Your browser doesn't support javascript.
loading
Mostrar: 20 | 50 | 100
Resultados 1 - 20 de 98
Filtrar
1.
Health Technol Assess ; 25(53): 1-52, 2021 09.
Artigo em Inglês | MEDLINE | ID: mdl-34505829

RESUMO

BACKGROUND: The use of placebo comparisons for randomised trials assessing the efficacy of surgical interventions is increasingly being considered. However, a placebo control is a complex type of comparison group in the surgical setting and, although powerful, presents many challenges. OBJECTIVES: To provide a summary of knowledge on placebo controls in surgical trials and to summarise any recommendations for designers, evaluators and funders of placebo-controlled surgical trials. DESIGN: To carry out a state-of-the-art workshop and produce a corresponding report involving key stakeholders throughout. SETTING: A workshop to discuss and summarise the existing knowledge and to develop the new guidelines. RESULTS: To assess what a placebo control entails and to assess the understanding of this tool in the context of surgery is considered, along with when placebo controls in surgery are acceptable (and when they are desirable). We have considered ethics arguments and regulatory requirements, how a placebo control should be designed, how to identify and mitigate risk for participants in these trials, and how such trials should be carried out and interpreted. The use of placebo controls is justified in randomised controlled trials of surgical interventions provided that there is a strong scientific and ethics rationale. Surgical placebos might be most appropriate when there is poor evidence for the efficacy of the procedure and a justified concern that results of a trial would be associated with a high risk of bias, particularly because of the placebo effect. CONCLUSIONS: The use of placebo controls is justified in randomised controlled trials of surgical interventions provided that there is a strong scientific and ethics rationale. Feasibility work is recommended to optimise the design and implementation of randomised controlled trials. An outline for best practice was produced in the form of the Applying Surgical Placebo in Randomised Evaluations (ASPIRE) guidelines for those considering the use of a placebo control in a surgical randomised controlled trial. LIMITATIONS: Although the workshop participants involved international members, the majority of participants were from the UK. Therefore, although every attempt was made to make the recommendations applicable to all health systems, the guidelines may, unconsciously, be particularly applicable to clinical practice in the UK NHS. FUTURE WORK: Future work should evaluate the use of the ASPIRE guidelines in making decisions about the use of a placebo-controlled surgical trial. In addition, further work is required on the appropriate nomenclature to adopt in this space. FUNDING: Funded by the Medical Research Council UK and the National Institute for Health Research as part of the Medical Research Council-National Institute for Health Research Methodology Research programme.


Assuntos
Efeito Placebo , Humanos , Projetos de Pesquisa
2.
Fam Pract ; 2021 May 11.
Artigo em Inglês | MEDLINE | ID: mdl-33972999

RESUMO

BACKGROUND: Antibiotics are over-prescribed for upper respiratory tract infection (URTI). It is unclear how factors known to influence prescribing decisions operate 'in the moment': dual process theories, which propose two systems of thought ('automatic' and 'analytical'), may inform this. OBJECTIVE(S): Investigate cognitive processes underlying antibiotic prescribing for URTI and the factors associated with inappropriate prescribing. METHODS: We conducted a mixed methods study. Primary care physicians in Scotland (n = 158) made prescribing decisions for patient scenarios describing sore throat or otitis media delivered online. Decision difficulty and decision time were recorded. Decisions were categorized as appropriate or inappropriate based on clinical guidelines. Regression analyses explored relationships between scenario and physician characteristics and decision difficulty, time and appropriateness. A subgroup (n = 5) verbalized their thoughts (think aloud) whilst making decisions for a subset of scenarios. Interviews were analysed inductively. RESULTS: Illness duration of 4+ days was associated with greater difficulty. Inappropriate prescribing was associated with clinical factors suggesting viral cause and with patient preference against antibiotics. In interviews, physicians made appropriate decisions quickly for easier cases, with little deliberation, reflecting automatic-type processes. For more difficult cases, physicians deliberated over information in some instances, but not in others, with inappropriate prescribing occurring in both instances. Some interpretations of illness duration and unilateral ear examination findings (for otitis media) were associated with inappropriate prescribing. CONCLUSION: Both automatic and analytical processes may lead to inappropriate prescribing. Interventions to support appropriate prescribing may benefit from targeting interpretation of illness duration and otitis media ear exam findings and facilitating appropriate use of both modes of thinking.

