Nabizadeh Azita, Amani Bahman, Kadivar Maliheh, Toroski Mahdi, Asl Akbar Abdollahi, Bayazidi Yahya, Mojahedian Mahdi, Davari Majid
Department of Pharmacoeconomics and Pharmaceutical Administration, Tehran University of Medical Sciences, Tehran, Iran.
Department of Health Science Education Development, Tehran University of Medical Sciences, Tehran, Iran.
J Res Pharm Pract. 2018 Oct-Dec;7(4):171-177. doi: 10.4103/jrpp.JRPP_18_24.
Gaucher's disease (GD) is one of the most common lysosomal diseases in humans. It results from β-glucosidase deficiency and leads to necrosis, especially in macrophages with the accumulation of glucosylceramidase in cells. Most of the deleterious effects of the disease are seen in the liver, spleen, and bone marrow. The aim of this study was to compare the efficacy of Imiglucerase with Eliglustat in treating patients with GD. PubMed/Medline, Cochrane Library, Scopus, Web of Science, Embase, and Google Scholar were searched from inception to August, 2018. Predefined inclusion criteria for included studies were based on search methodology and are as follows: All randomized, quasi-randomized controlled, and cohort studies about patients with GD Type 1 that Imiglucerase was compared with Eliglustat were included. Two authors independently choose the papers based on the inclusion criteria. From 2979 recognized studies, three studies including two randomized clinical trials and one cohort study were recognized to meet the inclusion criteria. The primary outcomes were hemoglobin level, platelets count, liver, and spleen size, and the secondary outcomes were the immunological side effects of the medicines and bone complications. The results showed that there is no meaningful difference between the two medicines in terms of increasing blood hemoglobin, platelets count, and reducing the liver and spleen size. The findings of this review showed that both medicines are effective in the treatment of GD Type 1 and there is no statistically significant difference between their efficacies.
戈谢病(GD)是人类最常见的溶酶体疾病之一。它由β-葡萄糖苷酶缺乏引起,会导致坏死,尤其是细胞中葡糖神经酰胺蓄积的巨噬细胞坏死。该疾病的大多数有害影响见于肝脏、脾脏和骨髓。本研究的目的是比较伊米苷酶与依利格鲁司特治疗戈谢病患者的疗效。检索了PubMed/Medline、Cochrane图书馆、Scopus、科学网、Embase和谷歌学术,检索时间从建库至2018年8月。纳入研究的预定义纳入标准基于检索方法,如下:所有关于1型戈谢病患者且将伊米苷酶与依利格鲁司特进行比较的随机、半随机对照和队列研究均纳入。两名作者根据纳入标准独立选择论文。从2979项已识别的研究中,三项研究(包括两项随机临床试验和一项队列研究)被认定符合纳入标准。主要结局为血红蛋白水平、血小板计数、肝脏和脾脏大小,次要结局为药物的免疫副作用和骨骼并发症。结果显示,两种药物在提高血血红蛋白、血小板计数以及减小肝脏和脾脏大小方面无显著差异。本综述的结果表明,两种药物对1型戈谢病均有效,且它们的疗效无统计学显著差异。