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Deferasirox 6 months after allo-HSCT in AML/MDS: a prospective propensity-matched study
Romain Buono1, Anne Huynh2, Edouard Forcade3,4
1Département Prévention, Cancer, Environnement, Centre Léon Bérard, Lyon, France.
Blood Advances
|March 11, 2026
Summary
Deferasirox therapy after allogeneic hematopoietic stem cell transplantation (allo-HSCT) effectively reduced iron overload and improved progression-free survival. This iron chelation therapy showed promise in lowering transplant-related mortality, supporting its use post-transplant.
Area of Science:
- Hematology
- Transplantation Immunology
- Pharmacology
Background:
- Iron overload is a significant complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT), linked to increased oxidative stress and poorer patient outcomes.
- While deferasirox is known to reduce ferritin levels, its impact on long-term survival post-allo-HSCT requires further investigation.
Purpose of the Study:
- To evaluate the effect of deferasirox on survival outcomes, including overall survival (OS) and progression-free survival (PFS), in patients with iron overload after allo-HSCT.
- To assess the impact of deferasirox on transplant-related mortality (TRM) and relapse rates.
- To determine the safety and tolerability of deferasirox initiated six months post-allo-HSCT.
Main Methods:
- A prospective observational study involving 41 patients with AML or MDS who received deferasirox six months after allo-HSCT.
- Patients were compared to propensity score-matched controls from the SFGM-TC registry (1:2 ratio).
- Statistical analyses included exact matching, inverse probability of treatment weighting (IPTW), and multivariable Cox models to assess endpoints like OS, PFS, TRM, and relapse.
Main Results:
- Deferasirox treatment led to a sustained reduction in ferritin levels from 1,700 µg/L to below 1,000 µg/L by 18 months.
- Consistent across analytical methods, deferasirox was associated with a significantly reduced hazard for progression or death (PFS: HR 0.41-0.44) and a reduced hazard for mortality (OS: HR 0.28-0.43).
- A significant reduction in TRM was observed (HR 0.21 in PSM and Cox models), with manageable adverse events, primarily renal.
Conclusions:
- Initiating deferasirox six months post-allo-HSCT is associated with effective iron reduction, improved PFS, and lower TRM, with acceptable safety.
- The study suggests deferasirox is a feasible intervention for managing iron overload after allo-HSCT.
- Further confirmation through randomized controlled trials is warranted to solidify these findings.
