Simon Florian, Al-Sawaf Othman, Seymour John F, Eichhorst Barbara
Faculty of Medicine and University Hospital Cologne, Department I of Internal Medicine, Center for Integrated Oncology Aachen Bonn Cologne Duesseldorf, University of Cologne, Cologne, Germany.
Department of Haematology Peter MacCallum Cancer Centre & Royal Melbourne Hospital, University of Melbourne, Melbourne, VIC, Australia.
Leukemia. 2025 Jan;39(1):25-28. doi: 10.1038/s41375-024-02483-5. Epub 2024 Dec 10.
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.
惰性成熟B细胞肿瘤是一类疾病，近期的治疗进展已使总体生存期（OS）延长至数年以上。虽然这值得患者和护理人员庆祝，但为了捕捉治疗效果而需要的越来越长的观察期使试验设计变得复杂，并且可能阻碍更快地获得更有效的治疗方法。替代终点是一种有可能实现更早研究结果的工具，然而，其有效性需要在每种疾病和治疗环境中得到证明。本文从这一角度讨论了成熟B细胞肿瘤替代终点的验证及现有数据，随后展望了可测量残留病评估等精确工具作为新型替代候选指标的潜力。
