The DEEP (DEferiprone Evaluations in paediatrics) project, a 6-year European Project (FP7) coordinated by CVBF and comprising 23 recruiting centres in European and non-European countries, scientific partners from several European countries and a pharmaceutical group based in Canada, is at the final rush and has presented its preliminary results in Milan on February 28^th – March 1^st, 2018.

The DEEP Project 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. The aim of DEEP is to provide data on deferiprone pharmacokinetics in younger children, to evaluate the safety of deferiprone in the clinical setting through a long-term observational study and to generate new comparative efficacy/safety data to be used to grant a Marketing Authorisation (MA) of a new liquid formulation of the drug, specifically developed for children use.

The project consisted of three studies, DEEP-1 (PK), DEEP-2 (efficacy and safety) and DEEP-3 (long-term safety).

The DEEP-1 was a multi-centre, oral single dose experimental and modelling study to evaluate the pharmacokinetics of deferiprone in patients under 6 years of age affected by transfusiondependent haemoglobinopathies (NCT01740713, clinicaltrials.gov). It was concluded in 2014, providing scientific evidence that the dosage per kilogram of deferiprone used in adults and older children can provide sufficient exposure to ensure efficacy also in younger children. These results allowed children aged under 6 years to start to be recruited in DEEP-2 Safety/Efficacy Study.

The DEEP-2 was a phase III multicentre, randomised, open label, non-inferiority active-controlled trial aiming at comparing the efficacy, in terms of changes of serum ferritin levels and cardiac iron overload, of deferiprone versus deferasirox in paediatric patients affected by hereditary haemoglobinopathies, requiring chronic transfusions and chelation (NCT01825512, clinicaltrials.gov). It has involved 393 randomized patients with the first patient first visit (FPFV) in January 2015 and the last patient last visit (LPLV) in September 2017.

The DEEP-3 was a long-term observational safety study which evaluated the nature and incidence of adverse effects of deferiprone in children and adolescents with beta-thalassaemia major. Started in 2013, and ended in October 2015, with 297 patients enrolled in the study, it confirmed that the safety profile of deferiprone in children and adolescents is in accordance with the available data in adults.

The meeting has been organized on occasion of the Rare Disease Day 2018, promoted by EURORDIS to raise awareness of rare diseases and their impact on patients’ lives amongst the general public and decision-makers. The Rare Disease Day has taken place every year since 2008, to call upon researchers, universities, students, companies, policy makers and clinicians to do more research and to make them aware of the importance of research for the rare diseases community.

DEEP considers the promotion and the coordination of research extremely important for health care system, and particularly for Rare Diseases, since they represent the tools to speed up the achievements in research and benefits for patients.