Department of Hematology, Zhongda Hospital, School of Medicine, Southeast University, Institute of Hematology Southeast University, Nanjing, China; Department of Hematology, Xuzhou Central Hospital, Xuzhou, China.
Department of Hematology, Zhongda Hospital, School of Medicine, Southeast University, Institute of Hematology Southeast University, Nanjing, China.
Bosn J Basic Med Sci. 2021 Aug 1;21(4):409-421. doi: 10.17305/bjbms.2020.5485.
Despite advances in the understanding of disease pathobiology, treatment for relapsed or refractory acute myeloid leukemia (R/R AML) remains challenging. The prognosis of R/R AML remains extremely poor despite chemotherapy and bone marrow transplants. Discoveries on recurrent and novel genetic mutations, such as FLT3-ITD and IDH1/IDH2, critical signaling pathways, and unique molecular markers expressed on the surface of leukemic cells have been under investigation for the management of R/R AML. Other than monoclonal antibodies, diabodies, and triabodies are new targeted therapies developed in recent years and will be the new direction of immunotherapy. Targeted agents combined intensive regimens can be viable options for salvage therapy and as bridges to allogeneic transplant. Future directions will focus on novel, efficient and targeted combinations, low-toxicity maintenance, and individualized precision strategies. Here, we review the major recent advances of targeted therapies in the treatment of R/R AML.
尽管对疾病发病机制的理解取得了进展，但复发或难治性急性髓系白血病（R/R AML）的治疗仍然具有挑战性。尽管进行了化疗和骨髓移植，但 R/R AML 的预后仍然极差。对复发性和新型基因突变（如 FLT3-ITD 和 IDH1/IDH2）、关键信号通路以及白血病细胞表面表达的独特分子标记的研究一直在进行中，以用于 R/R AML 的治疗。除了单克隆抗体外，近年来还开发了双抗体和三抗体等新型靶向治疗药物，它们将成为免疫治疗的新方向。靶向药物联合强化方案可能是挽救性治疗和异基因移植桥梁的可行选择。未来的方向将集中在新型、高效和靶向的联合治疗、低毒性维持以及个体化精准策略上。在这里，我们回顾了靶向治疗在 R/R AML 治疗方面的主要最新进展。
