Pediatric Studies Research Articles

Parameningeal Rhabdomyosarcoma: Results of the European Pediatric Soft Tissue Sarcoma Study Group RMS 2005 Study.

Parameningeal (PM) site is an unfavorable characteristic in rhabdomyosarcoma (RMS). We described the treatment and outcome for patients with PM RMS and investigated the prognostic value of risk factors. We scored PM site by originating site and by highest risk extension. Patients with PM RMS were treated within the European pediatric Soft tissue sarcoma Study Group (EpSSG) RMS 2005 study with risk-adapted, multi-modal treatment. Three-hundred-eighty-one patients with PM RMS were included. Radiotherapy was administered in 359 patients (77 with surgery). After a median follow-up of 75 months, 5-year event-free survival was 60% (95% confidence interval (CI) 55%-65%), 5-year overall survival was 65% (95% CI 60%-70%). The outcome for patients with PM RMS has not improved in comparison to previous historical studies, despite the more rigorous application of radiotherapy (94% of patients). Signs of meningeal involvement, PM site, and age at diagnosis remained prognostic risk factors. EudraCT number 2005-000217-35.

Interhypothalamic adhesions: prevalence, structure, and location-based classification map in pediatric patients undergoing MRI.

Interhypothalamic adhesions (IHAs) have been reported only in the pediatric population, with unknown prevalence and histological composition. We aim to demonstrate their prevalence, assess their persistence through longitudinal imaging, classify IHAs by anatomical distribution, explore their structure, and report associated pathologies. A retrospective review was conducted on consecutive pediatric brain MRI studies obtained between January 2012, and December 2013. The presence of an IHA was only confirmed when observed on at least two planes. For each IHA, cross-sectional area was calculated, and signal intensities were measured at the center on sagittal T2WIs. Signal intensities were also measured in both cerebral white matter and gray matter for normalization and comparison. Patient demographics and clinical information were collected from electronic charts. Out of 1550 patients (0-17.9 years), 33 (19 males, 14 females) had an IHA, resulting in a 2.13% prevalence. Follow-up images were available for 19 IHA-positive patients, and IHAs were again seen in 92% of the follow-up scans (71/77). Normalized IHA signal highly correlated with normalized gray matter signal (r = 0.83, P < 0.001), but not with normalized white matter signal (r = -0.16, p = 0.494). Common co-occurring pathologies included hydrocephalus (n = 9), prematurity (n = 8), and corpus callosum abnormalities (n = 7). All type 3 IHAs (3/3) were accompanied by pituitary pathologies. IHAs have a prevalence of 2.13% in our cohort, and the majority persist in longitudinal studies. They showed gray matter signal intensity and Type 3 IHAs exclusively accompanied pituitary abnormalities.

Anxiety, depression, and headache-related disability in a large pediatric clinic-based sample.

To explore the relationships among anxiety, depression, and headache-related disability in a pediatric clinic-based retrospective cross-sectional study. Anxiety and depression are commonly considered comorbidities of headache disorders and are frequently seen in children and adolescents. These conditions can contribute to disability and may have a cumulative impact. In this study, we tested whether self-reported anxiety and/or depression in youth were associated with headache-related disability. This was a retrospective cross-sectional study of children ages 6-17 years old who completed a headache intake questionnaire at the time of a new outpatient neurology visit. Those who reported on behavioral health symptoms, involvement of a behavioral health provider (i.e., yes/no), and the PedMIDAS (a validated metric of headache-related disability) were included. The relationship between anxiety and/or depression and headache-related disability was examined. Of the 12,660 questionnaires queried, 9118 met criteria for inclusion. Respondents were 64.0% female and had a median age of 13.5 years (interquartile range [IQR] 10.3, 15.7). Compared to patients without self-reported anxiety/depression, patients with anxiety and depression reported higher headache-related disability (M = 17.0, [IQR 6.0, 41.0]) even after accounting for covariates (estimated difference = 6.0, 95% confidence interval [CI: 4.4-7.5]). For participants with anxiety and/or depression, having a behavioral health provider was associated with greater headache-related disability (estimated difference = 7.0; 95% CI 4.6-9.3). Patients with self-reported anxiety and/or depression reported higher headache-related disability. Having a behavioral health provider was associated with greater headache-related disability, indicating the complexity and high level of need for this population. Further research is needed to understand the directionality of these results; however, patients with headache as well as depression and or anxiety are a vulnerable group who may benefit from an integrated care model.

