Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine
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 trans...
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| Format: | Article |
| Language: | English |
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Electronic Physician
2016-05-01
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| Series: | Electronic Physician |
| Subjects: | |
| Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4930264/ |
| _version_ | 1818024237326139392 |
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| author | Mohamed Abdel Malik Hassan Omar Atef Tolba |
| author_facet | Mohamed Abdel Malik Hassan Omar Atef Tolba |
| author_sort | Mohamed Abdel Malik Hassan |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-12-10T03:57:01Z |
| format | Article |
| id | doaj.art-8531080c2aca4d3cafd7ead8db3399cf |
| institution | Directory Open Access Journal |
| issn | 2008-5842 2008-5842 |
| language | English |
| last_indexed | 2024-12-10T03:57:01Z |
| publishDate | 2016-05-01 |
| publisher | Electronic Physician |
| record_format | Article |
| series | Electronic Physician |
| spelling | doaj.art-8531080c2aca4d3cafd7ead8db3399cf2022-12-22T02:03:06ZengElectronic PhysicianElectronic Physician2008-58422008-58422016-05-01852425243110.19082/2425Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamineMohamed Abdel Malik HassanOmar Atef TolbaIntroduction: 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.https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4930264/iron chelatorsiron overloadthalassemiaefficacy |
| spellingShingle | Mohamed Abdel Malik Hassan Omar Atef Tolba Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine Electronic Physician iron chelators iron overload thalassemia efficacy |
| title | Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine |
| title_full | Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine |
| title_fullStr | Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine |
| title_full_unstemmed | Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine |
| title_short | Iron chelation monotherapy in transfusion-dependent beta-thalassemia major patients: a comparative study of deferasirox and deferoxamine |
| title_sort | iron chelation monotherapy in transfusion dependent beta thalassemia major patients a comparative study of deferasirox and deferoxamine |
| topic | iron chelators iron overload thalassemia efficacy |
| url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4930264/ |
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