Victoria Hodgetts Morton, Catherine A Moakes, Jane Daniels, Lee Middleton, Andrew Shennan, Peter Brocklehurst, Fidan Israfil-Bayli, Andrew K Ewer, James Gray, Nigel Ab Simpson, Jane E Norman, Christoph Lees, Konstantinos Tryposkiadis, Clive Stubbs, Max Hughes, R Katie Morris, Philip Toozs-Hobson
<p><strong>Background: </strong>Second trimester miscarriage and preterm birth is a significant global problem. Surgical cervical cerclage is performed to prevent pregnancy loss and preterm birth. It utilises either a monofilament or braided suture. It is hypothesised that a braided material becomes colonised with pathogenic bacteria that causes vaginal dysbiosis, infection and cerclage failure.</p><p><strong>Objectives: </strong>The primary objective of the study was to examine the effectiveness of using a monofilament suture material as opposed to a braided suture material on pregnancy loss in women requiring a vaginal cervical cerclage.</p><p><strong>Design: </strong>Superiority open randomised controlled trial.</p><p><strong>Setting: </strong>Seventy-five maternity sites across the UK.</p><p><strong>Participants: </strong>Women experiencing a singleton pregnancy requiring a cervical cerclage.</p><p><strong>Interventions: </strong>Monofilament suture or braided suture.</p><p><strong>Main outcome measures: </strong>The primary outcome was pregnancy loss (miscarriage and perinatal mortality, including any stillbirth or neonatal death in the first week of life). Secondary outcomes included the core outcome set for preterm birth.</p><p><strong>Methods: </strong>Women were randomised on a 1 : 1 basis to monofilament or braided cerclage utilising a bespoke randomisation service with minimisation dependent on the site, indication for cerclage, intention to use progesterone and planned surgical technique. The inclusion criteria were three or more previous mid-trimester losses or preterm births, insertion of a cerclage in a previous pregnancy, a history of a mid-trimester loss or preterm birth with a shortened cervical length in the current pregnancy or in women who clinicians deemed at risk of preterm birth. The exclusion criteria were an emergency or rescue cerclage, age of < 18 years, being unable to give informed consent or the cerclage having to be placed abdominally. The original sample size was calculated based on a relative risk reduction of 41% from a pregnancy loss rate of 19% in the braided group to 11% in the monofilament group with 90% power and alpha at <i>p</i> = 0.05. The independent data monitoring committee noted a lower-than-anticipated pooled event rate within the trial and recommended an increase in sample size to 2050. The outcome data were collected using clinical record forms from the maternal and neonatal medical records and reported to Birmingham Clinical Trials Unit.</p><p><strong>Results: </strong>A total of 2049 women were randomised, after withdrawals and loss to follow-up, data on 1005 women in the monofilament group and 993 women in the braided group were included. The baseline demographics between the groups were similar. There was no evidence of a difference in pregnancy loss rates between the monofilament and braided groups (80/1003 vs. 75/993; adjusted risk ratio: 1.05, 95% confidence interval: 0.79 to 1.40; adjusted
{"title":"Cerclage suture type to prevent pregnancy loss in women requiring a vaginal cervical cerclage: the C-STICH RCT.","authors":"Victoria Hodgetts Morton, Catherine A Moakes, Jane Daniels, Lee Middleton, Andrew Shennan, Peter Brocklehurst, Fidan Israfil-Bayli, Andrew K Ewer, James Gray, Nigel Ab Simpson, Jane E Norman, Christoph Lees, Konstantinos Tryposkiadis, Clive Stubbs, Max Hughes, R Katie Morris, Philip Toozs-Hobson","doi":"10.3310/YKTW8402","DOIUrl":"10.3310/YKTW8402","url":null,"abstract":"<p><strong>Background: </strong>Second trimester miscarriage and preterm birth is a significant global problem. Surgical cervical cerclage is performed to prevent pregnancy loss and preterm birth. It utilises either a monofilament or braided suture. It is hypothesised that a braided material becomes colonised with pathogenic bacteria that causes vaginal dysbiosis, infection and cerclage failure.</p><p><strong>Objectives: </strong>The primary objective of the study was to examine the effectiveness of using a monofilament suture material as opposed to a braided suture material on pregnancy loss in women requiring a vaginal cervical cerclage.</p><p><strong>Design: </strong>Superiority open randomised controlled trial.</p><p><strong>Setting: </strong>Seventy-five maternity sites across the UK.</p><p><strong>Participants: </strong>Women experiencing a singleton pregnancy requiring a cervical cerclage.</p><p><strong>Interventions: </strong>Monofilament suture or braided suture.</p><p><strong>Main outcome measures: </strong>The primary outcome was pregnancy loss (miscarriage and perinatal mortality, including any stillbirth or neonatal death in the first week of life). Secondary outcomes included the core outcome set for preterm birth.</p><p><strong>Methods: </strong>Women were randomised on a 1 : 1 basis to monofilament or braided cerclage utilising a bespoke randomisation service with minimisation dependent on the site, indication for cerclage, intention to use progesterone and planned surgical technique. The inclusion criteria were three or more previous mid-trimester losses or preterm births, insertion of a cerclage in a previous pregnancy, a history of a mid-trimester loss or preterm birth with a shortened cervical length in the current pregnancy or in women who clinicians deemed at risk of preterm birth. The exclusion criteria were an emergency or rescue cerclage, age of < 18 years, being unable to give informed consent or the cerclage having to be placed abdominally. The original sample size was calculated based on a relative risk reduction of 41% from a pregnancy loss rate of 19% in the braided group to 11% in the monofilament group with 90% power and alpha at <i>p</i> = 0.05. The independent data monitoring committee noted a lower-than-anticipated pooled event rate within the trial and recommended an increase in sample size to 2050. The outcome data were collected using clinical record forms from the maternal and neonatal medical records and reported to Birmingham Clinical Trials Unit.</p><p><strong>Results: </strong>A total of 2049 women were randomised, after withdrawals and loss to follow-up, data on 1005 women in the monofilament group and 993 women in the braided group were included. The baseline demographics between the groups were similar. There was no evidence of a difference in pregnancy loss rates between the monofilament and braided groups (80/1003 vs. 75/993; adjusted risk ratio: 1.05, 95% confidence interval: 0.79 to 1.40; adjusted","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 40","pages":"1-44"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11403380/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142139881","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}
Moira Cruickshank, Jemma Hudson, Rodolfo Hernández, Magaly Aceves-Martins, Richard Quinton, Katie Gillies, Lorna S Aucott, Charlotte Kennedy, Paul Manson, Nicholas Oliver, Frederick Wu, Siladitya Bhattacharya, Waljit S Dhillo, Channa N Jayasena, Miriam Brazzelli
