Health technology assessment最新文献

{"title":"Automated devices for identifying peripheral arterial disease in people with leg ulceration: an evidence synthesis and cost-effectiveness analysis.","authors":"Dwayne Boyers, Moira Cruickshank, Lorna Aucott, Charlotte Kennedy, Paul Manson, Paul Bachoo, Miriam Brazzelli","doi":"10.3310/TWCG3912","DOIUrl":"10.3310/TWCG3912","url":null,"abstract":"<p><strong>Background: </strong>Peripheral artery disease is a common condition caused by narrowing/blockage of the arteries, resulting in reduced blood supply. Peripheral artery disease is associated with an increased risk of vascular complications, but early treatment reduces mortality and morbidity. Leg ulcers are long-lasting wounds, usually treated by compression therapy. Compression therapy is not suitable for people with peripheral artery disease, as it can affect the arterial blood supply. In clinical practice, people with peripheral artery disease are identified by measurement of the ankle-brachial pressure index using a sphygmomanometer and manual Doppler device. However, this method can be uncomfortable for people with leg ulcers and automated devices have been proposed as a more acceptable alternative. The objective of this appraisal was to summarise the clinical and cost-effectiveness evidence on the use of automated devices to detect peripheral artery disease in people with leg ulcers.</p><p><strong>Methods: </strong>.</p><p><strong>Clinical effectiveness: </strong>To identify reports of relevant studies, we searched major electronic databases and scrutinised the information supplied by the manufacturers of the automated devices under investigation. Due to the lack of evidence on people with leg ulcers, we considered evidence from studies of any design assessing automated devices versus an acceptable reference device in any population receiving ankle-brachial pressure index assessment. We summarised information on diagnostic accuracy of the automated devices and level of agreement with the reference device. For each device, when data permit, we pooled data across studies by conducting random-effects meta-analyses using a Hierarchical Summary Receiving Operating Characteristics model.</p><p><strong>Cost-effectiveness: </strong>An economic model comprising a decision tree (24 weeks) and Markov models to capture lifetime costs and quality-adjusted life-years associated with venous, arterial and mixed aetiology disease in leg ulcer patients. Analyses were conducted from a United Kingdom National Health Service and Personal Social Services perspective. Costs and quality-adjusted life-years were discounted at 3.5% per year. Deterministic and several probabilistic analyses were used to capture uncertainty surrounding a range of optimistic and pessimistic assumptions about the impact of automated tests on health outcomes (ulcer healing and requirement for invasive management of arterial disease).</p><p><strong>Results: </strong>.</p><p><strong>Clinical effectiveness: </strong>From the 116 records retrieved by the electronic searches, we included 24 studies evaluating five devices (BlueDop Vascular Expert, BOSO ABI-System 100, Dopplex Ability, MESI ankle-brachial pressure index MD and WatchBP Office ABI). Two studies assessing people with leg ulcers found that automated devices often gave higher ankle-brachial pressure index readings than ma","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 37","pages":"1-158"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11367298/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142054175","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Image directed redesign of bladder cancer treatment pathways: the BladderPath RCT.","authors":"Nicholas James, Sarah Pirrie, Wenyu Liu, James Catto, Kieran Jefferson, Prashant Patel, Ana Hughes, Ann Pope, Veronica Nanton, Harriet P Mintz, Allen Knight, Jean Gallagher, Richard T Bryan","doi":"10.3310/DEHT5407","DOIUrl":"10.3310/DEHT5407","url":null,"abstract":"<p><strong>Background: </strong>Transurethral resection of bladder tumour has been the mainstay of bladder cancer staging for > 60 years. Staging inaccuracies are commonplace, leading to delayed treatment of muscle-invasive bladder cancer. Multiparametric magnetic resonance imaging offers rapid, accurate and non-invasive staging of muscle-invasive bladder cancer, potentially reducing delays to radical treatment.</p><p><strong>Objectives: </strong>To assess the feasibility and efficacy of the introducing multiparametric magnetic resonance imaging ahead of transurethral resection of bladder tumour in the staging of suspected muscle-invasive bladder cancer.