Pub Date : 2024-11-19DOI: 10.1016/j.cct.2024.107750
Jitendra Ganju , Ron Xiaolong Yu
According to ICH E9(R1), defining the estimand comes before defining the analysis approach, and the strategies for addressing intercurrent events are key components of the estimand. With the composite strategy, the problem of missing data disappears, because it becomes part of the endpoint definition. It is this perspective that we adopt in addressing the problem of missing data. We propose comparing patients in a pairwise manner to determine which patient fared better, one patient from each group, taking into account the reason for and timing of missingness. For purposes of illustration, reasons for missingness are placed into three categories: (1) death or adverse events, (2) administration of rescue medication (treated as missing even if patient continues in the study, or a poor score is assigned), and (3) other reasons such as loss to follow-up or withdrawal of consent. Each pair of patients is compared on the endpoint of interest. The comparison outcomes are determined based on the presence of missing data, its category, and timing. For instance, if both patients in a pair have received rescue medication, the patient with the later time of rescue medication is considered to have fared better. The overall treatment effect is estimated from combining results across all pairwise comparisons. This method allows the reason and timing of missing data to contribute to the assessment of treatment effects, providing a solution to some limitations of existing methods. Thus, the pairwise comparison approach addresses the missing data problem transparently via the composite strategy.
根据 ICH E9(R1)，在确定分析方法之前，首先要确定估计指标，而处理并发症的策略是估计指标的关键组成部分。有了复合策略，缺失数据的问题就不复存在，因为它已成为终点定义的一部分。我们正是从这个角度来解决数据缺失问题的。我们建议对患者进行配对比较，以确定哪位患者的情况更好，每组一名患者，同时考虑缺失的原因和时间。为了便于说明，我们将缺失原因分为三类：(1) 死亡或不良事件；(2) 施用抢救药物（即使患者继续接受研究，或评分较差，也视为缺失）；(3) 其他原因，如失去随访或撤回同意。对每对患者进行相关终点的比较。比较结果根据是否存在缺失数据、缺失数据的类别和时间来确定。例如，如果一对患者中的两名患者都接受了抢救药物治疗，则认为接受抢救药物治疗时间较晚的患者情况较好。所有配对比较的总体治疗效果合并计算。这种方法允许缺失数据的原因和时间有助于评估治疗效果，解决了现有方法的一些局限性。因此，配对比较法通过综合策略透明地解决了数据缺失问题。
{"title":"A new way to address missing data in late-stage clinical trials","authors":"Jitendra Ganju , Ron Xiaolong Yu","doi":"10.1016/j.cct.2024.107750","DOIUrl":"10.1016/j.cct.2024.107750","url":null,"abstract":"<div><div>According to ICH E9(R1), defining the estimand comes before defining the analysis approach, and the strategies for addressing intercurrent events are key components of the estimand. With the composite strategy, the problem of missing data disappears, because it becomes part of the endpoint definition. It is this perspective that we adopt in addressing the problem of missing data. We propose comparing patients in a pairwise manner to determine which patient fared better, one patient from each group, taking into account the reason for and timing of missingness. For purposes of illustration, reasons for missingness are placed into three categories: (1) death or adverse events, (2) administration of rescue medication (treated as missing even if patient continues in the study, or a poor score is assigned), and (3) other reasons such as loss to follow-up or withdrawal of consent. Each pair of patients is compared on the endpoint of interest. The comparison outcomes are determined based on the presence of missing data, its category, and timing. For instance, if both patients in a pair have received rescue medication, the patient with the later time of rescue medication is considered to have fared better. The overall treatment effect is estimated from combining results across all pairwise comparisons. This method allows the reason and timing of missing data to contribute to the assessment of treatment effects, providing a solution to some limitations of existing methods. Thus, the pairwise comparison approach addresses the missing data problem transparently via the composite strategy.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107750"},"PeriodicalIF":2.0,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142681248","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}
Pub Date : 2024-11-19DOI: 10.1016/j.cct.2024.107746
Emma P. Keane , Michelle Guo , Lisa M. Gudenkauf , Annabella C. Boardman , M. Tim Song , Emma D. Wolfe , Isabella S. Larizza , Manfred N. Mate-Kole , Brian C. Healy , Jeff C. Huffman , Areej El-Jawahri , Hermioni L. Amonoo
Background
Despite the association between peer support interventions and improved patient-reported outcomes (PROs) across cancer populations, there is a lack of structured peer support interventions for patients with hematologic malignancies undergoing hematopoietic stem cell transplantation (HSCT).
