Department of Biostatistics, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, U.S.A.
Stat Med. 2014 Jan 30;33(2):181-92. doi: 10.1002/sim.5922. Epub 2013 Sep 4.
The number needed to treat is a tool often used in clinical settings to illustrate the effect of a treatment. It has been widely adopted in the communication of risks to both clinicians and non-clinicians, such as patients, who are better able to understand this measure than absolute risk or rate reductions. The concept was introduced by Laupacis, Sackett, and Roberts in 1988 for binary data, and extended to time-to-event data by Altman and Andersen in 1999. However, up to the present, there is no definition of the number needed to treat for time-to-event data with competing risks. This paper introduces such a definition using the cumulative incidence function and suggests non-parametric and semi-parametric inferential methods for right-censored time-to-event data in the presence of competing risks. The procedures are illustrated using the data from a breast cancer clinical trial.
需要治疗的人数是一种常用于临床环境的工具,用于说明治疗效果。它已被广泛应用于向临床医生和非临床医生(如患者)传达风险,因为他们比绝对风险或降低率更能理解这一指标。这一概念是由 Laupacis、Sackett 和 Roberts 于 1988 年提出的,用于二项数据,而由 Altman 和 Andersen 于 1999 年扩展到生存时间数据。然而,直到现在,对于存在竞争风险的生存时间数据,还没有需要治疗的人数的定义。本文使用累积发生率函数介绍了这样一个定义,并为存在竞争风险的右删失生存时间数据提出了非参数和半参数推断方法。这些程序使用乳腺癌临床试验的数据进行了说明。