WP4 – Performing an efficacy/safety trial to compare deferiprone and deferasirox (DEEP-2)

Despite the widespread use of deferiprone in children of all ages, no formal evaluation of DFP efficacy and safety in young children is available. Therefore, the objective of the present WP is to plan and conduct a randomised, controlled trial aimed at comparing the efficacy of deferiprone versus deferasirox in paediatric patients. The hypothesis to be tested is the non inferiority of the deferiprone versus deferasirox in reducing serum ferritin levels and/or cardiac iron overload.

Additional secondary objectives:

•    Evaluation of the treatment efficacy in terms of hepatic iron concentrations
•    Assessment of the safety profiles
•    Patient compliance
•    Evaluation of benefit of of treatments in terms of quality of life (QoL)

WP Leader: Aurelio Maggio (AORC)
Involved Partners: CVBF, UL, AOC, NKUA, UHCT, MPHS, CU, ApoPharma, GIAMBRONE, AORC, AODP, PB, FGB, CNGMO