<p><strong>Background: </strong>Low levels of testosterone cause male hypogonadism, which is associated with sexual dysfunction, tiredness and reduced muscle strength and quality of life. Testosterone replacement therapy is commonly used for ameliorating symptoms of male hypogonadism, but there is uncertainty about the magnitude of its effects and its cardiovascular and cerebrovascular safety.</p><p><strong>Aims of the research: </strong>The primary aim was to evaluate the safety of testosterone replacement therapy. We also assessed the clinical and cost-effectiveness of testosterone replacement therapy for men with male hypogonadism, and the existing qualitative evidence on men's experience and acceptability of testosterone replacement therapy.</p><p><strong>Design: </strong>Evidence synthesis and individual participant data meta-analysis of effectiveness and safety, qualitative evidence synthesis and model-based cost-utility analysis.</p><p><strong>Data sources: </strong>Major electronic databases were searched from 1992 to February 2021 and were restricted to English-language publications.</p><p><strong>Methods: </strong>We conducted a systematic review with meta-analysis of individual participant data according to current methodological standards. Evidence was considered from placebo-controlled randomised controlled trials assessing the effects of any formulation of testosterone replacement therapy in men with male hypogonadism. Primary outcomes were mortality and cardiovascular and cerebrovascular events. Data were extracted by one reviewer and cross-checked by a second reviewer. The risk of bias was assessed using the Cochrane Risk of Bias tool. We performed one-stage meta-analyses using the acquired individual participant data and two-stage meta-analyses to integrate the individual participant data with data extracted from eligible studies that did not provide individual participant data. A decision-analytic Markov model was developed to evaluate the cost per quality-adjusted life-years of the use of testosterone replacement therapy in cohorts of patients of different starting ages.</p><p><strong>Results: </strong>We identified 35 trials (5601 randomised participants). Of these, 17 trials (3431 participants) provided individual participant data. There were too few deaths to assess mortality. There was no difference between the testosterone replacement therapy group (120/1601, 7.5%) and placebo group (110/1519, 7.2%) in the incidence of cardiovascular and/or cerebrovascular events (13 studies, odds ratio 1.07, 95% confidence interval 0.81 to 1.42; <i>p</i> = 0.62). Testosterone replacement therapy improved quality of life and sexual function in almost all patient subgroups. In the testosterone replacement therapy group, serum testosterone was higher while serum cholesterol, triglycerides, haemoglobin and haematocrit were all lower. We identified several themes from five qualitative studies showing how symptoms of low testosterone affect men
{"title":"The effects and safety of testosterone replacement therapy for men with hypogonadism: the TestES evidence synthesis and economic evaluation.","authors":"Moira Cruickshank, Jemma Hudson, Rodolfo Hernández, Magaly Aceves-Martins, Richard Quinton, Katie Gillies, Lorna S Aucott, Charlotte Kennedy, Paul Manson, Nicholas Oliver, Frederick Wu, Siladitya Bhattacharya, Waljit S Dhillo, Channa N Jayasena, Miriam Brazzelli","doi":"10.3310/JRYT3981","DOIUrl":"10.3310/JRYT3981","url":null,"abstract":"<p><strong>Background: </strong>Low levels of testosterone cause male hypogonadism, which is associated with sexual dysfunction, tiredness and reduced muscle strength and quality of life. Testosterone replacement therapy is commonly used for ameliorating symptoms of male hypogonadism, but there is uncertainty about the magnitude of its effects and its cardiovascular and cerebrovascular safety.</p><p><strong>Aims of the research: </strong>The primary aim was to evaluate the safety of testosterone replacement therapy. We also assessed the clinical and cost-effectiveness of testosterone replacement therapy for men with male hypogonadism, and the existing qualitative evidence on men's experience and acceptability of testosterone replacement therapy.</p><p><strong>Design: </strong>Evidence synthesis and individual participant data meta-analysis of effectiveness and safety, qualitative evidence synthesis and model-based cost-utility analysis.</p><p><strong>Data sources: </strong>Major electronic databases were searched from 1992 to February 2021 and were restricted to English-language publications.</p><p><strong>Methods: </strong>We conducted a systematic review with meta-analysis of individual participant data according to current methodological standards. Evidence was considered from placebo-controlled randomised controlled trials assessing the effects of any formulation of testosterone replacement therapy in men with male hypogonadism. Primary outcomes were mortality and cardiovascular and cerebrovascular events. Data were extracted by one reviewer and cross-checked by a second reviewer. The risk of bias was assessed using the Cochrane Risk of Bias tool. We performed one-stage meta-analyses using the acquired individual participant data and two-stage meta-analyses to integrate the individual participant data with data extracted from eligible studies that did not provide individual participant data. A decision-analytic Markov model was developed to evaluate the cost per quality-adjusted life-years of the use of testosterone replacement therapy in cohorts of patients of different starting ages.</p><p><strong>Results: </strong>We identified 35 trials (5601 randomised participants). Of these, 17 trials (3431 participants) provided individual participant data. There were too few deaths to assess mortality. There was no difference between the testosterone replacement therapy group (120/1601, 7.5%) and placebo group (110/1519, 7.2%) in the incidence of cardiovascular and/or cerebrovascular events (13 studies, odds ratio 1.07, 95% confidence interval 0.81 to 1.42; <i>p</i> = 0.62). Testosterone replacement therapy improved quality of life and sexual function in almost all patient subgroups. In the testosterone replacement therapy group, serum testosterone was higher while serum cholesterol, triglycerides, haemoglobin and haematocrit were all lower. We identified several themes from five qualitative studies showing how symptoms of low testosterone affect men","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 43","pages":"1-210"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11404359/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142153874","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}
Carrie Stewart, Hangjian Wu, Uma Alagappan, Augusto Azuara-Blanco, Anthony J King, Andrew J Tatham, Rodolfo Hernández, Bruce Lowe, Darian Shotton, Nana Appiah, Taylor Coffey, Thenmalar Vadiveloo, Graeme MacLennan, Katie Gillies
<p><strong>Background: </strong>Glaucoma is a chronic disease of the optic nerve and a leading cause of severe visual loss in the UK. Once patients have been diagnosed, they need regular monitoring at hospital eye services. Recent advances in technology mean patients with glaucoma can now monitor their disease at home. This could be more convenient for patients and potentially reduce costs and increase capacity for the NHS. However, it is uncertain whether self-monitoring would be acceptable or possible for patients with glaucoma.</p><p><strong>Objectives: </strong>The objectives were to: identify which patients are most appropriate for home monitoring; understand views of key stakeholders (patients, clinicians, researchers) on whether home glaucoma monitoring is feasible and acceptable; develop a conceptual framework for the economic evaluation of home glaucoma monitoring; and explore the need for and provide evidence on the design of a future study to evaluate the clinical and cost-effectiveness of digital technologies for home monitoring of glaucoma.</p><p><strong>Design: </strong>In-home Tracking of glaucoma: Reliability, Acceptability, and Cost (I-TRAC) was a multiphase mixed-methods feasibility study with key components informed by theoretical and conceptual frameworks.</p><p><strong>Setting: </strong>Expert glaucoma specialists in the UK recruited through professional glaucoma societies; study site staff and patient participants recruited through three UK hospital eye services (England, Scotland, Northern Ireland); and UK research teams recruited though existing networks.</p><p><strong>Intervention: </strong>Home tonometer that measures intraocular pressure and a tablet computer with a visual function application. Patients were asked to use the technology weekly for 12 weeks.</p><p><strong>Results: </strong>Forty-two patients were recruited. Retention and completion of follow-up procedures was successful, with 95% (<i>n</i> = 40) completing the 3-month follow-up clinic visits. Adherence to the interventions was generally high [adherence to both devices (i.e. ≥ 80% adherence) was 55%]. Overall, patients and healthcare professionals were cautiously optimistic about the acceptability of digital technologies for home monitoring of patients with glaucoma. While most clinicians were supportive of the potential advantages glaucoma home monitoring could offer, concerns about the technologies (e.g. reliability and potential to miss disease progression) and how they would fit into routine care need to be addressed. Additionally, clarity is required on defining the ideal population for this intervention. Plans for how to evaluate value for money in a future study were also identified. However, the study also highlighted several unknowns relating to core components of a future evaluative study that require addressing before progression to a definitive effectiveness trial.</p><p><strong>Limitations: </strong>The main limitation relates to our sample an
