A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

82 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-03-01

Study Completion Date

2022-05-18

Brief Summary

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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.

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Detailed Description

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Conditions

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Fabry Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

CROSS_SECTIONAL

Study Groups

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Patient with Fabry Disease on Enzyme Replacement Therapy (ERT)

Patients with Fabry Disease receiving Enzyme Replacement Therapy (agalsidase alfa or agalsidase beta)

Enzyme Replacement Therapy (ERT)

Intervention Type DRUG

ERT infusion every other week

Caregiver

Caregiver of patient with Fabry Disease on Enzyme Replacement Therapy (ERT)

No interventions assigned to this group

Interventions

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Enzyme Replacement Therapy (ERT)

ERT infusion every other week

Intervention Type DRUG

Other Intervention Names

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agalsidase alpha agalsidase beta

Eligibility Criteria

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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.

-Self-identifies as a caregiver of a patient with FD for whom written informed consent has been obtained for inclusion in the study.

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 (and/or the patient with FD whom they support or care for) is unable or unwilling to give consent for study participation.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No