Health technology assessment最新文献

{"title":"Photobiomodulation in the management of oral mucositis for adult head and neck cancer patients receiving irradiation: the LiTEFORM RCT.","authors":"Michael Nugent,&nbsp;Valerie Bryant,&nbsp;Chrissie Butcher,&nbsp;Holly Fisher,&nbsp;Sean Gill,&nbsp;Rebecca Goranova,&nbsp;Shaun Hiu,&nbsp;Lyndsay Lindley,&nbsp;James O'Hara,&nbsp;Yemi Oluboyede,&nbsp;Joanne Patterson,&nbsp;Tim Rapley,&nbsp;Tomos Robinson,&nbsp;Nikki Rousseau,&nbsp;Vicky Ryan,&nbsp;Ramkumar Shanmugasundaram,&nbsp;Linda Sharp,&nbsp;Ruby Smith Whelan,&nbsp;Deborah D Stocken,&nbsp;Laura Ternent,&nbsp;Janet Wilson,&nbsp;Jenn Walker","doi":"10.3310/UWNB3375","DOIUrl":"https://doi.org/10.3310/UWNB3375","url":null,"abstract":"<p><strong>Background: </strong>Oral mucositis is a debilitating and painful complication of head and neck cancer irradiation that is characterised by inflammation of the mucous membranes, erythema and ulceration. Oral mucositis affects 6000 head and neck cancer patients per year in England and Wales. Current treatments have not proven to be effective. International studies suggest that low-level laser therapy may be an effective treatment.</p><p><strong>Objectives: </strong>To assess the clinical effectiveness and cost-effectiveness of low-level laser therapy in the management of oral mucositis in head and neck cancer irradiation. To identify barriers to and facilitators of implementing low-level laser therapy in routine care.</p><p><strong>Design: </strong>Placebo-controlled, individually randomised, multicentre Phase III superiority trial, with an internal pilot and health economic and qualitative process evaluations. The participants, outcome assessors and therapists were blinded.</p><p><strong>Setting: </strong>Nine NHS head and neck cancer sites in England and Wales.</p><p><strong>Participants: </strong>A total of 87 out of 380 participants were recruited who were aged ≥ 18 years and were undergoing head and neck cancer irradiation with ≥ 60 Gy.</p><p><strong>Intervention: </strong>Random allocation (1 : 1 ratio) to either low-level laser therapy or sham low-level laser therapy three times per week for the duration of irradiation. The diode laser had the following specifications: wavelength 660 nm, power output 75 mW, beam area 1.5 cm², irradiance 50 mW/cm², exposure time 60 seconds and fluence 3 J/cm². There were 20-30 spots per session. Sham low-level laser therapy was delivered in an identical manner.</p><p><strong>Main outcome measure: </strong>The mean Oral Mucositis Weekly Questionnaire-Head and Neck Cancer score at 6 weeks following the start of irradiation. Higher scores indicate a worse outcome.</p><p><strong>Results: </strong>A total of 231 patients were screened and, of these, 87 were randomised (low-level laser therapy arm, <i>n</i> = 44; sham arm, <i>n</i> = 43). The mean age was 59.4 years (standard deviation 8.8 years) and 69 participants (79%) were male. The mean Oral Mucositis Weekly Questionnaire-Head and Neck Cancer score at 6 weeks was 33.2 (standard deviation 10) in the low-level laser therapy arm and 27.4 (standard deviation 13.8) in the sham arm.</p><p><strong>Limitations: </strong>The trial lacked statistical power because it did not meet the recruitment target. Staff and patients willingly participated in the trial and worked hard to make the LiTEFORM trial succeed. However, the task of introducing, embedding and sustaining new low-level laser therapy services into a complex care pathway proved challenging. Sites could deliver low-level laser therapy to only a small number of patients at a time. The administration of low-level laser therapy was viewed as straightforward, but also tim","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9761526/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10859564","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":"Oral nutritional interventions in frail older people who are malnourished or at risk of malnutrition: a systematic review.","authors":"Katie Thomson,&nbsp;Stephen Rice,&nbsp;Oluwatomi Arisa,&nbsp;Eugenie Johnson,&nbsp;Louise Tanner,&nbsp;Christopher Marshall,&nbsp;Tumi Sotire,&nbsp;Catherine Richmond,&nbsp;Hannah O'Keefe,&nbsp;Wael Mohammed,&nbsp;Margot Gosney,&nbsp;Anne Raffle,&nbsp;Barbara Hanratty,&nbsp;Claire T McEvoy,&nbsp;Dawn Craig,&nbsp;Sheena E Ramsay","doi":"10.3310/CCQF1608","DOIUrl":"10.3310/CCQF1608","url":null,"abstract":"<p><strong>Background: </strong>Malnutrition worsens the health of frail older adults. Current treatments for malnutrition may include prescribed oral nutritional supplements, which are multinutrient products containing macronutrients and micronutrients.