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Our latest research, all in one place. Browse our collection of journal articles, reports and conference proceedings to see how we’re contributing to HEOR research. Remember to: 

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Peer-reviewed publication

A Machine Learning Approach to Identify Important Variables for Distinguishing between Fallers and Non-Fallers in Older Women

YHEC authors: Emily Gregg
Publication date: October 2023
Journal: Plos One

Abstract

Falls are a significant ongoing public health concern for older adults. At present, few studies have concurrently explored the influence of multiple measures when seeking to determine which variables are most predictive of fall risks. As such, this cross-sectional study aimed to identify those functional variables (i.e. balance, gait and clinical measures) and physical characteristics (i.e. strength and body composition) that could best distinguish between older female fallers and non-fallers, using a machine learning approach. Overall, 60 community-dwelling older women (=65 years), retrospectively classified as fallers (n = 21) or non-fallers (n = 39), attended three data collection sessions. Data (281 variables) collected from tests in five separate domains (balance, gait, clinical measures, strength and body composition) were analysed using random forest (RF) and leave-one-variable-out partial least squares correlation analysis (LOVO PLSCA) to assess variable importance. The strongest discriminators from each domain were then aggregated into a multi-domain dataset, and RF, LOVO PLSCA, and logistic regression models were constructed to identify the important variables in distinguishing between fallers and non-fallers. These models were used to classify participants as either fallers or non-fallers, with their performance evaluated using receiver operating characteristic (ROC) analysis. The study found that it is possible to classify fallers and non-fallers with a high degree of accuracy (e.g. logistic regression: sensitivity = 90%; specificity = 87%; AUC = 0.92; leave-one-out cross-validation accuracy = 63%) using a combination of 18 variables from four domains, with the gait and strength domains being particularly informative for screening programmes aimed at assessing falls risk.

Peer-reviewed publication

Development of a Health Economic Model to Evaluate the Cost-Effectiveness of Roxadustat in Treating Anemia Associated with Non-Dialysis-Dependent Chronic Kidney Disease

YHEC authors: Stuart Mealing, Joe Moss
Publication date: October 2023
Journal: Journal of Medical Economics

Abstract

BACKGROUND: Treatment for anemia of chronic kidney disease (CKD) largely consists of erythropoiesis-stimulating agents (ESAs) with iron supplementation. Although ESAs are well-established and efficacious, their use has been associated with considerable economic and humanistic burdens. Roxadustat, an oral medication, is a hypoxia-inducible factor prolyl hydroxylase inhibitor that targets multiple causes of CKD and has a similar efficacy and safety profile to ESAs. The cost-effectiveness of this treatment, however, has yet to be investigated.

OBJECTIVE: The study objective was to develop a health economic model to evaluate the cost-effectiveness of roxadustat compared with ESAs for treating anemia of non-dialysis-dependent (NDD) CKD.

METHODS: A cohort-based model was developed for a hypothetical cohort of 1,000 patients with anemia of NDD CKD, incorporating eight health states, representing the hemoglobin level of each patient. The model was informed by individual patient-level data from the roxadustat global phase 3 clinical trial program. Total and incremental costs as well as quality-adjusted life-years (QALYs) associated with roxadustat versus ESAs were estimated from the perspective of the UK National Health Service. Sensitivity analyses were performed to assess the robustness of the model. Analyses exploring alternative scenarios were also conducted.

RESULTS: On a per-person basis, over 1,000 simulations, roxadustat was found to be on average less costly (-£32) and more effective (+0.01 QALYs) than ESAs, with a dominant incremental cost-effectiveness ratio. The probability of cost-effectiveness at a £20,000 per QALY willingness-to-pay threshold from the UK perspective was 67%.

CONCLUSION: The model developed may be a useful instrument that, alongside expert clinical opinion, can inform clinical and policy decision-making regarding treatment of anemia of NDD CKD. The model highlights the cost-effectiveness of roxadustat, as well as its potential to have a meaningful impact in reducing the burden of anemia of NDD CKD.

