Childhood-onset Rheumatic Diseases Research Articles

Cytokine correlations in childhood-onset rheumatic diseases with pulmonary involvement

BackgroundThere was less research about cytokines in lung involvement of childhood-onset rheumatic diseases (RD) patients.ObjectiveIn this investigation, blood levels of 12 cytokines were tested in order to explore their associations with lung involvement in newly diagnosed childhood-onset RD patients.MethodsHigh-resolution computed tomography (HRCT) and pulmonary function tests (PFTs) were performed on 53 newly diagnosed RD patients without any treatment in our department between July 2021 and December 2023. Patients were separated into groups based on whether their lungs were involved or not according to abnormalities found on HRCT or PFTs. We examined the serum cytokines with 41 healthy, age and gender-matched controls.ResultsThe majority of serum cytokine levels were statistically different between the RD group with lung involvement and the control group. The RD group with lung involvement had considerably higher serum IL-6 and IFN-γ levels than the RD group without lung involvement or control group. The median serum IL-6 levels were 19 pg/ml [interquartile range (IQR) 6.9, 27.6], 4.4 pg/ml (IQR 2.0, 10.5), and 2.0 pg/ml (IQR 2.0, 2.0) in RD group with lung involvement, RD group without lung involvement, and control group, respectively. Similarly, the median serum IFN-γ levels were 6.0 pg/ml (IQR 5.0, 15), 5.0 pg/ml (IQR 5.0, 5.0), and 5.0 pg/ml (IQR 5.0, 5.0) in RD group with lung involvement, RD group without lung involvement, and control group, respectively. The receiver operating characteristic (ROC) curve study showed that IL-6 and IFN-γ had area under the curve (AUC) values of 0.782 and 0.742, respectively, indicating their significant diagnostic potential for lung involvement in RD patients.ConclusionOur findings imply that IL-6 and IFN-γ may be associated with the development of lung illnesses and may be involved in the pathophysiology of RD. Thus, in individuals with RD, elevated levels of IFN-γ over 5.42 pg/ml or IL-6 above 6.25 pg/ml may warrant suspicion of lung involvement.

Transition readiness assessment in Portuguese adolescents and young adults with pediatric-onset rheumatic diseases: a single-center study.

In this study, we aimed to assess the transition readiness levels amongst patients with childhood-onset rheumatic diseases. Additionally, we sought to identify and analyze predictive factors associated with better transition readiness skills in adolescent and young adult (AYAs) patients. This is a monocentric cross-sectional study that includes patients between 14 and 26 years of age who attended outpatient pediatric and young adult rheumatology appointments between October and December of 2023 and that were diagnosed with an immune-mediated rheumatic disease before reaching 18 years of age, with at least 1 year of disease duration. Patients were presented with a questionnaire that contained demographic and clinical questions, TRACS (Questionário de Preparação da Transição para a Autonomia nos Cuidados de Saúde) questionnaire - a validated Portuguese version of the Transition Readiness Assessment Questionnaire (TRAQ), and Hospital Anxiety and Depression Scale (HADS) questionnaire. Data was analyzed to assess the significant associations between the different variables and transition readiness outcome measured by the TRACS. Descriptive statistics, statistical comparisons and logistic regression analysis were performed. A total of 69 patients with a median age of 20 [17.5-22.5] were included in this study. The median TRACS score was 4.41 [4.09-4.74]. Significantly higher TRACS scores were observed in patients who were female, 18 years of age or older, had a higher level of education, were employed, had active disease or that belonged to middle-class (when compared to patients belonging to upper- middle class). The logistic regression analysis demonstrated that being a female or having an educational status equal to 12th grade or superior emerged as predictors of higher transition readiness levels. Our study identified female sex and higher level of education as predictors of increased transition readiness levels. Therefore, healthcare providers should consider these variables when assessing patients for transition readiness and focus on improving transition process, especially in male and less educated AYAs.

Transition to Adult Rheumatology Care: A Disease-Specific Guide.

