Hemophilia A, Prophylaxis of Bleeding
Conditions
Brief summary
Researchers are looking for a better way to treat people who have hemophilia A. Hemophilia A is a genetic bleeding disorder that is caused by the lack of a protein in the blood called clotting factor 8 (FVIII). FVIII is naturally found in the blood where it causes the blood to clump together to help prevent and stop bleeding. People with lower levels of FVIII or with FVIII that does not work properly may bleed for a long time from minor wounds, have painful bleeding into joints, or have internal bleeding. The study treatment, Jivi (also called damoctocog alfa pegol), is already available for doctors to prescribe to people with hemophilia A to treat and prevent bleeding. It works by replacing the missing FVIII, or the FVIII that does not work properly. People with hemophilia A need frequent injections of FVIII products into the vein. So called standard half-life (SHL) products need to be given 2 to 4 times a week for the prevention of bleeding. In recent years, new products like Jivi called extended half-life (EHL) products have become available. These products last longer in the body so that they require to be given less often with injections every 3-5 days. Thus, these treatments may be easier and more comfortable to stick to in daily life. There is no general plan concerning the best amount of treatment and the frequency of injections for the prevention of bleeding, since the severity may be different and individual risk factors have to be considered. Doctors often decide on a treatment plan based on their experience. The main purpose of this study is to learn how well a new scoring approach works to select a treatment plan for the prevention of bleeding in people with hemophilia A who switch their treatment from SHL products to Jivi. Different types of information are used to calculate the risk score like bleeding history, certain biological factors, and physical activity of the participant. All participants will receive Jivi for 6 months. In the first four weeks, all participants will receive Jivi 2 times a week at a dose level of 40 IU per kilogram body weight (also known as 40 IU/kg/dose, recommended maximum dose is 6,000 IU). Then, based on their risk score, each participant will be assigned to one of three treatment plans: * participants with a high risk remain on Jivi administration 2 times a week at 40 IU/kg/dose * participants with a medium risk will switch to Jivi administration every 5 days at 50 IU/kg/dose * participants with a low risk will switch to Jivi administration every 5 days at 50 IU/kg/dose and after 4 weeks to a less frequent administration (e.g., every 7 days) at 60 IU/kg/dose To check how well the new scoring approach works for choosing the right treatment plan, researchers will look at how many participants have a favourable outcome. This means that the participant has either fewer bleeding events vs. the pre-study treatment and takes Jivi less often or as often as the previous SHL treatment but with fewer bleeding events, or that the participant has a comparable number of bleeding events but needs to take Jivi less often than the previous treatment. Each participant will be in the study for approximately 7.5 months. During this time, 4 visits to the study site and 3 phone calls are planned. During the study, the doctors and their study team will: • do physical examinations • take blood samples • ask the participants questions about how they are feeling and what adverse events they are having. In addition, participants or their guardians are required to write down the dates of Jivi treatments and bleeding events in an electronic diary and to fill in different questionnaires on their quality of life, health status, work/ school productivity, pain, and treatment satisfaction. In addition, participants are expected to keep appointments for visits and to adhere to the assigned treatment regimen. An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments.
Interventions
BIOLOGICALDamoctocog alfa-pegol is a recombinant B-domain deleted human coagulation FVIII variant site specifically conjugated with a 60 kDa, branched (30 kDa each) polyethylene glycol (PEG).
Dosage Levels: * 40 IU/kg/dose two times per week * 50 IU/kg/dose every 5 days * 60 IU/kg/dose, Less frequent dosing (e.g. every 7 days), the total recommended maximum dose/infusion is 6000 IU.
