What question did this study set out to answer?

The review aims to evaluate the effectiveness and safety of dual oral iron chelation therapy for TDT patients not adequately managed with monotherapy.

April 22, 2026Annals of Hematology0 citationsOpen Access

Dual oral iron chelation with deferasirox and deferiprone in transfusion-dependent β-thalassemia: a narrative review of efficacy, safety, and practical application

VJVinson James KEKristina Ifeoma Emeghebo

Key Points

The review aims to evaluate the effectiveness and safety of dual oral iron chelation therapy for TDT patients not adequately managed with monotherapy.
Conducted a literature review using PubMed, Scopus, and regional databases from January 2016 to January 2025.
Evaluated clinical cohort studies and real-world audits of dual therapy (DFX + DFP) for iron reduction and safety outcomes.
Assessed outcomes like serum ferritin levels, MRI findings, and adherence qualitatively.
Patients showed mean serum ferritin declines of 25 to 45%.
Over 80% of pediatric patients achieved ferritin levels below 2000 ng/mL.
Treatment was associated with mild gastrointestinal effects and stable transaminase levels, with no agranulocytosis reported.

Abstract

For patients with transfusion-dependent β-thalassemia (TDT) and suboptimal response to iron chelator monotherapy, dual oral iron chelation (DOIC) with deferasirox (DFX) and deferiprone (DFP) represents a practical strategy to enhance iron removal. This narrative review synthesizes contemporary evidence for its use, with a focus on identifying clinical signals from real-world data and highlighting gaps requiring prospective investigation. We conducted a narrative review informed by a structured literature search of PubMed, Scopus, and regional databases (January 2016 to January 2025) for studies evaluating DOIC (DFX + DFP) in pediatric and adult TDT. Outcomes of interest included serum ferritin, MRI T2*, hepatic iron concentration, adherence, and safety. Study quality and risk of bias were assessed qualitatively using standard criteria for observational research. Evidence from clinical cohort studies and real-world audits indicates that DFX + DFP therapy produces additive or synergistic reductions in systemic iron burden, with mean serum ferritin declines of 25 to 45%. Recent real-world pediatric data (2024 to 2025) report over 80% of patients achieving ferritin below 2000 ng/mL, with high adherence rates (> 85%). MRI trends show improvement in hepatic and cardiac iron, though meaningful organ-specific changes typically require more than 24 months of therapy. The safety profile is consistent with established monotherapy profiles, primarily comprising mild gastrointestinal effects and transient transaminase elevations, with no reported agranulocytosis in reviewed cohorts. Study limitations include small samples, heterogeneous dosing protocols, and absence of randomized controlled trial data. DOIC with DFX and DFP is an effective and tolerable regimen for TDT patients with inadequate iron control on single agents. It leverages pharmacologic synergy within an all-oral, adherence-friendly regimen suitable for real-world use, including resource-limited settings. Prospective randomized trials with serial MRI endpoints are warranted to establish its superiority over optimized monotherapy for organ protection and long-term outcomes.

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