From Acute Pancreatitis to Diabetes: Risk Factors and Timeline Analysis in a Pediatric Population Study

Abstract Objectives Identify the risk profile for post-pancreatitis diabetes mellitus (PPDM) in children after a single acute pancreatitis (AP) episode, investigate episode severity's association with complications, length of stay (LOS), and intensive care unit (ICU) needs. Methods This cross-sectional study used the Pediatric Health Information System database for patients ≤19 years old with index AP admissions (2011-2020). A query (2012-2021) was performed for diabetes mellitus (DM). Patients were excluded if they had prior DM or pancreatic surgery on or before AP or DM diagnoses. Results Out of 12,822 patients with index AP admissions (median age: 13.4 years, 54% female), 686 (5.4%) developed PPDM, 320 (2.5%) during and 366 (2.9%) at a later admission. The median collective time to PPDM during and after index AP admission was 1.9 months (IQR: 0-20.8). Patients with PPDM experienced extended LOS, heightened ICU needs, and increased complications such as sepsis, pancreatic necrosis, cyst formation, along with higher rates of organ dysfunction and comorbidities. Conclusions Children face DM risks during or following AP episodes. Enhanced DM screening is crucial for patients with existing comorbidities, local or systemic complications or organ dysfunction. Close outpatient follow-up within 3-6 months of discharge from an index AP episode is recommended.

Interictal EEG spikes increase perfusion in low-grade epilepsy-associated tumors: a pediatric arterial spin labeling study.

Arterial spin labeling (ASL), a noninvasive magnetic resonance (MRI) perfusion sequence, holds promise in the presurgical evaluation of pediatric lesional epilepsy patients, including those with low-grade epilepsy-associated tumors (LEATs). The interpretation of ASL-derived perfusion patterns, however, presents challenges. Our study aims to elucidate these perfusion changes in children with LEATs, exploring their correlations with clinical, electroencephalography (EEG), and anatomical MRI findings. Our cohort included 15 children with LEAT-associated focal lesional epilepsy who underwent single-delay pseudo-continuous ASL imaging; eight were imaged under sedation. We assessed perfusion images both qualitatively and quantitatively, focusing on LEAT-related perfusion changes, as indicated by the asymmetry index (AI) and regional cerebral blood flow (rCBF). ASL revealed LEAT-related perfusion changes in all but two patients: 12 LEATs were hypoperfused and one was hyperperfused relative to the contralateral brain parenchyma (CBP). LEATs showed significantly lower perfusion compared to CBP (median: 38.7 vs. 59.1mL/100g/min for LEAT and CBP, respectively; p value = 0.004, Wilcoxon-Mann-Whitney), regardless of sedation. Notably, elevated AI and rCBF values correlated with interictal spikes on EEG (median: -0.008 and 0.84 vs -0.27 and 0.58, respectively), but not to other clinical, EEG, or MRI variables (p value = 0.036, Wilcoxon-Mann-Whitney). By highlighting the connection between LEAT and brain perfusion, and by correlating perfusion characteristics and epileptogenicity, our research enhanced our understanding of pediatric epilepsy associated with LEATs. Also, by proving the robustness of these findings to sedation we confirmed the importance of adding ASL to epilepsy protocols to as a valuable tool to supplement anatomical imaging.