Young adults with childhood-onset rheumatic diseases are more frequently establishing and continuing care with adult rheumatologists. The transfer of care can be challenging for both the young adult patients and their adult rheumatologists, in large part due to differences between pediatric-onset rheumatic diseases and their adult-onset counterparts, or due to the rarity of some pediatric-onset rheumatic conditions. Other challenges are due to cultural differences between pediatric and adult medical care and to the young adult needing to increasingly perform self-management skills that were previously managed by parents or other caregivers. In this review, we will provide a summary of strategies for working effectively with young adults as they transition to adult care. We will then discuss a subset of childhood-onset rheumatic diseases-including juvenile idiopathic arthritis, localized scleroderma, autoinflammatory diseases, pediatric-onset systemic lupus erythematosus, juvenile-onset dermatomyositis, and autoimmune encephalitis-for which clinical manifestations, management, and prognosis frequently differ between pediatric onset and adult onset. Our aim is to highlight differences that make caring for this population of transitioning young adults unique, providing tools and knowledge to empower the adult rheumatologist to care for these young adults in ways that are evidence-based, effective, efficient, and rewarding.

Biologics Therapy in Paediatric Rheumatic Diseases in Hong Kong

Background: There is a substantial expansion in the number of biologics available for the treatment of JIA and childhood rheumatic disease over the past decades. However, there is no report on their use and safety profile in the local paediatric population. Method of study: This is a multi-centred retrospective cohort study, recruiting patients followed up in three Paediatric departments in Hong Kong. Subjects who were followed up for childhood-onset rheumatic diseases and was treated with any of the biologic DMARs on or before the 31st December 2022, were reviewed. Results: A total of 96 patients who received biologic DMARs in the studied period were reviewed. Sixty patients were diagnosed to have JIA, cSLE (10), uveitis (8), refractory KD (7), JDM (2), COVID-MISC (3), and others (6). Concerning the JIA group, male: female is 1.4:1. 38% of the patients had polyarticular JIA and 50% had ERA who failed conventional treatment with various cDMARS. Concerning the nature of biologics, 85% of JIA patients used different types of TNF-blockers, including etanercept (38.3%), adalimumab (40%), golimumab (5%) and infliximab (3.3%). Six JIA patients used tocilizumab and one used abatacept as second-line treatment after fail response to TNF-blockers. One JIA patient was on tofacitinib to avoid subcutaneous injection. For other biologics, infliximab for refractory KD (8), rituximab for cSLE and JDM (7 and 1), belimumab in cSLE (2) and anakinra in COVID-MISC (4). One JIA-ERA patient developed lymphoma six months after stopping adalimumab and recovered after chemotherapy. One cSLE patient received rituximab and developed EBV-related CNS lymphoma with repeated infections that required hospitalization. Most patients tolerated the biologic DMARs well without complications. There was no tuberculosis infection or activation. Two cSLE patients developed herpes zoster after rituximab infusion and required hospital treatment. Two patients on adalimumab developed recurrent folliculitis and one had to stop treatment. One patient developed bacteria endophthalmitis while on adalimumab. Four patients on adalimumab developed injection site reactions and one patient need to change to another biologic. One patient on adalimumab developed positive lupus serology without clinical features. Conclusion: A broad spectrum of biologics has been used in children with various rheumatic diseases without major complications. Although rare, 2 patients developed lymphoma during the study period. Although there is no causal relationship identified with the use of biologics, continuous monitoring of the long-term safety of these drugs in the paediatric cohort is recommended.

Transition readiness in adolescents and young adults with chronic rheumatic disease in Oman: today\u2019s needs and future challenges

IntroductionIn Oman, the ““transition” of health care of adolescents to adult care occurs at a young age, like many other GCC countries for cultural reasons. In order to address this concern, this study was conducted to determine the transition readiness skills of adolescents and young adults with childhood onset rheumatic diseases using a cross-cultural adaptation of the UNC TRxANSITION scale.MethodsWe used a professionally translated/back translated, provider-administered UNC TRxANSITION Scale. This 32-question scale measures HCT in 10 domains including knowledge about diagnosis or treatment, diet, reproductive health, school/work, insurance, ability to self-manage and identification of new health providers. The maximum transitional score of 10, was categorized as low (1-4), moderate (4 - 7) and high (7 -10) transitional readiness scores.ResultsWe enrolled 81 Omani adolescents and young adults (AYA) with chronic childhood onset rheumatic diseases. The cohort consisted of 79% females, with mean age of 15.8 years (± 3.53) and mean disease duration of 6.95 years (± 4.83). Our cohort’s overall mean score is low 5.22 (±1.68). Only 14.8% of the cohort achieved a high transition score (≥7). Significant direct relationship was observed between age and the mean transition readiness score (r = .533, P < .001). The mean transition readiness score in the younger age group (10-13 years) was 4.07 (±1.29), the middle age group (14-18 years) was 5.43 (±1.27), while the older age group (19-21 year), was 6.12 (±1.81). Mean transition score of youngest age group was found to be significantly lower than the other two age groups (p = .003).ConclusionOverall, the transition readiness of AYA in Oman is low compared to other western countries indicating the need to initiate a health care transition preparation program for patients with chronic diseases across the country. In addition, we need to establish regional guidelines to address the transfer and transition policies to be in line to international recommendations.As transition continues after transfer, and is preferably guided by adolescent developmental status rather than chronological age, it would be preferable to refer to the transition and transfer policies 9rather than transitional age policy) to be in line to international recommendations.

