Haemophilia A, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor
Conditions
Keywords
Hemophilia A, Factor VIII
Brief summary
This multicenter, non-interventional, prospective study will collect information about activity status, bleeds, health-related quality of life (HRQoL), health status, and safety in participants with moderate or severe haemophilia A without factor VIII (FVIII) inhibitors, who are being treated in accordance with normal clinical practice.
Interventions
DRUGStandard of Care for Haemophilia A
There is no pre-determined studied medicinal product. Any treatment (including treatments started during the observational period) will be administered according to standard clinical practice, independently of participation in the current study, according to clinician's independent therapeutic decision.
Sponsors
Hoffmann-La Roche
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
* Must own a device compatible with the electronic Patient-Reported Outcome (ePRO) application and with the fitness tracker that will be provided to the patient * Must have on his/her own device a data traffic availability of at least 2 gigabytes (GB) in total per month intended only for use of study applications and data transfer. If the data traffic plan is exhausted, the participant must be able to connect to a wi-fi network at least once every day in order to transfer the data collected for the study purpose * Must accept to run on his/her own device the ePRO application and the fitness tracker application * Must be available to turn on daily the bluetooth connection of his/her own device in order to allow the synchronization with the fitness tracker * Ability and willingness to comply with all aspects of the protocol, including completion of questions on the ePRO application (for underage population, ePRO questions can be answered by legally authorized representative if deemed necessary) * Ability and willingness to wear the activity tracking device as indicated * Diagnosis of severe (FVIII \<1%) or moderate (FVIII ≥1% and ≤2%) congenital haemophilia A * No prior history of a positive inhibitor against FVIII. If participant has a previous history of inhibitor development, the participant must have successfully eradicated inhibitors since 3 years. * At least 150 exposure days of FVIII prior to enrolment
Exclusion criteria
* Bleeding disorder other than congenital haemophilia A * Ongoing (or planned during the study) immune tolerance induction or FVIII prophylaxis if the participant has currently low titre of inhibitors or had inhibitors in the past 3 years * Previous or concomitant autoimmune or connective tissue disease * History of or suspected allergy or intolerance to any of the component of the fitness device (e.g., aluminium anodised) * History of clinically significant hypersensitivity associated with monoclonal antibody * Obesity (Body Mass Index \[BMI\] ≥30 kilograms/metre squared of body surface area \[kg/m\^2}) * Clinically important cardiovascular, metabolic, endocrine disorders or any other concomitant diseases or conditions that could limit the mobility of participant or could represent any risk according to the Investigator's judgment, or that could interfere with the study evaluation parameters * Participation in any other interventional clinical trial, including Roche sponsored studies or in any other support program that may include drug administration other than standard clinical practice (e.g., compassionate use, use not in agreement with the authorized indications, patient support programs, etc.)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | From baseline to study completion (up to 18 months) | Physical activity data was collected as daily active minutes measured on each valid day by wearing a fitness tracker continuously during study participation. Valid day = a day in which fitness tracker was worn for at least 10 hours. Daily active minutes were saved in two different sets of data collected by fitness tracker. For each set minutes were categorized by intensity of activity done. First set = minutes categorized according to time collected in each heart zone. Overall heart zone minutes = fat-burn minutes (heart rate: 50-69% of maximum) + cardio minutes (medium-to-high intensity exercise zone corresponds to heart rate: 70-84% of maximum) + peak minutes (high-intensity exercise zone, corresponds to heart rate \> 85% of maximum). Second set =minutes categorized according to active zones. Overall active zone minutes=lightly active minutes +fairly active minutes+very active minutes. Moderate-to-Vigorous Physical Activity (MVPA) minutes=Fairly active minutes+Very active minutes. |
| Mean Number of Steps Taken Per Day by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | From baseline to study completion (up to 18 months) | Number of steps taken by the participants on each valid day were measured by wearing a fitness tracker continuously during study participation. Valid day was defined as a day in which fitness tracker was worn for at least 10 hours. |
| Mean Metabolic Equivalent of Tasks (METs) Per Day Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | From baseline to study completion (up to 18 months) | The MET is an objective measure of the ratio of the rate at which a person expends energy, relative to the mass of that person, while performing some specific physical activity compared to a reference. The METs were derived as: (amount of kilo calories (kcal) collected in the fat-burn, cardio, and peak minutes) / \[(sum of fat-burn, cardio and peak hours) \* weight of the participant in kilograms (kg)\]. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | From baseline to study completion (up to 18 months) | The activity weekly rate was derived on a per participant basis as ratio between the number of times that the type of activity is reported on the fitness tracker (numerator) and the length of the observational period (i.e. end of study date - baseline date + 1) (denominator) and then multiplied by 7. The physical activities were categorized in the following groups: exercises at intervals, gym weights, running, swimming, tapis roulant, walk, general exercises: other types of physical activity identified by the fitness tracker that are not already included in the above-mentioned categories. |
| Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | From baseline to study completion (up to 18 months) | The number of active minutes by intensity (lightly, fairly, and very active minutes) were obtained at the participant level on a per activity basis as the median of the values collected by the fitness tracker for each activity recorded during the observational period. In this analysis, the means and 95% confidence intervals were calculated using the median lightly active minutes by type of physical activity performed by participants with available data. The physical activities were categorized in the following groups: exercises at intervals, gym weights, running, swimming, tapis roulant, walk, general exercises: other types of physical activity identified by the fitness tracker that are not already included in the above-mentioned categories. |
| Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | From baseline to study completion (up to 18 months) | The number of active minutes by intensity (lightly, fairly, and very active minutes) were obtained at the participant level on a per activity basis as the median of the values collected by the fitness tracker for each activity recorded during the observational period. In this analysis, the means and 95% confidence intervals were calculated using the median fairly active minutes by type of physical activity performed by participants with available data. The physical activities were categorized in the following groups: exercises at intervals, gym weights, running, swimming, tapis roulant, walk, general exercises: other types of physical activity identified by the fitness tracker that are not already included in the above-mentioned categories. |
| Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | From baseline to study completion (up to 18 months) | The number of active minutes by intensity (lightly, fairly, and very active minutes) were obtained at the participant level on a per activity basis as the median of the values collected by the fitness tracker for each activity recorded during the observational period. The MVPA minutes is the sum of fairly active and very active minutes. In this analysis, the means and 95% confidence intervals were calculated using the median MVPA minutes by type of physical activity performed by participants with available data. The physical activities were categorized in the following groups: exercises at intervals, gym weights, running, swimming, tapis roulant, walk, general exercises: other types of physical activity identified by the fitness tracker that are not already included in the above-mentioned categories. |
| Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study | From baseline to study completion (up to 18 months) | A participant was defined adherent to WHO guidelines according to the following algorithm: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). A participant who was adherent to the WHO guidelines according to the above definition was defined as an active participant, otherwise as a sedentary participant. |
| Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study, Reported Via the ePRO Application | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | Bleeds that occurred the same day but in different body site were counted separately. |
| Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months). | The assessment of bleeds was divided into spontaneous bleeds, traumatic bleeds, and bleeds related to procedure/surgery. Spontaneous bleeds = the occurrence of hemorrhage where neither the participant nor a caregiver could identify a reason. Traumatic bleeds = hemorrhage occurring secondary to an event such as trauma, strenuous activity, or overuse. Bleeds related to procedure/surgery = such as hematomas resulting from any surgeries or invasive procedures. |
| Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Baseline and whenever bleeding occured from study initiation to completion (up to 18 months) | Sites of bleeds were defined as urine, faeces, and vomit (three sites grouped together), joint, muscle, and other bleeds. |
| Number of Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | From baseline to study completion (up to 18 months) | The following types of regimens used to treat haemophilia A are reported here: on-demand (to stop bleeding) and prophylaxis (regular infusions of clotting factor to prevent bleeds). Participants could have received more than one medication. |
| Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | From baseline to study completion (up to 18 months) | Participants could have received more than one medication. |
| Number of Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | From baseline to the time of switch to a new therapy (up to 18 months) | Participants could have received more than one medication. |
| Number of Treated Bleeds Over the Course of the Study | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | An event was considered a treated bleed if coagulation factors were administered to treat signs or symptoms of bleeding (e.g., pain, swelling, etc.). |
| Number of Bleeds Treated With Each Type of Treatment for Bleeding (Haemostatic Treatment, Treatment for Pain) | From baseline to whenever bleeding occurred (up to 18 months) | An event was considered a treated bleed if coagulation factors were administered to treat signs or symptoms of bleeding (e.g., pain, swelling, etc.). |
| Duration of Treatment for Bleeding Over the Course of the Study | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | — |
| Number of Bleeds Treated With Each Route of Administration Over the Course of the Study | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | Number of bleeds treated by intravenous, subcutaneous, intramuscular, nasal, ophthalmic, oral, rectal, and other routes of administration are reported here. |
| Number of Bleeds Which Needed to Receive a Transfusion | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | — |
| Number of Bleeds Which Needed to Administer an Additional Hemostatic Therapy | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | — |
| Number of Bleeds With Adherence to Treatment Regimen for Haemophilia A in the Week Preceding the Bleeding | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | The adherence to the treatment regimen for haemophilia A was analyzed as the number of bleeds with an answer equal to Yes and My therapeutic plan does not include any treatment in the last week in the week preceding the bleeding. |