{"title":"Feasibility of in-home monitoring for people with glaucoma: the I-TRAC mixed-methods study.","authors":"Carrie Stewart, Hangjian Wu, Uma Alagappan, Augusto Azuara-Blanco, Anthony J King, Andrew J Tatham, Rodolfo Hernández, Bruce Lowe, Darian Shotton, Nana Appiah, Taylor Coffey, Thenmalar Vadiveloo, Graeme MacLennan, Katie Gillies","doi":"10.3310/GTWD6802","DOIUrl":"10.3310/GTWD6802","url":null,"abstract":"<p><strong>Background: </strong>Glaucoma is a chronic disease of the optic nerve and a leading cause of severe visual loss in the UK. Once patients have been diagnosed, they need regular monitoring at hospital eye services. Recent advances in technology mean patients with glaucoma can now monitor their disease at home. This could be more convenient for patients and potentially reduce costs and increase capacity for the NHS. However, it is uncertain whether self-monitoring would be acceptable or possible for patients with glaucoma.</p><p><strong>Objectives: </strong>The objectives were to: identify which patients are most appropriate for home monitoring; understand views of key stakeholders (patients, clinicians, researchers) on whether home glaucoma monitoring is feasible and acceptable; develop a conceptual framework for the economic evaluation of home glaucoma monitoring; and explore the need for and provide evidence on the design of a future study to evaluate the clinical and cost-effectiveness of digital technologies for home monitoring of glaucoma.</p><p><strong>Design: </strong>In-home Tracking of glaucoma: Reliability, Acceptability, and Cost (I-TRAC) was a multiphase mixed-methods feasibility study with key components informed by theoretical and conceptual frameworks.</p><p><strong>Setting: </strong>Expert glaucoma specialists in the UK recruited through professional glaucoma societies; study site staff and patient participants recruited through three UK hospital eye services (England, Scotland, Northern Ireland); and UK research teams recruited though existing networks.</p><p><strong>Intervention: </strong>Home tonometer that measures intraocular pressure and a tablet computer with a visual function application. Patients were asked to use the technology weekly for 12 weeks.</p><p><strong>Results: </strong>Forty-two patients were recruited. Retention and completion of follow-up procedures was successful, with 95% (<i>n</i> = 40) completing the 3-month follow-up clinic visits. Adherence to the interventions was generally high [adherence to both devices (i.e. ≥ 80% adherence) was 55%]. Overall, patients and healthcare professionals were cautiously optimistic about the acceptability of digital technologies for home monitoring of patients with glaucoma. While most clinicians were supportive of the potential advantages glaucoma home monitoring could offer, concerns about the technologies (e.g. reliability and potential to miss disease progression) and how they would fit into routine care need to be addressed. Additionally, clarity is required on defining the ideal population for this intervention. Plans for how to evaluate value for money in a future study were also identified. However, the study also highlighted several unknowns relating to core components of a future evaluative study that require addressing before progression to a definitive effectiveness trial.</p><p><strong>Limitations: </strong>The main limitation relates to our sample an","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 44","pages":"1-163"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11404360/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142153875","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}
Sophie Beese, Tuba S Avşar, Malcolm Price, David Quinn, Hoong S Lim, Janine Dretzke, Chidubem O Ogwulu, Pelham Barton, Louise Jackson, David Moore
<p><strong>Background: </strong>Selected patients with advanced heart failure ineligible for heart transplantation could benefit from left ventricular assist device therapy as 'destination therapy'. There is evidence of the efficacy of destination therapy; however, it is not currently commissioned within the United Kingdom National Health Service due to the lack of economic evidence.</p><p><strong>Objective: </strong>What is the clinical and cost-effectiveness of a left ventricular assist device compared to medical management for patients with advanced heart failure ineligible for heart transplantation (destination therapy)?</p><p><strong>Methods: </strong>A systematic review of evidence on the clinical and cost-effectiveness of left ventricular assist devices as destination therapy was undertaken including, where feasible, a network meta-analysis to provide an indirect estimate of the relative effectiveness of currently available left ventricular assist devices compared to medical management. For the systematic reviews, data sources searched (up to 11 January 2022) were Cochrane CENTRAL, MEDLINE and EMBASE via Ovid for primary studies, and Epistemonikos and Cochrane Database of Systematic Reviews for relevant systematic reviews. Trial registers were also searched, along with data and reports from intervention-specific registries. Economic studies were identified in EconLit, CEA registry and the NHS Economic Evaluation Database (NHS EED). The searches were supplemented by checking reference lists of included studies. An economic model (Markov) was developed to estimate the cost-effectiveness of left ventricular assist devices compared to medical management from the United Kingdom National Health Service/personal social service perspective. Deterministic and probabilistic sensitivity analyses were conducted to explore uncertainties. Where possible, all analyses focused on the only currently available left ventricular assist device (HeartMate 3<sup>TM</sup>, Abbott, Chicago, IL, USA) in the United Kingdom.</p><p><strong>Results: </strong>The clinical effectiveness review included 134 studies (240 articles). There were no studies directly comparing HeartMate 3 and medical management (a randomised trial is ongoing). The currently available left ventricular assist device improves patient survival and reduces stroke rates and complications compared to earlier devices and relative to medical management. For example, survival at 24 months is 77% with the HeartMate 3 device compared to 59% with the HeartMate II (MOMENTUM 3 trial). An indirect comparison demonstrated a reduction in mortality compared to medical management [relative risk of death 0.25 (95% confidence interval 0.13 to 0.47); 24 months; this study]. The cost-effectiveness review included 5 cost analyses and 14 economic evaluations covering different generations of devices and with different perspectives. The reported incremental costs per quality-adjusted life-year gained compared to medical
