Takayasu arteritis (TA) is a true orphan disease. There are no published controlled clinical trials and the evidence on which to develop management strategies and therapeutic guidelines is rudimentary, particularly when compared to the advances seen in other vasculitides and inflammatory rheumatic diseases1. Additional significant challenges to more effective management exist. These include a lack of awareness of the disease and a resultant delay in diagnosis, as well as the absence of gold standards for the quantification of disease activity and imaging2. However, the outlook is beginning to change for the better, with early evidence pointing towards improved outcomes for patients3. This improvement reflects earlier use of combined immunosuppression and the increased availability of noninvasive imaging modalities, particularly 18F-fluorodeoxyglucose positron emission computerized tomography (18F-FDG-CT-PET), high-resolution ultrasound (US), magnetic resonance, and CT angiography (MRA and CTA).
Perhaps in light of the paucity of clinical trial evidence, corticosteroids remain the mainstay of therapy and achieve remission in 60% of cases4. However, when tapered, most patients relapse. Our practice is to initiate combination immunosuppressive therapy at diagnosis to optimize control of disease activity and steroid-sparing2. Small open-label studies support the efficacy of methotrexate, azathioprine, and mycophenolate5. Although cyclophosphamide is typically reserved for refractory or life-threatening disease, we have some concerns about its efficacy in TA, and its use is often contraindicated due to a need to preserve fertility in this young, predominantly female, patient group. Despite optimal combination immunosuppression, up to 30% of patients fail to respond adequately.
This observation led to the investigation of biologic therapies already available for other rheumatic diseases. Nearly a decade ago Hoffman and colleagues reported the initial open-label study demonstrating the efficacy of tumor necrosis factor-α (TNF-α) blockade in TA6. Since …
Address correspondence to Professor Mason; Vascular Sciences, Imperial College London, Hammersmith Hospital, Du Cane Road, London, W12 0NN, UK. E-mail: justin.mason{at}imperial.ac.uk