RT Journal Article
SR Electronic
T1 Outcome Measures Used in Clinical Trials for Behçet Syndrome: A Systematic Review
JF The Journal of Rheumatology
JO J Rheumatol
FD The Journal of Rheumatology
SP 599
OP 612
DO 10.3899/jrheum.131249
VO 41
IS 3
A1 Gulen Hatemi
A1 Peter A. Merkel
A1 Vedat Hamuryudan
A1 Maarten Boers
A1 Haner Direskeneli
A1 Sibel Z. Aydin
A1 Hasan Yazici
YR 2014
UL http://www.jrheum.org/content/41/3/599.abstract
AB Behçet syndrome (BS) is a multisystem vasculitis that is most active during young adulthood, causing serious disability and significant impairment in quality of life. Differences in the disease course, severity, and organ involvement between patients, depending on the age at presentation and sex, makes it impossible to determine a single management strategy. The diversity and variability in the outcome measures used in clinical trials in BS makes it difficult to compare the results or inform physicians about the best management strategy for individual patients. There is a large unmet need to determine or develop validated outcome measures for use in clinical trials in BS that are acceptable to researchers and regulatory agencies. We conducted a systematic review to describe the outcomes and outcome measures that have been used in clinical trials in BS. This review revealed the diversity and variability in the outcomes and outcome measures and the lack of standard definitions for most outcomes and rarity of validated outcome tools for disease assessment in BS. This systematic literature review will identify domains and candidate instruments for use in a Delphi exercise, the next step in the development of a core set of outcome measures that are properly validated and widely accepted by the collaboration of researchers from many different regions of the world and from different specialties, including rheumatology, ophthalmology, dermatology, gastroenterology, and neurology.