| Number of Participants Who Were Adherent to the Treatment Regimen for Haemophilia A Over the Course of the Study by Adherence Category (<80% vs. ≥80% of Study Weeks) | From baseline to study completion (up to 18 months) | The adherence to the treatment regimen for haemophilia A was analyzed as the number of participants with \<80% or ≥80% of weeks on study with Yes and My therapeutic plan does not include any treatment in the last week over the total number of weeks reported via the ePRO. Missing data from some weeks was treated as No. The total number of weeks on study for each participant was derived as (End of study date - Baseline date + 1) / 7. |
| Number of Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Baseline and every 3 months until study completion (up to 18 months) | Participants could have received more than one medication. |
| Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | Participants reported the intensity of pain in their main joints (elbow, knee, ankle) by marking on a visual analogue scale (VAS), a horizontal line 100 millimeters (mm) in length, anchored by word descriptors at each end, where the left extreme means No pain = 0 while the right extreme means Worst Pain Imaginable = 100. The VAS score is the distance (in mm) from the left end of the line to the point where the participant marks crossed the line. VAS scale was submitted to the participants every month during the study. |
| Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The EQ-5D-5L includes five levels of severity in each of the five EuroQol-5D dimensions and the EQ VAS. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each dimension having 5 levels (1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems). Response options are measured with a 5-point Likert scale. The 5D-5L systems are converted into a single index utility score between 0 to 1, where higher score indicates a better health state and lower score indicate worse health state. EQ-5D-5L questionnaire was submitted to the participants every 3 month from baseline during the study. |
| Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and the other section is an evaluation of the overall health status using VAS. The VAS records the respondent's self-rated health on a 20 centimeter (cm) vertical VAS from 0 to 100 on how good or bad their health is at assessment; 0 means the worst health imaginable, and 100 means the best health imaginable. |
| Haemophilia Joint Health Score (HJHS) Total Score Over Time | Baseline, Months 6 and 12 | The HJHS is an instrument developed to measure joint health status and to identify early signs of joint disease in children. This tool assesses joints most commonly affected by bleeding (knees, ankles, and elbows). Each of the 6 joints is assessed across 8 domains (Swelling, Duration of Swelling, Atrophy, Crepitus, Flexion Loss, Extension Loss, Pain, and Strength), and a Total Score is calculated as the sum of all joint domain scores. The score range is from 0 to 124 points (a score of 0 indicates no joint damage). A higher score indicates higher joint damage. |
| Mean Body Mass Index (BMI) Over Time | Baseline, Months 3, 6, 9, 12, 15 and 18 | — |
| Yearly Rate of Days Away From School or Work for Participants | Baseline and every month until study completion (up to 18 months) | The number of days away from school or work (participant) was analyzed every month as a continuous variable. The yearly rate was derived on a per participant basis as the proportion between the total number of days away from school and/or work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. |
| Yearly Rate of Days Away From Work for Parent/Caregiver | Baseline and every month until study completion (up to 18 months) | The number of days away from work was analyzed every month as a continuous variable. The yearly rate was derived on a per participant (for parents and caregivers) basis as the proportion between the total number of days away from work and duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. |
| Yearly Rate of Hospitalization Days | Baseline and every month until study completion (up to 18 months) | The yearly rate was derived on a per participant basis as the proportion between the total number of hospitalization days and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. |
| Age of Possible Early Retirement of Caregiver, as Reported by Caregivers in a Questionnaire | Baseline and every month until study completion (up to 18 months) | — |
| Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A | From baseline up to study completion (up to 18 months) | Participants who were adherent to the WHO guidelines for physical activity according to the below definition were defined as an active participant, otherwise as a sedentary participant. A participant was defined adherent to WHO guidelines according to the following algorithm: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or Very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). The participants were classified as 'Active' or 'Sedentary' based on data collected from baseline till the end of the study. |
| Annualized Bleeding Rates (ABR) in Active Versus Sedentary Participants | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | The annualized bleeding rate was derived as follows: total number of bleeds/total follow-up (person-year), where the individual's study duration was given by (\[end of study date-baseline date +1\] divided by 365.25). ABR was reported in active and sedentary participants. A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an active participant, otherwise as a sedentary participant. WHO guidelines for adherence: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or Very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). |
| Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | Participants reported intensity of pain in their main joints (elbow, knee, ankle) by marking on a VAS, a horizontal line 100 mm in length, anchored by word descriptors at each end, where the left extreme means No pain = 0 while right extreme means Worst Pain Imaginable = 100. VAS score is the distance (in mm) from left end of the line to the point where participant marks crossed the line. A participant who was adherent to WHO guidelines for physical activity according to the below definition was defined as an active participant, otherwise as a sedentary participant. WHO guidelines for adherence: 12-17 years old: MVPA minutes per day ≥60 minutes. 18-50 years old: Fairly active (moderate) minutes per week ≥150 minutes or Very active (vigorous) minutes per week ≥75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). VAS scale was submitted to the participants every month during the study. |
| Number of Active Versus Sedentary Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Baseline and every 3 months until study completion (up to 18 months) | A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an active participant, otherwise as a sedentary participant. WHO guidelines for adherence: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or Very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). Participants can have received more than one medication. |
| Number of Active Versus Sedentary Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | From baseline to study completion (up to 18 months) | The following types of regimens used to treat haemophilia A are reported here: on-demand (to stop bleeding) and prophylaxis (regular infusions of clotting factor to prevent bleeds). A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an active participant, otherwise as a sedentary participant. WHO guidelines for adherence: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or Very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). Participants can have received more than one medication. |
| Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | From baseline to study completion (up to 18 months) | A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an active participant, otherwise as a sedentary participant. WHO guidelines for adherence: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or Very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). |
| Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | From baseline to the time of switch to a new therapy (up to 18 months) | A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an active participant, otherwise as a sedentary participant. WHO guidelines for adherence: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or Very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). |
| HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | EQ-5D-5L is a standardized measure of health status developed by EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. EQ-5D-5L includes five levels of severity in each of five EuroQol-5D dimensions and EQ VAS. Descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each dimension having 5 levels (1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems). Response options are measured with a 5-point Likert scale. 5D-5L systems are converted into a single index utility score between 0-1, where higher score indicates a better health state and lower score indicate worse health state. A participant who was adherent to WHO guidelines for physical activity was defined as an active participant, otherwise as a sedentary participant. EQ-5D-5L questionnaire was submitted to participants every 3 month from baseline during the study. |
| Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities pain/discomfort, anxiety/depression) and the other section is an evaluation of the overall health status using VAS. The VAS records the respondent's self-rated health on a 20 centimeter (cm) vertical VAS from 0 to 100 on how good or bad their health is at assessment; 0 means the worst health imaginable, and 100 means the best health imaginable. A participant who was adherent to the WHO guidelines for physical activity was defined as an active participant, otherwise as a sedentary participant. |
| HJHS Total Score for Active Versus Sedentary Participants Over Time | Baseline, Months 6 and 12 | The HJHS is an instrument developed to measure joint health status and to identify early signs of joint disease in children. This tool assesses joints most commonly affected by bleeding (knees, ankles, and elbows). Each of the 6 joints is assessed across 8 domains (Swelling, Duration of Swelling, Atrophy, Crepitus, Flexion Loss, Extension Loss, Pain, and Strength), and a Total Score is calculated as the sum of all joint domain scores. The score range is from 0 to 124 points (a score of 0 indicates no joint damage). A higher score indicates higher joint damage. |
| Yearly Rate of Number of Days Away From School/Work for Active Versus Sedentary Participants | Baseline and every month until study completion (up to 18 months) | The number of days away from school or work (participant) was analyzed every month as a continuous variable. The yearly rate was derived on a per participant basis as the proportion between the total number of days away from school and/or work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. |
| Yearly Rate of Number of Days Away From Work for Active Versus Sedentary Participant's Parents or Caregivers | Baseline and every month until study completion (up to 18 months) | The number of days away from work was analyzed every month as a continuous variable. The yearly rate was derived on a per participant (for parents/caregivers) basis as the proportion between the total number of days away from work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. |
| Yearly Rate of Number of Hospitalization Days for Active Versus Sedentary Participants | Baseline and every month until study completion (up to 18 months) | The yearly rate was derived on a per participant basis as the proportion between the total number of hospitalization days and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. |
| Annualized Bleeding Rates in Active Versus Sedentary Participants by the Severity (Moderate Versus Severe) of Haemophilia A | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | The annualized bleeding rate was derived as follows: total number of bleeds/total follow-up (person-year), where the individual's study duration was given by (\[end of study date-baseline date +1\] divided by 365.25). A participant who was adherent to the WHO guidelines for physical activity was defined as an active participant, otherwise as a sedentary participant. Participants were classified for disease severity at baseline: severe (Factor VIII activity \<1%) or moderate (Factor VIII activity between ≥1% and ≤2%) haemophilia A. |
| Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17 and 18 | Participants reported the intensity of pain in their main joints (elbow, knee, ankle) by marking on a VAS, a horizontal line 100 mm in length, anchored by word descriptors at each end, where the left extreme means No pain = 0 while the right extreme means Worst Pain Imaginable = 100. The VAS score is the distance (in mm) from the left end of the line to the point where the participant marks crossed the line. VAS scale was submitted to the participants every month during the study. |
| Number of Participants With Moderate Versus Severe Haemophilia A by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Baseline and every 3 months until study completion (up to 18 months) | Participants could have received more than one medication. |
| Number of Participants With Moderate Versus Severe Haemophilia A by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | From baseline to study completion (up to 18 months) | The following types of regimens used to treat haemophilia A are reported here: on-demand (to stop bleeding) and prophylaxis (regular infusions of clotting factor to prevent bleeds). Participants could have received more than one medication. |
| Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | From baseline to study completion (up to 18 months) | Participants could have received more than one medication. |
| Number of Participants With Moderate Versus Severe Haemophilia A Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | From baseline to the time of switch to a new therapy (up to 18 months) | Participants could have received more than one medication. |
| HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The EQ-5D-5L includes five levels of severity in each of the five EuroQol-5D dimensions and the EQ VAS. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each dimension having 5 levels (1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems). Response options are measured with a 5-point Likert scale. The 5D-5L systems are converted into a single index utility score between 0 to 1, where higher score indicates a better health state and lower score indicate worse health state. EQ-5D-5L questionnaire was submitted to the participants every 3 month from baseline during the study. |
| Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and the other section is an evaluation of the overall health status using VAS. The VAS records the respondent's self-rated health on a 20 cm vertical VAS from 0 to 100 on how good or bad their health is at assessment; 0 means the worst health imaginable, and 100 means the best health imaginable. |
| HJHS Total Score for Participants With Moderate Versus Severe Haemophilia A | Baseline, Months 6 and 12 | The HJHS is an instrument developed to measure joint health status and to identify early signs of joint disease in children. This tool assesses joints most commonly affected by bleeding (knees, ankles, and elbows). Each of the 6 joints is assessed across 8 domains (Swelling, Duration of Swelling, Atrophy, Crepitus, Flexion Loss, Extension Loss, Pain, and Strength), and a Total Score is calculated as the sum of all joint domain scores. The total score range is from 0 to 124 points (a score of 0 indicates no joint damage). A higher score indicates higher joint damage. |
| Yearly Rate of Days Away From School for Participants With Moderate Versus Severe Haemophilia A | Baseline and every month until study completion (up to 18 months) | The number of days away from school or work (participant) was analyzed every month as a continuous variable. The yearly rate was derived on a per participant basis as the proportion between the total number of days away from school and/or work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. |
| Yearly Rate of Days Away From Work for Parents/Caregivers of Participants With Moderate Versus Severe Haemophilia A | Baseline and every month until study completion (up to 18 months) | The number of days away from work was analyzed every month as a continuous variable. The yearly rate was derived on a per participant (for parents/caregivers) basis as the proportion between the total number of days away from work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. |
| Yearly Rate of Hospitalization Days for Participants With Moderate Versus Severe Haemophilia A | Baseline and every month until study completion (up to 18 months) | The yearly rate was derived on a per participant basis as the proportion between the total number of hospitalization days and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. |
Countries
Italy
Participant flow
Recruitment details
107 patients were screened, but 4 patients did not meet the eligibility criteria; in total, 103 participants were enrolled and started the study. Participants took part in this study at 17 investigative sites in Italy from 24 February 2020 up to 28 April 2022.
Participants by arm
| Arm | Count |
|---|---|
| Standard of Care for Haemophilia A Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | 103 |
| Total | 103 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Participants withdrawal of consent | 5 |
| Overall Study | Protocol Violation | 10 |
Baseline characteristics
| Characteristic | Standard of Care for Haemophilia A |
|---|---|
| Age, Continuous | 28.3 years STANDARD_DEVIATION 10.4 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 10 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 93 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Is Haemophilia A diagnosis confirmed No | 0 Participants |
| Is Haemophilia A diagnosis confirmed Yes | 103 Participants |
| Race/Ethnicity, Customized American Indian or Alaska Native | 1 Participants |
| Race/Ethnicity, Customized Asian | 2 Participants |
| Race/Ethnicity, Customized Other | 1 Participants |
| Race/Ethnicity, Customized White | 99 Participants |
| Severity type Moderate Haemophilia | 6 Participants |
| Severity type Severe Haemophilia | 97 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 103 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 0 |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
Mean Metabolic Equivalent of Tasks (METs) Per Day Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study
The MET is an objective measure of the ratio of the rate at which a person expends energy, relative to the mass of that person, while performing some specific physical activity compared to a reference. The METs were derived as: (amount of kilo calories (kcal) collected in the fat-burn, cardio, and peak minutes) / \[(sum of fat-burn, cardio and peak hours) \* weight of the participant in kilograms (kg)\].
Time frame: From baseline to study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Mean Metabolic Equivalent of Tasks (METs) Per Day Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | 5.1 MET per day |
| Standard of Care: 18-30 Years | Mean Metabolic Equivalent of Tasks (METs) Per Day Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | 5.3 MET per day |
| Standard of Care:31-50 Years | Mean Metabolic Equivalent of Tasks (METs) Per Day Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | 4.5 MET per day |
p-value: 0.0026Kruskal-Wallis
Primary
Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study
Physical activity data was collected as daily active minutes measured on each valid day by wearing a fitness tracker continuously during study participation. Valid day = a day in which fitness tracker was worn for at least 10 hours. Daily active minutes were saved in two different sets of data collected by fitness tracker. For each set minutes were categorized by intensity of activity done. First set = minutes categorized according to time collected in each heart zone. Overall heart zone minutes = fat-burn minutes (heart rate: 50-69% of maximum) + cardio minutes (medium-to-high intensity exercise zone corresponds to heart rate: 70-84% of maximum) + peak minutes (high-intensity exercise zone, corresponds to heart rate \> 85% of maximum). Second set =minutes categorized according to active zones. Overall active zone minutes=lightly active minutes +fairly active minutes+very active minutes. Moderate-to-Vigorous Physical Activity (MVPA) minutes=Fairly active minutes+Very active minutes.
Time frame: From baseline to study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Active Zone Minutes | 226.5 number of daily active minutes |
| Standard of Care: 12-17 Years | Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Heart Zone Minutes | 53.2 number of daily active minutes |
| Standard of Care: 12-17 Years | Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | MVPA Minutes | 13.8 number of daily active minutes |
| Standard of Care: 18-30 Years | Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Active Zone Minutes | 309.4 number of daily active minutes |
| Standard of Care: 18-30 Years | Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Heart Zone Minutes | 24.6 number of daily active minutes |
| Standard of Care: 18-30 Years | Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | MVPA Minutes | 42.3 number of daily active minutes |
| Standard of Care:31-50 Years | Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Heart Zone Minutes | 39.2 number of daily active minutes |
| Standard of Care:31-50 Years | Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | MVPA Minutes | 28.6 number of daily active minutes |
| Standard of Care:31-50 Years | Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Active Zone Minutes | 284.1 number of daily active minutes |
Comparison: Heart zone minutes: Comparison among the age groups was performed by means of a Kruskal-Wallis test.p-value: 0.763Kruskal-Wallis
Comparison: Active zone minutes: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.0913Kruskal-Wallis
Comparison: MVPA minutes: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.0956Kruskal-Wallis
Primary
Mean Number of Steps Taken Per Day by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study
Number of steps taken by the participants on each valid day were measured by wearing a fitness tracker continuously during study participation. Valid day was defined as a day in which fitness tracker was worn for at least 10 hours.
Time frame: From baseline to study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Mean Number of Steps Taken Per Day by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | 5402.4 number of steps per day |
| Standard of Care: 18-30 Years | Mean Number of Steps Taken Per Day by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | 8788.3 number of steps per day |
| Standard of Care:31-50 Years | Mean Number of Steps Taken Per Day by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | 8279.8 number of steps per day |
p-value: 0.0649Kruskal-Wallis
Secondary
Age of Possible Early Retirement of Caregiver, as Reported by Caregivers in a Questionnaire
Time frame: Baseline and every month until study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Age of Possible Early Retirement of Caregiver, as Reported by Caregivers in a Questionnaire | 57.8 years |
Secondary
Annualized Bleeding Rates (ABR) in Active Versus Sedentary Participants
The annualized bleeding rate was derived as follows: total number of bleeds/total follow-up (person-year), where the individual's study duration was given by (\[end of study date-baseline date +1\] divided by 365.25). ABR was reported in active and sedentary participants. A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an active participant, otherwise as a sedentary participant. WHO guidelines for adherence: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or Very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes).
Time frame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Annualized Bleeding Rates (ABR) in Active Versus Sedentary Participants | 1.945 rate of bleeds per person-year |
| Standard of Care: 18-30 Years | Annualized Bleeding Rates (ABR) in Active Versus Sedentary Participants | 1.384 rate of bleeds per person-year |
Secondary
Annualized Bleeding Rates in Active Versus Sedentary Participants by the Severity (Moderate Versus Severe) of Haemophilia A
The annualized bleeding rate was derived as follows: total number of bleeds/total follow-up (person-year), where the individual's study duration was given by (\[end of study date-baseline date +1\] divided by 365.25). A participant who was adherent to the WHO guidelines for physical activity was defined as an active participant, otherwise as a sedentary participant. Participants were classified for disease severity at baseline: severe (Factor VIII activity \<1%) or moderate (Factor VIII activity between ≥1% and ≤2%) haemophilia A.
