Paediatrics Publications

Title

In silico validation of the autoinflammatory disease damage index

Authors

Nienke M. Ter Haar, University Medical Center Utrecht
Amber Laetitia Justine Van Delft, Wilhelmina Kinderziekenhuis
Kim Valerie Annink, Wilhelmina Kinderziekenhuis
Henk Van Stel, Wilhelmina Kinderziekenhuis
Sulaiman M. Al-Mayouf, King Faisal Specialist Hospital and Research Centre
Gayane Amaryan, National Paediatric Centre for Familial Mediterranean Fever and Gastroenterology Service
Jordi Anton, Hospital Sant Joan de Déu Barcelona
Karyl S. Barron, National Institute of Allergy and Infectious Diseases (NIAID)
Susanne Benseler, Alberta Children's Hospital
Paul A. Brogan, UCL Great Ormond Street Institute of Child Health
Luca Cantarini, Università degli Studi di Siena
Marco Cattalini, Spedali Civili Di Brescia
Alexis Virgil Cochino, Alessandrescu-Rusescu National Institute of Mother and Child Health
Fabrizio De Benedetti, IRCCS Ospedale Pediatrico Bambino Gesù
Fatma Dedeoglu, Children's Hospital Boston
Adriana Almeida De Jesus, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Erkan Demirkaya, Western UniversityFollow
Pavla Dolezalova, Všeobecná Fakultní Nemocnice v Praze
Karen L. Durrant, Autoinflammatory Alliance
Giovanna Fabio, Ospedale Maggiore Policlinico Milano
Romina Gallizzi
Raphaela Goldbach-Mansky, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Eric Hachulla, Université de Lille
Veronique Hentgen, CHV Centre Hospitalier de Versailles
Troels Herlin, Aarhus Universitetshospital
Michaël Hofer, Université de Lausanne (UNIL)
Hal M. Hoffman, University of California, San Diego
Antonella Insalaco, IRCCS Ospedale Pediatrico Bambino Gesù
Annette F. Jansson, Charité – Universitätsmedizin Berlin

Document Type

Article

Publication Date

11-1-2018

Journal

Annals of the Rheumatic Diseases

Volume

77

Issue

11

First Page

1599

Last Page

1605

URL with Digital Object Identifier

10.1136/annrheumdis-2018-213725

Abstract

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 â 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.

This document is currently not available here.

Share

COinS