Systemic corticosteroids are cornerstone therapies in pediatric inflammatory diseases. Despite their clinical importance, both parents and clinicians may harbor substantial concerns regarding steroid-related adverse effects. While “steroidophobia” has been extensively studied in the context of topical corticosteroids, no validated instrument exists to assess clinician-based attitudes toward systemic corticosteroid use. This study aimed to develop and validate novel psychometric tools to measure systemic steroidophobia among clinicians. A methodological, multicenter study was conducted between June and October 2025 among pediatric rheumatology specialists and fellows. Two draft instruments were developed: the Oral Corticosteroid Phobia Scale (21 items) and the Intravenous Corticosteroid Phobia Scale (22 items). Face and content validity were evaluated through expert review. Item analysis, exploratory and confirmatory factor analyses, internal consistency (Cronbach’s alpha), and test–retest reliability were performed. A total of 121 clinicians participated. After item reduction, the oral scale comprised 15 items and the intravenous scale 16 items, each demonstrating a two-factor structure. The Oral Corticosteroid Phobia Scale explained 63.5% of total variance (Cronbach’s alpha = 0.908), and the Intravenous Corticosteroid Phobia Scale explained 66.4% (Cronbach’s alpha = 0.935). Test–retest reliability was excellent (ICC = 0.902 and 0.930, respectively). Model fit indices indicated good construct validity. Lower phobia scores were observed among clinicians with longer pediatric rheumatology experience. The developed scales demonstrated acceptable validity and reliability for assessing clinician-based systemic steroidophobia. These tools may facilitate a more systematic and objective identification of attitudinal barriers toward corticosteroid use and may serve as formative educational instruments in residency training and continuing medical education. Further validation across different pediatric subspecialties and countries is warranted. The online version contains supplementary material available at 10.1186/s12969-026-01200-z.
The purpose of the study is to evaluate the demographic characteristics, clinical features, and management strategies of TNF inhibitor-related autoimmune disorders (TIRAIDs) in pediatric rheumatology practice. The medical records of 71 pediatric patients treated with a TNF inhibitor for a rheumatologic disease and who exhibited any TIRAIDs during a 10-year period were retrospectively evaluated. The prevalence of TIRAIDs was 2.8% among 2450 pediatric patients who needed to use any TNF inhibitor drugs due to a rheumatologic disease. There was a female predominance (n = 40, 56%). TIRAIDs were observed in 49 patients (69%) receiving etanercept, 16 patients (23%) receiving adalimumab, and 6 patients (8%) receiving infliximab. The median time to TIRAIDs was 21.6 months. Non-infectious uveitis (38%) was the most common TIRAID, followed by paradoxical psoriasis (PP) (25%), lupus-like disease (8%), multiple sclerosis (7%), episcleritis (7%), and hidradenitis suppurativa (1%). In 58% of patients, treatment was switched to another TNF inhibitor, while in 42%, it was switched to a non-TNF biological therapy and/or cDMARDs. Complete resolution of TIRAIDs was observed in 63% of the patients. C-reactive protein levels and Juvenile Arthritis Disease Activity Score-27 were significantly higher during the development of TIRAIDs in patients with juvenile idiopathic arthritis compared to the remission period (p < 0.001 and p = 0.041, respectively). Among TIRAIDs, non-infectious uveitis and PP were the most frequently observed manifestations, predominantly occurring during etanercept treatment. Disease activity tended to increase during the onset of TIRAIDs. Discontinuation of TNF inhibitor therapy and switching to another biological treatment may be effective in achieving clinical remission.
