Drug1st line3 diseases
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 level
- green
Used across diseases
| Disease | Response rate | Line | Evidence |
|---|---|---|---|
| Familial Mediterranean Fever | ~95% (attack prevention) | 1st | green |
| Hypocomplementemic Urticarial Vasculitis Syndrome | Variable; partial responses reported | 1st | amber |
| TNF Receptor-Associated Periodic Syndrome | 12.5% complete response | Alternative | red |
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
| Molecule | Role | Expression | Evidence |
|---|---|---|---|
| Pyrin (MEFV) | Inflammasome sensor protein (mutated in FMF) | Gain-of-function mutations | established |
| Serum Amyloid A (SAA) | Acute-phase reactant and amyloid precursor | Elevated | established |