Fabry Disease
Conditions
Brief summary
This is an international, non-interventional research study of adult patients with Fabry Disease and their caregivers. The study comprised a prospective time and motion evaluation and a cross-sectional evaluation of patient and caregiver-reported outcomes. The study evaluated the time associated with the preparation and administration of a single dose of enzyme replacement therapy (ERT) in patients by health care providers as well as the impact on Fabry patients and caregivers time and costs associated with an ERT treatment. The study also evaluated the patients' quality of life wellbeing, fatigue and work productivity.
Interventions
ERT infusion every other week
Sponsors
Amicus Therapeutics
Study design
Observational model
COHORT
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Inclusion criteria
Patient Inclusion Criteria: * Patients with a documented diagnosis of Fabry Disease (FD) * Patients who have received ≥4 doses of ERT (with agalsidase alfa or agalsidase beta) for the treatment of FD. * Patients who present to the participating hospital(s) or treatment centre(s) for administration of a dose of ERT (as part of their routine treatment) during the data collection period. Caregiver Inclusion Criteria: -Self-identifies as a caregiver of a patient with FD for whom written informed consent has been obtained for inclusion in the study. Patient
Exclusion criteria
* Patients who are unable or unwilling to give consent for study participation. * Patients whose ERT preparation and administration takes place exclusively in the home setting with no healthcare professional (HCP) involvement in preparation of the infusion. * For the time and motion evaluation: Patients whose ERT is administered by a HCP who does not consent to be observed. Caregiver
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease | up to 7 weeks | Total time (minutes) spent by HCPs on all activities related to administration of a single dose of ERT in patients with Fabry Disease including pre-infusion (pre-administration patient consultation; infusion and premedication preparation), ERT (and premedication) administration, and post-administration assessment and documentation. Results are shown for all patients with Fabry Disease on ERT in study |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Total Time Spent by HCPs on Each Separate Task Associated With the Preparation and Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta). | up to 7 weeks | Total time spent (in minutes) by healthcare professionals (HCP) on tasks associated with preparation and administration of a single dose of ERT (with agalsidase alfa or agalsidase beta) in patients with Fabry Disease including pre-administration activities (patient consultation, infusion/pre-medication preparation activities), administration of ERT (and pre-medications), and post-infusion activities (monitoring of patient during and after administration, completion of clinical documentation, and any other ERT-administration related activities). |
| Total Patient Time Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta). | up to 7 weeks | Total patient time associated with attendance for administration of a single dose of ERT includes the total time from when the patient left home, to when they returned home and based on the reported time spent travelling, total waiting time, the total time spent during infusion episode (pre, during and post) and the other recorded activities. |
| Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Brazil. | up to 7 weeks | Total out-of-pocket costs (eg, food, travel, internet) incurred by patients in Brazil that were directly related to attendance for the administration of a single dose of ERT. |
| Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Taiwan. | up to 7 weeks | Total out-of-pocket costs (eg, food, travel, internet) incurred by patients in Taiwan that were directly related to attendance for the administration of a single dose of ERT. |
| Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Japan. | up to 7 weeks | Total out-of-pocket costs (eg, food, travel, internet) incurred by patients in Japan that were directly related to attendance for the administration of a single dose of ERT. |
| Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Turkey | up to 7 weeks | Total out-of-pocket costs (eg, food, travel, internet) incurred by patients in Turkey that were directly related to attendance for the administration of a single dose of ERT. |
