Endomyocardial biopsy: safety and prognostic utility in paediatric and adult myocarditis in the European Society of Cardiology EURObservational Research Programme Cardiomyopathy and Myocarditis Long-Term Registry.

Background And Aims: Contemporary multicentre data on clinical and diagnostic spectrum and outcome in myocarditis are limited. Study aims were to describe baseline features, 1-year follow-up, and baseline predictors of outcome in clinically suspected or biopsy-proven myocarditis (2013 European Society of Cardiology criteria) in adult and paediatric patients from the EURObservational Research Programme Cardiomyopathy and Myocarditis Long-Term Registry.

Methods: Five hundred eighty-one (68.

A Population-Based Cohort Study on Diagnosis and Early Management of Anorectal Malformation in the UK and Ireland.

Background: This study describes the presentation and initial management of anorectal malformation (ARM); evaluating the frequency, causes and consequences of late diagnosis.

Methods: A prospective, population cohort study was undertaken for newly diagnosed ARMs in the UK and Ireland from 01/10/2015 and 30/09/2016. Follow-up was completed at one year.

Understanding the influence of surgical parameters on craniofacial surgery outcomes: a computational study.

Sagittal craniosynostosis (SC) is a congenital condition whereby the newborn skull develops abnormally owing to the premature ossification of the sagittal suture. Spring-assisted cranioplasty (SAC) is a minimally invasive surgical technique to treat SC, where metallic distractors are used to reshape the newborn's head. Although safe and effective, SAC outcomes remain uncertain owing to the limited understanding of skull-distractor interaction and the limited information provided by the analysis of single surgical cases.

Management of seizures in patients with primary mitochondrial diseases: consensus statement from the InterERNs Mitochondrial Working Group.

Background And Purpose: Primary mitochondrial diseases (PMDs) are common inborn errors of energy metabolism, with an estimated prevalence of one in 4300. These disorders typically affect tissues with high energy requirements, including heart, muscle and brain. Epilepsy may be the presenting feature of PMD, can be difficult to treat and often represents a poor prognostic feature.

Socioeconomic and ethnic disparities associated with access to cochlear implantation for severe-to-profound hearing loss: A multicentre observational study of UK adults.

Background: Patients with severe-to-profound hearing loss may benefit from management with cochlear implants. These patients need a referral to a cochlear implant team for further assessment and possible surgery. The referral pathway may result in varied access to hearing healthcare.

The Virtual Child.

We are building the world's first Virtual Child-a computer model of normal and cancerous human development at the level of each individual cell. The Virtual Child will "develop cancer" that we will subject to unlimited virtual clinical trials that pinpoint, predict, and prioritize potential new treatments, bringing forward the day when no child dies of cancer, giving each one the opportunity to lead a full and healthy life.

Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia.

Propionic acidaemia is a rare disorder caused by defects in the propionyl-coenzyme A carboxylase α or β (PCCA or PCCB) subunits that leads to an accumulation of toxic metabolites and to recurrent, life-threatening metabolic decompensation events. Here we report interim analyses of a first-in-human, phase 1/2, open-label, dose-optimization study and an extension study evaluating the safety and efficacy of mRNA-3927, a dual mRNA therapy encoding PCCA and PCCB. As of 31 May 2023, 16 participants were enrolled across 5 dose cohorts.

Designing, Developing, and Testing a Chatbot for Parents and Caregivers of Children and Young People With Rheumatological Conditions (the IMPACT Study): Protocol for a Co-Designed Proof-of-Concept Study.

Background: Pediatric rheumatology is a term that encompasses over 80 conditions affecting different organs and systems. Children and young people with rheumatological chronic conditions are known to have high levels of mental health problems and therefore are at risk of poor health outcomes. Clinical psychologists can help children and young people manage the daily difficulties of living with one of these conditions; however, there are insufficient pediatric psychologists in the United Kingdom.

Bulbar function in spinal muscular atrophy (SMA): State of art and new challenges. 21st July 2023, Rome, Italy.

Progressive bulbar involvement is frequent in spinal muscular atrophy, with prevalence and severity of deficits associated with type. The report provides an overview of the presentations made at the workshop grouped into 4 sessions: the first section was dedicated to videofluoroscopy with a revision of the existing protocols and discussion on which one should be used in routine clinical practice and in research settings. The second session was dedicated to interprofessional routine assessments of bulbar function, with a review of the recent clinical tools specifically developed for SMA.

