Yang Weili, Qian Haoran, Yang Litao, Wang Pengfei, Qian Hailong, Chu Binbin, Liu Zhuo, Sun Jingyu, Wu Dan, Sun Lifeng, Zhou Wenqiang, Hu Jingwei, Chen Xiaolei, Shou Chunhui, Ruan Lingxiang, Zhang Yunyun, Yu Jiren
Department of Gastrointestinal Surgery, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China.
Department of Gastrointestinal Surgery, Sir Run Run Shaw Hospital, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China.
Front Oncol. 2023 May 18;13:1180795. doi: 10.3389/fonc.2023.1180795. eCollection 2023.
Mutations in KIT proto-oncogene, receptor tyrosine kinase (KIT) and platelet-derived growth factor receptor-α (PDGFRA) render the available tyrosine kinase inhibitors (TKI) ineffective in treating advanced gastrointestinal stromal tumors (GIST). Ripretinib, a broad-spectrum switch-control kinase inhibitor, has shown increased efficacy and manageable safety, but real-world evidence remains scarce. This study evaluates the efficacy and safety of ripretinib among Chinese patients in a real-world setting.
Advanced GIST patients (N=23) receiving ripretinib following progression on previous lines of TKI treatment were enrolled to determine the efficacy [progression-free survival (PFS) and overall survival (OS)]. Safety was assessed by the incidence and severity of adverse events (AEs). All statistical analyses were performed using SPSS version 20.0 and a p-value of <0.05 was considered significant.
The median PFS (mPFS) of efficacy analysis set (EAS) (N=21) was 7.1 months. mPFS of patients receiving ripretinib following ≤2 lines of previous TKI treatment and ≥3 prior lines of therapy were 7.1 and 9.2 months, respectively. The median OS (mOS) was 12.0 months and shorter interval between the end of the latest TKI and ripretinib therapy was correlated with longer median PFS and OS (p=0.054 and p=0.046), respectively. Alopecia and asthenia were the most common AEs observed.
Compared to previous lines of TKI in advanced GIST patients, ripretinib showed superior efficacy with clinically manageable AEs. Real-world results are comparable to that of phase III INVICTUS study and its Chinese bridging study. Hence, ripretinib can be used for the clinical management of advanced GIST patients.
KIT原癌基因、受体酪氨酸激酶（KIT）和血小板衍生生长因子受体-α（PDGFRA）的突变使现有的酪氨酸激酶抑制剂（TKI）在治疗晚期胃肠道间质瘤（GIST）时无效。瑞派替尼是一种广谱开关控制激酶抑制剂，已显示出更高的疗效和可管理的安全性，但真实世界的证据仍然稀缺。本研究评估了瑞派替尼在真实世界中对中国患者的疗效和安全性。
纳入23例先前接受TKI治疗进展后接受瑞派替尼治疗的晚期GIST患者，以确定疗效[无进展生存期（PFS）和总生存期（OS）]。通过不良事件（AE）的发生率和严重程度评估安全性。所有统计分析均使用SPSS 20.0版进行，p值<0.05被认为具有统计学意义。
疗效分析集（EAS）（N = 21）的中位PFS（mPFS）为7.1个月。先前接受≤2线TKI治疗和≥3线先前治疗后接受瑞派替尼治疗的患者的mPFS分别为7.1个月和9.2个月。中位OS（mOS）为12.0个月，最新TKI治疗结束与瑞派替尼治疗之间的间隔时间越短，分别与更长的中位PFS和OS相关（p = 0.054和p = 0.046）。脱发和乏力是观察到的最常见的AE。
与晚期GIST患者先前使用的TKI相比，瑞派替尼显示出更高的疗效，且AE在临床上可管理。真实世界的结果与III期INVICTUS研究及其中国桥接研究的结果相当。因此，瑞派替尼可用于晚期GIST患者的临床管理。
