Kutny Matthew A, Gregory John, Feusner James H
Department of Pediatrics, Division of Hematology/Oncology, University of Alabama at Birmingham, Birmingham, AL, USA.
UMDNJ - New Jersey Medical School, Atlantic Health System, Goryeb Children's Hospital, Morristown, NJ, USA.
Best Pract Res Clin Haematol. 2014 Mar;27(1):69-78. doi: 10.1016/j.beha.2014.04.007. Epub 2014 Apr 26.
Acute promyelocytic leukaemia (APL) in children and adolescents shares many features with APL in adults. There are important distinctions, however, between these age groups in the presentation, complications and treatment outcomes. Paediatric patients are more likely to present with high risk features including elevated WBC count or microgranular variant (M3v). Yet the early death rate is lower in paediatric patients compared to adult patients. Overall outcomes such as CR, OS and EFS appear similar in paediatric and adult patients treated on similar regimens except that very young children may have a higher risk of relapse. While contemporary studies have clearly demonstrated improved survival in adults receiving ATO therapy, currently there is more limited data on the role of ATO in paediatric patients. Here we highlight the similarities and important distinctions between paediatric and adult APL while reviewing available data on treatment of paediatric APL.
儿童和青少年急性早幼粒细胞白血病（APL）与成人APL有许多共同特征。然而，在临床表现、并发症和治疗结果方面，这些年龄组之间存在重要差异。儿科患者更有可能表现出高风险特征，包括白细胞计数升高或微颗粒变异型（M3v）。然而，与成人患者相比，儿科患者的早期死亡率较低。除了非常年幼的儿童可能有较高的复发风险外，接受相似治疗方案的儿科和成人患者的总体结局，如完全缓解（CR）、总生存期（OS）和无事件生存期（EFS）似乎相似。虽然当代研究清楚地表明接受三氧化二砷（ATO）治疗的成人患者生存率有所提高，但目前关于ATO在儿科患者中的作用的数据更为有限。在此，我们在回顾儿科APL治疗可用数据的同时，强调儿科和成人APL之间的异同。