</p><p><strong>Design: </strong>Open-label, multistage randomised controlled study in three parts: feasibility, intermediate and final clinical stages. The COVID pandemic prevented completion of the final stage.</p><p><strong>Setting: </strong>Fifteen UK hospitals.</p><p><strong>Participants: </strong>Newly diagnosed bladder cancer patients of age ≥ 18 years.</p><p><strong>Interventions: </strong>Participants were randomised to Pathway 1 or 2 following visual assessment of the suspicion of non-muscle-invasive bladder cancer or muscle-invasive bladder cancer at the time of outpatient cystoscopy, based upon a 5-point Likert scale: Likert 1-2 tumours considered probable non-muscle-invasive bladder cancer; Likert 3-5 possible muscle-invasive bladder cancer. In Pathway 1, all participants underwent transurethral resection of bladder tumour. In Pathway 2, probable non-muscle-invasive bladder cancer participants underwent transurethral resection of bladder tumour, and possible muscle-invasive bladder cancer participants underwent initial multiparametric magnetic resonance imaging. Subsequent therapy was determined by the treating team and could include transurethral resection of bladder tumour.</p><p><strong>Main outcome measures: </strong>Feasibility stage: proportion with possible muscle-invasive bladder cancer randomised to Pathway 2 which correctly followed the protocol. Intermediate stage: time to correct treatment for muscle-invasive bladder cancer.</p><p><strong>Results: </strong>Between 31 May 2018 and 31 December 2021, of 638 patients approached, 143 participants were randomised; 52.1% were deemed as possible muscle-invasive bladder cancer and 47.9% probable non-muscle-invasive bladder cancer. Feasibility stage: 36/39 [92% (95% confidence interval 79 to 98%)] muscle-invasive bladder cancer participants followed the correct treatment by pathway. Intermediate stage: median time to correct treatment was 98 (95% confidence interval 72 to 125) days for Pathway 1 versus 53 (95% confidence interval 20 to 89) days for Pathway 2 [hazard ratio 2.9 (95% confidence interval 1.0 to 8.1)], <i>p</i> = 0.040. Median time to correct treatment for all participants was 37 days for Pathway 1 and 25 days for Pathway 2 [hazard ratio 1.4 (95% confidence interval 0.9 to 2.0)].</p><p><strong>Limitations: </strong>For pa","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 42","pages":"1-65"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11403381/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142153873","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Community-based complex interventions to sustain independence in older people, stratified by frailty: a systematic review and network meta-analysis.","authors":"Thomas Frederick Crocker, Natalie Lam, Joie Ensor, Magda Jordão, Ram Bajpai, Matthew Bond, Anne Forster, Richard D Riley, Deirdre Andre, Caroline Brundle, Alison Ellwood, John Green, Matthew Hale, Jessica Morgan, Eleftheria Patetsini, Matthew Prescott, Ridha Ramiz, Oliver Todd, Rebecca Walford, John Gladman, Andrew Clegg","doi":"10.3310/HNRP2514","DOIUrl":"10.3310/HNRP2514","url":null,"abstract":"<p><strong>Background: </strong>Sustaining independence is important for older people, but there is insufficient guidance about which community health and care services to implement.</p><p><strong>Objectives: </strong>To synthesise evidence of the effectiveness of community services to sustain independence for older people grouped according to their intervention components, and to examine if frailty moderates the effect.</p><p><strong>Review design: </strong>Systematic review and network meta-analysis.</p><p><strong>Eligibility criteria: </strong>Studies: Randomised controlled trials or cluster-randomised controlled trials. Participants: Older people (mean age 65+) living at home. Interventions: community-based complex interventions for sustaining independence. Comparators: usual care, placebo or another complex intervention.</p><p><strong>Main outcomes: </strong>Living at home, instrumental activities of daily living, personal activities of daily living, care-home placement and service/economic outcomes at 1 year.</p><p><strong>Data sources: </strong>We searched MEDLINE (1946-), Embase (1947-), CINAHL (1972-), PsycINFO (1806-), CENTRAL and trial registries from inception to August 2021, without restrictions, and scanned reference lists.</p><p><strong>Review methods: </strong>Interventions were coded, summarised and grouped. Study populations were classified by frailty. A random-effects network meta-analysis was used. We assessed trial-result risk of bias (Cochrane RoB 2), network meta-analysis inconsistency and certainty of evidence (Grading of Recommendations Assessment, Development and Evaluation for network meta-analysis).