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1.  Proceedings of the 14th annual conference of INEBRIA 
Holloway, Aisha S. | Ferguson, Jennifer | Landale, Sarah | Cariola, Laura | Newbury-Birch, Dorothy | Flynn, Amy | Knight, John R. | Sherritt, Lon | Harris, Sion K. | O’Donnell, Amy J. | Kaner, Eileen | Hanratty, Barbara | Loree, Amy M. | Yonkers, Kimberly A. | Ondersma, Steven J. | Gilstead-Hayden, Kate | Martino, Steve | Adam, Angeline | Schwartz, Robert P. | Wu, Li-Tzy | Subramaniam, Geetha | Sharma, Gaurav | McNeely, Jennifer | Berman, Anne H. | Kolaas, Karoline | Petersén, Elisabeth | Bendtsen, Preben | Hedman, Erik | Linderoth, Catharina | Müssener, Ulrika | Sinadinovic, Kristina | Spak, Fredrik | Gremyr, Ida | Thurang, Anna | Mitchell, Ann M. | Finnell, Deborah | Savage, Christine L. | Mahmoud, Khadejah F. | Riordan, Benjamin C. | Conner, Tamlin S. | Flett, Jayde A. M. | Scarf, Damian | McRee, Bonnie | Vendetti, Janice | Gallucci, Karen Steinberg | Robaina, Kate | Clark, Brendan J. | Jones, Jacqueline | Reed, Kathryne D. | Hodapp, Rachel M. | Douglas, Ivor | Burnham, Ellen L. | Aagaard, Laura | Cook, Paul F. | Harris, Brett R. | Yu, Jiang | Wolff, Margaret | Rogers, Meighan | Barbosa, Carolina | Wedehase, Brendan J. | Dunlap, Laura J. | Mitchell, Shannon G. | Dusek, Kristi A. | Gryczynski, Jan | Kirk, Arethusa S. | Oros, Marla T. | Hosler, Colleen | O’Grady, Kevin E. | Brown, Barry S. | Angus, Colin | Sherborne, Sidney | Gillespie, Duncan | Meier, Petra | Brennan, Alan | de Vargas, Divane | Soares, Janaina | Castelblanco, Donna | Doran, Kelly M. | Wittman, Ian | Shelley, Donna | Rotrosen, John | Gelberg, Lillian | Edelman, E. Jennifer | Maisto, Stephen A. | Hansen, Nathan B. | Cutter, Christopher J. | Deng, Yanhong | Dziura, James | Fiellin, Lynn E. | O’Connor, Patrick G. | Bedimo, Roger | Gibert, Cynthia | Marconi, Vincent C. | Rimland, David | Rodriguez-Barradas, Maria C. | Simberkoff, Michael S. | Justice, Amy C. | Bryant, Kendall J. | Fiellin, David A. | Giles, Emma L. | Coulton, Simon | Deluca, Paolo | Drummond, Colin | Howel, Denise | McColl, Elaine | McGovern, Ruth | Scott, Stephanie | Stamp, Elaine | Sumnall, Harry | Vale, Luke | Alabani, Viviana | Atkinson, Amanda | Boniface, Sadie | Frankham, Jo | Gilvarry, Eilish | Hendrie, Nadine | Howe, Nicola | McGeechan, Grant J. | Ramsey, Amy | Stanley, Grant | Clephane, Justine | Gardiner, David | Holmes, John | Martin, Neil | Shevills, Colin | Soutar, Melanie | Chi, Felicia W. | Weisner, Constance | Ross, Thekla B. | Mertens, Jennifer | Sterling, Stacy A. | Shorter, Gillian W. | Heather, Nick | Bray, Jeremy | Cohen, Hildie A. | McPherson, Tracy L. | Adam, Cyrille | López-Pelayo, Hugo | Gual, Antoni | Segura-Garcia, Lidia | Colom, Joan | Ornelas, India J. | Doyle, Suzanne | Donovan, Dennis | Duran, Bonnie | Torres, Vanessa | Gaume, Jacques | Grazioli, Véronique | Fortini, Cristiana | Paroz, Sophie | Bertholet, Nicolas | Daeppen, Jean-Bernard | Satterfield, Jason M. | Gregorich, Steven | Alvarado, Nicholas J. | Muñoz, Ricardo | Kulieva, Gozel | Vijayaraghavan, Maya | Adam, Angéline | Cunningham, John A. | Díaz, Estela | Palacio-Vieira, Jorge | Godinho, Alexandra | Kushir, Vladyslav | O’Brien, Kimberly H. M. | Aguinaldo, Laika D. | Sellers, Christina M. | Spirito, Anthony | Chang, Grace | Blake-Lamb, Tiffany | LaFave, Lea R. Ayers | Thies, Kathleen M. | Pepin, Amy L. | Sprangers, Kara E. | Bradley, Martha | Jorgensen, Shasta | Catano, Nico A. | Murray, Adelaide R. | Schachter, Deborah | Andersen, Ronald M. | Rey, Guillermina Natera | Vahidi, Mani | Rico, Melvin W. | Baumeister, Sebastian E. | Johansson, Magnus | Sinadinovic, Christina | Hermansson, Ulric | Andreasson, Sven | O’Grady, Megan A. | Kapoor, Sandeep | Akkari, Cherine | Bernal, Camila | Pappacena, Kristen | Morley, Jeanne | Auerbach, Mark | Neighbors, Charles J. | Kwon, Nancy | Conigliaro, Joseph | Morgenstern, Jon | Magill, Molly | Apodaca, Timothy R. | Borsari, Brian | Hoadley, Ariel | Scott Tonigan, J. | Moyers, Theresa | Fitzgerald, Niamh M. | Schölin, Lisa | Barticevic, Nicolas | Zuzulich, Soledad | Poblete, Fernando | Norambuena, Pablo | Sacco, Paul | Ting, Laura | Beaulieu, Michele | Wallace, Paul George | Andrews, Matthew | Daley, Kate | Shenker, Don | Gallagher, Louise | Watson, Rod | Weaver, Tim | Bruguera, Pol | Oliveras, Clara | Gavotti, Carolina | Barrio, Pablo | Braddick, Fleur | Miquel, Laia | Suárez, Montse | Bruguera, Carla | Brown, Richard L. | Capell, Julie Whelan | Paul Moberg, D. | Maslowsky, Julie | Saunders, Laura A. | McCormack, Ryan P. | Scheidell, Joy | Gonzalez, Mirelis | Bauroth, Sabrina | Liu, Weiwei | Lindsay, Dawn L. | Lincoln, Piper | Hagle, Holly | Wallhed Finn, Sara | Hammarberg, Anders | Andréasson, Sven | King, Sarah E. | Vargo, Rachael | Kameg, Brayden N. | Acquavita, Shauna P. | Van Loon, Ruth Anne | Smith, Rachel | Brehm, Bonnie J. | Diers, Tiffiny | Kim, Karissa | Barker, Andrea | Jones, Ashley L. | Skinner, Asheley C. | Hinman, Agatha | Svikis, Dace S. | Thacker, Casey L. | Resnicow, Ken | Beatty, Jessica R. | Janisse, James | Puder, Karoline | Bakshi, Ann-Sofie | Milward, Joanna M. | Kimergard, Andreas | Garnett, Claire V. | Crane, David | Brown, Jamie | West, Robert | Michie, Susan | Rosendahl, Ingvar | Andersson, Claes | Gajecki, Mikael | Blankers, Matthijs | Donoghue, Kim | Lynch, Ellen | Maconochie, Ian | Phillips, Ceri | Pockett, Rhys | Phillips, Tom | Patton, R. | Russell, Ian | Strang, John | Stewart, Maureen T. | Quinn, Amity E. | Brolin, Mary | Evans, Brooke | Horgan, Constance M. | Liu, Junqing | McCree, Fern | Kanovsky, Doug | Oberlander, Tyler | Zhang, Huan | Hamlin, Ben | Saunders, Robert | Barton, Mary B. | Scholle, Sarah H. | Santora, Patricia | Bhatt, Chirag | Ahmed, Kazi | Hodgkin, Dominic | Gao, Wenwu | Merrick, Elizabeth L. | Drebing, Charles E. | Larson, Mary Jo | Sharma, Monica | Petry, Nancy M. | Saitz, Richard | Weisner, Constance M. | Young-Wolff, Kelly C. | Lu, Wendy Y. | Blosnich, John R. | Lehavot, Keren | Glass, Joseph E. | Williams, Emily C. | Bensley, Kara M. | Chan, Gary | Dombrowski, Julie | Fortney, John | Rubinsky, Anna D. | Lapham, Gwen T. | Forray, Ariadna | Olmstead, Todd A. | Gilstad-Hayden, Kathryn | Kershaw, Trace | Dillon, Pamela | Weaver, Michael F. | Grekin, Emily R. | Ellis, Jennifer D. | McGoron, Lucy | McGoron, Lucy
doi:10.1186/s13722-017-0087-8
PMCID: PMC5606215
2.  Robot Assisted Training for the Upper Limb after Stroke (RATULS): study protocol for a randomised controlled trial 
Trials  2017;18:340.
