{"title":"High rheumatoid factor does not diminish efficacy of TNF inhibitors in seropositive JIA.","authors":"Boris Hügle, Gerd Horneff, Johannes-Peter Haas","doi":"10.1186/s12969-025-01125-z","DOIUrl":"10.1186/s12969-025-01125-z","url":null,"abstract":"<p><strong>Objectives: </strong>Rheumatoid factor (RF) binds to the immunoglobulin Fc portion, which might influence the efficacy of Fc-carrying TNF inhibitors (TNFi). This has been shown in studies in adults with RF-positive RA, but not yet in children. The aim of this study was to determine efficacy of TNFi in children with seropositive polyarthritis according to rheumatoid factor levels.</p><p><strong>Methods: </strong>Two databases were searched for patients with JIA/seropositive polyarthritis, admitted between November 2009 and March 2023. Data collected were demographic data, treatment with antirheumatic medications and JADAS27 and cJADAS27 prior to and after start of TNFi treatment. Changes in JADAS27 and cJADAS27 on TNFi were compared between patients with highly elevated RF (> 160 U/ml) and low titre RF (< 160 U/ml) using repeated measures ANOVA.</p><p><strong>Results: </strong>28 patients were included, 16 with RF < 160 U/ml at diagnosis, and 12 with RF ≥ 160 U/ml. 21 patients (75%) were treated with etanercept, three (11%) with adalimumab and four (14%) with golimumab, 23 patients additionally received methotrexate. Mean JADAS27 (cJADAS27) at treatment start was 23.0 ± 14.7 (21.0 ± 12.1), and 4.8 ± 5.0 (4.8 ± 4.7) at assessment after starting TNFi. Independent-samples t-test comparing percentage improvement as well as an ANCOVA determined that mean JADAS27 and cJADAS27 scores did not differ significantly across the two time points.</p><p><strong>Conclusions: </strong>Unlike in adults, efficacy of TNFi was not diminished by elevated levels of RF in this cohort of pediatric patients with seropositive polyarthritis. Further studies are necessary to confirm these findings.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"76"},"PeriodicalIF":2.8,"publicationDate":"2025-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12278679/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144683610","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Reem Abdwani, Mahadev J Mal, Eman Al Masroori, Ruqaiya Al Jashmi, Safiya Al Abrawi, Ibrahim Al-Zakwani
{"title":"Juvenile dermatomyositis in Oman: clinical patterns and disease trajectory from a National cohort.","authors":"Reem Abdwani, Mahadev J Mal, Eman Al Masroori, Ruqaiya Al Jashmi, Safiya Al Abrawi, Ibrahim Al-Zakwani","doi":"10.1186/s12969-025-01132-0","DOIUrl":"10.1186/s12969-025-01132-0","url":null,"abstract":"<p><strong>Objective: </strong>Juvenile dermatomyositis (JDM) is an uncommon autoimmune condition in children, often leading to prolonged disease burden and significant morbidity. Despite global advancements in understanding JDM, studies from the Middle East, particularly Oman, remain scarce. This study aims to characterize JDM from an Omani national cohort, evaluating clinical manifestations, laboratory features, disease course, and treatment outcomes.</p><p><strong>Methods: </strong>A retrospective review of all JDM patients diagnosed and managed by pediatric rheumatologist in tertiary centers in Oman was conducted. Patient demographics, clinical features, laboratory findings, treatment modalities, and disease outcomes were analyzed.</p><p><strong>Results: </strong>A total of 30 children diagnosed with JDM were included. They had an equal female to male distribution, 1:1 ratio. The median age at disease onset was 6.78 years (range: 2-13), with a median diagnostic delay of 8.4 months (range:1-23). The median follow-up period for these patients was 4 years (absolute range: 1 month-16 years). Classic JDM skin manifestations, including heliotrope rash (n = 25; 83%) and Gottron's papules (n = 23; 77%), were common. Proximal muscle weakness was observed in 28 (93%) patients, while 23 (77%) patients exhibited elevated muscle enzymes. MRI findings consistent with myositis were present in 70% (n = 19/27) of the subjects, and muscle biopsy confirmed JDM in 9 cases (30%). Among 25 patients