Sponsors
Bayer
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participants must be ≥ 12 years of age inclusive, at the time of signing the informed consent/assent. * Previously treated patients (≥ 150 EDs) with congenital hemophilia A. * Prophylaxis with any SHL FVIII product with a stable frequency for at least 6 consecutive months within the last 12 months prior to screening before entering the study and documented in medical records. Stable frequency is defined as a minimum 18 weeks of treatment in a 6 (consecutive) calendar month period in the 12 months prior to screening. Patients can be on any non-Jivi EHL between the 6-month stable SHL prophylaxis period and start of study treatment. * Documented bleeding rate (ABR) while on stable frequency SHL prophylaxis for at least 6 consecutive months within the last 12 months prior to screening. * No current evidence (≥ 0.6 BU/mL) of FVIII inhibitors. If a participant has had a positive inhibitor titer in the past (≥ 0.6 BU/mL on two occasions) but has been tolerized for at least 1 year since the last positive titer with at least 1 negative inhibitor assay test during that period, they can be enrolled. If a participant has had a positive inhibitor titer in the past (≥ 0.6 BU/mL) but did not require tolerization and has had at least 1 negative inhibitor assay test during a minimum period of at least 1 year since the last positive titer, they can be enrolled. * If they are human immunodeficiency virus (HIV) positive, cluster of differentiation 4 (CD4+) lymphocyte count should be \> 200/mm\^3 within 1 year before entering the study and documented in medical records. - * Participants who are willing to complete an electronic diary (eDiary). * Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. * For adolescent participants (≥ 12 to \< 18 years), a legal guardian must be available to help the study-site personnel ensure follow-up; accompany the participant to the study site on each assessment day according to the Schedule of Activities (SoA) (e.g. able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures); consistently and consecutively be available to provide information on the participant using the PROs during the scheduled study visits; accurately and reliably dispense study intervention as directed. * For adolescent participants, a legal guardian must be able to accurately maintain the child's take-home record, including items of general health.
Exclusion criteria
* Any other inherited or acquired bleeding disorder in addition to hemophilia A. Note: von Willebrand disease should be diagnosed per local clinical practice. Participants with a diagnosis of von Willebrand disease in medical records or diagnosed at the time of screening will be excluded. * Platelet count \< 100,000/mm\^3 * Evidence of inhibitor to FVIII (≥ 0.6 BU/mL) within the last 1 year * The participant is currently participating in another investigational drug study or has participated in a clinical study involving an investigational drug or device within 30 days of signing informed consent. * The participant has a planned major surgery. * Documentation of missing risk score parameters other than physical activity . * Known hypersensitivity to the drug substance, excipients, or mouse or hamster protein. * Any other significant medical condition that the investigator feels would be a risk to the participant or would impede the study. * Close affiliation with the investigational site; e.g. a close relative of the investigator, dependent person (e.g. employee or student of the investigational site). * Otherwise vulnerable participants (e.g. participants who are in custody by order of an authority). * Judgment by the investigator that the participant should not participate in the study if the participant is unlikely to comply with study procedures (i.e. eDiary completion, clinic visits, phone updates), restrictions, and requirements.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Number of Participants With Favorable Outcome on the Score-assigned Prophylaxis Regimen | up to 6 months | Overall number of participants with favorable outcomes after prophylaxis regimen assignment by a risk score, based on a participant's baseline phenotypic and biologic variables, when switching from a standard half-life (SHL) product to Jivi. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Total ABR From Pre-study | up to 6 months | To assess the efficacy of Jivi compared to a previous SHL treatment. |
| Change in the Frequency of Pre-study SHL Teratment to the Frequency of Jivi Administration (Infusions/Month) | up to 6 months | To assess the frequency of Jivi administration. |
| Occurrence of Participants With 0 and ≤ 1 Spontaneous Bleeds | up to 6 months | To assess the proportion of participants with 0 and ≤ 1 spontaneous bleeds. |
| Change in Haemophilia Quality of Life Questionnaire (Haem-A-QoL or Haemo-QoL) | up to 6 months | To assess participant quality of life (QoL) and physical activity, as measured by the Patient Reported Outcomes (PROs) Hemophilia A-specific quality of life and Hemophilia-specific quality of life. This includes 41 items covering 6 domains: Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact, and Treatment Concerns. The Haemo-QoL Short Form contains 35 items covering 9 domains: Physical Health, View of Yourself, Family, Friends, Others, Sports, Dealing, and Treatment. A higher score on the questionnaire represents greater impairment, and a negative change from baseline represents improvement. The percentage score is sum of item scores divided by the possible range. Individual item scores range from 1 to 5. |
| Annualized Bleeding Rate (ABR) (Total, Joint, Spontaneous) | up to 6 months | To assess the efficacy of Jivi |
| EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | up to 6 months | To assess participant quality of life (QoL) and physical activity, as measured by Patient-Reported Outcomes (PROs). Change to baseline means changes between Visit 2 and Visit 6 |
| Treatment Satisfaction Questionnaire for Medication (TSQM) | up to 6 months | To assess participant quality of life (QoL) and physical activity, as measured by Patient-Reported Outcomes (PROs). Score ranges from 10 (lowest satisfaction) to 100 (greatest satisfaction). |
| Work Productivity and Activity Impairment (WPAI) Questionnaire Scores | up to 6 months | Work Productivity and Activity Impairment Questionnaire scores for hemophilia effect over the last 7 days on productivity or ability at work, at school, and in regular daily activities. It ranges from 0% to 100%, where higher scores indicate greater work impairment and less productivity. To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs). Baseline = Visit 2, Change from Baseline = Visit 6 |
| Number of Target Joints and Change in Target Joint Status From Baseline | up to 6 months | To assess target joint status, per modified International Society on Thrombosis and Haemostasis (ISTH) guidelines |
| Patient's Global Impression of Change (PGI-C) | up to 6 months | To assess participant quality of life (QoL) and physical activity, as measured by Patient-Reported Outcomes (PROs). |
Countries
United States
Participant flow
Recruitment details
The study was conducted at 14 study centers in the US between 28 July 2022 (first particpant first visit) and 12 September 2024 (last participant last visit).
