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Challenges and lessons learned in recruiting participants for school-based disease prevention programs during COVID-19. 2019冠状病毒病期间招募校本疾病预防项目参与者的挑战和经验教训。
IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-24 eCollection Date: 2024-12-01 DOI: 10.1016/j.conctc.2024.101399
Yelena P Wu, Elise K Brunsgaard, Nic Siniscalchi, Tammy Stump, Heather Smith, Douglas Grossman, Jakob Jensen, David B Buller, Jennifer L Hay, Jincheng Shen, Benjamin A Haaland, Kenneth P Tercyak

Schools provide an ideal setting for delivery of disease prevention programs due to the ability to deliver health education and counseling, including health behavior interventions, to large numbers of students. However, the remote and hybrid learning models that arose during the coronavirus (COVID-19) pandemic created obstacles to these efforts. In this article, we provide insights on collaborating with schools to deliver disease prevention programming during the height of the COVID-19 pandemic, and in subsequent years. We illustrate these strategies by drawing upon our firsthand research experiences engaging high schools in a school-based cancer prevention trial focused on sun safety. Delivery of a cluster-randomized trial of a school-based skin cancer prevention program was initiated in the spring of 2020 at the onset of the COVID-19 pandemic in the U.S. We present multilevel evaluation data on strategies used to reach schools remotely and share lessons learned that may inform similar approaches moving forward during times of crises. Although the COVID-19 pandemic interrupted school-based recruitment for this trial, enrollment improved one year later and did not appear to differ between rural and urban schools. Recruitment strategies and trial-related procedures were modified to address new challenges brought about by the pandemic. Despite the COVID-19 crisis altering US classrooms, disease prevention programming can continue to be offered within schools, given close community partnerships and new adaptations to the ways in which such programming and research are conducted.

由于学校能够向大量学生提供健康教育和咨询,包括健康行为干预,因此学校为提供疾病预防项目提供了理想的环境。然而,在冠状病毒(COVID-19)大流行期间出现的远程和混合学习模式给这些努力带来了障碍。在本文中,我们提供了在COVID-19大流行高峰期以及随后几年与学校合作提供疾病预防规划的见解。我们通过利用我们的第一手研究经验来说明这些策略,这些经验让高中参与了一项以学校为基础的以阳光安全为重点的癌症预防试验。2020年春季,在美国COVID-19大流行爆发之际,一项基于学校的皮肤癌预防项目的集群随机试验开始了。我们提出了用于远程到达学校的策略的多层次评估数据,并分享了经验教训,这些经验教训可能会为危机时期的类似方法提供信息。尽管COVID-19大流行中断了以学校为基础的试验招募,但一年后入学率有所提高,农村和城市学校之间似乎没有差异。修订了征聘战略和与试验有关的程序,以应对大流行病带来的新挑战。尽管COVID-19危机改变了美国的课堂,但鉴于密切的社区伙伴关系以及对此类规划和研究进行方式的新调整,学校可以继续提供疾病预防规划。
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引用次数: 0
The effects of alpha-lipoic acid transdermal patch for local subcutaneous fat reduction: A randomized, placebo-controlled, clinical trial in overweight volunteers α-硫辛酸透皮贴片对减少局部皮下脂肪的效果:针对超重志愿者的随机安慰剂对照临床试验
IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-24 DOI: 10.1016/j.conctc.2024.101402
Kanidta Sooklert , Sasin Thamakaison , Siwaporn Nilyai , Sarocha Cherdchom , Rojrit Rojanathanes , Amornpun Sereemaspun

Background

Combating obesity is challenging, as anti-obesity compounds lose effectiveness or cause severe side effects when delivered via conventional routes. Thus, there is a need for new, effective treatment routes that are home-based and safe for long-term use. This double-blind, placebo-controlled clinical trial aimed to investigate the efficacy of a biocellulose transdermal patch containing α-lipoic acid (ALA), an anti-obesity compound, in reducing subcutaneous fat accumulation.