CTIM-34. EFFECTS OF NEOADJUVANT IMMUNE CHECKPOINT INHIBITION ON PEDIATRIC AND ADULT HIGH-GRADE GLIOMAS

Abstract High-grade gliomas (HGG) are lethal tumors with few long-term survivors affecting adult and pediatric patients. Studies investigating immune checkpoint inhibition (ICI) suggest administration of neoadjuvant anti-PD1 blockade (neo-aPD1) enhances local and systemic anti-tumor responses. This data is supported by increased T cell density in the tumor and proportion of cytotoxic T cells in peripheral blood. In a previous randomized clinical trial, neo-aPD1 improved survival in adult with recurrent glioblastoma. In this study, we utilized multiplex immunofluorescence (mIF), and single-cell RNA sequencing (scRNA-seq) to investigate the effects of neoadjuvant ICI on the TME and compare between pediatric and adult populations. Tissue samples from adult patients with recurrent GBM treated with neo-aPD1 (n=22) or adjuvant only therapy (n=5) were obtained from our UCLA off-label neo-PD1 study. Tissue samples were also obtained from pediatric patients with recurrent/progressive HGG treated with neoadjuvant ICI from the ongoing Pediatric Neuro-Oncology Consortium study PNOC19. Samples included diagnostic tissue from two patients and on study surgery tissue from four patients. All tissue samples were subjected to mIF and underwent imaging analysis to assess immune cell infiltration by quantifying lymphoid and myeloid cell densities across the two treatment groups. Our preliminary findings elucidated a trend of higher T cell infiltration in adult neoadjuvant compared to adjuvant-only treated patients (median density 58.11 vs 15.15 T-cells/mm2). Compared to their adult counterparts, neoadjuvant ICI treated pediatric patients had even more robust total T cell infiltration (median density 268.10 vs 58.11 T-cell/mm2, pediatric vs adult respectively). scRNA-seq analysis revealed a greater enrichment of monocytic macrophages in the pediatric population compared to tissue resident microglia in the adults. This early data requires analysis of additional samples to better understand differences in the anti-tumor immune response between adult and pediatric recurrent/progressive HGG patients.

Longer-term safety and efficacy of baricitinib for atopic dermatitis in pediatric patients 2 to

Background Baricitinib, an oral selective Janus kinase inhibitor, improved clinical signs and symptoms of moderate-to-severe atopic dermatitis (AD) at week 16 in the phase 3 pediatric study BREEZE-AD-PEDS. Objective To assess longer-term efficacy and safety of baricitinib in pediatric patients aged 2 to <18 years. Methods In BREEZE-AD-PEDS long-term extension, responders and partial responders (validated Investigator Global Assessment-Atopic Dermatitis [vIGA-AD®] 0/1/2) at Week 16 remained on double-blind treatment to which they were randomized (placebo, baricitinib [1-mg equivalent, 2-mg equivalent, or 4-mg equivalent); non-responders (vIGA-AD 3 or 4) at Week 16 transitioned to open-label baricitinib 4-mg equivalent. Safety was summarized for all randomized patients who received ≥1 dose of study treatment. Results In total 467 patients received baricitinib for 750.7 patient-years. Proportion of responders/partial responders (at Week 16) who achieved vIGA-AD 0/1 at Week 52 was greater for baricitinib 4-mg equivalent (56.8%) versus all other treatment groups (42.2%, 47.7%, and 39.7% for 2-mg equivalent, 1-mg equivalent, and placebo, respectively). Most treatment-emergent adverse events were mild/moderate in severity. No deaths, pulmonary emboli, deep vein thromboses or arterial thrombotic events, major adverse cardiovascular events, malignancies, tuberculosis events, or gastrointestinal perforations were reported. Conclusions Baricitinib demonstrated sustained long-term efficacy. No new safety signals were identified. Trial Registration ClinicalTrials.gov Identifier: NCT03952559

Clinicopathological Analysis of a European Cohort of MYOD1 Mutant Rhabdomyosarcomas in Children and Young Adults.

