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IL1-blocking therapy in colchicine-resistant familial Mediterranean fever

Birgit Maria Köhler1, Hanns-Martin Lorenz1, Norbert Blank1

  • 1Division of Rheumatology, Department of Internal Medicine, Heidelberg University Hospital, Heidelberg, Germany.

European Journal of Rheumatology
|December 4, 2018
PubMed
Abstract

Insights

Interleukin-1 (IL-1) blocking therapy effectively reduced familial Mediterranean fever (FMF) attacks in patients resistant to colchicine. This treatment demonstrated rapid symptom suppression and was well-tolerated long-term.

Area of Science:

  • Rheumatology
  • Genetics
  • Immunology

Background:

  • Familial Mediterranean fever (FMF) is a genetic autoinflammatory disorder.
  • Colchicine resistance affects 10%-20% of FMF patients, necessitating alternative treatments.
  • FMF can lead to severe complications like amyloidosis.

Purpose of the Study:

  • To evaluate the efficacy and safety of IL-1 blocking agents (anakinra or canakinumab) in FMF patients with inadequate response to colchicine.
  • To assess the impact of IL-1 blockade on FMF disease activity, severity scores, and patient-reported outcomes.

Main Methods:

  • A cohort study analyzing 31 adult FMF patients with colchicine resistance.
  • Patients were treated with anakinra or canakinumab.
  • Clinical, laboratory, MEFV mutation data, and patient-reported outcomes were collected and analyzed.

Main Results:

  • IL-1 blocking therapy rapidly suppressed FMF symptoms and inflammation within 2±3 days.
  • Significant reduction in FMF attack frequency (p<0.003) and improved FMF activity (p<0.0001) were observed.
  • FMF severity correlated with high-penetrance MEFV mutations; 39% of patients had amyloidosis.

Conclusions:

  • IL-1 blocking therapy is a well-tolerated and effective treatment for colchicine-resistant FMF.
  • Therapy provides sustained suppression of FMF symptoms and reduces attack frequency.
  • Long-term treatment (median 2 years) showed good tolerability.

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