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Communicating and understanding statistical measures when quantifying the between-group difference in competing risks.
International Journal of Epidemiology ( IF 7.7 ) Pub Date : 2023-09-21 , DOI: 10.1093/ije/dyad127
Hongji Wu 1 , Chengfeng Zhang 1 , Yawen Hou 2 , Zheng Chen 1
Affiliation  

Competing risks issues are common in clinical trials and epidemiological studies for patients in follow-up who may experience a variety of possible outcomes. Under such competing risks, two hazard-based statistical methods, cause-specific hazard (CSH) and subdistribution hazard (SDH), are frequently used to assess treatment effects among groups. However, the outcomes of the CSH-based and SDH-based methods have a close connection with the proportional hazards (CSH or SDH) assumption and may have an non-intuitive interpretation. Recently, restricted mean time lost (RMTL) has been used as an alternative summary measure for analysing competing risks, due to its clinical interpretability and robustness to the proportional hazards assumption. Considering the above approaches, we summarize the differences between hazard-based and RMTL-based methods from the aspects of practical interpretation, proportional hazards model assumption and the selection of restricted time points, and propose corresponding suggestions for the analysis of between-group differences under competing risks. Moreover, an R package 'cRMTL' and corresponding step-by-step guidance are available to help users for applying these approaches.

中文翻译:

在量化竞争风险的组间差异时沟通和理解统计指标。

对于可能经历各种可能结果的随访患者来说,竞争风险问题在临床试验和流行病学研究中很常见。在这种竞争风险下,两种基于危害的统计方法,即特定原因危害(CSH)和次分布危害(SDH),经常被用来评估群体间的治疗效果。然而,基于 CSH 和基于 SDH 的方法的结果与比例风险(CSH 或 SDH)假设密切相关,并且可能具有非直观的解释。最近,由于其临床可解释性和对比例风险假设的稳健性,限制平均损失时间(RMTL)已被用作分析竞争风险的替代汇总指标。考虑到上述方法,我们从实践解释、比例风险模型假设和限制时间点的选择等方面总结了基于风险的方法和基于RMTL的方法的差异,并为分析组间差异提出了相应的建议。竞争风险。此外,R 包“cRMTL”和相应的分步指南可帮助用户应用这些方法。
更新日期:2023-09-21
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