Journal of Scleroderma and Related Disorders最新文献

{"title":"Critical digital ischaemia in systemic sclerosis exacerbated by multiple myeloma: A case report.","authors":"Andre Silva Franco, Gabriel Berlingieri Polho, Ana Paula Luppino Assad, Renata Miossi, Percival Degrava Sampaio-Barros","doi":"10.1177/23971983231171248","DOIUrl":"10.1177/23971983231171248","url":null,"abstract":"<p><strong>Introduction: </strong>The overlapping of systemic sclerosis with hematologic malignancy has been described previously in the literature. This case report presents a patient with systemic sclerosis and multiple myeloma who had severe digital ischaemia that culminated in the amputation of several fingers.</p><p><strong>Case report: </strong>A 65-year-old White female patient was diagnosed with limited systemic sclerosis in 2002, smouldering multiple myeloma IgG/kappa in 2017 and liver cirrhosis in 2018 due to autoimmune hepatitis. In 2021, she was admitted to the emergency room with dry ischaemia of all fingers and toes despite optimized therapy, associated with visual blurring. The diagnostic hypothesis was hyperviscosity syndrome associated with multiple myeloma reactivation. The patient underwent chemotherapy and despite initial laboratory improvement, 19 digits required amputation.</p><p><strong>Conclusion: </strong>Although the association between systemic sclerosis and multiple myeloma is rare, it should be remembered in cases of significant worsening of Raynaud's phenomenon. Causes unrelated to systemic sclerosis should also be considered in the presence of severe exacerbations in patients with other comorbidities.</p>","PeriodicalId":17036,"journal":{"name":"Journal of Scleroderma and Related Disorders","volume":"8 3","pages":"NP9-NP13"},"PeriodicalIF":1.4,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10515990/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41129072","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 survey of Australian rheumatologists' perspectives of nutrition needs in systemic sclerosis.","authors":"De-Arne A Samm,&nbsp;Aimee R Macoustra,&nbsp;Rhiannon K Crane,&nbsp;Leah M McWilliams,&nbsp;Susanna M Proudman,&nbsp;Lee-Anne S Chapple","doi":"10.1177/23971983231185465","DOIUrl":"https://doi.org/10.1177/23971983231185465","url":null,"abstract":"<p><strong>Aim: </strong>Systemic sclerosis (scleroderma) is an incurable inflammatory condition synonymous with unique nutrition needs. As rheumatologists are frequently responsible for managing the various organ manifestations, this study aimed to understand the service needs and nutritional concerns of rheumatologists involved in the care of adults with systemic sclerosis.</p><p><strong>Methods: </strong>A 13-item online qualitative and quantitative survey was distributed via REDCap^® from January to March 2022 to rheumatologists who are members of the Australian Scleroderma Interest Group and consult patients with systemic sclerosis. Data were collected on rheumatologists' demographics, and their views on symptoms observed, nutrition concerns and priorities, and preferred dietetic service provision for their patients. Data are reported as number (%).</p><p><strong>Results: </strong>Of 27 eligible rheumatologists, 17 (63%) completed the survey. All rheumatologists reported gastrointestinal symptoms in their patients (<i>n</i> = 17, 100%); predominantly reflux (<i>n</i> = 17, 100%) and dysphagia (<i>n</i> = 17, 100%). Weight loss was observed by the majority of rheumatologists (<i>n</i> = 15, 88%). Rheumatologists reported patients used food avoidance/special diets to manage symptoms (<i>n</i> = 12, 71%). Dietetic consultation was reported as potentially beneficial by all rheumatologists, with the preferred time being when symptoms increase or change (<i>n</i> = 15, 88%), and the preferred approaches being written resources (<i>n</i> = 15, 88%), face-to-face (<i>n</i> = 14, 82%) and telephone consultation (<i>n</i> = 14, 82%). Advice on gaining weight (<i>n</i> = 14, 82%) and systemic sclerosis symptom management (<i>n</i> = 13, 77%) were the most desired education topics reported.</p><p><strong>Conclusion: </strong>Rheumatologists commonly observe gastrointestinal symptoms in patients with systemic sclerosis and report dietetics services would be advantageous in supporting their patients to gain weight and better manage their symptoms.