Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain

Abstract

Objective: To evaluate the efficacy of colchicine therapy in pediatric patients with PFAPA syndromewho present with an incomplete response to the standard treatment or with frequent episodes (aninterval of less than 14 days between two disease flares).Methods: A multicenter cohort study of children diagnosed with PFAPA syndrome and treated withcolchicine was performed in three separate hospitals located in Spain. The patients clinical and laboratory data were reviewed by accessing their medical records. Response to colchicine was evaluatedafter 12 months of treatment for frequency, duration, and intensity of PFAPA episodes.Results: A total of 13 children were included in our study, 43% of whom were boys. Median age of thecolchicine therapy initiation was 6 years (interquartile range (IQR)=3-9.5). Following a 12-month period of colchicine therapy (median dosage of 0.02 mg/kg/day; IQR=0.02-0.03), a significant decrease inthe median number of flares (median 8; IQR=7-14 vs 3; IQR=2-4; p=0.005) and the duration of diseaseepisodes (median 4 days; IQR=3.25-5.125 vs 1 day; IQR=1-2; p=0.003) was observed. Furthermore, thehighest degree of fever during disease flares was reduced from median 40ºC (IQR=39.5-40) to 38.5ºC(IQR=37.7-38.9) (p=0.002).Conclusion: Colchicine therapy decreased the frequency and intensity of PFAPA. The use of colchicinecould be an effective treatment in pediatric patients with PFAPA syndrome who present with frequentor severe relapses.