Background
Loss of arm function is a common and distressing consequence of stroke. We describe the protocol for a pragmatic, multicentre randomised controlled trial to determine whether robot-assisted training improves upper limb function following stroke.
Methods/design
Study design: a pragmatic, three-arm, multicentre randomised controlled trial, economic analysis and process evaluation.
Setting: NHS stroke services.
Participants: adults with acute or chronic first-ever stroke (1 week to 5 years post stroke) causing moderate to severe upper limb functional limitation.
Randomisation groups:
1. Robot-assisted training using the InMotion robotic gym system for 45 min, three times/week for 12 weeks
2. Enhanced upper limb therapy for 45 min, three times/week for 12 weeks
3. Usual NHS care in accordance with local clinical practice
Randomisation: individual participant randomisation stratified by centre, time since stroke, and severity of upper limb impairment.
Primary outcome: upper limb function measured by the Action Research Arm Test (ARAT) at 3 months post randomisation.
Secondary outcomes: upper limb impairment (Fugl-Meyer Test), activities of daily living (Barthel ADL Index), quality of life (Stroke Impact Scale, EQ-5D-5L), resource use, cost per quality-adjusted life year and adverse events, at 3 and 6 months.
Blinding: outcomes are undertaken by blinded assessors.
Economic analysis: micro-costing and economic evaluation of interventions compared to usual NHS care. A within-trial analysis, with an economic model will be used to extrapolate longer-term costs and outcomes.
Process evaluation: semi-structured interviews with participants and professionals to seek their views and experiences of the rehabilitation that they have received or provided, and factors affecting the implementation of the trial.
Sample size: allowing for 10% attrition, 720 participants provide 80% power to detect a 15% difference in successful outcome between each of the treatment pairs. Successful outcome definition: baseline ARAT 0–7 must improve by 3 or more points; baseline ARAT 8–13 improve by 4 or more points; baseline ARAT 14–19 improve by 5 or more points; baseline ARAT 20–39 improve by 6 or more points.
Discussion
The results from this trial will determine whether robot-assisted training improves upper limb function post stroke.
Trial registration
ISRCTN, identifier: ISRCTN69371850. Registered 4 October 2013.
Electronic supplementary material
The online version of this article (doi:10.1186/s13063-017-2083-4) contains supplementary material, which is available to authorized users.
doi:10.1186/s13063-017-2083-4
PMCID: PMC5520386
Stroke; Arm; Rehabilitation; Robotics; RCT; Cost-effectiveness analysis; Parallel process evaluation
3.  Choosing the target difference (‘effect size’) for a randomised controlled trial - DELTA2 guidance protocol 
Trials  2017;18:271.
Background
A key step in the design of a randomised controlled trial (RCT) is the estimation of the number of participants needed. By far the most common approach is to specify a target difference and then estimate the corresponding sample size; this sample size is chosen to provide reassurance that the trial will have high statistical power to detect such a difference between the randomised groups (at the planned statistical significance level). The sample size has many implications for the conduct of the study, as well as carrying scientific and ethical aspects to its choice. Despite the critical role of the target difference for the primary outcome in the design of an RCT, the manner in which it is determined has received little attention. This article reports the protocol of the Difference ELicitation in TriAls (DELTA2) project, which will produce guidance on the specification and reporting of the target difference for the primary outcome in a sample size calculation for RCTs.
Methods/design
The DELTA2 project has five components: systematic literature reviews of recent methodological developments (stage 1) and existing funder guidance (stage 2); a Delphi study (stage 3); a 2-day consensus meeting bringing together researchers, funders and patient representatives, as well as one-off engagement sessions at relevant stakeholder meetings (stage 4); and the preparation and dissemination of a guidance document (stage 5).
Discussion
Specification of the target difference for the primary outcome is a key component of the design of an RCT. There is a need for better guidance for researchers and funders regarding specification and reporting of this aspect of trial design. The aim of this project is to produce consensus based guidance for researchers and funders.
Electronic supplementary material
The online version of this article (doi:10.1186/s13063-017-1969-5) contains supplementary material, which is available to authorized users.
doi:10.1186/s13063-017-1969-5
PMCID: PMC5469157  PMID: 28606102
Target difference; Clinically important difference; Sample size; Guidance; Randomised controlled trial; Pilot study; Effect size
4.  Supporting Looked After Children and Care Leavers In Decreasing Drugs, and alcohol (SOLID): protocol for a pilot feasibility randomised controlled trial of interventions to decrease risky substance use (drugs and alcohol) and improve mental health of looked after children and care leavers aged 12–20 years 
Background
Looked after children (LAC) and care leavers are young people who have been placed under the legal care of local authorities, in many instances due to a history of abuse and/or neglect. These young people have a significantly increased risk of substance use and mental disorder compared to their peers. The aim of the SOLID study is to assess the feasibility and acceptability of a definitive three-arm multi-centre randomised controlled trial (RCT) that compares the effectiveness of two interventions that aim to reduce risky drug and alcohol use and improve mental health among LAC aged 12 to 20 years with usual care.
