Landgren Ola, Devlin Sean M
Division of Myeloma, Department of Medicine, Sylvester Myeloma Research Institute, Sylvester Comprehensive Cancer Center, University of Miami, Miami, Florida.
Memorial Sloan Kettering Cancer Center, New York, New York.
Blood Cancer Discov. 2025 Jan 8;6(1):13-22. doi: 10.1158/2643-3230.BCD-24-0292.
The acceptance of MRD-negative complete response as an endpoint that is reasonably likely to predict clinical benefit will allow for the design of streamlined clinical trials for accelerated approval, enabling significantly faster patient access to novel therapies. Cooperative efforts were required to obtain and analyze clinical trial data from multiple sponsors and to determine the best approach to analysis with a relatively limited number of available datasets. The process to evaluate MRD as an intermediate endpoint, undertaken jointly by myeloma researchers and industry, with feedback from the FDA, serves as a roadmap for other areas of oncology to develop intermediate endpoints.
将微小残留病阴性完全缓解作为一个合理可能预测临床获益的终点来接受，将有助于设计简化的临床试验以加速批准，从而使患者能显著更快地获得新型疗法。需要各方合作努力，以获取和分析来自多个申办方的临床试验数据，并在可用数据集相对有限的情况下确定最佳分析方法。骨髓瘤研究人员和行业共同开展的、并获得美国食品药品监督管理局反馈的将微小残留病评估为中间终点的过程，为肿瘤学其他领域开发中间终点提供了路线图。
