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用于乳糜泻的新型药物。

Emerging drugs for celiac disease.

作者信息

Freeman Hugh James

机构信息

University of British Columbia, Department of Medicine (Gastroenterology) , Vancouver, BC , Canada

出版信息

Expert Opin Emerg Drugs. 2015 Mar;20(1):129-35. doi: 10.1517/14728214.2015.985204. Epub 2014 Dec 13.

Abstract

INTRODUCTION

Celiac disease is an immune-mediated gluten-dependent disorder, primarily affecting the small intestine in genetically predisposed individuals. The disorder has a very heterogeneous clinical and histopathological spectrum. Current treatment with a gluten-free diet is very effective, but the diet is difficult to maintain and remains costly.

AREAS COVERED

Alternatives to the gluten-free diet have been proposed to either replace this current treatment, or at least, to supplement use of the gluten-free diet. Studies in the published English language literature relevant to this review were examined for this report.

EXPERT OPINION

Most recent published double-blind, placebo-controlled clinical trials have focused on an orally administered recombinant glutenase (ALV003) showing significant but limited benefit to celiac disease patients already compliant with a gluten-free diet. Other studies have addressed other immune mechanisms that may play a role in its pathogenesis and have not been so positive. Added investigations, particularly over the long-term, in other larger and more heterogeneous populations are needed.

摘要

引言

乳糜泻是一种免疫介导的麸质依赖性疾病,主要影响具有遗传易感性个体的小肠。该疾病具有非常异质性的临床和组织病理学谱。目前采用无麸质饮食的治疗方法非常有效,但这种饮食难以维持且成本高昂。

涵盖领域

已提出无麸质饮食的替代方案,以取代当前的这种治疗方法,或者至少补充无麸质饮食的使用。本报告对已发表的与本综述相关的英文文献中的研究进行了审查。

专家意见

最近发表的双盲、安慰剂对照临床试验主要集中在口服重组麸质酶(ALV003)上,该酶对已经遵循无麸质饮食的乳糜泻患者显示出显著但有限的益处。其他研究探讨了可能在其发病机制中起作用的其他免疫机制,但结果并不那么乐观。需要在其他更大且更具异质性的人群中进行更多研究,尤其是长期研究。

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