Steve Goodacre, Laura Sutton, Kate Ennis, Ben Thomas, Olivia Hawksworth, Khurram Iftikhar, Susan J Croft, Gordon Fuller, Simon Waterhouse, Daniel Hind, Matt Stevenson, Mike J Bradburn, Michael Smyth, Gavin D Perkins, Mark Millins, Andy Rosser, Jon Dickson, Matthew Wilson
<p><strong>Background: </strong>Guidelines for sepsis recommend treating those at highest risk within 1 hour. The emergency care system can only achieve this if sepsis is recognised and prioritised. Ambulance services can use prehospital early warning scores alongside paramedic diagnostic impression to prioritise patients for treatment or early assessment in the emergency department.</p><p><strong>Objectives: </strong>To determine the accuracy, impact and cost-effectiveness of using early warning scores alongside paramedic diagnostic impression to identify sepsis requiring urgent treatment.</p><p><strong>Design: </strong>Retrospective diagnostic cohort study and decision-analytic modelling of operational consequences and cost-effectiveness.</p><p><strong>Setting: </strong>Two ambulance services and four acute hospitals in England.</p><p><strong>Participants: </strong>Adults transported to hospital by emergency ambulance, excluding episodes with injury, mental health problems, cardiac arrest, direct transfer to specialist services, or no vital signs recorded.</p><p><strong>Interventions: </strong>Twenty-one early warning scores used alongside paramedic diagnostic impression, categorised as sepsis, infection, non-specific presentation, or other specific presentation.</p><p><strong>Main outcome measures: </strong>Proportion of cases prioritised at the four hospitals; diagnostic accuracy for the sepsis-3 definition of sepsis and receiving urgent treatment (primary reference standard); daily number of cases with and without sepsis prioritised at a large and a small hospital; the minimum treatment effect associated with prioritisation at which each strategy would be cost-effective, compared to no prioritisation, assuming willingness to pay £20,000 per quality-adjusted life-year gained.</p><p><strong>Results: </strong>Data from 95,022 episodes involving 71,204 patients across four hospitals showed that most early warning scores operating at their pre-specified thresholds would prioritise more than 10% of cases when applied to non-specific attendances or all attendances. Data from 12,870 episodes at one hospital identified 348 (2.7%) with the primary reference standard. The National Early Warning Score, version 2 (NEWS2), had the highest area under the receiver operating characteristic curve when applied only to patients with a paramedic diagnostic impression of sepsis or infection (0.756, 95% confidence interval 0.729 to 0.783) or sepsis alone (0.655, 95% confidence interval 0.63 to 0.68). None of the strategies provided high sensitivity (> 0.8) with acceptable positive predictive value (> 0.15). NEWS2 provided combinations of sensitivity and specificity that were similar or superior to all other early warning scores. Applying NEWS2 to paramedic diagnostic impression of sepsis or infection with thresholds of > 4, > 6 and > 8 respectively provided sensitivities and positive predictive values (95% confidence interval) of 0.522 (0.469 to 0.574) and 0.216 (
{"title":"Prehospital early warning scores for adults with suspected sepsis: the PHEWS observational cohort and decision-analytic modelling study.","authors":"Steve Goodacre, Laura Sutton, Kate Ennis, Ben Thomas, Olivia Hawksworth, Khurram Iftikhar, Susan J Croft, Gordon Fuller, Simon Waterhouse, Daniel Hind, Matt Stevenson, Mike J Bradburn, Michael Smyth, Gavin D Perkins, Mark Millins, Andy Rosser, Jon Dickson, Matthew Wilson","doi":"10.3310/NDTY2403","DOIUrl":"10.3310/NDTY2403","url":null,"abstract":"<p><strong>Background: </strong>Guidelines for sepsis recommend treating those at highest risk within 1 hour. The emergency care system can only achieve this if sepsis is recognised and prioritised. Ambulance services can use prehospital early warning scores alongside paramedic diagnostic impression to prioritise patients for treatment or early assessment in the emergency department.</p><p><strong>Objectives: </strong>To determine the accuracy, impact and cost-effectiveness of using early warning scores alongside paramedic diagnostic impression to identify sepsis requiring urgent treatment.</p><p><strong>Design: </strong>Retrospective diagnostic cohort study and decision-analytic modelling of operational consequences and cost-effectiveness.</p><p><strong>Setting: </strong>Two ambulance services and four acute hospitals in England.</p><p><strong>Participants: </strong>Adults transported to hospital by emergency ambulance, excluding episodes with injury, mental health problems, cardiac arrest, direct transfer to specialist services, or no vital signs recorded.</p><p><strong>Interventions: </strong>Twenty-one early warning scores used alongside paramedic diagnostic impression, categorised as sepsis, infection, non-specific presentation, or other specific presentation.</p><p><strong>Main outcome measures: </strong>Proportion of cases prioritised at the four hospitals; diagnostic accuracy for the sepsis-3 definition of sepsis and receiving urgent treatment (primary reference standard); daily number of cases with and without sepsis prioritised at a large and a small hospital; the minimum treatment effect associated with prioritisation at which each strategy would be cost-effective, compared to no prioritisation, assuming willingness to pay £20,000 per quality-adjusted life-year gained.</p><p><strong>Results: </strong>Data from 95,022 episodes involving 71,204 patients across four hospitals showed that most early warning scores operating at their pre-specified thresholds would prioritise more than 10% of cases when applied to non-specific attendances or all attendances. Data from 12,870 episodes at one hospital identified 348 (2.7%) with the primary reference standard. The National Early Warning Score, version 2 (NEWS2), had the highest area under the receiver operating characteristic curve when applied only to patients with a paramedic diagnostic impression of sepsis or infection (0.756, 95% confidence interval 0.729 to 0.783) or sepsis alone (0.655, 95% confidence interval 0.63 to 0.68). None of the strategies provided high sensitivity (> 0.8) with acceptable positive predictive value (> 0.15). NEWS2 provided combinations of sensitivity and specificity that were similar or superior to all other early warning scores. Applying NEWS2 to paramedic diagnostic impression of sepsis or infection with thresholds of > 4, > 6 and > 8 respectively provided sensitivities and positive predictive values (95% confidence interval) of 0.522 (0.469 to 0.574) and 0.216 (","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 16","pages":"1-93"},"PeriodicalIF":3.5,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11017155/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140318162","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}
Jo Worthington, Jessica Frost, Emily Sanderson, Madeleine Cochrane, Jessica Wheeler, Nikki Cotterill, Stephanie J MacNeill, Sian Noble, Miriam Avery, Samantha Clarke, Mandy Fader, Hashim Hashim, Lucy McGeagh, Margaret Macaulay, Jonathan Rees, Luke Robles, Gordon Taylor, Jodi Taylor, Joanne Thompson, J Athene Lane, Matthew J Ridd, Marcus J Drake
<p><strong>Background: </strong>Conservative therapies are recommended as initial treatment for male lower urinary tract symptoms. However, there is a lack of evidence on effectiveness and uncertainty regarding approaches to delivery.</p><p><strong>Objective: </strong>The objective was to determine whether or not a standardised and manualised care intervention delivered in primary care achieves superior symptomatic outcome for lower urinary tract symptoms to usual care.</p><p><strong>Design: </strong>This was a two-arm cluster randomised controlled trial.</p><p><strong>Setting: </strong>The trial was set in 30 NHS general practice sites in England.</p><p><strong>Participants: </strong>Participants were adult men (aged ≥ 18 years) with bothersome lower urinary tract symptoms.</p><p><strong>Interventions: </strong>Sites were randomised 1 : 1 to deliver the TReatIng Urinary symptoms in Men in Primary Health care using non-pharmacological and non-surgical interventions trial intervention or usual care to all participants. The TReatIng Urinary symptoms in Men in Primary Health care using non-pharmacological and non-surgical interventions intervention comprised a standardised advice booklet developed for the trial from the British Association of Urological Surgeons' patient information sheets, with patient and expert input. Patients were directed to relevant sections by general practice or research nurses/healthcare assistants following urinary symptom assessment, providing the manualised element. The healthcare professional provided follow-up contacts over 12 weeks to support adherence to the intervention.