The DEEP-1 pharmacokinetic study evaluated the systemic exposure of DFP in the paediatric population aged from 1 month to < 6 years for which PK profile of deferiprone is unknown. This lack of information implies that so far the dosage of deferiprone in this age subset can only be calculated empirically based on adult data. Thus, the DEEP-1 study was aimed at providing the daily dosage most appropriate to ensure safety and efficacy in young children before enrolling them in DEEP-2.
Deferiprone exposures from 18 evaluable children aged from 1 month to < 6 yearsaffected by hereditary haemoglobinopathies confirmed that a dosing regimen of 25 mg/kg t.i.d.(i.e. 75 mg/kg/die) is the most mappropriate also in this specific age subset, with the possibility of titration up to 33.3 mg/kg t.i.d. (i.e. 100 mg/kg/die), if necessary.
For the first time, we have scientific evidence that the dosage of deferiprone used in adults can provide sufficient exposure to ensure efficacy also in small children.