Enzyme replacement therapy for Fabry disease: A systematic review and meta-analysis.
Enzyme replacement therapy for Fabry disease: A systematic review and meta-analysis.
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Fabry病的酶替代疗法：系统评价和荟萃分析。
DOI：
10.1590/s1415-47572012000600009
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发表时间：
2012-12
影响因子：
2.1
通讯作者：
Schwartz IV
中科院分区：
文献类型：
--
作者：
Alegra T;Vairo F;de Souza MV;Krug BC;Schwartz IV
法布里病（FD）的特异性治疗方法是使用半乳糖苷酶α或β的酶替代疗法（ERT）。进行了一项系统性综述和荟萃分析，以评估ERT治疗FD的疗效和安全性。仅纳入了比较半乳糖苷酶α或β与安慰剂的双盲、随机临床试验（RCT）。出于分析目的，认为ERT与半乳糖苷酶α或β相似。确定了10项RCT，这些RCT显示神经性疼痛、心脏异常和神经酰胺三己糖苷（GL-3）水平有所改善。一项荟萃分析显示，发热、寒战、产生半乳糖苷酶IgG抗体的几率增加，与高血压的发生或心电图QRS波群持续时间缩短无显著相关性。本比较中纳入的RCT招募了少数患者，异质性很高，主要关注替代终点，限制了关于ERT治疗FD的真实的效果的任何结论。现有证据表明，不同患者亚组对ERT的反应不同，半乳糖苷酶可能减缓FD的进展，现有变化略有改善。然而，仍然存在许多不确定性，需要进一步研究。
The specific treatment available for Fabry disease (FD) is enzyme replacement therapy (ERT) with agalsidase alfa or beta. A systematic review and meta-analysis was conducted to assess the efficacy and safety of ERT for FD. Only double-blind, randomized clinical trials (RCTs) comparing agalsidase alfa or beta and placebo were included. ERT with either agalsidase alfa or beta was considered similar for the purposes of analysis. Ten RCTs were identified, which showed improvements in neuropathic pain, in heart abnormalities and in globotriaosylceramide (GL-3) levels. A meta-analysis showed increased odds for fever, rigors, development of IgG antibodies to agalsidase, and no significant association with development of hypertension or reduction in the QRS complex duration on electrocardiogram. The RCTs included in this comparison enrolled few patients, were highly heterogeneous, and were focused mainly on surrogate endpoints, limiting any conclusions as to the real effect of ERT for FD. The available evidence suggests that response to ERT is variable across patient subgroups and that agalsidase may slow progression of FD, with slight improvement of existing changes. Nevertheless, many uncertainties remain, and further studies are necessary.
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影响因子：
3.5
作者：
Sakuraba, H;Murata-Ohsawa, M;Itoh, K
通讯作者：
Itoh, K
影响因子：
5.7
作者：
Hughes, D. A.;Elliott, P. M.;Mehta, A. B.
通讯作者：
Mehta, A. B.
影响因子：
120.7
作者：
Meikle, PJ;Hopwood, JJ;Carey, WF
通讯作者：
Carey, WF
影响因子：
2
作者：
Bradburn, Michael J.;Deeks, Jonathan J.;Localio, A. Russell
通讯作者：
Localio, A. Russell
DOI：
10.1016/0197-2456(86)90046-2
发表时间：
1986-09-01
期刊：
CONTROLLED CLINICAL TRIALS
影响因子：
--
作者：
DERSIMONIAN, R;LAIRD, N
通讯作者：
LAIRD, N