Shah Manasi, Agarwal Bharat
Bridgeport Hospital, Yale University School of Medicine, Yale, USA.
Indian J Pediatr. 2008 Aug;75(8):831-7. doi: 10.1007/s12098-008-0155-x. Epub 2008 Sep 4.
Acute myeloid leukemia (AML) is the most common childhood malignancy. AML has therapeutically been difficult to treat. In 2001, the World Health Organization (WHO), in conjunction with the Society for Hematopathology and the European Association of Hematopathology, published a new classification for myeloid neoplasms. A number of chromosomal abnormalities are used to predict outcome and stratify therapeutic risk groups in children with AML. Recently, alterations in receptor tyrosine kinases, tyrosine phosphatases and in oncogenes such as RAS have been implicated in the pathogenesis of AML. This article aims to review the recent development in diagnosis, treatment and monitoring of AML. Better understanding of the molecular pathogenesis of AML has led to the development of target-specific therapies. Some of the new classes of drugs include monoclonal antibody directed against the CD33 antigen, farnesyltransferase inhibitors (FTI), and FMSlike tyrosine kinase 3 (FLT3) inhibitors. The role of allogenic SCT, particularly whether it should be done during first CR or reserved for second remission, remains the most controversial issue in pediatric AML. There is a need of collaboration with international pediatric cooperative oncology groups and definitive clinical trials in order to establish use of these newer molecules in pediatric populations.
急性髓系白血病(AML)是儿童期最常见的恶性肿瘤。AML在治疗上一直颇具难度。2001年,世界卫生组织(WHO)联合血液病理学协会及欧洲血液病理学协会发布了髓系肿瘤的新分类。多种染色体异常被用于预测AML患儿的预后并对治疗风险组进行分层。最近,受体酪氨酸激酶、酪氨酸磷酸酶以及RAS等癌基因的改变被认为与AML的发病机制有关。本文旨在综述AML在诊断、治疗及监测方面的最新进展。对AML分子发病机制的深入了解推动了靶向特异性疗法的发展。一些新型药物包括针对CD33抗原的单克隆抗体、法尼基转移酶抑制剂(FTI)以及FMS样酪氨酸激酶3(FLT3)抑制剂。异基因造血干细胞移植(SCT)的作用,尤其是应在首次完全缓解(CR)期进行还是留待第二次缓解期进行,仍是儿童AML中最具争议的问题。需要与国际儿科肿瘤协作组合作并开展确定性临床试验,以便在儿科人群中确定这些新型分子的应用。