Search

Objective: Dry eye disease (DED) is a condition associated with a myriad of systemic disorders. According to recent preliminary data, axial spondylarthritis (axial-SpA) could represent a new entity associated with DED. Therefore, this study aimed to assess DED in patients with axial SpA by performing quantitative and qualitative specific tests to investigate the potential association between DED and ocular surface damage in patients with axial-SpA and to identify potential variables associated with DED., Methods: A total of 71 patients with axial-SpA who fulfilled the Assessment of SpondyloArthritis International Society (ASAS) classification criteria and 19 healthy controls were enrolled in this study. Both the patients and the controls underwent a complete ocular assessment aimed at evaluating the tear film and ocular surface, which included the Schirmer test, tear break-up time (TBUT), fluorescein staining, and lissamine green staining. The Ocular Surface Disease Index (OSDI) questionnaire was administered to all patients., Results: DED symptoms were reported in 46 (64.8%) patients and three (15.8%) healthy controls ( p = 0.0004). The odds ratio for receiving a diagnosis of axial-SpA based on the presence of dry-eye-related symptoms was 9.2 (95% C.I. 2.72-42.52, p = 0.001). The Schirmer test values of < 6 mm/5 min were observed in 31 (43.7%) patients with axial-SpA and two (10.5%) healthy controls ( p = 0.013); a TBUT of <5 s was observed in 34 (47.9%) patients with axial-SpA and six (31.6%) healthy controls. The median OSDI score was found to be 22.9 (IQR = 29.35) among the patients with axial-SpA and 0.0 (IQR = 4.69) among the healthy controls ( p = 0.009). The fluorescein and lissamine green staining of the ocular surface indicated a significantly higher Oxford Grading Scale in the patients with axial-SpA than in the healthy controls., Conclusion: Patients with axial-SpA often complain of eye dryness, which may be quantified with the self-administered OSDI questionnaire and objectively assessed through the tests commonly used for the diagnosis of DED. Patients suspected of having axial-SpA should routinely be asked about dry eye symptoms and evaluated for potential corneal and conjunctival damage., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. The author(s) declared that they were an editorial board member of Frontiers, at the time of submission. This had no impact on the peer review process and the final decision., (Copyright © 2024 Vitale, Caggiano, Martín-Nares, Di Meglio, Sica, Hinojosa-Azaola, Perfetti, Pagliara, Guidetti, Fonollosa, Lopez, Sbalchiero, Sota, Carreño, Kawakami-Campos, Gentileschi, de-la-Torre, Tosi, Mazzei, Balistreri, Frediani, Cantarini and Fabiani.)

Introduction: Non-infectious inflammatory ocular diseases pose significant challenges in diagnosis and management, often requiring systemic immunosuppressive therapy. Since Janus kinase (JAK) inhibitors may represent a novel therapeutic option for these disorders, the present study aimed to expand current knowledge about their efficacy and safety in patients with these conditions., Methods: This prospective cohort study included 12 adult patients from the international AutoInflammatory Disease Alliance (AIDA) Network registries dedicated to non-infectious ocular inflammatory conditions. We assessed ocular flares, visual acuity, disease course, and complications before and after initiating JAK inhibitor therapy., Results: Ocular inflammation was related to a systemic disease in 8 (66.7%) patients as follows: spondyloarthritis ( n = 3), peripheral psoriatic arthritis ( n = 1), rheumatoid arthritis ( n = 1), antinuclear antibodies (ANA) positive juvenile idiopathic arthritis ( n = 1), Behçet's syndrome ( n = 1), Vogt-Koyanagi-Harada syndrome ( n = 1). In total, 4 patients received baricitinib, 1 patient received tofacitinib, and 7 patients underwent upadacitinib treatment. The overall average duration of JAK inhibitors treatment was 8.6 ± 5.5 months (ranging from 3 to 20 months). At the last assessment, ocular disease control was complete in 12/12 patients. One patient discontinued baricitinib due to poor compliance after a 12-month relapse-free period. The incidence of ocular flares was 125 episodes/1.000 person-months prior to the initiation of JAK inhibitors and 28.6 episodes/1.000 person-months thereafter. The incidence rate ratio for experiencing a relapse before starting a JAK inhibitor compared to the following period was 4.37 (95% CI 1.3-14.7, p -value: 0.02)., Conclusion: JAK inhibitors demonstrate efficacy and safety in controlling ocular inflammatory relapses, confirming that they represent a valuable treatment option for patients with non-infectious inflammatory ocular diseases resistant to conventional treatments., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. The authors declared that they were an editorial board member of Frontiers, at the time of submission. This had no impact on the peer review process and the final decision., (Copyright © 2024 Vitale, Palacios-Olid, Caggiano, Ragab, Hernández-Rodríguez, Pelegrín, Mejía-Salgado, Zarate-Pinzón, Gentileschi, Sota, Fonollosa, Carreño, Gaggiano, Amin, Balistreri, Narváez, Tosi, Frediani, Cantarini, de-la-Torre and Fabiani.)

