Accessibility to
iron chelators including
deferoxamine and
deferasirox remains obscured in many developing countries. To provide an alternative, the government
pharmaceutical organization of Thailand (GPO) manufactured
deferiprone which has similar bioequivalent to the standard product. Seventy-three pediatric patients with severe β
thalassemias, age range 3.2-19 years, were recruited to a 1-year multicenter prospective, single arm, open label, dose escalating Phase III study of
deferiprone to determine its clinical efficacy and safety. Sixty-four patients (87.6%) completed the study with good compliance (>94%). Average
deferiprone dose was 79.1±4.3 mg/kg/day. Overall, mean serum
ferritin (SF) levels at 1 year were not significantly changed from baseline. However, 45% of patients (response group) had SF reduced >15% from baseline at 1 year with a median reduction of 1,065 ng ml(-1) . Baseline SF was the major factor that predicts clinical efficacy; patients with baseline SF>3,500 ng ml(-1) had the most significant fall of SF at 1 year. A subgroup analysis by MRI-T2* confirmed that the response group had higher baseline liver
iron and
deferiprone could significantly reduce liver
iron overload and normalize levels of ALT at 1 year. Although, gastrointestinal irritation (20.5%) was the most common
drug-related adverse events (AEs) followed by transaminitis (16.4%) and
neutropenia (6.8%), all patients were well tolerated. There was no mortality and
agranulocytosis found in this trial. Monotherapy of
deferiprone with appropriate dose adjustment and monitoring for adverse events appeared to be an effective
chelation therapy in some patients with good compliance and acceptable safety profiles.