| 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 | 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. | Posted | | Mean | Standard Deviation | minutes | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease on ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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). | 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. | Posted | | Mean | Standard Deviation | minutes | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease on ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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. | 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. | Posted | | Mean | Standard Deviation | minutes | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease on ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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. | 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. | Posted | | Mean | Standard Deviation | R$ | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease on ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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. | 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. | Posted | | Mean | Standard Deviation | NT$ | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease on ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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. | 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. | Posted | | Mean | Standard Deviation | ¥ | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease on ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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. | 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. | Posted | | Mean | Standard Deviation | ₺ | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease on ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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 | 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. | Posted | | Count of Participants | | Participants | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease on ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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 | 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. | Posted | | Mean | Standard Deviation | hours | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease on ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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). | | Posted | | Mean | Standard Deviation | minutes | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Caregiver | Caregiver of patient with Fabry Disease on ERT |
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| 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). | Data presented are from Taiwan in New Taiwan dollars (NT$). No data were reported for the 1 caregiver in Turkey for this outcome measure. | Posted | | Mean | Standard Deviation | NT$ | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Caregiver | Caregiver of patient with Fabry Disease on ERT |
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| 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. | | Posted | | Count of Participants | | Participants | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Caregiver | Caregiver of patient with Fabry Disease on ERT |
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| 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. | Data are available from 3 caregivers in Taiwan. | Posted | | Median | Full Range | hours | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Caregiver | Caregiver of patient with Fabry Disease receiving ERT |
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| 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. | 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. | Posted | | Mean | Standard Deviation | score on a scale | | HRQoL was completed once (during the ERT infusion visit) prior to leaving the clinic | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease Receiving ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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. | 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. | Posted | | Mean | Standard Deviation | score on a scale | | WHO-5 was completed once (during the ERT infusion visit) prior to leaving the clinic | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease Receiving ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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. | 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. | Posted | | Mean | Standard Deviation | score on a 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 | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease on ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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. | 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. | Posted | | Mean | Standard Deviation | Percent | | WPAI completed once by patients during the period of 1 to 7 Days After ERT Infusion | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease Receiving ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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. | 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. | Posted | | Mean | Standard Deviation | Percent | | WPAI completed once by patients on the day of the next ERT infusion (approximately 15 days after the prior infusion) | | | | ID | Title | Description |
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| OG000 | Patients With Fabry Disease Receiving ERT | Patients with Fabry Disease receiving Enzyme Replacement Therapy (ERT) (agalsidase alfa or agalsidase beta) infusion every other week |
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| 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. | | Posted | | Mean | Standard Deviation | Percent | | WPAI-CG completed once by caregivers during the period of 1 to 7 days after ERT infusion | | | | ID | Title | Description |
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| OG000 | Caregiver | Caregiver of patient with Fabry Disease on ERT |
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| 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. | | Posted | | Mean | Standard Deviation | Percent | | WPAI-CG completed once by caregivers on day of the next ERT infusion (approximately 15 days after prior infusion) | | | | ID | Title | Description |
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| OG000 | Caregiver | Caregiver of patient with Fabry Disease on ERT |
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| 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. | | Posted | | Mean | Standard Deviation | score on a scale | | up to 7 weeks | | | | ID | Title | Description |
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| OG000 | Caregiver | Caregiver of patient with Fabry Disease on ERT |
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