阿加糖酶α酶替代疗法治疗法布里病的疗效：10年治疗后的心脏结局
Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years' treatment.
作者信息
Kampmann Christoph, Perrin Amandine, Beck Michael
机构信息
Section Head for Congenital Heart Defects, Center for Pediatric and Adolescent Medicine, University Medical Center, University of Mainz, Langenbeckstr. 1, Mainz, DE-55101, Germany.
Statistical Programmer, Rare Diseases Business Unit, Global Outcomes Research, Shire, Zug, Switzerland.
出版信息
Orphanet J Rare Dis. 2015 Sep 29;10:125. doi: 10.1186/s13023-015-0338-2.
BACKGROUND
To explore long-term effects of agalsidase alfa on Fabry disease cardiomyopathy in adults.
METHODS
Retrospective analysis of prospectively collected data at a single center in Mainz, Germany, revealed that 45 adult patients (21 men, 24 women) had received agalsidase alfa for approximately 10 years. Data were extracted for cardiac and heart failure status, echocardiographic evaluations of cardiac structure and function, and renal function at treatment start and during agalsidase alfa treatment.
RESULTS
After 10 years of agalsidase alfa treatment, heart failure classification had improved by at least 1 class in 22/42 patients, and angina scores were stable or improved in 41/42 patients. During treatment, no patients without left ventricular hypertrophy (LVH) at treatment initiation developed LVH, and no patients with LVH at treatment initiation showed a decline in left ventricular mass.
CONCLUSIONS
Approximately 10 years of agalsidase alfa treatment appeared to have beneficial effects for controlling progression and improving some symptoms of Fabry-associated cardiomyopathy.
背景
探讨阿加糖酶α对成年法布里病心肌病的长期影响。
方法
对德国美因茨一个中心前瞻性收集的数据进行回顾性分析，发现45例成年患者（21例男性，24例女性）接受阿加糖酶α治疗约10年。提取治疗开始时及阿加糖酶α治疗期间的心脏和心力衰竭状况、心脏结构和功能的超声心动图评估以及肾功能数据。
结果
阿加糖酶α治疗10年后，22/42例患者的心力衰竭分级至少改善了1级，41/42例患者的心绞痛评分稳定或改善。治疗期间，治疗开始时无左心室肥厚（LVH）的患者未发生LVH，治疗开始时患有LVH的患者左心室质量未下降。
结论
约10年的阿加糖酶α治疗似乎对控制法布里相关心肌病的进展和改善某些症状具有有益作用。
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