Deferiprone vs Deferasirox in paediatric haemoglobinopathies

Jul 6, 2020

The article “Evaluation of the effectiveness and safety of Deferiprone compared to Deferasirox in pediatric patients with hemoglobinopathies (DEEP-2)” was recently published on The Lancet Haematology Journal.

DEEP-2 is one of three studies foreseen in the DEEP (DEferiprone Evaluation in Paediatrics) project, a European funded Project (FP7) in which Fondazione Benzi was involved supporting the ethical and regulatory activities. DEEP Project aimed 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 population.

DEEP-2 study is a phase III multicentre, randomised, open label, non-inferiority active-controlled trial aiming at comparing for the first time the efficacy of Deferiprone versus Deferasirox involving 393 paediatric patients aged from 1 month to less than 18 years, affected by hereditary haemoglobinopathies.

Congenital hemoglobinopathies require treatments that include regular blood transfusions and iron chelators administered alone or in combination. Currently, patients have different options of chelation treatment, as parenteral Desferrioxamine, oral Deferiprone and Deferasirox. However, none of the existing chelators completely covers the pediatric needs. Thus, the availability of chelators really tailored for this population remains a huge problem and a relevant therapeutic need. For years, Deferiprone has been used ‘off-label’ in the pediatric population in lack of some crucial information.

Together with data from published pediatric studies, DEEP2 results provide with increased evidence on the efficacy and safety of Deferiprone in paediatric patients during 12 months of treatment.

Discover more about the study results by reading the article here!