A preliminary score for the assessment of disease activity in hereditary recurrent fevers: Results from the AIDAI (Auto-Inflammatory Diseases Activity Index) consensus conference

Maryam Piram, Joost Frenkel, Marco Gattorno, Seza Ozen, Helen J. Lachmann, Raphaela Goldbach-Mansky, Véronique Hentgen, Bénédicte Neven, Katia Stankovic Stojanovic, Anna Simon, Jasmin Kuemmerle-Deschner, Hal Hoffman, Silvia Stojanov, Agnès Duquesne, Pascal Pillet, Alberto Martini, Jacques Pouchot, Isabelle Koné-Paut

Research output: Contribution to journalArticlepeer-review

Abstract

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.

Original languageEnglish
Pages (from-to)309-314
Number of pages6
JournalAnnals of the Rheumatic Diseases
Volume70
Issue number2
DOIs
Publication statusPublished - Feb 2011

ASJC Scopus subject areas

  • Rheumatology
  • Immunology
  • Biochemistry, Genetics and Molecular Biology(all)
  • Immunology and Allergy

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