Children with autoimmune and immune-mediated rheumatic diseases (AIIRD) receiving immunosuppressive therapies (IST) are at increased risk of morbidity from vaccine-preventable infections. The 2022 American College of Rheumatology (ACR) guidelines recommend vaccinating all children with AIIRD, with specific recommendations based on IST. Children with AIIRD receiving IST or who are immunocompromised are eligible to receive non-live vaccines, however vaccination rates remain low. The aim of this study was to understand the North American pediatric rheumatology provider approach to vaccine review and reconciliation and identify possible barriers to immunization. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) Vaccination Workgroup surveyed member North American pediatric rheumatologists about their vaccination practices when caring for children with AIIRD receiving IST. A 44-question electronic survey was distributed from March-May 2022 among rheumatology providers in the CARRA network. The survey response rate was 64% and up to 90% of rheumatology providers endorsed diverse approaches to vaccine review, with variations related to frequency of review, based on rheumatic disease and medication. Notably, IST changes prompted providers to assess baseline receipt of live vaccines over non-live vaccines (p < 0.001), while providers who endorsed not performing or deferring vaccine review did so less frequently for live compared to non-live vaccines (p < 0.05). Although 26% of surveyed providers indicated they preferred all vaccines be administered by the patient’s primary care provider (PCP), surveyed providers frequently administered influenza and pneumococcal vaccines within their practices. Provider-identified barriers to immunization included lack of clinic resources (58%), patient or parent vaccine hesitancy (27%), and family concerns regarding vaccines (20%). Most subspecialty providers (78%) communicated vaccine recommendations to PCPs using a clinic note. Our results highlighted potential modifiable factors for optimizing immunization coverage among children with AIIRD receiving IST. Standardized vaccine review practices and improved communication between rheumatologists and primary care providers can maximize clinical opportunities for improving vaccination rates and increase awareness of the importance of vaccination against vaccine-preventable infections. The online version contains supplementary material available at 10.1186/s12969-026-01189-5.
We evaluated the PEDSnet clinical research network for study enrollment of juvenile spondyloarthritis, a rare rheumatic disease that includes enthesitis-related arthritis (ERA) and psoriatic arthritis (PsA). An electronic health record (EHR)-based typology was developed by an interdisciplinary team to query EHR data for a spectrum of pediatric rheumatic diseases (2009–2023) from 8 PEDSnet centers. The prevalence, characteristics and drug exposures for juvenile spondyloarthritis was explored to gauge feasibility of leveraging the network for study enrollment. Next, the typology was adapted to identify subjects for a clinical trial; the efficiency of EHR typology query was compared to standard screening efforts. Code sets for 35 pediatric rheumatology conditions were developed to identify potentially eligible subjects in the PEDSnet network. 2510 unique patients with juvenile spondyloarthritis across the PEDSnet health care systems over the years of study were identified. Median age at 1st rheumatology visit was 12.9 years, 50.4% were female, and the median time from 1st to last rheumatology visit was 4.2 years. The spondyloarthritis typology was adapted to screen for eligible patients for the BACK-OFF JSpA trial. Over 3-months at one institution, the query saved 19.5 h and 1.9 h of effort compared to manual screening of all juvenile arthritis or enthesitis-related arthritis patient charts, respectively. Results support the capacity of the PEDSnet clinical research network to facilitate identification of subjects for rare pediatric rheumatic disease studies. Typologies for these diseases were developed and can be leveraged for clinical trial recruitment to improve efficiency. The online version contains supplementary material available at 10.1186/s12969-025-01181-5.
Protocols concerning classification, monitoring and treatment were developed for the oligoarticular form of juvenile idiopathic arthritis (JIA) as part of a consensus process. The aim was to establish standardized, evidence-based protocols for managing persistent oligoarticular JIA. The group of authors initially formulated 23 statements and circulated them in an online survey to medical members of the Society for Paediatric and Adolescent Rheumatology (GKJR). A total of 80 of the 124 paediatric and adolescent rheumatologists took part in the survey, which corresponds to just under 65% of the paediatric and adolescent rheumatologists active at the time. In a final online meeting, comments from the survey were incorporated into the statements and then agreed upon by the group of authors. Finally, for newly occurring oligoarticular JIA, 20 statements and a summary consensus treatment protocol were developed to optimise the treatment of persistent oligoarticular JIA.