Time frame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of participants analyzed is the number of participants with available physical activities data in ITT population for sedentary and active participants.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Annualized Bleeding Rates in Active Versus Sedentary Participants by the Severity (Moderate Versus Severe) of Haemophilia A | Moderate Haemophilia | 0.368 rate of bleeds per person-year |
| Standard of Care: 12-17 Years | Annualized Bleeding Rates in Active Versus Sedentary Participants by the Severity (Moderate Versus Severe) of Haemophilia A | Severe Haemophilia | 1.517 rate of bleeds per person-year |
| Standard of Care: 18-30 Years | Annualized Bleeding Rates in Active Versus Sedentary Participants by the Severity (Moderate Versus Severe) of Haemophilia A | Moderate Haemophilia | 2.536 rate of bleeds per person-year |
| Standard of Care: 18-30 Years | Annualized Bleeding Rates in Active Versus Sedentary Participants by the Severity (Moderate Versus Severe) of Haemophilia A | Severe Haemophilia | 1.035 rate of bleeds per person-year |
Secondary
Duration of Treatment for Bleeding Over the Course of the Study
Time frame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Duration of Treatment for Bleeding Over the Course of the Study | 2.0 days |
Secondary
Haemophilia Joint Health Score (HJHS) Total Score Over Time
The HJHS is an instrument developed to measure joint health status and to identify early signs of joint disease in children. This tool assesses joints most commonly affected by bleeding (knees, ankles, and elbows). Each of the 6 joints is assessed across 8 domains (Swelling, Duration of Swelling, Atrophy, Crepitus, Flexion Loss, Extension Loss, Pain, and Strength), and a Total Score is calculated as the sum of all joint domain scores. The score range is from 0 to 124 points (a score of 0 indicates no joint damage). A higher score indicates higher joint damage.
Time frame: Baseline, Months 6 and 12
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Haemophilia Joint Health Score (HJHS) Total Score Over Time | Baseline | 10 scores on a scale |
| Standard of Care: 12-17 Years | Haemophilia Joint Health Score (HJHS) Total Score Over Time | Month 6 | 13.7 scores on a scale |
| Standard of Care: 12-17 Years | Haemophilia Joint Health Score (HJHS) Total Score Over Time | Month 12 | 12.5 scores on a scale |
Secondary
Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time
The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The EQ-5D-5L includes five levels of severity in each of the five EuroQol-5D dimensions and the EQ VAS. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each dimension having 5 levels (1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems). Response options are measured with a 5-point Likert scale. The 5D-5L systems are converted into a single index utility score between 0 to 1, where higher score indicates a better health state and lower score indicate worse health state. EQ-5D-5L questionnaire was submitted to the participants every 3 month from baseline during the study.
Time frame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Baseline | 0.93 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 1 | 0.89 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 3 | 0.90 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 4 | 0.94 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 5 | 0.94 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 6 | 0.90 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 7 | 0.92 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 8 | 0.93 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 9 | 0.88 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 10 | 0.80 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 11 | 0.90 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 12 | 0.92 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 13 | 0.62 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 14 | 0.86 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 15 | 0.85 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 17 | 0.90 scores on a scale |
| Standard of Care: 12-17 Years | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | Month 18 | 0.92 scores on a scale |
Secondary
Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score
The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and the other section is an evaluation of the overall health status using VAS. The VAS records the respondent's self-rated health on a 20 centimeter (cm) vertical VAS from 0 to 100 on how good or bad their health is at assessment; 0 means the worst health imaginable, and 100 means the best health imaginable.
Time frame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Baseline | 81.9 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 1 | 81.0 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 3 | 80.6 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 4 | 86.0 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 5 | 83.5 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 6 | 75.8 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 7 | 64 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 8 | 78.4 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 9 | 73.3 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 10 | 72.5 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 11 | 75.3 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 12 | 69.6 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 13 | 65 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 14 | 83.3 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 15 | 75 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 17 | 85 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | Month 18 | 97 scores on a scale |
Secondary
Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants
The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities pain/discomfort, anxiety/depression) and the other section is an evaluation of the overall health status using VAS. The VAS records the respondent's self-rated health on a 20 centimeter (cm) vertical VAS from 0 to 100 on how good or bad their health is at assessment; 0 means the worst health imaginable, and 100 means the best health imaginable. A participant who was adherent to the WHO guidelines for physical activity was defined as an active participant, otherwise as a sedentary participant.
Time frame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Baseline | 79.9 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 3 | 76.9 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 4 | 80.0 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 6 | 73.1 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 7 | 62.5 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 8 | 80.2 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 9 | 70.7 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 11 | 81.7 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 12 | 47.8 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 14 | 97.5 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 15 | 75 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 17 | 85.0 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 18 | 97 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Baseline | 78.1 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 1 | 81.0 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 12 | 89.3 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 3 | 81.8 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 9 | 67.0 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 4 | 97.0 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 13 | 65.0 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 6 | 76.1 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 11 | 66.7 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 7 | 55.0 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 14 | 67.0 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | Month 8 | 79.1 scores on a scale |
Secondary
Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A
The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and the other section is an evaluation of the overall health status using VAS. The VAS records the respondent's self-rated health on a 20 cm vertical VAS from 0 to 100 on how good or bad their health is at assessment; 0 means the worst health imaginable, and 100 means the best health imaginable.
Time frame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoint.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 11 | 73.5 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 8 | 48 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 6 | 84.4 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 13 | 65.0 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 9 | 82.3 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 3 | 81.2 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 4 | 75.0 scores on a scale |
| Standard of Care: 12-17 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Baseline | 88.3 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 8 | 79.7 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 9 | 72.0 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 10 | 72.5 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 11 | 75.5 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 12 | 69.6 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 14 | 83.3 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 15 | 75.0 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 17 | 85.0 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 18 | 97.0 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Baseline | 81.5 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 1 | 81.0 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 3 | 80.5 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 4 | 87.6 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 5 | 83.5 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 6 | 75.1 scores on a scale |
| Standard of Care: 18-30 Years | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | Month 7 | 64.0 scores on a scale |
Secondary
HJHS Total Score for Active Versus Sedentary Participants Over Time
The HJHS is an instrument developed to measure joint health status and to identify early signs of joint disease in children. This tool assesses joints most commonly affected by bleeding (knees, ankles, and elbows). Each of the 6 joints is assessed across 8 domains (Swelling, Duration of Swelling, Atrophy, Crepitus, Flexion Loss, Extension Loss, Pain, and Strength), and a Total Score is calculated as the sum of all joint domain scores. The score range is from 0 to 124 points (a score of 0 indicates no joint damage). A higher score indicates higher joint damage.
Time frame: Baseline, Months 6 and 12
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Number analyzed is the number of participants with data available for analysis at given timepoint.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | HJHS Total Score for Active Versus Sedentary Participants Over Time | Month 12 | 17.3 scores on a scale |
| Standard of Care: 12-17 Years | HJHS Total Score for Active Versus Sedentary Participants Over Time | Baseline | 11.8 scores on a scale |
| Standard of Care: 12-17 Years | HJHS Total Score for Active Versus Sedentary Participants Over Time | Month 6 | 29.3 scores on a scale |
| Standard of Care: 18-30 Years | HJHS Total Score for Active Versus Sedentary Participants Over Time | Month 6 | 8.7 scores on a scale |
| Standard of Care: 18-30 Years | HJHS Total Score for Active Versus Sedentary Participants Over Time | Baseline | 12.1 scores on a scale |
| Standard of Care: 18-30 Years | HJHS Total Score for Active Versus Sedentary Participants Over Time | Month 12 | 15.0 scores on a scale |
Secondary
HJHS Total Score for Participants With Moderate Versus Severe Haemophilia A
The HJHS is an instrument developed to measure joint health status and to identify early signs of joint disease in children. This tool assesses joints most commonly affected by bleeding (knees, ankles, and elbows). Each of the 6 joints is assessed across 8 domains (Swelling, Duration of Swelling, Atrophy, Crepitus, Flexion Loss, Extension Loss, Pain, and Strength), and a Total Score is calculated as the sum of all joint domain scores. The total score range is from 0 to 124 points (a score of 0 indicates no joint damage). A higher score indicates higher joint damage.
Time frame: Baseline, Months 6 and 12
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | HJHS Total Score for Participants With Moderate Versus Severe Haemophilia A | Baseline | 8.3 scores on a scale |
| Standard of Care: 12-17 Years | HJHS Total Score for Participants With Moderate Versus Severe Haemophilia A | Month 6 | 20.0 scores on a scale |
| Standard of Care: 12-17 Years | HJHS Total Score for Participants With Moderate Versus Severe Haemophilia A | Month 12 | 0.0 scores on a scale |
| Standard of Care: 18-30 Years | HJHS Total Score for Participants With Moderate Versus Severe Haemophilia A | Baseline | 10.1 scores on a scale |
| Standard of Care: 18-30 Years | HJHS Total Score for Participants With Moderate Versus Severe Haemophilia A | Month 6 | 13.3 scores on a scale |
| Standard of Care: 18-30 Years | HJHS Total Score for Participants With Moderate Versus Severe Haemophilia A | Month 12 | 12.8 scores on a scale |
Secondary
HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants
EQ-5D-5L is a standardized measure of health status developed by EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. EQ-5D-5L includes five levels of severity in each of five EuroQol-5D dimensions and EQ VAS. Descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each dimension having 5 levels (1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems). Response options are measured with a 5-point Likert scale. 5D-5L systems are converted into a single index utility score between 0-1, where higher score indicates a better health state and lower score indicate worse health state. A participant who was adherent to WHO guidelines for physical activity was defined as an active participant, otherwise as a sedentary participant. EQ-5D-5L questionnaire was submitted to participants every 3 month from baseline during the study.
