塞尔珀卡提尼布在晚期RET-突变性髓性甲状腺癌中进行的3期试验
Julien Hadoux1, Rossella Elisei1, Marcia S Brose1
1From the Service d'oncologie endocrinienne, département d'imagerie, Gustave Roussy and ENDOCAN-TUTHYREF Network, Villejuif (J.H.), and the Nuclear Medicine Department and Thyroid Unit, Centre François Baclesse, Caen (S.B.) - both in France; the Endocrine Unit, Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy (R.E.); the Department of Medical Oncology, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia (M.S.B.); the Department of Endocrinology, Instituto do Câncer do Estado de São Paulo, Universidade de São Paulo, and Instituto D'Or de Pesquisa e Ensino - both in São Paulo (A.O.H.); Sydney Medical School, University of Sydney, Sydney (B.G.R.); the Department of Thyroid and Neck Tumor, Tianjin Medical University Cancer Institute & Hospital, Tianjin, China (M.G.); the Department of Nuclear Medicine and Endocrine Oncology, Maria Sklodowska Curie National Research Institute of Oncology, Gliwice Branch, Poland (B.J.); Federal State Institution Medical Radiology Research Center, Obninsk, Russia (P.I.); the Department of Oncology, 2nd Faculty of Medicine of Charles University and Motol University Hospital, Prague, Czech Republic (K.K.); the Clinical Oncology Department, Weston Park Cancer Center, NHS Foundation Trust, Sheffield, United Kingdom (J.W.); the Department of Endocrinology Diabetology and Metabolism, Endocrine Tumour Center at West German Cancer Center, University Hospital Essen, University of Duisburg-Essen, Essen, Germany (D.F.); the Department of Internal Medicine, Seoul National University Hospital, Seoul, South Korea (B.K.); the Department of Medical Oncology, Memorial Sloan Kettering Cancer Center, New York (E.J.S.); the Department of Head and Neck Medical Oncology, National Cancer Center Hospital East, Kashiwa, Japan (M.T.); the Endocrine Neoplasia and Hormonal Disorders Department, University of Texas M.D. Anderson Cancer Center, Houston (M.I.H.); Eli Lilly, Indianapolis (R.S., Y.L., V.S., J.W., B.L., P.M.); the Medical Oncology Department, Vall d'Hebron Institute of Oncology, Universitat Autònoma de Barcelona, Barcelona (J.C.); and the Cancer Center, Massachusetts General Hospital, Boston (L.J.W.).
与标准疗法相比,塞尔珀卡提尼布显著改善了晚期RET突变性髓性甲状腺癌患者的无进展生存率和无治疗失败生存率. 这种RET抑制剂为这种罕见的癌症提供了优越的治疗选择.
科学领域:
- 在瘤学瘤学.
- 遗传学 是一个遗传学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 先进的RET突变髓性甲状腺癌 (MTC) 提出了一个治疗挑战.
- 塞尔珀卡提尼布是一种选择性RET抑制剂,在MTC中已证明有效.
- 与已批准的多酶抑制剂相对比较的疗效之前尚不清楚.
研究的目的:
- 为了比较selpercatinib与cabozantinib或vandetanib作为先进的RET-突变MTC的第一线治疗的疗效.
- 评估无进展生存期 (PFS) 作为主要终点.
- 评估治疗无失败生存期 (TFFS),整体反应和安全性.
主要方法:
- 第三阶段,随机试验设计.
- 291名患有渐进式RET突变MTC的患者随机分配给塞尔珀卡提尼布或医生选择的卡博赞提尼布/万达提尼布.
- 主要终点:通过盲目独立的中央审查进行PFS. 二级终点:TFFS,整体反应,安全性.
主要成果:
- 与对照组相比,塞尔珀卡提尼布表现出优异的PFS (未达到与16.8个月相比) 和TFFS (未达到与13.9个月相比) (P<0.001两者).
- 12个月的PFS为塞尔珀卡提尼布的86.8%,对照组为65.7%.
- 塞尔珀卡提尼布的整体响应率为69.4%,对照组为38.8%,因不良事件而减少剂量减少和中止.
结论:
- 在RET突变MTC患者中,塞尔珀卡提尼布显著改善PFS和TFFS.
- 与卡博赞提尼布或万德坦提尼布相比,塞尔珀卡提尼布是一种优越的第一线治疗选择.
- 塞尔珀卡提尼布的安全性概况更为有利,剂量修改和治疗中止率较低.
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