儿童非高危急性早幼粒细胞白血病诱导期加一剂蒽环类治疗的疗效和安全性SCCLG/SCCCG-APL协作组

Efficacy and safety of adding a dose of anthracyclines for induction treatment in pediatric non high-risk acute promyelocytic leukemia

目的成人非高危急性早幼粒细胞白血病(NHR-APL)的去化疗诱导治疗,其疗效和安全性在儿童患者中尚未得到验证,需开展儿童的研究。方法数据来自华南儿童APL协作组的前瞻性研究资料,≤16岁、完成诱导治疗的NHR-APL患儿纳入分析。在砷剂+全反式维甲酸诱导治疗的基础上,比较加或不加1剂量去甲氧柔红霉素/米托蒽醌10mg/m^(2)(化疗或无化疗组)的缓解情况和不良事件率。结果化疗组139例,无化疗组27例,两组的诱导治疗结果比较:达血液学完全缓解的中位天数分别为26.0(22.0,31.5)d和31.0(27.5,35.0)d(P=0.002);分子学完全缓解分别有29.5%和3.7%(P=0.005);外周血白细胞计数>10×10^(9)/L分别有43.2%和74.1%(P=0.003);最高白细胞中位数分别为8.6(IQR 4.9,18.4)×10^(9)/L和22.7(IQR 16.0,41.7)×10^(9)/L(P<0.001);确诊分化综合征分别有5.8%和33.3%(P<0.001);出凝血事件发生率分别为14.4%和51.9%(P<0.001)。感染、颅内高压、心和肝功能异常等不良事件的发生率两组相似(均P>0.1)。结论儿童NHR-APL可能有别于成人患者,与无化疗比较,诱导治疗加1剂量蒽环类疗效更好,并发症的风险更低。

Objective The efficacy and safety of chemotherapy-free therapy for adult non high-risk acute promyelocytic leukemia(NHR-APL)have not been validated in children,and pediatric research is needed.Methods The data came from the prospective study of the South China Children′s Leukemia/Cancer Group,and NHR-APL patients who completed induction therapy were included in the analysis.The remission and adverse event rates with or without 1 dose of idarubicin/mitoxantrone 10mg/m^(2)(chemotherapy or non-chemotherapy group)were compared,on the basis of induction therapy with arsenic and all trans retinoic acid.Results There were 139 patients in the chemotherapy group and 27 patients in the non chemotherapy group.Comparison of induction results between chemotherapy and non chemotherapy groups:the median time of hematological complete remission were 26.0(22.0,31.5)days and 31.0(27.5,35.0)days respectively(P=0.002),and there were 29.5%and 3.7%of cases achieved molecular complete remission(P=0.005).The incidence of white blood cells(WBC)>10×10^(9)/L during induction was 43.2%and 74.1%(P=0.003).The median highest WBC was 8.6(4.9,18.4)×10^(9)/L,and 22.7(16.0,41.7)×10^(9)/L(P<0.001).In two groups,5.8%and 33.3%of cases were diagnosed with differentiation syndrome,respectively(P<0.001).The incidence of coagulation events was 14.4%and 51.9%(P<0.001).In addition,the incidence of adverse events such as infection,intracranial hypertension,heart and liver disfunction,and gastrointestinal symptoms was similar in the two groups(All P>0.1).Conclusions Pediatric APL may be different from adult patients.Compared to non chemotherapy regimen,NHR APL patients only received one dose of anthracycline during induction have better induction response and lower complications.