【摘要】 目的 探索真实世界中局部晚期胃肠间质瘤（gastrointestinal stromal tumor, GIST）患者接受新辅助治疗的疗效及其影响因素。方法 回顾性纳入2014年4月1日至2025年4月1日在江苏省人民医院、南京鼓楼医院、江苏省肿瘤医院、徐州医科大学附属医院、江苏省苏北人民医院、连云港市第二人民医院、苏州大学附属第一医院、南京市第一医院、盐城市第一人民医院、无锡市人民医院、常州市第一人民医院、南通大学附属医院、南通市第一人民医院、扬州大学附属医院、常州市第二人民医院和无锡市第二人民医院共16家医院接受新辅助治疗的局部晚期GIST患者。通过实体瘤反应评价标准（Response Evaluation Criteria in Solid Tumors, RECIST） 1.1标准评估客观缓解率，分析人口学、临床病理特征和基因突变等因素与疗效和预后的关系，采用单因素差异分析、多因素logistic与Cox回归分析和Kaplan-Meier法进行统计学评估。结果 共收集137例GIST患者，104例接受伊马替尼治疗且资料完整的患者纳入分析。客观缓解率为43.3%，平均缩瘤率为26.96%,R0切除率达97.7%。单因素分析显示，客观缓解率在核分裂象≥5个/50 HPF患者中较低（P=0.029），在KIT原癌基因受体酪氨酸激酶（KIT proto-oncogene, receptor tyrosine kinase; KIT）基因外显子11缺失突变患者中有更低的趋势（P=0.075）。多因素logistic回归分析也显示，这2种因素与客观缓解情况有关（均P<0.05）。单因素Cox回归分析表明，核分裂象<5个/50 HPF降低术后复发风险（P=0.018），但多因素Cox回归分析未发现无瘤生存期的预后因素（均P>0.05）。结论 伊马替尼新辅助治疗可有效缩小GIST肿瘤体积并提高R0切除率，基因突变类型（如KIT基因外显子11缺失突变）和核分裂象活跃（≥5个/50 HPF）是客观缓解率低的潜在预测因素，核分裂象活跃者远期预后更差。对于新辅助治疗效果不佳和缩瘤效果不显著的患者，亟需寻求进一步缩瘤的治疗方案。更多还原

【Abstract】 Objective To investigate the efficacy of neoadjuvant therapy in patients with locally advanced gastrointestinal stromal tumors（GISTs） and its influencing factors in real-world settings. Methods Patients with locally advanced GISTs who received neoadjuvant therapy between April 1^st, 2014, and April 1^st, 2025, across 16 hospitals, including Jiangsu Provincial People’s Hospital, Nanjing Drum Tower Hospital, Jiangsu Cancer Hospital, Affiliated Hospital of Xuzhou Medical University, Northern Jiangsu People’s Hospital, Lianyungang Second People’s Hospital, the First Affiliated Hospital of Soochow University, Nanjing First Hospital, Yancheng No.1 People’s Hospital, Wuxi People’s Hospital, the First People’s Hospital of Changzhou, Affiliated Hospital of Nantong University, the First People’s Hospital of Nantong, Affiliated Hospital of Yangzhou University, the Second People’s Hospital of Changzhou, and Wuxi Second People’s Hospital, were retrospectively enrolled. O bjective response rate（ORR） was evaluated using Response Evaluation Criteria in Solid Tumors（RECIST） 1.1. The associations of objective response and prognosis with demographic features, clinicopathological characteristics, and genetic mutations were analyzed. Statistical methods included univariate analysis, multivariate logistic and Cox regression, and Kaplan-Meier analysis. Results Among 137 initially collected GIST patients, 104 cases treated with imatinib and with complete data were analyzed. The ORR was 43.3%, mean tumor reduction rate was 26.96%, and R0 resection rate was 97.7%. Univariate analysis showed lower ORR in patients with mitotic figures ≥5/50 HPF（P=0.029） and a trend toward lower ORR in those with KIT proto-oncogene, receptor tyrosine kinase（KIT） exon 11 deletion mutations（P=0.075）. Multivariate logistic regression confirmed that both factors were independently associated with reduced ORR（both P<0.05）. Univariate Cox regression indicated that mitotic figures <5/50 HPF reduced postoperative recurrence risk（P=0.018）, but multivariate Cox regression found no significant prognostic factors for disease-free survival（all P>0.05）. Conclusions Neoadjuvant imatinib effectively reduces tumor volume and achieves high R0 resection rate in locally advanced GISTs. KIT exon 11 deletion mutations and high mitotic activity（≥5/50 HPF） are potential predictors of suboptimal ORR, with the latter correlating with poorer long-term outcomes. For patients with inadequate tumor reduction, alternative strategies to enhance neoadjuvant efficacy are urgently needed.更多还原