Pub Date : 2024-08-03DOI: 10.1016/j.semarthrit.2024.152526
Caroline A. Flurey , Bethan Jones , Ummugulsum Gazel , Chikosolu Uzoka , Kate Rosser , Thomas Khoo , Marieke Voshaar , Wijnanda Hoogland , Beverley Shea , Lynn March , Dorcas Beaton , Peter Tugwell , Susanna Proudman , OMERACT remission in RA: Patient perspective working group
Aims
Our previous work identified pain, fatigue, and independence as missing from the ACR/EULAR rheumatoid arthritis (RA) remission criteria from the patient perspective. Validated measures exist for pain and fatigue, but not for independence. As a first step towards developing such a measure, this study aimed to understand ‘Independence’ in the context of RA remission from the patient perspective.
Methods
International qualitative research study comprising five focus groups of 19 participants with RA. Data were analysed using reflexive thematic analysis.
Results
Five overarching themes were identified, underpinned by a construct of “stages of independence”. Independence means at least being ‘physically and functionally able’ but may go beyond this and enable ‘participation beyond function’, ‘cognitive independence’, and ‘having or taking control’. There was no agreement on whether assistance is an aid to independence or undermines ability to achieve independence (‘assistance is complicated’). The construct “Stages of independence” acknowledges that Independence may mean different things to different patients and there may be other factors beyond disease activity that hold patients in each of these stages.
Conclusion
These novel data suggest a desirable definition of independence includes full active participation without the need to consider or work around disease activity, and cognitive independence from thoughts of RA. Independence in RA remission is a complex concept and next steps will be to seek patient and professional agreement on the most important issues raised in these focus groups to take forward to developing a measure for independence in the context of RA remission from the patient perspective.
{"title":"“It means almost forgetting that you've got a disease”: An OMERACT study to define independence in the context of rheumatoid arthritis remission from the patient perspective","authors":"Caroline A. Flurey , Bethan Jones , Ummugulsum Gazel , Chikosolu Uzoka , Kate Rosser , Thomas Khoo , Marieke Voshaar , Wijnanda Hoogland , Beverley Shea , Lynn March , Dorcas Beaton , Peter Tugwell , Susanna Proudman , OMERACT remission in RA: Patient perspective working group","doi":"10.1016/j.semarthrit.2024.152526","DOIUrl":"10.1016/j.semarthrit.2024.152526","url":null,"abstract":"<div><h3>Aims</h3><p>Our previous work identified pain, fatigue, and independence as missing from the ACR/EULAR rheumatoid arthritis (RA) remission criteria from the patient perspective. Validated measures exist for pain and fatigue, but not for independence. As a first step towards developing such a measure, this study aimed to understand ‘Independence’ in the context of RA remission from the patient perspective.</p></div><div><h3>Methods</h3><p>International qualitative research study comprising five focus groups of 19 participants with RA. Data were analysed using reflexive thematic analysis.</p></div><div><h3>Results</h3><p>Five overarching themes were identified, underpinned by a construct of “stages of independence”. Independence means at least being ‘physically and functionally able’ but may go beyond this and enable ‘participation beyond function’, ‘cognitive independence’, and ‘having or taking control’. There was no agreement on whether assistance is an aid to independence or undermines ability to achieve independence (‘assistance is complicated’). The construct “Stages of independence” acknowledges that Independence may mean different things to different patients and there may be other factors beyond disease activity that hold patients in each of these stages.</p></div><div><h3>Conclusion</h3><p>These novel data suggest a desirable definition of independence includes full active participation without the need to consider or work around disease activity, and cognitive independence from thoughts of RA. Independence in RA remission is a complex concept and next steps will be to seek patient and professional agreement on the most important issues raised in these focus groups to take forward to developing a measure for independence in the context of RA remission from the patient perspective.</p></div>","PeriodicalId":21715,"journal":{"name":"Seminars in arthritis and rheumatism","volume":null,"pages":null},"PeriodicalIF":4.6,"publicationDate":"2024-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0049017224001665/pdfft?md5=870f1a7769d0fd55e20652b34e7e72dd&pid=1-s2.0-S0049017224001665-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141913889","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}
Pub Date : 2024-08-03DOI: 10.1016/j.semarthrit.2024.152527
Anqi Zhang , Elisabeth Brouwer , Maria Sandovici , Arjan Diepstra , William F. Jiemy , Kornelis S.M. van der Geest
Objective
To summarize current insights on the immune pathology of bursitis caused by rheumatic inflammatory diseases, degenerative conditions, or mechanical stress and identify knowledge gaps in this field. Data on tenosynovitis pathology was included for comparison.
Methods
We performed a systematic review encompassing an electronic database search of all published literatures in PubMed/MEDLINE from inception to February 13, 2023, investigating the immunological changes occurring in the bursa of patients with inflammatory rheumatic diseases, degenerative conditions or mechanical stress (e.g., impingement syndrome).
Results
Thirty-two articles provided data on the immune pathology of bursal tissue inflammation were identified. Histological and immunological perturbations included alterations of tissue morphology, infiltration of macrophages and some T cells, and enhanced expression of proinflammatory cytokines, such as interleukin (IL)-6, IL-1β and tumor necrosis factor alpha (TNF-α). These changes were described for all three underlying causes, although studies on bursitis associated with rheumatic inflammatory diseases were rare. Fibrosis was only reported in subacromial bursitis caused by mechanical stress within our included studies.
Conclusion
Current insights on bursitis were outdated and studies on bursitis associated with rheumatic inflammatory diseases are particularly lacking. Substantial overlap of enhanced expression of IL-6, IL-1β, TNF-α and infiltrating macrophages were found in bursitis irrespective of the underlying cause. In depth investigation on bursitis such as high throughput multi-omics are urgently needed to guide disease-specific therapeutic management.
