scholarly journals Update on Outcome Measure Development for Large Vessel Vasculitis: Report from OMERACT 12

2015 ◽  
Vol 42 (12) ◽  
pp. 2465-2469 ◽  
Author(s):  
Sibel Zehra Aydin ◽  
Haner Direskeneli ◽  
Antoine Sreih ◽  
Fatma Alibaz-Oner ◽  
Ahmet Gul ◽  
...  
Keyword(s):  
Disease Activity ◽  
Outcome Measures ◽  
Research Agenda ◽  
Task Force ◽  
Large Vessel Vasculitis ◽  
Large Vessel ◽  
Disease Assessment ◽  
Activity Assessment ◽  
Delphi Exercise ◽  
Disease Activity Assessment

Objective.The rarity of large vessel vasculitis (LVV) is a major factor limiting randomized controlled trials in LVV, resulting in treatment choices in these diseases that are guided mainly by observational studies and expert opinion. Further complicating trials in LVV is the absence of validated and meaningful outcome measures. The Outcome Measures in Rheumatology (OMERACT) vasculitis working group initiated the Large Vessel Vasculitis task force in 2009 to develop data-driven, validated outcome tools for clinical investigation in LVV. This report summarizes the progress that has been made on a disease activity assessment tool and patient-reported outcomes in LVV as well as the group’s research agenda.Methods.The OMERACT LVV task force brought an international group of investigators and patient research partners together to work collaboratively on developing outcome tools. The group initially focused on disease activity assessment tools in LVV. Following a systematic literature review, an international Delphi exercise was conducted to obtain expert opinion on principles and domains for disease assessment. 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, all to ensure that the approach to disease assessment includes measures of patients’ perspectives and that patients have input into the research agenda and process.Results.The preliminary results of both the Delphi exercise and the qualitative interviews were discussed at the OMERACT 12 (2014) meeting and the completion of the analyses will produce an initial set of domains and instruments to form the basis of next steps in the research agenda.Conclusion.The research agenda continues to evolve, with the ultimate goal of developing an OMERACT-endorsed core set of outcome measures for use in clinical trials of LVV.

scholarly journals Assessment of Disease Activity in Large-vessel Vasculitis: Results of an International Delphi Exercise

2017 ◽  
Vol 44 (12) ◽  
pp. 1928-1932 ◽  
Author(s):  
Sibel Z. Aydin ◽  
Haner Direskeneli ◽  
Peter A. Merkel ◽  
Keyword(s):  
Disease Activity ◽  
Morning Stiffness ◽  
Large Vessel Vasculitis ◽  
Large Vessel ◽  
Clinical Activity ◽  
Activity Assessment ◽  
Core Set ◽  
Delphi Exercise ◽  
International Experts ◽  
Disease Activity Assessment

Objective.To arrive at consensus for candidate outcomes for disease activity assessment in large-vessel vasculitis (LVV) in clinical trials.Methods.A Delphi survey including 99 items was circulated among international experts for 3 rounds.Results.Fifty-seven items were accepted for both giant cell arteritis and Takayasu arteritis. Sixty-seven percent of experts voted to have a common approach for both diseases with additional disease-specific items such as weight loss, scalp tenderness/necrosis, morning stiffness, dizziness, visual symptoms, and imaging.Conclusion.This study highlights similarities and differences in experts’ perspectives for assessing clinical activity in LVV and may guide a consensus-driven core set of validated outcomes.

scholarly journals Disease activity assessment in large vessel vasculitis

Reumatismo ◽  
2011 ◽  
Vol 63 (2) ◽  
Author(s):  
L. Magnani ◽  
A. Versari ◽  
D. Salvo ◽  
M. Casali ◽  
G. Germanò ◽  
...  
Keyword(s):  
Disease Activity ◽  
Large Vessel Vasculitis ◽  
Large Vessel ◽  
Activity Assessment ◽  
Disease Activity Assessment

scholarly journals Development of a Core Set of Outcome Measures for Large-vessel Vasculitis: Report from OMERACT 2016

