Colchicine efficacy for undefined autoinflammatory diseases


Autoinflammatory diseases (AIDs) are a rare group of illnesses characterized by unprovoked episodes of fever and systemic inflammation.  Patients with defined AIDs benefit from evidence-based treatment guidelines1. Unfortunately, many patients with AIDs do not have a genetic diagnosis and their symptoms do not match any of the known AIDs. There is an unmet need to provide effective treatment to these patients with undefined AIDs (uAIDs). We examined the efficacy of colchicine in patients with uAIDs and identified patient characteristics and clinical factors that predicted a good colchicine response.


We conducted a retrospective chart review of patients with a clinical diagnosis of uAIDs who tolerated colchicine. Responses were defined as follows:

  • Good response: decrease in the frequency, severity, AND length of febrile episodes without requiring additional medications.
  • Partial response: decrease in the frequency, severity, OR length of episodes; additional medicines may have been required.
  • No response: no benefit was appreciated.

For statistical analyses, partial and non-responders were combined into a single group.


184 patients with uAIDs were identified and 68 had used colchicine.

  • 33 (48.5%) were good colchicine responders.
  • 30 (44.1%) were partial responders.
  • 5 (7.4%) were non-responders.

Patient characteristics based on colchicine response are shown in Table 1; ethnicities are shown in Figures 1 and 2. Clinical characteristics of febrile episodes, based on colchicine response, are shown in Table 2.


  • Colchicine was effective treatment for most patients with uAIDs, with 48% and 44% of patients having a good or partial response, respectively.
  • Patient characteristics, including the presence of mutations in AID genes (eg. MEFV), a family history of recurrent fevers, and age of disease onset did not predict colchicine response.
  • On the other hand, features of febrile episodes were useful in predicting colchicine response: patients were more likely to have a good response if they had vomiting during flares; abdominal pain approached statistical significance.
  • Although prior studies have shown colchicine to be beneficial for patients with recurrent aphthous stomatitis2, Behcet’s3, and PFAPA4, our cohort of patients with uAIDs and aphthous ulcers were less likely to benefit from colchicine than patients without aphthae.


1. Haar ter NM, Oswald M, Jeyaratnam J, et al. Recommendations for the management of autoinflammatory diseases. Ann Rheum Dis. 2015;74(9):1636-1644. doi:10.1136/annrheumdis-2015-207546.

2. Katz, J., Langevitz, P., Shemer, J., Barak, S. & Livneh, A. Prevention of recurrent aphthous stomatitis with colchicine: an open trial. Journal of American Dermatology 31, 459–461 (1994).

3. Colchicine versus placebo in Behçet’s disease: randomized, double-blind, controlled crossover trial. 19, 542–549 (2009).

4. Butbul Aviel, Y., Tatour, S., Gershoni-Baruch, R. & Brik, R. Colchicine as a therapeutic option in periodic fever, aphthous stomatitis, pharyngitis, cervical adenitis (PFAPA) syndrome. Seminars in arthritis and Rheumatism 45, 471–474 (2016).