Objective
To test the feasibility, acceptability, and preliminary efficacy of a novel five-session, phone-delivered peer support intervention (STEPP: Supporting Transplant Experiences with Peer Program) for improving quality of life (QOL) and reducing psychological distress in patients undergoing HSCT.
Methods
This single-center pilot randomized clinical trial (RCT) will compare the STEPP intervention to usual transplant care among 90 patients hospitalized for HSCT. Eligible participants include adults (age ≥ 18 years) with hematologic malignancies hospitalized for autologous or allogeneic HSCT. The STEPP intervention provides informational, emotional, and practical support. To test the primary aim of intervention feasibility, we have established a priori benchmarks of 60 % enrollment of eligible patients and 60 % completion of at least 3 out of 5 intervention sessions among those randomized to STEPP. Acceptability will be assessed using the Client Satisfaction Questionnaire, with scores ≥3.0/4.0 indicating greater intervention acceptability. To test the secondary aim of preliminary efficacy, we will examine changes in PROs (e.g., anxiety symptoms and QOL) from pre- to post-intervention.
Discussion
This pilot RCT of a structured, phone-delivered peer support intervention tailored to the needs of patients preparing to undergo HSCT will elucidate the feasibility, acceptability, and preliminary efficacy of the STEPP intervention. We will then be poised to conduct a future, full-scale RCT to establish the efficacy of STEPP on patient outcomes.
{"title":"A peer support intervention in patients with hematologic malignancies undergoing hematopoietic stem cell transplantation (HSCT): The STEPP randomized pilot trial design and methods","authors":"Emma P. Keane , Michelle Guo , Lisa M. Gudenkauf , Annabella C. Boardman , M. Tim Song , Emma D. Wolfe , Isabella S. Larizza , Manfred N. Mate-Kole , Brian C. Healy , Jeff C. Huffman , Areej El-Jawahri , Hermioni L. Amonoo","doi":"10.1016/j.cct.2024.107746","DOIUrl":"10.1016/j.cct.2024.107746","url":null,"abstract":"<div><h3>Background</h3><div>Despite the association between peer support interventions and improved patient-reported outcomes (PROs) across cancer populations, there is a lack of structured peer support interventions for patients with hematologic malignancies undergoing hematopoietic stem cell transplantation (HSCT).</div></div><div><h3>Objective</h3><div>To test the feasibility, acceptability, and preliminary efficacy of a novel five-session, phone-delivered peer support intervention (STEPP: Supporting Transplant Experiences with Peer Program) for improving quality of life (QOL) and reducing psychological distress in patients undergoing HSCT.</div></div><div><h3>Methods</h3><div>This single-center pilot randomized clinical trial (RCT) will compare the STEPP intervention to usual transplant care among 90 patients hospitalized for HSCT. Eligible participants include adults (age ≥ 18 years) with hematologic malignancies hospitalized for autologous or allogeneic HSCT. The STEPP intervention provides informational, emotional, and practical support. To test the primary aim of intervention feasibility, we have established a priori benchmarks of 60 % enrollment of eligible patients and 60 % completion of at least 3 out of 5 intervention sessions among those randomized to STEPP. Acceptability will be assessed using the Client Satisfaction Questionnaire, with scores ≥3.0/4.0 indicating greater intervention acceptability. To test the secondary aim of preliminary efficacy, we will examine changes in PROs (e.g., anxiety symptoms and QOL) from pre- to post-intervention.</div></div><div><h3>Discussion</h3><div>This pilot RCT of a structured, phone-delivered peer support intervention tailored to the needs of patients preparing to undergo HSCT will elucidate the feasibility, acceptability, and preliminary efficacy of the STEPP intervention. We will then be poised to conduct a future, full-scale RCT to establish the efficacy of STEPP on patient outcomes.</div><div><span><span>ClinicalTrials.gov</span><svg><path></path></svg></span>: NCT 06010017.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107746"},"PeriodicalIF":2.0,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142681255","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}
Pub Date : 2024-11-19DOI: 10.1016/j.cct.2024.107748
Rebecca North , Andy J. Liu , Carl Pieper , Susanne Danus , Connie R. Thacker , Marissa Ashner , Cathleen Colón-Emeric , Richard H. Lee
Older adults with Alzheimer's disease (AD), in addition to significant cognitive disability, have twice the risk of fracture compared to those with normal cognition. Fractures among older adults with AD are associated with substantial morbidity, loss of physical function, and significant mortality. Prior studies have shown a decreased risk of fracture among those taking acetylcholinesterase inhibitors, such as donepezil. With both cognitive and non-cognitive benefits, donepezil would be a valuable component in a fracture prevention program for older adults with AD. Though anti-amyloid therapies are now clinically available, donepezil may still have non-cognitive benefits. However, the specific effects of donepezil on bone metabolism are unknown. We have designed this randomized, double-blind, placebo-controlled clinical trial to investigate the effect of AD treatment with donepezil on bone metabolism. The study will measure the change in bone mineral density, bone turnover markers, and bone quality related to 12-months of donepezil therapy. This will be the first known study of changes in bone metabolism among older adults with AD.
