Prolonged remission after cyclophosphamide or tacrolimus treatment in childhood nephrotic syndrome: a cohort study.

Background: Steroid-sparing immunosuppression is used in 50% of children with nephrotic syndrome, to prevent relapses and steroid-related toxicity. However, rates and predictors of prolonged remission after cyclophosphamide and tacrolimus are uncertain.

Methods: Retrospective analysis of children (1-18 years) enrolled in a longitudinal cohort.

Targeted Child Mental Health Prevention and Parenting Support Within a Canadian Context: A Randomized Controlled Trial Evaluating the U.S.-Developed Family Check-Up®.

Canada lacks an approach to early childhood mental health prevention aimed at decreasing barriers to care among highest-needs families. In this Canadian randomized controlled trial, we aimed to evaluate whether participation in the Family Check-Up® (FCU®) would be associated with lower severity of child behavior problems (primary outcome) and caregiver psychological distress and daily parenting stress (secondary outcomes). Eligible caregivers of children aged 2-4 years with (i) high severity of behavior problems and/or (ii) above-average severity plus ≥ 1 family psychosocial risk factor were recruited from early education, community, and clinical settings in Hamilton, Ontario.

Critical care EEG monitoring in children with abusive head trauma: A retrospective study of seizure burden and predictors of neurological outcomes.

Objectives: Abusive Head Trauma (AHT) remains an important cause of acute seizures, morbidity, and mortality in children. We aimed to assess the clinical and electrographic seizure burden in children with AHT and to explore predictors of morbidity and mortality.

Methods: We conducted a retrospective chart review of all children admitted with AHT who underwent continuous electroencephalographic monitoring (cEEG) between January 1st, 2015, and April 15th, 2021.

Addressing unmet social needs of children with obesity: a pilot randomized controlled trial.

Background: Childhood obesity is an ongoing public health crisis, and recent clinical practice guidelines identify addressing the role of social inequities in the disparity of health among children with obesity as an area to address. This study aimed to assess the feasibility of a community navigation intervention in a pediatric weight management clinic.

Methods: A single-center pilot randomized controlled trial (RCT) recruiting families from a tertiary hospital pediatric weight management program to evaluate the feasibility of a community navigation intervention.

Early-Onset Trajectories of Emotional Dysregulation in Autistic Children.

Objective: Emotional dysregulation (ED) is a common and debilitating problem for autistic children and their families. However, little is known about early-onset patterns of dysregulation, associated risk factors, and child and family outcomes. This study aimed to characterize trajectories of ED in an inception cohort of autistic preschoolers.

Mean global field power is reduced in infantile epileptic spasms syndrome after response to vigabatrin.

Purpose: Infantile epileptic spasms syndrome (IESS) is associated with abnormal neuronal networks during a critical period of synaptogenesis and brain plasticity. Hypsarrhythmia is a visual EEG biomarker used to diagnose IESS, assess response to treatment, and monitor relapse. Computational EEG biomarkers hold promise in providing unbiased, reliable, and objective criteria for clinical management.

Symptom Screening for Hospitalized Pediatric Patients With Cancer: A Randomized Clinical Trial.

Importance: Pediatric patients with cancer experience severely bothersome symptoms during treatment. It was hypothesized that symptom screening and provision of symptom reports to the health care team would reduce symptom burden in pediatric patients with cancer.

Objective: To determine if daily symptom screening and provision of symptom reports to the health care team was associated with lower total symptom burden as measured by the Symptom Screening in Pediatrics Tool (SSPedi) compared to usual care among pediatric patients with cancer admitted to a hospital or seen in a clinic daily for at least 5 days.

Brief Report: Positive Caregiver Perceptions on Receiving Research Summary Reports in Autism Longitudinal Research.

Purpose: Although participating in research is often presented as something that can 'help others in the future', research participants may also benefit from accessing and understanding their own research data. However, participant attitudes toward receiving individual data via research reports are under researched. This study examined participant perceptions of research reports within the context of the pilot phase of the Pediatric Autism Research Cohort (PARC) Study.

Perceptions of physicians caring for pediatric patients with cancer in Europe: insights into the use of palliative care, its timing, and barriers to early integration.

Background: Integrating pediatric palliative care (PPC) into pediatric oncology standard care is essential. Therefore, it is important to assess physicians' knowledge and perceptions of PPC to optimize its practice.

Objective: To evaluate the knowledge, comfort levels, and perspectives of physicians regarding the timing and perceived barriers to integrating PPC into pediatric cancer care across Europe.

Support Experiences and Wishes of Bereaved Parents After the Loss of Their Child to Cancer.

Introduction: The death of a child has a tremendous impact on parents' lives. The experience of parents who have lost a child to cancer may differ from other bereavement experiences, including other childhood and adulthood causes of death, because of the uncertainty of the prognosis, the aggressive treatment, and the potential for regret about treatment decisions. Bereavement care remains scarce, and effective interventions to meet the diverse needs of parents have not been defined.

