The Effect of Rilonacept versus Placebo on Health-Related Quality of Life in Patients with Poorly Controlled Familial Mediterranean Fever

Abstract EN

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.