Patients with PAX3/7-FOXO1 fusion-negative rhabdomyosarcomas (fnRMS) harbouring the rare L122R MYOD1 mutation have significantly poorer prognosis than other fnRMS. We undertook a detailed clinicopathological evaluation of a cohort of patients with MYOD1 mutated fnRMS in order to improve risk stratification and treatment options. Histological, mutational and clinical data from a cohort of patients with MYOD1 mutant RMS treated in Europe were analysed. Thirty-two cases with mutant MYOD1 RMS were identified from patients enrolled in sequential European rhabdomyosarcoma clinical trials from 1992 to 2022 (n = 22) and non-trial cohorts (n = 10). Thirty cases had the recurrent L122R missense mutation, one case harboured a K124E mutation and one case had a truncating mutation (S63X). Increased MyoD1 and reduced MYF4 immunostaining were consistent features of MYOD1L122R-mutated RMS. Applying the risk stratification of the European paediatric Soft tissue sarcoma Study Group (EpSSG) RMS2005 trial, among 20 localised RMS cases that could be assigned a risk category, one was Very High Risk, 13 were High Risk and six were Standard Risk. Eight patients had distant metastases at diagnosis. Of the 25 patients with adequate clinical follow-up data, 15/25 (60%) patients had an event at a median time of 9months (12/15 included failure of local control) and 13/25 (52%) died of disease. This MYOD1 mutant cohort demonstrates increased MYOD and reduced MYF4 immunostaining, high risk of local failure and poor survival in agreement with other studies. Increased treatment intensity and improved local control should be considered for these patients.

Baseline Impulse Oscillometry Metrics Differentiate Asthmatic Children From Non-Asthmatic Children Across Ethnicity/Race: A Meta-Analysis.

Identifying the asthmatic early to prevent permanent airway remodeling and the progression of the disease is desirable. In children, baseline impulse oscillometry has been found effective in identifying asthma in some studies but not others. The purpose of our study was to utilize a meta-analysis to determine whether there were significant peripheral airway differences between asthmatic and non-asthmatic children across ethnicity/race, utilizing baseline impulse oscillometry (IOS) to establish its usefulness as a diagnostic tool in this age group. This was a comprehensive search of published literature on pediatric oscillometric studies evaluating younger children (mean age ranging from 4.3 to 6 years) and older children (mean age ranging from 8.7 to 11.4 years) from the United States, Europe, Asia, and Middle East. Inclusion criteria required the primary variable resistance at 5 Hertz (R5) (kPa/L/s) or (cmH2O/L/s) for both control and asthmatic subjects, and excluded studies if asthmatics had uncontrolled disease. Our data show that there are significantly higher R5 and area of reactance (AX) and lower reactance at 5 Hertz (X5) in both younger and older asthmatic children compared to healthy controls from various countries. This meta-analysis firmly establishes that baseline oscillometry metrics, resistance and reactance, are effective in identifying the asthmatic child across age and the ethnicities/races evaluated.

Clinical Presentation of Cat Scratch Disease in Pediatric Patients-A Single-Center Study.

Cat scratch disease (CSD) is the infectious disease caused by Bartonella henselae. Its typical presentation is regional lymphadenopathy. Also it may present with symptoms related to involved organs or disseminated disease with fever of unknown origin (FUO). Here children with CSD are evaluated to increase awareness about disease. A total of 29 children diagnosed with CSD between 2019 and 2022 were involved in the study. Patients' demographic characteristics, clinical, laboratory and radiological findings, treatments, and outcomes were analyzed. Seventeen of the patients were male, 12 were female, and their mean age was 116.5 ± 51 months. About 69.6% of them had a history of cat contact. Twenty-seven patient (93.1%) had lymphadenopathy, mostly axillary involved (61.5%). Other manifestations were disseminated disease presented with FUO, neuroretinitis, and encephalopathy. Twenty-seven patients (93.1%) had received antibiotics before admission without any improvement. Ultrasound showed that the affected lymph nodes were conglomerated, lobulated contoured, and cortical thickened, with one-third having cystic suppurative components. Serologic tests were positive in 24 of 27 patients. Twenty-one patients gave response to 5 days azithromycin treatment, in 8 patients this treatment extended to 10-14 days, rifampicin with/without doxycycline was given to 6 patients, and steroids were given to 3 patients. In case of regional lymphadenopathy, especially axillary, not responding to nonspecific antibiotics CSD should be suspected. Cat contact history and serological and ultrasonographic findings are useful for diagnosis. Even if CSD responds well to azithromycin, sometimes prolongation of azithromycin and addition of other antibiotic or steroid may be required.

