Effective clinical research into the causes and treatments associated with large vessel vasculitis can be a challenge. The rarity of the disease limits the potential for randomized, controlled trials and the paucity of generally accepted outcome measures makes it difficult to assess treatments. A study in The Journal of Rheumatology outlined a concerted effort to tackle both of these challenges.
Revelatory clinical research is more difficult for some conditions than others. Diseases such as HIV, diabetes, and many types of cancer—while severe and potentially life-threatening—are good targets for clinical research. Disease causes, effective medication therapy, and a plethora of patients for clinical trials add up to a rich and ripe environment for research.
For conditions such as large vessel vasculitis (LVV), effective clinical research is far from a given. Vasculitis is a group of disorders that destroy blood vessels through inflammation. Both arteries and veins are affected by vasculitis, which can be classified by the cause, the location, the type of vessel, or the size of the vessel. LVV is typically used to denote the spectrum of primary vasculitides that causes chronic granulomatous inflammation predominantly involving the aorta and its major branches. Common conditions such as rheumatoid arthritis, along with rarer conditions such as Behçet disease, are considered LVV conditions.
There are two distinct factors that make effective clinical research into the causes and treatments associated with LVV difficult to come by: the rarity of the disease limits the potential for randomized, controlled trials; and the paucity of generally accepted outcome measures makes it difficult to assess treatments. A new study in The Journal of Rheumatology outlined a concerted effort to tackle both of these challenges.
In 2009, the Outcome Measures in Rheumatology (OMERACT) vasculitis working group initiated the Large Vessel Vasculitis task force to develop data-driven, validated outcome tools for clinical investigation in LVV. The task force gathered an international group of investigators and patient research partners to compare notes and develop outcome tools. It is not randomized, controlled clinical research, but it is a big positive step forward in that direction.
The effort included two concurrent steps: an exhaustive literature review to “study and identify expert opinion on principles and domains for disease assessment;” and qualitative patient research, including patient interviews, focus groups, and information from research partners.
The OMERACT vasculitis working group’s LVV task force is also conducting qualitative research with patients, including interviews, focus groups, and engaging patients as research partners.According to the researchers, it’s important that “the approach to disease assessment includes measures of patients’ perspectives and that patients have input into the research agenda and process.”
The research is still in the beginning phases, with a great deal of analysis still to come. But it’s a promising initial step in arriving at a consensus for clinical outcome assessments. And the impacts could reach beyond treatment of LVV, as vasculitis can be an accompanying symptom in rheumatoid arthritis, cancer, and infections such as hepatitis C.