Pediatric Disease Research Articles

Longitudinal plasma metabolome patterns and relation to kidney function and proteinuria in pediatric chronic kidney disease.

Understanding plasma metabolome patterns in relation to changing kidney function in pediatric chronic kidney disease (CKD) is important for continued research for identifying novel biomarkers, characterizing biochemical pathophysiology, and developing targeted interventions. There is a limited number of studies of longitudinal metabolomics, and virtually none in pediatric CKD. The Chronic Kidney Disease in Children (CKiD) study is a multi-institutional, prospective cohort that enrolled children aged six-months to 16-years with estimated glomerular filtration rate (eGFR) 30-90ml/min/1.73m2. Untargeted metabolomics profiling was performed on plasma samples from the baseline, two-, and four-year study visits. There were technologic updates in the metabolomic profiling platform used between the baseline and follow-up assays. Statistical approaches were adopted to avoid direct comparison of baseline and follow-up measurements.To identify metabolite associations with eGFR or urine protein:creatinine (UPCR) among all three timepoints, we applied linear mixed effects (LME) models. To identify metabolites associated with time, we applied LME models to the two- and four-year follow-up data. We applied linear regression analysis to examine associations between change in metabolite level over time (∆level) and change in eGFR (∆eGFR) and UPCR (∆UPCR). We reported significance based on both the False Discovery Rate (FDR) <0.05 and p<0.05. There were 1156 person-visits (N: baseline=626, 2-year=254, 4-year=276) included. There were 622 metabolites with standardized measurements at all three timepoints. In LME modeling, 406 and 343 metabolites associated with eGFR and UPCR at FDR<0.05 respectively. Among 530 follow-up person-visits, 158 metabolites showed differences over time at FDR<0.05. For participants with complete data at both follow-up visits (N=123), we report 35 metabolites with ∆level∼∆eGFR associations significant at FDR<0.05. There were no metabolites with significant ∆level∼∆UPCR associations at FDR<0.05. We report 16 metabolites with ∆level∼∆UPCR associations at p<0.05 and associations with UPCR in LME modeling at FDR<0.05. We characterized longitudinal plasma metabolomic patterns associated with eGFR and UPCR in a large pediatric CKD population. Many of these metabolite signals have been associated with CKD progression, etiology, and proteinuria in previous CKD Biomarkers Consortium studies. There were also novel metabolite associations with eGFR and proteinuria detected.

A literary review on Ayurveda perspective of Pandu in Kaumarabhritya

In traditional science of Indian medical system, Childhood age is described as Kaumaravastha. The basic feature of this age group is to increase immunity, physical &amp; mental strength, management of nutritional supply &amp; control of prevalence of disease in of growing children. Kaumarbhritya not only to help paediatric disease but also describe dietary &amp; daily life routine for paediatric care. The Pandu is one of the common diseases of childhood as early growing life which resembles Anaemia as per modern science. The disease involves vitiation of Dhatus by Pitta predominant Tridosha, which leads to deficiency of Blood (Rakta) Fat (Meda) &amp; Ojas.[2] The condition is associated with laxity (Saithilya) of Dhatus, heaviness (Gaurava). Other predominant features are like diminish Varna (Complexion), Vala (Strength) &amp; Sneha (Smoothness) of the body. There are certain premonitory symptoms which indicate chances of Pandu like Hrudaya Spandana (Palpitation), Roukshya (Dryness), Swedabhava (Loss of sweating) &amp; Shrama (tiredness).[4] The Pandu may arise due to Sevan of Kshara, Amla, Lavana, Asatmya Bhojana. Hence Ayurveda recommended to take Satmya Bhojana &amp; all of the foods which increase the amount of blood. The foods like Kharjura, Kismis, Apple, Pomegranate should be given. The drugs to be used in child should have Madhura Rasa.There are various Ayurveda formulation also available for the management of Pandu such as Navayasa Choorna. Samsodhana Karma also indicated for the management of Pandu Roga. This Article described various Ayurveda perspective of Pandu Roga in children.

