The conditioning regimen of a total of 400 mg/m2 of decitabine prior to an allogeneic hematopoietic cell transplant (allo-HSCT) among patients with acute myeloid leukemia (AML) was well-tolerated and demonstrated promising efficacy, according to the results of a phase 2 trial presented at the 2023 Tandem Transplantation and Cellular Therapy Meetings of ASTCT and CIBMTR.

These data suggest there is “potential to become one of the standard conditionings in patients with relapsed/refractory AML undergoing allo-HSCT,” the authors said in their presentation.

The multicenter, single-arm phase 2 study treated 51 patients with relapsed/refractory AML with 200 mg/m2 of decitabine on 2 different days, followed by cytarabine plus busulfan and cyclophosphamide prior to allo-HSCT. The primary endpoints were 1-year and estimated 2-year cumulative incidence of relapse (CIR). The secondary endpoints included non-relapse mortality (NRM), leukemia-free survival (LFS), overall survival (OS), and graft vs host disease and relapse-free survival (GRFS).

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After a median follow-up of 12 months from the time of allo-HSCT, the 1-year CIR was 21.2% and estimated 2-year CIR was 31.5%. The 1- and 2-year NRM were 11.8% and 16.9%, respectively.

A total of 67.0% of patients achieved LFS at 1 year and 51.6% did at 2 years. GRFS was 49.0% and 33.6% at 1 and 2 years, respectively. There were 39.2% of patients who developed grade II-IV acute GVHD by 100 days, and the 1-year rate of moderate to severe chronic GVHD rate was 36.6%.

The total number of chemotherapy cycles received prior to allo-HSCT was significantly associated with a higher CIR (hazard ratio [HR], 1.52; 95% CI, 1.13-2.04; P =.006) in a multivariate analysis. A lower CIR was significantly associated with chronic GVHD (HR, 0.17; 95% CI, 0.03-0.82; P =.027).

The authors concluded that “’mega-dose’ decitabine is a novel conditioning regimen with perfect efficacy and safety.” They added that a phase 3 study of this regimen will begin in the near future.


Lv M, Yan C-H, Sun Y-Q, et al. Allogeneic hematopoietic stem cell transplantation with mega-dose decitabine conditioning for patients with relapsed/refractory AML: a multicenter prospective phase II study. Presented at: 2023 Tandem Meetings Transplantation and Cellular Therapy Meetings of ASTCT and CIBMTR. February 15-19, 2023. Abstract 55.