Queensland Children's Medical Research Institute, The University of Queensland, Brisbane, Australia.
Leukemia. 2013 Jul;27(7):1451-60. doi: 10.1038/leu.2013.106. Epub 2013 Apr 8.
Significant improvements in survival for children with acute myeloid leukaemia (AML) have been made over the past three decades, with overall survival rates now approximately 60-70%. However, these gains can be largely attributed to more intensive use of conventional cytotoxics made possible by advances in supportive care, and although over 90% of children achieve remission with frontline therapy, approximately one third in current protocols relapse. Furthermore, late effects of therapy cause significant morbidity for many survivors. Novel therapies are therefore desperately needed. Early-phase paediatric trials of several new agents such as clofarabine, sorafenib and gemtuzumab ozogamicin have shown encouraging results in recent years. Due to the relatively low incidence of AML in childhood, the success of paediatric early-phase clinical trials is largely dependent upon collaborative clinical trial design by international cooperative study groups. Successfully incorporating novel therapies into frontline therapy remains a challenge, but the potential for significant improvement in the duration and quality of survival for children with AML is high.
在过去的三十年中，儿童急性髓系白血病（AML）的生存率有了显著提高，总体生存率现在约为 60-70%。然而，这些进展在很大程度上归因于支持性治疗方面的进步使得常规细胞毒素的使用更加密集，尽管超过 90%的儿童通过一线治疗达到缓解，但目前方案中约有三分之一的患者复发。此外，治疗的后期影响导致许多幸存者出现严重的发病率。因此，迫切需要新的治疗方法。近年来，几种新药物如克拉屈滨、索拉非尼和吉妥珠单抗奥佐米星的儿科早期试验显示出令人鼓舞的结果。由于儿童 AML 的发病率相对较低，儿科早期临床试验的成功在很大程度上取决于国际合作研究组的协作临床试验设计。成功地将新疗法纳入一线治疗仍然是一个挑战，但提高 AML 儿童的生存时间和质量的潜力很高。