Outcome Measures of Clinical Trials in Pediatric Chronic Kidney Disease

Outcome Measures of Clinical Trials in Pediatric Chronic Kidney Disease

Statistical methods to control for confounders in rare disease settings that use external control

ABSTRACT In the drug development for rare disease, the number of treated subjects in the clinical trial is often very small, whereas the number of external controls can be relatively large. There is no clear guidance on choosing an appropriate statistical method to control baseline confounding in this situation. To fill this gap, we conduct extensive simulations to evaluate the performance of commonly used matching and weighting methods as well as the more recently developed targeted maximum likelihood estimation (TMLE) and cardinality matching in small sample settings, mimicking the motivating data from a pediatric rare disease. Among the methods examined, the performance of coarsened exact matching (CEM) and TMLE are relatively robust under various model specifications. CEM is only feasible when the number of controls far exceeds the number of treated, whereas TMLE has better performance with less extreme treatment allocation ratios. Our simulations suggest bootstrap is useful for variance estimation in small samples after matching.

Treatment of pediatric grave's disease: a single-center experience

Treatment of pediatric grave's disease: a single-center experience

Closing the gap between acceptable and ideal in catheterisation for paediatric and congenital heart disease-A global view.

In recent issues of the Journal of the Society for Cardiovascular Angiography and Interventions and the Journal of the American College of Cardiology: Cardiovascular Interventions, Holzer and colleagues presented an Expert Consensus Document titled: "PICS / AEPC / APPCS / CSANZ / SCAI / SOLACI: Expert consensus statement on cardiac catheterization for pediatric patients and adults with congenital heart disease." This Expert Consensus Document is a massively important contribution to the community of paediatric and congenital cardiac care. This document was developed as an Expert Consensus Document by the Pediatric and Congenital Interventional Cardiovascular Society, the Association for European Paediatric and Congenital Cardiology, the Asia-Pacific Pediatric Cardiac Society, the Cardiac Society of Australia and New Zealand, the Society for Cardiovascular Angiography and Interventions, and the Latin American Society of Interventional Cardiology, as well as the Congenital Cardiac Anesthesia Society and the American Association of Physicists in Medicine.As perfectly stated in the Preamble of this Expert Consensus Document, "This expert consensus document is intended to inform practitioners, payors, hospital administrators and other parties as to the opinion of the aforementioned societies about best practices for cardiac catheterisation and transcatheter management of paediatric and adult patients with congenital heart disease, with added accommodations for resource-limited environments." And, the fact that the authorship of this Expert Consensus Document includes global representation is notable, commendable, and important.This Expert Consensus Document has the potential to fill an important gap for this patient population. National guideline documents for specific aspects of interventions in patients with paediatric heart disease, including training guidelines, do exist. However, this current Expert Consensus Document authored by Holzer and colleagues provides truly globally applicable standards on cardiac catheterisation for both paediatric patients and adults with congenital heart disease (CHD).Our current Editorial provides different regional perspectives from senior physicians dedicated to paediatric and congenital cardiac care who are practicing in Europe, the Asia-Pacific region, Latin America, Australia/New Zealand, and North America. Establishing worldwide standards for cardiac catheterisation laboratories for children and adults with CHD is a significant stride towards improving the quality and consistency of care. These standards should not only reflect the current state of medical knowledge but should also be adaptable to future advancements, ultimately fostering better outcomes and enhancing the lives of individuals affected by CHD worldwide.Ensuring that these standards are accessible and adaptable across different healthcare settings globally is a critical step. Given the variability in resources and infrastructure globally, the need exists for flexibility and tailoring to implement recommendations.The potential impact of the Expert Consensus Document and its recommendations is likely significant, but heterogeneity of healthcare systems will pose continuing challenges on healthcare professionals. Indeed, this heterogeneity of healthcare systems will challenge healthcare professionals to finally close the gap between acceptable and ideal in the catheterisation of patients with paediatric and/or congenital heart disease.

Pediatric to Adult Transition in Inflammatory Bowel Disease: Consensus Guidelines for Australia and New Zealand.

