ObjectiveThe present paper describes the design, development, and implementation of the AutoInflammatory Disease Alliance (AIDA) International Registry specifically dedicated to patients with Schnitzler's syndrome.MethodsThis 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.ResultsSince 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.ConclusionsThis 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.
ObjectivesIn systemic sclerosis (SSc) American patients, anti-Th/To antibodies were reported to be associated with interstitial lung disease (ILD) and pulmonary arterial hypertension (PAH). Few data in European patients are available, so we aimed at describing the clinical associations of anti-Th/To antibodies, focusing on ILD outcome, organ damage and mortality in an Italian single-centre cohort. Methods Case-control study: anti-Th/To+ SSc patients vs. anti-topoisomerase (anti-topo)1+, anticentromere (ACA)+ and quadruple-negative (anti-topo 1-, ACA-, anti-RNAP3-, anti-Th/To-) SSc patients (1:3; matched for sex and age at SSc onset).Organ damage was assessed with the SCTC-Damage Index. ResultsThirteen anti-Th/To+ patients were evaluated: 100% had limited cutaneous involvement; 46% digital ulcers; none had PAH, synovitis, joint contractures. As compared to anti-topo 1+ and quadruple-negative patients, anti-Th/To+ patients developed less frequently ILD (40% vs. 85% and 84%), that required less immunosuppression (8% vs. 41% and 44%), and rarely had functional worsening (15.4% at 5 years), without development of long-term complications (no need for O 2 , pulmonary hypertension, death). In anti-Th/To+ patients, the Damage Index was lower than in anti-topo 1+ and quadruple-negative patients at various timepoints, and remained low during the long-term follow-up (median: 16 years). The 5-and 10-year survival of anti-Th/To+ patients was 92% and 72%, respectively, and did not differ from those of the SSc matched patients; none of the anti-Th/To+ patients died due to SSc, while mortality was mainly related to cancer. ConclusionsIn this study, anti-Th/To+ patients showed a mild SSc phenotype, characterised by low organ damage, favourable ILD outcome and good survival.
BackgroundEnrichment strategies from clinical trials for progressive systemic sclerosis-associated interstitial lung disease (SSc-ILD) have been partly successful but have not been tested in a real life cohort.ObjectivesAnalyse the efficacy, representativeness and feasibility of enrichment strategies in SSc-ILD patients from the EUSTAR cohort.MethodsWe applied the inclusion criteria of major recent SSc-ILD trials (focuSSced, SLS II and SENSCIS) in SSc-ILD patients and assessed progressive ILD, defined as absolute change in forced vital capacity (FVC) and as significant progression (FVC decline >10%) over time. Data were compared to all patients and patients not fulfilling any inclusion criteria.ResultsIn total, 2258 SSc-ILD patients were included, with 31.2% meeting SENSCIS, 5.8% SLS II, 1.6% focuSSced criteria and 1529 (67.7%) not meeting any criteria (Table 1). In the first 12+/-3 months, a slow FVC% decline of –0.1% was seen in the total, unselected cohort and in patients fulfilling SENSCIS criteria. Patients fulfilling criteria from focuSSced showed a strong FVC decline of –3.7%. Notably, patients enriched for SLS II criteria showed FVC improvement of +2.3% (Figure 1). Similarly, compared to the total unselected cohort, the number of significant progressive events was numerically higher in patients fulfilling focuSSced criteria, the same for SENSCIS