Colchicine

Microtubule disruption; reduces leukocyte motility and phagocytosis

Response rate
~95% (attack prevention)
Onset
Days-weeks (prophylactic)
Route
Oral, 1-2 mg/day (adults), 0.5-1 mg/day (children)
Line
1st
IgM effect
Normalises SAA/CRP in most patients

Evidence summary

Cornerstone lifelong treatment since 1972. Three seminal 1974 RCTs established efficacy. Prevents attacks in ~95% of patients and prevents AA amyloidosis development. Paradoxically, FMF mutations render pyrin inflammasome activation insensitive to colchicine in vitro — clinical efficacy likely through effects on leukocyte motility, adhesion, and phagocytosis. Safe in pregnancy. ~5-10% of patients are resistant, ~5-10% are intolerant (GI side effects).

Molecular targets (2)

MoleculeRoleExpressionEvidence
Pyrin (MEFV)Inflammasome sensor protein (mutated in FMF)Gain-of-function mutationsestablished
Serum Amyloid A (SAA)Acute-phase reactant and amyloid precursorElevatedestablished

Sources (6)

DetailsGoldstein RC et al. (1974) Prophylactic colchicine therapy in familial Mediterranean fever: a controlled, double-blind study · Ann Intern MedDOI
DetailsDinarello CA et al. (1974) Colchicine therapy for familial Mediterranean fever: a double-blind trial · N Engl J MedDOI
DetailsZemer D et al. (1974) A controlled trial of colchicine in preventing attacks of familial Mediterranean fever · N Engl J MedPubMed
DetailsOzen S et al. (2025) EULAR/PReS endorsed recommendations for the management of familial Mediterranean fever (FMF): 2024 update · Ann Rheum DisDOI
DetailsDemirkaya E et al. (2016) Efficacy and safety of treatments in familial Mediterranean fever: a systematic review · Rheumatol IntDOI
DetailsCochrane Collaboration (2022) Interventions for reducing inflammation in familial Mediterranean fever · Cochrane Database Syst RevPubMed