Researchers find new therapy for vasculitis
Forty years after the development of the only therapy for vasculitis, a rare but severe disease of blood vessels, researchers have found a new therapy that is at least as effective, requires fewer treatments and promises far fewer side effects. Federally-funded researchers reported Wednesday that treatment with the biotech drug rituximab, already marketed as Rituxan for the treatment of B cell lymphoma and rheumatoid arthritis, is slightly more effective than the existing drug, cyclophosphamide, at inducing remission from vasculitis and significantly more effective at inducing remission when the disease has relapsed.
“The reason this is a big deal is that this is a disease where people would come in and be told, ‘Listen, we are probably going to be able to get on top of your life-threatening disease by using cyclophosphamide, but you are going to have major side effects down the road from this drug,’” Dr. Robert Spiera of the Hospital for Special Surgery in New York, said in a statement. Spiera is a coauthor of the report, published in the New England Journal of Medicine. Side effects of cyclophosphamide can include infections, infertility and cancer.
Vasculitis is an inflammation of small to medium-sized blood vessels caused by the attack of antibodies on immune cells called neutrophils. The two main forms of this disease, autoimmune vasculitis and Wegener’s granulomatosis, are so-called rare or orphan diseases that affect fewer than 6,000 Americans. Forty years ago, more than 90% of patients diagnosed with the diseases could expect to die within three years. Then a young physician named Dr. Anthony Fauci, who now heads the National Institute of Allergy and Infectious Diseases -- which sponsored the new trial -- showed that six months of treatment with cyclophosphamide plus steroids, followed by daily doses of azathioprine plus steroids, could place more than 90% of patients in remission. But there is a high risk of relapse and, of course, the aforementioned side effects.
Anecdotal evidence suggested that rituximab might be beneficial for victims of the disease, so Dr. John Stone of the Massachusetts General Hospital and Dr. Ulrich Specks of the Mayo Clinic in Rochester, Minn., organized a clinical trial of 197 patients, about half of whom had previously received cyclophosphamide treatment but had suffered a relapse. They were randomly assigned to receive either intravenous rituximab therapy once a week for a month, plus steroids, or the standard cyclophosphamide regimen. After six months, 64% of patients in the rituximab group had no disease activity and were able to discontinue use of steroids, compared with 53% of those in the cyclophosphamide group. Among those who had previously been treated, 67% of those receiving rituximab had no disease activity and were able to stop steroid use, compared with 42% of the cyclophosphamide group.
The researchers plan to follow the patients for 18 months to monitor for relapses and long-term effects of the treatment.
