A systematic review of patient-reported outcome measures in patients with anti-neutrophil cytoplasmic antibody associated vasculitis

Abstract

Objectives ANCA associated vasculitis (AAV) is associated with significant morbidity, fatigue, pain and poor health-related quality of life (HRQoL). This review aims to assess the comprehensiveness of existing patient reported outcome measures (PROMs) used in AAV and identify associations with poorer HRQoL outcomes. Methods A literature review of studies using PROMs, including those labelled HRQoL in people with AAV as a primary or secondary study outcome were screened and reviewed up to July 2023. Quality was assessed using the Critical Appraisal Skills Programme. Results A total of 30 articles were included which utilised 22 different PROM tools. 76.7% (n = 23) used the SF-36 or a variation as a generic measure of health status and or HRQoL. Two studies developed a disease specific PROM. The AAV-PRO showed good psychometric properties but potential limitations in capturing all relevant aspects of the disease experience for AAV patients. Factors associated with poorer HRQoL included: neurological and sinonasal involvement, women and younger patients. 86.6% of studies showed no meaningful relationships between the SF-36 and BVAS, VDI or disease duration. Depression and anxiety were common and socioeconomic factors such as unemployment were significantly associated with poorer mental health outcomes. Glucocorticoids were found to be independently associated with worse SF-36 scores. Conclusion Generic PROMs are useful in measuring significant changes but lack sensitivity to specific symptoms and unique AAV-related issues, while existing disease specific PROMs have limitations and may not fully capture AAV patient's perspective on disease and treatment burden.

Treatment goals in ANCA-associated vasculitis: defining success in a new era

“…Evaluating success and optimizing outcomes in the management of AAV thus requires routine monitoring using a combination of assessments to evaluate the extent of AAV damage, susceptibility to comorbidities, and the impact of AAV symptoms and adverse events on the patient as well as AAV disease activity, and treatment strategy modified accordingly. Key validated tools ( 95 ) available for measuring patient outcomes are outlined in Table 2 . A recent evaluation of the performance of tools for assessing outcome found the instruments with the best performance in AAV were the Birmingham Vasculitis Activity Score (BVAS) for disease activity, the VDI for tissue damage, and the AAV-PRO for health-related quality of life ( 96 ).…”