The controversial role of deferiprone in the treatment of thalassemia

The role of the orally active iron (Fe) chelator deferiprone in the treatme nt of beta -thalassemia remains a controversial subject. Despite initial st udies showing high Fe chelation efficacy in vitro and also in animals and h uman subjects, several latter studies have not been so successful. In fact, it has been reported in several clinical trials that deferiprone after lon g-term treatment had either little effect or actually increased hepatic Fe loading. In addition, an increase in liver fibrosis was noted in one study. However more recently, results by other investigators have suggested that the drug may be used under some circumstances without marked toxicity. In p articular it has been demonstrated that the combination of deferoxamine (DF O) and deferiprone results in more Fe excretion than when either chelator i s used alone. Moreover, a combination of both drugs led to a decrease in de feriprone-mediated toxicity. Other studies performed in patients for up to in years showed no progressive fibrosis after deferiprone therapy, while a possible trend toward increasing fibrosis was noted in another investigatio n. Additional studies using larger numbers of deferiprone-treated patients are essential to determine the efficacy and safety of this drug, particular ly in relation to the development of fibrosis. The present review discusses the possible role of deferiprone in the treatment of Fe overload.