Asymptomatic SARS-CoV-2 seropositivity: patients with childhood-onset rheumatic diseases versus healthy children.

ObjectiveWe aimed to find out the asymptomatic severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) seroprevalence among pediatric patients with rheumatic diseases and healthy children and to compare them with each other.MethodsPatients with familial Mediterranean fever (FMF), juvenile idiopathic arthritis (JIA), and juvenile systemic lupus erythematosus (jSLE) and healthy children as healthy control (HC) group who remained asymptomatic during the pandemic are examined by ELISA immunoglobulin (Ig) A and IgG tests in this cross-sectional study.ResultsOverall, 149 subjects (90 females) were included in the study. While IgA was positive in 15 subjects (10%) (HC: 8, jSLE: 3, FMF: 2, JIA: 2; p = 0.196), IgG was positive in 14 subjects (9.4%) (HC: 7, JIA: 5, FMF: 1, jSLE: 1; p = 0.156). Nineteen subjects (12.75%) were IgA or IgG positive (HC: 8, JIA: 5, jSLE: 3, FMF: 3; p = 0.644). Although not significant, seropositivity was more often in HC group. Both IgA and IgG positivity were not found to be related to age, sex, underlying rheumatic diseases, and received treatments of the patients.ConclusionWe revealed that patients with childhood-onset rheumatic diseases, even if they receive immunosuppressive medication such as biologic or conventional disease-modifying anti-rheumatic drugs, might have an asymptomatic SARS-CoV-2 infection, similarly to their healthy peers. Key points• Although it has been already known that children are most likely to have asymptomatic SARS-CoV-2 infection, there is a lack of data on the disease course of children with rheumatic disease.• There was no significant difference regarding the asymptomatic SARS-CoV-2 seropositivity rates between healthy children and the patients with childhood-onset rheumatic diseases.• Patients with childhood-onset rheumatic diseases, even if they receive immunosuppressive medication, might have asymptomatic SARS-CoV-2 infection, similarly to their healthy peers.

Early experience of COVID-19 vaccine-related adverse events among adolescents and young adults with rheumatic diseases: A single-center study.

Considering the concerns regarding the coronavirus disease-2019 (COVID-19) vaccine safety among pediatric patients with inflammatory rheumatic diseases (IRD) due to a lack of data, an urgent need for studies evaluating safety profiles of vaccines emerged. Among participants vaccinated by CoronaVac inactive SARS-CoV-2 or BNT162b2 messenger RNA (mRNA) COVID-19 (Pfizer-BioNTech) vaccine, healthy children under 18 and patients under 21 with an at least 1-year follow-up period in our department for a childhood-onset rheumatic disease were included into this cross-sectional study. Overall, 246 subjects (141 [57.3%] females) (biologic group: 43, non-biologic group: 180, healthy control group: 23) were eligible for the study. The median age was 15.34 (12.02-20.92) years. The most common adverse events were fatigue (n=68, 27.6%), headache (n=44, 17.9%), myalgia (n=38, 15.4%), arthralgia (n=38, 15.4%), and fever (n=35, 14.2%). Only 3 subjects (2 patients with familial Mediterranean fever, and one healthy child) were considered to experienced serious adverse events, since they required hospitalization. Local reactions were seen in 20 (8.13%), and 27 patients (12.1%) had disease flares within 1 month after the vaccines. Although it was significantly higher in those who received the BNT162b2 mRNA vaccine (P<.001), there was no significant relationship between adverse event frequency and age, gender, the existing diseases, ongoing treatment regimens and pre-vaccination COVID-19 histories. Although immunogenicity studies for efficacy of the vaccines and long-term follow-up studies for adverse events monitoring are required, our study indicates an acceptable safety profile of COVID-19 vaccines and encourages children with IRD to be vaccinated.

