Abstract: SA-PO804
FollowME Fabry Pathfinders Registry: Renal Effectiveness in a Cohort of Patients on Migalastat Treatment for at Least Three Years
Session Information
- Genetic Diseases: Glomerulopathies - II
November 04, 2023 | Location: Exhibit Hall, Pennsylvania Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidneys
- 1202 Genetic Diseases of the Kidneys: Non-Cystic
Authors
- West, Michael L., Dalhousie University Faculty of Medicine, Halifax, Nova Scotia, Canada
- Hughes, Derralynn, Royal Free London NHS Foundation Trust and University College London, London, London, United Kingdom
- Sunder-Plassmann, Gere, Medizinische Universitat Wien Universitatsklinik fur Innere Medizin III, Wien, Wien, Austria
- Jovanovic, Ana, Northern Care Alliance NHS Foundation Trust, Salford, Manchester, United Kingdom
- Brand, Eva, Interdisciplinary Fabry Center Münster, University Hospital Münster, Münster, Germany
- Bichet, Daniel G., Hôpital du Sacré-Coeur, University of Montréal, Montréal, Quebec, Canada
- Pisani, Antonio, Azienda Ospedaliera Universitaria Federico II, Napoli, Campania, Italy
- Nowak, Albina, University Hospital Zurich and University of Zurich, Zurich, Switzerland
- Torra, Roser, Fundació Puigvert, Universitat Autònoma de Barcelona, Barcelona, Catalunya, Spain
- Khan, Aneal, University of Calgary Cumming School of Medicine, Calgary, Alberta, Canada
- Azevedo, Olga, Hospital Senhora da Oliveira, Guimarães, Portugal
- Lehman, Anna, The University of British Columbia, Vancouver, British Columbia, Canada
- Linhart, Ales, Univerzita Karlova, Praha, Prague, Czechia
- Rutecki, Jasmine L., Amicus Therapeutics Inc, Philadelphia, Pennsylvania, United States
- Giuliano, Joseph D., Amicus Therapeutics Inc, Philadelphia, Pennsylvania, United States
- Krusinska, Eva, Amicus Therapeutics Inc, Philadelphia, Pennsylvania, United States
Background
The followME Fabry Pathfinders registry (EUPAS20599) is evaluating real-world safety, effectiveness and patient-reported outcomes for patients with Fabry disease who were enrolled into one of three groups: migalastat-amenable GLA variants receiving migalastat, any GLA variant receiving enzyme replacement therapy and migalastat-amenable GLA variants not receiving Fabry disease-specific therapy (untreated).
Methods
We present effectiveness data across categories of kidney function at enrollment in a cohort of patients who had received ≥3 years of migalastat treatment. Enrolled patients were ≥12 years old with an estimated glomerular filtration rate (eGFR) >30 mL/min/1.73 m2.
Results
As of August 2022, 125 patients (60.0% males; median age, 58.0 years) with amenable GLA variants had a mean migalastat exposure of 3.9 years. At enrollment, mean±SD eGFR was 83.7 ± 22.5 mL/min/1.73 m2, and overall, 17 (14.7%) patients had an eGFR <60 mL/min/1.73 m2. Median urine albumin: creatinine ratio was 19.0 mg/g (range 0−1124, n=40). Mean (SD) eGFR annualized rate of change (mL/min/1.73 m2/year) in the overall cohort was −0.9 (4.9). When analyzed by eGFR category at enrollment annualized change was −1.0 (3.9) in patients with eGFR ≥90 (33.6%), −1.0 (5.9) in patients with eGFR ≥60–90 (43.2%), and −0.4 (3.5) in patients with eGFR ≥30–60 (12.8%). Overall, 94.4% of patients did not experience a renal Fabry-associated clinical event (FACE: doubling of serum creatinine level from the start of analysis [two consecutive values]; end-stage renal disease requiring long term dialysis or transplantation). Seven patients each experienced one renal FACE for an incidence of 14.2/1000 patient-years. When excluding patients with the mostly cardiac variant p.N215S (69.6%, n=87) mean eGFR rate of change was −1.4 (4.6) mL/min/1.73 m2/year, n=81) and renal FACEs incidence was 20.9/1000 patient-years.
Conclusion
These data support sustained effectiveness with migalastat, regardless of kidney function at enrollment, in an amenable real-world cohort of patients with Fabry disease.
Funding
- Commercial Support – Amicus Therapeutics