The incidence of pediatric inflammatory bowel disease (IBD) is rising, and there is an increasing need to support adolescents when they transition to adult care. Evidence supports the use of a structured transition process but there is great variation across Australasia. The study aim was to develop evidence and expert opinion-based consensus statements to guide transitional care services in IBD. A modified UCLA-RAND methodology was employed to develop consensus statements. An IBD expert steering committee was formed and a systematic literature review was conducted to guide the drafting of consensus statements. A multidisciplinary group was formed comprising 16 participants (clinicians, nurses, surgeons, psychologists), who anonymously voted on the appropriateness and necessity of the consensus statements using Likert scales (1 = lowest, 9 = highest) with a median ≥7 required for inclusion. Patient support groups, including direct input from young people with IBD, informed the final recommendations. Fourteen consensus statements were devised with key recommendations including use of a structured transition program and transition coordinator, mental health and transition readiness assessment, key adolescent discussion topics, allied health involvement, age for transition, and recommendations for clinical communication and handover, with individualized patient considerations. Each statement reached median ≥8 for appropriateness, and ≥7 for necessity, in the first voting round, and the results were discussed in an online meeting to refine statements. A multidisciplinary group devised consensus statements to optimize pediatric to adult transitional care for adolescents with IBD. These guidelines should support improved and standardized delivery of IBD transitional care within Australasia.

STEP-CD study: ustekinumab use in paediatric Crohn's disease-a multicentre retrospective study from paediatric IBD Porto group of ESPGHAN.

Ustekinumab is an effective therapy for adult Crohn's disease (CD), but data in paediatric CD patients are scarce. The aim of the study was to describe the real-life effectiveness and safety of ustekinumab in paediatric CD. This is a multicentre review of children with Crohn's disease treated with ustekinumab. The aim of our study was to describe the effectiveness and safety of ustekinumab in paediatric real-life practice. This is a study of the Paediatric IBD (inflammatory bowel disease) Porto group of ESPGHAN. Corticosteroid (CS)- and exclusive enteral nutrition (EEN)-free remission, defined as weighted Paediatric Crohn's Disease Activity Index (wPCDAI) < 12.5, and physician global assessment (PGA) were determined at weeks 12 and 52. A total of 101 children were included at a median age of 15.4 years (IQR 12.7-17.2) with a median follow-up of 7.4 months (IQR 5.6-11.8). Ninety-nine percent had received prior anti-TNF, 63% ≥ 2 anti-TNFα therapies and 22% vedolizumab. Baseline median wPCDAI was 39 (IQR 25-57.5) (71 (70%) patients with moderate-severe activity). Weeks 12 and 52 CS- and EEN-free remission were both 40.5%. Clinical response at week 6, iv induction route and older age at onset of ustekinumab treatment were predictive factors associated with clinical remission at week 12. Seven minor adverse events probably related to ustekinumab were reported. One patient died from an unrelated cause. Conclusion: Our results suggest that ustekinumab is effective and safe in children with chronically active or refractory CD. What is Known: • Ustekinumab is an effective therapy for adult moderate to severe Crohn's disease (CD). • Off-label use of ustekinumab in children is increasing especially in anti-TNF refractory CD. What is New: • Is the largest cohort of real-world use of ustekinumab in paediatric CD to date. • Clinical response at week 6, iv induction and older age at onset of ustekinumab were predictive factors associated with clinical response at week 12.

Advance Care Planning for Children With Rare Diseases: A Pilot RCT.

Pediatric rare diseases are often life-limiting conditions and/or require constant caregiving. Investigators assessed the initial efficacy of the FAmily CEntered (FACE) pediatric advance care planning (pACP), FACE-Rare, intervention on families' quality of life. A pilot-phase, single-blinded, intent-to-treat, randomized controlled clinical trial enrolled families from 1 pediatric quaternary hospital between 2021 and 2023. Intervention families received 3 weekly 60-minute (FACE-Rare pACP) sessions: (1) Carer Support Needs Assessment Tool or Action Plan, (2) Carer Support Needs Assessment Tol Action Plan Review, and (3) Pediatric Next Steps: Respecting Choices pACP. Controls received treatment as usual (TAU). Outcome measures were Beck Anxiety Inventory, Family Appraisal of Caregiving, Functional Assessment of Chronic Illness Therapy (FACIT)-Spirituality, and health care utilization. Generalized mixed effect models with γ response assessed the intervention effect at 3-month follow-up. Children (n = 21) were aged 1 to 10 years, 48% male, 24% Black; and 100% technology dependent. Primary family caregivers (n = 21) were aged 30 to 43 years, 19% male, 19% Black; and 27% household income below the Federal poverty level. Dyads underwent 1:1 randomization: 9 to FACE-Rare and 12 to TAU. TAU caregivers reported statistically lower meaning and peace than FACE-Rare caregivers (0.9, P = .03, confidence interval [CI]: 0.75-0.99). Black caregivers reported significantly less caregiver distress (0.7, P = .04, CI: 0.47-0.98) than non-Black caregivers. Poor families reported more anxiety (3.5, P = .002, CI: 1.62-7.94), more caregiver strain (1.2, P = .006, CI: 1.07-1.42); and less family well-being (0.8, P = .02, CI: 0.64-0.95). FACE®-Rare was feasible, acceptable, safe, and demonstrated initial efficacy, providing greater feelings of meaning and peace to caregivers. Poverty impacted well-being. A multisite trial is needed to determine generalizability.

