Validation of an acute kidney injury prediction model as a clinical decision support system.

Background: Acute kidney injury (AKI) is a critical clinical condition that requires immediate intervention. We developed an artificial intelligence (AI) model called PRIME Solution to predict AKI and evaluated its ability to enhance clinicians' predictions.

Methods: The PRIME Solution was developed using convolutional neural networks with residual blocks on 183,221 inpatient admissions from a tertiary hospital (2013-2017) and externally validated with 4,501 admissions at another tertiary hospital (2020-2021).

Dolutegravir metabolism: impact of genetic variations on uridine diphosphate glucuronosyltransferase subfamilies.

1. Dolutegravir (DTG) is a key drug used to treat human immunodeficiency virus type-1 (HIV-1) infections. Adverse events (AEs) of DTG treatment, including headache, anxiety, depression, insomnia, and abnormal dreams, are influenced by sex, body weight, age, and serum bilirubin levels.

Epidemiology of diagnostic errors in pediatric emergency departments using electronic triggers.

Objectives: We applied three electronic triggers to study frequency and contributory factors of missed opportunities for improving diagnosis (MOIDs) in pediatric emergency departments (EDs): return visits within 10 days resulting in admission (Trigger 1), care escalation within 24 h of ED presentation (Trigger 2), and death within 24 h of ED visit (Trigger 3).

Methods: We created an electronic query and reporting template for the triggers and applied them to electronic health record systems of five pediatric EDs for visits from 2019. Clinician reviewers manually screened identified charts and initially categorized them as "unlikely for MOIDs" or "unable to rule out MOIDs" without a detailed chart review.

The Outcome of Ongoing Adult Endocrine Engagement Following Transition From Paediatric Care.

Objective: Transition is important for continuity of care for patients with chronic health conditions. The aim of this service evaluation was to determine the effectiveness of a transition clinic at a tertiary hospital with long-term attendance in the adult endocrine service.

Design: Retrospective case notes review of patients seen by paediatric endocrinology at the Royal Hospital for Children, Glasgow, at the time of transition to adult services, between 2012 and 2022.

Virus-free CRISPR knock-in of a chimeric antigen receptor into KLRC1 generates potent GD2-specific natural killer cells.

Natural killer (NK) cells are an appealing off-the-shelf, allogeneic cellular therapy due to their cytotoxic profile. However, their activity against solid tumors remains suboptimal in part due to the upregulation of NK-inhibitory ligands, such as HLA-E, within the tumor microenvironment. Here, we utilize CRISPR-Cas9 to disrupt the KLRC1 gene (encoding the HLA-E-binding NKG2A receptor) and perform non-viral insertion of a GD2-targeting chimeric antigen receptor (CAR) within NK cells isolated from human peripheral blood.

Choroid plexus-targeted viral gene therapy for alpha-mannosidosis, a prototypical neurometabolic lysosomal storage disease.

The choroid plexuses (CP) are highly vascularized structures that project into the ventricles of the vertebrate brain. The polarized epithelia of the CP produce cerebrospinal fluid by transporting water and ions into the ventricles from the blood and normally secrete a large number of proteins. We assessed the feasibility of selective CP transduction with recombinant adeno-associated virus (rAAV) gene therapy vectors for treatment of lysosomal storage disease (LSD), a broad category of neurometabolic illness associated with significant burdens to affected patients and their families.

First-in-human study on tolerability, pharmacokinetics and pharmacodynamics of single and multiple escalating doses of XKH001, a recombinant humanized monoclonal antibody against IL-25 in healthy Chinese volunteers.

Background: XKH001 is a recombinant humanized IgG1 monoclonal antibody against IL-25 for the treatment of type 2 inflammatory diseases. This study aimed to evaluate the tolerability, pharmacokinetics, and pharmacodynamics of XKH001 in humans for the first time.

Research Design And Methods: This clinical investigation adopted a randomized, double-blind, and placebo-controlled single ascending dose (SAD) and multiple ascending dose (MAD) design.

