Impact of Disease Burden on Clinical Outcomes of AML Patients Receiving Allogeneic Hematopoietic Cell Transplantation: A Study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.

Abstract Pre-transplant detectable measurable residual disease (MRD) is still associated with high risk of relapse and poor outcomes in acute myeloid leukemia (AML). We aimed at evaluating the impact of disease burden on prediction of relapse and survival in patients receiving allogeneic hematopoietic cell transplantation (allo-HCT) in first remission (CR1). We identified a total of 3202 adult AML patients, of these 1776 patients were in CR1 and MRD positive and 1426 patients were primary refractory at time of transplant. After a median follow-up of 24.4 months, non-relapse mortality and relapse rate were significantly higher in the primary refractory group compared to the CR1 MRD positive group (Hazards Ratio (HR)=1.82 (95% CI: 1.47-2.24) p<0.001 and HR=1.54 (95% CI: 1.34-1.77), p<0.001), respectively. Leukemia-free survival (LFS) and overall survival (OS) were significantly worse in the primary refractory group (HR=1.61 (95% CI: 1.44-1.81), p<0.001 and HR= 1.71 (95% CI: 1.51-1.94), p<0.001, respectively). Our real-life data suggest that patients in CR1 and MRD positive at time of transplant could still be salvaged by allo-HCT with a 2-year OS of 63%, if negative MRD cannot be obtained and their outcomes are significantly better than patients transplanted with active disease.