Biomarkers predicting the effect of anti-TNF treatment in paediatric and adult inflammatory bowel disease.

Paediatric and adult inflammatory bowel disease (pIBD, aIBD) patients may lose response to anti-TNF treatment within the first year. Adult-extrapolated weight-based dosing is incorrect in children, due to age-related pharmacokinetic differences. We investigated biomarkers for initial and maintenance of response to infliximab (IFX) or adalimumab (ADA), comparing pIBD and aIBD patients. In this prospective, observational study, pIBD (n = 24) and aIBD (n = 21) patients were included when initiating anti-TNF. Escalation from standard dosing and continued anti-TNF at 12 and 18 months were assessed. Biomarkers included clinical laboratory parameters, faecal calprotectin (FCP) and IFX trough levels (TLs). Plasma proteomics was performed in pIBD. During our study, treatment escalation (in clinical loss of response) occurred more common in pIBD versus aIBD (p = 0.02). We established that IFX therapy escalation in pIBD patients was not due to low infliximab levels. We identified 9 pro-inflammatory proteins that were elevated in patients losing response. Anti-TNF exposure-response relationship may be different in pIBD versus aIBD. No biomarkers for maintained response were identified, but 9 inflammatory proteins were of interest as potential predictors for loss of response in pIBD.

Utilization and perspectives of weight loss medications in pediatric metabolic dysfunction-associated steatotic liver disease.

Metabolic dysfunction-associated steatotic liver disease (MASLD) is increasing globally in pediatric populations. Currently, MASLD management primarily relies on lifestyle interventions, which pose challenges in sustaining long-term weight loss. This study investigated the use of weight loss medications in MASLD care through an international survey of 166 pediatric gastroenterologists and hepatologists. The results indicated a notable interest in weight loss medications, with 38% of practitioners considering or using them, particularly glucagon-like peptide-1 receptor agonists. However, the survey also revealed a tendency among clinicians to refer patients to specialists, emphasizing the potential gap between acknowledgment and prescription practices. Challenges include the lack of guidelines and uncertainty regarding side effects. The study highlights a pressing need for education, with over 90% of the respondents expressing an interest. Our study highlights the current management of MASLD, the potential role of pharmacotherapy, and highlights avenues for improved care and education in this dynamic field.

Nontuberculous mycobacterial disease in children: A systematic review and meta-analysis

BackgroundThe prevalence of nontuberculous mycobacterial (NTM) disease in children is increasing worldwide. The clinical manifestations of pediatric NTM patients are significantly different from those of adult patients, but the knowledge of the disease is generally poor. MethodsEnglish databases (PubMed, Web of Science, Embase, BIOSIS) and Chinese databases (CNKI, Wanfan, VIP) were searched on October 15th, 2022. All the articles of cross-sectional and cohort studies reporting the species composition and lesion site of the NTM disease in children using well-recognized NTM species identification methods were taken into account. Using a random effects model, we assessed the disease lesion sites and the prevalence of different NTM species in pediatric NTM disease. Sources of heterogeneity were analyzed using Cochran's Q and the I2 statistic. All analyses were performed using CMA V3.0. ResultsThe prevalence rates of NTM disease in children ranged between 0.6 and 5.36/100,000 in different countries, and Europe reported the highest prevalence rate. The most common clinical lesion site was lymph node, accounting for 71.1 % (55.0 %–83.2 %), followed by lung (19.3 %, 9.8%–34.4 %）and then skin and soft tissue (16.6 %,13.5%–20.3 %). Mycobacterium avium complex (MAC) was the most isolated NTM pathogen in children, accounting for 54.9 % (39.4%–69.6 %). Inconsistent with adult patients, Mycobacterium avium accounted for a dominant proportion in MAC than Mycobacterium intracellulare. ConclusionsThe lymph node was the most affected organ in pediatric NTM disease, while Mycobacterium avium was the most isolated pathogenic species in children.

