Hyperammonemia and acute liver failure associated with deferasirox in two adolescents with sickle cell disease

Alison S Towerman^{1

2}, Kristin P Guilliams^{3

4

5}, Réjean Guerriero³, Marwan S Shinawi⁶, Janis M Stoll⁷, Daniel N Willis¹, Monica L Hulbert¹

Affiliations

¹ Division of Hematology/Oncology, Department of Pediatrics, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA.
² Trudy Busch Valentine School of Nursing, Saint Louis University, St. Louis, Missouri, USA.
³ Division of Pediatric and Developmental Neurology, Department of Neurology, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA.
⁴ Division of Critical Care Medicine, Department of Pediatrics, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA.
⁵ Mallinckrodt Institute of Radiology, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA.
⁶ Division of Genetics and Genomic Medicine, Department of Pediatrics, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA.
⁷ Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA.

Alison S Towerman et al. Br J Haematol. 2023 May.

. 2023 May;201(4):e30-e33.

doi: 10.1111/bjh.18770. Epub 2023 Mar 25.

Alison S Towerman^{1

2}, Kristin P Guilliams^{3

4

5}, Réjean Guerriero³, Marwan S Shinawi⁶, Janis M Stoll⁷, Daniel N Willis¹, Monica L Hulbert¹

¹ Division of Hematology/Oncology, Department of Pediatrics, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA.
² Trudy Busch Valentine School of Nursing, Saint Louis University, St. Louis, Missouri, USA.
³ Division of Pediatric and Developmental Neurology, Department of Neurology, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA.
⁴ Division of Critical Care Medicine, Department of Pediatrics, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA.
⁵ Mallinckrodt Institute of Radiology, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA.
⁶ Division of Genetics and Genomic Medicine, Department of Pediatrics, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA.
⁷ Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, Washington University in St. Louis School of Medicine, St. Louis, Missouri, USA.

No abstract available

References

1. DeBaun MR, Jordan LC, King AA, Schatz J, Vichinsky E, Fox CK, et al. American Society of Hematology 2020 guidelines for sickle cell disease: prevention, diagnosis, and treatment of cerebrovascular disease in children and adults. Blood Adv. 2020;4(8):1554-88.
1. Ramaswami A, Rosen DJ, Chu J, Wistinghausen B, Arnon R. Fulminant liver failure in a child with beta-thalassemia on Deferasirox: a case report. J Pediatr Hematol Oncol. 2017;39(3):235-7.
1. Martinelli D, Goffredo BM, Falvella FS, Marano M. Acute hyperammonemia in children under deferasirox treatment: cutting the gordian knot. Clin Toxicol (Phila). 2019;57(5):375-7.
1. Marano M, Bottaro G, Goffredo B, Stoppa F, Pisani M, Marinaro AM, et al. Deferasirox-induced serious adverse reaction in a pediatric patient: pharmacokinetic and pharmacogenetic analysis. Eur J Clin Pharmacol. 2016;72(2):247-8.
1. Pediatric Advisory Committee. Exjade®/Jadenu® (deferasirox) briefing document [Internet]. United States: Food and Drug Administration; 2018 Sept 20 (cited 2023 March 22). p. 1-14. Avalable from: https://www.fda.gov/media/164286/download