</p><p><strong>Main outcome measures: </strong>The primary outcome was the validated patient-reported International Prostate Symptom Score 12 months post consent. Rather than the minimal clinically important difference of 3.0 points for overall International Prostate Symptom Score, the sample size aimed to detect a difference of 2.0 points, owing to the recognised clinical impact of individual symptoms.</p><p><strong>Results: </strong>A total of 1077 men consented to the study: 524 in sites randomised to the intervention arm (<i>n</i> = 17) and 553 in sites randomised to the control arm (<i>n</i> = 13). A difference in mean International Prostate Symptom Score at 12 months was found (adjusted mean difference of -1.81 points, 95% confidence interval -2.66 to -0.95 points), with a lower score in the intervention arm, indicating less severe symptoms. Secondary outcomes of patient-reported urinary symptoms, quality of life specific to lower urinary tract symptoms and perception of lower urinary tract symptoms all showed evidence of a difference between the arms favouring the intervention. No difference was seen between the arms in the proportion of urology referrals or adverse events. In qualitative interviews, participants welcomed the intervention, describing positive effects on their symptoms, as well as on their understanding of conservative care and
{"title":"Lower urinary tract symptoms in men: the TRIUMPH cluster RCT.","authors":"Jo Worthington, Jessica Frost, Emily Sanderson, Madeleine Cochrane, Jessica Wheeler, Nikki Cotterill, Stephanie J MacNeill, Sian Noble, Miriam Avery, Samantha Clarke, Mandy Fader, Hashim Hashim, Lucy McGeagh, Margaret Macaulay, Jonathan Rees, Luke Robles, Gordon Taylor, Jodi Taylor, Joanne Thompson, J Athene Lane, Matthew J Ridd, Marcus J Drake","doi":"10.3310/GVBC3182","DOIUrl":"10.3310/GVBC3182","url":null,"abstract":"<p><strong>Background: </strong>Conservative therapies are recommended as initial treatment for male lower urinary tract symptoms. However, there is a lack of evidence on effectiveness and uncertainty regarding approaches to delivery.</p><p><strong>Objective: </strong>The objective was to determine whether or not a standardised and manualised care intervention delivered in primary care achieves superior symptomatic outcome for lower urinary tract symptoms to usual care.</p><p><strong>Design: </strong>This was a two-arm cluster randomised controlled trial.</p><p><strong>Setting: </strong>The trial was set in 30 NHS general practice sites in England.</p><p><strong>Participants: </strong>Participants were adult men (aged ≥ 18 years) with bothersome lower urinary tract symptoms.</p><p><strong>Interventions: </strong>Sites were randomised 1 : 1 to deliver the TReatIng Urinary symptoms in Men in Primary Health care using non-pharmacological and non-surgical interventions trial intervention or usual care to all participants. The TReatIng Urinary symptoms in Men in Primary Health care using non-pharmacological and non-surgical interventions intervention comprised a standardised advice booklet developed for the trial from the British Association of Urological Surgeons' patient information sheets, with patient and expert input. Patients were directed to relevant sections by general practice or research nurses/healthcare assistants following urinary symptom assessment, providing the manualised element. The healthcare professional provided follow-up contacts over 12 weeks to support adherence to the intervention.</p><p><strong>Main outcome measures: </strong>The primary outcome was the validated patient-reported International Prostate Symptom Score 12 months post consent. Rather than the minimal clinically important difference of 3.0 points for overall International Prostate Symptom Score, the sample size aimed to detect a difference of 2.0 points, owing to the recognised clinical impact of individual symptoms.</p><p><strong>Results: </strong>A total of 1077 men consented to the study: 524 in sites randomised to the intervention arm (<i>n</i> = 17) and 553 in sites randomised to the control arm (<i>n</i> = 13). A difference in mean International Prostate Symptom Score at 12 months was found (adjusted mean difference of -1.81 points, 95% confidence interval -2.66 to -0.95 points), with a lower score in the intervention arm, indicating less severe symptoms. Secondary outcomes of patient-reported urinary symptoms, quality of life specific to lower urinary tract symptoms and perception of lower urinary tract symptoms all showed evidence of a difference between the arms favouring the intervention. No difference was seen between the arms in the proportion of urology referrals or adverse events. In qualitative interviews, participants welcomed the intervention, describing positive effects on their symptoms, as well as on their understanding of conservative care and","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 13","pages":"1-162"},"PeriodicalIF":3.5,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11017146/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140174465","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}
Tony Kendrick, Christopher Dowrick, Glyn Lewis, Michael Moore, Geraldine M Leydon, Adam Wa Geraghty, Gareth Griffiths, Shihua Zhu, Guiqing Lily Yao, Carl May, Mark Gabbay, Rachel Dewar-Haggart, Samantha Williams, Lien Bui, Natalie Thompson, Lauren Bridewell, Emilia Trapasso, Tasneem Patel, Molly McCarthy, Naila Khan, Helen Page, Emma Corcoran, Jane Sungmin Hahn, Molly Bird, Mekeda X Logan, Brian Chi Fung Ching, Riya Tiwari, Anna Hunt, Beth Stuart
<p><strong>Background: </strong>Guidelines on the management of depression recommend that practitioners use patient-reported outcome measures for the follow-up monitoring of symptoms, but there is a lack of evidence of benefit in terms of patient outcomes.</p><p><strong>Objective: </strong>To test using the Patient Health Questionnaire-9 questionnaire as a patient-reported outcome measure for monitoring depression, training practitioners in interpreting scores and giving patients feedback.</p><p><strong>Design: </strong>Parallel-group, cluster-randomised superiority trial; 1 : 1 allocation to intervention and control.</p><p><strong>Setting: </strong>UK primary care (141 group general practices in England and Wales).</p><p><strong>Inclusion criteria: </strong>Patients aged ≥ 18 years with a new episode of depressive disorder or symptoms, recruited mainly through medical record searches, plus opportunistically in consultations.</p><p><strong>Exclusions: </strong>Current depression treatment, dementia, psychosis, substance misuse and risk of suicide.</p><p><strong>Intervention: </strong>Administration of the Patient Health Questionnaire-9 questionnaire with patient feedback soon after diagnosis, and at follow-up 10-35 days later, compared with usual care.</p><p><strong>Primary outcome: </strong>Beck Depression Inventory, 2nd edition, symptom scores at 12 weeks.</p><p><strong>Secondary outcomes: </strong>Beck Depression Inventory, 2nd edition, scores at 26 weeks; antidepressant drug treatment and mental health service contacts; social functioning (Work and Social Adjustment Scale) and quality of life (EuroQol 5-Dimension, five-level) at 12 and 26 weeks; service use over 26 weeks to calculate NHS costs; patient satisfaction at 26 weeks (Medical Informant Satisfaction Scale); and adverse events.</p><p><strong>Sample size: </strong>The original target sample of 676 patients recruited was reduced to 554 due to finding a significant correlation between baseline and follow-up values for the primary outcome measure.</p><p><strong>Randomisation: </strong>Remote computerised randomisation with minimisation by recruiting university, small/large practice and urban/rural location.</p><p><strong>Blinding: </strong>Blinding of participants was impossible given the open cluster design, but self-report outcome measures prevented observer bias. Analysis was blind to allocation.</p><p><strong>Analysis: </strong>Linear mixed models were used, adjusted for baseline depression, baseline anxiety, sociodemographic factors, and clustering including practice as random effect. Quality of life and costs were analysed over 26 weeks.</p><p><strong>Qualitative interviews: </strong>Practitioner and patient interviews were conducted to reflect on trial processes and use of the Patient Health Questionnaire-9 using the Normalization Process Theory framework.</p><p><strong>Results: </strong>Three hundred and two patients were recruited in intervention arm practices and 227 patients we
