文章：

盲人摸象与AML：我们能感受到进展吗？

The blind men and the AML elephant: can we feel the progress?

原文发布日期：2016-05-13

DOI: 10.1038/bcj.2016.33

类型: Review

开放获取: 是

英文摘要：

The pharmacological therapy of non-promyelocytic acute myeloid leukemia (AML) has remained unchanged for over 40 years with an anthracycline–cytarabine combination forming the backbone of induction treatments. Nevertheless, the survival of younger patients has increased due to improved management of the toxicity of therapies including stem cell transplantation. Older patients and those with infirmity that precludes treatment-intensification have, however, not benefited from improvements in supportive care and continue to experience poor outcomes. An increased understanding of the genomic heterogeneity of AML raises the possibility of treatment-stratification to improve prognosis. Thus, efforts to identify agents with non-conventional anti-leukemic effects have paralleled those aiming to optimize leukemia cell-kill with conventional chemotherapy, resulting in a number of randomized controlled trials (RCT). In the last 18 months, RCTs investigating the effects of vosaroxin, azacitidine and gemtuzumab ozogamycin and daunorubicin dose have been reported with some studies indicating a statistically significant survival benefit with the investigational agent compared with standard therapy and potentially, a new era in AML therapeutics. Given the increasing costs of cancer care, a review of these studies, with particular attention to the magnitude of clinical benefit with the newer agents would be useful, especially for physicians treating patients in single-payer health systems.

摘要翻译： 

非早幼粒细胞急性髓系白血病（AML）的药理学治疗40余年来始终未变，蒽环类药物与阿糖胞苷的组合构成诱导治疗的基石。然而，由于包括干细胞移植在内的治疗毒性管理改善，年轻患者的生存率有所提高。但老年患者及因体质虚弱无法接受强化治疗的患者，并未从支持治疗的进步中获益，预后依然较差。对AML基因组异质性认识的深入，为通过治疗分层改善预后提供了可能。因此，在优化传统化疗白血病细胞杀伤效果的同时，研究人员也在积极探索具有非传统抗白血病效应的药物，由此开展了多项随机对照试验（RCT）。过去18个月中，多项关于伏硫辛、阿扎胞苷、吉妥珠单抗奥佐米星及柔红霉素剂量的RCT研究结果陆续公布，部分研究显示试验药物相较标准疗法具有统计学显著的生存获益，这可能预示着AML治疗新时代的到来。鉴于肿瘤治疗费用日益增长，对这些研究进行综述——特别关注新型药物的临床获益程度——将具有重要价值，对在单一支付者医疗系统中执业的医师而言尤为如此。

原文链接：

The blind men and the AML elephant: can we feel the progress?