Objectives: Ultrasound has a paramount role in the diagnostic assessment of giant cell arteritis (GCA); Southend halo score (HS), halo count (HC), and OMERACT GCA Ultrasonography Score (OGUS) are the first quantitative scores proposed in this setting. The aim of this study was therefore to assess the diagnostic accuracy of these scores in a real-life scenario, as well as to evaluate their optimal cutoff, also with respect to disease extent, sex, and age., Methods: We retrospectively collected clinical, serological, and US findings of all patients referred for the first time to our vasculitis clinic in the suspicion of GCA., Results: A total of 79 patients were included, and a definite diagnosis of GCA was made in 43 patients. For OGUS, the ROC curve showed an optimal cut point of 0.81 (sensitivity 79.07% and specificity 97.22%). For HC and HS, the optimal cutoff values were > 1.5 (sensitivity 76.7% and specificity 97.2%) and > 14.5 (sensitivity 74.4% and specificity 97.2%), respectively. No relevant differences were assessed when patients were stratified according to disease extent, age, and sex. Compression sign (CS) was positive in 34 of 38 patients with cranial GCA and negative in all controls and LV-GCA., Conclusion: All three scores display good sensitivity and excellent specificity, although the cutoff was slightly different than proposed. In particular, for OGUS, a threshold of 0.81 could be employed for diagnostic purposes, although it was developed solely for monitoring. Due to its high sensitivity and specificity, CS should be always assessed in all patients referred with a suspicion of cranial GCA., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2024 Conticini, Falsetti, Al Khayyat, Grazzini, Baldi, Bellisai, Gentileschi, Bardelli, Fabiani, Cantarini, Dasgupta and Frediani.)

Introduction: The effectiveness of canakinumab may change according to the different times it is used after Still's disease onset. This study aimed to investigate whether canakinumab (CAN) shows differences in short- and long-term therapeutic outcomes, according to its use as different lines of biologic treatment., Methods: Patients included in this study were retrospectively enrolled from the AutoInflammatory Disease Alliance (AIDA) International Registry dedicated to Still's disease. Seventy-seven (51 females and 26 males) patients with Still's disease were included in the present study. In total, 39 (50.6%) patients underwent CAN as a first-line biologic agent, and the remaining 38 (49.4%) patients were treated with CAN as a second-line biologic agent or subsequent biologic agent., Results: No statistically significant differences were found between patients treated with CAN as a first-line biologic agent and those previously treated with other biologic agents in terms of the frequency of complete response ( p  =0.62), partial response ( p  =0.61), treatment failure ( p  >0.99), and frequency of patients discontinuing CAN due to lack or loss of efficacy ( p  =0.2). Of all the patients, 18 (23.4%) patients experienced disease relapse during canakinumab treatment, 9 patients were treated with canakinumab as a first-line biologic agent, and nine patients were treated with a second-line or subsequent biologic agent. No differences were found in the frequency of glucocorticoid use ( p  =0.34), daily glucocorticoid dosage ( p  =0.47), or concomitant methotrexate dosage ( p  =0.43) at the last assessment during CAN treatment., Conclusion: Canakinumab has proved to be effective in patients with Still's disease, regardless of its line of biologic treatment., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2023 Vitale, Caggiano, Sfikakis, Dagna, Lopalco, Ragab, La Torre, Almaghlouth, Maggio, Sota, Tufan, Hinojosa-Azaola, Iannone, Loconte, Laskari, Direskeneli, Ruscitti, Morrone, Mayrink Giardini, Panagiotopoulos, Di Cola, Martín-Nares, Monti, De Stefano, Kardas, Duran, Campochiaro, Tomelleri, Alabdulkareem, Gaggiano, Tarsia, Bartoloni, Romeo, Hussein, Laymouna, Parente de Brito Antonelli, Dagostin, Fotis, Bindoli, Navarini, Alibaz-Oner, Sevik, Frassi, Ciccia, Iacono, Crisafulli, Portincasa, Jaber, Kawakami-Campos, Wiesik-Szewczyk, Iagnocco, Simonini, Sfriso, Balistreri, Giacomelli, Conti, Frediani, Fabiani and Cantarini.)

