Health technology assessment最新文献

{"title":"Cefiderocol for treating severe aerobic Gram-negative bacterial infections: technology evaluation to inform a novel subscription-style payment model.","authors":"Beth Woods, Laetitia Schmitt, Dina Jankovic, Benjamin Kearns, Alison Scope, Shijie Ren, Tushar Srivastava, Chu Chang Ku, Jean Hamilton, Claire Rothery, Laura Bojke, Mark Sculpher, Sue Harnan","doi":"10.3310/YGWR4511","DOIUrl":"10.3310/YGWR4511","url":null,"abstract":"<p><strong>Background: </strong>To limit the use of antimicrobials without disincentivising the development of novel antimicrobials, there is interest in establishing innovative models that fund antimicrobials based on an evaluation of their value as opposed to the volumes used. The aim of this project was to evaluate the population-level health benefit of cefiderocol in the NHS in England, for the treatment of severe aerobic Gram-negative bacterial infections when used within its licensed indications. The results were used to inform the National Institute for Health and Care Excellence guidance in support of commercial discussions regarding contract value between the manufacturer and NHS England.</p><p><strong>Methods: </strong>The health benefit of cefiderocol was first derived for a series of high-value clinical scenarios. These represented uses that were expected to have a significant impact on patients' mortality risks and health-related quality of life. The clinical effectiveness of cefiderocol relative to its comparators was estimated by synthesising evidence on susceptibility of the pathogens of interest to the antimicrobials in a network meta-analysis. Patient-level costs and health outcomes of cefiderocol under various usage scenarios compared with alternative management strategies were quantified using decision modelling. Results were reported as incremental net health effects expressed in quality-adjusted life-years, which were scaled to 20-year population values using infection number forecasts based on data from Public Health England. The outcomes estimated for the high-value clinical scenarios were extrapolated to other expected uses for cefiderocol.</p><p><strong>Results: </strong>Among <i>Enterobacterales</i> isolates with the metallo-beta-lactamase resistance mechanism, the base-case network meta-analysis found that cefiderocol was associated with a lower susceptibility relative to colistin (odds ratio 0.32, 95% credible intervals 0.04 to 2.47), but the result was not statistically significant. The other treatments were also associated with lower susceptibility than colistin, but the results were not statistically significant. In the metallo-beta-lactamase <i>Pseudomonas aeruginosa</i> base-case network meta-analysis, cefiderocol was associated with a lower susceptibility relative to colistin (odds ratio 0.44, 95% credible intervals 0.03 to 3.94), but the result was not statistically significant. The other treatments were associated with no susceptibility. In the base case, patient-level benefit of cefiderocol was between 0.02 and 0.15 quality-adjusted life-years, depending on the site of infection, the pathogen and the usage scenario. There was a high degree of uncertainty surrounding the benefits of cefiderocol across all subgroups. There was substantial uncertainty in the number of infections that are suitable for treatment with cefiderocol, so population-level results are presented for a range of scenarios for the current in","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 28","pages":"1-238"},"PeriodicalIF":3.5,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11229178/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141467523","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":"Benefits and harms of antenatal and newborn screening programmes in health economic assessments: the VALENTIA systematic review and qualitative investigation.","authors":"Oliver Rivero-Arias, May Ee Png, Ashley White, Miaoqing Yang, Sian Taylor-Phillips, Lisa Hinton, Felicity Boardman, Abigail McNiven, Jane Fisher, Baskaran Thilaganathan, Sam Oddie, Anne-Marie Slowther, Svetlana Ratushnyak, Nia Roberts, Jenny Shilton Osborne, Stavros Petrou","doi":"10.3310/PYTK6591","DOIUrl":"10.3310/PYTK6591","url":null,"abstract":"<p><strong>Background: </strong>Health economic assessments are used to determine whether the resources needed to generate net benefit from an antenatal or newborn screening programme, driven by multiple benefits and harms, are justifiable. It is not known what benefits and harms have been adopted by economic evaluations assessing these programmes and whether they omit benefits and harms considered important to relevant stakeholders.