免疫抑制疗法治疗儿童再生障碍性贫血疗效分析

Efficacy of immunosuppressive therapy for children with aplastic anemia

目的探讨免疫抑制疗法(IST)治疗儿童再生障碍性贫血(AA)的疗效、安全性及影响疗效的主要因素。方法对2007年1月至2010年12月接受IST治疗的55例重型再生障碍性贫血(SAA)及51例慢性再生障碍性贫血(CAA)患儿的临床资料进行回顾性分析。结果①在CAA患儿中,抗胸腺球蛋白(ATG)联合环孢素A(CsA)治疗组总有效率明显高于CsA单独治疗组(80%vs 44%,P<0.05);在SAA患儿中,ATG+CsA联合治疗组总有效率亦高于CsA单独治疗组(75%vs 40%,P<0.05)。各组均无病例进展为骨髓增生异常综合征、阵发性夜间血红蛋白尿及急性髓细胞白血病等克隆性疾病。②ATG+CsA联合治疗组中,病程<6个月、骨髓造血面积>40%、治疗前无重症感染以及有G-CSF早期治疗反应的患儿治疗效果较好,而治疗效果与AA分型、年龄等指标无关。结论 ATG+CsA联合治疗是治疗儿童AA的一种安全有效的方法;病程长短、有无严重感染、骨髓造血面积及G-CSF早期治疗反应是影响疗效的主要因素。

Objective To study the effectiveness and safety of immunosuppressive therapy（IST） in the treatment of childhood aplastic anemia（AA） and to study the main factors influencing the effectiveness.Methods The clinical data of 55 children with severe aplastic anemia（SAA） and 51 children with chronic aplastic anemia（CAA） were retrospectively analyzed.All patients received IST from January 2007 to December 2010.Results In children with CAA,the effective rate of antithymocyte globulin（ATG） plus cyclosporine A（CsA） combination therapy was significantly higher than that of CsA alone（80% vs 44%;P0.05）;in children with SAA,the effective rate of ATG plus CsA combination therapy was also significantly higher than that of CsA alone（75% vs 40%;P0.05）.No patients developed clonal disease such as myelodysplastic syndrome,paroxysmal nocturn hemoglobinuria or acute myelocytic leukemia.In patients treated with the ATG plus CsA combination therapy,the response rate was relatively high for children whose disease course was less than six months,bone marrow hematopoietic area was more than 40%,had no severe infections,and experienced granulocyte colony stimulating factor（G-CSF） reaction during the early treatment;however,it was not related to AA subtypes and age.Conclusions ATG plus CsA combination therapy is effective and safe in the treatment of childhood AA.The disease course,bone marrow hematopoietic area,severe infections and G-CSF reaction to early treatment are the main factors influencing the therapeutic effects.