Faculty Bibliography

Background/Purpose: An unmet need for reliable, validated, and widely accepted outcome measures for trials in Behet's syndrome (BS) was identified through: i) a systematic review; ii) a survey among Behet's experts; and iii) an outcome measures interest group meeting during the 16th International Conference on Behet's Disease (1,2). The OMERACT Behet's Syndrome Working Group has been working to advance outcome measures in BS with the goal of creating a core set of data-driven measures for use in clinical trials. To identify domains, subdomains, and outcomes to be assessed in trials of BS, a Delphi exercise among BS experts and patients with BS has been initiated. This abstract describes the results for round 1 of the Delphi. Methods: A list of possible domains, subdomains, and outcomes was prepared using the results of a systematic literature review on outcomes assessed in previous studies in BS, patient priorities identified through qualitative interviews, and expert opinion. A 3- round Delphi was begun among physicians from different specialties experienced in BS and among patients with BS. The patient survey was the same as the physician survey with medical terms explained. The web-based survey was formatted in both English and Turkish and emailed to 123 physicians and 130 patients. Agreement by ³70% of either physicians or patients resulted in an item being accepted. Results: 74 physicians and 35 patients participated in Round 1. The physicians were experts in BS from 21 countries and from within a wide range of specialties, including Rheumatology (50%), Ophthalmology (12%), Internal Medicine (12%), Dermatology (16%), Gastroenterology (3%), and Neurology (1%). Among the participating patients there was good representation of each type of organ involvement. Table 1 shows the domains to be measured in all trials in BS that received ³70% endorsement by expert physicians and the additional subdomains endorsed for trials for each type of involvement. In addition to all of the domains identified by physicians, ³70% of patients endorsed the assessment of pain, fatigue, sleep, sexual functioning, psychological functioning, and acute phase reactants in all trials of BS. Conclusion: Multiple disease-related domains in BS have been identified by physicians and patients as important to address in clinical trials, suggesting that a core set for all trials will be needed and subdomains for subsets of disease (specific manifestations) will also be useful. Rating and ranking of these domains and subdomains in the next 2 rounds will enable the development of a core set of domains to be assessed in clinical trials of BS.(Table Presented)

BACKGROUND: Exercise is associated with major benefits in patients with rheumatic diseases for both cardiovascular and rheumatic status. However, information about exercise generally is not collected systematically in routine rheumatology care. A multidimensional health assessment questionnaire (MDHAQ), which was designed for busy clinical settings, includes a query about exercise status. We analyzed possible associations between change in MDHAQ exercise scores and other MDHAQ measures in patients with various rheumatic diseases over one year. METHODS: In one rheumatology clinical setting, all patients, regardless of diagnosis, complete an MDHAQ before seeing a rheumatologist. The MDHAQ includes scores for physical function, pain, and patient global estimate, compiled into an index, routine assessment of patient index data (RAPID3), as well as a self-report joint count and a query about exercise. Patients were classified into four groups according to their exercise status at baseline and one year later as: EXER-Yes (regular exercise), EXER-Yes; EXER-No (no regular exercise), EXER-Yes; EXER-Yes, EXER-No; and EXER-No, EXER-No. These groups were compared using the chi square and Kruskal-Wallis tests and analysis of variance (ANOVA). RESULTS: Patients who reported regular exercise at baseline were younger, had higher formal education, and better clinical status than other patients. The EXER-No, EXER-Yes group had greater improvement in other MDHAQ variables than patients in the other three groups. By contrast, the EXER-Yes, EXER-No group was the only group with poorer status one year later. CONCLUSIONS: The MDHAQ exercise query indicates that regular exercise is associated with better clinical status. Patients in the EXER-No, EXER-Yes group reported the best clinical improvement, although it is not known whether exercise preceded or followed the improved clinical status.

