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Deferasirox in Indian children with thalassemia major: 3 years experience

Mayank Dhamija1, Amita Mahajan, Manas Kalra

  • 1Department of Pediatric Hematology and Oncology, Rajiv Gandhi Cancer Institute and Research Centre, Rohini, New Delhi, India.

Indian Journal of Medical and Paediatric Oncology : Official Journal of Indian Society of Medical & Paediatric Oncology
|July 24, 2013
PubMed

Insights

Oral iron chelator deferasirox effectively reduced iron overload in Indian children with thalassemia major. The drug demonstrated safety and efficacy, with most patients showing improved serum ferritin levels over 36 months.

Area of Science:

  • Hematology
  • Pediatric Medicine
  • Pharmacology

Background:

  • Thalassemia major requires frequent blood transfusions, leading to transfusional hemosiderosis (iron overload).
  • Effective iron chelation therapy is crucial for managing iron overload in these patients.

Purpose of the Study:

  • To assess the efficacy and safety of deferasirox in Indian children with thalassemia major and high iron load.
  • To evaluate the impact of deferasirox on serum ferritin levels and identify potential toxicities.

Main Methods:

  • A cohort of 50 Indian children (age 2-18) with thalassemia major received deferasirox for 36 months.
  • Dosing was individualized based on serum ferritin, adjusted up to 40 mg/kg/day.
  • Regular monitoring of ferritin, liver enzymes, and renal function was performed.

Main Results:

  • 76% of patients showed a significant decline in serum ferritin levels (P<0.05).
  • Mean serum ferritin decreased from 4354 ng/mL at baseline to 3042 ng/mL at 36 months.
  • No severe toxicity was observed during the study period.

Conclusions:

  • Deferasirox (≥30 mg/kg) is effective and safe for reducing transfusional hemosiderosis in pediatric thalassemia major.
  • Dose escalation to 40 mg/kg/day was required for 70% of patients.
  • 30% of patients did not achieve negative iron balance even at maximum doses.
Abstract

Keywords:
Deferasiroxserum ferritinthalassemia major

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