Journal ArticleDOI
Long-term therapy with deferiprone.
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TLDR
The results of this study may provide the data required for an FDA decision regarding licensing of this agent for the treatment of iron overload, a goal supported by investigators worldwide.Abstract:
Data from several trials have provided direct and supportive evidence for the efficacy of deferiprone in the treatment of iron overload in thalassemia major. Deferiprone has been shown to induce sustained decreases in body iron to concentrations associated with survival free from the complications of iron overload in deferoxamine (DFO)-treated patients. Despite this evidence of efficacy, the risk of agranulocytosis mandates a careful evaluation in patients willing and able to use DFO. The incidence of agranulocytosis associated with deferiprone is under study in a prospective multicenter trial in Canada, Italy and the United States, under corporate sponsorship (Apotex Research, Canada). The results of this study should determine the risk associated with the use of this agent, and may provide the data required for an FDA decision regarding licensing of this agent for the treatment of iron overload, a goal supported by investigators worldwide.read more
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Journal ArticleDOI
Role of deferiprone in chelation therapy for transfusional iron overload.
TL;DR: Before 1987, iron chelation therapy for patients with thalassemia major and other refractory anemias requiring regular transfusions depended almost entirely on one drug, deferoxamine.
Journal ArticleDOI
Safety profile of the oral iron chelator deferiprone: a multicentre study
TL;DR: It is demonstrated that agranulocytosis is less common than previously predicted and, under the specific conditions of monitoring, the safety profile of deferiprone is characterized, and it is shown that neutropenia resolved after interruption of therapy.
Journal ArticleDOI
The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients
Adriana Ceci,Paola Baiardi,Mariagrazia Felisi,Maria Domenica Cappellini,Vittorio Carnelli,Vincenzo De Sanctis,Renzo Galanello,Aurelio Maggio,Giuseppe Masera,Antonio Piga,Francesco Schettini,Ippazio Stefàno,Fernando Tricta +12 more
TL;DR: These data show that the drug was effective in reducing serum ferritin levels and the incidence of adverse events was not greater than the frequency reported in clinical trials.
Journal ArticleDOI
Iron Chelators for Thalassaemia
TL;DR: The abnormalities in iron metabolism associated with thalassaemia major and the chelatable iron pools representing the targets of iron-chelating therapy are discussed and the development of new, orally effective, iron chelators which may be considered for present or future use are reviewed.
Journal ArticleDOI
Deferiprone: a review of its clinical potential in iron overload in beta-thalassaemia major and other transfusion-dependent diseases.
TL;DR: Deferiprone is the most promising oral iron chelator under development at present and shows promise as an adjunct to deferoxamine therapy in patients with insufficient response and may prove useful as a maintenance treatment to interpose between treatments.