Therapeutic effect of clofarabine in children with relapsed or refractory acute lymphoblastic leukemia

目的 探讨氯法拉滨应用于儿童复发/难治性急性淋巴细胞性白血病的疗效和不良反应.方法 26例复发/难治性急性淋巴细胞性白血病患儿接受氯法拉滨单药治疗,男22例,女4例,中位年龄9.5岁(4～17岁).患儿均接受连续5d静脉滴注氯法拉滨(52 mg/m2,每次超过2 h),其中13例患儿接受连续2次氯法拉滨单药化疗,1例患儿接受连续3次氯法拉滨单药化疗.结果 26例患儿第1次氯法拉滨化疗后11例(42％)获完全缓解,7例(27％)获部分缓解,总有效率69％,8例(31％)未缓解.26例患儿中,13例继续给予第2次氯法拉滨化疗后11例(85％)获完全缓解,1例(8％)部分缓解,1例(8％)未缓解.其中1例患儿接受3次氯法拉滨化疗均获完全缓解.化疗的不良反应主要为中性粒细胞减少、感染、肝功能损害、胃肠道反应,无化疗相关死亡病例.结论 氯法拉滨治疗儿童复发/难治性急性淋巴细胞性白血病有一定疗效,不良反应可以耐受,是一种新的治疗选择.To explore the efficacy and adverse effects of clofarabine for relapsed/refractory acute lymphoblastic leukemia in children.Twenty-six pediatric patients with relapsed/refractory acute lymphoblastic leukemia were treated with clofarabine. There were 22 males and 4 females, with a mean age of 9.5 years (ranging from 4 to 17 years). They received clofarabine 52 mg/m2 intravenously over 2 hours daily for 5 days. Thirteen patients received two cycles and one patient received three cycles.In the first cycle of clofarabine, complete remission was obtained in 11 children (42%) and partial remission was obtained in 7 children (27%). Eight children (31%) were considered unresponsive. In the second cycle, 11 (85%) of the 13 children obtained complete remission, 1 (8%) partial remission and 1 (8%) was unresponsive. One child received three cycles and obtained complete remission in each cycle. The common adverse events were myelosuppression, infection, liver dysfunction and gastrointestinal adverse reactions. There were no chemotherapy-related deaths.Clofarabine is effective in the treatment of children with relapsed/refractory acute lymphoblastic leukemia and its adverse effects can be tolerated. Clofarabine could be a promising new treatment for relapsed/refractory acute lymphoblastic leukemia.PubMed中文核心期刊要目总览(PKU)中国科技核心期刊(ISTIC)中国科学引文数据库(CSCD)06444-4471