Real-world experience of pegcetacoplan in paroxysmal nocturnal hemoglobinuria.

Pegcetacoplan significantly improves outcomes for patients with paroxysmal nocturnal hemoglobinuria (PNH) experiencing extravascular hemolysis (EVH) on eculizumab, leading to approval in 2021/2022 (USA/Europe). We report the first collaborative real-world evidence on pegcetacoplan use in UK and France. A total of 48 patients were either currently receiving or previously received pegcetacoplan (2019-2023).

Real-world efficacy of single-agent belantamab mafodotin in relapsed systemic AL amyloidosis.

Systemic light chain (AL) amyloidosis is a relapsing plasma cell disorder. Therapy is limited, particularly for triple-class refractory disease. We report the use of belantamab mafodotin, a BCMA-directed drug-antibody conjugate, for relapsed AL amyloidosis, including patients traditionally excluded from clinical trials.

Outcomes in ANCA-Associated Vasculitis in Scotland: Validation of the Renal Risk Score in a Complete National Cohort.

Introduction: Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) causes autoimmune-mediated inflammation of small blood vessels in multiple organs, including the kidneys. The ability to accurately predict kidney outcomes would enable a more personalized therapeutic approach.

Methods: We used our national renal biopsy registry to validate the ability of ANCA Renal Risk Score (ARRS) to predict end-stage kidney disease (ESKD) for individual patients.

Estimating the effect of physical activity on cognitive function within the UK Biobank cohort.

Background: Physical activity (PA) has been associated with benefits for cognitive function (CF), but previous estimates of the strength of this relationship may have been biased due to limitations in statistical modelling practices that are common among observational studies. We aimed to address this by using a rigorously constructed conceptual causal model to guide an empirical analysis estimating the effect of PA on CF in the UK Biobank cohort of middle-aged and older adults.

Methods: This study analysed a subsample of 334 227 adults from the UK Biobank prospective cohort study.

Incidence and Outcomes of Patients Receiving Chronic Kidney Replacement Therapy Admitted to Scottish ICUs Between 2009 and 2019-A National Observational Cohort Study.

Objectives: To determine the incidence and characteristics of ICU admissions in the Scottish population of patients treated with chronic kidney replacement therapy (KRT) over an 11-year period and determine factors associated with post-ICU admission mortality.

Design: Retrospective observational cohort study.

Setting: We analyzed admissions to Scottish intensive care environments between January 1, 2009, and December 31, 2019.

Comprehensive Geriatric Assessment for community-dwelling, high-risk, frail, older people.

Background: Comprehensive Geriatric Assessment (CGA) is a multidimensional interdisciplinary diagnostic process focused on determining an older person's medical, psychological and functional capability in order to develop a co-ordinated and integrated care plan. CGA is not limited simply to assessment, but also directs a holistic management plan for older people, which leads to tangible interventions. While there is established evidence that CGA reduces the likelihood of death and disability in acutely unwell older people, the effectiveness of CGA for community-dwelling, frail, older people at risk of poor health outcomes is less clear.

Clinical Performance of Triage Strategies for Hr-HPV-Positive Women; A Longitudinal Evaluation of Cytology, p16/K-67 Dual Stain Cytology, and HPV16/18 Genotyping.

Background: We evaluated the longitudinal performance of three options: HPV16/18 genotyping (HPV16/18), cytology (LBC), and p16/Ki-67 dual stain cytology (DS) for the triage of high-risk Human Papillomavirus-positive (Hr-HPV+) women within the cervical screening program in Scotland.

Methods: Data were derived from a cohort of Hr-HPV+ women (n = 385) who participated in PaVDaG (Papillomavirus Dumfries and Galloway) study. Performance of triage strategies for detecting high-grade disease was assessed at 3 (in women <50 years) or 5 years (in women >50 years).

Clinical performance of DNA and RNA based HPV tests for test of cure (TOC) post treatment for cervical intraepithelial neoplasia (CIN) - a retrospective study.

HPV testing as a "test of cure" (TOC) of women treated for cervical high-grade lesions can support and inform appropriate clinical management pathways. However, there is a lack of studies that report the discrete performance of different HPV assays in this context, including HPV mRNA based assays. To address this, we performed an analysis of the clinical performance of two hrHPV assays in the (TOC) setting; the recently launched DNA based Alinity m HR HPV (Abbott Molecular) and RNA based Aptima HPV assay (Hologic).

TEARS: a longitudinal investigation of the prevalence, psychological associations and trajectory of poststroke emotionalism.

Objective: There are few longitudinal studies of poststroke emotionalism (PSE) and our understanding of the psychological associations of PSE is limited, constraining assessment of existing interventions and the development of new therapies. This study aimed to assess the prevalence and course of PSE over the first year poststroke, and its psychological associations.

Methods: Consenting stroke survivors who were physically and cognitively able to participate were assessed within 2 weeks, 6 and 12 months of stroke to determine PSE point prevalence using a diagnostic, semistructured PSE interview (Testing Emotionalism After Recent Stroke-Diagnostic Interview).

The Impact of Vaccination on Incidence and Outcomes of SARS-CoV-2 Infection in Patients with Kidney Failure in Scotland.

Background: Patients with kidney failure requiring KRT are at high risk of complications and death following SARS-CoV-2 infection, with variable antibody responses to vaccination reported. We investigated the effects of COVID-19 vaccination on the incidence of infection, hospitalization, and death from COVID-19 infection.

Methods: The study design was an observational data linkage cohort study.

