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工具变量法揭示了绝经激素治疗在具有里程碑意义的女性健康倡议临床试验中的更大效果。

Instrumental Variables Methods Reveal Larger Effects of Menopausal Hormone Therapy in the Landmark Women's Health Initiative Clinical Trial.

作者信息

Angrist Joshua, Kowalski Amanda E, Ristovska Ljubica, Stefanick Marcia L

机构信息

Massachusetts Institute of Technology.

University of Michigan.

出版信息

AEA Pap Proc. 2025 May;115:215-220. doi: 10.1257/pandp.20251064.

DOI:10.1257/pandp.20251064
PMID:40880581
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12383917/
Abstract

Landmark results from the Women's Health Initiative trial showed that random assignment to menopausal hormone therapy (MHT) elevated risks of breast cancer and other adverse events. Recent analyses argue that MHT risks are small. These analyses report intention-to-treat (ITT) effects, ignoring the fact that many women assigned intervention were non-adherent, while many women assigned control initiated treatment. Instrumental variable (IV) methods and adherence data allow us to estimate effects of MHT on compliers who took MHT if and only if assigned. IV estimates show risks and benefits that are substantially larger than the ITT estimates used to inform MHT guidelines.

摘要

妇女健康倡议试验的标志性结果表明,随机分配接受绝经激素治疗(MHT)会增加患乳腺癌和其他不良事件的风险。最近的分析认为MHT的风险很小。这些分析报告的是意向性治疗(ITT)效应,却忽略了一个事实,即许多被分配接受干预的女性并未坚持治疗,而许多被分配为对照组的女性却开始了治疗。工具变量(IV)方法和依从性数据使我们能够估计MHT对那些只有在被分配的情况下才会服用MHT的依从者的影响。IV估计显示的风险和益处比用于制定MHT指南的ITT估计要大得多。

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本文引用的文献

1
Menopausal Hormone Therapy Use Among Postmenopausal Women.绝经后妇女的激素治疗使用情况。
JAMA Health Forum. 2024 Sep 6;5(9):e243128. doi: 10.1001/jamahealthforum.2024.3128.
2
The Women's Health Initiative Randomized Trials and Clinical Practice: A Review.《妇女健康倡议随机试验和临床实践:综述》。
JAMA. 2024 May 28;331(20):1748-1760. doi: 10.1001/jama.2024.6542.
3
Instrumental variables methods reconcile intention-to-screen effects across pragmatic cancer screening trials.工具变量法协调了实用癌症筛查试验中的意图筛查效应。
Proc Natl Acad Sci U S A. 2023 Dec 19;120(51):e2311556120. doi: 10.1073/pnas.2311556120. Epub 2023 Dec 15.
4
Behaviour within a Clinical Trial and Implications for Mammography Guidelines.临床试验中的行为及其对乳腺钼靶检查指南的影响。
Rev Econ Stud. 2023 Jan;90(1):432-462. doi: 10.1093/restud/rdac022. Epub 2022 May 9.
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Menopausal Hormone Therapy and Long-term All-Cause and Cause-Specific Mortality: The Women's Health Initiative Randomized Trials.绝经激素治疗与全因及特定病因长期死亡率:妇女健康倡议随机试验
JAMA. 2017 Sep 12;318(10):927-938. doi: 10.1001/jama.2017.11217.
6
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JAMA. 2013 Oct 2;310(13):1353-68. doi: 10.1001/jama.2013.278040.
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8
Risks and benefits of estrogen plus progestin in healthy postmenopausal women: principal results From the Women's Health Initiative randomized controlled trial.健康绝经后妇女使用雌激素加孕激素的风险与益处:妇女健康倡议随机对照试验的主要结果
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