In silico validation of the Autoinflammatory Disease Damage Index
Por:
Ter Haar NM, van Delft ALJ, Annink KV, van Stel H, Al-Mayouf SM, Amaryan G, Anton-Lopez J, Barron KS, Benseler S, Brogan PA, Cantarini L, Cattalini M, Cochino AV, de Benedetti F, Dedeoglu F, de Jesus AA, Demirkaya E, Dolezalova P, Durrant KL, Fabio G, Gallizzi R, Goldbach-Mansky R, Hachulla E, Hentgen V, Herlin T, Hofer M, Hoffman HM, Insalaco A, Jansson AF, Kallinich T, Kone-Paut I, Kozlova A, Kuemmerle-Deschner JB, Lachmann HJ, Laxer RM, Martini A, Nielsen S, Nikishina I, Ombrello AK, Özen S, Papadopoulou-Alataki E, Quartier P, Rigante D, Russo R, Simon A, Trachana M, Uziel Y, Ravelli A, Schulert G, Gattorno M and Frenkel J
Publicada:
1 nov 2018
Ahead of Print:
4 ago 2018
Resumen:
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.
Filiaciones:
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-Lopez 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, Charité 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, Charité 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
Green Submitted, Green Accepted, Bronze
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