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专家意见:在酶替代疗法(ERT)短缺期间,对法布雷病的临时治疗建议。

Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT).

机构信息

Department of Internal Medicine, Division of Endocrinology and Metabolism, Academic Medical Center, Amsterdam, The Netherlands.

出版信息

Mol Genet Metab. 2011 Jan;102(1):99-102. doi: 10.1016/j.ymgme.2010.11.155. Epub 2010 Nov 30.

Abstract

The shortage of enzyme for treatment of Fabry disease has caused anxiety among patients, physicians and governments. Following a request from the European Medicines Agency, consensus was reached on the temporary prioritization of patients for treatment based on disease severity and potential reversibility. Advice on follow-up of patients was agreed upon. This consensus is proposed to support the temporary guidelines issued throughout the period of ERT shortage, which will most likely last until April 2011.

摘要

治疗法布里病的酶短缺问题引起了患者、医生和政府的焦虑。在接到欧洲药品管理局的请求后,根据疾病严重程度和潜在可逆性,各方就临时确定治疗优先顺序达成了共识。还就患者的后续处理达成了一致意见。提出该共识是为了支持 ERT 短缺期间发布的临时指南,该指南很可能会持续到 2011 年 4 月。

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