Pediatric Rheumatic Diseases Research Articles

Ocular Manifestations of Pediatric Rheumatic Diseases

Ocular Manifestations of Pediatric Rheumatic Diseases

Impact of the COVID-19 Pandemic on the Management Course of Paediatric Rheumatic Diseases in a Tertiary Care Hospital of Bangladesh

Impact of the COVID-19 Pandemic on the Management Course of Paediatric Rheumatic Diseases in a Tertiary Care Hospital of Bangladesh

Implementation of Rheumatology Health Care Transition Processes and Adaptations to Systems Under Stress: A Mixed-Methods Study.

Despite poor health care transition outcomes among young adults with pediatric rheumatic diseases, adoption of transition best practices is low. We sought to understand how structured transition processes were operationalized within pediatric rheumatology practices and what factors were perceived to enable adaptations during a global pandemic. We conducted a mixed methods study of team leaders' experiences during an interim analysis of a pilot project to implement transition policy discussions at sites in the Childhood Arthritis and Rheumatology Research Alliance Transition Learning Collaborative. We combined quantitative assessments of organizational readiness for change (9 sites) and semistructured interviews of team leaders (8 sites) using determinants in the Exploration, Preparation, Implementation, Sustainment Framework. Engagement of nursing and institutional improvement efforts facilitated decisions to implement transition policies. Workflows incorporating educational processes by nonphysicians were perceived to be critical for success. When the pandemic disrupted contact with nonphysicians, capacity for automation using electronic medical record (EMR)-based tools was an important facilitator, but few sites could access these tools. Sites without EMR-based tools did not progress despite reporting high organizational readiness to implement change at the clinic level. Lastly, educational processes were often superseded by acute issues, such that youth with greater medical/psychosocial complexity may not receive the intervention. We generated several considerations to guide implementation of transition processes within pediatric rheumatology from the perspectives of team leaders. Careful assessment of institutional and nursing support is advisable before conducting complex transition interventions. Ideally, new strategies would ensure interventions reach youth with high complexity.

Blood transcriptomics to facilitate diagnosis and stratification in pediatric rheumatic diseases – a proof of concept study

BackgroundTranscriptome profiling of blood cells is an efficient tool to study the gene expression signatures of rheumatic diseases. This study aims to improve the early diagnosis of pediatric rheumatic diseases by investigating patients’ blood gene expression and applying machine learning on the transcriptome data to develop predictive models.MethodsRNA sequencing was performed on whole blood collected from children with rheumatic diseases. Random Forest classification models were developed based on the transcriptome data of 48 rheumatic patients, 46 children with viral infection, and 35 controls to classify different disease groups. The performance of these classifiers was evaluated by leave-one-out cross-validation. Analyses of differentially expressed genes (DEG), gene ontology (GO), and interferon-stimulated gene (ISG) score were also conducted.ResultsOur first classifier could differentiate pediatric rheumatic patients from controls and infection cases with high area-under-the-curve (AUC) values (AUC = 0.8 ± 0.1 and 0.7 ± 0.1, respectively). Three other classifiers could distinguish chronic recurrent multifocal osteomyelitis (CRMO), juvenile idiopathic arthritis (JIA), and interferonopathies (IFN) from control and infection cases with AUC ≥ 0.8. DEG and GO analyses reveal that the pathophysiology of CRMO, IFN, and JIA involves innate immune responses including myeloid leukocyte and granulocyte activation, neutrophil activation and degranulation. IFN is specifically mediated by antibacterial and antifungal defense responses, CRMO by cellular response to cytokine, and JIA by cellular response to chemical stimulus. IFN patients particularly had the highest mean ISG score among all disease groups.ConclusionOur data show that blood transcriptomics combined with machine learning is a promising diagnostic tool for pediatric rheumatic diseases and may assist physicians in making data-driven and patient-specific decisions in clinical practice.