</p><p><strong>Objective: </strong>To assess the effectiveness and cost-effectiveness of oral nutritional supplements (with or without other dietary interventions) in frail older people who are malnourished or at risk of malnutrition.</p><p><strong>Data sources: </strong>MEDLINE, EMBASE, Cochrane Library, Scopus, CINAHL (Cumulative Index to Nursing and Allied Health Literature) and grey literature were searched from inception to 13 September 2021.</p><p><strong>Review methods: </strong>A systematic review and meta-analysis was conducted to evaluate the effectiveness and cost-effectiveness of oral nutritional supplements in frail older people (aged ≥ 65 years) who are malnourished or at risk of malnutrition (defined as undernutrition as per National Institute for Health and Care Excellence guidelines). Meta-analysis and network meta-analysis were undertaken, where feasible, along with a narrative synthesis. A cost-effectiveness review was reported narratively. A de novo model was developed using effectiveness evidence identified in the systematic review to estimate the cost-effectiveness of oral nutritional supplements.</p><p><strong>Results: </strong>Eleven studies (<i>n</i> = 822 participants) were included in the effectiveness review, six of which were fully or partly funded by industry. Meta-analyses suggested positive effects of oral nutritional supplements compared with standard care for energy intake (kcal) (standardised mean difference 1.02, 95% confidence interval 0.15 to 1.88; very low quality evidence) and poor mobility (mean difference 0.03, <i>p</i> < 0.00001, 95% confidence interval 0.02 to 0.04; very low quality evidence) but no evidence of an effect for body weight (mean difference 1.31, 95% confidence interval -0.05 to 2.66; very low quality evidence) and body mass index (mean difference 0.54, 95% confidence interval -0.03 to 1.11; very low quality evidence). Pooled results for other outcomes were statistically non-significant. There was mixed narrative evidence regarding the effect of oral nutritional supplements on quality of life. Network meta-analysis could be conducted only for body weight and grip strength; there was evidence of an effect for oral nutritional supplements compared with standard care for body weight only. Study quality was mixed; the randomisation method was typically poorly reported. One economic evaluation, in a care home setting, was included. This was a well-conducted study showing that oral nutritional supplements could be cost-effective. Cost-effectiveness analysis suggested that oral nutritional supplements may only be cost-effective for people with lower body mass index (< 21 kg/m²) using cheaper oral nutritional supplements products that req","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9791461/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9153548","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":"Impact of video-assisted thoracoscopic lobectomy versus open lobectomy for lung cancer on recovery assessed using self-reported physical function: VIOLET RCT.","authors":"Eric Lim,&nbsp;Rosie A Harris,&nbsp;Holly E McKeon,&nbsp;Timothy Jp Batchelor,&nbsp;Joel Dunning,&nbsp;Michael Shackcloth,&nbsp;Vladimir Anikin,&nbsp;Babu Naidu,&nbsp;Elizabeth Belcher,&nbsp;Mahmoud Loubani,&nbsp;Vipin Zamvar,&nbsp;Lucy Dabner,&nbsp;Timothy Brush,&nbsp;Elizabeth A Stokes,&nbsp;Sarah Wordsworth,&nbsp;Sangeetha Paramasivan,&nbsp;Alba Realpe,&nbsp;Daisy Elliott,&nbsp;Jane Blazeby,&nbsp;Chris A Rogers","doi":"10.3310/THBQ1793","DOIUrl":"https://doi.org/10.3310/THBQ1793","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Lung cancer is the leading cause of cancer death. Surgery remains the main method of managing early-stage disease. Minimal-access video-assisted thoracoscopic surgery results in less tissue trauma than open surgery; however, it is not known if it improves patient outcomes.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To compare the clinical effectiveness and cost-effectiveness of video-assisted thoracoscopic surgery lobectomy with open surgery for the treatment of lung cancer.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design, setting and participants: &lt;/strong&gt;A multicentre, superiority, parallel-group, randomised controlled trial with blinding of participants (until hospital discharge) and outcome assessors conducted in nine NHS hospitals. Adults referred for lung resection for known or suspected lung cancer, with disease suitable for both surgeries, were eligible. Participants were followed up for 1 year.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Participants were randomised 1 : 1 to video-assisted thoracoscopic surgery lobectomy or open surgery. Video-assisted thoracoscopic surgery used one to four keyhole incisions without rib spreading. Open surgery used a single incision with rib spreading, with or without rib resection.