This study evaluates adherence to the oral chaperone therapy migalastat in patients with Fabry disease.
Fabry disease is a rare disease and part of the group of lysosomal storage disorders. Since 2016, chaperone therapy as a new therapeutic approach is available. This study is a prospective cohort study and observes patients under therapy with migalastat. This study is suggested to help estimating the adherence of the oral therapy. All patients in treatment with migalastat in the Fabry Center Wuerzburg (FAZiT) and selected patients of other cooperating Fabry Centers are included in this study if informed consent is provided.
Study Type
OBSERVATIONAL
Enrollment
30
Wuerzburg University Hospital
Würzburg, Bavaria, Germany
RECRUITINGAdherence to oral therapy with migalastat according to Medication Assessment Questionnaire
Pharmacological adherence according to adapted and translated 'Medication Assessment Questionnaire'.
Time frame: From date of inclusion up to104 weeks (2 years)
Quality of Life according to SF-36 and Wuerzburg pain questionnaire
Change of quality of life under migalastat therapy according to SF-36 and Wuerzburg pain questionnaire. Short Form (36), abbreviated SF-36, is a disease-specific measurement tool for increasing the health-related quality of life. The SF-36 is composed of eight scale-valued domains that correspond to the weighted sums of answers in each section. The range of values of each scale is 0-100 under the assumption that each question has the same weight. A score of 0 is the worst outcome (maximum disability) and a score of 100 is the best outcome (no disability). The eight domains of the SF-36 are vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning and mental health.
Time frame: From date of inclusion up to104 weeks (2 years)
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