Serologic Evaluation for Severe Acute Respiratory Syndrome Coronavirus 2 in Pediatric Heart Transplantation Recipients and Patients on a Pediatric Heart Transplantation Waiting List in a Quaternary Hospital

BackgroundThe COVID-19 pandemic has been a global reality for over three years. Serological studies have great importance for the comprehension of the virus's behavior in populations, as it can suggest the status of the epidemic in a community. This cross-sectional study aimed to analyze the serological profile for COVID-19 in patients before and after pediatric heart transplant. MethodsSerologies for IgG and IgM antibodies for Sars-CoV-2 were collected in patients of the Pediatric Cardiology and Congenital Heart Diseases unit of a Brazilian hospital between January and August 2022. In total, 174 patients were recruited: 28 on waiting list and 146 recipients. Information such as demographics (age, gender, state of origin), type of heart disease (congenital or acquired) and time after transplant were analyzed. ResultsOverall, 72 patients had a positive serology for anti-N antibodies (48,0%), being 62 transplant recipients and 10 patients on transplant waiting list, with positivity rates of 48,1% and 47,6%, respectively. Positivity rates for previously infected individuals were 62,5% and 62,1%, respectively. ConclusionsWe found that approximately half of the sample has developed IgM or IgG antibodies against the Sars-CoV-2 virus Serological studies are a relevant tool to public health for health promotion of vulnerable groups, and can be useful to future studies on the antibodies’ behavior. The duration and the level of protection given by these antibodies is still a subject being studied, and can become a key information for the protection of these populations if discovered in the future.

Increased Farnesoid X Receptor agonism in frontal cortex is associated with neuronal loss in a pig model of pediatric non-alcoholic fatty liver disease

Clinical studies suggest a link between non-alcoholic fatty liver disease (NAFLD) and neurodegeneration, but the mechanisms connecting both pathologies remain unknown. Rodent models of liver failure have shown neurological decline to be associated with increased bile acid (BA) levels and BA-activated farnesoid X receptor (FXR) signaling in the brain, which may disrupt brain’s cholesterol metabolism and promote neuronal death. We have previously established a pediatric animal model of NAFLD by feeding juvenile Iberian pigs a diet high in fat and fructose (HFF) for 10 weeks. The aim of this study was to determine whether BAs and FXR gene expression were increased in the frontal cortex (FC) of NAFLD pigs, and whether these changes were associated with neuronal loss. Eighteen 15-day-old Iberian pigs were assigned to receive for 10 weeks either a control (CON) or a HFF diet. FC was collected for immunohistochemistry, metabolomics, and transcriptomics analyses. Functional enrichment analyses were performed on differentially expressed genes to identify Gene Ontology (GO) terms using the Database for Annotation, Visualization, and Integrated Discovery. All HFF-fed pigs developed NAFLD. Immunostaining of mature neurons with NeuN was lower in FC of HFF compared with CON (P ≤ 0.05), whereas total BA levels increased (P ≤ 0.05) in FC of HFF-fed pigs. Brain levels of cholic and conjugated deoxycholic acids, which are FXR agonists, increased ( P ≤ 0.05) in HFF compared with CON. Conversely, tauroursodeoxycholic acid, which has strong FXR-antagonistic effect and has been shown to exert neuroprotective functions, was decreased (P ≤ 0.001) in HFF and was positively correlated with NeuN staining intensity (R2 = 0.623; P ≤ 0.001). Expression of FC genes encoding FXR, BA synthetic enzymes sterol 24- and 27-hydroxylases, and BA transporters Na+-taurocholate cotransporting polypeptide, organic anion transporting polypeptide, and organic solute transporters alpha and beta did not differ between groups. GO terms associated with Wnt/β-catenin pathway, which promotes neuronal survival and neurogenesis, were downregulated in HFF-fed pigs ( P ≤ 0.05), whereas mitochondrial respiratory chain complex and mitochondrion increased in HFF compared with CON ( P ≤ 0.01). In conclusion, increased BA levels and FXR agonism in frontal cortex of NAFLD pigs was associated with dysregulated Wnt/β-catenin signaling pathway and neuronal loss. California State University Agriculture Research Institute (grants 58873 and 58913), California Polytechnic State University internal funding programs Baker/Koob, RSCA, Frost, BiOWiSH Technologies, Hilmar Ingredients, and Acorn Seekers. This is the full abstract presented at the American Physiology Summit 2024 meeting and is only available in HTML format. There are no additional versions or additional content available for this abstract. Physiology was not involved in the peer review process.

