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.

A survey of resistance to colchicine treatment for French patients with familial Mediterranean fever

“…Thus, resistant patients seem to be good candidates for biologic treatment (i.e., anti-IL-1 treatments). Nevertheless, none of these drugs are being approved, and increasing their use may greatly increase the overall cost of care for FMF [ 6 , 7 , 11 ].…”

International Retrospective Chart Review of Treatment Patterns in Severe Familial Mediterranean Fever, Tumor Necrosis Factor Receptor–Associated Periodic Syndrome, and Mevalonate Kinase Deficiency/Hyperimmunoglobulinemia D Syndrome

“…The FMF patients included in this study notably had unusually severe forms of the disease, with an inadequate response to colchicine, and only represent a very small portion of the FMF population . Unfortunately no definition of colchicine resistance/intolerance has been widely agreed upon . However, the definition for this study is in accordance with multiple articles that have specifically addressed this issue .…”

Conventional and novel therapeutic options in children with familial Mediterranean fever: A rare autoinflammatory disease

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