</p><p><strong>Results: </strong>We included 129 studies (74,946 participants). Nineteen intervention components, including 'multifactorial-action' (multidomain assessment and management/individualised care planning), were identified in 63 combinations. The following results were of low certainty unless otherwise stated. For living at home, compared to no intervention/placebo, evidence favoured: multifactorial-action and review with medication-review (odds ratio 1.22, 95% confidence interval 0.93 to 1.59; moderate certainty) multifactorial-action with medication-review (odds ratio 2.55, 95% confidence interval 0.61 to 10.60) cognitive training, medication-review, nutrition and exercise (odds ratio 1.93, 95% confidence interval 0.79 to 4.77) and activities of daily living training, nutrition and exercise (odds ratio 1.79, 95% confidence interval 0.67 to 4.76). Four intervention combinations may reduce living at home. For instrumental activities of daily living, evidence favoured multifactorial-action and review with medication-review (standardised mean difference 0.11, 95% confidence interval 0.00 to 0.21; moderate certainty). Two interventions may reduce instrumental activities of daily living. For personal activities of daily living, evidence favoured exercise, multifactorial-action and review with medication-review and sel","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 48","pages":"1-194"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11403382/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285842","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development and validation of prediction models for fetal growth restriction and birthweight: an individual participant data meta-analysis.","authors":"John Allotey, Lucinda Archer, Dyuti Coomar, Kym Ie Snell, Melanie Smuk, Lucy Oakey, Sadia Haqnawaz, Ana Pilar Betrán, Lucy C Chappell, Wessel Ganzevoort, Sanne Gordijn, Asma Khalil, Ben W Mol, Rachel K Morris, Jenny Myers, Aris T Papageorghiou, Basky Thilaganathan, Fabricio Da Silva Costa, Fabio Facchinetti, Arri Coomarasamy, Akihide Ohkuchi, Anne Eskild, Javier Arenas Ramírez, Alberto Galindo, Ignacio Herraiz, Federico Prefumo, Shigeru Saito, Line Sletner, Jose Guilherme Cecatti, Rinat Gabbay-Benziv, Francois Goffinet, Ahmet A Baschat, Renato T Souza, Fionnuala Mone, Diane Farrar, Seppo Heinonen, Kjell Å Salvesen, Luc Jm Smits, Sohinee Bhattacharya, Chie Nagata, Satoru Takeda, Marleen Mhj van Gelder, Dewi Anggraini, SeonAe Yeo, Jane West, Javier Zamora, Hema Mistry, Richard D Riley, Shakila Thangaratinam","doi":"10.3310/DABW4814","DOIUrl":"10.3310/DABW4814","url":null,"abstract":"<p><strong>Background: </strong>Fetal growth restriction is associated with perinatal morbidity and mortality. Early identification of women having at-risk fetuses can reduce perinatal adverse outcomes.</p><p><strong>Objectives: </strong>To assess the predictive performance of existing models predicting fetal growth restriction and birthweight, and if needed, to develop and validate new multivariable models using individual participant data.</p><p><strong>Design: </strong>Individual participant data meta-analyses of cohorts in International Prediction of Pregnancy Complications network, decision curve analysis and health economics analysis.</p><p><strong>Participants: </strong>Pregnant women at booking. External validation of existing models (9 cohorts, 441,415 pregnancies); International Prediction of Pregnancy Complications model development and validation (4 cohorts, 237,228 pregnancies).</p><p><strong>Predictors: </strong>Maternal clinical characteristics, biochemical and ultrasound markers.</p><p><strong>Primary outcomes: </strong>fetal growth restriction defined as birthweight <10th centile adjusted for gestational age and with stillbirth, neonatal death or delivery before 32 weeks' gestation birthweight.</p><p><strong>Analysis: </strong>First, we externally validated existing models using individual participant data meta-analysis. If needed, we developed and validated new International Prediction of Pregnancy Complications models using random-intercept regression models with backward elimination for variable selection and undertook internal-external cross-validation. We estimated the study-specific performance (<i>c</i>-statistic, calibration slope, calibration-in-the-large) for each model and pooled using random-effects meta-analysis. Heterogeneity was quantified using τ<sup>2</sup> and 95% prediction intervals. We assessed the clinical utility of the fetal growth restriction model using decision curve analysis, and health economics analysis based on National Institute for Health and Care Excellence 2008 model.