{"title":"Patient-reported outcome measures for monitoring primary care patients with depression: the PROMDEP cluster RCT and economic evaluation.","authors":"Tony Kendrick, Christopher Dowrick, Glyn Lewis, Michael Moore, Geraldine M Leydon, Adam Wa Geraghty, Gareth Griffiths, Shihua Zhu, Guiqing Lily Yao, Carl May, Mark Gabbay, Rachel Dewar-Haggart, Samantha Williams, Lien Bui, Natalie Thompson, Lauren Bridewell, Emilia Trapasso, Tasneem Patel, Molly McCarthy, Naila Khan, Helen Page, Emma Corcoran, Jane Sungmin Hahn, Molly Bird, Mekeda X Logan, Brian Chi Fung Ching, Riya Tiwari, Anna Hunt, Beth Stuart","doi":"10.3310/PLRQ4216","DOIUrl":"10.3310/PLRQ4216","url":null,"abstract":"<p><strong>Background: </strong>Guidelines on the management of depression recommend that practitioners use patient-reported outcome measures for the follow-up monitoring of symptoms, but there is a lack of evidence of benefit in terms of patient outcomes.</p><p><strong>Objective: </strong>To test using the Patient Health Questionnaire-9 questionnaire as a patient-reported outcome measure for monitoring depression, training practitioners in interpreting scores and giving patients feedback.</p><p><strong>Design: </strong>Parallel-group, cluster-randomised superiority trial; 1 : 1 allocation to intervention and control.</p><p><strong>Setting: </strong>UK primary care (141 group general practices in England and Wales).</p><p><strong>Inclusion criteria: </strong>Patients aged ≥ 18 years with a new episode of depressive disorder or symptoms, recruited mainly through medical record searches, plus opportunistically in consultations.</p><p><strong>Exclusions: </strong>Current depression treatment, dementia, psychosis, substance misuse and risk of suicide.</p><p><strong>Intervention: </strong>Administration of the Patient Health Questionnaire-9 questionnaire with patient feedback soon after diagnosis, and at follow-up 10-35 days later, compared with usual care.</p><p><strong>Primary outcome: </strong>Beck Depression Inventory, 2nd edition, symptom scores at 12 weeks.</p><p><strong>Secondary outcomes: </strong>Beck Depression Inventory, 2nd edition, scores at 26 weeks; antidepressant drug treatment and mental health service contacts; social functioning (Work and Social Adjustment Scale) and quality of life (EuroQol 5-Dimension, five-level) at 12 and 26 weeks; service use over 26 weeks to calculate NHS costs; patient satisfaction at 26 weeks (Medical Informant Satisfaction Scale); and adverse events.</p><p><strong>Sample size: </strong>The original target sample of 676 patients recruited was reduced to 554 due to finding a significant correlation between baseline and follow-up values for the primary outcome measure.</p><p><strong>Randomisation: </strong>Remote computerised randomisation with minimisation by recruiting university, small/large practice and urban/rural location.</p><p><strong>Blinding: </strong>Blinding of participants was impossible given the open cluster design, but self-report outcome measures prevented observer bias. Analysis was blind to allocation.</p><p><strong>Analysis: </strong>Linear mixed models were used, adjusted for baseline depression, baseline anxiety, sociodemographic factors, and clustering including practice as random effect. Quality of life and costs were analysed over 26 weeks.</p><p><strong>Qualitative interviews: </strong>Practitioner and patient interviews were conducted to reflect on trial processes and use of the Patient Health Questionnaire-9 using the Normalization Process Theory framework.</p><p><strong>Results: </strong>Three hundred and two patients were recruited in intervention arm practices and 227 patients we","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 17","pages":"1-95"},"PeriodicalIF":3.5,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11017630/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140318163","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}
Elizabeth Cook, Joanne Laycock, Dhanupriya Sivapathasuntharam, Camila Maturana, Catherine Hilton, Laura Doherty, Catherine Hewitt, Catriona McDaid, David Torgerson, Peter Bates
<p><strong>Background: </strong>Lateral compression type-1 pelvic fractures are a common fragility fracture in older adults. Patients who do not mobilise due to ongoing pain are at greater risk of immobility-related complications. Standard treatment in the United Kingdom is provision of pain relief and early mobilisation, unlike fragility hip fractures, which are usually treated surgically based on evidence that early surgery is associated with better outcomes. Currently there is no evidence on whether patients with lateral compression type-1 fragility fractures would have a better recovery with surgery than non-surgical management.</p><p><strong>Objectives: </strong>To assess the clinical and cost effectiveness of surgical fixation with internal fixation device compared to non-surgical management of lateral compression type-1 fragility fractures in older adults.</p><p><strong>Design: </strong>Pragmatic, randomised controlled superiority trial, with 12-month internal pilot; target sample size was 600 participants. Participants were randomised between surgical and non-surgical management (1 : 1 allocation ratio). An economic evaluation was planned.</p><p><strong>Setting: </strong>UK Major Trauma Centres.</p><p><strong>Participants: </strong>Patients aged 60 years or older with a lateral compression type-1 pelvic fracture, arising from a low-energy fall and unable to mobilise independently to a distance of 3 m and back due to pelvic pain 72 hours after injury.</p><p><strong>Interventions: </strong>Internal fixation device surgical fixation and non-surgical management. Participants, surgeons and outcome assessors were not blinded to treatment allocation.</p><p><strong>Main outcome measures: </strong>Primary outcome - average patient health-related quality of life, over 6 months, assessed by the EuroQol-5 Dimensions, five-level version utility score. Secondary outcomes (over the 6 months following injury) - self-rated health, physical function, mental health, pain, delirium, displacement of pelvis, mortality, complications and adverse events, and resource use data for the economic evaluation.</p><p><strong>Results: </strong>The trial closed early, at the end of the internal pilot, due to low recruitment. The internal pilot was undertaken in two separate phases because of a pause in recruitment due to the coronavirus disease 2019 pandemic. The planned statistical and health economic analyses were not conducted. Outcome data were summarised descriptively. Eleven sites opened for recruitment for a combined total of 92 months. Three-hundred and sixteen patients were assessed for eligibility, of whom 43 were eligible (13.6%). The main reason for ineligibility was that the patient was able to mobilise independently to 3 m and back (<i>n</i> = 161). Of the 43 eligible participants, 36 (83.7%) were approached for consent, of whom 11 (30.6%) provided consent. The most common reason for eligible patients not consenting to take part was that they were unwilling
{"title":"Surgical versus non-surgical management of lateral compression type-1 pelvic fracture in adults 60 years and older: the L1FE RCT.","authors":"Elizabeth Cook, Joanne Laycock, Dhanupriya Sivapathasuntharam, Camila Maturana, Catherine Hilton, Laura Doherty, Catherine Hewitt, Catriona McDaid, David Torgerson, Peter Bates","doi":"10.3310/LAPW3412","DOIUrl":"10.3310/LAPW3412","url":null,"abstract":"<p><strong>Background: </strong>Lateral compression type-1 pelvic fractures are a common fragility fracture in older adults. Patients who do not mobilise due to ongoing pain are at greater risk of immobility-related complications. Standard treatment in the United Kingdom is provision of pain relief and early mobilisation, unlike fragility hip fractures, which are usually treated surgically based on evidence that early surgery is associated with better outcomes. Currently there is no evidence on whether patients with lateral compression type-1 fragility fractures would have a better recovery with surgery than non-surgical management.</p><p><strong>Objectives: </strong>To assess the clinical and cost effectiveness of surgical fixation with internal fixation device compared to non-surgical management of lateral compression type-1 fragility fractures in older adults.</p><p><strong>Design: </strong>Pragmatic, randomised controlled superiority trial, with 12-month internal pilot; target sample size was 600 participants. Participants were randomised between surgical and non-surgical management (1 : 1 allocation ratio). An economic evaluation was planned.</p><p><strong>Setting: </strong>UK Major Trauma Centres.</p><p><strong>Participants: </strong>Patients aged 60 years or older with a lateral compression type-1 pelvic fracture, arising from a low-energy fall and unable to mobilise independently to a distance of 3 m and back due to pelvic pain 72 hours after injury.</p><p><strong>Interventions: </strong>Internal fixation device surgical fixation and non-surgical management. Participants, surgeons and outcome assessors were not blinded to treatment allocation.</p><p><strong>Main outcome measures: </strong>Primary outcome - average patient health-related quality of life, over 6 months, assessed by the EuroQol-5 Dimensions, five-level version utility score. Secondary outcomes (over the 6 months following injury) - self-rated health, physical function, mental health, pain, delirium, displacement of pelvis, mortality, complications and adverse events, and resource use data for the economic evaluation.</p><p><strong>Results: </strong>The trial closed early, at the end of the internal pilot, due to low recruitment. The internal pilot was undertaken in two separate phases because of a pause in recruitment due to the coronavirus disease 2019 pandemic. The planned statistical and health economic analyses were not conducted. Outcome data were summarised descriptively. Eleven sites opened for recruitment for a combined total of 92 months. Three-hundred and sixteen patients were assessed for eligibility, of whom 43 were eligible (13.6%). The main reason for ineligibility was that the patient was able to mobilise independently to 3 m and back (<i>n</i> = 161). Of the 43 eligible participants, 36 (83.7%) were approached for consent, of whom 11 (30.6%) provided consent. The most common reason for eligible patients not consenting to take part was that they were unwilling","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 15","pages":"1-67"},"PeriodicalIF":3.5,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11017634/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140174467","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}