Methods
All LAC aged 12 to 20 years residing in four local authorities in North East England will be screened by their social worker for risky drug and alcohol use using the CRAFFT (Car, Relax, Alone, Forget, Friends and Trouble) screening tool. Those who score ≥2 will be invited to take part in the trial after further eligibility checks. Informed consent will be taken and baseline data collected. Participants will then be randomised into either (i) Motivational Enhancement Therapy, (ii) Social Behaviour and Network Therapy, or (iii) control–usual care. Follow-up data will be collected 12 months post-baseline. The baseline and follow-up questionnaires will measure self-reported drug and alcohol use, mental health and well-being and health-related quality of life. The follow-up will also collect data on placement stability and self-reported sexual, antisocial and criminal behaviour. Participants will also be asked about the use of health and social services. A detailed process evaluation, using both qualitative and quantitative methods, will be conducted and involve LAC, their carers, social workers and drug and alcohol practitioners.
Discussion
Despite having an increased likelihood of risky substance misuse, there is a lack of evidence outlining specific interventions to decrease drug and alcohol use targeting LAC. This feasibility study will provide the information needed to develop a definitive trial. LAC will benefit from the results of this study and the further development of the interventions.
Trial registration
ISRCTN80786829
doi:10.1186/s40814-017-0138-7
PMCID: PMC5439153
Looked after children; Drugs and alcohol; Feasibility; Social work; Intervention
5.  Views of Young People With Chronic Conditions on Transition From Pediatric to Adult Health Services 
Purpose
This study sought to identify and describe the views of young people with chronic conditions about the transition from pediatric to adult services.
Methods
Q methodology was used to identify young people’s views on transition. A set of 39 statements about transition was developed from an existing literature review and refined in consultation with local groups of young people. Statements were printed onto cards and a purposive sample of 44 young people with chronic health conditions was recruited, 41 remaining in the study. The young people were asked to sort the statement cards onto a Q-sort grid, according to their opinions from “strongly disagree” to “strongly agree.” Factor analysis was used to identify shared points of view (patterns of similarity between individual’s Q-sorts).
Results
Four distinct views on transition were identified from young people: (1) “a laid-back view of transition;” (2) “anxiety about transition;” (3) “wanting independence and autonomy during transition;” and (4) “valuing social interaction with family, peers, and professionals to assist transition.”
Conclusions
Successful transition is likely to be influenced by how young people view the process. Discussing and understanding young people’s views and preferences about transition should help clinicians and young people develop personalized planning for transition as a whole, and more specifically the point of transfer, leading to effective and efficient engagement with adult care.
doi:10.1016/j.jadohealth.2016.04.004
PMCID: PMC5245766  PMID: 27287962
Transition; Q methodology; Chronic conditions; Shared viewpoints
6.  Multicentre individual randomised controlled trial of screening and brief alcohol intervention to prevent risky drinking in young people aged 14–15 in a high school setting (SIPS JR-HIGH): study protocol 
BMJ Open  2016;6(12):e012474.
Introduction
Drinking has adverse impacts on health, well-being, education and social outcomes for adolescents. Adolescents in England are among the heaviest drinkers in Europe. Recently, the proportion of adolescents who drink alcohol has fallen, although consumption among those who do drink has actually increased. This trial seeks to investigate how effective and efficient an alcohol brief intervention is with 11–15 years olds to encourage lower alcohol consumption.
Methods and analysis
This is an individually randomised two-armed trial incorporating a control arm of usual school-based practice and a leaflet on a healthy lifestyle (excl. alcohol), and an intervention arm that combines usual practice with a 30 min brief intervention delivered by school learning mentors and a leaflet on alcohol. At least 30 schools will be recruited from four regions in England (North East, North West, London, Kent and Medway) to follow-up 235 per arm. The primary outcome is total alcohol consumed in the last 28 days, using the 28 day Timeline Follow Back questionnaire measured at the 12-month follow-up. The analysis of the intervention will consider effectiveness and cost-effectiveness. A qualitative study will explore, via 1:1 in-depth interviews with (n=80) parents, young people and school staff, intervention experience, intervention fidelity and acceptability issues, using thematic narrative synthesis to report qualitative data.
Ethics and dissemination
Ethical approval was granted by Teesside University. Dissemination plans include academic publications, conference presentations, disseminating to local and national education departments and the wider public health community, including via Fuse, and engaging with school staff and young people to comment on whether and how the project can be improved.
Trial registration trial
ISRCTN45691494; Pre-results.
doi:10.1136/bmjopen-2016-012474
PMCID: PMC5223663  PMID: 28011807
Alcohol; Brief Intervention; Randomised Controlled Trial; School Setting
7.  A feasibility study incorporating a pilot randomised controlled trial of oral feeding plus pre-treatment gastrostomy tube versus oral feeding plus as-needed nasogastric tube feeding in patients undergoing chemoradiation for head and neck cancer (TUBE trial): study protocol 
Background
There are 7000 new cases of head and neck squamous cell cancers (HNSCC) treated by the NHS each year. Stage III and IV HNSCC can be treated non-surgically by radio therapy (RT) or chemoradiation therapy (CRT). CRT can affect eating and drinking through a range of side effects with 90 % of patients undergoing this treatment requiring nutritional support via gastrostomy (G) or nasogastric (NG) tube feeding.
Long-term dysphagia following CRT is a primary concern for patients. The effect of enteral feeding routes on swallowing function is not well understood, and the two feeding methods have, to date, not been compared to assess which leads to a better patient outcome.
The purpose of this study is to explore the feasibility of conducting a randomised controlled trial (RCT) comparing these two options with particular emphasis on patient willingness to be randomised and clinician willingness to approach eligible patients.
Methods/design
This is a mixed methods multicentre study to establish the feasibility of a randomised controlled trial comparing oral feeding plus pre-treatment gastrostomy versus oral feeding plus as required nasogastric tube feeding in patients with HNSCC. A total of 60 participants will be randomised to the two arms of the study (1:1 ratio). The primary outcome of feasibility is a composite of recruitment (willingness to randomise and be randomised) and retention. A qualitative process evaluation investigating patient, family and friends and staff experiences of trial participation will also be conducted alongside an economic modelling exercise to synthesise available evidence and provide estimates of cost-effectiveness and value of information. Participants will be assessed at baseline (pre-randomisation), during CRT weekly, 3 months and 6 months.
Discussion
Clinicians are in equipoise over the enteral feeding options for patients being treated with CRT. Swallowing outcomes have been identified as a top priority for patients following treatment and this trial would inform a future larger scale RCT in this area to inform best practice.
Trial registration
ISRCTN48569216
doi:10.1186/s40814-016-0069-8
PMCID: PMC5154009  PMID: 27965848
Nasogastric tube; Gastrostomy; Swallow outcome; Chemoradiation therapy; Head and neck cancer
8.  Economic evaluations on centralisation of specialised healthcare services: a systematic review of methods 
BMJ Open  2016;6(5):e011214.
Objective
To systematically review and appraise the quality of economic evaluations assessing centralisation of specialised healthcare services.
