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
Posted on 2020-04-16 - 03:51
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.
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Amarilyo, Gil; Rothman, Deborah; Manthiram, Kalpana; Edwards, Kathryn M.; Li, Suzanne C.; Marshall, Gary S.; et al. (2020). 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. figshare. Collection. https://doi.org/10.6084/m9.figshare.c.4940046.v1
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AUTHORS (28)
GA
Gil Amarilyo
DR
Deborah Rothman
KM
Kalpana Manthiram
KE
Kathryn M. Edwards
SL
Suzanne C. Li
GM
Gary S. Marshall
CY
Cagri Yildirim-Toruner
KH
Kathleen Haines
PF
Polly J. Ferguson
GL
Geraldina Lionetti
JC
Julie Cherian
YZ
Yongdong Zhao
PD
Patricia DeLaMora
GS
Grant Syverson
SN
Simona Nativ
MT
Marinka Twilt
IM
Ian C. Michelow
YS
Yuriy Stepanovskiy
AT
Akaluck Thatayatikom
LH
Liora Harel