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中国癌症防治杂志

中国癌症防治杂志 ›› 2021, Vol. 13 ›› Issue (4): 370-375.doi: 10.3969/j.issn.1674-5671.2021.04.07

• 恶性血液病综合治疗专栏 • 上一篇    下一篇

芦可替尼治疗骨髓纤维化患者的疗效及其预测模型

  

  1. 复旦大学附属中山医院血液科，病理科
  • 出版日期:2021-08-25 发布日期:2021-09-17
  • 通讯作者: 刘澎 E-mail：liu.peng@zs-hospital.sh.cn
  • 基金资助:
    国家自然科学基金项目（81873450）

Establishment and analysis of therapeutic efficacy prediction model of ruxolitinib in patients with myelofibrosis

  • Online:2021-08-25 Published:2021-09-17

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摘要： 目的 探讨芦可替尼治疗骨髓纤维化的疗效及其预测指标。方法 选取2017年11月至2020年12月在复旦大学附属中山医院血液科诊治的采用芦可替尼治疗的64例骨髓纤维化患者为研究对象，以治疗12周时脾脏体积较基线缩小≥35%为终点事件。采用LASSO回归筛选芦可替尼治疗骨髓纤维化的疗效预测指标，二分类Logistic回归建立预测模型，受试者工作特征（ROC）曲线下面积（AUC）评价模型的预测效能。 结果 64例骨髓纤维化患者中，DIPSS评分为低危10例、中危-1 22例、中危-2 14例、高危18例。JAK2V617F基因突变45例，MPL W515L/K基因突变 3例，CALR基因突变8例，三阴性8例。LASSO回归筛选出13个变量与芦可替尼缩脾的疗效相关。基于Logistic回归以平均血小板体积（MPV）、外周血有原幼细胞（BLAST）、血小板计数（PLT）、红细胞体积分布宽度（RDW-SD）为预测因素成功构建疗效预测模型，其AUC为0.774，敏感度为81.0%，特异度为69.6%。64例骨髓纤维化患者中共有23例（35.94%）患者脾脏体积较基线缩小≥35%；严格按照血小板计数选择芦可替尼起始剂量的15例患者进行亚组分析，7例（46.67%）患者的脾脏体积较基线缩小≥35%，与所有患者相比差异有统计学意义（P<0.001）。结论 早期给予足量芦可替尼治疗的骨髓纤维化患者疗效良好，以MPV、BLAST、PLT、RDW-SD为预测因素构建的Logistic回归模型具有较好的疗效预测效能。

关键词: 骨髓纤维化, 芦可替尼, LASSO回归, Logisitc回归分析, 疗效

Abstract: Objective To investigate the efficacy of Ruxolitinib in the treatment of myelofibrosis and its predictive indices. Methods A total of 64 patients with myelofibrosis, treated with Ruxolitinib in the Department of Hematology, Zhongshan Hospital, Fudan University from November 2017 to December 2020, were collected as the study subjects. The endpoint was the reduction of spleen volume ≥35% from baseline at 12 weeks of treatment. LASSO regression was used to screen the predictive indices of the therapeutic effect of Ruxolitinib in the treatment of myelofibrosis. The regression model was established by the binary logistic regression, and the prediction efficiency of the model was evaluated by the areas under the curve (AUC) of receiver operating characteristic (ROC). Results Among the 64 patients with myelofibrosis, DIPSS scores were 10 low risk patients, 22 intermediate risk-1 patients, 14 intermediate risk-2 patients, and 18 high risk patients; 45 cases of JAK2V617F gene mutation, 3 cases of MPL W515L/K gene mutation, 8 cases of CALR gene mutation, and 8 cases of triple negative. The results of LASSO regression showed that 13 variables were related to the efficacy of Ruxolitinib. Using MPV, BLAST, PLT and RDW-SD as predictors, the predictive model of efficacy was successfully established based on Logistic regression, with AUC of 0.774, sensitivity of 81.0% and the specificity of 69.6%. There were 23 patients (35.94%) with myelofibrosis whose spleen volume decreased by ≥35% compared with baseline; 15 patients with the initial sufficient dose of Ruxolitinib were selected strictly for subgroup analysis according to the platelet count, and the result showed that the spleen volume of 7 patients (46.67%) was reduced by ≥35% compared with the baseline, which was statistically significant compared with all patients (P<0.001). Conclusions Early treatment with sufficient dose of Ruxolitinib shows good efficacy in myelofibrosis patients, and the Logistic regression model including MPV, BLAST, PLT and RDW-SD as the predictive factors has good prediction efficacy.

Key words: Myelofibrosis, Ruxolitinib, LASSO regression, Logistic regression, Therapeutic effect

中图分类号: 

  • R733