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

Autor: Antonella Didio, Adriana Ceci, Fedele Bonifazi, Donato Bonifazi, Viviana Giannuzzi, Rosa Conte, Mariagrazia Felisi
Rok vydání: 2019
Předmět:
DOI: 10.6084/m9.figshare.8187941.v1
Popis: 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 (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.
Databáze: OpenAIRE