Adapt to survive and thrive: the time is now for adaptive platform trials for preterm birth

IF 15.5 1区 医学 Q1 PEDIATRICS Lancet Child & Adolescent Health Pub Date : 2025-02-01 DOI:10.1016/S2352-4642(24)00328-6
Prof Brett J Manley PhD , Christopher J D McKinlay PhD , Prof Katherine J Lee PhD , Prof Katie M Groom PhD , Clare L Whitehead PhD
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Abstract

In this Viewpoint, we discuss the challenges facing perinatal clinical researchers, many of which are unique to this field, and how traditional two-arm randomised trials using frequentist analysis might no longer be fit for purpose for perinatology. We propose a solution: the adoption of adaptive platform trials (APTs) with Bayesian methodology to address perinatal research questions to improve outcomes of preterm birth. APTs use a master protocol as a foundation to efficiently assess multiple interventions simultaneously for a particular disease. APTs can study these interventions in a perpetual manner, with interventions allowed to enter or leave the platform on the basis of preplanned decision algorithms. In this Viewpoint, we outline the ways in which APTs can overcome some of the issues facing perinatal clinical research, and the challenges and essential requirements for the design and implementation of perinatal APTs that should be considered.
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适应生存和发展:现在是进行早产适应性平台试验的时候了。
在本观点中,我们讨论了围产期临床研究人员面临的挑战,其中许多挑战是该领域独有的,以及使用频率分析的传统双臂随机试验如何不再适合围产期目的。我们提出了一个解决方案:采用自适应平台试验(APTs)与贝叶斯方法来解决围产期研究问题,以改善早产的结局。apt使用主协议作为基础,有效地评估同时针对特定疾病的多种干预措施。apt可以永久地研究这些干预措施,根据预先计划的决策算法,允许干预措施进入或离开平台。在本观点中,我们概述了APTs可以克服围产期临床研究面临的一些问题的方法,以及围产期APTs设计和实施应考虑的挑战和基本要求。
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来源期刊
Lancet Child & Adolescent Health
Lancet Child & Adolescent Health Psychology-Developmental and Educational Psychology
CiteScore
40.90
自引率
0.80%
发文量
381
期刊介绍: The Lancet Child & Adolescent Health, an independent journal with a global perspective and strong clinical focus, presents influential original research, authoritative reviews, and insightful opinion pieces to promote the health of children from fetal development through young adulthood. This journal invite submissions that will directly impact clinical practice or child health across the disciplines of general paediatrics, adolescent medicine, or child development, and across all paediatric subspecialties including (but not limited to) allergy and immunology, cardiology, critical care, endocrinology, fetal and neonatal medicine, gastroenterology, haematology, hepatology and nutrition, infectious diseases, neurology, oncology, psychiatry, respiratory medicine, and surgery. Content includes articles, reviews, viewpoints, clinical pictures, comments, and correspondence, along with series and commissions aimed at driving positive change in clinical practice and health policy in child and adolescent health.
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