Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2016 May 25;8(5):2425-31.
doi: 10.19082/2425. eCollection 2016 May.

Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine

Mohamed Abdel Malik Hassan  1 , Omar Atef Tolba  2
Affiliations

Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine

Mohamed Abdel Malik Hassan et al. Electron Physician. .

Abstract

Introduction: Iron overload is the primary cause of mortality and morbidity in thalassemia major (TM) despite advances in chelation therapy. The aim of this study was to compare the effectiveness and safety of deferasirox (DFX) and deferoxamine (DFO) as iron-chelating agents in patients with transfusion-dependent β-thalassemia major.

Methods: This prospective randomized study included 60 patients with transfusion-dependent β-TM during the period from September 2014 to September 2015. Their ages were ≥ 6 years, and they had serum ferritin above 1500 μg/L and were on irregular DFO therapy. Patients had regular packed red cell transfusion in a dose of 10 mL/kg/session. They were randomized to receive DFX (single oral daily dose of 20-40 mg/kg/day) or DFO (20-50 mg/kg/day via subcutaneous infusion over 8-10 hours, 5 days a week). Iron overload was determined by serum ferritin level. The primary endpoint was decrease of serum ferritin level below 1500 μg/L. The secondary endpoint was drug safety.

Results: Both drugs significantly reduced serum ferritin (p < 0.001). At the end of follow-up, there were no significant differences between the two groups in serum ferritin levels (p = 0.673) and in percent reduction of ferritin (p = 0.315). There were no significant differences between the two groups in the total amount of blood transfusion (p = 0.166) and average iron intake (p = 0.227). There were no mortalities or any serious adverse effects, neutropenia, arthropathy, or pulmonary toxicity. Gastrointestinal upset and skin rash occurred more frequently with DFX than with DFO (p = 0.254 and 0.095, respectively).

Conclusion: With appropriate dosing and compliance with drugs, both DFX and DFO are generally well tolerated, safe, and effective in reducing serum ferritin levels in iron-overloaded, regularly-transfused thalassemia major patients. Therefore, oral DFX is recommended for more convenience and adherence to the treatment regimen.

Keywords: efficacy; iron chelators; iron overload; safety; thalassemia.

PubMed Disclaimer

References

    1. Shawky RM, Kamal TM. Thalassemia intermedia: An overview. Egyptian Journal of Medical Human Genetics. 2012;13(3):245–55. doi: 10.1016/j.ejmhg.2012.03.006. - DOI
    1. El-Beshlawy A, Manz C, Naja M, Eltagui M, Tarabishi C, Youssry I, et al. Iron chelation in thalassemia: combined or monotherapy? The Egyptian experience. Ann Hematol. 2008;87(7):545–50. doi: 10.1007/s00277-008-0471-2. - DOI - PubMed
    1. Aessopos A, Berdoukas V, Tsironi M. The heart in transfusion dependent homozygous thalassemia today-prediction, prevention and management. Eur J Haematol. 2008;80(2):93–106. doi: 10.1111/j.1600-0609.2007.01018. - DOI - PMC - PubMed
    1. Crisponi G, Remelli M. Iron chelating agents for the treatment of iron overload. Coord Chem Rev. 2008;252(10–11):1225–40. doi: 10.1016/j.ccr.2007.12.014. - DOI
    1. Gardenghi S, Marongiu MF, Ramos P, Guy E, Breda L, Chadburn A, et al. Ineffective erythropoiesis in beta-thalassemia is characterized by increased iron absorption mediated by down-regulation of hepcidin and up-regulation of ferroportin. Blood. 2007;109(11):5027–35. doi: 10.1182/blood-2006-09-048868. - DOI - PMC - PubMed

LinkOut - more resources