Time frame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Baseline | 0.90 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 3 | 0.89 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 4 | 0.95 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 6 | 0.87 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 7 | 0.87 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 8 | 0.9 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 9 | 0.85 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 11 | 0.89 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 12 | 0.86 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 14 | 0.89 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 15 | 0.85 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 17 | 0.90 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 18 | 0.92 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Baseline | 0.93 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 1 | 0.89 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 12 | 0.97 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 3 | 0.90 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 9 | 0.88 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 4 | 1 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 13 | 0.62 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 6 | 0.90 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 11 | 0.89 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 7 | 0.94 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 14 | 0.86 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | Month 8 | 0.91 scores on a scale |
Secondary
HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A
The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The EQ-5D-5L includes five levels of severity in each of the five EuroQol-5D dimensions and the EQ VAS. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each dimension having 5 levels (1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems). Response options are measured with a 5-point Likert scale. The 5D-5L systems are converted into a single index utility score between 0 to 1, where higher score indicates a better health state and lower score indicate worse health state. EQ-5D-5L questionnaire was submitted to the participants every 3 month from baseline during the study.
Time frame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoint.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 11 | 0.93 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 8 | 0.91 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 6 | 0.92 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 13 | 0.62 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 9 | 0.83 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 3 | 0.88 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 4 | 0.90 scores on a scale |
| Standard of Care: 12-17 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Baseline | 0.92 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 8 | 0.93 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 9 | 0.88 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 10 | 0.80 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 11 | 0.90 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 12 | 0.92 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 14 | 0.86 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 15 | 0.85 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 17 | 0.90 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 18 | 0.92 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Baseline | 0.93 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 1 | 0.89 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 3 | 0.90 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 4 | 0.95 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 5 | 0.94 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 6 | 0.90 scores on a scale |
| Standard of Care: 18-30 Years | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | Month 7 | 0.92 scores on a scale |
Secondary
Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study
The number of active minutes by intensity (lightly, fairly, and very active minutes) were obtained at the participant level on a per activity basis as the median of the values collected by the fitness tracker for each activity recorded during the observational period. In this analysis, the means and 95% confidence intervals were calculated using the median fairly active minutes by type of physical activity performed by participants with available data. The physical activities were categorized in the following groups: exercises at intervals, gym weights, running, swimming, tapis roulant, walk, general exercises: other types of physical activity identified by the fitness tracker that are not already included in the above-mentioned categories.
Time frame: From baseline to study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Number analyzed is the number of participants with data available for analysis.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Swimming | 1.7 fairly active minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Exercises at Intervals | 1.3 fairly active minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Gym Weights | 0.2 fairly active minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Running | 0.0 fairly active minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Walk | 13.2 fairly active minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | General | 13.6 fairly active minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Tapis Roulant | 0.0 fairly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Gym Weights | 8.3 fairly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Running | 1.1 fairly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Swimming | 6.3 fairly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Tapis Roulant | 2.3 fairly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Exercises at Intervals | 0.1 fairly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | General | 11.3 fairly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Walk | 9.8 fairly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Exercises at Intervals | 0.0 fairly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Swimming | 4.5 fairly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Walk | 9.6 fairly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | General | 7.9 fairly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Gym Weights | 8.7 fairly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Tapis Roulant | 1.5 fairly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Running | 0.3 fairly active minutes per activity |
Comparison: Running: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.0745Kruskal-Wallis
Comparison: Swimming: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.2177Kruskal-Wallis
Comparison: Tapis Roulant: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.2121Kruskal-Wallis
Comparison: Gym weights: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.2336Kruskal-Wallis
Comparison: Exercises at intervals: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.419Kruskal-Wallis
Comparison: General: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.0252Kruskal-Wallis
Comparison: Walk: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.2143Kruskal-Wallis
Secondary
Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study
The number of active minutes by intensity (lightly, fairly, and very active minutes) were obtained at the participant level on a per activity basis as the median of the values collected by the fitness tracker for each activity recorded during the observational period. In this analysis, the means and 95% confidence intervals were calculated using the median lightly active minutes by type of physical activity performed by participants with available data. The physical activities were categorized in the following groups: exercises at intervals, gym weights, running, swimming, tapis roulant, walk, general exercises: other types of physical activity identified by the fitness tracker that are not already included in the above-mentioned categories.
Time frame: From baseline to study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Number analyzed is the number of participants with data available for analysis.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Swimming | 2.7 lightly active minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Exercises at Intervals | 0.8 lightly active minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Gym Weights | 0.7 lightly active minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Running | 0.1 lightly active minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Walk | 3.9 lightly active minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | General | 11.2 lightly active minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Tapis Roulant | 0.5 lightly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Gym Weights | 3.7 lightly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Running | 0.5 lightly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Swimming | 2.8 lightly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Tapis Roulant | 1.2 lightly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Exercises at Intervals | 0.0 lightly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | General | 5.2 lightly active minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Walk | 3.3 lightly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Exercises at Intervals | 2.6 lightly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Swimming | 3.8 lightly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Walk | 4.7 lightly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | General | 5.8 lightly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Gym Weights | 2.8 lightly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Tapis Roulant | 5.0 lightly active minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Running | 0.3 lightly active minutes per activity |
Comparison: Running: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.6151Kruskal-Wallis
Comparison: Swimming: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.9332Kruskal-Wallis
Comparison: Tapis Roulant: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.4726Kruskal-Wallis
Comparison: Gym Weights: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.4111Kruskal-Wallis
Comparison: Exercises at Intervals: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.0892Kruskal-Wallis
Comparison: General: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.1642Kruskal-Wallis
Comparison: Walk: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.7256Kruskal-Wallis
Secondary
Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study
The number of active minutes by intensity (lightly, fairly, and very active minutes) were obtained at the participant level on a per activity basis as the median of the values collected by the fitness tracker for each activity recorded during the observational period. The MVPA minutes is the sum of fairly active and very active minutes. In this analysis, the means and 95% confidence intervals were calculated using the median MVPA minutes by type of physical activity performed by participants with available data. The physical activities were categorized in the following groups: exercises at intervals, gym weights, running, swimming, tapis roulant, walk, general exercises: other types of physical activity identified by the fitness tracker that are not already included in the above-mentioned categories.
Time frame: From baseline to study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Number analyzed is the number of participants with data available for analysis.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Swimming | 2.3 MVPA minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Exercise at Intervals | 4.3 MVPA minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Gym weights | 5.0 MVPA minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Running | 4.3 MVPA minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Walk | 24.2 MVPA minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | General | 32.9 MVPA minutes per activity |
| Standard of Care: 12-17 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Tapis Roulant | 0 MVPA minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Gym weights | 49.3 MVPA minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Running | 36.6 MVPA minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Swimming | 14.4 MVPA minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Tapis Roulant | 18.8 MVPA minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Exercise at Intervals | 0.1 MVPA minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | General | 34.3 MVPA minutes per activity |
| Standard of Care: 18-30 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Walk | 33.7 MVPA minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Exercise at Intervals | 0 MVPA minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Swimming | 6.4 MVPA minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Walk | 30.3 MVPA minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | General | 26.5 MVPA minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Gym weights | 29.6 MVPA minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Tapis Roulant | 17.5 MVPA minutes per activity |
| Standard of Care:31-50 Years | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Running | 12.8 MVPA minutes per activity |
Comparison: Running: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.0085Kruskal-Wallis
Comparison: Swimming: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.1098Kruskal-Wallis
Comparison: Tapis Roulant: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.2703Kruskal-Wallis
Comparison: Gym Weights: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.3366Kruskal-Wallis
Comparison: Exercises at Intervals: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.0992Kruskal-Wallis
Comparison: General: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.1194Kruskal-Wallis
Comparison: Walk: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.0549Kruskal-Wallis
Secondary
Mean Body Mass Index (BMI) Over Time
Time frame: Baseline, Months 3, 6, 9, 12, 15 and 18
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Mean Body Mass Index (BMI) Over Time | Baseline | 23.34 kilogram per square meter (kg/m2) |
| Standard of Care: 12-17 Years | Mean Body Mass Index (BMI) Over Time | Month 3 | 23.56 kilogram per square meter (kg/m2) |
| Standard of Care: 12-17 Years | Mean Body Mass Index (BMI) Over Time | Month 6 | 23.42 kilogram per square meter (kg/m2) |
| Standard of Care: 12-17 Years | Mean Body Mass Index (BMI) Over Time | Month 9 | 23.76 kilogram per square meter (kg/m2) |
| Standard of Care: 12-17 Years | Mean Body Mass Index (BMI) Over Time | Month 12 | 22.98 kilogram per square meter (kg/m2) |
Secondary
Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study
The activity weekly rate was derived on a per participant basis as ratio between the number of times that the type of activity is reported on the fitness tracker (numerator) and the length of the observational period (i.e. end of study date - baseline date + 1) (denominator) and then multiplied by 7. The physical activities were categorized in the following groups: exercises at intervals, gym weights, running, swimming, tapis roulant, walk, general exercises: other types of physical activity identified by the fitness tracker that are not already included in the above-mentioned categories.
