酶替代疗法Anderson-Fabry疾病。

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酶替代疗法Anderson-Fabry疾病。

科克伦数据库系统启2010年5月12日;(5):CD006663。cd006663.pub2 doi: 10.1002/14651858.。

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20464743 (在PubMed

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背景:Anderson-Fabry鞘糖脂代谢疾病的x染色体缺陷。进行性肾功能不全是一种发病率的主要来源,额外的并发症导致有氧运动——cerebro-vascular参与。生存是减少影响男性和女性携带者症状之一。目的:评价酶替代疗法的有效性和安全性与其他干预措施相比,安慰剂或不干预,治疗Anderson-Fabry疾病。搜索策略:我们搜索“临床试验”Cochrane图书馆、MEDLINE、EMBASE,紫丁香和囊性纤维化和遗传疾病组的先天性代谢试验注册(日期最近的搜索:07年2010年4月)。选择标准:随机对照试验的agalsidase阿尔法或β参与者被诊断为Anderson-Fabry疾病。数据收集和分析:两位作者选择的相关试验,评估方法学质量和提取数据。主要结果:五个研究比较agalsidase阿尔法或β187名参与者实现了选择标准。这两个试验比较agalsidase阿尔法安慰剂报道globotriaosylceramide浓度在血浆和组织;非标准聚合结果。一项研究报告的疼痛分数,有统计上显著的改善参与者接受治疗了三个月,平均差-2.10(95%可信区间(CI) -3.79 - -0.41); at up to five months, mean difference -1.90 (95% CI -3.65 to -0.15); and at up to six months, mean difference -2.00 (95% CI -3.66 to -0.34). There was a significant difference in pain-related quality of life at over five months and up to six months, mean difference -2.10 (95% CI -3.92 to -0.28) but not at other time-points. Neither study reported deaths.One of the three trials comparing agalsidase beta to placebo reported on globotriaosylceramide concentration in plasma and tissue and showed significant improvement: kidney, mean difference -1.70 (95% CI -2.09 to -1.31); heart, mean difference -0.90 (95% CI -1.18 to -0.62); and composite results (renal, cardiac, and cerebrovascular complications and death), mean difference -4.80 (95% CI -5.45 to -4.15). There was no significant difference between groups for death; no studies reported on pain. AUTHORS' CONCLUSIONS: Five small, poor quality randomised controlled trials provide no robust evidence for use of either agalsidase alfa and beta to treat Anderson-Fabry disease.

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药物靶点

药物	目标	类	生物	药理作用	行动
Agalsidase阿尔法	Globotriaosylceramide	集团	人类	未知的	代谢 配位体	细节
Agalsidaseβ	Globotriaosylceramide	集团	人类	是的	代谢 配位体	细节