{"title":"The immune pathology of bursitis in rheumatic inflammatory diseases, degenerative conditions and mechanical stress: A systematic review","authors":"Anqi Zhang , Elisabeth Brouwer , Maria Sandovici , Arjan Diepstra , William F. Jiemy , Kornelis S.M. van der Geest","doi":"10.1016/j.semarthrit.2024.152527","DOIUrl":"10.1016/j.semarthrit.2024.152527","url":null,"abstract":"<div><h3>Objective</h3><p>To summarize current insights on the immune pathology of bursitis caused by rheumatic inflammatory diseases, degenerative conditions, or mechanical stress and identify knowledge gaps in this field. Data on tenosynovitis pathology was included for comparison.</p></div><div><h3>Methods</h3><p>We performed a systematic review encompassing an electronic database search of all published literatures in PubMed/MEDLINE from inception to February 13, 2023, investigating the immunological changes occurring in the bursa of patients with inflammatory rheumatic diseases, degenerative conditions or mechanical stress (e.g., impingement syndrome).</p></div><div><h3>Results</h3><p>Thirty-two articles provided data on the immune pathology of bursal tissue inflammation were identified. Histological and immunological perturbations included alterations of tissue morphology, infiltration of macrophages and some T cells, and enhanced expression of proinflammatory cytokines, such as interleukin (IL)-6, IL-1β and tumor necrosis factor alpha (TNF-α). These changes were described for all three underlying causes, although studies on bursitis associated with rheumatic inflammatory diseases were rare. Fibrosis was only reported in subacromial bursitis caused by mechanical stress within our included studies.</p></div><div><h3>Conclusion</h3><p>Current insights on bursitis were outdated and studies on bursitis associated with rheumatic inflammatory diseases are particularly lacking. Substantial overlap of enhanced expression of IL-6, IL-1β, TNF-α and infiltrating macrophages were found in bursitis irrespective of the underlying cause. In depth investigation on bursitis such as high throughput multi-omics are urgently needed to guide disease-specific therapeutic management.</p></div>","PeriodicalId":21715,"journal":{"name":"Seminars in arthritis and rheumatism","volume":null,"pages":null},"PeriodicalIF":4.6,"publicationDate":"2024-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0049017224001677/pdfft?md5=1a9606a1942069ea00001dc1a1641360&pid=1-s2.0-S0049017224001677-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141985664","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}
Pub Date : 2024-07-31DOI: 10.1016/j.semarthrit.2024.152524
Laura Bricman , Clément Triaille , Emilie Sapart , Tatiana Sokolova , Aleksandra Avramovska , Francesco Natalucci , Thomas Kirchgesner , Patrick Durez
Background
Rheumatoid arthritis (RA) is classically considered a systemic disorder, but the role of local factors in driving synovial inflammation is increasingly being recognized. These joint-specific factors may consequently modulate disease phenotype.
Objectives
Our goal was to study the spatial distribution of swelling, tenderness and erosions in a large cohort of early RA (ERA) patients, to assess for patterns of simultaneously-involved joint clusters. We also aimed to investigate the link between arthritis localization and phenotypic features such as bone erosions and response to methotrexate therapy.
Methods
DMARD-naive patients from the ERA UCLouvain Brussels cohort were included. Forty-four joints were clinically assessed for swelling and tenderness before treatment, and 6 months later for methotrexate-treated patients. Clusters of joints were identified using Principal component analysis and Cramer's correlation coefficients. Frequency of bone erosions and joint-specific response to methotrexate were compared across different clusters.
Results
452 ERA patients were included. Analysis of the spatial distribution of swelling and tenderness allowed for the identification of 3 joint clusters that showed significant simultaneous involvement: (i) MTP1–5 joints, (ii) hand joints (MCPs and PIPs), and (iii) larger joints. These clusters were associated with different susceptibility to bone erosions and distinct clinical features, but similar local response (joint swelling resolution) to methotrexate.
Conclusion
This is the first study investigating the spatial distribution of arthritis in a large cohort of early RA using an unbiased approach. We identify clusters of simultaneously involved joints, supporting the importance of local factors in driving synovitis in RA.
{"title":"Analysis of synovitis patterns in early RA supports the importance of joint-specific factors","authors":"Laura Bricman , Clément Triaille , Emilie Sapart , Tatiana Sokolova , Aleksandra Avramovska , Francesco Natalucci , Thomas Kirchgesner , Patrick Durez","doi":"10.1016/j.semarthrit.2024.152524","DOIUrl":"10.1016/j.semarthrit.2024.152524","url":null,"abstract":"<div><h3>Background</h3><p>Rheumatoid arthritis (RA) is classically considered a systemic disorder, but the role of local factors in driving synovial inflammation is increasingly being recognized. These joint-specific factors may consequently modulate disease phenotype.</p></div><div><h3>Objectives</h3><p>Our goal was to study the spatial distribution of swelling, tenderness and erosions in a large cohort of early RA (ERA) patients, to assess for patterns of simultaneously-involved joint clusters. We also aimed to investigate the link between arthritis localization and phenotypic features such as bone erosions and response to methotrexate therapy.</p></div><div><h3>Methods</h3><p>DMARD-naive patients from the ERA UCLouvain Brussels cohort were included. Forty-four joints were clinically assessed for swelling and tenderness before treatment, and 6 months later for methotrexate-treated patients. Clusters of joints were identified using Principal component analysis and Cramer's correlation coefficients. Frequency of bone erosions and joint-specific response to methotrexate were compared across different clusters.</p></div><div><h3>Results</h3><p>452 ERA patients were included. Analysis of the spatial distribution of swelling and tenderness allowed for the identification of 3 joint clusters that showed significant simultaneous involvement: (i) MTP1–5 joints, (ii) hand joints (MCPs and PIPs), and (iii) larger joints. These clusters were associated with different susceptibility to bone erosions and distinct clinical features, but similar local response (joint swelling resolution) to methotrexate.</p></div><div><h3>Conclusion</h3><p>This is the first study investigating the spatial distribution of arthritis in a large cohort of early RA using an unbiased approach. We identify clusters of simultaneously involved joints, supporting the importance of local factors in driving synovitis in RA.</p></div>","PeriodicalId":21715,"journal":{"name":"Seminars in arthritis and rheumatism","volume":null,"pages":null},"PeriodicalIF":4.6,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141978862","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-24DOI: 10.1016/j.semarthrit.2024.152520
Wils Nielsen , Vibeke Strand , Lee Simon , Ellie Pinsker , Dennisse Bonilla , Eric Morand , Julian Thumboo , Martin Aringer , Marta Mosca , Ian Bruce , Ioannis Parodis , Alfred Kim , Maya Desai , Yvonne Enman , Beverley Shea , Daniel J. Wallace , Yashaar Chaichian , Sandra Navarra , Cynthia Aranow , Meggan Mackay , Zahi Touma
Background
Since the development of the OMERACT Systemic Lupus Erythematosus (SLE) Core Outcome Set (COS) in 1998, many new SLE domains have been identified and measures developed, creating a need to update the SLE COS. To revisit the 1998 SLE COS and research agenda domains, and generate new candidate domains, we conducted this study of patients with SLE and collaborators.