Analysis of platelet transfusion efficacy during extracorporeal membrane oxygenation (ECMO) treatment in pediatric patients post-cardiac surgery-a retrospective cohort study.

Postoperative extracorporeal membrane oxygenation (ECMO) may be necessary for pediatric patients following cardiac surgery, with associated risks of thrombocytopenia and bleeding. Prophylactic platelet transfusions are utilized to mitigate these risks, but the effectiveness of platelet transfusion cannot be reliably predicted. The aim of this study was to investigate the effect of platelet transfusion during postoperative treatment with ECMO in children undergoing cardiac surgery and to explore the optimal transfusion thresholds to reduce the number of platelet transfusions in patients and reduce the risk of death. We included in our study patients from the Pediatric Cardiac Surgery Department at the First Affiliated Hospital of Tsinghua University who underwent cardiac surgery and received ECMO treatment from January 1, 2019, to December 31, 2023, and received platelets transfusion at least once during the ECMO therapy. The platelet counts were determined both before and 24 hours posttransfusion of the platelet product. The corrected count increment (CCI) was calculated for the effectiveness estimation of platelet transfusion. The research subjects were divided into 3 groups based on the platelet count before transfusion (pretransfusion platelet count ≤30×109/L was the low-threshold group, pretransfusion count 31-50×109/L was the medium-threshold group, and ≥51×109/L was the high-threshold group) and the effective rates of each group were calculated. A total of 11 patients received 47 platelet transfusions, an average of 4.27 ± 1.67 per patient. According to the 24-hour postinfusion platelet (Plt) corrected critical control increase index (24-hour CCI) ≥4500, the infusion was considered to be effective, and ineffective when the CCI was <4500. Out of these, 22 transfusions (46.8%) proved effective, whereas 25 (53.2%) were deemed ineffective. The effective transfusion rates across the 3 groups were 69.2%, 50%, and 27.7%, respectively. The efficacy of platelet transfusion may be higher if a low threshold of platelet transfusion is chosen during ECMO treatment, on the premise of ensuring life safety.

Factors influencing the initiation of biologic therapy in children with severe asthma: Results of the pediatric asthma noninvasive diagnostic approaches (PANDA) study.

Despite the availability of biologics for severe pediatric asthma, real-life studies reporting on drivers behind initiating biologics and their alignment with the Global Initiative for Asthma (GINA) recommendations are lacking. We performed analysis within the pediatric asthma noninvasive diagnostic approaches study, a prospective cohort of 6- to 17-year-old children with severe asthma. Information was collected on demographic factors, symptom control, treatment, comorbidities, and diagnostic tests from medical records and questionnaires. We divided patients into "starters" or "nonstarters" based on the clinical decision to initiate biologics and performed multivariate logistic regression analysis to identify drivers behind initiating therapy. Additionally, we assessed patient suitability for biologics according to key factors in the GINA recommendations: Type 2 inflammation, frequency of exacerbations, and optimization of treatment adherence. In total, 72 children (mean age 11.5 ± 3.0 years, 65.3% male) were included (13 starters). Initiation of biologics was associated with a higher GINA treatment step (adjusted odds ratio's [aOR] = 5.0, 95%CI 1.33-18.76), steroid toxicity (aOR = 21.1, 95%CI 3.73-119.91), frequency of exacerbations (aOR = 1.6, 95%CI 1.10-2.39), improved therapy adherence (aOR = 1.7, 95%CI 1.10-2.46), Caucasian ethnicity (aOR = 0.20, 95%CI 0.05-0.80), ≥1 allergic sensitization (aOR = 0.06, 95%CI 0.004-0.97), and allergic rhinitis (aOR = 0.13, 95%CI 0.03-0.65). Furthermore, steroid toxicity was identified as an important factor for deviation from the current recommendations on biologic prescription. We identified multiple drivers and inhibitors for initiating biologics, and showed the clinical need for biologics in severe pediatric asthmatics suffering from steroid toxicity. These findings may help refine asthma management guidelines.

