Background Familial Mediterranean fever (FMF) is the most common auto inflammatory syndrome with recurrent inflammatory episodes. Colchicine is the main treatment of FMF. It is also effective in preventing secondary amyloidosis. There is increasing number of refractory cases which were successfully treated with biologics targeting IL-1 production in patients with FMF. However, currently there is no controlled study evaluating the safety and efficacy of IL-1 blockage in management of FMF patients who were unresponsive to colchicine.
Objectives The aim of this report was to evaluate and discuss treatment of pediatric FMF patients with anti-interleukin1 (IL1) agents.
Methods We retrospectively reviewed the medical records of FMF patients attended to our pediatric rheumatology department. Six patients diagnosed as colchicine resistant were participated in our series. Refractory FMF or unresponsive to colchicine was described as severe and frequent attacks and/or having high acute phase reactance levels despite having a maximum dose of colchicine (2mg/day). Disease course, adverse effects, duration of follow-up, treatment protocols, responses to the therapies were discussed.
Results Six patients (5 male, 1 female) having refractory FMF were identified. The mean age of the patients was 14.3 years (range 10.6-19). The mean time of colchicine usage was 6 years (range 2-10). The median follow-up time from starting of colchicine to the beginning of biologic agents was 5.1 years (range 1-10). The mean time of anti-IL 1 agents usage was 0.99 years (range 0.15-1,5). MEFV gene analyses revealed homozygote M694V mutations in 5 patients and heterozygote M694V mutations in one patient. They were all treated with anakinra and/or canakinumab along with the colchicine management. The use of anti-IL1 drugs were beneficial to all patients with complete remission. Injection- side reaction developed in only one patient and lasted in three weeks. None of them had any severe adverse effects due to the therapy.
Conclusions Anakinra and canakinumab seems to be effective in patients difficult to treat with colchicine as shown both in our series and in the literature. Therefor controlled trials are needed to evaluate the safety and long term efficacy of IL-1 targeting agents in resistant patients.
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Disclosure of Interest None declared