Objective
(1) To evaluate existing candidate SLE domains for inclusion in the SLE COS. (2) To generate additional candidate SLE domains for COS consideration. (3) To engage SLE collaborators, including patients, in developing the updated SLE COS.
Methods
The OMERACT SLE Working Group's steering committee developed a survey to assess the importance of candidate SLE domains and generate additional domains for consideration towards the SLE COS. Patients with SLE followed at the University of Toronto Lupus Clinic (patient group) and members of the OMERACT SLE Working Group (collaborator group) were invited to complete the survey between August 2022 and February 2023.
Results
A total of 175 patients were invited and 100 completed the survey. Of 178 collaborators invited, 145 completed the survey. Patients tended to prioritize life-impact domains while collaborators prioritized clinical domains. Both patients and collaborators recommended additional domains to those included in the 1998 SLE COS and research agenda.
Conclusion
The domain inclusion and importance results demonstrate that patients and collaborators prioritize different domains, so capturing the perspectives of both groups is essential to ensure a holistic assessment of SLE. The results of the study identify domains that already have a high level of agreement for potential inclusion in the SLE COS, domains that require further explanation, and novel domains that warrant consideration.
自 1998 年开发出 OMERACT 系统性红斑狼疮（SLE）核心成果集（COS）以来，许多新的系统性红斑狼疮领域已被确定并开发出测量方法，从而产生了更新系统性红斑狼疮核心成果集的需求。为了重新审视 1998 年系统性红斑狼疮 COS 和研究议程领域，并产生新的候选领域，我们对系统性红斑狼疮患者和合作者进行了这项研究。(1) 评估现有的系统性红斑狼疮候选领域，以便纳入系统性红斑狼疮 COS。(2）产生更多的系统性红斑狼疮候选领域供 COS 考虑。(3) 让系统性红斑狼疮合作者（包括患者）参与制定更新的系统性红斑狼疮 COS。OMERACT 系统性红斑狼疮工作组的指导委员会制定了一项调查，以评估候选系统性红斑狼疮领域的重要性，并产生更多领域供系统性红斑狼疮 COS 考虑。多伦多大学狼疮诊所的系统性红斑狼疮患者（患者组）和 OMERACT 系统性红斑狼疮工作组成员（合作者组）受邀在 2022 年 8 月至 2023 年 2 月期间完成调查。共有 175 名患者受邀，其中 100 人完成了调查。在受邀的 178 名合作者中，有 145 人完成了调查。患者倾向于优先考虑生活影响领域，而合作者则优先考虑临床领域。除了 1998 年系统性红斑狼疮 COS 和研究议程中包含的领域外，患者和合作者还建议增加其他领域。领域的纳入和重要性结果表明，患者和合作者优先考虑的领域不同，因此，要确保对系统性红斑狼疮进行全面评估，就必须掌握这两个群体的观点。研究结果确定了可能纳入系统性红斑狼疮 COS 的共识度较高的领域、需要进一步解释的领域以及值得考虑的新领域。
{"title":"OMERACT systemic lupus erythematosus domain survey","authors":"Wils Nielsen , Vibeke Strand , Lee Simon , Ellie Pinsker , Dennisse Bonilla , Eric Morand , Julian Thumboo , Martin Aringer , Marta Mosca , Ian Bruce , Ioannis Parodis , Alfred Kim , Maya Desai , Yvonne Enman , Beverley Shea , Daniel J. Wallace , Yashaar Chaichian , Sandra Navarra , Cynthia Aranow , Meggan Mackay , Zahi Touma","doi":"10.1016/j.semarthrit.2024.152520","DOIUrl":"10.1016/j.semarthrit.2024.152520","url":null,"abstract":"<div><h3>Background</h3><p>Since the development of the OMERACT Systemic Lupus Erythematosus (SLE) Core Outcome Set (COS) in 1998, many new SLE domains have been identified and measures developed, creating a need to update the SLE COS. To revisit the 1998 SLE COS and research agenda domains, and generate new candidate domains, we conducted this study of patients with SLE and collaborators.</p></div><div><h3>Objective</h3><p>(1) To evaluate existing candidate SLE domains for inclusion in the SLE COS. (2) To generate additional candidate SLE domains for COS consideration. (3) To engage SLE collaborators, including patients, in developing the updated SLE COS.</p></div><div><h3>Methods</h3><p>The OMERACT SLE Working Group's steering committee developed a survey to assess the importance of candidate SLE domains and generate additional domains for consideration towards the SLE COS. Patients with SLE followed at the University of Toronto Lupus Clinic (patient group) and members of the OMERACT SLE Working Group (collaborator group) were invited to complete the survey between August 2022 and February 2023.</p></div><div><h3>Results</h3><p>A total of 175 patients were invited and 100 completed the survey. Of 178 collaborators invited, 145 completed the survey. Patients tended to prioritize life-impact domains while collaborators prioritized clinical domains. Both patients and collaborators recommended additional domains to those included in the 1998 SLE COS and research agenda.</p></div><div><h3>Conclusion</h3><p>The domain inclusion and importance results demonstrate that patients and collaborators prioritize different domains, so capturing the perspectives of both groups is essential to ensure a holistic assessment of SLE. The results of the study identify domains that already have a high level of agreement for potential inclusion in the SLE COS, domains that require further explanation, and novel domains that warrant consideration.</p></div>","PeriodicalId":21715,"journal":{"name":"Seminars in arthritis and rheumatism","volume":null,"pages":null},"PeriodicalIF":4.6,"publicationDate":"2024-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141770864","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-23DOI: 10.1016/j.semarthrit.2024.152501
Claudia Mendoza-Pinto , Marcial Sánchez-Tecuatl , Roberto Berra-Romani , Iván Daniel Maya-Castro , Ivet Etchegaray-Morales , Pamela Munguía-Realpozo , Maura Cárdenas-García , Francisco Javier Arellano-Avendaño , Mario García-Carrasco
Objective
This study aimed to investigate the current status and performance of machine learning (ML) approaches in providing reproducible treatment response predictions.
