Daletzakis Antonios, Roes Kit C B, Jonker Marianne A
Biometrics Department, Netherlands Cancer Institute, Amsterdam, The Netherlands.
Science Department IQ Health, Section Biostatistics, Research Institute for Medical Innovation, Radboud University Medical Center, Nijmegen, The Netherlands.
Pharm Stat. 2025 Jan-Feb;24(1):e2468. doi: 10.1002/pst.2468.
The duration of response (DoR) is defined as the time from the onset of response to treatment up to progression of disease or death due to any reason, whichever occurs earlier. The expected DoR could be a suitable estimand to measure the efficacy of a treatment but is in practice difficult to estimate, since patients' follow-up times are often right-censored. Instead, the restricted mean duration of response (RMDoR) is often used. The RMDoR in a time is equal to the expected DoR restricted to the interval . In this paper, we consider the behaviour of the RMDoR as a function of and its suitability as a measure to quantify the efficacy of a treatment. Besides, we focus on the estimation of the RMDoR. In oncology, the events response to treatment and progression of disease are typically detected through time-scheduled scans and are therefore interval-censored. We describe multiple estimators for the RMDoR that deal with the interval censoring in different ways and study the performance of these estimators in single arm trials and randomised controlled trials.
缓解持续时间（DoR）定义为从治疗开始出现缓解到疾病进展或因任何原因死亡的时间，以较早发生者为准。预期缓解持续时间可能是衡量治疗效果的一个合适的估计量，但在实际中难以估计，因为患者的随访时间常常是右删失的。相反，常常使用受限平均缓解持续时间（RMDoR）。在时间(t)时的RMDoR等于限制在区间([0,t])内的预期缓解持续时间。在本文中，我们考虑RMDoR作为(t)的函数的行为及其作为量化治疗效果的一种度量的适用性。此外，我们专注于RMDoR的估计。在肿瘤学中，对治疗的反应和疾病进展事件通常通过定期扫描来检测，因此是区间删失的。我们描述了多种针对RMDoR的估计方法，它们以不同方式处理区间删失，并研究这些估计方法在单臂试验和随机对照试验中的性能。