Introduction: This 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 Behçet's disease (BD)., Methods: The 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., Results: Respondents 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 Behçet's Disease Quality of Life (BDQoL) score was 14 (IQR 11, range 0-30), indicating a medium quality of life, and the median Global Fatigue Index (GFI) was 38.7 (IQR 10.9, range 1-50), expressing a significant level of fatigue. The mean Beliefs about Medicines Questionnaire (BMQ) necessity-concern differential was 0.9 ± 1.1 (range - 1.8-4), 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 ( p  < 0.001), the presence of any major organ involvement ( p  < 0.031), the presence of gastro-intestinal ( p  < 0.001), neurological ( p  = 0.012) and musculoskeletal ( p  = 0.022) symptoms, recurrent fever ( p  = 0.002), and headache ( p  < 0.001) 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 ( F  = 14.519, OR 1.162 [CI 0.557-1.766], p  < 0.001)., Discussion: Preliminary 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., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2023 Gaggiano, Del Bianco, Sota, Gentileschi, Ruscitti, Giacomelli, Piga, Crisafulli, Monti, Emmi, De Paulis, Vitale, Tarsia, Caggiano, Nuzzolese, Parretti, Fabiani, Lopalco, Maier, Cattalini, Rigante, Govoni, Li Gobbi, Guiducci, Parronchi, Marino, Ciccia, Maggio, Aragona, Bartoloni, Iagnocco, Viapiana, Sebastiani, Guerriero, Insalaco, Del Giudice, Conti, Barone, Olivieri, Brucato, Carubbi, Triggianese, Mauro, Tosi, Fonollosa, Giardini, Ragab, Tharwat, Hernández-Rodríguez, Sfikakis, Laskari, Karamanakos, Espinosa, Shahram, Direskeneli, Hinojosa-Azaola, Opris-Belinski, AlMaghlouth, Hatemi, Eksin, Önen, Więsik-Szewczyk, Akkoç, Tufan, Şahin, Erten, Ozen, Batu, Frediani, Balistreri and Cantarini.)

