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IgA肾病:临床试验中药物治疗概述

IgA nephropathy: an overview of drug treatments in clinical trials.

作者信息

Selvaskandan Haresh, Gonzalez-Martin Guillermo, Barratt Jonathan, Cheung Chee Kay

机构信息

John Walls Renal Unit, University Hospitals Leicester NHS Trust, Leicester, UK.

Department of Cardiovascular Sciences, University of Leicester, Leicester, UK.

出版信息

Expert Opin Investig Drugs. 2022 Dec;31(12):1321-1338. doi: 10.1080/13543784.2022.2160315. Epub 2023 Jan 1.

DOI:10.1080/13543784.2022.2160315
PMID:36588457
Abstract

INTRODUCTION

IgA nephropathy (IgAN) is the commonest primary glomerulonephritis worldwide and may progress to end-stage kidney disease (ESKD) within a 10-20 year period. Its slowly progressive course has made clinical trials challenging to perform, however the acceptance of proteinuria reduction as a surrogate end point has significantly improved the feasibility of conducting clinical trials in IgAN, with several novel and repurposed therapies currently undergoing assessment. Already, interim results are demonstrating value to some of these, offering great hope to those with IgAN.

AREAS COVERED

This review explores the rationale, candidates, clinical precedents, and trial status of therapies that are currently or have recently been evaluated for efficacy in IgAN. All IgAN trials registered with the U.S. National Library of Medicine; ClinicalTrials.gov were reviewed.

EXPERT OPINION

For the first time, effective treatment options beyond supportive care are becoming available for those with IgAN. This is the culmination of commendable international efforts and signifies a new era for those with IgAN. As more therapies become available, future challenges will revolve around deciding which treatments are most appropriate for individual patients, which is likely to push IgAN into the realm of precision medicine.

摘要

引言

IgA肾病(IgAN)是全球最常见的原发性肾小球肾炎,可能在10至20年内进展为终末期肾病(ESKD)。其缓慢进展的病程使得进行临床试验具有挑战性,然而,将蛋白尿减少作为替代终点的接受度显著提高了在IgAN中开展临床试验的可行性,目前有几种新型和重新利用的疗法正在接受评估。已经有中期结果显示其中一些疗法具有价值,为IgAN患者带来了巨大希望。

涵盖领域

本综述探讨了目前或最近已评估其在IgAN中疗效的疗法的基本原理、候选药物、临床先例和试验状态。对在美国国立医学图书馆注册的所有IgAN试验;ClinicalTrials.gov进行了审查。

专家意见

对于IgAN患者来说,首次有了支持性治疗之外的有效治疗选择。这是国际上值得称赞的努力的成果,标志着IgAN患者迎来了一个新时代。随着更多疗法出现,未来的挑战将围绕确定哪些治疗最适合个体患者展开,这可能会将IgAN推向精准医学领域。

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