Clinicopathologic Characteristics and Prognosis of PDGFRA-Mutant Gastrointestinal Stromal Tumors: A Large-Scale, Multi-Institutional, Observational Study in China
Clinicopathologic Characteristics and Prognosis of PDGFRA-Mutant Gastrointestinal Stromal Tumors: A Large-Scale, Multi-Institutional, Observational Study in China
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PDGFRA 突变胃肠道间质瘤的临床病理特征和预后：中国的一项大规模、多机构观察性研究
DOI：
10.2139/ssrn.3783281
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发表时间：
2021-02
期刊：
影响因子：
--
通讯作者：
Kaixiong Tao
中科院分区：
文献类型：
--
作者：
Chengqi Zhang;Ming Wang;Jian Li;Xiaodong Gao;Bo Zhang;Han Liang;Ye Zhou;Guoqing Liao;Fan Feng;Yanbing Zhou;J Yu;Jun Zhang;Yongjian Zhou;Yingjiang Ye;Jiansi Chen;Qun Zhao;Kuntang Shen;Hui Cao;Kaixiong Tao
背景：评估PDGFRA突变的胃肠道间质瘤(GIST)患者的临床病理特征、辅助治疗效果和术后完全切除的预后，并建立该亚型的无复发生存(RFS)预后模型。方法：这项回顾性研究使用了2005年至2019年在中国16个大型医疗中心接受完全切除的原发PDGFRA突变的胃肠道间质瘤患者的数据。采用倾向性得分匹配(PSM)对混杂因素进行调整。采用多因素逐步COX回归模型建立预测模型，其中潜在的预测因素可用于常规临床实践，并使用风险评分函数。通过校准直方图和时间相关的接收器工作特性曲线对预测模型进行了交叉验证。结果：共纳入280例PDGFRA基因突变的GIST患者，其中D842V突变172例，非D842V突变108例。患者1、3、5年RFS率分别为95.9%、91.2%、89.5%。非D842V突变组的RFS明显优于D842V组(P=0.033)。接受或不接受伊马替尼辅助治疗的中高危非D842V突变GIST患者的RFS在PSM前后没有显著差异。多因素分析显示，D842V突变(P=0.017)、非胃肿瘤(P5%，P=0.005)是影响PDGFRA突变的胃肠道间质瘤患者预后的独立变量。评分模型显示，1、3、5年累积复发率的预测值与实际值吻合较好。Rnrn解释：PDGFRA突变的GIST患者术后预后良好。非D842V突变患者的预后可能好于D842V突变患者。佐剂伊马替尼对非D842V突变患者的益处可以忽略不计。预测模型显示出良好的预测准确性，提示其具有临床实用价值。rnrn基金声明：本研究得到国家自然科学基金中国(编号81874184)的支持。rnrn利益声明：无声明。rnrn伦理批准声明：各参与机构的机构审查委员会批准了该研究，并认为本研究不需要单独的知情同意。
Background: To evaluate clinicopathologic features, adjuvant therapy efficacy, and prognosis of post-complete resection in PDGFRA-mutant gastrointestinal stromal tumor (GIST) patients and establish a relapse-free survival (RFS) prognostic model for this GIST subgroup. rnrnMethods: This retrospective study used data from primary PDGFRA-mutant GIST patients who underwent complete resection (2005-2019) at 16 large-scale medical centers in China. Propensity score matching (PSM) was applied to adjust for confounding. Multivariate Cox regression models with stepwise were performed to build the prediction model, in which the potential predictors were available in routine clinical practice and using the risk score functions. The prediction model was cross-validated by calibration histogram and time-dependent receiver operating characteristic curves. rnrnFindings: A total of 280 PDGFRA-mutant (172 D842V-mutant; 108 non-D842V-mutant) GIST patients post-complete resection were enrolled. The 1-, 3-, and 5-year RFS rates of patients were 95.9, 91.2, and 89.5%, respectively. The RFS of the non-D842V-mutant group was superior to that of the D842V group (P=0.033). The RFS in intermediate/high-risk non-D842V-mutant GIST patients who received adjuvant therapy with or without imatinib did not significantly differ before and after PSM. Multivariate analysis revealed that D842V mutation (P=0.017), non-gastric tumor (P 5% (P=0.005) were the independent variables influencing PDGFRA-mutant GIST patient prognosis. The scoring model showed the predicted and actual cumulative 1-, 3- and 5-year follow-up relapse rates fit well. rnrnInterpretation: PDGFRA-mutant GIST patients had a favorable prognosis after surgery. Non-D842V-mutant patients might have better prognoses than D842V-mutant patients. The beneficial effect of adjuvant imatinib for non-D842V-mutant patients is negligible. The prognostic model demonstrated favorable prediction accuracy, suggesting its clinical utility.rnrnFunding Statement: This study was supported by the National Natural Science Foundation of China (No.81874184).rnrnDeclaration of Interests: None to declare.rnrnEthics Approval Statement: The institutional review boards of each participating institution approved the study and deemed that a separate informed consent was not necessary for this study.
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影响因子：
4.6
作者：
Yoo C;Ryu MH;Jo J;Park I;Ryoo BY;Kang YK
通讯作者：
Kang YK
用于识别“极高风险”患者的新型病理预后评分 (PPS)：对 506 名高风险胃肠道间质瘤 (GIST) 患者的多中心回顾性分析
影响因子：
3.2
作者：
Liu, Xuechao;Qiu, Haibo;Zhou, Zhiwei
通讯作者：
Zhou, Zhiwei
DOI：
--
发表时间：
1984-09
期刊：
Nihon Geka Gakkai zasshi
影响因子：
--
作者：
T. Onodera;N. Goseki;G. Kōsaki
通讯作者：
T. Onodera;N. Goseki;G. Kōsaki
影响因子：
39.2
作者：
Collins, Gary S.;Reitsma, Johannes B.;Moons, Karel G. M.
通讯作者：
Moons, Karel G. M.
影响因子：
168.9
作者：
Joensuu, Heikki;Hohenberger, Peter;Corless, Christopher L.
通讯作者：
Corless, Christopher L.