Development of the autoinflammatory disease damage index (ADDI).
Ter Haar NMLaboratory for Translational Immunology, University Medical Centre Utrecht, Utrecht, The Netherlands.
Annink KVDepartment of Paediatrics, University Medical Centre Utrecht, Utrecht, The Netherlands.
Al-Mayouf SMDepartment of Paediatrics, King Faisal Specialist Hospital & Research Centre, Riyadh, Saudi Arabia.
Amaryan GNational Paediatric Centre for Familial Mediterranean Fever and Gastroenterology Service, Arabkir Medical Centre-Institute of Child & Adolescent Health, Yerevan, Armenia.
Anton JPaediatric Rheumatology Unit, Hospital Sant Joan de Déu, Barcelona, Spain.
Barron KSDivision of Intramural Research and National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, USA.
Benseler SMDepartment of Paediatrics and Department of Rheumatology, Alberta Children's Hospital, Calgary, Canada.
Brogan PADepartment of Infection, Inflammation and Rheumatology, University College London Institute of Child Health, London, UK.
Cantarini LDepartment of Medical Sciences, Surgery and Neurosciences, Rheumatology Unit, University of Siena, Siena, Italy.
Cattalini MPaediatric Clinic, University of Brescia and Spedali Civili di Brescia, Brescia, Italy.
Cochino AVPaediatrics Department, National Institute for Mother and Child Health Alessandrescu-Rusescu, Bucharest, Romania.
De Benedetti FDivision of Rheumatology, Ospedale Pediatrico Bambino Gesù, Rome, Italy.
Dedeoglu FDivision of Immunology, Rheumatology Program, Boston Children's Hospital, Harvard Medical School, Boston, USA.
De Jesus AATranslational Autoinflammatory Disease Section, NIAID, National Institutes of Health, Bethesda, USA.
Della Casa Alberighi OUOSD Farmacologia Clinica e Clinical Trial-Scientific Direction, G. Gaslini Institute, Genova, Italy.
Demirkaya EDepartment of Paediatric Rheumatology, Gulhane Military Medical Faculty, Ankara, Turkey.
Dolezalova PDepartment of Paediatrics and Adolescent Medicine, Charles University, General University Hospital, Prague, Czech Republic.
Durrant KLAutoinflammatory Alliance, San Fransisco, USA.
Fabio GDepartment of Internal Medicine, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy.
Gallizzi RDepartment of Paediatrics, Rheumatology, AOU G Martino, Messina, Italy.
Goldbach-Mansky RTranslational Autoinflammatory Disease Section, NIAID, National Institutes of Health, Bethesda, USA.
Hachulla EDépartement de Médecine Interne et Immunologie Clinique, Université de Lille, Lille, France.
Hentgen VReference centre for autoinflammatory diseases (CEREMAI), Versailles Hospital, Le Chesnay, France.
Herlin TDepartment of Paediatrics, Aarhus University Hospital, Aarhus, Denmark.
Hofer MDepartment of Paediatric Rheumatology, University of Lausanne, Lausanne, Switzerland.
Hoffman HMDepartment of Paediatrics, University of California, San Diego, USA.
Jansson AFDepartment of Rheumatology&Immunology, Dr. von Hauner Childrens Hospital, Ludwig-Maximilians-University, Munich, Germany.
Kallinich TPaediatric Pneumology and Immunology and Interdisciplinary Centre for Social Paediatrics, Charité University Medicine Berlin, Berlin, Germany.
Koné-Paut IDepartment of Paediatric Rheumatology and CEREMAI, Bicêtre Hospital, APHP, University of Paris Sud, Paris, France.
Kozlova ADepartment of Immunology, Federal Research and Clinical Centre for Paediatric Haematology, Oncology and Immunology, Moscow, Russia.
Kuemmerle-Deschner JBDivision of Paediatric Rheumatology, Department of Paediatrics, University Hospital Tuebingen, Tuebingen, Germany.
Lachmann HJDivision of Medicine, University College London, London, UK.
Laxer RMDepartment of Paediatrics and Medicine, University of Toronto and the Hospital for Sick Children, Toronto, Canada.
Martini ADirezione Scientifica, G Gaslini Institute, Genova, Italy.
Nielsen SPaediatric Rheumatology unit 4272, Rigshospitalet, Copenhagen, Denmark.
Nikishina IDepartment of Paediatric Rheumatic diseases, V.A. Nasonova Research Institute of Rheumatology, Moscow, Russia.
Ombrello AKInflammatory Disease Section, National Human Genome Research Institute, National Institutes of Health, Bethesda, USA.
Ozen SDepartment of Paediatric Rheumatology, Hacettepe University, Ankara, Turkey.
Papadopoulou-Alataki EFourth Department of Paediatrics, Aristotle University of Thessaloniki, Thessaloniki, Greece.
Quartier PDepartment of Paediatric Immunology-Hematology and Rheumatology Unit and IMAGINE Institute, Institution Necker-Enfants Malades Hospital and Paris-Descartes University, Paris, France.
Rigante DInstitute of Paediatrics, Fondazione Policlinico Universitario A. Gemelli, Università Cattolica Sacro Cuore, Rome, Italy.
Russo RServicio de Inmunología y Reumatología, Hospital de Pediatría Garrahan, Buenos Aires, Argentina.
Simon AInternal Medicine, Radboud Expertise Centre for Immunodeficiency and Autoinflammation, Radboud University Medical Centre, Nijmegen, The Netherlands.
Trachana MPaediatric Immunology and Rheumatology Referral Centre, first Paediatric clinic, Aristotle University of Thessaloniki, Thessaloniki, Greece.
Uziel YDepartment of Paediatrics, Meir Medical Centre, Kfar Saba, Tel Aviv University, Sackler School of Medicine, Tel Aviv, Israel.
Ravelli AInstitution Università degli Studi di Genova and G. Gaslini Institute, Genova, Italy.
Gattorno MUOC Pediatria 2, G. Gaslini Institute, Genova, Italy.
Frenkel JDepartment of Paediatrics, University Medical Centre Utrecht, Utrecht, The Netherlands.
English
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 consensus building. Patients fulfilled a significant role in this process.