抗胸腺细胞球蛋白联合环胞菌素强烈免疫抑制治疗儿童极重型再生障碍性贫血疗效分析

Evaluation of the efficacy of immunosuppressive treatments with antithymocyteglobin combined with cyclosporine A(CSA) in children with acquired very severe aplastic anemia

目的评估抗胸腺细胞球蛋白(Antithymic Globlin,ATG)联合环孢菌素A(Cyclosporine A,CSA)治疗儿童极重型再生障碍性贫血(Very Severe Aplasia Anemia,VSAA)的治疗效果及安全性。方法前瞻性分析我院2006年5月至2007年9月期间14例儿童VSAA病人应用ATG+CSA的强烈免疫抑制治疗的疗效:起效时间、有效率、死亡率、不良反应、复发率、恶性疾病事件及生存率。结果对治疗有反应11例,起效时间为3至7个月,有效率为78.6%。应用ATG主要不良反应为过敏和血清病,无被迫停药病例。2例死亡,均死于ATG治疗后1个月内发生的败血症。所有病人均随访1年以上;随访过程中未见恶性疾病事件及复发病例;1年以上生存率为85.7%。结论儿童VSAA联合应用ATG+CSA的强烈免疫抑制治疗:起效快、反应率高、疗效好、不良反应可以控制,是治疗缺乏合适移植供体、而随时面对出血、感染等死亡威胁的VSAA儿童的有效的治疗方法。而ATG治疗后短期内感染是主要致死原因,需要积极控制。

Objective The present study aimed to evaluate the efficacy and safety of immunosuppressive treatments with cyelosporine A （CSA） combined with antithymocyteglobin （ATG） in children with acquired very severe aplastie anemia （VSAA）. Methods Fourteen eases with VSAA were treated with immunosuppressive agents mentioned above in our department from May 2006 to Sep 2007. The effective rate, relapse, adverse reactions, mortality and event free survival （EFS） were summarize. Results . The responsive time of the CSA combined with ATG was 3 months to 7 months, the effective rate was 78.6% （11/14）. In 11 eases who were responsive to therapy, the relapse rate was 0%. All eases had adverse reactions to the immunosuppressive agents. All cases were followed-up for more than 1 year, and the event free survival over one year was 85.7% （12/14）. Conclusion The immunosuppressive therapies for acquired severe aplastie anemia in childhood were effective. Incidence of adverse reactions was high, Methylprednisolone was good choice for it. The chief cause of death was infection after ATG therapy.