Mycophenolate mofetil is effective for managing symptoms of the most common type of lupus

Mayo Clinic investigators have found that the medication mycophenolate mofetil is effective for managing symptoms of systemic lupus erythematosus (SLE), the most common type of lupus, for patients whose SLE does not affect the kidneys.

Importantly, the researchers say the drug is well tolerated and has fewer side effects than many other medications used to treat the disease.

“This would be considered a nice addition to medications we can use for these patients -- an alternative and widely applicable to many patients,” says Kevin Moder, M.D., Mayo Clinic rheumatologist, who will present the findings at the American College of Rheumatology Annual Scientific Meeting in San Antonio, Texas.

Patients who have SLE, an autoimmune, inflammatory disease, may experience symptoms from fevers to joint pain to excessive fatigue to hair loss, among others. The disease also may affect major organs, and the kidneys are especially vulnerable. SLE affecting the kidneys is treated differently from non-renal SLE. The Food and Drug Administration has approved mycophenolate mofetil for SLE affecting the kidneys. The new Mayo Clinic research indicates, however, that the drug could help many more lupus patients.

Currently, there is no curative treatment for SLE. Also, many medications used to treat this disease have considerable side effects. For example, Dr. Moder explains that one currently available immunomodulatory treatment, cyclophosphamide, puts patients at risk for infertility and cancer.

“It’s a significant step if the medication is effective but has fewer side effects [than medications currently approved by the FDA for SLE],” says Dr. Moder.

Dr. Moder explains that while mycophenolate mofetil helps many patients, it is not 100 percent effective. He describes the drug, however, as “very well received” by patients on his study. “Mycophenolate mofetil was very well tolerated and it has very few side effects,” he says.

The Mayo Clinic investigators undertook this trial because mycophenolate mofetil had proven effective for treatment of SLE affecting the kidneys, and the drug appeared promising for SLE that does not affect the kidneys in animal models and early human studies. Mycophenolate mofetil is not currently FDA-approved for non-renal SLE, but it is gaining wide usage among physicians, according to Dr. Moder.

This study of mycophenolate mofetil was conducted in a six-month, open label, prospective trial. Of the 23 patients who started in the study, 18 completed it and 12 met the investigators’ criteria for a significant response to mycophenolate mofetil. Response to the drug was measured by reduction in dosage of prednisone, a commonly prescribed medication for SLE; scores on the Systemic Lupus Activity Measure, a widely used disease activity instrument; and in the level of double-stranded DNA, which is a marker for SLE. Patient and physician global assessment scores, subjective measurement vehicles indicating how patients are doing overall, also improved.

Next steps for mycophenolate mofetil research at Mayo Clinic, indicates Dr. Moder, may include studies combining this drug with other medications. Currently in the United States, approximately 1.4 million people have some form of lupus, according to the National Women’s Health Information Center.