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 Harel; Shoghik Akoghlanian; Lori Tucker; Mariana Correia Marques; Hemalatha Srinivasalu; Evan J Propst; Greg R Licameli; Fatma Dedeoglu; Sivia Lapidus

doi:10.1186/s12969-020-00424-x

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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

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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, …

Pediatric rheumatology online journal, Vol.18(1), pp.31-31

04/15/2020

DOI: 10.1186/s12969-020-00424-x

PMCID: PMC7157990

PMID: 32293478

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Abstract

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. 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. 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. 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.

Cimetidine - therapeutic use

Pharyngitis - physiopathology

Antipyretics - therapeutic use

Colchicine - therapeutic use

Humans

Fever - therapy

Child, Preschool

Lymphadenitis - therapy

Adrenal Cortex Hormones - therapeutic use

Histamine H2 Antagonists - therapeutic use

Fever - physiopathology

Syndrome

Tubulin Modulators - therapeutic use

Advisory Committees

Lymphadenitis - physiopathology

Stomatitis, Aphthous - therapy

Tonsillectomy

Stomatitis, Aphthous - physiopathology

Neck

Child

Pharyngitis - therapy

Details

Title: Subtitle: 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
Creators: Gil Amarilyo - Pediatric Rheumatology Unit, Schneider Children's Medical Center of Israel, Petach Tikva; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel. gamarilyo@clalit.org.il
Deborah Rothman - Massacusetts General Hospital for Children, Boston, MA, USA
Kalpana Manthiram - National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA
Kathryn M Edwards - Vanderbilt University School of Medicine, Nashville, TN, USA
Suzanne C Li - Joseph M Sanzari Children's Hospital, Hackensack Meridian Health, Hackensack, NJ, USA
Gary S Marshall - Department of Pediatrics, University of Louisville, Louisville, KY, USA
Cagri Yildirim-Toruner - Nationwide Children's Hospital, Columbus, OH, USA
Kathleen Haines - Joseph M Sanzari Children's Hospital, Hackensack Meridian Health, Hackensack, NJ, USA
Polly J Ferguson - Stead Family Department of Pediatrics, University of Iowa Carver College of Medicine, Iowa City, IA, USA
Geraldina Lionetti - UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA
Julie Cherian - Stony Brook University Hospital, Stony Brook, NY, USA
Yongdong Zhao - Seattle Children's Hospital, University of Washington, Seattle, WA, USA
Patricia DeLaMora - Weill Cornell Medical College, New York, NY, USA
Grant Syverson - Medical College of Wisconsin, Wauwatosa, WI, USA
Simona Nativ - Goryeb Children's Hospital, Morristown, NJ, USA
Marinka Twilt - Rheumatology, Department of Pediatrics, Alberta Children's Hospital, University of Calgary, Calgary, Alberta, Canada
Ian C Michelow - Alpert Medical School of Brown University, Providence, RI, USA
Yuriy Stepanovskiy - Shupyk National Medical Academy of Postgraduate Education, Kyiv, Ukraine
Akaluck Thatayatikom - Department of Pediatrics, University of Florida, Gainesville, FL, USA
Liora Harel - Pediatric Rheumatology Unit, Schneider Children's Medical Center of Israel, Petach Tikva; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
Shoghik Akoghlanian - Nationwide Children's Hospital, Columbus, OH, USA
Lori Tucker - BC Children's Hospital, Vancouver, BC, Canada
Mariana Correia Marques - Boston Children's Hospital, Harvard Medical School, Boston, MA, USA
Hemalatha Srinivasalu - Children's National Medical Center, Washington, DC, USA
Evan J Propst - Hospital for Sick Children, University of Toronto, Toronto, ON, Canada
Greg R Licameli - Boston Children's Hospital, Harvard Medical School, Boston, MA, USA
Fatma Dedeoglu - Boston Children's Hospital, Harvard Medical School, Boston, MA, USA
Sivia Lapidus - Joseph M Sanzari Children's Hospital, Hackensack Meridian Health, Hackensack, NJ, USA
Resource Type: Journal article
Publication Details: Pediatric rheumatology online journal, Vol.18(1), pp.31-31
DOI: 10.1186/s12969-020-00424-x
PMID: 32293478
PMCID: PMC7157990
NLM abbreviation: Pediatr Rheumatol Online J
ISSN: 1546-0096
eISSN: 1546-0096
Publisher: England
Language: English
Date published: 04/15/2020
Academic Unit: Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Rheumatology, Allergy, and Immunology
Record Identifier: 9984070713302771

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