Kaitlin A Quinn1, Sara Monti2, Robin Christensen3, David Jayne4, Carol A Langford5, Georgia E Lanier6, Alfred Mahr7, Christian Pagnoux8, Beverley Shea9, Maria Bjork Viðarsdóttir10, Gunnar Tomasson11, Peter A Merkel12. 1. Systemic Autoimmunity Branch, National Institutes of Health, NIAMS, Bethesda, MD, USA. Electronic address: Kaitlin.quinn@nih.gov. 2. Department of Rheumatology, Policlinico S. Matteo, IRCCS Fondazione, Pavia, Italy; University of Pavia, PhD in Experimental Medicine, Pavia, Italy. 3. Section for Biostatistics and Evidence-Based Research, the Parker Institute, Bispebjerg and Frederiksberg Hospital & Research Unit of Rheumatology, Department of Clinical Research, University of Southern Denmark, Odense University Hospital, Denmark. 4. Department of Medicine, University of Cambridge, United Kingdom. 5. Department of Rheumatic and Immunologic Diseases, Cleveland Clinic, Cleveland, OH, USA. 6. Sherborn, MA, USA. 7. Clinic for Rheumatology, Cantonal Hospital St. Gallen, Switzerland. 8. Vasculitis Clinic, Division of Rheumatology, Mount Sinai Hospital, University of Toronto, Toronto, Canada. 9. Ottawa Hospital Research Institute, School of Epidemiology and Public Health, University of Ottawa, Ottawa, Canada. 10. Reykjavik, Iceland. 11. Faculty of Medicine, University of Iceland and Landspitali University Hospital, Reykjavik, Iceland. 12. Division of Rheumatology, Department of Medicine, and Division of Epidemiology, Department of Biostatistics, Epidemiology, and Informatics, University of Pennsylvania, Philadelphia, PA, USA.
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.
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.
Authors: J H Stone; G S Hoffman; P A Merkel; Y I Min; M L Uhlfelder; D B Hellmann; U Specks; N B Allen; J C Davis; R F Spiera; L H Calabrese; F M Wigley; N Maiden; R M Valente; J L Niles; K H Fye; J W McCune; E W St Clair; R A Luqmani Journal: Arthritis Rheum Date: 2001-04
Authors: John H Stone; Peter A Merkel; Robert Spiera; Philip Seo; Carol A Langford; Gary S Hoffman; Cees G M Kallenberg; E William St Clair; Anthony Turkiewicz; Nadia K Tchao; Lisa Webber; Linna Ding; Lourdes P Sejismundo; Kathleen Mieras; David Weitzenkamp; David Ikle; Vicki Seyfert-Margolis; Mark Mueller; Paul Brunetta; Nancy B Allen; Fernando C Fervenza; Duvuru Geetha; Karina A Keogh; Eugene Y Kissin; Paul A Monach; Tobias Peikert; Coen Stegeman; Steven R Ytterberg; Ulrich Specks Journal: N Engl J Med Date: 2010-07-15 Impact factor: 91.245
Authors: Sara Monti; Kaitlin A Quinn; Robin Christensen; David Jayne; Carol Langford; Georgia E Lanier; Alfred Mahr; Christian Pagnoux; Maria Bjork Viðarsdóttir; Peter A Merkel; Gunnar Tomasson Journal: Semin Arthritis Rheum Date: 2020-09-29 Impact factor: 5.532
Authors: Loïc Guillevin; Christian Pagnoux; Alexandre Karras; Chahera Khouatra; Olivier Aumaître; Pascal Cohen; François Maurier; Olivier Decaux; Jacques Ninet; Pierre Gobert; Thomas Quémeneur; Claire Blanchard-Delaunay; Pascal Godmer; Xavier Puéchal; Pierre-Louis Carron; Pierre-Yves Hatron; Nicolas Limal; Mohamed Hamidou; Maize Ducret; Eric Daugas; Thomas Papo; Bernard Bonnotte; Alfred Mahr; Philippe Ravaud; Luc Mouthon Journal: N Engl J Med Date: 2014-11-06 Impact factor: 91.245
Authors: Nataliya Milman; Eilish McConville; Joanna C Robson; Annelies Boonen; Peter Tugwell; George A Wells; Dipayan Chaudhuri; Jill Dawson; Gunnar Tomasson; Susan Ashdown; Don Gebhart; Georgia Lanier; Jacqueline Peck; Carol A McAlear; Katherine S Kellom; Peter F Cronholm; Peter A Merkel Journal: J Rheumatol Date: 2019-02-01 Impact factor: 4.666
Authors: Christian Pagnoux; Alfred Mahr; Mohamed A Hamidou; Jean-Jacques Boffa; Marc Ruivard; Jean-Pierre Ducroix; Xavier Kyndt; François Lifermann; Thomas Papo; Marc Lambert; José Le Noach; Mehdi Khellaf; Dominique Merrien; Xavier Puéchal; Stéphane Vinzio; Pascal Cohen; Luc Mouthon; Jean-François Cordier; Loïc Guillevin Journal: N Engl J Med Date: 2008-12-25 Impact factor: 91.245
Authors: David R W Jayne; Gill Gaskin; Niels Rasmussen; Daniel Abramowicz; Franco Ferrario; Loic Guillevin; Eduardo Mirapeix; Caroline O S Savage; Renato A Sinico; Coen A Stegeman; Kerstin W Westman; Fokko J van der Woude; Robert A F de Lind van Wijngaarden; Charles D Pusey Journal: J Am Soc Nephrol Date: 2007-06-20 Impact factor: 10.121
Authors: Joanna C Robson; Jill Dawson; Helen Doll; Peter F Cronholm; Nataliya Milman; Katherine Kellom; Susan Ashdown; Ebony Easley; Don Gebhart; Georgia Lanier; John Mills; Jacqueline Peck; Raashid Ahmed Luqmani; Judy Shea; Gunnar Tomasson; Peter A Merkel Journal: Ann Rheum Dis Date: 2018-04-25 Impact factor: 19.103