{"title":"Effect of donepezil on bone metabolism among older adults with Alzheimer's disease","authors":"Rebecca North , Andy J. Liu , Carl Pieper , Susanne Danus , Connie R. Thacker , Marissa Ashner , Cathleen Colón-Emeric , Richard H. Lee","doi":"10.1016/j.cct.2024.107748","DOIUrl":"10.1016/j.cct.2024.107748","url":null,"abstract":"<div><div>Older adults with Alzheimer's disease (AD), in addition to significant cognitive disability, have twice the risk of fracture compared to those with normal cognition. Fractures among older adults with AD are associated with substantial morbidity, loss of physical function, and significant mortality. Prior studies have shown a decreased risk of fracture among those taking acetylcholinesterase inhibitors, such as donepezil. With both cognitive and non-cognitive benefits, donepezil would be a valuable component in a fracture prevention program for older adults with AD. Though anti-amyloid therapies are now clinically available, donepezil may still have non-cognitive benefits. However, the specific effects of donepezil on bone metabolism are unknown. We have designed this randomized, double-blind, placebo-controlled clinical trial to investigate the effect of AD treatment with donepezil on bone metabolism. The study will measure the change in bone mineral density, bone turnover markers, and bone quality related to 12-months of donepezil therapy. This will be the first known study of changes in bone metabolism among older adults with AD.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107748"},"PeriodicalIF":2.0,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142681258","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}
Pub Date : 2024-11-19DOI: 10.1016/j.cct.2024.107756
Stephanie Miles , Andrea Phillipou , Erica Neill , Amanda Newbigin , Hannah W. Kim , Kamryn T. Eddy , Jennifer J. Thomas
Avoidant/restrictive food intake disorder (ARFID) is a feeding and eating disorder for which there are very few evidence-based treatments. Cognitive-Behavioral Therapy for Avoidant/Restrictive Food Intake Disorder (CBT-AR) is a novel exposure-based treatment which is suitable for people aged ten and older. The primary aims of the study are to undertake a real-world evaluation of the feasibility, acceptability, and preliminary effectiveness of CBT-AR for young people aged 12–25 years old in an outpatient eating disorders service where the patient population has high levels of psychiatric comorbidity. Clinicians, patients, and parents/guardians will be involved in the evaluation. Assessments will be carried out at baseline, during weekly sessions, at the end of each treatment stage, at end of CBT-AR treatment, and at 3-month follow-up. Assessments will measure ARFID symptoms, mood, quality of life, therapeutic alliance, and feedback on the treatment. The study will take place over a 12-month period and will evaluate the use of CBT-AR within real-life clinical practice conditions, noting how and why deviations from the treatment have occurred. The findings of this research will inform future ARFID treatment delivery and the implementation of CBT-AR at outpatient mental health services.
{"title":"An evaluation of Cognitive-Behavioral Therapy for Avoidant/Restrictive Food Intake Disorder (CBT-AR) in a youth outpatient eating disorders service: A protocol paper","authors":"Stephanie Miles , Andrea Phillipou , Erica Neill , Amanda Newbigin , Hannah W. Kim , Kamryn T. Eddy , Jennifer J. Thomas","doi":"10.1016/j.cct.2024.107756","DOIUrl":"10.1016/j.cct.2024.107756","url":null,"abstract":"<div><div>Avoidant/restrictive food intake disorder (ARFID) is a feeding and eating disorder for which there are very few evidence-based treatments. Cognitive-Behavioral Therapy for Avoidant/Restrictive Food Intake Disorder (CBT-AR) is a novel exposure-based treatment which is suitable for people aged ten and older. The primary aims of the study are to undertake a real-world evaluation of the feasibility, acceptability, and preliminary effectiveness of CBT-AR for young people aged 12–25 years old in an outpatient eating disorders service where the patient population has high levels of psychiatric comorbidity. Clinicians, patients, and parents/guardians will be involved in the evaluation. Assessments will be carried out at baseline, during weekly sessions, at the end of each treatment stage, at end of CBT-AR treatment, and at 3-month follow-up. Assessments will measure ARFID symptoms, mood, quality of life, therapeutic alliance, and feedback on the treatment. The study will take place over a 12-month period and will evaluate the use of CBT-AR within real-life clinical practice conditions, noting how and why deviations from the treatment have occurred. The findings of this research will inform future ARFID treatment delivery and the implementation of CBT-AR at outpatient mental health services.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107756"},"PeriodicalIF":2.0,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142681256","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}
Pub Date : 2024-11-19DOI: 10.1016/j.cct.2024.107747
R. Gil-Gutiérrez , I. Medina-Martínez , C. Ballesteros-Rubio , F.J. De La Hera-Fernández , R. Ríos-Fernández , J.L. Callejas-Rubio , M. Zamora-Pasadas , I. Cantarero-Villanueva , M. Correa-Rodríguez , N. Ortego-Centeno , B. Rueda-Medina
Background
Health-related lifestyle management could improve related symptoms and adverse events in patients with systemic lupus erythematosus (SLE). The phenolic compounds in extra virgin olive oil (EVOO) and physical exercise (PE) have both shown benefits for autoimmune conditions, but no intervention has synergised the two approaches.
