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在高危胃肠道间质瘤病例中鉴别极高风险患者:基于多中心人群回顾性队列研究的列线图的开发与验证

Distinguishing very high-risk patients among high-risk gastrointestinal stromal tumor cases: development and validation of a nomogram based on a multicenter population-based retrospective cohort study.

作者信息

Jiang Qi, Fu Yang, Zhang Xinhua, Zhang Peng, Gao Zhidong, Zhang Jun, Zhao Xuefeng, Qiu Haibo, Zhang Zilong, Fu Yingfeng, Liu Jian, Yang Zhenhua, Zhang Bo, Gao Xiaodong, Tao Kaixiong

机构信息

Department of Gastrointestinal Surgery, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.

Department of Gastrointestinal Surgery, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, China.

出版信息

Ann Med. 2025 Dec;57(1):2520896. doi: 10.1080/07853890.2025.2520896. Epub 2025 Jun 20.

DOI:10.1080/07853890.2025.2520896
PMID:40539729
Abstract

BACKGROUND

High-risk gastrointestinal stromal tumors (GISTs) are highly heterogeneous. This study aimed to developa nomogram for predicting recurrence in patients with high-risk GISTand to provide guidance for adjuvant therapy.

METHODS

Data were retrospectively collected from 971 patients with high-risk GIST who underwent genetic testingat 13 centers in China. A nomogram was constructed in the training cohort and validated in the validation cohort.

RSEULTS

The training and validation cohorts included 696 and 275 patients, respectively. The nomogram incorporated blood plateletlevels, total protein, tumor location, tumor size, mitotic count, tumor rupture, Ki-67 index, and gene mutations. The C-index and AUC were 0.758 and 0.781 in the training cohort and 0.841 and 0.755, respectively, in the validation cohort. Calibration and DCA curves confirmed favorable discrimination, calibration accuracy, and clinical benefits. Using the nomogram, patients were categorized into a general high-risk groupand a very high-risk group. Among the general high-risk group, no significant difference in RFS was observed between patients who received adjuvant imatinib for 2.5 years or longer. Conversely, in the very high-risk group, patients receiving adjuvant imatinib for five or more years had markedly improved RFS.

CONCLUSIONS

Based on the largest nomogram-related multicenter study in high-risk GIST, the nomogram accurately predicted RFS in patients with high-risk GIST and provided guidance on adjuvant therapy for the first time. For general high-risk patients, 3 years of adjuvant imatinib is adequate, whereas very high-risk patients benefit significantly from more than 5 years of adjuvant imatinib.

摘要

背景

高危胃肠道间质瘤(GIST)具有高度异质性。本研究旨在开发一种用于预测高危GIST患者复发的列线图,并为辅助治疗提供指导。

方法

回顾性收集了来自中国13个中心的971例接受基因检测的高危GIST患者的数据。在训练队列中构建列线图,并在验证队列中进行验证。

结果

训练队列和验证队列分别包括696例和275例患者。该列线图纳入了血小板水平、总蛋白、肿瘤位置、肿瘤大小、有丝分裂计数、肿瘤破裂、Ki-67指数和基因突变。训练队列中的C指数和AUC分别为0.758和0.781,验证队列中分别为0.841和0.755。校准曲线和决策曲线分析证实了良好的区分度、校准准确性和临床获益。使用该列线图,患者被分为一般高危组和极高危组。在一般高危组中,接受辅助伊马替尼治疗2.5年或更长时间的患者之间无进展生存期(RFS)无显著差异。相反,在极高危组中,接受辅助伊马替尼治疗5年或更长时间的患者RFS明显改善。

结论

基于高危GIST中最大规模的与列线图相关的多中心研究,该列线图准确预测了高危GIST患者的RFS,并首次为辅助治疗提供了指导。对于一般高危患者,3年的辅助伊马替尼治疗就足够了,而极高危患者从超过5年的辅助伊马替尼治疗中显著获益。

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