{"title":"Clinical and cost-effectiveness of left ventricular assist devices as destination therapy for advanced heart failure: systematic review and economic evaluation.","authors":"Sophie Beese, Tuba S Avşar, Malcolm Price, David Quinn, Hoong S Lim, Janine Dretzke, Chidubem O Ogwulu, Pelham Barton, Louise Jackson, David Moore","doi":"10.3310/MLFA4009","DOIUrl":"10.3310/MLFA4009","url":null,"abstract":"<p><strong>Background: </strong>Selected patients with advanced heart failure ineligible for heart transplantation could benefit from left ventricular assist device therapy as 'destination therapy'. There is evidence of the efficacy of destination therapy; however, it is not currently commissioned within the United Kingdom National Health Service due to the lack of economic evidence.</p><p><strong>Objective: </strong>What is the clinical and cost-effectiveness of a left ventricular assist device compared to medical management for patients with advanced heart failure ineligible for heart transplantation (destination therapy)?</p><p><strong>Methods: </strong>A systematic review of evidence on the clinical and cost-effectiveness of left ventricular assist devices as destination therapy was undertaken including, where feasible, a network meta-analysis to provide an indirect estimate of the relative effectiveness of currently available left ventricular assist devices compared to medical management. For the systematic reviews, data sources searched (up to 11 January 2022) were Cochrane CENTRAL, MEDLINE and EMBASE via Ovid for primary studies, and Epistemonikos and Cochrane Database of Systematic Reviews for relevant systematic reviews. Trial registers were also searched, along with data and reports from intervention-specific registries. Economic studies were identified in EconLit, CEA registry and the NHS Economic Evaluation Database (NHS EED). The searches were supplemented by checking reference lists of included studies. An economic model (Markov) was developed to estimate the cost-effectiveness of left ventricular assist devices compared to medical management from the United Kingdom National Health Service/personal social service perspective. Deterministic and probabilistic sensitivity analyses were conducted to explore uncertainties. Where possible, all analyses focused on the only currently available left ventricular assist device (HeartMate 3<sup>TM</sup>, Abbott, Chicago, IL, USA) in the United Kingdom.</p><p><strong>Results: </strong>The clinical effectiveness review included 134 studies (240 articles). There were no studies directly comparing HeartMate 3 and medical management (a randomised trial is ongoing). The currently available left ventricular assist device improves patient survival and reduces stroke rates and complications compared to earlier devices and relative to medical management. For example, survival at 24 months is 77% with the HeartMate 3 device compared to 59% with the HeartMate II (MOMENTUM 3 trial). An indirect comparison demonstrated a reduction in mortality compared to medical management [relative risk of death 0.25 (95% confidence interval 0.13 to 0.47); 24 months; this study]. The cost-effectiveness review included 5 cost analyses and 14 economic evaluations covering different generations of devices and with different perspectives. The reported incremental costs per quality-adjusted life-year gained compared to medical ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 38","pages":"1-237"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11367304/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142072626","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}
Fiona Collinson, Kara-Louise Royle, Jayne Swain, Christy Ralph, Anthony Maraveyas, Tim Eisen, Paul Nathan, Robert Jones, David Meads, Tze Min Wah, Adam Martin, Janine Bestall, Christian Kelly-Morland, Christopher Linsley, Jamie Oughton, Kevin Chan, Elisavet Theodoulou, Gustavo Arias-Pinilla, Amy Kwan, Luis Daverede, Catherine Handforth, Sebastian Trainor, Abdulazeez Salawu, Christopher McCabe, Vicky Goh, David Buckley, Jenny Hewison, Walter Gregory, Peter Selby, Julia Brown, Janet Brown
<p><strong>Background: </strong>There is interest in using treatment breaks in oncology, to reduce toxicity without compromising efficacy.</p><p><strong>Trial design: </strong>A Phase II/III multicentre, open-label, parallel-group, randomised controlled non-inferiority trial assessing treatment breaks in patients with renal cell carcinoma.</p><p><strong>Methods: </strong>Patients with locally advanced or metastatic renal cell carcinoma, starting tyrosine kinase inhibitor as first-line treatment at United Kingdom National Health Service hospitals.</p><p><strong>Interventions: </strong>At trial entry, patients were randomised (1 : 1) to a drug-free interval strategy or a conventional continuation strategy. After 24 weeks of treatment with sunitinib/pazopanib, drug-free interval strategy patients took up a treatment break until disease progression with additional breaks dependent on disease response and patient choice. Conventional continuation strategy patients continued on treatment. Both trial strategies continued until treatment intolerance, disease progression on treatment, withdrawal or death.</p><p><strong>Objective: </strong>To determine if a drug-free interval strategy is non-inferior to a conventional continuation strategy in terms of the co-primary outcomes of overall survival and quality-adjusted life-years.</p><p><strong>Co-primary outcomes: </strong>For non-inferiority to be concluded, a margin of ≤ 7.5% in overall survival and ≤ 10% in quality-adjusted life-years was required in both intention-to-treat and per-protocol analyses. This equated to the 95% confidence interval of the estimates being above 0.812 and -0.156, respectively. Quality-adjusted life-years were calculated using the utility index of the EuroQol-5 Dimensions questionnaire.</p><p><strong>Results: </strong>Nine hundred and twenty patients were randomised (461 conventional continuation strategy vs. 459 drug-free interval strategy) from 13 January 2012 to 12 September 2017. Trial treatment and follow-up stopped on 31 December 2020. Four hundred and eighty-eight (53.0%) patients [240 (52.1%) vs. 248 (54.0%)] continued on trial post week 24. The median treatment-break length was 87 days. Nine hundred and nineteen patients were included in the intention-to-treat analysis (461 vs. 458) and 871 patients in the per-protocol analysis (453 vs. 418). For overall survival, non-inferiority was concluded in the intention-to-treat analysis but not in the per-protocol analysis [hazard ratio (95% confidence interval) intention to treat 0.97 (0.83 to 1.12); per-protocol 0.94 (0.80 to 1.09) non-inferiority margin: 95% confidence interval ≥ 0.812, intention to treat: 0.83 > 0.812 non-inferior, per-protocol: 0.80 < 0.812 not non-inferior]. Therefore, a drug-free interval strategy was not concluded to be non-inferior to a conventional continuation strategy in terms of overall survival. For quality-adjusted life-years, non-inferiority was concluded in both the intention-to-treat and per-prot
