胃肠道间质瘤的分子靶向治疗

Molecular targeted therapy for the treatment of gastrointestinal stromal tumor

分子靶向药物的出现，改变了胃肠道间质瘤（GIST）的治疗模式。伊马替尼400mg／d被推荐为转移性GIST的标准一线治疗方案。伊马替尼标准剂量治疗失败后，增加伊马替尼剂量或换用舒尼替尼治疗可进一步延长患者生存时间，同时新的分子靶向药物显示出了治疗GIST的潜在活性。GIST完整切除术后，伊马替尼辅助治疗可改善中高度复发风险患者的无复发生存率。伊马替尼术前治疗可提高手术切除率，但是否使患者生存获益尚未得到最终证实。c—kit和（或）血小板源性生长因子受体仪（PDGFRcα）基因突变可以预测伊马替尼与舒尼替尼的疗效，同时亦有助于辅助治疗获益人群的筛选。

Molecular targeted agents changed the therapeutic pattern on gastrointestinal stromal tumor （GIST）. Imatinib of 400 mg/d is recommended to be the first line therapy for metastatic GIST. Escalation of imatinib dose or sunitinib could improve the overall survival of patients when the treatment with standard dose of imatinib failed. In addition, new targeted agents showed potential anti-tumor efficacy. Imatinib adjuvant therapy could improve the recurrence-free survival of GIST patients with median or high risk after complete tumor resection. Preoperative treatment with imatinib raises tumor resection rate, but whether it could provide survival benefit has not been proved. C-kit/PDGFRot gene mutation can predict the efficacy of imatinib and sunitinib, and it is helpful to screen out the GIST patients to receive imatinib adiuvant therapy.