12 Hip involvement in juvenile idiopathic arthritis: frequency and associated factors

BackgroundJuvenile idiopathic arthritis (JIA) is a pediatric rheumatic disease with several subgroups. The hip is frequently affected. The frequency of this involvement can reach 50% especially in the severe and destructive forms. Arthroplasty may be indicated in advanced cases.ObjectivesTo assess the frequency and associated factors with hip involvement in (JIA).MethodsWe conducted a retrospective study including adults with long-standing JIA according to the International League of Associations for Rheumatology (ILAR) criteria over a period of 28 years (1994–2022). Demographic, clinical, biological, and radiographic data were collected. These parameters were compared according to the presence or absence of hip involvement.ResultsA total of 29 Patients were enrolled (12 men and 17 women), the mean age was 35.69 ± 11.72 [18–61] years. The mean age of disease onset was 11.10 ± 4.25 [2–16] years. The average diagnostic delay was 52.96 ± 95.97 [0–336] months. The average disease duration was 24.48 ± 12.76 [1–47] years.Sixteen patients had a polyarticular form. Mean CRP values were 42.74 ± 63.37 [2–218] mg/l, a biological inflammatory syndrome was present in 19 cases. Rheumatoid factor, ACPA and anti-nuclear antibodies were observed in 12, 7 and 5 cases respectively. At least one extra-articular manifestation was noted in 16 cases.Hip involvement was noted in 14 patients (48.3%). It was bilateral in 64.3% of cases (n = 9). Twenty-three hips were affected in total (56.9%). Hip involvement was diagnosed 14.5 ± 9.37 [1–28] years after disease onset. Arthroplasty was performed on 10 hips with a delay of 201.60 ± 104.75 [108–348] months between diagnosis of JIA and surgery.Hip involvement was associated with male gender in our study (75% vs 29.4%; p = 0.016). On the other hand, our study showed that age, age at onset, diagnostic delay, symptoms duration, smoking, BMI, extra-articular manifestations, CRP, rheumatoid factor, antinuclear antibodies, ACPA and erosive character were not associated with hip involvement.ConclusionOur study showed that hip involvement is common in JIA, mainly in male patients. It usually occurs late in the disease course. Since hip involvement is a cause of disability and functional impairment, it should be assessed regularly.

13 Juvenile idiopathic arthritis and cardiovascular risk factors

BackgroundJuvenile idiopathic arthritis (JIA) is the most common pediatric rheumatic disease. Although, some patients achieve remission, some cases of JIA may persist into adulthood. Patients with JIA and other inflammatory joint diseases have increased cardiovascular disease risk compared with the general population.ObjectivesTo study the cardiovascular risk factors in JIA and their association with disease parameters.MethodsThis was a retrospective study including adults with long-standing JIA according to the International League of Associations for Rheumatology (ILAR) criteria over a period of 28 years (1994–2022). We collected sociodemographic and anthropometric parameters, clinical data, results of biological assessments, and data on prescribed therapies.We studied the following cardiovascular risk factors: family history of cardiovascular event, physical inactivity, smoking, arterial hypertension, diabetes, dyslipidaemia and obesity.ResultsWe included 29 patients. The M/F sex ratio was 0.71, the mean age was 35.69 ± 11.72 [18–61] years. The mean age of disease onset was 11.10 ± 4.25 [2–16] years. The average diagnostic delay was 52.96 ± 95.97 [0–336] months. The average disease duration was 24.48 ± 12.76 [1–47] years. The mean BMI was 21.20 ± 4.88 kg/m2 [14.17–27.55].The polyarticular form was the most frequent, noted in 55.2% of cases (n = 16). Extra-articular manifestations were observed in 55.2% of cases. Mean CRP was 42.74 ± 63.37 [2–218] mg/l and biological inflammatory syndrome was present in 19 cases. Rheumatoid factor, ACPA and anti-nuclear antibodies were observed in 12, 7 and 5 cases respectively.Corticosteroid therapy and NSAIDs were prescribed to 18 of the subjects.Cardiovascular risk factors were present in 41.4% (n = 12) of cases: family history of cardiovascular event (n = 2 cases), physical inactivity (n = 5 cases), smoking (n = 3 cases), arterial hypertension (n = 4 cases), diabetes (n = 4 cases), dyslipidaemia (n = 4), and BMI ≥ 25 kg/m² (n = 4).Following parameters were significantly higher in patients with cardiovascular risk factors: the presence of a biological inflammatory syndrome (81.8% vs 35.3%; p = 0.016), the frequency of prescription of corticosteroids (91.7% vs 52.9%; p = 0.026) and NSAIDs (83.3% vs 47.1%; p = 0.047).However, no significant difference was noted when comparing these parameters: gender, age, age of disease onset, disease duration and presence of extra-articular manifestations. Moreover, cardiovascular risk factors were not associated with the presence of rheumatoid factor, ACPA, and antinuclear antibodies.ConclusionInflammation, corticosteroid therapy and NSAIDs are associated with the presence of cardiovascular risk factors in JIA. The evaluation and control of this risk must be regular during patient follow-up. Control of inflammation and rationalization of treatment are necessary.