| Percentage of Patients With Work Absence Due to Attendance for This ERT Episode. | up to 7 weeks | The percentage of patients known to have taken time off from work during their ERT episode |
| Number of Hours of Patient Work Absence Due to Attendance for ERT Episode. | up to 7 weeks | The number of paid and unpaid hours that patients were absent from work during their ERT episode |
| Total Caregiver Time Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT | up to 7 weeks | Total caregiver time (in minutes) associated with accompanying a patient with Fabry Disease for the administration of a single dose of ERT (with agalsidase alfa or agalsidase beta). |
| Total Costs Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT | up to 7 weeks | Total costs associated with accompanying a patient with Fabry Disease for the administration of a single dose of ERT (with agalsidase alfa or agalsidase beta). |
| Percentage of Caregivers With Work Absence Due to Accompanying the Patient for ERT Administration. | up to 7 weeks | Percentage of caregivers with work absence due to accompanying the patient for this episode of ERT administration. |
| Number of Hours of Caregivers Work Absence Due to Accompanying the Patient for ERT Administration. | up to 7 weeks | Number of hours of caregivers work absence due to accompanying the patient for this ERT administration episode. |
| Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses) | HRQoL was completed once (during the ERT infusion visit) prior to leaving the clinic | Health Related Quality of Life (HRQoL) measured by the 12 Item Short Form Survey \[SF-12\]). In the SF-12, patients were asked to rate aspects of their health across 8 domains; all domains have a maximum value of 5 (range 0-5) except physical functioning, for which the maximum score is 3 (range 0-3). Higher scores indicate better HRQoL while low scores indicate worse HRQoL. |
| Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit | WHO-5 was completed once (during the ERT infusion visit) prior to leaving the clinic | World Health Organization-5 Wellbeing Index \[WHO-5\] consists of 5 items and the timeframe for responses is based on the previous 2 weeks. The 5 items relate to feeling cheerful, calm, active, rested and being interested in life. For each question, patients rated the extent to which the statement applies between 0 (none of the time) and 5 (all of the time), with higher scores indicating better wellbeing. The WHO-5 total score is calculated by totaling the values of each of the five questions. The total scores range from 0 to 25, where 0 represents the worst possible quality of life and 25 represents the best possible quality of life. |
| Patients Level of Fatigue Measured by Fatigue Likert Scale | Questionnaire completed during ERT infusion (baseline), the same evening as date of ERT infusion, and once during the period of 1 to 7 days post-infusion | Questionnaire measuring Fatigue. Possible scores range from 1 (not at all tired) to 5 (extremely tired); higher scores therefore indicating worse fatigue. Negative value in change from during ERT infusion to evening of ERT infusion indicates more fatigue in the evening of infusion. Negative value in change from baseline (during ERT infusion) to 1-7 days after ERT infusion indicates more fatigue during the period occurring 1-7 days after ERT infusion. |
| Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion | WPAI completed once by patients during the period of 1 to 7 Days After ERT Infusion | Work Productivity and Activity Index (WPAI) consists of 6 items on the effect of health problems on ability to work and perform regular activities: 1=currently employed; 2=hours missed due to health problems; 3=hours missed other reasons; 4=hours actually worked; 5=degree health affected productivity while working; 6=degree health affected regular activities Score calculation (multiplied by 100 for percent): * Percent work time missed due to health: Q2/(Q2+Q4) * Percent impairment while working due to health: Q5/10 * Percent overall work impairment due to health: Q2/(Q2+Q4) + \[(1-(Q2/(Q2+Q4))) x (Q5/10)\] * Percent activity impairment due to health: Q6/10 Final scores are expressed as impairment percentages (range: 0-100%), with higher numbers indicating greater impairment/less productivity (ie, worse outcomes) and lower numbers indicating less impairment/greater productivity (ie, better outcomes). Data were collected 1 to 7 days after ERT infusion. |
| Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion | WPAI completed once by patients on the day of the next ERT infusion (approximately 15 days after the prior infusion) | Work Productivity and Activity Index (WPAI) consists of 6 items on the effect of health problems on ability to work and perform regular activities: 1=currently employed; 2=hours missed due to health problems; 3=hours missed other reasons; 4=hours actually worked; 5=degree health affected productivity while working; 6=degree health affected regular activities Score calculation (multiplied by 100 for percent): * Percent work time missed due to health: Q2/(Q2+Q4) * Percent impairment while working due to health: Q5/10 * Percent overall work impairment due to health: Q2/(Q2+Q4) + \[(1-(Q2/(Q2+Q4))) x (Q5/10)\] * Percent activity impairment due to health: Q6/10 Final scores are expressed as impairment percentages (range: 0-100%), with higher numbers indicating greater impairment/less productivity (ie, worse outcomes) and lower numbers indicating less impairment/greater productivity (ie, better outcomes). Data were collected on day of next ERT infusion. |
| Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion. | WPAI-CG completed once by caregivers during the period of 1 to 7 days after ERT infusion | Work Productivity and Activity Index (WPAI) consists of 6 items on the effect of health problems on ability to work and perform regular activities: 1=currently employed; 2=hours missed due to health problems; 3=hours missed other reasons; 4=hours actually worked; 5=degree health affected productivity while working; 6=degree health affected regular activities Score calculation (multiplied by 100 for percent): * Percent work time missed due to health: Q2/(Q2+Q4) * Percent impairment while working due to health: Q5/10 * Percent overall work impairment due to health: Q2/(Q2+Q4) + \[(1-(Q2/(Q2+Q4))) x (Q5/10)\] * Percent activity impairment due to health: Q6/10 Final scores are expressed as impairment percentages (range: 0-100%), with higher numbers indicating greater impairment/less productivity (ie, worse outcomes) and lower numbers indicating less impairment/greater productivity (ie, better outcomes). Data were collected 1 to 7 days after ERT infusion. |
| Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion. | WPAI-CG completed once by caregivers on day of the next ERT infusion (approximately 15 days after prior infusion) | Work Productivity and Activity Index (WPAI) consists of 6 items on the effect of health problems on ability to work and perform regular activities: 1=currently employed; 2=hours missed due to health problems; 3=hours missed other reasons; 4=hours actually worked; 5=degree health affected productivity while working; 6=degree health affected regular activities Score calculation (multiplied by 100 for percent): * Percent work time missed due to health: Q2/(Q2+Q4) * Percent impairment while working due to health: Q5/10 * Percent overall work impairment due to health: Q2/(Q2+Q4) + \[(1-(Q2/(Q2+Q4))) x (Q5/10)\] * Percent activity impairment due to health: Q6/10 Final scores are expressed as impairment percentages (range: 0-100%), with higher numbers indicating greater impairment/less productivity (ie, worse outcomes) and lower numbers indicating less impairment/greater productivity (ie, better outcomes). WPAI was completed by caregiver on day of next ERT infusion. |
| Level of Strain in Providing Care for a Patient With Fabry Disease (Caregiver Strain Index [CSI] Scores / Responses). | up to 7 weeks | The Caregiver Strain Index (CSI) consists of 12 questions with Yes/No answer and measures the strain of care provision in five domains (financial, physical, psychological, social and personal). Yes = 1 and No = 0. The range is from 0-12. Total scores are the sum of all responses. A score of 7 or higher indicates a high level of stress. |
Countries
Brazil, Japan, Taiwan, Turkey (Türkiye), United States
Participant flow
Pre-assignment details
76 patients with Fabry Disease on enzyme replacement therapy (ERT) and 6 caregivers of patients with Fabry Disease on ERT were enrolled into the study per protocol for a total of 82 participants. As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group ("Patients with Fabry Disease on ERT") and not separately.
