The goal of the Vasculitis Translational Research Program (VTRP) is to develop clinical and translational research initiatives across many types of systemic vasculitis. Vasculitis is a family of rare diseases characterized by inflammation within blood vessels. Tremendous progress over the last few decades has transformed vasculitis from frequently fatal into a chronic illness. However, treatment with potentially toxic medications, including glucocorticoids and other immune suppressants such as cyclophosphamide, is generally required to induce remission. Although most patients with vasculitis achieve remission with treatment, the majority of patients experience one or more relapses. Relapses often result in additional permanent organ damage and can be fatal. Currently, there are few known predictors of relapse in vasculitis, and there are no known predictors to reliably guide treatment decisions. The identification of novel therapeutic targets and of biomarkers that predict clinical outcomes to guide patient-specific therapeutic decisions is a major focus of the VTRP.
The VTRP was established in 2013 to evaluate patients with known vasculitis or with clinical suspicion of vasculitis. Diseases of interest include granulomatosis with polyangiitis (GPA, Wegener’s), microscopic polyangiitis (MPA), eosinophilic granulomatosis with polyangiitis (EGPA, Churg-Strauss), polyarteritis nodosa (PAN), giant cell arteritis (GCA), Takayasu’s arteritis (TAK), and idiopathic aortitis among other types of vasculitis.
Some of the objectives of the VTRP include:
- Comprehensive clinical evaluation and treatment recommendations for all patients within the program.
- Novel imaging studies to assess for vascular inflammation in medium and large vessel vasculitis.
- Characterization of novel aspects of neutrophil biology in ANCA-associated vasculitis, in collaboration with the NIAMS Systemic Autoimmunity Branch (Kaplan Lab).
- Discovery of novel biomarkers in peripheral blood and tissue through whole-genome gene expression profiling and other high-throughput techniques.
- Clinical trials of novel therapeutics in small, medium, and large vessel vasculitis.
- Collaboration with the Vasculitis Clinical Research Consortium.
Grayson PC, Amudala NA, McAlear CA, Leduc RL, Shereff D, Richesson R, Fraenkel L, Merkel PA. Causal Attributions about Disease Onset and Relapse in Patients with Systemic Vasculitis. J Rheumatol. 2014 May;41(5):923-30. doi: 10.3899/jrheum.131096
Mooney J, Spalding N, Poland F, Grayson P, Leduc R, McAlear CA, Richesson RL, Shereff D, Merkel PA, Watts RA. The informational needs of patients with ANCA-associated vasculitis--development of an informational needs questionnaire. Rheumatology (Oxford). 2014 Mar 12. [Epub ahead of print]
Kissin EY, Grayson PC, Cannella AC, Demarco PJ, Evangelisto A, Goyal J, Al Haj R, Higgs J, Malone DG, Nishio MJ, Tabechian D, Kaeley GS. Musculoskeletal ultrasound objective structured clinical examination: an assessment of the test. Arthritis Care Res (Hoboken). 2014 Jan;66(1):2-6