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    Steroid Sparing

    Retrospective series experience ranks mycophenolate mofetil as first-line adjuvant for pemphigus treatment

    Baltimore -- The immunosuppressant agent mycophenolate mofetil (CellCept) should be considered the steroid-sparing drug of choice in the management of pemphigus, according to Daniel Mimouni, M.D.

    Dr. Mimouni presented a retrospective analysis of data collected in a series of 42 patients with pemphigus vulgaris or pemphigus foliaceus who were either refractory to steroid monotherapy, would relapse during steroid dose-tapering attempts, or were experiencing serious side effects with other adjuvant treatments. The mycophenolate mofetil was very well tolerated, and with its addition 70 percent of those difficult patients achieved a complete remission, defined as disease control with use of prednisone <10 mg every other day.

    At the time the data were analyzed, a few other patients had achieved partial remission and were expected to go on to complete remission with continued treatment. The treatment was considered a failure in about 25 percent of the patients, of whom two stopped mycophenolate mofetil because of side effects and the rest could not undergo successful tapering of their steroid dose.

    "Not only is mycophenolate mofetil very effective when added to prednisone for treating pemphigus, but it is also very safe, especially compared to other steroid-sparing options, such as azathioprine. Therefore, after excluding patients with fulminant disease who require very aggressive therapy, we recommend that mycophenolate mofetil be considered the first-line adjuvant drug for pemphigus patients not responding to systemic-steroid treatment alone," Dr. Mimouni said.

    Dr. Mimouni is currently a dermatologist at Rabin Medical Center, Petah Tikva, Israel, but undertook the retrospective analysis when he was an immunodermatology fellow at Johns Hopkins School of Medicine, Baltimore. The patients in the series were treated by Grant J. Anhalt, M.D., professor of dermatology and pathology, and Hossein Nousari, M.D., co-director of immunodermatology. Those physicians began to explore the efficacy and safety of mycophenolate mofetil as an adjunctive treatment for pemphigus about five years ago based on published literature demonstrating its utility and favorable safety profile when used as a steroid-sparing agent to prevent acute graft rejection in transplant patients as well as more limited reports describing successful treatment of pemphigus patients.

    Gradual Tapering Employed The Johns Hopkins dermatologists initiate treatment of pemphigus using a moderate steroid dose, prescribing 1 mg/kg lean body weight. When added as a steroid-sparing agent, mycophenolate mofetil is administered twice daily at a relatively high dose of 35 to 45 mg/kg/day. Tapering of the steroid begins after about one to one-and-a-half months, although time to onset of efficacy of the mycophenolate mofetil as measured by clinical improvement, and changes in antibody levels is about three months.

    In the series of patients who achieved a complete remission with combined mycophenolate mofetil/prednisone treatment, the median time to that event was nine months after initiation of mycophenolate mofetil. Overall, mycophenolate mofetil was administered for an average of 18 to 24 months.

    Abdominal pain was the most common side effect associated with mycophenolate mofetil. However, 80 percent of the patients had no adverse reactions at all, and the gastrointestinal-related disturbances that occurred were generally mild and improved with continued treatment.

    One of the two adverse reaction-related drop-outs complained of severe nausea and the second patient developed neutropenia, but that could not be proven to be caused by the mycophenolate mofetil.

    "Systemic steroids have been a tremendous advance in the management of pemphigus, which, before steroids were introduced in the 1950s, was often fatal. Nevertheless, steroid treatment is successful as monotherapy in only a minority of patients, about 30 percent, and the rest require additional treatment," Dr. Mimouni said.

    Dr. Mimouni and colleagues have written a paper describing their experience using mycophenolate mofetil as adjuvant therapy in pemphigus patients. That article is in press in Archives of Dermatology.

    CellCept is a product of Roche Laboratories. The research was supported by an educational grant from the company, but none of the physicians has any financial interest in it.

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