小儿再生障碍性贫血疗效总结

Evaluation on the therapeutic efficacy of the combined immunosuppression therapy in children with aplastic anemia

目的总结72例小儿再生障碍性贫血(以下简称再障)的疗效。方法72例患儿中30例慢性再障,42例急性再障。慢性再障给予安雄、CsA治疗,急性再障给予安雄、CsA、大剂量丙种球蛋白、ATG治疗,其中2例接受异基因造血干细胞移植。结果72例再障患儿中慢性再障组和急性再障组在性别和发病年龄上无明显差异(P>0.05)。30例慢性再障病人中17例进步或治愈,11例无效或进展,无死亡病例;42例急性再障病人中14例进步或治愈,20例无效或进展,7例死亡,两组病人达进步或治愈者均无复发病例,两组总有效率有显著差异(56.67%vs33.33%P<0.05),两组病人总的生存率统计学上有差异(100%vs83.33%P<0.05)。两组出现疗效的时间无差异(P>0.05),而在中位输血时间间隔上,两组有显著差异(P<0.005)。在42例急性再障病人中,28例接受ATG治疗,14例未接受ATG治疗,ATG组中有11例出现进步或治愈,未接受ATG组中仅3例出现疗效,两组有明显统计学差异(42.13%vs21.43%P<0.05),72例再障病人中有3例转变为AML(分别为M2,M3,M5),2例接受了异基因造血干细胞移植后治愈。结论慢性再障治疗疗效及生存率明显高于急性再障,而联合免疫治疗是治疗急性再障除了骨髓移植外的最有效的方法。

Objective Beginning from Aug. 1996 - Setp. 2004. 85 children with aplastic anemia aged from 1 - 14 years were enrolled in hematology dept. 72 patients were followed up until Mar. 2005. To analyse 72 children with aplastic anemia, the therapeutic efficacy of the combined immunosuppression therapy and the prognosis of these patients was evaluated. Methods In total of 72 children with aplastic anemia, 30 cases with chronic aplastic anemia received androgen, CSA, while 42 cases with acute aplastic anemia received androgen, CSA, high-dose IGIV, ATG. 2 cases with acute aplastic anemia received Allo-genic peripheral blood stem cell transplantion（Allo-PBSCT）. The clinical characteristics, therapeutic effect, relapse rate, mortality, the incidence of second malignant diseases and survival rate were analysed. The standard for diagnosis and therapentic effect evaluation was based on the criteria the 4 th Chinese national conference on aplastic anemia. Results There was no statistical difference in sex and age distribution be- tween the children with chronic aplastic anemia or acute aplastic anemia. （P 〉 0.05）. 17 cases of 30 cases with chronic group had got improvement or cure, 11 cases with chronic type failed, with no case died. 14 cases from acute group had achieved improvement or cure, 20 cases failed, 7 cases died, with no case relapsed in both groups. The effective rate in chronic group was higher than that in acute group（56.67% vs 33.33% , P 〈0.05 ）. The overall survival rate was different between these two groups （100% vs 83.33% P 〉 0.05 ）. The time of effect occurred was of no marked difference between these two groups （ P 〉 0.05 ）. While the average interval of transfusion was of significant difference between these two groups （ P 〈 0. 005 ）. 28 cases of 42 cases with acute group received ATG, with 11 cases achieved improvement or cure, while only 3 cases without ATG treatment had effect with statistical difference between ATG group and no ATG group （42.13% vs 21.43%, P 〈0.05）. 3 of 72 patients with aplastic anemia developed AML （M1, M3, M5 respectively） , then they received chemotherapy, while 2 patients were cured after receivine Allo-PBSCT. Conclusion The therapeutic effect and survival rate in children with chronic aplastic anemia was significantly higher than that of children with acute aplastic anemia, indicating the combined immunosuppressive therapy was the most effective therapy in addition to the Allo-PBSCT in the treatment of children with acute aplastic anemia.