Department of Gastrointestinal Surgery, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, No. 1277 Jiefang Avenue, Wuhan, 430022, Hubei Province, China.
Department of Gastrointestinal Surgery, Renji Hospital, Shanghai Jiao Tong University School of Medicine, No. 160 Pujian Road, Shanghai, 200127, China.
Adv Ther. 2022 Jun;39(6):2920-2931. doi: 10.1007/s12325-022-02115-3. Epub 2022 Apr 24.
To evaluate clinicopathologic features and prognosis of post-complete resection in patients with PDGFRA-mutant gastrointestinal stromal tumor (GIST), and even to establish a relapse-free survival (RFS) prognostic model for this subgroup.
This retrospective study used data from patients with primary PDGFRA-mutant GIST who underwent complete resection (2005-2019) at 16 large-scale medical centers in China. Stepwise multivariate Cox regression models 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.
A total of 280 patients with PDGFRA-mutant (172 D842V-mutant and 108 non-D842V-mutant) GIST after complete resection were enrolled. Most tumors originated in the stomach (89.6%). The 1-, 3-, and 5-year RFS rates 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). Multivariate analysis demonstrated that D842V mutation (P = 0.017), non-gastric tumor (P < 0.001), and Ki-67 > 5% (P = 0.005) were the independent variables influencing the prognosis of patients with PDGFRA-mutant GIST. The scoring model showed the predicted and actual cumulative 1-, 3- and 5-year follow-up relapse rates fit well.
PDGFRA-mutant GIST mostly originated in the stomach and had a favorable prognosis after surgery. Non-D842V-mutant patients might have better prognoses than D842V-mutant patients. The prognostic model demonstrated favorable prediction accuracy, suggesting its clinical utility.
本研究旨在评估 PDGFRA 突变型胃肠道间质瘤（GIST）患者完全切除术后的临床病理特征和预后，并建立该亚组患者无复发生存（RFS）的预测模型。
本回顾性研究纳入了 2005 年至 2019 年在中国 16 家大型医疗机构接受完全切除术治疗的原发性 PDGFRA 突变型 GIST 患者。采用逐步多因素 Cox 回归模型构建预测模型，模型中的潜在预测因素在常规临床实践中均可用，并通过风险评分函数进行分析。通过校准直方图和时间依赖性接收者操作特征曲线对预测模型进行交叉验证。
共纳入 280 例完全切除术后的 PDGFRA 突变型（172 例 D842V 突变型和 108 例非 D842V 突变型）GIST 患者，大多数肿瘤起源于胃（89.6%）。1、3 和 5 年 RFS 率分别为 95.9%、91.2%和 89.5%。非 D842V 突变型组的 RFS 优于 D842V 突变型组（P=0.033）。多因素分析表明，D842V 突变（P=0.017）、非胃肿瘤（P<0.001）和 Ki-67>5%（P=0.005）是影响 PDGFRA 突变型 GIST 患者预后的独立变量。评分模型显示预测和实际累积 1、3 和 5 年随访复发率拟合良好。
PDGFRA 突变型 GIST 主要起源于胃，术后预后良好。非 D842V 突变型患者的预后可能优于 D842V 突变型患者。该预后模型具有良好的预测准确性，提示其具有临床应用价值。