Methods

One hundred and sixteen overweight participants (average age 37.96 ± 7.80 years) were recruited for the study. They were randomly assigned to apply either the calcium citrate nanoparticle-encapsulated ALA transdermal patch or a placebo on their arm. The participants’ body weight, height, blood lipid profile (cholesterol, triglyceride, low-density lipoprotein, and high-density lipoprotein), arm circumference, triceps skin fold, and subcutaneous fat thickness were recorded at baseline and at the 2-week follow-up.

Results

The mean arm circumference did not show any significant difference from baseline, whereas the triceps skinfold and subcutaneous fat thickness showed a significant reduction. The 2-week treatment did not significantly alter the plasma LDL, HDL, and triglyceride levels of the participants, but it significantly reduced the total cholesterol level.

Conclusion

This study reports the successful reduction of subcutaneous fat of the calcium citrate nanoparticle-encapsulated ALA transdermal patches. The transdermal patches could be used as a safe and effective home-based solution for combating obesity.
背景防治肥胖症具有挑战性,因为通过传统途径给药时,抗肥胖化合物会失去疗效或产生严重的副作用。因此,我们需要新的、有效的、可在家中长期安全使用的治疗途径。这项双盲、安慰剂对照临床试验旨在研究含有抗肥胖化合物α-硫辛酸(ALA)的生物纤维素透皮贴片在减少皮下脂肪堆积方面的疗效。他们被随机分配在手臂上贴上柠檬酸钙纳米胶囊 ALA 透皮贴或安慰剂。研究人员在基线和两周随访时记录了参与者的体重、身高、血脂(胆固醇、甘油三酯、低密度脂蛋白和高密度脂蛋白)、臂围、肱三头肌皮肤褶皱和皮下脂肪厚度。结论本研究报告了柠檬酸钙纳米胶囊 ALA 透皮贴片成功减少皮下脂肪的情况。这种透皮贴片可作为一种安全有效的家庭肥胖症防治方案。
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引用次数: 0
Noninvasive anatomical assessment for ruling out hemodynamically relevant coronary artery anomalies in adults – A comparison of coronary-CT to invasive coronary angiography: The NARCO study design 用于排除与血流动力学相关的成人冠状动脉异常的无创解剖评估 - 冠状动脉 CT 与有创冠状动脉造影的比较:NARCO 研究设计
IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-19 DOI: 10.1016/j.conctc.2024.101394
Marius R. Bigler , Anselm W. Stark , Isaac Shiri , Joel Illi , Matthias Siepe , Federico Caobelli , Andreas A. Giannopoulos , Ronny R. Buechel , Andreas Haeberlin , Dominik Obrist , Lorenz Räber , Christoph Gräni

Background

Anomalous aortic origin of a coronary artery (AAOCA) is a rare congenital heart disease, potentially leading to myocardial ischemia and adverse cardiac events. As the sole presence of AAOCA does not always imply a revascularization, a detailed anatomical and functional analysis is crucial for clinical decision-making. Currently, invasive coronary angiography is the gold-standard method for a thorough hemodynamic assessment of AAOCA. However, due to its invasive nature, the development of noninvasive diagnostic alternatives is desired.

Methods

In the NARCO trial, patients with AAOCA will undergo coronary computed tomography angiography (CCTA) to assess anatomical high-risk features followed by a vessel-based (i.e. invasive measurement with fractional flow reserve and intravascular imaging under a dobutamine-volume challenge) and a myocardium-based (i.e. nuclear imaging) ischemia testing. Comparison of noninvasive and invasive imaging will be performed. Additionally, explorative analysis of post-processing advanced computational fluid dynamics (CFD) and 3D printing will be performed to unravel the pathophysiologic mechanism of myocardial ischemia in AAOCA.

Aims

Our primary aim is to define characteristics of anatomical high-risk features (using CCTA) to rule out noninvasively hemodynamically relevant anomalous vessels in AAOCA patients. The secondary aim is to investigate the underlying pathophysiology of AAOCA-related hemodynamic relevance using advanced techniques such as CFD and 3D printing.

