The effect of migalastat on long-term renal outcomes in enzyme replacement therapy (ERT)–naive and ERT-experienced patients with Fabry disease is not well defined. An integrated posthoc analysis of the phase 3 clinical trials and open-label extension studies was conducted to evaluate long-term changes in renal function in patients with Fabry disease and amenable GLA variants who were treated with migalastat for ≥2 years during these studies. The analysis included ERT-naive (n = 36 [23 females]; mean age 45 years; mean baseline estimated glomerular filtration rate (eGFR), 91.4 mL/min/mL/1.73 m²) and ERT-experienced (n = 42 [24 females]; mean age, 50 years; mean baseline eGFR, 89.2 mL/min/1.73m²) patients with amenable variants who received migalastat 123 mg every other day for ≥2 years. The annualized rate of change from baseline to last observation in estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation (eGFR_CKD-EPI) was calculated by both simple linear regression and a random coefficient model. In ERT-naive patients, mean annualized rates of change from baseline in eGFR_CKD-EPI were − 1.6 mL/min/1.73 m² overall and − 1.8 mL/min/1.73 m² and − 1.4 mL/min/1.73 m² in male and female patients, respectively, as estimated by simple linear regression. In ERT-experienced patients, mean annualized rates of change from baseline in eGFR_CKD-EPI were − 1.6 mL/min/1.73 m² overall and − 2.6 mL/min/1.73 m² and − 0.8 mL/min/1.73 m² in male and female patients, respectively. Mean annualized rate of change in eGFR_CKD-EPI in ERT-naive patients with the classic phenotype (defined by white blood cell alpha galactosidase A [α-Gal A] activity of <3% of normal and multiorgan system involvement) was −1.7 mL/min/1.73 m². When calculated using the random coefficient model, which adjusted for sex, age, and baseline renal function, the annualized eGFR_CKD-EPI change was minimal (mean: −0.1 and 0.1 mL/min/1.73 m² in ERT-naive and ERT-experienced patients, respectively). In conclusion, patients with Fabry disease and amenable GLA variants receiving long-term migalastat treatment (≤8.6 years) maintained renal function irrespective of treatment status, sex, or phenotype.

米加司他对未接受过酶替代疗法 (ERT) 和接受过 ERT 的法布里病患者的长期肾脏结局的影响尚未明确。对 3 期临床试验和开放标签扩展研究进行了综合事后分析，以评估在这些研究期间接受米加司他治疗 ≥ 2 年的法布里病和适合 GLA变异患者的肾功能长期变化。分析包括未接受过 ERT（n  = 36 [23 名女性]；平均年龄 45 岁；平均基线估计肾小球滤过率 (eGFR)，91.4 mL/min/mL/1.73 m ²）和经历过 ERT（n  = 42 [ 24 名女性]；平均年龄 50 岁；平均基线 eGFR，89.2 mL/min/1.73m ²) 接受米加司他 123 mg 每隔一天服用 ≥ 2 年的可接受变异的患者。通过简单线性回归和随机系数模型计算使用慢性肾脏病流行病学协作方程 (eGFR _CKD-EPI )估算的肾小球滤过率从基线到最后一次观察的年化变化率。在未接受过 ERT 治疗的患者中，eGFR _CKD-EPI相对于基线的平均年化变化率总体为 − 1.6 mL/min/1.73 m ²和 − 1.8 mL/min/1.73 m ²和 − 1.4 mL/min/1.73 m ²分别通过简单线性回归估计的男性和女性患者。_{在接受过 ERT 治疗的患者中，eGFR CKD-EPI}相对于基线的平均年化变化率在男性和女性患者中分别为 − 1.6 mL/min/1.73 m ²和 − 2.6 mL/min/1.73 m ²和 − 0.8 mL/min/1.73 m ²。具有经典表型（定义为白细胞 α 半乳糖苷酶 A [α-Gal A] 活性 < 正常和多器官系统受累的 3%）的 ERT 初治患者的 eGFR CKD-EPI 平均年化变化率为_-1.7 mL /分钟/1.73 米²。当使用针对性别、年龄和基线肾功能进行调整的随机系数模型进行计算时，年化 eGFR _CKD-EPI变化很小（平均值：-0.1 和 0.1 mL/min/1.73 m ²分别在未接受过 ERT 治疗和接受过 ERT 治疗的患者中）。总之，无论治疗状态、性别或表型如何，接受长期米加司他治疗（≤8.6 年）的法布里病和适合GLA变异的患者均能维持肾功能。