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FollowME Fabry Pathfinders Registry: Renal Effectiveness in a Cohort of Patients on Migalastat Treatment for at Least Three Years
Simon McErlane
2024 ; 2024(1):
논문분류 :
춘계학술대회 초록집
Objectives: The followME Fabry Pathfinders registry (EUPAS20599) is evaluating real-world safety, effectiveness and patient-reported outcomes for patients living with Fabry disease. We present effectiveness data across categories of kidney function at enrollment in a cohort of patients who had received ≥3 years of migalastat treatment. Methods: Patients were enrolled into one of three groups: migalastat-amenable GLA variants receiving migalastat (reported here), any GLA variant receiving enzyme replacement therapy, and migalastat-amenable GLA variants not receiving Fabry disease-specific therapy (untreated). Enrolled patients were ≥16 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 patients (13.9%) 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, mean (SD) 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, 99.2% 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). One patient experienced one renal FACE for an incidence of 2.0 events per 1000 patient-years. When excluding patients with the predominantly cardiac variant p.N215S (30.4%, n=38), mean eGFR rate of change was −1.4 (4.6) mL/min/1.73 m2/year, n=81. Conclusions: These data support sustained effectiveness with migalastat, regardless of kidney function at enrollment, in an amenable real-world cohort of patients with Fabry disease.
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