3.
Trials ; 22(1): 361, 2021 May 24.
Artigo em Inglês | MEDLINE | ID: mdl-34030707

RESUMO

BACKGROUND: There is an ethical imperative to offer the results of trials to those who participated. Existing research highlights that less than a third of trials do so, despite the desire of participants to receive the results of the trials they participated in. This scoping review aimed to identify, collate, and describe the available evidence relating to any aspect of disseminating trial results to participants. METHODS: A scoping review was conducted employing a search of key databases (MEDLINE, EMBASE, PsycINFO, and the Cumulative Index to Nursing & Allied Health Literature (CINAHL) from January 2008 to August 2019) to identify studies that had explored any aspect of disseminating results to trial participants. The search strategy was based on that of a linked existing review. The evidence identified describes the characteristics of included studies using narrative description informed by analysis of relevant data using descriptive statistics. RESULTS: Thirty-three eligible studies, including 12,700 participants (which included patients, health care professionals, trial teams), were identified and included. Reporting of participant characteristics (age, gender, ethnicity) across the studies was poor. The majority of studies investigated dissemination of aggregate trial results. The most frequently reported mode of disseminating of results was postal. Overall, the results report that participants evaluated receipt of trial results positively, with reported benefits including improved communication, demonstration of appreciation, improved retention, and engagement in future research. However, there were also some concerns about how well the dissemination was resourced and done, worries about emotional effects on participants especially when reporting unfavourable results, and frustration about the delay between the end of the trial and receipt of results. CONCLUSIONS: This scoping review has highlighted that few high-quality evaluative studies have been conducted that can provide evidence on the best ways to deliver results to trial participants. There have been relatively few qualitative studies that explore perspectives from diverse populations, and those that have been conducted are limited to a handful of clinical areas. The learning from these studies can be used as a platform for further research and to consider some core guiding principles of the opportunities and challenges when disseminating trial results to those who participated.


Assuntos
Ansiedade , Pessoal de Saúde , Humanos , Pesquisa Qualitativa
4.
J Intensive Care Soc ; 22(2): 127-135, 2021 May.
Artigo em Inglês | MEDLINE | ID: mdl-34025752

RESUMO

Background: There is increasing evidence that access to critical care services is not equitable. We aimed to investigate whether location of residence in Scotland impacts on the risk of admission to an Intensive Care Unit and on outcomes. Methods: This was a population-based Bayesian spatial analysis of adult patients admitted to Intensive Care Units in Scotland between January 2011 and December 2015. We used a Besag-York-Mollié model that allows us to make direct probabilistic comparisons between areas regarding risk of admission to Intensive Care Units and on outcomes. Results: A total of 17,596 patients were included. The five-year age- and sex-standardised admission rate was 352 per 100,000 residents. There was a cluster of Council Areas in the North-East of the country which had lower adjusted admission rates than the Scottish average. Midlothian, in South East Scotland had higher spatially adjusted admission rates than the Scottish average. There was no evidence of geographical variation in mortality. Conclusion: Access to critical care services in Scotland varies with location of residence. Possible reasons include differential co-morbidity burden, service provision and access to critical care services. In contrast, the probability of surviving an Intensive Care Unit admission, if admitted, does not show geographical variation.

5.
BMJ Open ; 11(4): e049093, 2021 04 29.
Artigo em Inglês | MEDLINE | ID: mdl-33926985

RESUMO

OBJECTIVES: Randomised controlled trials conducted using cohorts and routinely collected data, including registries, electronic health records and administrative databases, are increasingly used in healthcare intervention research. A Consolidated Standards of Reporting Trials (CONSORT) statement extension for trials conducted using cohorts and routinely collected data (CONSORT-ROUTINE) has been developed with the goal of improving reporting quality. This article describes the processes and methods used to develop the extension and decisions made to arrive at the final checklist. METHODS: The development process involved five stages: (1) identification of the need for a reporting guideline and project launch; (2) conduct of a scoping review to identify possible modifications to CONSORT 2010 checklist items and possible new extension items; (3) a three-round modified Delphi study involving key stakeholders to gather feedback on the checklist; (4) a consensus meeting to finalise items to be included in the extension, followed by stakeholder piloting of the checklist; and (5) publication, dissemination and implementation of the final checklist. RESULTS: 27 items were initially developed and rated in Delphi round 1, 13 items were rated in round 2 and 11 items were rated in round 3. Response rates for the Delphi study were 92 of 125 (74%) invited participants in round 1, 77 of 92 (84%) round 1 completers in round 2 and 62 of 77 (81%) round 2 completers in round 3. Twenty-seven members of the project team representing a variety of stakeholder groups attended the in-person consensus meeting. The final checklist includes five new items and eight modified items. The extension Explanation & Elaboration document further clarifies aspects that are important to report. CONCLUSION: Uptake of CONSORT-ROUTINE and accompanying Explanation & Elaboration document will improve conduct of trials, as well as the transparency and completeness of reporting of trials conducted using cohorts and routinely collected data.


