Subcutaneous iron chelation therapy, oral iron chelation therapy, or both in patients with thalassemia?

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Abstract

Background

This study aimed to compare the efficacy of different iron-chelating agents, namely deferoxamine (DFO), deferiprone (DFP), and deferasirox and combined DFO and DFP in reducing serum ferritin levels in thalassemic patients attending the Pediatric Hematology Clinic of the Children and Obstetrics and Gynecology Hospital, Minia University.

Patients and methods

A total of 57 patients with thalassemia major attending the Pediatric Hematology Clinic of Minia University were included in the study. They were classified into four groups according to type of chelation: the DFO group included 12 patients, the DFP group included 18 patients, the deferasirox group included 15 patients, and the combined DFO and DFP group included 12 patients. The patients were subjected to thorough history taking, clinical examination, and laboratory investigation (complete blood count, liver function and renal function tests, and levels of hepatitis B surface antigen, hepatitis C antibody, and initial serum ferritin at the start of the study and final serum ferritin levels after 1 year of chelation).

Results

The mean serum ferritin levels decreased significantly in all groups after 1 year (P<0.001), with more reduction in the combined therapy group. Four (22.2%) patients developed elevated liver transaminases and six (33.3%) developed mild bone pain and arthralgia during treatment with oral DFP. However, none of our patients developed neutropenia/agranulocytosis or significant gastrointestinal tract symptoms. There were no detectable side effects on administering deferasirox, except for some gastrointestinal tract pain that improved with continued treatment.

Conclusion

The three iron-chelating agents are effective in reducing the serum ferritin levels in thalassemic children, with the combined DFO and DFP therapy being more effective.

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