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用Vismodegib或Sonidegib持续抑制高林综合征相关的基础细胞癌症:一个案例系列

Raquel Wescott¹, Wolfram Samlowski^1,2,3

¹School of Medicine, University of Nevada, Reno, NV 89557, USA.

Current oncology (Toronto, Ont.)

|October 27, 2023

概括

像sonidegib和vismodegib这样的刺抑制剂有效治疗戈林综合征,显著减少新的基底细胞癌症. 持续治疗可以抑制瘤的进展,并改善患者的治疗结果.

科学领域:

在瘤学瘤学.
皮肤病学皮肤病学
遗传学遗传学是一个

背景情况:

无基底细胞癌综合征 (戈林综合征) 是一种遗传性疾病,其特征是多种基底细胞癌.
刺信号通路中的突变驱动了戈林综合征中的瘤发展.
维斯莫迪吉布和索尼迪吉布是向疗法,可以抑制刺途径.

研究的目的:

评估Gorlin综合征患者的sonidegib和vismodegib的疗效和毒性.
评估减少新基底细胞癌的发展和响应的持续时间.
分析治疗修改及其对耐受性和疗效的影响.

主要方法:

从2012年3月到2022年3月,对10名戈林综合征患者进行了回顾性分析,这些患者接受了sonidegib (n=6) 或vismodegib (n=4) 治疗.
在治疗前和治疗期间评估新的瘤发展.
评估治疗反应的持续时间,毒性和剂量调整的需要.

主要成果:

所有患者在接受了刺抑制剂治疗后实现了完全缓解.
观察到新的基底细胞癌症的显著减少,从每名患者的平均28.3到1.4 (p=0.0048).
随访时间中位数为30.7个月,新癌症发病的时间中位数为47.3个月. 三名患者出现了复发,六名患者需要因毒性而修改治疗.

结论:

持续的刺抑制剂治疗有效地抑制了戈林综合征中基底细胞癌的进展.
调整药物给药时间表可以改善治疗耐受性而不会影响疗效,从而可能延长反应持续时间.
刺抑制剂是治疗戈林综合征的一个有希望的治疗选择,尽管耐药性和毒性需要仔细监测.

关键词:

戈林综合征 (Gorlin综合征) 是一种在 PTCH 突变中.刺抑制剂刺抑制剂无基底细胞癌综合征孙德基比比是什么意思孙德基比在Vismodegib的位置上

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