Children with rheumatic diseases experience social determinants of health that influence access to care and outcomes, yet data in pediatric rheumatology are limited. We aimed to characterize caregiver-reported social determinants of health in juvenile idiopathic arthritis, systemic lupus erythematosus, and juvenile dermatomyositis and assess disparities by diagnosis, language, ethnicity, insurance, and neighborhood disadvantage. We conducted a cross-sectional study of 417 caregiver surveys administered in English or Spanish during routine clinic visits (February to May 2023). Surveys assessed education, health literacy, food security, housing stability, transportation, and social work needs. Neighborhood deprivation was quantified using the Area Deprivation Index. Associations between SDoH and sociodemographic factors, insurance, diagnosis, and area of deprivation index were analyzed using univariate and post-hoc tests with complete case analysis. Mean cohort age was 12.1 years; 74.1% were female, 77.0% White, and 36.1% Hispanic/Latino. Overall, 39.1% of caregivers reported ≥ 1 unmet social need, yet only 8.9% requested social work support. Unmet needs were highest among Spanish-speaking families (83%), Hispanic/Latino caregivers (56%), and publicly or uninsured households (59% and 45%). Systemic Lupus (44%) and Juvenile Dermatomyositis (34%) caregivers reported more food insecurity than Juvenile Arthritis (18%). Higher area of deprivation index was associated with lower caregiver education, limited health literacy, food insecurity, transportation barriers, and housing instability (all P < 0.05). Children with rheumatic disease experience substantial social determinants of health-related inequities, especially those with Systemic lupus or Juvenile dermatomyositis, Hispanic/Latino ethnicity, Spanish-speaking, or non/underinsurance. Incorporating area of deprivation index-informed social determinants of health screening into pediatric rheumatology may help identify high-risk families and guide targeted, equitable interventions. The online version contains supplementary material available at 10.1186/s12969-026-01207-6.
The phenotypic heterogeneity and variable disease course of CAPS cannot be explained solely by NLRP3 mutations, suggesting additional environmental or modifying factors. This study aimed to characterize the phenotypic and genotypic features of Turkish-origin pediatric CAPS patients residing in Turkey (Turkish cohort) and Germany (German cohort). This multicenter, retrospective study included Turkish-origin pediatric patients meeting CAPS criteria from centers in Germany and Turkey. Demographic, genetic, clinical, and treatment data were collected, and disease activity was assessed using PGA and PPGA. The study included 51 Turkish-origin pediatric CAPS patients (living in Turkey: 23; Germany: 28) with a median age of disease onset at 1 year and diagnosis at 4 years. The Turkish cohort had more pathogenic mutations and more severe phenotypes, while the German cohort predominantly carried VUS/Q703K variants with a mild phenotype. Diagnostic delay and attack duration were longer in the Turkish cohort; urticarial rash, fatigue, thoracic pain, fever, and aphthous ulcers were more frequent, whereas diarrhea, lymphadenopathy, and infection-triggered attacks predominated in the German cohort. Baseline disease activity was higher in the Turkish cohort, and most patients achieved remission at last visit under therapy—mainly above-standard doses of Canakinumab—whereas over half of the German cohort reached remission without treatment. VUS and Q703K subgroup: Among patients with similar genotypes (VUS/Q703K), the Turkish cohort presented with older age at diagnosis, longer diagnostic delay, more moderate phenotypes, and higher baseline disease activity, whereas the German cohort exhibited milder phenotypes, infection-triggered attacks, and gastrointestinal involvement. The Turkish cohort received Canakinumab or Anakinra more frequently, often with dose adjustments and above-standard doses, achieving complete remission frequently under therapy at last visit, while the German cohort more often reached remission without treatment. Non-remission and partial remission were observed exclusively in the Turkish cohort. These findings suggest that Turkish-origin pediatric CAPS patients living in Turkey, including those with similar VUS/Q703K genotypes, may experience higher disease activity and greater treatment dependence than their German counterparts. However, as most patients in our cohort carried low-penetrance VUS variants, these observations are not directly generalizable to individuals with clearly pathogenic NLRP3 genotypes. Overall, this supports the notion that factors beyond genetics, potentially including environmental or modifying influences, contribute to CAPS severity and warrant further investigation. The online version contains supplementary material available at 10.1186/s12969-026-01214-7.