A novel transition clinic structure for adolescent and young adult patients with childhood onset rheumatic disease improves transition outcomes

BackgroundThe transition of health care from Pediatric to Adult providers for adolescents and young adults with chronic disease is associated with poor outcomes. Despite the importance of this transition, over 80% of these patients do not receive the services necessary to transition to Adult health care. In 2018, we initiated a transition clinic structure, integrating an Internal Medicine - Pediatrics trained Adult Rheumatologist in a Pediatric Rheumatology clinic to guide this transition. Our goal was to improve transition outcomes. We report the methods of this clinic and its preliminary outcomes.MethodsFor patients referred to the transition clinic, the Adult Rheumatologist assumed medical management and implemented a six-part modular transition curriculum. This curriculum included a Transition Policy, Transition Readiness Assessment, medication review and education, diagnosis review and education, and counseling on differences between Pediatric and Adult-oriented care. Eligible patients and their families were enrolled in a prospective observational outcomes research registry. Initial data from this transition clinic is reported including adherence with certain aspects of the transition curriculum and clinic utilization.ResultsThe transition clinic Adult Rheumatologist saw 177 patients in 2 years, and 57 patients were eligible for, approached, and successfully enrolled in the registry. From this registry, all patients reviewed the Transition Policy with the Adult Rheumatologist and 45 (78.9%) completed at least one Transition Readiness Assessment. Of the 22 patients for whom transition was indicated, all were successfully transitioned to an Adult Rheumatologist. 17 (77.3%) continued care post-transition with the transition clinic Adult Rheumatologist, and 5 (22.7%) continued care post-transition with a different Adult Rheumatologist. The median time between the last transition clinic visit and first Adult clinic visit was 5.1 months.ConclusionsOur experience demonstrated the success of our clinic model regarding participation in the transition curriculum and improved clinic utilization data. Our results are an improvement over transition rates reported elsewhere that did not implement our model. We believe that this structure could be applied to other primary care and subspecialty clinics.Trial registrationThis research was approved by the University of Utah Institutional Review Board (IRB) in January 2019 (IRB_00115964). Patients were retrospectively registered if involved prior to this date.

Clinical characteristics and COVID-19 outcomes in a regional cohort of pediatric patients with rheumatic diseases

BackgroundThis study aimed to assess the baseline characteristics and clinical outcomes of coronavirus disease 2019 (COVID-19) in pediatric patients with rheumatic and musculoskeletal diseases (RMD) and identify the risk factors associated with symptomatic or severe disease defined as hospital admission, intensive care admission or death.MethodsAn observational longitudinal study was conducted during the first year of the SARS-CoV-2 pandemic (March 2020–March 2021). All pediatric patients attended at the rheumatology outpatient clinics of six tertiary referral hospitals in Madrid, Spain, with a diagnosis of RMD and COVID-19 were included. Main outcomes were symptomatic disease and hospital admission. The covariates were sociodemographic and clinical characteristics and treatment regimens. We ran a multivariable logistic regression model to assess associated factors for outcomes.ResultsThe study population included 77 pediatric patients. Mean age was 11.88 (4.04) years Of these, 30 patients (38.96%) were asymptomatic, 41 (53.25%) had a mild-moderate COVID-19 and 6 patients (7.79%) required hospital admission. The median length of hospital admission was 5 (2–20) days, one patient required intensive care and there were no deaths. Previous comorbidities increased the risk for symptomatic disease and hospital admission. Compared with outpatients, the factor independently associated with hospital admission was previous use of glucocorticoids (OR 3.51; p = 0.00). No statistically significant risk factors for symptomatic COVID-19 were found in the final model.ConclusionNo differences in COVID-19 outcomes according to childhood-onset rheumatic disease types were found. Results suggest that associated comorbidities and treatment with glucocorticoids increase the risk of hospital admission.

New-onset hypogammaglobulinaemia and infectious complications associated with rituximab use in childhood-onset rheumatic diseases.

