抗胸腺细胞球蛋白联合环孢素治疗重型再生障碍性贫血

Antithymocyte Globulin Combined with Ciclosporine Therapy on Severe Aplastic Anemia in Children

目的观察抗胸腺细胞球蛋白(ATG)联合环孢素治疗重型再生障碍性贫血(SAA)的疗效及安全性。方法回顾性分析本院2009年1月-2010年3月收治的14例应用ATG联合环孢素治疗的儿童SAA的疗效及不良反应。14例患儿均采用ATG联合环孢素治疗,ATG用量为5mg·kg-1·d-1,静脉滴注,共5d,应用ATG前清除感染灶;同时静脉滴注甲泼尼龙2～3mg·kg-1·d-1,以减轻变态反应;环孢素的起始用量为3～5mg·kg-1·d-1,根据血药质量浓度调整环孢素用量,维持环孢素血药谷质量浓度为100～200μg·L-1;辅以成分输血、粒细胞集落刺激因子及抗感染治疗。结果 14例患儿中有效10例(71.43%),无效4例(28.57%);14例患儿中骨髓增生活跃者8例,其中有效7例(87.5%),无效1例(12.5%);骨髓增生低下者6例,有效3例(50.0%),无效3例(50.0%);在14例应用ATG治疗的患儿中,1例患儿在治疗过程中出现变态反应,2例在应用ATG2周左右出现血清病;1例患儿治疗过程中因PLT过低、颅内出血死亡。结论 ATG联合环孢素治疗儿童SAA有效,且相对安全,尤其是骨髓增生尚活跃者,有可能达到治愈。

Objective To observe the curative effect and safety of antithymocyte globulin （ATG） combined with ciclosporine therapy on severe aplastic anemia （SAA） in children.Methods A retrospective analysis of clinical outcome and adverse events of 14 children with SAA from Jan.2009 to Mar.2010 in the First Affiliated Hospital of Zhengzhou University was performed.All of the 14 children with SAA treated with ATG combined with ciclosporine.ATG 5 mg·kg-1·d-1,5 days,methyprednisone 2-3 mg·kg-1·d-1 to reduce anaphylaxis,the focus of infection should be cleared before treatment;ciclosporine 3-5 mg·kg-1·d-1,and the dose adjusted to the ciclosporine trough plasma concentration about 100-200 μg·L-1,assisting with transfusion and granulocyte colony-stimulating factor.Results A total of 14 cases were investigated,10 cases （71.43%） were effective,other 4 cases （28.57%） were ineffective.Seven in 8 cases whose marrow hyperplasia active got remission （87.5%）,and 1 case was ineffective （12.5%）;3 in 6 cases whose marrow hyperplasia decreased were effective （50.0%）,and 3 cases were ineffective （50.0%）.In the 14 children,1 case occurred allergic reaction,2 cases got serum sickness like reaction,and 1 case died because of introacranial hemorrhage.Conclusions ATG combined with ciclosporine therapy on SAA is effective and safe,and the children with medullary active hyperplasia may receive complete remission.