Need for better reporting of trials with surrogate endpoints: SPIRIT|CONSORT-SURROGATE extensions.
Need for better reporting of trials with surrogate endpoints: SPIRIT|CONSORT-SURROGATE extensions.
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DOI：
10.1136/jech-2022-219294
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发表时间：
2022-06-24
影响因子：
6.3
通讯作者：
Taylor, Rod S.
中科院分区：
文献类型：
--
作者：
Ciani, Oriana;Manyara, Anthony;Taylor, Rod S.
理想情况下，卫生干预措施有效性的证据应来自随机试验，这些试验可评估参与者的相关最终结果（PRFO），如健康状况或生存。12然而，此类试验往往需要大量样本，随访时间长，资源密集，费用昂贵。替代终点或“替代终点”已被用作PRFOs的代理和预测因子，以提高试验效率。在过去的二十年中，美国和欧洲的药物许可允许在批准新疗法时使用生物标志物（客观测量的分子、组织学、放射学或生理学特征）作为替代品，例如，用于心血管死亡的收缩压和/糖化血红蛋白（HbA1c），用于艾滋病发展的HIV病毒载量和用于总生存的肿瘤反应。34然而，重要的是要认识到替代方法在更广泛的医疗保健评估环境中的应用（包括公共卫生、诊断、外科、精神卫生、初级保健、康复干预措施的试验）和所谓的中间结果（可较早测量并具有预测性的PRFO因果路径上的结果）作为替代方法的使用，例如，通过旨在改善临终关怀的干预措施将死亡率纳入临终关怀5；对心血管事件的水果和蔬菜的摄入是一种旨在提高心血管风险的行为干预。6
Evidence for the effectiveness of health interventions should ideally come from randomised trials that assess a participant relevant final outcome (PRFO), such as health status or survival. 1 2 However, such trials often require large sample sizes, long follow-up times and are resource intensive and costly. 2 Surrogate endpoints or ‘surrogates’ have been used to improve trial efficiency by acting as a proxy and predictor for PRFOs. 3 Over the last two decades, drug licensing in the USA and Europe has allowed the use of biomarkers (an objectively measured molecular, histologic, radiographic or physiologic characteristic) as surrogates in the approval of new therapies, for example, systolic blood pressure and/glycosylated haemoglobin (HbA1c) for cardiovascular death, HIV viral load for development of AIDS and tumour response for overall survival. 3 4 However, it is important to recognise the application of surrogates in the wider setting of healthcare evaluation (including trials of public health, diagnostic, surgical, mental health, primary care, rehabilitation interventions) and the use of so-called intermediate outcomes (outcome on the causal path for PRFO that can be measured earlier and are predictive) as surrogates, for example, hospice enrolment for mortality with an intervention aimed at improving end of life care 5; fruit and vegetable consumption for cardiovascular events for a behavioural intervention designed to improve cardiovascular risk. 6
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DOI：
10.1136/bmj.f457
发表时间：
2013-01-29
期刊：
BMJ (Clinical research ed.)
影响因子：
--
作者：
Ciani O;Buyse M;Garside R;Pavey T;Stein K;Sterne JA;Taylor RS
通讯作者：
Taylor RS
影响因子：
120.7
作者：
Casarett, D;Karlawish, J;Asch, DA
通讯作者：
Asch, DA
影响因子：
15.3
作者：
Moher, David;Hopewell, Sally;Altman, Douglas G.
通讯作者：
Altman, Douglas G.
影响因子：
39.2
作者：
Chan, An-Wen;Tetzlaff, Jennifer M.;Moher, David
通讯作者：
Moher, David
DOI：
10.1136/bmj.c3653
发表时间：
2010-08-18
期刊：
BMJ (Clinical research ed.)
影响因子：
--
作者：
la Cour JL;Brok J;Gøtzsche PC
通讯作者：
Gøtzsche PC