一线应用兔抗胸腺细胞球蛋白联合环孢素治疗儿童重型再生障碍性贫血

目的 评价兔抗胸腺细胞球蛋白(ATG)联合环孢素(CsA)一线治疗儿童重型再生障碍性贫血(SAA)的疗效及不良反应.方法 回顾性分析我院贫血诊疗中心一线使用兔ATG联合CsA方案治疗初治SAA患儿资料.结果 71例SAA患儿,中位年龄12岁.其中SAA 38例,极重型AA(VSAA)33例.除3例接受治疗后早期死亡外,46例(67.6%)获得治疗反应,患儿脱离输注血液制品的中位时间为53 d.免疫抑制治疗(IST)后3个月33例(48.5%)、6个月45例(67.2%)患儿获得治疗反应.年龄＜10岁、10～15岁、＞15～18岁者治疗有效率分别为57.7%、56.5%和94.7%.3例复发.3例无效者接受二次IST,1例治疗后进展为骨髓增生异常综合征.60例患儿出现血清病反应.结论 一线应用兔ATG联合CsA治疗儿童SAA/VSAA,治疗反应率为67.6%,不良反应轻微.

Combination of rabbit antithymocyte globulin plus cyclosporin A as first-line immunosuppressive therapy for the childhood with severe aplastic anemia

Objective To analyse the efficacy and side-effects of rabbit antithymocyte globulin (ATG) and cyclosporin A(CsA) as the first-line therapy for childhood severe aplastic anemia(SAA).Methods Seventy-one childhood SAA patients treated with rabbit ATG + CsA as first line therapy were retrospectively analysed.Results Seventy-one SAA patients,including 38 SAA and 33 very severe aplastic anemia (VSAA),were enrolled.The median age was 12 years.Of these patients,3 died within 3 months after the immunosuppressive therapy(IST).The overall response rate was 67.6% (46/68) and the median time to transfusion independent was 53 days.Thirty-three patients(48.5%) obtained remission in 3 months after the IST and 45 (67.2%) in 6 months.The response rates were 57.7% (15/26),56.5% (13/23) and 94.7% (18/19) for patients less than 10 years old,10-15 year-old and 15-18 year-old,respectively.Sixty patients suffered from serum sickness on the IST.Three patients relapsed and another 3 unrespond patients received retreatment of IST,and one patient progressed to myelodysplastic syndromes(MDS).Conclusion Rabbit ATG in combination with CsA as first line therapy for childhood SAA/VSAA can lead to overall response rate of 67.6% with minor adverse effects.