Methods
This systematic review was conducted in accordance with the PRISMA statement and the CHARMS checklist. We searched PubMed, Cochrane Library, Web of Science, Scopus, and EBSCO databases for cohort studies that derived and/or validated ML models focused on predicting rheumatoid arthritis (RA) treatment response. We extracted data and critically appraised studies based on the Transparent Reporting of a Multivariable Prediction Model for Individual Prognosis or Diagnosis (TRIPOD) and Prediction Model Risk of Bias Assessment Tool (PROBAST) guidelines.
Results
From 210 unduplicated records identified by the literature search, we retained 29 eligible studies. Of these studies, 10 developed a predictive model and reported a mean adherence to the TRIPOD guidelines of 45.6 % (95 % CI: 38.3–52.8 %). The remaining 19 studies not only developed a predictive model but also validated it externally, with a mean adherence of 42.9 % (95 % CI: 39.1–46.6 %). Most of the articles had an unclear risk of bias (41.4 %), followed by a high risk of bias, which was present in 37.9 %.
Conclusions
In recent years, ML methods have been increasingly used to predict treatment response in RA. Our critical appraisal revealed unclear and high risk of bias in most of the identified models, suggesting that researchers can do more to address the risk of bias and increase transparency, including the use of calibration measures and reporting methods for handling missing data.
{"title":"Machine learning in the prediction of treatment response in rheumatoid arthritis: A systematic review","authors":"Claudia Mendoza-Pinto , Marcial Sánchez-Tecuatl , Roberto Berra-Romani , Iván Daniel Maya-Castro , Ivet Etchegaray-Morales , Pamela Munguía-Realpozo , Maura Cárdenas-García , Francisco Javier Arellano-Avendaño , Mario García-Carrasco","doi":"10.1016/j.semarthrit.2024.152501","DOIUrl":"10.1016/j.semarthrit.2024.152501","url":null,"abstract":"<div><h3>Objective</h3><p>This study aimed to investigate the current status and performance of machine learning (ML) approaches in providing reproducible treatment response predictions.</p></div><div><h3>Methods</h3><p>This systematic review was conducted in accordance with the PRISMA statement and the CHARMS checklist. We searched PubMed, Cochrane Library, Web of Science, Scopus, and EBSCO databases for cohort studies that derived and/or validated ML models focused on predicting rheumatoid arthritis (RA) treatment response. We extracted data and critically appraised studies based on the Transparent Reporting of a Multivariable Prediction Model for Individual Prognosis or Diagnosis (TRIPOD) and Prediction Model Risk of Bias Assessment Tool (PROBAST) guidelines.</p></div><div><h3>Results</h3><p>From 210 unduplicated records identified by the literature search, we retained 29 eligible studies. Of these studies, 10 developed a predictive model and reported a mean adherence to the TRIPOD guidelines of 45.6 % (95 % CI: 38.3–52.8 %). The remaining 19 studies not only developed a predictive model but also validated it externally, with a mean adherence of 42.9 % (95 % CI: 39.1–46.6 %). Most of the articles had an unclear risk of bias (41.4 %), followed by a high risk of bias, which was present in 37.9 %.</p></div><div><h3>Conclusions</h3><p>In recent years, ML methods have been increasingly used to predict treatment response in RA. Our critical appraisal revealed unclear and high risk of bias in most of the identified models, suggesting that researchers can do more to address the risk of bias and increase transparency, including the use of calibration measures and reporting methods for handling missing data.</p></div><div><h3>Funding</h3><p>None.</p></div>","PeriodicalId":21715,"journal":{"name":"Seminars in arthritis and rheumatism","volume":null,"pages":null},"PeriodicalIF":4.6,"publicationDate":"2024-07-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0049017224001410/pdfft?md5=8c5c0cc16bb161ec1cfa1a22e67d7aa7&pid=1-s2.0-S0049017224001410-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141770862","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}
Evidence on abatacept (ABA) utility for rheumatoid arthritis (RA) – associated interstitial lung disease (ILD) is growing. Clinical trials have shown equivalence in subcutaneous (SC) and intravenous (IV) administration of ABA for articular manifestations. However, this has not been studied in respiratory outcomes.
Objective
To compare the effectiveness of ABA in RA-ILD patients according to the route of administration.
Methods
National retrospective multicentre study of RA-ILD patients on treatment with ABA. They were divided into 2 groups: a) IV, and b) SC. The following outcomes were analysed from baseline to final follow-up using linear mixed models: a) forced vital capacity (FVC), b) diffusing capacity of the lungs for carbon monoxide (DLCO), c) chest high resolution computed tomography (HRCT), d) dyspnoea, e) RA activity, and f) sparing corticosteroids effect.
