What is this summary about?This summary reviews three evinacumab studies (two clinical trials and one computer-based model study with real-world data) in children with homozygous familial hypercholesterolemia (HoFH). Evinacumab 15 mg/kg administered into the vein (an intravenous infusion) every 4 weeks was tested in children (aged 5 to 11 years) in the pediatric study and adolescents (aged 12 to 17 years) in a long-term study. Computer-based models were used to predict how the same dose would work in younger children (aged 6 months to 4 years) by using data from clinical trials. This was later verified with real-world data from six children aged 1 to 4 years.What were the results of these studies?Across the clinical studies, evinacumab decreased ‘bad’ cholesterol (low-density lipoprotein cholesterol, or LDL-C) by around 40 to 50% and was generally well tolerated, with no new safety signals identified. In the computer-based model of children from 6 months to 4 years old, similar LDL-C reductions to those from observed real-world data in children 1 to 4 years old were predicted.What do the results mean?These findings supported the recent approval of evinacumab in children as young as 6 months of age in many countries.
INTRODUCTION Heart failure with preserved ejection fraction (HFpEF) accounts for over half of all heart failure hospitalisations and is increasing in prevalence, largely driven by obesity and Type 2 Diabetes Mellitus (T2DM). Glucagon-like-peptide receptor agonists (GLP-1 RAs) and the dual GLP-1/GIP agonist, tirzepatide, have been shown to be safe and effective medications in the treatment of T2DM and obesity as well as in obesity-related HFpEF. AREAS COVERED This review summarises the pathophysiology of obesity-related HFpEF, mechanisms by which GLP-1 RAs and tirzepatide may improve symptoms and outcomes, and summarise evidence from recent randomised controlled and observational trials across the disease spectrum they have been studied in. Therapeutic implications and future research directions are also discussed. A structured literature search was performed using PubMed/MEDLINE and Cochrane Central Register of Controlled Trials (CENTRAL) up to August 2025. EXPERT OPINION GLP-1 receptor agonists and tirzepatide represent a promising therapeutic advance for obesity-related HFpEF. Benefits are likely mediated through weight loss dependent and weight-loss independent mechanisms. Important uncertainties remain regarding their role in non-obese HFpEF, optimal patient selection, and their safety in HFrEF.
INTRODUCTION Ticagrelor is an oral reversible platelet P2Y12 inhibitor that improves ischaemic outcomes in atherosclerotic cardiovascular disease, including the prevention of stent thrombosis after percutaneous coronary intervention, at the cost of increased bleeding risk. AREAS COVERED A comprehensive literature search was performed across PubMed and Web of Science (inception to January 2026). This case-based review outlines the current guidance surrounding bleeding events and emergent surgery on oral P2Y12 inhibitors, treatment options for reversing their effects, and describes a novel ticagrelor reversal agent in two cases of life-threatening bleeding, one related to gastrointestinal haemorrhage and one associated with emergent coronary artery bypass graft surgery. EXPERT OPINION Ticagrelor is a first-line treatment for acute coronary syndromes but is associated with heightened bleeding risk, especially when combined with aspirin. Ticagrelor's reversibility may offer advantages over the irreversibly-acting thienopyridine P2Y12 inhibitors. Patients treated with potent antiplatelet regimens may require rapid and effective restoration of haemostatic activity in addition to supportive measures in those presenting with life-threatening haemorrhage or requiring emergent surgery. We summarise the current available treatment options to reverse antiplatelet activity and highlight the role of ticagrelor reversal with bentracimab in improving haemostasis in emergency settings.