</p><p><strong>Objectives: </strong>(1) To identify the benefits and harms adopted by health economic assessments in this area, and to assess how they have been measured and valued; (2) to identify attributes or relevance to stakeholders that ought to be considered in future economic assessments; and (3) to make recommendations about the benefits and harms that should be considered by these studies.</p><p><strong>Design: </strong>Mixed methods combining systematic review and qualitative work.</p><p><strong>Systematic review methods: </strong>We searched the published and grey literature from January 2000 to January 2021 using all major electronic databases. Economic evaluations of an antenatal or newborn screening programme in one or more Organisation for Economic Co-operation and Development countries were considered eligible. Reporting quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards checklist. We identified benefits and harms using an integrative descriptive analysis and constructed a thematic framework.</p><p><strong>Qualitative methods: </strong>We conducted a meta-ethnography of the existing literature on newborn screening experiences, a secondary analysis of existing individual interviews related to antenatal or newborn screening or living with screened-for conditions, and a thematic analysis of primary data collected with stakeholders about their experiences with screening.</p><p><strong>Results: </strong>The literature searches identified 52,244 articles and reports, and 336 unique studies were included. Thematic framework resulted in seven themes: (1) diagnosis of screened for condition, (2) life-years and health status adjustments, (3) treatment, (4) long-term costs, (5) overdiagnosis, (6) pregnancy loss and (7) spillover effects on family members. Diagnosis of screened-for condition (115, 47.5%), life-years and health status adjustments (90, 37.2%) and treatment (88, 36.4%) accounted for most of the benefits and harms evaluating antenatal screening. The same themes accounted for most of the benefits and harms included in studies assessing newborn screening. Long-term costs, overdiagnosis and spillover effects tended to be ignored. The wide-reaching family implications of screening were considered important to stakeholders. We observed good overlap between the thematic framework and the qualitative evidence.</p><p><strong>Limitations: </strong>Dual data extraction within the systematic literature review was not feasible due to the large number of studies included. I","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 25","pages":"1-180"},"PeriodicalIF":3.5,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11228689/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141467520","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":"Comparison of surgical or non-surgical management for non-acute anterior cruciate ligament injury: the ACL SNNAP RCT.","authors":"David J Beard, Loretta Davies, Jonathan A Cook, Jamie Stokes, Jose Leal, Heidi Fletcher, Simon Abram, Katie Chegwin, Akiko Greshon, William Jackson, Nicholas Bottomley, Matthew Dodd, Henry Bourke, Beverly A Shirkey, Arsenio Paez, Sarah E Lamb, Karen L Barker, Michael Phillips, Mark Brown, Vanessa Lythe, Burhan Mirza, Andrew Carr, Paul Monk, Carlos Morgado Areia, Sean O'Leary, Fares Haddad, Chris Wilson, Andrew Price","doi":"10.3310/VDKB6009","DOIUrl":"10.3310/VDKB6009","url":null,"abstract":"<p><strong>Background: </strong>Anterior cruciate ligament injury of the knee is common and leads to decreased activity and risk of secondary osteoarthritis of the knee. Management of patients with a non-acute anterior cruciate ligament injury can be non-surgical (rehabilitation) or surgical (reconstruction). However, insufficient evidence exists to guide treatment.</p><p><strong>Objective(s): </strong>To determine in patients with non-acute anterior cruciate ligament injury and symptoms of instability whether a strategy of surgical management (reconstruction) without prior rehabilitation was more clinically and cost-effective than non-surgical management (rehabilitation).</p><p><strong>Design: </strong>A pragmatic, multicentre, superiority, randomised controlled trial with two-arm parallel groups and 1:1 allocation. Due to the nature of the interventions, no blinding could be carried out.</p><p><strong>Setting: </strong>Twenty-nine NHS orthopaedic units in the United Kingdom.</p><p><strong>Participants: </strong>Participants with a symptomatic (instability) non-acute anterior cruciate ligament-injured knee.</p><p><strong>Interventions: </strong>Patients in the surgical management arm underwent surgical anterior cruciate ligament reconstruction as soon as possible and without any further rehabilitation. Patients in the rehabilitation arm attended physiotherapy sessions and only were listed for reconstructive surgery on continued instability following rehabilitation. Surgery following initial rehabilitation was an expected outcome for many patients and within protocol.