Pediatric Hematology and Oncology, Department of Pediatrics, University Hospital "Carl Gustav Carus", TU Dresden, Dresden, Germany.
Institute for Medical Informatics and Biometry, Faculty of Medicine "Carl Gustav Carus", TU Dresden, Dresden, Germany.
Leukemia. 2018 Jul;32(7):1657-1669. doi: 10.1038/s41375-018-0179-9. Epub 2018 Jun 20.
A total of 156 patients (age range 1.3-18.0 years, median 13.2 years; 91 (58.3%) male) with newly diagnosed CML (N = 146 chronic phase (CML-CP), N = 3 accelerated phase (CML-AP), N = 7 blastic phase (CML-BP)) received imatinib up-front (300, 400, 500 mg/m, respectively) within a prospective phase III trial. Therapy response, progression-free survival, causes of treatment failure, and side effects were analyzed in 148 children and adolescents with complete data. Event-free survival rate by 18 months for patients in CML-CP (median follow-up time 25 months, range: 1-120) was 97% (95% CI, 94.2-99.9%). According to the 2006 ELN-criteria complete hematologic response by month 3, complete cytogenetic response (CCyR) by month 12, and major molecular response (MMR) by month 18 were achieved in 98, 63, and 59% of the patients, respectively. By month 36, 86% of the patients achieved CCyR and 74% achieved MMR. Thirty-eight patients (27%) experienced imatinib failure because of unsatisfactory response or intolerance (N = 9). In all, 28/148 patients (19%) underwent stem cell transplantation (SCT). In the SCT sub-cohort 2/23 patients diagnosed in CML-CP, 0/1 in CML-AP, and 2/4 in CML-BP, respectively, died of relapse (N = 3) or SCT-related complications (N = 2). This large pediatric trial extends and confirms data from smaller series that first-line imatinib in children is highly effective.
共有 156 名新诊断为 CML 的患者（年龄 1.3-18.0 岁，中位数 13.2 岁；91 名男性（58.3%））接受了伊马替尼一线治疗（分别为 300、400、500mg/m2），这些患者来自一项前瞻性 III 期试验。对 148 名具有完整数据的儿童和青少年进行了治疗反应、无进展生存、治疗失败的原因和副作用分析。CML-CP 患者（中位随访时间 25 个月，范围 1-120）18 个月时无事件生存率为 97%（95%CI，94.2-99.9%）。根据 2006 年 ELN 标准，3 个月时达到完全血液学缓解，12 个月时达到完全细胞遗传学缓解（CCyR），18 个月时达到主要分子缓解（MMR）的患者分别为 98%、63%和 59%。36 个月时，86%的患者达到 CCyR，74%的患者达到 MMR。38 名患者（27%）因反应不满意或不耐受而发生伊马替尼失败（N=9）。共有 28/148 名患者（19%）接受了干细胞移植（SCT）。在 SCT 亚组中，2/23 名被诊断为 CML-CP 的患者、0/1 名被诊断为 CML-AP 的患者和 2/4 名被诊断为 CML-BP 的患者分别因复发（N=3）或与 SCT 相关的并发症（N=2）而死亡。这项大型儿科试验扩展并证实了来自较小系列的研究数据，即儿童一线使用伊马替尼具有高度疗效。
