Development and Implementation of the AIDA International Registry for Patients With Still's Disease

Abstract

ObjectiveAim of this paper is to present the design, construction, and modalities of dissemination of the AutoInflammatory Disease Alliance (AIDA) International Registry for patients with systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD), which are the pediatric and adult forms of the same autoinflammatory disorder.MethodsThis Registry is a clinical, physician-driven, population- and electronic-based instrument implemented for the retrospective and prospective collection of real-world data. The collection of data is based on the Research Electronic Data Capture (REDCap) tool and is intended to obtain evidence drawn from routine patients' management. The collection of standardized data is thought to bring knowledge about real-life clinical research and potentially communicate with other existing and future Registries dedicated to Still's disease. Moreover, it has been conceived to be flexible enough to easily change according to future scientific acquisitions.ResultsStarting from June 30th to February 7th, 2022, 110 Centers from 23 Countries in 4 continents have been involved. Fifty-four of these have already obtained the approval from their local Ethics Committees. Currently, the platform counts 290 users (111 Principal Investigators, 175 Site Investigators, 2 Lead Investigators, and 2 data managers). The Registry collects baseline and follow-up data using 4449 fields organized into 14 instruments, including patient's demographics, history, clinical manifestations and symptoms, trigger/risk factors, therapies and healthcare access.ConclusionsThis international Registry for patients with Still's disease will allow a robust clinical research through collection of standardized data, international consultation, dissemination of knowledge, and implementation of observational studies based on wide cohorts of patients followed-up for very long periods. Solid evidence drawn from “real-life” data represents the ultimate goal of this Registry, which has been implemented to significantly improve the overall management of patients with Still's disease. NCT 05200715 available at https://clinicaltrials.gov/.

Development and Implementation of the AIDA International Registry for Patients With VEXAS Syndrome

“…Regardless of the real impact that this disease has in the population, VEXAS is to be considered a rare disease at present. Therefore, as for other autoinflammatory diseases ( 6 – 8 ), we have developed and launched a Registry capable of gathering the few real-life data available worldwide. While waiting for randomized controlled clinical trials, which are likely to take many years for their conduction, a registry dedicated to VEXAS syndrome can lead to the rapid collection of real-life data from a sufficiently large number of patients.…”

“…As for other AIDA registries ( 6 – 8 ), the assessment of the socioeconomic influence of the disease on the national healthcare systems, on patients' social role and job impact represent an intriguing subject of analysis. Other objectives will be identified based on the challenges that clinical practice and scientific research will bring forward in the coming years.…”

“…Regarding autoinflammatory diseases, the AutoInflammatory Disease Alliance (AIDA) has already developed and launched eight international registries for patients with many rare autoinflammatory diseases ( 6 – 8 ). The AIDA Project has already allowed the construction of an international Network of physicians and researchers interested in putting together information to expand current evidence about monogenic autoinflammatory diseases, Still's disease, Schnitzler's syndrome, Behçet's disease, periodic fever, aphthous stomatitis, pharyngitis, cervical adenitis (PFAPA) syndrome, non-infectious uveitis, non-infectious scleritis, and undifferentiated systemic autoinflammatory diseases (USAIDs).…”

Development and implementation of the AIDA international registry for patients with Schnitzler's syndrome

“…The Registry for Schnitzler's syndrome is classified as a clinical-, physician-driven, population- and electronic-based registry; it was developed along with other autoinflammatory disease-specific registries in the context of AIDA network ( 7 – 9 ).…”

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

“…In this context, the PFAPA syndrome has been included by the AutoInflammatory Disease Alliance (AIDA) among autoinflammatory disorders to be better investigated through a dedicated International Registry. Indeed, this registry goes to add other already developed and activated registries dedicated to other autoinflammatory diseases, as non-infectious uveitis, non-infectious scleritis, Behçet's disease, monogenic autoinflammatory diseases, Still's disease, Schnitzler's syndrome, vacuoles/E1 enzyme/X-linked autoinflammatory, somatic (or VEXAS) syndrome and undifferentiated systemic autoinflammatory diseases (i.e., USAIDs) (18)(19)(20).…”