An international Delphi exercise to identify items of importance for measuring response to treatment in ANCA-associated vasculitis

Abstract

ObjectiveANCA-associated vasculitis (AAV) is characterized by fluctuating levels of disease activity, but no formal criteria exist to measure response to treatment. This Delphi exercise aimed to reach consensus about which measures are considered by patients and physicians to be most important when assessing response to treatment in clinical trials of AAV. MethodsAn international 3-round online Delphi exercise was conducted. Survey participants included patients with AAV and physicians with expertise in AAV. Survey participants were asked to rate (on a scale of 1–9) the importance of each item when assessing response to treatment in AAV. Items scored 7–9 by ≥70% participants were considered highly important. Results89 patients and 176 physicians completed three rounds of the Delphi exercise. The most highly rated items of response involved disease activity [extent of organ involvement, physician global assessment], mortality [survival], and patient-reported outcomes [patient global assessment and health-related quality of life measures]. Achievement of specific BVAS scores were highly rated only by physicians. Items highly rated only by patients included laboratory measures [changes on urinalysis and acute phase reactants], pain, and fatigue. Additional items related to damage and adverse events were highly rated by both groups. ConclusionThere is consensus between patients and physicians on many items considered important to measure when assessing response to treatment in AAV. There are some items considered important by only patients or only physicians. These data will inform the next steps in the development criteria of response to treatment in AAV.