tested for myositis specific antibodies, NXP2 (n = 3), Anti-TIF1 (n = 2), Anti-Mi-2 (n = 1), and MDA5 (n = 1) were detected, showing expected correlations with disease phenotype. Corticosteroids were universally administered, with methotrexate (n = 25; 83%) and IVIG (n = 15; 50%) as common adjuncts. Calcinosis was observed in 8 patients (27%), and was managed with various treatment modalities including pamidronate (n = 3), diltiazem (n = 2), and infliximab (n = 1). At the last follow-up, 18 patients (60%) were in clinical remission, 50% (n = 15) followed a polyphasic or chronic disease course, and 2 patients succumbed to disease-related complications.</p><p><strong>Conclusions: </strong>This study provides comprehensive characterization of pediatric JDM in Oman. The findings highlight regional variations in disease presentation, autoantibody profiles, and treatment responses, underscoring the need for early diagnosis and individualized management strategies. Continued follow-up is essential to optimize long-term outcomes and improve survival rates in this patient population.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"75"},"PeriodicalIF":2.8,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12273291/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144661020","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
John Storwick, Carrie Ye, Shahin Jamal, Nancy Maltez, Mercedes Chan
{"title":"Immune checkpoint inhibitors and the pediatric rheumatologist: a pediatric needs assessment.","authors":"John Storwick, Carrie Ye, Shahin Jamal, Nancy Maltez, Mercedes Chan","doi":"10.1186/s12969-025-01127-x","DOIUrl":"10.1186/s12969-025-01127-x","url":null,"abstract":"<p><strong>Background: </strong>The use of immune checkpoint inhibitor (ICI) therapy is increasing in pediatric oncology. ICIs can cause rheumatic-immune related adverse events (Rh-irAEs) such as inflammatory arthritis and myositis. Few case reports detail Rh-irAEs and their management in the pediatric population. Our objective was to assess the familiarity of pediatric rheumatologists (PRs) worldwide with Rh-irAEs, gauge confidence in managing these conditions, and identify knowledge gaps to guide future educational efforts.</p><p><strong>Methods: </strong>We circulated an online survey to 2084 PRs via the \"Dr. Peter Dent Pediatric Rheumatology Bulletin Board.\" Responses were collected from June 2024 to September 2024. We collected data on practitioner demographics, knowledge of ICIs and Rh-irAEs, confidence in managing Rh-irAEs, and preferred educational resources.</p><p><strong>Results: </strong>Sixty-nine participants responded, of which 55 (80%) were PRs from academic centers. Despite global distribution, 56 (81%) responses came from North America. Thirty-four (49%) respondents were not aware of ICIs and their related mechanisms, indications, and side effects, and 40 (58%) were not familiar with irAEs. Fifty-five (80%) had never managed a patient with Rh-irAEs. Among those who had (14/69, 21%), the median number of cases managed was 2.0 (IQR 0.0). Thirty-nine respondents were \"not confident at all\" managing Rh-irAEs, 34 were \"not confident at all\" managing pre-existing autoimmune diseases (PAD) in ICI users, and 46 were \"not confident at all\" advising oncology colleagues on initiating or discontinuing ICIs in the context of Rh-irAEs or pre-existing autoimmune diseases (PAD). No respondents felt \"completely confident\" managing these conditions. Participants identified knowledge gaps in long-term management, acute management, and recognition and diagnosis. Forty-three indicated the need for pediatric-specific clinical guidelines. Of the 14 respondents with clinical experience treating Rh-irAEs, treatment varied, with 4 using nonsteroidal anti-inflammatory drugs, 3 using prednisone, and 4 combining prednisone with methotrexate. Long-term management also varied, with 5 using methotrexate, and 3 using tumor necrosis factor inhibitors.</p><p><strong>Conclusions: </strong>Significant knowledge gaps and a lack of confidence exist among PRs managing ICI-related Rh-irAEs. As ICI use increases in pediatric oncology, PRs' exposure to Rh-irAEs will follow. Targeted educational programs and clinical guidelines may be valuable to address these gaps.