Aim
To analyse the effects of an intervention combining EVOO and a multicomponent health promotion and PE programme on disease activity, clinical characteristics, cardiovascular risk, physical fitness, and the molecular level in SLE sufferers.
Methods
Three-arm prospective randomised controlled 24-week clinical trial. 90 participants will be randomised into one of three groups: control; EVOO supplements; or EVOO and multicomponent health promotion and PE programme.
Results
Pre-, mid- and post-intervention assessments will record disease activity, clinical characteristics, nutritional evaluation, cardiovascular risk assessment, physical condition and functioning, and molecular markers.
Conclusions
The proposed trial will help clarify whether a combined intervention adding an EVOO supplement to a Mediterranean Diet intake pattern and adherence to an active-healthy lifestyle are beneficial for SLE patients, as well as the need for health and pharmacological care, increasing knowledge of the organic mechanisms mediated by EVOO and PE adherence, allowing new useful biomarkers to be characterised at the diagnostic/prognostic level.
{"title":"Effects of an intervention with EVOO and physical exercise in systemic lupus erythematosus patients: Efinutriles trial protocol","authors":"R. Gil-Gutiérrez , I. Medina-Martínez , C. Ballesteros-Rubio , F.J. De La Hera-Fernández , R. Ríos-Fernández , J.L. Callejas-Rubio , M. Zamora-Pasadas , I. Cantarero-Villanueva , M. Correa-Rodríguez , N. Ortego-Centeno , B. Rueda-Medina","doi":"10.1016/j.cct.2024.107747","DOIUrl":"10.1016/j.cct.2024.107747","url":null,"abstract":"<div><h3>Background</h3><div>Health-related lifestyle management could improve related symptoms and adverse events in patients with systemic lupus erythematosus (SLE). The phenolic compounds in extra virgin olive oil (EVOO) and physical exercise (PE) have both shown benefits for autoimmune conditions, but no intervention has synergised the two approaches.</div></div><div><h3>Aim</h3><div>To analyse the effects of an intervention combining EVOO and a multicomponent health promotion and PE programme on disease activity, clinical characteristics, cardiovascular risk, physical fitness, and the molecular level in SLE sufferers.</div></div><div><h3>Methods</h3><div>Three-arm prospective randomised controlled 24-week clinical trial. 90 participants will be randomised into one of three groups: control; EVOO supplements; or EVOO and multicomponent health promotion and PE programme.</div></div><div><h3>Results</h3><div>Pre-, mid- and post-intervention assessments will record disease activity, clinical characteristics, nutritional evaluation, cardiovascular risk assessment, physical condition and functioning, and molecular markers.</div></div><div><h3>Conclusions</h3><div>The proposed trial will help clarify whether a combined intervention adding an EVOO supplement to a Mediterranean Diet intake pattern and adherence to an active-healthy lifestyle are beneficial for SLE patients, as well as the need for health and pharmacological care, increasing knowledge of the organic mechanisms mediated by EVOO and PE adherence, allowing new useful biomarkers to be characterised at the diagnostic/prognostic level.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107747"},"PeriodicalIF":2.0,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142681259","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}
Pub Date : 2024-11-19DOI: 10.1016/j.cct.2024.107755
Genevieve Dupuis , Ryan E. Ferguson , Areef Ishani , William C. Cushman , Sarah M. Leatherman
Background
The Diuretic Comparison Project (DCP) was a multi-center, embedded pragmatic trial conducted within the VA healthcare system comparing chlorthalidone and hydrochlorothiazide in preventing major adverse cardiovascular events and non-cancer deaths in hypertensive patients. Study procedures were decentralized, and facility leadership first had to agree and accept study procedures before staff approached participants. Recent evidence suggests facilities that choose and choose not to participate in trials may differ and this study considered such differences within DCP.
