Abstract
Background: Thalassemia is an inherited hemoglobin disorder; mostly require life-long blood transfusions, leading to chronic iron overload which causes growth failure, delayed sexual development in adolescents and vital organ dysfunctions. So, children with thalassemia need lifelong iron chelation therapy. Hence, this study conducted with the aim of to evaluate efficacy and safety of Deferasirox in pediatric thalassemia patients.
Materials and methods: This is an observational, prospective, single-centered hospital-based study conducted in a tertiary care teaching hospital from June 2018 to December 2019. Total sixty children (age 2-18 years) with beta thalassemia major and hemoglobin E beta thalassemia with iron overload were enrolled to commence deferasirox. Efficacy and safety we observed by measuring serum ferritin three monthly and SGPT, SGOT & serum creatinine monthly.
Results: The serum ferritin level of 72% patients reduced significantly after 12 months in comparison to baseline level. There was no serious adverse effect except mild abdominal pain, nausea & vomiting and transaminitis.
Conclusion: Deferasirox is efficacious in reducing iron overload of the body when administer at optimum dose over at least one year and presenting a safe as well as convenient alternative for most of the transfusion dependent pediatric thalassemia patients.