Department of Paediatrics, Section of Haematology, Oncology, and Bone Marrow Transplantation, University of Colorado Denver School of Medicine, Aurora, CO, USA.
Br J Haematol. 2010 Nov;151(4):295-311. doi: 10.1111/j.1365-2141.2010.08282.x. Epub 2010 Aug 31.
Modifications to the treatment of acute lymphoblastic leukaemia (ALL) in children have led to a dramatic increase in survival in the past 40 years. Despite this success, a significant subset of paediatric leukaemia patients either relapse or fail to ever achieve a complete remission. Additionally, some patients necessitate treatment with intensified chemotherapy regimens due to clinical or laboratory findings which identify them as high risk. These patients are unlikely to respond to further minor adjustments to the dosing or timing of administration of the same chemotherapy medications. Many novel targeted therapies for the treatment of childhood ALL provide potential mechanisms to further improve cure rates, and provide the possibility of minimizing toxicity to non-malignant cells, given their specificity to malignant cell phenotypes. This article explores many of the potential targeted therapies in varying stages of development, from those currently in clinical trials to those still being refined in the research laboratory.
过去 40 年来，对儿童急性淋巴细胞白血病 (ALL) 的治疗方法的改进导致了生存率的显著提高。尽管取得了这一成功，但仍有相当一部分儿科白血病患者要么复发，要么从未完全缓解。此外，由于临床或实验室发现将其确定为高风险，一些患者需要接受强化化疗方案治疗。这些患者不太可能对同一化疗药物的剂量或给药时间的进一步微调做出反应。许多针对儿童 ALL 的新型靶向治疗为进一步提高治愈率提供了潜在的机制，并有可能最大限度地减少对非恶性细胞的毒性，因为它们对恶性细胞表型具有特异性。本文探讨了许多处于不同开发阶段的潜在靶向治疗方法，从目前正在临床试验中的方法到仍在研究实验室中进行改进的方法。
