Using QALYs as an Outcome for Assessing Global Prediction Accuracy in Diabetes Simulation Models.

Objectives: (1) To demonstrate the use of quality-adjusted life-years (QALYs) as an outcome measure for comparing performance between simulation models and identifying the most accurate model for economic evaluation and health technology assessment. QALYs relate directly to decision making and combine mortality and diverse clinical events into a single measure using evidence-based weights that reflect population preferences. (2) To explore the usefulness of Q, the proportional reduction in error, as a model performance metric and compare it with other metrics: mean squared error (MSE), mean absolute error, bias (mean residual), and .

How are maternal and fetal outcomes incorporated when measuring benefits of interventions in pregnancy? Findings from a systematic review of cost-utility analyses.

Objective: Medical interventions used in pregnancy can affect the length and quality of life of both the pregnant person and fetus. The aim of this systematic review was to identify and describe the theoretical frameworks that underpin outcome measurement in cost-utility analyses of pregnancy interventions.

Methods: Searches were conducted in the Paediatric Economic Database Evaluation (PEDE) database (up to 2017), as well as Medline, Embase and EconLit (2017-2019).

Using orthopaedic health care resources efficiently: A cost analysis of day surgery for unicompartmental knee replacement.

Background: Day surgery for unicompartmental knee replacement (UKR) could potentially reduce hospital costs. We aimed to measure the impact of introducing a day surgery UKR pathway on mean length of stay (LOS) and costs for the UK NHS, compared to an accelerated inpatient pathway. Secondly, the study aimed to compare the magnitude of costs using three costing approaches: top-down costing; simple micro-costing; and real-world costing.

Estimating Risk Factor Time Paths Among People with Type 2 Diabetes and QALY Gains from Risk Factor Management.

Objectives: Most type 2 diabetes simulation models utilise equations mapping out lifetime trajectories of risk factors [e.g. glycated haemoglobin (HbA)].

Stereotactic radiotherapy for neovascular age-related macular degeneration (STAR): a pivotal, randomised, double-masked, sham-controlled device trial.

Background: Neovascular age-related macular degeneration (nAMD) is a leading cause of blindness. The first-line therapy is anti-vascular endothelial growth factor (anti-VEGF) agents delivered by intravitreal injection. Ionising radiation mitigates key pathogenic processes underlying nAMD, and therefore has therapeutic potential.

Co-aggregation with Apolipoprotein E modulates the function of Amyloid-β in Alzheimer's disease.

Which isoforms of apolipoprotein E (apoE) we inherit determine our risk of developing late-onset Alzheimer's Disease (AD), but the mechanism underlying this link is poorly understood. In particular, the relevance of direct interactions between apoE and amyloid-β (Aβ) remains controversial. Here, single-molecule imaging shows that all isoforms of apoE associate with Aβ in the early stages of aggregation and then fall away as fibrillation happens.

Radiological outcomes following surgical fixation with wires versus moulded cast for patients with a dorsally displaced fracture of the distal radius: a radiographic analysis from the DRAFFT2 trial.

Aims: The primary aim of this study was to report the radiological outcomes of patients with a dorsally displaced distal radius fracture who were randomized to a moulded cast or surgical fixation with wires following manipulation and closed reduction of their fracture. The secondary aim was to correlate radiological outcomes with patient-reported outcome measures (PROMs) in the year following injury.

Methods: Participants were recruited as part of DRAFFT2, a UK multicentre clinical trial.

Rationing in an Era of Multiple Tight Constraints: Is Cost-Utility Analysis Still Fit for Purpose?

Cost-utility analysis may not be sufficient to support reimbursement decisions when the assessed health intervention requires a large proportion of the healthcare budget or when the monetary healthcare budget is not the only resource constraint. Such cases include joint replacement, coronavirus disease 2019 (COVID-19) interventions and settings where all resources are constrained (e.g.

Health poverty among people with type 2 diabetes mellitus (T2DM) in Malaysia.

In the context of the escalating burden of diabetes in low and middle-income countries (LMICs), there is a pressing concern about the widening disparities in care and outcomes across socioeconomic groups. This paper estimates health poverty measures among individuals with type 2 diabetes mellitus (T2DM) in Malaysia. Using data from the National Diabetes Registry between 2009 and 2018, the study linked 932,855 people with T2DM aged 40-75 to death records.

Trends in all-cause mortality among adults with diagnosed type 2 diabetes in West Malaysia: 2010 - 2019.

Aims: We determined 10-year all-cause mortality trends in diagnosed type 2 diabetes (T2D) population in West Malaysia, a middle-income country in the Western-Pacific region.

Methods: One million T2D people aged 40-79 registered in the National Diabetes Registry (2009-2018) were linked to death records (censored on 31 December 2019). Standardized absolute mortality rates and standardized mortality ratios (SMRs) were estimated relative to the Malaysian general population, and standardized to the 2019 registry population with respect to sex, age group, and disease duration.

Effectiveness of nail bed repair in children with or without replacing the fingernail: NINJA multicentre randomized clinical trial.

Background: Surgery for nail bed injuries in children is common. One of the key surgical decisions is whether to replace the nail plate following nail bed repair. The aim of this RCT was to assess the clinical effectiveness and cost-effectiveness of nail bed repair with fingernail replacement/substitution compared with repair without fingernail replacement.

