Department of Endocrinology and Clinical Nutrition, University Hospital Zurich, Zurich, Switzerland; Department of Internal Medicine, Psychiatric Hospital of the University of Zurich, Zurich, Switzerland.
Takeda Pharmaceuticals International AG, Zurich, Switzerland.
Mol Genet Metab. 2024 Sep-Oct;143(1-2):108561. doi: 10.1016/j.ymgme.2024.108561. Epub 2024 Aug 3.
Treatment with agalsidase alfa in patients with Fabry disease is most effective when initiated early in the disease course; however, the clinical benefits in elderly patients are less well established. This analysis assesses outcomes in patients aged 65 years or older from the Fabry Outcome Survey (FOS) who were treated with agalsidase alfa.
FOS data were extracted for adult patients aged 65 years or older who received agalsidase alfa, had baseline data and at least 3 years of post-baseline data, and had undergone no renal transplantation and/or dialysis before treatment. The data of patients who had undergone renal transplantation and/or dialysis during follow-up were excluded from estimated glomerular filtration rate (eGFR) analysis after the date of the renal transplantation and/or dialysis. Adult patients were stratified into two groups: those who started treatment before 65 years of age and who were still being treated when aged 65 years or older (group A), and those who started treatment when aged 65 years or older (group B). Mean annual changes in left ventricular mass index (LVMI), eGFR and proteinuria were assessed in group A (before and after the age of 65 years to understand if there was an age-related effect once patients turned 65 years of age) and in group B.
Estimated mean (standard error [SE]) annual changes in LVMI were 0.46 (0.26) g/m and 0.21 (0.42) g/m in patients in group A when they were younger than 65 years and when they were aged 65 years or older, respectively, and 0.12 (0.65) g/m in patients in group B. For eGFR, mean (SE) annual changes were 0.83 (2.12) mL/min/1.73 m and 2.64 (2.18) mL/min/1.73 m in patients in group A when they were younger than 65 years and when they were aged 65 years or older, respectively, and 2.31 (1.44) mL/min/1.73 m in patients in group B. Proteinuria remained relatively stable in both subgroups of group A (before and after the age of 65 years) and group B.
Continuation and initiation of agalsidase alfa treatment in patients aged 65 years or older with Fabry disease were associated with stabilization of proteinuria and minimal increases in cardiac (LVMI) and renal (eGFR) outcomes.
法布里病患者尽早接受阿加糖酶阿尔法治疗效果最佳；然而，老年患者的临床获益尚不明确。本分析评估了接受阿加糖酶阿尔法治疗的法布里病患者中年龄在 65 岁及以上患者的治疗结果。
提取了年龄在 65 岁及以上、接受过阿加糖酶阿尔法治疗、有基线数据且至少有 3 年随访数据、未接受过肾移植和/或透析的成年患者的法布里结局调查（FOS）数据。在接受肾移植和/或透析后随访期间接受肾移植和/或透析的患者的数据被排除在肾小球滤过率（eGFR）分析之外，排除时间为肾移植和/或透析日期之后。将成年患者分为两组：一组为 65 岁之前开始治疗且在 65 岁及以上时仍在接受治疗的患者（A 组），另一组为 65 岁及以上开始治疗的患者（B 组）。在 A 组（65 岁之前和之后）和 B 组中评估左心室质量指数（LVMI）、eGFR 和蛋白尿的年平均变化。
A 组患者在 65 岁之前和之后的 LVMI 年平均变化分别为 0.46（0.26）g/m 和 0.21（0.42）g/m，B 组患者为 0.12（0.65）g/m。对于 eGFR，A 组患者在 65 岁之前和之后的年平均变化分别为 0.83（2.12）mL/min/1.73m 和 2.64（2.18）mL/min/1.73m，B 组患者为 2.31（1.44）mL/min/1.73m。A 组（65 岁之前和之后）和 B 组患者的蛋白尿相对稳定。
65 岁及以上法布里病患者继续或开始接受阿加糖酶阿尔法治疗与蛋白尿稳定以及心脏（LVMI）和肾脏（eGFR）结局的微小改善相关。
