77例骨髓增生异常综合征5q-综合征患者临床特征、疗效和生存分析  

Analysis of clinical characteristics,treatment response rate and survival of 77 myelodysplastic syndrome patients with del(5q)syndrome

作　　者：刘丹 徐泽锋 秦铁军 李承文 胡耐博 潘丽娟 曲士强 李冰 肖志坚 Liu Dan;Xu Zefeng;Qin Tiejun;Li Chengwen;Hu Naibo;Pan Lijuan;Qu Shiqiang;Li Bing;Xiao Zhijian(Institute of Hematology and Blood Diseases Hospital,CAMS&PUMC,State Key Laboratory of Experimental Hematology,National Clinical Research Center for Hematological Disorders,Tianjin 300020,China)

机构地区：[1]中国医学科学院、北京协和医学院血液学研究所、血液病医院,实验血液学国家重点实验室,国家血液系统疾病临床医学研究中心,天津300020

出　　处：《中华血液学杂志》2019年第11期895-900,共6页Chinese Journal of Hematology

基　　金：国家自然科学基金(81870104、81470297);天津市自然科学基金重点项目(18JCZDJC34900);中国医学科学院医学与健康科技创新工程(2016-I2M-1-001)。

摘　　要：目的探讨符合WHO(2016)诊断分型标准的骨髓增生异常综合征(MDS)5q-综合征患者的临床特征、来那度胺(LEN)疗效和生存情况。方法回顾性分析2008年1月至2018年4月于中国医学科学院血液病医院就诊的77例符合WHO(2016)诊断分型标准的MDS 5q-综合征患者临床资料,比较单纯5q-与5q-伴1个非-7/7q-的其他染色体异常(ACA)患者临床特征、疗效及生存情况,并比较LEN与非LEN药物治疗的疗效与生存情况。结果77例患者中,单纯5q-者64例,5q-伴ACA者13例,确诊时5q-伴ACA患者的中位年龄显著低于单纯5q-患者[58(29~64)岁对63(31~82)岁,z=2.164,P=0.030]。5q-伴ACA患者CD41免疫组化染色小巨核细胞(直径≤40μm)检出率(91.7%,11/12)显著高于单纯5q-患者(60.0%,33/55)(P=0.046)。可评估LEN疗效的29例患者中,单纯5q-患者(19例)与5q-伴ACA患者(10例)血液学总反应率(78.9%对80.0%)、血液学完全缓解(CR)率(57.9%对60.0%)、细胞遗传学反应(CyR)率[69.2%(9/13)对66.7%(4/6)]、完全细胞遗传学反应(CCyR)率[61.5%(8/13)对33.3%(2/6)]相当。单纯5q-与5q-伴ACA患者中位总生存(OS)时间差异无统计学意义(62个月对78个月,P=0.313)。29例LEN组患者的血液学总反应率(79.3%对36.0%)、CR率(58.6%对8.0%)、CyR率[68.4%(13/19)对11.1%(1/9)]、CCyR率[52.6%(10/19)对0(0/9)]均高于非LEN组患者(25例),但两组患者中位OS时间差异无统计学意义(78个月对62个月,P=0.297)。结论单纯5q-与5q-伴ACA患者临床特征大体相似,两组患者LEN疗效、中位OS时间无明显差异。LEN治疗5q-综合征患者的疗效肯定,优于非LEN药物。Objective To observe the clinical characteristics,treatment responses and prognosis of patients with myelodysplastic syndrome(MDS)-del(5q)syndrome who met WHO(2016)diagnostic typing criteria.Methods A total of 77 patients with del(5q)syndrome,according to WHO(2016)classification,were retrospectively analyzed between January 2008 and April 2018 in the Blood Diseases Hospital,Chinese Academy of Medical Sciences.Clinical characteristics,lenalidomide(LEN)efficacy and survivals were compared between the patients with del(5q)alone and those with one additional cytogenetic abnormality(ACA)with the exception of monosomy 7 or del(7q).Treatment response and overall survival(OS)were compared between patients who were treated with LEN and traditional non-LEN drugs.Results Of 77 patients,64 were isolated del(5q)and 13 were del(5q)with ACA.There were significant differences of the median age and percentage of patients who had small megakaryocytes in bone marrow smear by immunohistochemistry(CD41)between the patients with isolated del(5q)and the patients with del(5q)+ACA[58(29-64)years old vs 63(31-82)years old,z=2.164,P=0.030;and 91.7%vs 60.0%,P=0.046,respectively].The overall hematological response rate(78.9%vs 80.0%),complete hematological remission(CR)rate(57.9%vs 60.0%),cytogenetic response(CyR)rate[69.2%(9/13)vs 66.7%(4/6)]and complete cytogenetic response(CCyR)rate[61.5%(8/13)vs 33.3%(2/6)]of LEN were similar between the patients with isolated del(5q)(n=19)and with del(5q)+ACA(n=10),as well as the median Overall survival(OS)between these two groups of patients(62 months vs 78 months,P=0.388).The hematological response rate(79.3%vs 36.0%),CR rate(58.6%vs 8.0%),CyR rate[68.4%(13/19)vs 11.1%(1/9)]and CCyR rate[52.6%(10/19)vs 0(0/9)]were higher among patients treated with LEN(n=29)than those treated with non-LEN therapy(n=25).There was no statistically significant difference in OS between the patients with LEN or non-LEN therapy(78 months vs 62 months,P=0.297).Conclusion Comparing del(5q)syndrome patients with isolated del(5

关 键 词：骨髓增生异常综合征 5Q-综合征 治疗反应 来那度胺 生存分析 

分 类 号：R5[医药卫生—内科学]