The role of mycophenolate mofetil in clinical renal transplantation.

Mycophenolate mofetil (MM) is the ester derivative of mycophenolic acid (MPA), which exerts immunosuppressive activity by inhibiting de novo purine biosynthesis. Following animal models and pilot studies in human renal allograft recipients that confirmed MM's ability to prevent and half rejection episodes, 3 large multicenter studies of 500 patients each were conducted in North America, Europe, and Australia to compare MM with azathioprine or placebo, all drugs being used in conjunction with prednisone and cyclosporine. The results demonstrated a statistically significant reduction in episodes of allograft rejection in the MM groups with a concomitant reduction in the use of antirejection therapy, especially antilymphocyte antibody therapy. Adverse-event profiles were similar for all groups, although there were slightly more gastrointestinal and tissue-invasive cytomegalovirus (CMV) episodes in the MM groups. It is concluded that MM, which recently received Food and Drug Administration (FDA) approval in the United States, should be an important addition to the transplant physician's immunosuppressive armamentarium.