Methods
A cross-sectional comparison of facilities participating in DCP was conducted with data from the study start (June 2016) including: 2016 American Community Survey, 2016 Strategic Analytics for Improvement and Learning reports, star ratings, and 2014 hospital complexity. Characteristics of participating and non-participating centers were compared using logistic regression, including county-level socio-economic features and hospital-level performance.
Results
Of 144 VA medical centers, leadership at 75 centers (52 %) initiated participation. Facilities in highly-urban and higher median income counties were more likely to participate, as were higher-complexity facilities. Facilities with research experience were 2.18 times as likely to participate. No other hospital performance metrics or county-level demographics were associated with participation.
Conclusions
Overall, this study suggests research exposure and quality care metrics may impact a facility's decision to participate. These results highlight key considerations for recruitment to multi-site, and particularly pragmatic, clinical trials. Consideration of supporting facilities that have not historically participated in research may be fruitful for recruitment. These results emphasize the importance of education about pragmatic study design and its integration with clinical care.
{"title":"Characteristics of VA hospitals by participation status in a large pragmatic embedded clinical trial","authors":"Genevieve Dupuis , Ryan E. Ferguson , Areef Ishani , William C. Cushman , Sarah M. Leatherman","doi":"10.1016/j.cct.2024.107755","DOIUrl":"10.1016/j.cct.2024.107755","url":null,"abstract":"<div><h3>Background</h3><div>The Diuretic Comparison Project (DCP) was a multi-center, embedded pragmatic trial conducted within the VA healthcare system comparing chlorthalidone and hydrochlorothiazide in preventing major adverse cardiovascular events and non-cancer deaths in hypertensive patients. Study procedures were decentralized, and facility leadership first had to agree and accept study procedures before staff approached participants. Recent evidence suggests facilities that choose and choose not to participate in trials may differ and this study considered such differences within DCP.</div></div><div><h3>Methods</h3><div>A cross-sectional comparison of facilities participating in DCP was conducted with data from the study start (June 2016) including: 2016 American Community Survey, 2016 Strategic Analytics for Improvement and Learning reports, star ratings, and 2014 hospital complexity. Characteristics of participating and non-participating centers were compared using logistic regression, including county-level socio-economic features and hospital-level performance.</div></div><div><h3>Results</h3><div>Of 144 VA medical centers, leadership at 75 centers (52 %) initiated participation. Facilities in highly-urban and higher median income counties were more likely to participate, as were higher-complexity facilities. Facilities with research experience were 2.18 times as likely to participate. No other hospital performance metrics or county-level demographics were associated with participation.</div></div><div><h3>Conclusions</h3><div>Overall, this study suggests research exposure and quality care metrics may impact a facility's decision to participate. These results highlight key considerations for recruitment to multi-site, and particularly pragmatic, clinical trials. Consideration of supporting facilities that have not historically participated in research may be fruitful for recruitment. These results emphasize the importance of education about pragmatic study design and its integration with clinical care.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107755"},"PeriodicalIF":2.0,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142681257","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}
Pub Date : 2024-11-17DOI: 10.1016/j.cct.2024.107744
Keith A. Marsolo , Andrea Cheville , Edward R. Melnick , Jeffrey G. Jarvik , Gregory E. Simon , Kathleen A. Sluka , Leslie J. Crofford , Karen L. Staman , Rachel L. Richesson , Judith M. Schlaeger , Lesley H. Curtis
The NIH Pragmatic Trials Collaboratory supports the design and conduct of 32 embedded pragmatic clinical trials, and many of these trials rely on data from the electronic health record (EHR) to monitor outcomes and/or use functionality provided by the EHR platform to deliver the intervention. Given the complexity and dynamic nature of EHR systems, study teams have encountered challenges in use of the EHR for these purposes, including challenges related to local implementation of trial interventions, rapid technology evolution, EHR updates, and transitions in EHR systems. In this article, we share case examples and lessons learned, and suggest that teams need to be aware of—and perhaps proactively investigate— possible changes to EHR systems and data that will affect the delivery of interventions and the integrity and safety of pragmatic clinical trials.
