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多变量置换检验用于比较匹配数据的生存曲线。

Multivariate permutation test to compare survival curves for matched data.

机构信息

Center of Biostatistics for Clinical Epidemiology, Department of Health Sciences, University of Milano-Bicocca, 20900, Monza, Italy.

出版信息

BMC Med Res Methodol. 2013 Feb 11;13:16. doi: 10.1186/1471-2288-13-16.

DOI:10.1186/1471-2288-13-16
PMID:23399031
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3574851/
Abstract

BACKGROUND

In the absence of randomization, the comparison of an experimental treatment with respect to the standard may be done based on a matched design. When there is a limited set of cases receiving the experimental treatment, matching of a proper set of controls in a non fixed proportion is convenient.

METHODS

In order to deal with the highly stratified survival data generated by multiple matching, we extend the multivariate permutation testing approach, since standard nonparametric methods for the comparison of survival curves cannot be applied in this setting.

RESULTS

We demonstrate the validity of the proposed method with simulations, and we illustrate its application to data from an observational study for the comparison of bone marrow transplantation and chemotherapy in the treatment of paediatric leukaemia.

CONCLUSIONS

The use of the multivariate permutation testing approach is recommended in the highly stratified context of survival matched data, especially when the proportional hazards assumption does not hold.

摘要

背景

在缺乏随机化的情况下，可以基于匹配设计来比较实验治疗与标准治疗。当实验组的病例数量有限时，方便采用非固定比例的适当对照组进行匹配。

方法

为了处理由多次匹配产生的高度分层生存数据，我们扩展了多元置换检验方法，因为标准的非参数生存曲线比较方法在此情况下无法应用。

结果

我们通过模拟验证了所提出方法的有效性，并展示了其在骨髓移植和化疗治疗儿科白血病的观察性研究数据中的应用。

结论

在高度分层的生存匹配数据背景下，特别是当比例风险假设不成立时，推荐使用多元置换检验方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/918f/3574851/08ccc7f6fdeb/1471-2288-13-16-2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/918f/3574851/9b6467ce2384/1471-2288-13-16-1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/918f/3574851/08ccc7f6fdeb/1471-2288-13-16-2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/918f/3574851/9b6467ce2384/1471-2288-13-16-1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/918f/3574851/08ccc7f6fdeb/1471-2288-13-16-2.jpg

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本文引用的文献

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Biometrics. 2008 Sep;64(3):733-740. doi: 10.1111/j.1541-0420.2007.00975.x. Epub 2008 Jan 11.
2
Analyzing survival curves at a fixed point in time.在固定时间点分析生存曲线。
Stat Med. 2007 Oct 30;26(24):4505-19. doi: 10.1002/sim.2864.
3
A weighted Kaplan-Meier estimator for matched data with application to the comparison of chemotherapy and bone-marrow transplant in leukaemia.
通过整合EgoNet算法和通路分析来研究骨肉瘤中的自我模块和通路。
Braz J Med Biol Res. 2017 Feb 16;50(2):e5793. doi: 10.1590/1414-431X20165793.
4
Obesity-hypoventilation syndrome: increased risk of death over sleep apnea syndrome.肥胖低通气综合征：相较于睡眠呼吸暂停综合征，死亡风险增加。
PLoS One. 2015 Feb 11;10(2):e0117808. doi: 10.1371/journal.pone.0117808. eCollection 2015.
Stat Med. 2002 Dec 30;21(24):3847-64. doi: 10.1002/sim.1357.
4
A class of permutation tests for stratified survival data.一类用于分层生存数据的置换检验。
Biometrics. 1999 Dec;55(4):1156-61. doi: 10.1111/j.0006-341x.1999.01156.x.
5
Allogeneic bone marrow transplantation versus chemotherapy in high-risk childhood acute lymphoblastic leukaemia in first remission. Associazione Italiana di Ematologia ed Oncologia Pediatrica (AIEOP) and the Gruppo Italiano Trapianto di Midollo Osseo (GITMO).首次缓解期高危儿童急性淋巴细胞白血病的异基因骨髓移植与化疗对比研究。意大利儿科血液学与肿瘤学协会（AIEOP）及意大利骨髓移植研究组（GITMO）
Br J Haematol. 1997 Feb;96(2):387-94. doi: 10.1046/j.1365-2141.1997.d01-2033.x.
6
Some permutation tests for survival data.一些针对生存数据的排列检验。
Biometrics. 1996 Mar;52(1):87-97.