ANCA-associated vasculitis: new options beyond steroids and cytotoxic drugs

Small vessel vasculitic syndromes--Wegener's granulomatosis, microscopic polyangiitis and renal limited vasculitis (which are associated with circulating antineutrophil cytoplasmic autoantibodies)--are an important cause of renal failure. Present immunosuppressive regimens that are based on cyclophosphamide have significantly increased survival rates. However, these treatments are toxic, increase the risk of infection and do not cure disease. Therefore, newer approaches are required. Understanding disease pathogenesis has allowed rational use for newer therapies such as rituximab, which depletes B cells. Unfortunately, blockade of promising targets such as TNF-alpha, which was thought to be a pivotal cytokine in inflammation, has not shown benefit in a randomised controlled trial. Better understanding of the pathogenesis of the disease is the key to the development of novel targeted therapies, which are urgently required to improve patient prognosis. Gene therapy with targeted delivery of specific proteins is an exciting future prospect.