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

内容摘要

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.