临床试验中的疾病进展通常通过放射学测量来定义。然而，临床进展可能对患者更有意义，即使不满足进展的放射学标准也可能发生，并且通常需要在临床实践中改变治疗。本研究的目的是确定在测试转移性实体瘤全身疗法的 III 期试验中，基于进展的试验终点中临床进展标准的使用情况。对 III 期试验的主要手稿和方案进行了审查，以确定临床事件（例如顽固性疼痛、肿瘤出血或神经系统损害）是否可能构成进展事件。单变量逻辑回归计算试验水平协变量与临床进展之间关联的比值比 (OR) 和 95% CI。总共纳入了 216 项试验，招募了 148,190 名患者，发表日期为 2006 年至 2020 年。13% 的试验 (n = 27) 的进展标准中纳入了临床状态的重大变化，最常见的是作为次要终点 (n = 22）。只有 59% 的试验 (n = 16) 报告了构成复合替代终点的不同临床进展结果。与其他疾病部位相比，泌尿生殖试验更有可能包括临床进展定义（16/33 [48%] vs. 11/183 [6%]；OR，14.72；95% CI，5.99 至 37.84；p < .0001 ）。虽然主要的肿瘤相关临床事件很少被视为疾病进展事件，但对临床进展的更多关注可能会提高转移性​​实体瘤患者替代终点的意义和临床适用性。© 2024 UICC。

Disease progression in clinical trials is commonly defined by radiologic measures. However, clinical progression may be more meaningful to patients, may occur even when radiologic criteria for progression are not met, and often requires a change in therapy in clinical practice. The objective of this study was to determine the utilization of clinical progression criteria within progression-based trial endpoints among phase III trials testing systemic therapies for metastatic solid tumors. The primary manuscripts and protocols of phase III trials were reviewed for whether clinical events, such as refractory pain, tumor bleeding, or neurologic compromise, could constitute a progression event. Univariable logistic regression computed odds ratios (OR) and 95% CI for associations between trial-level covariates and clinical progression. A total of 216 trials enrolling 148,190 patients were included, with publication dates from 2006 through 2020. A major change in clinical status was included in the progression criteria of 13% of trials (n = 27), most commonly as a secondary endpoint (n = 22). Only 59% of trials (n = 16) reported distinct clinical progression outcomes that constituted the composite surrogate endpoint. Compared with other disease sites, genitourinary trials were more likely to include clinical progression definitions (16/33 [48%] vs. 11/183 [6%]; OR, 14.72; 95% CI, 5.99 to 37.84; p < .0001). While major tumor-related clinical events were seldom considered as disease progression events, increased attention to clinical progression may improve the meaningfulness and clinical applicability of surrogate endpoints for patients with metastatic solid tumors.© 2024 UICC.