Alanna Brown, Paloma Ferrando-Vivas, Mariana Popa, Gema Milla de la Fuente, John Pappachan, Brian H Cuthbertson, Laura Drikite, Richard Feltbower, Theodore Gouliouris, Isobel Sale, Robert Shulman, Lyvonne N Tume, John Myburgh, Kerry Woolfall, David A Harrison, Paul R Mouncey, Kathryn Rowan, Nazima Pathan
<p><strong>Background: </strong>Healthcare-associated infections are a major cause of morbidity and mortality in critically ill children. In adults, data suggest the use of selective decontamination of the digestive tract may reduce the incidence of healthcare-associated infections. Selective decontamination of the digestive tract has not been evaluated in the paediatric intensive care unit population.</p><p><strong>Objectives: </strong>To determine the feasibility of conducting a multicentre, cluster-randomised controlled trial in critically ill children comparing selective decontamination of the digestive tract with standard infection control.</p><p><strong>Design: </strong>Parallel-group pilot cluster-randomised controlled trial with an integrated mixed-methods study.</p><p><strong>Setting: </strong>Six paediatric intensive care units in England.</p><p><strong>Participants: </strong>Children (> 37 weeks corrected gestational age, up to 16 years) requiring mechanical ventilation expected to last for at least 48 hours were eligible for the PICnIC pilot cluster-randomised controlled trial. During the ecology periods, all children admitted to the paediatric intensive care units were eligible. Parents/legal guardians of recruited patients and healthcare professionals working in paediatric intensive care units were eligible for inclusion in the mixed-methods study.</p><p><strong>Interventions: </strong>The interventions in the PICnIC pilot cluster-randomised controlled trial included administration of selective decontamination of the digestive tract as oro-pharyngeal paste and as a suspension given by enteric tube during the period of mechanical ventilation.</p><p><strong>Main outcome measures: </strong>The decision as to whether a definitive cluster-randomised controlled trial is feasible is based on multiple outcomes, including (but not limited to): (1) willingness and ability to recruit eligible patients; (2) adherence to the selective decontamination of the digestive tract intervention; (3) acceptability of the definitive cluster-randomised controlled trial; (4) estimation of recruitment rate; and (5) understanding of potential clinical and ecological outcome measures.</p><p><strong>Results: </strong>A total of 368 children (85% of all those who were eligible) were enrolled in the PICnIC pilot cluster-randomised controlled trial across six paediatric intensive care units: 207 in the baseline phase (Period One) and 161 in the intervention period (Period Two). In sites delivering selective decontamination of the digestive tract, the majority (98%) of children received at least one dose of selective decontamination of the digestive tract, and of these, 68% commenced within the first 6 hours. Consent for the collection of additional swabs was low (44%), though data completeness for potential outcomes, including microbiology data from routine clinical swab testing, was excellent. Recruited children were representative of the wider paediatric intensiv
{"title":"Use of selective gut decontamination in critically ill children: PICnIC a pilot RCT and mixed-methods study.","authors":"Alanna Brown, Paloma Ferrando-Vivas, Mariana Popa, Gema Milla de la Fuente, John Pappachan, Brian H Cuthbertson, Laura Drikite, Richard Feltbower, Theodore Gouliouris, Isobel Sale, Robert Shulman, Lyvonne N Tume, John Myburgh, Kerry Woolfall, David A Harrison, Paul R Mouncey, Kathryn Rowan, Nazima Pathan","doi":"10.3310/HDKV1008","DOIUrl":"10.3310/HDKV1008","url":null,"abstract":"<p><strong>Background: </strong>Healthcare-associated infections are a major cause of morbidity and mortality in critically ill children. In adults, data suggest the use of selective decontamination of the digestive tract may reduce the incidence of healthcare-associated infections. Selective decontamination of the digestive tract has not been evaluated in the paediatric intensive care unit population.</p><p><strong>Objectives: </strong>To determine the feasibility of conducting a multicentre, cluster-randomised controlled trial in critically ill children comparing selective decontamination of the digestive tract with standard infection control.</p><p><strong>Design: </strong>Parallel-group pilot cluster-randomised controlled trial with an integrated mixed-methods study.</p><p><strong>Setting: </strong>Six paediatric intensive care units in England.</p><p><strong>Participants: </strong>Children (> 37 weeks corrected gestational age, up to 16 years) requiring mechanical ventilation expected to last for at least 48 hours were eligible for the PICnIC pilot cluster-randomised controlled trial. During the ecology periods, all children admitted to the paediatric intensive care units were eligible. Parents/legal guardians of recruited patients and healthcare professionals working in paediatric intensive care units were eligible for inclusion in the mixed-methods study.</p><p><strong>Interventions: </strong>The interventions in the PICnIC pilot cluster-randomised controlled trial included administration of selective decontamination of the digestive tract as oro-pharyngeal paste and as a suspension given by enteric tube during the period of mechanical ventilation.</p><p><strong>Main outcome measures: </strong>The decision as to whether a definitive cluster-randomised controlled trial is feasible is based on multiple outcomes, including (but not limited to): (1) willingness and ability to recruit eligible patients; (2) adherence to the selective decontamination of the digestive tract intervention; (3) acceptability of the definitive cluster-randomised controlled trial; (4) estimation of recruitment rate; and (5) understanding of potential clinical and ecological outcome measures.</p><p><strong>Results: </strong>A total of 368 children (85% of all those who were eligible) were enrolled in the PICnIC pilot cluster-randomised controlled trial across six paediatric intensive care units: 207 in the baseline phase (Period One) and 161 in the intervention period (Period Two). In sites delivering selective decontamination of the digestive tract, the majority (98%) of children received at least one dose of selective decontamination of the digestive tract, and of these, 68% commenced within the first 6 hours. Consent for the collection of additional swabs was low (44%), though data completeness for potential outcomes, including microbiology data from routine clinical swab testing, was excellent. Recruited children were representative of the wider paediatric intensiv","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 8","pages":"1-84"},"PeriodicalIF":3.5,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11017160/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139989802","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}
Owen Price, Cat Papastavrou Brooks, Isobel Johnston, Peter McPherson, Helena Goodman, Andrew Grundy, Lindsey Cree, Zahra Motala, Jade Robinson, Michael Doyle, Nicholas Stokes, Christopher J Armitage, Elizabeth Barley, Helen Brooks, Patrick Callaghan, Lesley-Anne Carter, Linda M Davies, Richard J Drake, Karina Lovell, Penny Bee
