1. Consensus treatment plans for periodic fever, aphthous stomatitis, pharyngitis and adenitis syndrome (PFAPA): a framework to evaluate treatment responses from the childhood arthritis and rheumatology research alliance (CARRA) PFAPA work group
- Author
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Gil, Amarilyo, Deborah, Rothman, Kalpana, Manthiram, Kathryn M, Edwards, Suzanne C, Li, Gary S, Marshall, Cagri, Yildirim-Toruner, Kathleen, Haines, Polly J, Ferguson, Geraldina, Lionetti, Julie, Cherian, Yongdong, Zhao, Patricia, DeLaMora, Grant, Syverson, Simona, Nativ, Marinka, Twilt, Ian C, Michelow, Yuriy, Stepanovskiy, Akaluck, Thatayatikom, Liora, Harel, Shoghik, Akoghlanian, Lori, Tucker, Mariana Correia, Marques, Hemalatha, Srinivasalu, Evan J, Propst, Greg R, Licameli, Fatma, Dedeoglu, Sivia, Lapidus, and Jonathan, Hausmann
- Subjects
Pediatrics ,lcsh:Diseases of the musculoskeletal system ,Childhood arthritis ,medicine.medical_treatment ,Disease ,0302 clinical medicine ,Adrenal Cortex Hormones ,Immunology and Allergy ,030212 general & internal medicine ,Child ,PFAPA ,lcsh:RJ1-570 ,Pharyngitis ,Syndrome ,Tubulin Modulators ,Histamine H2 Antagonists ,Periodic fever ,Consensus treatment plan ,Child, Preschool ,Stomatitis, Aphthous ,medicine.symptom ,Periodic fever syndrome ,Cimetidine ,Research Article ,medicine.medical_specialty ,Antipyretics ,Fever ,Advisory Committees ,Placebo ,03 medical and health sciences ,Rheumatology ,Lymphadenitis ,Internal medicine ,medicine ,Humans ,Recurrent fever ,Tonsillectomy ,030203 arthritis & rheumatology ,business.industry ,lcsh:Pediatrics ,medicine.disease ,Pediatrics, Perinatology and Child Health ,lcsh:RC925-935 ,Periodic fever, aphthous stomatitis, pharyngitis and adenitis ,business ,Colchicine ,Neck - Abstract
Background Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome is the most common periodic fever syndrome in children. There is considerable heterogeneity in management strategies and a lack of evidence-based treatment guidelines. Consensus treatment plans (CTPs) are standardized treatment regimens that are derived based upon best available evidence and current treatment practices that are a way to enable comparative effectiveness studies to identify optimal therapy and are less costly to execute than randomized, double blind placebo controlled trials. The purpose of this project was to develop CTPs and response criteria for PFAPA. Methods The CARRA PFAPA Working Group is composed of pediatric rheumatologists, infectious disease specialists, allergists/immunologists and otolaryngologists. An extensive literature review was conducted followed by a survey to assess physician practice patterns. This was followed by virtual and in-person meetings between 2014 and 2018. Nominal group technique (NGT) was employed to develop CTPs, as well as inclusion criteria for entry into future treatment studies, and response criteria. Consensus required 80% agreement. Results The PFAPA working group developed CTPs resulting in 4 different treatment arms: 1. Antipyretic, 2. Abortive (corticosteroids), 3. Prophylaxis (colchicine or cimetidine) and 4. Surgical (tonsillectomy). Consensus was obtained among CARRA members for those defining patient characteristics who qualify for participation in the CTP PFAPA study. Conclusion The goal is for the CTPs developed by our group to lead to future comparative effectiveness studies that will generate evidence-driven therapeutic guidelines for this periodic inflammatory disease.
- Published
- 2019