Leukemia Service, Department of Medicine, Roswell Park Comprehensive Cancer Center , Buffalo, NY, USA.
Expert Rev Clin Pharmacol. 2020 Dec;13(12):1389-1400. doi: 10.1080/17512433.2020.1850255. Epub 2021 Jan 7.
: Acute myeloid leukemia (AML) is a deadly disease associated with poor outcomes. For over four decades, therapeutic options for AML were limited to high dose cytotoxic chemotherapy. Scientific breakthroughs have not only enhanced our understanding of the molecular underpinnings of this disease but also resulted in the development of several targeted therapies with superior efficacy and lesser toxicities than conventional chemotherapy. The FDA approval of small molecule inhibitors for specific AML subsets highlights the importance of genetic and molecular profiling to optimally personalize AML therapy in the modern era. : In this article, we review the medical literature from PubMed on recent FDA approved drugs for AML by their mechanism of action: small molecule inhibitors, antibody-drug conjugate, cytotoxic, and epigenetic agents. We describe how to incorporate these agents into the current treatment paradigm for specific AML patients. : Knowing the molecular characteristics of patients with AML is of utmost importance to plan the best management. There are promising drugs targeting leukemogenesis by various mechanisms. It is important to consider clinical trial options for patients if and when available. We have provided a brief overview of the most promising agents on the horizon for AML therapy.
急性髓系白血病（AML）是一种预后不良的致命疾病。四十多年来，AML 的治疗选择仅限于大剂量细胞毒性化疗。科学的突破不仅增强了我们对这种疾病分子基础的理解，而且还开发了几种靶向治疗方法，其疗效优于传统化疗，毒性更小。FDA 批准针对特定 AML 亚群的小分子抑制剂突出了基因和分子分析在现代个性化 AML 治疗中的重要性。在本文中，我们通过作用机制综述了 PubMed 上最近获得 FDA 批准用于 AML 的药物的医学文献：小分子抑制剂、抗体药物偶联物、细胞毒性药物和表观遗传学药物。我们描述了如何将这些药物纳入特定 AML 患者的当前治疗方案。了解 AML 患者的分子特征对于制定最佳治疗方案至关重要。有多种机制的靶向白血病发生的有前途的药物。如果有临床试验选择，应考虑为患者提供这些选择。我们简要概述了 AML 治疗中最有前途的药物。
