RMD OpenPub Date : 2024-12-20DOI: 10.1136/rmdopen-2024-004943
Elisabetta Casto, Alessandro Celi, Rosalinda Madonna, Raffaele De Caterina, Federica Di Cianni, Rosaria Talarico, Marta Mosca, Marco De Carlo, Andrea Maria D'Armini, Laura Carrozzi, Roberta Pancani
{"title":"Anticoagulation in venous thromboembolism in Behçet's syndrome: friend or foe?","authors":"Elisabetta Casto, Alessandro Celi, Rosalinda Madonna, Raffaele De Caterina, Federica Di Cianni, Rosaria Talarico, Marta Mosca, Marco De Carlo, Andrea Maria D'Armini, Laura Carrozzi, Roberta Pancani","doi":"10.1136/rmdopen-2024-004943","DOIUrl":"10.1136/rmdopen-2024-004943","url":null,"abstract":"","PeriodicalId":21396,"journal":{"name":"RMD Open","volume":"10 4","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11664342/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142966410","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
RMD OpenPub Date : 2024-12-20DOI: 10.1136/rmdopen-2024-004948
Mate Lorincz, Mikkel Østergaard, Marie Wetterslev, Inge Juul Sørensen, Ole Rintek Madsen, Sara Nysom Christiansen, Merete Lund Hetland, Mads Bakkegaard, Mette Klarlund, Anne Duer, Mikael Boesen, Kasper Kjærulf Gosvig, Susanne Juhl Pedersen
{"title":"Construct validity and responsiveness of ASAS Health Index assessed in two longitudinal studies of tumour necrosis factor alpha inhibitor initiation and dose reduction in patients with axial spondyloarthritis.","authors":"Mate Lorincz, Mikkel Østergaard, Marie Wetterslev, Inge Juul Sørensen, Ole Rintek Madsen, Sara Nysom Christiansen, Merete Lund Hetland, Mads Bakkegaard, Mette Klarlund, Anne Duer, Mikael Boesen, Kasper Kjærulf Gosvig, Susanne Juhl Pedersen","doi":"10.1136/rmdopen-2024-004948","DOIUrl":"10.1136/rmdopen-2024-004948","url":null,"abstract":"<p><strong>Background: </strong>The Assessment of SpondyloArthritis international Society Health Index (ASAS HI) is a novel questionnaire of global functioning for patients with axial spondyloarthritis (SpA).</p><p><strong>Objective: </strong>The objective was to assess the construct validity, discriminatory ability and responsiveness of ASAS HI in relation to patient-reported outcome measures (PROMs), MRI and radiography.</p><p><strong>Methods: </strong>Data from two longitudinal studies with tumour necrosis factor inhibitor (TNFi) initiation (novel MRI And biomarkers in Golimumab-treated patients with axial spondyloarthritis (MANGO): n=45) respectively tapering (Dose adjustment of Biological treatment in patients with SpA (DOBIS): n=106) were used. Analyses included a wide panel of PROMs, MRI and radiography scores of the spine and sacroiliac joints (SIJs).</p><p><strong>Results: </strong>In the MANGO study, 30 (68%) patients were clinical responders at week 16. In the DOBIS study, 105 (99%) patients flared after mean (SD; min-max) 31 (17; 2.7-81) weeks. After initiation of TNF inhibitor in MANGO, ASAS HI significantly decreased from baseline to week 4, 16 and 52. In DOBIS, ASAS significantly increased from baseline to the flare visit and significantly decreased from the flare visit to week 96. In multivariate regression models, ASAS HI was independently associated with Spondyloarthritis Research Consortium of Canada MRI SIJ Inflammation score, Canada-Denmark MRI Spine Inflammation score, EuroQol, Ankylosing Spondylitis Disease Activity Index, Bath Ankylosing Spondylitis Disease Activity Index and Patient Global. Patients stratified according to ASAS HI health status groups (good, moderate, poor) at baseline and change categories (absolute and percentage change) from baseline to week 16/flare showed good discriminatory ability for almost all outcome variables (p≤0.001). ASAS HI had a large responsiveness in MANGO (standardised response mean (SRM)=-1.3, effect size (ES)=-1.7) and moderate responsiveness in DOBIS (SRM=0.7, ES=0.6).</p><p><strong>Conclusion: </strong>ASAS HI showed good construct validity, discriminatory ability and responsiveness.</p><p><strong>Trial registration number: </strong>ClinicalTrials.gov: NCT02011386.</p>","PeriodicalId":21396,"journal":{"name":"RMD Open","volume":"10 4","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11664365/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142966412","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
RMD OpenPub Date : 2024-12-12DOI: 10.1136/rmdopen-2024-004176