Results
A total of 397 patients were included (94 IV-ABA and 303 SC-ABA), median follow-up of 24 [10–48] months. After adjustment for possible confounders, FVC and DLCO remained stable during the first 24 months without differences between IV-ABA and SC-ABA (p = 0.6304 and 0.5337). Improvement/ stability of lung lesions in HRCT was observed in 67 % of patients (75 % IV-ABA, 64 % SC-ABA; p = 0.07). Dyspnoea stabilized/ improved in 84 % of patients (90 % IV-ABA, 82 % SC-ABA; p = 0.09). RA - disease activity improved in both groups. No statistically significant differences regarding any of the variables studied between the two groups were found. ABA was withdrawn in 87 patients (21.9 %), 45 % IV-ABA and 37 % SC-ABA (p = 0.29). ILD worsening and articular inefficacy were the most common reasons for ABA discontinuation.
Conclusion
In patients with RA-ILD, ABA seems to be equally effective regardless of the route of administration.
{"title":"Subcutaneous vs intravenous abatacept in rheumatoid arthritis-interstitial lung disease. National multicentre study of 397 patients","authors":"Marta López-Maraver , Ana Serrano-Combarro , Belén Atienza-Mateo , Natividad del Val , Ivette Casafont-Solé , Rafael B. Melero-Gonzalez , Alba Pérez-Linaza , Jerusalem Calvo Gutiérrez , Natalia Mena-Vázquez , Nuria Vegas-Revenga , Lucía Domínguez-Casas , Jesús Loarce Martos , Cilia Amparo Peralta Ginés , Carolina Diez Morrondo , Lorena Pérez Albaladejo , Rubén López Sánchez , Mª Guadalupe Manzano Canabal , Anahy Mª Brandy-García , Patricia López Viejo , Gema Bonilla , Ricardo Blanco","doi":"10.1016/j.semarthrit.2024.152517","DOIUrl":"10.1016/j.semarthrit.2024.152517","url":null,"abstract":"<div><h3>Background</h3><p>Evidence on abatacept (ABA) utility for rheumatoid arthritis (RA) – associated interstitial lung disease (ILD) is growing. Clinical trials have shown equivalence in subcutaneous (SC) and intravenous (IV) administration of ABA for articular manifestations. However, this has not been studied in respiratory outcomes.</p></div><div><h3>Objective</h3><p>To compare the effectiveness of ABA in RA-ILD patients according to the route of administration.</p></div><div><h3>Methods</h3><p>National retrospective multicentre study of RA-ILD patients on treatment with ABA. They were divided into 2 groups: <strong>a)</strong> IV, and <strong>b)</strong> SC. The following outcomes were analysed from baseline to final follow-up using linear mixed models: <strong>a)</strong> forced vital capacity (FVC), <strong>b)</strong> diffusing capacity of the lungs for carbon monoxide (DLCO), <strong>c)</strong> chest high resolution computed tomography (HRCT), <strong>d)</strong> dyspnoea, <strong>e)</strong> RA activity, and <strong>f)</strong> sparing corticosteroids effect.</p></div><div><h3>Results</h3><p>A total of 397 patients were included (94 IV-ABA and 303 SC-ABA), median follow-up of 24 [10–48] months. After adjustment for possible confounders, FVC and DLCO remained stable during the first 24 months without differences between IV-ABA and SC-ABA (<em>p</em> = 0.6304 and 0.5337). Improvement/ stability of lung lesions in HRCT was observed in 67 % of patients (75 % IV-ABA, 64 % SC-ABA; <em>p</em> = 0.07). Dyspnoea stabilized/ improved in 84 % of patients (90 % IV-ABA, 82 % SC-ABA; <em>p</em> = 0.09). RA - disease activity improved in both groups. No statistically significant differences regarding any of the variables studied between the two groups were found. ABA was withdrawn in 87 patients (21.9 %), 45 % IV-ABA and 37 % SC-ABA (<em>p</em> = 0.29). ILD worsening and articular inefficacy were the most common reasons for ABA discontinuation.</p></div><div><h3>Conclusion</h3><p>In patients with RA-ILD, ABA seems to be equally effective regardless of the route of administration.</p></div>","PeriodicalId":21715,"journal":{"name":"Seminars in arthritis and rheumatism","volume":null,"pages":null},"PeriodicalIF":4.6,"publicationDate":"2024-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141770861","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-18DOI: 10.1016/j.semarthrit.2024.152521
Stefano Di Donato , Suiyuan Huang , John D Pauling , Francesco Del Galdo , Maya Sabbagh , Dinesh Khanna , Michael Hughes
Objectives
Raynaud's phenomenon (RP) is a symptom complex associated with digital vascular compromise. Our aim was to examine for clinically relevant differences between primary RP (PRP) and secondary RP (SRP) to connective tissue disease.
Methods
We report cross-sectional results from the Patient Survey of experiences of Raynaud's Phenomenon (PASRAP), which aimed to explore the broad-ranging impact of RP. The survey was widely distributed online including via social medial. Participation was voluntary and responses were anonymous.
Results
1229 respondents completed PASRAP with self-reported RP: PRP 218 (17.7 %) and SRP 1011 (82.3 %) of which 903 (92.9 %) Systemic Sclerosis. The mean (SD) age was significantly lower in respondents with PRP (41.7 [11.8] vs 54.2 [12.4] years, P<0.0001). During attacks, more subjects with SRP reported cyanotic colour changes (92.2 % vs 86.5 %, P=0.0089). Patients with PRP experienced more pain (72.1 % vs 55.9 %, P<0.0001), numbness (80.3 % vs 69.4 %, P=0.0016), stinging/throbbing (93.4 % vs 80.8 %, P<0.0001), and tingling (84.0 % vs 77.5 %, P=0.0345). Only half of respondents’ symptoms were adequately controlled by their current medication(s), more commonly in SRP (55.2 % vs 45.2 %, P=0.0084). There were important differences in the triggers, number, and seasonal variation of RP attacks.
Conclusion
There are clinically relevant differences between PRP and SRP concerning the multifaceted lived patient experience of RP. Neurosensory symptoms are more common in PRP. Patients with SRP are older and present with more colour changes, overrepresented by cyanosis, and with less complete resolution of symptoms between attacks. These data provide novel insights for future RP clinical trial design.