Methods
A systematic review to identify economic evaluations on centralisation of any specialised healthcare service. Full economic evaluations comparing costs and consequences of centralisation of any specialised healthcare service were eligible for inclusion. Methodological characteristics of included studies were appraised using checklists adapted from recommended guidelines.
Results
A total of 64 full-text articles met the inclusion criteria. Two studies were conducted in the UK. Most of the studies used volume of activity as a proxy measure of centralisation. The methods used to assess centralisation were heterogeneous. Studies differed in terms of study design used and aspect of centralisation they considered. There were major limitations in studies. Only 12 studies reported the study perspective. Charges which are not true representation of costs were used by 17 studies to assess cost outcomes. Only 10 reported the detailed breakdown of the cost components used in their analysis. Discounting was necessary in 14 studies but was reported only in 7 studies. Sensitivity analyses were included by less than one-third of the studies. The applicability of the identified studies to a setting other than the one they were conducted in is questionable, given variations in the organisation of services and healthcare costs. Centralisation as a concept has also been variably and narrowly defined as activity of specific services which may not reflect the wider aspects of centralisation.
Conclusions
Confounded and biased information coming from studies without standardised methods may mislead decision-makers towards making wrong decisions on centralisation. It is important to improve the methodology and reporting of economic evaluations in order to provide more robust and transferable evidence. Wider aspects of healthcare centralisation should be considered in the estimates of costs and health outcomes.
doi:10.1136/bmjopen-2016-011214
PMCID: PMC4861117  PMID: 27154484
centralisation; healthcare; review; HEALTH ECONOMICS
9.  Prevalence of alcohol related attendance at an inner city emergency department and its impact: a dual prospective and retrospective cohort study 
Emergency Medicine Journal : EMJ  2015;33(3):187-193.
Background
Alcohol related hospital attendances are a potentially avoidable burden on emergency departments (EDs). Understanding the number and type of patients attending EDs with alcohol intoxication is important in estimating the workload and cost implications. We used best practice from previous studies to establish the prevalence of adult alcohol related ED attendances and estimate the costs of clinical management and subsequent health service use.
Methods
The setting was a large inner city ED in northeast England, UK. Data were collected via (i) retrospective review of hospital records for all ED attendances for four pre-specified weeks in 2010/2011 to identify alcohol related cases along with 12 months of follow-up of the care episode and (ii) prospective 24/7 assessment via breath alcohol concentration testing of patients presenting to the ED in the corresponding weeks in 2012/2013.
Results
The prevalence rates of alcohol related attendances were 12% and 15% for the retrospective and prospective cohorts, respectively. Prospectively, the rates ranged widely from 4% to 60% across week days, rising to over 70% at weekends. Younger males attending in the early morning hours at weekends made up the largest proportion of alcohol related attendances. The mean cost per attendance was £249 (SD £1064); the mean total cost for those admitted was £851 (SD £2549). The most common reasons for attending were trauma related injuries followed by psychiatric problems.
Conclusions
Alcohol related attendances are a major and avoidable burden on emergency care. However, targeted interventions at weekends and early morning hours could capture the majority of cases and help prevent future re-attendance.
doi:10.1136/emermed-2014-204581
PMCID: PMC4789717  PMID: 26698364
alcohol abuse; emergency departments
10.  Open urethroplasty versus endoscopic urethrotomy - clarifying the management of men with recurrent urethral stricture (the OPEN trial): study protocol for a randomised controlled trial 
Trials  2015;16:600.
Background
Urethral stricture is a common cause of difficulty passing urine in men with prevalence of 0.5 %; about 62,000 men in the UK. The stricture is usually sited in the bulbar part of the urethra causing symptoms such as reduced urine flow. Initial treatment is typically by endoscopic urethrotomy but recurrence occurs in about 60 % of men within 2 years. The best treatment for men with recurrent bulbar stricture is uncertain. Repeat endoscopic urethrotomy opens the narrowing but it usually scars up again within 2 years requiring repeated procedures. The alternative of open urethroplasty involves surgically reconstructing the urethra, which may need an oral mucosal graft. It is a specialist procedure with a longer recovery period but may give lower risk of recurrence. In the absence of firm evidence as to which is best, individual men have to trade off the invasiveness and possible benefit of each option. Their preference will be influenced by individual social circumstances, availability of local expertise and clinician guidance. The open urethroplasty versus endoscopic urethrotomy (OPEN) trial aims to better guide the choice of treatment for men with recurrent urethral strictures by comparing benefit over 2 years in terms of symptom control and need for further treatment.
Methods/Design
OPEN is a pragmatic, UK multicentre, randomised trial. Men with recurrent bulbar urethral strictures (at least one previous treatment) will be randomised to undergo endoscopic urethrotomy or open urethroplasty. Participants will be followed for 24 months after randomisation, measuring symptoms, flow rate, the need for re-intervention, health-related quality of life, and costs. The primary clinical outcome is the difference in symptom control over 24 months measured by the area under the curve (AUC) of a validated score. The trial has been powered at 90 % with a type I error rate of 5 % to detect a 0.1 difference in AUC measured on a 0–1 scale. The analysis will be based on all participants as randomised (intention-to-treat). The primary economic outcome is the incremental cost per quality-adjusted life year. A qualitative study will assess willingness to be randomised and hence ability to recruit to the trial.
Discussion
The OPEN Trial seeks to clarify relative benefit of the current options for surgical treatment of recurrent bulbar urethral stricture which differ in their invasiveness and resources required. Our feasibility study identified that participation would be limited by patient preference and differing recruitment styles of general and specialist urologists. We formulated and implemented effective strategies to address these issues in particular by inviting participation as close as possible to diagnosis. In addition re-calculation of sample size as recruitment progressed allowed more efficient design given the limited target population and funding constraints. Recruitment is now to target.
Trial registration
ISRCTN98009168 Date of registration: 29 November 2012.
Electronic supplementary material
The online version of this article (doi:10.1186/s13063-015-1120-4) contains supplementary material, which is available to authorized users.
doi:10.1186/s13063-015-1120-4
PMCID: PMC4697334  PMID: 26718754
Bulbar urethra; Urethral stricture; Randomised trial urethroplasty; Endoscopic urethrotomy; Cost-effectiveness analysis
11.  A feasibility study of a Family Focused Treatment for Adolescents with Bipolar Disorder—the FAB study 
Background
The aim of this study is to examine the feasibility of a future definitive randomised controlled trial of Family Focused Treatment for Adolescents UK (FFT-A UK) in the management of early-onset bipolar disorder (EOBD) (under 18 years). The FFT-A has been evaluated in the USA to augment the pharmacological treatment of adolescents with bipolar disorder (BD). The FFT-A UK has been condensed to 16 sessions over approximately 6 months to be utilised within the UK National Health Service.
Research from the USA suggests that families experience high levels of distress, stress, burden and family disharmony when living with a young person who has BD. The FFT-A UK is a family-based approach designed to increase understanding of BD (psycho-education), improve communication and increase ability to problem-solve.