Pre-assignment details
A total of 21 participants were enrolled. 21 participants were assigned to treatment.
Participants by arm
| Arm | Count |
|---|---|
| Overall 2x/week (40 IU/kg/dose) with Jivi for 4 weeks, continued according to assigned prophylaxis regimen. | 21 |
| Total | 21 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Overall |
|---|---|
| Age, Customized Adolescents (12-17 years) | 3 Participants |
| Age, Customized Adults (18-64 years) | 17 Participants |
| Age, Customized From 65-84 years | 1 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 10 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 11 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 19 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 21 |
| other Total, other adverse events | 7 / 21 |
| serious Total, serious adverse events | 2 / 21 |
Outcome results
Primary
Overall Number of Participants With Favorable Outcome on the Score-assigned Prophylaxis Regimen
Overall number of participants with favorable outcomes after prophylaxis regimen assignment by a risk score, based on a participant's baseline phenotypic and biologic variables, when switching from a standard half-life (SHL) product to Jivi.
Time frame: up to 6 months
Population: all patients enrolled, followed for at least 4 months and not having switched from the prophylaxis regimen, determined by the risk-score; This analysis included only one single arm. Within this single arm, each participant was assigned a prophylaxis regimen according to a risk score, based on a participant's phenotypic and biologic variables.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Damoctocog Alfa-pegol Prophylaxis Regimens | Overall Number of Participants With Favorable Outcome on the Score-assigned Prophylaxis Regimen | 12 participants |
Secondary
Annualized Bleeding Rate (ABR) (Total, Joint, Spontaneous)
To assess the efficacy of Jivi
Time frame: up to 6 months
Population: all patients enrolled, followed for at least 4 months and not having switched from the prophylaxis regimen, determined by the risk-score; This analysis included only one single arm. Within this single arm, each participant was assigned a prophylaxis regimen according to a risk score, based on a participant's phenotypic and biologic variables.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Damoctocog Alfa-pegol Prophylaxis Regimens | Annualized Bleeding Rate (ABR) (Total, Joint, Spontaneous) | joint bleeds | 2.800 bleeds per year | Standard Deviation 4.16 |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Annualized Bleeding Rate (ABR) (Total, Joint, Spontaneous) | spontaneous bleeds | 1.788 bleeds per year | Standard Deviation 3.968 |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Annualized Bleeding Rate (ABR) (Total, Joint, Spontaneous) | total bleeds | 4.172 bleeds per year | Standard Deviation 5.516 |
Secondary
Change in Haemophilia Quality of Life Questionnaire (Haem-A-QoL or Haemo-QoL)
To assess participant quality of life (QoL) and physical activity, as measured by the Patient Reported Outcomes (PROs) Hemophilia A-specific quality of life and Hemophilia-specific quality of life. This includes 41 items covering 6 domains: Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact, and Treatment Concerns. The Haemo-QoL Short Form contains 35 items covering 9 domains: Physical Health, View of Yourself, Family, Friends, Others, Sports, Dealing, and Treatment. A higher score on the questionnaire represents greater impairment, and a negative change from baseline represents improvement. The percentage score is sum of item scores divided by the possible range. Individual item scores range from 1 to 5.
Time frame: up to 6 months
Population: All participants enrolled and followed for at least 4 months, only 10 participants completed this patient-reported outcome; This analysis included only one single arm. Within this single arm, each participant was assigned a prophylaxis regimen according to a risk score, based on a participant's phenotypic and biologic variables.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Damoctocog Alfa-pegol Prophylaxis Regimens | Change in Haemophilia Quality of Life Questionnaire (Haem-A-QoL or Haemo-QoL) | Change from baseline at visit 6 | -4.7 Percentage of scores on a scale | Standard Deviation 11.7 |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Change in Haemophilia Quality of Life Questionnaire (Haem-A-QoL or Haemo-QoL) | Visit 2 (Baseline) | 31.5 Percentage of scores on a scale | Standard Deviation 16.38 |
Secondary
Change in the Frequency of Pre-study SHL Teratment to the Frequency of Jivi Administration (Infusions/Month)
To assess the frequency of Jivi administration.