Conclusions

The NARCO trial will help to optimize AAOCA patient selection for revascularization by improving risk stratification and ruling out hemodynamic relevance noninvasively and, therefore, preventing unnecessary downstream testing and/or costly interventions in patients with AAOCA.
背景冠状动脉主动脉起源异常(AAOCA)是一种罕见的先天性心脏病,可能导致心肌缺血和不良心脏事件。由于仅存在 AAOCA 并不意味着一定要进行血管重建,因此详细的解剖和功能分析对临床决策至关重要。目前,有创冠状动脉造影术是对 AAOCA 进行全面血液动力学评估的黄金标准方法。方法在 NARCO 试验中,AAOCA 患者将接受冠状动脉计算机断层扫描(CCTA),以评估解剖学上的高危特征,然后进行基于血管(即多巴酚丁胺容量挑战下的有创测量、分数血流储备和血管内成像)和基于心肌(即核成像)的缺血测试。将对无创和有创成像进行比较。此外,还将对后处理高级计算流体动力学(CFD)和三维打印进行探索性分析,以揭示 AAOCA 患者心肌缺血的病理生理机制。结论NARCO试验将有助于优化AAOCA患者的血管再通选择,改善风险分层和无创排除血流动力学相关性,从而避免对AAOCA患者进行不必要的下游检测和/或昂贵的干预。
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引用次数: 0
Defining methods to improve eSource site start-up practices 确定改进电子资源网站启动做法的方法
IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-14 DOI: 10.1016/j.conctc.2024.101391
Amy E. Cramer , Linda S. King , Michael T. Buckley , Peter Casteleyn , Cory Ennis , Muayad Hamidi , Gonçalo M.C. Rodrigues , Denise C. Snyder , Aruna Vattikola , Eric L. Eisenstein

Background

eSource software that transfers patient electronic health record data into a clinical trial electronic case report form holds promise for increasing data quality while reducing data collection, monitoring and source document verification costs. Integrating eSource into multicenter clinical trial start-up procedures could facilitate the use of eSource technologies in clinical trials.

Methods

We conducted a qualitative integrative analysis to identify eSource site start-up key steps, challenges that might occur in executing those steps, and potential solutions to those challenges. We then conducted a value analysis to determine the challenges and solutions with the greatest impacts for eSource implementation teams.

Results

There were 16 workshop participants: 10 pharmaceutical sponsor, 3 academic site, and 1 eSource vendor representative. Participants identified 36 Site Start-Up Key Steps, 11 Site Start-Up Challenges, and 14 Site Start-Up Solutions for eSource-enabled studies. Participants also identified 77 potential impacts of the Challenges upon the Site Start-Up Key Steps and 70 ways in which the Solutions might impact Site Start-Up Challenges. The most important Challenges were: [1] not being able to identify a site eSource champion and [2] not agreeing on an eSource approach. The most important Solutions were: [1] eSource vendors accepting electronic data in the Health Level 7 Fast Healthcare Interoperability Resources (HL7® FHIR®) standard, [2] creating standard content for eSource-related legal documents, and [3] creating a common eSource site readiness checklist.

