Journal of Blood Disorders & Transfusion

Abstract

Safety and Efficacy of Early Use of Iron Chelation in Infants and Toddlers with Transfusion-Dependent Thalassemia: A Multicenter Prospective Study

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, compliance on iron chelator with reporting the Adverse Events (AEs) and anthropometric measures. Laboratory investigations including monthly complete blood count (CBC) and Serum Ferritin (SF), while measurement of liver transaminases and serum creatinine was every 3 months.

Results: Three-hundred and sixty pediatric Transfusion-Dependent Thalassemia (TDT) patients (60%males) were enrolled. Their mean age at enrolment was of 1.72 ± 0.49 months, with a mean of 8.9 ± 0.32 blood transfusions. All were naive to chelation and their initial SF ranged from 820 ng/ml-1202 ng/ml and 480 ng/ml-1070 ng/ml in DFX in a dose of 20 mg/kg/day-25 mg/kg/day and DFP in a dose 50 mg/kg/day-75 mg/kg/day groups respectively; adding during the study period a median of 16 transfusions. With a comparable transfused iron input. At 12 months of regular transfusion-chelation; SF was<1000 ng/ml in almost 62% (60% and 70% on the patients on DFX and DFF respectively) and at 24 months in almost 56% (52% and 64%) respectively, differences were statistically significant. 77% of DFX patients underwent dose increase above 25 mg⁄kg⁄day, while 12.5% of DFP group had increased their dose to above 75 mg/kg/day. Compliance was comparable.

Conclusion: Our results confirmed previous observations on safety and efficacy of both iron chelators in TDT naïve to chelation who had started it at an earlier age. AEs in this real practice study were less compared with published clinical trial data, with significant deviation from guidelines.

Published Date: 2023-09-27; Received Date: 2023-08-21