Deferasirox 6 months after allo-HSCT in AML/MDS: a prospective propensity-matched study

Iron overload after allogeneic hematopoietic stem cell transplantation (allo-HSCT) is associated with oxidative stress and adverse outcomes. Although deferasirox reduces ferritin, its effect on survival remains incompletely characterized. We conducted a prospective observational study across 7 French centers, enrolling 41 patients with acute myeloid leukemia or myelodysplastic syndromes who initiated deferasirox 6 months after allo-HSCT. Eligibility required complete remission, iron overload (ferritin level >1000 μg/L), and preserved organ function. Outcomes were compared with propensity score-matched controls (1:2) from the SFGM-TC registry. End points were overall survival (OS), progression-free survival (PFS), transplant-related mortality (TRM), relapse, and safety. Analyses employed exact matching, inverse probability of treatment weighting (IPTW), and multivariable Cox models. Ferritin level decreased from 1700 μg/L to <1000 μg/L by 18 months. Across analytic methods, deferasirox was associated with a reduced hazard of progression or death (PFS: hazard ratio [HR], 0.41-0.44) and a reduced hazard of mortality (OS: HR, 0.28-0.43). TRM was significantly reduced in propensity score matching and Cox models (HR, 0.21), whereas the IPTW analysis yielded a comparable point estimate (HR, 0.24) that did not reach the significance threshold (P = .063). The probability of relapse (estimated via cumulative incidence of relapse) did not differ significantly. Adverse events were predominantly renal and manageable. Initiating deferasirox 6 months after allo-HSCT was associated with sustained ferritin reduction, a reduced hazard of progression or death (PFS), lower TRM, and a favorable although nonsignificant hazard of mortality (OS), with acceptable safety. These findings support deferasirox as a feasible posttransplant intervention, warranting confirmation in randomized trials.