Clinical Trials最新文献

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Pragmatic monitoring of emerging efficacy data in randomized controlled trials. 对随机对照试验中新出现的疗效数据进行务实监测。
IF 2.2 3区 医学
Clinical Trials Pub Date : 2025-04-01 Epub Date: 2024-11-09 DOI: 10.1177/17407745241290729
Shrikant I Bangdiwala, Salim Yusuf
{"title":"Pragmatic monitoring of emerging efficacy data in randomized controlled trials.","authors":"Shrikant I Bangdiwala, Salim Yusuf","doi":"10.1177/17407745241290729","DOIUrl":"10.1177/17407745241290729","url":null,"abstract":"<p><p>Monitoring the conduct of phase III randomized controlled trials is driven by ethical reasons to protect the study integrity and the safety of trial participants. We propose a group sequential, pragmatic approach for monitoring the accumulating efficacy information in randomized controlled trials. The \"Population Health Research Institute boundary\" is simple to implement and sensible, as it considers the reduction in uncertainty with increasing information as the study progresses. It is also pragmatic, since it takes into consideration the typical monitoring behavior of monitoring committees of large multicenter trials and is relatively easily implemented. It not only controls the overall Lan-DeMets type I error probability (alpha) spent, but performs better than other group sequential boundaries for the total nominal study alpha. We illustrate the use of our monitoring approach in the early termination of two past completed trials.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"155-160"},"PeriodicalIF":2.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11986074/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142616308","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Ethical considerations for sharing aggregate results from pragmatic clinical trials. 分享实用临床试验综合结果的伦理考虑。
IF 2.2 3区 医学
Clinical Trials Pub Date : 2025-04-01 Epub Date: 2024-11-25 DOI: 10.1177/17407745241290782
Stephanie R Morain, Abigail Brickler, Joseph Ali, Patricia Pearl O'Rourke, Kayte Spector-Bagdady, Benjamin Wilfond, Vasiliki Rahimzadeh, Caleigh Propes, Kayla Mehl, David Wendler
{"title":"Ethical considerations for sharing aggregate results from pragmatic clinical trials.","authors":"Stephanie R Morain, Abigail Brickler, Joseph Ali, Patricia Pearl O'Rourke, Kayte Spector-Bagdady, Benjamin Wilfond, Vasiliki Rahimzadeh, Caleigh Propes, Kayla Mehl, David Wendler","doi":"10.1177/17407745241290782","DOIUrl":"10.1177/17407745241290782","url":null,"abstract":"<p><p>A growing literature has explored the ethical obligations and current practices related to sharing aggregate results with research participants. However, no prior work has examined these issues in the context of pragmatic clinical trials. Several characteristics of pragmatic clinical trials may complicate both the ethics and the logistics of sharing aggregate results. Among these characteristics include that pragmatic clinical trials may affect the rights, welfare, and interests of not only patient-subjects but also clinicians, meaning that results may be owed to a broader range of groups than typically considered in other research contexts. In addition, some pragmatic clinical trials are conducted under a waiver of informed consent, meaning sharing results may alert participants that they were enrolled without their consent. This article explores the ethical dimensions that can inform decision-making about sharing aggregate results from pragmatic clinical trials, and provides recommendations for that sharing. A central insight is that healthcare institutions-as key partners for the conduct of pragmatic clinical trials-must also be key partners in decision-making about sharing aggregate pragmatic clinical trial results. We conclude with insights for future research.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"248-254"},"PeriodicalIF":2.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11991889/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142715141","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Detecting irregularities in randomized controlled trials using machine learning. 利用机器学习检测随机对照试验中的违规行为。
IF 2.2 3区 医学
Clinical Trials Pub Date : 2025-04-01 Epub Date: 2024-11-25 DOI: 10.1177/17407745241297947
Walter Nelson, Jeremy Petch, Jonathan Ranisau, Robin Zhao, Kumar Balasubramanian, Shrikant I Bangdiwala
{"title":"Detecting irregularities in randomized controlled trials using machine learning.","authors":"Walter Nelson, Jeremy Petch, Jonathan Ranisau, Robin Zhao, Kumar Balasubramanian, Shrikant I Bangdiwala","doi":"10.1177/17407745241297947","DOIUrl":"10.1177/17407745241297947","url":null,"abstract":"<p><strong>Background: </strong>Over the course of a clinical trial, irregularities may arise in the data. Trialists implement human-intensive, expensive central statistical monitoring procedures to identify and correct these irregularities before the results of the trial are analyzed and disseminated. Machine learning algorithms have shown promise for identifying center-level irregularities in multi-center clinical trials with minimal human intervention. We aimed to characterize the form-level data irregularities in several historical clinical trials and evaluate the ability of a machine learning-based outlier detection algorithm to identify them.