塞尔珀卡提尼布在RET突变型红细胞瘤中的临床活性
Barbara Deschler-Baier1, Bhavana Konda2, Erminia Massarelli3
1Comprehensive Cancer Center, University Hospital Würzburg, 97080 Würzburg, Germany.
The Journal of clinical endocrinology and metabolism
|April 25, 2024
概括
塞尔珀卡提尼布有效治疗有偶发性或多发性内分泌新陈代谢2型 (MEN2) 综合征的患者的RET突变性染细胞瘤. 这种RET激酶抑制剂在这些罕见的瘤中显示出显著的抗瘤活性和持久的反应.
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:
- 在各种固体瘤中发现了激活RET变化,包括色细胞瘤,与零星病例和多重内分泌新陈代谢2型 (MEN2) 综合征相关.
- 塞尔珀卡丁尼布是一种选择性的RET激酶抑制剂,在LIBRETTO-001研究中证明了对RET改变的固体瘤的疗效.
研究的目的:
- 报告用selpercatinib治疗的染细胞瘤患者的初始结果.
- 评估塞尔珀卡提尼布在特定的一组发红细胞瘤病例中的疗效和安全性.
主要方法:
- 病例系列描述了第一批6名用selpercatinib治疗的染细胞瘤患者.
- 这些患者被纳入了LIBRETTO-001临床试验 (NCT03157128).
主要成果:
- 六分之四的患者实现了部分反应或完全反应;两个患者的病情稳定.
- 治疗持续时间各不相同,一些患者接受塞尔珀卡提尼布超过56个月.
- 安全性概况与之前的塞尔珀卡提尼布研究一致.
结论:
- 塞尔珀卡提尼布证明了作为RET突变细胞瘤治疗的有效性.
- 这些发现扩大了对有针对性的RET抑制有反应的RET激活瘤的范围.
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