ANCA-Associated Vasculitis Patient-Reported Outcome (AAV PRO)

AAV PRO was developed to capture patient-reported outcomes specific to AAV, including organ-specific symptoms, systemic-symptoms, treatment side effects, social and emotional impact, concerns about the future and physical function. It aims to provide a comprehensive understanding of the patient experience beyond clinical measures alone, allowing healthcare providers and researchers to better assess disease activity, treatment efficacy, and overall patient needs.

The identified domains offer a comprehensive profile of the impact of AAV on patients’ everyday life and were felt by the patient partners to represent “what AAV was to them”.

The underpinning qualitative work for the AAV-PRO included in-depth interviews with patients with granulomatosis with polyangiitis, microscopic polyangiitis and eosinophilic granulomatosis, from rheumatology and nephrology departments in the UK, USA and Canada. Fifty patient 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 after cognitive testing and linguistic translatability assessment. Further information can be found here: https://pubmed.ncbi.nlm.nih.gov/29379322/

A large- scale survey using the long-list of 35-item candidate questionnaire items was then conducted, to determine the final scale structure and validate the AAV-PRO. Further information can be found here: https://pubmed.ncbi.nlm.nih.gov/29695498/

Participants were recruited via the patient organisation Vasculitis UK and the Vasculitis Patient-Powered Research Network in the US. 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.

There were 626 participants with AAV; >25% reporting ‘active disease’. Exploratory factor analysis (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’ (p<0.001). Construct validity was demonstrated by correlations between domain scores and the EQ-5D-5L (range r=-0.55 to 0.78), all p<0.0001. In participants reporting ‘no change’ (n=97) during the test-retest, intraclass correlation coefficient values were high (range 0.89-0.96) for each domain. For more information, please refer to the validation paper.

The AAV PRO questionnaire contains 29 items (questions).

Each item has 5 response options scored from 0 to 4 (higher score representing greater severity).

Questions group together into 6 domains: Organ-Specific Symptoms (5 items), Systemic
Symptoms (4 items), Treatment Side Effects (5 items), Social and Emotional Impact (6 items),
Concerns about the Future (5 items), Physical Function (4 items).

The AAV PRO was originally validated for paper-and-pen completion, any transitions to digital format should be approached with care and reviewed by our team. You can contact us if you have any questions around digital migrations.

Pen and Paper

You may also be interested in the following PROs developed by the same team:

1. GCA-PRO. The GCA-PRO is a validated outcome measure for use in clinical trials, observation studies and clinical practice. It can also be used as a communication tool between patients and their clinicians, to highlight areas of greatest importance to patients, within face to face or online consultations.
2. Steroid PRO. The Steroid PRO is the first validated and reliable patient-reported outcome measure designed to assess the impact of glucocorticoid-specific therapy on patients’ well-being and quality of life.