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

Abstract

Objective: The antineutrophil 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, USA, 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, USA, 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 HRQoL in AAV to date, and the results have underpinned the development of 35 candidate items for a disease-specific, patient-reported outcome questionnaire. Keywords: ANCA-associated vasculitis, quality of life, patient-reported outcomes, granulomatosis with polyangiitis, microscopic polyangiitis, eosinophilic granulomatosis with polyangiitis IntroductionGranulomatosis with polyangiitis (GPA), microscopic polyangiitis (MPA), and eosinophilic granulomatosis with polyangiitis (EGPA) are organ-and life-threatening multisystem diseases of small arteries, known as the antineutrophil cytoplasmic antibody (ANCA)-associated vasculitides (AAVs). 1 Patients present with inflammation in multiple body areas, including the lungs, ear, nose, kidneys, joints, and nerves, 2 with fevers, weight loss, and fatigue.3 Treatment is with glucocorticoids and other immunosuppressants. 4 A third of patients will have irreversible damage at diagnosis, 5 and 40% will suffer relapse. 6 One-year mortality in AAV has been >15%, 7 a poorer survival rate than breast or prostate cancer; 8 however, mortality in AAV has improved, likely due to the adoption of newer treatment regimens.9,10 Surveys have demonstrated impairments in health-related quality of life (HRQoL) in AAV, in particular fatigue. 18Robson et al A clinician-derived survey to assess patient perspectives in granulomatosis with polyangiitis was developed in 1998; 19 however, there have...

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

“…It has good face, content and construct validity, is reliable, feasible and discriminates among disease states. Patients have played a key role within every stage of development 22. This article describes the underlying structure of the final AAV-PRO and its validation in terms of reliability, feasibility, discrimination and construct validity.…”

“…Stage 1: qualitative analysis, item production and testing in the UK, USA and Canada resulting in a 35-iten candidate ANCA-associated vasculitis patient-reported outcomes (AAV-PRO) questionnaire (completed) 22. Stage 2: large-scale parallel survey of people with AAV in the UK and USA, to investigate the underlying scale structure of the AAV-PRO.…”

Utility of the Brief Illness Perception Questionnaire to Monitor Patient Beliefs in Systemic Vasculitis

“…The demographic characteristics by diagnosis were consistent with known characteristic distributions for these types of vasculitis. For example, the ratios between female and male participants in this study were characteristic of each disease (25)(26)(27).…”

Ab0606 long-Term Biological Treatment in Large Vessels Vasculitis: A Retrospective Single-Center Study on 30 Patients From 2011 to 2018

“…Methods: A secondary analysis of all interview transcripts from a previous project exploring health-related quality of life in relation to AAV was performed. 4 Deductive analysis was used to map appropriate areas discussed by patients to the self-management framework proposed by Lorig and Holman. 1 Inductive analysis was used to identify emergent themes that did not fit into this framework.…”

“…Thirty-four patients were diagnosed within the last 2 years and 16 were diagnosed more than 2 years ago. 4 From the core tasks proposed in Lorig and Holman's framework, patients with AAV appear to attribute particular importance to maintaining life roles and to a lesser extent medication management and dealing with the emotions of chronic disease. Core skills necessary to self-manage in AAV include learning to take action as part of self-efficacy, forming productive health-care partnerships, learning about decision making (requiring disease and self-awareness), utilising resources effectively, and problemsolving.…”