The effect of rilonacept versus placebo on health-related quality of life in patients with poorly controlled familial Mediterranean fever

Biomed Res Int. 2014;2014:854842. doi: 10.1155/2014/854842. Epub 2014 May 15.

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.

Conclusion: 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.

Publication types

  • Clinical Trial
  • Comparative Study
  • Randomized Controlled Trial
  • Research Support, N.I.H., Intramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adult
  • Double-Blind Method
  • Familial Mediterranean Fever / drug therapy*
  • Female
  • Humans
  • Male
  • Quality of Life
  • Recombinant Fusion Proteins / therapeutic use*
  • Surveys and Questionnaires
  • Treatment Outcome
  • Young Adult

Substances

  • Recombinant Fusion Proteins
  • rilonacept

Associated data

  • ClinicalTrials.gov/NCT00582907