Teresa Grohmann, Arani Vivekanantham, Laura C Coates, Stephen Pennington, Oliver FitzGerald
{"title":"Clinical, genetic and omics-based biomarkers that might support the identification of the development of psoriatic arthritis in individuals with psoriasis: a narrative review of the literature.","authors":"Teresa Grohmann, Arani Vivekanantham, Laura C Coates, Stephen Pennington, Oliver FitzGerald","doi":"10.1136/rmdopen-2024-004176","DOIUrl":"10.1136/rmdopen-2024-004176","url":null,"abstract":"<p><p>It is known that 25%-30% of individuals with cutaneous psoriasis (PsC) will develop psoriatic arthritis (PsA). To date, the reasons for the development of PsA in individuals with PsC have not been identified. Furthermore, there are considerable delays in the diagnosis and treatment of PsA, which lead to joint and bone deformation and chronic pain. It is therefore important to develop more precise diagnostic and screening tools. In this narrative review of the literature, clinical risk factors and novel molecular biomarkers (genetic markers, blood and inflammatory markers, lipid, metabolite and protein biomarkers) have been evaluated. The review included 38 publications that were reported between May 2020 and May 2024. Similar to previous reviews, nail involvement was one of the strongest clinical risk factors for the development of PsA, while molecular biomarkers did not provide a clear and robust differentiation between PsC and PsA groups. The seemingly poor performance of molecular markers may be largely attributed to small study populations and heterogeneity in study designs. Data and sample sharing in large consortia such as HIPPOCRATES (Health initiatives in Psoriasis and PsOriatic arthritis ConsoRTium European States) could help to overcome the limitations of small studies and enable the development of more robust diagnostic and screening tools for PsA.</p>","PeriodicalId":21396,"journal":{"name":"RMD Open","volume":"10 4","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647361/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142822589","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
RMD OpenPub Date : 2024-12-12DOI: 10.1136/rmdopen-2024-004527corr1
{"title":"Correction: Frailty is independently associated with subclinical cardiovascular disease in patients with systemic lupus erythematosus.","authors":"","doi":"10.1136/rmdopen-2024-004527corr1","DOIUrl":"10.1136/rmdopen-2024-004527corr1","url":null,"abstract":"","PeriodicalId":21396,"journal":{"name":"RMD Open","volume":"10 4","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647324/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142822607","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
RMD OpenPub Date : 2024-12-12DOI: 10.1136/rmdopen-2024-004494
Iain B McInnes, Philipp Sewerin, Mohamed Sharaf, Michela Efficace, Frédéric Lavie, Miriam Zimmermann, Laura C Coates
{"title":"Sustained response to guselkumab regardless of baseline characteristics in patients with active psoriatic arthritis and inadequate response to TNF inhibitors: results from the phase 3b COSMOS clinical trial.","authors":"Iain B McInnes, Philipp Sewerin, Mohamed Sharaf, Michela Efficace, Frédéric Lavie, Miriam Zimmermann, Laura C Coates","doi":"10.1136/rmdopen-2024-004494","DOIUrl":"10.1136/rmdopen-2024-004494","url":null,"abstract":"<p><strong>Objectives: </strong>To prospectively evaluate the effect of guselkumab through 48 weeks across various clinical outcomes in subgroups of patients with psoriatic arthritis (PsA) and inadequate response to tumour necrosis factor inhibitors (TNFi-IR) from the phase 3b COSMOS trial. Subgroups were defined by baseline demographics, disease characteristics and prior/ongoing therapies.</p><p><strong>Methods: </strong>Patients with active PsA (tender joint count (TJC) and swollen joint count (SJC) both ≥3) and TNFi-IR were randomised 2:1 to receive guselkumab 100 mg at week 0, week 4, then every 8 weeks through week 44 or to placebo with cross-over to guselkumab 100 mg at week 16 (early escape) or week 24 (planned). Guselkumab effect on joints (American College of Rheumatology (ACR) 20/50/70, enthesitis, dactylitis), skin (Psoriasis Area and Severity Index 90/100, Investigator's Global Assessment 0/1), patient-reported outcomes (PROs) (Functional Assessment of Chronic Illness Therapy-Fatigue, Health Assessment Questionnaire-Disability Index) and composite outcome measures (PsA Disease Activity Score low disease activity, minimal disease activity) were evaluated by baseline patient age, sex, body mass index, SJC, TJC, PsA duration, %body surface area, C reactive protein, pain Visual Analogue Scale, number of prior TNFi and discontinuation reason, and conventional synthetic disease-modifying antirheumatic drug status. Results are descriptive only.