<p><strong>Background: </strong>Containment (e.g. physical restraint and seclusion) is used frequently in mental health inpatient settings. Containment is associated with serious psychological and physical harms. De-escalation (psychosocial techniques to manage distress without containment) is recommended to manage aggression and other unsafe behaviours, for example self-harm. All National Health Service staff are trained in de-escalation but there is little to no evidence supporting training's effectiveness.</p><p><strong>Objectives: </strong>Objectives were to: (1) qualitatively investigate de-escalation and identify barriers and facilitators to use across the range of adult acute and forensic mental health inpatient settings; (2) co-produce with relevant stakeholders an intervention to enhance de-escalation across these settings; (3) evaluate the intervention's preliminary effect on rates of conflict (e.g. violence, self-harm) and containment (e.g. seclusion and physical restraint) and understand barriers and facilitators to intervention effects.</p><p><strong>Design: </strong>Intervention development informed by Experience-based Co-design and uncontrolled pre and post feasibility evaluation. Systematic reviews and qualitative interviews investigated contextual variation in use and effects of de-escalation. Synthesis of this evidence informed co-design of an intervention to enhance de-escalation. An uncontrolled feasibility trial of the intervention followed. Clinical outcome data were collected over 24 weeks including an 8-week pre-intervention phase, an 8-week embedding and an 8-week post-intervention phase.</p><p><strong>Setting: </strong>Ten inpatient wards (including acute, psychiatric intensive care, low, medium and high secure forensic) in two United Kingdom mental health trusts.</p><p><strong>Participants: </strong>In-patients, clinical staff, managers, carers/relatives and training staff in the target settings.</p><p><strong>Interventions: </strong>Enhancing de-escalation techniques in adult acute and forensic units: Development and evaluation of an evidence-based training intervention (EDITION) interventions included de-escalation training, two novel models of reflective practice, post-incident debriefing and feedback on clinical practice, collaborative prescribing and ward rounds, practice changes around admission, shift handovers and the social and physical environment, and sensory modulation and support planning to reduce patient distress.</p><p><strong>Main outcome measures: </strong>Outcomes measured related to feasibility (recruitment and retention, completion of outcome measures), training outcomes and clinical and safety outcomes. Conflict and containment rates were measured via the Patient-Staff Conflict Checklist. Clinical outcomes were measured using the Attitudes to Containment Measures Questionnaire, Attitudes to Personality Disorder Questionnaire, Violence Prevention Climate Scale, Capabilities, Opportunities, and Motiv
{"title":"Development and evaluation of a de-escalation training intervention in adult acute and forensic units: the EDITION systematic review and feasibility trial.","authors":"Owen Price, Cat Papastavrou Brooks, Isobel Johnston, Peter McPherson, Helena Goodman, Andrew Grundy, Lindsey Cree, Zahra Motala, Jade Robinson, Michael Doyle, Nicholas Stokes, Christopher J Armitage, Elizabeth Barley, Helen Brooks, Patrick Callaghan, Lesley-Anne Carter, Linda M Davies, Richard J Drake, Karina Lovell, Penny Bee","doi":"10.3310/FGGW6874","DOIUrl":"10.3310/FGGW6874","url":null,"abstract":"<p><strong>Background: </strong>Containment (e.g. physical restraint and seclusion) is used frequently in mental health inpatient settings. Containment is associated with serious psychological and physical harms. De-escalation (psychosocial techniques to manage distress without containment) is recommended to manage aggression and other unsafe behaviours, for example self-harm. All National Health Service staff are trained in de-escalation but there is little to no evidence supporting training's effectiveness.</p><p><strong>Objectives: </strong>Objectives were to: (1) qualitatively investigate de-escalation and identify barriers and facilitators to use across the range of adult acute and forensic mental health inpatient settings; (2) co-produce with relevant stakeholders an intervention to enhance de-escalation across these settings; (3) evaluate the intervention's preliminary effect on rates of conflict (e.g. violence, self-harm) and containment (e.g. seclusion and physical restraint) and understand barriers and facilitators to intervention effects.</p><p><strong>Design: </strong>Intervention development informed by Experience-based Co-design and uncontrolled pre and post feasibility evaluation. Systematic reviews and qualitative interviews investigated contextual variation in use and effects of de-escalation. Synthesis of this evidence informed co-design of an intervention to enhance de-escalation. An uncontrolled feasibility trial of the intervention followed. Clinical outcome data were collected over 24 weeks including an 8-week pre-intervention phase, an 8-week embedding and an 8-week post-intervention phase.</p><p><strong>Setting: </strong>Ten inpatient wards (including acute, psychiatric intensive care, low, medium and high secure forensic) in two United Kingdom mental health trusts.</p><p><strong>Participants: </strong>In-patients, clinical staff, managers, carers/relatives and training staff in the target settings.</p><p><strong>Interventions: </strong>Enhancing de-escalation techniques in adult acute and forensic units: Development and evaluation of an evidence-based training intervention (EDITION) interventions included de-escalation training, two novel models of reflective practice, post-incident debriefing and feedback on clinical practice, collaborative prescribing and ward rounds, practice changes around admission, shift handovers and the social and physical environment, and sensory modulation and support planning to reduce patient distress.</p><p><strong>Main outcome measures: </strong>Outcomes measured related to feasibility (recruitment and retention, completion of outcome measures), training outcomes and clinical and safety outcomes. Conflict and containment rates were measured via the Patient-Staff Conflict Checklist. Clinical outcomes were measured using the Attitudes to Containment Measures Questionnaire, Attitudes to Personality Disorder Questionnaire, Violence Prevention Climate Scale, Capabilities, Opportunities, and Motiv","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 3","pages":"1-120"},"PeriodicalIF":3.5,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11017147/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139722314","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}
Steven J Edwards, Charlotta Karner, Tracey Jhita, Samantha Barton, Gemma Marceniuk, Zenas Z N Yiu, Miriam Wittmann
<p><strong>Background: </strong>Atopic dermatitis is a chronic relapsing inflammatory skin condition. One of the most common skin disorders in children, atopic dermatitis typically manifests before the age of 5 years, but it can develop at any age. Atopic dermatitis is characterised by dry, inflamed skin accompanied by intense itchiness (pruritus).</p><p><strong>Objectives: </strong>To appraise the clinical and cost effectiveness of abrocitinib, tralokinumab and upadacitinib within their marketing authorisations as alternative therapies for treating moderate-to-severe atopic dermatitis compared to systemic immunosuppressants (first-line ciclosporin A or second-line dupilumab and baricitinib).</p><p><strong>Data sources: </strong>Studies were identified from an existing systematic review (search date 2019) and update searches of electronic databases (MEDLINE, EMBASE, CENTRAL) to November 2021, from bibliographies of retrieved studies, clinical trial registers and evidence provided by the sponsoring companies of the treatments under review.</p><p><strong>Methods: </strong>A systematic review of the clinical effectiveness literature was carried out and a network meta-analysis undertaken for adults and adolescents at different steps of the treatment pathway. The primary outcome of interest was a combined response of Eczema Area and Severity Index 50 + Dermatology Life Quality Index ≥ 4; where this was consistently unavailable for a step in the pathway, an analysis of Eczema Area and Severity Index 75 was conducted. A de novo economic model was developed to assess cost effectiveness from the perspective of the National Health Service in England. The model structure was informed through systematic review of the economic literature and by consulting clinical experts. Effectiveness data were obtained from the network meta-analysis. Costs and utilities were obtained from the evidence provided by sponsoring companies and standard UK sources.</p><p><strong>Results: </strong>Network meta-analyses indicate that abrocitinib 200 mg and upadacitinib 30 mg may be more effective, and tralokinumab may be less effective than dupilumab and baricitinib as second-line systemic therapies. Abrocitinib 100 mg and upadacitinib 15 mg have a more similar effectiveness to dupilumab. Upadacitinib 30 and 15 mg are likely to be more effective than ciclosporin A as a first-line therapy. Upadacitinib 15 mg, abrocitinib 200 and 100 mg may be more effective than dupilumab in adolescents. The cost effectiveness of abrocitinib and upadacitinib for both doses is dependent on the subgroup of interest. Tralokinumab can be considered cost-effective as a second-line systemic therapy owing to greater cost savings per quality-adjusted life-year lost.</p><p><strong>Conclusions: </strong>The primary strength of the analysis of the three new drugs compared with current practice for each of the subpopulations is the consistent approach to the assessment of clinical and cost effectiveness. Howeve
