Effect of Deferasirox Six Months after allo-HSCT on AML/MDS Outcomes: a Propensity-Score Matched Study

Iron overload after allogeneic hematopoietic stem cell transplantation (allo-HSCT) is associated with oxidative stress and adverse outcomes. While deferasirox reduces ferritin, its effect on survival remains incompletely characterized. We conducted a prospective observational study across seven French centers, enrolling 41 patients with AML or MDS who initiated deferasirox six months after allo-HSCT. Eligibility required complete remission, iron overload (ferritin >1,000 µg/L), and preserved organ function. Outcomes were compared with propensity score-matched controls (1:2) from the SFGM-TC registry. Endpoints 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 decreased from 1,700 µg/L to <1,000 µg/L by 18 months. Across analytic methods, deferasirox was associated with a reduced hazard of progression or death (PFS: HR 0.41-0.44) and a reduced hazard of mortality (OS: HR 0.28-0.43). TRM was significantly reduced in PSM and Cox models (HR 0.21), while 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 [CIR]) did not differ significantly. Adverse events were predominantly renal and manageable. Initiating deferasirox six months after allo-HSCT was associated with sustained ferritin reduction, a reduced hazard of progression or death (PFS), lower TRM and a favorable though non-significant hazard of mortality (OS), with acceptable safety. These findings support deferasirox as a feasible post-transplant intervention, warranting confirmation in randomized trials.