criteria and even slightly lower for patients fulfilling the SLS2 criteria.Table 1.Demographics and baseline clinical characteristics of EUSTAR patientsNot fulfilling any criteria (n=1529)focuSSced (n=36)SLS II (n=132)SENSCIS (n=704)Age, years (SD)58.4 (2.9)51.5 (12.2)†51.2 (12.7) †54.2 (13.8) †Male, n (%)231 (15.1)7 (19)35 (27)**156 (21)*Disease duration, months (SD)156.3 (99.4)16.1 (13.9)†40.7 (25.2) †39.4 (23.9) †DcSSc, n (%)597 (43.8)36 (100) †85 (65) †35 (52) †ATA, n (%)735 (51.1)24 (67)*85 (69) †370 (56)mRSS, mean (SD)9.5 (8.3)21 (6.5)*13 (9.6)*11 (9.2)GERD, n (%)1002 (65.9)25 (69)92 (70)430 (62)ESR, mean (SD)27 (20.5)43.1 (23) †29.6 (19.6) †24.7 (20.7)MMF, n (%)75 (16.5)0 (0) †0 (0) †52 (22) †MTX, n (%)42 (9.2)0 (0) †2 (5)20 (9)FVC % predicted, mean (SD)85.7 (22.5)88 (13.6)*66 (9.1) †88 (19.8)DLCO% predicted, mean (SD)58.9 (21.5)61 (12.7)49(14.6)†59 (14.2)NYHA class, n (%)3261 (17.8)6 (19)28 (21)72 (10)*440 (2.7)0 (0)3 (2)4 (1)**P-value: 0.001–0.05; †P<0.001, between focuSSced, SENSCIS or SLS compared with not fulfilling any study criteria.In the second 12 months period, SENSCIS enriched patients had a further absolute FVC% decline as described for the total cohort. In contrast, patients fulfilling the focuSSced and SLS II inclusion criteria showed numerical improvement of lung function in the second period (Figure 1). There were no significant associations of enrichment criteria and ILD progression in the second period.Over the mean observation period of 2.3 years, patients not fulfilling any inclusion criteria showed the same FVC decline of –0.9 (12.1) as observed for the total cohort (–0.9% (12.6)). There were numerical differences in FVC changes in the enriched patient cohorts, varying from –2.8% FVC decline in patients fulfilling the focuSSced criteria to +3.4% FVC improvement with SLS II criteria.ConclusionApplication of enrichment criteria from previous clinical trials showed enrichment for progression with variable success but led to selected patient populations reducing feasibility of recruitment. These findings are important for future clinical trial design and interpretation of the results of published trials.AcknowledgementsWe thank all EUSTAR collaborators.Disclosure of InterestsAnna-Maria Hoffmann-Vold Speakers bureau: Actelion, Boehringer Ingelheim, Jansen, Lilly, Medscape, Merck Sharp & Dohme, Roche, Consultant of: Actelion, ARXX, Bayer, Boehringer Ingelheim, Jansen, Lilly, Medscape, Merck Sharp & Dohme, Roche, Grant/research support from: Boehringer Ingelheim, Cathrine Brunborg: None declared, Paolo Airò Speakers bureau: Bristol-Myers-Squibb, Boehringer Ingelheim, Consultant of: Bristol-Myers-Squibb, Grant/research support from: Bristol-Myers-Squibb, Roche, Jannsen, CSL Behring, Lidia P. Ananyeva Speakers bureau: Boehringer Ingelheim, Consultant of: Boehringer Ingelheim, László Czirják Speakers bureau: Boehringer Ingelheim, Consultant of: Boehringer Ingelheim, Actelion (now GSK), MSD, Novartis, Pfizer, Roche, Lilly, Grant/research support from: Boehringer Ingelheim, Actelion (now GSK), MSD, Novartis, Pfizer, Serena Guiducci: None declared, Eric Hachulla Speakers bureau: GSK, Roche-Chugai, Johnson & Johnson, Boehringer Ingelheim, Consultant of: CSL Behring, GSK, Roche-Chugai, Johnson & Johnson, Boehringer Ingelheim, Grant/research support from: CSL Behring, Boehringer Ingelheim, GSK, Roche-Chugai, Sanofi Genzyme, Mengtao Li: None declared, Carina Mihai Speakers bureau: MEDtalks Switzerland, Mepha, Grant/research support from: Roche, Boehringer Ingelheim, Janssen, Gabriela Riemekasten Speakers