Literature DB >> 24237762

PFAPA syndrome: clinical characteristics and treatment outcomes in a large single-centre cohort.

Petra Król1, Marek Böhm, Viktor Sula, Petra Dytrych, Rami Katra, Dana Nemcová, Pavla Dolezalová.   

Abstract

OBJECTIVES: This paper aims to describe clinical and laboratory features and disease outcome in a single-centre cohort of patients with PFAPA syndrome (Periodic Fever, Aphtous stomatitis, Pharyngitis, and Adenitis) and to test performance of diagnostic and therapeutic algorithms.
METHODS: Patients fulfilling criteria were selected from the fever clinic population. Prospective follow-up together with recruitment of newly diagnosed patients followed pre-defined guidelines. Diagnostic and therapeutic algorithms and definitions of outcome and therapy response were formulated. Paired blood samples during febrile and afebrile periods were compared.
RESULTS: Out of 176 patients referred for suspected periodic fever 125 children fulfilled criteria. Their age at onset was 23 months, median episode duration 3.5 days at 4-week intervals. Fever was associated with pharyngitis (91%), cervical adenitis (78%) and aphtae (41%). Among therapeutic options, episodic prednisone proved to be the most common first-line treatment. Administered to 77 patients, it reduced symptoms in 94%. Tonsillectomy led to the full symptom resolution in all 18 patients. Forty-six patients reached disease remission.
CONCLUSIONS: Distribution of typical symptoms, response to therapies and disease outcome in a large patient cohort were documented. We offer diagnostic and therapeutic algorithms that have proven effective during this prospective trial. Our findings support the general belief of benign nature of this aetiologically unclear condition, despite proportion of patients having persistent disease for years. Maintenance of normal findings in afebrile intervals, striking response to a single dose of prednisone and normal growth and development together with spontaneous tendency towards prolongation of afebrile intervals are important confirmatory features of PFAPA syndrome.

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Year:  2013        PMID: 24237762

Source DB:  PubMed          Journal:  Clin Exp Rheumatol        ISSN: 0392-856X            Impact factor:   4.473


  12 in total

1.  Physicians' perspectives on the diagnosis and management of periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome.

Authors:  Kalpana Manthiram; Suzanne C Li; Jonathan S Hausmann; Gil Amarilyo; Karyl Barron; Hanna Kim; Simona Nativ; Geraldina Lionetti; Andrew Zeft; Donald Goldsmith; David Kimberlin; Kathryn Edwards; Fatma Dedeoglu; Sivia Lapidus
Journal:  Rheumatol Int       Date:  2017-03-07       Impact factor: 2.631

2.  Unique histologic features of tonsils from patients with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome.

Authors:  Kalpana Manthiram; Hernan Correa; Kelli Boyd; Joseph Roland; Kathryn Edwards
Journal:  Clin Rheumatol       Date:  2017-07-26       Impact factor: 2.980

3.  Periodic fever, aphthous stomatitis, pharyngitis and cervical adenitis syndrome in children: a brief literature review.

Authors:  Cristina Terumy Okamoto; Hanne Lise Chaves; Mateus José Schmitz
Journal:  Rev Paul Pediatr       Date:  2022-06-10

4.  Family History in Periodic Fever, Aphthous Stomatitis, Pharyngitis, Adenitis (PFAPA) Syndrome.

Authors:  Kalpana Manthiram; Emily Nesbitt; Thomas Morgan; Kathryn M Edwards
Journal:  Pediatrics       Date:  2016-09       Impact factor: 7.124

5.  Health-related quality of life in children with PFAPA syndrome.

Authors:  Claire Grimwood; Isabelle Kone-Paut; Maryam Piram; Linda Rossi-Semerano; Véronique Hentgen
Journal:  Orphanet J Rare Dis       Date:  2018-08-09       Impact factor: 4.123

6.  Colchicine Effectiveness and Safety in Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis.

Authors:  Tatjana Welzel; Maren Ellinghaus; Anna L Wildermuth; Norbert Deschner; Susanne M Benseler; Jasmin B Kuemmerle-Deschner
Journal:  Front Pediatr       Date:  2021-11-25       Impact factor: 3.418

7.  Development and implementation of the AIDA International Registry for patients with Periodic Fever, Aphthous stomatitis, Pharyngitis, and cervical Adenitis syndrome.

Authors:  Francesca Della Casa; Antonio Vitale; Marco Cattalini; Francesco La Torre; Giovanna Capozio; Emanuela Del Giudice; Maria Cristina Maggio; Giovanni Conti; Maria Alessio; Benson Ogunjimi; Gaafar Ragab; Giacomo Emmi; Emma Aragona; Teresa Giani; Giuseppe Lopalco; Paola Parronchi; Farhad Shahram; Elena Verrecchia; Francesca Ricci; Fabio Cardinale; Silvia Di Noi; Rossana Nuzzolese; Riccardo Lubrano; Serena Patroniti; Roberta Naddei; Vito Sabato; Mohamed A Hussein; Laura Dotta; Violetta Mastrorilli; Stefano Gentileschi; Abdurrahman Tufan; Valeria Caggiano; Mohamed Tharwat Hegazy; Jurgen Sota; Ibrahim A Almaghlouth; Amr Ibrahim; Ewa Wiȩsik-Szewczyk; Burcugul Ozkiziltas; Salvatore Grosso; Micol Frassi; Maria Tarsia; Rosa Maria R Pereira; Maged Taymour; Carla Gaggiano; Sergio Colella; Claudia Fabiani; Maria Morrone; Piero Ruscitti; Bruno Frediani; Veronica Spedicato; Henrique A Mayrink Giardini; Alberto Balistreri; Donato Rigante; Luca Cantarini
Journal:  Front Pediatr       Date:  2022-07-22       Impact factor: 3.569

Review 8.  New insights on multigenic autoinflammatory diseases.

Authors:  Petros Efthimiou; Olga Petryna; Priscila Nakasato; Apostolos Kontzias
Journal:  Ther Adv Musculoskelet Dis       Date:  2022-09-03       Impact factor: 3.625

9.  The role of tonsillectomy in the Periodic Fever, Aphthous stomatitis, Pharyngitis and cervical Adenitis syndrome; a literature review.

Authors:  Jostein Førsvoll; Knut Øymar
Journal:  BMC Ear Nose Throat Disord       Date:  2018-02-22

10.  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.

Authors:  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
Journal:  Pediatr Rheumatol Online J       Date:  2020-04-15       Impact factor: 3.054

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