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阿加糖酶α酶替代疗法的长期疗效:使用解构复合事件进行分析。

Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events.

作者信息

Beck Michael, Hughes Derralynn, Kampmann Christoph, Pintos-Morell Guillem, Ramaswami Uma, West Michael L, Giugliani Roberto

机构信息

University Medical Center, Mainz, Germany.

Royal Free London NHS Foundation Trust, University College London, London, UK.

出版信息

Mol Genet Metab Rep. 2017 Nov 9;14:31-35. doi: 10.1016/j.ymgmr.2017.10.008. eCollection 2018 Mar.

Abstract

This is a retrospective analysis of Fabry Outcome Survey data from children/adults ( = 677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining cerebrovascular, cardiac, and renal morbidity endpoints separately. Cardiac events occurred at younger ages than cerebrovascular or renal events, cerebrovascular events were more frequent in females than males, and males were more likely to experience cardiac and renal events at a younger age than females.

摘要

这是一项对677名接受阿加糖酶α酶替代疗法、中位治疗时间为3年的儿童/成人的法布里病结果调查数据进行的回顾性分析,分别考察脑血管、心脏和肾脏发病终点。心脏事件比脑血管或肾脏事件发生的年龄更小,脑血管事件在女性中比男性更常见,并且男性比女性更有可能在更年轻的年龄发生心脏和肾脏事件。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9504/5684503/f78c1d4f19a7/gr1.jpg

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