Health related quality of life in ANCA associated vasculitis and item generation for a disease specific patient reported outcome measure

ABSTRACTObjective: The anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitides (AAVs) are multisystem diseases of the small blood vessels. Patients experience irreversible damage and psychological effects from AAV and its treatment. An international collaboration was created to investigate the impact of AAV on health-related quality of life (HRQoL), and develop a disease-specific patient-reported outcome measure to assess outcomes of importance to patients.Methods: Patients with AAV from the UK, US, and Canada were interviewed to identify salient aspects of HRQoL affected by AAV. The study was overseen by a steering committee including four patient research partners. Purposive sampling of interviewees ensured representation of a range of disease manifestations and demographics. Inductive analysis was used to identify themes of importance to patients; these were further confirmed by a free-listing exercise in the US. Individual themes were recast into candidate items, which were scrutinized by patients, piloted through cognitive interviews and received a linguistic and translatability evaluation. Results: Fifty interviews, conducted to saturation, with patients from the UK, US and Canada, identified 55 individual themes of interest within seven broad domains: general health perceptions, impact on function, psychological perceptions, social perceptions, social contact, social role and symptoms. Individual themes were constructed into >100 candidate questionnaire items which were then reduced and refined to 35 candidate items.Conclusion: This is the largest international qualitative analysis of health related quality of life in ANCA associated vasculitis to date, the results have underpinned the development of 35 candidate items for a disease-specific, patient-reported outcome questionnaire

Patient perceptions of glucocorticoids in anti-neutrophil cytoplasmic antibody-associated vasculitis

© 2017, The Author(s). Granulomatosis with polyangiitis (GPA), microscopic polyangiitis (MPA), and eosinophilic granulomatosis with polyangiitis (EGPA) are multisystem diseases of small blood vessels, collectively known as the anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitides (AAV). This study explores the patient’s perspective on the use of glucocorticoids, which are still a mainstay of treatment in AAV. Patients with AAV from the UK, USA, and Canada were interviewed, using purposive sampling to include a range of disease manifestations and demographics. The project steering committee, including patient partners, designed the interview prompts and cues about AAV, its treatment, and impact on health-related quality of life. Interviews were transcribed and analysed to establish themes grounded in the data. A treatment-related code was used to focus analysis of salient themes related to glucocorticoid therapy. Fifty interviews were conducted. Individual themes related to therapy with glucocorticoids emerged from the data and were analysed. Three overarching themes emerged: (1) Glucocorticoids are effective at the time of diagnosis and during relapse, and withdrawal can potentiate a flare, (2) glucocorticoids are associated with salient emotional, physical, and social effects (depression, anxiety, irritation, weight gain and change in appearance, diabetes mellitus, effect on family and work); and (3) patient perceptions of balancing the risks and benefits of glucocorticoids. Patients identified the positive aspects of treatment with glucocorticoids; they are fast-acting and effective, but, they voiced concerns about adverse effects and the uncertainty of the dose-reduction process. These results may be informative in the development of novel glucocorticoid-sparing regimens

CanVasc Consensus Recommendations for the Management of Antineutrophil Cytoplasm Antibody-associated Vasculitis: 2020 Update

Objective In 2015, the Canadian Vasculitis Research Network (CanVasc) created recommendations for the management of antineutrophil cytoplasm antibody (ANCA)-associated vasculitides (AAV) in Canada. The current update aimed to revise existing recommendations and create additional recommendations, as needed, based on a review of new available evidence. Methods A needs assessment survey of CanVasc members informed questions for an updated systematic literature review (publications spanning May 2014-September 2019) using Medline, Embase, and Cochrane. New and revised recommendations were developed and categorized according to the level of evidence and strength of each recommendation. The CanVasc working group used a two-step modified Delphi procedure to reach >80% consensus on the inclusion, wording and grading of each new and revised recommendation. Results Eleven new and 16 revised recommendations were created, and 12 original (2015) recommendations were retained. New and revised recommendations are discussed in detail within this document. Five original recommendations were removed, of which 4 were incorporated into the explanatory text. The supplementary appendix for practical use was revised to reflect the updated recommendations. Conclusion The 2020 updated recommendations provide rheumatologists, nephrologists, and other specialists caring for patients with AAV in Canada with new management guidance, based on current evidence and consensus from Canadian experts