</p><p><strong>Methods: </strong>Data irregularities previously identified by humans in historical clinical trials were ascertained by comparing preliminary snapshots of the trial databases to the final, locked databases. We measured the ability of a machine learning based outlier detection algorithm to identify form-level irregularities using concordance (area under the receiver operator characteristic), positive predictive value (precision), and sensitivity (recall).</p><p><strong>Results: </strong>We examined preliminary snapshots of seven historical clinical trials which randomized a total of 77,001 participants. We extracted a total of 1,267,484 completed entries from 358 case report forms containing irregularities from all snapshots across all trials, containing a total of 24,850 form-wide irregularities (median per-form form-level irregularity rate: 1.81%). Our proposed machine learning algorithm detects form-level irregularities with a median concordance of 0.74 (interquartile range = 0.57-0.89), slightly exceeding the performance of a previously proposed machine learning approach with a median area under the receiver operator characteristic of 0.73 (interquartile range = 0.54-0.88).</p><p><strong>Conclusion: </strong>Data irregularities in historical clinical trials were ascertained by comparing preliminary snapshots of the trial database to the final database. These irregularities can be categorized according to their scope. Irregularities can be successfully detected by a machine learning algorithm as early or earlier than a human can, without human intervention. Such an approach may complement existing techniques for central statistical monitoring in large multi-center randomized controlled trials and possibly improve the efficiency of costly data verification processes.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"178-187"},"PeriodicalIF":2.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142715120","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
UK paediatric clinical trial protocols: A review of guidance for participant management and care in the event of premature termination. 英国儿科临床试验协议:对提前终止试验时的参与者管理和护理指南的审查。
IF 2.2 3区 医学
Clinical Trials Pub Date : 2025-04-01 Epub Date: 2024-11-19 DOI: 10.1177/17407745241296864
Helen Pluess-Hall, Paula Smith, Julie Menzies
{"title":"UK paediatric clinical trial protocols: A review of guidance for participant management and care in the event of premature termination.","authors":"Helen Pluess-Hall, Paula Smith, Julie Menzies","doi":"10.1177/17407745241296864","DOIUrl":"10.1177/17407745241296864","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background/aims: &lt;/strong&gt;Clinical trials provide an opportunity to identify new treatments and can offer patients access to treatments otherwise unavailable. However, approximately 10% of paediatric clinical trials discontinue before the trial has completed. If this premature termination is because the trial treatment(s) being investigated are identified to be ineffective or unsafe, it results in the abrupt discontinuation of the investigational medicinal product for participants. For some participants, there may not be other treatment options to pursue at the trial-end. Trials prematurely terminating can be a distressing experience for all involved and currently there is little published evidence about the guidance provided to healthcare professionals in the event of premature trial termination. The study protocol is the source of guidance for healthcare professionals delivering clinical research, detailing how to conduct all aspects of the trial. The aim was to quantify the proportion of clinical trial protocols that included premature trial termination and subsequently those that provided instructions related to participant management and care. In addition, to analyse the context in which premature termination was included and the detail of any instructions for participant management and care.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;The ClinicalTrials.gov database was searched by a single reviewer for UK interventional drug trials enrolling children with an available study protocol. Protocols were searched to assess if the risk of premature trial termination was identified, the context for premature termination being included, if information was provided to support the management and care of participants should this situation occur and the detail of those instructions. Data were summarised descriptively.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Of 245 clinical trial protocols, 235 (95.9%) identified the possibility of premature trial termination, the majority within the context of the sponsor asserting their right to terminate the trial (82.7%, 115/235) and providing reasons why the trial could be stopped (65.5%, 91/235). Forty-two percent (98/235) provided guidance for participant management and care, most commonly to contact/inform the participant (45.9%, 45/98). Directions varied in the quantity and level of detail.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;This review of UK clinical trial protocol highlights that information surrounding premature termination is lacking, with only 42% providing guidance on the care of trial participants. While this ensures regulatory compliance, it fails to consider the challenge for healthcare professionals in managing participants on-going care or the duty of care owed to participants. Further research is required to understand if additional documents are being used in practice, and if these meet the needs of healthcare professionals in supporting research participants and families during premature trial termination","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"220-226"},"PeriodicalIF":2.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11986081/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142667166","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Statistical properties of items and summary scores from the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE®) in a diverse cancer sample. 不同癌症样本中患者报告结果版《不良事件通用术语标准》(PRO-CTCAE®)项目和总分的统计特性。
