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

Objective: Familial Mediterranean fever (FMF) is the most common monogenic autoinflammatory disease. Colchicine is the primary treatment for FMF, although some patients do not respond well or are unable to tolerate it. For these patients, the addition of interleukin-1 (IL-1) antagonists is the preferred option. However, the impact of colchicine treatment alongside the use of IL-1 antagonists remains unclear.

Methods: We recruited adult FMF patients who satisfied the Eurofever and Pediatric Rheumatology International Trials Organization classification criteria and were receiving IL-1 antagonist treatment from our FMF cohort. All the patients exhibited colchicine intolerance or resistance. As per the FMF cohort protocol, the patients were longitudinally followed up, including assessments of their C-reactive protein, erythrocyte sedimentation rate, autoinflammatory disease activity index (AIDAI), and autoinflammatory damage index (ADDI).

Results: Among the 125 patients (68 female and 57 male), 96 received a combination of IL-1 antagonists and the maximum tolerated dose of colchicine, whereas 29 were treated exclusively with IL-1 antagonists due to colchicine intolerance. The patients' inflammatory markers, AIDAI activity, and ADDI damage scores did not differ significantly between the two groups during the follow-up period. Notably, the drug retention rates were significantly higher in the patients treated solely with IL-1 antagonists.

Conclusion: While the typical approach is to maintain colchicine treatment alongside the use of IL-1 antagonists, for patients who cannot tolerate effective colchicine doses, IL-1 antagonists alone may effectively control FMF disease activity.