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Endocrine Society Recommends Limited Use of Propylthiouracil in Children CHEVY CHASE, Md -- April 16, 2009 -- On the basis of several lines of evidence and frequency estimates, the Endocrine Society endorses the recommendation that propylthiouracil (PTU) no longer be used as first-line treatment for Graves' disease in children because of PTU-related liver failure in the paediatric population. In a letter to the editor published in the April 9 issue of the New England Journal of Medicine, Scott A. Rivkees, MD, Yale University School of Medicine, New Haven, Connecticut, and Donald R. Mattison, MD, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Bethesda, Maryland, highlight concerns about potential severe liver disease in children from use of PTU -- an adverse effect not seen with the other thionamide derivative, methimazole. Some of the evidence was presented at the NICHD workshop "Hepatic Toxicity Following Treatment for Pediatric Graves' Disease" on October 28, 2008. Despite the relative rarity of Graves' disease in the young, children and adolescents account for as many as 13 of 42 cases of serious PTU-related liver failure reported to date in the medical literature and a similar disproportionate number (4 of 13) of those requiring liver transplant for this indication between 1990 and 2002. Although precise numbers are not known with certainty, it can be estimated that there are 4,000 children treated each year for Graves' disease in the US. If 40% are treated with PTU, then as many as 1 to 2 children a year could develop severe liver disease, with some requiring a liver transplant or potentially suffering a fatal outcome. Even 1 excess death is too many if it can be prevented. The Society therefore suggests that until data from more thorough prospective studies or peer-reviewed retrospective studies become available, it would seem reasonable to use methimazole preferentially in the paediatric population. SOURCE: The Endocrine Society E-mail this page to a friend or colleague! To print, use this version