Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences

Study Overview
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Brief Summary
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In the shared decision-making process, patients should express their expectations and preferences regarding treatment to the physician. A specific questionnaire addressing needs and expectations of Fabry patients has been built at the initiative of Amicus. In addition, this questionnaire also evaluates the benefit of treatment from the patient's perspective. Nothing is known until now on patient's expectations, potential clustering of patients regarding their expectations and evaluation of treatment benefit from the patients perspective. Study objectives are differentiated according to the study phase (inclusion and follow-up). At inclusion, the primary objective is to cluster patients according to their needs and expectations regarding treatment. During follow-up, the primary objective is to evaluate treatment benefit in relation with patients needs and expectations.

Detailed Description
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Prospective, longitudinal, non-comparative, open-label, multicentre, non interventional cohort study. Decision to treat the patient should be made independently of the patient's participation in the study.

Official Title
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Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences (SATIS-Fab)

Conditions
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Fabry Disease, Anderson Fabry Disease

Intervention / Treatment
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* Behavioral: Characterization of patients expectations and preferences regarding their treatment


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients ≥ 16 years old Diagnosed with Fabry disease With amenable mutation Decision by clinician to start or pursue ongoing ERT or migalastat Non-opposition form to participate in the study signed Exclusion Criteria: - Concomitant patient participation in an interventional clinical study (Category 1 interventional research or category 2 interventional research according to the Jardé law classification in France)

Ages Eligible for Study
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Minimum Age: 16 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Whole cohort<br>The whole cohort will be divided into clusters according to patients expectations and preferences regarding their treatment | Behavioral: Characterization of patients expectations and preferences regarding their treatment<br>* Patients will be clustered before and after a principal components analysis (PCA) on PNQ. Observations will be clustered by the Ward method. The number of clusters will be chosen using the Kolinski criteria. Characteristics of patients will be described by cluster.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Homogeneous clusters of patients according to their needs and expectations towards the treatment received. | Number of clusters will be defined using a non-supervised classification method. | Baseline | 
| Evaluate treatment benefit in relation to patients needs and expectations towards their specific treatment. | Patients Benefit Index (PBI): descriptive statistics. The PBI will be calculated by reference to the PNQ measured at the preceding visit The proportion of patients with PBI ≥ 1 will be described with a two-sided 95% confidence interval. | 12 month |