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.


Journal

Pediatric rheumatology online journal
ISSN: 1546-0096
Titre abrégé: Pediatr Rheumatol Online J
Pays: England
ID NLM: 101248897

Informations de publication

Date de publication:
15 Apr 2020
Historique:
received: 16 12 2019
accepted: 03 04 2020
entrez: 16 4 2020
pubmed: 16 4 2020
medline: 5 3 2021
Statut: epublish

Résumé

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.

Sections du résumé

BACKGROUND 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 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 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 CONCLUSIONS
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.

Identifiants

pubmed: 32293478
doi: 10.1186/s12969-020-00424-x
pii: 10.1186/s12969-020-00424-x
pmc: PMC7157990
doi:

Substances chimiques

Adrenal Cortex Hormones 0
Antipyretics 0
Histamine H2 Antagonists 0
Tubulin Modulators 0
Cimetidine 80061L1WGD
Colchicine SML2Y3J35T

Types de publication

Consensus Development Conference Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

31

Investigateurs

Ronald Laxer (R)
Lisa Imundo (L)
Paul Tsoukas (P)
Peter Wright (P)
Kelly Brown (K)
Rima Khasawneh (R)
Rosie Scuccimarri (R)
Evan Mulvihill (E)
Meghan Aabo (M)
Edwin Anderson (E)
Leslie Abramson (L)
Daniela Adelean (D)
Danielle Dumez (D)
Marla Guzman (M)
Renee Pang (R)
Ellen Go (E)
Katalin Koranyi (K)
Donald Goldsmith (D)
Hanna Kim (H)
Andrew Zeft (A)
Rayfel Schnieder (R)
Victoria Statler (V)
Lauren Steele (L)
Lori Broderick (L)
Hal Hoffman (H)
Sriharsha Cherukumilli Grevich (SC)
Elizabeth Chalom (E)
Michal Cidon (M)
Robert Sundel (R)
Nadine Saad (N)
Deborah McCurdy (D)
Grant Schulert (G)
Ali Yalcindag (A)
Eric Yen (E)
Sara Stern (S)
Karen Durrant (K)
Yonatan Butbul (Y)
Jonathan Hausmann (J)

Références

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Auteurs

Gil Amarilyo (G)

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 (D)

Massacusetts General Hospital for Children, Boston, MA, USA.

Kalpana Manthiram (K)

National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.

Kathryn M Edwards (KM)

Vanderbilt University School of Medicine, Nashville, TN, USA.

Suzanne C Li (SC)

Joseph M Sanzari Children's Hospital, Hackensack Meridian Health, Hackensack, NJ, USA.

Gary S Marshall (GS)

Department of Pediatrics, University of Louisville, Louisville, KY, USA.

Cagri Yildirim-Toruner (C)

Nationwide Children's Hospital, Columbus, OH, USA.

Kathleen Haines (K)

Joseph M Sanzari Children's Hospital, Hackensack Meridian Health, Hackensack, NJ, USA.

Polly J Ferguson (PJ)

Stead Family Department of Pediatrics, University of Iowa Carver College of Medicine, Iowa City, IA, USA.

Geraldina Lionetti (G)

UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.

Julie Cherian (J)

Stony Brook University Hospital, Stony Brook, NY, USA.

Yongdong Zhao (Y)

Seattle Children's Hospital, University of Washington, Seattle, WA, USA.

Patricia DeLaMora (P)

Weill Cornell Medical College, New York, NY, USA.

Grant Syverson (G)

Medical College of Wisconsin, Wauwatosa, WI, USA.

Simona Nativ (S)

Goryeb Children's Hospital, Morristown, NJ, USA.

Marinka Twilt (M)

Rheumatology, Department of Pediatrics, Alberta Children's Hospital, University of Calgary, Calgary, Alberta, Canada.

Ian C Michelow (IC)

Alpert Medical School of Brown University, Providence, RI, USA.

Yuriy Stepanovskiy (Y)

Shupyk National Medical Academy of Postgraduate Education, Kyiv, Ukraine.

Akaluck Thatayatikom (A)

Department of Pediatrics, University of Florida, Gainesville, FL, USA.

Liora Harel (L)

Pediatric Rheumatology Unit, Schneider Children's Medical Center of Israel, Petach Tikva; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

Shoghik Akoghlanian (S)

Nationwide Children's Hospital, Columbus, OH, USA.

Lori Tucker (L)

BC Children's Hospital, Vancouver, BC, Canada.

Mariana Correia Marques (MC)

Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.

Hemalatha Srinivasalu (H)

Children's National Medical Center, Washington, DC, USA.

Evan J Propst (EJ)

Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.

Greg R Licameli (GR)

Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.

Fatma Dedeoglu (F)

Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.

Sivia Lapidus (S)

Joseph M Sanzari Children's Hospital, Hackensack Meridian Health, Hackensack, NJ, USA.

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