Study Shows IVIG Safe, Effective Treatment for Muscle Disease

For release: Wednesday, December 29, 1993

Patients with a painful and debilitating muscle disease called dermatomyositis showed dramatic improvement on a treatment regimen of intravenous immune globulin (IVIG) during a recent double-blind, placebo-controlled trial. The study, which was conducted at the National Institute of Neurological Disorders and Stroke (NINDS), will be published in the December 30 issue of the New England Journal of Medicine .*

"The IVIG treatment resulted in some of the most significant improvements I have ever seen in patients with neurological disease. In addition, the treatment was safe and well tolerated," said Marinos C. Dalakas, M.D., chief of the NINDS Neuromuscular Diseases Section and author of the study.

"What's exciting about this study is the promise it holds for treating a variety of neuromuscular disorders," said Patricia A. Grady, Ph.D., acting director of the NINDS. "We are always encouraged when the scientific knowledge gained from one study can extend to helping an even broader group of patients."

Dermatomyositis belongs to a family of auto-immune diseases called "inflammatory myopathies" (including polymyositis and inclusion body myositis) that cause progressive muscle weakness and inflammation, and affect about 1 out of 100,000 persons. Patients with dermatomyositis have difficulty performing everyday activities like getting dressed, getting up from a chair, and climbing stairs. As the disease progresses, patients are often unable to walk. Dermatomyositis is also characterized by a chronic scaly skin rash on the eyelids and hands. The disease affects both children and adults and females more often than males.

The study was double-blinded, meaning that neither the doctor nor the patients knew which therapy patients were receiving. In the trial, 15 dermatomyositis patients were randomly assigned to receive one monthly infusion of high-dose immune globulin or placebo for three months, at the end of which they had the option of crossing over to the other treatment. (Initially, 8 were assigned to immune globulin and 7 to placebo.) After crossover a total of 12 patients had received immune globulin. Nine of these patients, who were severely disabled, experienced major improvement and resumed almost normal function, two of the patients showed mild improvement, and one had no change in condition.

Of the 11 placebo-treated patients, none showed a major improvement, 3 had a mild improvement, and 3 had no change in their condition and 5 had a worsening of the condition. Four of the patients who crossed over to the placebo after major improvement with IVIG therapy returned to their original condition of disability--two returned to wheelchairs.

The study authors described the dramatic response in the group treated with IVIG: "These patients had not felt so strong since the onset of their disease two to six years earlier...They were able to get up from the wheelchair, run, climb stairs and behave normally."

One of the patients who received the IVIG treatment, said: "It seemed unreal! I was able to do many of the things I had been unable to do for more than a year."

The clinical assessment of the patients' physical improvements was based on changes in muscle strength, neuromuscular symptoms as measured by testing the function of specific muscle groups, and changes in the rash, photographed before and after the infusions. The investigators also performed repeat muscle biopsies on the patients, which allowed them to measure muscle fibers and look at other cellular changes.

The study was prompted by the need for a safer and more effective therapy for dermatomyositis patients who have become resistant to existing treatments. The standard therapies have been corticosteroids and other immunosuppressive drugs, which for many patients are not effective in controlling the disease. Those drugs can also cause a variety of serious side effects including hypertension, bone marrow suppression, lung disease and infections.

Researchers were particularly encouraged by the safety of the IVIG treatment. The only reported side effects were headaches during the course of the 12-hour infusion, but even so, two patients who reported severe headaches stated that the benefit from the treatment far outweighed the adverse effect.

Dr. Dalakas said this study illustrated some of the specific actions of the IVIG treatment on the immune system in the patients. Because immunoglobulins are the major carriers of the body's disease-fighting antibodies, the variety of antibodies contained in the drug preparation--made up from a pool of 5000 normal donors--seemed to counteract the antibodies found in the patient. The IVIG also interfered with the action in the patient's immune system that generated muscle-damaging factors.

IVIG has proven effective for the treatment of other neuromuscular diseases including Guillain-Barre syndrome. The treatment is now being studied by NINDS for polymyositis, myasthenia gravis and neuropathies. IVIG has also been tried as a treatment for multiple sclerosis, with varying results.

"The major concern about the IVIG therapy right now is its expense," said Dr. Dalakas. Cost estimates for a single infusion range from $8,000 to $26,000. Patients require the infusions about every six weeks in order to maintain the clinical benefits.

The NINDS, one of the National Institutes of Health located in Bethesda, Md., is the nation's principal supporter of research on the brain and nervous system. The Institute is a lead agency for the Congressionally designated Decade of the Brain.

*"A Controlled Trial of High-Dose Intravenous Immune Globulin Infusions As Treatment for Dermatomyositis." Marinos C. Dalakas, M.D., Isabel Illa, M.D., James M. Dambrosia, Ph.D., Shawke A. Soueidan, M.D., Daniel P. Stein, M.D., Carlos Otero, M.D., Steven T. Dinsmore, D.O., and Susan McCrosky, R.N., New England Journal of Medicine; December 30, 1993; pp 1993-2000 Volume 329, Number 27.

Date Last Modified: Tuesday, October 28, 2008

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