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Abstract：
BACKGROUND: 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.
METHODS: 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.
RESULTS: 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).
CONCLUSIONS: 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.
METHODS: 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.
RESULTS: 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).
CONCLUSIONS: 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.
摘要：
背景：大多数胃肠道间质瘤（GIST）存在c-KIT或PDGFRA突变。酪氨酸激酶抑制剂(TKIs)的施用显著改善了患有GIST的患者的存活率。我们旨在评估台湾晚期或复发性GIST患者的临床结局。
方法：纳入2010年至2020年确诊的患者。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。分析无进展生存期(PFS)和总生存期(OS)并用Kaplan-Meier法绘制。Cox回归分析影响生存的预后因素。
结果：共纳入224例接受TKIs治疗的晚期或复发性GIST患者。所有患者均接受伊马替尼治疗。93例和42例患者接受舒尼替尼和瑞戈非尼治疗，分别。使用伊马替尼治疗的患者48个月PFS和OS分别为50.5%和79.5%，分别。在多变量Cox回归分析中，c-KIT外显子9和PDGFRA突变是PFS不良的预后因素，PDGFRA突变是伊马替尼治疗患者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。接受regorafenib治疗的患者的中位PFS为7.14个月（95％CI，3.39-10.89）。
结论：我们展示了接受TKIs治疗的晚期GIST患者的真实临床结果，并确定了突变状态作为患者生存的独立预后因素。
方法：纳入2010年至2020年确诊的患者。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。分析无进展生存期(PFS)和总生存期(OS)并用Kaplan-Meier法绘制。Cox回归分析影响生存的预后因素。
结果：共纳入224例接受TKIs治疗的晚期或复发性GIST患者。所有患者均接受伊马替尼治疗。93例和42例患者接受舒尼替尼和瑞戈非尼治疗，分别。使用伊马替尼治疗的患者48个月PFS和OS分别为50.5%和79.5%，分别。在多变量Cox回归分析中，c-KIT外显子9和PDGFRA突变是PFS不良的预后因素，PDGFRA突变是伊马替尼治疗患者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。接受regorafenib治疗的患者的中位PFS为7.14个月（95％CI，3.39-10.89）。
结论：我们展示了接受TKIs治疗的晚期GIST患者的真实临床结果，并确定了突变状态作为患者生存的独立预后因素。