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;The primary outcome was self-reported physical function (using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30) at 5 weeks. Secondary outcomes included upstaging to pathologic node stage 2 disease, time from surgery to hospital discharge, pain in the first 2 days, prolonged pain requiring analgesia at &gt; 5 weeks, adverse health events, uptake of adjuvant treatment, overall and disease-free survival, quality of life (Quality of Life Questionnaire Core 30, Quality of Life Questionnaire Lung Cancer 13 and EQ-5D) at 2 and 5 weeks and 3, 6 and 12 months, and cost-effectiveness.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;A total of 503 patients were randomised between July 2015 and February 2019 (video-assisted thoracoscopic surgery, &lt;i&gt;n&lt;/i&gt; = 247; open surgery, &lt;i&gt;n&lt;/i&gt; = 256). One participant withdrew before surgery. The mean age of patients was 69 years; 249 (49.5%) patients were men and 242 (48.1%) did not have a confirmed diagnosis. Lobectomy was performed in 453 of 502 (90.2%) participants and complete resection was achieved in 429 of 439 (97.7%) participants. Quality of Life Questionnaire Core 30 physical function was better in the video-assisted thoracoscopic surgery group than in the open-surgery group at 5 weeks (video-assisted thoracoscopic surgery, &lt;i&gt;n&lt;/i&gt; = 247; open surgery, &lt;i&gt;n&lt;/i&gt; = 255; mean difference 4.65, 95% confidence interval 1.69 to 7.61; &lt;i&gt;p&lt;/i&gt; = 0.0089). Upstaging from clinical node stage 0 to pathologic node stage 1 and from clinical node stage 0 or 1 to pathologic node stage 2 was similar (&lt;i&gt;p&lt;/i&gt; ≥ 0.50). Pain scores were similar on day 1, but lower in the video-assisted thoracoscopic surgery group on day 2 (mean differe","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9791462/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10491186","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":"Planned delivery for pre-eclampsia between 34 and 37 weeks of gestation: the PHOENIX RCT.","authors":"Lucy C Chappell, Peter Brocklehurst, Marcus Green, Pollyanna Hardy, Rachael Hunter, Alice Beardmore-Gray, Ursula Bowler, Anna Brockbank, Virginia Chiocchia, Alice Cox, Kate Duhig, Jessica Fleminger, Carolyn Gill, Melanie Greenland, Eleanor Hendy, Ann Kennedy, Paul Leeson, Louise Linsell, Fergus P McCarthy, Jamie O'Driscoll, Anna Placzek, Lucilla Poston, Stephen Robson, Pauline Rushby, Jane Sandall, Laura Scholtz, Paul T Seed, Jenie Sparkes, Kayleigh Stanbury, Sue Tohill, Basky Thilaganathan, John Townend, Edmund Juszczak, Neil Marlow, Andrew Shennan","doi":"10.3310/CWWH0622","DOIUrl":"10.3310/CWWH0622","url":null,"abstract":"<p><strong>Background: </strong>In women with late preterm pre-eclampsia (i.e. at 34⁺⁰ to 36⁺⁶ weeks' gestation), the optimal delivery time is unclear because limitation of maternal-fetal disease progression needs to be balanced against infant complications. The aim of this trial was to determine whether or not planned earlier initiation of delivery reduces maternal adverse outcomes without substantial worsening of perinatal or infant outcomes, compared with expectant management, in women with late preterm pre-eclampsia.</p><p><strong>Methods: </strong>We undertook an individually randomised, triple non-masked controlled trial in 46 maternity units across England and Wales, with an embedded health economic evaluation, comparing planned delivery and expectant management (usual care) in women with late preterm pre-eclampsia. The co-primary maternal outcome was a maternal morbidity composite or recorded systolic blood pressure of ≥ 160 mmHg (superiority hypothesis). The co-primary short-term perinatal outcome was a composite of perinatal deaths or neonatal unit admission (non-inferiority hypothesis). Analyses were by intention to treat, with an additional per-protocol analysis for the perinatal outcome. The primary 2-year infant neurodevelopmental outcome was measured using the PARCA-R (Parent Report of Children's Abilities-Revised) composite score. The planned sample size of the trial was 900 women; the trial is now completed. We undertook two linked substudies.