Rheumatology Advances in Practice最新文献

{"title":"Clinical characteristics and quality of life in children with PFAPA syndrome and Behçet's disease.","authors":"Asli Gürel Bedir, Sara Sebnem Kilic","doi":"10.1093/rap/rkaf015","DOIUrl":"10.1093/rap/rkaf015","url":null,"abstract":"<p><strong>Objective: </strong>Periodic fever, aphthous stomatitis, pharyngitis, cervical adenitis (PFAPA) syndrome and Behçet's disease (BD) are non-monogenic autoinflammatory disorders with common clinical characteristics and genetic features. We aimed to review both patients' clinical characteristics and quality of life.</p><p><strong>Methods: </strong>Retrospective data were collected on patients' clinical and laboratory characteristics with PFAPA and BD between 2019 and 2022. The quality of life questionnaire (Pediatric Quality of Life Inventory) form was completed by the parents of all patients and the control group.</p><p><strong>Results: </strong>The study included 150 cases aged between 2 and 18, including 60 with PFAPA, 30 BD patients and 60 healthy volunteers. The duration of attacks in males with PFAPA was longer than that in females (<i>P</i> = 0.015). During attacks, the mean acute phase reactants of PFAPA patients were higher than those of BD (<i>P</i> = 0.010). In addition, there was a statistically significant relationship between the use of colchicine and a decrease in attack frequency in BD patients at 89.29% (<i>P</i> = 0.001 < 0.05) and in PFAPA patients at 88% (<i>P</i> = 0.001 < 0.05). Precisely, 61.67% of PFAPA (<i>n</i> = 37) and 73.33% of BD patients (<i>n</i> = 22) exhibited poor quality of life regarding daily activities and school attendance.</p><p><strong>Conclusion: </strong>Limited data on the quality of life of paediatric BD and PFAPA syndrome are available. During the attacks of patients with PFAPA and BD, acute-phase reactants were higher in PFAPA patients. Colchicine is an effective medication in reducing attacks in both groups. Effective and timely treatment is indispensable to improve quality of life.</p>","PeriodicalId":21350,"journal":{"name":"Rheumatology Advances in Practice","volume":"9 1","pages":"rkaf015"},"PeriodicalIF":2.1,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829805/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143433596","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Occupational history questionnaire for job coding and exposure assessment in systemic autoimmune rheumatic diseases.","authors":"Anne-Catherine Dens, Hannelore Celen, Stijn Michiels, Michaël Doumen, Sofia Pazmino, Lode Godderis, Patrick Verschueren, Hans Kromhout, Steven Ronsmans, Ellen De Langhe","doi":"10.1093/rap/rkaf016","DOIUrl":"10.1093/rap/rkaf016","url":null,"abstract":"<p><strong>Objectives: </strong>Systemic autoimmune rheumatic diseases (SARDs) develop in genetically susceptible individuals when exposed to environmental factors such as respirable crystalline silica (RCS) particles. Assessing occupational exposure in population-based studies is critical but resource intensive, often requiring expert interviews. This study aimed to develop and validate a self-administered occupational history questionnaire that allows for International Standard Classification of Occupations 1968 (ISCO-68) coding and exposure assessment as a cost-effective alternative to traditional interviews.</p><p><strong>Methods: </strong>Seventy RA patients participated by completing a standardized telephone interview and the newly developed self-administered questionnaire. Participants were also asked to recruit two gender- and age-matched family members for comparison. Independent observers assigned ISCO-68 codes to the reported jobs and a job exposure matrix was used to link each job to RCS exposure. Agreement between the interview and questionnaire was assessed by comparing reported working years, ISCO-68 job codes and RCS exposure. Cohen's κ and intraclass correlation were calculated to evaluate agreement and interobserver variability.</p><p><strong>Results: </strong>The patient response rate was 77%, but family member controls had a low response rate (6.45%), likely impacted by the COVID-19 pandemic. Agreement for reported working years was 91%, with a Cohen's κ of 0.87 for ever/never RCS exposure. Manual ISCO coding introduced variability, but interobserver reliability remained high (intraclass correlation coefficient = 0.91).