50 The spectrum of pediatric lupus: data from the Kenya Pediatric Rheumatology Registry (KAPRI)

BackgroundPediatric rheumatic diseases are chronic illnesses that impart a significant disease burden upon children and their families (1–3). Furthermore, they have the potential to cause physical disability, diminished quality of life and significant direct and indirect costs (1–3). Determination of the burden and clinical characteristics of pediatric lupus is a critical first step to improving access to care and optimizing use of existing health systems for the well-being of these patients (4). Childhood-onset systemic lupus erythematosus (cSLE) is a prototype autoimmune condition characterized by simultaneous or sequential organ and system involvement, with unpredictable flare and high levels of morbidity and mortality (5). A pediatric rheumatology registry is critical in defining the spectrum of pediatric lupus within the region, the clinical characteristics of the disease, the outcomes and responses to various interventions. The Kenya Pediatric Rheumatology Registry (KAPRI) registry offers a unique opportunity to pioneer and spearhead a systematic and organized format in collecting pertinent clinical information that will offer information on current clinical scenarios and offer a platform to conduct other research projects to improve pediatric lupus healthcare in sub-Sahara Africa. Our objective was to determine the clinic-epidemiological profile of paediatric lupus patients by describing their baseline patient characteristics and clinical features at the Aga Khan University Medical College East Africa who were enrolled into the KAPRI registry from inception in March 2019 to December 2021.MethodsAll patient records were selected from the KAPRI registry database using the ICD 10 code M32 that denotes Systemic Lupus Erythematosus (SLE) Age, gender, laboratory, clinical features at diagnosis, treatment options offered at time of diagnosis were extracted from the database.ResultsAmong the 207 patients enrolled thus far in the registry, 7 had a diagnosis of cSLE (3.4%). The commonest symptom among the patients were joint pain, fever and rash. Other clinical features and outcomes are highlighted in the table 1 below;ConclusionOver a 2-year period, 57% of our cSLE patients (4 of 7) achieved remission while 43% (3of 7) were lost to follow up. Further studies are required to elucidate predictors of good clinical response and reasons for loss to follow up among our cSLE patients.

Update of evidence- and consensus-based guidelines for the treatment of juvenile idiopathic arthritis (JIA) by the German Society of Pediatric and Juvenile Rheumatic Diseases (GKJR): New perspectives on interdisciplinary care

BackgroundNew therapeutic strategies for juvenile idiopathic arthritis (JIA) have evolved within the past ten years, and as a result, an update of the 2011 recommendations of the German management guidelines was initiated. MethodsA systemic literature review was performed, overarching principles were proposed and pre-selected via an online survey followed by two multidisciplinary consensus conferences. Pharmacological and non-pharmacological treatments were discussed, statements were proposed and ultimately agreed upon by nominal group technique (NGT). Results12 overarching therapeutic principles, as well as 9 recommendations on pharmacological and 5 on non-pharmacological treatments for JIA were agreed upon. ConclusionThis report summarizes the recent update of the interdisciplinary, consensus-based German guidelines on the management of JIA. The multi- and interdisciplinary participation of all caregivers was central for this patient-focused update. With these guidelines, physicians can choose an evidence-based approach, which allows better tailored treatment in this vulnerable cohort of children and adolescents.