{"title":"Abrocitinib, tralokinumab and upadacitinib for treating moderate-to-severe atopic dermatitis.","authors":"Steven J Edwards, Charlotta Karner, Tracey Jhita, Samantha Barton, Gemma Marceniuk, Zenas Z N Yiu, Miriam Wittmann","doi":"10.3310/LEXB9006","DOIUrl":"10.3310/LEXB9006","url":null,"abstract":"<p><strong>Background: </strong>Atopic dermatitis is a chronic relapsing inflammatory skin condition. One of the most common skin disorders in children, atopic dermatitis typically manifests before the age of 5 years, but it can develop at any age. Atopic dermatitis is characterised by dry, inflamed skin accompanied by intense itchiness (pruritus).</p><p><strong>Objectives: </strong>To appraise the clinical and cost effectiveness of abrocitinib, tralokinumab and upadacitinib within their marketing authorisations as alternative therapies for treating moderate-to-severe atopic dermatitis compared to systemic immunosuppressants (first-line ciclosporin A or second-line dupilumab and baricitinib).</p><p><strong>Data sources: </strong>Studies were identified from an existing systematic review (search date 2019) and update searches of electronic databases (MEDLINE, EMBASE, CENTRAL) to November 2021, from bibliographies of retrieved studies, clinical trial registers and evidence provided by the sponsoring companies of the treatments under review.</p><p><strong>Methods: </strong>A systematic review of the clinical effectiveness literature was carried out and a network meta-analysis undertaken for adults and adolescents at different steps of the treatment pathway. The primary outcome of interest was a combined response of Eczema Area and Severity Index 50 + Dermatology Life Quality Index ≥ 4; where this was consistently unavailable for a step in the pathway, an analysis of Eczema Area and Severity Index 75 was conducted. A de novo economic model was developed to assess cost effectiveness from the perspective of the National Health Service in England. The model structure was informed through systematic review of the economic literature and by consulting clinical experts. Effectiveness data were obtained from the network meta-analysis. Costs and utilities were obtained from the evidence provided by sponsoring companies and standard UK sources.</p><p><strong>Results: </strong>Network meta-analyses indicate that abrocitinib 200 mg and upadacitinib 30 mg may be more effective, and tralokinumab may be less effective than dupilumab and baricitinib as second-line systemic therapies. Abrocitinib 100 mg and upadacitinib 15 mg have a more similar effectiveness to dupilumab. Upadacitinib 30 and 15 mg are likely to be more effective than ciclosporin A as a first-line therapy. Upadacitinib 15 mg, abrocitinib 200 and 100 mg may be more effective than dupilumab in adolescents. The cost effectiveness of abrocitinib and upadacitinib for both doses is dependent on the subgroup of interest. Tralokinumab can be considered cost-effective as a second-line systemic therapy owing to greater cost savings per quality-adjusted life-year lost.</p><p><strong>Conclusions: </strong>The primary strength of the analysis of the three new drugs compared with current practice for each of the subpopulations is the consistent approach to the assessment of clinical and cost effectiveness. Howeve","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 4","pages":"1-113"},"PeriodicalIF":3.5,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11017148/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139722324","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}
Adwoa Parker, Catherine Arundel, Laura Clark, Elizabeth Coleman, Laura Doherty, Catherine Elizabeth Hewitt, David Beard, Peter Bower, Cindy Cooper, Lucy Culliford, Declan Devane, Richard Emsley, Sandra Eldridge, Sandra Galvin, Katie Gillies, Alan Montgomery, Christopher J Sutton, Shaun Treweek, David J Torgerson
<p><strong>Background: </strong>Randomised controlled trials ('trials') are susceptible to poor participant recruitment and retention. Studies Within A Trial are the strongest methods for testing the effectiveness of strategies to improve recruitment and retention. However, relatively few of these have been conducted.</p><p><strong>Objectives: </strong>PROMoting THE Use of Studies Within A Trial aimed to facilitate at least 25 Studies Within A Trial evaluating recruitment or retention strategies. We share our experience of delivering the PROMoting THE Use of Studies Within A Trial programme, and the lessons learnt for undertaking randomised Studies Within A Trial.</p><p><strong>Design: </strong>A network of 10 Clinical Trials Units and 1 primary care research centre committed to conducting randomised controlled Studies Within A Trial of recruitment and/or retention strategies was established. Promising recruitment and retention strategies were identified from various sources including Cochrane systematic reviews, the Study Within A Trial Repository, and existing prioritisation exercises, which were reviewed by patient and public members to create an initial priority list of seven recruitment and eight retention interventions. Host trial teams could apply for funding and receive support from the PROMoting THE Use of Studies Within A Trial team to undertake Studies Within A Trial. We also tested the feasibility of undertaking co-ordinated Studies Within A Trial, across multiple host trials simultaneously.</p><p><strong>Setting: </strong>Clinical trials unit-based trials recruiting or following up participants in any setting in the United Kingdom were eligible.</p><p><strong>Participants: </strong>Clinical trials unit-based teams undertaking trials in any clinical context in the United Kingdom.</p><p><strong>Interventions: </strong>Funding of up to £5000 and support from the PROMoting THE Use of Studies Within A Trial team to design, implement and report Studies Within A Trial.</p><p><strong>Main outcome measures: </strong>Number of host trials funded.</p><p><strong>Results: </strong>Forty-two Studies Within A Trial were funded (31 host trials), across 12 Clinical Trials Units. The mean cost of a Study Within A Trial was £3535. Twelve Studies Within A Trial tested the same strategy across multiple host trials using a co-ordinated Study Within A Trial design, and four used a factorial design. Two recruitment and five retention strategies were evaluated in more than one host trial. PROMoting THE Use of Studies Within A Trial will add 18% more Studies Within A Trial to the Cochrane systematic review of recruitment strategies, and 79% more Studies Within A Trial to the Cochrane review of retention strategies. For retention, we found that pre-notifying participants by card, letter or e-mail before sending questionnaires was effective, as was the use of pens, and sending personalised text messages to improve questionnaire response. We highlight key lesson
{"title":"Undertaking Studies Within A Trial to evaluate recruitment and retention strategies for randomised controlled trials: lessons learnt from the PROMETHEUS research programme.","authors":"Adwoa Parker, Catherine Arundel, Laura Clark, Elizabeth Coleman, Laura Doherty, Catherine Elizabeth Hewitt, David Beard, Peter Bower, Cindy Cooper, Lucy Culliford, Declan Devane, Richard Emsley, Sandra Eldridge, Sandra Galvin, Katie Gillies, Alan Montgomery, Christopher J Sutton, Shaun Treweek, David J Torgerson","doi":"10.3310/HTQW3107","DOIUrl":"10.3310/HTQW3107","url":null,"abstract":"<p><strong>Background: </strong>Randomised controlled trials ('trials') are susceptible to poor participant recruitment and retention. Studies Within A Trial are the strongest methods for testing the effectiveness of strategies to improve recruitment and retention. However, relatively few of these have been conducted.</p><p><strong>Objectives: </strong>PROMoting THE Use of Studies Within A Trial aimed to facilitate at least 25 Studies Within A Trial evaluating recruitment or retention strategies. We share our experience of delivering the PROMoting THE Use of Studies Within A Trial programme, and the lessons learnt for undertaking randomised Studies Within A Trial.