"Large-Scale, Multicenter, Prospective Registry Study of Ripretinib in Advanced GIST: A Real-World Study from China"

作者全名："Zhang, Xinhua; Zhang, Peng; Qiu, Haibo; Fang, Yong; Liu, Heli; Zhou, Yongjian; Xu, Hao; Yu, JiRen; Zhang, Jun; Wang, Ming; Shen, Lin; Li, Jian"

作者地址："[Zhang, Xinhua] Sun Yat Sen Univ, Affiliated Hosp 1, Guangzhou, Peoples R China; [Zhang, Peng] Huazhong Univ Sci & Technol, Union Hosp, Tongji Med Coll, Wuhan, Peoples R China; [Qiu, Haibo] Sun Yat Sen Univ, Canc Ctr, Guangzhou, Peoples R China; [Fang, Yong] Fudan Univ, Zhongshan Hosp, Shanghai, Peoples R China; [Liu, Heli] Cent South Univ, Xiangya Hosp, Changsha, Peoples R China; [Zhou, Yongjian] Fujian Med Univ, Union Hosp, Fuzhou, Peoples R China; [Xu, Hao] Nanjing Med Univ, Affiliated Hosp 1, Nanjing, Peoples R China; [Yu, JiRen] Zhejiang Univ, Affiliated Hosp 1, Sch Med, Hangzhou, Peoples R China; [Zhang, Jun] Chongqing Med Univ, Affiliated Hosp 1, Chongqing, Peoples R China; [Wang, Ming] Shanghai Jiao Tong Univ, Renji Hosp, Sch Med, Shanghai, Peoples R China; [Shen, Lin; Li, Jian] Peking Univ Canc Hosp & Inst, 52 Fucheng Rd, Beijing, Peoples R China"

通信作者："Li, J (通讯作者)，Peking Univ Canc Hosp & Inst, 52 Fucheng Rd, Beijing, Peoples R China."

来源：ADVANCES IN THERAPY

ESI学科分类：PHARMACOLOGY & TOXICOLOGY

WOS号：WOS:001016035800002

JCR分区：Q2

影响因子：3.4

年份：2023

卷号：40

期号：9

开始页：3817

结束页：3829

文献类型：Article

关键词：GIST; Ripretinib; Real-world study; Predictive factors

摘要："IntroductionTyrosine-kinase inhibitors (TKIs) have become the standard treatment for patients with advanced gastrointestinal stromal tumor (GIST); however, secondary mutations can still drive disease progression. Studies have shown that ripretinib, a novel switch-control TKI, inhibits various primary and secondary drug-resistant mutations. There is a paucity of data on the effectiveness and safety of ripretinib in a real-world setting. This prospective, large-scale, real-world registry study aimed to evaluate the effectiveness and safety of ripretinib as a fourth-line treatment in Chinese patients with advanced GIST.MethodsPatients & GE; 18 years of age having recurrent/metastatic GIST were enrolled. Key endpoints were median progression-free survival (mPFS), median overall survival (mOS), and adverse events (AEs) incidence. Univariate and multivariate analyses were conducted to identify various parameters associated with PFS.ResultsA total of 240 patients were enrolled. After a median follow-up period of 6.5 months, the mPFS [95% confidence interval (CI)] was 7.70 (6.60, 8.60) months and the mOS was not reached. Multivariate analysis revealed association of Eastern Cooperative Oncology Group (ECOG) performance status score with PFS and superior benefits for non-gastric was observed as compared to gastric GISTs [hazard ratio (HR) 0.58, 95% CI (0.39-0.86)]. Disease control rate and tumor shrinkage (any magnitude) was 73% and 43%, respectively. Ripretinib was also effective in the subgroup of patients with different gene mutations. The toxicities were tolerable, and most reported AEs were alopecia (17.1%) and hand-foot syndrome (15.4%).ConclusionRipretinib demonstrated effectiveness and a tolerable safety profile, making it a viable option as a fourth- or later-line treatment in Chinese patients with advanced GISTs, especially for non-gastric GISTs."

基金机构：China Primary Health Care Foundation

基金资助正文：No funding or sponsorship was received for this study. The Rapid Service and Open Access Fees were funded by The China Primary Health Care Foundation.