Sebastian Botzenhardt from University Hospital of Erlangen and his co-writers have just published the results of the DEEP-3 study in the January issue of Haematologica on behalf of the DEEP consortium.
DEEP (Deferiprone Evaluation in Paediatrics) is funded EU-funded project aimed to investigate safety and efficacy of deferiprone in children and adolescences from the Mediterranean region affected by beta-thalassemia major.
An early iron chelation therapy is crucial in the treatment of the beta-thalassemia major to avoid complication related to the chronic transfusional iron overload. Deferiprone is an iron chelator that showed a superior efficacy in reducing cardiac iron, especially in combination with deferoxamine. Notwithstanding, utilization in children is still limited since studies in paediatric patients are rare.
DEEP-3 is a multi-center, retro- and prospective, non-interventional cohort study aimed to investigate the long-term safety of deferiprone therapy alone and in combination with deferoxamine. The study was conducted in 16 hospitals in six Mediterranean countries between November 2012 and April 2016 – Albania (1), Cyprus (1), Egypt (1), Greece (1), Italy (11) and Tunisia (1) – to assess the nature and incidence of serious and non-serious adverse drug reactions (ADR) to deferiprone and potential risk factors for ADRs and deferiprone withdrawals. Patients treated with deferiprone were followed up throughout their treatment, from its start up to the end of the study or the patient’s withdrawal.
The authors concluded that the safety profile of deferiprone in children and adolescents is in accordance with the available data in adults and no unexpected ADRs were observed. However, agranulocytosis was also present in this paediatric population. Arthropathy, including arthralgia and swelling, mostly in the knee joints, was the most common ADR and needs to be closely monitored.
The full publication is available at the following link: http://www.haematologica.org/content/103/1/e1.long