Pediatric connective tissue disease-associated interstitial lung disease (CTD-ILD) is a rare but serious condition with limited clinical data; this study aimed to evaluate its clinical features, treatment strategies, and outcomes. A single-center retrospective study included 50 pediatric CTD-ILD patients with complete follow-up data from June 2018 to February 2024. Collected variables included age at CTD-ILD diagnosis (years), sex, underlying CTD, respiratory symptoms, chest HRCT findings, pulmonary function parameters (VC, FVC, FEV₁, FEV₁/FVC%, TLC, DLco), treatment regimens, and outcomes. All patients underwent HRCT, and follow-up data were obtained from electronic medical records. The median onset age was 10 years, with a female-to-male ratio of 4.56:1. Systemic lupus erythematosus (27/50, 54.00%) was the most common underlying disease, followed by MCTD and pSS. Nearly one-third of the patients (18/50, 36.0%) were asymptomatic; the most common symptom was cough (17/50, 34.0%), followed by dyspnea (8/50, 16.0%) and hypoxemia (6/50, 12.0%), while hemoptysis was rare (1/50, 2.0%). On HRCT, ground-glass opacities were the most common finding (25/50, 50.0%), followed by reticulation (11/50, 22.0%). All patients received corticosteroids for induction; CTX was used in 24/50 (48.0%) and MMF in 23/50 (46.0%). During maintenance, MMF was used in 45/50 (90.0%) of cases. Pulmonary function improved significantly at six and 12 months (P < 0.05), and HRCT showed radiological improvement in all patients with no progression at 1 year. Pediatric CTD-ILD often has an insidious onset with non-specific respiratory symptoms. Chest HRCT and pulmonary function testing are important tools for early evaluation and detection. In our cohort, treatment with corticosteroids and immunosuppressants was associated with improvements in lung function and imaging findings, supporting the potential benefit of early recognition and timely management.
Macrophage activation syndrome (MAS) complicating pediatric systemic lupus erythematosus (cSLE) is a life-threatening hyperinflammatory state driven by dysregulated cytokine release. A recent systematic literature review identified only a limited number of cSLE-MAS cases treated with IL-1 inhibitors, with variable efficacy, underscoring the need for evidence-based salvage strategies when first-line biologic therapy fails. We report a 17-year-old female with cSLE who developed secondary hemophagocytic lymphohistiocytosis/MAS fulfilling HLH-2004 criteria approximately 17 months after initial diagnosis, in the setting of medication nonadherence and nutritional failure. The MAS course was refractory to intravenous immunoglobulin, pulse methylprednisolone, dexamethasone, cyclosporine, and intravenous anakinra, and was further complicated by a secondary Clostridioides difficile superinfection precipitating a MAS flare with ferritin peaking at > 10,000 ng/mL. Following transfer to our institution, subcutaneous canakinumab at standard weight-based dosing produced prompt defervescence, sustained ferritin normalization, and clinical stabilization permitting transition to outpatient immunomodulation. This case highlights canakinumab as a viable salvage therapeutic option for patients with biologic-refractory cSLE-MAS. Emerging evidence supports an expanding role for selective IL-1β blockade in pediatric lupus-associated hyperinflammatory syndromes; prospective studies are needed to define optimal dosing, sequencing, and treatment duration.
Kawasaki disease is an acute systemic vasculitis that predominantly affects children under 5 years old and represents the leading cause of their acquired heart disease. The most serious complication involves the coronary arteries, leading to the formation of coronary artery aneurysms. The standard first-line therapy for KD consists of high-dose intravenous immunoglobulin combined with aspirin. However, for IVIG-resistant patients and adjunctive treatments such as interleukin-1 (IL-1) receptor antagonists. Despite promising experimental and clinical evidence, no comprehensive evaluation has systematically summarized the efficacy and safety of anakinra in KD patients unresponsive to conventional therapy. Therefore, this systematic review aims to compile and critically assess clinical data published between 2010 and 2024 on anakinra use in pediatric Kawasaki disease patients. Across approximately 60 reported pediatric cases derived from clinical trials, observational studies, and case reports, anakinra administration was consistently associated with rapid fever resolution (typically within 24–48 h) and marked reduction in inflammatory markers, particularly C-reactive protein. Coronary artery outcomes were variable and frequently reported qualitatively. In studies providing Z-score measurements, stabilization or regression of coronary dilatation was described in several cases, including occasional regression of giant aneurysms; however, progression despite therapy was also observed. Interpretation of vascular outcomes is limited by heterogeneity in timing of initiation, concomitant therapies, small sample sizes, and lack of comparator groups. Overall, evidence quality was judged to be low to very low according to GRADE principles due to methodological limitations and imprecision. Current data should therefore be considered exploratory and hypothesis-generating. Well-designed randomized controlled trials with standardized coronary outcome reporting are required to determine the definitive efficacy and long-term cardiovascular impact of anakinra in refractory Kawasaki disease.