{"title":"Temporary treatment cessation compared with continuation of tyrosine kinase inhibitors for adults with renal cancer: the STAR non-inferiority RCT.","authors":"Fiona Collinson, Kara-Louise Royle, Jayne Swain, Christy Ralph, Anthony Maraveyas, Tim Eisen, Paul Nathan, Robert Jones, David Meads, Tze Min Wah, Adam Martin, Janine Bestall, Christian Kelly-Morland, Christopher Linsley, Jamie Oughton, Kevin Chan, Elisavet Theodoulou, Gustavo Arias-Pinilla, Amy Kwan, Luis Daverede, Catherine Handforth, Sebastian Trainor, Abdulazeez Salawu, Christopher McCabe, Vicky Goh, David Buckley, Jenny Hewison, Walter Gregory, Peter Selby, Julia Brown, Janet Brown","doi":"10.3310/JWTR4127","DOIUrl":"https://doi.org/10.3310/JWTR4127","url":null,"abstract":"<p><strong>Background: </strong>There is interest in using treatment breaks in oncology, to reduce toxicity without compromising efficacy.</p><p><strong>Trial design: </strong>A Phase II/III multicentre, open-label, parallel-group, randomised controlled non-inferiority trial assessing treatment breaks in patients with renal cell carcinoma.</p><p><strong>Methods: </strong>Patients with locally advanced or metastatic renal cell carcinoma, starting tyrosine kinase inhibitor as first-line treatment at United Kingdom National Health Service hospitals.</p><p><strong>Interventions: </strong>At trial entry, patients were randomised (1 : 1) to a drug-free interval strategy or a conventional continuation strategy. After 24 weeks of treatment with sunitinib/pazopanib, drug-free interval strategy patients took up a treatment break until disease progression with additional breaks dependent on disease response and patient choice. Conventional continuation strategy patients continued on treatment. Both trial strategies continued until treatment intolerance, disease progression on treatment, withdrawal or death.</p><p><strong>Objective: </strong>To determine if a drug-free interval strategy is non-inferior to a conventional continuation strategy in terms of the co-primary outcomes of overall survival and quality-adjusted life-years.</p><p><strong>Co-primary outcomes: </strong>For non-inferiority to be concluded, a margin of ≤ 7.5% in overall survival and ≤ 10% in quality-adjusted life-years was required in both intention-to-treat and per-protocol analyses. This equated to the 95% confidence interval of the estimates being above 0.812 and -0.156, respectively. Quality-adjusted life-years were calculated using the utility index of the EuroQol-5 Dimensions questionnaire.</p><p><strong>Results: </strong>Nine hundred and twenty patients were randomised (461 conventional continuation strategy vs. 459 drug-free interval strategy) from 13 January 2012 to 12 September 2017. Trial treatment and follow-up stopped on 31 December 2020. Four hundred and eighty-eight (53.0%) patients [240 (52.1%) vs. 248 (54.0%)] continued on trial post week 24. The median treatment-break length was 87 days. Nine hundred and nineteen patients were included in the intention-to-treat analysis (461 vs. 458) and 871 patients in the per-protocol analysis (453 vs. 418). For overall survival, non-inferiority was concluded in the intention-to-treat analysis but not in the per-protocol analysis [hazard ratio (95% confidence interval) intention to treat 0.97 (0.83 to 1.12); per-protocol 0.94 (0.80 to 1.09) non-inferiority margin: 95% confidence interval ≥ 0.812, intention to treat: 0.83 > 0.812 non-inferior, per-protocol: 0.80 < 0.812 not non-inferior]. Therefore, a drug-free interval strategy was not concluded to be non-inferior to a conventional continuation strategy in terms of overall survival. For quality-adjusted life-years, non-inferiority was concluded in both the intention-to-treat and per-prot","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 45","pages":"1-171"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11403377/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285839","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}
Jill Colquitt, Mary Jordan, Rachel Court, Emma Loveman, Janette Parr, Iman Ghosh, Peter Auguste, Mubarak Patel, Chris Stinton
<p><strong>Background: </strong>Lung cancer is one of the most common types of cancer in the United Kingdom. It is often diagnosed late. The 5-year survival rate for lung cancer is below 10%. Early diagnosis may improve survival. Software that has an artificial intelligence-developed algorithm might be useful in assisting with the identification of suspected lung cancer.</p><p><strong>Objectives: </strong>This review sought to identify evidence on adjunct artificial intelligence software for analysing chest X-rays for suspected lung cancer, and to develop a conceptual cost-effectiveness model to inform discussion of what would be required to develop a fully executable cost-effectiveness model for future economic evaluation.</p><p><strong>Data sources: </strong>The data sources were MEDLINE All, EMBASE, Cochrane Database of Systematic Reviews, Cochrane CENTRAL, Epistemonikos, ACM Digital Library, World Health Organization International Clinical Trials Registry Platform, clinical experts, Tufts Cost-Effectiveness Analysis Registry, company submissions and clinical experts. Searches were conducted from 25 November 2022 to 18 January 2023.</p><p><strong>Methods: </strong>Rapid evidence synthesis methods were employed. Data from companies were scrutinised. The eligibility criteria were (1) primary care populations referred for chest X-ray due to symptoms suggestive of lung cancer or reasons unrelated to lung cancer; (2) study designs that compared radiology specialist assessing chest X-ray with adjunct artificial intelligence software versus radiology specialists alone and (3) outcomes relating to test accuracy, practical implications of using artificial intelligence software and patient-related outcomes. A conceptual decision-analytic model was developed to inform a potential full cost-effectiveness evaluation of adjunct artificial intelligence software for analysing chest X-ray images to identify suspected lung cancer.</p><p><strong>Results: </strong>None of the studies identified in the searches or submitted by the companies met the inclusion criteria of the review. Contextual information from six studies that did not meet the inclusion criteria provided some evidence that sensitivity for lung cancer detection (but not nodule detection) might be higher when chest X-rays are interpreted by radiology specialists in combination with artificial intelligence software than when they are interpreted by radiology specialists alone. No significant differences were observed for specificity, positive predictive value or number of cancers detected. None of the six studies provided evidence on the clinical effectiveness of adjunct artificial intelligence software. The conceptual model highlighted a paucity of input data along the course of the diagnostic pathway and identified key assumptions required for evidence linkage.</p><p><strong>Limitations: </strong>This review employed rapid evidence synthesis methods. This included only one reviewer conducting all el
背景：肺癌是英国最常见的癌症之一：肺癌是英国最常见的癌症之一。肺癌通常诊断较晚。肺癌的 5 年生存率低于 10%。早期诊断可提高生存率。采用人工智能算法的软件可能有助于识别疑似肺癌：本综述旨在确定用于分析疑似肺癌胸部 X 光片的辅助人工智能软件的证据，并开发一个概念性成本效益模型，为讨论开发一个完全可执行的成本效益模型所需的条件提供信息，以便进行未来的经济评估：数据来源：MEDLINE All、EMBASE、Cochrane 系统综述数据库、Cochrane CENTRAL、Epistemonikos、ACM 数字图书馆、世界卫生组织国际临床试验注册平台、临床专家、塔夫茨成本效益分析注册中心、公司提交的资料和临床专家。检索时间为 2022 年 11 月 25 日至 2023 年 1 月 18 日：方法：采用快速证据综合方法。对来自公司的数据进行了仔细审查。资格标准为：(1) 因肺癌症状或与肺癌无关的原因而转诊进行胸部 X 光检查的初级保健人群；(2) 将放射科专家使用辅助人工智能软件评估胸部 X 光与放射科专家单独评估胸部 X 光进行比较的研究设计；(3) 与测试准确性、使用人工智能软件的实际意义和患者相关结果有关的结果。我们建立了一个概念性决策分析模型，为分析胸部X光图像以确定疑似肺癌的辅助人工智能软件的潜在全面成本效益评估提供依据：在搜索中发现或由公司提交的研究中，没有一项符合审查的纳入标准。不符合纳入标准的六项研究的背景信息提供了一些证据，表明由放射科专家结合人工智能软件解读胸部 X 光片时，肺癌检测灵敏度（但不包括结节检测）可能高于仅由放射科专家解读胸部 X 光片时。在特异性、阳性预测值或检测出的癌症数量方面没有观察到明显差异。六项研究均未提供有关辅助人工智能软件临床效果的证据。概念模型强调了诊断过程中输入数据的匮乏，并确定了证据关联所需的关键假设：本综述采用了快速证据综合方法。局限性：本综述采用了快速证据综合方法，其中包括只有一名综述员负责综述的全部内容，并仅以英语进行有针对性的检索。未发现符合条件的研究：目前还没有适用于本综述的关于使用辅助人工智能软件检测胸部 X 光片上疑似肺癌的证据，无论是从初级保健转诊的肺癌症状患者，还是因其他原因从初级保健转诊的患者：未来的研究：需要了解辅助人工智能软件检测肺结节和癌症的准确性，以及其对临床决策和患者预后的影响。为概念模型生成关键输入参数的研究将有助于完善模型结构，并将其转换为完整的工作模型，以分析人工智能软件在该适应症方面的成本效益：本研究注册为 PROSPERO CRD42023384164：该奖项由美国国家健康与护理研究所（NIHR）的证据合成计划（NIHR奖项编号：NIHR135755）资助，全文发表于《健康技术评估》（Health Technology Assessment）第28卷第50期。更多奖项信息请参阅 NIHR Funding and Awards 网站。