Conclusions

Site start-up for eSource-enabled multi-center clinical trials is a complex socio-technical problem. This study's Start-Up Solutions provide initial steps for scalable eSource implementation.
背景eSource软件能将患者电子健康记录数据传输到临床试验电子病例报告表中,有望提高数据质量,同时降低数据收集、监控和源文件验证成本。将 eSource 整合到多中心临床试验启动程序中可促进 eSource 技术在临床试验中的应用。方法我们进行了定性综合分析,以确定 eSource 研究机构启动的关键步骤、执行这些步骤时可能遇到的挑战以及应对这些挑战的潜在解决方案。然后,我们进行了价值分析,以确定对 eSource 实施团队影响最大的挑战和解决方案:研讨会共有 16 位参与者:10 位制药赞助商、3 位学术机构和 1 位 eSource 供应商代表。与会者确定了启用 eSource 研究的 36 个研究机构启动关键步骤、11 个研究机构启动挑战和 14 个研究机构启动解决方案。与会者还确定了 77 个 "挑战 "对 "研究机构启动关键步骤 "的潜在影响,以及 70 种 "解决方案 "可能对 "研究机构启动挑战 "产生影响的方式。最重要的挑战是[1] 无法确定站点电子资源负责人;[2] 无法就电子资源方法达成一致。最重要的解决方案是[1] 电子源供应商接受健康等级 7 快速医疗保健互操作性资源 (HL7® FHIR®) 标准中的电子数据,[2] 为电子源相关法律文件创建标准内容,以及 [3] 创建通用的电子源研究机构准备情况检查表。本研究的 "启动解决方案 "为可扩展的 eSource 实施提供了初始步骤。
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引用次数: 0
A Bayesian adaptive feasibility design for rare diseases 针对罕见疾病的贝叶斯自适应可行性设计
IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-09 DOI: 10.1016/j.conctc.2024.101392
Maureen M. Churipuy , Shirin Golchi , Marie Hudson , Sabrina Hoa
It is important for researchers to carefully assess the feasibility of a clinical trial prior to the launch of the study. One feasibility aspect that needs to be considered includes whether investigators can expect to successfully achieve the sample size needed for their trial. In this manuscript, we present a Bayesian design in which data collected during a pilot study is used to predict the feasibility of a planned phase III trial. Specifically, we outline a model that predicts a target sample size obtained from the Gamma-Poisson distribution. In a simulation study, we showcase the utility of the proposed design by applying it to a phase III trial designed to assess the efficacy of mycophenolate mofetil in individuals with mild systemic sclerosis. We demonstrate that the predictive nature of the proposed design is particularly useful for rare disease clinical trials and has the potential to greatly increase their efficiency.
对于研究人员来说,在启动研究之前仔细评估临床试验的可行性非常重要。需要考虑的一个可行性方面包括研究人员是否有望成功达到试验所需的样本量。在本手稿中,我们介绍了一种贝叶斯设计,利用试验研究期间收集的数据来预测计划中的 III 期试验的可行性。具体来说,我们概述了一个模型,该模型可预测从伽马-泊松分布中获得的目标样本量。在一项模拟研究中,我们将所提出的设计应用于一项 III 期试验,以评估霉酚酸酯对轻度系统性硬化症患者的疗效,从而展示了该设计的实用性。我们证明,所提设计的预测性对罕见病临床试验特别有用,并有可能大大提高其效率。
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引用次数: 0
Reasons for declining participation in inpatient research among historically minoritized racial and ethnic communities: A scoping review 历史上少数种族和民族社区参与住院病人研究减少的原因:范围审查
IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-07 DOI: 10.1016/j.conctc.2024.101386
Poyani Bavishi , Alyssa A. Grimshaw , Oscar F. Rojas Perez , Brian D. Kiluk , E. Jennifer Edelman

Background

To promote equitable recruitment for studies conducted in the inpatient hospital setting, we sought to characterize reasons why individuals, both from historically minoritized racial and ethnic groups and the broader patient population, refuse participation in clinical trials within inpatient settings.

Methods

An exhaustive search of the literature was conducted in Cochrane Library, Google Scholar, Embase, MEDLINE, PubMed, Scopus, and Web of Science databases to find relevant articles published from the inception of each database to April 30, 2023. Studies recruiting patients during their inpatient stay and reporting reasons for refusing participation in clinical trials met the inclusion criteria.

Results

The search resulted in 2264 citations, of which 22 were included. Fourteen did not report data related to race, while 19 reported no ethnicity data. Reasons for refusal across trials included study burden and inconvenience (n = 16), transportation and logistical issues (n = 13), lack of interest in research (n = 12), and refusal to be randomized (n = 10). Prominent concepts included the importance of incorporating social support systems in consenting processes, lack of efforts to include data or recruitment efforts for individuals from minoritized groups, and physician involvement in recruitment.