2017 ◽  
Vol 44 (12) ◽  
pp. 1933-1937 ◽  
Author(s):  
Antoine G. Sreih ◽  
Fatma Alibaz-Oner ◽  
Tanaz A. Kermani ◽  
Sibel Z. Aydin ◽  
Peter F. Cronholm ◽  
...  
Keyword(s):  
Focus Groups ◽  
Outcome Measures ◽  
Large Vessel Vasculitis ◽  
Assessment Tools ◽  
Large Vessel ◽  
Disease Assessment ◽  
Patient Interviews ◽  
Core Set ◽  
Delphi Exercise ◽  
Disease Specific

Objective.Among the challenges in conducting clinical trials in large-vessel vasculitis (LVV), including both giant cell arteritis (GCA) and Takayasu arteritis (TA), is the lack of standardized and meaningful outcome measures. The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group initiated an international effort to develop and validate data-driven outcome tools for clinical investigation in LVV.Methods.An international Delphi exercise was completed to gather opinions from clinical experts on LVV-related domains considered important to measure in trials. Patient interviews and focus groups were completed to identify outcomes of importance to patients. The results of these activities were presented and discussed in a “Virtual Special Interest Group” using telephone- and Internet-based conferences, discussions through electronic mail, and an in-person session at the 2016 OMERACT meeting. A preliminary core set of domains common for all forms of LVV with disease-specific elements was proposed.Results.The majority of experts agree with using common outcome measures for GCA and TA, with the option of supplementation with disease-specific items. Following interviews and focus groups, pain, fatigue, and emotional effect emerged as health-related quality of life domains important to patients. Current disease assessment tools, including the Birmingham Vasculitis Activity Score, were found to be inadequate to assess disease activity in GCA and standardized assessment of imaging tests were felt crucial to study LVV, especially TA.Conclusion.Initial data from a clinician Delphi exercise and structured patient interviews have provided themes toward an OMERACT-endorsed core set of domains and outcome measures.

scholarly journals Update on Outcome Measure Development in Large-vessel Vasculitis: Report from OMERACT 2018

2019 ◽  
Vol 46 (9) ◽  
pp. 1198-1201 ◽  
Author(s):  
Sibel Z. Aydin ◽  
Joanna C. Robson ◽  
Antoine G. Sreih ◽  
Catherine Hill ◽  
Fatma Alibaz-Oner ◽  
...  
Keyword(s):  
Outcome Measures ◽  
Patient Reported Outcome ◽  
Large Vessel Vasculitis ◽  
Large Vessel ◽  
Disease States ◽  
Core Set ◽  
Patient Reported ◽  
Delphi Exercise ◽  
Patient Acceptable Symptom State ◽  
Selection Of

Objective.The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group seeks to develop validated outcome measures for use in trials for large-vessel vasculitis (LVV).Methods.An international Delphi exercise conducted among investigators identified items considered important to measure active disease. In parallel, qualitative research with patients was conducted, including interviews and focus groups.Results.Next steps prioritized by the group for LVV include (1) defining disease states (remission, flare, and patient-acceptable symptom state) and (2) selection of patient-reported outcome tools.Conclusion.The ultimate goal is to develop an OMERACT-endorsed core set of outcome measures for use in clinical trials of LVV.

scholarly journals AB0373 PSYCHOMETRIC PROPERTIES OF OUTCOME MEASUREMENT INSTRUMENTS FOR LARGE VESSEL VASCULITIS: A SYSTEMATIC LITERATURE REVIEW

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 1213.1-1213
Author(s):  
G. Boleto ◽  
A. Berti ◽  
P. A. Merkel ◽  
S. Aydin ◽  
H. Direskeneli ◽  
...  
Keyword(s):  
Disease Activity ◽  
Psychometric Properties ◽  
Outcome Measures ◽  
Outcome Measurement ◽  
Large Vessel Vasculitis ◽  
Large Vessel ◽  
Measurement Instruments ◽  
Core Domain ◽  
Patient Reported ◽  
Disease Damage