</p><p><strong>Main outcome measures: </strong>The primary outcome was the Knee Injury and Osteoarthritis Outcome Score 4 at 18 months post randomisation. Secondary outcomes included return to sport/activity, intervention-related complications, patient satisfaction, expectations of activity, generic health quality of life, knee-specific quality of life and resource usage.</p><p><strong>Results: </strong>Three hundred and sixteen participants were recruited between February 2017 and April 2020 with 156 randomised to surgical management and 160 to rehabilitation. Forty-one per cent (<i>n</i> = 65) of those allocated to rehabilitation underwent subsequent reconstruction within 18 months with 38% (<i>n</i> = 61) completing rehabilitation and not undergoing surgery. Seventy-two per cent (<i>n</i> = 113) of those allocated to surgery underwent reconstruction within 18 months. Follow-up at the primary outcome time point was 78% (<i>n</i> = 248; surgical, <i>n</i> = 128; rehabilitation, <i>n</i> = 120). Both groups improved over time. Adjusted mean Knee Injury and Osteoarthritis Outcome Score 4 scores at 18 months had increased to 73.0 in the surgical arm and to 64.6 in the rehabilitation arm. The adjusted mean difference was 7.9 (95% confidence interval 2.5 to 13.2; <i>p</i> = 0.005) in favour of surgical management. The per-protocol analyses supported the intention-to-treat results, with a","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 27","pages":"1-97"},"PeriodicalIF":3.5,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11228690/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141467522","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":"Evidence of quality of life for hospitalised patients with COVID-19: a scoping review.","authors":"Edward Jd Webb, Natalie King, Daniel Howdon, Enitan D Carrol, Joanne Euden, Philip Howard, Philip Pallmann, Martin J Llewelyn, Emma Thomas-Jones, Bethany Shinkins, Jonathan Sandoe","doi":"10.3310/ATPR4281","DOIUrl":"https://doi.org/10.3310/ATPR4281","url":null,"abstract":"<p><strong>Background: </strong>Information on the quality of life of people hospitalised with COVID-19 is important, both in assessing the burden of disease and the cost-effectiveness of treatments. However, there were potential barriers to collecting such evidence.</p><p><strong>Objective: </strong>To review the existing evidence on quality of life for people hospitalised with COVID-19, with a focus on the amount of evidence available and methods used.</p><p><strong>Design: </strong>A scoping review with systematic searches.</p><p><strong>Results: </strong>A total of 35 papers were selected for data extraction. The most common study type was economic evaluation (<i>N</i> = 13), followed by cross-sectional (<i>N</i> = 10). All economic evaluations used published utility values for other conditions to represent COVID-19 inpatients' quality of life. The most popular quality-of-life survey measure was the Pittsburgh Sleep Quality Index (<i>N</i> = 8). There were 12 studies that used a mental health-related survey and 12 that used a sleep-related survey. Five studies used EQ-5D, but only one collected responses from people in the acute phase of COVID-19. Studies reported a negative impact on quality of life for people hospitalised with COVID-19, although many studies did not include a formal comparison group.</p><p><strong>Limitations: </strong>Although it used systematic searches, this was not a full systematic review.</p><p><strong>Conclusion: </strong>Quality-of-life data were collected from people hospitalised with COVID-19 from relatively early in the pandemic. However, there was a lack of consensus as to what survey measures to use, and few studies used generic health measures. Economic evaluations for COVID-19 treatments did not use utilities collected from people with COVID-19. In future health crises, researchers should be vigilant for opportunities to collect quality-of-life data from hospitalised patients but should try to co-ordinate as well as ensuring generic health measures are used more.</p><p><strong>Funding: </strong>This article presents independent research funded by the National Institute for Health and Care Research (NIHR) <i>Health Technology Assessment</i> programme as award number NIHR132254.