Fulminant Liver Failure in a Child with β-Thalassemia on Deferasirox: A Case Report

Archie Ramaswami, Danya J. Rosen, Jaime Chu, Birte Wistinghausen, Ronen Arnon

Research output: Contribution to journalArticlepeer-review

17 Scopus citations


Deferesirox (DFX), an oral chelating agent, is used to treat chronic iron overload in several hematological diseases such as β-thalassemia, sickle cell disease, and myelodysplastic anemia. DFX is generally well tolerated with the exception of gastrointestinal disturbances and rash, although cases of renal toxicity, as well as acute and chronic liver failure, have been reported in adults and children. Here we describe a 3-year-old girl with β-thalassemia undergoing treatment with DFX who presented with acute liver failure and Fanconi's syndrome. It is important for pediatric gastroenterologists, hepatologists, and hematologists to be aware that the commonly used drug DFX can lead to acute liver failure in children, and liver function should be monitored closely in all patients taking DFX.

Original languageEnglish
Pages (from-to)235-237
Number of pages3
JournalJournal of Pediatric Hematology/Oncology
Issue number3
StatePublished - 2017


  • Fanconi syndrome
  • chelation
  • deferasirox
  • hepatotoxicity
  • β-thalassemia


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