Associations of food group intakes with serum carbon isotope ratio values in youth: results from two prospective pediatric cohort studies

BackgroundThe carbon isotope ratio (CIR) is a candidate biomarker for sugar sweetened beverage (SSB) intake in the U.S. However, research specific to youth, who differ in their physiology and dietary patterns compared to adults, is lacking. ObjectivesWe evaluated longitudinal associations of SSB intakes across childhood/adolescence with serum CIR. We also explored the relationship between other dietary intakes and serum CIR. MethodsData were from participants in two longitudinal, pediatric cohorts in Colorado: Exploring Perinatal Outcomes among CHildren Study (EPOCH) (visits at median 10 and 16 y, n=150) and Healthy Start Study (visits at median 5 and 9 y, n=166). Serum CIR was measured using isotope ratio mass spectrometry. Diet was assessed by food frequency questionnaires (EPOCH) or 24-hour diet recalls (Healthy Start). We assessed associations of longitudinal dietary intakes (log2-transformed, standardized) with serum CIR using linear mixed models adjusted for age, sex, and energy intake, and associations of change values between visits using linear regression models. ResultsIn linear mixed models, higher SSB intake across visits was associated with higher serum CIR in both cohorts (β [95% CI]: 0.11[0.06,0.15] in EPOCH and 0.14[0.07,0.21] in Healthy Start). Higher meat intake and a higher dietary animal protein ratio were also positively associated with serum CIR in both cohorts (β [95% CI]: 0.08[0.05,0.12] and 0.18[0.13,0.23] in EPOCH; 0.08[0.01,0.16] and 0.28[0.21,0.35] in Healthy Start). In change analyses, there were positive associations for changes in the dietary animal protein ratio between visits with changes in serum CIR in both cohorts, but not for changes in SSB intake. ConclusionsOur findings support serum CIR as a potential biomarker of SSB intake in youth cross-sectionally; however, there was not a strong link between change values over longer-term follow-up. Meat/animal protein intake was also consistently and, at times, more strongly associated with serum CIR.

Evaluation of Smell Function in Patients with Childhood Epilepsy with Centrotemporal Spikes at a Pediatric Neurology Clinic-A Case-Control Study.

Objective: Childhood epilepsy with centrotemporal spikes (CECTS) is associated with cognitive, behavioral, and language difficulties. These epileptic discharges predominantly occur in the temporal lobe, which is known to be involved in olfactory functions. This study aims to assess olfactory dysfunction in patients with CECTS. Methods: This study included patients diagnosed with CECTS who were attending follow-ups at the Department of Child Neurology between January 2022 and July 2023. Olfactory function was evaluated using the Sniffin' Sticks (Burghart GmbH, Wedel, Germany) 12-point screening test, which was administered to 44 patients and 35 controls. The smell test and the final control EEGs were performed simultaneously. Results: A total of 44 patients and 35 control subjects were enrolled in this study. The smell scores were significantly lower in the patient group compared to the control group (p = 0.029). The patient group had a very high prevalence of anosmia compared to the control group. The normosmia rate in the control group was significantly higher. No significant difference was observed in the smell scores based on EEG findings or antiepileptic drug type. Conclusions: Olfactory dysfunction was identified in patients with CECTS compared to healthy controls, yielding results consistent with findings for other types of epilepsy. Olfactory dysfunction was detected in a greater frequency among the patients diagnosed with CECTS than among the healthy control group, and similar results were obtained with other types of epilepsy. It was deduced that these patients may experience problems with smell sensitivity throughout their lives. The most important result of this study is that this condition should be taken into account in regard to patients' well-being and lives.

