Safety and efficacy of a liquid formulation of deferiprone (GPO-LONE ®) monotherapy among iron-overloaded young children with transfusion-dependent thalassemia
Liquid formulation of deferiprone among young thalassemia children
Keywords:
Liquid formulation, Deferiprone, Iron overload, ThalassemiaAbstract
Abstract:
Background: Iron chelation for children aged 2-6 years is limited. Aim: Safety and efficacy of a liquid formulation of deferiprone (GPO-L-ONE ®) monotherapy among young children was reported. Subjects and Methods: Deferiprone in a daily dose of 50-100 mg/kg divided in 3 doses was given to thalassemia children aged 2-12 years with transfusion-induced iron overload. Efficacy was defined as responder and nonresponder by the ≥ 15 or < 15% reduced serum ferritin compared with the initial ferritin level. Results: Nineteen patients (7 males and 12 females) with thalassemia at the median age of 4.1 years were enrolled. The median daily iron load from regular transfusion was 0.41 mg/kg. Eighteen and 8 of 19 patients completed the first and second year study, respectively. The maximum daily dose of deferiprone was 100 mg/kg. The median baseline ferritin 1,470 ng/mL changed in +633 and +64.5 ng/mL at the end of the first and second years, respectively. However, only 4 of 19 patients (21%) were defined as responders with significantly reduced serum ferritin starting from the 6th month of treatment. Ten adverse events of transminitis (n = 3), severe neutropenia (n = 1), neutropenia (n = 2) and thrombocytopenia (n = 4) were found among 5 patients who spontaneously recovered without medical intervention. Conclusion: A liquid formulation of deferiprone (GPO-L-ONE®) at a daily dose of 100 mg/kg was tolerable among young children. However, efficacy was limited by responsiveness to negative iron balance in 4 of 19 patients (21%).
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References
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