Introduction: Familial Mediterranean fever (FMF) is the most common hereditary autoinflammatory syndrome. The treatment of choice is colchicine. However, ~40% of patients are only partial responders and 5-10% are non-responders. Advances in the understanding of the role of pyrin in the regulation of interleukin (IL)-1β activation has led to use of anti-IL-1 agents for colchicine-resistant FMF.
Areas covered: The authors performed a literature search of anti-IL-1 treatment for FMF, particularly canakinumab, a humanized IL-1β antibody, by searching PubMed/Medline/Scopus since 2001 and proceedings of major rheumatologic conferences since 2011 for unpublished studies.
Expert opinion: Many reports of successful treatments with anti-IL-1 agents were published since 2007. In 2011, the first case reports of successful treatment with canakinumab were reported. Successful phase II trials reported in 2014 and 2015 led to a double-blind, randomized, placebo-controlled phase III trial in patients with colchicine-resistant FMF. Significantly more canakinumab treated patients attained the very stringent primary outcome measure and secondary outcomes vs. those treated with placebo. The safety profile was similar to canakinumab trials for other indications. Canakinumab appears to be an excellent alternative for the vast majority of patients with colchicine-resistant FMF, with an adequate safety profile.
Keywords: Amyloidosis; anakinra; canakinumab; colchicine; familial Mediterranean fever; interleukin-1; pyrin; rilonacept; treatment.