Dataset Information

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

ABSTRACT:

Objective

The aim of this paper is to present the AutoInflammatory Disease Alliance (AIDA) international Registry dedicated to Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) syndrome, describing its design, construction, and modalities of dissemination.

Methods

This Registry is a clinical, physician-driven, population- and electronic-based instrument designed for the retrospective and prospective collection of real-life data. Data gathering is based on the Research Electronic Data Capture (REDCap) tool and is intended to obtain real-world evidence for daily patients' management. The Registry may potentially communicate with other on-line tools dedicated to VEXAS syndrome, thus enhancing international collaboration and data sharing for research purposes. The Registry is practical enough to be easily modified to meet future needs regarding VEXAS syndrome.

Results

To date (April 22nd, 2022), 113 Centers from 23 Countries in 4 continents have been involved; 324 users (114 Principal Investigators, 205 Site Investigators, 2 Lead Investigators, and 3 data managers) are currently able to access the registry for data entry (or data sharing) and collection. The Registry includes 4,952 fields organized into 18 instruments designed to fully describe patient's details about demographics, clinical manifestations, symptoms, histologic details about skin and bone marrow biopsies and aspirate, laboratory features, complications, comorbidities, therapies, and healthcare access.

Conclusion

This international Registry for patients with VEXAS syndrome will allow the achievement of a comprehensive knowledge about this new disease, with the final goal to obtain real-world evidence for daily clinical practice, especially in relation to the comprehension of this disease about the natural history and the possible therapeutic approaches. This Project can be found on https://clinicaltrials.gov NCT05200715.

SUBMITTER: Vitale A

PROVIDER: S-EPMC9309690 | biostudies-literature |

REPOSITORIES: biostudies-literature

ACCESS DATA

Similar Datasets

Project description:ObjectiveThis paper points out the design, development and deployment of the AutoInflammatory Disease Alliance (AIDA) International Registry dedicated to pediatric and adult patients affected by Undifferentiated Systemic AutoInflammatory Diseases (USAIDs).MethodsThis is an electronic registry employed for real-world data collection about demographics, clinical, laboratory, instrumental and socioeconomic data of USAIDs patients. Data recruitment, based on the Research Electronic Data Capture (REDCap) tool, is designed to obtain standardized information for real-life research. The instrument is endowed with flexibility, and it could change over time according to the scientific acquisitions and potentially communicate with other similar tools; this platform ensures security, data quality and data governance.ResultsThe focus of the AIDA project is connecting physicians and researchers from all over the world to shed a new light on heterogeneous rare diseases. Since its birth, 110 centers from 23 countries and 4 continents have joined the AIDA project. Fifty-four centers have already obtained the approval from their local Ethics Committees. Currently, the platform counts 290 users (111 Principal Investigators, 179 Site Investigators, 2 Lead Investigators, and 2 data managers). The Registry is collecting baseline and follow-up data using 3,769 fields organized into 23 instruments, which include demographics, history, symptoms, trigger/risk factors, therapies, and healthcare information access for USAIDs patients.ConclusionsThe development of the AIDA International Registry for USAIDs patients will facilitate the online collection of real standardized data, connecting a worldwide group of researchers: the Registry constitutes an international multicentre observational groundwork aimed at increasing the patient cohort of USAIDs in order to improve our knowledge of this peculiar cluster of autoinflammatory diseases. NCT05200715 available at https://clinicaltrials.gov/.

Project description:Background Patients with Hemolytic Uremic Syndrome (HUS) face late diagnosis and lack of appropriate treatment because of a lack of knowledge and experience in this field. A prerequisite for such knowledge is the development of research infrastructures such as a registry system. Therefore, this study aimed to develop and describe the HUS registry in accordance with the Iranian health system and implement its software system. Methods We first interviewed 10 pediatric nephrologists and after analyzing the interviews, we identified the features and requirements and the data related to HUS. Then, during two rounds of the Delphi technique (the first round with 23 participants and the second round with 18 participants), the model of this registry was finalized based on the agreement of at least 75% of specialists. At the next step, based on the agreed requirements, IRI.HUS.Reg (Iranian Hemolytic Uremic Syndrome Registry) software was developed and implemented in a pediatric hospital. Results We classified 369 meaning units of interviews in 41 codes and 7 final themes including purposes of the registry (10 codes), inclusion criteria (7 codes), data collection method (4 codes), data quality control (6 codes), data sources (4 codes), data analysis (3 codes) and software features (7 codes). These 7 feature groups (67 subgroups) and 12 data classes (138 data elements) include demographic data, referrals, examinations, clinical signs, causes, laboratory tests, medical histories, paraclinical measures, treatments, outcomes, patient’s status at discharge, and follow-up data were reviewed by the Delphi panelists, and finally, 64 features and 131 data elements were accepted by at least 78% agreement. Then, we developed and implemented a registry software system in a hospital. Conclusion We implemented IRI.HUS.Reg based on related features, 12 data classes agreed by specialists, literature review, and comparison with other existing registries. Therefore, the data collected in this registry can be compared with other data from existing registries in other countries. Supplementary Information The online version contains supplementary material available at 10.1186/s13023-022-02376-9.