Treat to Target (Drug-Free) Inactive Disease in JIA: To What Extent Is This Possible?

Background: Treat to target (T2T) is a strategy that has been increasingly employed in the management of several chronic diseases, with demonstrated improved outcomes. The use of T2T in juvenile idiopathic arthritis (JIA), a common rheumatic disease of childhood, is still in its infancy, and the feasibility of its use in attaining drug-free clinical remission is unclear. Aims: We aim to explore the current literature of the use of T2T in JIA, and to review the potential benefits and limitations of this approach in regard to this chronic disease. Sources: A comprehensive PubMed search was conducted using relevant keywords, with full text articles in English included in the review. Content: T2T is an appealing strategy for improving outcomes of pediatric rheumatic diseases given the limited availability of therapeutics and potential cumulative effects of long-term immunosuppression. The application in a cohort of children, however, is limited by heterogeneity of disease, availability of high-quality evidence, and patient and parental preferences. Unlike adult rheumatoid arthritis, the ‘window of opportunity’ has not been definitively demonstrated in large scale trials, and although early studies of T2T in JIA have been favorable, the timing and means of escalation (especially with regard to biologics) need clarification. Implications: This review outlines several issues of implementing T2T in JIA, including the important extra-articular manifestations of disease and non-pharmacological management, that should be considered in future consensus guidelines.

Epidemiology conduction of paediatric rheumatic diseases based on the registry database of the Pediatric Rheumatology Association of Japan.

Although epidemiological surveys of paediatric rheumatic diseases in Japan have been conducted, they were single surveys with no continuity. This is the first report of the Pediatric Rheumatology Association of Japan registry database, which was established to continuously collect data for paediatric rheumatic diseases. Pediatric Rheumatology International Collaborate Unit Registry version 2 (PRICUREv2) is a registry database established by the Pediatric Rheumatology Association of Japan. The registry data were analysed for the age of onset, time to diagnosis, sex differences, seasonality, and other factors. Our data showed the same trend regarding rates of paediatric rheumatic diseases reported in Japan and other countries. The age of onset was lower in juvenile idiopathic arthritis (JIA) and juvenile dermatomyositis and higher in systemic lupus erythematosus and Sjögren's syndrome. The time to diagnosis was relatively short in JIA and systemic lupus erythematosus but longer in juvenile dermatomyositis and Sjögren's syndrome. Rheumatoid factor-positive polyarticular JIA showed a seasonality cluster with regard to onset. PRICUREv2 aided the retrieval and evaluation of current epidemiological information on patients with paediatric rheumatic diseases. It is expected that the data collection will be continued and will be useful for expanding research in Japan.

Real-life data on efficacy and safety of original Adalimumab and biosimilar Adalimumab (ABP 501) in pediatric rheumatic diseases

ABSTRACT Background We aimed to compare the efficacy and safety of the original product (OP) and biosimilar product (BP) of adalimumab in pediatric rheumatic diseases. Research design and methods The study group consisted of patients who had received original or biosimilar adalimumab (ABP 501) therapy for at least 3 months. The patients were divided into uveitis and arthritis groups based on the indication of adalimumab treatment. Assessment of disease activity was performed by Juvenile Arthritis Disease Activity Score in patients with juvenile idiopathic arthritis, and by standardization of uveitis nomenclature criteria in patients with uveitis. Results The study included 140 patients, of which 87 were treated with OP and 53 with BP. In the arthritis group, 26 (63.4%) and 20 (57.1%) patients reached inactive disease according to JADAS-27 in the original and biosimilar adalimumab groups, respectively. In the uveitis group the mean number of exacerbations throughout the treatment period was 0.84 ± 1.07 in the OP group, and 0.58 ± 0.79 in the BP group. There were 71 treatment-emergent adverse events in the OP group and 38 in the BP group. Conclusion There was no significant difference between the biosimilar and the original product in efficacy and safety.