美国国立卫生研究院（NIH）实用临床试验协作组支持 32 项嵌入式实用临床试验的设计和实施，其中许多试验依赖电子病历（EHR）中的数据来监测结果和/或使用 EHR 平台提供的功能来实施干预。鉴于电子病历系统的复杂性和动态性，研究团队在将电子病历用于这些目的时遇到了挑战，包括与试验干预措施的本地实施、快速技术演进、电子病历更新和电子病历系统过渡有关的挑战。在这篇文章中，我们分享了案例和经验教训，并建议研究小组需要意识到--或许可以主动调查--电子病历系统和数据可能发生的变化，这些变化将影响干预措施的实施以及务实临床试验的完整性和安全性。
{"title":"Impact of electronic health record updates and changes on the delivery and monitoring of interventions in embedded pragmatic clinical trials","authors":"Keith A. Marsolo , Andrea Cheville , Edward R. Melnick , Jeffrey G. Jarvik , Gregory E. Simon , Kathleen A. Sluka , Leslie J. Crofford , Karen L. Staman , Rachel L. Richesson , Judith M. Schlaeger , Lesley H. Curtis","doi":"10.1016/j.cct.2024.107744","DOIUrl":"10.1016/j.cct.2024.107744","url":null,"abstract":"<div><div>The NIH Pragmatic Trials Collaboratory supports the design and conduct of 32 embedded pragmatic clinical trials, and many of these trials rely on data from the electronic health record (EHR) to monitor outcomes and/or use functionality provided by the EHR platform to deliver the intervention. Given the complexity and dynamic nature of EHR systems, study teams have encountered challenges in use of the EHR for these purposes, including challenges related to local implementation of trial interventions, rapid technology evolution, EHR updates, and transitions in EHR systems. In this article, we share case examples and lessons learned, and suggest that teams need to be aware of—and perhaps proactively investigate— possible changes to EHR systems and data that will affect the delivery of interventions and the integrity and safety of pragmatic clinical trials.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107744"},"PeriodicalIF":2.0,"publicationDate":"2024-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142674989","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}
Pub Date : 2024-11-17DOI: 10.1016/j.cct.2024.107754
John Lin , Rachel L. Winer , Christina Bliss Barsness , Jay Desai , Kristi Fordyce , Rahel Ghebre , Anisa M. Ibrahim , Sharif Mohamed , Timothy Ramer , Adam A. Szpiro , Bryan J. Weiner , Sophia Yohe , Rebekah Pratt
Background
Somali American individuals have lower cervical cancer screening rates than the U.S. general population. Offering HPV self-sampling in primary care clinics could increase screening rates in Somali American individuals by addressing screening barriers.
Methods
The Isbaar Project is a Hybrid Type 2 effectiveness-implementation study of a patient-centered, culturally tailored HPV self-sampling intervention for Somali American individuals. Guided by the Consolidated Framework for Implementation Research and Social Cognitive Theory, we conducted focus groups with Somali American individuals, and interviews with clinicians and clinic staff to inform refinement and development of implementation strategies. HPV self-sampling was then implemented as a usual care screening option at 3 community-based primary care clinics in Minneapolis, Minnesota in February 2023. The primary objective is to assess the effect of implementing in-clinic HPV self-sampling on screening completion in Somali American individuals. The secondary objective is to assess the effect of implementing HPV self-sampling on screening completion in all patients. Using difference-in-difference methods, we will evaluate changes in screening rates one-year pre and post implementation and compare changes with control clinics followed over the same time period. Using RE-AIM, we will conduct a post-implementation mixed methods analysis of processes and strategies needed to successfully implement HPV self-sampling in primary care.
Conclusions
The study was designed to evaluate a real-world in-clinic HPV self-sampling intervention for Somali American individuals, generating data on both effectiveness and implementation applicable to other community-based clinics in the U.S. The objective of this report is to describe the rationale and design of the study.
