Novel Therapeutics for the Treatment of Familial Mediterranean Fever: From Colchicine to Biologics

Familial Mediterranean fever (FMF), an inherited autosomal recessive disorder, is characterized by sporadic, paroxysmal attacks of fever and serosal inflammation, lasting 1–3 days. Patients may develop renal amyloidosis, arthritis, serositis, and skin and oral lesions. Diagnosis is based on clinical features, response to treatment with colchicine, and genetic analysis. Colchicine prevents attacks and renal amyloidosis, in addition to reversing proteinuria. Nonresponders may receive novel therapy, including interleukin (IL)-1 receptor antagonists and IL-1 decoy receptor. Recently, new options have been considered. Clinical Pharmacology & Therapeutics (2013); 95 1, 89–97 advance online publication 2 October 2013. doi:10.1038/clpt.2013.148