New treatment for severe vasculitis?

September 6, 2010

A new strategy for treating patients who have antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis, a severe form of vasculitis, provides the same benefits as the current standard of care used for more than 40 years but requires less frequent treatments.

A new strategy for treating patients who have antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis, a severe form of vasculitis, provides the same benefits as the current standard of care used for more than 40 years but requires less frequent treatments. Early results of an NIH-sponsored study suggested that patients with disease relapses-typically recurrences of fever, fatigue, kidney damage, or bleeding in the lungs-respond better to the new regimen.

The current standard of care for ANCA-associated vasculitis combines a 3- to 6-month course of daily cyclophosphamide plus corticosteroids, followed by long-term daily azathioprine (AZA) plus corticosteroids. Before this treatment regimen became available, about 80% of patients died as a result of kidney failure or bleeding in the lungs within 2 years of disease onset, it was noted. Now more than 90% of patients experience remission after they receive cyclophosphamide-based therapy, but a high rate of relapse and a need for re-treatment remain, according to researchers.

The study was designed to find an alternative therapy for patients with ANCA-associated vasculitis to induce disease remission and reduce or eliminate maintenance corticosteroid use and to find a less toxic therapy that also prolongs remission. The 197 participants were divided randomly into 2 groups, one receiving intravenous rituximab therapy once a week for 1 month plus corticosteroids, and the other receiving 3 to 6 months of daily cyclophosphamide therapy plus corticosteroids, followed by daily AZA.

After 6 months, 64% of patients in the rituximab group and 53% of those in the cyclophosphamide group had no disease activity and were able to discontinue the use of corticosteroids; 67% of patients with relapsing disease in the rituximab group had no disease activity and were able to discontinue all corticosteroid use after therapy, compared with 42% in the cyclophosphamide group. The investigators observed no major differences in the overall adverse
effects in patients from the 2 groups.

The study was conducted by the Immune Tolerance Network. It appears online in the New England Journal of Medicine (http://www.nejm.org). For more information about the NIH and its programs, visit http://www.nih.gov. For a comprehensive discussion of vasculitis, go to www.musculoskeletalnetwork.com to see“Meeting the challenge of the vasculitides” [“Article Archive,” December 9, 2009], authored by Karen Adams, MD, and Simon Carette, MD, at University Health Network and Mount Sinai Hospital in Toronto.