Time frame: From baseline to study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Number analyzed is the number of participants with data available for analysis.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Swimming | 0.03 number of times per week |
| Standard of Care: 12-17 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Exercises at Intervals | 0.02 number of times per week |
| Standard of Care: 12-17 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Gym Weights | 0.03 number of times per week |
| Standard of Care: 12-17 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Running | 0.06 number of times per week |
| Standard of Care: 12-17 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Walk | 0.53 number of times per week |
| Standard of Care: 12-17 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | General | 0.86 number of times per week |
| Standard of Care: 12-17 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Tapis Roulant | 0.06 number of times per week |
| Standard of Care: 18-30 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Gym Weights | 0.63 number of times per week |
| Standard of Care: 18-30 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Running | 0.12 number of times per week |
| Standard of Care: 18-30 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Swimming | 0.06 number of times per week |
| Standard of Care: 18-30 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Tapis Roulant | 0.11 number of times per week |
| Standard of Care: 18-30 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Exercises at Intervals | 0.02 number of times per week |
| Standard of Care: 18-30 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | General | 0.86 number of times per week |
| Standard of Care: 18-30 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Walk | 1.30 number of times per week |
| Standard of Care:31-50 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Exercises at Intervals | 0.02 number of times per week |
| Standard of Care:31-50 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Swimming | 0.03 number of times per week |
| Standard of Care:31-50 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Walk | 0.88 number of times per week |
| Standard of Care:31-50 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | General | 0.51 number of times per week |
| Standard of Care:31-50 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Gym Weights | 0.13 number of times per week |
| Standard of Care:31-50 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Tapis Roulant | 0.05 number of times per week |
| Standard of Care:31-50 Years | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Running | 0.18 number of times per week |
Comparison: Running: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.9493Kruskal-Wallis
Comparison: Swimming: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.8305Kruskal-Wallis
Comparison: Tapis Roulant: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.8725Kruskal-Wallis
Comparison: Gym Weights: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.338Kruskal-Wallis
Comparison: Exercises at Intervals: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.9706Kruskal-Wallis
Comparison: General: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.1727Kruskal-Wallis
Comparison: Walk: Comparison among age groups was performed using Kruskal-Wallis test.p-value: 0.0099Kruskal-Wallis
Secondary
Number of Active Versus Sedentary Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study
A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an active participant, otherwise as a sedentary participant. WHO guidelines for adherence: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or Very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). Participants can have received more than one medication.
Time frame: Baseline and every 3 months until study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Etoricoxib | 2 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Paracetamol | 1 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Etoricoxib | 2 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Paracetamol | 2 Participants |
Secondary
Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study
A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an active participant, otherwise as a sedentary participant. WHO guidelines for adherence: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or Very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes).
Time frame: From baseline to study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Moroctocog Alfa | 3 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Turoctocog Alfa Pegol | 1 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Lonoctocog Alfa | 3 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Damoctocog Alfa Pegol | 0 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Efmoroctocog Alfa | 8 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Factor VIII (Antihemophilic Factor) | 0 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Rurioctocog Alfa Pegol | 2 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Emicizumab | 6 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Turoctocog Alfa | 1 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Tranexamic Acid | 1 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Simoctocog Alfa | 3 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | On Demand for Haemophilia: Octocog Alfa | 0 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Octocog Alfa | 8 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | On Demand for Haemophilia: Octocog Alfa | 1 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Octocog Alfa | 10 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Efmoroctocog Alfa | 7 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Moroctocog Alfa | 2 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Turoctocog Alfa | 3 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Lonoctocog Alfa | 0 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Rurioctocog Alfa Pegol | 1 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Simoctocog Alfa | 0 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Turoctocog Alfa Pegol | 2 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Damoctocog Alfa Pegol | 1 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Factor VIII (Antihemophilic Factor) | 1 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Emicizumab | 3 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Tranexamic Acid | 1 Participants |
Secondary
Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A
Participants who were adherent to the WHO guidelines for physical activity according to the below definition were defined as an active participant, otherwise as a sedentary participant. A participant was defined adherent to WHO guidelines according to the following algorithm: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or Very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). The participants were classified as 'Active' or 'Sedentary' based on data collected from baseline till the end of the study.
Time frame: From baseline up to study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A | Sedentary Participants | 3 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A | Active Participants | 3 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A | Unknown | 0 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A | Sedentary Participants | 54 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A | Active Participants | 34 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A | Unknown | 9 Participants |
Secondary
Number of Active Versus Sedentary Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study
The following types of regimens used to treat haemophilia A are reported here: on-demand (to stop bleeding) and prophylaxis (regular infusions of clotting factor to prevent bleeds). A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an active participant, otherwise as a sedentary participant. WHO guidelines for adherence: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or Very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). Participants can have received more than one medication.
Time frame: From baseline to study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia | 28 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | On Demand for Haemophilia | 0 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia | 25 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | On Demand for Haemophilia | 1 Participants |
Secondary
Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used
A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an active participant, otherwise as a sedentary participant. WHO guidelines for adherence: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or Very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes).
Time frame: From baseline to the time of switch to a new therapy (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Rurioctocog Alfa Pegol | 1 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Damoctocog Alfa Pegol | 1 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Emicizumab | 5 Participants |
| Standard of Care: 12-17 Years | Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Turoctocog Alfa Pegol | 1 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Emicizumab | 0 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Damoctocog Alfa Pegol | 1 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Rurioctocog Alfa Pegol | 0 Participants |
| Standard of Care: 18-30 Years | Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Turoctocog Alfa Pegol | 4 Participants |
Secondary
Number of Bleeds Treated With Each Route of Administration Over the Course of the Study
Number of bleeds treated by intravenous, subcutaneous, intramuscular, nasal, ophthalmic, oral, rectal, and other routes of administration are reported here.
Time frame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of bleeds analyzed=number of bleeds during analysis at given timepoint.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Bleeds Treated With Each Route of Administration Over the Course of the Study | Intravenous | 90 number of treated bleeds |
| Standard of Care: 12-17 Years | Number of Bleeds Treated With Each Route of Administration Over the Course of the Study | Subcutaneous | 0 number of treated bleeds |
| Standard of Care: 12-17 Years | Number of Bleeds Treated With Each Route of Administration Over the Course of the Study | Intramuscular | 0 number of treated bleeds |
| Standard of Care: 12-17 Years | Number of Bleeds Treated With Each Route of Administration Over the Course of the Study | Nasal | 0 number of treated bleeds |
| Standard of Care: 12-17 Years | Number of Bleeds Treated With Each Route of Administration Over the Course of the Study | Ophthalmic | 0 number of treated bleeds |
| Standard of Care: 12-17 Years | Number of Bleeds Treated With Each Route of Administration Over the Course of the Study | Oral | 4 number of treated bleeds |
| Standard of Care: 12-17 Years | Number of Bleeds Treated With Each Route of Administration Over the Course of the Study | Rectal | 0 number of treated bleeds |
| Standard of Care: 12-17 Years | Number of Bleeds Treated With Each Route of Administration Over the Course of the Study | Others | 0 number of treated bleeds |
Secondary
Number of Bleeds Treated With Each Type of Treatment for Bleeding (Haemostatic Treatment, Treatment for Pain)
An event was considered a treated bleed if coagulation factors were administered to treat signs or symptoms of bleeding (e.g., pain, swelling, etc.).
Time frame: From baseline to whenever bleeding occurred (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of bleeds analyzed=number of bleeds during analysis at given timepoint.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Bleeds Treated With Each Type of Treatment for Bleeding (Haemostatic Treatment, Treatment for Pain) | Haemostatic Treatment | 93 number of treated bleeds |
| Standard of Care: 12-17 Years | Number of Bleeds Treated With Each Type of Treatment for Bleeding (Haemostatic Treatment, Treatment for Pain) | Treatment for Pain | 1 number of treated bleeds |
Secondary
Number of Bleeds Which Needed to Administer an Additional Hemostatic Therapy
Time frame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of bleeds analyzed=number of bleeds during analysis at given timepoint.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Standard of Care: 12-17 Years | Number of Bleeds Which Needed to Administer an Additional Hemostatic Therapy | 4 number of bleeds |
Secondary
Number of Bleeds Which Needed to Receive a Transfusion
Time frame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of bleeds analyzed=number of bleeds during analysis at given timepoint
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Standard of Care: 12-17 Years | Number of Bleeds Which Needed to Receive a Transfusion | 0 number of bleeds |
Secondary
Number of Bleeds With Adherence to Treatment Regimen for Haemophilia A in the Week Preceding the Bleeding
The adherence to the treatment regimen for haemophilia A was analyzed as the number of bleeds with an answer equal to Yes and My therapeutic plan does not include any treatment in the last week in the week preceding the bleeding.
Time frame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of bleeds analyzed=number of bleeds during analysis at given timepoint.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Standard of Care: 12-17 Years | Number of Bleeds With Adherence to Treatment Regimen for Haemophilia A in the Week Preceding the Bleeding | 77 Number of bleeds |
Secondary
Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application
Sites of bleeds were defined as urine, faeces, and vomit (three sites grouped together), joint, muscle, and other bleeds.
