In silico validation of the Autoinflammatory Disease Damage Index.

Autor:	Ter Haar NM; Laboratory for Translational Immunology, University Medical Centre Utrecht, Utrecht, The Netherlands.; Department of Paediatric Immunology and Rheumatology, University Medical Centre Utrecht, Utrecht, The Netherlands., van Delft ALJ; Department of Paediatrics, Universitair Medisch Centrum Utrecht-Locatie Wilhelmina Kinderziekenhuis, Utrecht, The Netherlands., Annink KV; Department of Paediatrics, Universitair Medisch Centrum Utrecht-Locatie Wilhelmina Kinderziekenhuis, Utrecht, The Netherlands., van Stel H; Department of Paediatrics, Universitair Medisch Centrum Utrecht-Locatie Wilhelmina Kinderziekenhuis, Utrecht, The Netherlands., Al-Mayouf SM; Department of Pediatrics, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia., Amaryan G; National Paediatric Centre for Familial Mediterranean Fever and Gastroenterology Service, Arabkir Medical Centre-Institute of Child and Adolescent Health, Yerevan, Armenia., Anton J; Paediatric Rheumatology Unit, Hospital Sant Joan de Déu, Barcelona, Spain., Barron KS; Division of Intramural Research and National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA., Benseler S; Departments of Paediatrics and Rheumatology, Alberta Children's Hospital, Calgary, Alberta, Canada., Brogan PA; Department of Infection, Inflammation and Rheumatology, University College London Institute of Child Health, London, UK., Cantarini L; Rheumatology Unit, Department of Medical Sciences, Surgery and Neurosciences, University of Siena, Siena, Italy., Cattalini M; Paediatric Clinic, University of Brescia and Spedali Civili di Brescia, Brescia, Italy., Cochino AV; Department of Paediatrics, National Institute for Mother and Child Health Alessandrescu-Rusescu, Bucharest, Romania., de Benedetti F; Division of Rheumatology, Ospedale Pediatrico Bambino Gesù, Rome, Italy., Dedeoglu F; Rheumatology Program, Division of Immunology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA., de Jesus AA; Translational Autoinflammatory Disease Section, NIAMS/NIH, Bethesda, Maryland, USA., Demirkaya E; Western University Children's Hospital, London Health Sciences Centre, London, UK., Dolezalova P; Department of Paediatrics and Adolescent Medicine, Charles University, General University Hospital, Praha, Czech Republic., Durrant KL; Autoinflammatory Alliance, San Francisco, California, USA., Fabio G; Department of Internal Medicine, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy., Gallizzi R; Department of Paediatric Rheumatology, AOUG Martino, Messina, Italy., Goldbach-Mansky R; Translational Autoinflammatory Disease Section, NIAMS/NIH, Bethesda, Maryland, USA., Hachulla E; Département de Médecine Interne et Immunologie Clinique, Université de Lille, Lille, France., Hentgen V; Reference Centre for Autoinflammatory Diseases (CEREMAI), Versailles Hospital, Le Chesnay, France., Herlin T; Departmentof Paediatrics, Aarhus University Hospital, Aarhus, Denmark., Hofer M; Department of Paediatric Rheumatology, University of Lausanne, Lausanne, Switzerland.; Departmentof Paediatric Rheumatology, University Hospital of Geneva, Geneva, Switzerland., Hoffman HM; Department of Paediatrics, University of California, San Diego, California, USA., Insalaco A; Dipartimento di Medicina Pediatrica, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy., Jansson AF; Paediatric Pneumology and Immunology and Interdisciplinary Centre for Social Paediatrics, Charite University Medicine Berlin, Berlin, Germany.; Department of Rheumatology and Immunology, Dr von Hauner Children's Hospital, Ludwig-Maximilians-University, Munich, Germany., Kallinich T; Paediatric Pneumology and Immunology and Interdisciplinary Centre for Social Paediatrics, Charite University Medicine Berlin, Berlin, Germany., Kone-Paut I; Department of Paediatric Rheumatology and CEREMAI, Bicêtre Hospital, APHP, University of Paris Sud, Paris, France., Kozlova A; Department of Immunology, Federal Research and Clinical Centre for Paediatric Haematology, Oncology and Immunology, Moscow, Russian Federation., Kuemmerle-Deschner JB; Division of Pediatric Rheumatology, Department of Pediatrics, University Hospital Tübingen, Tübingen, Germany., Lachmann HJ; Division of Medicine, University College London, London, UK., Laxer RM; Department of Paediatrics and Medicine, University of Toronto and the Hospital for Sick Children, Toronto, Ontario, Canada., Martini A; Direzione Scientifica, Istituto Giannina Gaslini, Genova, Liguria, Italy., Nielsen S; Paediatric Rheumatology Unit 4272, Rigshospitalet, Copenhagen, Denmark., Nikishina I; Department of Paediatric Rheumatic Diseases, VA Nasonova Research Institute of Rheumatology, Moscow, Russian Federation., Ombrello AK; Inflammatory Disease Section, National Human Genome Research Institute, Bethesda, Maryland, USA., Özen S; Pediatric Rheumatology, Hacettepe University, Ankara, Turkey., Papadopoulou-Alataki E; Fourth Department