WP5 - Performing a long-term safety study in paediatric patients (DEEP-3)

The primary objective of WP5 is to evaluate long-term safety of deferiprone use in paediatric patients by conducting a multi-centre, multi-national, observational cohort study (DEEP-3) with both retrospective and prospective data collection. The study involves 18 recruiting centres from 7 different countries: Albania, Cyprus, Greece, Egypt, Tunisia, Italy and the UK.

The DEEP-3 study has the aim to investigate the long-term safety of deferiprone in children with β-thalassaemia major aged 1 month to less than 18 years. Primarily, the study determines the nature and incidences for serious adverse drug reactions (ADR) to deferiprone such as agranulocytosis and neutropenia. Additionally, incidences of non-serious ADRs and risk factors for ADRs related to deferiprone therapy are investigated.

Anonymised patient data such as demographic data, transfusion regimen, chelation therapy, concomitant medications and diagnoses and laboratory data are collected via an electronic case report form (e-CRF). Any adverse medical finding which might be related to deferiprone therapy is documented by the investigators and further assessed by the study team. It is anticipated to recruit 400 patients; all of them will be followed-up for at least two years or until cessation of deferiprone therapy.

WP Leader: Antje Neubert (UKER)
Involved Partners: CVBF, AOC, NKUA, UHCT, MPHS, CU, AORC, AODP, PB, FGB, UKER, CNGMO