去甲基化单药与联合改良IAG方案治疗中高危骨髓增生异常综合征患者疗效及安全性的临床分析  

Clinical analysis of the efficacy and safety of demethylation monotherapy and combined with modified IAG regimen in the treatment of patients with Intermediate to high risk myelodysplastic syndrome

作　　者：陈程 王珊 孙爱宁[1] CHEN Cheng;WANG Shan;SUN Ai-ning(The First Affiliated Hospital of Soochow University,Jiangsu Institute of Hematology,Key Laboratory of Thrombosis and Hemostasis of the Ministry of Health,Collaborative Innovation Center for Hematology,Suzhou 215006,China.)

机构地区：[1]苏州大学附属第一医院,江苏省血液研究所,卫生部血栓与止血重点实验室,血液学协同创新中心,江苏苏州215006

出　　处：《中国处方药》2022年第12期23-27,共5页Journal of China Prescription Drug

摘　　要：目的 比较去甲基化药物(HMA)单药和HMA联合改良IAG(伊达比星+阿糖胞苷+粒细胞集落刺激因子)方案治疗中高危骨髓增生异常综合征(MDS)患者的疗效和安全性。方法 收集2015年3月~2021年3月收治于苏州大学附属第一医院,并行HMA单药或联合改良IAG方案治疗的初诊中高危MDS患者资料,分析临床特征及相关基因突变对疗效的影响,并比较组间不良反应发生情况。结果 经过两个疗程的治疗,联合化疗组总体有效(ORR)率及完全缓解(CR)率明显高于单药治疗组(P=0.012,P <0.001)。多因素分析显示,患者初诊时平均红细胞血红蛋白量(MCH)水平是影响ORR的独立因素(P=0.049),NPM1基因突变是影响CR的独立因素(P=0.009),5号染色体长臂缺失(5q-)是影响骨髓完全缓解(mCR)率的独立因素(P=0.041);联合化疗组不良反应尤其是Ⅲ~Ⅳ级骨髓抑制的发生率显著高于单药治疗组,组间差异有统计学意义(P=0.002);联合化疗组感染发生率也较高,但经积极抗感染治疗后组间差异缩小(P=0.890)。结论 HMA联合改良IAG方案治疗中高危MDS患者较HMA单药疗效好,但Ⅲ~Ⅳ级骨髓抑制的不良反应发生率更高。MCH较高水平、合并NPM1基因突变及5q-的患者接受HMA或联合改良IAG方案治疗的效果均优于平均水平。Objective To compare the therapeutic effect and safty of hypomethylating agents(HMA) monotherapy or HMA combined with modified IAG(Idarubicin + Cytarabine + Recombinant human granulocyte colony-stimulating factor) regimen for the treatment of patients with intermediate and high-risk myelodysplastic syndrome(MDS). Methods The data of newly diagnosed intermediate and high-risk MDS patients admitted to the First Affiliated Hospital of Soochow University from March 2015 to March 2021 and treated with HMA alone or combined with the modified IAG regimen were collected, the effect of clinical characteristics and related gene mutations on the curative efficacy were analyzed, and the incidence of adverse reactions between groups were compared. Results After two courses of treatment, the overall response rate(ORR) and complete remission(CR) rates in the combined chemotherapy group were significantly higher than those in the monotherapy group(P = 0.012, P < 0.001). Multivariate analysis showed that the mean corpuscular hemoglobin(MCH) level at the initial diagnosis was an independent factor affecting ORR( P = 0.049), NPM1 gene mutation was an independent factor affecting CR(P = 0.009), the deletion of the long arm of chromosome 5(5q-) was an independent factor affecting the rate of bone marrow complete remission(mCR)(P = 0.041).The incidence of adverse reactions, especially grades Ⅲ~Ⅳ myelosuppression in the combined chemotherapy group was significantly higher than that in the monotherapy group, and the difference between groups was statistically significant(P = 0.002). The incidence of infection in the combined chemotherapy group was also higher than that in the monotherapy group, but the difference between groups was reduced through active anti-infection treatments(P = 0.890). Conclusion For the treatment of intermediate and high-risk MDS patients, HMA combined with modified IAG regimen has better curative effect than HMA monotherapy, but the incidence of adverse reactions of grade Ⅲ~Ⅳ myelosuppression was higher.

关 键 词：骨髓增生异常综合征 去甲基化药物 联合化疗 NPM1 5号染色体长臂缺失(5q-) 

分 类 号：R551.3[医药卫生—血液循环系统疾病]