Colchicine prophylaxis in pediatric PFAPA: a systematic review.

Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) is the most common periodic fever syndrome in children. On-demand corticosteroids stop attacks but may shorten fever-free intervals. This systematic review assessed the effectiveness of daily colchicine prophylaxis in pediatric PFAPA. Furthermore, MEFV status association with colchicine efficacy has been explored. PubMed, Scopus, and Web of Science were systematically searched from inception to 6 November 2025 to identify trials and observational studies presenting participants with a diagnosis of PFAPA, colchicine prophylaxis as interventions, and attack frequency/attack-free interval as outcomes. All eligible records identified up to that date were screened.

conclusionColchicine reduced attack frequency, lengthened attack-free intervals, and lowered steroid use, with benefits often within 1 month. A short randomized comparison showed similar 3-month efficacy to cimetidine. Adverse events were mostly mild gastrointestinal; discontinuations were uncommon. MEFV variants as predictors of response remain uncertain. Current evidence supports colchicine as an efficacy and generally well-tolerated preventive option. WHAT IS KNOWN: • PFAPA is the most common periodic fever in children. On-demand corticosteroids stop attacks but may shorten symptom-free intervals; preventive options include cimetidine, selective tonsillectomy, and biologics. • Colchicine modulates innate immunity and it is effective in familial Mediterranean fever; MEFV variants occur in a subset of PFAPA, supporting interest in repurposing. WHAT IS NEW: • Continuous colchicine reduced attack frequency, prolonged attack-free intervals, and lowered steroid use; adverse events were mostly mild gastrointestinal, with few treatment discontinuations. • Clinical improvement often appeared by about 1 month and stabilized by 3 months; this supports early reassessment to adjust dose or change therapy. MEFV is not clearly associated.