{"title":"Design of a pragmatic trial integrating human papillomavirus (HPV) self-sampling into primary care to reduce cervical cancer screening disparities in Somali American individuals: The Isbaar project","authors":"John Lin , Rachel L. Winer , Christina Bliss Barsness , Jay Desai , Kristi Fordyce , Rahel Ghebre , Anisa M. Ibrahim , Sharif Mohamed , Timothy Ramer , Adam A. Szpiro , Bryan J. Weiner , Sophia Yohe , Rebekah Pratt","doi":"10.1016/j.cct.2024.107754","DOIUrl":"10.1016/j.cct.2024.107754","url":null,"abstract":"<div><h3>Background</h3><div>Somali American individuals have lower cervical cancer screening rates than the U.S. general population. Offering HPV self-sampling in primary care clinics could increase screening rates in Somali American individuals by addressing screening barriers.</div></div><div><h3>Methods</h3><div>The Isbaar Project is a Hybrid Type 2 effectiveness-implementation study of a patient-centered, culturally tailored HPV self-sampling intervention for Somali American individuals. Guided by the Consolidated Framework for Implementation Research and Social Cognitive Theory, we conducted focus groups with Somali American individuals, and interviews with clinicians and clinic staff to inform refinement and development of implementation strategies. HPV self-sampling was then implemented as a usual care screening option at 3 community-based primary care clinics in Minneapolis, Minnesota in February 2023. The primary objective is to assess the effect of implementing in-clinic HPV self-sampling on screening completion in Somali American individuals. The secondary objective is to assess the effect of implementing HPV self-sampling on screening completion in all patients. Using difference-in-difference methods, we will evaluate changes in screening rates one-year pre and post implementation and compare changes with control clinics followed over the same time period. Using RE-AIM, we will conduct a post-implementation mixed methods analysis of processes and strategies needed to successfully implement HPV self-sampling in primary care.</div></div><div><h3>Conclusions</h3><div>The study was designed to evaluate a real-world in-clinic HPV self-sampling intervention for Somali American individuals, generating data on both effectiveness and implementation applicable to other community-based clinics in the U.S. The objective of this report is to describe the rationale and design of the study.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107754"},"PeriodicalIF":2.0,"publicationDate":"2024-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142674720","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}
Pub Date : 2024-11-17DOI: 10.1016/j.cct.2024.107749
Rebekah Laidsaar-Powell , Sarah Giunta , Lisa Beatty , Phyllis Butow , Daniel Costa , Aaron Lam , Ilona Juraskova , Olivia Cook , Fiona Crawford-Williams , Nicole M. Rankin , Joanne Shaw
Background
Family or friend carers of people with cancer report high levels of depression, anxiety, caregiving strain, and unmet needs. Limited strategies for identification and management of distress have been established among cancer carers. This paper describes the protocol of two linked studies: Study 1a, a distress screening and stepped care pathway feasibility study and Study 1b, a hybrid implementation-effectiveness Randomised Controlled Trial (RCT) to assess the benefit of a comprehensive, carer-centred online Cognitive Behavioural Therapy (iCBT) program for carers with anxiety and depression.
Methods
For Study 1a, 300 cancer carers will be screened for distress. Carers with low distress will be referred to publicly available carer resources. Carers scoring 4 and higher on the distress thermometer will complete depression and anxiety measures. Carers with high anxiety/depression will be recommended psychological therapy. Carers with mild/moderate anxiety and/or depression will be allocated to the Carers iCBT Program, evaluated via a RCT with waitlist control group (Study 1b). For Study 1b, intervention group carers will receive access to a 6-lesson self-directed online iCBT program. Waitlist-controls will access the intervention at 14 weeks. Intervention and control groups will complete baseline, 6 week, and 14 week self-report measures; controls will complete additional measures at 20 and 28 weeks. A sample size of n = 166 carers in the iCBT RCT is needed.
Conclusions
If acceptable, feasible and effective, this pathway and iCBT intervention could offer a sustainable, scalable and low-cost approach to identifying and managing distress in carers, and potentially improving patient and carer outcomes.
Trial Registration: Australian and New Zealand Clinical Trial Registry number: ACTRN12623001341617p.
{"title":"CarersCanADAPT: Study protocol of a stepped care pathway and hybrid type 1 effectiveness-implementation trial of an online cognitive behavioural therapy (iCBT) program for cancer carers with anxiety and depression","authors":"Rebekah Laidsaar-Powell , Sarah Giunta , Lisa Beatty , Phyllis Butow , Daniel Costa , Aaron Lam , Ilona Juraskova , Olivia Cook , Fiona Crawford-Williams , Nicole M. Rankin , Joanne Shaw","doi":"10.1016/j.cct.2024.107749","DOIUrl":"10.1016/j.cct.2024.107749","url":null,"abstract":"<div><h3>Background</h3><div>Family or friend carers of people with cancer report high levels of depression, anxiety, caregiving strain, and unmet needs. Limited strategies for identification and management of distress have been established among cancer carers. This paper describes the protocol of two linked studies: Study 1a, a distress screening and stepped care pathway feasibility study and Study 1b, a hybrid implementation-effectiveness Randomised Controlled Trial (RCT) to assess the benefit of a comprehensive, carer-centred online Cognitive Behavioural Therapy (iCBT) program for carers with anxiety and depression.