Self-sampling as the principal modality for population based cervical screening: Five-year follow-up of the PaVDaG study.

Self-sampling provides a powerful means to engage women in cervical screening. In the original Papillomavirus Dumfries and Galloway study (PaVDaG), we demonstrated cross-sectional similarity of high-risk human papillomavirus (Hr-HPV) testing on self-taken vaginal vs clinician-taken samples for the detection of cervical intraepithelial neoplasia 2 or worse (CIN2+). Few data exist on the longitudinal performance of self-sampling; we present longitudinal outcomes of PaVDaG.

What is the precaecocolic fascia?

The precaecocolic fascia, previously known as Jackson's membrane, is a variable vascular peritoneal fold between the ascending colon and the right posterolateral abdominal wall. First described in 1913, it was originally thought to be of developmental or inflammatory origin and associated with abdominal pain. This investigation aimed to review its frequency, form and structure and look for evidence of association with malformation of the bowel, or previous inflammation.

Adult-onset recurrent Kawasaki disease: an unusual cause of fever of unknown origin.

We present the case of a 20-year-old man with a background of transposition of the great arteries presenting with fever of unknown origin, who developed a shock like syndrome with respiratory failure necessitating intubation. After extensive investigation, a diagnosis of adult-onset Kawasaki disease was made, and he was successfully treated with IVIg and corticosteroids. We present the clinical findings clinicians should be aware of, and review the literature on managing this rare presentation in adult, highlighting the importance of early diagnosis in improving outcomes.

Practical Pharmacist-Led Interventions to Improve Antimicrobial Stewardship in Ghana, Tanzania, Uganda and Zambia.

The World Health Organisation (WHO) and others have identified, as a priority, the need to improve antimicrobial stewardship (AMS) interventions as part of the effort to tackle antimicrobial resistance (AMR). An international health partnership model, the Commonwealth Partnerships for Antimicrobial Stewardship (CwPAMS) programme, was established between selected countries in Africa (Ghana, Tanzania, Zambia and Uganda) and the UK to support AMS. This was funded by UK aid under the Fleming Fund and managed by the Commonwealth Pharmacists Association (CPA) and Tropical Health and Education Trust (THET).

An international genome-wide meta-analysis of primary biliary cholangitis: Novel risk loci and candidate drugs.

Backgrounds & Aims: Primary biliary cholangitis (PBC) is a chronic liver disease in which autoimmune destruction of the small intrahepatic bile ducts eventually leads to cirrhosis. Many patients have inadequate response to licensed medications, motivating the search for novel therapies. Previous genome-wide association studies (GWAS) and meta-analyses (GWMA) of PBC have identified numerous risk loci for this condition, providing insight into its aetiology.

ANCA-associated renal vasculitis is associated with rurality but not seasonality or deprivation in a complete national cohort study.

Background: Small studies suggest an association between ANCA-associated vasculitis (AAV) incidence and rurality, seasonality and socioeconomic deprivation. We examined the incidence of kidney biopsy-proven AAV and its relationship with these factors in the adult Scottish population.

Methods: Using the Scottish Renal Biopsy Registry, all adult native kidney biopsies performed between 2014 and 2018 with a diagnosis of granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) were identified.

X Chromosome Contribution to the Genetic Architecture of Primary Biliary Cholangitis.

Background & Aims: Genome-wide association studies in primary biliary cholangitis (PBC) have failed to find X chromosome (chrX) variants associated with the disease. Here, we specifically explore the chrX contribution to PBC, a sexually dimorphic complex autoimmune disease.

Methods: We performed a chrX-wide association study, including genotype data from 5 genome-wide association studies (from Italy, United Kingdom, Canada, China, and Japan; 5244 case patients and 11,875 control individuals).

The challenges of defining sample adequacy in an era of HPV based cervical screening.

Background: The implementation of Human Papillomavirus based cervical screening continues apace on a global scale. Understanding the basis and burden of inadequate or invalid samples is important to ensure confidence in high quality laboratory results and inform the development of new technologies. Here we present population based data from Scotland and Denmark which detail the extent of invalid samples for HPV detection in both clinician-taken and self-taken samples.

Clinical Impact of Residual C-Peptide Secretion in Type 1 Diabetes on Glycemia and Microvascular Complications.

Objective: To quantify the relationship of residual C-peptide secretion to glycemic outcomes and microvascular complications in type 1 diabetes.

Research Design And Methods: C-peptide was measured in an untimed blood sample in the Scottish Diabetes Research Network Type 1 Bioresource (SDRNT1BIO) cohort of 6,076 people with type 1 diabetes monitored for an average of 5.2 years.

One-year outcomes from a phase 3 randomized trial of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who received prior eculizumab.

Ravulizumab every 8 weeks showed non-inferiority to eculizumab every 2 weeks in a 26-week, phase 3, randomized controlled trial in adults with paroxysmal nocturnal hemoglobinuria (PNH) who were clinically stable on eculizumab (NCT03056040). We report results from the first 26 weeks of the extension period in which patients continued ravulizumab (n = 96) or switched from eculizumab to ravulizumab (n = 95). At week 52, mean (SD) lactate dehydrogenase levels increased 8.

A systematic review of the physical activity levels of acutely ill older adults in Hospital At Home settings: an under-researched field.

Purpose: The purpose of this review was to identify, evaluate and synthesise existing evidence reporting the physical activity levels of acutely ill older patients in a 'Hospital At Home' setting and compare this to patients with similar characteristics treated in a traditional hospital inpatient setting. Functional changes and any adverse outcomes due to physical activity (e.g.