摘要
目的:分析40例儿童骨髓增生异常综合征(MDS)的临床特点及预后,为临床诊疗提供参考。方法:回顾性总结苏州大学附属儿童医院血液科2011年1月1日至2017年12月31日收治的40例初诊为MDS患儿的临床特点、危险分层和不同治疗方案对预后的影响。Kaplan-Meier生存曲线分析患儿3年总生存率和无事件生存率。结果:40例患儿中,男女比例1.4∶1.0,男性略多于女性,中位年龄6.0岁。其中儿童难治性血细胞减少症是最常见类型,染色体异常11例,合并基因异常12例。接受造血干细胞移植(HSCT)15例,未进行HSCT而行单纯药物治疗25例。接受HSCT治疗和非HSCT治疗的3年总生存率分别为(72.2±12.2)%和(35.3±10.2)%(P=0.039),无事件生存率分别为(65.0±12.9)%和(19.2±8.4)%(P=0.012)。COX回归分析显示,年龄<7岁(P=0.033)、初诊血小板<50×10^(9)/L(P=0.007)、存在复杂核型和/或基因突变(P=0.0002)、未接受HSCT治疗(P=0.016)是影响儿童MDS预后的高危因素。40例患儿依据国际预后积分系统(IPSS)、WHO分型预后积分系统(WPSS)及修订的国际预后积分系统(IPSS-R)3种方法分类,分析显示以上3类风险评估方法对儿童MDS进行风险评估都存在局限性,不能全面评估儿童MDS预后。结论:儿童MDS以儿童难治性血细胞减少症多见。COX多因素分析显示,年龄<7岁、初诊血小板<50×10^(9)/L、存在复杂核型和/或基因突变、未接受HSCT治疗是影响患儿预后的高危因素。HSCT是目前治愈儿童MDS的最有效手段。目前临床评估儿童MDS预后的IPSS-R等方法具有明显局限性。
Objective:To analyze the clinical characteristics and prognosis of 40 children with myelodysplastic syndrome(MDS),and provide ideas for clinical diagnosis and treatment.Methods:The clinical characteristics,risk stratification,and different treatment regimens of 40 cases with MDS admitted in Department of Hematology of Children’ s Hospital of Soochow University from January 1,2011 to December 31,2017 was retrospectively analyzed.Kaplan-Meier survival curve were used to estimate 3-year overall survival(OS) rate and event-free survival(EFS) rate.Results:In 40 cases,the ratio of male to female was 1.4:1.0,male was more than female,and median age was 6.0 years old.Among them,refractory cytopenia(MDS-RCC) was the most common type,and 11 cases were chromosomal abnormalities,21 cases genetic abnormalities.Fifteen cases accepted hematopoietic stem cell transplantation(HSCT)treatment,while 25 cases did not but drug therapy alone.The 3-year OS rate of the cases who accepted HSCT or not was(72.2 ± 12.2)% and(35.3 ± 10.2)%(P=0.039),3-year EFS rate was(65.0 ± 12.9)% and(19.2 ± 8.4)%(P=0.012),respectively.Cox regression analysis showed that age <7 years old(P=0.0333),initial diagnosed platelet <50 × 10^(9)/L(P=0.007),presence of complex karyotypes and/or gene mutations(P=0.0002),and treatment without HSCT(P=0.016)were the high-risk factors of prognosis.All the children were classified according to IPSS,WPSS and IPSS-R,while analysis result showed that the above three risk assessment had limitations for risk assessment of MDS in children,they could not comprehensively assess the prognosis of children with MDS.Conclusion:MDS-RCC in children is more common.Cox multivariate analysis shows that age <7 years old,initial diagnosed platelet <50 × 10^(9)/L,presence of complex karyotypes and/or gene mutation,and treatment without HSCT are the high-risk factors of prognosis in children with MDS.HSCT is the most effective treatment to cure children with MDS at present.The current methods such as IPSS-R commonly used in assessment of prognosis in children with MDS show obvious limitation.
作者
郑夏
徐基昕
蒋卫芹
孔令军
赵文理
ZHENG Xia;XU Ji-Xin;JIANG Wei-Qin;KONG Ling-Jun;ZHAO Wen-Li(Department of Pediatrics,Huashan Hospital North Fudan University,Shanghai 201900,China;Department of Hematology.Zhengzhou Children's Hospital,Zhengzhou 450000,Henan Province,China;Department of Emergency,.Children's Hospital of Shanghai,Shanghai 200333,China;Department of Hematology,Children's Hospital of Soochow University,Suzhou 215025,Jiangsu Province,China;Department of Hematology,SJTU Affiliated Siuxth People's Hospital South Campus,Shanghai Fengxian District Central Hospital,Shanghai 201400,China)
出处
《中国实验血液学杂志》
CAS
CSCD
北大核心
2022年第1期195-200,共6页
Journal of Experimental Hematology
基金
上海市卫健委重点科研项目(201640028)
上海市奉贤区科委重点科研项目(奉科20160801)。
关键词
骨髓增生异常综合征
儿童
临床特点
危险分层
预后
myelodysplastic syndrome
children
clinical characteristics
risk stratification
prognosis
