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.
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。