Discussion

To enhance participation among historically minoritized communities in clinical trials, greater efforts must be made to collect demographic information and document refusal reasons to inform future recruitment methods. Strategies include proactively accounting for culture and language differences in study design and recruitment and intentionally engaging social support networks. Limiting study burden and logistics and optimizing collaborations between clinical and research teams would promote accessibility and foster patient trust.
背景为了促进在医院住院环境中进行的研究的公平招募,我们试图找出历史上少数种族和民族群体以及更广泛的患者群体拒绝参与住院环境中临床试验的原因。方法在Cochrane Library、Google Scholar、Embase、MEDLINE、PubMed、Scopus和Web of Science数据库中进行了详尽的文献检索,以找到从每个数据库建立之初到2023年4月30日期间发表的相关文章。在住院期间招募患者并报告拒绝参与临床试验原因的研究符合纳入标准。其中 14 篇未报告种族相关数据,19 篇未报告种族数据。各试验的拒绝原因包括研究负担和不便(16 例)、交通和后勤问题(13 例)、对研究缺乏兴趣(12 例)以及拒绝随机化(10 例)。突出的概念包括在同意过程中纳入社会支持系统的重要性、缺乏针对少数群体个人的数据或招募工作,以及医生参与招募。策略包括在研究设计和招募中主动考虑文化和语言差异,并有意识地让社会支持网络参与进来。限制研究负担和后勤工作,优化临床和研究团队之间的合作,将促进研究的可及性并增进患者的信任。
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引用次数: 0
Modeling severe uncontrolled asthma: Transitioning away from health states 模拟严重失控的哮喘:从健康状态过渡
IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-07 DOI: 10.1016/j.conctc.2024.101390
Tereza Lanitis , Asif H. Khan , Irina Proskorovsky , Ivan Houisse , Andreas Kuznik , Siddhesh Kamat , Conrado Franco-Villalobos , Florence Joulain

Background

Models developed to date to simulate long-term outcomes of asthma have been criticized for lacking granularity and ignoring disease heterogeneity.

Objective

To propose an alternative approach to modeling asthma and apply it to model long-term outcomes in a population with moderate-to-severe type 2 asthma (patients with raised fractional exhaled nitric oxide or eosinophils) and treated with conventional therapy.

Methods

A discretely integrated condition event (DICE) approach was adopted, simulating individual profiles with asthma over patients’ lifetime in terms of exacerbations, asthma-related death, and death unrelated to asthma. The timing of these events is dependent on profile characteristics including lung function, asthma control, exacerbation history, and other baseline characteristics or contextual factors. Predictive equations were derived from a clinical trial to model time to exacerbation, change in asthma control, lung function, and utility. Real-world studies were used to supplement data gaps. Outcomes evaluated included life expectancy, quality-adjusted life-years (QALY), number of exacerbations, and lung function over time.

Results

Average annual rates of severe and moderate exacerbations were 1.82 and 3.08 respectively, with rates increasing over time. Lung function declined at a higher rate compared with the general population. Average life expectancy was 75.2 years, compared with 82.4 years in a matched general population. The majority of life-years were spent with uncontrolled asthma and impaired lung function.

Conclusion

Patients with moderate-to-severe type 2 asthma and a history of exacerbations suffer from frequent exacerbations and reduced lung function and life expectancy. Capturing multiple conditions to simulate long-term outcomes in patients with asthma may provide more realistic projections of exacerbation rates.
背景迄今为止,为模拟哮喘的长期预后而开发的模型因缺乏精细度和忽视疾病的异质性而饱受诟病。方法 采用离散综合条件事件(DICE)方法,模拟哮喘患者一生中哮喘加重、哮喘相关死亡和与哮喘无关死亡的个体特征。这些事件发生的时间取决于个人特征,包括肺功能、哮喘控制情况、哮喘加重史以及其他基线特征或背景因素。通过临床试验得出了预测方程,以模拟哮喘恶化的时间、哮喘控制的变化、肺功能和效用。真实世界研究用于补充数据缺口。评估的结果包括预期寿命、质量调整生命年(QALY)、哮喘加重次数和肺功能随时间的变化。与普通人群相比,肺功能的下降率更高。平均预期寿命为 75.2 岁,而相匹配的普通人群为 82.4 岁。结论患有中重度 2 型哮喘并有病情加重病史的患者病情会频繁加重,肺功能和预期寿命都会缩短。捕捉多种情况来模拟哮喘患者的长期预后,可以更真实地预测病情恶化率。
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引用次数: 0
A protocol for modifying progesterone to increase postpartum cigarette smoking abstinence and reduce secondhand smoke exposure in infants 调整黄体酮以提高产后戒烟率并减少婴儿二手烟暴露的方案
IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-06 DOI: 10.1016/j.conctc.2024.101389
Nermine Abdelwahab , Alicia Allen , Katherine Harrison , Ashley Petersen , Sharon Allen
New interventions are necessary to increase postpartum abstinence from cigarette smoking. Sex hormones, specifically progesterone, have been found to be protective against drug-taking behaviors. Our pilot double-blind, randomized, controlled trial, although underpowered, suggested a trend toward a higher prevalence of smoking abstinence among postpartum participants receiving exogenous progesterone compared to those receiving placebo. This paper outlines the protocol used in our study to evaluate the efficacy of modifying progesterone to increase postpartum smoking abstinence and, subsequently, decrease secondhand smoke exposure in infants. In the intervention arm, participants will receive open-label exogenous oral progesterone (200 mg twice daily). Using a concurrent control group that does not receive progesterone treatment, we hypothesize that progesterone treatment will increase postpartum smoking abstinence as measured using a 7-day point prevalence at six months post-treatment allocation, as well as reduce smoking-related risk factors. Secondary objectives include examining the impact of this maternal smoking intervention on infant health. In addition to describing the protocol, we also discuss the protocol changes made due to the COVID-19 pandemic. Upon completion, this study will provide new information on how sex hormones may influence smoking cessation in postpartum populations, which can have broad public health implications.

