Multicellular tumor-stromal interactions recapitulate aspects of therapeutic response and human oncogenic signaling in a 3D disease model for H3K27M-altered DIPG.

It has become evident from decades of clinical trials that multimodal therapeutic approaches with focus on cell intrinsic and microenvironmental cues are needed to improve understanding and treat the rare, inoperable, and ultimately fatal diffuse intrinsic pontine glioma (DIPG), now categorized as a diffuse midline glioma. In this study we report the development and characterization of an in vitro system utilizing 3D Tumor Tissue Analogs (TTA), designed to replicate the intricate DIPG microenvironment. The innate ability of fluorescently labeled human brain endothelial cells, microglia, and patient-derived DIPG cell lines to self-assemble has been exploited to generate multicellular 3D TTAs that mimic tissue-like microstructures, enabling an in- depth exploration of the spatio-temporal dynamics between neoplastic and stromal cells.

Xanthogranulomatous Epithelial Tumor in a Pediatric Patient. A Potential Diagnostic Pitfall With Non-Langerhans Cell Histiocytic Neoplasms, Particularly Juvenile Xanthogranuloma.

Xanthogranulomatous epithelial tumor is a recently described neoplasm harboring recurrent HMGA2::NCOR2 fusions, which, owing to its extensive xanthogranulomatous appearance with abundant foamy histiocytes and frequent Touton-type giant cells, may be confused with other inflammatory and neoplastic entities. We present a case arising in the right arm subcutaneous tissues of a 10-year-old boy, which was initially interpreted as a non-Langerhans cell histiocytic neoplasm compatible with Juvenile xanthogranuloma.

2D Speckle Tracking Strain Echocardiography in Multisystem Inflammatory Syndrome in Children: A Multicenter Analysis From the MUSIC Study.

Background: 2D-speckle tracking echocardiography may help detect subclinical ventricular dysfunction, but data in multisystem inflammatory syndrome in children (MIS-C) are scarce. We investigated left ventricular (LV) strain parameters in MIS-C and their association with outcomes.

Methods: We performed an ambi-directional, 32-center cohort study on hospitalized MIS-C patients (March 2020-November 2021) with at least 1 echocardiogram read by the Core Lab.

Integration of Genome and Epigenetic Testing in the Diagnostic Evaluation of Developmental Delay: Differentiating Börjeson-Forssman-Lehmann (BFLS) and White-Kernohan (WHIKERS) Syndromes.

: More than 1500 genes are associated with developmental delay and intellectual disability, with variants in many of these genes contributing to a shared phenotype. The discovery of variants of uncertain significance (VUS) found in these genes during genetic testing can lead to ambiguity and further delay in diagnosis and medical management. Phenotyping, additional genetic testing, and functional studies can all add valuable information to help reclassify these variants.

A 10-Year Review of the Efficacy of Cranial Remolding Orthosis Treatment and Factors That Influence Outcomes for Infants with Isolated Deformational Plagiocephaly.

Background/objectives: The purpose of this study was to examine the overall efficacy and treatment outcomes of CROs in the treatment of isolated deformational plagiocephaly and investigate the variables that influence treatment efficacy.

Methods: This was a 10-year retrospective review of N = 27, 990 infants with Isolated Deformational Plagiocephaly (IDP) who completed Cranial Remolding Orthosis (CRO) treatment between 3 and 18 months of age.

Results: There was a significant overall mean change in CVAI(S) of -3.

Clinical characteristics and treatment outcomes in patients with Niemann-Pick disease type C (NP-C): a cross-sectional study.

Background: Niemann-Pick disease type C (NP-C) is a rare autosomal recessive lysosomal storage disorder characterized by progressive neurodegeneration. This study aimed to characterize the clinical features and treatment outcomes of NP-C in Iranian patients.

Methods: We conducted a cross-sectional study of 58 patients with NP-C diagnosed between March 2013 and March 2024 at Mofid Children's Hospital, Tehran.

Opportunities and challenges surrounding the use of implantable haemodynamic monitor in paediatric Fontan patients living in high-altitude, rural settings.

Background: Implantable haemodynamic monitors allow remote monitoring of Fontan circulation. We report unique opportunities and challenges related to device use in rural, high-altitude regions.

