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.

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.

• Authors: Philip J Hashkes, Steven J Spalding, Rula Hajj-Ali, Edward H Giannini, Anne Johnson, Karyl S Barron, Michael H Weisman, Noune Pashinian, Andreas O Reiff, Jonathan Samuels, Dowain Wright, Daniel J Lovell, Bin Huang
• Journal: BioMed research international (Biomed Res Int) Vol. 2014 Pg. 854842 ( 2014) ISSN: 2314-6141 [Electronic] United States
• PMID: 25147819 (Publication Type: Clinical Trial, Comparative Study, Journal Article, 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.)

Chemical References

• Recombinant Fusion Proteins
• rilonacept

Topics

• 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