A Randomized, Placebo-Controlled, Double-Blind Trial of the Effect of Combined Therapy with Deferoxamine and Deferiprone on Myocardial Iron in Thalassemia Major Using Cardiovascular Magnetic Resonance

Authors
Tanner, M.A., Galanello, R., Dessi, C., Smith, G. C., Westwood, M.A., Agus, A., Roughton, M., Assomull, R., Nair, S.V., Walker, J.M., Pennell, D.

Journal
Circulation, 2007, 15 (13)

Background
The leading cause of death in thalassaemia major patients is cardiac complications secondary to iron overload. Ferriprox® has been shown to remove cardiac iron, improving cardiac function and patient survival. This study aims to determine if Ferriprox in combination with desferoxamine, provides additional benefits versus deferoxamine alone.

Methods
167 thalassaemia major patients treated with standard deferoxamine therapy had their cardiac iron load assessed by MRI T2*. 65 patients identified as having mild to moderate myocardial iron loading were entered into the study. This group was randomized to continue deferoxamine therapy alone, or begin combination therapy with Ferriprox and deferoxamine.

The primary end point of the study was the change in myocardial T2* over 12 months, with secondary endpoints of endothelial and cardiac function.

Results
There were significant improvements in all measures (T2*, cardiac and endothelial function, and serum ferritin) for the combined therapy group vs. the deferoxamine only group.

Conclusion
Combination therapy with Ferriprox and deferoxamine provided superior reduction in myocardial iron load, improved cardiac and endothelial function, and reduced serum ferritin load versus chelation monotherapy with deferoxamine.

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