Peer-reviewed publication

Plus Sutures for Preventing Surgical Site Infection: A Systematic Review of Clinical Outcomes with Economic and Environmental Models

YHEC authors: Mary Edwards, Sara Graziadio
Publication date: October 2023
Journal: BMC Surgery

Abstract

BACKGROUND: Surgical site infections (SSIs) represent ~ 20% of all hospital-acquired infections in surgical patients and are associated with prolonged hospital stay, admission to intensive care, and mortality. We conducted a systematic review with economic and environmental models to assess whether triclosan-coated sutures (Plus Sutures) provide benefits over non-coated sutures in the reduction of SSI risk.

METHODS: Searches were conducted in fifteen databases. A total of 1,991 records were retrieved. Following deduplication and screening by two independent reviewers, 31 randomized controlled trials in adults and children were included in the review. Similarity of the studies was assessed by narrative review and confirmed by quantitative assessment. A fixed effects meta-analysis of SSI incidence model including all groups of patients estimated a risk ratio of 0.71 (95% confidence interval: 0.64 to 0.79) indicating those in the Plus Sutures group had a 29% reduction in the risk of developing an SSI compared with those in the control group (p < 0.001). Safety outcomes were analysed qualitatively. RESULTS: The economic model estimated the use of Plus Sutures to result in average cost savings of £13.63 per patient. Plus Sutures remained cost-saving in all subgroup analyses with cost-savings ranging between £11 (clean wounds) and £140 (non-clean wounds). The environmental impact of SSI is substantial, and the model suggests that the introduction of Plus Sutures could result in potential environmental benefits. CONCLUSIONS: The evidence suggests that Plus Sutures are associated with a reduced incidence of SSI across all surgery types alongside cost savings when compared with standard sutures.

Peer-reviewed publication

The Burden of Mitochondrial Disease with Associated Seizures: Systematic Literature Reviews of Health-Related Quality of Life, Utilities, Costs and Healthcare Resource Use Data

YHEC authors: Emily Gregg, Chris Bartlett, Mich Arber, Deborah Watkins, Sara Gradiadio
Publication date: October 2023
Journal: Orphanet Journal of Rare Diseases

Abstract

BACKGROUND: Mitochondrial disease is a degenerative, progressive, heterogeneous group of genetic disorders affecting children and adults. Mitochondrial disease is associated with morbidity and mortality, with predominantly neurological and neuromuscular symptoms including dystonia, weakness, encephalopathy, developmental delay and seizures. Seizures are one of the most common and severe manifestations of mitochondrial disease. These seizures are typically refractory to common anti-seizure therapies. There are no approved disease-modifying treatments for mitochondrial disease. Our objective was to conduct two systematic literature reviews to identify health-related quality of life (HRQoL), utilities, costs and healthcare resource use data in mitochondrial disease with associated seizures.

METHODS: A range of databases and information sources were searched up to July 2022 to identify eligible studies. Search strategies included a range of variant terms for mitochondrial disease and HRQoL, utilities, cost and healthcare resource use outcomes. Two reviewers independently assessed articles against the eligibility criteria; studies were extracted by one reviewer and checked by a second. Risk of bias was assessed for studies reporting HRQoL data. Results were narratively assessed.

RESULTS: Seven studies were eligible for the HRQoL and utilities review. The studies used different tools to report data, and despite the variability in methods, HRQoL scores across the studies showed moderate/severe disease in patients with mitochondrial disease with associated seizures. Parents of patients with mitochondrial disease with associated seizures were characterised by high total parenting stress. No studies reported utilities data. Two case reports and one retrospective review of medical records of children who died in hospital were eligible for the costs and resource use review. These provided limited information on the duration of hospital stay, in an intensive care unit (ICU), on mechanical ventilation. No studies reported costs data.

CONCLUSION: These reviews highlight the limited HRQoL, utilities, costs and resource use data and the variability of instruments used in mitochondrial disease with associated seizures. However, the data available indicate that mitochondrial disease with associated seizures affects patients' and caregivers' HRQoL alike. No robust conclusion can be drawn on the impact of mitochondrial disease with associated seizures on hospital or ICU length of stay.

Trial registration PROSPERO: CRD42022345005.