</p><p><strong>Conclusion: </strong>The self-administered occupational history questionnaire provides a valid, cost-effective and time-efficient alternative to telephone interviews for assessing occupational history and estimating RCS exposure in epidemiological research. Future studies should explore automated coding systems and improved strategies for control recruitment.</p>","PeriodicalId":21350,"journal":{"name":"Rheumatology Advances in Practice","volume":"9 1","pages":"rkaf016"},"PeriodicalIF":2.1,"publicationDate":"2025-02-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11882302/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143568078","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Universal health-related social needs screening in a paediatric rheumatology clinic.","authors":"Mia T Chandler, Maria Alfieri Biancarelli, Anthony Dekermanji, Margaret Taggart, Benjamin G Ethier, Joyce C Chang, Brittany Esty, Mary Beth F Son, Melissa M Hazen","doi":"10.1093/rap/rkaf014","DOIUrl":"10.1093/rap/rkaf014","url":null,"abstract":"<p><strong>Objectives: </strong>Health-related social needs (HRSN) have a significant impact on health outcomes. While screening is prevalent in paediatric primary care settings, little evidence exists regarding the feasibility of HRSN screening in paediatric rheumatology clinics. Individuals with paediatric rheumatic disease have frequent longitudinal visits, therefore it is important to understand social needs that may hinder optimal health outcomes in this population. The objectives of this study were to understand the burden of HRSN and to contextualize the feasibility of universal HRSN screening in a busy paediatric rheumatology clinic.</p><p><strong>Methods: </strong>The study team secured funding for a full-time social worker and developed a needs assessment questionnaire (NAQ) through an iterative process. The paper-based NAQ was distributed by administrative clinical staff to all patients at each in-person visit. Medical providers returned completed NAQs to a designated collection tray. The responses were reviewed and analysed. The social worker addressed positive screens among families who opted in for contact.</p><p><strong>Results: </strong>More than 1/3 of patients returned an NAQ (985/2819), 604/985 (61%) of which revealed one or more concerns. The most frequent social concern was school or work related. The social worker responded by phone or patient electronic portal message to returned NAQs that identified unmet needs.</p><p><strong>Conclusion: </strong>A school or work concern was a frequent health-related social need in the paediatric rheumatology clinic. The prevalence of unmet HRSN, despite primary care access, shows the importance of screening in rheumatology clinics. Sustainable funding is needed to scale efforts to assess and address them.</p>","PeriodicalId":21350,"journal":{"name":"Rheumatology Advances in Practice","volume":"9 2","pages":"rkaf014"},"PeriodicalIF":2.1,"publicationDate":"2025-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11954546/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143754365","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Phase 3, multicentre, randomized, double-blind, placebo-controlled, parallel-group study of ustekinumab in patients with Takayasu arteritis.","authors":"Hajime Yoshifuji, Tomonori Ishii, Hiroki Ohashi, Katsunori Yoshizawa, Maki Mihoya, Kazuko Nishikawa, Yoshikazu Nakaoka","doi":"10.1093/rap/rkaf013","DOIUrl":"10.1093/rap/rkaf013","url":null,"abstract":"<p><strong>Objectives: </strong>Takayasu arteritis (TAK) is a rare, chronic large vessel vasculitis with unmet treatment needs. This phase 3 study aimed to evaluate efficacy, safety, pharmacokinetics and immunogenicity of ustekinumab (UST) in Japanese patients with TAK.</p><p><strong>Methods: </strong>Patients with TAK who had relapsed ≤12 weeks prior to study intervention administration and achieved remission thereafter with standard-of-care including corticosteroid intensification were randomized 1:1 to receive UST or matching placebo with protocol-defined oral glucocorticoid taper regimen. The double-blind (DB) phase was up to the patient's relapse/total of 35 relapse events, followed by the open-label extension (OLE) phase. Primary endpoint was the time to relapse of TAK per protocol-defined criteria through the end of the DB phase.