This study aimed to analyze the allele frequencies and genotype distributions of key FKBP4 single nucleotide polymorphisms (SNPs) (rs11833878, rs1981655, and rs41456246) in pediatric systemic lupus erythematosus (pSLE) patients versus healthy controls and to evaluate their association with glucocorticoid (GC) treatment efficacy. Forty-five patients with pSLE and 45 age- and sex-matched healthy controls were recruited between October 2022 and June 2024. Genotyping was performed using the imLDR® kit. The patients received standard GC treatment. Clinical assessments (SLEDAI-2K, laboratory parameters, and GC dosage) were performed at baseline, 3, and 6 months. Genotypes at rs11833878 and rs41456246 were significantly correlated with clinical indicators. rs11833878 was associated with a significant difference in baseline platelet (PLT) count (P = 0.037) and PLT improvement at 3 months (P = 0.016). From baseline to 6 months, it correlated with SLEDAI-2K reduction (P = 0.019) and PLT change (P = 0.035). Between 3 and 6 months, it was associated with a 24-hour urinary protein reduction (P = 0.047). rs41456246 was associated with a significant difference in baseline white blood cell (WBC) count (P = 0.011) and GC dosage at 3 months (P = 0.029). From baseline to 6 months, it correlated with the magnitude of GC tapering (P = 0.021) and PLT improvement (P = 0.034). Between 3 and 6 months, it was associated with the extent of GC tapering (P = 0.011). No significant associations were found for rs1981655. FKBP4 polymorphisms may modulate the therapeutic response in pSLE, potentially influencing GC receptor function. rs11833878 was linked to improvements in PLT, SLEDAI-2K score, and proteinuria, whereas rs41456246 was associated with WBC, GC dosage, and PLT recovery. Genotyping these loci may serve as a valuable reference for developing personalized treatment strategies.
Anakinra is a recombinant human interleukin-1 receptor antagonist primarily administered by subcutaneous injection for the treatment of autoinflammatory conditions. Intravenous use of anakinra is only sparsely described in the literature. The aim of this study was to assess the safety of intravenous use of anakinra in a cohort of pediatric patients. This is a multicenter, retrospective cohort study. All patients who received intravenous anakinra from January 1st, 2017, to February 29th 2024 were enrolled. Collected data comprised: demographic characteristics, underlying clinical conditions, infusion-related data, anakinra-related adverse events and clinical response. The case series included 113 patients: 64 (56.6%) with underlying rheumatologic diseases, 27 (23.9%) with onco-hematologic diseases, 22 (19.5%) with severe systemic infections. Fifty-nine patients (52.2%) were admitted to intensive care units. The intravenous anakinra dose ranged from 2 to 20 mg/kg/day, and treatment duration ranged from 1 to 80 days. Adverse events were observed in 10 of 113 treated children (8.8%). The most common events were transient elevation of liver or pancreatic enzymes in seven patients (6.2%) and maculopapular rash in two patients (1.2%). One patient (0.9%) experienced an anaphylactoid reaction immediately after the infusion. Sixteen patients (14.2%) died. Among those who died, ten were receiving ongoing anakinra treatment, with a median treatment duration of 27 days (range 2–42), while six patients had discontinued the drug several days earlier. Intravenous administration of anakinra appears to be safe and not associated with severe adverse events. Reported side effects were transient, not life-threatening, and resolved either with specific treatment or after drug discontinuation. Intravenous anakinra may therefore be considered a safe therapeutic option for selected life-threatening acute clinical conditions.
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Methotrexate (MTX) is the anchor and most prescribed disease-modifying anti-rheumatic drug (DMARD) for inflammatory rheumatic diseases (IRDs). MTX can be very efficacious but can also have serious, life-threatening side effects. Adequate education and follow-up of patients/carers are therefore essential, and dedicated rheumatology nurse consultations are an important part of this. However, many patients across European countries lack access to nurse consultations, and there are no agreed-upon, defined standards of care for this topic. To develop points to consider (PtC), based on the best available evidence and experts' opinion, on the nursing education of patients (or carers) with IRDs taking MTX. A task force of adult and pediatric nurses (n=19) from 16 European countries, one rheumatologist, one pharmacist, and three patient-representatives, was established by the Portuguese Association of Health Professionals in Rheumatology. The group convened virtually to discuss the protocol for developing the PtC, including the research questions for a scoping review and for a European survey to collect patients'/careers', nurses' and rheumatologists' experiences and perceptions about MTX education. The results from these studies informed the development of the PtC statements, which were discussed and voted on in two virtual meetings and one online questionnaire. EULAR Standard Operating Procedures for the development of recommendations/PtC were followed. The consensus resulted in three overarching principles and six PtC. All PtC were based on available scientific evidence, and all obtained high levels of agreement (>8/10). These PtC emphasize the need for continuous, tailored education by trained nurses, the availability of diverse educational methods, and the support for self-management and adherence strategies. A set of PtC has been developed to improve the quality of care provided to patients with IRDs and their carers regarding the education and support nurses should provide on MTX use. The ultimate goal is to optimize MTX intake, improve efficacy, reduce side effects and ensure adherence to treatment. A plan is underway for the European implementation of these PtC, recognizing the crucial relevance of multi-professional rheumatology teamwork.
