title: A preliminary score for the assessment of disease activity in hereditary recurrent fevers: results from the AIDAI (Auto-Inflammatory Diseases Activity Index) Consensus Conference.
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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Department of Pediatrics and Pediatric Rheumatology, National Reference Center for Auto-Inflammatory Disorders, Bicêtre University Hospital, Paris XI University, Le Kremlin-Bicêtre, France.
authors.forename: Maryam
authors.initials: M
authors.lastname: Piram
authors.forename: Joost
authors.initials: J
authors.lastname: Frenkel
authors.forename: Marco
authors.initials: M
authors.lastname: Gattorno
authors.forename: Seza
authors.initials: S
authors.lastname: Ozen
authors.forename: Helen J
authors.initials: HJ
authors.lastname: Lachmann
authors.forename: Raphaela
authors.initials: R
authors.lastname: Goldbach-Mansky
authors.forename: Véronique
authors.initials: V
authors.lastname: Hentgen
authors.forename: Bénédicte
authors.initials: B
authors.lastname: Neven
authors.forename: Katia Stankovic
authors.initials: KS
authors.lastname: Stojanovic
authors.forename: Anna
authors.initials: A
authors.lastname: Simon
authors.forename: Jasmin
authors.initials: J
authors.lastname: Kuemmerle-Deschner
authors.forename: Hal
authors.initials: H
authors.lastname: Hoffman
authors.forename: Silvia
authors.initials: S
authors.lastname: Stojanov
authors.forename: Agnès
authors.initials: A
authors.lastname: Duquesne
authors.forename: Pascal
authors.initials: P
authors.lastname: Pillet
authors.forename: Alberto
authors.initials: A
authors.lastname: Martini
authors.forename: Jacques
authors.initials: J
authors.lastname: Pouchot
authors.forename: Isabelle
authors.initials: I
authors.lastname: Koné-Paut
mesh_terms: D056587:Cryopyrin-Associated Periodic Syndromes / Q000175:diagnosis; D003697:Delphi Technique; D010505:Familial Mediterranean Fever / Q000175:diagnosis; D005334:Fever; D056660:Hereditary Autoinflammatory Diseases / Q000175:diagnosis*; D006801:Humans; D008499:Medical Records; D054078:Mevalonate Kinase Deficiency / Q000175:diagnosis; D012720:Severity of Illness Index