Methods/Design
The trial will examine the feasibility of a randomised, parallel group, non-blinded design and the procedures of a subsequent definitive trial. Thirty-three young people with BD and their families will be recruited. Participants will complete measures at baseline, on completion of the 6-month treatment and again after a further 6 months. The self-report measures include the Warwick Edinburgh Mental Well-being Scale, The McMaster Family Assessment Device (FAD), Conflict Behaviour Questionnaire aka ‘Interaction Behaviour Questionnaire’, EuroQuol EQ-5D-3L and EQ-5D-Y. Primary outcomes will be rates of eligibility, recruitment and retention, estimates of the variability in the self-report measures and assessment of the intervention delivery in the study population. Participants’ qualitative views on the measures and intervention will be sought to confirm the acceptability of intervention and study design. The health economics component will establish how cost-effectiveness will be assessed in a future definitive trial.
Discussion
The study will produce a full trial protocol and amendments to the FFT-A UK to inform a well-designed multi-centre randomised controlled trial (RCT) as an adjunct to pharmacotherapy in the management of EOBD.
Trial registration
Current Controlled Trials ISRCTN59769322
doi:10.1186/s40814-015-0038-7
PMCID: PMC5153680  PMID: 27965821
Early onset; Bipolar disorder; Psychological intervention; Trial; FFT-A UK; Young people; Adolescents; FAB
12.  The NULevel trial of a scalable, technology-assisted weight loss maintenance intervention for obese adults after clinically significant weight loss: study protocol for a randomised controlled trial 
Trials  2015;16:421.
Background
Effective weight loss interventions are widely available but, after weight loss, most individuals regain weight. This article describes the protocol for the NULevel trial evaluating the effectiveness and cost-effectiveness of a systematically developed, inexpensive, scalable, technology-assisted, behavioural intervention for weight loss maintenance (WLM) in obese adults after initial weight loss.
Methods/Design
A 12-month single-centre, two-armed parallel group, participant randomised controlled superiority trial is underway, recruiting a total of 288 previously obese adults after weight loss of ≥5 % within the previous 12 months. Participants are randomly assigned to intervention or control arms, with a 1:1 allocation, stratified by sex and percentage of body weight lost (<10 % vs ≥10 %). Change in weight (kg) from baseline to 12 months is the primary outcome. Weight, other anthropometric variables and 7-day physical activity (assessed via accelerometer) measures are taken at 0 and 12 months. Questionnaires at 0, 6 and 12 months assess psychological process variables, health service use and participant costs. Participants in the intervention arm initially attend an individual face-to-face WLM consultation with an intervention facilitator and then use a mobile internet platform to self-monitor and report their diet, daily activity (via pedometer) and weight through daily weighing on wirelessly connected scales. Automated feedback via mobile phone, tailored to participants’ weight regain and goal progress is provided. Participants in the control arm receive quarterly newsletters (via links embedded in text messages) and wirelessly connected scales. Qualitative process evaluation interviews are conducted with a subsample of up to 40 randomly chosen participants. Acceptability and feasibility of procedures, cost-effectiveness, and relationships among socioeconomic variables and WLM will also be assessed.
Discussion
It is hypothesised that participants allocated to the intervention arm will show significantly lower levels of weight regain from baseline than those in the control arm. To date, this is the first WLM trial using remote real-time weight monitoring and mobile internet platforms to deliver a flexible, efficient and scalable intervention, tailored to the individual. This trial addresses a key research need and has the potential to make a vital contribution to the evidence base to inform future WLM policy and provision.
Trial registration
http://www.isrctn.com/ISRCTN14657176 (registration date 20 March 2014).
doi:10.1186/s13063-015-0931-7
PMCID: PMC4580361  PMID: 26395774
Behaviour; Randomised controlled trial; Clinical protocol; Obesity; Overweight; Weight loss; Weight loss maintenance
13.  A mixed methods study to assess the feasibility of a randomised controlled trial of invasive urodynamic testing versus clinical assessment and non-invasive tests prior to surgery for stress urinary incontinence in women: the INVESTIGATE-I study 
Trials  2015;16:400.
Background
The position of invasive urodynamic testing (IUT) in diagnostic pathways for urinary incontinence is unclear, and systematic reviews have called for further trials evaluating clinical utility. The objective of this study was to inform the decision whether to proceed to a definitive randomised trial of IUT compared to clinical assessment with non-invasive tests, prior to surgery in women with stress urinary incontinence (SUI) or stress-predominant mixed urinary incontinence (MUI).
Methods
A mixed methods study comprising a pragmatic multicentre randomised pilot trial, a qualitative face-to face interview study with patients eligible for the trial, an exploratory economic evaluation including value of information study, a survey of clinicians’ views about IUT, and qualitative telephone interviews with purposively sampled survey respondents. Only the first and second of these elements are reported here.
Trial participants were randomised to either clinical assessment with non-invasive tests (control arm) or clinical assessment with non-invasive tests plus IUT (intervention arm).
The main outcome measures of these feasibility studies were confirmation that units can identify and recruit eligible women, acceptability of investigation strategies and data collection tools, and acquisition of outcome data to determine the sample size for a definitive trial. The primary outcome proposed for a definitive trial was ICIQ-FLUTS (total score) 6 months after surgery or the start of nonsurgical treatment.
Results
Of 284 eligible women, 222 (78 %) were recruited, 165/219 (75 %) returned questionnaires at baseline, and 125/200 returned them (63 %) at follow-up. Most women underwent surgery; management plans were changed in 19 (19 %) participants following IUT.
Participants interviewed were positive about the trial and the associated documentation.
Conclusions
All elements of a definitive trial were rehearsed. Such a trial would require between 232 and 922 participants, depending on the target difference in the primary outcome. We identified possible modifications to our protocol for application in a definitive trial including clarity over inclusion/exclusions, screening processes, reduction in secondary outcomes, and modification to patient questionnaire booklets and bladder diaries. A definitive trial of IUT versus clinical assessment prior to surgery for SUI or stress predominant MUI is feasible and remains relevant.
Trial registration
Current Controlled Trials: ISRCTN 71327395, registered 7 June 2010.
doi:10.1186/s13063-015-0928-2
PMCID: PMC4563900  PMID: 26350343
feasibility studies; pilot studies; interview studies; randomised controlled trial; stress urinary incontinence; urodynamics
14.  Is it worthwhile to conduct a randomized controlled trial of glaucoma screening in the United Kingdom? 
Objectives
To assess the value of conducting a glaucoma screening randomized controlled trial in the UK.
Methods
Decision model based economic evaluation and value of information analysis. Model derived from a previous health technology assessment. Model updated in terms of structure and parameter estimates with data from surveys, interviews with members of the public and health care providers and routine sources.
Results
On average, across a range of ages of initiating screening (40–60 years), glaucoma prevalence (1–5%), screening uptake (30–100%), and the performance of current case finding, screening was not cost-effective at a £30,000 threshold per quality adjusted life year (QALY) from the perspective of the National Health Service (NHS). The societal value of removing all uncertainty around glaucoma screening is £107 million at a threshold of £20,000 per QALY. For informing policy decisions on glaucoma screening, reducing uncertainty surrounding the NHS and personal social care cost of sight impairment (£74 million) was of most value, followed by reducing uncertainty in test performance (£14 million) and uptake of either screening or current eye care (£8 million each).
