In silico validation of the Autoinflammatory Disease Damage Index.
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Ter Haar NM
Laboratory for Translational Immunology, University Medical Centre Utrecht, Utrecht, The Netherlands.
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van Delft ALJ
Department of Paediatrics, Universitair Medisch Centrum Utrecht-Locatie Wilhelmina Kinderziekenhuis, Utrecht, The Netherlands.
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Annink KV
Department of Paediatrics, Universitair Medisch Centrum Utrecht-Locatie Wilhelmina Kinderziekenhuis, Utrecht, The Netherlands.
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van Stel H
Department of Paediatrics, Universitair Medisch Centrum Utrecht-Locatie Wilhelmina Kinderziekenhuis, Utrecht, The Netherlands.
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Al-Mayouf SM
Department of Pediatrics, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia.
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Amaryan G
National Paediatric Centre for Familial Mediterranean Fever and Gastroenterology Service, Arabkir Medical Centre-Institute of Child and Adolescent Health, Yerevan, Armenia.
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Anton J
Paediatric Rheumatology Unit, Hospital Sant Joan de Déu, Barcelona, Spain.
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Barron KS
Division of Intramural Research and National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
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Benseler S
Departments of Paediatrics and Rheumatology, Alberta Children's Hospital, Calgary, Alberta, Canada.
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Brogan PA
Department of Infection, Inflammation and Rheumatology, University College London Institute of Child Health, London, UK.
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Cantarini L
Rheumatology Unit, Department of Medical Sciences, Surgery and Neurosciences, University of Siena, Siena, Italy.
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Cattalini M
Paediatric Clinic, University of Brescia and Spedali Civili di Brescia, Brescia, Italy.
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Cochino AV
Department of Paediatrics, National Institute for Mother and Child Health Alessandrescu-Rusescu, Bucharest, Romania.
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de Benedetti F
Division of Rheumatology, Ospedale Pediatrico Bambino Gesù, Rome, Italy.
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Dedeoglu F
Rheumatology Program, Division of Immunology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
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de Jesus AA
Translational Autoinflammatory Disease Section, NIAMS/NIH, Bethesda, Maryland, USA.
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Demirkaya E
Western University Children's Hospital, London Health Sciences Centre, London, UK.
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Dolezalova P
Department of Paediatrics and Adolescent Medicine, Charles University, General University Hospital, Praha, Czech Republic.
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Durrant KL
Autoinflammatory Alliance, San Francisco, California, USA.
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Fabio G
Department of Internal Medicine, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy.
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Gallizzi R
Department of Paediatric Rheumatology, AOUG Martino, Messina, Italy.
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Goldbach-Mansky R
Translational Autoinflammatory Disease Section, NIAMS/NIH, Bethesda, Maryland, USA.
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Hachulla E
Département de Médecine Interne et Immunologie Clinique, Université de Lille, Lille, France.
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Hentgen V
Reference Centre for Autoinflammatory Diseases (CEREMAI), Versailles Hospital, Le Chesnay, France.
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Herlin T
Departmentof Paediatrics, Aarhus University Hospital, Aarhus, Denmark.
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Hofer M
Department of Paediatric Rheumatology, University of Lausanne, Lausanne, Switzerland.
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Hoffman HM
Department of Paediatrics, University of California, San Diego, California, USA.
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Insalaco A
Dipartimento di Medicina Pediatrica, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy.
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Jansson AF
Paediatric Pneumology and Immunology and Interdisciplinary Centre for Social Paediatrics, Charite University Medicine Berlin, Berlin, Germany.
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Kallinich T
Paediatric Pneumology and Immunology and Interdisciplinary Centre for Social Paediatrics, Charite University Medicine Berlin, Berlin, Germany.
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Kone-Paut I
Department of Paediatric Rheumatology and CEREMAI, Bicêtre Hospital, APHP, University of Paris Sud, Paris, France.
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Kozlova A
Department of Immunology, Federal Research and Clinical Centre for Paediatric Haematology, Oncology and Immunology, Moscow, Russian Federation.
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Kuemmerle-Deschner JB
Division of Pediatric Rheumatology, Department of Pediatrics, University Hospital Tübingen, Tübingen, Germany.
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Lachmann HJ
Division of Medicine, University College London, London, UK.
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Laxer RM
Department of Paediatrics and Medicine, University of Toronto and the Hospital for Sick Children, Toronto, Ontario, Canada.
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Martini A
Direzione Scientifica, Istituto Giannina Gaslini, Genova, Liguria, Italy.
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Nielsen S
Paediatric Rheumatology Unit 4272, Rigshospitalet, Copenhagen, Denmark.
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Nikishina I
Department of Paediatric Rheumatic Diseases, VA Nasonova Research Institute of Rheumatology, Moscow, Russian Federation.
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Ombrello AK
Inflammatory Disease Section, National Human Genome Research Institute, Bethesda, Maryland, USA.
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Özen S
Pediatric Rheumatology, Hacettepe University, Ankara, Turkey.
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Papadopoulou-Alataki E
Fourth Department of Pediatrics, Aristotle University of Thessaloniki, Thessaloniki, Greece.
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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.
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Rigante D
Institute of Paediatrics, Fondazione Policlinico Universitario A Gemelli, Università Cattolica Sacro Cuore, Rome, Italy.
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Russo R
Servicio de Inmunología/Reumatología, Hospital de Pediatria Juan P Garrahan, Buenos Aires, Argentina.
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Simon A
Department of General Internal Medicine, Radboud Expertise Centre for Immunodeficiency and Autoinflammation, Radboud University Medical Centre, Nijmegen, The Netherlands.
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Trachana M
Paediatric Immunology and Rheumatology Referral Centre, First Paediatric Clinic, Aristotle University of Thessaloniki, Thessaloniki, Greece.
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Uziel Y
Department of Paediatrics, Meir Medical Centre, Kfar Saba, Israel.
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Ravelli A
Institution Università degli Studi di Genova and G Gaslini Institute, Genova, Italy.
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Schulert G
Division of Rheumatology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
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Gattorno M
Institution Università degli Studi di Genova and G Gaslini Institute, Genova, Italy.
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Frenkel J
Department of Paediatrics, Universitair Medisch Centrum Utrecht-Locatie Wilhelmina Kinderziekenhuis, Utrecht, The Netherlands.
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Published in:
- Annals of the rheumatic diseases. - 2018
English
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.
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green
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https://sonar.ch/global/documents/106476
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