</p><p><strong>Results: </strong>The study was terminated early due to patient recruitment challenge. Of 14 patients randomized, 8 relapsed during the DB phase (UST: 4/6; placebo: 4/8). The median time to relapse (weeks) was 11.14 (95% CI: 4.14, not estimated [NE]) for UST and 12.64 (95% confidence interval [CI]: 12.14, NE) for placebo (hazard ratio [HR] = 1.86 [95% CI: 0.41, 8.47]). In the DB phase, one patient in each group reported serious adverse event (SAE; UST: vascular pseudoaneurysm and brachiocephalic artery stenosis; placebo: cholecystitis); none were related to study intervention. Through the OLE phase, 1/4 (25.0%) patients in the UST-UST group (vascular graft infection considered related to study intervention) and none in the placebo-UST had SAEs. There were no serious infections/deaths throughout the study.</p><p><strong>Conclusion: </strong>The efficacy of UST in patients with TAK cannot be adequately assessed as the pre-determined sample size was not reached, and the study was prematurely terminated. No new safety signal of UST was identified.</p><p><strong>Trial registration: </strong>Clinicaltrials.gov, https://clinicaltrials.gov, NCT04882072; jrct.niph.go.jp, https://jrct.niph.go.jp, jRCT2061210007; Clinical Registry, CR108981.</p>","PeriodicalId":21350,"journal":{"name":"Rheumatology Advances in Practice","volume":"9 2","pages":"rkaf013"},"PeriodicalIF":2.1,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11919447/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143658546","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"BASDAI and ASDAS disease states in relationship to ASAS40 response: post hoc analysis of ixekizumab in radiographic axial spondyloarthritis.","authors":"Martin Rudwaleit, Victoria Navarro-Compán, Hagen Russ, Tommaso Panni, Erica Filippi, Mani Haschemi Nassab, Soyi Liu-Leage, Vincent Goëb, Francesco Ciccia, Jean Dudler","doi":"10.1093/rap/rkaf012","DOIUrl":"https://doi.org/10.1093/rap/rkaf012","url":null,"abstract":"<p><strong>Objectives: </strong>To explore the relationship between Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) and Axial Spondyloarthritis Disease Activity Score (ASDAS) used in clinical practice and the Assessment of SpondyloArthritis international Society 40% (ASAS40) response, the primary endpoint in clinical trials in axial spondyloarthritis (axSpA).</p><p><strong>Methods: </strong>Data from COAST-V, a phase 3 trial of ixekizumab <i>vs</i> placebo in biologic-naïve radiographic axSpA (r-axSpA) patients, were analysed. Patients treated with ixekizumab every 4 weeks were categorized using the ASAS40 response at week 16 and 52. The association between BASDAI and ASDAS disease states, respectively, and ASAS40 response achieved/not achieved was investigated. Additionally, back pain, fatigue, Bath Ankylosing Spondylitis Functional Index, ASAS Health Index and 36-item Short Form Health Survey Physical Component Summary scores corresponding to these states were assessed. Results were reported descriptively.</p><p><strong>Results: </strong>After 16  weeks, 48.1% (39/81) of patients achieved an ASAS40 response. Among them, 71.8% (<i>n</i> = 28) and 43.6% (<i>n</i> = 17) achieved BASDAI <3 and BASDAI <2, respectively; 76.9% (<i>n</i> = 30) and 33.3% (<i>n</i> = 13) attained ASDAS <2.1 and ASDAS <1.3, respectively. Among ASAS40 responders at week 52 [53.1% (43/81)], 83.8% (<i>n</i> = 36) and 51.2% (<i>n</i> = 22) of patients achieved BASDAI <3 and BASDAI <2, respectively; 93.1% (<i>n</i> = 40) and 41.9% (<i>n</i> = 18) attained ASDAS <2.1 and ASDAS <1.3. Lower BASDAI and ASDAS disease states corresponded well with less back pain, fatigue and functioning impairment and better health-related quality of life.</p><p><strong>Conclusions: </strong>More than 70% of biologic-naïve r-axSpA patients who achieved an ASAS40 response, also attained low disease activity or inactive disease as measured by the BASDAI or ASDAS. Findings may help clinicians translate results from clinical trials into daily practice.