Abstract
Control of purine synthesis has been studied in 3 children with a syndrome of hyperuricemia and cerebral dysfunction. Azathioprine was employed as an agent recently shown to inhibit purine synthesis in adults. Elevated plasma concentrations of uric acid were reduced to within normal limits in an adult but not in the 3 children. There was significant evidence of marrow depression in the adult but no hematological effect in the children. These observations are interpreted to indicate a requirement for conversion of azathioprine to the nucleotide of 6-mercaptopurine for action on both uric-acid metabolism and on the bone marrow.
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