</p><p><strong>Design: </strong>A network of 10 Clinical Trials Units and 1 primary care research centre committed to conducting randomised controlled Studies Within A Trial of recruitment and/or retention strategies was established. Promising recruitment and retention strategies were identified from various sources including Cochrane systematic reviews, the Study Within A Trial Repository, and existing prioritisation exercises, which were reviewed by patient and public members to create an initial priority list of seven recruitment and eight retention interventions. Host trial teams could apply for funding and receive support from the PROMoting THE Use of Studies Within A Trial team to undertake Studies Within A Trial. We also tested the feasibility of undertaking co-ordinated Studies Within A Trial, across multiple host trials simultaneously.</p><p><strong>Setting: </strong>Clinical trials unit-based trials recruiting or following up participants in any setting in the United Kingdom were eligible.</p><p><strong>Participants: </strong>Clinical trials unit-based teams undertaking trials in any clinical context in the United Kingdom.</p><p><strong>Interventions: </strong>Funding of up to £5000 and support from the PROMoting THE Use of Studies Within A Trial team to design, implement and report Studies Within A Trial.</p><p><strong>Main outcome measures: </strong>Number of host trials funded.</p><p><strong>Results: </strong>Forty-two Studies Within A Trial were funded (31 host trials), across 12 Clinical Trials Units. The mean cost of a Study Within A Trial was £3535. Twelve Studies Within A Trial tested the same strategy across multiple host trials using a co-ordinated Study Within A Trial design, and four used a factorial design. Two recruitment and five retention strategies were evaluated in more than one host trial. PROMoting THE Use of Studies Within A Trial will add 18% more Studies Within A Trial to the Cochrane systematic review of recruitment strategies, and 79% more Studies Within A Trial to the Cochrane review of retention strategies. For retention, we found that pre-notifying participants by card, letter or e-mail before sending questionnaires was effective, as was the use of pens, and sending personalised text messages to improve questionnaire response. We highlight key lesson","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 2","pages":"1-114"},"PeriodicalIF":3.5,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11017159/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139702330","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}
Tamara Ondruskova, Rachel Royston, Michael Absoud, Gareth Ambler, Chen Qu, Jacqueline Barnes, Rachael Hunter, Monica Panca, Marinos Kyriakopoulos, Kate Oulton, Eleni Paliokosta, Aditya Narain Sharma, Vicky Slonims, Una Summerson, Alastair Sutcliffe, Megan Thomas, Brindha Dhandapani, Helen Leonard, Angela Hassiotis
<p><strong>Background: </strong>Stepping Stones Triple P is an adapted intervention for parents of young children with developmental disabilities who display behaviours that challenge, aiming at teaching positive parenting techniques and promoting a positive parent-child relationship.</p><p><strong>Objective: </strong>To evaluate the clinical and cost-effectiveness of level 4 Stepping Stones Triple P in reducing behaviours that challenge in children with moderate to severe intellectual disabilities.</p><p><strong>Design, setting, participants: </strong>A parallel two-arm pragmatic multisite single-blind randomised controlled trial recruited a total of 261 dyads (parent and child). The children were aged 30-59 months and had moderate to severe intellectual disabilities. Participants were randomised, using a 3 : 2 allocation ratio, into the intervention arm (Stepping Stones Triple P; <i>n</i> = 155) or treatment as usual arm (<i>n</i> = 106). Participants were recruited from four study sites in Blackpool, North and South London and Newcastle.</p><p><strong>Intervention: </strong>Level 4 Stepping Stones Triple P consists of six group sessions and three individual phone or face-to-face contacts over 9 weeks. These were changed to remote sessions after 16 March 2020 due to the coronavirus disease 2019 pandemic.</p><p><strong>Main outcome measure: </strong>The primary outcome measure was the parent-reported Child Behaviour Checklist, which assesses the severity of behaviours that challenge.</p><p><strong>Results: </strong>We found a small non-significant difference in the mean Child Behaviour Checklist scores (-4.23, 95% CI -9.98 to 1.52, <i>p</i> = 0.146) in the intervention arm compared to treatment as usual at 12 months. Per protocol and complier average causal effect sensitivity analyses, which took into consideration the number of sessions attended, showed the Child Behaviour Checklist mean score difference at 12 months was lower in the intervention arm by -10.77 (95% CI -19.12 to -2.42, <i>p</i> = 0.014) and -11.53 (95% CI -26.97 to 3.91, <i>p</i> = 0.143), respectively. The Child Behaviour Checklist mean score difference between participants who were recruited before and after the coronavirus disease 2019 pandemic was estimated as -7.12 (95% CI -13.44 to -0.81) and 7.61 (95% CI -5.43 to 20.64), respectively (<i>p</i> = 0.046), suggesting that any effect pre-pandemic may have reversed during the pandemic. There were no differences in all secondary measures. Stepping Stones Triple P is probably value for money to deliver (-£1057.88; 95% CI -£3218.6 to -£46.67), but decisions to roll this out as an alternative to existing parenting interventions or treatment as usual may be dependent on policymaker willingness to invest in early interventions to reduce behaviours that challenge. Parents reported the intervention boosted their confidence and skills, and the group format enabled them to learn from others and benefit from peer support. There were 20 s
背景阶梯石三P "是一种经过调整的干预措施，适用于有挑战行为的发育障碍幼儿的父母，旨在教授积极的养育技巧和促进积极的亲子关系：评估第四级 "阶梯石三P "在减少中重度智障儿童挑战行为方面的临床和成本效益：一项平行双臂多地点单盲随机对照试验共招募了 261 对父母和儿童。这些儿童的年龄在 30-59 个月之间，患有中度至重度智障。参与者按照 3 : 2 的分配比例被随机分配到干预组（阶梯石三P；n = 155）或常规治疗组（n = 106）。参与者从布莱克浦、伦敦北部和南部以及纽卡斯尔的四个研究地点招募：4 级阶梯石三P疗法包括六次小组课程和三次个人电话或面对面联系，为期 9 周。由于2019年冠状病毒疾病大流行，这些课程在2020年3月16日后改为远程课程。主要结果测量：主要结果测量是家长报告的儿童行为清单，该清单评估挑战行为的严重程度：结果：我们发现，与 12 个月时的常规治疗相比，干预组的儿童行为检查表平均得分差异很小（-4.23，95% CI -9.98 至 1.52，p = 0.146）。根据干预方案和干预者平均因果效应进行的敏感性分析（考虑了参加治疗的次数）显示，干预组在12个月时的儿童行为检查表平均得分差异分别为-10.77（95% CI -19.12至-2.42，p = 0.014）和-11.53（95% CI -26.97至3.91，p = 0.143）。据估计，2019年冠状病毒病大流行前后招募的参与者之间的儿童行为检查表平均得分差异分别为-7.12（95% CI -13.44至-0.81）和7.61（95% CI -5.43至20.64）（p = 0.046），这表明大流行前的任何影响可能在大流行期间逆转。所有次要指标均无差异。阶梯石三P "的实施可能物有所值（-1057.88英镑；95% CI-3218.6英镑至-46.67英镑），但是否将其作为现有亲职干预或常规治疗的替代方案进行推广，可能取决于政策制定者是否愿意投资于早期干预，以减少挑战行为。家长们表示，干预措施增强了他们的信心和技能，小组形式使他们能够向他人学习，并从同伴支持中受益。研究期间共报告了20起严重不良事件，但均与干预措施无关：局限性："踏脚石三重P "干预组的参与率较低，而且在招募和实施干预时遇到了与2019年冠状病毒病相关的挑战：第四级 "踏脚石 "三重P疗法并没有减少中度至重度智障幼儿的早发挑战行为。然而，对那些接受了足够剂量干预的儿童来说，其行为还是有影响的。鉴于挑战行为对人和人的社会网络造成的长期后果，"踏脚石 "三P疗法很有可能至少不会增加成本，因此值得考虑将其作为一种早期治疗方案：今后的工作：进一步的研究应探讨在这一人群中实施针对挑战行为的亲子小组，以及最佳的实施模式，以最大限度地提高参与度和后续效果：本研究已注册为 NCT03086876 (https://www.clinicaltrials.gov/ct2/show/NCT03086876?term=Hassiotis±Angela&draw=1&rank=1)：该奖项由英国国家健康与护理研究所（NIHR）健康技术评估项目资助（NIHR奖项编号：HTA 15/162/02），全文发表于《健康技术评估》（Health Technology Assessment）第28卷第6期。更多奖项信息请参阅 NIHR Funding and Awards 网站。
{"title":"Clinical and cost-effectiveness of an adapted intervention for preschoolers with moderate to severe intellectual disabilities displaying behaviours that challenge: the EPICC-ID RCT.","authors":"Tamara Ondruskova, Rachel Royston, Michael Absoud, Gareth Ambler, Chen Qu, Jacqueline Barnes, Rachael Hunter, Monica Panca, Marinos Kyriakopoulos, Kate Oulton, Eleni Paliokosta, Aditya Narain Sharma, Vicky Slonims, Una Summerson, Alastair Sutcliffe, Megan Thomas, Brindha Dhandapani, Helen Leonard, Angela Hassiotis","doi":"10.3310/JKTY6144","DOIUrl":"10.3310/JKTY6144","url":null,"abstract":"<p><strong>Background: </strong>Stepping Stones Triple P is an adapted intervention for parents of young children with developmental disabilities who display behaviours that challenge, aiming at teaching positive parenting techniques and promoting a positive parent-child relationship.</p><p><strong>Objective: </strong>To evaluate the clinical and cost-effectiveness of level 4 Stepping Stones Triple P in reducing behaviours that challenge in children with moderate to severe intellectual disabilities.</p><p><strong>Design, setting, participants: </strong>A parallel two-arm pragmatic multisite single-blind randomised controlled trial recruited a total of 261 dyads (parent and child). The children were aged 30-59 months and had moderate to severe intellectual disabilities. Participants were randomised, using a 3 : 2 allocation ratio, into the intervention arm (Stepping Stones Triple P; <i>n</i> = 155) or treatment as usual arm (<i>n</i> = 106). Participants were recruited from four study sites in Blackpool, North and South London and Newcastle.</p><p><strong>Intervention: </strong>Level 4 Stepping Stones Triple P consists of six group sessions and three individual phone or face-to-face contacts over 9 weeks. These were changed to remote sessions after 16 March 2020 due to the coronavirus disease 2019 pandemic.