Development of the autoinflammatory disease damage index (ADDI)
Por:
Ter Haar NM, Annink KV, Al-Mayouf SM, Amaryan G, Anton-Lopez J, Barron KS, Benseler SM, Brogan PA, Cantarini L, Cattalini M, Cochino AV, De Benedetti F, Dedeoglu F, De Jesus AA, Della Casa Alberighi O, 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, Koné-Paut I, Kozlova A, Kuemmerle-Deschner JB, Lachmann HJ, Laxer RM, Martini A, Nielsen S, Nikishina I, Ombrello AK, Ozen S, Papadopoulou-Alataki E, Quartier P, Rigante D, Russo R, Simon A, Trachana M, Uziel Y, Ravelli A, Gattorno M and Frenkel J
Publicada:
1 may 2017
Ahead of Print:
3 nov 2016
Resumen:
Objectives Autoinflammatory diseases cause systemic inflammation that can result in damage to multiple organs. A validated instrument is essential to quantify damage in individual patients and to compare disease outcomes in clinical studies. Currently, there is no such tool. Our objective was to develop a common autoinflammatory disease damage index (ADDI) for familial Mediterranean fever, cryopyrin-associated periodic syndromes, tumour necrosis factor receptor-associated periodic fever syndrome and mevalonate kinase deficiency.
Methods We developed the ADDI by consensus building. The top 40 enrollers of patients in the Eurofever Registry and 9 experts from the Americas participated in multiple rounds of online surveys to select items and definitions. Further, 22 (parents of) patients rated damage items and suggested new items. A consensus meeting was held to refine the items and definitions, which were then formally weighted in a scoring system derived using decision-making software, known as 1000minds.
Results More than 80% of the experts and patients completed the online surveys. The preliminary ADDI contains 18 items, categorised in the following eight organ systems: reproductive, renal/amyloidosis, developmental, serosal, neurological, ears, ocular and musculoskeletal damage. The categories renal/amyloidosis and neurological damage were assigned the highest number of points, serosal damage the lowest number of points. The involvement of (parents of) patients resulted in the inclusion of, for example, chronic musculoskeletal pain.
Conclusions An instrument to measure damage caused by autoinflammatory diseases is developed based on consensusbuilding. Patients fulfilled a significant role in this process.
Filiaciones:
Ter Haar NM:
Laboratory for Translational Immunology, University Medical Centre Utrecht, Utrecht, The Netherlands
Department of Paediatric Immunology, University Medical Centre Utrecht, Utrecht, The Netherlands
Annink KV:
Department of Paediatrics, University Medical Centre Utrecht, Utrecht, The Netherlands
Al-Mayouf SM:
Department of Paediatrics, King Faisal Specialist Hospital & Research Centre, Riyadh, Saudi Arabia
Amaryan G:
National Paediatric Centre for Familial Mediterranean Fever and Gastroenterology Service, Arabkir Medical Centre-Institute of Child & 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, USA
Benseler SM:
Department of Paediatrics and Department of Rheumatology, Alberta Children's Hospital, Calgary, Canada
Brogan PA:
Department of Infection, Inflammation and Rheumatology, University College London Institute of Child Health, London, UK
Cantarini L:
Department of Medical Sciences, Surgery and Neurosciences, Rheumatology Unit, University of Siena, Siena, Italy
Cattalini M:
Paediatric Clinic, University of Brescia and Spedali Civili di Brescia, Brescia, Italy
Cochino AV:
Paediatrics Department, 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:
Division of Immunology, Rheumatology Program, Boston Children's Hospital, Harvard Medical School, Boston, USA
De Jesus AA:
Translational Autoinflammatory Disease Section, NIAID, National Institutes of Health, Bethesda, USA
Della Casa Alberighi O:
UOSD Farmacologia Clinica e Clinical Trial-Scientific Direction, G. Gaslini Institute, Genova, Italy
Demirkaya E:
Department of Paediatric Rheumatology, Gulhane Military Medical Faculty, Ankara, Turkey
Dolezalova P:
Department of Paediatrics and Adolescent Medicine, Charles University, General University Hospital, Prague, Czech Republic
Durrant KL:
Autoinflammatory Alliance, San Fransisco, USA
Fabio G:
Department of Internal Medicine, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy
Gallizzi R:
Department of Paediatrics, Rheumatology, AOU G Martino, Messina, Italy
Goldbach-Mansky R:
Translational Autoinflammatory Disease Section, NIAID, National Institutes of Health, Bethesda, 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:
Department of Paediatrics, Aarhus University Hospital, Aarhus, Denmark
Hofer M:
Department of Paediatric Rheumatology, University of Lausanne, Lausanne, Switzerland
Department of Paediatric Rheumatology, University Hospital of Geneva, Geneva, Switzerland
Hoffman HM:
Department of Paediatrics, University of California, San Diego, USA
Insalaco A:
Dipartimento di Medicina Pediatrica, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy
Jansson AF:
Department of Rheumatology&Immunology, Dr. von Hauner Childrens Hospital, Ludwig-Maximilians-University, Munich, Germany
Kallinich T:
Paediatric Pneumology and Immunology and Interdisciplinary Centre for Social Paediatrics, Charité University Medicine Berlin, Berlin, Germany
Koné-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, Russia
Kuemmerle-Deschner JB:
Division of Paediatric Rheumatology, Department of Paediatrics, University Hospital Tuebingen, Tuebingen, 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, Canada
Martini A:
Direzione Scientifica, G Gaslini Institute, Genova, Italy
Nielsen S:
Paediatric Rheumatology unit 4272, Rigshospitalet, Copenhagen, Denmark
Nikishina I:
Department of Paediatric Rheumatic diseases, V.A. Nasonova Research Institute of Rheumatology, Moscow, Russia
Ombrello AK:
Inflammatory Disease Section, National Human Genome Research Institute, National Institutes of Health, Bethesda, USA
Ozen S:
Department of Paediatric Rheumatology, Hacettepe University, Ankara, Turkey
Papadopoulou-Alataki E:
Fourth Department of Paediatrics, 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, France
Rigante D:
Institute of Paediatrics, Fondazione Policlinico Universitario A. Gemelli, Università Cattolica Sacro Cuore, Rome, Italy
Russo R:
Servicio de Inmunología y Reumatología, Hospital de Pediatría Garrahan, Buenos Aires, Argentina
Simon A:
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, Tel Aviv University, Sackler School of Medicine, Tel Aviv, Israel
Ravelli A:
Institution Università degli Studi di Genova and G. Gaslini Institute, Genova, Italy
Gattorno M:
UOC Pediatria 2, G. Gaslini Institute, Genova, Italy
Frenkel J:
Department of Paediatrics, University Medical Centre Utrecht, Utrecht, The Netherlands
Green Submitted, Green Published
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