Conclusions
A glaucoma screening trial in the UK is unlikely to be the best use of research resources. Further research to quantify the costs of sight impairment falling on the NHS and personal social services is a priority. Further development of glaucoma tests and research into strategies to promote the uptake of screening or current eye care such as through the use of a behavioural intervention would be worthwhile.
doi:10.1177/1355819613499748
PMCID: PMC4509868  PMID: 24088295
decision analysis; health policy; public health; ophthalmology
15.  The NAtional randomised controlled Trial of Tonsillectomy IN Adults (NATTINA): a clinical and cost-effectiveness study: study protocol for a randomised control trial 
Trials  2015;16:263.
Background
The role of tonsillectomy in the management of adult tonsillitis remains uncertain and UK regional variation in tonsillectomy rates persists. Patients, doctors and health policy makers wish to know the costs and benefits of tonsillectomy against conservative management and whether therapy can be better targeted to maximise benefits and minimise risks of surgery, hence maximising cost-effective use of resources. NATTINA incorporates the first attempt to map current NHS referral criteria against other metrics of tonsil disease severity.
Methods/design
A UK multi-centre, randomised, controlled trial for adults with recurrent tonsillitis to compare the clinical and cost-effectiveness of tonsillectomy versus conservative management.
An initial feasibility study comprises qualitative interviews to investigate the practicality of the protocol, including willingness to randomise and be randomised. Approximately 20 otolaryngology staff, 10 GPs and 15 ENT patients will be recruited over 5 months in all 9 proposed main trial participating sites.
A 6-month internal pilot will then recruit 72 patients across 6 of the 9 sites. Participants will be adults with recurrent acute tonsillitis referred by a GP to secondary care. Randomisation between tonsillectomy and conservative management will be according to a blocked allocation method in a 1:1 ratio stratified by centre and baseline disease severity.
If the pilot is successful, the main trial will recruit a further 528 patients over 18 months in all 9 participating sites. All participants will be followed up for a total of 24 months, throughout which both primary and secondary outcome data will be collected. The primary outcome is the number of sore throat days experienced over the 24-month follow-up. The pilot and main trials include an embedded qualitative process evaluation.
Discussion
NATTINA is designed to evaluate the relative effectiveness and efficiency of tonsillectomy versus conservative management in patients with recurrent sore throat who are eligible for surgery. Most adult tonsil disease and surgery has an impact on economically active age groups, with individual and societal costs through loss of earnings and productivity. Avoidance of unnecessary operations and prioritisation of those individuals likely to gain most from tonsillectomy would reduce costs to the NHS and society.
Trial registration
ISRCTN55284102, Date of Registration: 4 August 2014
doi:10.1186/s13063-015-0768-0
PMCID: PMC4467045  PMID: 26047934
Tonsillitis; Sore throat; Tonsillectomy; Surgery; Randomised controlled trial
16.  The ETTAA study protocol: a UK-wide observational study of ‘Effective Treatments for Thoracic Aortic Aneurysm’ 
BMJ Open  2015;5(6):e008147.
Introduction
Chronic thoracic aortic aneurysm (CTAA) affecting the arch or descending aorta is an indolent but life-threatening condition with a rising prevalence as the UK population ages. Treatment may be in the form of open surgical repair (OSR) surgery, endovascular stent grafting (ESG) or best medical therapy (BMT). Currently, there is no consensus on the best management strategy, and no UK-specific economic studies that assess outcomes beyond the chosen procedure, but this is required in the context of greater demand for treatment and limited National Health Service (NHS) resources.
Methods and analysis
This is a prospective, multicentre observational study with statistical and economic modelling of patients with CTAA affecting the arch or descending aorta. We aim to gain an understanding of how treatments are currently chosen, and to determine the clinical effectiveness and cost-effectiveness of the three available treatment strategies (BMT, ESG and OSR). This will be achieved by: (1) following consecutive patients who are referred to the teams collaborating in this proposal and collecting data regarding quality of life (QoL), medical events and hospital stays over a maximum of 5 years; (2) statistical analysis of the comparative effectiveness of the three treatments; and (3) economic modelling of the comparative cost-effectiveness of the three treatments. Primary study outcomes are: aneurysm growth, QoL, freedom from reintervention, freedom from death or permanent neurological injury, incremental cost per quality-adjusted life year gained.
Ethics and dissemination
The study will generate an evidence base to guide patients and clinicians to determine the indications and timing of treatment, as well as informing healthcare decision-makers about which treatments the NHS should provide. The study has achieved ethical approval and will be disseminated primarily in the form of a Health Technology Assessment monograph at its completion.
Trial registration number
ISRCTN04044627.
doi:10.1136/bmjopen-2015-008147
PMCID: PMC4458682  PMID: 26038360
VASCULAR MEDICINE
17.  Does quality of life improve in octogenarians following cardiac surgery? A systematic review 
BMJ Open  2015;5(4):e006904.
Objectives
Current outcome measures in cardiac surgery are largely described in terms of mortality. Given the changing demographic profiles and increasingly aged populations referred for cardiac surgery this may not be the most appropriate measure. Postoperative quality of life is an outcome of importance to all ages, but perhaps particularly so for those whose absolute life expectancy is limited by virtue of age. We undertook a systematic review of the literature to clarify and summarise the existing evidence regarding postoperative quality of life of older people following cardiac surgery. For the purpose of this review we defined our population as people aged 80 years of age or over.
Methods
A systematic review of MEDLINE, EMBASE, Cochrane Library, trial registers and conference abstracts was undertaken to identify studies addressing quality of life following cardiac surgery in patients 80 or over.
Results
Forty-four studies were identified that addressed this topic, of these nine were prospective therefore overall conclusions are drawn from largely retrospective observational studies. No randomised controlled data were identified.
Conclusions
Overall there appears to be an improvement in quality of life in the majority of elderly patients following cardiac surgery, however there was a minority in whom quality of life declined (8–19%). There is an urgent need to validate these data and if correct to develop a robust prediction tool to identify these patients before surgery. Such a tool could guide informed consent, policy development and resource allocation.
doi:10.1136/bmjopen-2014-006904
PMCID: PMC4420984  PMID: 25922099
GERIATRIC MEDICINE
18.  Specifying the target difference in the primary outcome for a randomised controlled trial: guidance for researchers 
Trials  2015;16:12.
Background
Central to the design of a randomised controlled trial is the calculation of the number of participants needed. This is typically achieved by specifying a target difference and calculating the corresponding sample size, which provides reassurance that the trial will have the required statistical power (at the planned statistical significance level) to identify whether a difference of a particular magnitude exists. Beyond pure statistical or scientific concerns, it is ethically imperative that an appropriate number of participants should be recruited. Despite the critical role of the target difference for the primary outcome in the design of randomised controlled trials, its determination has received surprisingly little attention. This article provides guidance on the specification of the target difference for the primary outcome in a sample size calculation for a two parallel group randomised controlled trial with a superiority question.
