Pemoline (Cylert) is a central nervous system stimulant first introduced into clinical trials in the 1970s. Currently, it is most commonly used to treat children with attention deficit disorder (ADD). There have been scattered reports of hepatotoxicity with pemoline usually involving transient elevation of liver chemistries.3-6  Overt liver failure has been rarely reported.7-10  During the last 3 years we encountered four cases of marked liver dysfunction in children on pemoline. Two of these progressed to fulminant hepatic failure requiring liver transplantation. In those cases where autoantibodies were measured, there was evidence for autoimmune activation (see Tables1 and 2).

This patient was an adopted 14-year-old girl with ADD unresponsive to methylphenidate. Pemoline, 37.5 mg per day, was instituted with some improvement in school performance. Liver chemistries were not routinely monitored. Six months later, jaundice and fatigue developed. Physical examination was notable for jaundice and no...

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