Department of Internal Medicine, Section of Hematology, Yale School of Medicine, New Haven, Connecticut.
Leukemia Service, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
Curr Opin Oncol. 2021 Nov 1;33(6):658-669. doi: 10.1097/CCO.0000000000000778.
Disease relapse remains the major cause of death in patients with acute myeloid leukemia (AML) and is driven by the persistence of leukemic cells following induction chemotherapy or allogeneic hematopoietic cell transplant (allo-HCT). Maintenance therapies to extend the duration of remission and to improve survival have been proposed for several years with mixed results but have experienced a renaissance recently.
The oral hypomethylating agent CC-486 has been the first agent to show an overall survival (OS) benefit compared with observation in AML patients in remission following intensive chemotherapy who are not proceeding to allo-HCT. In the realm of maintenance therapy following allo-HCT, the FLT3 inhibitor sorafenib has yielded superior results in terms of OS and relapse-free survival in randomized trials compared with observation. Several open questions remain regarding patient selection, timing, duration and safety of maintenance therapies. Various targeted agents are currently tested in clinical trials and could potentially enable an even more individualized therapeutic approach.
Maintenance therapies following intensive chemotherapy or allo-HCT offer a new therapeutic paradigm for an increasing number of AML patients and have been shown to result in an OS benefit in selected patients.
疾病复发仍然是急性髓细胞白血病(AML)患者死亡的主要原因,其由诱导化疗或异基因造血细胞移植(allo-HCT)后白血病细胞的持续存在所驱动。多年来,人们一直提出维持治疗以延长缓解期并改善生存,但结果喜忧参半,但最近又重新兴起。
口服低甲基化剂 CC-486 是第一个与观察相比,在接受强化化疗后缓解且不进行 allo-HCT 的 AML 患者中显示出总生存期(OS)获益的药物。在 allo-HCT 后的维持治疗领域,FLT3 抑制剂索拉非尼在随机试验中在 OS 和无复发生存方面的结果优于观察。关于维持治疗的患者选择、时机、持续时间和安全性仍存在一些悬而未决的问题。目前正在临床试验中测试各种靶向药物,可能使更个体化的治疗方法成为可能。
强化化疗或 allo-HCT 后的维持治疗为越来越多的 AML 患者提供了新的治疗范例,并已在选定患者中显示出 OS 获益。