Time frame: up to 6 months
Population: all patients enrolled, followed for at least 4 months; This analysis included only one single arm. Within this single arm, each participant was assigned a prophylaxis regimen according to a risk score, based on a participant's phenotypic and biologic variables.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Damoctocog Alfa-pegol Prophylaxis Regimens | Change in the Frequency of Pre-study SHL Teratment to the Frequency of Jivi Administration (Infusions/Month) | -6.876 infusions per month | Standard Deviation 4.7557 |
Secondary
Change in Total ABR From Pre-study
To assess the efficacy of Jivi compared to a previous SHL treatment.
Time frame: up to 6 months
Population: all patients enrolled, followed for at least 4 months and not having switched from the prophylaxis regimen, determined by the risk-score; This analysis included only one single arm. Within this single arm, each participant was assigned a prophylaxis regimen according to a risk score, based on a participant's phenotypic and biologic variables.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Damoctocog Alfa-pegol Prophylaxis Regimens | Change in Total ABR From Pre-study | -8.005 bleeds per year | Standard Deviation 15.242 |
Secondary
EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire
To assess participant quality of life (QoL) and physical activity, as measured by Patient-Reported Outcomes (PROs). Change to baseline means changes between Visit 2 and Visit 6
Time frame: up to 6 months
Population: All participants enrolled and followed for at least 4 months, only 11 participants completed this patient-reported outcome; This analysis included only one single arm. Within this single arm, each participant was assigned a prophylaxis regimen according to a risk score, based on a participant's phenotypic and biologic variables.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | walking | improved, less impaired | 1 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | anxious or depressed | improved, less impaired | 2 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | walking | no change | 5 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | walking | worsened, more impaired | 5 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | self-care | improved, less impaired | 1 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | self-care | no change | 10 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | self-care | worsened, more impaired | 0 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | usual activity | improved, less impaired | 0 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | usual activity | no change | 11 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | usual activity | worsened, more impaired | 0 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | pain or discomfort | improved, less impaired | 0 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | pain or discomfort | no change | 9 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | pain or discomfort | worsened, more impaired | 2 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | anxious or depressed | no change | 6 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | EuroQoL 5 Dimensions (EQ-5D-5L) Questionnaire | anxious or depressed | worsened, more impaired | 3 Participants |
Secondary
Number of Target Joints and Change in Target Joint Status From Baseline
To assess target joint status, per modified International Society on Thrombosis and Haemostasis (ISTH) guidelines
Time frame: up to 6 months
Population: All participants enrolled and followed for at least 4 months; This analysis included only one single arm. Within this single arm, each participant was assigned a prophylaxis regimen according to a risk score, based on a participant's phenotypic and biologic variables.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Damoctocog Alfa-pegol Prophylaxis Regimens | Number of Target Joints and Change in Target Joint Status From Baseline | minus four (change from baseline) | 1 Joints |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Number of Target Joints and Change in Target Joint Status From Baseline | minus three (change from baseline) | 4 Joints |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Number of Target Joints and Change in Target Joint Status From Baseline | minus two (change from baseline) | 2 Joints |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Number of Target Joints and Change in Target Joint Status From Baseline | minus one (change from baseline) | 4 Joints |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Number of Target Joints and Change in Target Joint Status From Baseline | zero (change from baseline) | 8 Joints |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Number of Target Joints and Change in Target Joint Status From Baseline | zero (baseline) | 8 Joints |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Number of Target Joints and Change in Target Joint Status From Baseline | one (baseline) | 4 Joints |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Number of Target Joints and Change in Target Joint Status From Baseline | two (baseline) | 2 Joints |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Number of Target Joints and Change in Target Joint Status From Baseline | three (baseline) | 3 Joints |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Number of Target Joints and Change in Target Joint Status From Baseline | four (baseline) | 2 Joints |
Secondary
Occurrence of Participants With 0 and ≤ 1 Spontaneous Bleeds
To assess the proportion of participants with 0 and ≤ 1 spontaneous bleeds.
Time frame: up to 6 months
Population: All participants enrolled and followed for at least 4 months; This analysis included only one single arm. Within this single arm, each participant was assigned a prophylaxis regimen according to a risk score, based on a participant's phenotypic and biologic variables.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Damoctocog Alfa-pegol Prophylaxis Regimens | Occurrence of Participants With 0 and ≤ 1 Spontaneous Bleeds | 0 spontaneous bleed | 13 participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Occurrence of Participants With 0 and ≤ 1 Spontaneous Bleeds | <=1 spontaneous bleeds | 13 participants |
Secondary
Patient's Global Impression of Change (PGI-C)
To assess participant quality of life (QoL) and physical activity, as measured by Patient-Reported Outcomes (PROs).