Risk-stratification tools for emergency department patients with syncope: A systematic review and meta-analysis of direct evidence for SAEM GRACE.

Objectives: Approximately 10% of patients with syncope have serious or life-threatening causes that may not be apparent during the initial emergency department (ED) assessment. Consequently, researchers have developed clinical decision rules (CDRs) to predict adverse outcomes and risk stratify ED syncope patients. This systematic review and meta-analysis (SRMA) aims to cohere and synthesize the best current evidence regarding the methodological quality and predictive accuracy of CDRs for developing an evidence-based ED syncope management guideline.

The haemophilia joint health score for the assessment of joint health in patients with haemophilia.

Introduction: The haemophilia joint health score (HJHS) is a tool used to assess joint changes in patients with haemophilia. There is lack of consensus on the interpretation of HJHS scores and their clinical relevance.

Aim: To evaluate available literature reporting HJHS changes over time and assess a possible cut-off value for clinically relevant outcomes and the ideal follow-up for a meaningful score change.

Accuracy of point-of-care SARS-CoV-2 detection using buccal swabs in pediatric emergency departments.

To optimize the identification of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)-infected children, specimen collection and testing method are crucial considerations. Ideally, specimen collection is easy and causes minimal discomfort, and the laboratory approach is simple, accurate, and rapid. In this prospective cohort study we evaluated the accuracy of a point-of care nucleic acid device using caregiver/patient self-collected buccal swabs.

Substrate specificity controlled by the exit site of human P4-ATPases, revealed by de novo point mutations in neurological disorders.

The maintenance of lipid asymmetry on the plasma membrane is regulated by flippases, such as ATP8A2, ATP11A, and ATP11C, which translocate phosphatidylserine and phosphatidylethanolamine from the outer leaflet to the inner leaflet. We previously identified a patient-derived point mutation (Q84E) in ATP11A at the phospholipid entry site, which acquired the ability to flip phosphatidylcholine (PtdCho). This mutation led to elevated levels of sphingomyelin (SM) in the outer leaflet of the plasma membrane.

Cost-utility of nelarabine for the first-line treatment of newly diagnosed pediatric T-cell acute lymphoblastic leukemia in Canada.

Background: The Children's Oncology Group (COG)-AALL0434 trial investigated the addition of nelarabine to the augmented Berlin-Frankfurt-Münster (aBFM) protocol in patients (1.0-30.99 years) with newly diagnosed T-cell acute lymphoblastic leukemia (T-ALL).

A validated heart-specific model for splice-disrupting variants in childhood heart disease.

Background: Congenital heart disease (CHD) is the most common congenital anomaly. Almost 90% of isolated cases have an unexplained genetic etiology after clinical testing. Non-canonical splice variants that disrupt mRNA splicing through the loss or creation of exon boundaries are not routinely captured and/or evaluated by standard clinical genetic tests.

"Surgery First" vs. "Endoscopy First" for Pediatric Choledocholithiasis Presenting at the End of the Week - A CARES Working Group Study.

Background: Choledocholithiasis in children is commonly managed with an "endoscopy-first" (EF) strategy (endoscopic retrograde cholangiopancreatography (ERCP) followed by laparoscopic cholecystectomy (LC)). Because ERCP availability is often limited at the end of the week (EoW), we hypothesized that a "surgery-first" (SF) approach (LC with intraoperative cholangiogram (IOC) ± transcystic laparoscopic common bile exploration (LCBDE)) would decrease length of stay (LOS) and time to definitive intervention (TTDI).

Methods: A multicenter, retrospective cohort study was conducted on pediatric patients from 2018 to 2023 with suspected choledocholithiasis.

Effectiveness of surgical interventions for managing obesity in children and adolescents: A systematic review and meta-analysis framed using minimal important difference estimates based on GRADE guidance to inform a clinical practice guideline.

Objective: To summarize the literature on bariatric surgery for managing pediatric obesity, including intervention effects to improve patient-reported outcome measures (PROMs), cardiometabolic risk factors, anthropometry, and assess adverse events (AEs).

Methods: Eligible studies were published between January 2012 and January 2022 and included randomized controlled trials (RCTs) and observational (controlled and uncontrolled) studies before and after surgery with a mean age <18 years old. Outcomes and subgroups were selected a priori by stakeholders; estimates of effect for outcomes were presented relative to minimal important differences (MIDs) and GRADE certainty of evidence.

Diagnosis of TET3-Related Beck-Fahrner Syndrome in an Individual With Chorioretinal and Iris Colobomata Using a DNA Methylation Signature.

Disorders of developmental delay can occur from pathogenic variants in genes responsible for epigenetic regulation. Heterozygous and biallelic pathogenic variants in TET3 have recently been described in TET3-related Beck-Fahrner syndrome (TET3-BEFAHRS), representing an autosomal dominant disorder with variable expressivity. Typical features include intellectual disability and developmental delay.