Vaccination completeness in children with rheumatic diseases: A longitudinal, observational multicenter cohort study in Switzerland

IntroductionChildren with pediatric inflammatory rheumatic diseases (PRD) have an increased infection risk. Vaccinations are effective to avoid vaccine-preventable diseases. This study aimed to assess the vaccination completeness in Swiss PRD patients stratified by immunosuppressive treatment (IST).Materials and methodsThis multicenter observational cohort study of PRD patients was performed in Basel, Geneva, Lucerne, Lausanne, and Zurich in PRD patients aged < 18 years included in the Juvenile Inflammatory Rheumatism Cohort. Completeness was assessed for i) the overall vaccination status (Swiss national immunization program (NIP) and specific additional PRD-recommended vaccinations), ii) for all and each vaccination of the NIP at PRD diagnosis and reference date (RefD) and iii) all and each specific additional PRD-recommended vaccination at RefD. Completeness was assessed over the disease course and stratified by IST.ResultsOf 616 eligible patients, 234 children were analyzed. Of these, 147 (63%) were girls. Median age at PRD diagnosis was 6.5 years (IQR 2.9–10.3) and 10.9 years at RefD (6.9–14.3). The median follow-up since PRD diagnosis was 3 years (1.1–5.5). 120/234 children received IST. At RefD, overall vaccination completeness was 3.8% (9/234 children), completeness for the NIP vaccinations was 70.1% (164/234 children; IST 65%, no IST: 75.4%) and for all specific additional PRD-recommended vaccinations was 3.8% (9/234 children; IST 2.5%; no IST 5.3%). Vaccination completeness against pneumococcal disease, hepatitis B virus, and human papilloma virus (HPV) was 50.4, 20, 37.9%, respectively. In 25/35 children with negative varicella zoster virus history vaccination status was complete (IST: 94.4%, no IST: 47%). Annual non-live influenza vaccination was complete in 24.2% of children during IST; adherence decreased over the disease course.DiscussionThis study identified a low overall vaccination completeness in children with PRD. Particularly, the completeness of specific additional PRD-recommended vaccinations was low. If not performed early after PRD diagnosis, vaccination status remained frequently incomplete. Close collaboration between pediatrician and rheumatologist to improve vaccination completeness is essential. Exchange of vaccination records, standardized assessment of specific PRD-recommended vaccinations and those of the NIP, and annual reminder for influenza vaccination are crucial to improve vaccination completeness in this vulnerable pediatric population.

Efficacy and Safety of NSAIDs in Infants: A Comprehensive Review of the Literature of the Past 20 Years.

Non-steroidal anti-inflammatory drugs (NSAIDs) are commonly used in infants, children, and adolescents worldwide; however, despite sufficient evidence of the beneficial effects of NSAIDs in children and adolescents, there is a lack of comprehensive data in infants. The present review summarizes the current knowledge on the safety and efficacy of various NSAIDs used in infants for which data are available, and includes ibuprofen, dexibuprofen, ketoprofen, flurbiprofen, naproxen, diclofenac, ketorolac, indomethacin, niflumic acid, meloxicam, celecoxib, parecoxib, rofecoxib, acetylsalicylic acid, and nimesulide. The efficacy of NSAIDs has been documented for a variety of conditions, such as fever and pain. NSAIDs are also the main pillars of anti-inflammatory treatment, such as in pediatric inflammatory rheumatic diseases. Limited data are available on the safety of most NSAIDs in infants. Adverse drug reactions may be renal, gastrointestinal, hematological, or immunologic. Since NSAIDs are among the most frequently used drugs in the pediatric population, safety and efficacy studies can be performed as part of normal clinical routine, even in young infants. Available data sources, such as (electronic) medical records, should be used for safety and efficacy analyses. On a larger scale, existing data sources, e.g. adverse drug reaction programs/networks, spontaneous national reporting systems, and electronic medical records should be assessed with child-specific methods in order to detect safety signals pertinent to certain pediatric age groups or disease entities. To improve the safety of NSAIDs in infants, treatment needs to be initiated with the lowest age-appropriate or weight-based dose. Duration of treatment and amount of drug used should be regularly evaluated and maximum dose limits and other recommendations by the manufacturer or expert committees should be followed. Treatment for non-chronic conditions such as fever and acute (postoperative) pain should be kept as short as possible. Patients with chronic conditions should be regularly monitored for possible adverse effects of NSAIDs.

