抗胸腺细胞球蛋白治疗儿童再生障碍性贫血的临床研究  

Treatment of childhood aplastic anemia with antithymocyte globulin, management of side effects and long-term follow-up

作　　者：傅晓燕[1] 谢晓恬[1] 蒋莎义[1] 石苇[1] 邵越霞[1] 

机构地区：[1]同济大学附属同济医院儿科,上海200065

出　　处：《中华儿科杂志》2011年第3期226-230,共5页Chinese Journal of Pediatrics

摘　　要：目的 总结以抗胸腺细胞球蛋白（ATG）为主的免疫抑制疗法治疗儿童再生障碍性贫血时,ATG的实施方法、不良反应防治和长期随访措施等与远期疗效的相关性.方法 儿童再生障碍性贫血35例,其中极重型再生障碍性贫血（VSAA）6例,急性再生障碍性贫血（SAA）11例,慢性重型SAA8例,中型再生障碍性贫血（MAA）10例.ATG治疗期间措施包括：治疗前清除感染灶;积极防治过敏反应;密切观察和处理因ATG相关血小板减少所致严重出血和免疫抑制所致严重感染;积极防治血清病;重视长期随访中的治疗措施与质量.结果 ATG治疗后,所有病例均出现60%以上的淋巴细胞绝对计数下降.平均随访28个月,总有效率为77.14%（27/35）,显效率为57.14%（20/35）.VSAA、SAA和MAA间疗效无明显差异.ATG不良反应观察结果：①48.6%出现轻度类过敏反应;②血清病发生率42.9%,平均病程3.6 d;③9例（25.7%）外周血小板（BPC）计数＜10×109/L;④8例（22.9%）在ATG治疗后1个月内发生感染;⑤未发生ATG治疗相关死亡.Genzyme和Fresenius两种不同ATG制剂,在疗效和ATG相关不良反应发生率方面差异均无统计学意义.结论 ATG治疗儿童SAA和MAA疗效显著,但需积极预防和控制ATG不良反应,避免治疗相关死亡.长期辅助治疗和提高随访质量,也是确保疗效的重要环节.Objective To evaluate the efficacy of antithymocyte globulin （ATG） based immunosupression therapy for childhood aplastic anemia, to reduce the adverse effects and to observe the long-term outcome. Method Thirty-five children with aplastic anemia （AA） were enrolled in this study.Six of the cases had very severe AA （VSAA）, 11 had severe AA （SAA）-Ⅰ, 8 had SAA-Ⅱ and 10 had moderate AA （MAA）. All these patients were treated with ATG plus Cyclosporin A （CSA）. The following measures were taken during the ATG therapy： infection of the patients had been controlled before ATG treatment. Comprehensive anti-allergic measures were implemented. Close attention was paid to the hemorrhage related with platelet reduction caused by ATG and severe infection of the patients. Result Shortly after the ATG usage, all the patients had a significant decrease of absolute peripheral lymphoblast count by more than 60 percent. With a mean follow-up time of 28 months, the total effective rate was 77.14％ （ 27/35）, significant response rate was 57.14％（20/35）. There was no significant difference among VSAA, SAA and MAA groups in the response rate. Adverse reactions included the following：① 48.6％ （17/35） patients presented mild anaphylactoid reaction during the first day of ATG treatment; ②42.9％（15/35） cases presented serum sickness 5-11 days after the last dose of ATG with a mean duration of 3. 6 days, all the patients were cured effectively with methylprednisolone; ③25.7％ （9/35）patient＇s peripheral blood platelet count was reduced, might be caused by ATG, to below 10 × 109/L, but no patient had severe hemorrhagic complication after platelet transfusion was performed; ④22.9％ （ 8/35 ） of patients got infection within a month after ATG therapy, including 3 cases with clinical septicemia, all the 3 cases recovered after antibiotics treatment. There was no ATG treatment-related death in this series. Conclusion ATG is a very effective therapy for children with SAA and MAA.

关 键 词：贫血 再生障碍性 抗淋巴细胞血清 免疫抑制法 药物毒性 治疗结果 儿童 

分 类 号：R556.5[医药卫生—血液循环系统疾病]