Lupus nephritis (LN) affects approximately 60–70% of children with systemic lupus erythematosus (cSLE). Clinical practice for childhood lupus nephritis (cLN) management in Saudi Arabia varies significantly between pediatric rheumatologists and nephrologists. Unified, evidence-based national guidelines are needed to standardize care and improve outcomes. We aimed to develop the first evidence-based Saudi consensus recommendations for the diagnosis, monitoring, and treatment of childhood LN through collaboration between pediatric rheumatologists and nephrologists. A multidisciplinary working group developed evidence-based recommendations for cLN under the Saudi Rheumatology Society and Saudi Society of Nephrology and Transplantation. PICO questions guided a comprehensive literature search in Embase, PubMed, and Cochrane, extending the EULAR SHARE review (up to July 2013) to include studies through December 2022. Two independent reviewers screened, selected, and assessed studies, consulting adult LN guidelines when pediatric evidence was lacking. Recommendations were drafted and refined through a two-round e-Delphi process involving 20 national experts, with ≥70% agreement required for inclusion. Study quality was appraised using a modified Downs and Black checklist, and each statement was assigned a Level of Evidence and Grade of Recommendation per Oxford criteria. The consensus panel established comprehensive recommendations for early diagnosis, therapeutic goals, treatment by LN class, and supportive care in cLN. Early evaluation for renal involvement in all cSLE patients and timely biopsy were emphasized, using the 2018 ISN/RPS classification with added histopathologic criteria. Prognostic targets include ≥25% proteinuria reduction at 3 months, ≥50% at 6 months, and <0.7 g/day at 12 months. Class-specific therapy was outlined: corticosteroids (low–moderate dose) for Class I–II, with DMARDs if needed; high-dose corticosteroids + MMF as first-line for proliferative LN, cyclophosphamide as an alternative, and multi-target or biologic regimens for refractory disease. For pure membranous LN, corticosteroids + MMF are preferred, with other agents as second-line. Adjuvant recommendations include hydroxychloroquine, early ACEi/ARB, infection prevention, and ovarian protection during cyclophosphamide. These evidence-based, locally adapted guidelines aim to standardize and optimize the management of cLN in Saudi Arabia, improving early diagnosis, guiding treatment selection by LN class, and promoting supportive strategies to enhance renal outcomes, survival, and quality of life. The online version contains supplementary material available at 10.1186/s12969-026-01215-6.
This study sought to evaluate the clinical implications of thrombocytopenia in pediatric patients diagnosed with systemic lupus erythematosus (SLE) and to explore its relationship with various disease features. Furthermore, the research aimed to identify risk factors that affect the occurrence of SLE-associated thrombocytopenia. A single-center retrospective study was conducted involving 236 pediatric patients diagnosed with SLE at Children's Hospital of Fudan University from January 2020 and December 2025. Clinical information and laboratory parameters, such as complement levels, autoantibody profiles, and platelet counts, were systematically collected. Participants were divided into two groups and those without, based on their platelet counts at the time they were diagnosed with SLE. The presence of thrombocytopenia was determined at diagnosis, and further subgroup analyses were carried out based on the severity of the condition. All statistical analyses, such as logistic regression and one-way ANOVA, were conducted using SPSS version 26.0. Thrombocytopenia was observed in 19.5% (46 out of 236) of the patients. In comparison to the cohort without thrombocytopenia, the thrombocytopenia group demonstrated significantly increased incidences of leukopenia, leukocyte reduction, and positivity for antiphospholipid antibody IgM, anti-β2-glycoprotein-1 antibody, and lupus anticoagulant (P < 0.05). Furthermore, severe thrombocytopenia (defined as a platelet count below 50 × 10⁹/L) was correlated with a markedly higher prevalence of lupus anticoagulant positivity relative to the mild-to-moderate thrombocytopenia subgroup. Logistic regression analysis revealed that leukopenia, elevated erythrocyte sedimentation rate (ESR), positivity for Anti-β2 glycoprotein 1 antibodies, high lupus anticoagulant, and neuropsychiatric manifestations were significantly associated with the presence of thrombocytopenia. Thrombocytopenia frequently occurs in pediatric patients with SLE and demonstrates a significant correlation with leukopenia, the presence of antiphospholipid antibodies, and involvement of major organs. Additionally, further multicenter prospective investigations are necessary to clarify the contribution of platelets to the pathogenesis of SLE. In clinical practice, when thrombocytopenia is identified in pediatric SLE patients, thorough evaluation for antiphospholipid antibodies and neuropsychiatric systemic lupus erythematosus (NPSLE) is warranted.