Current Status of Pediatric Rheumatic Disease Care in Japan

Current Status of Pediatric Rheumatic Disease Care in Japan

Non-criteria antiphospholipid antibodies and pediatric rheumatic disease: a case series

BackgroundNon-criteria antiphospholipid antibodies (NC-aPL) are a relatively undefined subgroup of antiphospholipid antibodies (aPL). Knowledge about NC-aPL in adults is limited and even less is known in pediatric patients. Routine tests for antiphospholipid syndrome (APS)—a clinical state marked by the presence of aPL in association with vascular thrombosis—usually include lupus anticoagulant (LAC), anti-cardiolipin (aCL) and -beta-2 glycoprotein I (aβ2GPI). LAC is a functional screen for prothrombotic aPL, while the latter tests identify specific autoantibodies. Specific targets of NC-aPL include, but are not limited to, phosphatidylethanolamine, phosphatidylserine, and prothrombin.Presentation of casesWe present single-center data from eight pediatric patients with NC-aPL identified during a three-year period. All patients had presenting features raising suspicion for APS. Most patients were female with a primary rheumatic disease. One patient had a stroke. Another patient had alveolar hemorrhage and pulmonary hypertension. Raynaud’s phenomenon, rashes involving distal extremities, and headaches were common. Most patients had a positive LAC, yet their routine aPL tests were negative, prompting testing for NC-aPL.ConclusionsOur findings suggest NC-aPL are associated with typical signs and symptoms of APS in pediatric patients. Pediatricians and pediatric subspecialists should consider NC-aPL when clinical suspicion is high and routine aPL tests are negative, particularly when LAC is positive. While guidelines for NC-aPL do not yet exist for children or adults, these autoantibodies have pathogenic potential. Actionable items could include evaluation for the presence of other (primary) rheumatic diseases, and consultation with hematologists and/or obstetricians regarding anticoagulation/platelet inhibition and thrombosis education. Future guidelines regarding NC-aPL will only be generated by gathering more data, ideally prospectively.

Real-life vaccination coverage in Slovak children with rheumatic diseases.

BackgroundEvidence-based recommendations for vaccination of patients with pediatric rheumatic diseases (PRDs) are available, their implementation in practice is unknown.ObjectivesTo analyze real-life vaccination coverage in children with PRDs and identify reasons for incomplete vaccination.MethodsUp-to-date information on vaccination status of Slovak children followed at a tertiary pediatric rheumatology center was retrieved from pediatricians over an 18-month period and compared to the standard Slovak Immunization Schedule. Reasons for missed vaccinations were analyzed retrospectively.ResultsVaccination records of 156 patients (median age 10 years, 2–18) with PRDs (JIA n = 108, systemic diseases n = 21, autoinflammatory diseases n = 16, uveitis n = 9, others n = 2) were available for analysis. 117 (75.0%) were completely vaccinated, 2 (1.3%) had not received any vaccine due to reasons unrelated to PRD. 37 (23.7%) remaining patients missed altogether 48 mandatory vaccinations. In 58.3% (n = 28, in 24 patients) no PRD related reasons for missing vaccinations were identified. Only 20 vaccinations (18 live-attenuated and 2 non-live in 19 patients) were missed due to ongoing immunosuppressive treatment or PRD activity. Patients aged 11–14 years were more likely to be incompletely vaccinated than other age groups (48.8% vs. 15.9%, p < 0.001), mainly due to missed MMR booster. Systemic immunosuppressive treatment was a significant predictor for incomplete vaccination status (OR 5.03, 95% CI 1.13–22.31, p = 0.03).ConclusionFull vaccination is possible in a high proportion of PRD patients. In addition to immunosuppressive therapy, reasons unrelated to PRDs are a frequent and possibly inadequate cause of missed vaccinations. Periodic vaccination status assessments are needed in pediatric rheumatology care.

