ABSTRACT
Azathioprine is coverted intracellularly to purine thioanalogues that inhibit normal purine biosynthesis in DNA, RNA and coenzymes. The apparent efficacy of azathioprine in the first trial in adult newly diagnosed diabetics might be attributed to the uncontrolled nature of that trial and/or to a slower natural history of beta cell destruction in adults. Azathioprine was given to alternate patients age 15 to 50 years presenting with acute, symptomatic hyperglycemia requiring insulin treatment. The apparent success of azathioprine in the randomized pilot trial provided justification for undertaking a large double-blind controlled trial. Azathioprine treatment was associated with an increase in fasting C-peptide, but this effect was not sustained, indicating that the drug retards but does not prevent progressive beta cell destruction. A standard dose of azathioprine did not influence the remission phase in children with newly diagnosed Type I diabetes. Whether a higher dose of azathioprine, other agents, or combinations thereof would be effective remains to be determined.
