Deferasirox 6 months after allo-HSCT in AML/MDS: a prospective propensity-matched study
Abstract
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.
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BibTeX
@article{buono2026deferasirox,
title = {{Deferasirox 6 months after allo-HSCT in AML/MDS: a prospective propensity-matched study}},
author = {Romain Buono and Anne Huynh and Edouard Forcade and Hélène Labussière and Patrice Ceballos and Jérôme Cornillon and Quentin Cabrera and Maud D’aveni and Marie Robin and Raynier Devillier and Nicole Raus and Mauricette Michallet},
journal = {Blood Advances},
year = {2026},
month = may,
doi = {10.1182/bloodadvances.2025018894},
url = {https://hal.univ-lorraine.fr/hal-05787682v1},
note = {CC BY-NC-ND},
}
RIS
TY - JOUR
TI - Deferasirox 6 months after allo-HSCT in AML/MDS: a prospective propensity-matched study
AU - Romain Buono
AU - Anne Huynh
AU - Edouard Forcade
AU - Hélène Labussière
AU - Patrice Ceballos
AU - Jérôme Cornillon
AU - Quentin Cabrera
AU - Maud D’aveni
AU - Marie Robin
AU - Raynier Devillier
AU - Nicole Raus
AU - Mauricette Michallet
PY - 2026
DA - 2026/05/26
JO - Blood Advances
DO - 10.1182/bloodadvances.2025018894
UR - https://hal.univ-lorraine.fr/hal-05787682v1
AB - 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.
SN - 2473-9529
SN - 2473-9537
ER -