Pattern of iron chelation therapy in Egyptian beta thalassemic patients: Mansoura University Children's Hospital experience

Abstract

Background: The simultaneous use of deferoxamine (DFO) and deferiprone (DFP) has an additive effect in iron excretion in transfusion-dependent thalassemic patients.

Aim of the work: To evaluate the efficacy and safety of a prospective alternating therapy with DFO and DFP in patients with beta-thalassemia major (TM) and increased serum ferritin with DFO monotherapy alone.

Patient and methods: Sixty patients with beta-TM (mean age +/- SD, 13.05 +/- 6.1, range 10-20 years) with iron overload (serum ferritin > 2000 ng/ml) were studied. They received DFO at a daily dose of 40 mg/kg/day for 5-7 nights/week for the past several years. These patients were randomly assigned either to continue treatment with DFO alone (DFO group, n = 30) or prospectively receive additional alternating therapy with DFP at 75 mg/kg/day for 4 days/week and DFO for the other 2 days/week (alternating therapy group, n = 30). The efficacy of both groups was assessed by measurements of serum ferritin, echocardiography, and 24 h urine iron excretion (UIE) levels throughout 1 year follow-up.

Results: In the 60 evaluable patients, the mean serum ferritin ( +/- SD) fell dramatically from 4500 ( +/- 1250) ng/ml at the start of the study to 1250 ( +/- 750) ng/ml (alternate therapy group; P < 0.001) at the end of the study. There was also a significant improvement in the myocardial function as assessed by the ejection fraction (P < 0.002) and fractional shortening (P < 0.01) in those patients on alternate therapy for 1 year. Their mean urinary iron excretion elevated from 0.41 +/- 0.27 to 0.76 +/- 0.49 mg/kg/24 h (P < 0.003). There was a significant difference between both groups as regard the studied parameters at the end of the study. Whereas, there was no statistical difference as regard the studied parameters at the start and the end of the study in the DFO group. No significant adverse effects had occurred in both groups that necessitated withdrawal from the study.

Conclusions: beta-Thalassemic major patients with transfusional iron overload can be safely and effectively treated with an alternate therapy of DFO/DFP with a progressive fall in the mean serum ferritin and significant improvement of myocardial performance.