IF 2.2 3区 医学
Clinical Trials Pub Date : 2025-04-01 Epub Date: 2024-10-23 DOI: 10.1177/17407745241286065
Carolyn Mead-Harvey, Ethan Basch, Lauren J Rogak, Blake T Langlais, Gita Thanarajasingam, Brenda F Ginos, Minji K Lee, Claire Yee, Sandra A Mitchell, Lori M Minasian, Tito R Mendoza, Antonia V Bennett, Deborah Schrag, Amylou C Dueck, Gina L Mazza
{"title":"Statistical properties of items and summary scores from the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE<sup>®</sup>) in a diverse cancer sample.","authors":"Carolyn Mead-Harvey, Ethan Basch, Lauren J Rogak, Blake T Langlais, Gita Thanarajasingam, Brenda F Ginos, Minji K Lee, Claire Yee, Sandra A Mitchell, Lori M Minasian, Tito R Mendoza, Antonia V Bennett, Deborah Schrag, Amylou C Dueck, Gina L Mazza","doi":"10.1177/17407745241286065","DOIUrl":"10.1177/17407745241286065","url":null,"abstract":"<p><strong>Background/aims: </strong>The Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE<sup>®</sup>) was developed to capture symptomatic adverse events from the patient perspective. We aim to describe statistical properties of PRO-CTCAE items and summary scores and to provide evidence for recommendations regarding PRO-CTCAE administration and reporting.</p><p><strong>Methods: </strong>Using data from the PRO-CTCAE validation study (NCT02158637), prevalence, means, and standard deviations of PRO-CTCAE items, composite scores, and mean and maximum scores across attributes (frequency, severity, and/or interference) of symptomatic adverse events were calculated. For each adverse event, correlations and agreement between attributes, correlations between attributes and composite scores, and correlations between composite, mean, and maximum scores were estimated.</p><p><strong>Results: </strong>PRO-CTCAE items were completed by 899 patients with various cancer types. Most patients reported experiencing one or more adverse events, with the most prevalent being fatigue (87.7%), sad/unhappy feelings (66.0%), anxiety (63.6%), pain (63.2%), insomnia (61.8%), and dry mouth (60.0%). Attributes were moderately to strongly correlated within an adverse event (<i>r</i> = 0.53 to 0.77, all <i>p</i> < 0.001) but not fully concordant (κ<sub>weighted</sub> = 0.26 to 0.60, all <i>p</i> < 0.001), with interference demonstrating lowest mean scores and prevalence among attributes of the same adverse event. Attributes were moderately to strongly correlated with composite scores (<i>r</i> = 0.67 to 0.97, all <i>p</i> < 0.001). Composite scores were moderately to strongly correlated with mean and maximum scores for the same adverse event (<i>r</i> = 0.69 to 0.94, all <i>p</i> < 0.001). Correlations between composite scores of different adverse events varied widely (<i>r</i> = 0.04 to 0.68) but were moderate to strong for conceptually related adverse events.</p><p><strong>Conclusions: </strong>Results provide evidence for PRO-CTCAE administration and reporting recommendations that the full complement of attributes be administered for each adverse event, and that attributes as well as summary scores be reported.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"161-169"},"PeriodicalIF":2.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11986086/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142496568","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Response to Cleland and Anzar. 对克利兰和安扎尔的回应。
IF 2.2 3区 医学
Clinical Trials Pub Date : 2025-03-31 DOI: 10.1177/17407745251324843
Janet Wittes, David L DeMets, KyungMann Kim, Dennis G Maki, Marc A Pfeffer, J Michael Gaziano, Panagiota Kitsantas, Charles H Hennekens, Sarah K Wood
{"title":"Response to Cleland and Anzar.","authors":"Janet Wittes, David L DeMets, KyungMann Kim, Dennis G Maki, Marc A Pfeffer, J Michael Gaziano, Panagiota Kitsantas, Charles H Hennekens, Sarah K Wood","doi":"10.1177/17407745251324843","DOIUrl":"https://doi.org/10.1177/17407745251324843","url":null,"abstract":"","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"17407745251324843"},"PeriodicalIF":2.2,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751501","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Quality management of a multi-center randomized controlled feeding trial: A prospective observational study. 多中心随机对照喂养试验的质量管理:一项前瞻性观察研究。
IF 2.2 3区 医学
Clinical Trials Pub Date : 2025-03-30 DOI: 10.1177/17407745251324653
Xiayan Chen, Huijuan Li, Lin Feng, Xi Lan, Shuyi Li, Yanfang Zhao, Guo Zeng, Huilian Zhu, Jianqin Sun, Yanfang Wang, Yangfeng Wu