</p>","PeriodicalId":21350,"journal":{"name":"Rheumatology Advances in Practice","volume":"9 2","pages":"rkaf012"},"PeriodicalIF":2.1,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11978384/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143812225","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Ultrasound-guided synovial tissue biopsy for people with rheumatoid arthritis: a micro-costing study.","authors":"Sainan Chang, Felice Rivellese, Katherine Payne, Zoë Ide, Anne Barton, Costantino Pitzalis, Sean P Gavan","doi":"10.1093/rap/rkaf011","DOIUrl":"10.1093/rap/rkaf011","url":null,"abstract":"<p><strong>Objective: </strong>Identify and quantify the resource use and cost per patient of performing a US-guided synovial tissue biopsy for people with RA within a routine healthcare setting.</p><p><strong>Method: </strong>A micro-costing study was performed from a healthcare system perspective (National Health Service England). A service pathway conceptual model described how the procedure will be embedded within routine care to inform management decisions. Consumables were estimated from existing standard operating procedures for synovial biopsies. Staff time was estimated by expert input (clinical rheumatologist and patient). The time for tissue sample acquisition was obtained from data within the biopsy-driven R4RA trial. Unit costs were sourced from historic purchase prices, published salary scales and public-facing list prices. One-way sensitivity analysis identified key drivers of total cost per patient. Scenario analyses explored the cost impact if less senior healthcare staff performed the biopsy or if an additional outpatient appointment was required to identify joint suitability.</p><p><strong>Results: </strong>The total cost of US-guided synovial tissue biopsy was £356.24/patient (best-case estimate: £185.30; worst-case estimate: £812.46). The key driver of total cost was the time taken to perform tissue sample acquisition. The total cost was lower if a registrar performed the biopsy (£294.24) and higher if an additional outpatient appointment was required for joint selection (£438.98).</p><p><strong>Conclusion: </strong>Interventions requiring synovial tissue samples to inform treatment selection or prognosis are emerging. The findings can inform cost parameters for future cost-effectiveness analyses. These results will help resource allocation and clinical decisions to improve the value of treatments for RA.</p>","PeriodicalId":21350,"journal":{"name":"Rheumatology Advances in Practice","volume":"9 1","pages":"rkaf011"},"PeriodicalIF":2.1,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11855308/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143503656","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A novel classifier of radiographic knee osteoarthritis for use on knee DXA images is predictive of joint replacement in UK Biobank.","authors":"Rhona A Beynon, Fiona R Saunders, Raja Ebsim, Benjamin G Faber, Mijin Jung, Jennifer S Gregory, Claudia Lindner, Richard M Aspden, Nicholas C Harvey, Timothy Cootes, Jonathan H Tobias","doi":"10.1093/rap/rkaf009","DOIUrl":"10.1093/rap/rkaf009","url":null,"abstract":"<p><strong>Objectives: </strong>DXA scans may offer a novel means of evaluating radiographic knee OA (rKOA) in large population studies and through opportunistic screening. We aimed to develop and apply a semi-automated method for assessing rKOA using ≈20 000 knee DXA images from UK Biobank (UKB) and assess its face validity by checking for expected relationships with clinical outcomes.</p><p><strong>Methods: </strong>Right knee DXA scans were manually annotated for osteophytes to derive corresponding grades. Joint space narrowing (JSN) grades in the medial joint compartment were determined from automatically measured minimum joint space width. Overall rKOA grade (0-4) was determined by combining osteophyte and JSN grades. Logistic regression was employed to investigate the associations of osteophyte, JSN and rKOA grades with knee pain and hospital-diagnosed KOA. Cox proportional hazards modelling was used to examine the associations of these variables with risk of subsequent total knee replacement (TKR).</p><p><strong>Results: </strong>Of the 19 595 participants included (mean age 63.7 years), 19.5% had rKOA grade ≥1 (26.1% female, 12.5% male). Grade ≥1 osteophytes and grade ≥1 JSN were associated with knee pain, hospital-diagnosed KOA and TKR. Higher rKOA grades were linked to stronger associations with these clinical outcomes, with the most pronounced effects observed for TKR. Hazard ratios for the association of rKOA grades with TKR were 3.28, 8.75 and 28.63 for grades 1, 2 and 3-4, respectively.