Assuntos
Projetos de Pesquisa , Dados de Saúde Coletados Rotineiramente , Lista de Checagem , Consenso , Técnica Delfos
7.
J Clin Epidemiol ; 137: 14-22, 2021 09.
Artigo em Inglês | MEDLINE | ID: mdl-33652081

RESUMO

OBJECTIVE: To develop a core outcome set for the evaluation of interventions that aim to improve how people make decisions about whether to participate in randomized controlled trials (of healthcare interventions), the ELICIT Study. STUDY DESIGN: International mixed-method study involving a systematic review of existing outcomes, semi-structured interviews, an online Delphi survey, and a face-to-face consensus meeting. RESULTS: The literature review and stakeholder interviews (n = 25) initially identified 1045 reported outcomes that were grouped into 40 individually distinct outcomes. These 40 outcomes were scored for importance in two rounds of an online Delphi survey (n = 79), with 18 people attending the consensus meeting. Consensus was reached on 12 core outcomes: therapeutic misconception; comfort with decision; authenticity of decision; communication about the trial; empowerment; sense of altruism; equipoise; knowledge; salience of questions; understanding, how helpful the process was for decision making; and trial attrition. CONCLUSION: The ELICIT core outcome set is the first internationally agreed minimum set of outcomes deemed essential to be measured in all future studies evaluating interventions to improve decisions about participating in an randomized controlled trial. Use of the ELICIT core set will ensure that results from these trials are comparable and relevant to all stakeholders. REGISTRATION: COMET database - http://www.comet-initiative.org/Studies/Details/595.


Assuntos
Consentimento Livre e Esclarecido/normas , Ensaios Clínicos Controlados Aleatórios como Assunto/normas , Humanos , Cooperação Internacional , Resultado do Tratamento
8.
Pilot Feasibility Stud ; 7(1): 53, 2021 Feb 18.
Artigo em Inglês | MEDLINE | ID: mdl-33602340

RESUMO

BACKGROUND: There is a critical need for an intervention to improve nurses' eating and physical activity behaviours. As nurses spend a substantial proportion of their waking hours at work, concerted efforts to deliver such interventions in the workplace is growing. This study formed part of a multiphase programme of research that aimed to systematically develop an evidence-based and theory-informed workplace intervention to promote changes in eating and physical activity among nurses. METHODS: The intervention was developed iteratively, in line with Medical Research Council complex intervention guidelines. It involved four activities: (1) identifying the evidence base, (2) understanding the determinants of nurses' eating and physical activity behaviour change through theory-based qualitative interviews and survey, (3) identifying intervention options using the Behaviour Change Wheel, and (4) specifying intervention content and implementation options using a taxonomy of behaviour change techniques. RESULTS: Data from 13 randomised controlled trials indicated that workplace-based behaviour change interventions targeted to this population are effective in changing behaviour. The evidence base was, however, limited in quantity and quality. Nurses' beliefs about important factors determining their eating and physical activity behaviour were identified across 16 qualitative interviews and 245 survey responses, and key determinants included environmental context and resources, behavioural regulation, emotion, beliefs about consequences, knowledge and optimism. Based on these findings, 22 behaviour change techniques suitable for targeting the identified determinants were identified and combined into a potential workplace intervention. CONCLUSIONS: An evidence-based and theory-informed intervention tailored to the target population and setting has been explicitly conceptualised using a systematic approach. The proposed intervention addresses previous evidence gaps for the user population of nurses. Further to this, such an intervention, if implemented, has the potential to impact nurses' eating and physical activity behaviours and in turn, the health of nurses and the quality of healthcare delivery.

