Long Term Deferiprone Chelation Therapy

A wide variety of compounds have been tested in animals and humans in order to find drugs which are capable of removing iron from patients with refractory anaemias receiving regular blood transfusions. Parenteral preparations which have been evaluated clinically include desferrioxamine (DFO), diethylene triamine penta-acetic acid (DTPA), desferrithiocin and HBED (N,NL bis (2-hydroxy-benzyl ethylenediamine - NNI diacetic acid). Orally active chelators include 2,3 dihydroxybenzoic acid, pyridoxal isonicotinyl hydrazone, 1,2,dimethylpyrid-4-one (L1, deferiprone) and 1,2 diethylpyrid-4-one). The only two compounds in wide clinical use are DFO and deferiprone. DFO discussed in detail elsewhere in this volume remains standard therapy but because of cost, failure of compliance and, less frequently, toxicity or sensitivity, it is not satisfactory for many patients worldwide, especially in poorer countries where thalassaemia major and other transfusion dependent genetic disorders of haemoglobin are most frequent and antenatal diagnosis and prevention is inadequate or non-existent. In India, deferiprone, known as Kelfer (Cipla, Bombay, India) has been licensed since 1990. In the European Community Countries deferiprone, known as Ferriprox, has been licensed for those patients for whom DFO is contra-indicated or who exhibit serious toxicity to DFO therapy. In this review we shall describe the long term results of therapy with deferiprone either alone or in combination with DFO. Other recent reviews of this drug include those of Addis et al1, Basman Balfour et al2Diav-Citrin et al3Hershko & Hoffbrand4Olivieri5.