Deferiprone is an effective and safe iron chelation therapy for young patients with transfusion-dependent hemoglobinopathies, according to results from a phase 3 trial published in Lancet Haematology.

A team of researchers conducted a multicenter, randomized, open-label analysis (DEEP-2; ClinicalTrials.gov Identifier: NCT01825512) that included pediatric patients aged 1 month to 18 years with transfusion-dependent hemoglobinopathies. The study was performed at 21 research sites in Europe and northern Africa.

The goal of the study was to demonstrate noninferiority for deferiprone in comparison with deferasirox. Patients were stratified by age and randomly assigned to either groups deferiprone (194 patients) or deferasirox (199 patients) treatment groups.

The primary endpoint for efficacy was the proportion of patients showing successful chelation. This was a composite endpoint that involved meeting criteria for changes to serum ferritin levels and cardiac magnetic resonance imaging (MRI) T2-star results after 1 year of therapy.


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The most common hemoglobinopathies in this study were β-thalassemia major (90% of patients) and sickle cell disease (7% of patients).

Median study follow-ups were 379 days for the deferiprone group and 381 days for the deferasirox group. The study investigators reported that deferiprone demonstrated noninferiority, with treatment success in per-protocol population analysis occurring in 55.2% of evaluable patients receiving deferiprone, compared with 54.8% of evaluable patients receiving deferasirox (P =.79).

The study investigators reported that treatment-related and serious adverse events occurred at similar rates between treatment groups. Arthralgia and gastrointestinal issues occurred more with deferiprone, and abnormal renal function occurred more with deferasirox.

In the safety analysis, reversible agranulocytosis was reported in 3 patients on deferiprone, while 2 patients receiving deferasirox experienced renal/urinary disorders that were reversible. There were no fatalities reported.

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The study investigators concluded that the data from this large, randomized trial support deferiprone therapy in pediatric patients who have transfusion-dependent hemoglobinopathies. “The main clinical implication of this study is that paediatric patients might now have more than one efficacious and safe option for oral iron chelation therapy,” the study investigators concluded.

Disclosure: Several study authors have declared affiliations with the pharmaceutical industry. Please see the original reference for a full list of authors’ disclosures.

Reference

Maggio A, Kattamis A, Felisi M, et al. Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial. Lancet Haematol. 2020;7(6):e469-e478.