10.
Health Technol Assess ; 24(20): 1-98, 2020 04.
Artigo em Inglês | MEDLINE | ID: mdl-32369436

RESUMO

BACKGROUND: Late-stage medial compartment knee osteoarthritis can be treated using total knee replacement or partial (unicompartmental) knee replacement. There is high variation in treatment choice and insufficient evidence to guide selection. OBJECTIVE: To assess the clinical effectiveness and cost-effectiveness of partial knee replacement compared with total knee replacement in patients with medial compartment knee osteoarthritis. The findings are intended to guide surgical decision-making for patients, surgeons and health-care providers. DESIGN: This was a randomised, multicentre, pragmatic comparative effectiveness trial that included an expertise component. The target sample size was 500 patients. A web-based randomisation system was used to allocate treatments. SETTING: Twenty-seven NHS hospitals (68 surgeons). PARTICIPANTS: Patients with medial compartment knee osteoarthritis. INTERVENTIONS: The trial compared the overall management strategy of partial knee replacement treatment with total knee replacement treatment. No specified brand or subtype of implant was investigated. MAIN OUTCOME MEASURES: The Oxford Knee Score at 5 years was the primary end point. Secondary outcomes included activity scores, global health measures, transition items, patient satisfaction (Lund Score) and complications (including reoperation, revision and composite 'failure' - defined by minimal Oxford Knee Score improvement and/or reoperation). Cost-effectiveness was also assessed. RESULTS: A total of 528 patients were randomised (partial knee replacement, n = 264; total knee replacement, n = 264). The follow-up primary outcome response rate at 5 years was 88% and both operations had good outcomes. There was no significant difference between groups in mean Oxford Knee Score at 5 years (difference 1.04, 95% confidence interval -0.42 to 2.50). An area under the curve analysis of the Oxford Knee Score at 5 years showed benefit in favour of partial knee replacement over total knee replacement, but the difference was within the minimal clinically important difference [mean 36.6 (standard deviation 8.3) (n = 233), mean 35.1 (standard deviation 9.1) (n = 231), respectively]. Secondary outcome measures showed consistent patterns of benefit in the direction of partial knee replacement compared with total knee replacement although most differences were small and non-significant. Patient-reported improvement (transition) and reflection (would you have the operation again?) showed statistically significant superiority for partial knee replacement only, but both of these variables could be influenced by the lack of blinding. The frequency of reoperation (including revision) by treatment received was similar for both groups: 22 out of 245 for partial knee replacement and 28 out of 269 for total knee replacement patients. Revision rates at 5 years were 10 out of 245 for partial knee replacement and 8 out of 269 for total knee replacement. There were 28 'failures' of partial knee replacement and 38 'failures' of total knee replacement (as defined by composite outcome). Beyond 1 year, partial knee replacement was cost-effective compared with total knee replacement, being associated with greater health benefits (measured using quality-adjusted life-years) and lower health-care costs, reflecting lower costs of the index surgery and subsequent health-care use. LIMITATIONS: It was not possible to blind patients in this study and there was some non-compliance with the allocated treatment interventions. Surgeons providing partial knee replacement were relatively experienced with the procedure. CONCLUSIONS: Both total knee replacement and partial knee replacement are effective, offer similar clinical outcomes and have similar reoperation and complication rates. Some patient-reported measures of treatment approval were significantly higher for partial knee replacement than for total knee replacement. Partial knee replacement was more cost-effective (more effective and cost saving) than total knee replacement at 5 years. FUTURE WORK: Further (10-year) follow-up is in progress to assess the longer-term stability of these findings. TRIAL REGISTRATION: Current Controlled Trials ISRCTN03013488 and ClinicalTrials.gov NCT01352247. FUNDING: This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 24, No. 20. See the NIHR Journals Library website for further project information.


Assuntos
Artroplastia do Joelho/economia , Protocolos Clínicos , Análise Custo-Benefício/estatística & dados numéricos , Osteoartrite do Joelho/cirurgia , Resultado do Tratamento , Idoso , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Satisfação do Paciente , Anos de Vida Ajustados por Qualidade de Vida , Reoperação/estatística & dados numéricos , Inquéritos e Questionários , Reino Unido
11.
J Clin Epidemiol ; 125: 30-37, 2020 09.
Artigo em Inglês | MEDLINE | ID: mdl-32422248

RESUMO

OBJECTIVE: It is now more than 50 years since the concepts of explanatory and pragmatic attitudes toward trials were first discussed by Schwartz and Lellouch in their influential 1967 paper. Since then, there has been increasing focus on design aspects that may be consistent with more pragmatic attitudes within clinical trials, and a number of tools developed to assist investigators prospectively think about their trial design. Researchers have subsequently expressed interest in using these tools retrospectively to characterize trials as pragmatic or explanatory. RESULTS: We suggest that recent attempts to retrospectively dichotomize trials solely on the basis of quantitative scoring of trial design features are flawed. Instead, we argue that there is a need to consider both the intent and design when assessing the degree of pragmatism within a trial. CONCLUSION: The practical implication of our suggestion for trial reporting is that investigators should explicitly state the intent of the trial through a clear articulation of the decision that they hope will be informed by the trial results. This should be coupled with a completed PRagmatic-Explanatory Continuum Indicator Summary 2 assessment (or similar) with an explanation of study design choices to appropriately assess whether the study design is consistent with the study intent. We believe this will assist reviewers and knowledge users in making assessments of trials.