Comparing parental distress and children’s difficulties between parents of children with rheumatic diseases and parents of healthy children in families facing the COVID-19 pandemic

The COVID-19 pandemic could be a threat for the health status of children with a chronic condition. The present study aimed to explore parents’ and children’s psychological adjustment during the current pandemic, pursuing a triple objective: to compare the psychological adjustment of parents of children with pediatric rheumatic diseases (PRDs) and parents of healthy children; to analyze children’s psychological symptoms (emotional problems and hyperactivity) before and during the COVID-19 pandemic, and with or without a PRDs diagnosis; to explore the associations of children’s emotional problems and hyperactivity with parents’ psychological adjustment, parent–child interactions and belonging or not to families with PRDs. This cross-sectional study involved 56 parents of children with PRDs and 53 parents of healthy children. Self-report questionnaires about parents’ depression, anxiety, parenting stress, and children’s emotional symptoms and hyperactivity-inattention were administered. No differences were detected on psychological adjustment between parents of children with PRDs and parents of healthy children. Parents of children with PRDs reported statistically significant higher levels of children’s emotional problems and hyperactivity before the pandemic, compared to parents of healthy children; during COVID-19 pandemic, emotional symptoms increased for both groups, while hyperactivity-inattention symptoms increased only in the group of healthy children. Children’s emotional difficulties were associated with higher levels of parental anxiety, worse parent–child interaction and having PRDs; children’s hyperactivity symptoms were related to parent–child difficult interaction and higher levels of parental depression. Findings suggest the importance to target the children in relation to their parents, when approaching the psychological aspects of PRDs.

Impact of mitral regurgitation on left ventricular remodeling and function in children with rheumatic heart disease.

The impact of mitral regurgitation (MR) from pediatric rheumatic heart disease (RHD) and its effect on left ventricular (LV) remodeling and function following surgical intervention is uncertain. The objective is to explore the impact of mitral valve (MV) surgeries on myocardial mechanics, remodeling and function and identify pre-operative predictors of post-operative dysfunction which may contribute to the optimal timing of intervention. A retrospective review of echocardiographic data was performed of eighteen pediatricpatients with RHD (median 9yrs, IQR 6-12) who underwent MV surgery. Echocardiograms pre-operatively and a median of 13.5months (IQR 10.2-15) following intervention were compared to controls. Pre-operative LV end-diastolic indexed volumes (LVEDVi) were significantly increased compared to controls and remained persistently larger post-operatively. LV ejection fraction (LVEF) (pre 62.6% ± 6.1, post 51.7% ± 9.7, p = 0.002), and global longitudinal strain (GLS) (pre -24.3 ± 4.1, post -18.2 ± 2.6, p < 0.001) decreased post-operatively at mid-term follow-up. Pre-operative LVEDVi was a significant predictor of post-operative LVEF, with a cut-off of ≥ 102ml/m2 associated with LV dysfunction (LVEF < 55%; sensitivity 70%, specificity 75%). Pre-operative LVEDVi also negatively correlated with GLS (r = -0.58, p = 0.01). LV dimensions and volumes remain persistently larger than controls while LV function decreases post-surgical alleviation of MR in paediatric RHD. Pre-operative LVEDVi predicted post-operative LV dysfunction and utilising LV indexed volumes in directing timing of surgical planning should be considered. Further studies are required to investigate whether timely alleviation of MR before significant LV dilatation and remodeling occur may substantially prevent LV dysfunction and improve outcomes.

