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Long-Term Deferiprone May Protect Heart Against Toxic Iron Overload In Patients With Thalassaemia Major
A DGReview of :"Comparative effects of deferiprone and deferoxamine on survival and cardiac disease in patients with thalassemia major: a retrospective analysis"
Haematologica
05/22/2003
By Mary Beth Nierengarten
In the first retrospective analysis of the comparative effects of deferiprone and deferoxamine on mortality and cardiac morbidity in patients with thalassaemia major, long-term therapy with deferiprone was associated with a significant reduction in cardiac dysfunction over time and improved survival compare to deferoxamine.
Most patients with thalassaemia major die from iron-induced cardiac disease, despite chelation therapy with deferoxamine, and to date, no study has compared the frequency of cardiac complications and survival between deferiprone and deferoxamine in these patients.
Antonio Piga and colleagues from the University of Turin, Italy, retrospectively evaluated 129 patients with thalassaemia major who met study criteria, 54 of whom received deferiprone (average dose of 73.7+11.2 mg/kg/day) and 75 deferoxamine (average dose 39.2+4.7 mg/kg per infusion, administered 6+1 times/week on average). Treatment compliance was 89 and 85% for the deferiprone- and deferoxamine-treated groups, respectively.
On an average of 6 years follow-up, patients treated with deferiprone had significantly fewer cases of cardiac dysfunction (defined as worsening of pre-existing cardiac abnormalities or development of new cardiac disease) than patients treated with deferoxamine: 4% (2 of 54) vs. 20% (15 of 75), respectively (p=0.007).
Deferiprone-treated patients also had significantly improved 5-year survival (p=0.003), with no deaths compared to 4 deaths out of the 75 patients treated with deferoxamine.
The authors speculate that the improved outcomes with deferiprone suggests that it has a cardioprotective effect that may be due to its lipophilicity, low molecular weight, and neutral charge of pH 7.4, which may allow it to cross cell-membranes and bind intracellular iron.
The authors conclude that "in patients with thalassaemia major, long-term therapy with deferiprone results in a greater cardioprotective effect against toxicity of iron overload than does subcutaneous deferoxamine." They recommend the need for prospective studies to confirm these results.
Haematologica 2003 May;88(5):489-96.
"Comparative effects of deferiprone and deferoxamine on survival and cardiac disease in patients with thalassemia major: a retrospective analysis"
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