Participants by arm
| Arm | Count |
|---|---|
| Patients With Fabry Disease on ERT Patients with Fabry Disease receiving Enzyme Replacement Therapy (agalsidase alfa or agalsidase beta) infusion every other week | 76 |
| Caregiver Caregiver of patient with Fabry Disease on ERT | 6 |
| Total | 82 |
Baseline characteristics
| Characteristic | Patients With Fabry Disease on ERT | Total | Caregiver |
|---|---|---|---|
| Age, Continuous | 48.9 years | 48.9 years | — |
| Age, Customized Caregiver age range 31 to 40 years | 0 Participants | 2 Participants | 2 Participants |
| Age, Customized Caregiver age range 41 to 50 years | 0 Participants | 3 Participants | 3 Participants |
| Age, Customized Caregiver age range 61 to 70 years | 0 Participants | 1 Participants | 1 Participants |
| ERT Administered to Patients Agalsidase alfa/biosimilar | 28 Participants | 28 Participants | 0 Participants |
| ERT Administered to Patients Agalsidase beta/biosimilar | 48 Participants | 48 Participants | 0 Participants |
| Race and Ethnicity Not Collected | — | 0 Participants | — |
| Region of Enrollment Brazil | 23 participants | 23 participants | 0 participants |
| Region of Enrollment Japan | 4 participants | 4 participants | 0 participants |
| Region of Enrollment Taiwan | 30 participants | 35 participants | 5 participants |
| Region of Enrollment Turkey | 19 participants | 20 participants | 1 participants |
| Sex: Female, Male Female | 31 Participants | 36 Participants | 5 Participants |
| Sex: Female, Male Male | 45 Participants | 46 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 0 | 0 / 0 |
| other Total, other adverse events | 0 / 0 | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 | 0 / 0 |
Outcome results
Primary
Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease
Total time (minutes) spent by HCPs on all activities related to administration of a single dose of ERT in patients with Fabry Disease including pre-infusion (pre-administration patient consultation; infusion and premedication preparation), ERT (and premedication) administration, and post-administration assessment and documentation. Results are shown for all patients with Fabry Disease on ERT in study
Time frame: up to 7 weeks
Population: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Patients With Fabry Disease on ERT | Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease | 151.9 minutes | Standard Deviation 62.5 |
Secondary
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion.
Work Productivity and Activity Index (WPAI) consists of 6 items on the effect of health problems on ability to work and perform regular activities: 1=currently employed; 2=hours missed due to health problems; 3=hours missed other reasons; 4=hours actually worked; 5=degree health affected productivity while working; 6=degree health affected regular activities Score calculation (multiplied by 100 for percent): * Percent work time missed due to health: Q2/(Q2+Q4) * Percent impairment while working due to health: Q5/10 * Percent overall work impairment due to health: Q2/(Q2+Q4) + \[(1-(Q2/(Q2+Q4))) x (Q5/10)\] * Percent activity impairment due to health: Q6/10 Final scores are expressed as impairment percentages (range: 0-100%), with higher numbers indicating greater impairment/less productivity (ie, worse outcomes) and lower numbers indicating less impairment/greater productivity (ie, better outcomes). Data were collected 1 to 7 days after ERT infusion.
Time frame: WPAI-CG completed once by caregivers during the period of 1 to 7 days after ERT infusion
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patients With Fabry Disease on ERT | Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion. | % Work Time Missed Due to Health (Absenteeism) | 0 Percent | Standard Deviation 0 |
| Patients With Fabry Disease on ERT | Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion. | % Impairment While Working Due to Health (Presenteeism) | 17.5 Percent | Standard Deviation 20.6 |
| Patients With Fabry Disease on ERT | Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion. | % Overall Work Impairment Due to Health | 17.5 Percent | Standard Deviation 20.6 |
| Patients With Fabry Disease on ERT | Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - 1 to 7 Days After ERT Infusion. | % Activity Impairment Due to Health | 18.3 Percent | Standard Deviation 18.3 |
Secondary
Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion.
Work Productivity and Activity Index (WPAI) consists of 6 items on the effect of health problems on ability to work and perform regular activities: 1=currently employed; 2=hours missed due to health problems; 3=hours missed other reasons; 4=hours actually worked; 5=degree health affected productivity while working; 6=degree health affected regular activities Score calculation (multiplied by 100 for percent): * Percent work time missed due to health: Q2/(Q2+Q4) * Percent impairment while working due to health: Q5/10 * Percent overall work impairment due to health: Q2/(Q2+Q4) + \[(1-(Q2/(Q2+Q4))) x (Q5/10)\] * Percent activity impairment due to health: Q6/10 Final scores are expressed as impairment percentages (range: 0-100%), with higher numbers indicating greater impairment/less productivity (ie, worse outcomes) and lower numbers indicating less impairment/greater productivity (ie, better outcomes). WPAI was completed by caregiver on day of next ERT infusion.
