CAJ | 학술논문

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.
목적 평개토항흉선세포구단백(ATG)연합배포소(CsA)일선치료인동중형재생장애성빈혈(SAA)적료효급불량반응.방법 회고성분석아원빈혈진료중심일선사용토ATG연합CsA방안치료초치SAA환인자료.결과 71례SAA환인,중위년령12세.기중SAA 38례,겁중형AA(VSAA)33례.제3례접수치료후조기사망외,46례(67.6%)획득치료반응,환인탈리수주혈액제품적중위시간위53 d.면역억제치료(IST)후3개월33례(48.5%)、6개월45례(67.2%)환인획득치료반응.년령＜10세、10～15세、＞15～18세자치료유효솔분별위57.7%、56.5%화94.7%.3례복발.3례무효자접수이차IST,1례치료후진전위골수증생이상종합정.60례환인출현혈청병반응.결론 일선응용토ATG연합CsA치료인동SAA/VSAA,치료반응솔위67.6%,불량반응경미.
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.