Dataset Information

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

ABSTRACT:

Objective

ANCA-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.

Methods

An 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.

Results

89 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.

Conclusion

There 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.

SUBMITTER: Quinn KA

PROVIDER: S-EPMC9262834 | biostudies-literature | 2022 Aug

REPOSITORIES: biostudies-literature

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Publications

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

Quinn Kaitlin A KA Monti Sara S Christensen Robin R Jayne David D Langford Carol A CA Lanier Georgia E GE Mahr Alfred A Pagnoux Christian C Shea Beverley B Viðarsdóttir Maria Bjork MB Tomasson Gunnar G Merkel Peter A PA

Seminars in arthritis and rheumatism 20220428

<h4>Objective</h4>ANCA-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.<h4>Methods</h4>An international 3-round online Delphi exercise was conducted. Survey participants included patients with AAV and phy ...[more]

PMID: 35523065

Similar Datasets

Project description:Rationale & objectiveWe sought to elicit patient preferences regarding the use of plasma exchange in antineutrophil cytoplasmic antibody-associated vasculitis (AAV) and its tradeoffs of risk of kidney failure and risk of serious infection.Study designPatient survey.Setting & participantsThe online survey was circulated to adults with AAV via kidney and vasculitis networks in Canada, the United Kingdom, and the United States.OutcomesRespondents reviewed the estimated 1-year risks of kidney failure and serious infection in AAV with and without plasma exchange across 5 serum creatinine categories (150, 250, 350, 450, and 600 μmol/L). For each scenario, participants indicated whether or not they would choose plasma exchange.Analytical approachResponses were assessed with multilevel multivariable logistic regression models to identify predictors of respondent choice regarding treatment with plasma exchange.ResultsThe 470 respondents from the 13 countries (United States 61.7%, United Kingdom 20.0%, Canada 13.8%, and other countries 4.5%) had a mean age of 58.6 (SD 14.3) years, 70.2% women. Respondents were more likely to choose plasma exchange in scenarios at high risk of kidney failure and serious infection (creatinine level of 350 or 450 μmol/L) compared with lower risk scenarios or the highest risk scenario. However, 145 (30.9%) chose plasma exchange across all scenarios, whereas 80 (17.0%) declined plasma exchange across all scenarios. Respondents from the United Kingdom (OR, 2.61; 95% CI, 1.09-6.22) who received previous dialysis (OR, 2.70; 95% CI, 1.12-6.52) or received previous plasma exchange (OR, 5.62; 95% CI, 2.72-11.61) were more likely to choose plasma exchange, whereas older respondents (OR, 0.98; 95% CI, 0.96-0.99 per 1 year increase) were less likely.LimitationsUnclear generalizability to non-English-speaking, older, and less health literate adults, possible responder bias, survivor bias, lack of individualized risk assessments for kidney failure, and serious infection.ConclusionsPatients with AAV do not express a consistent choice for plasma exchange, which highlights the need for shared decision making.

Project description:Antineutrophil cytoplasm antibody (ANCA)-associated vasculitides are small-vessel vasculitides that include granulomatosis with polyangiitis (formerly Wegener's granulomatosis), microscopic polyangiitis, and eosinophilic granulomatosis with polyangiitis (Churg-Strauss syndrome). Renal-limited ANCA-associated vasculitides can be considered the fourth entity. Despite their rarity and still unknown cause(s), research pertaining to ANCA-associated vasculitides has been very active over the past decades. The pathogenic role of antimyeloperoxidase ANCA (MPO-ANCA) has been supported using several animal models, but that of antiproteinase 3 ANCA (PR3-ANCA) has not been as strongly demonstrated. Moreover, some MPO-ANCA subsets, which are directed against a few specific MPO epitopes, have recently been found to be better associated with disease activity, but a different method than the one presently used in routine detection is required to detect them. B cells possibly play a major role in the pathogenesis because they produce ANCAs, as well as neutrophil abnormalities and imbalances in different T-cell subtypes [T helper (Th)1, Th2, Th17, regulatory cluster of differentiation (CD)4+ CD25+ forkhead box P3 (FoxP3)+ T cells] and/or cytokine-chemokine networks. The alternative complement pathway is also involved, and its blockade has been shown to prevent renal disease in an MPO-ANCA murine model. Other recent studies suggested strongest genetic associations by ANCA type rather than by clinical diagnosis. The induction treatment for severe granulomatosis with polyangiitis and microscopic polyangiitis is relatively well codified but does not (yet) really differ by precise diagnosis or ANCA type. It comprises glucocorticoids combined with another immunosuppressant, cyclophosphamide or rituximab. The choice between the two immunosuppressants must consider the comorbidities, past exposure to cyclophosphamide for relapsers, plans for pregnancy, and also the cost of rituximab. Once remission is achieved, maintenance strategy following cyclophosphamide-based induction relies on less toxic agents such as azathioprine or methotrexate. The optimal maintenance strategy following rituximab-based induction therapy remains to be determined. Preliminary results on rituximab for maintenance therapy appear promising. Efforts are still under way to determine the optimal duration of maintenance therapy, ideally tailored according to the characteristics of each patient and the previous treatment received.

Dataset Information

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

Objective

Methods

Results

Conclusion

Publications

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

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