Large-scale evaluation of outcomes after a genetic diagnosis in children with severe developmental disorders.

Purpose: We sought to evaluate outcomes for clinical management after a genetic diagnosis from the Deciphering Developmental Disorders study.

Methods: Individuals in the Deciphering Developmental Disorders study who had a pathogenic/likely pathogenic genotype in the DECIPHER database were selected for inclusion ( = 5010). Clinical notes from regional clinical genetics services notes were reviewed to assess predefined clinical outcomes relating to interventions, prenatal choices, and information provision.

Use of virtual reality to remotely train healthcare professionals in paediatric emergency tracheostomy skills: protocol for a multi-centre, non-inferiority educational interventional study with historical controls.

Background: The insertion of a tracheostomy is an established technique used to wean patients off ventilatory support, manage secretions in complex conditions, and as a potentially life-saving procedure to bypass upper airway obstruction. Life-threatening complications during aftercare are not uncommon and may be influenced by a lack of education of carers or healthcare providers of children and young people living with a tracheostomy. Education programmes designed and supported by the National Tracheostomy Safety Project are effective, but resources are not available to educate the workforce at scale.

Procalcitonin-guided duration of antibiotic treatment in children hospitalised with confirmed or suspected bacterial infection in the UK (BATCH): a pragmatic, multicentre, open-label, two-arm, individually randomised, controlled trial.

Background: Procalcitonin is a rapid response biomarker specific for bacterial infection, which is not routinely used in the UK National Health Service. We aimed to assess whether using a procalcitonin-guided algorithm would safely reduce the duration of antibiotic therapy compared with usual care, in which C-reactive protein is the commonly used biomarker.

Methods: The BATCH trial was a pragmatic, multicentre, open-label, parallel, two-arm, individually randomised, controlled trial conducted in 15 hospitals in England and Wales.

Symptomatic glucocorticoid-induced adrenal suppression in the United Kingdom and Ireland: a BPSU study.

Objective: To determine the incidence and describe the presentation and management of unexpected symptomatic glucocorticoid-induced adrenal suppression (AS) in children and young people aged 0-15 years.

Setting And Design: Surveillance study of symptomatic glucocorticoid (GC)-induced AS with supportive biochemical evidence or presenting as an adrenal crisis, reported via the British Paediatric Surveillance Unit (BPSU) from September 2020 to September 2022.

Results: Over a 25-month period, 190 reports of symptomatic GC-induced AS/adrenal crisis were made, of which 22 were confirmed cases: 18 AS and 4 adrenal crises.

Guardian Knowledge in Primary Congenital Glaucoma: A Cross-Sectional Study.

Prcis: Guardian education level and frequency of surgical interventions are key determinants of knowledge in primary congenital glaucoma, highlighting the need for targeted educational strategies.

Background: Management of congenital glaucoma poses unique challenges, particularly concerning the patient guardians' understanding of the condition, which is crucial for treatment adherence and follow-up compliance. This study aimed to assess guardians' knowledge levels and identify the influencing factors.

Variations and Opportunities in Postnatal Management of Hemolytic Disease of the Fetus and Newborn.

Importance: Preventive efforts in pregnancy-related alloimmunization have considerably decreased the prevalence of hemolytic disease of the fetus and newborn (HDFN). International studies are therefore essential to obtain a deeper understanding of the postnatal management and outcomes of HDFN. Taken together with numerous treatment options, large practice variations among centers may exist.

Developing an adaptive paediatric intensive care unit platform trial with key stakeholders: a qualitative study.

Objectives: Platform trials were used successfully in adult populations during the COVID-19 pandemic. By testing multiple treatments within a single trial, platform trials can help identify the most effective treatments (and any interactions between treatments) for patients more quickly and with less burden for patients and their families. The aim of this qualitative research was to inform the design of the first adaptive platform trial for paediatric intensive care in the UK with young people, parents/carers and paediatric intensive care unit (PICU) staff.

Impact of surrogates for insulin resistance on mortality and life expectancy in primary care: a nationwide cross-sectional study with registry linkage (LIPIDOGRAM2015).

Background: Insulin resistance (IR) is an important risk factor for multiple chronic diseases, increasing mortality and reducing life expectancy. The associations between emerging surrogates for IR, triglyceride-glucose index (TyG) and TyG-related indicators, with all-cause mortality and life expectancy in middle-aged and older patients in primary care are unclear.

Methods: This study originated from the Polish primary care cohort LIPIDOGRAM2015, including patients aged ≥45 years.

Automating radiological measurements of the hip in children with cerebral palsy.

Aims: The aims of this study were to develop an automatic system capable of calculating four radiological measurements used in the diagnosis and monitoring of cerebral palsy (CP)-related hip disease, and to demonstrate that these measurements are sufficiently accurate to be used in clinical practice.

Methods: We developed a machine-learning system to automatically measure Reimer's migration percentage (RMP), acetabular index (ACI), head shaft angle (HSA), and neck shaft angle (NSA). The system automatically locates points around the femoral head and acetabulum on pelvic radiographs, and uses these to calculate measurements.

