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Safety of oral iron chelator deferiprone in young thalassaemics

Rahul Naithani1, Jagdish Chandra, Sharad Sharma

  • 1Division of Hematology, Department of Pediatrics, Kalawati Saran Children's Hospital, Lady Hardinge Medical College, New Delhi, India. dr_raul6@hotmail.com

European Journal of Haematology
|February 8, 2005
PubMed

Insights

Deferiprone is used for iron chelation in young thalassemia patients. Thrombocytopenia is a significant side effect requiring monitoring, but it resolves upon discontinuation.

Area of Science:

  • Hematology
  • Pediatric Medicine
  • Pharmacology

Background:

  • Deferiprone is a widely used iron chelator for thalassemia patients.
  • Existing safety and efficacy data primarily focus on older children and adults.

Purpose of the Study:

  • To evaluate the safety profile of deferiprone in pediatric patients under six years of age.
  • To identify and quantify adverse events associated with deferiprone treatment in this age group.

Main Methods:

  • Retrospective analysis of follow-up data from 44 pediatric patients (<6 years) receiving deferiprone.
  • Systematic monitoring for gastrointestinal and osteoarticular side effects.
  • Regular complete blood count monitoring every 2-4 weeks.

Main Results:

  • Nausea and vomiting occurred in 27.2% of patients; joint symptoms in 9.1%.
  • Neutropenia was rare (4.5%), with no cases of agranulocytosis.
  • Thrombocytopenia affected 45.45% of patients, typically appearing 3-12 months after therapy initiation.

Conclusions:

  • Thrombocytopenia is a key adverse event in young thalassemia patients on deferiprone, necessitating vigilant blood count monitoring.
  • Symptoms of thrombocytopenia generally resolve after deferiprone discontinuation, supporting its continued use with careful observation.
Abstract

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