</p>","PeriodicalId":17036,"journal":{"name":"Journal of Scleroderma and Related Disorders","volume":"8 3","pages":"203-209"},"PeriodicalIF":2.0,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10515988/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41099797","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":"Myositis mimics in scleroderma: A case series of neuromuscular diseases that can co-exist in scleroderma","authors":"Emma Joyce, Fredrick M Wigley, Laura K Hummers, Julie J Paik","doi":"10.1177/23971983231197442","DOIUrl":"https://doi.org/10.1177/23971983231197442","url":null,"abstract":"We present a case series of four patients with systemic sclerosis and skeletal myopathy. While idiopathic inflammatory myopathies, or myositis, are thought to be the most common type of muscle disease seen in systemic sclerosis, we highlight four cases where unique clinical findings and careful assessment ruled out myositis mimics. Key diagnostic tools that can be helpful for clinicians to diagnose a neuromuscular disease are also detailed in this report.","PeriodicalId":17036,"journal":{"name":"Journal of Scleroderma and Related Disorders","volume":"10 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135154406","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Altered pathways of keratinization, extracellular matrix generation, angiogenesis, and stromal stem cells proliferation in patients with systemic sclerosis.","authors":"Amelia Spinella, Domenico Lo Tartaro, Lara Gibellini, Marco de Pinto, Valentina Pinto, Elisa Bonetti, Francesca Lolli, Melba Lattanzi, Federica Lumetti, Gabriele Amati, Giorgio De Santis, Andrea Cossarizza, Carlo Salvarani, Dilia Giuggioli","doi":"10.1177/23971983221130145","DOIUrl":"10.1177/23971983221130145","url":null,"abstract":"<p><strong>Objective: </strong>Systemic sclerosis is characterized by endothelial dysfunction, autoimmunity abnormalities, and fibrosis of the skin and internal organs. The pathogenetic mechanisms underlying systemic sclerosis vasculopathy are still not clarified. A complex cellular and extracellular network of interactions has been studied, but it is currently unclear what drives the activation of fibroblasts/myofibroblasts and the extracellular matrix deposition.</p><p><strong>Methods: </strong>Using RNA sequencing, the aim of the work was to identify potential functional pathways implied in systemic sclerosis pathogenesis and markers of endothelial dysfunction and fibrosis in systemic sclerosis patients. RNA-sequencing analysis was performed on RNA obtained from biopsies from three systemic sclerosis patients and three healthy controls enrolled in our University Hospital. RNA was used to generate sequencing libraries that were sequenced according to proper transcriptomic analyses. Subsequently, we performed gene set enrichment analysis of differentially expressed genes on the entire list of genes that compose the RNA-sequencing expression matrix.</p><p><strong>Results: </strong>Gene set enrichment analysis revealed that healthy controls were characterized by gene signatures related to stromal stem cells proliferation, cytokine-cytokine receptor interaction, macrophage-enriched metabolic network, whereas systemic sclerosis tissues were enriched in signatures associated with keratinization, cornification, retinoblastoma 1 and tumor suppressor 53 signaling.</p><p><strong>Conclusion: </strong>According to our data, RNA-sequencing and pathway analysis revealed that systemic sclerosis subjects display a discrete pattern of gene expression associated with keratinization, extracellular matrix generation, and negative regulation of angiogenesis and stromal stem cells proliferation. Further analysis on larger numbers of patients is needed; however, our findings provide an interesting framework for the development of biomarkers useful to explore potential future therapeutic approaches.</p>","PeriodicalId":17036,"journal":{"name":"Journal of Scleroderma and Related Disorders","volume":"8 2","pages":"151-166"},"PeriodicalIF":2.0,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10242696/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9599246","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":"Cardiotoxicity in autologous haematopoietic stem cell transplantation for systemic sclerosis.","authors":"Ross Penglase, Laila Girgis, Helen Englert, Xavier Brennan, Andrew Jabbour, Eugene Kotlyar, David Ma, John Moore","doi":"10.1177/23971983221145639","DOIUrl":"10.1177/23971983221145639","url":null,"abstract":"<p><p>Autologous haematopoietic stem cell transplantation is now well-established as an effective treatment for severe systemic sclerosis with clear demonstration of favourable end-organ and survival outcomes. Treatment-related cardiotoxicity remains the predominant safety concern and contraindicates autologous haematopoietic stem cell transplantation in patients with severe cardiopulmonary disease. In this review, we describe the cardiovascular outcomes of autologous haematopoietic stem cell transplantation recipients, discuss the potential mechanisms of cardiotoxicity and propose future mitigating strategies.