Time frame: up to 6 months
Population: All participants enrolled and followed for at least 4 months, only 9 participants completed this patient-reported outcome; This analysis included only one single arm. Within this single arm, each participant was assigned a prophylaxis regimen according to a risk score, based on a participant's phenotypic and biologic variables.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Damoctocog Alfa-pegol Prophylaxis Regimens | Patient's Global Impression of Change (PGI-C) | status minimally improved | 1 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Patient's Global Impression of Change (PGI-C) | status minimally worse | 1 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Patient's Global Impression of Change (PGI-C) | status much improved | 2 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Patient's Global Impression of Change (PGI-C) | status not changed | 4 Participants |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Patient's Global Impression of Change (PGI-C) | status very much improved | 1 Participants |
Secondary
Treatment Satisfaction Questionnaire for Medication (TSQM)
To assess participant quality of life (QoL) and physical activity, as measured by Patient-Reported Outcomes (PROs). Score ranges from 10 (lowest satisfaction) to 100 (greatest satisfaction).
Time frame: up to 6 months
Population: All participants enrolled and followed for at least 4 months, only 5 participants completed this patient-reported outcome; This analysis included only one single arm. Within this single arm, each participant was assigned a prophylaxis regimen according to a risk score, based on a participant's phenotypic and biologic variables.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Damoctocog Alfa-pegol Prophylaxis Regimens | Treatment Satisfaction Questionnaire for Medication (TSQM) | Visit 2 (baseline) | 37.9 Percentage | Standard Deviation 7.44 |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Treatment Satisfaction Questionnaire for Medication (TSQM) | Change from baseline at visit 6 | -1.8 Percentage | Standard Deviation 6.42 |
Secondary
Work Productivity and Activity Impairment (WPAI) Questionnaire Scores
Work Productivity and Activity Impairment Questionnaire scores for hemophilia effect over the last 7 days on productivity or ability at work, at school, and in regular daily activities. It ranges from 0% to 100%, where higher scores indicate greater work impairment and less productivity. To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs). Baseline = Visit 2, Change from Baseline = Visit 6
Time frame: up to 6 months
Population: All participants enrolled and followed for at least 4 months, participants' number completing this patient-reported outcome varied depending on parameter;This analysis included only one single arm. Within this single arm, each participant was assigned a prophylaxis regimen according to a risk score, based on a participant's phenotypic and biologic variables.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Damoctocog Alfa-pegol Prophylaxis Regimens | Work Productivity and Activity Impairment (WPAI) Questionnaire Scores | Percent Work Time Missed (change from baseline) 4 participants | 19.4 Percentage | Standard Deviation 24.21 |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Work Productivity and Activity Impairment (WPAI) Questionnaire Scores | Percent Working Impairment (baseline) 6 participants | 26.7 Percentage | Standard Deviation 22.51 |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Work Productivity and Activity Impairment (WPAI) Questionnaire Scores | Percent Working Impairment (change from baseline) 3 participants | 10.0 Percentage | Standard Deviation 20 |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Work Productivity and Activity Impairment (WPAI) Questionnaire Scores | Percent Class Time Missed (change from baseline) 2 participants | 1.4 Percentage | Standard Deviation 1.96 |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Work Productivity and Activity Impairment (WPAI) Questionnaire Scores | Percent Class Attending Impairment (baseline) 4 participants | 15.0 Percentage | Standard Deviation 19.15 |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Work Productivity and Activity Impairment (WPAI) Questionnaire Scores | Percent Class Attending Impairment (change) 2 participants | 0 Percentage | Standard Deviation 28.28 |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Work Productivity and Activity Impairment (WPAI) Questionnaire Scores | Percent Daily Activities Impairment (baseline) 12 participants | 33.3 Percentage | Standard Deviation 29.34 |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Work Productivity and Activity Impairment (WPAI) Questionnaire Scores | Percent Work Time Missed (baseline) 7 participants | 1.8 Percentage | Standard Deviation 4.64 |
| Damoctocog Alfa-pegol Prophylaxis Regimens | Work Productivity and Activity Impairment (WPAI) Questionnaire Scores | Percent Class Time Missed (baseline) 4 participants | 5.0 Percentage | Standard Deviation 10 |