</div></div><div><h3>Methods</h3><div>For Study 1a, 300 cancer carers will be screened for distress. <em>Carers with low distress</em> will be referred to publicly available carer resources. Carers scoring 4 and higher on the distress thermometer will complete depression and anxiety measures. <em>Carers with high anxiety/depression</em> will be recommended psychological therapy. <em>Carers with mild/moderate anxiety and/or depression</em> will be allocated to the Carers iCBT Program, evaluated via a RCT with waitlist control group (Study 1b). For Study 1b, intervention group carers will receive access to a 6-lesson self-directed online iCBT program. Waitlist-controls will access the intervention at 14 weeks. Intervention and control groups will complete baseline, 6 week, and 14 week self-report measures; controls will complete additional measures at 20 and 28 weeks. A sample size of <em>n</em> = 166 carers in the iCBT RCT is needed.</div></div><div><h3>Conclusions</h3><div>If acceptable, feasible and effective, this pathway and iCBT intervention could offer a sustainable, scalable and low-cost approach to identifying and managing distress in carers, and potentially improving patient and carer outcomes.</div><div><strong>Trial Registration:</strong> Australian and New Zealand Clinical Trial Registry number: ACTRN12623001341617p.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107749"},"PeriodicalIF":2.0,"publicationDate":"2024-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142674791","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}
Pub Date : 2024-11-17DOI: 10.1016/j.cct.2024.107751
Thanh K. Thai , Christine A. Board , Joshua R. Nugent , Jessica L. Johnston , Esther Y. Huynh , Cindy Hanh Chen , Andy H. Chan , Richard W. Grant , Lisa K. Gilliam , Anjali Gopalan
Therapeutic inertia, the delay in the appropriate initiation and intensification of recommended therapies, is a major contributor to the lack of improvement in type 2 diabetes outcomes over the past decade. Therapeutic inertia during the period following diagnosis, when improvements in glycemic control can have lasting benefits, is often overlooked. Technology and team-based care approaches can effectively address therapeutic inertia. Here, we describe the protocol for a randomized, quality improvement trial targeting metformin-related therapeutic inertia among adults with recently diagnosed type 2 diabetes and a hemoglobin A1c <8 %. Service areas within an integrated healthcare delivery system were randomized to 1) usual care, 2) physician education on therapeutic inertia, and 3) physician education on therapeutic inertia paired with proactive outreach by a clinical pharmacist. The physician education sessions were offered to primary care providers working in the service areas randomized to Arm 2 and Arm 3, and proactive outreach by a clinical pharmacist was performed for patients of the providers in service areas randomized to Arm 3. Outcomes will be abstracted from the EHR at 6, 12, and 18 months following the physician education sessions. The primary outcome will be the proportion of patients with an HbA1c <7 % at each of the follow-up time points. Outcome abstraction and analyses will occur in late 2024. This trial seeks to rigorously evaluate care strategies that can shift stagnant type 2 diabetes outcomes. Our protocol, along with the pending results, may offer examples to other healthcare systems working to improve type 2 diabetes care.
{"title":"Overcoming therapeutic inertia in newly diagnosed type 2 diabetes: Protocol of a randomized, quality improvement trial","authors":"Thanh K. Thai , Christine A. Board , Joshua R. Nugent , Jessica L. Johnston , Esther Y. Huynh , Cindy Hanh Chen , Andy H. Chan , Richard W. Grant , Lisa K. Gilliam , Anjali Gopalan","doi":"10.1016/j.cct.2024.107751","DOIUrl":"10.1016/j.cct.2024.107751","url":null,"abstract":"<div><div>Therapeutic inertia, the delay in the appropriate initiation and intensification of recommended therapies, is a major contributor to the lack of improvement in type 2 diabetes outcomes over the past decade. Therapeutic inertia during the period following diagnosis, when improvements in glycemic control can have lasting benefits, is often overlooked. Technology and team-based care approaches can effectively address therapeutic inertia. Here, we describe the protocol for a randomized, quality improvement trial targeting metformin-related therapeutic inertia among adults with recently diagnosed type 2 diabetes and a hemoglobin A1c <8 %. Service areas within an integrated healthcare delivery system were randomized to 1) usual care, 2) physician education on therapeutic inertia, and 3) physician education on therapeutic inertia paired with proactive outreach by a clinical pharmacist. The physician education sessions were offered to primary care providers working in the service areas randomized to Arm 2 and Arm 3, and proactive outreach by a clinical pharmacist was performed for patients of the providers in service areas randomized to Arm 3. Outcomes will be abstracted from the EHR at 6, 12, and 18 months following the physician education sessions. The primary outcome will be the proportion of patients with an HbA1c <7 % at each of the follow-up time points. Outcome abstraction and analyses will occur in late 2024. This trial seeks to rigorously evaluate care strategies that can shift stagnant type 2 diabetes outcomes. Our protocol, along with the pending results, may offer examples to other healthcare systems working to improve type 2 diabetes care.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107751"},"PeriodicalIF":2.0,"publicationDate":"2024-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142667152","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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