Small soluble α-synuclein aggregates are the toxic species in Parkinson's disease.

Soluble α-synuclein aggregates varying in size, structure, and morphology have been closely linked to neuronal death in Parkinson's disease. However, the heterogeneity of different co-existing aggregate species makes it hard to isolate and study their individual toxic properties. Here, we show a reliable non-perturbative method to separate a heterogeneous mixture of protein aggregates by size.

Moulded cast compared with K-wire fixation after manipulation of an acute dorsally displaced distal radius fracture: the DRAFFT 2 RCT.

Background: Patients with a displaced fracture of the distal radius are frequently offered surgical fixation. Manipulation of the fracture and moulded plaster casting is an alternative treatment that avoids metal implants, but evidence of its effectiveness is lacking.

Objective: To compare functional outcomes, quality-of-life outcomes, complications and resource use among patients with a dorsally displaced fracture of the distal radius treated with manipulation and surgical fixation with Kirschner wires (K-wires) and those treated with manipulation and moulded cast.

Lifetime cost-effectiveness simulation of once-weekly exenatide in type 2 diabetes: A cost-utility analysis based on the EXSCEL trial.

Aims: The Exenatide Study of Cardiovascular Event Lowering (EXSCEL) trial assessed once-weekly exenatide (EQW) vs. placebo, added to usual care in 14,752 patients with type 2 diabetes mellitus (Clinicaltrials.gov: NCT01144338).

Soluble amyloid beta-containing aggregates are present throughout the brain at early stages of Alzheimer's disease.

Protein aggregation likely plays a key role in the initiation and spreading of Alzheimer's disease pathology through the brain. Soluble aggregates of amyloid beta are believed to play a key role in this process. However, the aggregates present in humans are still poorly characterized due to a lack of suitable methods required for characterizing the low concentration of heterogeneous aggregates present.

Progressive exercise compared with best-practice advice, with or without corticosteroid injection, for rotator cuff disorders: the GRASP factorial RCT.

Background: Rotator cuff-related shoulder pain is very common, but there is uncertainty regarding which modes of exercise delivery are optimal and the long-term benefits of corticosteroid injections.

Objectives: To assess the clinical effectiveness and cost-effectiveness of progressive exercise compared with best-practice physiotherapy advice, with or without corticosteroid injection, in adults with a rotator cuff disorder.

Design: This was a pragmatic multicentre superiority randomised controlled trial (with a 2 × 2 factorial design).

Progressive exercise compared with best practice advice, with or without corticosteroid injection, for the treatment of patients with rotator cuff disorders (GRASP): a multicentre, pragmatic, 2 × 2 factorial, randomised controlled trial.

Background: Corticosteroid injections and physiotherapy exercise programmes are commonly used to treat rotator cuff disorders but the treatments' effectiveness is uncertain. We aimed to compare the clinical effectiveness and cost-effectiveness of a progressive exercise programme with a single session of best practice physiotherapy advice, with or without corticosteroid injection, in adults with a rotator cuff disorder.

Methods: In this pragmatic, multicentre, superiority, randomised controlled trial (2 × 2 factorial), we recruited patients from 20 UK National Health Service trusts.

Statistical and health economic analysis plan for a randomized controlled trial of surgical fixation with K-wires versus plaster casting in the treatment of dorsally displaced distal radius fractures: DRAFFT2.

Aims: Describe a statistical and economic analysis plan for the Distal Radius Acute Fracture Fixation Trial 2 (DRAFFT2) randomized controlled trial.

Methods: DRAFFT2 is a multicentre, parallel, two-arm randomized controlled trial. It compares surgical fixation with K-wires versus plaster cast in adult patients who have sustained a dorsally displaced fracture of the distal radius.

Economic Evaluation of Factorial Trials: Cost-Utility Analysis of the Atorvastatin in Factorial With Omega EE90 Risk Reduction in Diabetes 2 × 2 × 2 Factorial Trial of Atorvastatin, Omega-3 Fish Oil, and Action Planning.

Objectives: We applied principles for conducting economic evaluations of factorial trials to a trial-based economic evaluation of a cluster-randomized 2 × 2 × 2 factorial trial. We assessed the cost-effectiveness of atorvastatin, omega-3 fish oil, and an action-planning leaflet, alone and in combination, from a UK National Health Service perspective.

Methods: The Atorvastatin in Factorial With Omega EE90 Risk Reduction in Diabetes (AFORRD) Trial randomized 800 patients with type 2 diabetes to atorvastatin, omega-3, or their respective placebos and randomized general practices to receive a leaflet-based action-planning intervention designed to improve compliance or standard care.

Pre-Fontan Cardiac Catheterization Data as a Predictor of Prolonged Hospital Stay and Post-Discharge Adverse Outcomes Following the Fontan Procedure: A Single-Center Study.

Despite improved outcomes following modifications to the Fontan technique, significant morbidity and mortality persist. We sought to determine if abnormal pre-Fontan catheterization hemodynamic data will predict postoperative prolonged hospital stay (PHLOS) and adverse post-discharge outcomes. This is a retrospective study of patients who underwent the Fontan procedure at Children's Hospital of New Orleans from 2008 to 2018.