Food Allergy and Mental Health in Children and Adolescents-The Role of Shared Familial Environment.

Background: Evidence suggests a link between food allergy and poor mental health, however, this may be explained by shared genetic and environmental factors. We aimed to investigate the association between food allergy of different severity and mental health in children, and the role of familial factors.

Methods: This population-based, longitudinal cohort study is based on the Child and Adolescent Twin Study in Sweden with questionnaire data reported by parents and/or children.

The Utility of the Radiocapitellar View in Pediatric Medial Epicondyle Fracture Evaluation.

Background: Anteroposterior (AP) and lateral radiographs often underestimate displacement in medial epicondyle fractures, further complicating the already debated operative indications. The radiocapitellar (RC) view is an alternative radiograph that allows for the separation of the humeroradial and humeroulnar joints. This study investigates the utility of the RC view in measuring displacement and identifying the need for operative intervention in pediatric medial epicondyle fractures.

Feasibility of home-based urine collection in children under 5 years in the ORIGINS birth cohort study: mixed method protocol and sample completion results.

Background: Urine is an attractive biospecimen for nutritional status and population health surveys. It is an excellent non-invasive alternative to blood for appropriate biomarkers in young children and is suitable for home-based collection, enabling representative collections across a population. However, the bulk of literature in this population is restricted to collection in primary care settings.

An exceptionally rare case of a diffuse midline glioma with concomitant H3.1 K27M and G34R mutations in the HIST1H3C (H3C3) gene.

Histone mutations (H3 K27M, H3 G34R/V) are molecular features defining subtypes of paediatric-type diffuse high-grade gliomas (HGG) (diffuse midline glioma (DMG), H3 K27-altered, diffuse hemispheric glioma (DHG), H3 G34-mutant). The WHO classification recognises in exceptional cases, these mutations co-occur. We report one such case of a 2-year-old female presenting with neurological symptoms; MRI imaging identified a brainstem lesion which was biopsied.

SNORA37/CMTR1/ELAVL1 feedback loop drives gastric cancer progression via facilitating CD44 alternative splicing.

Background: Emerging evidence shows that small nucleolar RNA (snoRNA), a type of highly conserved non-coding RNA, is involved in tumorigenesis and aggressiveness. However, the roles of snoRNAs in regulating alternative splicing crucial for cancer progression remain elusive.

Methods: High-throughput RNA sequencing and comprehensive analysis were performed to identify crucial snoRNAs and downstream alternative splicing events.

Liver transplantation for homozygous familial hypercholesterolemia: a retrospective analysis from Chinese experience.

Background: Homozygous familial hypercholesterolaemia (HoFH) increases risk of premature cardiovascular events and cardiac death. In severe cases of HoFH, clinical signs and symptoms cannot be controlled well by non-surgical treatments, liver transplantation (LT) currently represents the viable option.

Method: To assess the clinical efficacy, prognosis, and optimal timing of LT for HoFH, a retrospective analysis was conducted on the preoperative, surgical conditions, and postoperative follow-up of children who received an LT for HoFH at the Beijing Friendship Hospital over the period from December 2014 to August 2022.

Financial crisis and its association with parental stress and children's mental health in Lebanon.

Background: Lebanon has experienced a series of devastating crises that continue to have significant adverse effects on the mental health of parents and their children, especially those who are unemployed, burdened with debt or financial difficulties, and have pre-existing mental health conditions. Accordingly, this study aimed to assess the effect of financial insecurities on parents in Lebanon amid the multiple crises, and the impact of parents' mental health on their children's emotional and behavioral wellbeing.

Methods: A cross-sectional study including 589 parents in Lebanon was performed using convenience sampling of parents of any gender with children aged 4 to 18 from the five Lebanese governorates.

Congenital muscular dystrophies and myopathies: the leading cause of genetic muscular disorders in eleven Chinese families.

