Colchicine-intolerant familial mediterranean fever patients: A comparative study between different colchicine doses and IL-1 inhibitor monotherapy

Area of Science:

• Rheumatology
• Genetics
• Immunology

Background:

• Familial Mediterranean Fever (FMF) is a prevalent monogenic autoinflammatory disorder.
• Colchicine is the standard FMF treatment, but efficacy is limited by intolerance or resistance in some patients.
• Interleukin-1 (IL-1) antagonists are recommended for refractory FMF, yet their use with colchicine requires further investigation.

Purpose of the Study:

• To evaluate the efficacy and outcomes of IL-1 antagonist monotherapy versus combination therapy in FMF patients with colchicine intolerance or resistance.
• To compare inflammatory markers, disease activity, and damage scores between treatment groups.

Main Methods:

• Adult FMF patients meeting Eurofever/Pediatric Rheumatology International Trials Organization criteria were enrolled.
• Patients were categorized based on treatment: IL-1 antagonist plus tolerated colchicine dose, or IL-1 antagonist monotherapy due to colchicine intolerance.
• Longitudinal follow-up included assessments of C-reactive protein, erythrocyte sedimentation rate, Autoinflammatory Disease Activity Index (AIDAI), and Autoinflammatory Damage Index (ADDI).

Main Results:

• Of 125 patients, 96 received combination therapy and 29 received IL-1 antagonist monotherapy.
• No significant differences in inflammatory markers, AIDAI, or ADDI were observed between groups.
• Drug retention rates were significantly higher in patients treated solely with IL-1 antagonists.

Conclusions:

• IL-1 antagonists alone can effectively control FMF disease activity in patients with colchicine intolerance.
• Monotherapy with IL-1 antagonists offers a viable alternative for FMF management, demonstrating comparable efficacy and improved tolerability.
• These findings challenge the routine combination approach for colchicine-intolerant FMF patients.