</p><p><strong>Results: </strong>Between 29 September 2014 and 10 December 2018, 901 women were recruited; 450 women [448 women (two withdrew consent) and 471 infants] were allocated to planned delivery and 451 women (451 women and 475 infants) were allocated to expectant management. The incidence of the co-primary maternal outcome was significantly lower in the planned delivery group [289 (65%) women] than in the expectant management group [338 (75%) women] (adjusted relative risk 0.86, 95% confidence interval 0.79 to 0.94; <i>p</i> = 0.0005). The incidence of the co-primary perinatal outcome was significantly higher in the planned delivery group [196 (42%) infants] than in the expectant management group [159 (34%) infants] (adjusted relative risk 1.26, 95% confidence interval 1.08 to 1.47; <i>p</i> = 0.0034), but indicators of neonatal morbidity were similar in both groups. At 2-year follow-up, the mean PARCA-R scores were 89.5 points (standard deviation 18.2 points) for the planned delivery group (290 infants) and 91.9 points (standard deviation 18.4 points) for the expectant management group (256 infants), both within the normal developmental range (adjusted mean difference -2.4 points, 95% confidence interval -5.4 to 0.5 points; non-inferiority <i>p</i> = 0.147). Planned delivery was significantly cost-saving (-£2711, 95% confidence interval -£4840 to -£637) compared with expectant management. There were nine serious adverse events in the planned delivery group and","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2022-11-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10068586/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9244116","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":"Increasing comprehensiveness and reducing workload in a systematic review of complex interventions using automated machine learning.","authors":"Olalekan A Uthman, Rachel Court, Jodie Enderby, Lena Al-Khudairy, Chidozie Nduka, Hema Mistry, G J Melendez-Torres, Sian Taylor-Phillips, Aileen Clarke","doi":"10.3310/UDIR6682","DOIUrl":"10.3310/UDIR6682","url":null,"abstract":"<p><strong>Background: </strong>As part of our ongoing systematic review of complex interventions for the primary prevention of cardiovascular diseases, we have developed and evaluated automated machine-learning classifiers for title and abstract screening. The aim was to develop a high-performing algorithm comparable to human screening.</p><p><strong>Methods: </strong>We followed a three-phase process to develop and test an automated machine learning-based classifier for screening potential studies on interventions for primary prevention of cardiovascular disease. We labelled a total of 16,611 articles during the first phase of the project. In the second phase, we used the labelled articles to develop a machine learning-based classifier. After that, we examined the performance of the classifiers in correctly labelling the papers. We evaluated the performance of the five deep-learning models [i.e. parallel convolutional neural network ( CNN ), stacked CNN , parallel-stacked CNN , recurrent neural network ( RNN ) and CNN-RNN]. The models were evaluated using recall, precision and work saved over sampling at no less than 95% recall.</p><p><strong>Results: </strong>We labelled a total of 16,611 articles, of which 676 (4.0%) were tagged as 'relevant' and 15,935 (96%) were tagged as 'irrelevant'. The recall ranged from 51.9% to 96.6%. The precision ranged from 64.6% to 99.1%. The work saved over sampling ranged from 8.9% to as high as 92.1%. The best-performing model was parallel CNN , yielding a 96.4% recall, as well as 99.1% precision, and a potential workload reduction of 89.9%.</p><p><strong>Future work and limitations: </strong>We used words from the title and the abstract only. More work needs to be done to look into possible changes in performance, such as adding features such as full document text. The approach might also not be able to be used for other complex systematic reviews on different topics.</p><p><strong>Conclusion: </strong>Our study shows that machine learning has the potential to significantly aid the labour-intensive screening of abstracts in systematic reviews of complex interventions. Future research should concentrate on enhancing the classifier system and determining how it can be integrated into the systematic review workflow.</p><p><strong>Funding: </strong>This project was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme and will be published in <i>Health Technology Assessment</i>. See the NIHR Journals Library website for further project information.</p>","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2022-11-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10068584/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9599620","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":"SeHCAT (tauroselcholic [75selenium] acid) for the investigation of bile acid diarrhoea in adults: a systematic review and cost-effectiveness analysis","authors":"M. Westwood, I. C. Ramos, N. Armstrong, Edyta Ryczek, H. Penton, M. Holleman, C. Noake, M. Al","doi":"10.3310/jtfo0945","DOIUrl":"https://doi.org/10.3310/jtfo0945","url":null,"abstract":"\u0000 \u0000 Tauroselcholic [75selenium] acid (SeHCAT™) (GE Healthcare, Chicago, IL, USA) is a radiopharmaceutical that may be useful in diagnosing bile acid diarrhoea.