</p><p><strong>Results: </strong>Baseline characteristics were similar between guselkumab (n=189) and placebo (n=96) groups. The benefit of guselkumab over placebo in achieving ACR 20 (primary endpoint; 50% vs 28%) and ACR 50 (23% vs 8%) response at week 24 was observed within all subgroups. Furthermore, response rates in the guselkumab group increased between week 24 and week 48 within almost all subgroups. Similar response patterns at week 24 and through week 48 were observed across various clinical outcomes.</p><p><strong>Conclusions: </strong>Guselkumab every 8 weeks led to consistent improvements through week 24 in joint, skin, PRO and composite outcomes versus placebo across diverse baseline-defined subgroups of TNFi-IR patients with PsA. Response rates increased or were durable through week 48 within most subgroups.</p>","PeriodicalId":21396,"journal":{"name":"RMD Open","volume":"10 4","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647317/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142822609","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
RMD OpenPub Date : 2024-12-12DOI: 10.1136/rmdopen-2023-003946corr2
{"title":"Correction: comparison of active tuberculosis occurrence associated with janus kinase inhibitors and biological DMARDs in rheumatoid arthritis.","authors":"","doi":"10.1136/rmdopen-2023-003946corr2","DOIUrl":"10.1136/rmdopen-2023-003946corr2","url":null,"abstract":"","PeriodicalId":21396,"journal":{"name":"RMD Open","volume":"10 4","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647380/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142822604","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Higher risk profile among patients with TET2-mutated giant cell arteritis: a cluster analysis.","authors":"Alexis F Guedon, Asmaa Ouafdi, Nabil Belfeki, Azeddine Dellal, Nouha Ghriss, Marc Scheen, Fadi Haidar, Olivier Espitia, Jean-Yves Scoazec, Olivier Fain, Christophe Marzac, Olivier Hermine, Eric Solary, Julien Rossignol, Arsène Mekinian","doi":"10.1136/rmdopen-2024-004694","DOIUrl":"10.1136/rmdopen-2024-004694","url":null,"abstract":"<p><strong>Objective: </strong>We aimed to assess the prevalence of clonal haematopoiesis (CH) in patients with giant cell arteritis (GCA) compared with controls and individuals with other autoimmune diseases (AIDs) and to identify high-risk clinical/genetic profiles that could influence disease outcomes.</p><p><strong>Methods: </strong>In a prospective observational study at three hospitals, we included 49 patients diagnosed with GCA, 48 patients with other AIDs and 27 control participants. We used next-generation sequencing to detect clonal haematopoiesis (CH) among them.</p><p><strong>Results: </strong>CH was detected in 55.1% of patients with GCA, 59.3% of controls and 18.8% of patients with other AIDs. The most commonly mutated genes in GCA and control groups were <i>DNMT3A</i> and <i>TET2</i>. No significant differences in CH prevalence were found between patients with GCA and controls or other AID when adjusted for age and sex. Cluster analysis revealed two distinct groups within the patients with GCA, one of which displayed a higher prevalence of TET2 and JAK2 variants, and was associated with worse prognosis.</p><p><strong>Conclusions: </strong>CH is prevalent among patients with GCA but does not differ significantly from controls or other autoimmune conditions. However, specific genetic profiles, particularly mutations in TET2 and JAK2, are associated with a higher risk cluster within the GCA cohort. This observation highlights the interest of detecting CH in patients with GCA in both routine practice and clinical trials for better risk stratification. Further prospective studies are needed to determine if management tailored to the genetic profile would improve outcomes.</p>","PeriodicalId":21396,"journal":{"name":"RMD Open","volume":"10 4","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647381/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142808003","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