Assuntos
Tomada de Decisões , Ensaios Clínicos Pragmáticos como Assunto/métodos , Humanos , Projetos de Pesquisa
12.
Lancet ; 395(10226): 828-838, 2020 03 07.
Artigo em Inglês | MEDLINE | ID: mdl-32145797

RESUMO

Placebo comparisons are increasingly being considered for randomised trials assessing the efficacy of surgical interventions. The aim of this Review is to provide a summary of knowledge on placebo controls in surgical trials. A placebo control is a complex type of comparison group in the surgical setting and, although powerful, presents many challenges. This Review outlines what a placebo control entails and present understanding of this tool in the context of surgery. We consider when placebo controls in surgery are acceptable (and when they are desirable) in terms of ethical arguments and regulatory requirements, how a placebo control should be designed, how to identify and mitigate risk for participants in these trials, and how such trials should be done and interpreted. Use of placebo controls is justified in randomised controlled trials of surgical interventions provided there is a strong scientific and ethical rationale. Surgical placebos might be most appropriate when there is poor evidence for the efficacy of the procedure and a justified concern that results of a trial would be associated with high risk of bias, particularly because of the placebo effect. Feasibility work is recommended to optimise the design and implementation of randomised controlled trials. This Review forms an outline for best practice and provides guidance, in the form of the Applying Surgical Placebo in Randomised Evaluations (known as ASPIRE) checklist, for those considering the use of a placebo control in a surgical randomised controlled trial.


Assuntos
Placebos , Ensaios Clínicos Controlados Aleatórios como Assunto , Procedimentos Cirúrgicos Operatórios , Guias como Assunto , Humanos , Ensaios Clínicos Controlados Aleatórios como Assunto/ética , Ensaios Clínicos Controlados Aleatórios como Assunto/normas , Projetos de Pesquisa
13.
J Clin Epidemiol ; 119: 109-116, 2020 03.
Artigo em Inglês | MEDLINE | ID: mdl-31786153

RESUMO

OBJECTIVES: To examine key methodological considerations for using a placebo intervention in randomized controlled trials (RCTs) evaluating invasive procedures, including surgery. STUDY DESIGN AND SETTING: RCTs comparing an invasive procedure with a placebo were included in this systematic review. Articles published from database inception to December 31, 2017, were retrieved from Ovid MEDLINE, Ovid EMBASE and CENTRAL electronic databases, by handsearching references and expert knowledge. Data on trial characteristics (clinical area, nature of invasive procedure, number of patients and centers) and key methodological (rationale for using placebos, minimization of risk, information provision, offering the treatment intervention to patients randomized to placebo, delivery of cointerventions, and intervention standardization and fidelity) were extracted and summarized descriptively. RESULTS: One hundred thirteen articles reporting 96 RCTs were identified. Most were conducted in gastrointestinal surgery (n = 40, 42%) and evaluated minimally invasive procedures (n = 44, 46%). Over two-thirds randomized fewer than 100 patients (n = 65, 68%) and a third were single center (n = 31, 32%). A third (n = 33, 34%) did not report a rationale for using a placebo. Most common strategies to minimize patient risk were operator skill (n = 22, 23%) and independent data monitoring (n = 28, 29%). Provision of patient information regarding placebo use was infrequently reported (n = 11, 11%). Treatment interventions were offered to patients randomized to placebo in 43 trials (45%). Cointerventions were inconsistently reported, but 64 trials (67%) stated that anesthesia was matched between groups. Attempts to standardize interventions and monitor their delivery were reported in n = 7, (7%) and n = 4, (4%) trials, respectively. CONCLUSION: Most placebo-controlled trials in surgery evaluate minor surgical procedures and currently there is inconsistent reporting of key trial methods. There is a need for guidance to optimize the transparency of trial reporting in this area.


Assuntos
Coleta de Dados/métodos , Melhoria de Qualidade , Ensaios Clínicos Controlados Aleatórios como Assunto/métodos , Projetos de Pesquisa , Procedimentos Cirúrgicos Operatórios/métodos , Humanos , Placebos
14.
N Engl J Med ; 381(10): 912-922, 2019 09 05.
Artigo em Inglês | MEDLINE | ID: mdl-31483962

