Beijing Key Laboratory of Pediatric Hematology Oncology, Beijing, China.
National Key Discipline of Pediatrics (Capital Medical University), Beijing, China.
J Clin Oncol. 2021 Oct 1;39(28):3161-3170. doi: 10.1200/JCO.20.03096. Epub 2021 Jun 2.
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 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 ( = .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 ( = .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.
砷剂联合全反式维 A 酸（ATRA）是成人急性早幼粒细胞白血病（APL）的标准治疗方法。然而，基于更大的样本量，尚未有研究报道该治疗方法在儿科 APL 患者中的安全性和有效性。
我们在中国 38 家医院进行了一项多中心试验。根据基线白细胞计数和 突变情况，将新诊断的 APL 患者分为两个风险组。标准风险组给予 ATRA 联合三氧化二砷或口服砷剂，不化疗；高风险组给予 ATRA、三氧化二砷或口服砷剂联合低剂量蒽环类药物。主要终点为 2 年无事件生存和总生存。
我们纳入了 193 例 APL 患者。中位随访 28.9 个月后，标准风险组的 2 年总生存率为 99%（95%CI，97 至 100），高风险组为 95%（95%CI，90 至 100）（ =.088）。标准风险组的 2 年无事件生存率为 97%（95%CI，93 至 100），高风险组为 90%（95%CI，83 至 96）（ =.252）。治疗后砷的血浆水平显著升高，治疗期间稳定的有效浓度范围为 42.9 至 63.2ng/mL。此外，治疗结束后 6 个月，血浆、尿液、头发和指甲中的砷水平迅速降至正常。
砷剂联合 ATRA 对儿科 APL 患者有效且安全，尽管仍需要长期随访。
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