</p>","PeriodicalId":17036,"journal":{"name":"Journal of Scleroderma and Related Disorders","volume":"8 2","pages":"87-100"},"PeriodicalIF":2.0,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10242691/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10646367","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":"Subcutaneous immunoglobulin therapy for refractory skin thickening in rapidly progressive systemic sclerosis: A case report and literature review.","authors":"Fabio Cacciapaglia, Stefano Stano, Marco Fornaro, Florenzo Iannone","doi":"10.1177/23971983221124180","DOIUrl":"10.1177/23971983221124180","url":null,"abstract":"<p><p>The use of immunoglobulin is a therapeutic option with increasing evidence of efficacy for different rheumatologic autoimmune systemic diseases. Some studies concerning immunoglobulin use in systemic sclerosis have been published with encouraging results. We present the case of a young woman diagnosed with rapidly progressive diffuse cutaneous systemic sclerosis, refractory to therapy with methotrexate and rituximab, which presented a relevant skin improvement after one year of subcutaneous immunoglobulin (2 g/kg cumulative monthly dose, refracted in weekly administrations). Furthermore, a narrative literature review of the evidence for alternative treatments with a focus on immunoglobulin use for systemic sclerosis skin involvement was carried out.</p>","PeriodicalId":17036,"journal":{"name":"Journal of Scleroderma and Related Disorders","volume":"8 2","pages":"101-106"},"PeriodicalIF":2.0,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10242697/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9599244","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":"Relapsing polychondritis in systemic sclerosis: A rare vasculitic mimic.","authors":"Carolina Teles,&nbsp;Chiranthi Kongala Liyanage,&nbsp;Geoffrey Chow,&nbsp;Christopher P Denton,&nbsp;Voon Ong","doi":"10.1177/23971983221141599","DOIUrl":"https://doi.org/10.1177/23971983221141599","url":null,"abstract":"<p><strong>Introduction: </strong>Relapsing polychondritis is a rare, immune-mediated disease characterised by inflammation of cartilaginous structures. Auricular chondritis, sparing the fatty lobule, is the most typical feature, followed by nose and laryngotracheal involvement. Albeit rare, neurologic involvement is reported with relapsing polychondritis. Cranial nerve involvement is the most frequent neurologic manifestation and is probably due to an underlying vasculitic process. Approximately one-third of relapsing polychondritis patients can overlap with other systemic diseases, including other autoimmune connective tissue diseases, but association with systemic sclerosis has very rarely been described.</p><p><strong>Case description: </strong>A 63-year-old woman presented with acute new-onset severe dysphagia, accompanied by hoarseness and preceded by pain, swelling and erythema of the left pinna, unresponsive to antibiotics. She had a history of long-standing limited cutaneous systemic sclerosis. Cranial nerve examination revealed right-sided palatal palsy, and left vocal cord palsy was found on fibreoptic nasendoscopy. Magnetic resonance imaging of the head and neck showed bilateral enhancement of an extracranial segment of the glossopharyngeal and vagus nerves. Clinical features and imaging findings were consistent with relapsing polychondritis, which successfully responded to high-dose steroids.</p><p><strong>Conclusions: </strong>This is a case of relapsing polychondritis mimicking progression of systemic sclerosis, showcasing its challenging features. It emphasises the importance of early diagnosis and prompt management with potential impact on the outcome, while highlighting the complex interplay between these two disease entities and vasculitic mechanisms, which may reflect the shared network of genetic predisposition across autoimmune rheumatic diseases.