Efficacy of anakinra treatment in pediatric rheumatic diseases: Our single-center experience.

This study aims to present our experience on anakinra, a recombinant interleukin-1 (IL-1) receptor antagonist, and efficacy results in pediatric rheumatic diseases in our clinic. Between July 1st, 2016 and July 1st, 2020, a total of 33 pediatric patients (18 males, 15 females; mean age: 6±3.4 years; range 4 to 13 years) with pediatric rheumatic diseases who were treated with anakinra were retrospectively analyzed. The patients with over one-month treatment period and followed for at least one year were included. Demographic and clinical findings, outcomes, adverse events, prior and/or additional treatments were collected at baseline, at 3 and 12 months of therapy. There were 33 patients with different pediatric rheumatic diseases (11 with systemic juvenile idiopathic arthritis [sJIA] complicated by macrophage activation syndrome [MAS], six with hyperimmunoglobulin-D syndrome, five with cryopyrin-associated periodic syndrome, five with familial Mediterranean fever, four with idiopathic recurrent pericarditis, one with NLRP12-associated periodic fever syndrome and one with unclassified systemic autoinflammatory disease), in the study group. The complete response was observed 69.7% of patients, partial response in 24.2%, and no response in 6.1% at three months of treatment. Inactive disease status was achieved in 45.5% of the patients with remission-on medication and 18.2% of the patients with remission-off medication at the end of a year. Anakinra was switched to other biological treatments in 51.5% of patients (n=17). Biological switch to canakinumab and tocilizumab were observed in 70.6% and 29.4% of these patients. Except for local reactions (n=2), no adverse events were observed in any of the patients. Anakinra appears to be a promising treatment alternative owing to its rapid effect as a result of its short half-life in autoinflammatory conditions. While short-term therapy seems to be sufficient for the sJIA complicated by MAS, the patients with systemic autoinflammatory diseases maintenance a more anakinra-dependent course.

Evaluation of pediatric rheumatologists’ knowledge, attitudes, and behavior regarding vaccination in pediatric rheumatic diseases

Aim: Developments in diagnostic methods and advances in the treatment of pediatric rheumatic diseases (PRDs) have improved the quality of life in this patient group. However, vaccine-preventable infectious diseases are still outstanding causes of morbidity and mortality in immunocompromised patients more than in healthy population. Pediatric rheumatologists play a critical role in increasing vaccination rates since they have ample opportunity to administer vaccinations. The aim of this study is to determine the knowledge, attitudes, and behaviors of pediatric rheumatologists about vaccination in rheumatic diseases.&#x0D; Material and Method: Between March 2022-May 2022, an online questionnaire of 20 items was used to evaluate the knowledge, attitudes, and behaviors of pediatric rheumatologists in Turkey with respect to vaccination.&#x0D; Results: A total of 81 participants answered all survey questions completely. The ages of the study group ranged from 30 to 48 years, at a mean of 37.5±3.8 years. Most of the pediatric rheumatologists (n:76, 93.8%) were working in a tertiary pediatric hospital; 27.2% did not consider themselves primarily responsible for the vaccination of children with PRDs, and 30.9% did not refer their patients to the department that administers the vaccine either before or during immunosuppressive therapy. In addition, it was found that only 14.8% regularly questioned the vaccination history of patients at each outpatient visit. One-third of study group recommended the implementation of non-scheduled vaccines and the most recommended non-scheduled vaccine was seasonal influenza (n:48, 59.3%). The comparative analysis between pediatric rheumatology residents and staff physicians showed no statistically significant difference in the level of knowledge except in the question concerning live vaccines.&#x0D; Conclusion: This study revealed that there are still serious problems in clinical practice about the vaccination of PRDs patients. For this purpose, pediatric rhematologists’ awareness and knowledge about vaccination should be increased with a special education program.