耐药性结核病治疗,III 期平台试验的案例。

IF 8.4 2区 医学 Q1 PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH
Tom A Yates, Samara Barnes, Martin Dedicoat, Onn Min Kon, Heinke Kunst, Marc Lipman, Kerry A Millington, Andrew J Nunn, Patrick Pj Phillips, Jessica L Potter, S Bertel Squire
{"title":"耐药性结核病治疗,III 期平台试验的案例。","authors":"Tom A Yates, Samara Barnes, Martin Dedicoat, Onn Min Kon, Heinke Kunst, Marc Lipman, Kerry A Millington, Andrew J Nunn, Patrick Pj Phillips, Jessica L Potter, S Bertel Squire","doi":"10.2471/BLT.23.290948","DOIUrl":null,"url":null,"abstract":"<p><p>Most phase III trials in drug-resistant tuberculosis have either been underpowered to quantify differences in microbiological endpoints or have taken up to a decade to complete. Composite primary endpoints, dominated by differences in treatment discontinuation and regimen changes, may mask important differences in treatment failure and relapse. Although new regimens for drug-resistant tuberculosis appear very effective, resistance to new drugs is emerging rapidly. There is a need for shorter, safer and more tolerable regimens, including those active against bedaquiline-resistant tuberculosis. Transitioning from multiple regimen A versus regimen B trials to a single large phase III platform trial would accelerate the acquisition of robust estimates of relative efficacy and safety. Further efficiencies could be achieved by adopting modern adaptive platform designs. Collaboration among trialists, affected community representatives, funders and regulators is essential for developing such a phase III platform trial for drug-resistant tuberculosis treatment regimens.</p>","PeriodicalId":9465,"journal":{"name":"Bulletin of the World Health Organization","volume":null,"pages":null},"PeriodicalIF":8.4000,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362690/pdf/","citationCount":"0","resultStr":"{\"title\":\"Drug-resistant tuberculosis treatments, the case for a phase III platform trial.\",\"authors\":\"Tom A Yates, Samara Barnes, Martin Dedicoat, Onn Min Kon, Heinke Kunst, Marc Lipman, Kerry A Millington, Andrew J Nunn, Patrick Pj Phillips, Jessica L Potter, S Bertel Squire\",\"doi\":\"10.2471/BLT.23.290948\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Most phase III trials in drug-resistant tuberculosis have either been underpowered to quantify differences in microbiological endpoints or have taken up to a decade to complete. Composite primary endpoints, dominated by differences in treatment discontinuation and regimen changes, may mask important differences in treatment failure and relapse. Although new regimens for drug-resistant tuberculosis appear very effective, resistance to new drugs is emerging rapidly. There is a need for shorter, safer and more tolerable regimens, including those active against bedaquiline-resistant tuberculosis. Transitioning from multiple regimen A versus regimen B trials to a single large phase III platform trial would accelerate the acquisition of robust estimates of relative efficacy and safety. Further efficiencies could be achieved by adopting modern adaptive platform designs. Collaboration among trialists, affected community representatives, funders and regulators is essential for developing such a phase III platform trial for drug-resistant tuberculosis treatment regimens.</p>\",\"PeriodicalId\":9465,\"journal\":{\"name\":\"Bulletin of the World Health Organization\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":8.4000,\"publicationDate\":\"2024-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362690/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Bulletin of the World Health Organization\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.2471/BLT.23.290948\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Bulletin of the World Health Organization","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.2471/BLT.23.290948","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH","Score":null,"Total":0}
引用次数: 0

摘要

大多数耐药性结核病的 III 期试验要么没有足够的能力量化微生物终点的差异,要么需要长达十年的时间才能完成。以治疗中断和治疗方案改变差异为主的综合主要终点可能会掩盖治疗失败和复发方面的重要差异。尽管治疗耐药性结核病的新方案似乎非常有效,但对新药的耐药性正在迅速出现。我们需要更短、更安全、更耐受的治疗方案,包括那些对贝达喹啉耐药结核病有效的治疗方案。从多个治疗方案 A 与治疗方案 B 的对比试验过渡到单一的大型 III 期平台试验,将加快获得对相对疗效和安全性的可靠估计。采用现代适应性平台设计可进一步提高效率。试验人员、受影响社区代表、资助者和监管者之间的合作对于开发这种针对耐药结核病治疗方案的 III 期平台试验至关重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Drug-resistant tuberculosis treatments, the case for a phase III platform trial.

Most phase III trials in drug-resistant tuberculosis have either been underpowered to quantify differences in microbiological endpoints or have taken up to a decade to complete. Composite primary endpoints, dominated by differences in treatment discontinuation and regimen changes, may mask important differences in treatment failure and relapse. Although new regimens for drug-resistant tuberculosis appear very effective, resistance to new drugs is emerging rapidly. There is a need for shorter, safer and more tolerable regimens, including those active against bedaquiline-resistant tuberculosis. Transitioning from multiple regimen A versus regimen B trials to a single large phase III platform trial would accelerate the acquisition of robust estimates of relative efficacy and safety. Further efficiencies could be achieved by adopting modern adaptive platform designs. Collaboration among trialists, affected community representatives, funders and regulators is essential for developing such a phase III platform trial for drug-resistant tuberculosis treatment regimens.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
Bulletin of the World Health Organization
Bulletin of the World Health Organization 医学-公共卫生、环境卫生与职业卫生
CiteScore
11.50
自引率
0.90%
发文量
317
审稿时长
3 months
期刊介绍: The Bulletin of the World Health Organization Journal Overview: Leading public health journal Peer-reviewed monthly journal Special focus on developing countries Global scope and authority Top public and environmental health journal Impact factor of 6.818 (2018), according to Web of Science ranking Audience: Essential reading for public health decision-makers and researchers Provides blend of research, well-informed opinion, and news
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信