Beckground: Despite the recent advances in the field of autoinflammatory diseases, most patients with recurrent fever episodes do not have any defined diagnosis. The present study aims at describing a cohort of patients suffering from apparently unexplained recurrent fever, in whom non-radiographic axial spondylarthritis (SpA) represented the unique diagnosis identified after a complete clinical and radiologic assessment., Materials and Methods: Patients' data were obtained from the international registry on Undifferentiated Systemic AutoInflammatory Diseases (USAIDs) developed by the AutoInflammatory Disease Alliance (AIDA) network., Results: A total of 54 patients with recurrent fever episodes were also affected by non-radiographic axial SpA according to the international classification criteria. SpA was diagnosed after the start of fever episodes in all cases; the mean age at the diagnosis of axial SpA was 39.9 ± 14.8 years with a diagnostic delay of 9.3 years. The highest body temperature reached during flares was 42°C, with a mean temperature of 38.8 ± 1.1°C. The most frequent manifestations associated to fever were: arthralgia in 33 (61.1%) cases, myalgia in 24 (44.4%) cases, arthritis in 22 (40.7%) cases, headache in 15 (27.8%) cases, diarrhea in 14 (25.9%) cases, abdominal pain in 13 (24.1%) cases, and skin rash in 12 (22.1%) cases. Twenty-four (44.4%) patients have taken daily or on-demand non-steroidal anti-inflammatory drugs (NSAIDs) and 31 (57.4%) patients have been treated with daily or on demand oral glucocorticoids. Colchicine was used in 28 (51.8%) patients, while other conventional disease modifying anti-rheumatic drugs (cDMARDs) were employed in 28 (51.8%) patients. Forty (74.1%) patients underwent anti-tumor necrosis factor (TNF) agents and 11 (20.4%) were treated with interleukin (IL)-1 inhibitors. The response to TNF inhibitors on recurrent fever episodes appeared more effective than that observed with anti-IL-1 agents; colchicine and other cDMARDs were more useful when combined with biotechnological agents., Conclusion: Signs and symptoms referring to axial SpA should be inquired in patients with apparently unexplained recurrent fever episodes. The specific treatment for axial SpA may lead to a remarkable improvement in the severity and/or frequency of fever episodes in patients with unexplained fevers and concomitant axial SpA., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2023 Vitale, Caggiano, Silva, Oliveira, Ruscitti, Ciccia, Vasi, Tufan, Lopalco, AlMaghlouth, Sota, Wiesik-Szewczyk, Gaggiano, Giardini, Spedicato, Ragab, Iannone, Balistreri, Frassi, Hernández-Rodríguez, Fabiani, Falsetti, Di Meglio, Frediani, Mazzei, Rigante, Faria and Cantarini.)

Objective: To evaluate the potential role of Streptococcus salivarius K12 (SSK12) in controlling febrile flares in patients with Periodic Fever, Aphthous stomatitis, Pharyngitis, and cervical Adenitis (PFAPA) syndrome. Further aims were to assess the impact of SSK12 on (i) flare duration, (ii) variation in the degree of the highest body temperature during flares, (iii) steroid-sparing effect, and (iv) change of PFAPA accompanying symptoms before and after SSK12 introduction., Patients and Methods: The medical charts from 85 pediatric patients with PFAPA syndrome (49 males and 36 females) enrolled in the AIDA registry and treated with SSK12 for a median period of 6.00 ± 7.00 months in the period between September 2017 and May 2022 were examined. Children recruited had a median time of disease duration of 19.00 ± 28.00 months., Results: The number of febrile flares significantly decreased comparing the 12 months before [median (IQR), 13.00 (6.00)] and after SSK12 initiation [median (IQR), 5.50 (8.00), p < 0.001]. The duration of fever was significantly reduced from 4.00 (2.00) days to 2.00 (2.00) days [ p < 0.001]. Similarly, the highest temperature in°C was found significantly lower in the last follow-up assessment [median (IQR), 39.00 (1.00)] compared to the period prior to SSK12 start [median (IQR), 40.00 (1.00), p < 0.001]. Steroid load (mg/year) of betamethasone (or any equivalent steroid) significantly decreased between 12 months before treatment with SSK12 [median (IQR), 5.00 (8.00) mg/year] and the last follow-up visit [median (IQR), 2.00 (4.00) mg/year, p < 0.001]. The number of patients experiencing symptoms including pharyngitis/tonsillitis ( p < 0.001), oral aphthae ( p < 0.001) and cervical lymphadenopathy ( p < 0.001) significantly decreased following SSK12., Conclusion: SSK12 prophylaxis given for at least 6.00 months was found to reduce febrile flares of PFAPA syndrome: in particular, it halved the total number per year of fever flares, shortened the duration of the single febrile episode, lowered body temperature by 1°C in the febrile flare, provided a steroid-sparing effect, and significantly reduced the accompanying symptoms related to the syndrome., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2023 La Torre, Sota, Insalaco, Conti, Del Giudice, Lubrano, Breda, Maggio, Civino, Mastrorilli, Loconte, Natale, Celani, Romeo, Patroniti, Gentile, Vitale, Caggiano, Gaggiano, Diomeda, Cattalini, Lopalco, Emmi, Parronchi, Gentileschi, Cardinale, Aragona, Shahram, Marino, Barone, Moscheo, Ozkiziltas, Carubbi, Alahmed, Iezzi, Ogunjimi, Mauro, Tarsia, Mahmoud, Giardini, Sfikakis, Laskari, Więsik-Szewczyk, Hernández-Rodríguez, Frediani, Gómez-Caverzaschi, Tufan, Almaghlouth, Balistreri, Ragab, Fabiani, Cantarini and Rigante.)

