7 research outputs found
Development and Implementation of the AIDA International Registry for Patients with Non-Infectious Uveitis
Introduction: The aim of this paper is to point out the design, development and deployment of the AutoInflammatory Disease Alliance (AIDA) International Registry for paediatric and adult patients with non-infectious uveitis (NIU). Methods: This is a physician-driven, population- and electronic-based registry implemented for both retrospective and prospective collection of real-world demographics, clinical, laboratory, instrumental and socioeconomic data of patients with uveitis and other non-infectious inflammatory ocular diseases recruited through the AIDA Network. Data recruitment, based on the Research Electronic Data Capture (REDCap) tool, is thought to collect standardised information for real-life research and has been developed to change over time according to future scientific acquisitions and potentially communicate with other similar instruments. Security, data quality and data governance are cornerstones of this platform. Results: Ninety-five centres have been involved from 19 countries and four continents from 24 March to 16 November 2021. Forty-eight out of 95 have already obtained the approval from their local ethics committees. At present, the platform counts 259 users (95 principal investigators, 160 site investigators, 2 lead investigators, and 2 data managers). The AIDA Registry collects baseline and follow-up data using 3943 fields organised into 13 instruments, including patient's demographics, history, symptoms, trigger/risk factors, therapies and healthcare utilization for patients with NIU. Conclusions: The development of the AIDA Registry for patients with NIU will facilitate the collection of standardised data leading to real-world evidence and enabling international multicentre collaborative research through inclusion of patients and their families worldwide
Musculoskeletal manifestations in children with Behçet's syndrome: data from the AIDA Network Behçet's Syndrome Registry
This study aims to describe musculoskeletal manifestations (MSM) in children with Behçet's syndrome (BS), their association with other disease manifestations, response to therapy, and long-term prognosis. Data were retrieved from the AIDA Network Behçet's Syndrome Registry. Out of a total of 141 patients with juvenile BS, 37 had MSM at disease onset (26.2%). The median age at onset was 10.0 years (IQR 7.7). The median follow-up duration was 21.8 years (IQR 23.3). Recurrent oral (100%) and genital ulcers (67.6%) and pseudofolliculitis (56.8%) were the most common symptoms associated with MSM. At disease onset, 31 subjects had arthritis (83.8%), 33 arthralgia (89.2%), and 14 myalgia (37.8%). Arthritis was monoarticular in 9/31 cases (29%), oligoarticular in 10 (32.3%), polyarticular in 5 (16.1%), axial in 7 (22.6%). Over time, arthritis became chronic-recurrent in 67.7% of cases and 7/31 patients had joint erosions (22.6%). The median Behçet's Syndrome Overall Damage Index was 0 (range 0-4). Colchicine was inefficacious for MSM in 4/14 cases (28.6%), independently from the type of MSM (pâ=â0.46) or the concomitant therapy (pâ=â0.30 for cDMARDs, pâ=â1.00 for glucocorticoids); cDMARDs and bDMARDs were inefficacious for MSM in 6/19 (31.4%) and 5/12 (41.7%) cases. The presence of myalgia was associated with bDMARDs inefficacy (pâ=â0.014). To conclude, MSM in children with BS are frequently associated with recurrent ulcers and pseudofolliculitis. Arthritis is mostly mono- or oligoarticular, but sacroiliitis is not unusual. Prognosis of this subset of BS is overall favorable, though the presence of myalgia negatively affects response to biologic therapies. ClinicalTrials.gov Identifier: NCT05200715 (registered on December 18, 2021)
A patient-driven registry on Behçetâs disease: the AIDA for patients pilot project
