Efficacy and safety of ripretinib in the treatment of advanced gastrointestinal stromal tumors:a multicenter retrospective study

DU Yuqiang; TIAN Hongkun; YE Shengwei; FENG Yongdong; JIANG Qi; LIU Weizhen; ZENG Xiangyu; TAO Kaixiong; ZHANG Peng

doi:10.3969/j.issn.1009-0460.2023.10.003

Abstract：Objective To investigate the efficacy and safety of ripretinib in the treatment of recurrent metastatic or unresectable gastrointestinal stromal tumor(GIST).Methods Clinical data of advanced GIST patients treated with ripretinib from three hospitals in Hubei Province from April 2021 to January 2023 were retrospectively collected,including lines of treatment,efficacy,drug discontinuation,and incidence of adverse events.Results A total of 29 advanced GIST patients were included.The median age at the time of ripretinib administration was 60(28-85)years.Ripretinib was used as a fourth-line treatment in 15 cases,third-line treatment in 11 cases,and second-line treatment in 3 cases.In the first evaluation after ripretinib treatment in 29 advanced GIST patients,partial response(PR)was observed in 5 cases,stable disease(SD)in 16 cases,and progressive disease(PD)in 8 cases.The objective response rate(ORR)was 17.2%,and the disease control rate(DCR)was 72.4%.Adverse events were observed in 25 patients,including grade 1-2 adverse events in 23 cases(79.3%)and grade 3 adverse events in 2 cases(6.9%).By the last follow-up,14 out of 29 patients had died.The 1-year overall survival rate(OS)was 56.4%,and the 1-year cancer-specificsurvival rate(CSS)was 62.1%for the entire group of patients.11 patients discontinued ripretinib treatment,with 1 patient due to gastrointestinal bleeding,and 10 patients due to financialreasons,among which 4 cases experienced disease progression,and the doctor recommended increasing the dose of ripretinib,but the patients chose to discontinue ripretinib due to economic burden.Compared to patients who discontinued ripretinib,those who continued treatment had a significantly longer 1-year OS rate(69.6%vs.36.4%,P＜0.01)and 1-year CSS rate(80.4%vs.36.4%,P＜0.01).Conclusion Ripretinib can effectively control the disease progression of advanced GIST in patients who have failed first-line or multiple-line treatments,with mild adverse events and good tolerability.Compared to patients who discontinued ripretinib,those who continued treatment had more significant survival benefits.