RESUMO

BACKGROUND: Endovenous laser ablation and ultrasound-guided foam sclerotherapy are recommended alternatives to surgery for the treatment of primary varicose veins, but their long-term comparative effectiveness remains uncertain. METHODS: In a randomized, controlled trial involving 798 participants with primary varicose veins at 11 centers in the United Kingdom, we compared the outcomes of laser ablation, foam sclerotherapy, and surgery. Primary outcomes at 5 years were disease-specific quality of life and generic quality of life, as well as cost-effectiveness based on models of expected costs and quality-adjusted life-years (QALYs) gained that used data on participants' treatment costs and scores on the EuroQol EQ-5D questionnaire. RESULTS: Quality-of-life questionnaires were completed by 595 (75%) of the 798 trial participants. After adjustment for baseline scores and other covariates, scores on the Aberdeen Varicose Vein Questionnaire (on which scores range from 0 to 100, with lower scores indicating a better quality of life) were lower among patients who underwent laser ablation or surgery than among those who underwent foam sclerotherapy (effect size [adjusted differences between groups] for laser ablation vs. foam sclerotherapy, -2.86; 95% confidence interval [CI], -4.49 to -1.22; P<0.001; and for surgery vs. foam sclerotherapy, -2.60; 95% CI, -3.99 to -1.22; P<0.001). Generic quality-of-life measures did not differ among treatment groups. At a threshold willingness-to-pay ratio of £20,000 ($28,433 in U.S. dollars) per QALY, 77.2% of the cost-effectiveness model iterations favored laser ablation. In a two-way comparison between foam sclerotherapy and surgery, 54.5% of the model iterations favored surgery. CONCLUSIONS: In a randomized trial of treatments for varicose veins, disease-specific quality of life 5 years after treatment was better after laser ablation or surgery than after foam sclerotherapy. The majority of the probabilistic cost-effectiveness model iterations favored laser ablation at a willingness-to-pay ratio of £20,000 ($28,433) per QALY. (Funded by the National Institute for Health Research; CLASS Current Controlled Trials number, ISRCTN51995477.).


Assuntos
Procedimentos Endovasculares , Terapia a Laser , Qualidade de Vida , Escleroterapia , Varizes/terapia , Adulto , Análise Custo-Benefício , Procedimentos Endovasculares/economia , Feminino , Seguimentos , Humanos , Análise de Intenção de Tratamento , Terapia a Laser/economia , Masculino , Pessoa de Meia-Idade , Anos de Vida Ajustados por Qualidade de Vida , Escleroterapia/economia , Escleroterapia/métodos , Inquéritos e Questionários , Resultado do Tratamento , Ultrassonografia de Intervenção , Varizes/cirurgia
15.
Lancet ; 394(10200): 746-756, 2019 08 31.
Artigo em Inglês | MEDLINE | ID: mdl-31326135

RESUMO

BACKGROUND: Late-stage isolated medial knee osteoarthritis can be treated with total knee replacement (TKR) or partial knee replacement (PKR). There is high variation in treatment choice and little robust evidence to guide selection. The Total or Partial Knee Arthroplasty Trial (TOPKAT) therefore aims to assess the clinical effectiveness and cost-effectiveness of TKR versus PKR in patients with medial compartment osteoarthritis of the knee, and this represents an analysis of the main endpoints at 5 years. METHODS: Our multicentre, pragmatic randomised controlled trial was done at 27 UK sites. We used a combined expertise-based and equipoise-based approach, in which patients with isolated osteoarthritis of the medial compartment of the knee and who satisfied general requirements for a medial PKR were randomly assigned (1:1) to receive PKR or TKR by surgeons who were either expert in and willing to perform both surgeries or by a surgeon with particular expertise in the allocated procedure. The primary endpoint was the Oxford Knee Score (OKS) 5 years after randomisation in all patients assigned to groups. Health-care costs (in UK 2017 prices) and cost-effectiveness were also assessed. This trial is registered with ISRCTN (ISRCTN03013488) and ClinicalTrials.gov (NCT01352247). FINDINGS: Between Jan 18, 2010, and Sept 30, 2013, we assessed 962 patients for their eligibility, of whom 431 (45%) patients were excluded (121 [13%] patients did not meet the inclusion criteria and 310 [32%] patients declined to participate) and 528 (55%) patients were randomly assigned to groups. 94% of participants responded to the follow-up survey 5 years after their operation. At the 5-year follow-up, we found no difference in OKS between groups (mean difference 1·04, 95% CI -0·42 to 2·50; p=0·159). In our within-trial cost-effectiveness analysis, we found that PKR was more effective (0·240 additional quality-adjusted life-years, 95% CI 0·046 to 0·434) and less expensive (-£910, 95% CI -1503 to -317) than TKR during the 5 years of follow-up. This finding was a result of slightly better outcomes, lower costs of surgery, and lower follow-up health-care costs with PKR than TKR. INTERPRETATION: Both TKR and PKR are effective, offer similar clinical outcomes, and result in a similar incidence of re-operations and complications. Based on our clinical findings, and results regarding the lower costs and better cost-effectiveness with PKR during the 5-year study period, we suggest that PKR should be considered the first choice for patients with late-stage isolated medial compartment osteoarthritis. FUNDING: National Institute for Health Research Health Technology Assessment Programme.