{"title":"Artificial intelligence software for analysing chest X-ray images to identify suspected lung cancer: an evidence synthesis early value assessment.","authors":"Jill Colquitt, Mary Jordan, Rachel Court, Emma Loveman, Janette Parr, Iman Ghosh, Peter Auguste, Mubarak Patel, Chris Stinton","doi":"10.3310/LKRT4721","DOIUrl":"https://doi.org/10.3310/LKRT4721","url":null,"abstract":"<p><strong>Background: </strong>Lung cancer is one of the most common types of cancer in the United Kingdom. It is often diagnosed late. The 5-year survival rate for lung cancer is below 10%. Early diagnosis may improve survival. Software that has an artificial intelligence-developed algorithm might be useful in assisting with the identification of suspected lung cancer.</p><p><strong>Objectives: </strong>This review sought to identify evidence on adjunct artificial intelligence software for analysing chest X-rays for suspected lung cancer, and to develop a conceptual cost-effectiveness model to inform discussion of what would be required to develop a fully executable cost-effectiveness model for future economic evaluation.</p><p><strong>Data sources: </strong>The data sources were MEDLINE All, EMBASE, Cochrane Database of Systematic Reviews, Cochrane CENTRAL, Epistemonikos, ACM Digital Library, World Health Organization International Clinical Trials Registry Platform, clinical experts, Tufts Cost-Effectiveness Analysis Registry, company submissions and clinical experts. Searches were conducted from 25 November 2022 to 18 January 2023.</p><p><strong>Methods: </strong>Rapid evidence synthesis methods were employed. Data from companies were scrutinised. The eligibility criteria were (1) primary care populations referred for chest X-ray due to symptoms suggestive of lung cancer or reasons unrelated to lung cancer; (2) study designs that compared radiology specialist assessing chest X-ray with adjunct artificial intelligence software versus radiology specialists alone and (3) outcomes relating to test accuracy, practical implications of using artificial intelligence software and patient-related outcomes. A conceptual decision-analytic model was developed to inform a potential full cost-effectiveness evaluation of adjunct artificial intelligence software for analysing chest X-ray images to identify suspected lung cancer.</p><p><strong>Results: </strong>None of the studies identified in the searches or submitted by the companies met the inclusion criteria of the review. Contextual information from six studies that did not meet the inclusion criteria provided some evidence that sensitivity for lung cancer detection (but not nodule detection) might be higher when chest X-rays are interpreted by radiology specialists in combination with artificial intelligence software than when they are interpreted by radiology specialists alone. No significant differences were observed for specificity, positive predictive value or number of cancers detected. None of the six studies provided evidence on the clinical effectiveness of adjunct artificial intelligence software. The conceptual model highlighted a paucity of input data along the course of the diagnostic pathway and identified key assumptions required for evidence linkage.</p><p><strong>Limitations: </strong>This review employed rapid evidence synthesis methods. This included only one reviewer conducting all el","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 50","pages":"1-75"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11403378/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285844","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}
Barry Wright, Kerry Jane Bell, Jane E Blackwell, Catarina Teige, Laura Mandefield, Han I Wang, Charlie Welch, Arabella Scantlebury, Jude Watson, Dean McMillan, Emma Standley, Leah Attwell, Hayley Carrick, Amelia Taylor, Olivia Taylor, Rachel Hodkinson, Hannah Edwards, Hannah Pearson, Steve Parrott, David Marshall, Danielle Varley, Rebecca Hargate, Ann Mclaren, Catherine Elizabeth Hewitt
<p><strong>Background: </strong>Differences in the way autistic children experience the world can contribute to anxiety and stress. Carol Gray's Social Stories™ are a highly personalised intervention to support children by providing social information about specific situations in an individual story.</p><p><strong>Objectives: </strong>This randomised controlled trial aimed to establish whether Social Stories are clinically effective and cost-effective in improving social responsiveness and social and emotional health in children on the autism spectrum in schools.</p><p><strong>Design: </strong>A multisite pragmatic cluster randomised controlled trial comparing Social Stories with care as usual.</p><p><strong>Setting: </strong>Eighty-seven schools (clusters) across Yorkshire and the Humber.</p><p><strong>Participants: </strong>Two hundred and forty-nine children were randomised via a bespoke system hosted at York Trials Unit (129 Social Stories and 120 care as usual). Recruitment was completed in May 2021. Participants were children aged 4-11 years with a diagnosis of autism, alongside teachers, interventionists and caregivers. Recruitment was via schools, NHS trusts, support groups and local publicity.</p><p><strong>Intervention: </strong>The intervention included training for educational professionals and caregivers covering psychoeducation and implementation of Social Stories. Stories were written around contextualised goals around the child's need for social information. Interventionists read the Social Story™ with the child at least six times over 4 weeks during school.</p><p><strong>Main outcome measure: </strong>The primary outcome was the Social Responsiveness Scale-2 completed by teachers at 6 months (the primary end point), which measures social awareness, cognition, communication and behaviour. Data were collected from caregivers and educational professionals at 6 weeks and 6 months through questionnaires. Blinding of participants was not possible.</p><p><strong>Results: </strong>At 6 months, the estimated difference in expected teacher-reported Social Responsiveness Scale-2 T-score (the primary end point) was -1.61 (95% confidence interval -4.18 to 0.96, <i>p</i> = 0.220), slightly favouring the intervention group. The estimated differences for the parent-reported secondary outcomes at 6 months were small and generally favoured the control group except the measure of children's quality-adjusted life-year (+ 0.001, 95% confidence interval -0.032 to 0.035) and parental stress (-1.49, 95% confidence interval -5.43 to 2.46, <i>p</i> = 0.460), which favoured the intervention group. Children in the intervention group met their individual goals more frequently than children who received usual care alone (0.97 confidence interval 0.21 to 1.73, <i>p</i> = 0.012). The intervention is likely to save small costs (-£191 per child, 95% confidence interval -767.7 to 337.7) and maintain a similar quality of life compared to usual care. The probability
{"title":"Impact of Social Stories on social and emotional health of autism spectrum primary school children: the ASSSIST2 RCT with economic evaluation.","authors":"Barry Wright, Kerry Jane Bell, Jane E Blackwell, Catarina Teige, Laura Mandefield, Han I Wang, Charlie Welch, Arabella Scantlebury, Jude Watson, Dean McMillan, Emma Standley, Leah Attwell, Hayley Carrick, Amelia Taylor, Olivia Taylor, Rachel Hodkinson, Hannah Edwards, Hannah Pearson, Steve Parrott, David Marshall, Danielle Varley, Rebecca Hargate, Ann Mclaren, Catherine Elizabeth Hewitt","doi":"10.3310/JBTM8017","DOIUrl":"10.3310/JBTM8017","url":null,"abstract":"<p><strong>Background: </strong>Differences in the way autistic children experience the world can contribute to anxiety and stress. Carol Gray's Social Stories™ are a highly personalised intervention to support children by providing social information about specific situations in an individual story.</p><p><strong>Objectives: </strong>This randomised controlled trial aimed to establish whether Social Stories are clinically effective and cost-effective in improving social responsiveness and social and emotional health in children on the autism spectrum in schools.</p><p><strong>Design: </strong>A multisite pragmatic cluster randomised controlled trial comparing Social Stories with care as usual.</p><p><strong>Setting: </strong>Eighty-seven schools (clusters) across Yorkshire and the Humber.</p><p><strong>Participants: </strong>Two hundred and forty-nine children were randomised via a bespoke system hosted at York Trials Unit (129 Social Stories and 120 care as usual). Recruitment was completed in May 2021. Participants were children aged 4-11 years with a diagnosis of autism, alongside teachers, interventionists and caregivers. Recruitment was via schools, NHS trusts, support groups and local publicity.