Use of Cefiderocol in the Management of Children With Infection or Colonization With Multi-drug Resistant Gram-negative Bacteria: A Retrospective, Single-center Case Series.

Cefiderocol, a novel siderophore cephalosporin, represents a treatment option for infections with multidrug-resistant Gram-negative bacteria, of which rates are rising worldwide. Clinical data on its use in children is limited. In our pediatric case series, the largest reported to date, cefiderocol seems safe and well tolerated, with more favorable clinical outcomes when compared to the literature reviewing adult cases.

Association Between Chest Compression Pause Duration and Survival After Pediatric In-Hospital Cardiac Arrest.

Background: The association between chest compression (CC) pause duration and pediatric in-hospital cardiac arrest survival outcomes is unknown. The American Heart Association has recommended minimizing pauses in CC in children to <10 seconds, without supportive evidence. We hypothesized that longer maximum CC pause durations are associated with worse survival and neurological outcomes.

A practical evidence-based approach to management of type 2 diabetes in children and young people (CYP): UK consensus.

Background: Type 2 diabetes in young people is an aggressive disease with a greater risk of complications leading to increased morbidity and mortality during the most productive years of life. Prevalence in the UK and globally is rising yet experience in managing this condition is limited. There are no consensus guidelines in the UK for the assessment and management of paediatric type 2 diabetes.

Early Intervention and Use of Autologous Grafts in Growing Skull Fractures Results in Better Outcomes: Experience From a Tertiary Pediatric Neurosurgery Center.

Background And Objectives: Growing skull fracture (GSF) is a rare complication of pediatric head trauma. Definitive treatment is surgical repair. We have attempted to assess whether use of autologous grafts for duraplasty and cranioplasty leads to better outcomes.

Analyzing entropy features in time-series data for pattern recognition in neurological conditions.

In the field of medical diagnosis and patient monitoring, effective pattern recognition in neurological time-series data is essential. Traditional methods predominantly based on statistical or probabilistic learning and inference often struggle with multivariate, multi-source, state-varying, and noisy data while also posing privacy risks due to excessive information collection and modeling. Furthermore, these methods often overlook critical statistical information, such as the distribution of data points and inherent uncertainties.

Paediatric meningitis in the conjugate vaccine era and a novel clinical decision model to predict bacterial aetiology.

Objectives: The aims of this study were to assess aetiology and clinical characteristics in childhood meningitis, and develop clinical decision rules to distinguish bacterial meningitis from other similar clinical syndromes.

Methods: Children aged <16 years hospitalised with suspected meningitis/encephalitis were included, and prospectively recruited at 31 UK hospitals. Meningitis was defined as identification of bacteria/viruses from cerebrospinal fluid (CSF) and/or a raised CSF white blood cell count.

Practice patterns in reporting and documentation of Charles Bonnet syndrome: a retrospective review following COVID-19.

Background: Charles Bonnet syndrome (CBS) is characterized by visual hallucinations occurring in people with visual impairment. CBS can negatively impact psychological well-being, and the COVID-19 pandemic period was associated with an exacerbation of symptoms.

Objectives: To compare clinical practice patterns and reporting of CBS at a tertiary eye care center between an interval prior to the COVID-19 pandemic and an interval during the pandemic.

Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.

Stem cell gene therapy and hematopoietic stem cell transplantation (SCT) require conditioning to ablate the recipient's hematopoietic stem cells (HSCs) and create a niche for gene-corrected/donor HSCs. Conventional conditioning agents are non-specific, leading to off-target toxicities and resulting in significant morbidity and mortality. We developed tissue-specific anti-human CD45 antibody-drug conjugates (ADCs), using rat IgG2b anti-human CD45 antibody clones YTH24.

Accelerometer and Survey Assessed Physical Activity in Children With Epilepsy: A Case-Controlled Study.

Purpose: Anecdotal evidence suggests that children with epilepsy (CWE) are limited in the frequency of their daily physical activity (PA). However, there is limited research utilizing device-based measures of PA. We compared levels of PA and sedentary behavior in CWE (11-15 y) and age- and gender-matched healthy controls.

Extended pelvic lymph node dissection during robotic prostatectomy: antegrade versus retrograde technique.

Background: Robot-assisted radical prostatectomy (RARP) with extended lymphadenectomy (ePLND) is the gold standard for surgical treatment of prostate cancer (PCa). Recently, the en-bloc ePLND has been proposed but no studies reported on the standardization of the technique. The aim of the study is to describe different standardized en-bloc ePLND, the antegrade and the retrograde ePLND, and to compare their surgical and oncological outcomes.