免疫抑制治疗儿童再生障碍性贫血疗效分析

目的探讨提高儿童再生障碍性贫血(再障,aplastic anemia,AA)疗效的治疗方法。方法回顾分析56例接受正规治疗且随访≥3个月的再障患儿的临床资料,56例按治疗方案分为2组,一组为单用环胞素A(CsA)治疗(A组),另一组抗胸腺细胞球蛋白(ATG)联合CsA免疫治疗(B组),比较两组患儿的疗效及生存率。结果 31例慢性再障(CAA)患儿中接受A组治疗30例,总体有效率为83.3%(25/30),仅1例CAA患儿接受B组治疗。25例重型再障(SAA)患儿接受B组治疗16例,治疗有效率为50.0%(8/16),较接受A组治疗的9例SAA患儿的有效率(44.4%)高,但差异无统计学意义(P>0.05)。接受B组治疗的16例SAA患儿,12个月生存率为66.7%,2年生存率70.0%。结论 ATG联合CsA免疫治疗是儿童再障,尤其是无合适供体进行造血干细胞移植的SAA患儿首选方案,但应注意足量、持续用药,并努力降低ATG治疗期间感染发生率,这对提高儿童SAA治愈率有重要意义。临床儿科杂志,2012,30(5):428-430]

Clinical analysis of immunosuppressive therapy in childhood aplastic anemia

Objective To explore the treatment experiences in childhood aplastic anemia(AA) to improve the therapeutic efficacy.Methods Clinical data of 56 aplastic anemia cases was collected and reviewed retrospectively from January 1999 to November 2009.All patients were accepted regular treatment as well as followed up over 3 months.They were divided into 2 groups according to treatment protocol.One group(group A) was administered with cyclosporine A(CsA) only and the other group(group B) with antithymocyte globulin(ATG) combined with CsA.The treatment efficacy and survival rate were compared between these two groups.Results Among 30 of 31 children with chronic aplastic anemia(CAA) who enrolled into group A the total treatment effective rate was 83.3%,5 of them failed the treatment(16.7%).The other one child with CAA was enrolled into group B.Sixteen of 25 children with severe aplastic anemia(SAA) were enrolled into group B,the treatment effective rate was 50.0%,higher than that of 9 SAA children enrolled into group A(44.4%) without significant difference(P > 0.05).The survival rates of 12-month and 2-year follow up with SAA cases enrolled in group B were 66.7% and 70.0% respectively.Conclusions The immunosuppressive therapy with ATG and CsA is the preferred treatment for SAA children,especially for those lacking matched sibling donors for hematopoietic stem cell transplantation.To achieve higher efficacy rate and better clinical outcome,sufficient and sustained medication together with the efforts to cut down the infection rate during ATG treatment is needed.