Clinical trials registration #

NCT04783857.
有必要采取新的干预措施来提高产后戒烟率。研究发现,性激素(尤其是孕酮)对吸毒行为具有保护作用。我们的试验性双盲随机对照试验虽然研究力量不足,但结果表明,与服用安慰剂的参与者相比,服用外源性黄体酮的产后参与者的戒烟率呈上升趋势。本文概述了我们的研究方案,该方案旨在评估调整孕酮以提高产后戒烟率并进而减少婴儿二手烟暴露的疗效。在干预组中,参与者将接受开放标签的外源性口服黄体酮(200 毫克,每天两次)。我们假设黄体酮治疗将提高产后戒烟率(以治疗后 6 个月的 7 天点吸烟率来衡量),并减少与吸烟相关的风险因素。次要目标包括研究这种产妇吸烟干预对婴儿健康的影响。除了介绍方案外,我们还讨论了因 COVID-19 大流行而对方案做出的修改。这项研究完成后,将为性激素如何影响产后人群的戒烟提供新的信息,从而对公共卫生产生广泛的影响。临床试验注册 #NCT04783857。
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引用次数: 0
An automated platform trial framework for A/B testing 用于 A/B 测试的自动化平台试验框架
IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-04 DOI: 10.1016/j.conctc.2024.101388
Wenru Zhou , Miranda Kroehl , Maxene Meier , Alexander Kaizer
This paper proposes a platform trial for conducting A/B tests with multiple arms and interim monitoring to investigate the impact of several factors on the expected sample size and probability of early stopping. We examined the performance of three stopping boundaries: O’Brien Fleming (OBF) stopping for either futility or difference (both), Pocock stopping for futility only, and fixed sample size design. We simulated twelve scenarios of different orders of arms based on various effect sizes, as well as considered 1 or 3 interim looks. The overall findings are summarizing in a flowchart to provide intuitive guidance for the design of the platform based on the simulation. We found that it is better to use OBF stopping for both if there is any effective variant and the trial is sufficiently powered to detect the expected effect size. If the study is underpowered to detect a difference, we recommend fixed sample size design to gather as much information as possible, however if the expected sample size is important to minimize, we recommend using Pocock boundaries with futility monitoring. Our results aimed at helping high-tech companies conduct their own studies without requiring extensive knowledge of clinical trial design and statistical methodology.
本文提出了一种进行多臂 A/B 试验和中期监测的平台试验,以研究多个因素对预期样本量和提前停止概率的影响。我们考察了三种停止界限的性能:奥布莱恩-弗莱明(O'Brien Fleming,OBF)停止无效或差异(两者),波科克(Pocock)停止无效,以及固定样本量设计。我们根据不同的效应大小模拟了 12 种不同数量级的手臂,并考虑了 1 或 3 次中期观察。我们以流程图的形式总结了总体研究结果,以便为基于模拟的平台设计提供直观指导。我们发现,如果存在任何有效的变体,且试验有足够的动力来检测预期效应大小,那么最好都使用 OBF 停止。如果研究动力不足,无法检测到差异,我们建议采用固定样本量设计,以收集尽可能多的信息;但如果预期样本量对最小化很重要,我们建议采用带有无效性监测的 Pocock 边界。我们的研究成果旨在帮助高科技公司开展自己的研究,而无需掌握丰富的临床试验设计和统计方法知识。
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引用次数: 0
Personalizing cognitive processing therapy with a case formulation approach to intentionally target impairment in psychosocial functioning associated with PTSD 采用病例制定法个性化认知处理疗法,有意识地针对与创伤后应激障碍相关的社会心理功能损伤进行治疗
IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-01 DOI: 10.1016/j.conctc.2024.101385
T.E. Galovski , L.B. McSweeney , R.D.V. Nixon , J.S. Wachen , B.N. Smith , S. Noorbaloochi , D. Vogt , B.L. Niles , S.M. Kehle-Forbes
Posttraumatic stress disorder (PTSD) is a debilitating condition often accompanied by significant functional impairments affecting quality of life and well-being. While Cognitive Processing Therapy (CPT) is a leading, evidence-based psychotherapy for PTSD, demonstrating substantial efficacy in core symptom reduction, its impact on psychosocial functioning is less well-established. The Personalizing Cognitive Processing Therapy with a Case Formulation Approach (Personalizing Approaches to Therapy: PATh) study aims to enhance CPT by explicitly targeting functional impairments and idiosyncratic challenges to optimal therapy outcomes (COTOs), comparing its efficacy against standard CPT in improving psychosocial functioning, quality of life, well-being, and core PTSD and depression symptoms. This randomized controlled trial involves 200 Veterans across eight Veterans Health Administration clinical sites, assigned to either Case Formulation CPT (CF-CPT) or standard CPT. Providers will deliver up to 20 sessions per patient, with assessments at baseline, mid-treatment, post-treatment, and three months follow-up. It is hypothesized that Veterans receiving CF-CPT will show greater improvements in functioning, quality of life, well-being, and symptom reduction, alongside higher treatment completion rates compared to standard CPT. Secondary outcomes will examine specific clinical challenges and their influence on treatment outcomes. This study investigates whether a personalized, flexible CPT protocol can enhance functional recovery in PTSD treatment without compromising the efficacy of the traditional approach, potentially impacting clinical practices and patient outcomes by promoting holistic recovery for veterans with PTSD.
创伤后应激障碍(PTSD)是一种使人衰弱的疾病,通常伴有严重的功能障碍,影响生活质量和幸福感。认知处理疗法(CPT)是治疗创伤后应激障碍的主要循证心理疗法,在减轻核心症状方面具有显著疗效,但其对社会心理功能的影响却没有得到充分证实。个性化认知处理疗法与病例制定方法(个性化治疗方法:PATh)研究旨在通过明确针对功能障碍和对最佳治疗结果(CTOs)的特殊挑战来加强认知处理疗法,比较其与标准认知处理疗法在改善心理社会功能、生活质量、幸福感以及创伤后应激障碍和抑郁症核心症状方面的疗效。这项随机对照试验涉及退伍军人健康管理局 8 个临床站点的 200 名退伍军人,他们将被分配接受病例制定 CPT(CF-CPT)或标准 CPT。医护人员将为每位患者提供多达 20 次治疗,并在基线、治疗中期、治疗后和三个月的随访中进行评估。根据假设,与标准 CPT 相比,接受 CF-CPT 治疗的退伍军人在功能、生活质量、幸福感和症状减轻方面会有更大的改善,同时治疗完成率也会更高。次要结果将检查具体的临床挑战及其对治疗结果的影响。本研究探讨了个性化、灵活的 CPT 方案能否在不影响传统方法疗效的前提下,提高创伤后应激障碍治疗的功能恢复,从而通过促进创伤后应激障碍退伍军人的整体康复,对临床实践和患者疗效产生潜在影响。
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Contemporary Clinical Trials Communications
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