{"title":"Quality management of a multi-center randomized controlled feeding trial: A prospective observational study.","authors":"Xiayan Chen, Huijuan Li, Lin Feng, Xi Lan, Shuyi Li, Yanfang Zhao, Guo Zeng, Huilian Zhu, Jianqin Sun, Yanfang Wang, Yangfeng Wu","doi":"10.1177/17407745251324653","DOIUrl":"https://doi.org/10.1177/17407745251324653","url":null,"abstract":"<p><p>BackgroundNutrition and dietary trials are often prone to bias, leading to inaccurate or questionable estimates of intervention efficacy. However, reports on quality management practices of well-controlled dietary trials are scarce. This study aims to introduce the quality management system of the Diet, ExerCIse and CarDiovascular hEalth-Diet Study and report its performance in ensuring study quality.MethodsThe quality management system consisted of a study coordinating center, trial governance, and quality control measures covering study design, conduct, and data analysis and reporting. Metrics for evaluating the performance of the system were collected throughout the whole trial development and conducted from September 2016 to June 2021, covering major activities at the coordinating center, study sites, and central laboratories, with a focus on the protocol amendments, protocol deviations (eligibility, fidelity, confounders management, loss to follow-up and outside-of-window visits, and blindness success), and measurement accuracy.ResultsThree amendments to the study protocol enhanced feasibility. All participants (265) met the eligibility criteria. Among them, only 3% were lost to the primary outcome follow-up measurement. More than 95% of participants completed the study, they consumed more than 96% of the study meals, and more than 94% of participants consumed more than 18 meals per week, with no between-group differences. Online monitoring of nutrient targets for the intervention diet showed that all targets were achieved except for the fiber intake, which was 4.3 g less on average. Only 3% experienced a body weight change greater than 2.0 kg, and 3% had medication changes which were not allowed by the study. James' blinding index at the end of the study was 0.68. The end digits of both systolic and diastolic blood pressure readings were distributed equally. For laboratory measures, 100% of standard samples, 97% of blood-split samples, and 87% of urine-split samples had test results within the acceptable range. Only 1.4% of data items required queries, for which only 30% needed corrections.DiscussionThe Diet, ExerCIse and CarDiovascular hEalth-Diet Study quality management system provides a framework for conducting a high-quality dietary intervention clinical trial.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"17407745251324653"},"PeriodicalIF":2.2,"publicationDate":"2025-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751500","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Building a professionally recognised clinical trial workforce: Is it time for an education and accreditation strategy? 建立一支专业认可的临床试验队伍:是时候实施教育和认证战略了吗?
IF 2.2 3区 医学
Clinical Trials Pub Date : 2025-03-27 DOI: 10.1177/17407745251328287
Simone Spark, Prudence Perry, Thobekile Mthethwa-Pitt, Dragan Ilic, Anne Woollett, Sophia Zoungas, Marina Skiba
{"title":"Building a professionally recognised clinical trial workforce: Is it time for an education and accreditation strategy?","authors":"Simone Spark, Prudence Perry, Thobekile Mthethwa-Pitt, Dragan Ilic, Anne Woollett, Sophia Zoungas, Marina Skiba","doi":"10.1177/17407745251328287","DOIUrl":"https://doi.org/10.1177/17407745251328287","url":null,"abstract":"<p><p>Evidence-based medicine relies heavily on well-conducted clinical trials. Australia lacks a discipline-specific education pathway to provide the specialist skills necessary to conduct clinical trials to the highest standards. Unlike allied health professionals, clinical trialists who currently possess the specialist skills to conduct clinical trials do not receive professional recognition. The National Health and Medical Research Council defines 'clinical trialist' to include site staff as well as investigators. In this perspective piece, we explore the importance of discipline-specific education in creating a job-ready workforce of clinical trialists; the need for recognition of clinical trialists as an allied health profession in concert with their existing medical, nursing and other professional qualifications and outline a proposed specialist education and accreditation strategy.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"17407745251328287"},"PeriodicalIF":2.2,"publicationDate":"2025-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143718174","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Design considerations for randomized comparisons of neoadjuvant-adjuvant versus adjuvant-only cancer immunotherapy when tumor measurement schedules do not align (SWOG S1801). 当肿瘤测量计划不一致时,新辅助与仅辅助的癌症免疫治疗随机比较的设计考虑(SWOG S1801)。
IF 2.2 3区 医学
Clinical Trials Pub Date : 2025-03-18 DOI: 10.1177/17407745251321371
Megan Othus, Elad Sharon, Michael C Wu, Vernon K Sondak, Antoni Ribas, Sapna P Patel