</p><p><strong>Results: </strong>Of the 119 published models, one birthweight model (Poon) could be validated. None reported fetal growth restriction using our definition. Across all cohorts, the Poon model had good summary calibration slope of 0.93 (95% confidence interval 0.90 to 0.96) with slight overfitting, and underpredicted birthweight by 90.4 g on average (95% confidence interval 37.9 g to 142.9 g). The newly developed International Prediction of Pregnancy Complications-fetal growth restriction model included maternal age, height, parity, smoking status, ethnicity, and any history of hypertension, pre-eclampsia, previous stillbirth or small for gestational age baby and gestational age at delivery. This allowed predictions conditional on a range of assumed gestational ages at delivery. The pooled apparent <i>c</i>-statistic and calibration were 0.96 (95% confidence interval 0.51 to 1.0), and 0.95 (95% confidence interval 0.67","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 47","pages":"1-119"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11404361/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285841","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Lenvatinib plus pembrolizumab for untreated advanced renal cell carcinoma: a systematic review and cost-effectiveness analysis.","authors":"Nigel Fleeman, Rachel Houten, Sarah Nevitt, James Mahon, Sophie Beale, Angela Boland, Janette Greenhalgh, Katherine Edwards, Michelle Maden, Devarshi Bhattacharyya, Marty Chaplin, Joanne McEntee, Shien Chow, Tom Waddell","doi":"10.3310/TRRM4238","DOIUrl":"10.3310/TRRM4238","url":null,"abstract":"<p><strong>Background: </strong>Renal cell carcinoma is the most common type of kidney cancer, comprising approximately 85% of all renal malignancies. Patients with advanced renal cell carcinoma are the focus of this National Institute for Health and Care Excellence multiple technology appraisal. A patient's risk of disease progression depends on a number of prognostic risk factors; patients are categorised as having intermediate/poor risk or favourable risk of disease progression.</p><p><strong>Objectives: </strong>The objectives of this multiple technology appraisal were to appraise the clinical effectiveness and cost-effectiveness of lenvatinib plus pembrolizumab versus relevant comparators listed in the final scope issued by the National Institute for Health and Care Excellence: sunitinib, pazopanib, tivozanib, cabozantinib and nivolumab plus ipilimumab.</p><p><strong>Methods: </strong>The assessment group carried out clinical and economic systematic reviews and assessed the clinical and cost-effectiveness evidence submitted by Eisai, Hatfield, Hertfordshire, UK (the manufacturer of lenvatinib) and Merck Sharp & Dohme, Whitehouse Station, NJ, USA (the manufacturer of pembrolizumab). The assessment group carried out fixed-effects network meta-analyses using a Bayesian framework to generate evidence for clinical effectiveness. As convergence issues occurred due to sparse data, random-effects network meta-analysis results were unusable. The assessment group did not develop a de novo economic model, but instead modified the partitioned survival model provided by Merck Sharp & Dohme.</p><p><strong>Results: </strong>The assessment group clinical systematic review identified one relevant randomised controlled trial (CLEAR trial). The CLEAR trial is a good-quality, phase III, multicentre, open-label trial that provided evidence for the efficacy and safety of lenvatinib plus pembrolizumab compared with sunitinib. The assessment group progression-free survival network meta-analysis results for all three risk groups should not be used to infer any statistically significant difference (or lack of statistically significant difference) for any of the treatment comparisons owing to within-trial proportional hazards violations or uncertainty regarding the validity of the proportional hazards assumption. The assessment group overall survival network meta-analysis results for the intermediate-/poor-risk subgroup suggested that there was a numerical, but not statistically significant, improvement in the overall survival for patients treated with lenvatinib plus pembrolizumab compared with patients treated with cabozantinib or nivolumab plus ipilimumab. Because of within-trial proportional hazards violations or uncertainty regarding the validity of the proportional hazards assumption, the assessment group overall survival network meta-analysis results for the favourable-risk subgroup and the all-risk population should not be used to infer any statistically