Methods
This work was part of the DELTA (Difference ELicitation in TriAls) project. Draft guidance was developed by the project steering and advisory groups utilising the results of the systematic review and surveys. Findings were circulated and presented to members of the combined group at a face-to-face meeting, along with a proposed outline of the guidance document structure, containing recommendations and reporting items for a trial protocol and report. The guidance and was subsequently drafted and circulated for further comment before finalisation.
Results
Guidance on specification of a target difference in the primary outcome for a two group parallel randomised controlled trial was produced. Additionally, a list of reporting items for protocols and trial reports was generated.
Conclusions
Specification of the target difference for the primary outcome is a key component of a randomized controlled trial sample size calculation. There is a need for better justification of the target difference and reporting of its specification.
Electronic supplementary material
The online version of this article (doi:10.1186/s13063-014-0526-8) contains supplementary material, which is available to authorized users.
doi:10.1186/s13063-014-0526-8
PMCID: PMC4302137  PMID: 25928502
Target difference; clinically important difference; sample size; randomised controlled trial; guidance
19.  Methods for Specifying the Target Difference in a Randomised Controlled Trial: The Difference ELicitation in TriAls (DELTA) Systematic Review 
PLoS Medicine  2014;11(5):e1001645.
Jonathan Cook and colleagues systematically reviewed the literature for methods of determining the target difference for use in calculating the necessary sample size for clinical trials, and discuss which methods are best for various types of trials.
Please see later in the article for the Editors' Summary
Background
Randomised controlled trials (RCTs) are widely accepted as the preferred study design for evaluating healthcare interventions. When the sample size is determined, a (target) difference is typically specified that the RCT is designed to detect. This provides reassurance that the study will be informative, i.e., should such a difference exist, it is likely to be detected with the required statistical precision. The aim of this review was to identify potential methods for specifying the target difference in an RCT sample size calculation.
Methods and Findings
A comprehensive systematic review of medical and non-medical literature was carried out for methods that could be used to specify the target difference for an RCT sample size calculation. The databases searched were MEDLINE, MEDLINE In-Process, EMBASE, the Cochrane Central Register of Controlled Trials, the Cochrane Methodology Register, PsycINFO, Science Citation Index, EconLit, the Education Resources Information Center (ERIC), and Scopus (for in-press publications); the search period was from 1966 or the earliest date covered, to between November 2010 and January 2011. Additionally, textbooks addressing the methodology of clinical trials and International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) tripartite guidelines for clinical trials were also consulted. A narrative synthesis of methods was produced. Studies that described a method that could be used for specifying an important and/or realistic difference were included. The search identified 11,485 potentially relevant articles from the databases searched. Of these, 1,434 were selected for full-text assessment, and a further nine were identified from other sources. Fifteen clinical trial textbooks and the ICH tripartite guidelines were also reviewed. In total, 777 studies were included, and within them, seven methods were identified—anchor, distribution, health economic, opinion-seeking, pilot study, review of the evidence base, and standardised effect size.
Conclusions
A variety of methods are available that researchers can use for specifying the target difference in an RCT sample size calculation. Appropriate methods may vary depending on the aim (e.g., specifying an important difference versus a realistic difference), context (e.g., research question and availability of data), and underlying framework adopted (e.g., Bayesian versus conventional statistical approach). Guidance on the use of each method is given. No single method provides a perfect solution for all contexts.
Please see later in the article for the Editors' Summary
Editors' Summary
Background
A clinical trial is a research study in which human volunteers are randomized to receive a given intervention or not, and outcomes are measured in both groups to determine the effect of the intervention. Randomized controlled trials (RCTs) are widely accepted as the preferred study design because by randomly assigning participants to groups, any differences between the two groups, other than the intervention under study, are due to chance. To conduct a RCT, investigators calculate how many patients they need to enroll to determine whether the intervention is effective. The number of patients they need to enroll depends on how effective the intervention is expected to be, or would need to be in order to be clinically important. The assumed difference between the two groups is the target difference. A larger target difference generally means that fewer patients need to be enrolled, relative to a smaller target difference. The target difference and number of patients enrolled contribute to the study's statistical precision, and the ability of the study to determine whether the intervention is effective. Selecting an appropriate target difference is important from both a scientific and ethical standpoint.
Why Was This Study Done?
There are several ways to determine an appropriate target difference. The authors wanted to determine what methods for specifying the target difference are available and when they can be used.
What Did the Researchers Do and Find?
To identify studies that used a method for determining an important and/or realistic difference, the investigators systematically surveyed the research literature. Two reviewers screened each of the abstracts chosen, and a third reviewer was consulted if necessary. The authors identified seven methods to determine target differences. They evaluated the studies to establish similarities and differences of each application. Points about the strengths and limitations of the method and how frequently the method was chosen were also noted.
What Do these Findings Mean?
The study draws attention to an understudied but important part of designing a clinical trial. Enrolling the right number of patients is very important—too few patients and the study may not be able to answer the study question; too many and the study will be more expensive and more difficult to conduct, and will unnecessarily expose more patients to any study risks. The target difference may also be helpful in interpreting the results of the trial. The authors discuss the pros and cons of different ways to calculate target differences and which methods are best for which types of studies, to help inform researchers designing such studies.
Additional Information
Please access these websites via the online version of this summary at http://dx.doi.org/10.1371/journal.pmed.1001645.
Wikipedia has an entry on sample size determination that discusses the factors that influence sample size calculation, including the target difference and the statistical power of a study (statistical power is the ability of a study to find a difference between treatments when a true difference exists). (Note: Wikipedia is a free online encyclopedia that anyone can edit; available in several languages.)
The University of Ottawa has an article that explains how different factors influence the power of a study
doi:10.1371/journal.pmed.1001645
PMCID: PMC4019477  PMID: 24824338
21.  Developing Effective and Efficient care pathways in chronic Pain: DEEP study protocol 
BMC Oral Health  2014;14:6.
Background
Pain affecting the face or mouth and lasting longer than three months (“chronic orofacial pain”, COFP) is relatively common in the UK. This study aims to describe and model current care pathways for COFP patients, identify areas where current pathways could be modified, and model whether these changes would improve outcomes for patients and use resources more efficiently.
Methods/Design
The study takes a prospective operations research approach. A cohort of primary and secondary care COFP patients (n = 240) will be recruited at differing stages of their care in order to follow and analyse their journey through care. The cohort will be followed for two years with data collected at baseline 6, 12, 18, and 24 months on: 1) experiences of the care pathway and its impacts; 2) quality of life; 3) pain; 4) use of health services and costs incurred; 5) illness perceptions. Qualitative in-depth interviews will be used to collect data on patient experiences from a purposive sub-sample of the total cohort (n = 30) at baseline, 12 and 24 months. Four separate appraisal groups (public, patient, clincian, service manager/commissioning) will then be given data from the pathway analysis and asked to determine their priority areas for change. The proposals from appraisal groups will inform an economic modelling exercise. Findings from the economic modelling will be presented as incremental costs, Quality Adjusted Life Years (QALYs), and the incremental cost per QALY gained. At the end of the modelling a series of recommendations for service change will be available for implementation or further trial if necessary.
