Arsenic combined with all-trans retinoic acid (ATRA) is the standard of care for adult acute promyelocytic leukemia (APL). However, the safety and effectiveness of this treatment in pediatric patients with APL have not been reported on the basis of larger sample sizes.We conducted a multicenter trial at 38 hospitals in China. Patients with newly diagnosed APL were stratified into two risk groups according to baseline WBC count and FLT3-ITD mutation. ATRA plus arsenic trioxide or oral arsenic without chemotherapy were administered to the standard-risk group, whereas ATRA, arsenic trioxide, or oral arsenic plus reduced-dose anthracycline were administered to the high-risk group. Primary end points were event-free survival and overall survival at 2 years.We enrolled 193 patients with APL. After a median follow-up of 28.9 months, the 2-year overall survival rate was 99% (95% CI, 97 to 100) in the standard-risk group and 95% (95% CI, 90 to 100) in the high-risk group (P = .088). The 2-year event-free survival was 97% (95% CI, 93 to 100) in the standard-risk group and 90% (95% CI, 83 to 96) in the high-risk group (P = .252). The plasma levels of arsenic were significantly elevated after treatment, with a stable effective level ranging from 42.9 to 63.2 ng/mL during treatment. In addition, plasma, urine, hair, and nail arsenic levels rapidly decreased to normal 6 months after the end of treatment.Arsenic combined with ATRA is effective and safe in pediatric patients with APL, although long-term follow-up is still needed.
Huyong Zheng, Hui Jiang, Shaoyan Hu, Ning Liao, Diying Shen, Xin Tian, Guoping Hao, Runming Jin, Jianxin Li, Yongjun Fang, Xiuli Ju, Ansheng Liu, Ningling Wang, Xiaowen Zhai, Jiashi Zhu, Qun Hu, Limin Li, Wei Liu, Lirong Sun, Li Wang, Yunpeng Dai, Xiaoqin Feng, Fu Li, Hui Liang, Xinhui Luo, Mei Yan, Qingning Yin, Yan Chen, Yueqin Han, Lijun Qu, Yanling Tao, Hui Gao, Zhixu He, Limin Lin, Jixia Luo, Kaili Pan, Jingrong Zhang, Rong Zhang, Min Zhou, Yuanyuan Zhang, Linya Wang, Ruidong Zhang, Peifang Xiao, Yayun Ling, Xiaoxia Peng, Yaguang Peng, Tianyou Wang, Chinese Children's Leukemia Group