大多数胃肠道间质瘤 (GIST) 都存在 c-KIT 或 PDGFRA 突变。酪氨酸激酶抑制剂（TKI）的使用显着改善了 GIST 患者的生存率。我们的目的是评估台湾晚期或复发性 GIST 患者的临床结果。招募了 2010 年至 2020 年间诊断的患者。收集的数据包括基线特征、治疗模式、治疗结果、遗传畸变和生存状态。使用 Kaplan-Meier 方法分析并绘制无进展生存期 (PFS) 和总生存期 (OS)。采用Cox回归分析分析生存预后因素。共纳入224例接受TKI治疗的晚期或复发GIST患者。所有患者均接受伊马替尼治疗。 93 名患者和 42 名患者分别接受了舒尼替尼和瑞格非尼治疗。伊马替尼治疗患者的 48 个月 PFS 和 OS 率分别为 50.5% 和 79.5%。在多变量 Cox 回归分析中，c-KIT 外显子 9 和 PDGFRA 突变是伊马替尼治疗患者 PFS 不良的预后因素，PDGFRA 突变是 OS 不良的预后因素。接受舒尼替尼治疗的患者的中位 PFS 为 12.76 个月（95% 置信区间 (CI)，11.01-14.52）。具有 c-KIT 外显子 9 突变的患者比具有其他遗传畸变的患者具有更长的 PFS。使用瑞戈非尼治疗的患者的中位 PFS 为 7.14 个月（95% CI，3.39-10.89）。我们展示了接受 TKI 治疗的晚期 GIST 患者的真实临床结果，并将突变状态确定为患者生存的独立预后因素。© 2024 . 作者。

Most gastrointestinal stromal tumors (GISTs) harbor c-KIT or PDGFRA mutations. Administration of tyrosine kinase inhibitors (TKIs) has significantly improved the survival of patients with GISTs. We aimed to evaluate the clinical outcome of advanced or recurrent GIST patients in Taiwan.Patients diagnosed between 2010 and 2020 were enrolled. The collected data included baseline characteristics, treatment pattern, treatment outcome, genetic aberrations and survival status. Progression-free survival (PFS) and overall survival (OS) were analyzed and plotted with the Kaplan-Meier method. Cox regression analysis was used to analyze the prognostic factors of survival.A total of 224 patients with advanced or recurrent GISTs treated with TKIs were enrolled. All patients received imatinib treatment. Ninety-three and 42 patients received sunitinib and regorafenib treatment, respectively. The 48-month PFS and OS rates for patients treated with imatinib were 50.5% and 79.5%, respectively. c-KIT exon 9 and PDGFRA mutations were prognostic factors for a poor PFS and PDGFRA mutation was a prognostic factor for a poor OS in patients treated with imatinib in multivariate Cox regression analysis. The median PFS of patients who received sunitinib treatment was 12.76 months (95% confidence interval (CI), 11.01-14.52). Patients with c-KIT exon 9 mutations had a longer PFS than those with other genetic aberrations. The median PFS of patients treated with regorafenib was 7.14 months (95% CI, 3.39-10.89).We present real-world clinical outcomes for advanced GIST patients treated with TKIs and identify mutational status as an independent prognostic factor for patient survival.© 2024. The Author(s).