Time frame: WPAI-CG completed once by caregivers on day of the next ERT infusion (approximately 15 days after prior infusion)
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patients With Fabry Disease on ERT | Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion. | % Work Time Missed Due to Health (Absenteeism) | 5.0 Percent | Standard Deviation 10 |
| Patients With Fabry Disease on ERT | Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion. | % Impairment While Working Due to Health (Presenteeism) | 17.5 Percent | Standard Deviation 20.6 |
| Patients With Fabry Disease on ERT | Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion. | % Overall Work Impairment Due to Health | 21.0 Percent | Standard Deviation 24.3 |
| Patients With Fabry Disease on ERT | Caregiver's Levels of Work Impairment (Work Productivity and Activity Index [WPAI]-CG Scores) - Day of Next ERT Infusion. | % Activity Impairment Due to Health | 16.7 Percent | Standard Deviation 18.6 |
Secondary
Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses)
Health Related Quality of Life (HRQoL) measured by the 12 Item Short Form Survey \[SF-12\]). In the SF-12, patients were asked to rate aspects of their health across 8 domains; all domains have a maximum value of 5 (range 0-5) except physical functioning, for which the maximum score is 3 (range 0-3). Higher scores indicate better HRQoL while low scores indicate worse HRQoL.
Time frame: HRQoL was completed once (during the ERT infusion visit) prior to leaving the clinic
Population: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately. The HRQoL was completed only one time per patient.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patients With Fabry Disease on ERT | Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses) | Bodily Pain | 3.6 score on a scale | Standard Deviation 1.2 |
| Patients With Fabry Disease on ERT | Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses) | General Health | 2.9 score on a scale | Standard Deviation 1.1 |
| Patients With Fabry Disease on ERT | Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses) | Physical Functioning | 2.2 score on a scale | Standard Deviation 0.7 |
| Patients With Fabry Disease on ERT | Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses) | Role Physical | 3.3 score on a scale | Standard Deviation 1.1 |
| Patients With Fabry Disease on ERT | Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses) | Role Emotional | 3.6 score on a scale | Standard Deviation 1.1 |
| Patients With Fabry Disease on ERT | Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses) | Mental Health | 3.5 score on a scale | Standard Deviation 0.9 |
| Patients With Fabry Disease on ERT | Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses) | Vitality | 3.1 score on a scale | Standard Deviation 1.1 |
| Patients With Fabry Disease on ERT | Health Related Quality of Life (HRQoL) (12 Item Short Form Survey [SF-12] Scores / Responses) | Social Function | 3.6 score on a scale | Standard Deviation 1.2 |
Secondary
Level of Strain in Providing Care for a Patient With Fabry Disease (Caregiver Strain Index [CSI] Scores / Responses).
The Caregiver Strain Index (CSI) consists of 12 questions with Yes/No answer and measures the strain of care provision in five domains (financial, physical, psychological, social and personal). Yes = 1 and No = 0. The range is from 0-12. Total scores are the sum of all responses. A score of 7 or higher indicates a high level of stress.
Time frame: up to 7 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Patients With Fabry Disease on ERT | Level of Strain in Providing Care for a Patient With Fabry Disease (Caregiver Strain Index [CSI] Scores / Responses). | 4.0 score on a scale | Standard Deviation 2.3 |
Secondary
Number of Hours of Caregivers Work Absence Due to Accompanying the Patient for ERT Administration.
Number of hours of caregivers work absence due to accompanying the patient for this ERT administration episode.
Time frame: up to 7 weeks
Population: Data are available from 3 caregivers in Taiwan.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Patients With Fabry Disease on ERT | Number of Hours of Caregivers Work Absence Due to Accompanying the Patient for ERT Administration. | Number of Paid Hours Absent from Work | 6.0 hours |
| Patients With Fabry Disease on ERT | Number of Hours of Caregivers Work Absence Due to Accompanying the Patient for ERT Administration. | Number of Unpaid Hours Absent from Work | 3.0 hours |
Secondary
Number of Hours of Patient Work Absence Due to Attendance for ERT Episode.