Background: Congenital muscular dystrophies (CMDs) and myopathies (CMYOs) are a clinically and genetically heterogeneous group of neuromuscular disorders that share common features, such as muscle weakness, hypotonia, characteristic changes on muscle biopsy and motor retardation. In this study, we recruited eleven families with early-onset neuromuscular disorders in China, aimed to clarify the underlying genetic etiology.

Methods: Essential clinical tests, such as biomedical examination, electromyography and muscle biopsy, were applied to evaluate patient phenotypes.

Diagnostic accuracy of artificial intelligence algorithms to predict remove all macroscopic disease and survival rate after complete surgical cytoreduction in patients with ovarian cancer: a systematic review and meta-analysis.

Background: Complete Cytoreduction (CC) in ovarian cancer (OC) has been associated with better outcomes. Outcomes after CC have a multifactorial and interrelated cause that may not be predictable by conventional statistical methods. Artificial intelligence (AI) may be more accurate in predicting outcomes.

Combined detection of monocyte distribution width and procalcitonin for diagnosing and prognosing neonatal sepsis.

Background: To assess the value of combined Monocyte Distribution Width (MDW) and Procalcitonin (PCT) detection in diagnosing and predicting neonatal sepsis outcomes.

Methods: This retrospective study, conducted from January 2022 to December 2023.A retrospective analysis of 39 neonatal sepsis and 30 non-infectious systemic inflammatory response syndrome (SIRS) cases was conducted.

Development of a disease diagnostic model to predict the occurrence of central precocious puberty of female.

Objectives: To develop a clinical model for predicting the occurrence of Central Precocious Puberty based on the breast development outcomes in chinese girls.

Methods: This is a retrospective study, which included a total of 1,001 girls aged 6-9 years old who visited the outpatient clinic of Beijing Children's Hospital from January 2017 to October 2022 for "breast development". Participants were categorized into pubertal development (PD) cohort and simple premature breast development (PT) according to the criteria, and information was collected and tested for relevant indicators.

Interpreting positive celiac serology in children with new-onset type 1 diabetes.

Objectives: The association of celiac disease (CD) in type 1 diabetes mellitus (T1DM) is well-established, yet variation exists in screening practices. This study measures the accuracy of early screening with tissue transglutaminase Immunoglobulin A (TTG-IgA) and endomysial antibody (EMA) in newly diagnosed T1DM.

Methods: This is a retrospective study of children with T1DM between 2013 and 2019 with early CD screening and follow-up.

Super-photostable organic dye for long-term live-cell single-protein imaging.

Organic dyes play a crucial role in live-cell imaging because of their advantageous properties, such as photostability and high brightness. Here we introduce a super-photostable and bright organic dye, Phoenix Fluor 555 (PF555), which exhibits an order-of-magnitude longer photobleaching lifetime than conventional organic dyes without the requirement of any anti-photobleaching additives. PF555 is an asymmetric cyanine structure in which, on one side, the indole in the conventional Cyanine-3 is substituted with 3-oxo-quinoline.

Resolving tissue complexity by multimodal spatial omics modeling with MISO.

Spatial molecular profiling has provided biomedical researchers valuable opportunities to better understand the relationship between cellular localization and tissue function. Effectively modeling multimodal spatial omics data is crucial for understanding tissue complexity and underlying biology. Furthermore, improvements in spatial resolution have led to the advent of technologies that can generate spatial molecular data with subcellular resolution, requiring the development of computationally efficient methods that can handle the resulting large-scale datasets.

Updates on neonatal cell and novel therapeutics: Proceedings of the Second Neonatal Cell Therapies Symposium (2024).

Cell therapies as treatments for neonatal conditions have attracted significant research and parent interest over the last two decades. Mesenchymal stromal cells, umbilical cord blood cells and neural stem cells translate from lab, to preclinical and into clinical trials, with contributions being made from all over the world. Effective and timely translation involves frequent reflection and consultation from research-adjacent fields (i.