Time frame: Baseline and whenever bleeding occured from study initiation to completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Urine, Faeces, and Vomit Bleeds | 0 Bleeds | 17 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Urine, Faeces, and Vomit Bleeds | 1 or More Bleeds | 0 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Joint Bleeds | 0 Bleeds | 13 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Joint Bleeds | 1 or More Bleeds | 4 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Muscle Bleeds | 0 Bleeds | 15 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Muscle Bleeds | 1 or More Bleeds | 2 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Other Bleeds | 0 Bleeds | 15 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Other Bleeds | 1 or More Bleeds | 2 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Joint Bleeds | 0 Bleeds | 31 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Other Bleeds | 0 Bleeds | 40 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Joint Bleeds | 1 or More Bleeds | 15 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Muscle Bleeds | 0 Bleeds | 40 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Muscle Bleeds | 1 or More Bleeds | 6 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Urine, Faeces, and Vomit Bleeds | 0 Bleeds | 45 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Urine, Faeces, and Vomit Bleeds | 1 or More Bleeds | 1 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Other Bleeds | 1 or More Bleeds | 6 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Joint Bleeds | 0 Bleeds | 26 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Urine, Faeces, and Vomit Bleeds | 1 or More Bleeds | 4 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Urine, Faeces, and Vomit Bleeds | 0 Bleeds | 36 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Joint Bleeds | 1 or More Bleeds | 14 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Other Bleeds | 0 Bleeds | 34 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Muscle Bleeds | 1 or More Bleeds | 4 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Muscle Bleeds | 0 Bleeds | 36 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Other Bleeds | 1 or More Bleeds | 6 Participants |
Secondary
Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application
The assessment of bleeds was divided into spontaneous bleeds, traumatic bleeds, and bleeds related to procedure/surgery. Spontaneous bleeds = the occurrence of hemorrhage where neither the participant nor a caregiver could identify a reason. Traumatic bleeds = hemorrhage occurring secondary to an event such as trauma, strenuous activity, or overuse. Bleeds related to procedure/surgery = such as hematomas resulting from any surgeries or invasive procedures.
Time frame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months).
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Spontaneous Bleed | 0 Bleeds | 15 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Spontaneous Bleed | 1 or More Bleeds | 2 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Traumatic Bleed | 0 Bleeds | 14 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Traumatic Bleed | 1 or More Bleeds | 3 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Bleeds Related to Procedures/Surgeries | 0 Bleeds | 17 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Bleeds Related to Procedures/Surgeries | 1 or More Bleeds | 0 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Bleeds Related to Procedures/Surgeries | 1 or More Bleeds | 0 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Spontaneous Bleed | 0 Bleeds | 36 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Traumatic Bleed | 1 or More Bleeds | 18 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Bleeds Related to Procedures/Surgeries | 0 Bleeds | 46 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Spontaneous Bleed | 1 or More Bleeds | 10 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Traumatic Bleed | 0 Bleeds | 28 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Spontaneous Bleed | 1 or More Bleeds | 7 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Traumatic Bleed | 0 Bleeds | 26 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Bleeds Related to Procedures/Surgeries | 1 or More Bleeds | 1 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Traumatic Bleed | 1 or More Bleeds | 14 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Spontaneous Bleed | 0 Bleeds | 33 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | Bleeds Related to Procedures/Surgeries | 0 Bleeds | 39 Participants |
Secondary
Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study, Reported Via the ePRO Application
Bleeds that occurred the same day but in different body site were counted separately.
Time frame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study, Reported Via the ePRO Application | 0 Bleeds | 11 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study, Reported Via the ePRO Application | 1 or More Bleeds | 6 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study, Reported Via the ePRO Application | 0 Bleeds | 25 Participants |
| Standard of Care: 18-30 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study, Reported Via the ePRO Application | 1 or More Bleeds | 21 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study, Reported Via the ePRO Application | 0 Bleeds | 22 Participants |
| Standard of Care:31-50 Years | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study, Reported Via the ePRO Application | 1 or More Bleeds | 18 Participants |
Secondary
Number of Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study
Participants could have received more than one medication.
Time frame: Baseline and every 3 months until study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Etoricoxib | 5 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Paracetamol | 4 Participants |
Secondary
Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study
Participants could have received more than one medication.
Time frame: From baseline to study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Octocog Alfa | 39 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Efmoroctocog alfa | 25 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Moroctocog Alfa | 8 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Turoctocog Alfa | 8 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Lonoctocog Alfa | 6 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Rurioctocog Alfa Pegol | 6 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Damoctocog Alfa Pegol | 3 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Simoctocog Alfa | 3 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Turoctocog Alfa Pegol | 3 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Factor VIII (Antihaemophilic Factor) | 2 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Factor VIII, Recombinant | 1 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Emicizumab | 17 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Desmopressin | 1 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia: Tranexamic Acid | 2 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | On Demand for Haemophilia: Octocog Alfa | 1 Participants |
Secondary
Number of Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study
The following types of regimens used to treat haemophilia A are reported here: on-demand (to stop bleeding) and prophylaxis (regular infusions of clotting factor to prevent bleeds). Participants could have received more than one medication.
Time frame: From baseline to study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis For Haemophilia | 102 Participants |
| Standard of Care: 12-17 Years | Number of Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | On Demand for Haemophilia | 1 Participants |
Secondary
Number of Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used
Participants could have received more than one medication.
Time frame: From baseline to the time of switch to a new therapy (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Turoctocog Alfa Pegol | 7 Participants |
| Standard of Care: 12-17 Years | Number of Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Damoctocog Alfa Pegol | 3 Participants |
| Standard of Care: 12-17 Years | Number of Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Rurioctocog Alfa Pegol | 3 Participants |
| Standard of Care: 12-17 Years | Number of Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Emicizumab | 8 Participants |
Secondary
Number of Participants Who Were Adherent to the Treatment Regimen for Haemophilia A Over the Course of the Study by Adherence Category (<80% vs. ≥80% of Study Weeks)
The adherence to the treatment regimen for haemophilia A was analyzed as the number of participants with \<80% or ≥80% of weeks on study with Yes and My therapeutic plan does not include any treatment in the last week over the total number of weeks reported via the ePRO. Missing data from some weeks was treated as No. The total number of weeks on study for each participant was derived as (End of study date - Baseline date + 1) / 7.
Time frame: From baseline to study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. A total of 4 participants had missing data and were excluded from this analysis
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Participants Who Were Adherent to the Treatment Regimen for Haemophilia A Over the Course of the Study by Adherence Category (<80% vs. ≥80% of Study Weeks) | Adherence <80% of Weeks | 72 Participants |
| Standard of Care: 12-17 Years | Number of Participants Who Were Adherent to the Treatment Regimen for Haemophilia A Over the Course of the Study by Adherence Category (<80% vs. ≥80% of Study Weeks) | Adherence ≥80% of Weeks | 27 Participants |
Secondary
Number of Participants With Moderate Versus Severe Haemophilia A by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study
Participants could have received more than one medication.
Time frame: Baseline and every 3 months until study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Etoricoxib | 0 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Paracetamol | 0 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Etoricoxib | 5 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Paracetamol | 4 Participants |
Secondary
Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A
Participants could have received more than one medication.
Time frame: From baseline to study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Turoctocog Alfa | 0 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Simoctocog Alfa | 1 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Octocog Alfa | 1 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Turoctocog Alfa Pegol | 0 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Lonoctocog Alfa | 0 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Factor VIII (Antihaemophilic Factor) | 0 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Moroctocog Alfa | 1 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Factor VIII, Recombinant | 0 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Rurioctocog Alfa Pegol | 0 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Desmopressin | 0 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Efmoroctocog Alfa | 2 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Tranexamic Acid | 1 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Damoctocog Alfa Pegol | 0 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | On Demand; Octocog Alfa | 1 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Emicizumab | 0 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | On Demand; Octocog Alfa | 0 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Emicizumab | 17 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Octocog Alfa | 38 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Efmoroctocog Alfa | 23 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Moroctocog Alfa | 7 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Turoctocog Alfa | 8 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Lonoctocog Alfa | 6 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Rurioctocog Alfa Pegol | 6 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Damoctocog Alfa Pegol | 3 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Simoctocog Alfa | 2 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Turoctocog Alfa Pegol | 3 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Factor VIII (Antihaemophilic Factor) | 2 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Factor VIII, Recombinant | 1 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Desmopressin | 1 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Prophylaxis; Tranexamic Acid | 1 Participants |
Secondary
Number of Participants With Moderate Versus Severe Haemophilia A by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study
The following types of regimens used to treat haemophilia A are reported here: on-demand (to stop bleeding) and prophylaxis (regular infusions of clotting factor to prevent bleeds). Participants could have received more than one medication.
Time frame: From baseline to study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia | 5 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | On Demand for Haemophilia | 1 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | Prophylaxis for Haemophilia | 97 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | On Demand for Haemophilia | 0 Participants |
Secondary
Number of Participants With Moderate Versus Severe Haemophilia A Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used
Participants could have received more than one medication.
Time frame: From baseline to the time of switch to a new therapy (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Turoctocog Alfa Pegol | 2 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Damoctocog Alfa Pegol | 0 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Rurioctocog Alfa Pegol | 0 Participants |
| Standard of Care: 12-17 Years | Number of Participants With Moderate Versus Severe Haemophilia A Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Emicizumab | 0 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Emicizumab | 8 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Turoctocog Alfa Pegol | 5 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Rurioctocog Alfa Pegol | 3 Participants |
| Standard of Care: 18-30 Years | Number of Participants With Moderate Versus Severe Haemophilia A Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Damoctocog Alfa Pegol | 3 Participants |
Secondary
Number of Treated Bleeds Over the Course of the Study
An event was considered a treated bleed if coagulation factors were administered to treat signs or symptoms of bleeding (e.g., pain, swelling, etc.).
Time frame: Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of bleeds analyzed=number of bleeds during analysis at given timepoint.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Standard of Care: 12-17 Years | Number of Treated Bleeds Over the Course of the Study | 94 number of treated bleeds |
Secondary
Pain Intensity, as Reported by Participants Using a Visual Analogue Scale
Participants reported the intensity of pain in their main joints (elbow, knee, ankle) by marking on a visual analogue scale (VAS), a horizontal line 100 millimeters (mm) in length, anchored by word descriptors at each end, where the left extreme means No pain = 0 while the right extreme means Worst Pain Imaginable = 100. The VAS score is the distance (in mm) from the left end of the line to the point where the participant marks crossed the line. VAS scale was submitted to the participants every month during the study.
