Iron chelator deferiprone is widely used in paediatric β-thalassaemia patients with transfusional iron overload, although only limited study data are available particularly for children younger than 10 years. However, due to the off-patent status it is widely available and economical.
With respect to safety, long-term data are of special interest because early and constant iron elimination is necessary to avoid long-term effects of iron overload.
The DEEP project investigates efficacy and safety of deferiprone in children and adolescents and seeks approval for paediatric use (Paediatric Use Marketing Authorisation, PUMA). It is funded by the European Commission’s 7th Framework Programme (FP7). In this context, three studies are carried out between January 2011 and December 2014:
DEEP-1 examines pharmacokinetics and pharmacodynamics of deferiprone in children aged between one month and six years.
DEEP-2 is a randomised clinical trial comparing efficacy and safety of deferiprone with the oral iron chelator deferasirox in children between the age one month and less than 18 years.
The DEEP-3 study is sponsored by the Universitätsklinikum Erlangen and coordinated by the Department of Paediatrics and Adolescent Medicine. It investigates the long-term safety of deferiprone therapy in children with β-thalassemia aged one month to less than 18 years. It is a non-interventional, multicentre, multinational cohort study with retro- and prospective data collection. The study includes 16 recruiting centres from six different countries: Albania, Egypt, Greece, Italy, Tunisia and Cyprus. The main objective of the study is the nature and incidence of serious adverse drug reactions (ADRs) during deferiprone therapy. In addition, non-serious ADRs and risk factors for ADRs related to deferiprone are investigated. It is anticipated to recruit 400 patients; all of them will be followed-up for at least two years or until cessation of deferiprone therapy.
For more information, please visit http://www.deepproject.eu/