\u0000 \u0000 \u0000 \u0000 To assess the clinical effectiveness and cost-effectiveness of SeHCAT for the investigation of adults with chronic unexplained diarrhoea, diarrhoea-predominant irritable bowel syndrome or functional diarrhoea (suspected primary bile acid diarrhoea), and adults with chronic diarrhoea and Crohn’s disease who have not undergone ileal resection (suspected secondary bile acid diarrhoea).\u0000 \u0000 \u0000 \u0000 Sixteen databases were searched to November 2020. The review process included measures to minimise error and bias. Results were summarised by primary or secondary bile acid diarrhoea and study quality was considered. The cost-effectiveness analysis combined a short-term (6-month) decision-analytic model (diagnosis and initial treatment response) and a lifetime Markov model comprising three health states (diarrhoea, no diarrhoea and death), with transitions determined by probabilities of response to treatment. Analyses were conducted from an NHS and Personal Social Services perspective.\u0000 \u0000 \u0000 \u0000 Twenty-four studies were included in this review. Of these, 21 were observational studies, reporting some outcome data for patients treated with bile acid sequestrants, and in which only patients with a positive SeHCAT test were offered bile acid sequestrants. The median rate of response to bile acid sequestrants, among patients with a 7-day SeHCAT retention value of ≤ 15%, was 68% (range 38–86%) (eight studies). The estimated sensitivity of SeHCAT (≤ 15% threshold) to predict positive response to colestyramine was 100% (95% confidence interval 54.1% to 100%) and the specificity estimate was 91.2% (95% confidence interval 76.3% to 98.1%) (one study). The median proportion of treated patients who were intolerant/discontinued bile acid sequestrants was 15% (range 4–27%) (eight studies). There was insufficient information to determine whether or not intolerance varied between colestyramine, colestipol and colesevelam. For both populations, the SeHCAT 15% (i.e. a SeHCAT retention value of ≤ 15%) strategy dominated other strategies or resulted in incremental cost-effectiveness ratios of < £20,000–30,000 per quality-adjusted life-year gained. For the suspected primary bile acid diarrhoea population, SeHCAT 15% was the strategy most likely to be cost-effective: 67% and 73% probability at threshold incremental cost-effectiveness ratios of £20,000 and £30,000 per quality-adjusted life-year gained, respectively. For the Crohn’s disease population, these probabilities were 89% and 92% at £20,000 and £30,000 per quality-adjusted life-year gained, respectively. Cost-effectiveness was mostly led by treatment response. SeHCAT 15% was the strategy with the highest response rate in the majority of scenarios explored.\u0000 \u0000 \u0000 \u0000 There is a lack of evidence linking the use of SeHCAT testing to patient-relevant outcomes. The optimal","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2022-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48649054","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":"Assessing a 12-month course of oral alendronate for adults with avascular necrosis of the hip: MANTIS RCT with internal pilot.","authors":"Sion Glyn-Jones,&nbsp;Muhammad K Javaid,&nbsp;David Beard,&nbsp;Julia Newton,&nbsp;Robert Kerslake,&nbsp;Callum McBryde,&nbsp;Tim Board,&nbsp;Susan J Dutton,&nbsp;Melina Dritsaki,&nbsp;Vikas Khanduja,&nbsp;Magbor Akanni,&nbsp;Shaun Sexton,&nbsp;John Skinner,&nbsp;Nicholas Peckham,&nbsp;Ruth Knight,&nbsp;Ines Rombach,&nbsp;Loretta Davies,&nbsp;Vicki Barber","doi":"10.3310/AVZV0799","DOIUrl":"https://doi.org/10.3310/AVZV0799","url":null,"abstract":"<p><strong>Background: </strong>People with avascular necrosis of the hip have very limited treatment options currently available to stop the progression of this disease; this often results in the need for a hip replacement. There is some weak evidence that a class of drugs called bisphosphonates may delay the course of the disease, and this trial was commissioned and set up to provide robust evidence regarding the use of bisphosphonates in adults aged ≥ 18 years with this condition.</p><p><strong>Objectives: </strong>The aim of the Managing Avascular Necrosis Treatments: an Interventional Study ( MANTIS ) trial was to evaluate the clinical effectiveness and cost-effectiveness of a 12-month course of alendronate in the treatment of avascular necrosis.</p><p><strong>Design: </strong>This was a 66-month, definitive, multisite, two-arm, parallel-group, placebo-controlled, double-blind, randomised controlled trial, with an internal pilot phase.