Effectiveness of Deferasirox in Pediatric Thalassemia Patients: Experience from a Tertiary Care Hospital of Odisha

Mousumee Panigrahi, Trupti Rekha Swain, Rabindra Kumar Jena, Ashutosh Panigrahi, Nishant Debta, Mousumee Panigrahi, Trupti Rekha Swain, Rabindra Kumar Jena, Ashutosh Panigrahi, Nishant Debta

Abstract

Background and objectives: Patients with beta-thalassemia require lifelong blood transfusions, leading to chronic iron overload, which can lead to growth retardation, as well as hinder sexual development during the adolescent period and dysfunction of organs such as heart, pancreas, and endocrine glands. These patients are in need of lifelong transfusion therapy and hence lifelong iron chelation therapy as well. Hence, this study was aimed to assess the effectiveness of deferasirox for iron chelation in pediatric thalassemia cases in a tertiary care hospital of Eastern India.

Subjects and methods: This prospective, observational, hospital-based study was conducted from June 2015 to December 2016. Two hundred and fifty patients were assessed for eligibility, of which 174 were included. Effectiveness of deferasirox was observed by measuring serum ferritin levels which were monitored at the end of every 3 months till 1 year. We also evaluated the compliance with deferasirox therapy in the same study cohort.

Results: The serum ferritin level reduced significantly at the end of 12 months in comparison to baseline (P = 0.04). There was a mean absolute decrease in serum ferritin only in the dose range of 21-30 mg/kg/day. Approximately 90% of the patients had 100% compliance with deferasirox therapy.

Conclusions: Deferasirox is an effective iron chelator when started at an optimum time and with optimum dose. At least 1 year of deferasirox therapy is needed for a significant lowering of serum ferritin levels of pediatric thalassemia patients on multiple blood transfusions.

Keywords: Beta-thalassemia; blood transfusions; chelating agents; deferasirox; iron.

Conflict of interest statement

There are no conflicts of interest.

Copyright: © 2020 Indian Journal of Pharmacology.

Figures

Figure 1
Figure 1
Flow diagram of study design
Figure 2
Figure 2
Percentage of responders to deferasirox therapy as per baseline serum ferritin level
Figure 3
Figure 3
Percentage of responders as per dose of deferasirox therapy
Figure 4
Figure 4
Mean absolute change in serum ferritin as per baseline serum ferritin
Figure 5
Figure 5
Mean absolute change in serum ferritin as per dose range of deferasirox
Figure 6
Figure 6
Variation in mean serum ferritin levels over 1 year

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