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제목	FollowME Fabry Pathfinders Registry: Cardiac and Renal Effectiveness in a Multi-National, Multi-Center Cohort of Patients on Migalastat Treatment for 5 Years
저자	Haiyoung Jung
출판정보	2026; 2026(1):
키워드	Fabry disease, Migalastat, Renal, Efficacy, Registry
초록	Objectives: The ongoing, real-world followME Fabry Pathfinders registry (EUPAS20599) is designed to evaluate natural history and treatment outcomes in Fabry disease (FD). Here, we present 5-year effectiveness and safety data for migalastat in patients with FD. Methods: Data were collected and analyzed from patients ≥12 years of age with confirmed FD, migalastat-amenable GLA variants, an estimated glomerular filtration rate (eGFR) ≥30mL/min/1.73m2 and who had received migalastat for ≥4.9 years. Results: As of August 2025, 156 patients (64.1% male) were included. Median migalastat exposure was 5.7 years (range: 4.9–7.5). At enrollment, median age was 56.0 years (range: 16–77); p.N215S was the most common GLA variant (n=41; 26.3%). The Chronic Kidney Disease Epidemiology Collaboration equation was used to calculate eGFR. At baseline, 56 (35.9%) of patients had eGFR (mL/min/1.73m2) ≥90, 54 (34.6%) ≥60–<90, 10 (6.4%) ≥30–<60 and 36 (23.1%) had missing values; 39 had a proteinuria category ≥1 and 27 had missing values. Female baseline median eGFR was 90.8mL/min/1.73m2 (n=43; range: 57.7–128.1) with median annualized rate of change (ARC) −1.40mL/min/1.73m2/year (n=55; 95% confidence interval [95%CI] −2.04, −0.72); baseline median left ventricular mass index (LVMi) was 85.5g/m2 (n=35; range: 47.0–232.5) and median LVMi ARC was −0.69g/m2/year (n=46; 95%CI −2.36, 1.68). Male baseline median eGFR was 85.8mL/min/1.73m2 (n=77; range: 37.8–133.1) with median ARC −1.51mL/min/1.73m2/year (n=99; 95%CI −2.06, −0.68); baseline median LVMi was 132.3g/m2 (n=55; range: 54.3–296.2) and median LVMi ARC was −1.53g/m2/year (n=67; 95%CI −4.72, 0.40). Combined, median eGFR ARC was −1.47mL/min/1.73m2/year (n=154; 95%CI −1.99, −0.98; Figure 1) and median LVMi ARC was −0.95g/m2/year (n=113; 95%CI −2.36, 0.23; Figure 2). Two patients (1.3%) had any treatment-related treatment-emergent serious adverse event; neither discontinued migalastat. Conclusion: In this real-world observational study, migalastat preserved cardiac size and renal function over median 5.7 years with no new safety findings reported.
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