To investigate the incidence and risk factors for hypogammaglobulinaemia and infectious complications associated with rituximab treatment in childhood-onset rheumatic diseases. We performed a single-centre retrospective study of patients (n = 85) treated at Boston Children's Hospital (BCH) from 2009 to 2019. Study subjects included patients (ages 6-24 years) who received rituximab for the treatment of a childhood-onset rheumatic disease. New-onset hypogammaglobulinaemia developed in 23 (27.1%) patients within 18 months of rituximab induction treatment. Twenty-two patients (25.9%) developed at least one infectious complication in the 18 months following the first rituximab infusion; of these, 11 (50%) had serious infections requiring inpatient treatment. After adjusting for potential confounders, exposure to pulse corticosteroid therapy in the month prior to rituximab use was a significant predictor of both new-onset hypogammaglobulinaemia (odds ratio [OR] 3.94; 95% CI: 1.07, 16.0; P = 0.044) and infectious complications (OR 15.3; 95% CI: 3.04, 126.8; P = 0.003). Post-rituximab hypogammaglobulinaemia was the strongest predictor of serious infectious complications (OR 7.89; 95% CI: 1.41, 65.6; P = 0.028). Younger age at rituximab use was also a significant predictor of new-onset hypogammaglobulinaemia (OR 0.83; 95% CI: 0.70, 0.97; P = 0.021). Compared with other rheumatic diseases, patients with vasculitis had a higher likelihood of developing infectious complications, including serious infections. Although rituximab was well tolerated in terms of infectious complications in the majority of patients with childhood-onset rheumatic diseases, a substantial proportion developed new-onset hypogammaglobulinaemia and infectious complications following treatment. Our study highlights a role for heightened vigilance of rituximab-associated hypogammaglobulinaemia and infections in paediatric patients with rheumatic conditions.

Assessment of transition readiness in adolescents in Thailand with rheumatic diseases: a cross-sectional study

BackgroundMost childhood-onset rheumatic diseases are chronic health conditions, which need long-term care throughout adulthood. A well-organized transition care is challenging and patient assessment of transition skills is needed for transfer preparation to an adult care setting. The Transition Readiness Assessment Questionnaire (TRAQ) is used to assess transition skills in chronically ill patients. Currently, limited transition skill assessment data exist in pediatric patients with rheumatic diseases, especially in Asian countries. This study aimed to determine the transition readiness skills in patients with rheumatic diseases and ascertain predictive factors contributing to high transition readiness skills.MethodsThis is a cross-sectional study. All patients with rheumatic diseases aged 15–20 years were recruited. The TRAQ was cross-culturally adapted into the Thai language with good internal consistency and reliability. Patients completed the Thai TRAQ at the recent clinic visit and took the retest at a 2-week interval. Demographic data, baseline characteristics, clinical manifestations, and disease status were collected. Descriptive and logistic regression analyses were performed.ResultsA total of 111 patients with a mean age of 17.4 ± 1.8 years were included. Median (IQR) disease duration was 6.4 (3.2–9.0) years. The most common rheumatic disease was juvenile idiopathic arthritis (48.6%), followed by systemic lupus erythematosus (35.1%). The mean TRAQ score was 3.85 ± 0.69. Independent visits (OR 4.35, 95% CI 1.23–15.37) was a predictor of a high TRAQ score. Furthermore, dependent visits (OR 7.84, 95% CI 2.41–25.50) was a predictor of low TRAQ score in the “appointment keeping” domain, whereas inactive disease (OR 4.54, 95% CI 1.25–16.55) was a predictor of a low TRAQ score in “tracking health issues” domain. Lack of knowledge and skills on health insurance coverage, financial management, appointment arrangement, and coping with their illness were issues causing lower TRAQ score.ConclusionsPatients, who had independent visits, had a higher chance to obtain higher TRAQ scores, whereas patients, who had an inactive disease or dependent visits, had less transition readiness skills. Physicians and parents should prepare to transfer patients to adult care settings, mainly encouraging independent living skills.

