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

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 HarelShoghik Akoghlanian, Lori Tucker, Mariana Correia Marques, Hemalatha Srinivasalu, Evan J. Propst, Greg R. Licameli, Fatma Dedeoglu, Sivia Lapidus, Ronald Laxer, Lisa Imundo, Paul Tsoukas, Peter Wright, Kelly Brown, Rima Khasawneh, Rosie Scuccimarri, Evan Mulvihill, Meghan Aabo, Edwin Anderson, Leslie Abramson, Daniela Adelean, Danielle Dumez, Marla Guzman, Renee Pang, Ellen Go, Katalin Koranyi, Donald Goldsmith, Hanna Kim, Andrew Zeft, Rayfel Schnieder, Victoria Statler, Lauren Steele, Lori Broderick, Hal Hoffman, Sriharsha Cherukumilli Grevich, Elizabeth Chalom, Michal Cidon, Robert Sundel, Nadine Saad, Deborah McCurdy, Grant Schulert, Ali Yalcindag, Eric Yen, Sara Stern, Karen Durrant, Yonatan Butbul, Jonathan Hausmann

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish
Article number31
JournalPediatric Rheumatology
Volume18
Issue number1
DOIs
StatePublished - 15 Apr 2020

Keywords

  • Consensus treatment plan
  • PFAPA
  • Periodic fever
  • Recurrent fever

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