Funk Christopher Ronald, Waller Edmund K
Department of Hematology & Medical Oncology, Winship Cancer Institute, Emory University School of Medicine, Atlanta, GA.
Department of Hematology & Medical Oncology, Winship Cancer Institute, Emory University School of Medicine, Atlanta, GA.
Clin Lymphoma Myeloma Leuk. 2025 Sep;25(9):615-624. doi: 10.1016/j.clml.2025.04.001. Epub 2025 Apr 8.
Acute myeloid leukemia (AML) is a heterogeneous malignant disorder of myeloid precursor cells, with high relapse rates, particularly in patients who fail to achieve morphological remission after induction therapy. Allogeneic hematopoietic cell transplantation (allo-HCT) can induce durable remissions through the graft-versus-leukemia (GVL) effect, yet current approaches of allo-HSCT often fail, with relapse rates of ∼40% within 6 months post-transplant. Outcomes following allo-HCT are inversely proportional to leukemia burden at the time of transplant. Both morphological relapse (≥5% blasts in the marrow) or minimal residual disease (MRD) positivity predict significantly reduced overall survival rates. Emerging strategies to improve outcomes in patients with high leukemic burden include aggressive bridging therapies (encompassing intensive salvage chemotherapy, hypomethylating agents, targeted inhibitors, and sequential induction-conditioning approaches such as FLAMSA), tailored conditioning regimens, post-transplant maintenance therapy, and innovative graft engineering methods. Graft engineering strategies, such as ORCA-T, which engineers stem cell grafts with a defined ratio of T-regulatory cells to effector T cells, are particularly promising and under evaluation in phase III clinical trials. These approaches aim to improve upon the poor outcomes for patients with persistent/relapsed AML undergoing allo-HCT.
急性髓系白血病(AML)是一种髓系前体细胞的异质性恶性疾病,复发率高,尤其是在诱导治疗后未达到形态学缓解的患者中。异基因造血细胞移植(allo-HCT)可通过移植物抗白血病(GVL)效应诱导持久缓解,但目前的allo-HSCT方法常常失败,移植后6个月内的复发率约为40%。allo-HCT后的结局与移植时的白血病负担成反比。形态学复发(骨髓中原始细胞≥5%)或微小残留病(MRD)阳性均预示总体生存率显著降低。改善高白血病负担患者结局的新兴策略包括积极的桥接治疗(包括强化挽救化疗、低甲基化药物、靶向抑制剂以及如FLAMSA等序贯诱导预处理方法)、定制的预处理方案、移植后维持治疗以及创新的移植物工程方法。移植物工程策略,如ORCA-T,可将干细胞移植物设计为具有特定比例的调节性T细胞与效应T细胞,特别有前景,目前正在III期临床试验中进行评估。这些方法旨在改善接受allo-HCT的持续性/复发性AML患者的不良结局。