摘要急性髓细胞白血病(AML)为成年人最常见的急性白血病,目前AML治疗仍以联合化疗及造血干细胞移植(HSCT)为主.近年来,随着人类基因组计划的完成、第二代基因测序技术等分子生物学领域的进展,为开发AML药物带来了新的方向.目前,AML的新药包括FMS样酪氨酸激酶(FLT)3抑制剂、异柠檬酸脱氢酶(IDH)1/2抑制剂、组蛋白去乙酰化酶(HDAC)抑制剂、针对表观遗传学药物、新一代的化疗药物如CPX-351,还包括以CD33抗体为代表的抗体免疫治疗及以嵌合抗原受体T细胞(CAR-T)免疫疗法为代表的细胞免疫治疗.本文重点对近5年来相关临床试验中取得较大进展,并且有望应用于临床AML治疗的新药研究进展进行综述.

abstractsAcute myeloid leukemia (AML) is the most common subtype of leukemia in adult.Standard combined chemotherapy and hematopoietic stem cell transplantation (HSCT) are the major treatment of AML.In recent years,advances in molecular biology area,such as the completion of the human genome project,and next-generation sequencing,have brought new direction to anti-AML drugs,such as FMS-like tyrosine kinase (FLT) inhibitor,isocitrate dehydrogenase (IDH) 1/2 inhibitor,histone deacetylase (HDAC) inhibitor,epigenetic drugs,novel chemotherapeutic drug CPX-351,as well as CD33 antibody as the representative of antibody immunotherapy and chimeric antigen receptor T cell (CAR-T) immunotherapy.In this brief review,we presents an overview of novel agents currently in clinical trials that have data published or be hopeful to be used in clinic in treatment of AML.