Objectives: Assess the performance of implantable haemodynamic monitor in Fontan circulation and identify potential sources of measurement discrepancy defined as non-physiological, negative, or significantly lower reading than baseline.

A Phase 4, Open-Label, Non-Randomized, Multicenter Study to Evaluate Efficacy (and Safety) of Intravenous Administration of Optison for Contrast-Enhanced Echocardiography in Pediatric Patients.

Contrast-enhanced echocardiography (CE-echo) is commonly used in adult patients to optimize endocardial border delineation (EBD) but there is limited experience in children. A prospective, open-label, non-randomized, multicenter study was performed to evaluate the efficacy of intravenous Optison for CE-echo in pediatric patients and determine the optimal dose of Optison. Subjects ≥ 9 and < 18 years with suboptimal echocardiograms (defined as ≥ 2 contiguous segments that could not be adequately visualized) were enrolled.

Favorable clinical outcomes and complications of endoscopic third ventriculostomy in Chiari Malformation Type I: A systematic review and meta-analysis.

Introduction: Chiari Malformation Type I (CM-I) is characterized by cerebellar tonsil herniation, often requiring posterior fossa decompression. However, when cerebrospinal fluid flow remains obstructed, additional interventions such as endoscopic third ventriculostomy (ETV) or ventriculoperitoneal shunting (VPS) may be necessary. Despite the frequent use of ETV in CM-I cases, evidence supporting its efficacy remains limited.

The growth modulating effects of tether tension on vertebral growth are biphasic: a study of posterior vertebral body tethering (pVBT) in a novel kyphotic porcine model.

Purpose: To measure the effects of posterior vertebral tethering (pVBT) on disc pressure and the effect of tether tension on growth modulation in the hyperkyphotic swine model, and to use computational modeling to predict growth modulation in scenarios unable to be tested in the animal model.

Methods: Swine were divided into non-operative control, single-level apical pVBT, or multi-level posterior pVBT groups. Pulsed fluorochrome labeling was used to measure regional vertebral growth rates, digital radiographs to assess changes in vertebral alignment, and pressure transducers to measure intervertebral disc pressures.

Is There a Role for ERCP in the Management of Gallstone Pancreatitis in Children? A Western Pediatric Surgery Research Consortium Study.

Background: Guidelines for adult gallstone pancreatitis (GP) in adults recommend endoscopic retrograde cholangiopancreatography (ERCP) for ongoing biliary obstruction. Studies in children are limited by small sample sizes. We sought to explore whether factors predictive of choledocholithiasis (CDL) are correlated with ERCP findings of stones in pediatric GP.

Functional Data Analysis of the Power-Duration Relationship in Cyclists.

Purpose: To extract, prioritize, and model the highly conserved variations in mean-maximal power (MMP) data in cyclists utilizing functional principal component (FPC) analysis.

Methods: A 3-parameter model (F3 model) was derived from the first 3 functions identified by FPC analysis of a large MMP data set. The F3 model was assessed for goodness of fit to a reserved out-of-sample partition of the MMP data set.

Type IV Hereditary Sensory and Autonomic Neuropathy: Challenges and Functional Outcomes.

Background: Type IV Hereditary Sensory and Autonomic Neuropathy (HSAN IV) is an exceedingly rare autosomal recessive disease classically characterized by generalized loss of temperature/pain sensation, intellectual disability, and anhidrosis. HSAN IV patients are subject to repeated orthopaedic injuries and complications. There is some data on the clinical presentation, but limited data on the diagnostic and treatment challenges, and functional outcomes with HSAN IV.

Pediatric Pharmacy Association 2025 KIDs List of Key Potentially Inappropriate Drugs in Pediatrics.

Objective: The objective was to update the KIDs List, a list of drugs and excipients that are potentially inappropriate for use in pediatric patients, accounting for emerging pharmacologic agents and published evidence.

Methods: A panel of 12 pediatric pharmacists from the Pediatric Pharmacy Association (PPA) evaluated primary, secondary, and tertiary literature; FDA Pediatric Safety Communications; the UpToDate Lexidrug database; and product information for drugs that may be considered potentially inappropriate for use in pediatric patients. A PubMed search identified new publications from October 1, 2017, to November 1, 2023.

Analgesia, Sedation, and Neuromuscular Blockade in Pediatric Severe Traumatic Brain Injury: Secondary Analysis of the "Approaches and Decisions in Acute Pediatric TBI Trial" (ADAPT).