The number of paid and unpaid hours that patients were absent from work during their ERT episode
Time frame: up to 7 weeks
Population: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately. Note: data were missing for 54% \[41/76\] of patients.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patients With Fabry Disease on ERT | Number of Hours of Patient Work Absence Due to Attendance for ERT Episode. | Number of Paid Hours Absent from Work | 6.6 hours | Standard Deviation 5.4 |
| Patients With Fabry Disease on ERT | Number of Hours of Patient Work Absence Due to Attendance for ERT Episode. | Number of Unpaid Hours Absent from Work | 5.3 hours | Standard Deviation 5.4 |
Secondary
Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit
World Health Organization-5 Wellbeing Index \[WHO-5\] consists of 5 items and the timeframe for responses is based on the previous 2 weeks. The 5 items relate to feeling cheerful, calm, active, rested and being interested in life. For each question, patients rated the extent to which the statement applies between 0 (none of the time) and 5 (all of the time), with higher scores indicating better wellbeing. The WHO-5 total score is calculated by totaling the values of each of the five questions. The total scores range from 0 to 25, where 0 represents the worst possible quality of life and 25 represents the best possible quality of life.
Time frame: WHO-5 was completed once (during the ERT infusion visit) prior to leaving the clinic
Population: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patients With Fabry Disease on ERT | Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit | WHO-5 Total Score | 15.1 score on a scale | Standard Deviation 5.6 |
| Patients With Fabry Disease on ERT | Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit | Item 1: felt cheerful and in good spirts | 3.1 score on a scale | Standard Deviation 1.3 |
| Patients With Fabry Disease on ERT | Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit | Item 2: felt calm and relaxed | 3.2 score on a scale | Standard Deviation 1.3 |
| Patients With Fabry Disease on ERT | Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit | Item 3: felt active and vigorous | 2.8 score on a scale | Standard Deviation 1.4 |
| Patients With Fabry Disease on ERT | Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit | Item 4: wake up feeling fresh and restful | 2.6 score on a scale | Standard Deviation 1.5 |
| Patients With Fabry Disease on ERT | Patients General Wellbeing Measured by World Health Organization-5 Wellbeing Index (WHO-5) Scores / Responses During ERT Infusion Visit | Item 5: daily life filled with things that interest me | 3.4 score on a scale | Standard Deviation 1.2 |
Secondary
Patients Level of Fatigue Measured by Fatigue Likert Scale
Questionnaire measuring Fatigue. Possible scores range from 1 (not at all tired) to 5 (extremely tired); higher scores therefore indicating worse fatigue. Negative value in change from during ERT infusion to evening of ERT infusion indicates more fatigue in the evening of infusion. Negative value in change from baseline (during ERT infusion) to 1-7 days after ERT infusion indicates more fatigue during the period occurring 1-7 days after ERT infusion.
Time frame: Questionnaire completed during ERT infusion (baseline), the same evening as date of ERT infusion, and once during the period of 1 to 7 days post-infusion
Population: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patients With Fabry Disease on ERT | Patients Level of Fatigue Measured by Fatigue Likert Scale | Level of fatigue during infusion | 2.4 score on a scale | Standard Deviation 1.4 |
| Patients With Fabry Disease on ERT | Patients Level of Fatigue Measured by Fatigue Likert Scale | Change in level of fatigue from during ERT infusion to evening of ERT infusion | -0.2 score on a scale | Standard Deviation 1.4 |
| Patients With Fabry Disease on ERT | Patients Level of Fatigue Measured by Fatigue Likert Scale | Change in level of fatigue from during ERT infusion to 1-7 days after ERT infusion | 0.1 score on a scale | Standard Deviation 1.3 |
Secondary
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion
Work Productivity and Activity Index (WPAI) consists of 6 items on the effect of health problems on ability to work and perform regular activities: 1=currently employed; 2=hours missed due to health problems; 3=hours missed other reasons; 4=hours actually worked; 5=degree health affected productivity while working; 6=degree health affected regular activities Score calculation (multiplied by 100 for percent): * Percent work time missed due to health: Q2/(Q2+Q4) * Percent impairment while working due to health: Q5/10 * Percent overall work impairment due to health: Q2/(Q2+Q4) + \[(1-(Q2/(Q2+Q4))) x (Q5/10)\] * Percent activity impairment due to health: Q6/10 Final scores are expressed as impairment percentages (range: 0-100%), with higher numbers indicating greater impairment/less productivity (ie, worse outcomes) and lower numbers indicating less impairment/greater productivity (ie, better outcomes). Data were collected 1 to 7 days after ERT infusion.