</p><p><strong>Conclusions: </strong>Our DXA-derived measure of rKOA demonstrated a progressive relationship with clinical outcomes. These findings support the use of DXA for classifying rKOA in large epidemiological studies and in future population-based screening.</p>","PeriodicalId":21350,"journal":{"name":"Rheumatology Advances in Practice","volume":"9 1","pages":"rkaf009"},"PeriodicalIF":2.1,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11846665/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143483945","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Anti-citrullinated protein antibody detection by hemagglutination.","authors":"Ilmar Kruis, Jyoti Kumari, Annemarie van der Heijden, Amrah Weijn, Wilma Vree Egberts, Iris Rose Peeters, Noortje van Herwaarden, Martin Salden, Ger J M Pruijn","doi":"10.1093/rap/rkaf010","DOIUrl":"10.1093/rap/rkaf010","url":null,"abstract":"<p><strong>Objectives: </strong>ACPAs play an important role in the classification of RA and can be detected by serological tests. Several ACPA detection assays are available for clinical use, which are all based on ELISA(-like) tests with citrullinated peptides (CCP2) or alternative citrullinated proteins/peptides. We aimed to facilitate ACPA detection in low-volume laboratories and resource-poor environments by developing a rapid and easy-to-perform test.</p><p><strong>Methods: </strong>We designed and generated an agglutination mediator by protein engineering. A single-chain antibody fragment that binds to glycophorin A, one of the major surface proteins of erythrocytes, was conjugated to a CCP2-like peptide. This agglutination mediator was used to assess ACPA in RA and PsA patients and in healthy individuals.</p><p><strong>Results: </strong>The agglutination mediator bound to erythrocytes was reactive with ACPA and induced haemagglutination in an ACPA-dependent fashion, which can be detected by the naked eye. The applicability was assessed by the analysis of fresh blood samples from 204 RA patients, 77 PsA patients and 100 healthy individuals. Agglutination was observed in up to 61% of the RA samples, which correlated well with the results obtained with a standardized anti-CCP2 ELISA (63-67%). Depending on the minimal agglutination score, agglutination was observed in only 3-21% of the PsA samples and in 1% of the healthy controls.</p><p><strong>Conclusion: </strong>We conclude that the agglutination mediator allows rapid and efficient detection of ACPA by haemagglutination in human blood samples and offers a low-threshold method for ACPA detection.</p>","PeriodicalId":21350,"journal":{"name":"Rheumatology Advances in Practice","volume":"9 1","pages":"rkaf010"},"PeriodicalIF":2.1,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829806/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143433593","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Complement proteins in axial spondyloarthritis: associations with disease activity and TNFi treatment response in a multicentre RCT.","authors":"Clara Mistegaard, Anne Troldborg, Murat Torgutalp, Anne Gitte Loft, Steffen Thiel, Burkhard Muche, Valeria Rios Rodriguez, Mikhail Protopopov, Joachim Listing, Joachim Sieper, Denis Poddubnyy, Fabian Proft","doi":"10.1093/rap/rkaf007","DOIUrl":"10.1093/rap/rkaf007","url":null,"abstract":"<p><strong>Objectives: </strong>To investigate lectin pathway proteins (LPPs) and complement activation marker C3dg as biomarkers for disease activity and treatment response in a multicentre, randomized controlled trial of axial spondyloarthritis (axSpA) patients initiating TNF inhibitor (TNFi) therapy.</p><p><strong>Methods: </strong>Serum samples from 108 patients with active radiographic axSpA from the CONSUL study, collected before and after 12 weeks of TNFi therapy, were measured using immunoassays for LPPs (MBL, CL-L1, M-, L-, and H-ficolin, MASP-1, -2, and -3, MAp44) and the complement activation marker C3dg.