Concept

β-thalassaemia major is one of the most severe forms of anaemia. The recommended treatment consists in regular blood transfusions combined with chelating therapy to remove harmful iron accumulation in the body. The first chelating agent approved for clinical use was deferoxamine that, despite its potentially satisfactory therapeutic effects, is not usable by many patients due to toxicity. Moreover, its subcutaneous route of administration leads to non-compliance in most young patients. On August 1999, the oral iron chelator deferiprone was authorised in Europe where it is currently indicated for the treatment of iron overload in patients with β-thalassaemia major when deferoxamine is contraindicated or inadequate. Despite a wide experience of deferiprone with thalassaemic patients, limited data are available on its use in 2-10 years old children and the need for additional data in this class of age is expressly stated in the 2009 PDCO (the Paediatric Committee at the European Medicinal Agency) Priority List. In addition, the anticipated benefit in controlling cardiac iron overload in all the paediatric population as well as in other chronic transfusion-dependent anaemias, has led the PDCO to expand the request for additional data to the cited patients populations.

The DEEP PProject has been developed with the specific intent to integrate the existing information on deferiprone use in paediatric patients, thus covering the lack of information and providing a valid support to the use of the drug in this class of age. According to recent scientific advancements and the opinion expressed by the PDCO, the Project has been expanded to include the whole paediatric population and other chronic transfusion-dependent anaemia. The aim DEEP is to provide data on pharmacokinetics, and comparative efficacy/safety of deferiprone in order to generate new data to be used for the registration of a new liquid formulation of deferiprone, specifically developed for children use. At the end of the proposed set of studies, deferiprone will be available as first line treatmentwith the description of efficacious dosages in children under 18 years. In addition, a three-year safety study will evaluate all deferiprone uses in the clinical setting.