Virtual Children’s Hospital for Learning Pediatric Nursing

Abstract Background In the context of a funded Erasmus+ program, a platform of a Virtual Children’s Hospital for Learning Pediatric Nursing - VLPN is being developed. The education of nurses working with children, adolescents and families, needs to include specialized knowledge, skills, attitudes and behaviors that will empower them to provide optimal and high-quality care. The virtual platform enables the improvement of pediatric nursing competences of the nursing students through interactive and self-paced activities and content. Objectives 1. To develop an immersive and interactive virtual reality learning environment (VRLE), a virtual children’s hospital and new teaching and learning methods and practices, utilizing virtual reality and simulation in teaching. 2. To promote the international collaboration of students and lecturers through the VRLE Results The results are preliminary. 1. The VRLE includes educational materials developed to be integrated in the pediatric nursing studies, easy to access and use by the students and teachers with innovative self-pace learning material i.e. videos, 3D-models, interactive tests. Digital patients can react to the provided care with speech, movement, facial expressions and gestures, something that is not possible with the manikins in the Labs. 2. Nursing students and lecturers from different countries can participate in joint remote seminars or workshops in the VRLE, sharing and comparing their country and culture-specific differences and similarities in the nursing practices. VRLE and digital materials enables synchronous and asynchronous learning. Conclusions VRLE is highly conducive to clinical and procedure-focused training by enabling simulation. VR methods offer nursing students the opportunity to practice skills in a safe and risk-free environment in an immersive way. It can be accessed by multiple students, teachers or nurses at the same time from different locations in different countries. Key messages • New innovative VRLE enhances nursing students’ knowledge and skills that are needed when working with sick children and their families. • International collaboration, interaction and mobilization are enabled by using a virtual platform instead of traveling, thus strengthening the co-operation in European higher educational institutes.

Towards equitable reporting of Indigenous status, ethnicity, language and country of birth in Australian paediatric clinical studies: A scoping review.

This scoping review aims to expansively review the reporting of Indigenous status, ethnicity, culture, language and country of birth in Australian paediatric clinical studies. Scoping review of Australian clinical studies, including randomised controlled trials, non-randomised controlled trials, cluster randomised controlled trials and quasi-experimental studies, with paediatric participants (<18 years) or mixed adult and paediatric participants. PubMed, Cumulated Index to Nursing and Allied Health Literature and Embase databases were searched for clinical studies published 1 January 2018 to 28 November 2022. Of the 2717 studies identified in the search, 209 clinical studies were included. Overall, 131 (62.7%) clinical studies captured in this review did not report any of the variables of interest. When reported, terms used by study authors varied extensively and subsequently five study-defined categories emerged 'Indigenous status', 'race', 'race and ethnicity', 'ethnicity', or 'natural skin colour'. 'Indigenous status' was most reported (n = 37, 17.7%), followed by 'ethnicity and/or cultural background' (n = 15, 7.2%), 'race and ethnicity' (n = 4, 1.9%), race (n = 1, 0.5%) and 'natural skin colour' (n = 1, 0.5%). Furthermore, language used at home was reported in 27 studies (12.9%) and country of birth in 23 studies (11.0%). This review demonstrated very low reporting of Indigenous status, ethnicity, culture, language and country of birth in Australian paediatric clinical studies. Poor reporting has raised concerns surrounding generalisability of findings from these trials in addition to equity. The recent international shift encompassing improved clinical trial reporting requirements, for ethnicity and race, require prompt establishment in the Australian clinical trial domain.

Research staff’s experiences of how the COVID-19 pandemic impacted recruitment for a paediatric network study

ABSTRACT Purpose Since the COVID-19 pandemic, a paediatric network study with clinical sites across Canada suffered a reduction in participation. When research studies fail to meet enrolment targets, it can reduce the strength and validity of the results. This study explores research staff’s experiences of how the COVID-19 pandemic impacted recruitment for a paediatric network study. Methods This study was conducted using a qualitative design. Focus group sessions were used to gain the perspective of research staff involved in recruitment and transcripts were analysed using Colaizzi’s seven-step method of data analysis. Results Analysis revealed four major themes: (1) the COVID-19 pandemic had an impact on research activity; (2) families of children with medical complexity perform a risk-benefit assessment when deciding whether to take part in research; (3) a trusting relationship with clinicians is a key factor in research recruitment; and (4) research needs to be flexible in order to adapt to evolving contexts. Conclusion This study identified both COVID-19 and non-COVID-19-related factors that impacted study recruitment for a paediatric network study. Understanding and addressing these challenges will mitigate the negative impacts on health outcomes that can occur when research studies fail to meet enrolment targets.