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"72"},"PeriodicalIF":2.8,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12261645/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144644161","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Identifying the characteristics of and developing a predictive model for differentiating cancer-related musculoskeletal symptoms from juvenile idiopathic arthritis.","authors":"Khwanjira Archawanantakul, Soamarat Vilaiyuk, Samart Pakakasama, Butsabong Lerkvaleekul","doi":"10.1186/s12969-025-01121-3","DOIUrl":"10.1186/s12969-025-01121-3","url":null,"abstract":"<p><strong>Background: </strong>Musculoskeletal (MSK) symptoms are a frequent presentation in pediatric patients and may arise from a range of conditions, including juvenile idiopathic arthritis (JIA) and malignancies. Differentiating cancer-related MSK symptoms from JIA at initial presentation remains challenging due to overlapping clinical features. Delays in the diagnosis of malignancy can result in significant morbidity, underscoring the need for reliable diagnostic tools. The ONCOREUM score was developed to distinguish malignancies presenting with arthropathy from JIA and demonstrated high performance in initial validation. However, its utility is limited to patients with arthropathy and does not extend to other forms of MSK involvement. This study aimed to validate the ONCOREUM score in patients with arthropathy and to develop an expanded predictive model to distinguish cancer-related MSK symptoms from JIA.</p><p><strong>Methods: </strong>Patients aged < 16 years diagnosed with cancer or JIA were included. This retrospective study was conducted in two phases: (1) Evaluating the ability of the ONCOREUM score to identify cancer with arthropathy, (2) Developing a model to differentiate cancer-related MSK symptoms from JIA using stepwise logistic regression analysis.</p><p><strong>Results: </strong>A total of 1,026 patients were reviewed (646 cancer, 380 JIA). In phase 1, 26 cancer patients (4.0%) and 351 JIA patients (92.4%) were included. The ONCOREUM score (cutoff = - 6) had a sensitivity of 88.5% and specificity of 65.0%, with an AUC of 0.77. In phase 2, MSK symptoms were present in 84 (13%) cancer cases (61 hematologic, 23 solid tumors). The best-fitting model was obtained through multivariable analysis: back pain (OR 15.58, 95% CI 2.77-87.64, p < 0.02), nocturnal pain (OR 789.97, 95% CI 51.26-12,175.54, p < 0.0001), limb bone pain (OR 24.11, 95% CI 6.91-84.12, p < 0.0001), pallor (OR 6.30, 95% CI 1.55-25.60, p < 0.01), morning stiffness (OR 0.03, 95% CI 0.002-0.57, p < 0.02), additive arthritis (OR 0.003, 95% CI 0.00-0.04, p < 0.0001), and monoarticular involvement (OR 0.02, 95% CI 0.00-0.23, p < 0.002). This model yielded an AUC of 0.99 (95% CI 0.98-0.99).</p><p><strong>Conclusions: </strong>The refined predictive model is a promising clinical tool for differentiating cancer-associated MSK symptoms from JIA.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"73"},"PeriodicalIF":2.8,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12261695/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144644160","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kim Nguyen, Shima Yasin, Ndate Fall, Alexei A Grom, Hermine I Brunner, Christopher Towe, Grant S Schulert
{"title":"Utility of patient-reported outcomes for pulmonary symptoms and sleep disturbance and impairment in children with systemic juvenile idiopathic arthritis.","authors":"Kim Nguyen, Shima Yasin, Ndate Fall, Alexei A Grom, Hermine I Brunner, Christopher Towe, Grant S Schulert","doi":"10.1186/s12969-025-01126-y","DOIUrl":"10.1186/s12969-025-01126-y","url":null,"abstract":"<p><strong>Background: </strong>Patient-reported outcomes (PROs) are critical assessment tools for clinical practice, observational studies, and interventional trials. While families of children with systemic juvenile idiopathic arthritis (SJIA) and SJIA-associated lung disease (SJIA-LD) report significant limitations in their quality of life, existing PROs for juvenile idiopathic arthritis may not properly measure the full impact of these disorders. Our objective was to utilize a newly developed lung symptom survey as well as existing, validated Patient-Reported Outcomes Measurement Information System (PROMIS) measures in children with SJIA with and without LD.