目的：评价伊马替尼治疗晚期、高危胃肠道间质瘤（GIST）的临床疗效及安全性。方法：回顾2011年1月—2016年6月期间收治的173例GIST患者资料，其中晚期患者73例，高危患者100例，分别比较两类患者中服用伊马替尼患者与未服药患者的预后情况。结果：73例晚期患者平均随访31（6~66）个月；Cox回归分析显示，其中服用伊马替尼患者总生存期（OS）明显长于未服用伊马替尼患者（1年OS：100.0% vs. 78.6%，2年OS：93.1% vs. 26.1%；HR=0.040，95% CI=0.011~0.152，P=0.000）。100例高危患者平均随访45（6~73）个月；亚组分析示，其中术后服用伊马替尼1年患者较未服药患者无复发生存期（RFS）明显延长（3年RFS：66.7% vs. 38.5%；HR=0.341，95% CI=0.134~0.868，P=0.024），而服用伊马替尼2年患者较服用伊马替尼1年患者RFS也明显提高（1年RFS：100.0% vs. 100.0%，2年RFS：100.0% vs. 88.9%，3年RFS：91.7% vs. 66.7%；HR=0.108，95% CI=0.015~0.778，P=0.027）；5例术后服用3年伊马替尼患者3年RFS为100.0%。服用伊马替尼的主要不良反应为浮肿、白细胞下降、胃肠道反应等，以1~2级为主。结论：伊马替尼治疗晚期、高危GIST有较好的安全性，能有效提高患者生存率；高危患者术后建议至少服用3年伊马替尼，但是否延长服药年限仍需更多的临床研究证实。
Clinical efficacy and safety of imatinib in treatment of advanced and high-risk gastrointestinal stromal tumor
Objective: To assess the clinical efficacy and safety of imatinib treatment for advanced and high-risk gastrointestinal stromal tumor (GIST). Methods: The clinical data of 173 GIST patients treated between January 2011 and June 2016 were reviewed. Of the patients, 73 cases had advanced GIST, and 100 cases had high-risk GIST. The outcomes were compared between patients with and without imatinib treatment in these two categories of patients, respectively. Results: The 73 patients with advanced GIST were followed up for 31 (6–66) months, and of them, the Cox regression analysis showed that the overall survival (OS) in patients with imatinib treatment was significantly prolonged compared with those without imatinib treatment (1-year OS: 100.0% vs. 78.6%, 2-year OS: 93.1% vs. 26.1%; HR=0.040, 95% CI=0.011–0.152, P=0.000). Follow-up was conducted for 45 (6–73) months in the 100 patients with high-risk GIST, of whom, the subgroup analysis showed that the recurrence free survival (RFS) in patients receiving postoperative 1-year imatinib treatment was significantly longer than that in those without imatinib treatment (3-year RFS: 66.7% vs. 38.5%; HR=0.341, 95% CI=0.134–0.868, P=0.024) and moreover, the RFS in patients receiving postoperative 2-year imatinib treatment was significantly longer than those receiving 1-year imatinib treatment (1-year RFS: 100.0% vs. 100.0%, 2-year RFS: 100.0% vs. 88.9%, 3-year RFS: 91.7% vs. 66.7%; HR=0.108, 95% CI=0.015–0.778, P=0.027); the 3-year RFS was 100.0% in the 5 high-risk patients who received postoperative 3-year imatinib treatment. The common adverse effects from imatinib treatment were edema, leukopenia and gastrointestinal disorders, and most of them were grade 1 to 2 in severity. Conclusion: Imatinib has favourable safety in treatment of advanced and high-risk GIST, and it can effectively improve the survival of the patients. For those high-risk patients, at least 3 years’ postoperative imatinib administration is recommended, but whether to extend the 3-year time limit of drug administration needs to be determined by further clinical studies.