Factors affecting care burden and life satisfaction among parents of children with type 1 diabetes

AimParents in the caregiving role for children with type 1 diabetes (T1D) are affected from many aspects including physically, psychologically, socially, financially and spiritually. The aim of this study was to determine the factors affecting the care burden and life satisfaction of parents caring for a child with T1D and to examine the relationship between them. MethodThis research was completed with 293 parents of children with T1D aged 3–18 years, attending the pediatric health and diseases clinics in two state hospitals located in [redacted for peer review] from May 2023 to September 2023. For collection of data, a survey form, the Zarit Burden Interview (ZBI) and Adult Life Satisfaction Scale (ALSS) were used. Analysis of data used percentages, means, one-way ANOVA (post-hoc Tukey test), independent samples t-test, regression and correlation tests. Statistical significance level was accepted as p < 0.05. ResultsParents had mean care burden scale scores of 56.30 ± 12.4, with mean adult life satisfaction scale scores of 69.39 ± 11.61. According to the regression analysis results, the ZBI scores were affected by the child's general health status after T1D diagnosis, impact on responsibilities after T1D diagnosis and diabetes management skills (p < 0.05). ALSS scores were affected by the variables of caregiver assessment of health status before and after the child's T1D diagnosis, income level, impact on responsibilities after T1D diagnosis and diabetes management skills (p < 0.05). There was a negative weak level of significant correlation identified between the ZBI and ALSS total scores (r = −0.36; p < 0.05). ConclusionGeneral health status after receiving T1D diagnosis, impact on responsibilities after diabetes diagnosis and diabetes management skills are factors affecting the care burden and life satisfaction of parents. Additionally, a negative relationship was determined between parents' care burden and life satisfaction. Nurses should develop family-centered care plans and work to reduce factors increasing care burden while increasing life satisfaction.

Transitional Medicine of Intractable Primary Dyslipidemias in Japan.

Transitional medicine refers to the seamless continuity of medical care for patients with childhood-onset diseases as they grow into adulthood. The transition of care must be seamless in medical treatment as the patients grow and in other medical aids such as subsidies for medical expenses in the health care system. Inappropriate transitional care, either medical or social, directly causes poorer prognosis for many early-onset diseases, including primary dyslipidemia caused by genetic abnormalities. Many primary dyslipidemias are designated as intractable diseases in the Japanese health care system for specific medical aids, as having no curative treatment and requiring enormous treatment costs for lipid management and prevention of complications. However, there are problems in transitional medicine for primary dyslipidemia in Japan. As for the medical treatment system, the diagnosis rate remains low due to the shortage of specialists, their insufficient link with generalists and other field specialists, and poor linkage between pediatricians and physicians for adults. In the medical care system, there is a mismatch of diagnostic criteria of primary dyslipidemias between children and adults for medical care expense subsidization, as between The Program for the Specific Pediatric Chronic Diseases and the Program for Designated Adult Intractable Diseases. This could lead some patients subsidized in their childhood to no longer be under the coverage of the aids after transition. This review intends to describe these issues in transitional medicine of primary dyslipidemia in Japan as a part of the efforts to resolve the problems by the Committee on Primary Dyslipidemia under the Research Program on Rare and Intractable Disease of the Ministry of Health, Labour and Welfare of Japan.