</p>","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-23"},"PeriodicalIF":3.6,"publicationDate":"2024-05-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141155181","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":"Clinical and cost-effectiveness of pessary self-management versus clinic-based care for pelvic organ prolapse in women: the TOPSY RCT with process evaluation.","authors":"Carol Bugge, Suzanne Hagen, Andrew Elders, Helen Mason, Kirsteen Goodman, Melanie Dembinsky, Lynn Melone, Catherine Best, Sarkis Manoukian, Lucy Dwyer, Aethele Khunda, Margaret Graham, Wael Agur, Suzanne Breeman, Jane Culverhouse, Angela Forrest, Mark Forrest, Karen Guerrero, Christine Hemming, Doreen McClurg, John Norrie, Ranee Thakar, Rohna Kearney","doi":"10.3310/NWTB5403","DOIUrl":"10.3310/NWTB5403","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Pelvic organ prolapse is common, causes unpleasant symptoms and negatively affects women's quality of life. In the UK, most women with pelvic organ prolapse attend clinics for pessary care.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;To determine the clinical effectiveness and cost-effectiveness of vaginal pessary self-management on prolapse-specific quality of life for women with prolapse compared with clinic-based care; and to assess intervention acceptability and contextual influences on effectiveness, adherence and fidelity.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;A multicentre, parallel-group, superiority randomised controlled trial with a mixed-methods process evaluation.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Women attending UK NHS outpatient pessary services, aged ≥ 18 years, using a pessary of any type/material (except shelf, Gellhorn or Cube) for at least 2 weeks. Exclusions: women with limited manual dexterity, with cognitive deficit (prohibiting consent or self-management), pregnant or non-English-speaking.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Intervention: &lt;/strong&gt;The self-management intervention involved a 30-minute teaching appointment, an information leaflet, a 2-week follow-up telephone call and a local clinic telephone helpline number. Clinic-based care involved routine appointments determined by centres' usual practice.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Allocation: &lt;/strong&gt;Remote web-based application; minimisation was by age, pessary user type and centre.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Blinding: &lt;/strong&gt;Participants, those delivering the intervention and researchers were not blinded to group allocation.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Outcomes: &lt;/strong&gt;The patient-reported primary outcome (measured using the Pelvic Floor Impact Questionnaire-7) was prolapse-specific quality of life, and the cost-effectiveness outcome was incremental cost per quality-adjusted life-year (a specifically developed health Resource Use Questionnaire was used) at 18 months post randomisation. Secondary outcome measures included self-efficacy and complications. Process evaluation data were collected by interview, audio-recording and checklist. Analysis was by intention to treat.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Three hundred and forty women were randomised (self-management, &lt;i&gt;n&lt;/i&gt; = 169; clinic-based care, &lt;i&gt;n&lt;/i&gt; = 171). At 18 months post randomisation, 291 questionnaires with valid primary outcome data were available (self-management, &lt;i&gt;n&lt;/i&gt; = 139; clinic-based care, &lt;i&gt;n&lt;/i&gt; = 152). Baseline economic analysis was based on 264 participants (self-management, &lt;i&gt;n&lt;/i&gt; = 125; clinic-based care, &lt;i&gt;n&lt;/i&gt; = 139) with valid quality of life and resource use data. Self-management was an acceptable intervention. There was no group difference in prolapse-specific quality of life at 18 months (adjusted mean difference -0.03, 95% confidence interval -9.32 to 9.25). There was fidelity to intervention delivery. Self-management was cost-effective at a willingness-to-pay threshold of £20,000 per quality-adjusted life-year","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 23","pages":"1-121"},"PeriodicalIF":3.5,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11145464/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141070849","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":"Doppler ultrasound surveillance of recently formed haemodialysis arteriovenous fistula: the SONAR observational cohort study.","authors":"James Richards, Dominic Summers, Anna Sidders, Elisa Allen, Mohammed Ayaz Hossain, Subhankar Paul, Matthew Slater, Matthew Bartlett, Regin Lagaac, Emma Laing, Valerie Hopkins, Chloe Fitzpatrick-Creamer, Cara Hudson, Joseph Parsons, Samuel Turner, Andrew Tambyraja, Subash Somalanka, James Hunter, Sam Dutta, Neil Hoye, Sarah Lawman, Tracey Salter, Mohammed Farid Aslam, Atul Bagul, Rajesh Sivaprakasam, George E Smith, Helen L Thomas, Zia Moinuddin, Simon R Knight, Nicholas Barnett, Reza Motallebzadeh, Gavin J Pettigrew","doi":"10.3310/YTBT4172","DOIUrl":"10.3310/YTBT4172","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Arteriovenous fistulas are considered the best option for haemodialysis provision, but as many as 30% fail to mature or suffer early failure.