</p>","PeriodicalId":17036,"journal":{"name":"Journal of Scleroderma and Related Disorders","volume":"8 2","pages":"NP4-NP8"},"PeriodicalIF":2.0,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10242694/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9599245","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":"Occupation as a gendered-role and outcome in systemic sclerosis.","authors":"Fatema Alkhamees,&nbsp;Oriana Hoi Yun Yu,&nbsp;Mianbo Wang,&nbsp;Marie Hudson","doi":"10.1177/23971983221143599","DOIUrl":"https://doi.org/10.1177/23971983221143599","url":null,"abstract":"<p><strong>Objective: </strong>Sex and gender are of growing scientific interest in disease onset and course. While sex differences have been shown to exist in systemic sclerosis, there is a paucity of data on gender. Our objective was to examine the association between occupation, a gender-related role and outcomes in systemic sclerosis.</p><p><strong>Methods: </strong>An occupation score ranging from 0 to 100, with lower scores representing occupations traditionally held by men and higher scores traditionally held by women, was constructed using the National Occupational Classification 2016 and data from Statistics Canada. Subjects in the Canadian Scleroderma Research Group registry were assigned an occupation score based on self-reported occupation. Multivariate models, adjusted for sex, age, smoking and education were used to estimate the independent effect of occupation score on systemic sclerosis outcomes.</p><p><strong>Results: </strong>We included 1104 subjects, of which 961 were females (87%) and 143 (13%) males. There were differences between females versus males: disease duration (9.9 vs 7.6 years, <i>p</i> = 0.002), diffuse disease (35% vs 54%, <i>p</i> < 0.001), interstitial lung disease (28% vs 37%, <i>p</i> = 0.021) and pulmonary hypertension (10% vs 4%, <i>p</i> = 0.033), but not pain, response to treatment and mortality. The median occupation scores differed between females and males (84.3 (interquartile range 56.8, 89.4) vs 24.9 (4.3, 54.1), <i>p</i> < 0.001). The Spearman correlation between sex and occupation score was 0.44, indicating a weak correlation. In adjusted analyses, occupation score was not an independent predictor of disease subset (diffuse vs limited), interstitial lung disease, pulmonary hypertension, pain, response to treatment or mortality.</p><p><strong>Conclusion: </strong>We did not find independent associations between an occupation score, a gender-related role and outcomes in systemic sclerosis. These results should be interpreted with caution as occupation may be a poor measure of gender. Future research using a validated measure of gender will be needed to generate robust data on the effect of gender in systemic sclerosis.</p>","PeriodicalId":17036,"journal":{"name":"Journal of Scleroderma and Related Disorders","volume":"8 2","pages":"131-136"},"PeriodicalIF":2.0,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/4c/02/10.1177_23971983221143599.PMC10242688.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9599243","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":"COVID-19 and protection of vaccination in patients with systemic sclerosis-associated interstitial lung disease.","authors":"Stylianos Panopoulos,&nbsp;Vasilios Tzilas,&nbsp;Vasiliki-Kalliopi Bournia,&nbsp;Anastasios Karamanakos,&nbsp;Katerina Laskari,&nbsp;Demosthenes Bouros,&nbsp;Maria Tektonidou,&nbsp;Petros P Sfikakis","doi":"10.1177/23971983221143252","DOIUrl":"https://doi.org/10.1177/23971983221143252","url":null,"abstract":"<p><strong>Objectives: </strong>Data on COVID-19 in patients with interstitial lung disease are scarce and whether SARS-CoV-2 may trigger interstitial lung disease progression remains unknown. We aimed to analyze outcomes of COVID-19 in patients with systemic sclerosis-associated interstitial lung disease, including possible thoracic radiographic progression.</p><p><strong>Patients and methods: </strong>All 43 patients with systemic sclerosis-associated interstitial lung disease followed in our center (mean ± SD, 55.2 ± 11.6 years, 36 female) with confirmed SARS-CoV2 infection up to 1 September 2022 were analyzed. Individual interstitial lung disease extent on high resolution CT (HRCT) performed before (up to 3 months) and after COVID-19 (2-5 months) was compared.