目的雷诺现象（Raynaud's phenomenon，RP）是一种与数字血管损害相关的症状综合。我们的目的是研究原发性雷诺现象（PRP）和继发性雷诺现象（SRP）与结缔组织疾病之间的临床相关性差异。方法我们报告了雷诺现象患者经历调查（PASRAP）的横断面结果，该调查旨在探索雷诺现象的广泛影响。该调查通过社交媒体等方式在网上广泛传播。结果有 1229 名受访者完成了 PASRAP，他们自述患有雷诺现象：PRP 218 人（17.7%），SRP 1011 人（82.3%），其中 903 人（92.9%）患有系统性硬化症。PRP受访者的平均（标清）年龄明显较低（41.7 [11.8] 岁 vs 54.2 [12.4]岁，P<0.0001）。在发作期间，更多的 SRP 患者报告了发绀的颜色变化（92.2% vs 86.5%，P=0.0089）。PRP 患者经历更多的疼痛（72.1 % vs 55.9 %，P<0.0001）、麻木（80.3 % vs 69.4 %，P=0.0016）、刺痛/刺痒（93.4 % vs 80.8 %，P<0.0001）和刺痛（84.0 % vs 77.5 %，P=0.0345）。只有半数受访者的症状得到了现有药物的充分控制，这在 SRP 中更为常见（55.2% vs 45.2%，P=0.0084）。在 RP 发作的诱因、次数和季节性变化方面存在重要差异。神经感觉症状在 PRP 中更为常见。SRP 患者年龄较大，表现出更多的颜色变化，其中以紫绀居多，而且在两次发作之间症状的缓解程度较差。这些数据为未来的 RP 临床试验设计提供了新的见解。
{"title":"Clinically relevant differences between primary Raynaud's phenomenon and secondary to connective tissue disease","authors":"Stefano Di Donato , Suiyuan Huang , John D Pauling , Francesco Del Galdo , Maya Sabbagh , Dinesh Khanna , Michael Hughes","doi":"10.1016/j.semarthrit.2024.152521","DOIUrl":"10.1016/j.semarthrit.2024.152521","url":null,"abstract":"<div><h3>Objectives</h3><p>Raynaud's phenomenon (RP) is a symptom complex associated with digital vascular compromise. Our aim was to examine for clinically relevant differences between primary RP (PRP) and secondary RP (SRP) to connective tissue disease.</p></div><div><h3>Methods</h3><p>We report cross-sectional results from the Patient Survey of experiences of Raynaud's Phenomenon (PASRAP), which aimed to explore the broad-ranging impact of RP. The survey was widely distributed online including via social medial. Participation was voluntary and responses were anonymous.</p></div><div><h3>Results</h3><p>1229 respondents completed PASRAP with self-reported RP: PRP 218 (17.7 %) and SRP 1011 (82.3 %) of which 903 (92.9 %) Systemic Sclerosis. The mean (SD) age was significantly lower in respondents with PRP (41.7 [11.8] vs 54.2 [12.4] years, <em>P</em><0.0001). During attacks, more subjects with SRP reported cyanotic colour changes (92.2 % vs 86.5 %, <em>P</em>=0.0089). Patients with PRP experienced more pain (72.1 % vs 55.9 %, <em>P</em><0.0001), numbness (80.3 % vs 69.4 %, <em>P</em>=0.0016), stinging/throbbing (93.4 % vs 80.8 %, <em>P</em><0.0001), and tingling (84.0 % vs 77.5 %, <em>P</em>=0.0345). Only half of respondents’ symptoms were adequately controlled by their current medication(s), more commonly in SRP (55.2 % vs 45.2 %, <em>P</em>=0.0084). There were important differences in the triggers, number, and seasonal variation of RP attacks.</p></div><div><h3>Conclusion</h3><p>There are clinically relevant differences between PRP and SRP concerning the multifaceted lived patient experience of RP. Neurosensory symptoms are more common in PRP. Patients with SRP are older and present with more colour changes, overrepresented by cyanosis, and with less complete resolution of symptoms between attacks. These data provide novel insights for future RP clinical trial design.</p></div>","PeriodicalId":21715,"journal":{"name":"Seminars in arthritis and rheumatism","volume":null,"pages":null},"PeriodicalIF":4.6,"publicationDate":"2024-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141846274","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-15DOI: 10.1016/j.semarthrit.2024.152519
Samuel Maxwell , Laura Ross , Shereen Oon , Ian P. Wicks , Jessica Day
Background
Muscle biopsy is an important test in the evaluation of individuals with suspected myopathy, including those with suspected idiopathic inflammatory myopathy (IIM). Various approaches, including open surgical biopsy, needle biopsy and conchotome forceps, have been reported. However the real-world utilisation of these approaches remains unclear. There are no established guidelines for the use of muscle biopsy, or selection of biopsy technique, in investigating IIM and international practices are not well-documented. This study describes current approaches to muscle biopsy amongst clinicians with expertise in IIM.
Methods
A survey regarding muscle biopsy practices was disseminated among members of the International Myositis Assessment and Clinical Studies (IMACS) group. Data were analysed using descriptive statistics.
Results
One-hundred and sixteen clinicians completed the survey, primarily rheumatologists. Open surgical biopsy was the most commonly employed technique (74.5 %), followed by needle (11.3 %) and conchotome (9.4 %) approaches. Clinical examination was the most common method of muscle selection, with 85.2 % of respondents reporting they ‘always or almost always’ relied on it. MRI and electromyography were also frequently utilised for muscle selection (51.9 %, 45.4 % respectively). There was variability in the perceived utility of muscle biopsy in certain clinical contexts, such as presence of myositis specific antibodies or cutaneous manifestations of dermatomyositis. While respondents generally reported low complication rates following muscle biopsy, non-diagnostic histopathology was commonly reported, regardless of procedural approach.
