地西他滨单药或联合三氧化二砷治疗骨髓增生异常综合征效果分析  

Efficacy analysis of decitabine monotherapy or combined with arsenic trioxide in treatment of myelodysplastic syndromes

作　　者：严泽莹 王莹 刘之茵 李佳明 孙海敏 陈钰 张苏江 Yan Zeying;Wang Ying;Liu Zhiyin;Li Jiaming;Sun Haimin;Chen Yu;Zhang Sujiang(Department of Hematology, North Hospital of Ruijin Hospital Affiliated to Medical College of Shanghai Jiaotong University, Shanghai 201800, China)

机构地区：[1]上海交通大学医学院附属瑞金医院北院血液科,201800

出　　处：《白血病．淋巴瘤》2019年第7期385-389,共5页Journal of Leukemia & Lymphoma

摘　　要：目的探讨地西他滨单药或联合三氧化二砷治疗骨髓增生异常综合征(MDS)患者的效果和耐受性.方法总结回顾上海交通大学医学院附属瑞金医院北院2014年1月至2017年4月收治的初发MDS患者32例临床资料,并进行随访.23例患者使用地西他滨联合三氧化二砷治疗(地西他滨20mg·m^-2·d^-1,第1天至第5天;三氧化二砷0.16mg/kg,第1天至第5天;4~6周重复1次),9例患者使用地西他滨单药治疗(地西他滨20mg·m^-2·d^-1,第1天至第5天,4~6周重复1次).分析患者临床病理特征及基因突变对疗效的影响.结果32例MDS患者中,男性18例,女性14例;年龄17~72岁,中位年龄56岁.基因检测发现TP53突变10例,TET2突变8例,U2AF1突变4例,RUNX1突变3例,ASXL1突变3例,NRAS突变2例,DNMT3A突变2例,JAK2V617突变1例.随访时间2~23个月,中位随访时间8个月.总有效21例(65.6%),其中完全缓解(CR)10例(31.3%),骨髓完全缓解(mCR)5例(15.6%),血液学改善6例(18.7%),联合用药组与单药治疗组有效率和CR率比较差异均无统计学意义(P=0.441,P=0.681).TP53突变的10例患者中7例CR.多因素分析显示,TP53突变是治疗获得CR的独立影响因素(P=0.037).治疗后所有患者均出现骨髓抑制,肺部感染16例.结论地西他滨联合三氧化二砷治疗MDS效果较好、耐受性高.有TP53突变的患者接受地西他滨单药或联合三氧化二砷疗效好于平均水平.Objective To analyze the therapeutic effect and tolerability of decitabine monotherapy or combined with arsenic trioxide for the treatment of patients with myelodysplastic syndromes(MDS).Methods Clinical characteristics of 32 patients with primary MDS in North Hospital of Ruijin Hospital Affiliated to Medical College of Shanghai Jiaotong University from January 2014 to April 2017 were retrospectively analyzed.The clinical data of these patients were collected,and the patients were followed up.Decitabine combined with arsenic trioxide was used in 23 cases,decitabine(20 mg·m-2·d-1)and arsenic trioxide(0.16 mg/kg)were administrated from day 1 to day 5 and was repeated every 4-6 weeks.For the remaining 9 cases,only decitabine was applied,decitabine(20 mg·m^-2·d^-1)was administrated from day 1 to day 5 and was repeated every 4-6 weeks.The clinicopathological characteristics and the effect of genetic mutations on the efficacy of treatment were investigated.Results Of the 32 patients with primary MDS,18 were male and 14 were female.The patients were 17-72 years old with a median age of 56 years old.Genetic analysis revealed 10 cases with TP53 mutations,8 cases with TET2 mutations,4 cases with U2AF1 mutations,3 cases with RUNX1 mutations,3 cases with ASXL1 mutations,2 cases with NRAS mutations,2 cases with DNMT3A mutations and 1 case with JAK2 V617 mutation.The follow-up time was 2-23 months with a median follow-up time of 8 months.A total of 21 cases(65.6%)attained treatment response.Among them,there were 10 cases(31.3%)with complete remission(CR),5 cases(15.6%)with bone marrow complete remission(MCR),and 6 cases(18.7%)with hematological improvement.There was no significant difference in the efficiency and CR rate between the combination group and the monotherapy group(P=0.441,P=0.681).Ten cases were found to have TP53 mutations,of which 7 cases had CR.Multivariate analysis demonstrated that TP53 mutation was an independent risk factor for CR(P=0.037).All patients developed myelosuppression after treatment,of which

关 键 词：骨髓增生异常综合征 地西他滨 三氧化二砷 疗效 

分 类 号：R551.3[医药卫生—血液循环系统疾病]