Efficacy and safety of anti‐interleukin‐1 in children with colchicine‐resistant familial Mediterranean fever

Abstract Background The aim of this study was to evaluate the efficacy and safety of anti‐interleukin‐1 (IL‐1) therapies in colchicine‐resistant pediatric patients with familial Mediterranean fever (FMF). Methods In this study, we retrospectively evaluated 656 children with FMF and 27 patients who had been treated with anti‐IL‐1 therapies (anakinra/canakinumab) . Clinical and laboratory features, MEFV gene mutations, treatment responses were investigated. Results Twenty of the patients were treated with anakinra (the treatment of 6 patients who initially used anakinra was switched to canakinumab in the follow‐up period), and 13 patients were treated with canakinumab. Clinical symptom and severity scores decreased in all patients A decrease in acute phase reactants was also observed in patients. A total of 18 (66%) patients had a M694V homozygous mutation, while 24 (89%) patients had a M694V mutation, at least in one allele. Conclusions FMF patients with colchicine resistance may progress to amyloidosis. IL‐1 antagonist treatment could be used safely with a favorable outcome in pediatric patients with FMF resistance to colchicine therapy and/or who have renal amyloidosis.