Forced displacement and migration are on the rise worldwide. Asylum seeking and refugee minors (ASRM) are particularly exposed to risk factors for mental health problems. Yet, there is a lack of comprehensive data on the prevalence of specific mental health problems as well as applied screening and follow-up care in Germany. Using the online platform REDCap, we conducted the cross-sectional SAVE-KID survey among health and social care professionals (HSCP) working with ASRM in Germany (n = 201; 44% medical doctors, 38% social workers) to assess the estimated mental health burden among ASRM, the conducted screening measures, and provided mental health care as well as the extent to which communication problems affect care for ASRM. Here we show, that on average, 21% of ASRM are reported with one or more listed mental health problem. Only 37% receive follow-up. Less than 24% of participants conduct mental health screening by informal questions, interviews, trained staff or questionnaires. 84% of participants report frequent communication problems. Most used aids are online tools or relatives' translations. SAVE-KID describes an imbalance between the occurrence of and screening for mental health problems among ASRM. Comprehensive, systematic detection of mental health problems remains challenging due to communication problems, lack of specialized staff and diagnostic tools as well as follow-up care structures. Children and adolescents who apply for asylum or refugee status in Germany are exposed to numerous mental health risks. We conducted an online survey called SAVE-KID, in which 201 health and social care professionals participated. Respondents reported that about one in five minors had a mental health problem, but only one-third of them received follow-up care. Less than a quarter of professionals used structured mental health screening methods. Most professionals reported serious communication difficulties and often had to rely on online tools or family members for translations. These findings show that mental health problems are widespread among underage asylum seekers and refugees, but effective screening and care are hampered by communication barriers, a lack of trained staff, and inadequate diagnosis and support structures.
This study aims to report the clinical, laboratory, and imaging manifestations of juvenile Sjögren’s disease (SjD) and highlight the role of large salivary gland ultrasound and small salivary gland biopsy for the purpose of diagnosis and classification in this age group. Currently, there are no unified classification criteria for juvenile SjD, and pediatric-specific literature is scarce. The current literature lacks validated pediatric-specific classification and diagnostic tools. Many tests used in adult classification criteria are not routinely assessed in children because they can be difficult to perform on younger patients and lack age-specific thresholds for abnormal results. This study aims to help build the foundation for future proposals to specifically classify SjD in the pediatric age group. This was a single-center exploratory pilot retrospective study of medical records of patients diagnosed with juvenile SjD over the past five years. Fifteen patients with juvenile SjD were included. The median age of disease onset was 11.5 years (range 8.5–17.5 years), and the median age at diagnosis was 12.5 years (range 8.5–17.5 years). Ten patients had primary SjD, and five patients had SjD secondary to systemic lupus erythematosus and limited scleroderma. The clinical manifestations at onset included musculoskeletal manifestations (n = 9), recurrent parotitis (n = 7), mucocutaneous manifestations (n = 6), cervical lymphadenopathy (n = 6), Raynaud’s phenomenon or acrocyanosis (n = 3), fever (n = 2), dysphagia (n = 2), respiratory symptoms (n = 2), and sicca manifestations (n = 2). One patient with primary SjD presented with life-threatening macrophage activation syndrome. Sarcoidosis, IgG-4-related disease, and relevant infections were ruled out in all patients. Anti-SSA antibodies were positive in 13/15 patients. All patients had abnormal large salivary gland ultrasound findings, including heterogeneous echogenicity (14/15), gland enlargement (14/15), hyperemia (11/14), cystic changes (10/15), cervical lymphadenopathy (8/15), and intraductal mucus plugging or calcifications (3/15). Small salivary gland biopsies from 13/13 patients were negative for granulomas and malignancies and revealed lymphocytic sialoadenitis with a focus score of ≥1. Recurrent parotitis and scarce sicca manifestations are two distinct clinical features of juvenile versus adult SjD. Large salivary gland ultrasound study is a promising practical tool that should be considered in suspected juvenile SjD patients. These clinical and imaging features should be considered in any proposed classification criteria for juvenile SjD.
