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

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, 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

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.

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.

References

    1. Sohar E, Gafni J, Pras M, Heller H. Familial Mediterranean fever. A survey of 470 cases and review of the literature. The American Journal of Medicine. 1967;43(2):227–253.
    1. The International FMF Consortium. Ancient missense mutations in a new member of the RoRet gene family are likely to cause familial Mediterranean fever. Cell. 1997;90:797–807.
    1. The French FMF Consortium. A candidate gene for familial Mediterranean fever. Nature Genetics. 1997;17:25–31.
    1. Ben-Chetrit E, Touitou I. Familial Mediterranean fever in the world. Arthritis Care and Research. 2009;61(10):1447–1453.
    1. Goldfinger SE. Colchicine for familial Mediterranean fever. The New England Journal of Medicine. 1972;287(25):p. 1302.
    1. Zemer D, Revach M, Pras M, et al. A controlled trial of colchicine in preventing attacks of familial Mediterranean fever. The New England Journal of Medicine. 1974;291(18):932–934.
    1. Dinarello CA, Wolff SM, Goldfinger SE, Dale DC, Alling DW. Colchicine therapy for familial Mediterranean fever. A double blind trial. The New England Journal of Medicine. 1974;291(18):934–937.
    1. Zemer D, Pras M, Sohar E, Modan M, Cabili S, Gafni J. Colchicine in the prevention and treatment of amyloidosis of familial Mediterranean fever. The New England Journal of Medicine. 1986;314(16):1001–1005.
    1. Lidar M, Scherrmann JM, Shinar Y, et al. Colchicine nonresponsiveness in familial Mediterranean fever: clinical, genetic, pharmacokinetic, and socioeconomic characterization. Seminars in Arthritis and Rheumatism. 2004;33(4):273–282.
    1. Ben-Chetrit E, Ozdogan H. Non-response to colchicine in FMF—definition, causes and suggested solutions. Clinical and Experimental Rheumatology. 2008;26(supplement 50):S49–S51.
    1. Buskila D, Zaks N, Neumann L, et al. Quality of life of patients with familial Mediterranean fever. Clinical and Experimental Rheumatology. 1997;15:355–360.
    1. Press J, Neumann L, Abu-Shakra M, Bolotin A, Buskila D. Living with a child with familial Mediterranean fever: does it affect the quality of life of the parents? Clinical and Experimental Rheumatology. 2000;18(1):103–106.
    1. Makay B, Ünsal E, Arslan N, Varni JW. Health-related quality of life of school-age children with familial Mediterranean fever. Clinical and Experimental Rheumatology. 2009;27(supplement 53):S96–S101.
    1. Deger SM, Ozturk MA, Demirag MD, et al. Health-related quality of life and its associations with mood condition in familial Mediterranean fever patients. Rheumatology International. 2011;31(5):623–628.
    1. Sahin S, Yalcin I, Senel S, et al. Assessment life quality of familial Mediterranean fever patients by short form-36 and its relationship with disease parameters. European Review for Medical and Pharmacological Sciences. 2013;17:958–963.
    1. Giese A, Kurucay M, Kilic L, et al. Quality of life in adult patients with familial Mediterranean fever living in Germany or Turkey compared to healthy subjects: a study evaluating the effect of disease severity and country of residence. Rheumatology International. 2013;33:1713–1719.
    1. Alayli G, Durmus D, Ozkaya O, Sen HE, Genc G, Kuru O. Frequency of juvenile fibromyalgia syndrome in children with familial Mediterranean fever: effects on depression and quality of life. Clinical and Experimental Rheumatology. 2011;29(supplement 69):S127–S132.
    1. Langevitz P, Buskila D, Finkelstein R, et al. Fibromyalgia in familial Mediterranean fever. Journal of Rheumatology. 1994;21(7):1335–1337.
    1. Kasapçopur Ö, Tengirsek M, Ercan G, et al. Hypermobility and fibromyalgia frequency in childhood familial Mediterranean fever. Clinical and Experimental Rheumatology. 2004;22(supplement 34):p. S79.
    1. Arcalyst. [Rilonacept] Prescribing Information. Tarrytown, NY, USA: Regeneron Pharmaceuticals, Inc.; 2009.
    1. Hashkes PJ, Spalding SJ, Giannini EH, et al. Rilonacept for colchicine-resistant or -intolerant familial Mediterranean fever: a randomized trial. Annals of Internal Medicine. 2012;157:533–541.
    1. Landgraf JM, Abetz L, Ware JE. The CHQ User's Manual. Boston, Mass, USA: The Health Institute, New England Medical Center; 1996.
    1. Ruperto N, Ravelli A, Pistorio A, et al. Cross-cultural adaptation and psychometric evaluation of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ) in 32 countries. Review of the general methodology. Clinical and Experimental Rheumatology. 2001;19(4):S1–S9.
    1. Amaryan G, Astvatsatryan V, Gabrielyan E, Panossian A, Panosyan V, Wikman G. Double-blind, placebo-controlled, randomized, pilot clinical trial of ImmunoGuard—a standardized fixed combination of Andrographis paniculata Nees, with Eleutherococcus senticosus Maxim, Schizandra chinensis Bail. and Glycyrrhiza glabra L. extracts in patients with familial Mediterranean fever. Phytomedicine. 2003;10(4):271–285.
    1. Kone-Paut I, Lachmann HJ, Kuemmerle-Deschner JB, et al. Sustained remission of symptoms and improved health-related quality of life in patients with cryopyrin-associated periodic syndrome treated with canakinumab: results of a double-blind placebo-controlled randomized withdrawal study. Arthritis Research & Therapy. 2011;13:p. R202.
    1. Ozçakar ZB, Yalçinkaya F, Yuksel S, Acar B, Gokmen D, Ekim M. Possible effect of subclinical inflammation on daily life in familial Mediterranean fever. Clinical Rheumatology. 2006;25:149–152.
    1. Yenokyan G, Armenian HK. Triggers for attacks in familial Mediterranean fever: application of the case-crossover design. American Journal of Epidemiology. 2012;175:1054–1061.
    1. Gidron Y, Berkovitch M, Press J. Psychosocial correlates of incidence of attacks in children with familial Mediterranean fever. Journal of Behavioral Medicine. 2003;26(2):95–104.
    1. Onat AM, Öztürk MA, Özçakar L, et al. Selective serotonin reuptake inhibitors reduce the attack frequency in familial Mediterranean fever. Tohoku Journal of Experimental Medicine. 2007;211(1):9–14.
    1. Lepore L, Paloni G, Caorsi R, et al. Follow-up and quality of life of patients with cryopyrin-associated periodic syndromes treated with anakinra. Journal of Pediatrics. 2010;157(2):310–315.

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