Report

[GID-HTE10021] Digital Technologies for Managing Low Back Pain: External Assessment Group Report

YHEC authors: Heather Davies, Lavinia Ferrante di Ruffano, Rachael McCool, Hayden Holmes, Robert Malcolm, Paul Miller, Samuel Harper, Emma Carr, James Scott, Emma Bishop
Publication date: September 2023
Publishers: National Insitute for Health and Care Excellence

Abstract

No abstract available.

Peer-reviewed publication

Care Pathway Analysis and Evidence Gaps in Adult-Onset Still’s Disease: Interviews with Experts from the UK, France, Italy, and Germany

YHEC authors: Emily Gregg, Sara Graziadio
Publication date: September 2023
Journal: Frontiers in Medicine

Abstract

INTRODUCTION: Adult-onset Still's disease (AOSD) is a rare systemic inflammatory disease of unknown etiology. Published AOSD data are limited, and clinical guidelines were lacking until recently. Managing AOSD remains largely empirical with uncertainties and high variability about the optimal care pathway. Therefore, we used a qualitative approach to collect clinical judgments from the UK, Italy, France and Germany to inform the development of an agreed care pathway. Our work aimed to decrease the uncertainty associated with clinical practice, inform future research in AOSD, and help identify standardized definitions and outcomes in this population.

METHODS: Semi-structured interviews and thematic analysis were conducted. Eleven clinicians were interviewed between May and July 2022: four were based in Italy, three in the UK, two in France, and two in Germany.

RESULTS: In this work, we identified the structure of the typical care pathway for AOSD patients, which can be used to inform future economic models in AOSD. The general structure of the pathway was similar across countries. Non-steroidal anti-inflammatory drugs are prescribed during the diagnostic workup while an additive approach is commonly used in confirmed cases: corticosteroids, conventional synthetic disease-modifying antirheumatic drugs, then biologic disease-modifying antirheumatic drugs (bDMARDs) (dose increased before switching). For severe presentations, more aggressive approaches with higher doses and early use of bDMARDs are used. The main elements of variation among countries and clinicians were the criteria used for diagnosis; order of bDMARDs and preferential treatments for articular and systemic patients; and tests for patient monitoring. There is also a lack of standardized outcome measures making comparisons and evidence synthesis challenging.

CONCLUSION: We identified important evidence gaps for clinical practice, e.g., reliable tests or scores predictive of disease progression and treatment outcome, and recommendations for research, e.g., reporting of compliance rates and use of the Yamaguchi criteria for clinical study inclusion. Consensus is needed around the use of the Systemic score in clinical practice and the clinical utility of this score. A standardized definition of remission is also required in AOSD, and further research should look to identify and validate the specific laboratory markers to be considered when assessing remission.

Peer-reviewed publication

A Mapping Exercise Using Automated Techniques to Develop a Search Strategy to Identify Systematic Review Tools

YHEC authors: Christopher Marshall
Publication date: September 2023
Journal: Research Synthesis Methods

Abstract

The Systematic Review Toolbox aims provide a web-based catalogue of tools that support various tasks within the systematic review and wider evidence synthesis process. Identifying publications surrounding specific systematic review tools is currently challenging, leading to a high screening burden for few eligible records. We aimed to develop a search strategy that could be regularly and automatically run to identify eligible records for the SR Toolbox, thus reducing time on task and burden for those involved. We undertook a mapping exercise to identify the PubMed IDs of papers indexed within the SR Toolbox. We then used the Yale MeSH Analyser and Visualisation of Similarities (VOS) Viewer text-mining software to identify the most commonly used MeSH terms and text words within the eligible records. These MeSH terms and text words were combined using Boolean Operators into a search strategy for Ovid MEDLINE. Prior to the mapping exercise and search strategy development, 81 software tools and 55 'Other' tools were included within the SR Toolbox. Since implementation of the search strategy, 146 tools have been added. There has been an increase in tools added to the toolbox since the search was developed and its corresponding auto-alert in MEDLINE was originally set up. Developing a search strategy based on a mapping exercise is an effective way of identifying new tools to support the systematic review process. Further research could be conducted to help prioritise records for screening to reduce reviewer burden further and to adapt the strategy for disciplines beyond healthcare.

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