The safety, tolerability, and efficacy of a liquid formulation of deferiprone in young children with transfusional iron overload

Moshen El Alfy, Teny Tjitra Sari, Chan Lee Lee, Fernando Tricta, Amal El-Beshlawy

Research output: Contribution to journalArticle

42 Citations (Scopus)

Abstract

Limited data are available on the use of deferiprone in children younger than 10 years of age. This study evaluated the safety and efficacy of a new liquid formulation of deferiprone for the treatment of transfusional iron overload in children 1-10 years old. One hundred children (91 thalassemia major, 8? Hb E-β thalassemia, and 1 sickle cell disease) were enrolled for a 6-month treatment with deferiprone (50 to 100 mg/kg/d). The safety profile was similar to or better than that reported in earlier studies with deferiprone tablets in older children and adults. No unexpected adverse reactions were observed. Gastrointestinal intolerance (GI) was observed in 11% and an increased serum ALT in 12% of the children. Both events were transient. Mild neutropenia, observed in 6% of patients, did not progress to agranulocytosis and resolved despite continuous deferiprone treatment. Two patients experienced agranulocytosis that resolved without complications upon discontinuation of therapy. Deferiprone use was associated with a significant decline in mean serum ferritin level from 2532±1463 μg/L at baseline to 2176±1144? μg/L (P<0.0005). The results of this study show a favorable benefit/risk ratio of deferiprone oral solution for the treatment of young children with transfusional iron overload.

Original languageEnglish
Pages (from-to)601-605
Number of pages5
JournalJournal of Pediatric Hematology/Oncology
Volume32
Issue number8
DOIs
Publication statusPublished - 1 Nov 2010

Keywords

  • deferiprone
  • pediatric
  • safety
  • transfusional iron overload

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