Department of Hematology, First Affiliated Hospital, Harbin Medical University, Harbin, China.
Blood. 2010 Mar 4;115(9):1697-702. doi: 10.1182/blood-2009-07-230805. Epub 2009 Dec 22.
The aim of this study was to determine the efficacy and safety of treatment of pediatric acute promyelocytic leukemia (APL) with single-agent arsenic trioxide (ATO). A total of 19 children (< or = 15 years of age) with newly diagnosed APL were treated with single-agent ATO for remission induction and postremission therapy. Seventeen of the children (89.5%) achieved complete hematologic remission, and 2 early deaths occurred from intracranial hemorrhage. ATO-induced leukocytosis was observed in 13 (68.4%) patients. Other ATO-related toxicities were minimal and transient. Postremission ATO therapy continued for 3 years; the most common side effect was ATO-induced neutropenia. With a median follow-up of 53 months (range, 23-76 months), the calculated 5-year overall survival and event-free survival were 83.9% and 72.7%, respectively, which are comparable with results achieved by the use of ATRA plus chemotherapy, which is the standard therapy for APL. No chronic arsenic toxicity or second malignancies were found during the follow-up period, and arsenic retention was not significant in patients off treatment more than 24 months. ATO resistance was observed in only 1 patient with a complex karyotype. The results indicate the high efficacy and safety of single-agent ATO regimens in the treatment of children with de novo APL.
本研究旨在确定用三氧化二砷（ATO）单药治疗儿童急性早幼粒细胞白血病（APL）的疗效和安全性。19 例新诊断为 APL 的儿童（年龄≤15 岁）接受ATO 单药诱导缓解和缓解后治疗。17 例儿童（89.5%）达到完全血液学缓解，2 例早期死亡是由颅内出血引起的。13 例（68.4%）患者出现 ATO 诱导的白细胞增多。其他与 ATO 相关的毒性反应轻微且短暂。缓解后 ATO 治疗持续 3 年；最常见的副作用是 ATO 诱导的中性粒细胞减少症。中位随访时间为 53 个月（范围，23-76 个月），计算出的 5 年总生存率和无事件生存率分别为 83.9%和 72.7%，与 ATRA 联合化疗的结果相当，后者是 APL 的标准治疗方法。在随访期间未发现慢性砷毒性或第二恶性肿瘤，且停药超过 24 个月的患者砷保留量不显著。仅 1 例具有复杂核型的患者观察到 ATO 耐药。结果表明，ATO 单药方案治疗新诊断的 APL 儿童具有高效、安全的特点。