Assuntos
Artroplastia do Joelho/economia , Artroplastia do Joelho/métodos , Osteoartrite do Joelho/cirurgia , Idoso , Análise Custo-Benefício , Feminino , Seguimentos , Custos de Cuidados de Saúde/estatística & dados numéricos , Humanos , Tempo de Internação/estatística & dados numéricos , Masculino , Pessoa de Meia-Idade , Duração da Cirurgia , Dor Pós-Operatória/etiologia , Anos de Vida Ajustados por Qualidade de Vida , Reoperação/estatística & dados numéricos , Inquéritos e Questionários , Resultado do Tratamento
16.
Trials ; 20(1): 401, 2019 Jul 05.
Artigo em Inglês | MEDLINE | ID: mdl-31277693

RESUMO

Ethical requirements of informed consent stipulate that patients approached to participate in a clinical trial be provided with written information that must cover key aspects of the trial. For consent to be deemed "informed", potential participants should be provided with a range of information about the trials (e.g., the trial aims, the anticipated benefits and potential risks of the trial, and their right to withdraw consent at any time). However, it is well documented that simple provision of this information does not ensure that participants make truly informed decisions. Decision aids, tools that have been shown in a treatment and screening context to support better-quality decisions, are emerging as a possible vehicle to support decision making about trial participation. However, information on how they should best be developed and evaluated in a clinical trial context is lacking. Therefore, this article, drawing on theoretical and empirical insights, outlines a framework for the development and evaluation of decision aids for people considering taking part in a clinical trial.


Assuntos
Comportamento de Escolha , Ensaios Clínicos como Assunto/métodos , Técnicas de Apoio para a Decisão , Consentimento Livre e Esclarecido , Seleção de Pacientes , Sujeitos da Pesquisa/psicologia , Comportamento de Escolha/ética , Ensaios Clínicos como Assunto/ética , Humanos , Consentimento Livre e Esclarecido/ética , Seleção de Pacientes/ética , Participação dos Interessados
17.
Res Integr Peer Rev ; 3: 9, 2018.
Artigo em Inglês | MEDLINE | ID: mdl-30397513

RESUMO

Background: Randomized controlled trials (RCTs) are often complex and expensive to perform. Less than one third achieve planned recruitment targets, follow-up can be labor-intensive, and many have limited real-world generalizability. Designs for RCTs conducted using cohorts and routinely collected health data, including registries, electronic health records, and administrative databases, have been proposed to address these challenges and are being rapidly adopted. These designs, however, are relatively recent innovations, and published RCT reports often do not describe important aspects of their methodology in a standardized way. Our objective is to extend the Consolidated Standards of Reporting Trials (CONSORT) statement with a consensus-driven reporting guideline for RCTs using cohorts and routinely collected health data. Methods: The development of this CONSORT extension will consist of five phases. Phase 1 (completed) consisted of the project launch, including fundraising, the establishment of a research team, and development of a conceptual framework. In phase 2, a systematic review will be performed to identify publications (1) that describe methods or reporting considerations for RCTs conducted using cohorts and routinely collected health data or (2) that are protocols or report results from such RCTs. An initial "long list" of possible modifications to CONSORT checklist items and possible new items for the reporting guideline will be generated based on the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) and The REporting of studies Conducted using Observational Routinely-collected health Data (RECORD) statements. Additional possible modifications and new items will be identified based on the results of the systematic review. Phase 3 will consist of a three-round Delphi exercise with methods and content experts to evaluate the "long list" and generate a "short list" of key items. In phase 4, these items will serve as the basis for an in-person consensus meeting to finalize a core set of items to be included in the reporting guideline and checklist. Phase 5 will involve drafting the checklist and elaboration-explanation documents, and dissemination and implementation of the guideline. Discussion: Development of this CONSORT extension will contribute to more transparent reporting of RCTs conducted using cohorts and routinely collected health data.

