IL-1 BLOCKERS in pediatric Familial Mediterranean Fever patients: A Single‑Center, Retrospective Analysis
2Rheumatology Unit, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel-Hashomer, ישראל
3Department of Pediatrics A, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel-Hashomer, Israel, ישראל
4Pediatric Nephrology Unit, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel-Hashomer, ישראל
Introduction: Familial Mediterranean fever (FMF) is an autosomal recessive disorder, caused by a point mutation in the Mediterranean Fever (MEFV) gene, located on chromosome 16p. It is an autoinflammatory disease characterized by interleukin (IL)-1 overproduction. Since the first report in 1972, Colchicine is the mainstay drug of treatment for FMF. Yet, Colchicine is not sufficient in 5 to 10% of the patients. Recently, anti-interleukin 1 agents are successfully used for those patients.
Aim: Evaluating the disease characteristics, indications, and treatment responses of pediatric FMF patients treated with anti-interleukin 1 agents in our pediatric rheumatology department.
Methods: Overall, more than 600 pediatric FMF patients were followed in our center between the years 2006-2019. Twenty of them were treated with anti-interleukin 1 agents. Data were collected retrospectively from the patients` computerized charts.
Results: Of the 20 included patients, 80% (n=16) were homozygotes to the reported MEFV mutation, 15% (n=3) were heterozygotes and 5% (n=1) had no mutation. Indications for anti-interleukin 1 agent treatment were either colchicine resistance (n=19) or colchicine intolerance (n=11). Complete response was obtained in 9/20 (45%) among all patients and partial response in 9/20 (45%) with 2 patients (10%) showing no response.
Conclusion: Pediatric FMF patients with colchicine resistance or intolerance may find anti-interleukin 1 agents as an effective additive treatment.
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