IntroductionThis paper describes the creation and preliminary results of a patient-driven registry for the collection of patient-reported outcomes (PROs) and patient-reported experiences (PREs) in Behçetâs disease (BD).MethodsThe project was coordinated by the University of Siena and the Italian patient advocacy organization SIMBA (Associazione Italiana Sindrome e Malattia di Behçet), in the context of the AIDA (AutoInflammatory Diseases Alliance) Network programme. Quality of life, fatigue, socioeconomic impact of the disease and therapeutic adherence were selected as core domains to include in the registry.ResultsRespondents were reached via SIMBA communication channels in 167 cases (83.5%) and the AIDA Network affiliated clinical centers in 33 cases (16.5%). The median value of the Behçetâs Disease Quality of Life (BDQoL) score was 14 (IQR 11, range 0â30), indicating a medium quality of life, and the median Global Fatigue Index (GFI) was 38.7 (IQR 10.9, range 1â50), expressing a significant level of fatigue. The mean Beliefs about Medicines Questionnaire (BMQ) necessity-concern differential was 0.9â±â1.1 (range â 1.8â4), showing that the registry participants prioritized necessity belief over concerns to a limited extent. As for the socioeconomic impact of BD, in 104 out of 187 cases (55.6%), patients had to pay from their own pocket for medical exams required to reach the diagnosis. The low family socioeconomic status (pâ<â0.001), the presence of any major organ involvement (pâ<â0.031), the presence of gastro-intestinal (pâ<â0.001), neurological (pâ=â0.012) and musculoskeletal (pâ=â0.022) symptoms, recurrent fever (pâ=â0.002), and headache (pâ<â0.001) were associated to a higher number of accesses to the healthcare system. Multiple linear regression showed that the BDQoL score could significantly predict the global socioeconomic impact of BD (Fâ=â14.519, OR 1.162 [CI 0.557â1.766], pâ<â0.001).DiscussionPreliminary results from the AIDA for Patients BD registry were consistent with data available in the literature, confirming that PROs and PREs could be easily provided by the patient remotely to integrate physician-driven registries with complementary and reliable information
Efficacy and safety of tocilizumab in adult-onset Still's disease: Real-life experience from the international AIDA registry
Long-term efficacy and safety of tocilizumab (TCZ) in adult-onset Still's disease (AOSD) mostly derive from small case series. Herein we report a registry-based study investigating TCZ efficacy and safety in a cohort of patients with AOSD evaluated by clinical and serum inflammatory markers as well as drug retention rate analysis
The Autoinflammatory Diseases Alliance Registry of monogenic autoinflammatory diseases
The present manuscript aims to describe an international, electronic-based, user-friendly and interoperable patient registry for monogenic autoinflammatory diseases (mAIDs), developed in the contest of the Autoinflammatory Diseases Alliance (AIDA) Network
Development and Implementation of the AIDA International Registry for Patients with Non-Infectious Uveitis
Introduction The aim of this paper is to point out the design,
development and deployment of the AutoInflammatory Disease Alliance
(AIDA) International Registry for paediatric and adult patients with
non-infectious uveitis (NIU). Methods This is a physician-driven,
population- and electronic-based registry implemented for both
retrospective and prospective collection of real-world demographics,
clinical, laboratory, instrumental and socioeconomic data of patients
with uveitis and other non-infectious inflammatory ocular diseases
recruited through the AIDA Network. Data recruitment, based on the
Research Electronic Data Capture (REDCap) tool, is thought to collect
standardised information for real-life research and has been developed
to change over time according to future scientific acquisitions and
potentially communicate with other similar instruments. Security, data
quality and data governance are cornerstones of this platform. Results
Ninety-five centres have been involved from 19 countries and four
continents from 24 March to 16 November 2021. Forty-eight out of 95 have
already obtained the approval from their local ethics committees. At
present, the platform counts 259 users (95 principal investigators, 160
site investigators, 2 lead investigators, and 2 data managers). The AIDA
Registry collects baseline and follow-up data using 3943 fields
organised into 13 instruments, including patient's demographics,
history, symptoms, trigger/risk factors, therapies and healthcare
utilization for patients with NIU. Conclusions The development of the
AIDA Registry for patients with NIU will facilitate the collection of
standardised data leading to real-world evidence and enabling
international multicentre collaborative research through inclusion of
patients and their families worldwide