An overview of the efficacy and safety of deferiprone in paediatric patients with congenital haemoglobinopathies and chronic iron overload

Introduction: Congenital hemoglobinopathies represent the most severe forms of anemia in Europe requiring treatment that include regular blood transfusions and iron chelators administered alone or in combination. However, none of the existing chelators completely covers the pediatric needs.Areas covered: The introduction of deferiprone on the market has represented an important advancement in congenital hemoglobinopathies. Moreover, several studies have shown that deferiprone combined therapy with desferrioxamine is particularly effective in cardiac iron overload. Despite a wide experience on deferiprone use, limited data were available in the pediatric population (<10 years of age) at the time of the marketing authorization. This review investigates and discusses the evidence more recently provided by deferiprone pediatric studies.Expert opinion: Available deferiprone efficacy and safety data in pediatrics are increasing and demonstrate that DFP is adequate and efficacious at the same dosage used in adults. No significantly increased risk for agranulocytosis and other relevant adverse events/adverse drug reactions is reported, but caution is recommended in very young children and in combined therapy if two chelators (deferiprone + desferrioxamine or deferiprone + deferasirox) are administered simultaneously. The results of larger and statistically powered trials are expected to solve the still existing controversies in pediatric uses of deferiprone.