Mycophenolate Mofetil to Treat Wegener's Granulomatosis and Related Vascular Inflammatory Conditions

This study will examine the safety and effectiveness of the drug mycophenolate mofetil (MPM) in treating Wegener's granulomatosis and related inflammatory vessel diseases. Blood vessel inflammation in these patients may involve different parts of the body, including the brain, nerves, eyes, sinuses, lungs, kidneys, intestinal tract, skin, joints, heart, and other sites. The more severe the involvement, the more likely the disease will be life-threatening. Standard treatment consists of combination drug therapy with prednisone and a cytotoxic agent-usually cyclophosphamide or methotrexate. However, some patients in whom this treatment is initially successful have a disease relapse; other patients cannot take the medications because of other health problems or because of severe side effects of the drugs. MPM is approved by the Food and Drug Administration to prevent kidney transplant rejection. It is chemically similar to another cytotoxic drug called azathioprine, which has been beneficial in maintaining remission in patients with Wegener's granulomatosis who have been treated successfully with cyclophosphamide. Because MPM is more effective than azathioprine in preventing organ rejection, it may also prove beneficial as a second-line treatment for Wegener's granulomatosis. Patients with Wegener's granulomatosis or related inflammatory vessel diseases who have had a relapse following treatment with cyclophosphamide and methotrexate or who cannot take one or both of these drugs may be eligible for this study. Only patients who have been treated at NIH in the methotrexate protocol or the cyclophosphamide switching to methotrexate protocol, or who have received the exact same treatment from their own physician may participate. Participants will have a complete medical evaluation including laboratory studies. Consultations, X-rays and biopsies of affected organs may also be done if indicated for diagnosis or treatment. Patients with active disease will be given MPM an