Background PFAPA syndrome (periodic fever, aphtosis, pharyngitis and adenitis) is the most frequent cause of recurrent fever of inflammatory origin in pediatrics. Available treatments are designed to interrupt the episodes and others to prevent the recurrence of the febrile episodes (baseline treatment).
Objectives Evaluation of the efficacy of the different treatments in our international cohort of PFAPA patients and comparison of these data with the results from the literature.
Methods We extracted the information regarding the treatments of 301 PFAPA patients who were included between 2007 and 2009 in our database. The treatment response was evaluated as absent, incomplete or complete. Literature search was done by medline and English and French written publications were analyzed.
Results Half of the patients were treated with corticosteroids which have an excellent efficacy to interrupt the febrile episodes with a response rate in 94%. NSAIDs and Paracetamol were taken in nearly 70% of patients with only a response rate (mainly incomplete) of 55% and 33% respectively. Only 30% (91 patients) underwent a baseline treatment. Among these treatments, adenotonsillectomy has the best response (90%) before tonsillectomy alone (68%). Colchicine, cimetidine and adenoidectomy alone were effective in only a minority of patients: cimetidine 25% (6/23), colchicine 33% (5/15), adenoidectomy 14% (1/7). In literature one retrospective study shows a response to colchicine with prolongation of fever free interval in 89%. Otherwise the literature contains only 2 randomized studies on PFAPA treatment; the other publications are non randomized trials, description of cohorts (15-105 patients) or case reports. The criteria used for selecting patients with PFAPA varied between the studies and are not always precisely mentioned.
Conclusions In accordance with the literature, we show the efficacy of corticosteroids in treatment of febrile episodes and that adenotonsillectomy prevents febrile episodes in a significant number of patients. With regard to this cohort and the literature we recommend starting the treatment with corticosteroids. Colchicine can be added in association with the corticosteroids in PFAPA patients who have febrile episodes with short intervals and an important repercussion on every-day live. In children with failure of medical treatment and important repercussion of the quality of life, surgery should be considered. However the evidence showed in the publications on the efficacy of treatments in PFAPA lack strong evidences. The principal weaknesses are the absence of precise and validated diagnostic criteria and the low number of randomized studies. Our work underlines the necessity of controlled trials in large and homogenous cohorts of patients with PFAPA.
Disclosure of Interest None Declared