title: Efficacy and safety of the interleukin-1 antagonist rilonacept in Schnitzler syndrome: an open-label study.
abstract: BACKGROUND
  Schnitzler syndrome (SchS) is a rare disease with suspected autoinflammatory background that shares several clinical symptoms, including urticarial rash, fever episodes, arthralgia, and bone and muscle pain with cryopyrin-associated periodic syndromes (CAPS). Cryopyrin-associated periodic syndromes respond to treatment with interleukin-1 antagonists, and single case reports of Schnitzler syndrome have shown improvement following treatment with the interleukin-1 blocker anakinra. This study evaluated the effects of the interleukin-1 antagonist rilonacept on the clinical signs and symptoms of SchS.
  
  
  METHODS
  Eight patients with SchS were included in this prospective, single-center, open-label study. After a 3-week baseline, patients received a subcutaneous loading dose of rilonacept 320 mg followed by weekly subcutaneous doses of 160 mg for up to 1 year. Efficacy was determined by patient-based daily health assessment forms, physician's global assessment (PGA), and measurement of inflammatory markers including C-reactive protein (CRP), serum amyloid A (SAA), and S100 calcium-binding protein A12 (S100A12).
  
  
  RESULTS
  Treatment with rilonacept resulted in a rapid clinical response as demonstrated by significant reductions in daily health assessment scores and PGA scores compared with baseline levels (P < 0.05). These effects, which were accompanied by reductions in CRP and SAA, continued over the treatment duration. Rilonacept treatment was well tolerated. There were no treatment-related severe adverse events and no clinically significant changes in laboratory safety parameters.
  
  
  CONCLUSIONS
  Rilonacept was effective and well tolerated in patients with SchS and may represent a promising potential therapeutic option.
authors.affiliation: Department of Dermatology and Allergy, Allergie-Centrum-Charité, Charité - Universitätsmedizin Berlin, Berlin, Germany.
authors.forename: K
authors.initials: K
authors.lastname: Krause
authors.forename: K
authors.initials: K
authors.lastname: Weller
authors.forename: R
authors.initials: R
authors.lastname: Stefaniak
authors.forename: H
authors.initials: H
authors.lastname: Wittkowski
authors.forename: S
authors.initials: S
authors.lastname: Altrichter
authors.forename: F
authors.initials: F
authors.lastname: Siebenhaar
authors.forename: T
authors.initials: T
authors.lastname: Zuberbier
authors.forename: M
authors.initials: M
authors.lastname: Maurer
mesh_terms: D000368:Aged; D015415:Biomarkers / Q000378:metabolism; D005260:Female; D006801:Humans; D018836:Inflammation Mediators / Q000378:metabolism; D007375:Interleukin-1 / Q000037:antagonists & inhibitors*; D008297:Male; D008875:Middle Aged; D011993:Recombinant Fusion Proteins / Q000009:adverse effects / Q000627:therapeutic use*; D019873:Schnitzler Syndrome / Q000188:drug therapy* / Q000378:metabolism / Q000473:pathology; D016896:Treatment Outcome; D014581:Urticaria / Q000188:drug therapy / Q000473:pathology
chemical_list: D015415:Biomarkers; D018836:Inflammation Mediators; D007375:Interleukin-1; D011993:Recombinant Fusion Proteins; C531377:rilonacept