18.
BMC Med Ethics ; 19(1): 90, 2018 11 20.
Artigo em Inglês | MEDLINE | ID: mdl-30458809

RESUMO

BACKGROUND: Randomized controlled trial (RCT) trial designs exist on an explanatory-pragmatic spectrum, depending on the degree to which a study aims to address a question of efficacy or effectiveness. As conceptualized by Schwartz and Lellouch in 1967, an explanatory approach to trial design emphasizes hypothesis testing about the mechanisms of action of treatments under ideal conditions (efficacy), whereas a pragmatic approach emphasizes testing effectiveness of two or more available treatments in real-world conditions. Interest in, and the number of, pragmatic trials has grown substantially in recent years, with increased recognition by funders and stakeholders worldwide of the need for credible evidence to inform clinical decision-making. This increase has been accompanied by the onset of learning healthcare systems, as well as an increasing focus on patient-oriented research. However, pragmatic trials have ethical challenges that have not yet been identified or adequately characterized. The present study aims to explore the views of key stakeholders with respect to ethical issues raised by the design and conduct of pragmatic trials. It is embedded within a large, four-year project that seeks to develop guidance for the ethical design and conduct of pragmatic trials. As a first step, this study will address important gaps in the current empirical literature with respect to identifying a comprehensive range of ethical issues arising from the design and conduct of pragmatic trials. By opening up a broad range of topics for consideration within our parallel ethical analysis, we will extend the current debate, which has largely emphasized issues of consent, to the range of ethical considerations that may flow from specific design choices. METHODS: Semi-structured interviews with key stakeholders (e.g. trialists, methodologists, lay members of study teams, bioethicists, and research ethics committee members), across multiple jurisdictions, identified based on their known experience and/or expertise with pragmatic trials. DISCUSSION: We expect that the study outputs will be of interest to a wide range of knowledge users including trialists, ethicists, research ethics committees, journal editors, regulators, healthcare policymakers, research funders and patient groups. All publications will adhere to the Tri-Agency Open Access Policy on Publications.


Assuntos
Atitude do Pessoal de Saúde , Ensaios Clínicos Pragmáticos como Assunto/ética , Protocolos Clínicos , Humanos , Entrevistas como Assunto , Ensaios Clínicos Pragmáticos como Assunto/métodos , Ensaios Clínicos Controlados Aleatórios como Assunto/ética , Ensaios Clínicos Controlados Aleatórios como Assunto/métodos , Projetos de Pesquisa , Pesquisadores/psicologia
20.
Trials ; 19(1): 525, 2018 Sep 27.
Artigo em Inglês | MEDLINE | ID: mdl-30261933

RESUMO

BACKGROUND: There is a widely recognized need for more pragmatic trials that evaluate interventions in real-world settings to inform decision-making by patients, providers, and health system leaders. Increasing availability of electronic health records, centralized research ethics review, and novel trial designs, combined with support and resources from governments worldwide for patient-centered research, have created an unprecedented opportunity to advance the conduct of pragmatic trials, which can ultimately improve patient health and health system outcomes. Such trials raise ethical issues that have not yet been fully addressed, with existing literature concentrating on regulations in specific jurisdictions rather than arguments grounded in ethical principles. Proposed solutions (e.g. using different regulations in "learning healthcare systems") are speculative with no guarantee of improvement over existing oversight procedures. Most importantly, the literature does not reflect a broad vision of protecting the core liberty and welfare interests of research participants. Novel ethical guidance is required. We have assembled a team of ethicists, trialists, methodologists, social scientists, knowledge users, and community members with the goal of developing guidance for the ethical design and conduct of pragmatic trials. METHODS: Our project will combine empirical and conceptual work and a consensus development process. Empirical work will: (1) identify a comprehensive list of ethical issues through interviews with a small group of key informants (e.g. trialists, ethicists, chairs of research ethics committees); (2) document current practices by reviewing a random sample of pragmatic trials and surveying authors; (3) elicit views of chairs of research ethics committees through surveys in Canada, UK, USA, France, and Australia; and (4) elicit views and experiences of community members and health system leaders through focus groups and surveys. Conceptual work will consist of an ethical analysis of identified issues and the development of new ethical solutions, outlining principles, policy options, and rationales. The consensus development process will involve an independent expert panel to develop a final guidance document. DISCUSSION: Planned output includes manuscripts, educational materials, and tailored guidance documents to inform and support researchers, research ethics committees, journal editors, regulators, and funders in the ethical design and conduct of pragmatic trials.


Assuntos
Comitês de Ética em Pesquisa/ética , Ética em Pesquisa , Ensaios Clínicos Pragmáticos como Assunto/ética , Projetos de Pesquisa , Consenso , Conferências de Consenso como Assunto , Comitês de Ética em Pesquisa/normas , Humanos , Comunicação Interdisciplinar , Estudos Multicêntricos como Assunto , Guias de Prática Clínica como Assunto , Ensaios Clínicos Pragmáticos como Assunto/métodos , Ensaios Clínicos Pragmáticos como Assunto/normas , Projetos de Pesquisa/normas , Literatura de Revisão como Assunto , Participação dos Interessados
SELEÇÃO DE REFERÊNCIAS
DETALHE DA PESQUISA
...