Targeted active surveillance of branded generic deferasirox in pediatric thalassemia patients in a tertiary care hospital of India: A pharmacovigilance study.

Abstract

Introduction: Deferasirox is a newer oral iron chelator which needs to be given for at least 1 year to see a significant decrease in serum ferritin level in thalassemia patients with chronic iron overload. This study aimed to assess the safety profile of branded generic deferasirox in pediatric thalassemia cases, as it has not been studied in eastern India, especially in real-world settings.

Subjects and methods: It was an observational hospital-based study in a government tertiary care teaching hospital where branded generic deferasirox is distributed free of cost to patients. One hundred and seventy-four patients were included. Safety assessment was done through active questioning about the symptoms starting after deferasirox therapy and through laboratory parameters and clinical examination.

Results: Sixty percent of patients developed adverse drug reactions (ADRs) with deferasirox during the 1-year follow-up. ADRs were more common in males than females. The highest number of ADRs was related to the gastrointestinal and hepatobiliary system. The ADRs were either mild or moderate. Compliance and number of ADRs were found to be inversely correlated.

Conclusion: ADRs of deferasirox are acceptable looking at the benefits. Regular monitoring and management of ADRs will facilitate patient compliance. However, healthcare professionals need to be alert while prescribing this drug and report any ADR, even if it is not labeled. This study found increased serum lipase and mouth ulcer as adverse effects, which are not yet labeled. These can be signals for further analysis by regulatory authorities.