Leukemia ( IF 12.8 ) Pub Date : 2024-12-10 , DOI: 10.1038/s41375-024-02483-5 Florian Simon, Othman Al-Sawaf, John F. Seymour, Barbara Eichhorst
Indolent mature B-cell neoplasms are a group of diseases in which recent therapeutic advances have led to an improved overall survival (OS) extending beyond several years. While cause of celebration for patients and caregivers, the increasingly long observation periods necessary to capture treatment effects are complicating trial design and possibly hindering swift access to more effective therapies. Surrogate endpoints are a tool with the potential of earlier study readouts, however, their validity needs to be proven in each individual disease and therapeutic setting. The validation of surrogate endpoints and available data for mature B-cell neoplasms are discussed within this perspective article, followed by an outlook on the potential of precise tools such as measurable residual disease assessment as novel surrogate candidates.
中文翻译:
成熟 B 细胞肿瘤中的替代终点 – 有意义还是误导?
惰性成熟 B 细胞肿瘤是一组疾病,其中最近的治疗进展导致总生存期 (OS) 改善,持续时间超过数年。虽然患者和护理人员值得庆祝,但捕捉治疗效果所需的观察期越来越长,这使得试验设计复杂化,并可能阻碍快速获得更有效的疗法。替代终点是一种具有早期研究读数潜力的工具,但是,它们的有效性需要在每种疾病和治疗环境中得到证明。这篇前瞻性文章讨论了成熟 B 细胞肿瘤的替代终点和可用数据的验证,然后展望了精确工具(例如可测量的残留病评估)作为新型替代候选者的潜力。