Wang Shu-Chun, Zou Yao, Liu Xiao-Ming, Chen Xiao-Juan, Zhang Li, Yang Wen-Yu, Liu Tian-Feng, Guo Ye, Chen Yu-Mei, Zhu Xiao-Fan
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College，Tianjin 300020, China.
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College，Tianjin 300020, China,E-mail:
Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2020 Feb;28(1):225-229. doi: 10.19746/j.cnki.issn.1009-2137.2020.01.038.
To analyze the clinical efficacy and side effects of reduced-dose of cyclophosphamide combined cyclosporine A for severe aplastic anemia(SAA) children.
Ten pediatric patients with SAA from January 2008 to May 2012 were enrolled. All the patients were treated with reduced dose of cyclophosphamide combined cyclosporine A. The dose of cyclophosphamide was 30 mg/(kg·d)×4 d, the dose of cyclosporine A gradually increased >15 mg/L accroding to the blood concentration.
The median follow-up time of the 10 pediatric patients was 100 months (6-126 months). Among 10 children with SAA, 4 cases achieved complete response(CR), 3 cases obtained partial response (PR) and the overall response rate was 70%, the remaining 3 cases showed no response (NR). One refractory patient treated by cyclophosphamide was progressed to paroxysmal nocturnal hemoglobinuria(PNH) at 25 months and was dead at 42 months after therapy.
The results show that reduced-dose cyclophosphamide (30 mg/kg·d for 4 consecutive days) combinated with CsA (initial dose 4 mg/kg·d, and drugvallery concentration >150 ng/ml) can make 7 of 10 children with severe aplastic anemia achieve complete response or partial response, and this regimen may be the second line regimen selected for some SAA children.
分析小剂量环磷酰胺联合环孢素A治疗儿童重型再生障碍性贫血（SAA）的临床疗效及副作用。
选取2008年1月至2012年5月的10例儿童SAA患者。所有患者均接受小剂量环磷酰胺联合环孢素A治疗。环磷酰胺剂量为30mg/（kg·d）×4天，环孢素A剂量根据血药浓度逐渐增加至＞15mg/L。
10例儿童患者的中位随访时间为100个月（6 - 126个月）。10例SAA患儿中，4例获得完全缓解（CR），3例获得部分缓解（PR），总缓解率为70%，其余3例无反应（NR）。1例接受环磷酰胺治疗的难治性患者在25个月时进展为阵发性睡眠性血红蛋白尿（PNH），并在治疗后42个月死亡。
结果表明，小剂量环磷酰胺（30mg/kg·d连续4天）联合环孢素A（初始剂量4mg/kg·d，血药谷浓度＞150ng/ml）可使10例儿童重型再生障碍性贫血中的7例获得完全缓解或部分缓解，该方案可能是部分SAA儿童的二线治疗方案。