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To assess the feasibility of performing a randomised controlled trial that examines whether, by informing early and effective salvage intervention of fistulas that would otherwise fail, Doppler ultrasound surveillance of developing arteriovenous fistulas improves longer-term arteriovenous fistula patency.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;A prospective multicentre observational cohort study (the 'SONAR' study).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Seventeen haemodialysis centres in the UK.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Consenting adults with end-stage renal disease who were scheduled to have an arteriovenous fistula created.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Intervention: &lt;/strong&gt;Participants underwent Doppler ultrasound surveillance of their arteriovenous fistulas at 2, 4, 6 and 10 weeks after creation, with clinical teams blinded to the ultrasound surveillance findings.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;Fistula maturation at week 10 defined according to ultrasound surveillance parameters of representative venous diameter and blood flow (wrist arteriovenous fistulas: ≥ 4 mm and &gt; 400 ml/minute; elbow arteriovenous fistulas: ≥ 5 mm and &gt; 500 ml/minute). Mixed multivariable logistic regression modelling of the early ultrasound scan data was used to predict arteriovenous fistula non-maturation by 10 weeks and fistula failure at 6 months.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;A total of 333 arteriovenous fistulas were created during the study window (47.7% wrist, 52.3% elbow). By 2 weeks, 37 (11.1%) arteriovenous fistulas had failed (thrombosed), but by 10 weeks, 219 of 333 (65.8%) of created arteriovenous fistulas had reached maturity (60.4% wrist, 67.2% elbow). Persistently lower flow rates and venous diameters were observed in those fistulas that did not mature. Models for arteriovenous fistulas' non-maturation could be optimally constructed using the week 4 scan data, with fistula venous diameter and flow rate the most significant variables in explaining wrist fistula maturity failure (positive predictive value 60.6%, 95% confidence interval 43.9% to 77.3%), whereas resistance index and flow rate were most significant for elbow arteriovenous fistulas (positive predictive value 66.7%, 95% confidence interval 48.9% to 84.4%). In contrast to non-maturation, both models predicted fistula maturation much more reliably [negative predictive values of 95.4% (95% confidence interval 91.0% to 99.8%) and 95.6% (95% confidence interval 91.8% to 99.4%) for wrist and elbow, respectively]. Additional follow-up and modelling on a subset (&lt;i&gt;n&lt;/i&gt; = 192) of the original SONAR cohort (the SONAR-12M study) revealed the rates of primary, assisted primary and secondary patency arteriovenous fistulas at 6 months were 76.5, 80.7 and 83.3, respectively. Fistula vein size, flow r","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 24","pages":"1-54"},"PeriodicalIF":3.5,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11145465/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141070876","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":"Advice only versus advice and a physiotherapy programme for acute traumatic anterior shoulder dislocation: the ARTISAN RCT.","authors":"Rebecca Kearney, David Ellard, Helen Parsons, Aminul Haque, James Mason, Henry Nwankwo, Helen Bradley, Steve Drew, Chetan Modi, Howard Bush, David Torgerson, Martin Underwood","doi":"10.3310/CMYW9226","DOIUrl":"10.3310/CMYW9226","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;The extra benefit of a programme of physiotherapy in addition to advice alone, following first-time traumatic shoulder dislocation, is uncertain. We compared the clinical and cost-effectiveness of a single session of advice with a single session of advice and a programme of physiotherapy.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;The primary objective was to quantify and draw inferences about observed differences in the Oxford Shoulder Instability Score between the trial treatment groups 6 months post randomisation, in adults with a first-time traumatic shoulder dislocation.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;A pragmatic, multicentre, superiority, randomised controlled trial with embedded qualitative study.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Forty-one hospitals in the UK NHS.