Time frame: WPAI completed once by patients during the period of 1 to 7 Days After ERT Infusion
Population: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patients With Fabry Disease on ERT | Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion | % Work Time Missed Due to Health (Absenteeism) | 5.0 Percent | Standard Deviation 9.5 |
| Patients With Fabry Disease on ERT | Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion | % Impairment While Working Due to Health (Presenteeism) | 28.6 Percent | Standard Deviation 29.5 |
| Patients With Fabry Disease on ERT | Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion | % Overall Work Impairment Due to Health | 28.9 Percent | Standard Deviation 27.7 |
| Patients With Fabry Disease on ERT | Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - 1 to 7 Days After ERT Infusion | % Activity Impairment Due to Health | 30.8 Percent | Standard Deviation 29.3 |
Secondary
Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion
Work Productivity and Activity Index (WPAI) consists of 6 items on the effect of health problems on ability to work and perform regular activities: 1=currently employed; 2=hours missed due to health problems; 3=hours missed other reasons; 4=hours actually worked; 5=degree health affected productivity while working; 6=degree health affected regular activities Score calculation (multiplied by 100 for percent): * Percent work time missed due to health: Q2/(Q2+Q4) * Percent impairment while working due to health: Q5/10 * Percent overall work impairment due to health: Q2/(Q2+Q4) + \[(1-(Q2/(Q2+Q4))) x (Q5/10)\] * Percent activity impairment due to health: Q6/10 Final scores are expressed as impairment percentages (range: 0-100%), with higher numbers indicating greater impairment/less productivity (ie, worse outcomes) and lower numbers indicating less impairment/greater productivity (ie, better outcomes). Data were collected on day of next ERT infusion.
Time frame: WPAI completed once by patients on the day of the next ERT infusion (approximately 15 days after the prior infusion)
Population: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patients With Fabry Disease on ERT | Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion | % Work Time Missed Due to Health (Absenteeism) | 3.7 Percent | Standard Deviation 7.3 |
| Patients With Fabry Disease on ERT | Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion | % Impairment While Working Due to Health (Presenteeism) | 22.6 Percent | Standard Deviation 24.9 |
| Patients With Fabry Disease on ERT | Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion | % Overall Work Impairment Due to Health | 25.7 Percent | Standard Deviation 26.4 |
| Patients With Fabry Disease on ERT | Patients Levels of Work Impairment (Work Productivity and Activity Index [WPAI] Scores) - Day of Next ERT Infusion | % Activity Impairment Due to Health | 32.3 Percent | Standard Deviation 30.4 |
Secondary
Percentage of Caregivers With Work Absence Due to Accompanying the Patient for ERT Administration.
Percentage of caregivers with work absence due to accompanying the patient for this episode of ERT administration.
Time frame: up to 7 weeks
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Patients With Fabry Disease on ERT | Percentage of Caregivers With Work Absence Due to Accompanying the Patient for ERT Administration. | 3 Participants |
Secondary
Percentage of Patients With Work Absence Due to Attendance for This ERT Episode.
The percentage of patients known to have taken time off from work during their ERT episode
Time frame: up to 7 weeks
Population: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately. Note: data were missing for 54% \[41/76\] of patients.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Patients With Fabry Disease on ERT | Percentage of Patients With Work Absence Due to Attendance for This ERT Episode. | 16 Participants |
Secondary
Total Caregiver Time Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT
Total caregiver time (in minutes) associated with accompanying a patient with Fabry Disease for the administration of a single dose of ERT (with agalsidase alfa or agalsidase beta).
Time frame: up to 7 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Patients With Fabry Disease on ERT | Total Caregiver Time Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT | 160 minutes | Standard Deviation 70.43 |
Secondary
Total Costs Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT
Total costs associated with accompanying a patient with Fabry Disease for the administration of a single dose of ERT (with agalsidase alfa or agalsidase beta).
