Department of Paediatrics, Kyoto Prefectural University of Medicine, Graduate School of Medical Science, Kyoto, Japan.
Br J Haematol. 2012 Oct;159(2):204-10. doi: 10.1111/bjh.12030. Epub 2012 Aug 28.
The acute myeloid leukaemia (AML) 99 trial conducted previously in Japan for the treatment of de novo paediatric AML showed excellent results, with a 5-year overall survival (OS) and event-free survival (EFS) of 75·6% and 61·6%, respectively. To examine reproducibility of these results in another cohort, the outcome of 146 newly diagnosed AML paediatric patients prospectively registered in the Japan Association of Childhood Leukaemia Study (JACLS) from 2003 to 2006 was compared to that of 240 patients in the original AML 99 clinical trial. The 5-year EFS and OS achieved in the new cohort was 66·7 ± 4·0% and 77·7 ± 8·0% respectively, which were comparable to those obtained in the original AML 99 clinical trial, although less frequent core-binding factor (CBF) AML (29·5% vs. 37%) and an almost equal frequency of allogeneic haematopoietic stem cell transplantation (allo-HSCT) during first complete remission (16·5% vs. 19%) were observed. The 5-year EFS in patients with a normal karyotype (NK) (n = 35, 54·9 ± 15·1%) was inferior in the present cohort when compared to the original AML99 trial. This study confirmed the excellent outcome of the original AML99 protocol.
此前在日本开展的治疗初治小儿急性髓细胞白血病（AML）的 AML 99 试验取得了优异的结果，5 年总生存率（OS）和无事件生存率（EFS）分别为 75.6%和 61.6%。为了在另一队列中检验这些结果的重现性，比较了 2003 年至 2006 年日本儿童白血病研究协会（JACLS）前瞻性登记的 146 例新诊断的 AML 儿科患者的结局与原始 AML 99 临床试验中的 240 例患者的结局。新队列中 5 年 EFS 和 OS 分别为 66.7±4.0%和 77.7±8.0%，与原始 AML 99 临床试验中获得的结果相当，尽管 CBF 急性髓细胞白血病（AML）（29.5%比 37%）的频率较低，并且首次完全缓解时同种异体造血干细胞移植（allo-HSCT）的频率几乎相等（16.5%比 19%）。核型正常（NK）（n=35）患者的 5 年 EFS（54.9±15.1%）较原始 AML99 试验差。本研究证实了原始 AML99 方案的优异结果。