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Adults with a radiologically confirmed first-time traumatic anterior shoulder dislocation, being managed non-operatively. People with neurovascular complications or bilateral dislocations, and those unable to adhere to trial procedures or unable to attend physiotherapy within 6 weeks of injury, or who had previously been randomised, were excluded.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;All participants received the same initial shoulder examination followed by advice to aid self-management, lasting up to 1 hour and administered by a physiotherapist (control). Participants randomised to receive an additional programme of physiotherapy were offered sessions lasting for up to 30 minutes, over a maximum duration of 4 months from the date of randomisation (intervention).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;The primary outcome measure was the Oxford Shoulder Instability Score. This is a self-completed outcome measure containing 12 questions (0-4 points each), with possible scores from 0 (worst function) to 48 (best function). Measurements were collected at 6 weeks, 3 months, 6 months and 12 months by postal questionnaire; 6 months was the primary outcome time point. The primary health outcome for economic evaluation was the quality-adjusted life-year, in accordance with National Institute of Health and Care Excellence guidelines.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Between 14 November 2018 and 14 March 2022, 482 participants were randomised to advice (&lt;i&gt;n&lt;/i&gt; = 240) or advice and a programme of physiotherapy (&lt;i&gt;n&lt;/i&gt; = 242). Participants were 34% female, with a mean age of 45 years, and treatment arms were balanced at baseline. There was not a statistically significant difference in the primary outcome between advice only and advice plus a programme of physiotherapy at 6 months for the primary intention-to-treat adjusted analysis (favours physiotherapy: 1.5, 95% confidence interval -0.3 to 3.5) or at earlier 3-month and 6-week time points on the Oxford Shoulder Instability Score (0-48; higher scores indicate better function). The probability of physiotherapy being cost-effective at a willingness-to-pay threshold of £30,0","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 22","pages":"1-94"},"PeriodicalIF":3.5,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11089447/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140851355","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":"Establishing a research partnership to investigate functional loss and rehabilitation towards the end of life.","authors":"Matthew Maddocks, Lisa Jane Brighton, Louise Connell, Alison Cowley, Barry Laird, Guy Peryer, Carmine Petrasso, Lucy Ziegler, Rowan Harwood","doi":"10.3310/PTHC7598","DOIUrl":"10.3310/PTHC7598","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Functional loss, the inability to perform necessary or desired tasks, is a common consequence of life-limiting illnesses and associated symptoms (pain, fatigue, breathlessness, etc.) and causes suffering for patients and families. Rehabilitation, a set of interventions designed to address functional loss, is recognised as essential within palliative care, as it can improve quality of life and reduce care costs. However, not everyone has equal access to rehabilitation. Despite limited life expectancy or uncertain ability to benefit from interventions, palliative rehabilitation services are often absent. This is partly due to a lack of high-quality research around optimal models of rehabilitation. Research in this area is methodologically challenging and requires multidisciplinary and cross-speciality collaboration.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Aim and objectives: &lt;/strong&gt;We aimed to establish and grow a United Kingdom research partnership across diverse areas, commencing with partners from Edinburgh, East Anglia, Lancashire, Leeds, London and Nottingham, around the topic area of functional loss and rehabilitation in palliative and end-of-life care. The objectives were to (1) develop a multidisciplinary, cross-speciality research partnership, (2) generate high-priority unanswered research questions with stakeholders, (3) co-design and submit high-quality competitive research proposals, including (4) sharing topic and methodological expertise, and (5) to build capacity and capability to deliver nationally generalisable studies.