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.
Departments of *Pediatric Gastroenterology and Hepatology
‡Pediatric Hepatology, Recanati/Miller Transplant Institute
§Pediatric Hematology and Oncology, Mount Sinai Medical Center, New York, NY
†Department of Pediatric Gastroenterology and Hepatology, Yale School of Medicine, New Haven, CT
The authors declare no conflict of interest.
Reprints: Archie Ramaswami, MD, Mount Sinai Medical Center, Recanati-Miller Transplant Institute, Mount Sinai School of Medicine, 1 Gustave L. Levy Place, Box 1104, New York, NY 10029 (e-mail: Archana.Ramaswami@mssm.edu).
Received February 29, 2016
Accepted June 24, 2016