Objective: Interleukin (IL)-1 inhibitors are largely employed in patients with Still's disease; in cases with refractory arthritis, IL-6 inhibitors have shown to be effective on articular inflammatory involvement. The aim of the present study is to assess any difference in the effectiveness of the IL-1β antagonist canakinumab prescribed as first-line biologic agent between the systemic and the chronic-articular Still's disease., Methods: Data were drawn from the retrospective phase of the AutoInflammatory Disease Alliance (AIDA) international registry dedicated to Still's disease. Patients with Still's disease classified according to internationally accepted criteria (Yamaguchi criteria and/or Fautrel criteria) and treated with canakinumab as first-line biologic agent were enrolled., Results: A total of 26 patients (17 females, 9 males; 18 patients developing Still's disease after the age of 16 years) were enrolled; 16 (61.5%) patients suffered from the systemic pattern of the disease; 10 (38.5%) patients suffered from the chronic-articular type. No differences were observed between the systemic and the chronic-articular Still's disease in the frequency of complete response, of flares after the start of canakinumab ( p = 0.701) and in the persistence in therapy ( p = 0.62). No statistical differences were observed between the two groups after 3 months, 12 months and at the last assessment in the decrease of: the systemic activity score ( p = 0.06, p = 0.17, p = 0.17, respectively); the disease activity score on 28 joints ( p = 0.54, p = 0.77, p = 0.98, respectively); the glucocorticoid dosage ( p = 0.15, p = 0.50, and p = 0.50, respectively); the use of concomitant disease modifying anti-rheumatic drugs ( p = 0.10, p = 1.00, and p = 1.00, respectively). No statistically significant differences were observed in the decrease of erythrocyte sedimentation rate ( p = 0.34), C reactive protein ( p = 0.48), and serum ferritin levels ( p = 0.34) after the start of canakinumab., Conclusion: Canakinumab used for Still's disease has been effective in controlling both clinical and laboratory manifestations disregarding the type of disease course when used as first-line biotechnological agent. These excellent results might have been further enhanced by the early start of IL-1 inhibition., Competing Interests: Payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events: LC, Novartis. PR, Sobi, Abbvie, Novartis Ely Lilly, and BMS. CC, Sobi Novartis, Jannsen, and Boehringer. EW-S, Novartis and Sobi. LD, Novartis, Sobi, Roche, Galapagos, Jannsen, and Pfizer. PS, Abbvie, UCB, Novartis, Lilly, Genesis, Enorasis, Amgen, and Yansen. Support for attending meetings and travel: CC, Novartis and Amgen. EW-S, Sobi. LD, Novartis. Participation on a Data Safety Monitoring Board or Advisory Board: CC, Boehringer. LD, Novartis. Consulting fees: LD, Novartis, SOBI, Roche, Galapagos, Jannsen, and Pfizer. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2022 Vitale, Caggiano, Maggio, Lopalco, Emmi, Sota, La Torre, Ruscitti, Bartoloni, Conti, Fabiani, Mattioli, Gaggiano, Cardinale, Dagna, Campochiaro, Giacomelli, Balistreri, Laskari, Tufan, Ragab, Almaghlouth, Więsik-Szewczyk, Pereira, Frediani, Iannone, Sfikakis and Cantarini.)

