是否到了抵制使用 RECIST 作为罕见肿瘤试验主要终点的时候了？
Is It Time to Resist Using RECIST as a Primary Endpoint for Rare Tumor Trials?
机构信息
Division of Medical Oncology, Department of Medicine, School of Medicine, Washington University in St. Louis, St. Louis, Missouri.
Siteman Cancer Center, St. Louis, Missouri.
出版信息
Clin Cancer Res. 2024 Oct 15;30(20):4552-4553. doi: 10.1158/1078-0432.CCR-24-1628.
Epithelioid hemangioendothelioma is an ultra-rare cancer driven by YAP-CAMTA1 fusion. Based on the link of the fusion to the MEK pathway, SARC33 was performed. It is a phase 2 trial examining trametinib that missed its primary objective by RECIST but demonstrated patient-reported outcome benefits in improved pain. See related article by Schuetze et al., p. 4584.
上皮样血管内皮细胞瘤是一种由 YAP-CAMTA1 融合驱动的超罕见癌症。基于该融合与 MEK 通路的关联，进行了 SARC33 试验。这是一项检查曲美替尼的 2 期临床试验，该试验未能达到 RECIST 的主要目标，但在改善疼痛方面显示出患者报告的结局获益。见 Schuetze 等人的相关文章，第 4584 页。
Zotero 插件