Browsing by Author "Telesca, S"

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    Musculoskeletal manifestations in children with Behcet's syndrome: data from the AIDA Network Behcet's Syndrome Registry
    Gaggiano, C; Maselli, A; Sfikakis, PP; Laskari, K; Ragab, G; Hegazy, MT; Laymouna, AH; Lopalco, G; Almaghlouth, IA; Asfina, KN; Alahmed, O; Mayrink, HAG; Antonelli, IPD; Cattalini, M; Piga, M; Sota, J; Gentileschi, S; Maggio, MC; Opris-Belinski, D; Hatemi, G; Insalaco, A; Olivieri, AN; Tufan, A; Karadeniz, H; Kardas, RC; La Torre, F; Cardinale, F; Marino, A; Guerriero, S; Ruscitti, P; Tarsia, M; Vitale, A; Caggiano, V; Telesca, S; Iannone, F; Parretti, V; Frassi, M; Aragona, E; Ciccia, F; Wiesik-Szewczyk, E; Ionescu, R; Sahin, A; Akkoç, N; Hinojosa-Azaola, A; Tharwat, S; Hernández-Rodríguez, J; Espinosa, G; Conti, G; Del Giudice, E; Govoni, M; Emmi, G; Fabiani, C; Balistreri, A; Frediani, B; Rigante, D; Cantarini, L
    This study aims to describe musculoskeletal manifestations (MSM) in children with Behcet's syndrome (BS), their association with other disease manifestations, response to therapy, and long-term prognosis. Data were retrieved from the AIDA Network Behcet'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 Behcet'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 (p = 0.46) or the concomitant therapy (p = 0.30 for cDMARDs, p = 1.00 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 (p = 0.014). 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).
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    Development and Implementation of the AIDA International Registry for Patients With VEXAS Syndrome
    Vitale, A; Caggiano, V; Della Casa, F; Hernandez-Rodriguez, J; Frassi, M; Monti, S; Tufan, A; Telesca, S; Conticini, E; Ragab, G; Lopalco, G; Almaghlouth, I; Pereira, RMR; Yildirim, D; Cattalini, M; Marino, A; Giani, T; La Torre, F; Ruscitti, P; Aragona, E; Wiesik-Szewczyk, E; Del Giudice, E; Sfikakis, PP; Govoni, M; Emmi, G; Maggio, MC; Giacomelli, R; Ciccia, F; Conti, G; Ait-Idir, D; Lomater, C; Sabato, V; Piga, M; Sahin, A; Opris-Belinski, D; Ionescu, R; Bartoloni, E; Franceschini, F; Parronchi, P; de Paulis, A; Espinosa, G; Maier, A; Sebastiani, GD; Insalaco, A; Shahram, F; Sfriso, P; Minoia, F; Alessio, M; Makowska, J; Hatemi, G; Akkoc, N; Li Gobbi, F; Gidaro, A; Olivieri, AN; Al-Mayouf, SM; Erten, S; Gentileschi, S; Vasi, I; Tarsia, M; Mahmoud, AAMA; Frediani, B; Alzahrani, MF; Laymouna, AH; Ricci, F; Cardinale, F; Jahnz-Rozyk, K; Tosi, GM; Crisafulli, F; Balistreri, A; Dagostin, MA; Ghanema, M; Gaggiano, C; Sota, J; Di Cola, I; Fabiani, C; Giardini, HAM; Renieri, A; Fabbiani, A; Carrer, A; Bocchia, M; Caroni, F; Rigante, D; Cantarini, L
    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 22(nd), 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.