O01 Genetic risk factors associated with increased risk of uveitis in patients with juvenile idiopathic arthritis

Abstract Background/Aims Juvenile idiopathic arthritis (JIA) is a childhood-onset rheumatic disease, which is associated with increased risk of uveitis. Approximately 80% of childhood chronic anterior uveitis cases are JIA-associated (JIAU), where it is considered a serious complication with the potential to lead to permanent blindness. Studies have reported associations of increased risk of JIAU to genetic variation within human leukocyte antigen (HLA) genes, in particular amino acid positions of HLA-DRB1. Here we report the results of the largest fine-mapping study of HLA genes in JIAU to date. Methods Genotyping was performed using Illumina Infinium CoreExome and Infinium OnmiExpress arrays. Samples were excluded with a call rate &amp;lt;0.98, discrepancy between genetically inferred sex and database records, inferred relatedness (Identity-by-decent), or ancestral outlier based on principal component analysis (PCA). SNPs were excluded with a call rate &amp;lt;0.98 or a minor allele frequency (MAF) &amp;lt;0.01. HLA alleles, amino acids, and SNPs were imputed using SNP2HLA. Analysis was performed on markers with an information score &amp;gt;0.9 and MAF &amp;gt; 0.01 using logistic regression, or omnibus test for multi-allelic markers, including 3 PCs as covariates. Independent effects were identified using forward stepwise logistic regression by inclusion of previously identified variants as covariates. Results We analysed 7,425 markers across the HLA region in 450 JIAU cases and 2,024 JIA cases. We defined study-wide significance at a Bonferroni corrected threshold of 6.7x10-6. The most significant association was at amino acid position 13 of DRB1 (p-value = 3.0x10-30) where the presence of serine (OR 1.7, 95% CI 1.4-1.9) or glycine (OR 2.0, 95% CI 1.6-2.5) were associated with increased risk of uveitis. Conditioning on DRB1 position 13, found a further association to amino acid position 67 of DRB1 (p-value = 2.4x10-6, unconditioned p-value = 3.2x10-23). The presence of isoleucine (OR 1.5, 95% CI 1.2-1.9) and phenylalanine (OR 1.8 95%, CI 1.4-2.2) at position 67 were associated with increased risk. No further study-wide associations were found at DRB1. Conditioning on all HLA-DRB1 alleles identified an independent effect at amino acid position 69 in DPB1 (p-value = 5.3x10-7, unconditioned p-value = 1.1x10-10). The presence of glutamic acid was associated with increased risk (OR 1.7, 95% CI 1.4-2.0). Conclusion This is the largest genetic study of HLA regions in JIAU and further resolves the genetic risk factors in this key susceptibility region. The analysis in this study has independently validated the association signal at position 13 of HLA-DRB1 (highly correlated with position 11) that had been reported in a previous study. Conditional analysis of HLA-DRB1 revealed a novel secondary association signal at DRB1 to amino acid position 67. Conditional analysis on all DRB1 alleles confirmed association to position 69 of HLA-DPB1 where previous reports of this signal had been only modestly associated. Disclosure M. Tordoff: None. S.L. Smith: None. E. Lopez-Isac: None. A. Morris: None. S. Eyre: None. W. Thomson: None. J. Bowes: None.

The Coming-of-Age Transition Care for Adolescents with Rheumatic Disease-Where Are We and What Have We Done in Asia?

The transition from pediatric to adult health care is a challenging yet important process in rheumatology as most childhood-onset rheumatic diseases persist into adulthood. Numerous reports on unmet needs as well as evidence of negative impact from poor transition have led to increased efforts to improve transition care, including international guidelines and recommendations. In line with these recommendations, transition programs along with transition readiness assessment tools have been established. Despite these efforts, there are still a lot of work to be done for transition care in rheumatology. This review article focuses on how transition care in rheumatology has developed in recent years and highlights the gaps in current practices.

Outcomes and predictors of juvenile idiopathic arthritis in Southeast Asia: a Singapore longitudinal study over a decade.

