Colchicine prophylaxis in pediatric PFAPA: a systematic review.
Colchicine prophylaxis reduces pediatric PFAPA attacks, with benefits often within one month.
Colchicine prophylaxis in pediatric PFAPA: a systematic review.
UNLABELLED: Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) is the most common periodic fever syndrome in children.
This systematic review assessed the effectiveness of daily colchicine prophylaxis in pediatric PFAPA.
A short randomized comparison showed similar 3-month efficacy to cimetidine.
On-demand corticosteroids stop attacks but may shorten fever-free intervals.
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.
MEFV is not clearly associated.
Colchicine 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.