Effect of Combined versus Monotherapy with Deferoxamine and Deferiprone in Iron Overloaded Thalassemia Patients: a Randomized Clinical Trial

Authors

1 Department of Pediatrics, Faculty of Medicine, Urmia University of Medical Sciences, Urmia, Iran.

2 Department of Pediatrics, Faculty of Medicine, Alborz University of Medical Sciences, Karaj, Iran.

3 Department of Community Medicine, Faculty of Medicine, Alborz University of Medical Sciences, Karaj, Iran.

4 Department of Psychology, Ilam Branch, Islamic Azad University, Ilam, Iran.

Abstract

Background: Patients with transfusional iron overload have depended on iron chelation therapy and improving chelation regimens have been of the highest priority. The aim of this study was to compare effect of combined versus monotherapy with Deferoxamine (DFO) and Deferiprone (DFP) in iron overloaded beta thalassemia (BT) major patients
Materials and Methods
We studied 36 BT major patients (mean age 7.6±4.6; range 3–16 years) attending the Ormieh Motahari hospital for regular transfusional support. Patients were randomly allocated to receive one of the following two treatments: DFO in combination with DFP (n=12), DFO alone (n=12) and DFP alone (n=12). Serum ferritin level, liver enzymes, blood urea nitrogen, and creatinine and side effects were monitored over a 12 months period.
Results: After one year, serum ferritin decreased more significantly in patients on DFO+DFP therapy compared to patients who only received DFO or DFP alone (P<0.01). Side effects of DFP, including gastrointestinal upset (nausea, vomiting and abdominal pain) and mild agranulocytosis occurred in five (41.7%) and two (8.3%) patients, respectively but none led to discontinuation of the treatment.
Conclusion: In comparison to the standard chelation monotherapy of DFO, combination treatment with additional DFP reduced serum ferritin and is effective procedure in clinical management of iron overload in patients with BT major.

Keywords