Background: Patient-reported assessments have a considerable weight both in disease activity assessment in practice and response criteria in clinical trials of rheumatoid arthritis (RA). Discrepancies and disagreement on global disease activity between patients and physicians may result in disagreement in treatment decisions in routine care and impact assessment of treatment response in clinical trials as well. Objectives: We aimed to illustrate the amount of discrepancy between patient and doctor global assessments (PGA and DGA) in routine care and asses the determinants thereof. Methods: We used data from RA patients registered in the Turkish Rheumatoid Arthritis Registry (TRAV), a prospective registry established in 2010 in order to record clinical data at each visit of all RA patients seen at three academic rheumatology centers in a routine care setting. Currently 1923 RA patients are registered in TRAV. We used the initial visits of these patients to define a global assessment discrepancy (GAD) variable, GAD=PGA-DGA. We calculated the average GAD, overall correlation of PGA with DGA and constructed linear regression models to determine the predictors of PGA, DGA and GAD. Age, gender, physical function (MD-HAQ score), pain score (numeric rating scale; NRS), fatigue score (NRS), patient's joint assessment (PJA), physician reported tender (TJC) and swollen joint counts (SJC), erythrocyte sedimentation rate (ESR) and center of follow-up were included in these models as potential predictors. Results: The mean age (standard deviation) of the registered patients was 52.0 (12.8) and 1561 (81%) were females. The average PGA was 3.59 (2.99) and the average DGA was 2.85 (2.63) Overall correlation of PGA and DGA was low (0.18) and average GAD was 0.70 (95% CI: 0.53-0.87). Confidence intervals around GAD indicated that on average patients rated their current disease activity as significantly worse than that of physicians. In the regression analyses; MD-HAQ, pain scores and center were common predictors of both PGA and DGA. Additional predictors of PGA were age, fatigue scores and PJA whereas additional predictors of DGA were ESR, PJA, TJC and SJC. Predictors of GAD were pain, fatigue, TJC, SJC, ESR and treatment center (see table). Conclusions: We found a weak correlation between patient and doctor global assessments of overall disease activity in RA patients under routine care. Higher pain and fatigue scores and different treatment centers were associated with a PGA higher than DGA whereas increasing TJC, SJC and ESR were associated with a DGA higher than PGA suggesting that patients and physicians prioritized different aspects of RA in their global assessments. Variation of the discrepancy by centers warrants further research with respect to a possible impact in multi-center treatment trials. (Table Presented)

PURPOSE OF REVIEW: Currently, there are no diagnostic criteria for vasculitides. To this end, there is a current European League Against Rheumatism and American College of Rheumatology initiative for formulating separate classification and diagnostic criteria for different forms of vasculitis. The authors of this review previously disagreed to separate classification and diagnostic criteria. They now expand this disagreement in light of both of more recent information and a reassessment of older communications. RECENT FINDINGS: We still can find no clear methodologies proposed to prepare separate diagnostic and classification criteria. Furthermore, the inadequate importance given to probabilities in discussing disease criteria was strikingly apparent. Among 77 articles on diagnostic/classification criteria making, not more than 4% discussed Bayes' theorem or predictive values or confidence intervals. The misconceptions related to the worry about circularity and the proper role of nomenclature in classification and diagnostic criteria continue. SUMMARY: Separate diagnostic and classification criteria are unrealistic. Classification criteria and nomenclature are only tools to a proper diagnosis, essentially not different for patient care or research. A frank discussion of probabilities in diagnosis is essential not only with the patients but also with all the stakeholders.

OBJECTIVES: To evaluate clinical response by baseline disease duration using 2-year data from the AMPLE trial. METHODS: Patients were randomised to subcutaneous abatacept 125 mg weekly or adalimumab 40 mg bi-weekly, with background methotrexate. As part of a post hoc analysis, the achievement of validated definitions of remission (Clinical Disease Activity Index (CDAI) 6 months). Disease Activity Score 28 (C-reactive protein) <2.6 or 6 months) across multiple clinical measures. CONCLUSIONS: Abatacept or adalimumab with background methotrexate were associated with similar onset and sustainability of response over 2 years. Patients treated early or later in the disease course achieved comparable clinical responses. TRIAL REGISTRATION NUMBER: NCT00929864, Post-results.