Patient-Reported Improvements in Patients with PNH Treated with Iptacopan from Two Phase 3 Studies.

Iptacopan, a first-in-class, oral, selective complement factor B inhibitor, demonstrated efficacy and safety as monotherapy in C5 inhibitor (C5i)-experienced (APPLY-PNH [NCT04558918]) and C5i-naive (APPOINT-PNH [NCT04820530]) patients with paroxysmal nocturnal hemoglobinuria (PNH). In APPLY-PNH and APPOINT-PNH, changes in fatigue (FACIT-Fatigue) and health-related quality of life (HRQOL; EORTC QLQ-C30) from baseline to Day 168 were evaluated. The proportion of patients achieving meaningful within-patient change (MWPC) on the FACIT-Fatigue and 4 EORTC QLQ-C30 subscales (physical functioning, role functioning, fatigue, dyspnea) was evaluated using anchor-based thresholds.

Schizophrenia more employable than depression? Language-based artificial intelligence model ratings for employability of psychiatric diagnoses and somatic and healthy controls.

Artificial Intelligence (AI) assists recruiting and job searching. Such systems can be biased against certain characteristics. This results in potential misrepresentations and consequent inequalities related to people with mental health disorders.

Biomarkers in biliary atresia - an elusive promise of predicting outcome.

Research on biomarkers in BA promises to predict and change outcomes in BA. This goal is still unrealized at the moment. The study by Kamp et al attempts to understand the drivers of fibrosis and elucidate the role of amyloid-related genes in the pathophysiology of BA.

Intestinal icrobiota Transplant Prior to llogeneic tem Cell ransplant (MAST) trial: study protocol for a multicentre, double-blinded, placebo-controlled, phase IIa trial.

Introduction: Lower diversity of the gut microbiome prior to allogeneic haematopoietic cell transplantation (HCT) correlates with reduced survival after the intervention. Most patients undergoing HCT for a haematological malignancy have previously received intensive chemotherapy, resulting in prolonged neutropenic episodes requiring broad-spectrum antibiotics; use of these has been linked to reduced microbiome diversity. Intestinal microbiota transplant (IMT) is a novel treatment approach that restores this diversity.

In Situ Light-Source Delivery During 5-Aminulevulinic Acid-Guided High-Grade Glioma Resection: Spatial, Functional and Oncological Informed Surgery.

Background/objectives: 5-aminulevulinic acid (5-ALA)-guided surgery for high-grade gliomas remains a challenge in neuro-oncological surgery. Inconsistent fluorescence visualisation, subjective quantification and false negatives due to blood, haemostatic agents or optical impediments from the external light source are some of the limitations of the present technology.

Methods: The preliminary results from this single-centre retrospective study are presented from the first 35 patients operated upon with the novel Nico Myriad Spectra System©.

Expedited Conveyance of Out-of-Hospital-Cardiac Arrest patients with STEMI and shockable rhythms to Cardiac Arrest Centres - A Feasibility Pilot Study of the British Cardiovascular Intervention Society conveyance algorithm.

Background And Aims: Guidelines suggest non-traumatic out-of-hospital cardiac arrest (OHCA) be conveyed to cardiac arrest centres (CAC). We hypothesised that (a) a pre-hospital conveyance algorithm based on initial presenting rhythm following OHCA is feasible and (b) that would demonstrate survival advantage.

Methods: This observational pilot study included all consecutive patients with OHCA from suspected cardiac aetiology from the county of Essex, United Kingdom from April 2022-April 2023.

Clinical outcomes of closed versus open simple ankle fractures patterns requiring soft tissue reconstruction: A prospective comparative observational study.

Background: Contemporary guidelines advocate for initial debridement and single-stage definitive fixation with immediate soft tissue reconstruction for open fractures. This study aims to evaluate the effectiveness of single-stage stabilization and immediate definitive soft tissue coverage in open ankle fractures compared to closed fractures.

Methods: We compared all isolated open ankle fractures (OF) treated between January 2017 and June 2019 to a control group of operatively managed closed ankle fractures (CF).

Implant waste and associated costs in trauma and orthopaedic surgery: a systematic review.

Purpose: Trauma and orthopaedic (T&O) surgery relies on medical implants and materials, often resulting in metalwork wastage (prosthesis, screws, nails, and plates). This places an economic strain on healthcare services and the environment. Our primary outcome is to quantify the implant wastage across the literature, and secondarily investigate the associated costs in this specialty.

Current concepts of fourth-generation minimally invasive and open hallux valgus surgery.

Hallux valgus (HV) presents as a common forefoot deformity that causes problems with pain, mobility, footwear, and quality of life. The most common open correction used in the UK is the Scarf and Akin osteotomy, which has good clinical and radiological outcomes and high levels of patient satisfaction when used to treat a varying degrees of deformity. However, there are concerns regarding recurrence rates and long-term outcomes.

Comparative Efficacy of Videolaryngoscopy and Direct Laryngoscopy in Patients Living With Obesity: A Meta-Analysis.

Intubation in patients living with obesity poses unique challenges due to altered airway anatomy and reduced physiological reserve, increasing the risk of complications. In synthesizing evidence from multiple trials, our meta-analysis suggests that videolaryngoscopy may provide a higher likelihood of achieving successful intubation on the first attempt compared to direct laryngoscopy while not substantially increasing the procedure time. Videolaryngoscopy was associated with a significant increase in first-pass intubation success compared to direct laryngoscopy, with a pooled risk ratio (RR) of 0.