To assess short- and long-term outcomes and predictors of juvenile idiopathic arthritis (JIA) children treated with contemporary therapy and compare those with reports elsewhere. Children with JIA were recruited from our web-based REgistry for Childhood Onset Rheumatic Diseases (RECORD) from 1997 to 2015. Disease status was defined using modified Wallace criteria. Nonparametric statistics described the data. Kaplan-Meier survival and logistic regression analyses were used to estimate probabilities and to determine predictors of outcomes. A total of 251 children with JIA (62% males, 71% Chinese) were included. Median follow-up duration was 2.9years (range 0.1-17.5). Short-term clinical inactive disease (CID) was attained in 37% with 62% systemic JIA (sJIA) and 47% persistent oligoarthritis (oJIA). Methotrexate (OR 0.34) decreased but sJIA (OR 3.25) increased chance of attaining CID at 6months. Overall, 79% of patients achieved CID within 2years (sJIA 92%, the highest, and RF+ polyarthritis 50%, the lowest probability). Biologics were associated with CID attainment (OR 2.73). One-half of patients flare after CID, median 1.2years (IQR 0.71-1.97). Late CID achievement predicted flare (OR 2.15). Only 15% had clinical remission off medication (none RF+ polyarthritis and 7% ERA). Only 13% of patients had active arthritis as young adults and 22% had active arthritis at last visit. Despite high proportion of JIA patients attaining CID, only one-fourth could stop all medications for at least 1year. Persistent oJIA patients were less likely to achieve clinical remission on medication and ERA patients had the least chance stopping medications. One-tenth of patients had active arthritis as young adults. Key Points • Majority of Asian children with JIA attained inactive disease within 2years after diagnosis. • Outcome predictors were different from reports from the West. • Despite high inactive disease numbers, only one-in-four JIA patients discontinued treatment within 5years.

Clinical Characteristics and Factors Associated With Disability and Impaired Quality of Life in Children With Juvenile Systemic Sclerosis: Results From the Childhood Arthritis and Rheumatology Research Alliance Legacy Registry

To investigate clinical manifestations of juvenile systemic sclerosis (SSc; scleroderma), including disease characteristics and patient quality of life, using the multinational Childhood Arthritis and Rheumatology Research Alliance (CARRA) Legacy Registry. Patients with juvenile SSc were prospectively enrolled between 2010 and 2013. The diagnosis of juvenile SSc was determined by the enrolling pediatric rheumatologist, with the requirement for disease onset prior to age 18 years. Collected data included demographics, disease characteristics, medication exposure, and quality of life metrics. In total, 64 patients with juvenile SSc were enrolled a median of 3.6 years after disease onset, which occurred at a median age of 10.3 years. The most common organ manifestations were dermatologic and vascular, followed by musculoskeletal, gastrointestinal, and pulmonary; in 38% of patients, ≥4 organ systems were affected. Patients with juvenile SSc had significantly more disability at enrollment compared with CARRA Legacy Registry patients with juvenile idiopathic arthritis, dermatomyositis, or systemic lupus erythematosus. Although physician-reported measures correlated most closely with arthritis, dermatologic manifestations, and pulmonary manifestations, poor patient-reported measures were associated with gastrointestinal involvement. During >50 person-years of follow-up, most organ manifestations remained stable, and no mortality or development of new solid organ involvement after enrollment was reported. In the first multicenter prospective cohort of patients with juvenile SSc in North America, the disease burden was high: multiorgan manifestations were common, and functional disability was greater than that observed in patients with other childhood-onset rheumatic diseases. Gastrointestinal involvement had the greatest impact on quality of life.

Early use of alendronate as a protective factor against the development of glucocorticoid-induced bone loss in childhood-onset rheumatic diseases: a cross-sectional study

BackgroundBisphosphonates are recommended for use as first-line therapy for the prevention and treatment of glucocorticoid-induced osteoporosis in adults. However, the appropriate usage of bisphosphonates for the prevention or treatment of glucocorticoid-induced osteoporosis in children remains unclear.MethodsWe performed a cross-sectional study to clarify the factors associated with the development of glucocorticoid-induced bone loss and osteoporosis in patients with childhood-onset rheumatic disease and to investigate the impact of the early use of alendronate. We recruited 39 patients with childhood-onset rheumatic disease who were evaluated to detect bone loss or osteoporosis at 3 months to 1.5 years after the initiation of treatment. The primary outcome of the study was the presence of bone loss or osteoporosis at the initial evaluation of the bone mineral density after at least 3 months of glucocorticoid therapy.ResultsBone loss and a history of fracture were found in 56 and 18% of the participants, respectively. Weekly oral alendronate therapy (median, 25.4 mg/m2) had been started by the time of the evaluation of osteoporosis in 46% of the participants and within 3 months after the start of glucocorticoid in 31% of the participants. There were no significant differences between the participants with bone loss (wBL group) and without bone loss (w/oBL group) in terms of gender, primary disease, or the age at the onset of primary disease. In terms of glucocorticoid use, there was no significant difference in the age at the start of glucocorticoid therapy, the length of glucocorticoid use, or the dose of glucocorticoids. The proportion of patients in the w/oBL group who received alendronate within 3 months after the start of glucocorticoid therapy was significantly greater than that in the wBL group. In the logistic regression analysis, only “alendronate therapy within 3 months after the start of glucocorticoid therapy” had a statistically significant effect on the development of bone loss (OR, 0.08; 95% CI, 0.02–0.43). The analysis did not reveal any factors associated with the development of osteoporosis.ConclusionsThe early use of alendronate may have a preventive effect against the development of bone loss in glucocorticoid-treated patients with childhood-onset rheumatic disease.

