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Large-Scale, Multicenter, Prospective Registry Study of Ripretinib in Advanced GIST: A Real-World Study from China
Xinhua Zhang1, Peng Zhang2, Haibo Qiu3
1The First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.
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.
Insights
Ripretinib shows effectiveness and a tolerable safety profile for advanced gastrointestinal stromal tumor (GIST) patients in China. It is a viable fourth-line treatment option, particularly for non-gastric GIST.
Area of Science:
- Oncology
- Medical Research
- Pharmacology
Background:
- Tyrosine-kinase inhibitors (TKIs) are standard for advanced gastrointestinal stromal tumor (GIST).
- Secondary mutations can cause disease progression despite TKI treatment.
- Ripretinib is a novel TKI targeting various drug-resistant mutations.
Purpose of the Study:
- To evaluate the real-world effectiveness and safety of ripretinib.
- To assess ripretinib as a fourth-line treatment for advanced GIST in Chinese patients.
Main Methods:
- Prospective, large-scale, real-world registry study.
- Enrolled patients aged ≥18 years with recurrent/metastatic GIST.
- Key endpoints: median progression-free survival (mPFS), median overall survival (mOS), and adverse events (AEs).
Main Results:
- 240 patients enrolled; median follow-up 6.5 months.
- mPFS was 7.70 months; mOS was not reached.
- Disease control rate 73%, tumor shrinkage 43%. Ripretinib effective across gene mutation subgroups.
- Tolerable toxicities: alopecia (17.1%), hand-foot syndrome (15.4%).
- ECOG status and non-gastric GIST associated with PFS.
Conclusions:
- Ripretinib demonstrated effectiveness and a tolerable safety profile in Chinese advanced GIST patients.
- Ripretinib is a viable fourth- or later-line treatment option.
- Non-gastric GISTs showed superior benefits.
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