</p><p><strong>Methods: </strong>Participants were parents/guardians of SJIA patients ≤ 18 years and were invited to participate using the Cincinnati Children's Hospital Medical Center (CCHMC) JIA Registry, and memberships in the Systemic JIA Foundation, and SJIA Facebook Group. Participants provided proxy-reports for their child using several PRO questionnaires [CCHMC Lung Symptom Survey; PROMIS Asthma Impact, Sleep Disturbance, Sleep Impairment Forms] and selected demographic and SJIA specific information.</p><p><strong>Results: </strong>There were 139 responses, of which 40.3% (n = 57) reported some lung disease including 12.9% (n = 20) with interstitial lung disease (ILD), pulmonary alveolar proteinosis (PAP) and/or pulmonary artery hypertension (PAH). All SJIA patients with any lung disease and those with ILD/PAP/PAH had significantly higher total questionnaire scores than patients without lung disease on the CCHMC Lung Symptoms Survey. Both the full survey and individual questions showed good ability to distinguish patients with ILD/PAP/PAH from those without (area under the curve (AUC) > 0.7). The majority of patients reported some level of sleep disturbance (n = 71/139 = 51.1%) and sleep impairment 53.2% (n = 74) regardless of presence or absence of lung disease, including moderate to severe sleep impairment and/or disturbance in 48% of SJIA patients.</p><p><strong>Conclusions: </strong>Children with SJIA and lung problems had higher scores on the CCHMC Lung Symptom Survey; however, these measures did not discriminate between SJIA-LD and other pulmonary conditions such as asthma. Based on PROMIS measures, a majority of children with SJIA had sleep disturbance and impairment, regardless of steroid use or presence of lung disease.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"74"},"PeriodicalIF":2.3,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12265269/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144644062","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"The incidence and risk factors of uveitis in children with juvenile idiopathic arthritis (JIA): a meta -analysis and literature review.","authors":"Xin Peng, Qiao Liu, Li Lin, Liqun Dong","doi":"10.1186/s12969-025-01122-2","DOIUrl":"10.1186/s12969-025-01122-2","url":null,"abstract":"<p><strong>Background: </strong>Uveitis is a serious complication of juvenile idiopathic arthritis (JIA). Despite its seriousness, a comprehensive understanding of its incidence and early risk factors remains elusive. This knowledge gap poses challenges for formulating tailored clinical early identification and prevention strategies. Therefore, our study aims to review the incidence and risk factors of uveitis in JIA patients, and provide evidence-based insights for developing specific clinical risk identification and prevention strategies.</p><p><strong>Methods: </strong>We systematically searched databases including PubMed, Cochrane, Embase, and Web of Science until December 31, 2023. The quality of included studies was assessed through the Newcastle-Ottawa Scale (NOS). Incidence data were synthesized from cohort studies, and meta-analysis was conducted through R language.</p><p><strong>Results: </strong>Our review encompassed 28 original studies involving 22,834 JIA patients, among whom 3,381 developed uveitis during the follow-up period. Meta-analysis revealed an overall prevalence of uveitis at 12.7% (95% CI: 10.5 - 15.1%), with rates of 14.3% (95% CI: 11.9 - 15.1%) in European populations, 6.5% (95% CI: 4.0 - 9.5%) in Asian populations, and 13.4% (95% CI: 9.5 - 17.8%) in North America. Identified risk factors for the development of uveitis included early age at JIA onset, ANA-positive, and increased ESR.