Assessment of transition readiness in patients with childhood-onset rheumatic diseases and their guardians

Assessment of transition readiness in patients with childhood-onset rheumatic diseases and their guardians

Biologic Agents in the Treatment of Childhood-Onset Rheumatic Disease

Biologic Agents in the Treatment of Childhood-Onset Rheumatic Disease

The new Childhood Arthritis and Rheumatology Research Alliance (CARRA) registry: design, rationale, and characteristics of patients enrolled in the first 12\xa0months

BackgroundHerein we describe the history, design, and rationale of the new Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry and present the characteristics of patients with juvenile idiopathic arthritis (JIA) enrolled in the first 12 months of operation.MethodsThe CARRA Registry began prospectively collecting data in the United States and Canada in July 2015 to evaluate the safety of therapeutic agents in persons with childhood-onset rheumatic disease, initially restricted to JIA. Secondary objectives include the evaluation of disease outcomes and their associations with medication use and other factors. Data are collected every 6 months and include clinical assessments, detailed medication use, patient-reported outcomes, and safety events. Follow-up is planned for at least 10 years for each participant and is facilitated by a telephone call center.ResultsAs of July 2016, 1192 patients with JIA were enrolled in the CARRA Registry at 49 clinical sites. At enrollment, their median age was 12.4 years old and median disease duration was 2.6 years. Owing to preferential enrollment, patients with systemic JIA (13%) and with a polyarticular course (75%) were over-represented compared to patients in typical clinical practice. Approximately 49% were currently using biologic agents and ever use of oral glucocorticoids was common (47%). The CARRA Registry provides safety surveillance data to pharmaceutical companies to satisfy their regulatory requirements, and several independently-funded sub-studies that use the Registry infrastructure are underway.ConclusionThe new CARRA Registry successfully enrolled nearly 1200 participants with JIA in the first 12 months of its operation. Sustainable funding has been secured from multiple sources. The CARRA Registry may serve as a model for the study of other uncommon diseases.

Survey of attitudes of non-pediatric rheumatologists among councilors of the Japan College of Rheumatology regarding transitional care

Objectives: The transition from pediatric to adult healthcare systems has recently received worldwide attention. Surveys of the attitudes of Japanese non-pediatric rheumatologists regarding transitional care were conducted.Methods: Non-pediatric rheumatologists among councilors of the Japan College of Rheumatology were enrolled in the surveys. Experiences of adult patients with childhood-onset rheumatic diseases, ideal medical care for these patients, and factors that made the transition to adult care difficult were examined via e-mail.Results: Overall, 201 non-pediatric rheumatologists (21.2%) responded to the surveys. Ninety-one percent had previous experience with patients with childhood-onset rheumatic disorders. Transition to non-pediatric institutes was supported by about 90% of respondents. However, only 32% of non-pediatric rheumatologists had no hesitation about caring for adults with childhood-onset rheumatology disorders. Two main factors prevented smooth transitions to non-pediatric care: inadequacy of non-pediatric care (57%) and lack of independence from parents/family (53%). The majority of non-pediatric rheumatologists hesitated about medical care for patients with autoinflammatory syndromes, whereas they became familiar with articular juvenile idiopathic arthritis without hesitation (86.6%); 93% of respondents requested more opportunities to learn about pediatric rheumatology disorders.Conclusions: Sharing additional knowledge about pediatric rheumatology within the non-pediatric rheumatology field is required.