Frequency, characteristics, predictors and treatment of relapsing myelin oligodendrocyte glycoprotein antibody–associated disease (MOGAD)

BackgroundMyelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) may have a monophasic or relapsing disease course. To date, factors that may predict a relapsing disease course remain largely unknown and only limited data exist regarding the efficacy of different utilized immunotherapy regimens at preventing or reducing relapses. ObjectivesTo assess the characteristics, predictors, and immunotherapy of relapsing MOGAD. MethodsThis multicenter retrospective analysis included all MOGAD cases at the University of Florida, Baylor College of Medicine and the University of California San Diego with minimum follow-up time of 6 months. Cox proportional hazards regression analyses, corrected for age and sex, were performed to evaluate hazard ratios (HR) of predictors of a relapsing disease course and to compare relapse hazards for utilized immunotherapies. ResultsThe majority of included participants (51/79 [64.6 %]) had a relapsing course, and of these individuals, 68.6 % (35/51) experienced their first relapse within the first year. However, 10/51 (19.6 %) participants experienced their first relapse ≥5 years (5–15 years) after the initial presentation. Predictors of a relapsing course were CSF pleocytosis (>150 cells/mm3; HR 3.3 [1.18 – 9.24]; p = 0.023), a pediatric disease onset at age < 9 years (HR 2.69 [1.07–6.75]; p = 0.035), and an initial presentation with the clinical syndrome of meningoencephalitis (HR 3.42 [1.28 – 9.17]; p = 0.015),. In participants with a relapsing course, 13/24 (54.2 %) patients remained relapse-free on rituximab, 4/8 (50 %) on mycophenolate mofetil, and 11/14 (78.6 %) on scheduled immunoglobulins. Patients treated with immunoglobulins had significantly fewer relapses compared to patients treated with other immunotherapies (HR: 0.1 [0.2 – 0.63]; p = 0.014). ConclusionsIn our cohort, the majority of MOGAD patients relapsed. The initial relapse occurred most frequently within the first year, but first relapses also took place over a decade after the initial presentation. Prepubertal onset, severe CSF pleocytosis, and the clinical syndrome of meningoencephalitis may be predictors of a relapsing course. Of the currently available off-label steroid-sparing treatments, scheduled immunoglobulins may be the most effective in relapse prevention.

Evaluation of the Effect of Medications Used in Children with Rheumatic Diseases on Dental Maturation.

The study's main objective is to compare dental maturation in healthy children and those with rheumatic diseases. It also aims to determine the age of children with Pediatric Rheumatic Diseases (PRD), specifically focusing on comparing those receiving medical treatment to those who are not, and to differentiate between biological and non-biological medication recipients, to identify potential differences. This observational cross-sectional study was conducted to assess the dental maturation stages in a cohort of 278 children (126 boys, 152 girls) aged between 6 and 14 years between September 2021 and December 2023. The study population included individuals with PRD requiring dental treatment and systemically healthy children. Digital panoramic radiographs were utilized for the evaluation of dental maturation in both groups. A statistically perfect agreement was found among the dental age (DA) calculated by the researchers (ICC=1.000; p&lt;0.01). A statistically perfect level of agreement was found between the chronological age (CA) of the cases and the DA measured by the Willems method (ICC=0.921; p&lt;0.01). It was found that the difference between the estimated DA and CA was higher in girls and boys in the PRD group who were taking medication. No significant differences were found in DA estimated based on gender and treatment status among individuals with PRD. The Willems method tended to underestimate the age of girls receiving biological treatment while overestimating the age in all other groups.

Orbital apex syndrome in pediatric thyroid eye disease: a case report and literature review.

Orbital apex syndrome in pediatric thyroid eye disease: a case report and literature review.

Pilot and feasibility of the SMART IBD mobile app to improve self-management in pediatric inflammatory bowel disease.

Access to evidence-based self-management support in pediatric inflammatory bowel disease (IBD) is a significant challenge. Digital therapeutic solutions can increase access and provide data to patients and providers that would otherwise not be available. We have iteratively developed a mobile application, Self-Management Assistance with Recommended Treatment (SMART) IBD, that allows patients to access self-management support and record symptoms and medication adherence. We conducted a pilot and feasibility study for this digital therapeutic tool in which patients used SMART IBD for 30 days. Results indicated that patients rated the app quality as good and accessed the app adequately overall, with some pages being used often. Medication adherence increased over the course of the study and was associated with sleep duration, mood, and stool consistency and blood content. Overall, this study demonstrated adequate feasibility for the SMART IBD app and initial findings suggest that additional research is needed to explore the potential impact of this tool in clinical care.