摘要
目的通过对应用联合免疫抑制疗法(IST)的儿童获得性再生障碍性贫血(AA)临床资料分析,总结IST对儿童AA的疗效及预后相关因素,以及阵发性睡眠性血红蛋白尿(PNH)克隆的转化情况。方法回顾性分析安徽医科大学第二附属医院2009年6月—2019年8月应用ATG联合CSA治疗的59例AA患儿的临床资料以及AA患儿不同病程阶段T细胞亚群的变化,总结分析IST对AA患儿的疗效。采用SPSS 16.0统计软件进行数据分析。结果59例初诊AA患儿,中位随访时间24个月。(1)IST后1个月、3个月、6个月、9个月、12个月的有效率分别为22%、39%、57%、61%、71%。截止2019年6月随访18例AA患儿的5年无事件生存率达66%。(2)治疗后CD3+T、CD4+T细胞升高,CD8+T细胞下降,CD4+T/CD8+T比值升高,差异有显著性(P<0.05)。(3)对38例AA患儿进行PNH克隆的随访,发现初诊AA患儿中有11例PNH克隆阳性,IST后3个月PNH克隆均转阴。初诊有无PNH克隆与疗效无相关性。结论IST治疗儿童AA疗效确切,可以有效地改善AA患儿的细胞免疫功能。初诊时有部分AA患儿存在PNH克隆,IST前后伴PNH克隆不影响疗效。
Objective To analyze the clinical data of acquired aplastic anemia(AA)in children with combined immunosuppressive therapy(IST),and to summarize the therapeutic effect and prognostic factors of IST in children with AA,as well as the transformation of paroxysmal sleep hemoglobinuria(PNH)clones.Methods The clinical data of 59 children with AA treated by ATG combined with CSA in the second affiliated hospital of Anhui medical university from June 2009 to August 2019 were retrospectively analyzed,also the changes of T-cell subsets in children with AA at different stages of disease,and the efficacy of IST in children with AA were studied.Results Among 59 newly diagnosed children with AA,the median follow-up time was 24 months.①1 month、3 months、6 months、9 months、12 months after IST,the response rate was 22%、39%、57%、61%、71%respectively.Up to June 2019,the 5-year event-free survival rate of 18 cases was 66%.②After the treatment,the proportion of CD3+and CD4+cells was increased,CD8+cells was decreased,and the ratio of CD4+/CD8+was increased,with statistically significant differences(P<0.05).③Follow-up of PNH cloning was conducted in 38 children with AA,and it was found that among the newly diagnosed children with AA,11 children with AA showed positive PNH cloning,and PNH cloning all turned negative after IST three months.Conclusions IST is effective in treatment of children with AA and can effectively improve the cellular immune function.PNH cloning was found in some children with AA at the initial diagnosis,and PNH cloning does not affect the efficacy.
作者
陈洁洁
刘亢亢
储金华
黄玲玲
杨林海
谢志伟
涂松济
王宁玲
CHEN Jiejie;LIU Kangkang;CHU Jinhua;HUANG Lingling;YANG Linhai;XIE Zhiwei;TU Songji;WANG Ningling(Department of Paediatrics,the Second Affiliated Hospital of Anhui Medical University,Hefei 230601,China)
出处
《中国小儿血液与肿瘤杂志》
CAS
2020年第3期133-137,143,共6页
Journal of China Pediatric Blood and Cancer