Objective: 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., Methods: This is an electronic platform, based on the Research Electronic Data Capture (REDCap) tool, used for real-world data collection of demographics, clinical, laboratory, instrumental and socioeconomic data of mAIDs patients. The instrument has flexibility, may change over time based on new scientific acquisitions, and communicate potentially with other similar registries; security, data quality and data governance are corner stones of the platform., Results: AIDA project will share knowledge and expertise on mAIDs. Since its start, 118 centers from 24 countries and 4 continents have joined the AIDA project. Fifty-nine centers have already obtained the approval from their local Ethics Committees. Currently, the platform counts 337 users (122 Principal Investigators, 210 Site Investigators, 2 Lead Investigators, and 3 data managers). The Registry collects baseline and follow-up data using 3,748 fields organized into 21 instruments, which include demographics, patient history, symptoms, trigger/risk factors, therapies, and healthcare information for mAIDs patients., Conclusions: The AIDA mAIDs Registry, acts both as a research tool for future collaborative real-life studies on mAIDs and as a service to connect all the figures called to participate. On this basis, the registry is expected to play a pivotal role in generating new scientific evidence on this group of rare diseases, substantially improving the management of patients, and optimizing the impact on the healthcare system. NCT05200715 available at https://clinicaltrials.gov., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2022 Gaggiano, Vitale, Tufan, Ragab, Aragona, Wiesik-Szewczyk, Ait-Idir, Conti, Iezzi, Maggio, Cattalini, Torre, Lopalco, Verrecchia, Paulis, Sahin, Insalaco, Sfikakis, Marino, Frassi, Ogunjimi, Opris-Belinski, Parronchi, Emmi, Shahram, Ciccia, Piga, Hernández-Rodríguez, Pereira, Alessio, Naddei, Olivieri, Giudice, Sfriso, Ruscitti, Gobbi, Kucuk, Sota, Hussein, Malizia, Jahnz-Różyk, Sari-Hamidou, Romeo, Ricci, Cardinale, Iannone, Casa, Natale, Laskari, Giani, Franceschini, Sabato, Yildirim, Caggiano, Hegazy, Marzo, Kucharczyk, Khellaf, Tarsia, Almaghlouth, Laymouna, Mastrorilli, Dotta, Benacquista, Grosso, Crisafulli, Parretti, Giordano, Mahmoud, Nuzzolese, Musso, Chighizola, Gentileschi, Morrone, Cola, Spedicato, Giardini, Vasi, Renieri, Fabbiani, Mencarelli, Frediani, Balistreri, Tosi, Fabiani, Lidar, Rigante and Cantarini.)

Objective: The present paper describes the design, development, and implementation of the AutoInflammatory Disease Alliance (AIDA) International Registry specifically dedicated to patients with Schnitzler's syndrome., Methods: This is a clinical physician-driven, population- and electronic-based registry implemented for the retrospective and prospective collection of real-life data from patients with Schnitzler's syndrome; the registry is based on the Research Electronic Data Capture (REDCap) tool, which is designed to collect standardized information for clinical research, and has been realized to change over time according to future scientific acquisitions and potentially communicate with other existing or future similar registries., Results: Since its launch, 113 centers from 23 countries in 4 continents have been involved. Fifty-seven have already obtained the approval from their local Ethics Committees. The platform counts 324 users (114 Principal Investigators, 205 Site Investigators, 2 Lead Investigators, and 3 data managers) at current (April 28th, 2022). The registry collects baseline and follow-up data using 3,924 fields organized into 25 instruments, including patient's demographics, history, clinical manifestations and symptoms, trigger/risk factors, laboratory, instrumental exams, therapies, socioeconomic information, and healthcare access., Conclusions: This International Registry for patients with Schnitzler's syndrome facilitates standardized data collection, enabling international collaborative projects through data sharing and dissemination of knowledge; in turn, it will shed light into many blind spots characterizing this complex autoinflammatory disorder., (Copyright © 2022 Sota, Vitale, Więsik-Szewczyk, Frassi, Lopalco, Emmi, Govoni, de Paulis, Marino, Gidaro, Monti, Opris-Belinski, Pereira, Jahnz-Rózyk, Gaggiano, Crisafulli, Iannone, Mattioli, Ruffilli, Mormile, Rybak, Caggiano, Airò, Tufan, Gentileschi, Ragab, Almaghlouth, Aboul-Fotouh Khalil, Cattalini, La Torre, Tarsia, Giardini, Ali Saad, Bocchia, Caroni, Giani, Cinotti, Ruscitti, Rubegni, Dagostin, Frediani, Guler, Della Casa, Maggio, Recke, von Bubnoff, Krause, Balistreri, Fabiani, Rigante and Cantarini.)

