Colchicine prophylaxis in pediatric PFAPA: a systematic review
Rosario Francesco Dipasquale1, Paola Sinopoli2, Alessia Mendicino2
1Paediatrics, Department of Health Sciences, University of Catanzaro "Magna Graecia", 88100, Catanzaro, Italy. rosariofrancesco.dipasquale@studenti.unicz.it.
Insights
Daily colchicine prophylaxis effectively reduces PFAPA flare-ups and lengthens symptom-free periods in children. This treatment is generally well-tolerated, offering a viable alternative to on-demand steroids for managing periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA).
Area of Science:
- Pediatric Rheumatology
- Immunology
- Pharmacology
Background:
- Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) is the most prevalent periodic fever syndrome in pediatric populations.
- While corticosteroids manage acute PFAPA attacks, they may reduce subsequent symptom-free intervals, necessitating exploration of preventive strategies.
- Colchicine, known for its efficacy in familial Mediterranean fever, is being investigated for PFAPA due to shared innate immunity pathways and occasional MEFV variants in PFAPA patients.
Purpose of the Study:
- To systematically review the efficacy of daily colchicine prophylaxis in reducing attack frequency and prolonging attack-free intervals in pediatric PFAPA.
- To assess the impact of colchicine on the need for steroid use in PFAPA management.
- To explore the potential association between MEFV gene variants and colchicine response in PFAPA patients.
Main Methods:
- A systematic literature search was conducted across PubMed, Scopus, and Web of Science up to November 6, 2025.
- Included studies were trials and observational studies involving pediatric PFAPA patients treated with colchicine prophylaxis.
- Outcomes assessed included attack frequency, attack-free intervals, steroid use, and adverse events.
Main Results:
- Daily colchicine significantly decreased PFAPA attack frequency and extended symptom-free periods, with benefits often observed within one month.
- Colchicine prophylaxis led to a reduction in the requirement for steroid medication.
- Adverse events were predominantly mild gastrointestinal issues, and treatment discontinuation rates were low; MEFV variant association with efficacy remains unclear.
Conclusions:
- Continuous colchicine prophylaxis is an effective and generally well-tolerated preventive treatment option for pediatric PFAPA.
- The observed benefits in reducing attack frequency and steroid use support colchicine as a valuable therapeutic strategy.
- Further research is needed to clarify the role of MEFV variants in predicting colchicine response in PFAPA.
Abstract:
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.
Conclusion:
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. 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.
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