signi","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 49","pages":"1-190"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11404358/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285843","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Reducing self-harm in adolescents: the RISA-IPD individual patient data meta-analysis and systematic review.","authors":"David Cottrell, Alex Wright-Hughes, Amanda Farrin, Rebecca Walwyn, Faraz Mughal, Alex Truscott, Emma Diggins, Donna Irving, Peter Fonagy, Dennis Ougrin, Daniel Stahl, Judy Wright","doi":"10.3310/GTNT6331","DOIUrl":"https://doi.org/10.3310/GTNT6331","url":null,"abstract":"<p><strong>Background: </strong>Self-harm is common in adolescents and a major public health concern. Evidence for effective interventions is lacking. An individual patient data meta-analysis has the potential to provide more reliable estimates of the effects of therapeutic interventions for self-harm than conventional meta-analyses, to explore which treatments are best suited to certain groups.</p><p><strong>Method: </strong>A systematic review and individual patient data meta-analysis of randomised controlled trials of therapeutic interventions to reduce repeat self-harm in adolescents who had a history of self-harm and presented to clinical services. Primary outcome was repetition of self-harm. The methods employed for searches, study screening and selection, and risk of bias assessment are described, with an overview of the outputs of the searching, selection and quality assessment processes. Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidance is followed.</p><p><strong>Results: </strong>We identified a total 39 eligible studies, from 10 countries, where we sought Individual Patient Data (IPD), of which the full sample of participants were eligible in 18 studies and a partial sample of participants were eligible in 21 studies. We obtained IPD from 26 studies of 3448 eligible participants. For our primary outcome, repetition of self-harm, only 6 studies were rated as low risk of bias with 10 rated as high risk (although 2 of these were for secondary outcomes only).</p><p><strong>Conclusions: </strong>Obtaining individual patient data for meta-analyses is possible but very time-consuming, despite clear guidance from funding bodies that researchers should share their data appropriately. More attention needs to be paid to seeking appropriate consent from study participants for (pseudo) anonymised data-sharing and institutions need to collaborate on agreeing template data-sharing agreements. Researchers and funders need to consider issues of research design more carefully. Our next step is to analyse all the data we have collected to see if it will tell us more about how we might prevent repetition of self-harm in young people.</p><p><strong>Funding: </strong>This article presents independent research funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme as award number 17/117/11. A plain language summary of this research article is available on the NIHR Journals Library Website https://doi.org/10.3310/GTNT6331.</p>","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-42"},"PeriodicalIF":3.5,"publicationDate":"2024-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141723589","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Immunogenicity and seroefficacy of pneumococcal conjugate vaccines: a systematic review and network meta-analysis.","authors":"Shuo Feng, Julie McLellan, Nicola Pidduck, Nia Roberts, Julian Pt Higgins, Yoon Choi, Alane Izu, Mark Jit, Shabir A Madhi, Kim Mulholland, Andrew J Pollard, Simon Procter, Beth Temple, Merryn Voysey","doi":"10.3310/YWHA3079","DOIUrl":"10.3310/YWHA3079","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Vaccination of infants with pneumococcal conjugate vaccines is recommended by the World Health Organization. Evidence is mixed regarding the differences in immunogenicity and efficacy of the different pneumococcal vaccines.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;The primary objective was to compare the immunogenicity of pneumococcal conjugate vaccine-10 versus pneumococcal conjugate vaccine-13. The main secondary objective was to compare the seroefficacy of pneumococcal conjugate vaccine-10 versus pneumococcal conjugate vaccine-13.