Su Wei-Chih, Huang Ching-Wen, Chen Yen-Cheng, Chang Tsung-Kun, Chen Po-Jung, Yeh Yung-Sung, Yin Tzu-Chieh, Tsai Hsiang-Lin, Wang Jaw-Yuan
Graduate Institute of Clinical Medicine, College of Medicine, Kaohsiung Medical University, Kaohsiung 80708, Taiwan, R.O.C.
Division of Colorectal Surgery, Department of Surgery, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung 80708, Taiwan, R.O.C.
Oncol Lett. 2025 Apr 14;29(6):299. doi: 10.3892/ol.2025.15045. eCollection 2025 Jun.
Curative resection is typically recommended for treating gastrointestinal stromal tumors (GISTs). Exceptions are made for locally advanced GISTs (LAGISTs) where radical resection may be impossible. First-line imatinib therapy can be employed to treat GISTs harboring mutations in the tyrosine-protein kinase KIT () and platelet-derived growth factor receptor α () genes to reduce the tumor size to resectable levels and minimize surgical risks. The present study investigated the treatment outcomes of patients with LAGISTs with different and gene mutations who received first-line imatinib therapy. A total of 37 patients with LAGISTs who underwent first-line imatinib treatment were included, and the median follow-up period was 41 months. Treatment regimens included imatinib, with subsequent therapies, such as sunitinib and regorafenib, administered upon imatinib failure. The genetic profiles of and were analyzed. Of the 37 patients, 24 (64.9%) successfully underwent curative resection. The median progression-free survival (PFS) was 36 months and the median overall survival (OS) was 41 months. Patients presented with tumors with various genetic mutations, which differentially affected their PFS and OS and adverse events were typically manageable. However, the gene mutation status was not significantly associated with treatment response or surgical resectability (both P>0.05). The present study elucidated the effects of first-line therapy on LAGISTs with genetic mutations, underscoring the effectiveness of imatinib treatment and the value of continual patient monitoring. Additional studies with long-term follow-up are required to evaluate treatment outcomes.
对于胃肠道间质瘤（GIST）的治疗，通常建议进行根治性切除。对于局部晚期GIST（LAGIST），若无法进行根治性切除则为例外情况。一线伊马替尼治疗可用于治疗携带酪氨酸蛋白激酶KIT（）和血小板衍生生长因子受体α（）基因突变的GIST，以将肿瘤大小缩小至可切除水平并降低手术风险。本研究调查了接受一线伊马替尼治疗的不同KIT和PDGFRA基因突变的LAGIST患者的治疗结果。共有37例接受一线伊马替尼治疗的LAGIST患者纳入研究，中位随访期为41个月。治疗方案包括伊马替尼，后续治疗如舒尼替尼和瑞戈非尼在伊马替尼治疗失败后使用。分析了KIT和PDGFRA的基因谱。37例患者中，24例（64.9%）成功接受了根治性切除。中位无进展生存期（PFS）为36个月，中位总生存期（OS）为41个月。患者呈现出具有各种基因突变的肿瘤，这些突变对其PFS、OS有不同影响，且不良事件通常可控。然而，基因突变状态与治疗反应或手术可切除性均无显著相关性（P均>0.05）。本研究阐明了一线治疗对具有基因突变的LAGIST的影响，强调了伊马替尼治疗的有效性以及持续监测患者的价值。需要进行长期随访的更多研究来评估治疗结果。
