title: Treatment of autoinflammatory diseases: results from the Eurofever Registry and a literature review.
abstract: OBJECTIVE
  To evaluate the response to treatment of autoinflammatory diseases from an international registry and an up-to-date literature review.
  
  
  METHODS
  The response to treatment was studied in a web-based registry in which clinical information on anonymised patients with autoinflammatory diseases was collected retrospectively as part of the Eurofever initiative. Participating hospitals included paediatric rheumatology centres of the Paediatric Rheumatology International Trial Organisation network and adult centres with a specific interest in autoinflammatory diseases. The following diseases were included: familial Mediterranean fever (FMF), cryopyrin-associated periodic syndromes (CAPS), tumour necrosis factor (TNF)-receptor associated periodic syndrome (TRAPS), mevalonate kinase deficiency (MKD), pyogenic arthritis pustulosis acne (PAPA) syndrome, deficiency of interleukin-1 receptor antagonist (DIRA), NLRP12-related periodic fever and periodic fever aphthosis pharyngitis adenitis (PFAPA) syndrome. Cases were independently validated by experts for each disease. A literature search regarding treatment of the abovementioned diseases was also performed using Medline and Embase.
  
  
  RESULTS
  22 months from the beginning of the enrolment, complete information on 496 validated patients was available. Data from the registry in combination with evidence from the literature confirmed that colchicine is the treatment of choice for FMF and IL-1 blockade for DIRA and CAPS. Corticosteroids on demand probably represent a valid therapeutic strategy for PFAPA, but also for MKD and TRAPS. Patients with poorly controlled MKD, TRAPS, PAPA or FMF may benefit from IL-1 blockade; anti-TNF treatment may represent a possible valuable alternative.
  
  
  CONCLUSIONS
  In the absence of high-grade evidence, these results could serve as a basis for therapeutic guidelines and to identify candidate drugs for future therapeutic trials.
authors: Department of Paediatrics, University Medical Center Utrecht, Utrecht, The Netherlands.
authors: Nienke
authors: N
authors: Ter Haar
authors: Helen
authors: H
authors: Lachmann
authors: Seza
authors: S
authors: Özen
authors: Pat
authors: P
authors: Woo
authors: Yosef
authors: Y
authors: Uziel
authors: Consuelo
authors: C
authors: Modesto
authors: Isabelle
authors: I
authors: Koné-Paut
authors: Luca
authors: L
authors: Cantarini
authors: Antonella
authors: A
authors: Insalaco
authors: Bénédicte
authors: B
authors: Neven
authors: Michael
authors: M
authors: Hofer
authors: Donato
authors: D
authors: Rigante
authors: Sulaiman
authors: S
authors: Al-Mayouf
authors: Isabelle
authors: I
authors: Touitou
authors: Romina
authors: R
authors: Gallizzi
authors: Efimia
authors: E
authors: Papadopoulou-Alataki
authors: Silvana
authors: S
authors: Martino
authors: Jasmin
authors: J
authors: Kuemmerle-Deschner
authors: Laura
authors: L
authors: Obici
authors: Nicolae
authors: N
authors: Iagaru
authors: Anna
authors: A
authors: Simon
authors: Susan
authors: S
authors: Nielsen
authors: Alberto
authors: A
authors: Martini
authors: Nicolino
authors: N
authors: Ruperto
authors: Marco
authors: M
authors: Gattorno
authors: Joost
authors: J
authors: Frenkel
mesh_terms: D000152:Acne Vulgaris / Q000453:epidemiology / Q000628:therapy*; D001170:Arthritis, Infectious / Q000453:epidemiology / Q000628:therapy*; D056587:Cryopyrin-Associated Periodic Syndromes / Q000453:epidemiology / Q000628:therapy*; D005060:Europe / Q000453:epidemiology; D010505:Familial Mediterranean Fever / Q000453:epidemiology / Q000628:therapy*; D006801:Humans; D054078:Mevalonate Kinase Deficiency / Q000453:epidemiology / Q000628:therapy*; D017511:Pyoderma Gangrenosum / Q000453:epidemiology / Q000628:therapy*; D012042:Registries / Q000706:statistics & numerical data*