Outcome measures used in clinical trials for Behçet syndrome: a systematic review.

J Rheumatol

From the Division of Rheumatology, Department of Internal Medicine, Cerrahpasa Medical School, Istanbul University, Istanbul, Turkey; Division of Rheumatology and Department of Epidemiology, University of Pennsylvania, Philadelphia, PA, USA; Department of Clinical Epidemiology, VU University Medical Center, Amsterdam, The Netherlands; Department of Rheumatology, Marmara University School of Medicine, Istanbul; Department of Rheumatology, Istanbul Medeniyet University, Goztepe Training and Research Hospital, Istanbul, Turkey.

Published: March 2014

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.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4508275PMC
http://dx.doi.org/10.3899/jrheum.131249DOI Listing

Publication Analysis

Top Keywords

outcome measures
24
measures clinical
16
clinical trials
16
behçet syndrome
8
systematic review
8
management strategy
8
diversity variability
8
validated outcome
8
outcomes outcome
8
outcome
7

Similar Publications

Importance: Care management benefits community-dwelling patients with dementia, but studies include few patients with moderate to severe dementia or from racial and ethnic minority populations, lack palliative care, and seldom reduce health care utilization.

Objective: To determine whether integrated dementia palliative care reduces dementia symptoms, caregiver depression and distress, and emergency department (ED) visits and hospitalizations compared with usual care in moderate to severe dementia.

Design, Setting, And Participants: A randomized clinical trial of community-dwelling patients with moderate to severe dementia and their caregivers enrolled from March 2019 to December 2020 from 2 sites in central Indiana (2-year follow-up completed on January 7, 2023).

View Article and Find Full Text PDF

Importance: Hypertension is the primary cardiovascular risk factor in Africa. Recently revised World Health Organization guidelines recommend starting antihypertensive dual therapy; clinical efficacy and tolerability of low-dose triple combination remain unclear.

Objectives: To compare the effect of 3 treatment strategies on blood pressure control among persons with untreated hypertension in Africa.

View Article and Find Full Text PDF

Sensitivity to Environmental Stress and Adversity and Lung Cancer.

JAMA Netw Open

January 2025

Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health, Rockville, Maryland.

Importance: Sensitivity to environmental stress and adversity may influence lung cancer risk, highlighting a critical link between psychosocial factors and cancer etiology.

Objective: To evaluate whether genetically estimated sensitivity to environmental stress and adversity is associated with lung cancer risk.

Design, Setting, And Participants: Data were obtained from a genome-wide association study identifying 37 independent genetic variants strongly associated with sensitivity to environmental stress and adversity and a cross-ancestry genome-wide meta-analysis from the International Lung Cancer Consortium.

View Article and Find Full Text PDF

Longitudinal Analysis of Obesity Drug Use and Public Awareness.

JAMA Netw Open

January 2025

Johns Hopkins Ciccarone Center for the Prevention of Cardiovascular Disease, Johns Hopkins University School of Medicine, Baltimore, Maryland.

Importance: Obesity, a chronic disease with escalating global prevalence, poses considerable health risks. Glucagon-like peptide 1 receptor agonists (GLP-1RAs), including liraglutide, semaglutide, and tirzepatide, have demonstrated efficacy for weight loss in clinical trials. The paradigm shift in the approach to obesity management drugs (OMDs) may offer an opportunity to examine online search activity and prescription trends.

View Article and Find Full Text PDF

Background: Interest in noninvasive treatment of basal cell carcinoma (BCC) has been increasing. For superficial BCC, it has been demonstrated that imiquimod cream, 5%, has high long-term efficacy, but for nodular BCC (nBCC), long-term evidence is sparse.

Objectives: To evaluate whether superficial curettage (SC) followed by imiquimod cream, 5%, is noninferior to surgical excision (SE) in nBCC after 5 years of treatment.

View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!