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

Abstract

ObjectiveAim of this paper is to illustrate the methodology, design, and development of the AutoInflammatory Disease Alliance (AIDA) International Registry dedicated to patients with the Periodic Fever, Aphthous stomatitis, Pharyngitis, and cervical Adenitis (PFAPA) syndrome.MethodsThis is a physician-driven, non-population- and electronic-based registry proposed to gather real-world demographics, clinical, laboratory, instrumental and socioeconomic data from PFAPA patients. Data recruitment is realized through the on-line Research Electronic Data Capture (REDCap) tool. This registry is thought to collect standardized information for clinical research leading to solid real-life evidence. The international scope and the flexibility of the registry will facilitate the realization of cutting-edge study projects through the constant updating of variables and the possible merging and transfer of data between current and future PFAPA registries.ResultsA total of 112 centers have already been involved from 23 countries and 4 continents starting from August 24th, 2021, to April 6th, 2022. In total 56/112 have already obtained the formal approval from their local Ethics Committees. The platform counts 321 users (113 principal investigators, 203 site investigators, two lead investigators, and three data managers). The registry collects retrospective and prospective data using 3,856 fields organized into 25 instruments, including PFAPA patient's demographics, medical histories, symptoms, triggers/risk factors, therapies, and impact on the healthcare systems.ConclusionsThe development of the AIDA International Registry for PFAPA patients will enable the on-line collection of standardized data prompting real-life studies through the connection of worldwide groups of physicians and researchers. This project can be found on https://clinicaltrials.gov NCT 05200715.

Increase in pediatric recurrent fever evaluations during the first year of the COVID-19 pandemic in North America

“…Nonetheless, strengths of our study include the use of an aggregate of ICD-10 codes (including but not limited to PFAPA), which allowed for a large number of patients evaluated for rare diseases to be studied at many North American pediatric academic centers. While we did not develop or utilize an autoinflammatory disease registry in this CARRA study, its nature supports previously noted benefits of access to registries for generating new data to study these rare diseases and optimize use of global healthcare systems to better care for patients in the future (24,25). Second, this project demonstrated the CARRA PFAPA/AID Working Group's ability to assemble a large network to answer emerging clinical questions using aggregate patient data.…”

Preliminary data revealing efficacy of Streptococcus salivarius K12 (SSK12) in Periodic Fever, Aphthous stomatitis, Pharyngitis, and cervical Adenitis (PFAPA) syndrome: A multicenter study from the AIDA Network PFAPA syndrome registry

“…We examined the medical charts of 85 PFAPA patients (49 males, 36 females) receiving SSK12 in the period September 2017–May 2022. This cohort was selected among 233 PFAPA patients enrolled in the AIDA registry ( 13 ). Figure 1 shows the flow-diagram of the selection process of eligible patients, forming the final cohort of participants.…”

“…The cohort was selected among 233 patients with PFAPA syndrome enrolled in the AIDA International Registry, a physician-driven electronic-based platform designed for both retrospective and prospective collection of data. Solid evidence drawn from the collection of “real-life” standardized data represents the main goal of the Registry, that will hopefully lead to an overall improvement of the disease management ( 13 ). Patients with insufficient follow-up data or missing baseline values were excluded from the final cohort as well as patients with a follow-up shorter than 3 months.…”