bureau: Boehringer Ingelheim, Consultant of: Boehringer Ingelheim, Petros Sfikakis Consultant of: Boehringer Ingelheim, Gabriele Valentini Consultant of: Boehringer Ingelheim, Sanofi, Grant/research support from: BMS, Otylia Kowal-Bielecka Speakers bureau: Boehringer Ingelheim, Novartis, Pfizer, Gilead Sciences, Janssen-Cilag, MEDAC, MSD, Abbvie, Sandoz, Consultant of: Boehringer Ingelheim, Health Care system Navigator, CSL Behring, MSD, Novartis, Grant/research support from: CSL Behring, Boehringer Ingelheim, Abbvie, Roche, MEDAC, Yannick Allanore Speakers bureau: Boehringer, Abbvie, Consultant of: Boehringer, Bayer, Astra-Zeneca, Prometheus, Sanofi, Genentech/Roche, Boehringer, Grant/research support from: Alpine Immunosciences, OSE Immunotherapeutics, Medsenic, Oliver Distler Speakers bureau: Bayer, Boehringer Ingelheim, Janssen, Medscape, Consultant of: Abbvie, Acceleron, Alcimed, Amgen, AnaMar, Arxx, AstraZeneca, Baecon, Blade, Bayer, Boehringer Ingelheim, Corbus, CSL Behring, 4P Science, Galapagos, Glenmark, Horizon, Inventiva, Kymera, Lupin, Miltenyi Biotec, Mitsubishi Tanabe, MSD, Novartis, Prometheus, Roivant, Sanofi and Topadur, Grant/research support from: Kymera, Mitsubishi Tanabe, Boehringer Ingelheim
Introduction: In 2015 the historic Jones criteria for the diagnosis of Acute Rheumatic Fever (ARF) were revised introducing two different sets of criteria for low-risk and for moderate/high-risk populations (according to ARF incidence). In Italy the exact ARF incidence is unknown but small regional or local reports suggest an incidence of 2-5/100.000 per year, suggesting that our population might be considered at moderate risk for ARF. Objectives: To evaluate the performance of the revised Jones criteria in a retrospective population and to compare it with the performance of the previous version of Jones criteria. Methods: We conducted a retrospective study on 288 patients with ARF (108 female; median age 8.5 years, IQR 7.1-10.3) diagnosed from 2001 to 2015 in a Pediatric Rheumatology Division by pediatric rheumatologists, discharged with an ICD 9 code consistent with ARF. We retrospectively applied the two sets (for low-risk and for moderate/high-risk) of the 2015 revised Jones criteria and the 1992 version of the Jones criteria. Results: Of 288 patients, 253 (87.8%) met the 1992 version of the Jones criteria, 237 (82.3%) met the revised criteria for low-risk populations and 259 (89.9%) for moderate/high-risk populations. None of these differences was significant. Prevalence of major and minor criteria is shown in Table. With the exception of difference in arthritis, the 1992 version and the 2015 revised version did not show major differences. Of the 288 patients with a clinical diagnosis of ARF 29 did not meet any version of the Jones criteria. Patients in this group presented with isolated chorea or silent carditis without other manifestations. Prevalence of the clinical characteristics and comparison among the 1992 version of Jones criteria and the 2015 revised Jones criteria (low risk and moderate-high risk populations): Values are expressed in Number (percentage). *p value (Fisher Exact test) Conclusion: The revised Jones criteria for low-risk populations are slightly more sensitive than the 1992 version of Jones criteria, while the revised Jones criteria for moderate/high populations are slightly less sensitive than the 1992 version. In this population, the revised criteria did not substantially modify the diagnosis of ARF. Approximately 10% of patients presented with isolated chorea or silent carditis.