Background:Giant cell arteritis (GCA) and Takayasu arteritis (TAK) are two forms of chronic progressive large-vessel vasculitis (LVV) of unknown etiology. In 2016, the OMERACT Vasculitis Working Group proposed the development of a Core Domain Set of outcome measures for LVV including organ and arterial function, fatigue, pain, biomarkers, and death (1). Understanding the psychometric properties of outcome measurement instruments is essential when selecting among instruments to use in research; a summary of such properties for measures of LVV has not been developed.Objectives:To systematically review and summarize the psychometric properties of outcome measurement instruments used to measure the domains of the OMERACT core domain set for LVV.Methods:A comprehensive search of several databases (Medline, EMBASE, Cochrane, among others) from inception to July 14, 2020 was conducted. Articles were included if they covered psychometric properties of instruments used in LVV. Following the COSMIN and OMERACT frameworks, different psychometric properties (validity, inter- and intra-observer reliability, sensitivity to change, and feasibility) of outcome measurement instruments used in LVV (GCA and TAK) were assessed. Risk of bias was assessed according to the COSMIN checklist.Results:Among the 3534 articles identified, 15 studies focusing on the development or validation of psychometric properties on LVV met the predefined criteria. Two were development studies and 13 were validation studies. These studies provided information on 13 instruments: 5 instruments specific to TAK, 2 specific to systemic vasculitides, and 6 general, non-disease-specific instruments. No instruments specific to GCA were identified.Of the main psychometric properties assessed in the included studies, 40% had a low, 47% had moderate, and 13% had high risk of bias. Construct validity was the property most frequently assessed (in 93% of the tools) (Figure 1).In TAK, the Indian Takayasu Clinical Activity Score 2010 (ITAS2010) showed good consistency (r=0.97), reliability (intra-observer, ICC=0.60; inter-observer, ICC=0.92) and validity (correlation with Physician Global Assessment (PGA) (r=0.73)) for disease activity. Regarding disease damage, the Disease Extent Index-Takayasu (DEI-Tak) showed good validity (correlation with NIH score 94%, k=0.85). Non-specific vasculitis instruments such as the Vasculitis Damage Index (VDI) and the Birmingham Vasculitis Activity Score (BVAS) showed moderate validity in the assessment of disease damage in GCA (cumulative glucocorticoid dose and disease duration, r=0.30 and r=0.29) and TAK (cumulative glucocorticoid dose and disease duration, r=0.29 and r=0.25) in the former and disease activity in GCA in the later (PGA, r=0.50).Six non-vasculitis-specific patient-reported outcomes (PROs) instruments were identified, all showing low to moderate validity in GCA/TAK.Conclusion:The psychometric properties of 13 outcome measures to study LVV covering the OMERACT domains of disease activity, damage, and patient-reported outcomes were assessed. ITAS2010, DEI-Tak, VDI, and BVAS were the instruments with better psychometric properties for disease activity and/or damage. Disease activity and/or damage instruments specific for GCA, and validated PROs for both GCA and TAK are needed.References:[1]Sreih GA, Alibaz-Oner F, Kermani TA, Aydin SZ, Cronholm PF, Davis T, et al. Development of a Core Set of Outcome Measures for Large-vessel Vasculitis: Report from OMERACT 2016 | The Journal of Rheumatology [Internet]. [cité 26 avr 2020]. Disponible sur: http://www.jrheum.org/content/44/12/1933.longDisclosure of Interests:None declared

scholarly journals Development of Outcome Measures for Large-vessel Vasculitis for Use in Clinical Trials: Opportunities, Challenges, and Research Agenda

2011 ◽  
Vol 38 (7) ◽  
pp. 1471-1479 ◽  
Author(s):  
HANER DIRESKENELI ◽  
SIBEL Z. AYDIN ◽  
TANAZ A. KERMANI ◽  
ERIC L. MATTESON ◽  
MAARTEN BOERS ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Research Agenda ◽  
Trial Design ◽  
Large Vessel Vasculitis ◽  
The Other ◽  
Large Vessel ◽  
Therapeutic Trials ◽  
Core Set ◽  
Existing Data

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.

scholarly journals THU0538 IN PSORIATIC ARTHRITIS PATIENTS CONSIDERED IN REMISSION BY THEIR RHEUMATOLOGIST, CAN DISCORDANCE IN DISEASE ACTIVITY ASSESSMENT BETWEEN PATIENT AND RHEUMATOLOGIST BE EXPLAINED BY RESIDUAL INFLAMMATION AS MEASURED BY ULTRASONOGRAPHIC EXAMINATION?