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. This Project can be found on https://clinicaltrials.gov NCT05200715., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2022 Vitale, Caggiano, Della Casa, Hernández-Rodríguez, Frassi, Monti, Tufan, Telesca, Conticini, Ragab, Lopalco, Almaghlouth, Pereira, Yildirim, Cattalini, Marino, Giani, La Torre, Ruscitti, Aragona, Wiesik-Szewczyk, Del Giudice, Sfikakis, Govoni, Emmi, Maggio, Giacomelli, Ciccia, Conti, Ait-Idir, Lomater, Sabato, Piga, Sahin, Opris-Belinski, Ionescu, Bartoloni, Franceschini, Parronchi, de Paulis, Espinosa, Maier, Sebastiani, Insalaco, Shahram, Sfriso, Minoia, Alessio, Makowska, Hatemi, Akkoç, Li Gobbi, Gidaro, Olivieri, Al-Mayouf, Erten, Gentileschi, Vasi, Tarsia, Mahmoud, Frediani, Fares Alzahrani, Laymouna, Ricci, Cardinale, Jahnz-Rózyk, Tosi, Crisafulli, Balistreri, Dagostin, Ghanema, Gaggiano, Sota, Di Cola, Fabiani, Giardini, Renieri, Fabbiani, Carrer, Bocchia, Caroni, Rigante and Cantarini.)

Objective: This 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)., Methods: This 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., Results: The 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., Conclusions: The 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/., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2022 Della Casa, Vitale, Lopalco, Ruscitti, Ciccia, Emmi, Cattalini, Wiesik-Szewczyk, Maggio, Ogunjimi, Sfikakis, Tufan, Al-Mayouf, Del Giudice, Aragona, La Torre, Sota, Colella, Di Cola, Iacono, Mattioli, Jahnz-Rózyk, Joos, Laskari, Gaggiano, Abbruzzese, Cipriani, Rozza, AlSaleem, Yildirim, Tarsia, Ragab, Ricci, Cardinale, Korzeniowska, Frassi, Caggiano, Saad, Pereira, Berlengiero, Gentileschi, Guerriero, Giani, Gelardi, Iannone, Giardini, Almaghlouth, Kardas, Ait-Idir, Frediani, Balistreri, Fabiani, Rigante and Cantarini.)

Objective: Aim 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., Methods: This 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., Results: Starting 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., Conclusions: This 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. NCT05200715 available at https://clinicaltrials.gov/., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2022 Vitale, Della Casa, Lopalco, Pereira, Ruscitti, Giacomelli, Ragab, La Torre, Bartoloni, Del Giudice, Lomater, Emmi, Govoni, Maggio, Maier, Makowska, Ogunjimi, Sfikakis, Sfriso, Gaggiano, Iannone, Dagostin, Di Cola, Navarini, Ahmed Mahmoud, Cardinale, Riccucci, Paroli, Marucco, Mattioli, Sota, Abbruzzese, Antonelli, Cipriani, Tufan, Fabiani, Ramadan, Cattalini, Kardas, Sebastiani, Giardini, Hernández-Rodríguez, Mastrorilli, Więsik-Szewczyk, Frassi, Caggiano, Telesca, Giordano, Guadalupi, Giani, Renieri, Colella, Cataldi, Gentile, Fabbiani, Al-Maghlouth, Frediani, Balistreri, Rigante and Cantarini.)