Using the International Classification of Function, Disability and Health (ICF) to Compare Areas of ANCA-Associated Vasculitits (AAV) Measured in Clinical Trials to those Important to Patients with AAV and Clinicians who are Involved in their Care

Background: The International Classification of Function, Disability and Health (ICF) describes health using 1424 categories from 4 components: body functions (BF), body structures (BS), activities and participation (AP) and contextual factors (environmental (EF) and personal (PF)). In this study the ICF was used to describe and compare aspects of ANCA-Associated Vasculitis (AAV) measured in clinical trials and those important to clinicians and patients. Methods: Individual interviews and focus groups were used to capture the perspective of AAV patients. Clinicians’ perspective was obtained with an email-based questionnaire. Outcomes used in AAV randomized trials were extracted from results of a systematic review of literature. Identified concepts were mapped to the ICF according to previously published ICF linking rules, and the resulting lists of relevant AAV outcomes were compared descriptively. Results: Twelve individual interviews and 2 focus groups represented the patient perspective while responses from 27 clinicians yielded the clinicians’ perspective. Systematic literature review identified 67 clinical trials and 28 abstracts from which measured outcomes were extracted. All three perspectives demonstrated detailed coverage of ICF components BF and BS. In the component AP patients and clinicians identified similar ICF categories, a number of which were under-sampled by AAV trials. Contextual factors appear to be significantly more relevant to patients than clinicians and researchers. Conclusion: Patients and clinicians have different views of the relevance of various AAV outcomes, and these views differ from what is measured in clinical trials of AAV. This highlights the need for a broad and standardized approach to developing and selecting outcomes for complex medical conditions such as AAV

Updating OMERACT core set of domains for ANCA-associated vasculitis: Patient perspective using the international classification of function, disability, and health

Objective: Aspects of ANCA-associated vasculitis (AAV) prioritized by patients with AAV were described using the International Classification of Function, Disability, and Health. Methods: Items identified during 14 individual interviews were incorporated into ICF-based questionnaire administered to participants of 2 vasculitis patient symposia, 36 in UK and 63 in USA. Results: Categories identified as at least “moderately relevant” by ≥ 5% of subjects included 44 body functions, 14 body structures, 35 activities and participation, 31 environmental factors, and 38 personal factors. Conclusion: Identified categories differ from those captured by the current OMERACT core set and those prioritized by vasculitis experts

Validation of the ANCA-associated vasculitis patient-reported outcomes (AAV-PRO) questionnaire

© 2018 Article author(s). Objectives To finalise and validate a disease-specific patient-reported outcome (PRO) measure: The ANCA-associated vasculitis patient-reported outcome (AAV-PRO) questionnaire. Using a 35-item candidate questionnaire developed following 50 qualitative interviews in the UK, USA and Canada, a longitudinal survey was conducted to determine the final scale structure and validate the AAV-PRO. Methods Participants were recruited via Vasculitis UK and the Vasculitis Patient-Powered Research Network. The 35-item candidate questionnaire was completed at baseline and 3 months; UK participants completed the EuroQol-5D-5L (EQ-5D-5L), while US participants completed a test-retest exercise, 3-5 days after baseline. Scale structure was defined using exploratory factor analysis (EFA) and Rasch analysis. Convergent and known groups validity, test-retest reliability and longitudinal construct validity were assessed. Results There were 626 participants with AAV; >25% reporting active disease'. EFA and Rasch analysis supported a 29-item profile measure comprising six domains: organ-specific symptoms', 'systemic symptoms', treatment side effects', 'social and emotional impact', 'concerns about the future' and 'physical function'. Mean domain scores were higher for participants with 'active disease' versus 'remission' (

Development of a core set of outcome measures for large-vessel vasculitis: Report from OMERACT 2016

Copyright © 2017. All rights reserved. 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

Update on outcome measure development for large vessel vasculitis: Report from OMERACT 12

© Copyright 2015 The Journal of Rheumatology. All rights reserved. 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