ATG-F治疗儿童再生障碍性贫血疗效研究  

Efficacy of ATG-F in the treatment of children with aplastic anemia

作　　者：石苇[1] 谢晓恬[1] 何薇[1] 周宁[1] 卢双龙[1] 邵越霞[1] 周晓迅[1] 乔晓红[1] 

机构地区：[1]上海市同济医院(同济大学附属同济医院)儿科,上海200065

出　　处：《中国小儿血液与肿瘤杂志》2015年第2期74-78,共5页Journal of China Pediatric Blood and Cancer

基　　金：上海市科委重点科研计划项目(项目编号:11JC1411900);上海市卫生局重大科研项目(项目编号:2010002)

摘　　要：目的探索抗人T-细胞兔免疫球蛋白（ATG-F，德国Fresenius）治疗儿童再生障碍性贫血（简称再障）的临床疗效。方法采用ATG-F联合环孢菌素A（CSA）的联合免疫抑制疗法（IST）治疗儿童再障共31例，其中极重型再障（VSAA）4例，重型再障（SAA）16例，依赖输血型非重型再障（NSAA）11例。实施ATG相关不良反应综合防治措施，并动态监测ATG—F治疗期间与治疗后外周血淋巴细胞绝对计数（ALC）。结果参照国际Camitta再障疗效标准，总有效率为71．0％（22／31例），其中SAA／VSAA和NSAA的总有效率分别为65．0％（13／20例）和81．8％（9／11例），总有效率差异无显著性。ATG—F相关类过敏反应、血清病和感染等不良反应发生率分别为35．5％（11例）、32．3％（10例）和16．1％（5例），均得以及时控制，无治疗相关死亡。动态监测显示，ATG—F治疗期间ALC下降幅度达到70％～80％，并可持续较长时间，直至治疗后90d仍未恢复到治疗前ALC计数水平的50％。结论本研究显示，ATG-F通过大幅度清除ALC以达到抑制异常T细胞之效应，治疗儿童再障疗效显著；采用审慎实施与防护措施，可有效防治ATG相关不良反应。ATG—F可以作为儿童再障IST治疗的ATG剂型选择之一。Objectiv （Fresenius, ATG-F） in the e To explore the clinical efficacy of Rabbit antithymocyte globulin treatment of children with aplastic anemia （AA）. Methods There were 31 cases of children with aplastic anemia in our study, which including 4 with very severe aplastic anemia （VSAA） , 16 with severe aplastic anemia （SAA） and 11 with blood transfusion dependent non-severe aplastic anemia （NSAA）, combined immunosuppressive therapies （IST） comprising ATG-F and cyclosporin A （CSA） were employed. The comprehensive measures for prevention and control of ATG related adverse reaction were applied and the number of actural lymphocyte counts （ALC） in peripheral blood of the patients were monitored during and after the ATG-F therapy. Results According to the international criteria for curative response to AA （Camitta）, the total response rates were 70.97% （22/ 31 cases） , in which SAA/VSAA and NSAA of the total e response rates were 65% （13/20 cases） and 81.82% （19/11 cases） respectively, the statistics showed there were no significant difference in total response rates in patients with SAA/VSAA and NSAA. The ATG related adverse reaction including anaphylaxis, serum sickness and infection occurred in 35.48% of cases （ 11 cases）, 32.26% （ 10 cases） and 16.13% （5 cases） respectively. All the adverse reactions were well controlled, and no ATG treatment related death developed. ALC decreased to 70-80% during the ATG-F therapy, and maintained to at least 50% less than that before therapy, for up to 90 days after treatment ALC has not restored to the level of 50% before treatment. Conclusions Our data showed that ATG-F therapy could reach positive outcomes in treatment of children with aplastic anemia by significantly inhibiting abnormal T cells, via eliminating ALC in the peripheral blood, with controllable toxicities.

关 键 词：贫血 再生障碍性 免疫抑制治疗 抗人T-细胞兔免疫球蛋白 疗效 淋巴 细胞绝对计数 

分 类 号：R725.5[医药卫生—儿科]