BackgroundSystemic sclerosis (SSc) is a multisystem autoimmune disorder with skin sclerosis and digital ulcers (DU) as typical cutaneous lesions. Patients are categorised as having either limited or diffuse skin involvement, depending on the distribution of skin sclerosis which is important as diffuse cutaneous SSc is associated with higher morbidity and mortality.ObjectivesGiven the paucity of pivotal data on the temporal evolution of skin manifestations during the early course of SSc, our aim was to longitudinally map the onset and identify risk factors for skin sclerosis and DU in patients with SSc from an early time point after the onset of Raynaud's phenomenon (RP) in the EUSTAR cohort.MethodsSSc patients followed in the prospective multinational EUSTAR database were investigated if they had a baseline visit within one year after RP onset. Outcome measures were analysed as a function of time after RP onset using Kaplan-Meier methods and included the evolution of the modified Rodnan skin score (mRSS), the presence of skin sclerosis, the presence of diffuse cutaneous involvement and the presence of DU. Cox proportional hazards regression analysis was used to evaluate risk factors.Results707 SSc patients in the EUSTAR database had a baseline visit within one year after RP onset. The median peak mRSS was 15 points and was reached 1.0 years after RP onset. There was no difference in the median time to reach the mRSS peak between patients with limited and patients with diffuse cutaneous SSc (p=0.36). All patients who developed moderate to severe skin sclerosis (defined as a mRSS≥2 at any body area) did so within 6.5 years after RP onset. The 1-year probability to develop a mRSS≥2 in at least one area of the upper and lower extremities was 68% and 25%, respectively. A quarter of patients developed diffuse cutaneous involvement in the first year after RP onset (Figure 1). This probability increased to 35.4% during the subsequent two years. Only a minority of patients developed diffuse cutaneous SSc thereafter. The probability to develop DU increased continuously to a maximum of 70% at the end of the 10 year observation. The main risk factors for diffuse cutaneous SSc were the presence of anti-RNA polymerase III autoantibodies, followed by anti-topoisomerase autoantibodies and male sex. The main risk factor for incident DU was the presence of anti-topoisomerase autoantibodies.ConclusionsEarly after RP onset, cutaneous manifestations exhibit rapid kinetics in SSc. This should be accounted for in trials aiming to prevent skin worsening. Furthermore, our findings will enable physicians to counsel and manage patients presenting early more accurately.AcknowledgementsEUSTAR acknowledges the prior support that EULAR has provided for the maintenance of the EUSTAR database.Disclosure of InterestV. Jaeger: None declared, E. Wirz: None declared, Y. Allanore Grant/research support from: Actelion, Bayer, Biogen Idec, BMS, Genentech/ Roche, Inventive, Medac, Pfizer, Sanofi/Genzyme, Servier and UCB, Consultant for: Actelion, Bayer, ...
BackgroundThe research of circulating biomarkers in the field of chronic arthritis is still an unmet need, especially for the subgroup of seronegative arthritis [e.g., negative for anti-citrullinated peptides autoantibodies (ACPA) and/or rheumatoid factors (RF)]. In fact, in this clinical subset, there is a lack of soluble molecules which could help for a diagnostic or prognostic purpose, and the differential diagnosis between psoriatic arthritis (PsA) and seronegative rheumatoid arthritis (RA) is still a challenge. C-X-C motif chemokine 13 (CXCL13) is one of the most promising biomarkers recently identified, because of its association with active synovitis (1) and with a poor prognosis in RA (2), although there are still inadequate results to justify its routine use. No data are available of the determination of CXCL13 serum levels in PsA.ObjectivesTo analyze CXCL13 serum levels in a cohort of PsA patients with a history of peripheral involvement in comparison with RA.MethodsCross sectional analysis of consecutive patients with peripheral PsA [n:81; male/female=44/37; median age (25°-75° percentile)=54 (46-62) years; 28-joint Disease Activity Score-C Reactive Protein (CRP-DAS28)=1.9 (1.6-2.5); active psoriasis=43%; DAPSA (Disease Activity in PSoriatic Arthritis) score=8 (3-14)] and RA [n:143; male/female=30/113; age =62 (50-70) years; seropositive=67%; CRP-DAS28=2.1 (1.5-2.8)] was performed. 100 sex and age-matched healthy controls (HC) were enrolled. CXCL13 serum levels were assessed through commercial ELISA test (R&D).ResultsCXCL13 serum levels were higher in all the subgroups of patients [PsA: 50.9 (34.5-80.2) pg/mL, p<0.01; RA: 77.2 (52.9-107.7) pg/mL, p<0.01; RA ACPA+: 77.7 (55.9-110.7) pg/mL, p<0.01; RA ACPA-: 69.5 (49.3-104) pg/mL, p<0.01] than in HC (22.3 (17.7-33.8) pg/mL). No significant differences were found among RA patients according with their seropositivity (p=0.378). CXCL13 serum levels were lower in PsA patients than in RA patients, independently from their seropositivity [vs RA ACPA+, p<0.01; vs RA ACPA-, p=0.012]. CXCL13 serum levels were positively correlated with CRP in PsA patients (r=0.30; p=0.008), but not with DAPSA score.ConclusionThese results confirm the value of CXCL13 as a biomarker in the field of chronic arthritis. Its higher levels in seronegative RA than in PsA suggests its possible value in the differential diagnosis of these two subsets of arthritis.References[1]Bechman et al. BMC Rheumatology 2020;[2]Bugatti et al. Rheumatology 2014.Disclosure of InterestsNone declared
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.