</p><p><strong>Intervention: </strong>The intervention included training for educational professionals and caregivers covering psychoeducation and implementation of Social Stories. Stories were written around contextualised goals around the child's need for social information. Interventionists read the Social Story™ with the child at least six times over 4 weeks during school.</p><p><strong>Main outcome measure: </strong>The primary outcome was the Social Responsiveness Scale-2 completed by teachers at 6 months (the primary end point), which measures social awareness, cognition, communication and behaviour. Data were collected from caregivers and educational professionals at 6 weeks and 6 months through questionnaires. Blinding of participants was not possible.</p><p><strong>Results: </strong>At 6 months, the estimated difference in expected teacher-reported Social Responsiveness Scale-2 T-score (the primary end point) was -1.61 (95% confidence interval -4.18 to 0.96, <i>p</i> = 0.220), slightly favouring the intervention group. The estimated differences for the parent-reported secondary outcomes at 6 months were small and generally favoured the control group except the measure of children's quality-adjusted life-year (+ 0.001, 95% confidence interval -0.032 to 0.035) and parental stress (-1.49, 95% confidence interval -5.43 to 2.46, <i>p</i> = 0.460), which favoured the intervention group. Children in the intervention group met their individual goals more frequently than children who received usual care alone (0.97 confidence interval 0.21 to 1.73, <i>p</i> = 0.012). The intervention is likely to save small costs (-£191 per child, 95% confidence interval -767.7 to 337.7) and maintain a similar quality of life compared to usual care. The probability","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 39","pages":"1-121"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142106913","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}
Simon D Kyle, Peter Bower, Ly-Mee Yu, Aloysius Niroshan Siriwardena, Yaling Yang, Stavros Petrou, Emma Ogburn, Nargis Begum, Leonie Maurer, Barbara Robinson, Caroline Gardner, Stephanie Armstrong, Julie Pattinson, Colin A Espie, Paul Aveyard
<p><strong>Background: </strong>Insomnia is a prevalent and distressing sleep disorder. Multicomponent cognitive-behavioural therapy is the recommended first-line treatment, but access remains extremely limited, particularly in primary care where insomnia is managed. One principal component of cognitive-behavioural therapy is a behavioural treatment called sleep restriction therapy, which could potentially be delivered as a brief single-component intervention by generalists in primary care.</p><p><strong>Objectives: </strong>The primary objective of the Health-professional Administered Brief Insomnia Therapy trial was to establish whether nurse-delivered sleep restriction therapy in primary care improves insomnia relative to sleep hygiene. Secondary objectives were to establish whether nurse-delivered sleep restriction therapy was cost-effective, and to undertake a process evaluation to understand intervention delivery, fidelity and acceptability.</p><p><strong>Design: </strong>Pragmatic, multicentre, individually randomised, parallel-group, superiority trial with embedded process evaluation.</p><p><strong>Setting: </strong>National Health Service general practice in three regions of England.</p><p><strong>Participants: </strong>Adults aged ≥ 18 years with insomnia disorder were randomised using a validated web-based randomisation programme.</p><p><strong>Interventions: </strong>Participants in the intervention group were offered a brief four-session nurse-delivered behavioural treatment involving two in-person sessions and two by phone. Participants were supported to follow a prescribed sleep schedule with the aim of restricting and standardising time in bed. Participants were also provided with a sleep hygiene leaflet. The control group received the same sleep hygiene leaflet by e-mail or post. There was no restriction on usual care.</p><p><strong>Main outcome measures: </strong>Outcomes were assessed at 3, 6 and 12 months. Participants were included in the primary analysis if they contributed at least one post-randomisation outcome. The primary end point was self-reported insomnia severity with the Insomnia Severity Index at 6 months. Secondary outcomes were health-related and sleep-related quality of life, depressive symptoms, work productivity and activity impairment, self-reported and actigraphy-defined sleep, and hypnotic medication use. Cost-effectiveness was evaluated using the incremental cost per quality-adjusted life-year. For the process evaluation, semistructured interviews were carried out with participants, nurses and practice managers or general practitioners. Due to the nature of the intervention, both participants and nurses were aware of group allocation.</p><p><strong>Results: </strong>We recruited 642 participants (<i>n</i> = 321 for sleep restriction therapy; <i>n</i> = 321 for sleep hygiene) between 29 August 2018 and 23 March 2020. Five hundred and eighty participants (90.3%) provided data at a minimum of one follow-up ti
资助：该奖项由国家健康与护理研究所（NIHR）的健康技术评估计划资助（NIHR奖项编号：16/84/01），全文发表于《健康技术评估》第28卷第36期。如需了解更多奖项信息，请访问 NIHR Funding and Awards 网站。
{"title":"Nurse-delivered sleep restriction therapy to improve insomnia disorder in primary care: the HABIT RCT.","authors":"Simon D Kyle, Peter Bower, Ly-Mee Yu, Aloysius Niroshan Siriwardena, Yaling Yang, Stavros Petrou, Emma Ogburn, Nargis Begum, Leonie Maurer, Barbara Robinson, Caroline Gardner, Stephanie Armstrong, Julie Pattinson, Colin A Espie, Paul Aveyard","doi":"10.3310/RJYT4275","DOIUrl":"10.3310/RJYT4275","url":null,"abstract":"<p><strong>Background: </strong>Insomnia is a prevalent and distressing sleep disorder. Multicomponent cognitive-behavioural therapy is the recommended first-line treatment, but access remains extremely limited, particularly in primary care where insomnia is managed. One principal component of cognitive-behavioural therapy is a behavioural treatment called sleep restriction therapy, which could potentially be delivered as a brief single-component intervention by generalists in primary care.</p><p><strong>Objectives: </strong>The primary objective of the Health-professional Administered Brief Insomnia Therapy trial was to establish whether nurse-delivered sleep restriction therapy in primary care improves insomnia relative to sleep hygiene. Secondary objectives were to establish whether nurse-delivered sleep restriction therapy was cost-effective, and to undertake a process evaluation to understand intervention delivery, fidelity and acceptability.</p><p><strong>Design: </strong>Pragmatic, multicentre, individually randomised, parallel-group, superiority trial with embedded process evaluation.</p><p><strong>Setting: </strong>National Health Service general practice in three regions of England.</p><p><strong>Participants: </strong>Adults aged ≥ 18 years with insomnia disorder were randomised using a validated web-based randomisation programme.</p><p><strong>Interventions: </strong>Participants in the intervention group were offered a brief four-session nurse-delivered behavioural treatment involving two in-person sessions and two by phone. Participants were supported to follow a prescribed sleep schedule with the aim of restricting and standardising time in bed. Participants were also provided with a sleep hygiene leaflet. The control group received the same sleep hygiene leaflet by e-mail or post. There was no restriction on usual care.</p><p><strong>Main outcome measures: </strong>Outcomes were assessed at 3, 6 and 12 months. Participants were included in the primary analysis if they contributed at least one post-randomisation outcome. The primary end point was self-reported insomnia severity with the Insomnia Severity Index at 6 months. Secondary outcomes were health-related and sleep-related quality of life, depressive symptoms, work productivity and activity impairment, self-reported and actigraphy-defined sleep, and hypnotic medication use. Cost-effectiveness was evaluated using the incremental cost per quality-adjusted life-year. For the process evaluation, semistructured interviews were carried out with participants, nurses and practice managers or general practitioners. Due to the nature of the intervention, both participants and nurses were aware of group allocation.</p><p><strong>Results: </strong>We recruited 642 participants (<i>n</i> = 321 for sleep restriction therapy; <i>n</i> = 321 for sleep hygiene) between 29 August 2018 and 23 March 2020. Five hundred and eighty participants (90.3%) provided data at a minimum of one follow-up ti","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 36","pages":"1-107"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11367301/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142054174","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}
