Jiangsu Institute of Hematology, National Clinical Research Center for Hematologic Diseases, The First Affiliated Hospital of Soochow University, Suzhou, China; Institute of Blood and Marrow Transplantation, Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China.
Jiangsu Institute of Hematology, National Clinical Research Center for Hematologic Diseases, The First Affiliated Hospital of Soochow University, Suzhou, China.
Transplant Cell Ther. 2021 Feb;27(2):173.e1-173.e9. doi: 10.1016/j.jtct.2020.10.010. Epub 2020 Dec 11.
The use of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for consolidation therapy in patients with core binding factor (CBF) acute myelogenous leukemia (AML) with intermediate- and adverse-risk genetics remains controversial. We retrospectively analyzed the clinical outcomes of 286 CBF-AML patients with intermediate- and adverse-risk genetics in first complete remission following consolidation with chemotherapy (n = 122), auto-HSCT (n = 27), or allo-HSCT (n = 137) between January 2009 and December 2018 at our center. Patients with allo-HSCT showed superior 5-year overall survival (OS; 74% versus 38% or 49%; P < .001) and progression-free survival (PFS; 74% versus 26% or 49%; P < .001) and lower cumulative incidence of relapse (CIR; 9% versus 69% or 31%; P < .001) compared with chemotherapy alone or auto-HSCT. In the allo-HSCT group, minimal residual disease (MRD) at the second and third months after allo-HSCT could predict relapse in t(8;21) patients (2 months: P = .002; 3 months: P < .001) but not in inv(16) patients. Moreover, positive MRD after 2 courses of consolidation chemotherapy before allo-HSCT was an independent risk factor for survival in CBF-AML patients with intermediate- and adverse-risk genetics, whereas haploidentical donor (haplo-) HSCT could overcome the adverse prognosis (5-year OS, 87%; 5-year PFS, 81%; 5-year CIR, 7%). Allo-HSCT could be the optimal first-line consolidation therapy for patients with intermediate- and adverse-risk genetics, and haplo-HSCT could improve survival for patients with positive MRD after 2 courses of consolidation chemotherapy.
异基因造血干细胞移植（allo-HSCT）用于中危和高危遗传学核心结合因子（CBF）急性髓系白血病（AML）患者的巩固治疗仍存在争议。我们回顾性分析了 2009 年 1 月至 2018 年 12 月期间在我中心接受巩固化疗（n=122）、自体外周血造血干细胞移植（n=27）或 allo-HSCT（n=137）的 286 例中危和高危遗传学 CBF-AML 患者的临床结局。allo-HSCT 组患者 5 年总生存率（OS；74%对 38%或 49%；P<0.001）和无进展生存率（PFS；74%对 26%或 49%；P<0.001）更高，累积复发率（CIR；9%对 69%或 31%；P<0.001）更低，与单纯化疗或自体外周血造血干细胞移植相比。在 allo-HSCT 组中，allo-HSCT 后第 2 和第 3 个月的微小残留病（MRD）可预测 t（8；21）患者的复发（2 个月：P=0.002；3 个月：P<0.001），但不能预测 inv（16）患者的复发。此外，allo-HSCT 前巩固化疗 2 个疗程后阳性 MRD 是中危和高危遗传学 CBF-AML 患者生存的独立危险因素，而单倍体相合供者（haplo-）HSCT 可克服不良预后（5 年 OS，87%；5 年 PFS，81%；5 年 CIR，7%）。allo-HSCT 可作为中危和高危遗传学患者的最佳一线巩固治疗，而对于巩固化疗 2 个疗程后阳性 MRD 的患者，haplo-HSCT 可改善生存。