Efficacy and safety of low-dose tolvaptan (7.5mg) in the treatment of inpatient hyponatraemia: a retrospective study.

Objectives: The recommended dose of tolvaptan for hyponatraemia secondary to SIADH is 15mg. We evaluated the efficacy of an initial 7.5mg dose and determined the frequency where sodium (Na+) correction exceeded safe limits, defined as an increment of ≥10 mmol/L, within the initial 8 or 24 hours of administration.

Cardiovascular-Liver-Metabolic Health: Recommendations in Screening, Diagnosis, and Management of Metabolic Dysfunction-Associated Steatotic Liver Disease in Cardiovascular Disease via Modified Delphi Approach.

There is a new awareness of the widespread nature of metabolic dysfunction-associated steatotic liver disease (MASLD) and its connection to cardiovascular disease (CVD). This has catalyzed collaboration between cardiologists, hepatologists, endocrinologists, and the wider multidisciplinary team to address the need for earlier identification of those with MASLD who are at increased risk for CVD. The overlap in the pathophysiologic processes and parallel prevalence of CVD, metabolic syndrome, and MASLD highlight the multisystem consequences of poor cardiovascular-liver-metabolic health.

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.

Impact of hyperkalaemia on renin-angiotensin-aldosterone (RAAS) inhibitor reduction or withdrawal following hospitalisation.

Background: Inhibitors of the renin-angiotensin-aldosterone system (RAAS), such as ACE inhibitors (ACEi), angiotensin-II receptor blockers and mineralocorticoid receptor antagonists, reduce morbidity and mortality in hypertension, congestive heart failure and chronic kidney disease. However, their use can lead to hyperkalaemia. We examined the proportions of RAAS inhibitor (RAASi) reduction or withdrawal, across GFR strata, following hospitalisation and the effect on patient mortality.

Long-Term Efficacy and Safety of Danicopan as Add-On Therapy to Ravulizumab or Eculizumab in PNH With Significant EVH.

Complement C5 inhibitor treatment with ravulizumab or eculizumab for paroxysmal nocturnal hemoglobinuria (PNH) improves outcomes and survival. Some patients remain anemic due to clinically significant extravascular hemolysis (cs-EVH: hemoglobin [Hgb] ≤9.5 g/dL and absolute reticulocyte count [ARC] ≥120×109/L).

Corrigendum to "Genetic Variants of Are Major Determinants of Kidney Failure in People of African Ancestry With HIV" [ Volume 7, Issue 4, April 2022, Pages 786-796].

[This corrects the article DOI: 10.1016/j.ekir.

Genital bleeding in prepubertal girls: a systematic review.

Introduction: This systematic review aims to collate evidence of the causes of genital bleeding, other than child sexual abuse and accidental injuries, presenting in prepubertal girls. It provides an update to the 2015 Royal College of Paediatrics and Child Health publication 'The Physical Signs of Child Sexual Abuse', an evidence-based review also known as the Purple Book.

Methods: MEDLINE, EMBASE and Scopus databases were searched for studies published between March 2014 and June 2023.

Pulsed-field ablation of atrial fibrillation with a pentaspline catheter across National Health Service England centres.

Introduction: Pulsed-field ablation (PFA) is a novel modality for pulmonary vein isolation in patients with atrial fibrillation (AF). We describe the initial uptake and experience of PFA using a pentaspline catheter across selected National Health Service England (NHSE) centres.

Methods: Data collected by NHSE Specialised Services Development Programme regarding AF ablation procedures using a single-shot, pentaspline, multielectrode PFA catheter (FARAWAVE, Boston Scientific) between June 2022 and August 2024 were aggregated and analysed to examine procedural metrics, acute efficacy and safety outcomes over 3-month follow-up.

Weight bearing versus non-weight bearing total contact cast in the management of active Charcot foot: A systematic review.

Aim: Diabetic Charcot neuro-osteoarthropathy carries a significant worldwide disease burden including diabetic foot infection, ulceration and amputation. The current accepted standard of treatment during the active phase of Charcot neuro-osteoarthropathy is offloading with total contact casting; however, controversy remains regarding weight-bearing status during this period.

Methods: A systematic review was performed following PRISMA guidelines of Pubmed, EMBASE, MEDLINE and the Cochrane central register of controlled trials for clinical studies from inception until June 2024 investigating weight-bearing and non-weight-bearing total contact casting for active Charcot neuro-osteoarthropathy.

Exploring links between oral health and infective endocarditis.

Infective endocarditis (IE) is a bacterial infection of the heart's inner lining. A low incidence rate combined with a high mortality rate mean that IE can be difficult to treat effectively. There is currently substantial evidence supporting a link between oral health and IE with the oral microbiome impacting various aspects of IE, including pathogenesis, diagnosis, treatment, and mortality rates.

'What is 'wellbeing' to doctors in training, and how do we improve it? Results of a quantitative survey and thematic analysis of internal medicine trainees.

Introduction: The wellbeing of medical trainees has come into sharp focus in recent years, particularly since the COVID-19 pandemic. Workforce pressures are well documented, as is the need to retain staff. There are a number of initiatives to try and improve the wellbeing of staff, including the introduction of wellbeing and education dellows for stage 1 internal medicine trainees in some regions.