</p><p><strong>Results: </strong>At SARS-CoV-2 infection, 9/43 patients were unvaccinated, whereas 5, 26, and 3 had received 2, 3, or 4 doses of an mRNA vaccine, respectively. Thirty-one patients were either on monotherapy with immunosuppressives (mycophenolate, <i>n</i> = 7; cyclophosphamide, <i>n</i> = 2; methotrexate, <i>n</i> = 10; tocilizumab, <i>n</i> = 7; rituximab, <i>n</i> = 1; etanercept, <i>n</i> = 1), or their combinations (<i>n</i> = 3). Eight patients (20%), of whom four unvaccinated, required hospitalization for pneumonia and three (7%) died of acute respiratory failure (<i>n</i> = 2, both unvaccinated) or cardiac arrest. Lack of vaccination was the only independent predictor for hospitalization (OR = 7.98, 95% CI: 1.25-51.09) and marginally for death (OR = 32.7, 95% CI: 0.97-1110.98), regardless of the presence of diffuse systemic sclerosis, interstitial lung disease extent greater than 20% or immunosuppressive treatment. In 22 patients with available HRCT pairs (vaccinated = 20), the interstitial lung disease extent before COVID-19 (20.4%± 17.8%) remained unchanged (22.4% ± 18.5%) in all but one patient.</p><p><strong>Conclusion: </strong>SARS-CoV-2 vaccination is of outmost importance for every systemic sclerosis patient with interstitial lung disease. COVID-19 does not seem to promote progression of systemic sclerosis-associated interstitial lung disease in vaccinated patients, but further studies are warranted.</p>","PeriodicalId":17036,"journal":{"name":"Journal of Scleroderma and Related Disorders","volume":"8 2","pages":"113-119"},"PeriodicalIF":2.0,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9755035/pdf/10.1177_23971983221143252.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9593195","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":"Gender differences in juvenile systemic sclerosis patients: Results from the international juvenile scleroderma inception cohort.","authors":"Ivan Foeldvari, Jens Klotsche, Ozgur Kasapcopur, Amra Adrovic, Maria Teresa Terreri, Ana Paula Sakamoto, Valda Stanevicha, Jordi Anton, Brian M Feldman, Flavio Sztajnbok, Raju Khubchandani, Ekaterina Alexeeva, Maria Katsicas, Sujata Sawhney, Vanessa Smith, Simone Appenzeller, Tadej Avcin, Mikhail Kostik, Thomas Lehman, Edoardo Marrani, Dieneke Schonenberg-Meinema, Walter-Alberto Sifuentes-Giraldo, Natalia Vasquez-Canizares, Mahesh Janarthanan, Monika Moll, Dana Nemcova, Anjali Patwardhan, Maria Jose Santos, Cristina Battagliotti, Lillemor Berntson, Blanca Bica, Jürgen Brunner, Rolando Cimaz, Patricia Costa-Reis, Despina Eleftheriou, Liora Harel, Gerd Horneff, Sindhu R Johnson, Daniela Kaiser, Tilmann Kallinich, Dragana Lazarevic, Kirsten Minden, Susan Nielsen, Farzana Nuruzzaman, Siri Opsahl Hetlevik, Yosef Uziel, Nicola Helmus, Kathryn S Torok","doi":"10.1177/23971983221143244","DOIUrl":"10.1177/23971983221143244","url":null,"abstract":"<p><strong>Objective: </strong>To compare organ involvement and disease severity between male and female patients with juvenile onset systemic sclerosis.</p><p><strong>Methods: </strong>Demographics, organ involvement, laboratory evaluation, patient-reported outcomes and physician assessment variables were compared between male and female juvenile onset systemic sclerosis patients enrolled in the prospective international juvenile systemic sclerosis cohort at their baseline visit and after 12 months.</p><p><strong>Results: </strong>One hundred and seventy-five juvenile onset systemic sclerosis patients were evaluated, 142 females and 33 males. Race, age of onset, disease duration, and disease subtypes (70% diffuse cutaneous) were similar between males and females. Active digital ulceration, very low body mass index, and tendon friction rubs were significantly more frequent in males. Physician global assessment of disease severity and digital ulcer activity was significantly higher in males. Composite pulmonary involvement was also more frequent in males, though not statistically significantly. After 12 months, they are the pattern of differences changed female patients had significantly more frequent pulmonary involvement.</p><p><strong>Conclusion: </strong>In this cohort, juvenile onset systemic sclerosis had a more severe course in males at baseline and but the pattern changed after 12 months. Some differences from adult findings persisted, there is no increased signal of pulmonary arterial hypertension or heart failure in male pediatric patients. While monitoring protocols of organ involvement in juvenile onset systemic sclerosis need to be identical for males and females.</p>","PeriodicalId":17036,"journal":{"name":"Journal of Scleroderma and Related Disorders","volume":"8 2","pages":"120-130"},"PeriodicalIF":2.0,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10242693/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9653004","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}