Time frame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 18 | 1.00 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Baseline | 14.24 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 1 | 18.38 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 2 | 16.91 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 3 | 17.53 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 4 | 21.77 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 5 | 18.76 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 6 | 18.98 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 7 | 19.89 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 8 | 17.85 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 9 | 17.58 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 10 | 18.20 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 11 | 20.26 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 12 | 19.85 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 13 | 29.50 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 14 | 21.40 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 15 | 20.75 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 16 | 12.50 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Month 17 | 15 scores on a scale |
Secondary
Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale
Participants reported intensity of pain in their main joints (elbow, knee, ankle) by marking on a VAS, a horizontal line 100 mm in length, anchored by word descriptors at each end, where the left extreme means No pain = 0 while right extreme means Worst Pain Imaginable = 100. VAS score is the distance (in mm) from left end of the line to the point where participant marks crossed the line. A participant who was adherent to WHO guidelines for physical activity according to the below definition was defined as an active participant, otherwise as a sedentary participant. WHO guidelines for adherence: 12-17 years old: MVPA minutes per day ≥60 minutes. 18-50 years old: Fairly active (moderate) minutes per week ≥150 minutes or Very active (vigorous) minutes per week ≥75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). VAS scale was submitted to the participants every month during the study.
Time frame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified timepoint.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Baseline | 17.86 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 3 | 22.80 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 4 | 27.56 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 5 | 23.28 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 6 | 24.09 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 7 | 22.52 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 8 | 22.02 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 9 | 17.80 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 10 | 20.16 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 11 | 20.72 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 12 | 22.68 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 13 | 7.50 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 14 | 8.33 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 15 | 16.67 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 16 | 12.50 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 17 | 15 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 18 | 1 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 1 | 21.29 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 2 | 18.32 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Baseline | 14.12 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 8 | 19.98 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 1 | 15.73 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 12 | 25 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 2 | 16.75 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 9 | 24.81 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 3 | 17.17 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 14 | 33 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 4 | 24.22 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 10 | 20.98 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 5 | 20 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 13 | 51.50 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 6 | 20.67 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 11 | 21.09 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 7 | 20.78 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Month 15 | 33 scores on a scale |
Secondary
Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale
Participants reported the intensity of pain in their main joints (elbow, knee, ankle) by marking on a VAS, a horizontal line 100 mm in length, anchored by word descriptors at each end, where the left extreme means No pain = 0 while the right extreme means Worst Pain Imaginable = 100. The VAS score is the distance (in mm) from the left end of the line to the point where the participant marks crossed the line. VAS scale was submitted to the participants every month during the study.
Time frame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17 and 18
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoint.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Baseline | 23.33 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 1 | 23.67 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 2 | 19.20 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 3 | 21.00 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 4 | 29.00 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 5 | 26.20 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 6 | 26.40 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 7 | 28.75 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 8 | 27.63 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 9 | 17.00 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 10 | 19.38 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 11 | 11.50 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 12 | 19.33 scores on a scale |
| Standard of Care: 12-17 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 13 | 70.00 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 18 | 1.00 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 7 | 19.29 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 13 | 16.00 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 8 | 17.21 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 14 | 21.40 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 9 | 17.64 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Baseline | 13.66 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 15 | 20.75 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 1 | 17.96 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 10 | 18.12 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 2 | 16.75 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 16 | 12.50 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 3 | 17.29 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 11 | 20.95 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 4 | 21.28 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 17 | 15.00 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 5 | 18.20 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 12 | 19.88 scores on a scale |
| Standard of Care: 18-30 Years | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Month 6 | 18.45 scores on a scale |
Secondary
Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study
A participant was defined adherent to WHO guidelines according to the following algorithm: 12-17 years old: MVPA minutes per day \>= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week \>= 150 minutes or very active (vigorous) minutes per week \>= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). A participant who was adherent to the WHO guidelines according to the above definition was defined as an active participant, otherwise as a sedentary participant.
Time frame: From baseline to study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Standard of Care: 12-17 Years | Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study | Yes (Active Subjects) | 0 percentage of participants |
| Standard of Care: 12-17 Years | Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study | No (Sedentary Subjects) | 100 percentage of participants |
| Standard of Care: 18-30 Years | Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study | Yes (Active Subjects) | 68.2 percentage of participants |
| Standard of Care: 18-30 Years | Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study | No (Sedentary Subjects) | 31.8 percentage of participants |
| Standard of Care:31-50 Years | Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study | Yes (Active Subjects) | 42.3 percentage of participants |
| Standard of Care:31-50 Years | Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study | No (Sedentary Subjects) | 57.7 percentage of participants |
Comparison: Comparison among age groups was performed using Chi-squared test.p-value: 0.0088Chi-squared
Secondary
Yearly Rate of Days Away From School for Participants With Moderate Versus Severe Haemophilia A
The number of days away from school or work (participant) was analyzed every month as a continuous variable. The yearly rate was derived on a per participant basis as the proportion between the total number of days away from school and/or work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25.
Time frame: Baseline and every month until study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Yearly Rate of Days Away From School for Participants With Moderate Versus Severe Haemophilia A | 0.0 days/year |
| Standard of Care: 18-30 Years | Yearly Rate of Days Away From School for Participants With Moderate Versus Severe Haemophilia A | 0.0 days/year |
Secondary
Yearly Rate of Days Away From School or Work for Participants
The number of days away from school or work (participant) was analyzed every month as a continuous variable. The yearly rate was derived on a per participant basis as the proportion between the total number of days away from school and/or work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25.
Time frame: Baseline and every month until study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Yearly Rate of Days Away From School or Work for Participants | 0 days/year |
Secondary
Yearly Rate of Days Away From Work for Parent/Caregiver
The number of days away from work was analyzed every month as a continuous variable. The yearly rate was derived on a per participant (for parents and caregivers) basis as the proportion between the total number of days away from work and duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25.
Time frame: Baseline and every month until study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Yearly Rate of Days Away From Work for Parent/Caregiver | 0 days/year |
Secondary
Yearly Rate of Days Away From Work for Parents/Caregivers of Participants With Moderate Versus Severe Haemophilia A
The number of days away from work was analyzed every month as a continuous variable. The yearly rate was derived on a per participant (for parents/caregivers) basis as the proportion between the total number of days away from work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25.
Time frame: Baseline and every month until study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Yearly Rate of Days Away From Work for Parents/Caregivers of Participants With Moderate Versus Severe Haemophilia A | 0.0 days/year |
| Standard of Care: 18-30 Years | Yearly Rate of Days Away From Work for Parents/Caregivers of Participants With Moderate Versus Severe Haemophilia A | 0 days/year |
Secondary
Yearly Rate of Hospitalization Days
The yearly rate was derived on a per participant basis as the proportion between the total number of hospitalization days and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25.
Time frame: Baseline and every month until study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Yearly Rate of Hospitalization Days | 0.0 days/year |
Secondary
Yearly Rate of Hospitalization Days for Participants With Moderate Versus Severe Haemophilia A
The yearly rate was derived on a per participant basis as the proportion between the total number of hospitalization days and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25.
Time frame: Baseline and every month until study completion (up to 18 months)
Population: ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Yearly Rate of Hospitalization Days for Participants With Moderate Versus Severe Haemophilia A | 0 days/year |
| Standard of Care: 18-30 Years | Yearly Rate of Hospitalization Days for Participants With Moderate Versus Severe Haemophilia A | 0 days/year |
Secondary
Yearly Rate of Number of Days Away From School/Work for Active Versus Sedentary Participants
The number of days away from school or work (participant) was analyzed every month as a continuous variable. The yearly rate was derived on a per participant basis as the proportion between the total number of days away from school and/or work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25.
Time frame: Baseline and every month until study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Yearly Rate of Number of Days Away From School/Work for Active Versus Sedentary Participants | 0.0 days/year |
| Standard of Care: 18-30 Years | Yearly Rate of Number of Days Away From School/Work for Active Versus Sedentary Participants | 0.0 days/year |
Secondary
Yearly Rate of Number of Days Away From Work for Active Versus Sedentary Participant's Parents or Caregivers
The number of days away from work was analyzed every month as a continuous variable. The yearly rate was derived on a per participant (for parents/caregivers) basis as the proportion between the total number of days away from work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25.
Time frame: Baseline and every month until study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Yearly Rate of Number of Days Away From Work for Active Versus Sedentary Participant's Parents or Caregivers | 0.0 days/year |
| Standard of Care: 18-30 Years | Yearly Rate of Number of Days Away From Work for Active Versus Sedentary Participant's Parents or Caregivers | 0.0 days/year |
Secondary
Yearly Rate of Number of Hospitalization Days for Active Versus Sedentary Participants
The yearly rate was derived on a per participant basis as the proportion between the total number of hospitalization days and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25.
Time frame: Baseline and every month until study completion (up to 18 months)
Population: Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Standard of Care: 12-17 Years | Yearly Rate of Number of Hospitalization Days for Active Versus Sedentary Participants | 0 days |
| Standard of Care: 18-30 Years | Yearly Rate of Number of Hospitalization Days for Active Versus Sedentary Participants | 0 days |