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 |
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Rok vydání: | 2019 |
Předmět: |
Pediatrics
medicine.medical_specialty Anemia business.industry Health Policy medicine.disease 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine chemistry 030220 oncology & carcinogenesis medicine Pharmacology (medical) Deferiprone business Pharmacology Toxicology and Pharmaceutics (miscellaneous) 030217 neurology & neurosurgery Paediatric patients Pediatric population |
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 |
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