Canakinumab in Children with Familial Mediterranean Fever: A Single-Center, Retrospective Analysis

Area of Science:

• Pediatric Rheumatology
• Autoinflammatory Diseases
• Immunology

Background:

• Familial Mediterranean Fever (FMF) is an autoinflammatory condition driven by Interleukin-1 (IL-1) overproduction.
• Colchicine is the primary treatment, but some pediatric FMF patients exhibit resistance or intolerance.
• Alternative therapies are crucial for managing refractory cases and preventing complications like amyloidosis.

Purpose of the Study:

• To evaluate the efficacy and safety of canakinumab, an IL-1β inhibitor, in pediatric patients with Familial Mediterranean Fever (FMF).
• To assess treatment response in FMF patients with colchicine resistance, intolerance, or specific disease manifestations.
• To analyze the impact of canakinumab on disease activity markers and clinical outcomes in this cohort.

Main Methods:

• A retrospective, single-cohort study of 14 pediatric FMF patients treated with canakinumab.
• Data collected included disease characteristics, reasons for canakinumab initiation, and treatment response.
• Evaluation of clinical outcomes, including attack frequency, proteinuria, and inflammatory markers.

Main Results:

• Canakinumab was indicated for colchicine resistance (11 patients), renal amyloidosis (1), and persistent arthritis (2).
• Complete response was achieved in 71.5% of all patients and 86% of those with a typical FMF phenotype.
• Significant reductions observed in attack frequency, proteinuria, erythrocyte sedimentation rate, and C-reactive protein.

Conclusions:

• Canakinumab demonstrates potential as an effective treatment for pediatric FMF patients with colchicine resistance, renal amyloidosis, or chronic oligoarthritis.
• Further investigation is warranted for canakinumab's efficacy in FMF patients with co-existing conditions, such as rheumatoid factor-positive polyarticular juvenile idiopathic arthritis.