Department of Child Health, Cairo University, Cairo, Egypt
Research Article
Safety and Efficacy of Early Use of Iron Chelation in Infants and Toddlers with Transfusion-Dependent Thalassemia: A Multicenter Prospective Study
Author(s): Mohsen Elalfy, Amira Adly*, Amal Elbeshlawy, Mona Hamdy, Tamer Hassan, Mohamed Meabed, Rasha El Ashry, Khalid Elsayeh, Seham Rageb, Mohamed Elshanshoury, Naglaa Shahein, Mahmoud El-Hawy, Gihan Lofty, Saad Shehata, Islam Elghamry, Yasser Wali and Omar Elalfy
Background: There are limited data on the efficacy and safety of oral iron chelators in infants and toddlers and
cite the Deferiprone (DFP) and Deferasirox (DFX) label that states that those chelators were not studied in children
under the age of 2 and 3 years respectively. The current study was designed to assess the long-term safety and efficacy
of DFX and DFP in this young population.
Methods: A 2-year prospective multi-center, investigator-initiated non-interventional study of infants and children
with TDT after at least 4 blood transfusions, naïve to chelation. The children started either on DFX at a dose of 20
mg/kg/day-25 mg/kg/day or DFP at a dose of 50 mg/kg/day-75 mg/kg/day. Throughout the study all patients were
subjected to through history and clinical examination stressing on detailed transfusion data, compl.. View more»