RMD OpenPub Date : 2024-12-10DOI: 10.1136/rmdopen-2024-004097
Aoife M O'Byrne, Lisa G M van Baarsen
{"title":"Lymph nodes as gatekeepers of autoimmune diseases.","authors":"Aoife M O'Byrne, Lisa G M van Baarsen","doi":"10.1136/rmdopen-2024-004097","DOIUrl":"10.1136/rmdopen-2024-004097","url":null,"abstract":"<p><p>Secondary lymphoid organs such as lymph nodes (LNs) are the home of peripheral tolerance mechanisms which control autoreactive T cells and prevent immune responses to self-antigen. In systemic autoimmunity, there is a clear failure of these peripheral tolerance mechanisms that leads to chronic inflammation and tissue destruction, highlighting the role for LNs as possible gatekeepers of autoimmunity. In recent years there has been a shift in research focus towards tissue sites in autoimmune diseases ranging from type 1 diabetes to rheumatoid arthritis in an effort to better characterise pathogenesis and guide diagnostic and therapeutic decisions. Although this has yielded great insight, it fails to tackle the initial break in tolerance that initiates disease progression which is most likely originating in peripheral LNs. In the majority of autoimmune diseases a preclinical phase is recognised. This is characterised by the presence of autoantibodies, which is indicative of a break in immune tolerance, and the absence of clinically apparent inflammation or tissue destruction. This review explores how our current knowledge of LNs in the preclinical and established phases of autoimmune diseases provides insight into possibly shared pathological mechanisms that drive disease progression and highlight the gaps in our knowledge that may help uncover new therapeutic avenues for intervention and prevention.</p>","PeriodicalId":21396,"journal":{"name":"RMD Open","volume":"10 4","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647372/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142808005","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
RMD OpenPub Date : 2024-12-10DOI: 10.1136/rmdopen-2024-004775
Dafne Capelusnik, Elena Nikiphorou, Annelies Boonen, Robin Christensen, Désirée van der Heijde, Robert Landewé, Astrid van Tubergen, Sofia Ramiro
{"title":"Comparing the construct validity of measurement instruments for pain and stiffness in patients with axial spondyloartwhritis: cross-sectional analysis in the OASIS cohort.","authors":"Dafne Capelusnik, Elena Nikiphorou, Annelies Boonen, Robin Christensen, Désirée van der Heijde, Robert Landewé, Astrid van Tubergen, Sofia Ramiro","doi":"10.1136/rmdopen-2024-004775","DOIUrl":"10.1136/rmdopen-2024-004775","url":null,"abstract":"<p><strong>Objectives: </strong>To compare the construct validity, including discrimination between known groups, of three pain and three morning stiffness (MS) measurement instruments.</p><p><strong>Methods: </strong>Patients with radiographic axial spondyloarthritis with 8-year data from the Outcome in Ankylosing Spondylitis International Study cohort were assessed cross-sectionally. Three instruments for pain and three for MS, all self-reported and scored 0-10, were compared. Construct validity was evaluated by testing (1) hypothesis of correlations' strength and (2) discrimination between known groups using standardised mean differences (SMD) across external constructs. Influence of contextual factors (CFs) on SMDs was investigated.</p><p><strong>Results: </strong>Of 85 patients, mean age was 54 (SD 11), mean symptom duration 31 (11) years, 71% males. All six instruments showed a good construct validity by fulfilling >75% of the hypotheses for the strength of correlation. Neck/back/hip pain (Bath Ankylosing Spondylitis Disease Activity Index-Question 2, BASDAI-Q2) and total back pain had higher SMDs compared with back pain at night across all between-group comparisons, with BASDAI-Q2 performing mostly slightly better (eg, SMD for external construct Axial Spondyloarthritis Disease Activity Score (ASDAS; ≥2.1 vs <2.1): 1.87 (BASDAI-Q2) vs 1.56 (total back pain) vs 1.07 (back pain at night)). MS-severity and severity/duration had higher SMDs across all external constructs (with MS-severity slightly better), while MS-duration performed worse (eg, SMD external construct ASDAS: 1.51 (MS-severity) and 1.39 (MS-severity/duration) vs 1.16 (MS-duration)). Influence of CFs on known group discrimination was limited.