Phase 3 Trial of Deferiprone in Paediatric patients with Transfusion-dependent Haemoglobinopathies

A phase 3 trial study published in the June 1 issue of The Lancet of Haematology evaluating the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2). Maggio A. and colleagues show the the non-inferiority of deferiprone versus deferasirox. DEEP-2 was a phase 3, multicentre, randomised trial in paediatric patients (aged 1 month to 18 years) with transfusion-dependent haemoglobinopathies. The study was done in 21 research hospitals and universities in Italy, Egypt, Greece, Albania, Cyprus, Tunisia, and the UK. Participants were receiving at least 150 mL/kg per year of red blood cells for the past 2 years at the time of enrolment and were receiving deferoxamine or deferasirox. Between March 17, 2014, and June 2016, 393 of the 435 patients were randomly assigned to a treatment group (194 to the deferiprone group; 199 to the deferasirox group). Non-inferiority of deferiprone versus deferasirox was established and compliance was similar between treatment groups: 183 (95%) of 193 patients in the deferiprone group versus 192 (97%) of 197 patients in the deferisirox group.In paediatric patients with transfusion-dependent haemoglobinopathies, deferiprone was effective and safe in inducing control of iron overload during 12 months of treatment. Considering the need for availability of more chelation treatments in paediatric populations, deferiprone offers a valuable treatment option for this age group.

More information: Original publication.