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Randomized Controlled Trial
. 2014:2014:854842.
doi: 10.1155/2014/854842. Epub 2014 May 15.

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

Philip J Hashkes  1 , Steven J Spalding  2 , Rula Hajj-Ali  2 , Edward H Giannini  3 , Anne Johnson  3 , Karyl S Barron  4 , Michael H Weisman  5 , Noune Pashinian  5 , Andreas O Reiff  6 , Jonathan Samuels  7 , Dowain Wright  8 , Daniel J Lovell  3 , Bin Huang  3
Affiliations
Randomized Controlled Trial

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

Philip J Hashkes et al. Biomed Res Int. 2014.

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.

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Figures

Figure 1
Figure 1
Health-related quality of life scores measured at screening and baseline visits and after treatment with rilonacept and placebo. Results are reported as a mean and 95% confidence interval. The 1st row includes the physical summary score and the 4 individual concepts weighted positively in calculating this score. The 2nd row includes the psychosocial summary score and the 6 concepts weighted positively in calculating this score. The 3rd row includes those concepts not used in calculating the summary scores. PhS: physical summary score; PF: physical function; BP: bodily pain and discomfort; GH: general health perceptions; RP: role/social limitations due to physical health; PsS: psychosocial summary score; REB: role/social limitations due to emotional or behavioral difficulties; BE: behavior; MH: mental health; SE: self-esteem; PE: emotional impact on parent/patient; PT: parental/patient time impact; FA: family activities; FC: family cohesion; GBE: global behavior; GGH: global health; CH: change in health.
Figure 2
Figure 2
Changes in health-related quality of life scores after use of rilonacept or placebo when compared to the screening and baseline visits. The order of the concepts is the same as Figure 1. CH was multiplied by 20 to align score with other concepts. PhS: physical summary score; PF: physical function; BP: bodily pain and discomfort; GH: general health perceptions; RP: role/social limitations due to physical health; PsS: psychosocial summary score; REB: role/social limitations due to emotional or behavioral difficulties; BE: behavior; MH: mental health; SE: self-esteem; PE: emotional impact on parent/patient; PT: parental/patient time impact; FA: family activities; FC: family cohesion; GBE: global behavior; GGH: global health; CH: change in health.
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References

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