Vaccination against RSV infections - update and perspective

Almost 60 years after a disastrous clinical vaccine trial in children, which resulted in enhanced disease and even deaths, the world of RSV vaccination is currently undergoing a dramatic positive change and development, closely linked to advances in new vaccine technologies. Three licensed safe and highly efficacious vaccines, Abrysvo, Arexvy and mRESVIA, reduce the incidence of RSV lower respiratory disease by 80% in people older than 60 years of age. Questions regarding long-term protection and effectiveness in specific risk groups with chronic medical conditions remain, and furthermore, innovative and safe concepts to actively vaccinate pregnant women and infants to prevent severe RSV infections - also in these high-risk populations - are eagerly awaited.Passive vaccination with the long-acting monoclonal antibody Nirsevimab for prevention of severe disease in the first RSV season of infants is a major innovation in global health and the importance and benefits of reducing the number of intramuscular injections for high-risk children is immense. In the coming years, results of numerous pediatric candidate RSV vaccine studies are expected, although particular caution seems advisable for historical reasons. In summary, the field of RSV vaccination has been revolutionized in the last 2 years and we will see further significant progress soon.

Reducing research participant disparities through use of a co-developed video intervention: A cohort nested pre-post study

Background Evidence on effective approaches for the recruitment of diverse populations remains limited. This study aimed to assess whether a co-developed study information video would improve participant ethnic diversity within a paediatric study cohort. Methods Pre-post study nested within the Uveitis in childhood prospective national inception cohort study, UNICORNS. Intervention of interest: study information and recruitment video with format and content co-developed with a patient expert group. Outcome of interest: the proportion of families from ethnic minority groups, and of families with primary caregivers without a higher educational degree who were recruited to the study, and the proportion of families who engaged in study activities (returned patient reported outcome metrics following recruitment) pre and post intervention implementation. Results During the study period, 191 children were recruited to UNICORNS (110 pre-intervention era, 81 post-intervention era). A larger proportion of families recruited in the post-intervention era were of an ethnic minority background (36% versus 20%, Fisher’s exact test p=0.02). A larger proportion of families returned self-descriptions of their sociodemographic background (100% versus 87%, Fisher’s exact test p=0.0003) and a larger proportion returned study specific patient reported outcome measures (70% versus 55%, Fisher’s exact test p=0.003). Conclusion Co-created digital research information resources can be an effective tool for ensuring better representation in research studies and may also support participant engagement overall. Our reported methods and findings should support other researchers in developing patient-centred and patient co-developed strategies and tools for the purposes of equitable access to medical research.

Low prevalence of spin in conclusions of interventional pediatric orthopedic studies

Interpretation bias as a factor of research reporting quality has not been thoroughly investigated in the conclusions of pediatric orthopedic publications. Our objective was to investigate the prevalence, subtypes, and severity of research reporting bias or spin of the conclusions in full-texts and abstracts of published studies investigating the effects of treatment/intervention in the pediatric orthopedic literature. We systematically searched ten high-ranking orthopedic journals on MEDLINE/PubMed. Inclusion criteria were pediatric orthopedic studies investigating the effects of treatment/intervention. We used descriptive statistics to report the prevalence, subtype, and severity of reporting bias in the studies’ conclusions according to validated classification criteria. We checked the results to ensure that data were neither misreported nor misinterpreted/extrapolated in the conclusions of the full-texts and their abstracts. Out of 93 included studies in the final analysis, 17 (18%) had at least one count of bias. Nine (10%) studies had bias in both full-text and the corresponding abstract conclusions. In four (4%) studies, bias was restricted to conclusions of abstracts only, and in four (4%) studies was restricted to a bias criterion assigned to the classification criteria of conclusions of full-text only and not the abstract. We analyzed 2511 spin/bias items across 93 studies and reported 30 (1%) counts of bias in conclusions of full-text and/or abstracts. The intervention was surgical in (71%) of studies. Interventional pediatric orthopedic studies published in high-ranking journals showed a low prevalence of reporting bias, namely, misleading reporting, misinterpretation, and inadequate extrapolation of conclusions. A comparative analysis with lower-ranking journals as a control group may reveal if our favorable results are an attribute of journal rank/quality. In general, editorial policies should emphasize skilled interpretation and extrapolation of research results.