Deferasirox 360mg tablets

Iron chelation treatments as adjuvant therapy can reduce iron stores to minimize the related morbidity and mortality in patients with thalassemia major. Objective: To compare Deferasirox (DFX) and Desferrioxamine (DFO) in terms of mean serum ferritin levels in patients of β-thalassemia major having Iron overload. Methods: This randomized controlled trial was conducted at the Thalassemia Center of Hematology Department, “The Children's Hospital and The Institute of Child Health”, Multan, Pakistan from January 2023 to September 2023. After randomization, children in DFO group were given DFO in a dose of 50mg/kg, through subcutaneous route by infusion pump five days a week. Children in DFX group were given DFX in a dose of 30mg/kg, orally in tablet form once daily. Baseline serum ferritin levels were measured and the change in mean serum ferritin level for each group was calculated and compared for both groups after 6-months of treatment. Results: In a total of 142 children, 87 (61.3%) children were male. The mean age was 7.08 ± 2.41 years. The mean number of blood transfusions at the time of enrollment were 13.4 ± 4.2. After 6 months of treatment in DFO versus DFX groups, the net change in mean serum ferritin levels from baseline to post-treatment was 947.2 ± 454.0 µg/L for DFO and 1053.5 ± 389.8 µg/L for DFX, with no statistically significant difference between the groups (p=0.1367). Conclusions: Once-daily oral deferasirox has good compliance, acceptable tolerability and appears to have similar efficacy to desferrioxamine in reducing iron burden of transfused patients with beta thalassemia major.

Abstract licence: CC BY 4.0

IntroductionAbnormal hemoglobin, or hemoglobinopathy, affects about 7% of the global population. Major hemoglobinopathies like beta-thalassemia and sickle cell disease require regular blood transfusions, leading to chronic iron overload. This review examines the efficacy and safety of deferiprone, an oral iron chelator, in managing iron overload in pediatric patients with transfusion-dependent conditions.MethodsData were sourced from PubMed, Google Scholar, and relevant articles, focusing on randomized controlled trials (RCTs) published between 2010 and 2023. The search terms included "deferiprone," "iron chelation," "transfusion," "iron overload," "hemoglobinopathies," and "thalassemia." Three RCTs met the inclusion criteria, involving 521 pediatric patients.ResultsThe START trial demonstrated that early-start deferiprone significantly reduced iron load compared to placebo, with no severe adverse events. The DEEP-2 study found deferiprone non-inferior to deferasirox in terms of efficacy and safety. Another trial highlighted the benefits of early deferiprone therapy in delaying iron overload symptoms without serious side effects. Common adverse effects included pyrexia, nasopharyngitis, and decreased neutrophil count, but no significant differences in growth parameters, creatinine, or prolactin levels were observed.ConclusionDeferiprone shows significant promise in managing iron overload in pediatric patients, with comparable effectiveness to existing therapies and a favorable safety profile. Its oral administration is advantageous for young children. However, long-term studies are needed to fully understand its safety and efficacy. Addressing challenges such as patient compliance and adverse effects through education, personalized medicine, and advanced monitoring techniques can further improve treatment outcomes for beta-thalassemia patients.

Abstract licence: CC BY-ND