PMID- 37356078
OWN - NLM
STAT- MEDLINE
DCOM- 20230816
LR  - 20231007
IS  - 1865-8652 (Electronic)
IS  - 0741-238X (Print)
IS  - 0741-238X (Linking)
VI  - 40
IP  - 9
DP  - 2023 Sep
TI  - Large-Scale, Multicenter, Prospective Registry Study of Ripretinib in Advanced 
      GIST: A Real-World Study from China.
PG  - 3817-3829
LID - 10.1007/s12325-023-02576-0 [doi]
AB  - INTRODUCTION: Tyrosine-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. METHODS: Patients >/= 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. RESULTS: A 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%). CONCLUSION: Ripretinib 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. TRIAL REGISTRATION: ClinicalTrials.gov identifier, NCT05697107.
CI  - (c) 2023. The Author(s).
FAU - Zhang, Xinhua
AU  - Zhang X
AD  - The First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.
FAU - Zhang, Peng
AU  - Zhang P
AD  - Union Hospital Affiliated to Tongji Medical College, Huazhong University of 
      Science and Technology, Wuhan, China.
FAU - Qiu, Haibo
AU  - Qiu H
AD  - Sun Yat-Sen University Cancer Center, Guangzhou, China.
FAU - Fang, Yong
AU  - Fang Y
AD  - Zhongshan Hospital, Fudan University, Shanghai, China.
FAU - Liu, Heli
AU  - Liu H
AD  - Xiangya Hospital, Central South University, Changsha, China.
FAU - Zhou, Yongjian
AU  - Zhou Y
AD  - Fujian Medical University Union Hospital, Fuzhou, China.
FAU - Xu, Hao
AU  - Xu H
AD  - The First Affiliated Hospital of Nanjing Medical University, Nanjing, China.
FAU - Yu, JiRen
AU  - Yu J
AD  - The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, 
      China.
FAU - Zhang, Jun
AU  - Zhang J
AD  - The First Affiliated Hospital of Chongqing Medical University, Chongqing, China.
FAU - Wang, Ming
AU  - Wang M
AD  - Renji Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 
      China.
FAU - Shen, Lin
AU  - Shen L
AD  - Peking University Cancer Hospital and Institute, 52 Fucheng Road, Beijing, China.
FAU - Li, Jian
AU  - Li J
AD  - Peking University Cancer Hospital and Institute, 52 Fucheng Road, Beijing, China. 
      oncogene@163.com.
LA  - eng
SI  - ClinicalTrials.gov/NCT05697107
PT  - Journal Article
PT  - Multicenter Study
DEP - 20230625
PL  - United States
TA  - Adv Ther
JT  - Advances in therapy
JID - 8611864
RN  - 9XW757O13D (ripretinib)
MH  - Humans
MH  - *Gastrointestinal Stromal Tumors/drug therapy
MH  - Neoplasm Recurrence, Local
MH  - Registries
MH  - Prospective Studies
PMC - PMC10427548
OTO - NOTNLM
OT  - GIST
OT  - Predictive factors
OT  - Real-world study
OT  - Ripretinib
COIS- Xinhua Zhang, Peng Zhang, Haibo Qiu, Yong Fang, Heli Liu, Yongjian Zhou, Hao Xu, 
      JiRen Yu, Jun Zhang, Ming Wang, Lin Shen, and Jian Li have nothing to disclose.
EDAT- 2023/06/25 19:15
MHDA- 2023/08/16 06:43
PMCR- 2023/06/25
CRDT- 2023/06/25 14:47
PHST- 2023/02/20 00:00 [received]
PHST- 2023/06/02 00:00 [accepted]
PHST- 2023/08/16 06:43 [medline]
PHST- 2023/06/25 19:15 [pubmed]
PHST- 2023/06/25 14:47 [entrez]
PHST- 2023/06/25 00:00 [pmc-release]
AID - 10.1007/s12325-023-02576-0 [pii]
AID - 2576 [pii]
AID - 10.1007/s12325-023-02576-0 [doi]
PST - ppublish
SO  - Adv Ther. 2023 Sep;40(9):3817-3829. doi: 10.1007/s12325-023-02576-0. Epub 2023 
      Jun 25.