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We searched the Cochrane Library, EMBASE, Global Health, MEDLINE, ClinicalTrials.gov and trialsearch.who.int up to July 2022. Studies were eligible if they directly compared either pneumococcal conjugate vaccine-7, pneumococcal conjugate vaccine-10 or pneumococcal conjugate vaccine-13 in randomised trials of children under 2 years of age, and provided immunogenicity data for at least one time point. Individual participant data were requested and aggregate data used otherwise. Outcomes included the geometric mean ratio of serotype-specific immunoglobulin G and the relative risk of seroinfection. Seroinfection was defined for each individual as a rise in antibody between the post-primary vaccination series time point and the booster dose, evidence of presumed subclinical infection. Each trial was analysed to obtain the log of the ratio of geometric means and its standard error. The relative risk of seroinfection ('seroefficacy') was estimated by comparing the proportion of participants with seroinfection between vaccine groups. The log-geometric mean ratios, log-relative risks and their standard errors constituted the input data for evidence synthesis. For serotypes contained in all three vaccines, evidence could be synthesised using a network meta-analysis. For other serotypes, meta-analysis was used. Results from seroefficacy analyses were incorporated into a mathematical model of pneumococcal transmission dynamics to compare the differential impact of pneumococcal conjugate vaccine-10 and pneumococcal conjugate vaccine-13 introduction on invasive pneumococcal disease cases. The model estimated the impact of vaccine introduction over a 25-year time period and an economic evaluation was conducted.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;In total, 47 studies were eligible from 38 countries. Twenty-eight and 12 studies with data available were included in immunogenicity and seroefficacy analyses, respectively. Geometric mean ratios comparing pneumococcal conjugate vaccine-13 versus pneumococcal conjugate vaccine-10 favoured pneumococcal conjugate vaccine-13 for serotypes 4, 9V and 23F at 1 month after primary vaccination series, with 1.14- to 1.54-fold significantly higher immunoglobulin G responses with pneumococcal conjugate vaccine-13. Risk of seroinfection prior to the time of booster dose was lower for pneumococcal conjugate vaccine-13 for serotype 4, 6B, 9V, 18C an","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 34","pages":"1-109"},"PeriodicalIF":3.5,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11284620/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141751570","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Devices for remote continuous monitoring of people with Parkinson's disease: a systematic review and cost-effectiveness analysis.","authors":"Edward Cox, Ros Wade, Robert Hodgson, Helen Fulbright, Thai Han Phung, Nicholas Meader, Simon Walker, Claire Rothery, Mark Simmonds","doi":"10.3310/YDSL3294","DOIUrl":"10.3310/YDSL3294","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Parkinson's disease is a brain condition causing a progressive loss of co ordination and movement problems. Around 145,500 people have Parkinson's disease in the United Kingdom. Levodopa is the most prescribed treatment for managing motor symptoms in the early stages. Patients should be monitored by a specialist every 6-12 months for disease progression and treatment of adverse effects. Wearable devices may provide a novel approach to management by directly monitoring patients for bradykinesia, dyskinesia, tremor and other symptoms. They are intended to be used alongside clinical judgement.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;To determine the clinical and cost-effectiveness of five devices for monitoring Parkinson's disease: Personal KinetiGraph, Kinesia 360, KinesiaU, PDMonitor and STAT-ON.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We performed systematic reviews of all evidence on the five devices, outcomes included: diagnostic accuracy, impact on decision-making, clinical outcomes, patient and clinician opinions and economic outcomes. We searched MEDLINE and 12 other databases/trial registries to February 2022. Risk of bias was assessed. Narrative synthesis was used to summarise all identified evidence, as the evidence was insufficient for meta-analysis. One included trial provided individual-level data, which was re-analysed. A de novo decision-analytic model was developed to estimate the cost-effectiveness of Personal KinetiGraph and Kinesia 360 compared to standard of care in the UK NHS over a 5-year time horizon. The base-case analysis considered two alternative monitoring strategies: one-time use and routine use of the device.