Zachary S Wallace1, Arezou Khosroshahi2, Mollie D Carruthers3, Cory A Perugino1, Hyon Choi1, Corrado Campochiaro4, Emma L Culver5, Frank Cortazar1, Emanuel Della-Torre4, Mikael Ebbo6, Ana Fernandes1, Luca Frulloni4, Phil A Hart7, Omer Karadag8, Shigeyuki Kawa9, Mitsuhiro Kawano10, Myung-Hwan Kim11, Marco Lanzillotta12, Shoko Matsui13, Kazuichi Okazaki14, Jay H Ryu15, Takako Saeki16, Nicolas Schleinitz17, Paula Tanasa2, Hisanori Umehara18, George Webster19, Wen Zhang20, John H Stone1. 1. Massachusetts General Hospital and Harvard Medical School, Boston, Massachusetts. 2. Emory University School of Medicine, Atlanta, Georgia. 3. University of British Columbia, Vancouver, British Columbia, Canada. 4. IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Milan, Italy. 5. John Radcliffe Hospital, Oxford, UK. 6. La Timone University Hospital, Marseille. 7. University of Verona, Verona, Italy. 8. Ohio State University Wexner Medical Center, Columbus. 9. Hacettepe University Faculty of Medicine, Ankara, Turkey. 10. Shinshu University, Matsumoto, Japan. 11. Kanazawa University Graduate School of Medicine, Kanazawa, Japan. 12. University of Ulsan College of Medicine, Asan Medical Center, Seoul, South Korea. 13. University of Toyama, Toyama, Japan. 14. Kansai Medical University, Osaka, Japan. 15. Mayo Clinic, Rochester, Minnesota. 16. Nagaoka Red Cross Hospital, Nagaoka, Japan. 17. Aix-Marseille Université, Assistance Publique Hôpitaux de Marseille, Marseille, France. 18. Hayashi General Hospital, Fukui, Japan. 19. University College London Hospitals, London, UK. 20. Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, China.
Abstract
OBJECTIVE: IgG4-related disease (IgG4-RD) can cause fibroinflammatory lesions in nearly any organ, leading to organ dysfunction and failure. The IgG4-RD Responder Index (RI) was developed to help investigators assess the efficacy of treatment in a structured manner. The aim of this study was to validate the RI in a multinational investigation. METHODS: The RI guides investigators through assessments of disease activity and damage in 25 domains, incorporating higher weights for disease manifestations that require urgent treatment or that worsen despite treatment. After a training exercise, investigators reviewed 12 written IgG4-RD vignettes based on real patients. Investigators calculated both an RI score as well as a physician's global assessment (PhGA) score for each vignette. In a longitudinal assessment, 3 investigators used the RI in 15 patients with newly active disease who were followed up over serial visits after treatment. We assessed interrater and intrarater reliability, precision, validity, and responsiveness. RESULTS: The 26 physician investigators included representatives from 6 specialties and 9 countries. The interrater and intrarater reliability of the RI was strong (0.89 and 0.69, respectively). Correlations (construct validity) between the RI and PhGA were high (Spearman's r = 0.9, P < 0.0001). The RI was sensitive to change (discriminant validity). Following treatment, there was significant improvement in the RI score (mean change 10.5 [95% confidence interval (95% CI) 5.4-12], P < 0.001), which correlated with the change in the PhGA. Urgent disease and damage were captured effectively. DISCUSSION: In this international, multispecialty study, we observed that the RI is a valid and reliable disease activity assessment tool that can be used to measure response to therapy.
OBJECTIVE: IgG4-related disease (IgG4-RD) can cause fibroinflammatory lesions in nearly any organ, leading to organ dysfunction and failure. The IgG4-RD Responder Index (RI) was developed to help investigators assess the efficacy of treatment in a structured manner. The aim of this study was to validate the RI in a multinational investigation. METHODS: The RI guides investigators through assessments of disease activity and damage in 25 domains, incorporating higher weights for disease manifestations that require urgent treatment or that worsen despite treatment. After a training exercise, investigators reviewed 12 written IgG4-RD vignettes based on real patients. Investigators calculated both an RI score as well as a physician's global assessment (PhGA) score for each vignette. In a longitudinal assessment, 3 investigators used the RI in 15 patients with newly active disease who were followed up over serial visits after treatment. We assessed interrater and intrarater reliability, precision, validity, and responsiveness. RESULTS: The 26 physician investigators included representatives from 6 specialties and 9 countries. The interrater and intrarater reliability of the RI was strong (0.89 and 0.69, respectively). Correlations (construct validity) between the RI and PhGA were high (Spearman's r = 0.9, P < 0.0001). The RI was sensitive to change (discriminant validity). Following treatment, there was significant improvement in the RI score (mean change 10.5 [95% confidence interval (95% CI) 5.4-12], P < 0.001), which correlated with the change in the PhGA. Urgent disease and damage were captured effectively. DISCUSSION: In this international, multispecialty study, we observed that the RI is a valid and reliable disease activity assessment tool that can be used to measure response to therapy.
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
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Authors: J-Matthias Löhr; Ulrich Beuers; Miroslav Vujasinovic; Domenico Alvaro; Jens Brøndum Frøkjær; Frank Buttgereit; Gabriele Capurso; Emma L Culver; Enrique de-Madaria; Emanuel Della-Torre; Sönke Detlefsen; Enrique Dominguez-Muñoz; Piotr Czubkowski; Nils Ewald; Luca Frulloni; Natalya Gubergrits; Deniz Guney Duman; Thilo Hackert; Julio Iglesias-Garcia; Nikolaos Kartalis; Andrea Laghi; Frank Lammert; Fredrik Lindgren; Alexey Okhlobystin; Grzegorz Oracz; Andrea Parniczky; Raffaella Maria Pozzi Mucelli; Vinciane Rebours; Jonas Rosendahl; Nicolas Schleinitz; Alexander Schneider; Eric Fh van Bommel; Caroline Sophie Verbeke; Marie Pierre Vullierme; Heiko Witt Journal: United European Gastroenterol J Date: 2020-06-18 Impact factor: 4.623
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