title: The effect of rilonacept versus placebo on health-related quality of life in patients with poorly controlled familial Mediterranean fever.
abstract: OBJECTIVE
  To examine the effect of rilonacept on the health-related quality of life (HRQoL) in patients with poorly controlled familial Mediterranean fever (FMF).
  
  
  METHODS
  As part of a randomized, double-blinded trial comparing rilonacept and placebo for the treatment of FMF, patients/parents completed the modified Child Health Questionnaire (CHQ) at baseline, and at the start and end of each of 4 treatment courses, 2 each with rilonacept and placebo.
  
  
  RESULTS
  Fourteen subjects were randomized; mean age was 24.4 ± 11.8 years. At baseline the physical HRQoL score was significantly less (24.2 ± 49.5) but the psychosocial score was similar to the population norm (49.5 ± 10.0). There were significant improvements in most HRQoL concepts after rilonacept but not placebo. Significant differences between rilonacept and placebo were found in the physical (33.7 ± 16.4 versus 23.7 ± 14.5, P = 0.021) but not psychosocial scores (51.4 ± 10.3 versus 49.8 ± 12.4, P = 0.42). The physical HRQoL was significantly impacted by the treatment effect and patient global assessment.
  
  
  CONCLUSIONS
  Treatment with rilonacept had a beneficial effect on the physical HRQoL in patients with poorly controlled FMF and was also significantly related to the patient global assessment. This trial is registered with ClinicalTrials.gov Identifier NCT00582907.
authors.affiliation: Cleveland Clinic Foundation, 9500 Euclid Avenue, Cleveland, OH 44195, USA ; Pediatric Rheumatology Unit, Shaare Zedek Medical Center, P.O. Box 3235, 91031 Jerusalem, Israel.
authors.forename: Philip J
authors.initials: PJ
authors.lastname: Hashkes
authors.affiliation: Cleveland Clinic Foundation, 9500 Euclid Avenue, Cleveland, OH 44195, USA.
authors.forename: Steven J
authors.initials: SJ
authors.lastname: Spalding
authors.affiliation: Cleveland Clinic Foundation, 9500 Euclid Avenue, Cleveland, OH 44195, USA.
authors.forename: Rula
authors.initials: R
authors.lastname: Hajj-Ali
authors.affiliation: Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH 45229, USA.
authors.forename: Edward H
authors.initials: EH
authors.lastname: Giannini
authors.affiliation: Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH 45229, USA.
authors.forename: Anne
authors.initials: A
authors.lastname: Johnson
authors.affiliation: Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, DHHS, 33 North Drive, MSC 3207, Bethesda, MD 20892, USA.
authors.forename: Karyl S
authors.initials: KS
authors.lastname: Barron
authors.affiliation: Cedars-Sinai Medical Center, 8700 Beverly Boulevard, Los Angeles, CA 90048, USA.
authors.forename: Michael H
authors.initials: MH
authors.lastname: Weisman
authors.affiliation: Cedars-Sinai Medical Center, 8700 Beverly Boulevard, Los Angeles, CA 90048, USA.
authors.forename: Noune
authors.initials: N
authors.lastname: Pashinian
authors.affiliation: Children's Hospital of Los Angeles, 4650 Sunset Boulevard, Los Angeles, CA 90027, USA.
authors.forename: Andreas O
authors.initials: AO
authors.lastname: Reiff
authors.affiliation: NYU Langone Hospital for Joint Diseases, 301 East 17th Street, New York, NY 10003, USA.
authors.forename: Jonathan
authors.initials: J
authors.lastname: Samuels
authors.affiliation: Children's Hospital Central California, 9300 Valley Children's Place, Madera, CA 93636, USA.
authors.forename: Dowain
authors.initials: D
authors.lastname: Wright
authors.affiliation: Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH 45229, USA.
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: Lovell
authors.affiliation: Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH 45229, USA.
authors.forename: Bin
authors.initials: B
authors.lastname: Huang
mesh_terms: D000328:Adult; D004311:Double-Blind Method; D010505:Familial Mediterranean Fever / Q000188:drug therapy*; D005260:Female; D006801:Humans; D008297:Male; D011788:Quality of Life; D011993:Recombinant Fusion Proteins / Q000627:therapeutic use*; D011795:Surveys and Questionnaires; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D011993:Recombinant Fusion Proteins; C531377:rilonacept