</p><p><strong>Results: </strong>After 12 weeks of TNFi therapy, serum levels of LPPs L-ficolin, M-ficolin, and MASP-2 decreased, while MASP-3 increased after adjustment for baseline CRP. Baseline L-ficolin levels correlated positively with baseline ASDAS-CRP and BASFI. C3dg correlated positively with ASDAS-CRP. Conversely, MASP-1 and MAp44 correlated negatively with ASDAS-CRP. Assessed by univariate logistic regression, C3dg and MASP-1 were associated with treatment response of clinically important improvement (ΔASDAS-CRP ≥1.1) and inactive disease (ASDAS-CRP <1.3) at week 12, respectively. Only C3dg remained significant in a multivariate regression analysis.</p><p><strong>Conclusion: </strong>In this study, complement LPPs L-ficolin, M-ficolin, and MASP-2 levels decrease following initiation of TNFi therapy, whereas alternative pathway critical component MASP-3 increases. Baseline L-ficolin and C3dg correlated positively with ASDAS-CRP, potentially by CRP influence. Nevertheless, baseline C3dg levels were associated with treatment response (ASDAS-CRP <1.3) at week 12. Our results provide important perspectives on the inflammatory processes in axSpA, shedding light on the involvement of the complement system related to disease activity, treatment response, and potentially to prognosis.</p>","PeriodicalId":21350,"journal":{"name":"Rheumatology Advances in Practice","volume":"9 1","pages":"rkaf007"},"PeriodicalIF":2.1,"publicationDate":"2025-01-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825385/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143433599","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Challenges in the transition of care for rare connective tissue diseases: results from the 2023 ERN ReCONNET Transition of Care Task Force survey.","authors":"Edoardo Marrani, Mojca Zajc Avramovic, Diana Marinello, Rosaria Talarico, Chiara Baldini, Eva Collado-González, João Eurico Fonseca, Linda Schraven, Filipa Oliveira Ramos, Paola Triggianese, Arjan Vissink, Marta Mosca, Tadej Avcin, Gabriele Simonini","doi":"10.1093/rap/rkae149","DOIUrl":"10.1093/rap/rkae149","url":null,"abstract":"<p><strong>Objectives: </strong>Two different European Reference Networks cover CTDs with paediatric onset, the European Reference Network on Rare and Complex Connective Tissue Diseases (ERN ReCONNET) and the European Reference Network on Rare Immunological Disorders (ERN RITA). The transition of care is a significant focus, with ReCONNET centres actively addressing this through updated programs. Despite these efforts, challenges persist. We aimed to inventory transitional care programs for rare CTDs across Europe.</p><p><strong>Methods: </strong>In April 2023, the ERN ReCONNET Transition of Care Task Force, consisting of expert clinicians, patient advocates and coordination team members, created a survey to assess transitional care practices. The survey was distributed to ERN ReCONNET and ERN RITA centres and responses received by 15 March 2024 were analysed.</p><p><strong>Results: </strong>A total of 67 responses from 59 centres across 20 European countries were collected. Paediatric rheumatologists typically initiated the transition process (49% of centres). Twenty centres had joint clinics. Despite positive self-assessments of transitional programs, significant limitations were noted. Transition policies varied, with only 40% of centres having a formal standardized policy and less than half of the centres adhering to available transition of care guidelines. Transfer readiness was evaluated using validated questionnaires in 13% of centres, while 29% transitioned patients based solely on age without any readiness assessments. The main challenges included finding adult-oriented centres and the lack of guidelines or engagement from adult centres. Adult healthcare providers also noted a lack of training in adolescent medicine.</p><p><strong>Conclusion: </strong>The survey highlighted diverse transition practices and resources across centres, with challenges in readiness evaluation and the use of guidelines. Despite these obstacles, respondents rated ongoing transition processes positively. Enhancing patient perspectives in the transition process is crucial to meet their needs during this critical phase.</p>","PeriodicalId":21350,"journal":{"name":"Rheumatology Advances in Practice","volume":"9 1","pages":"rkae149"},"PeriodicalIF":2.1,"publicationDate":"2025-01-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11780841/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143067872","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}