Discussion
The recent white paper on health and the report from the NHS Forum identified chronic conditions as priority areas and whilst technology can improve outcomes, so can simple, appropriate and well-defined clinical care pathways. Understanding the opportunity cost related to care pathways benefits the wider NHS. This research develops a method to help design efficient systems built around one condition (COFP), but the principles should be applicable to a wide range of other chronic and long-term conditions.
doi:10.1186/1472-6831-14-6
PMCID: PMC3909482  PMID: 24447722
Orofacial pain; Health economics; Quality of life; Qualitative methods; Chronic pain; Care pathways
23.  Issues in the incorporation of economic perspectives and evidence into Cochrane reviews 
Systematic Reviews  2013;2:83.
Background
Methods for systematic reviews of the effects of health interventions have focused mainly on addressing the question of 'What works?’ or 'Is this intervention effective in achieving one or more specific outcomes?’ Addressing the question 'Is it worth it given the resources available?’ has received less attention. This latter question can be addressed by applying an economic lens to the systematic review process.
This paper reflects on the value and desire for the consideration by end users for coverage of an economic perspective in a Cochrane review and outlines two potential approaches and future directions.
Methods
Two frameworks to guide review authors who are seeking to include an economic perspective are outlined. The first involves conducting a full systematic review of economic evaluations that is integrated into a review of intervention effects. The second involves developing a brief economic commentary. The two approaches share a set of common stages but allow the tailoring of the economic component of the Cochrane review to the skills and resources available to the review team.
Results
The number of studies using the methods outlined in the paper is limited, and further examples are needed both to explore the value of these approaches and to further develop them. The rate of progress will hinge on the organisational leadership, capacity and resources available to the CCEMG, author teams and other Cochrane entities. Particular methodological challenges to overcome relate to understanding the key economic trade-offs and casual relationships for a given decision problem and informing the development of evaluations designed to support local decision-makers.
Conclusions
Methods for incorporating economic perspectives and evidence into Cochrane intervention reviews are established. Their role is not to provide a precise estimate of 'cost-effectiveness’ but rather to help end-users of Cochrane reviews to determine the implications of the economic components of reviews for their own specific decisions.
doi:10.1186/2046-4053-2-83
PMCID: PMC3849717  PMID: 24050504
Cost-utility analysis; Cost-effectiveness analysis; Systematic review; Meta-analysis; Cochrane collaboration
24.  Study protocol: longitudinal study of the transition of young people with complex health needs from child to adult health services 
BMC Public Health  2013;13:675.
Background
Young people with complex health needs have impairments that can limit their ability to carry out day-to-day activities. As well as coping with other developmental transitions, these young people must negotiate the transfer of their clinical care from child to adult services. The process of transition may not be smooth and both health and social outcomes may suffer.
Increasingly, policy-makers have recognised the need to ensure a smoother transition between children’s and adult services, with processes that are holistic, individualised, and person-centred; however, there is little outcome data to support proposed models of care. This study aims to identify the features of transitional care that are potentially effective and efficient for young people with complex health needs making their transition.
Methods/Design
Longitudinal cohort study. 450 young people aged 14 years to 18 years 11 months (with autism spectrum disorder and an additional mental health problem, cerebral palsy or diabetes) will be followed through their transition from child to adult services and will contribute data at baseline, 12, 24 and 36 months. We will collect data on: health and wellbeing outcomes (participation, quality of life, satisfaction with services, generic health status (EQ-5D-Y) and condition specific measure of disease control or management); exposure to proposed beneficial features of services (such as having a key worker, appropriate involvement of parents); socio-economic characteristics of the sample; use of condition-related health and personal social services; preferences for the characteristics of transitional care.
We will us regression techniques to explore how outcomes vary by exposure to service features and by characteristics of the young people. These data will populate a decision-analytic model comparing the costs and benefits of potential alternative ways of organising transition services.
In order to better understand mechanisms and aid interpretation, we will undertake qualitative work with 15 young people, including interviews, non-participant observation and diary collection.
Discussion
This study will evaluate the effect of service components of transitional care, rather than evaluation of specific models that may be unsustainable or not generalisable. It has been developed in response to numerous national and international calls for such evaluation.
doi:10.1186/1471-2458-13-675
PMCID: PMC3724698  PMID: 23875722
Transition; Adolescence; Chronic illness; Disability; Evaluation; Protocol
25.  Surgery versus Active Monitoring in Intermittent Exotropia (SamExo): study protocol for a pilot randomised controlled trial 
Trials  2012;13:192.
Background
Childhood intermittent exotropia [X(T)] is a type of strabismus (squint) in which one eye deviates outward at times, usually when the child is tired. It may progress to a permanent squint, loss of stereovision and/or amblyopia (reduced vision). Treatment options for X(T) include eye patches, glasses, surgery and active monitoring. There is no consensus regarding how this condition should be managed, and even when surgery is the preferred option clinicians disagree as to the optimal timing. Reports on the natural history of X(T) are limited, and there is no randomised controlled trial (RCT) evidence on the effectiveness or efficiency of surgery compared with active monitoring. The SamExo (Surgery versus Active Monitoring in Intermittent Exotropia) pilot study has been designed to test the feasibility of such a trial in the UK.
Methods
Design: an external pilot patient randomised controlled trial.
Setting: four UK secondary ophthalmology care facilities at Newcastle NHS Hospitals Foundation Trust, Sunderland Eye Infirmary, Moorfields Eye Hospital and York NHS Trust.
Participants: children aged between 6 months and 16 years referred with suspected and subsequently diagnosed X(T). Recruitment target is a total of 144 children over a 9-month period, with 120 retained by 9-month outcome visit.
Randomisation: permuted blocks stratified by collaborating centre, age and severity of X(T).
Interventions: initial clinical assessment; randomisation (eye muscle surgery or active monitoring); 3-, 6- and 9-month (primary outcome) clinical assessments; participant/proxy completed questionnaire covering time and travel costs, health services use and quality of life (Intermittent Exotropia Questionnaire); qualitative interviews with parents to establish reasons for agreeing or declining participation in the pilot trial.
Outcomes: recruitment and retention rates; nature and extent of participation bias; nature and extent of biases arising from crossover or loss to follow-up; reasons for agreeing/declining participation; variability of cure rates (to inform power calculations for a definitive RCT); completion rates of outcome measures.
Discussion
The SamExo pilot trial will provide important pointers regarding the feasibility of a full RCT of immediate surgery versus deferred surgery/active monitoring. The results of this pilot, including differences in cure rates, will inform the design of a definitive RCT.
Trial registration
ISRCTN44114892
doi:10.1186/1745-6215-13-192
PMCID: PMC3521171  PMID: 23072556
Intermittent exotropia; Divergent strabismus; Surgery; Feasibility studies; Pilot study; Randomised controlled trial; Children; Parents; Qualitative research

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