Liu Li-Yuan, Wang Hui-Jun, Zhang Li, Jing Li-Ping, Zhou Kang, Yang Dong-Lin, Li Hong-Qiang, Liu Qing-Guo, Yan Zhang-Song, Liu Yong-Ze, Wu Yu-Hong, Chu Yu-Lin, Zhang Feng-Kui
Institute of Hematology and Blood Diseases Hospital, CAMS&PUMC, Tianjin 300020, China.
Zhonghua Xue Ye Xue Za Zhi. 2009 Nov;30(11):749-53.
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).
Seventy-one childhood SAA patients treated with rabbit ATG + CsA as first line therapy were retrospectively analysed.
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).
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.
分析兔抗胸腺细胞球蛋白（ATG）和环孢素A（CsA）作为儿童重型再生障碍性贫血（SAA）一线治疗方案的疗效及副作用。
回顾性分析71例接受兔ATG + CsA一线治疗的儿童SAA患者。
纳入71例SAA患者，其中38例为SAA，33例为极重型再生障碍性贫血（VSAA）。中位年龄为12岁。这些患者中，3例在免疫抑制治疗（IST）后3个月内死亡。总缓解率为67.6%（46/68），输血独立的中位时间为53天。33例患者（48.5%）在IST后3个月获得缓解，45例（67.2%）在6个月获得缓解。年龄小于10岁、10 - 15岁和15 - 18岁患者的缓解率分别为57.7%（15/26）、56.5%（13/23）和94.7%（18/19）。60例患者在IST期间出现血清病。3例患者复发，另外3例无反应患者接受IST再治疗，1例患者进展为骨髓增生异常综合征（MDS）。
兔ATG联合CsA作为儿童SAA/VSAA的一线治疗方案可使总缓解率达67.6%，且不良反应较小。