</p><p><strong>Conclusions: </strong>The recommended Assessment of SpondyloArthritis international Society Core Outcome Set (ASAS-COS) pain measurement instrument total back pain BASDAI-Q2 has the best known group discrimination. For MS, the ASAS-COS stiffness measure (MS-severity/duration) performs well although MS-severity even slightly better. Known group discrimination is overall stable across CFs.</p>","PeriodicalId":21396,"journal":{"name":"RMD Open","volume":"10 4","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647358/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142808000","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
RMD OpenPub Date : 2024-12-09DOI: 10.1136/rmdopen-2024-004918
Alexis F Guédon, Fabrice Carrat, Luc Mouthon, David Launay, Benjamin Chaigne, Grégory Pugnet, Jean-Christophe Lega, Arnaud Hot, Vincent Cottin, Christian Agard, Yannick Allanore, Anne Laure Fauchais, Alain Lescoat, Robin Dhote, Thomas Papo, Emmanuel Chatelus, Bernard Bonnotte, Jean-Emmanuel Kahn, Elisabeth Diot, Achille Aouba, Nadine Magy-Bertrand, Viviane Queyrel, Alain Le Quellec, Pierre Kieffer, Zahir Amoura, Brigitte Granel, Jean Baptiste Gaultier, Marie-Hélène Balquet, Denis Wahl, Olivier Lidove, Olivier Espitia, Ariel Cohen, Olivier Fain, Eric Hachulla, Arsène Mekinian, Sébastien Rivière
{"title":"Vasodilator drugs and heart-related outcomes in systemic sclerosis: an exploratory analysis.","authors":"Alexis F Guédon, Fabrice Carrat, Luc Mouthon, David Launay, Benjamin Chaigne, Grégory Pugnet, Jean-Christophe Lega, Arnaud Hot, Vincent Cottin, Christian Agard, Yannick Allanore, Anne Laure Fauchais, Alain Lescoat, Robin Dhote, Thomas Papo, Emmanuel Chatelus, Bernard Bonnotte, Jean-Emmanuel Kahn, Elisabeth Diot, Achille Aouba, Nadine Magy-Bertrand, Viviane Queyrel, Alain Le Quellec, Pierre Kieffer, Zahir Amoura, Brigitte Granel, Jean Baptiste Gaultier, Marie-Hélène Balquet, Denis Wahl, Olivier Lidove, Olivier Espitia, Ariel Cohen, Olivier Fain, Eric Hachulla, Arsène Mekinian, Sébastien Rivière","doi":"10.1136/rmdopen-2024-004918","DOIUrl":"10.1136/rmdopen-2024-004918","url":null,"abstract":"<p><strong>Background and aims: </strong>Systemic sclerosis (SSc) is an autoimmune connective disease characterised by excessive extracellular matrix deposition and widespread skin and internal organ fibrosis including various cardiac manifestations. Heart involvement is one of the leading causes of death among patients with SSc. In this study, we aimed to assess the effect of various vasodilator treatments.</p><p><strong>Methods: </strong>We used data from a national multicentric prospective study using the French SSc national database. We estimated the average treatment effect (ATE) of sildenafil, bosentan, angiotensin-converting enzyme (ACE) inhibitors and iloprost on diastolic dysfunction, altered ejection fraction <50% and pulmonary arterial hypertension (PAH) using a causal method, namely the longitudinal targeted minimum loss-based estimation, to adjust for confounding and informative censoring.</p><p><strong>Results: </strong>We included 1048 patients with available data regarding treatment. Regarding sildenafil analyses, the ATE on diastolic dysfunction at 3 years was -2.83% (95% CI -4.06; -1.60, p<0.00001), and the estimated ATE on altered ejection fraction <50% was -0.88% (95% CI -1.70; -0.05, p=0.037). We did not find a significative effect on PAH. Regarding bosentan, ACE inhibitors and iloprost, none of them neither showed a significant effect on diastolic dysfunction, altered ejection fraction <50% or PAH.</p><p><strong>Conclusions: </strong>Using causal methods, our study is the first and largest suggesting that sildenafil might have benefits among SSc patients regarding diastolic dysfunction and altered ejection fraction occurrence. However, further studies assessing the effect of vasodilators on heart-related outcome among SSc patients are needed to confirm those exploratory results.</p>","PeriodicalId":21396,"journal":{"name":"RMD Open","volume":"10 4","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11629012/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142807953","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}