</p><p><strong>Conclusion: </strong>The notable prevalence of uveitis in JIA demands clinical vigilance. Our study findings highlight that age, ANA status, and ESR correlate with risk of complicated uveitis. Future research endeavors could focus on constructing a concise risk assessment tool incorporating more potent independent factors. Such a tool would enhance screening efficacy within this demographic, facilitating tailored preventive strategies.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"71"},"PeriodicalIF":2.8,"publicationDate":"2025-07-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12257714/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144638721","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Elke Schubert-Hjalmarsson, Jonatan Fridolfsson, Daniel Arvidsson, Mats Börjesson, Mari Lundberg
{"title":"Exploring physical activity patterns in adolescents with hypermobility spectrum disorder or hypermobile Ehlers-Danlos Syndrome.","authors":"Elke Schubert-Hjalmarsson, Jonatan Fridolfsson, Daniel Arvidsson, Mats Börjesson, Mari Lundberg","doi":"10.1186/s12969-025-01124-0","DOIUrl":"10.1186/s12969-025-01124-0","url":null,"abstract":"<p><strong>Background: </strong>Pain and fatigue are cardinal symptoms in adolescents with Hypermobility Spectrum Disorder (HSD) and Hypermobile Ehlers-Danlos Syndrome (hEDS). Adolescents with HSD/hEDS are assumed to be less physically active as compared to healthy peers, possibly contributing to poorer health, but objectively measured data are lacking. The primary study aim was to investigate physical activity patterns (daytime and nighttime movement behavior) using accelerometers in adolescents with HSD/hEDS versus a control group. The secondary aim was investigation of any association between fatigue and movement behavior, acknowledging pain catastrophizing as a confounder.</p><p><strong>Methods: </strong>Thirty-seven adolescents with HSD/hEDS and 45 healthy adolescents (aged 13-17 years) participated. Physical activity was measured with Axivity AX3 triaxial accelerometer and an activity-sleep diary was used for assessing time in bed. Fatigue was assessed with the Pediatric Quality of Life Inventory - Multidimensional Fatigue Scale and pain catastrophizing with the Pain Catastrophizing Scale for children.</p><p><strong>Results: </strong>Adolescents with HSD/hEDS spent significantly more time in sedentary behavior (SED), less time in moderate-to-vigorous physical activity (MVPA) and exhibited significantly more sleep movement during night compared to the control group. An association between fatigue and SED, MVPA daytime or sleep movement in adolescents with HSD/hEDS, with pain catastrophizing as confounder, could not be confirmed.</p><p><strong>Conclusion: </strong>According to this study, adolescents with HSD/hEDS exhibited physical activity behaviors at levels that are associated to poorer health compared to healthy peers. Measures need to be taken to design health promoting programs for these adolescents, including physical activity and sleep health, using a biopsychosocial approach that considers physical, psychological, and social factors.</p><p><strong>Clinical trial registration: </strong>linicalTrials.gov PRS: Protocol Section NCT05633225.</p>","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"69"},"PeriodicalIF":2.8,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12235839/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144592938","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Takeshi Yamamoto, Eri Hayata, Hironori Sato, Taiji Nakano, Hiromichi Hamada
{"title":"Efficacy of mycophenolate mofetil for the maintenance therapy of anti-AQP4 antibody-positive NMOSD complying with probable sjögren's disease in a 2-year-old girl: a case report.","authors":"Takeshi Yamamoto, Eri Hayata, Hironori Sato, Taiji Nakano, Hiromichi Hamada","doi":"10.1186/s12969-025-01123-1","DOIUrl":"10.1186/s12969-025-01123-1","url":null,"abstract":"","PeriodicalId":54630,"journal":{"name":"Pediatric Rheumatology","volume":"23 1","pages":"70"},"PeriodicalIF":2.8,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12235836/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144592937","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}