of Pediatrics, Aristotle University of Thessaloniki, Thessaloniki, Greece., Quartier P; Department of Paediatric Immunology-Hematology and Rheumatology Unit and IMAGINE Institute, Institution Necker-Enfants Malades Hospital and Paris Descartes University, Paris, Île-de-France, France., Rigante D; Institute of Paediatrics, Fondazione Policlinico Universitario A Gemelli, Università Cattolica Sacro Cuore, Rome, Italy., Russo R; Servicio de Inmunología/Reumatología, Hospital de Pediatria Juan P Garrahan, Buenos Aires, Argentina., Simon A; Department of General Internal Medicine, Radboud Expertise Centre for Immunodeficiency and Autoinflammation, Radboud University Medical Centre, Nijmegen, The Netherlands., Trachana M; Paediatric Immunology and Rheumatology Referral Centre, First Paediatric Clinic, Aristotle University of Thessaloniki, Thessaloniki, Greece., Uziel Y; Department of Paediatrics, Meir Medical Centre, Kfar Saba, Israel., Ravelli A; Institution Università degli Studi di Genova and G Gaslini Institute, Genova, Italy., Schulert G; Division of Rheumatology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.; Department of Pediatrics, University of Cincinnati, Cincinnati, Ohio, USA., Gattorno M; Institution Università degli Studi di Genova and G Gaslini Institute, Genova, Italy., Frenkel J; Department of Paediatrics, Universitair Medisch Centrum Utrecht-Locatie Wilhelmina Kinderziekenhuis, Utrecht, The Netherlands.
Jazyk:	angličtina
Zdroj:	Annals of the rheumatic diseases [Ann Rheum Dis] 2018 Nov; Vol. 77 (11), pp. 1599-1605. Date of Electronic Publication: 2018 Aug 04.
DOI:	10.1136/annrheumdis-2018-213725
Abstrakt:	Introduction: Autoinflammatory diseases can cause irreversible tissue damage due to systemic inflammation. Recently, the Autoinflammatory Disease Damage Index (ADDI) was developed. The ADDI is the first instrument to quantify damage in familial Mediterranean fever, cryopyrin-associated periodic syndromes, mevalonate kinase deficiency and tumour necrosis factor receptor-associated periodic syndrome. The aim of this study was to validate this tool for its intended use in a clinical/research setting. Methods: The ADDI was scored on paper clinical cases by at least three physicians per case, independently of each other. Face and content validity were assessed by requesting comments on the ADDI. Reliability was tested by calculating the intraclass correlation coefficient (ICC) using an 'observer-nested-within-subject' design. Construct validity was determined by correlating the ADDI score to the Physician Global Assessment (PGA) of damage and disease activity. Redundancy of individual items was determined with Cronbach's alpha. Results: The ADDI was validated on a total of 110 paper clinical cases by 37 experts in autoinflammatory diseases. This yielded an ICC of 0.84 (95% CI 0.78 to 0.89). The ADDI score correlated strongly with PGA-damage (r=0.92, 95% CI 0.88 to 0.95) and was not strongly influenced by disease activity (r=0.395, 95% CI 0.21 to 0.55). After comments from disease experts, some item definitions were refined. The interitem correlation in all different categories was lower than 0.7, indicating that there was no redundancy between individual damage items. Conclusion: The ADDI is a reliable and valid instrument to quantify damage in individual patients and can be used to compare disease outcomes in clinical studies. Competing Interests: Competing interests: Novartis Pharma financially supported meetings with the methodologist. PAB: consultancy/speaking fees from Novartis, Roche, SOBI, UCB. FdB: Novartis, Novimmune, Hoffmann-La Roche, SOBI, AbbVie. LC: speaker’s fee from Novartis and SOBI. MC: consultancy fees for Novartis, SOBI and Abbvie. KLD: consultancy work for SOBI and Novartis; donations, honorariums and unrestricted grants have been received by the Autoinflammatory Alliance from SOBI, Novartis and Regeneron. RG: consultant for Abbvie. RGM: study support from SOBI, Novartis, Regeneron. VH: honorariums and educational grants from Novartis; honorariums from SOBI. MH: consultant for Novartis. HMH: consultant for Novartis and SOBI; speaker for Novartis. TK: research grant by Novartis; speaker’s bureau by Roche, BMS, Novartis and SOBI. JKD: consultant/speaker for Novartis and SOBI, and has received grant support from SOBI and Novartis. RML: ad board and consultant for Abbvie and Novartis. PQ: investigator, consultant and speaker’s bureau for Novartis and SOBI. MG: consultant for and unrestricted grants to Eurofever and speaker’s fee from SOBI and Novartis. YU: grant/research support from Novartis; consultant for Novartis; speaker’s bureau of Abbvie, Neopharm, Novartis, Roche. JF: consultant for Novartis. GS: consulting fees for Novartis. FD: attended to Novartis advisory board meeting. (© Author(s) (or their employer(s)) 2018. No commercial re-use. See rights and permissions. Published by BMJ.)
Databáze:	MEDLINE
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