Conclusion
Clinicians managing IIM report muscle biopsy to be well tolerated however, non-diagnostic results are common. Substantial heterogeneity regarding perceived indications for biopsy, procedural approaches, and muscle selection strategies were observed within this expert group. Future research is needed to establish best practice and determine the role of muscle biopsy in the context of continued advancements in serological profiling of IIM.
{"title":"Muscle biopsy practices in the evaluation of idiopathic inflammatory myopathies: An international survey of expert clinicians","authors":"Samuel Maxwell , Laura Ross , Shereen Oon , Ian P. Wicks , Jessica Day","doi":"10.1016/j.semarthrit.2024.152519","DOIUrl":"10.1016/j.semarthrit.2024.152519","url":null,"abstract":"<div><h3>Background</h3><p>Muscle biopsy is an important test in the evaluation of individuals with suspected myopathy, including those with suspected idiopathic inflammatory myopathy (IIM). Various approaches, including open surgical biopsy, needle biopsy and conchotome forceps, have been reported. However the real-world utilisation of these approaches remains unclear. There are no established guidelines for the use of muscle biopsy, or selection of biopsy technique, in investigating IIM and international practices are not well-documented. This study describes current approaches to muscle biopsy amongst clinicians with expertise in IIM.</p></div><div><h3>Methods</h3><p>A survey regarding muscle biopsy practices was disseminated among members of the International Myositis Assessment and Clinical Studies (IMACS) group. Data were analysed using descriptive statistics.</p></div><div><h3>Results</h3><p>One-hundred and sixteen clinicians completed the survey, primarily rheumatologists. Open surgical biopsy was the most commonly employed technique (74.5 %), followed by needle (11.3 %) and conchotome (9.4 %) approaches. Clinical examination was the most common method of muscle selection, with 85.2 % of respondents reporting they ‘always or almost always’ relied on it. MRI and electromyography were also frequently utilised for muscle selection (51.9 %, 45.4 % respectively). There was variability in the perceived utility of muscle biopsy in certain clinical contexts, such as presence of myositis specific antibodies or cutaneous manifestations of dermatomyositis. While respondents generally reported low complication rates following muscle biopsy, non-diagnostic histopathology was commonly reported, regardless of procedural approach.</p></div><div><h3>Conclusion</h3><p>Clinicians managing IIM report muscle biopsy to be well tolerated however, non-diagnostic results are common. Substantial heterogeneity regarding perceived indications for biopsy, procedural approaches, and muscle selection strategies were observed within this expert group. Future research is needed to establish best practice and determine the role of muscle biopsy in the context of continued advancements in serological profiling of IIM.</p></div>","PeriodicalId":21715,"journal":{"name":"Seminars in arthritis and rheumatism","volume":null,"pages":null},"PeriodicalIF":4.6,"publicationDate":"2024-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0049017224001598/pdfft?md5=c37a42ae131cc948916a9214c57b31d7&pid=1-s2.0-S0049017224001598-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141701238","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}
Pub Date : 2024-07-15DOI: 10.1016/j.semarthrit.2024.152515
Chengchen Zhang , Alejandro Amill-Rosario , Abree Johnson , Haeyoung Lee , O'Mareen Spence , Driss Oraichi , Harry Seifert , Valentine Franck , Susan Gamble , Huifeng Yun , Susan dosReis
Objective
Assess the risk of incident gout following exposure to recombinant zoster vaccine (RZV).
Methods
This case-only, self-controlled risk interval study included a cohort of US fee-for-service Medicare (Part A, B, and D) beneficiaries aged ≥65 years. The exposure was receipt of at least one dose of the two-dose RZV regimen in 2018 or 2019. The risk and control windows were days 1–30 and days 31–60, respectively, following vaccination. Incident gout was defined as the first episode of gout during the risk or control window, with no evidence of gout in the last 365 days. We estimated the relative risk (RR) and 95 % confidence interval (CI) of incident gout in the risk window relative to the control window, using conditional Poisson regression models. Sensitivity analyses included a dose-compliant subanalysis of individuals who received dose 2 60–183 days after dose 1; dose-specific analysis; seasonality adjustment; and COVID-19 adjustment for potential detection bias due to the pandemic.
Results
The 1290 RZV-exposed individuals with incident gout were primarily White (86.98 %), male (61.16 %), and aged 70–79 years (55.82 %). The RR of incident gout was 1.00 (95 % CI 0.90, 1.12). In the dose-compliant sensitivity analysis (n = 959 cases of incident gout), the RR of incident gout was 0.99 (95 % CI 0.87, 1.13). The findings were unchanged in the dose-specific, seasonality, and COVID-19 sensitivity analyses.
Conclusion
The findings suggest that RZV is not significantly associated with an increased risk of incident gout in the Medicare population aged ≥65 years.
