Corticosteroid use in PFAPA syndrome: clinical practice data from the JIR-CliPS Survey Study and a comprehensive literature review.

Objectives: Corticosteroids are used to abort disease flares in PFAPA syndrome. We aimed to obtain a global overview of physicians' corticosteroid usage strategies and analyze the data in the literature regarding corticosteroid use in PFAPA syndrome.

Methods: The JIR-CliPS PFAPA questionnaire included nine questions on corticosteroid use in addition to the demographic data questions. The survey was distributed via e-mail to potential respondents. The MEDLINE/PubMed and Scopus databases were searched systematically to extract the data regarding corticosteroid use in PFAPA syndrome.

Results: From 47 countries, 144 physicians (F/M = 2.6; 67.4% pediatric rheumatologists) answered the survey. Most respondents (n = 133; 92.4%) prescribe corticosteroids in PFAPA flares. The most frequently prescribed corticosteroid was prednisolone (63.2%). The definition of response to corticosteroid was indicated as "response within 12 h" by the highest number of respondents (n = 61; 42.4%). When corticosteroids cause an increase in attack frequency, most (57.9%) consider another treatment if this causes a decrease in quality of life. Forty-four (30.6%) respondents were "routinely" prescribing corticosteroids to PFAPA patients, and this practice was more frequent among more experienced physicians (p< 0.001).We identified 46 articles in the literature describing 4564 PFAPA patients treated with corticosteroids. Prednisone was the most frequently preferred corticosteroid (48.2%). Response to corticosteroids was around 95%, although an increase in attack frequency was noted in almost 35% of the patients.

Conclusion: Physicians frequently use corticosteroids for PFAPA in their routine clinical practice. Regarding treatment modification, the quality of life was a prominent consideration for physicians.