Juvenile idiopathic arthritis (JIA) is the most common rheumatic disease in children. Uveitis is the most frequent extra-articular manifestation of JIA and a major cause of visual morbidity. Despite advances in immunomodulatory therapy, many patients reach adulthood with active ocular inflammation or vision-threatening complications. The transition from pediatric to adult care represents a vulnerable period. The primary objective of our study is to describe ophthalmologic and rheumatologic disease characteristics at the time of transition from pediatric to adult care. We conducted a retrospective cohort study of patients with JIA and past or present uveitis who transitioned to adult rheumatology at Cochin Hospital between 2016 and 2024. Clinical, ophthalmologic, and therapeutic data were collected from electronic medical records. Descriptive statistics were performed. Comparative analyses were exploratory and intended to describe differences between subgroups rather than to test predefined hypotheses. A total of 46 patients were included. Median age at JIA diagnosis was 7.5 years [IQR 2.0-16.0] and median age at first uveitis was 6.0 years [IQR 3.0-12.2]. Median follow-up after transition was 2.44 years [IQR 1.17-3.94]. Most patients were female (80%, n = 37) and had oligoarticular JIA (59%, n = 27). Chronic uveitis predominated (83%, n = 38). Ocular complications occurred in 46% (n = 17), including cataract (24%, n = 11), glaucoma (20%, n = 9), and keratitis (7%, n = 3). Over half (57%, n = 13/23) experienced ≥ 5 flares since diagnosis. Biologic DMARDs were prescribed in 53% (n = 23/43), predominantly anti-TNF agents. This study highlights the substantial burden of JIA-associated uveitis at the time of transition to adult care, characterized by frequent complications, persistent disease activity, and a high need for biologic therapy. Our findings emphasize the necessity of structured and continuous ophthalmologic follow-up across all JIA subtypes, alongside close multidisciplinary collaboration, to prevent long-term ocular damage and preserve visual function.
To assess temporomandibular joint (TMJ) alterations using magnetic resonance imaging (MRI) in pediatric and young adult patients with juvenile idiopathic arthritis (JIA) and non-JIA inflammatory arthropathies, and to evaluate the added value of ultrasound (US). The study prospectively included three groups of patients: children with JIA; young adults under 35 years with JIA, and young adults under 35 years with non-JIA inflammatory arthropathies. All patients underwent joint count assessment, clinical examination of the TMJs, evaluation of global disease activity and TMJ MRI to identify inflammation or structural damage. TMJ US was additionally performed in the two young adult patient groups. Associations between clinical findings and MRI results were then analyzed using appropriate group comparison tests. Concordance between MRI and US findings was also calculated. A total of 41 patients were enrolled: 14 in the pediatric JIA group, 19 in the adult JIA group, and 8 in the adult non-JIA group. MRI analysis revealed significantly greater joint damage in adults compared to pediatric patients (p = 0.05). Tenderness on physical examination was significantly associated with MRI-detected joint damage (p = 0.01), whereas retrognathia was associated with MRI evidence of inflammation (p = 0.02) in all patients. The negative predictive value of clinical examination for detecting TMJ alterations was low (47.8%). US, performed in 18 out of the 27 adults, was compared with MRI findings and demonstrate poor concordance, with a sensitivity of 48% (95%CI 0.28-0.68), and a specificity of 73% (95%CI 0.47-0.99). TMJs alterations were common across all three patient groups. Clinical examination showed limited association with MRI-detected morphological lesions. MRI remains the gold standard for TMJ assessment, while the diagnostic performance of US requires optimization to enhance both sensitivity and specificity. "Comité Local d'Ethique Recherche (IRB) du CHU de Montpellier" no. IRB-MTP_2022_06_202201149.