Building global collaborative research networks in paediatric critical care: a roadmap.

Paediatric critical care units are designed for children at a vulnerable stage of development, yet the evidence base for practice and policy in paediatric critical care remains scarce. In this Health Policy, we present a roadmap providing strategic guidance for international paediatric critical care trials. We convened a multidisciplinary group of 32 paediatric critical care experts from six continents representing paediatric critical care research networks and groups.

Longer-term survival, quality of life, and cost-effectiveness of conservative versus liberal oxygenation targets in critically ill children: a pre-specified analysis from Oxy-PICU, a multicentre, open, parallel-group, randomised controlled trial.

Background: Peripheral oxygen saturation (SpO) above 94% is typical in children in paediatric intensive critical care units (PICUs) who are receiving invasive ventilation and supplemental oxygen. In a previous report from the Oxy-PICU trial, we showed that lower (conservative) oxygenation targets (SpO 88-92%) are beneficial, showing small but statistically significant differences in duration of organ support and large but non-significant cost reductions at 30 days. In this pre-specified analysis of the Oxy-PICU trial, we compare longer-term outcomes and cost-effectiveness of conservative versus liberal (SpO >94%) oxygenation targets in children with emergency PICU admission.

Accuracy of frameless robot-assisted stereoelectroencephalography depth electrode implantation using the neurolocate registration system in paediatric patients.

Background: We assessed the accuracy and performed a directional analysis of robot-assisted implantation of stereoelectroencephalography (SEEG) depth electrodes in children using the frameless neurolocate 3D registration module.

Methods: Thirteen children with epilepsy undergoing stereotactic robot-assisted insertion of SEEG electrodes were included. Six children were operated on with standard frame-based registration while 7 with the use of the frameless neurolocate registration module.

Status of surface electromyography assessment as part of clinical gait analysis in the management of patients with cerebral palsy - Outcomes of a Delphi process.

Background: The assessment of gait disorders in patients with neuromotor conditions, such as cerebral palsy (CP), has been a focus of clinical and research attention, with electromyography (EMG) offering a nuanced understanding of neurological and neuromuscular disorders. However, the interpretation of EMG data in the context of gait analysis remains challenging due to the complexity of neuromotor dynamics and variability in assessment methodologies.

Research Question: To which consensus can we get in a group of experts in the fields of neurological and neuromuscular disorders, biomechanics, and clinical gait analysis to establish standardized protocols and a common language for the measurement and analysis of EMG data in gait disorders, particularly in people living with CP?

Methods: A three-round Delphi process was conducted from February to September 2023 to gather opinions of 53 experts on the use of surface EMG data during gait in the context of CP.

Machine Learning-based Prognostic Subgrouping of Glioblastoma: A Multi-center Study.

Background: Glioblastoma is the most aggressive adult primary brain cancer, characterized by significant heterogeneity, posing challenges for patient management, treatment planning, and clinical trial stratification.

Methods: We developed a highly reproducible, personalized prognostication and clinical subgrouping system using machine learning (ML) on routine clinical data, MRI, and molecular measures from 2,838 demographically diverse patients across 22 institutions and 3 continents. Patients were stratified into favorable, intermediate, and poor prognostic subgroups (I, II, III) using Kaplan-Meier analysis (Cox proportional model and hazard ratios [HR]).

The effects of whey protein supplementation on indices of cardiometabolic health: A systematic review and meta-analysis of randomized controlled trials.

Introduction: The increasing prevalence of cardiometabolic diseases highlights the urgent need for practical interventions to mitigate their associated public health burden. Whey protein supplementation has emerged as a potential intervention for improving markers of cardiometabolic health. The aim of this systematic review and meta-analysis was to examine the effect of whey protein ingestion on cardiometabolic profile in adults.

Opportunities for nurses aspiring to or undertaking clinical academic development globally: protocol for a scoping review.

Introduction: Nurse researchers often lack awareness of how to start a clinical academic research career and often lack clear entry routes. This scoping review aims to identify the range and nature of clinical academic opportunities available for nurses. This will also identify the knowledge gaps and provide the basis for future research.

Paediatric meningiomas: a multi-centre case series of 27 patients.

Purpose: This study presents a series of paediatric meningiomas and discusses aetiology, risk factors and outcomes with comparison to current literature.

Methods: This is a retrospective review of surgically treated paediatric meningiomas from three UK centres: the University Hospital of Wales, Alder Hey Children's Hospital and Royal Manchester Children's Hospital. Twenty-seven patients aged 16 and under at the time of their first procedure were identified over a 15-year period (1 January 2007 and 1 March 2023).

Expert consensus recommendations for the diagnosis and treatment of chronic non-bacterial osteitis (CNO) in adults.

Background: There is considerable practice variation in labelling, diagnosis and treatment of adults with sterile bone inflammation. We developed a expert consensus recommendations on the disease definition, diagnosis and treatment of this rare condition.

Methods: Systematic literature review and Grading of Recommendations, Assessment, Development and Evaluations-based appraisal of evidence, two Delphi surveys and three digital and in-person consensus meetings with a multidisciplinary expert panel and patient representatives.