Wegener’s granulomatosis (WG) is a chronic, multisystem disease, characterized by a small vessel vasculitis. First line therapy is cyclophosphamide. Despite the use of other immunosuppressive agents, including methotrexate, azathioprine and mycophenolate mofetil, to maintain remission, relapse often occurs – with many patients requiring further courses of cyclophosphamide over many years.
Rituximab is being tested in a wide variety of small vessel vascultides but no prospective studies have been done in any of them, to my knowlledge. One pilot trial for Wegener’s showed a high response rate in a small group of patients. However, there was also the RAVE trial which became the basis for the FDA approval. The U.S. Food and Drug Administration approved Rituxan (rituximab), in combination with glucocorticoids (steroids) on April 12, 2011 to treat patients with Wegener’s granulomatosis (WG) and microscopic polyangiitis (MPA), two rare disorders that cause blood vessel inflammation (vasculitis).
Alistair J. Ferraro , Clara J. Day , Mark T. Drayson , and Caroline O. Savage
Effective therapeutic use of rituximab in refractory Wegener’s granulomatosis
Nephrol. Dial. Transplant. 20: 622-625
C M G Cheung, P I Murray, and C O S Savage
Successful treatment of Wegener’s granulomatosis associated scleritis with rituximab
Br J Ophthalmol, November 1, 2005; 89(11): 1542 – 1542.
K. A. Keogh, S. R. Ytterberg, F. C. Fervenza, K. A. Carlson, D. R. Schroeder, and U. Specks Rituximab for Refractory Wegener’s Granulomatosis: Report of a Prospective, Open-Label Pilot Trial
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Despina Eleftheriou , Marianna Melo , Stephen D. Marks , Kjell Tullus , John Sills , Gavin Cleary , Pavla Dolezalova , Seza Ozen , Clarissa Pilkington , Pat Woo , Nigel Klein , Michael J. Dillon , and Paul A. Brogan
Biologic therapy in primary systemic vasculitis of the young
Rheumatology Advance Access published on August 1, 2009, DOI 10.1093/rheumatology/kep148.
Rheumatology 48: 978-986.