Background: Sedative, analgesia, and neuromuscular blocking (NMB) medications may be necessary in the acute management of pediatric severe traumatic brain injury (sTBI), yet limited data exist to guide their use. We aimed to describe the use of continuous infusions of these medications in children with sTBI, to determine temporal trends during the first week of management, and to evaluate associations with in-hospital mortality.

Methods: We conducted a secondary analysis of the Approaches and Decisions in Acute Pediatric Traumatic Brain Injury Trial (NCT04077411, 2014-2017), a prospective multicenter observational study of patients < 18 years old with sTBI (Glasgow Coma Scale ≤ 8) who underwent intracranial pressure monitoring.

Colorectal cancer in a 13-year-old with constitutional mismatch repair deficiency and heterozygosity.

Constitutional mismatch repair deficiency (CMMRD) is a rare hereditary cancer syndrome resulting from biallelic mutations in DNA mismatch repair (MMR) genes that lead to early-onset cancers in children, including lymphoma and colorectal cancer (CRC). This case report presents a 13-year-old boy diagnosed with CMMRD due to a homozygous mutation and a heterozygous mutation. The patient's initial misdiagnosis as neurofibromatosis type 1 (NF1) highlights the overlap between CMMRD and NF1, as their overlapping genetic pathologies can yield similar clinical manifestations.

Have we hit the JAK-pot? Success of selective JAK 1 inhibitor following failure of pan-JAK inhibitor in refractory pediatric ulcerative colitis.

The treatment options available for pediatric ulcerative colitis (UC) are challenging due to few with Federal Drug Administration approval. Newer medications approved for adults include additional biologics with differing mechanisms of action and small molecule drugs, such as Janus kinase (JAK) inhibitors. Our case outlines a patient with refractory UC who failed mesalamine, adalimumab, tofacitinib, and vedolizumab.

Comparison of secondary surgery before and after centralisation of cleft services in the UK: a whole-island cross-sectional analysis.

Objective: Cleft lip and palate significantly impact a child's speech and facial appearance. Children undergo cleft repairs in infancy, but poor results from these initial repairs often lead to secondary surgery. In the late 1990s, cleft care provision in the UK was centralised to approximately 11 managed clinical networks or centres.

Fetal body composition and organ growth in pregnancies complicated by first trimester vaginal bleeding status: NICHD Fetal 3D Study.

Objective: To explore associations between self-reported first trimester vaginal bleeding status and fetal body composition and organ volumes measured by 3-Dimensional (3D) ultrasound across pregnancy.

Study Design: The NICHD Fetal 3D Study (2015-2019) included individuals with singleton pregnancies at low risk for fetal growth abnormalities (n=2,634). Fetal body composition measures of arm, abdomen, and thigh, and organ volumes including cerebellum, lung, kidney, and liver, were measured up to five times between 15-40 weeks using 3D ultrasound.

Factors Associated With Symptom Burden Among Pediatric Patients With Cancer.

Purpose: The objective was to identify factors associated with self-reported symptom burden measured using Symptom Screening in Pediatrics Tool (SSPedi) in pediatric patients with cancer.

Methods: This was a secondary analysis of a cluster randomized trial enrolling pediatric patients newly diagnosed with cancer. Twenty sites were randomized to routine symptom screening versus usual care.

Molecular and clinical spectrum of epilepsy-dyskinesia syndromes: a cross-sectional study of 609 patients.

Epilepsy-dyskinesia syndromes (EDS) are a complex group of neurogenetic disorders characterized by the co-occurrence of epilepsy and movement disorders. Despite their increasing clinical recognition, the molecular and clinical spectrum of EDS remain poorly understood. While numerous genetic etiologies have been implicated, systematic characterization across diverse populations is lacking.

Diagnostic, therapeutic, and surgical repair trends in slipping rib syndrome: a survey of surgeons in the United States.

Background: Diagnostic, therapeutic and surgical techniques for slipping rib syndrome (SRS) are evolving to improve clinical outcomes and recurrence. Our objective was to evaluate current practice patterns in SRS diagnosis and treatment among surgeons nationally.

Methods: An electronic survey was nationally distributed to surgeons who were identified as slipped rib repair providers to assess trends in diagnosis and treatment.