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

Amicus Therapeutics France SAS69 enrolled

Overview

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.

Prospective, longitudinal, non-comparative, open-label, multicentre, non interventional cohort study.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Fabry Disease Anderson Fabry Disease

Interventions

Patients will be clustered before and after a principal components analysis (PCA) on Patients Needs Questionnaire (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.

Eligibility

Sex: ALLMin age: 16 Years

Medical Language ↔ Plain English

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)

Locations (15)

CHU d'Angers

Angers, France

CHU Pellegrin

Bordeaux, France

Hôpital Côte de Nacre

Caen, France

CHU de Dijon

Dijon, France

CHRU de Lille

Lille, France

Hôpital Femme Mère Enfant

Lyon, France

Hôpital de la Conception

Marseille, France

CHU de Nantes

Nantes, France

Hopital Tenon

Paris, France

Groupe Hospitalier Diaconesses Croix Saint-Simon

Paris, France

...and 5 more locations

Outcomes

Primary Outcomes

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.

Time frame: Baseline

Treatment benefit, derived from Patients Benefit Index (PBI), 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.

Time frame: 12 month