</p><p><strong>Setting: </strong>Eight secondary care NHS hospitals across the UK.</p><p><strong>Participants: </strong>Planned trial size - 280 adult patients with avascular necrosis.</p><p><strong>Intervention: </strong>Participants in the intervention group received 70 mg of alendronate (an oral bisphosphonate) weekly for 12 months.</p><p><strong>Main outcomes: </strong>The main outcomes were Oxford Hip Score at 12 months (short-term outcome) and the time to decision that a hip replacement is required at 36 months (long-term outcome).</p><p><strong>Results: </strong>Twenty-one patients were recruited and randomised to receive either the intervention drug, alendronate, or a placebo-matched tablet.</p><p><strong>Limitations: </strong>This trial was principally limited by low disease prevalence. Other limitations included the late disease stage at which participants were identified and the rapid progression of the disease.</p><p><strong>Future work: </strong>This trial was limited by a low recruitment rate. Avascular necrosis of the hip should be treated as a rare disease. Future trials would need to recruit many more sites and recruit over a longer time period, and, for this reason, a registry may provide a more effective means of collecting data pertaining to this disease.</p><p><strong>Conclusions: </strong>The MANTIS trial was terminated at the end of the pilot phase, because it did not meet its go/no-go criteria. The main issue was a poor recruitment rate, owing to a lower than expected disease prevalence and difficulties in identifying the condition at a sufficiently early stage. Those patients who were identified and screened either were too advanced in their disease progression or were already taking medication. We would not recommend that a short-term interventional study is conducted on this condition until its prevalence, geographic foci and natural history and better understood. The difficulty of acquiring this understanding is likely to be a barrier in most health-care markets. One means of develop","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9638884/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10778558","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":"Defining the optimum strategy for identifying adults and children with coeliac disease: systematic review and economic modelling.","authors":"Martha Mc Elwenspoek, Howard Thom, Athena L Sheppard, Edna Keeney, Rachel O'Donnell, Joni Jackson, Cristina Roadevin, Sarah Dawson, Deborah Lane, Jo Stubbs, Hazel Everitt, Jessica C Watson, Alastair D Hay, Peter Gillett, Gerry Robins, Hayley E Jones, Sue Mallett, Penny F Whiting","doi":"10.3310/ZUCE8371","DOIUrl":"10.3310/ZUCE8371","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Coeliac disease is an autoimmune disorder triggered by ingesting gluten. It affects approximately 1% of the UK population, but only one in three people is thought to have a diagnosis. Untreated coeliac disease may lead to malnutrition, anaemia, osteoporosis and lymphoma.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;The objectives were to define at-risk groups and determine the cost-effectiveness of active case-finding strategies in primary care.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;(1) Systematic review of the accuracy of potential diagnostic indicators for coeliac disease. (2) Routine data analysis to develop prediction models for identification of people who may benefit from testing for coeliac disease. (3) Systematic review of the accuracy of diagnostic tests for coeliac disease. (4) Systematic review of the accuracy of genetic tests for coeliac disease (literature search conducted in April 2021). (5) Online survey to identify diagnostic thresholds for testing, starting treatment and referral for biopsy. (6) Economic modelling to identify the cost-effectiveness of different active case-finding strategies, informed by the findings from previous objectives.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Data sources: &lt;/strong&gt;For the first systematic review, the following databases were searched from 1997 to April 2021: MEDLINE&lt;sup&gt;®&lt;/sup&gt; (National Library of Medicine, Bethesda, MD, USA), Embase&lt;sup&gt;®&lt;/sup&gt; (Elsevier, Amsterdam, the Netherlands), Cochrane Library, Web of Science™ (Clarivate™, Philadelphia, PA, USA), the World Health Organization International Clinical Trials Registry Platform ( WHO ICTRP ) and the National Institutes of Health Clinical Trials database. For the second systematic review, the following databases were searched from January 1990 to August 2020: MEDLINE, Embase, Cochrane Library, Web of Science, Kleijnen Systematic Reviews ( KSR ) Evidence, WHO ICTRP and the National Institutes of Health Clinical Trials database. For prediction model development, Clinical Practice Research Datalink GOLD, Clinical Practice Research Datalink Aurum and a subcohort of the Avon Longitudinal Study of Parents and Children were used; for estimates for the economic models, Clinical Practice Research Datalink Aurum was used.