Hisham Mehanna, Jonathan J Deeks, Kristien Boelaert, Gitta Madani, Paul Sidhu, Paul Nankivell, Neil Sharma, Rebecca Woolley, Judith Taylor, Tessa Fulton-Lieuw, Andrew Palmer
<p><strong>Background: </strong>Strain and shear wave elastography which is commonly used with concurrent real-time imaging known as real-time ultrasound shear/strain wave elastography is a new diagnostic technique that has been reported to be useful in the diagnosis of nodules in several organs. There is conflicting evidence regarding its benefit over ultrasound-guided fine-needle aspiration cytology alone in thyroid nodules.</p><p><strong>Objectives: </strong>To determine if ultrasound strain and shear wave elastography in conjunction with fine-needle aspiration cytology will reduce the number of patients who have a non-diagnostic first fine-needle aspiration cytology results as compared to conventional ultrasound-only guided fine-needle aspiration cytology.</p><p><strong>Design: </strong>A pragmatic, unblinded, multicentre randomised controlled trial.</p><p><strong>Setting: </strong>Eighteen centres with a radiology department across England.</p><p><strong>Participants: </strong>Adults who had not undergone previous fine-needle aspiration cytology with single or multiple nodules undergoing investigation.</p><p><strong>Interventions: </strong>Ultrasound shear/strain wave elastography-ultrasound guided fine-needle aspiration cytology (intervention arm) - strain or shear wave elastography-guided fine-needle aspiration cytology. Ultrasound-only guided fine-needle aspiration cytology (control arm) - routine ultrasound-only guided fine-needle aspiration cytology (the current standard recommended by the British Thyroid Association guidelines).</p><p><strong>Main outcome measure: </strong>The proportion of patients who have a non-diagnostic cytology (Thy 1) result following the first fine-needle aspiration cytology.</p><p><strong>Randomisation: </strong>Patients were randomised at a 1 : 1 ratio to the interventional or control arms.</p><p><strong>Results: </strong>A total of 982 participants (80% female) were randomised: 493 were randomised to ultrasound shear/strain wave elastography-ultrasound guided fine-needle aspiration cytology and 489 were randomised to ultrasound-only guided fine-needle aspiration cytology. There was no evidence of a difference between ultrasound shear/strain wave elastography and ultrasound in non-diagnostic cytology (Thy 1) rate following the first fine-needle aspiration cytology (19% vs. 16% respectively; risk difference: 0.030; 95% confidence interval -0.007 to 0.066; <i>p</i> = 0.11), the number of fine-needle aspiration cytologies needed (odds ratio: 1.10; 95% confidence interval 0.82 to 1.49; <i>p</i> = 0.53) or in the time to reach a definitive diagnosis (hazard ratio: 0.94; 95% confidence interval 0.81 to 1.10; <i>p</i> = 0.45). There was a small, non-significant reduction in the number of thyroid operations undertaken when ultrasound shear/strain wave elastography was used (37% vs. 40% respectively; risk difference: -0.02; 95% confidence interval -0.06 to 0.009; <i>p</i> = 0.15), but no difference in the number of op
{"title":"Real-time ultrasound elastography in the diagnosis of newly identified thyroid nodules in adults: the ElaTION RCT.","authors":"Hisham Mehanna, Jonathan J Deeks, Kristien Boelaert, Gitta Madani, Paul Sidhu, Paul Nankivell, Neil Sharma, Rebecca Woolley, Judith Taylor, Tessa Fulton-Lieuw, Andrew Palmer","doi":"10.3310/PLEQ4874","DOIUrl":"https://doi.org/10.3310/PLEQ4874","url":null,"abstract":"<p><strong>Background: </strong>Strain and shear wave elastography which is commonly used with concurrent real-time imaging known as real-time ultrasound shear/strain wave elastography is a new diagnostic technique that has been reported to be useful in the diagnosis of nodules in several organs. There is conflicting evidence regarding its benefit over ultrasound-guided fine-needle aspiration cytology alone in thyroid nodules.</p><p><strong>Objectives: </strong>To determine if ultrasound strain and shear wave elastography in conjunction with fine-needle aspiration cytology will reduce the number of patients who have a non-diagnostic first fine-needle aspiration cytology results as compared to conventional ultrasound-only guided fine-needle aspiration cytology.</p><p><strong>Design: </strong>A pragmatic, unblinded, multicentre randomised controlled trial.</p><p><strong>Setting: </strong>Eighteen centres with a radiology department across England.</p><p><strong>Participants: </strong>Adults who had not undergone previous fine-needle aspiration cytology with single or multiple nodules undergoing investigation.</p><p><strong>Interventions: </strong>Ultrasound shear/strain wave elastography-ultrasound guided fine-needle aspiration cytology (intervention arm) - strain or shear wave elastography-guided fine-needle aspiration cytology. Ultrasound-only guided fine-needle aspiration cytology (control arm) - routine ultrasound-only guided fine-needle aspiration cytology (the current standard recommended by the British Thyroid Association guidelines).</p><p><strong>Main outcome measure: </strong>The proportion of patients who have a non-diagnostic cytology (Thy 1) result following the first fine-needle aspiration cytology.</p><p><strong>Randomisation: </strong>Patients were randomised at a 1 : 1 ratio to the interventional or control arms.</p><p><strong>Results: </strong>A total of 982 participants (80% female) were randomised: 493 were randomised to ultrasound shear/strain wave elastography-ultrasound guided fine-needle aspiration cytology and 489 were randomised to ultrasound-only guided fine-needle aspiration cytology. There was no evidence of a difference between ultrasound shear/strain wave elastography and ultrasound in non-diagnostic cytology (Thy 1) rate following the first fine-needle aspiration cytology (19% vs. 16% respectively; risk difference: 0.030; 95% confidence interval -0.007 to 0.066; <i>p</i> = 0.11), the number of fine-needle aspiration cytologies needed (odds ratio: 1.10; 95% confidence interval 0.82 to 1.49; <i>p</i> = 0.53) or in the time to reach a definitive diagnosis (hazard ratio: 0.94; 95% confidence interval 0.81 to 1.10; <i>p</i> = 0.45). There was a small, non-significant reduction in the number of thyroid operations undertaken when ultrasound shear/strain wave elastography was used (37% vs. 40% respectively; risk difference: -0.02; 95% confidence interval -0.06 to 0.009; <i>p</i> = 0.15), but no difference in the number of op","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 46","pages":"1-51"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11403383/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285840","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}
Dwayne Boyers, Moira Cruickshank, Lorna Aucott, Charlotte Kennedy, Paul Manson, Paul Bachoo, Miriam Brazzelli
<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
{"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}
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