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Comparative efficacy of iron chelation therapies in thalassemia major: a randomized controlled trial


Article Information

Title: Comparative efficacy of iron chelation therapies in thalassemia major: a randomized controlled trial

Authors: Zainab W Al-maaroof, Widad Hamza Shekair

Journal: Anaesthesia, Pain and Intensive Care

HEC Recognition History
Category From To
Y 2023-07-01 2024-09-30
Y 2022-07-01 2023-06-30
Y 2021-07-01 2022-06-30
Y 2020-07-01 2021-06-30

Publisher: Faculty of Anaesthesia, Pain and Intensive Care, AFMS

Country: Pakistan

Year: 2025

Volume: 29

Issue: 6

Language: en

DOI: 10.35975/apic.v29i6.2916

Keywords: ThalassemiaIronDeferasiroxDFODFPDFXDeferoxamineDeferiprone

Categories

Abstract

Background & objective: Thalassemia major, a transfusion-dependent genetic disorder, leads to iron overload in the body and necessitates frequent sessions of iron chelation therapy. Despite the availability of deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX), the optimal chelation strategies remain controversial.
We compared the efficacy and safety of DFO, DFP, and DFX in reducing iron overload parameters, including serum ferritin, liver iron concentration (LIC), and cardiac T2* MRI. We also compared the adverse events with the use of these three agents.
Methodology: A 12-month randomized controlled trial included 150 thalassemia major patients, randomized to receive either DFO (40–50 mg/kg/day subcutaneously), DFP (75 mg/kg/day per os), or DFX (20–40 mg/kg/day per os). Serum ferritin level, LIC, cardiac T2* MRI, and adverse events were assessed. Statistical analyses were performed using ANOVA) and chi-square tests.
Results: DFX showed the greatest reduction in serum ferritin (2100 ± 700 ng/mL vs. DFO: 2500 ± 750, DFP: 2400 ± 800; P = 0.02) and LIC (8.5 ± 2.5 mg/g vs. DFO: 10.2 ± 2.8, DFP: 9.8 ± 3.0; P = 0.01). DFP exhibited superior cardiac iron clearance (cardiac T2* MRI: 22.0 ± 5.5 ms vs. DFO: 20.5 ± 5.0, DFX: 21.0 ± 5.2; P = 0.03). The adverse events were mild (injection-site reactions for DFO and gastrointestinal disturbances for DFP/DFX).
Conclusion: DFX is optimal for systemic and hepatic iron reduction in thalassemia major patients, whereas DFP excels in cardiac iron clearance. Therapy should be personalized based on the iron deposition profiles.
Abbreviations: DFO:deferoxamine, DFP: deferiprone. DFX: deferasirox  
Keywords: Thalassemia; Iron; DFO; DFP; DFX; Deferoxamine; Deferiprone; Deferasirox  
Citation: Al-maaroof ZW, Shekair WH. Comparative efficacy of iron chelation therapies in thalassemia major: a randomized controlled trial. Anaesth. pain intensive care 2025;29(5):567-572. DOI: 10.35975/apic.v29i5.2916
Received: June 12, 2025; Revised: July 25, 2024; Accepted: July 26, 2025


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