Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Cervical Adenitis (PFAPA) Management Across Countries: A Survey to Inform Future Comparative Effectiveness Research

Mehmet YILDIZ¹, Samira Nazzar Romero², Lakshmi Moorthy³, Edan Itzkovitz⁴, Halide Ozge Basaran⁵, Tara Lozy⁶, Patricia DeLaMora⁷, Tatjana Welzel⁸, Ruby Haviv⁹, Kalpana Manthiram¹⁰, Camilo Vargas¹¹, Erdal Sag¹², amit ziv¹³, Adi Pappo¹⁴, Özlem Satirer¹⁵, Maryam Piram¹⁶, Gülşah Kavrul Kayaalp¹⁷, Nuray Aktay Ayaz¹⁸, Antonella Insalaco¹⁹, Jasmin Kuemmerle-Deschner²⁰, Dilan Dissanayake⁴, Kader Cetin Gedik²¹, Nadine Saad²², Nataša Toplak²³, Ozgur Kasapcopur²⁴, Marinka Twilt²⁵, Isabelle koné-Paut²⁶, Ronald M. Laxer²⁷, Marco Gattorno²⁸, Seza zen²⁹, Fatma Dedeoglu³⁰ and Sivia Lapidus³¹, ¹Division of Pediatric Rheumatology, Cerrahpasa Medical Faculty, Istanbul University-Cerrahpasa, Istanbul, Turkey, Istanbul, Turkey, ²Division of Rheumatology, Nemours Children's Health, University of Central Florida College of Medicine, Orlando, United States, Orlando, FL, ³Department of Pediatrics, Rutgers-Robert Wood Johnson Medical School, New Brunswick, NJ, United States, Metuchen, NJ, ⁴Division of Rheumatology, Department of Paediatrics, The Hospital for Sick Children, University of Toronto, Toronto, Canada, Toronto, ON, Canada, ⁵Department of Pediatrics, Hacettepe University, Ankara, Türkiye, New York City, NY, ⁶Joseph M. Sanzari Children's Hospital, Hackensack Meridian Health, Center for Discovery and Innovation, Hackensack Meridian Health and Hackensack Meridian School of Medicine, ⁷Department of Pediatrics, School of Medicine, New York Medical College, Valhalla, United States, ⁸Pediatric Rheumatology, University Children's Hospital Basel (UKBB), University of Basel, Basel, Switzerland, ⁹Pediatric Rheumatology Unit, Department of Pediatrics, Meir Medical Center, Kfar Saba, Israel., ¹⁰Laboratory of Immune System Biology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health, Bethesda, United States, Bethesda, MD, ¹¹Faculty of Medicine, Universidad del Valle, Cali, Colombia, Cali, Colombia, ¹²Department of Pediatric Rheumatology, Hacettepe University Faculty of Medicine, Ankara, Turkey, and Division, Ankara, Turkey, ¹³Pediatric Rheumatology Unit, Department of Pediatrics, Meir Medical Center, Kfar Saba, Israel, herzelia, Israel, ¹⁴Pediatric Rheumatology Unit, Schneider Children's Medical Center of Israel, Petach Tikva, Israel, HaMerkaz, Israel, ¹⁵Department of Paediatrics and Autoinflammation reference Center Tuebingen (arcT), Universitatsklinikum Tubingen, Tubingen, Baden-Württemberg, Germany, ¹⁶Department of Pediatrics, CHU Sainte-Justine, Université de Montréal, Montreal, QC, Canada; CHU Sainte-Justine Research Center, Université de Montréal, Montreal, QC, Canada, Montral, Canada, ¹⁷Department of Pediatric Rheumatology, Istanbul Faculty of Medicine, Istanbul University, Istanbul, Turkey, ¹⁸Department of Pediatric Rheumatology, Istanbul Faculty of Medicine, Istanbul University, Istanbul, Turkey, Istanbul, Turkey, ¹⁹Division of Rheumatology, ERN RITA Center, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy, Rome, Italy, ²⁰Department of Paediatrics and Autoinflammation reference Center Tuebingen (arcT), Universitatsklinikum Tubingen, Tubingen, Baden-Württemberg, Germany, Tuebingen, Germany, ²¹Division of Pediatric Rheumatology, Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, USA; Pediatric Autoinflammatory Diseases Center, University of Pittsburgh, Pittsburgh, United States, Pittsburgh, PA, ²²University of Michigan, Michigan Medicine, Ann Arbor, Michigan, United States, Ann Arbor, MI, ²³Department of Allergology, Rheumatology and Clinical Immunology, University Children's Hospital, Ljubljana, Slovenia, ²⁴Istanbul University-Cerrahpasa, Department of Pediatric Rheumatology, Istanbul, Türkiye, istanbul, Turkey, ²⁵Alberta Children's Hospital, Calgary, AB, Canada, ²⁶Department of Pediatrics, Kremlin-Bicêtre University Hospital, Paris-Saclay University, Paris, France, ²⁷The Hospital for Sick Children, Toronto, ON, Canada, ²⁸Division of Rheumatology and Autoinflammatory Diseases, IRCCS Istituto Giannina Gaslini, Genoa, Italy, Genova, Italy, ²⁹Hacettepe University Medical Faculty, Ankara, Turkey, ³⁰Boston Children's Hospital, Boston, MA, ³¹Joseph M. Sanzari Children's Hospital, Hackensack Meridian Health, Center for Discovery and Innovation, Hackensack Meridian Health and Hackensack Meridian School of Medicine, Montclair, NJ

Meeting: 2026 Pediatric Rheumatology Symposium

Session Information

Date: Thursday, March 19, 2026

Title: Posters: Clinical and Therapeutic Aspects I

Session Time: 6:00PM-7:00PM

Background/Purpose: Background: PFAPA is the most common autoinflammatory condition of childhood. Its frequency, clinical features, and response to treatments vary across populations. To standardize care, the Childhood Arthritis and Rheumatology Research Alliance (CARRA) published consensus treatment plans in 2020. However, real-world evidence of their effectiveness is limited.
Purpose: To assess current treatment practices for PFAPA across international centers and estimate potential enrollment for future comparative effectiveness studies.

