title: Development of outcome measures for large-vessel vasculitis for use in clinical trials: opportunities, challenges, and research agenda.
abstract: Giant cell (GCA) and Takayasu's arteritis (TAK) are 2 forms of large-vessel vasculitis (LVV) that involve the aorta and its major branches. GCA has a predilection for the cranial branches, while TAK tends to affect the extracranial branches. Both disorders may also cause nonspecific constitutional symptoms. Although some clinical features are more common in one or the other disorder and the ages of initial presentation differ substantially, there is enough clinical and histopathologic overlap between these disorders that some investigators suggest GCA and TAK may be 2 processes within the spectrum of a single disease. There have been few randomized therapeutic trials completed in GCA, and none in TAK. The lack of therapeutic trials in LVV is only partially explained by the rarity of these diseases. It is likely that the lack of well validated outcome measures for LVV and uncertainties regarding trial design contribute to the paucity of trials for these diseases. An initiative to develop a core set of outcome measures for use in clinical trials of LVV was launched by the international OMERACT Vasculitis Working Group in 2009 and subsequently endorsed by the OMERACT community at the OMERACT 10 meeting. Aims of this initiative include: (1) to review the literature and existing data related to outcome assessments in LVV; (2) to obtain the opinion of experts and patients on disease content; and (3) to formulate a research agenda to facilitate a more data-based approach to outcomes development.
authors: Marmara University School of Medicine, Istanbul, Turkey.
authors: Haner
authors: H
authors: Direskeneli
authors: Sibel Z
authors: SZ
authors: Aydin
authors: Tanaz A
authors: TA
authors: Kermani
authors: Eric L
authors: EL
authors: Matteson
authors: Maarten
authors: M
authors: Boers
authors: Karen
authors: K
authors: Herlyn
authors: Raashid A
authors: RA
authors: Luqmani
authors: Tuhina
authors: T
authors: Neogi
authors: Philip
authors: P
authors: Seo
authors: Ravi
authors: R
authors: Suppiah
authors: Gunnar
authors: G
authors: Tomasson
authors: Peter A
authors: PA
authors: Merkel
mesh_terms: D002986:Clinical Trials as Topic; D013700:Giant Cell Arteritis / Q000401:mortality / Q000628:therapy*; D006801:Humans; D017063:Outcome Assessment, Health Care / Q000379:methods / Q000639:trends*; D011788:Quality of Life; D015203:Reproducibility of Results; D012106:Research / Q000639:trends*; D015996:Survival Rate; D013625:Takayasu Arteritis / Q000401:mortality / Q000628:therapy*; D016896:Treatment Outcome