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Fifty-seven studies of Personal KinetiGraph, 15 of STAT-ON, 3 of Kinesia 360, 1 of KinesiaU and 1 of PDMonitor were included. There was some evidence to suggest that Personal KinetiGraph can accurately measure bradykinesia and dyskinesia, leading to treatment modification in some patients, and a possible improvement in clinical outcomes when measured using the Unified Parkinson's Disease Rating Scale. The evidence for STAT-ON suggested it may be of value for diagnosing symptoms, but there is currently no evidence on its clinical impact. The evidence for Kinesia 360, KinesiaU and PDMonitor is insufficient to draw any conclusions on their value in clinical practice. The base-case results for Personal KinetiGraph compared to standard of care for one-time and routine use resulted in incremental cost-effectiveness ratios of £67,856 and £57,877 per quality-adjusted life-year gained, respectively, with a beneficial impact of the Personal KinetiGraph on Unified Parkinson's Disease Rating Scale domains III and IV. The incremental cost-effectiveness ratio results for Kinesia 360 compared to standard of care for one-time and routine use were £38,828 and £67,203 per quality-adjusted life-year gained, respectively.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Limitations: &lt;/strong&gt;The evidence was limited i","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 30","pages":"1-187"},"PeriodicalIF":3.5,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11331379/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141633281","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Emollient application from birth to prevent eczema in high-risk children: the BEEP RCT.","authors":"Lucy E Bradshaw, Laura A Wyatt, Sara J Brown, Rachel H Haines, Alan A Montgomery, Michael R Perkin, Tracey H Sach, Sandra Lawton, Carsten Flohr, Matthew J Ridd, Joanne R Chalmers, Joanne Brooks, Richard Swinden, Eleanor J Mitchell, Stella Tarr, Nicola Jay, Kim S Thomas, Hilary Allen, Michael J Cork, Maeve M Kelleher, Eric L Simpson, Stella T Lartey, Susan Davies-Jones, Robert J Boyle, Hywel C Williams","doi":"10.3310/RHDN9613","DOIUrl":"10.3310/RHDN9613","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Atopic eczema is a common childhood skin problem linked with asthma, food allergy and allergic rhinitis that impairs quality of life.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;To determine whether advising parents to apply daily emollients in the first year can prevent eczema and/or other atopic diseases in high-risk children.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;A United Kingdom, multicentre, pragmatic, two-arm, parallel-group randomised controlled prevention trial with follow-up to 5 years.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Twelve secondary and four primary care centres.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Healthy infants (at least 37 weeks' gestation) at high risk of developing eczema, screened and consented during the third trimester or post delivery.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Infants were randomised (1 : 1) within 21 days of birth to apply emollient (Doublebase Gel®; Dermal Laboratories Ltd, Hitchin, UK or Diprobase Cream®) daily to the whole body (excluding scalp) for the first year, plus standard skin-care advice (emollient group) or standard skin-care advice only (control group). Families were not blinded to allocation.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;Primary outcome was eczema diagnosis in the last year at age 2 years, as defined by the UK Working Party refinement of the Hanifin and Rajka diagnostic criteria, assessed by research nurses blinded to allocation. Secondary outcomes up to age 2 years included other eczema definitions, time to onset and severity of eczema, allergic rhinitis, wheezing, allergic sensitisation, food allergy, safety (skin infections and slippages) and cost-effectiveness.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;One thousand three hundred and ninety-four newborns were randomised between November 2014 and November 2016; 693 emollient and 701 control. Adherence in the emollient group was 88% (466/532), 82% (427/519) and 74% (375/506) at 3, 6 and 12 months. At 2 years, eczema was present in 139/598 (23%) in the emollient group and 150/612 (25%) in controls (adjusted relative risk 0.95, 95% confidence interval 0.78 to 1.16; &lt;i&gt;p&lt;/i&gt; = 0.61 and adjusted risk difference -1.2%, 95% confidence interval -5.9% to 3.6%). Other eczema definitions supported the primary analysis. Food allergy (milk, egg, peanut) was present in 41/547 (7.5%) in the emollient group versus 29/568 (5.1%) in controls (adjusted relative risk 1.47, 95% confidence interval 0.93 to 2.33). Mean number of skin infections per child in the first year was 0.23 (standard deviation 0.68) in the emollient group versus 0.15 (standard deviation 0.46) in controls; adjusted incidence rate ratio 1.55, 95% confidence interval 1.15 to 2.09. The adjusted incremental cost per percentage decrease in risk of eczema at 2 years was £5337 (£7281 unadjusted). No difference between the groups in eczema or other atopic diseases was observed during follow-up to age 5 years via parental questionnaires.