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 508-508
Author(s):  
M. Moly ◽  
C. Lukas ◽  
J. Morel ◽  
B. Combe ◽  
G. Mouterde
Keyword(s):  
Psoriatic Arthritis ◽  
Disease Activity ◽  
Demographic Data ◽  
Univariate Analysis ◽  
Power Doppler ◽  
Minimal Disease Activity ◽  
Activity Assessment ◽  
Grey Scale ◽  
Minimal Disease ◽  
Disease Activity Assessment

Background:Psoriatic arthritis (PsA) is a heterogeneous disease and its assessment is sometimes difficult. Perception of disease activity by patient and physician is frequently discordant in patients in clinical remission. Ultrasound (US) is an imaging technique, which can detect inflammation in PsA.Objectives:The aim of our study was to assess whether persistence of disease activity evaluated by the patient, considered in remission by his rheumatologist, was associated with inflammation measured by US.Methods:We performed a transversal monocentric study. PsA patients were included if they met the CASPAR criteria and were considered in remission by their rheumatologist. Demographic data, characteristics of the disease and treatments were collected. Discordance was defined by a difference between patient’s and rheumatologist’s global assessment ≥30/100 on a Visual Analogic Scale. An US examination was performed on 50 joints, 28 tendons and 14 entheses by an independent investigator. Synovial or tendon sheath hypertrophy and PD signal were evaluated on a semi-quantitative scale, B Mode and PD signal abnormalities on entheses were searched, according to the EULAR-OMERACT scoring system. US remission was defined by no power Doppler (PD) signal on joints, tendons and entheses and minimal US activity by maximum one PD signal on the same sites. Univariate and multivariate analyses were performed to evaluate factors associated with US abnormalities.Results:Sixty-two PsA patients were included. 40.3% were women, the mean (SD) age was 55 (14) years, 42% were in US remission and 71% in minimal US activity (Table 1), 19.4% had ≥1 PD synovitis and 88.7% had a B mode synovitis, 95.2% had a B mode abnormality on entheses and 51.6% had ≥1 PD signal on entheses. Thirty nine percent had a discordant disease activity assessment with their rheumatologist. In univariate analysis, discordance was not associated with US remission (OR=1.71 (95%CI 0.61-4.83), p=0.224) or US minimal disease activity (OR=0.99 (95%CI 0.32-3.05), p=0.602). In multivariate analysis, US remission was independently associated with female gender (OR=3.94 (95%CI 1.20-12.9), p=0.024) and younger age (OR=0.95 (95%CI 0.91-0.99), p=0.027). Minimal US activity was associated with history of enthesis lesion (OR=11.26 (95%CI 1.34-94.93), p=0.026) and age (OR=0.95 (95%CI 0.90-1), p=0.044).Table 1.Ultrasound characteristics of the 62 PsA patients.N (%)Ultrasound remission26 (41.9)Ultrasound minimal disease activity44 (71)Patients with ≥1 grey scale synovitis55 (88.7)Patients with ≥1 Power Doppler synovitis12 (19.4)Patients with ≥1 grey scale tenosynovitis15 (24.2)Patients with ≥1 Power Doppler tenosynovitis1 (1.6)Patients with ≥1 grey scale enthesitis lesion (thickness, hypo echogenicity, calcification, enthesophyte, erosion, bursitis)59 (95.2)Patients with ≥1 Power Doppler enthesitis32 (51.6)Conclusion:Our study showed persistent inflammation evaluated by US in PsA patients considered in remission by their rheumatologist. However, prevalence of residual inflammation evaluated by US was not higher in patients with self-assessment of their disease discordant from their rheumatologist.Disclosure of Interests:Marie Moly: None declared, Cédric Lukas: None declared, Jacques Morel: None declared, Bernard Combe Grant/research support from: Novartis, Pfizer, Roche-Chugai, Consultant of: AbbVie; Gilead Sciences, Inc.; Janssen; Eli Lilly and Company; Pfizer; Roche-Chugai; Sanofi, Speakers bureau: Bristol-Myers Squibb; Gilead Sciences, Inc.; Eli Lilly and Company; Merck Sharp & Dohme; Pfizer; Roche-Chugai; UCB, Gael Mouterde: None declared

Intraepithelial Autoimmune Bullous Dermatoses Disease Activity Assessment and Therapy

2021 ◽  
Author(s):  
Carmen M. Montagnon ◽  
Julia S. Lehman ◽  
Dedee F. Murrell ◽  
Michael J. Camilleri ◽  
Stanislav N. Tolkachjov
Keyword(s):  
Disease Activity ◽  
Activity Assessment ◽  
Disease Activity Assessment
Sign in / Sign up

Export Citation Format

Share Document