在扩大访问计划中,ripretinib在晚期胃肠道筋瘤中的有效性和安全性:一项队列研究
Su Yin Lim1, Laura Ferro-López1, Elizabeth Barquin1
1The Royal Marsden NHS Foundation Trust, London SW3 6JJ, UK.
Cancers
|March 13, 2024
概括
利普雷提尼布在先进的胃肠道 stromal 瘤 (GIST) 患者中提供了长时间的无进展生存 (PFS) 和整体生存 (OS). 这种氨酸激酶抑制剂在英国扩展访问计划 (EAP) 中表现出良好的安全性.
科学领域:
- 在瘤学瘤学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 先进的胃肠道 stromal 瘤 (GIST) 通常会对标准疗法产生耐药性.
- 利普雷丁尼布是一种新型的氨酸激酶抑制剂,向GIST中的特定突变.
- 扩展获取计划 (EAP) 为未满足需求的患者提供获得试验药物的机会.
研究的目的:
- 在英国EAP中评估ripretinib在晚期GIST患者的疗效和安全性.
- 评估无进展生存期 (PFS),停止治疗的时间 (TTD) 和整体生存期 (OS).
- 分析治疗超出进展 (TBP) 和客观反应率 (ORR).
主要方法:
- 在2020年1月至2021年10月期间接受ripretinib治疗的45名GIST患者的回顾性评估.
- 收集的数据包括临床医生记录的评估和mRECIST 1.1.
- 生存分析使用卡普兰-梅尔法和考克斯回归.
主要成果:
- 里普利提尼布150毫克每天一次 (OD) 的PFS中位数为7.9个月,升级150毫克每天两次 (BD) 的PFS为5.4个月.
- 整体PFS和OS的中位数分别为9.7个月和14.0个月.
- 客观反应率为16.7% (OD) 和10.0% (BD);没有观察到新的安全信号.
结论:
- 在英国EAP内,ripretinib在晚期GIST患者中表现出长期的临床益处.
- 安全性概况与之前的临床试验一致.
- 利普雷丁尼布为GIST患者提供了一种有价值的治疗选择,这些患者对标准疗法有抗性.
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