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Review methods: &lt;/strong&gt;For review 1, cohort and case-control studies reporting on a diagnostic indicator in a population with and a population without coeliac disease were eligible. For review 2, diagnostic cohort studies including patients presenting with coeliac disease symptoms who were tested with serological tests for coeliac disease and underwent a duodenal biopsy as reference standard were eligible. In both reviews, risk of bias was assessed using the quality assessment of diagnostic accuracy studies 2 tool. Bivariate random-effects meta-analyses were fitted, in which binomial likelihoods for the numbers of true positives and true negatives were assumed.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;People with dermatitis herpetif","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":null,"pages":null},"PeriodicalIF":3.5,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9638887/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9146153","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":"Photodynamic versus white-light-guided resection of first-diagnosis non-muscle-invasive bladder cancer: PHOTO RCT.","authors":"Rakesh Heer, Rebecca Lewis, Anne Duncan, Steven Penegar, Thenmalar Vadiveloo, Emma Clark, Ge Yu, Paramananthan Mariappan, Joanne Cresswell, John McGrath, James N'Dow, Ghulam Nabi, Hugh Mostafid, John Kelly, Craig Ramsay, Henry Lazarowicz, Angela Allan, Matthew Breckons, Karen Campbell, Louise Campbell, Andy Feber, Alison McDonald, John Norrie, Giovany Orozco-Leal, Stephen Rice, Zafer Tandogdu, Ernest Taylor, Laura Wilson, Luke Vale, Graeme MacLennan, Emma Hall","doi":"10.3310/PLPU1526","DOIUrl":"10.3310/PLPU1526","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Around 7500 people are diagnosed with non-muscle-invasive bladder cancer in the UK annually. Recurrence following transurethral resection of bladder tumour is common, and the intensive monitoring schedule required after initial treatment has associated costs for patients and the NHS. In photodynamic diagnosis, before transurethral resection of bladder tumour, a photosensitiser that is preferentially absorbed by tumour cells is instilled intravesically. Transurethral resection of bladder tumour is then conducted under blue light, causing the photosensitiser to fluoresce. Photodynamic diagnosis-guided transurethral resection of bladder tumour offers better diagnostic accuracy than standard white-light-guided transurethral resection of bladder tumour, potentially reducing the chance of subsequent recurrence.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;The objective was to assess the clinical effectiveness and cost-effectiveness of photodynamic diagnosis-guided transurethral resection of bladder tumour.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;This was a multicentre, pragmatic, open-label, parallel-group, non-masked, superiority randomised controlled trial. Allocation was by remote web-based service, using a 1 : 1 ratio and a minimisation algorithm balanced by centre and sex.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;The setting was 22 NHS hospitals.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Patients aged ≥ 16 years with a suspected first diagnosis of high-risk non-muscle-invasive bladder cancer, no contraindications to photodynamic diagnosis and written informed consent were eligible.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Photodynamic diagnosis-guided transurethral resection of bladder tumour and standard white-light cystoscopy transurethral resection of bladder tumour.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;The primary clinical outcome measure was the time to recurrence from the date of randomisation to the date of pathologically proven first recurrence (or intercurrent bladder cancer death). The primary health economic outcome was the incremental cost per quality-adjusted life-year gained at 3 years.