RT Journal Article
SR Electronic
T1 A preliminary score for the assessment of disease activity in hereditary recurrent fevers: results from the AIDAI (Auto-Inflammatory Diseases Activity Index) Consensus Conference
JF Annals of the Rheumatic Diseases
JO Ann Rheum Dis
FD BMJ Publishing Group Ltd and European League Against Rheumatism
SP 309
OP 314
DO 10.1136/ard.2010.132613
VO 70
IS 2
A1 Maryam Piram
A1 Joost Frenkel
A1 Marco Gattorno
A1 Seza Ozen
A1 Helen J Lachmann
A1 Raphaela Goldbach-Mansky
A1 Véronique Hentgen
A1 Bénédicte Neven
A1 Katia Stankovic Stojanovic
A1 Anna Simon
A1 Jasmin Kuemmerle-Deschner
A1 Hal Hoffman
A1 Silvia Stojanov
A1 Agnès Duquesne
A1 Pascal Pillet
A1 Alberto Martini
A1 Jacques Pouchot
A1 Isabelle Koné-Paut
YR 2011
UL http://ard.bmj.com/content/70/2/309.abstract
AB Background The systemic autoinflammatory disorders (SAID) share many clinical manifestations, albeit with variable patterns, intensity and frequency. A common definition of disease activity would be rational and useful in the management of these lifelong diseases. Moreover, standardised disease activity scores are required for the assessment of new therapies in constant development. The aim of this study was to develop preliminary activity scores for familial Mediterranean fever, mevalonate kinase deficiency, tumour necrosis factor receptor-1-associated periodic syndrome and cryopyrin-associated periodic syndromes (CAPS). Methods The study was conducted using two well-recognised consensus formation methods: the Delphi technique and the nominal group technique. The results from a two-step survey and data from parent/patient interviews were used as preliminary data to develop the agenda for a consensus conference to build a provisional scoring system. Results 24 of 65 experts in SAID from 20 countries answered the web questionnaire and 16 attended the consensus conference. There was consensus agreement to develop separate activity scores for each disease but with a common format based on patient diaries. Fever and disease-specific clinical variables were scored according to their severity. A final score was generated by summing the score of all the variables divided by the number of days over which the diary was completed. Scores varied from 0 to 16 (0–13 in CAPS). These scores were developed for the purpose of clinical studies but could be used in clinical practice. Conclusion Using widely recognised consensus formation techniques, preliminary scores were obtained to measure disease activity in four main SAID. Further prospective validation study of this instrument will follow.