</p><p><strong>Main outcome measure: </strong>The primary outcome measure was the parent-reported Child Behaviour Checklist, which assesses the severity of behaviours that challenge.</p><p><strong>Results: </strong>We found a small non-significant difference in the mean Child Behaviour Checklist scores (-4.23, 95% CI -9.98 to 1.52, <i>p</i> = 0.146) in the intervention arm compared to treatment as usual at 12 months. Per protocol and complier average causal effect sensitivity analyses, which took into consideration the number of sessions attended, showed the Child Behaviour Checklist mean score difference at 12 months was lower in the intervention arm by -10.77 (95% CI -19.12 to -2.42, <i>p</i> = 0.014) and -11.53 (95% CI -26.97 to 3.91, <i>p</i> = 0.143), respectively. The Child Behaviour Checklist mean score difference between participants who were recruited before and after the coronavirus disease 2019 pandemic was estimated as -7.12 (95% CI -13.44 to -0.81) and 7.61 (95% CI -5.43 to 20.64), respectively (<i>p</i> = 0.046), suggesting that any effect pre-pandemic may have reversed during the pandemic. There were no differences in all secondary measures. Stepping Stones Triple P is probably value for money to deliver (-£1057.88; 95% CI -£3218.6 to -£46.67), but decisions to roll this out as an alternative to existing parenting interventions or treatment as usual may be dependent on policymaker willingness to invest in early interventions to reduce behaviours that challenge. Parents reported the intervention boosted their confidence and skills, and the group format enabled them to learn from others and benefit from peer support. There were 20 s","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 6","pages":"1-94"},"PeriodicalIF":3.5,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11017145/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139702331","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}
Alex Todhunter-Brown, Lorna Booth, Pauline Campbell, Brenda Cheer, Julie Cowie, Andrew Elders, Suzanne Hagen, Karen Jankulak, Helen Mason, Clare Millington, Margaret Ogden, Charlotte Paterson, Davina Richardson, Debs Smith, Jonathan Sutcliffe, Katie Thomson, Claire Torrens, Doreen McClurg
<p><strong>Background: </strong>Up to 30% of children have constipation at some stage in their life. Although often short-lived, in one-third of children it progresses to chronic functional constipation, potentially with overflow incontinence. Optimal management strategies remain unclear.</p><p><strong>Objective: </strong>To determine the most effective interventions, and combinations and sequences of interventions, for childhood chronic functional constipation, and understand how they can best be implemented.</p><p><strong>Methods: </strong>Key stakeholders, comprising two parents of children with chronic functional constipation, two adults who experienced childhood chronic functional constipation and four health professional/continence experts, contributed throughout the research. We conducted pragmatic mixed-method reviews. For all reviews, included studies focused on any interventions/strategies, delivered in any setting, to improve any outcomes in children (0-18 years) with a clinical diagnosis of chronic functional constipation (excluding studies of diagnosis/assessment) included. Dual reviewers applied inclusion criteria and assessed risk of bias. One reviewer extracted data, checked by a second reviewer. <b>Scoping review:</b> We systematically searched electronic databases (including Medical Literature Analysis and Retrieval System Online, Excerpta Medica Database, Cumulative Index to Nursing and Allied Health Literature) (January 2011 to March 2020) and grey literature, including studies (any design) reporting any intervention/strategy. Data were coded, tabulated and mapped. Research quality was not evaluated. <b>Systematic reviews of the evidence of effectiveness:</b> For each different intervention, we included existing systematic reviews judged to be low risk of bias (using the Risk of Bias Assessment Tool for Systematic Reviews), updating any meta-analyses with new randomised controlled trials. Where there was no existing low risk of bias systematic reviews, we included randomised controlled trials and other primary studies. The risk of bias was judged using design-specific tools. Evidence was synthesised narratively, and a process of considered judgement was used to judge certainty in the evidence as high, moderate, low, very low or insufficient evidence. <b>Economic synthesis:</b> Included studies (any design, English-language) detailed intervention-related costs. Studies were categorised as cost-consequence, cost-effectiveness, cost-utility or cost-benefit, and reporting quality evaluated using the consensus health economic criteria checklist. <b>Systematic review of implementation factors:</b> Included studies reported data relating to implementation barriers or facilitators. Using a best-fit framework synthesis approach, factors were synthesised around the consolidated framework for implementation research domains.</p><p><strong>Results: </strong>Stakeholders prioritised outcomes, developed a model which informed evidence synth
{"title":"Strategies used for childhood chronic functional constipation: the SUCCESS evidence synthesis.","authors":"Alex Todhunter-Brown, Lorna Booth, Pauline Campbell, Brenda Cheer, Julie Cowie, Andrew Elders, Suzanne Hagen, Karen Jankulak, Helen Mason, Clare Millington, Margaret Ogden, Charlotte Paterson, Davina Richardson, Debs Smith, Jonathan Sutcliffe, Katie Thomson, Claire Torrens, Doreen McClurg","doi":"10.3310/PLTR9622","DOIUrl":"10.3310/PLTR9622","url":null,"abstract":"<p><strong>Background: </strong>Up to 30% of children have constipation at some stage in their life. Although often short-lived, in one-third of children it progresses to chronic functional constipation, potentially with overflow incontinence. Optimal management strategies remain unclear.</p><p><strong>Objective: </strong>To determine the most effective interventions, and combinations and sequences of interventions, for childhood chronic functional constipation, and understand how they can best be implemented.</p><p><strong>Methods: </strong>Key stakeholders, comprising two parents of children with chronic functional constipation, two adults who experienced childhood chronic functional constipation and four health professional/continence experts, contributed throughout the research. We conducted pragmatic mixed-method reviews. For all reviews, included studies focused on any interventions/strategies, delivered in any setting, to improve any outcomes in children (0-18 years) with a clinical diagnosis of chronic functional constipation (excluding studies of diagnosis/assessment) included. Dual reviewers applied inclusion criteria and assessed risk of bias. One reviewer extracted data, checked by a second reviewer. <b>Scoping review:</b> We systematically searched electronic databases (including Medical Literature Analysis and Retrieval System Online, Excerpta Medica Database, Cumulative Index to Nursing and Allied Health Literature) (January 2011 to March 2020) and grey literature, including studies (any design) reporting any intervention/strategy. Data were coded, tabulated and mapped. Research quality was not evaluated. <b>Systematic reviews of the evidence of effectiveness:</b> For each different intervention, we included existing systematic reviews judged to be low risk of bias (using the Risk of Bias Assessment Tool for Systematic Reviews), updating any meta-analyses with new randomised controlled trials. Where there was no existing low risk of bias systematic reviews, we included randomised controlled trials and other primary studies. The risk of bias was judged using design-specific tools. Evidence was synthesised narratively, and a process of considered judgement was used to judge certainty in the evidence as high, moderate, low, very low or insufficient evidence. <b>Economic synthesis:</b> Included studies (any design, English-language) detailed intervention-related costs. Studies were categorised as cost-consequence, cost-effectiveness, cost-utility or cost-benefit, and reporting quality evaluated using the consensus health economic criteria checklist. <b>Systematic review of implementation factors:</b> Included studies reported data relating to implementation barriers or facilitators. Using a best-fit framework synthesis approach, factors were synthesised around the consolidated framework for implementation research domains.</p><p><strong>Results: </strong>Stakeholders prioritised outcomes, developed a model which informed evidence synth","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 5","pages":"1-266"},"PeriodicalIF":3.5,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11017632/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139722325","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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