Methods: The PFAPA Evaluation of Treatment Algorithms Study (PETALS) consortium conducted a survey (Feb–Mar 2025) across centers in Europe and the Americas. Each center provided estimated numbers of newly diagnosed and follow-up PFAPA patients, as well as estimates of the percentage of patients on each of the treatment options. Based on the information provided by the centers we calculated the number of patients that we would potentially have in each treatment option. The estimated percentage of patients on each treatment option was grouped into three categories: low (0-30% of patients), moderate (31-60% of patients), and high (61-100% of patients). The Cochran-Armitage test was used to compare the proportion of treatment strategies in the Americas and Europe.

Results: Twenty centers participated in the survey. The number of newly diagnosed and follow up PFAPA patients varied considerably across centers, with a balanced distribution between Europe and the Americas. (Figure 1). Figure 2 illustrates the estimated number of PFAPA patients who would be eligible for each treatment option within one year across participating centers.
For antipyretics 20% of the centers in the Europe reported high use of antipyretics and 40% reported moderate use. (Figure 3) One hundred percent of the centers in the Americas report low use of acetaminophen (p 0.01). Cimetidine is used moderately in 13% of the participating centers from Americas, otherwise there is low use reported by the rest of the cohort. Colchicine use is similar in the center in the Americas and Europe with 1/3 of the centers from both geographical areas reporting moderate use and 2/3 with low use. Steroids are used in at least 31% of patients in all the centers in the Americas and in 60% of European sites. Tonsillectomy with and without adenoidectomy was used more frequently in American centers (p 0.02).

Conclusion: Our survey demonstrated adequate patient availability for most of the treatment options. Steroids and colchicine are the most used strategies, while cimetidine and tonsillotomy are less used across the board. There is a systematic difference in the proportion of treatment usage for antipyretics and for tonsillectomy with or without adenoidectomy, with these options being more frequently used in Europe and in the Americas, respectively. No significant differences were found in the other treatment preferences. These findings support the feasibility of conducting comparative effectiveness studies and highlight the importance of international collaboration.

Figure 1. Newly diagnosed and established PFAPA cases in the last 12 months

Figure 2. Estimated number of patients for each treatment

Figure 3. Utilization of the management options for PFAPA in the Americas and Europe

Disclosures: M. YILDIZ: None; S. Nazzar Romero: None; L. Moorthy: None; E. Itzkovitz: None; H. Basaran: None; T. Lozy: Eli Lilly, 11; P. DeLaMora: None; T. Welzel: None; R. Haviv: None; K. Manthiram: None; C. Vargas: None; E. Sag: None; a. ziv: None; A. Pappo: None; Ö. Satirer: None; M. Piram: Novartis, 6, 12, Conference sponsor, Pfizer, 12, Event sponsor and conference grant; G. Kavrul Kayaalp: None; N. Aktay Ayaz: None; A. Insalaco: None; J. Kuemmerle-Deschner: Novartis, 1, 5, 6, Sobi, 5, 6; D. Dissanayake: None; K. Cetin Gedik: None; N. Saad: None; N. Toplak: None; O. Kasapcopur: None; M. Twilt: None; I. koné-Paut: Amgen, 2, CHUGAI, 2, 11, frezenius Kabi, 2, Novartis, 2, PATENT VALOR, 10, Pfizer, 6, SOBI, 2; R. Laxer: Akros pharma, 2, Celltrion, 2, Eli Lilly, 2, Novartis, 2, Sanofi-Aventis, 1, 2, UpToDate, 9; M. Gattorno: Fresinus-Kabi, 6, Novartis, 6, SOBI, 6; S. Özen: Novartis, 2, 6, Sobi, 2, 6; F. Dedeoglu: Sobi, 6, UptoDate, 9; S. Lapidus: None.

To cite this abstract in AMA style:

YILDIZ M, Nazzar Romero S, Moorthy L, Itzkovitz E, Basaran H, Lozy T, DeLaMora P, Welzel T, Haviv R, Manthiram K, Vargas C, Sag E, ziv a, Pappo A, Satirer Ö, Piram M, Kavrul Kayaalp G, Aktay Ayaz N, Insalaco A, Kuemmerle-Deschner J, Dissanayake D, Cetin Gedik K, Saad N, Toplak N, Kasapcopur O, Twilt M, koné-Paut I, Laxer R, Gattorno M, Özen S, Dedeoglu F, Lapidus S. Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Cervical Adenitis (PFAPA) Management Across Countries: A Survey to Inform Future Comparative Effectiveness Research [abstract]. Arthritis Rheumatol. 2026; 78 (suppl 3). https://acrabstracts.org/abstract/periodic-fever-aphthous-stomatitis-pharyngitis-and-cervical-adenitis-pfapa-management-across-countries-a-survey-to-inform-future-comparative-effectiveness-research/. Accessed .

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