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Limitations: &lt;/strong&gt;Two emol","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 29","pages":"1-116"},"PeriodicalIF":3.5,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11261424/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141633280","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Accuracy of glomerular filtration rate estimation using creatinine and cystatin C for identifying and monitoring moderate chronic kidney disease: the eGFR-C study.","authors":"Edmund J Lamb, Jonathan Barratt, Elizabeth A Brettell, Paul Cockwell, R Nei Dalton, Jon J Deeks, Gillian Eaglestone, Tracy Pellatt-Higgins, Philip A Kalra, Kamlesh Khunti, Fiona C Loud, Ryan S Ottridge, Aisling Potter, Ceri Rowe, Katie Scandrett, Alice J Sitch, Paul E Stevens, Claire C Sharpe, Bethany Shinkins, Alison Smith, Andrew J Sutton, Maarten W Taal","doi":"10.3310/HYHN1078","DOIUrl":"10.3310/HYHN1078","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Estimation of glomerular filtration rate using equations based on creatinine is widely used to manage chronic kidney disease. In the UK, the Chronic Kidney Disease Epidemiology Collaboration creatinine equation is recommended. Other published equations using cystatin C, an alternative marker of kidney function, have not gained widespread clinical acceptance. Given higher cost of cystatin C, its clinical utility should be validated before widespread introduction into the NHS.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;Primary objectives were to: (1) compare accuracy of glomerular filtration rate equations at baseline and longitudinally in people with stage 3 chronic kidney disease, and test whether accuracy is affected by ethnicity, diabetes, albuminuria and other characteristics; (2) establish the reference change value for significant glomerular filtration rate changes; (3) model disease progression; and (4) explore comparative cost-effectiveness of kidney disease monitoring strategies.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;A longitudinal, prospective study was designed to: (1) assess accuracy of glomerular filtration rate equations at baseline (&lt;i&gt;n&lt;/i&gt; = 1167) and their ability to detect change over 3 years (&lt;i&gt;n&lt;/i&gt; = 875); (2) model disease progression predictors in 278 individuals who received additional measurements; (3) quantify glomerular filtration rate variability components (&lt;i&gt;n&lt;/i&gt; = 20); and (4) develop a measurement model analysis to compare different monitoring strategy costs (&lt;i&gt;n&lt;/i&gt; = 875).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Primary, secondary and tertiary care.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Adults (≥ 18 years) with stage 3 chronic kidney disease.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration and Modification of Diet in Renal Disease equations.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;Measured glomerular filtration rate was the reference against which estimating equations were compared with accuracy being expressed as P30 (percentage of values within 30% of reference) and progression (variously defined) studied as sensitivity/specificity. A regression model of disease progression was developed and differences for risk factors estimated. Biological variation components were measured and the reference change value calculated. Comparative costs of monitoring with different estimating equations modelled over 10 years were calculated.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Accuracy (P30) of all equations was ≥ 89.5%: the combined creatinine-cystatin equation (94.9%) was superior (&lt;i&gt;p&lt;/i&gt; &lt; 0.001) to other equations. Within each equation, no differences in P30 were seen across categories of age, gender, diabetes, albuminuria, body mass index, kidney function level and ethnicity. All equations showed poor (&lt; 63%) sensitivity for detecting patients showing kidney function decline crossing clinically significant thresholds (e.g.","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 35","pages":"1-169"},"PeriodicalIF":3.5,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11331378/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141758373","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}