{"title":"Design considerations for randomized comparisons of neoadjuvant-adjuvant versus adjuvant-only cancer immunotherapy when tumor measurement schedules do not align (SWOG S1801).","authors":"Megan Othus, Elad Sharon, Michael C Wu, Vernon K Sondak, Antoni Ribas, Sapna P Patel","doi":"10.1177/17407745251321371","DOIUrl":"https://doi.org/10.1177/17407745251321371","url":null,"abstract":"<p><p>BackgroundIn 2022, SWOG S1801 was the first trial to demonstrate that single-agent anti-PD-1 checkpoint inhibition used as neoadjuvant-adjuvant therapy leads to significantly improved outcomes compared to adjuvant-only therapy. Endpoints in trials comparing neoadjuvant-adjuvant to adjuvant strategies need special consideration to ensure that event measurement timing is appropriately accounted for in analyses to avoid biased comparisons artificially favoring one arm over another.MethodsThe S1801 trial is used a case study to evaluate the issues involved in selecting endpoints for trials comparing neoadjuvant-adjuvant versus adjuvant-only strategies.ResultsDefinitions and timing of measurement of events is provided. Trial scenarios when recurrence-free versus event-free survival should be used are provided.ConclusionsIn randomized trials comparing neoadjuvant-adjuvant to adjuvant-only strategies, event-free survival endpoints measured from randomization are required for unbiased comparison of the arms. The time at which events can be measured on each arm needs to be carefully considered. If measurement of events occurs at different times on the randomized arms, modified definitions of event-free survival must be used to avoid bias.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"17407745251321371"},"PeriodicalIF":2.2,"publicationDate":"2025-03-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143656289","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Evaluating the use of text-message reminders and personalised text-message reminders on the return of participant questionnaires in trials, a systematic review and meta-analysis. 评估短信提醒和个性化短信提醒在试验参与者问卷返回中的使用情况,系统回顾和荟萃分析。
IF 2.2 3区 医学
Clinical Trials Pub Date : 2025-03-12 DOI: 10.1177/17407745251320888
Laura Doherty, Catherine Arundel, Elizabeth Coleman, Ailish Byrne, Katherine Jones
{"title":"Evaluating the use of text-message reminders and personalised text-message reminders on the return of participant questionnaires in trials, a systematic review and meta-analysis.","authors":"Laura Doherty, Catherine Arundel, Elizabeth Coleman, Ailish Byrne, Katherine Jones","doi":"10.1177/17407745251320888","DOIUrl":"https://doi.org/10.1177/17407745251320888","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Randomised controlled trials are widely accepted as the gold standard research methodology for the evaluation of interventions. However, they often display poor participant retention. To prevent this, various participant interventions have been identified and evaluated through the use of studies within a trial. Two such interventions are participant short message service reminders (also known as text-messages) and personalised participant short message service reminders, designed to encourage a participant to return a study questionnaire. While previous studies within a trial have evaluated the effectiveness of these two retention strategies, trialists continue to spend both time and money on these strategies while the evidence remains inconclusive.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This systematic review and meta-analysis compared the use of short message service reminders with no short message service reminder and personalised short message service reminders with non-personalised short message service reminders, on participant retention. Eligible studies were identified through advanced searches of electronic databases (MEDLINE, EMBASE and Cochrane Library) and hand-searching of alternative information sources. The review primary outcome was the proportion of study questionnaires returned for the individual study within a trial primary analysis time points.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Nine eligible studies within a trial were identified, of which four compared short message service versus no short message service and five compared personalised short message service versus non-personalised short message service. For those that compared personalised short message service versus non-personalised short message service, only three were deemed appropriate for meta-analysis. The primary outcome results for short message service versus no short message service concluded that short message service led to a statistically non-significant increase in the odds of study questionnaire return by 9% (odds ratio = 1.09, 95% confidence interval = 0.92 to 1.30). Similarly, comparison of personalised short message service versus non-personalised short message service concluded that personalised short message service caused a statistically non-significant increase in odds by 22% (odds ratio = 1.22, 95% confidence interval = 0.95 to 1.59).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;The effectiveness of both short message service and personalised short message service as retention tools remains inconclusive and further study within a trial evaluations are required. However, as short message services are low in cost, easy to use and generally well accepted by participants, it is suggested that trialists adopt a pragmatic approach and utilise these reminders until further research is conducted. Given both the minimal addition in cost for studies already utilising short message service reminders and some evidence of effect, personalisation shoul","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"17407745251320888"},"PeriodicalIF":2.2,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143604142","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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