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Activities: &lt;/strong&gt;The partnership was established with professionals from across England and Scotland with complementary areas of expertise including complex palliative and geriatric research, physiotherapy, nursing, palliative medicine and psychology. Research questions were generated through a modified version of the Child Health and Nutrition Research Initiative, which allowed for the collation and refinement of research questions relating to functional loss and rehabilitation towards the end of life. Partnership members were supported through a series of workshops to transform research ideas into proposals for submission to stage one calls by the National Institute for Health and Care Research. The partnership not only supported students, clinicians and public members with training opportunities but also supported clinicians in securing protected time from clinical duties to allow them to focus on developing local research initiatives.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Reflections: &lt;/strong&gt;Through our partnership we established a network that offered researchers, clinicians, students and public members the chance to develop novel skills and explore opportunities for personal and professional development around the topic area of functional loss and rehabilitation in palliative and end-of-life care. The partnership was crucial to foster collaboration and facilitate exchange of ideas, knowledge and experience","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-12"},"PeriodicalIF":3.6,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11070894/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140854536","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":"A randomised, double blind, placebo-controlled trial of a two-week course of dexamethasone for adult patients with a symptomatic Chronic Subdural Haematoma (Dex-CSDH trial).","authors":"Peter J Hutchinson, Ellie Edlmann, John G Hanrahan, Diederik Bulters, Ardalan Zolnourian, Patrick Holton, Nigel Suttner, Kevin Agyemang, Simon Thomson, Ian A Anderson, Yahia Al-Tamimi, Duncan Henderson, Peter Whitfield, Monica Gherle, Paul M Brennan, Annabel Allison, Eric P Thelin, Silvia Tarantino, Beatrice Pantaleo, Karen Caldwell, Carol Davis-Wilkie, Harry Mee, Elizabeth A Warburton, Garry Barton, Aswin Chari, Hani J Marcus, Sarah Pyne, Andrew T King, Antonio Belli, Phyo K Myint, Ian Wilkinson, Thomas Santarius, Carole Turner, Simon Bond, Angelos G Kolias","doi":"10.3310/XWZN4832","DOIUrl":"10.3310/XWZN4832","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Chronic subdural haematoma is a collection of 'old blood' and its breakdown products in the subdural space and predominantly affects older people. Surgical evacuation remains the mainstay in the management of symptomatic cases.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;The Dex-CSDH (DEXamethasone in Chronic SubDural Haematoma) randomised trial investigated the clinical effectiveness and cost-effectiveness of dexamethasone in patients with a symptomatic chronic subdural haematoma.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;This was a parallel, superiority, multicentre, pragmatic, randomised controlled trial. Assigned treatment was administered in a double-blind fashion. Outcome assessors were also blinded to treatment allocation.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Neurosurgical units in the UK.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Eligible participants included adults (aged ≥ 18 years) admitted to a neurosurgical unit with a symptomatic chronic subdural haematoma confirmed on cranial imaging.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Participants were randomly assigned in a 1 : 1 allocation to a 2-week tapering course of dexamethasone or placebo alongside standard care.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;The primary outcome was the Modified Rankin Scale score at 6 months dichotomised to a favourable (score of 0-3) or an unfavourable (score of 4-6) outcome. Secondary outcomes included the Modified Rankin Scale score at discharge and 3 months; number of chronic subdural haematoma-related surgical interventions undertaken during the index and subsequent admissions; Barthel Index and EuroQol 5-Dimension 5-Level utility index score reported at discharge, 3 months and 6 months; Glasgow Coma Scale score reported at discharge and 6 months; mortality at 30 days and 6 months; length of stay; discharge destination; and adverse events. An economic evaluation was also undertaken, during which the net monetary benefit was estimated at a willingness-to-pay threshold of £20,000 per quality-adjusted life-year.