Time frame: up to 7 weeks
Population: Data presented are from Taiwan in New Taiwan dollars (NT$). No data were reported for the 1 caregiver in Turkey for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Patients With Fabry Disease on ERT | Total Costs Associated With Accompanying a Patient With Fabry Disease for the Administration of a Single Dose of ERT | 2085 NT$ | Standard Deviation 1728.6 |
Secondary
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Brazil.
Total out-of-pocket costs (eg, food, travel, internet) incurred by patients in Brazil that were directly related to attendance for the administration of a single dose of ERT.
Time frame: up to 7 weeks
Population: Patients with Fabry Disease in Brazil. As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately. Data were not available for 2 patients.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Patients With Fabry Disease on ERT | Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Brazil. | 166.5 R$ | Standard Deviation 214.8 |
Secondary
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Japan.
Total out-of-pocket costs (eg, food, travel, internet) incurred by patients in Japan that were directly related to attendance for the administration of a single dose of ERT.
Time frame: up to 7 weeks
Population: Patients with Fabry Disease in Japan. As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Patients With Fabry Disease on ERT | Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Japan. | 8781.0 ¥ | Standard Deviation 15956.6 |
Secondary
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Taiwan.
Total out-of-pocket costs (eg, food, travel, internet) incurred by patients in Taiwan that were directly related to attendance for the administration of a single dose of ERT.
Time frame: up to 7 weeks
Population: Patients with Fabry Disease in Taiwan. As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately. Data were not available for 1 patient.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Patients With Fabry Disease on ERT | Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Taiwan. | 709.5 NT$ | Standard Deviation 855.9 |
Secondary
Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Turkey
Total out-of-pocket costs (eg, food, travel, internet) incurred by patients in Turkey that were directly related to attendance for the administration of a single dose of ERT.
Time frame: up to 7 weeks
Population: Patients with Fabry Disease in Turkey. As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately. Data were not available for 1 patient.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Patients With Fabry Disease on ERT | Total Costs Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta) - Turkey | 470.8 ₺ | Standard Deviation 1005.5 |
Secondary
Total Patient Time Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta).
Total patient time associated with attendance for administration of a single dose of ERT includes the total time from when the patient left home, to when they returned home and based on the reported time spent travelling, total waiting time, the total time spent during infusion episode (pre, during and post) and the other recorded activities.
Time frame: up to 7 weeks
Population: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately. Data were not available for 1 patient.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Patients With Fabry Disease on ERT | Total Patient Time Associated With Attendance for the Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta). | 364.1 minutes | Standard Deviation 193.9 |
Secondary
Total Time Spent by HCPs on Each Separate Task Associated With the Preparation and Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta).
Total time spent (in minutes) by healthcare professionals (HCP) on tasks associated with preparation and administration of a single dose of ERT (with agalsidase alfa or agalsidase beta) in patients with Fabry Disease including pre-administration activities (patient consultation, infusion/pre-medication preparation activities), administration of ERT (and pre-medications), and post-infusion activities (monitoring of patient during and after administration, completion of clinical documentation, and any other ERT-administration related activities).
Time frame: up to 7 weeks
Population: As was pre-specified in the study protocol, the Agalsidase Alfa and Agalsidase Beta arms are reported as a combined group (Patients with Fabry Disease on ERT) and not separately.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patients With Fabry Disease on ERT | Total Time Spent by HCPs on Each Separate Task Associated With the Preparation and Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta). | Time Spent by HCP on Pre-infusion activities | 20.9 minutes | Standard Deviation 13.4 |
| Patients With Fabry Disease on ERT | Total Time Spent by HCPs on Each Separate Task Associated With the Preparation and Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta). | Time spent by HCP on ERT administration | 118.2 minutes | Standard Deviation 56.3 |
| Patients With Fabry Disease on ERT | Total Time Spent by HCPs on Each Separate Task Associated With the Preparation and Administration of a Single Dose of ERT (With Agalsidase Alfa or Agalsidase Beta). | Time spent by HCP on post-infusion activities | 12.8 minutes | Standard Deviation 9.6 |