Pediatric rheumatology

Colchicine prophylaxis efficacy in PFAPA syndrome: A comparative cohort study

Semin Arthritis RheumPublished Retrospective cohort study

Authors' conclusion

Colchicine prophylaxis significantly reduces PFAPA attack frequency, with therapeutic benefits that are independent of FMF genetic status.

Study context

Population
Pediatric PFAPA patients
Participants
55 patients
Exposure
Colchicine prophylaxis vs standard care alone
  • Colchicinen = 15
  • Standard caren = 40reference
Outcome
Change in inter-attack interval duration from baseline to study completion
Methodological detailscomparative cohort
  • Comparative cohort

Main finding

As reported in the abstract

Change in inter-attack interval

Colchicine

60

median · IQR 51 · days

Standard care

0

median · IQR 0 · days

P < 0.001

Observational data: associations, not effects.

Further findings

Baseline FMF mutation prevalence

P = 0.002

Treatment response by FMF genetic status

Colchicine's therapeutic benefit was consistent regardless of FMF genetic status

Original publication

Colchicine prophylaxis efficacy in PFAPA syndrome: A comparative cohort study. Semin Arthritis Rheum. Published 9 February 2026. doi:10.1016/j.semarthrit.2026.152941

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