{"title":"Risk of incident gout following exposure to recombinant zoster vaccine in US adults aged ≥50 years","authors":"Chengchen Zhang , Alejandro Amill-Rosario , Abree Johnson , Haeyoung Lee , O'Mareen Spence , Driss Oraichi , Harry Seifert , Valentine Franck , Susan Gamble , Huifeng Yun , Susan dosReis","doi":"10.1016/j.semarthrit.2024.152515","DOIUrl":"10.1016/j.semarthrit.2024.152515","url":null,"abstract":"<div><h3>Objective</h3><p>Assess the risk of incident gout following exposure to recombinant zoster vaccine (RZV).</p></div><div><h3>Methods</h3><p>This case-only, self-controlled risk interval study included a cohort of US fee-for-service Medicare (Part A, B, and D) beneficiaries aged ≥65 years. The exposure was receipt of at least one dose of the two-dose RZV regimen in 2018 or 2019. The risk and control windows were days 1–30 and days 31–60, respectively, following vaccination. Incident gout was defined as the first episode of gout during the risk or control window, with no evidence of gout in the last 365 days. We estimated the relative risk (RR) and 95 % confidence interval (CI) of incident gout in the risk window relative to the control window, using conditional Poisson regression models. Sensitivity analyses included a dose-compliant subanalysis of individuals who received dose 2 60–183 days after dose 1; dose-specific analysis; seasonality adjustment; and COVID-19 adjustment for potential detection bias due to the pandemic.</p></div><div><h3>Results</h3><p>The 1290 RZV-exposed individuals with incident gout were primarily White (86.98 %), male (61.16 %), and aged 70–79 years (55.82 %). The RR of incident gout was 1.00 (95 % CI 0.90, 1.12). In the dose-compliant sensitivity analysis (<em>n</em> = 959 cases of incident gout), the RR of incident gout was 0.99 (95 % CI 0.87, 1.13). The findings were unchanged in the dose-specific, seasonality, and COVID-19 sensitivity analyses.</p></div><div><h3>Conclusion</h3><p>The findings suggest that RZV is not significantly associated with an increased risk of incident gout in the Medicare population aged ≥65 years.</p></div>","PeriodicalId":21715,"journal":{"name":"Seminars in arthritis and rheumatism","volume":null,"pages":null},"PeriodicalIF":4.6,"publicationDate":"2024-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141693562","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-15DOI: 10.1016/j.semarthrit.2024.152516
Emily Zhang , Sarah Capponi , Rebecca Scobell , Gabrielle Alonzi , Madeline Hlobik , Ankana Daga , Esra Meidan , Holly Wobma , Liyoung Kim , Lauren A. Henderson , Siobhan Case , Peter A. Nigrovic , John H. Stone , Karen H. Costenbader , Mary Beth F. Son , Joyce C. Chang
Objectives
The pediatric Glucocorticoid Toxicity Index (pGTI) is a new, pediatric-specific tool to quantify glucocorticoid (GC)-related morbidity in children. We evaluated the feasibility and construct validity of retrospective pGTI scoring in patients with pediatric-onset systemic lupus erythematosus (pSLE) and identified risk factors for cumulative toxicity.
Methods
We conducted a retrospective cohort study of patients with pSLE treated with GCs at two pediatric centers (1999–2023). GC exposure was estimated using interval-averaged oral prednisone-equivalent dose and cumulative prednisone-equivalent dose. We scored change in GC toxicity every 6 months (±2) using a modified pGTI including 7 of 10 domains. We calculated the Cumulative Worsening Score (CWS), a continuous summation of toxicity accrued. Mixed effects linear regression was used to identify factors associated with CWS.
Results
There were 126 patients with pSLE, including 88 with nephritis, with a median of 6 visits/patient. Nearly half (47 %) experienced toxicity in the Blood Pressure domain. Other common toxicities were mood disturbance (25 %), followed by increased body mass index (BMI), striae, and sleep disturbance (21 % each). Decreased growth velocity was observed in 18 %. There was modest correlation between cumulative GC dose and CWS (rho 0.3; p < 0.01). Greater cumulative toxicity was associated with younger age, elevated BMI, and rituximab use at the time of GC initiation, albeit indications for the latter were not captured.
Conclusions
Patients with pSLE experience a high burden of GC toxicity, particularly related to blood pressure, BMI, sleep, and growth. Standardized, pediatric-specific GC toxicity assessment is feasible in real-world settings and can facilitate evaluation of strategies to reduce morbidity in children requiring chronic GC treatment.
{"title":"Real-world application of the pediatric Glucocorticoid Toxicity Index in childhood-onset lupus","authors":"Emily Zhang , Sarah Capponi , Rebecca Scobell , Gabrielle Alonzi , Madeline Hlobik , Ankana Daga , Esra Meidan , Holly Wobma , Liyoung Kim , Lauren A. Henderson , Siobhan Case , Peter A. Nigrovic , John H. Stone , Karen H. Costenbader , Mary Beth F. Son , Joyce C. Chang","doi":"10.1016/j.semarthrit.2024.152516","DOIUrl":"10.1016/j.semarthrit.2024.152516","url":null,"abstract":"<div><h3>Objectives</h3><p>The pediatric Glucocorticoid Toxicity Index (pGTI) is a new, pediatric-specific tool to quantify glucocorticoid (GC)-related morbidity in children. We evaluated the feasibility and construct validity of retrospective pGTI scoring in patients with pediatric-onset systemic lupus erythematosus (pSLE) and identified risk factors for cumulative toxicity.</p></div><div><h3>Methods</h3><p>We conducted a retrospective cohort study of patients with pSLE treated with GCs at two pediatric centers (1999–2023). GC exposure was estimated using interval-averaged oral prednisone-equivalent dose and cumulative prednisone-equivalent dose. We scored change in GC toxicity every 6 months (±2) using a modified pGTI including 7 of 10 domains. We calculated the Cumulative Worsening Score (CWS), a continuous summation of toxicity accrued. Mixed effects linear regression was used to identify factors associated with CWS.</p></div><div><h3>Results</h3><p>There were 126 patients with pSLE, including 88 with nephritis, with a median of 6 visits/patient. Nearly half (47 %) experienced toxicity in the Blood Pressure domain. Other common toxicities were mood disturbance (25 %), followed by increased body mass index (BMI), striae, and sleep disturbance (21 % each). Decreased growth velocity was observed in 18 %. There was modest correlation between cumulative GC dose and CWS (rho 0.3; <em>p</em> < 0.01). Greater cumulative toxicity was associated with younger age, elevated BMI, and rituximab use at the time of GC initiation, albeit indications for the latter were not captured.</p></div><div><h3>Conclusions</h3><p>Patients with pSLE experience a high burden of GC toxicity, particularly related to blood pressure, BMI, sleep, and growth. Standardized, pediatric-specific GC toxicity assessment is feasible in real-world settings and can facilitate evaluation of strategies to reduce morbidity in children requiring chronic GC treatment.</p></div>","PeriodicalId":21715,"journal":{"name":"Seminars in arthritis and rheumatism","volume":null,"pages":null},"PeriodicalIF":4.6,"publicationDate":"2024-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141708440","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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