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;We enrolled 538 participants from 22 UK hospitals between 11 November 2014 and 6 February 2018. Of these, 269 were allocated to photodynamic diagnosis and 269 were allocated to white light. A total of 112 participants were excluded from the analysis because of ineligibility (&lt;i&gt;n&lt;/i&gt; = 5), lack of non-muscle-invasive bladder cancer diagnosis following transurethral resection of bladder tumour (&lt;i&gt;n&lt;/i&gt; = 89) or early cystectomy (&lt;i&gt;n&lt;/i&gt; = 18). In total, 209 photodynamic diagnosis and 217 white-light participants were included in the clinical end-point analysis population. All randomised participants were included in the cost-effectiveness analysis. Over a median follow-up period of 21 months for the photodynamic diagnosis group and 22 months for the white-light group, there were 86 recurrences (3-year recurrence-fr","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":null,"pages":null},"PeriodicalIF":3.5,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9639219/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9417072","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":"One-session treatment compared with multisession CBT in children aged 7-16 years with specific phobias: the ASPECT non-inferiority RCT.","authors":"Barry Wright,&nbsp;Lucy Tindall,&nbsp;Alexander J Scott,&nbsp;Ellen Lee,&nbsp;Katie Biggs,&nbsp;Cindy Cooper,&nbsp;Penny Bee,&nbsp;Han-I Wang,&nbsp;Lina Gega,&nbsp;Emily Hayward,&nbsp;Kiera Solaiman,&nbsp;M Dawn Teare,&nbsp;Thompson Davis,&nbsp;Karina Lovell,&nbsp;Jon Wilson,&nbsp;Dean McMillan,&nbsp;Amy Barr,&nbsp;Hannah Edwards,&nbsp;Jennifer Lomas,&nbsp;Chris Turtle,&nbsp;Steve Parrott,&nbsp;Catarina Teige,&nbsp;Tim Chater,&nbsp;Rebecca Hargate,&nbsp;Shehzad Ali,&nbsp;Sarah Parkinson,&nbsp;Simon Gilbody,&nbsp;David Marshall","doi":"10.3310/IBCT0609","DOIUrl":"https://doi.org/10.3310/IBCT0609","url":null,"abstract":"BACKGROUND\u0000Up to 10% of children and young people have a specific phobia that can significantly affect their mental health, development and daily functioning. Cognitive-behavioural therapy-based interventions remain the dominant treatment, but limitations to their provision warrant investigation into low-intensity alternatives. One-session treatment is one such alternative that shares cognitive-behavioural therapy principles but has a shorter treatment period.\u0000\u0000\u0000OBJECTIVE\u0000This research investigated the non-inferiority of one-session treatment to cognitive-behavioural therapy for treating specific phobias in children and young people. The acceptability and cost-effectiveness of one-session treatment were examined.\u0000\u0000\u0000DESIGN\u0000A pragmatic, multicentre, non-inferiority randomised controlled trial, with embedded economic and qualitative evaluations.\u0000\u0000\u0000SETTINGS\u0000There were 26 sites, including 12 NHS trusts.\u0000\u0000\u0000PARTICIPANTS\u0000Participants were aged 7-16 years and had a specific phobia defined in accordance with established international clinical criteria.\u0000\u0000\u0000INTERVENTIONS\u0000Participants were randomised 1 : 1 to receive one-session treatment or usual-care cognitive-behavioural therapy, and were stratified according to age and phobia severity. Outcome assessors remained blind to treatment allocation.\u0000\u0000\u0000MAIN OUTCOME MEASURES\u0000The primary outcome measure was the Behavioural Avoidance Task at 6 months' follow-up. Secondary outcomes included the Anxiety Disorder Interview Schedule, Child Anxiety Impact Scale, Revised Children's Anxiety and Depression Scale, a goal-based outcome measure, Child Health Utility 9D, EuroQol-5 Dimensions Youth version and resource usage. Treatment fidelity was assessed using the Cognitive Behaviour Therapy Scale for Children and Young People and the One-Session Treatment Rating Scale.\u0000\u0000\u0000RESULTS\u0000A total of 274 participants were recruited, with 268 participants randomised to one-session treatment (n = 134) or cognitive-behavioural therapy (n = 134). A total of 197 participants contributed some data, with 149 participants in the intention-to-treat analysis and 113 in the per-protocol analysis. Mean Behavioural Avoidance Task scores at 6 months were similar across treatment groups when both intention-to-treat and per-protocol analyses were applied [cognitive-behavioural therapy: 7.1 (intention to treat), 7.4 (per protocol); one-session treatment: 7.4 (intention to treat), 7.6 (per protocol); on the standardised scale adjusted mean difference for cognitive-behavioural therapy compared with one-session treatment -0.123, 95% confidence interval -0.449 to 0.202 (intention to treat), mean difference -0.204, 95% confidence interval -0.579 to 0.171 (per protocol)]. These findings were wholly below the standardised non-inferiority limit of 0.4, which suggests that one-session treatment is non-inferior to cognitive-behavioural therapy. No between-group differences in secondary outcome measures were found. The health economics evaluation suggested that, c","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9638885/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10412775","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}