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;A total of 748 patients were included after randomisation: 375 were assigned to dexamethasone and 373 were assigned to placebo. The mean age of the patients was 74 years and 94% underwent evacuation of their chronic subdural haematoma during the trial period. A total of 680 patients (91%) had 6-month primary outcome data available for analysis: 339 in the placebo arm and 341 in the dexamethasone arm. On a modified intention-to-treat analysis of the full study population, there was an absolute reduction in the proportion of favourable outcomes of 6.4% (95% confidence interval 11.4% to 1.4%; &lt;i&gt;p&lt;/i&gt; = 0.01) in the dexamethasone arm compared with the control arm at 6 months. At 3 months, the between-group difference was also in favour of placebo (-8.2%, 95% confidence interval -13.3% to -3.1%). Serious adverse events occurred in 60 out of 375 (16.0%) in the dexamethasone arm and 24 out of 373 (6.4%) in the placeb","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 12","pages":"1-122"},"PeriodicalIF":3.5,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11017629/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140174464","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":"Effectiveness of septoplasty compared to medical management in adults with obstruction associated with a deviated nasal septum: the NAIROS RCT.","authors":"Sean Carrie, Tony Fouweather, Tara Homer, James O'Hara, Nikki Rousseau, Leila Rooshenas, Alison Bray, Deborah D Stocken, Laura Ternent, Katherine Rennie, Emma Clark, Nichola Waugh, Alison J Steel, Jemima Dooley, Michael Drinnan, David Hamilton, Kelly Lloyd, Yemi Oluboyede, Caroline Wilson, Quentin Gardiner, Naveed Kara, Sadie Khwaja, Samuel Chee Leong, Sangeeta Maini, Jillian Morrison, Paul Nix, Janet A Wilson, M Dawn Teare","doi":"10.3310/MVFR4028","DOIUrl":"10.3310/MVFR4028","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;The indications for septoplasty are practice-based, rather than evidence-based. In addition, internationally accepted guidelines for the management of nasal obstruction associated with nasal septal deviation are lacking.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;The objective was to determine the clinical effectiveness and cost-effectiveness of septoplasty, with or without turbinate reduction, compared with medical management, in the management of nasal obstruction associated with a deviated nasal septum.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;This was a multicentre randomised controlled trial comparing septoplasty, with or without turbinate reduction, with defined medical management; it incorporated a mixed-methods process evaluation and an economic evaluation.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;The trial was set in 17 NHS secondary care hospitals in the UK.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;A total of 378 eligible participants aged &gt; 18 years were recruited.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Participants were randomised on a 1: 1 basis and stratified by baseline severity and gender to either (1) septoplasty, with or without turbinate surgery (&lt;i&gt;n&lt;/i&gt; = 188) or (2) medical management with intranasal steroid spray and saline spray (&lt;i&gt;n&lt;/i&gt; = 190).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;The primary outcome was the Sino-nasal Outcome Test-22 items score at 6 months (patient-reported outcome). The secondary outcomes were as follows: patient-reported outcomes - Nasal Obstruction Symptom Evaluation score at 6 and 12 months, Sino-nasal Outcome Test-22 items subscales at 12 months, Double Ordinal Airway Subjective Scale at 6 and 12 months, the Short Form questionnaire-36 items and costs; objective measurements - peak nasal inspiratory flow and rhinospirometry. The number of adverse events experienced was also recorded. A within-trial economic evaluation from an NHS and Personal Social Services perspective estimated the incremental cost per (1) improvement (of ≥ 9 points) in Sino-nasal Outcome Test-22 items score, (2) adverse event avoided and (3) quality-adjusted life-year gained at 12 months. An economic model estimated the incremental cost per quality-adjusted life-year gained at 24 and 36 months. A mixed-methods process evaluation was undertaken to understand/address recruitment issues and examine the acceptability of trial processes and treatment arms.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;At the 6-month time point, 307 participants provided primary outcome data (septoplasty, &lt;i&gt;n&lt;/i&gt; = 152; medical management, &lt;i&gt;n&lt;/i&gt; = 155). An intention-to-treat analysis revealed a greater and more sustained improvement in the primary outcome measure in the surgical arm. The 6-month mean Sino-nasal Outcome Test-22 items scores were -20.0 points lower (better) for participants randomised to septoplasty than for those randomised to medical management [the score for the septoplasty arm was 19.9 and the score for the medical management arm was","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 10","pages":"1-213"},"PeriodicalIF":3.5,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11017631/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140109862","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}