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Extrapolating Survival From Randomized Clinical Trial Data—Possibilities and Caution
JAMA Cardiology ( IF 24.0 ) Pub Date : 2021-11-01 , DOI: 10.1001/jamacardio.2021.2629
Laine Elliott Thomas 1, 2, 3 , Ann Marie Navar 4, 5 , Michael J Pencina 1, 2, 3
Affiliation  

Many therapies in cardiovascular disease, including heart failure (HF), are studied in clinical trials that report hazard ratios (HRs) over a follow-up period far shorter than the anticipated treatment duration. The HR has known limitations, and alternative measures, such as restricted mean survival time, have been advocated to summarize survival over limited follow-up.1 However, in chronic diseases, such as HF, treatment effect measures, such as restricted mean survival time, that are limited to clinical trial follow-up may underestimate the gains that could accrue over a lifetime.2 If patients are expected to continue treatment indefinitely, estimates of lifetime survival benefit can help inform patient, health system, and payer decision-making.



中文翻译:

从随机临床试验数据推断生存期——可能性和注意事项

在临床试验中研究了包括心力衰竭 (HF) 在内的心血管疾病的许多疗法,这些试验报告了远短于预期治疗持续时间的随访期间的风险比 (HR)。HR 有已知的局限性,并且提倡替代措施,例如限制平均生存时间,以在有限的随访中总结生存情况。1然而,在慢性病(如 HF)中,治疗效果指标(如限制平均生存时间)仅限于临床试验随访,可能会低估可能在一生中累积的收益。2如果预计患者将无限期地继续治疗,则终生生存获益的估计有助于为患者、卫生系统和付款人的决策提供信息。

更新日期:2021-11-08
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