Background/Objectives: In glioblastoma trials, efficacy evaluation often deviates from the standard Response Evaluation Criteria in Solid Tumors (RECIST), an objective response rate (ORR) method, because of the unique nature of brain tumors. In phase II trials from the fiscal years (FYs) 2017–2019, primary endpoints (PEs) were overall survival (OS) at 29%, ORR at 20%, progression-free survival (PFS) at 17%, and OS rate at 10%. Clinical trial methodologies have likely evolved in recent years. This study analyzed trends in efficacy endpoint settings for phase II trials from FY2020 to FY2022 compared with FY2017–2019.Methods: Using Clarivate’s Cortellis™ Clinical Trial Intelligence database, 116 phase II glioblastoma trials initiated between April 2020 and March 2023 were identified. After exclusions, 88 trials were analyzed. Trial characteristics, PEs, secondary endpoints (SEs), and designs were summarized and compared to prior data.Results: Of 101 PEs in the 88 trials, approximately half targeted newly diagnosed patients, and most tested pharmaceutical products. The most common PEs were FS (22%), OS (20%), and PFS rate (17%), while among 299 SEs, OS (15%), PFS (15%), and quality of life (14%) were most frequent. Time-to-event outcomes were employed in 74 (73%) trials, whereas ORR was used as a PE in only 7 trials (8%). ORR as a PE was significantly lower than in FY2017–2019 (p= 0.022).Conclusions: Recent glioblastoma trials show increased diversity in efficacy endpoints with less reliance on ORR compared to earlier periods, reflecting evolving strategies to address the unique challenges of glioblastoma treatment and evaluation.
背景/目的：在胶质母细胞瘤临床试验中，由于脑肿瘤的特殊性，疗效评估常偏离实体瘤疗效评价标准（RECIST）这一客观缓解率（ORR）方法。2017-2019财年的II期试验中，主要终点（PEs）分布为总生存期（OS）占29%、ORR占20%、无进展生存期（PFS）占17%、OS率占10%。近年来临床试验方法学可能已发生演变。本研究通过对比2020-2022财年与2017-2019财年的数据，分析了II期试验疗效终点设置的趋势变化。 方法：通过科睿唯安Cortellis™临床试验情报数据库，筛选出2020年4月至2023年3月期间启动的116项胶质母细胞瘤II期试验。经排除后，最终对88项试验进行分析。总结试验特征、主要终点、次要终点（SEs）及试验设计，并与早期数据进行对比。 结果：在88项试验的101个主要终点中，约半数针对新诊断患者，且多数为药物试验。最常见的主要终点包括无进展生存期（22%）、总生存期（20%）和PFS率（17%）；而在299个次要终点中，总生存期（15%）、无进展生存期（15%）和生活质量（14%）最为常见。74项试验（73%）采用时间-事件结局作为终点，仅7项试验（8%）将ORR设为主要终点。ORR作为主要终点的使用率显著低于2017-2019财年（p=0.022）。 结论：近期胶质母细胞瘤试验显示疗效终点呈现更高多样性，对ORR的依赖较早期显著降低，这反映了为应对胶质母细胞瘤治疗与评估的特殊挑战而不断演进的策略调整。
肿瘤(癌症)患者之家