Contemporary Clinical Trials Communications最新文献_第7页

TEleRehabilitation foR Aphasia (TERRA) phase II trial design 远程康复治疗失语症（TERRA） II期试验设计

IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL

Contemporary Clinical Trials Communications

Pub Date : 2024-12-01 DOI: 10.1016/j.conctc.2024.101406

Christy Cassarly , Alexandra Basilakos , Lisa Johnson , Janina Wilmskoetter , Jordan Elm , Argye E. Hillis , Leonardo Bonilha , Chris Rorden , Gregory Hickok , Dirk-Bart den Ouden , Julius Fridriksson

Background and purpose

Despite comprehensive evidence that supports the utility of aphasia therapy in persons with chronic (≥6 months) stroke-induced aphasia, the amount of therapy provided to patients in the United States is typically far less than what is likely necessary to maximize recovery. Two potential contributors to this discrepancy are limited access to rehabilitation services due to the availability of providers and logistical difficulties with transportation. One way to increase access to aphasia therapy is to rely on telerehabilitation.

Methods

The TEleRehabilitation foR Aphasia (TERRA) trial is a prospective, randomized, rater-blinded, multicenter phase II non-inferiority trial to evaluate telerehabilitation for aphasia therapy in persons with chronic post-stroke aphasia. Participants are randomized (1:1) to receive either aphasia remote therapy or in-clinic therapy for 30 total days of treatment (15 days of a semantically focused approach and 15 days of a phonologically focused approach) for 45 min per day. A total of 100 adults (ages 21–80) with a history of left hemisphere ischemic or hemorrhagic stroke incurred at least 12 months prior to study enrollment will be randomized. The trial will be conducted at the clinical research facilities at two sites: the Medical University of South Carolina and the University of South Carolina.

Conclusions

This paper details the design of the TERRA trial, which aims to test whether aphasia therapy delivered by a remote speech-language pathologist through videoconferencing (i.e., via telerehabilitation) is not clinically worse than in-clinic therapy for individuals with chronic post-stroke aphasia to provide an opportunity to move to a definitive phase III trial.

背景和目的尽管有全面的证据支持对慢性（≥6个月）卒中引起的失语症患者进行失语症治疗的有效性，但在美国，提供给患者的治疗量通常远远少于可能最大化恢复所需的量。造成这一差异的两个可能因素是，由于提供人员有限，获得康复服务的机会有限，以及运输方面的后勤困难。增加获得失语症治疗的一种方法是依赖远程康复。远程康复治疗失语症（TERRA）试验是一项前瞻性、随机、非盲法、多中心II期非劣效性试验，旨在评估远程康复治疗失语症对慢性脑卒中后失语症患者的疗效。参与者随机（1:1）接受失语症远程治疗或临床治疗，共30天，每天45分钟（15天为语义集中治疗，15天为语音集中治疗）。共有100名成年人（年龄21-80岁）在研究入组前至少12个月发生过左半球缺血性或出血性中风病史，将被随机分组。该试验将在两个地点的临床研究机构进行：南卡罗来纳医科大学和南卡罗来纳大学。本文详细介绍了TERRA试验的设计，该试验旨在测试由远程语言病理学家通过视频会议（即通过远程康复）提供的失语症治疗是否比临床治疗对慢性中风后失语症患者的临床治疗更差，从而为进入最终的III期试验提供机会。

{"title":"TEleRehabilitation foR Aphasia (TERRA) phase II trial design","authors":"Christy Cassarly , Alexandra Basilakos , Lisa Johnson , Janina Wilmskoetter , Jordan Elm , Argye E. Hillis , Leonardo Bonilha , Chris Rorden , Gregory Hickok , Dirk-Bart den Ouden , Julius Fridriksson","doi":"10.1016/j.conctc.2024.101406","DOIUrl":"10.1016/j.conctc.2024.101406","url":null,"abstract":"<div><h3>Background and purpose</h3><div>Despite comprehensive evidence that supports the utility of aphasia therapy in persons with chronic (≥6 months) stroke-induced aphasia, the amount of therapy provided to patients in the United States is typically far less than what is likely necessary to maximize recovery. Two potential contributors to this discrepancy are limited access to rehabilitation services due to the availability of providers and logistical difficulties with transportation. One way to increase access to aphasia therapy is to rely on telerehabilitation.</div></div><div><h3>Methods</h3><div>The TEleRehabilitation foR Aphasia (TERRA) trial is a prospective, randomized, rater-blinded, multicenter phase II non-inferiority trial to evaluate telerehabilitation for aphasia therapy in persons with chronic post-stroke aphasia. Participants are randomized (1:1) to receive either aphasia remote therapy or in-clinic therapy for 30 total days of treatment (15 days of a semantically focused approach and 15 days of a phonologically focused approach) for 45 min per day. A total of 100 adults (ages 21–80) with a history of left hemisphere ischemic or hemorrhagic stroke incurred at least 12 months prior to study enrollment will be randomized. The trial will be conducted at the clinical research facilities at two sites: the Medical University of South Carolina and the University of South Carolina.</div></div><div><h3>Conclusions</h3><div>This paper details the design of the TERRA trial, which aims to test whether aphasia therapy delivered by a remote speech-language pathologist through videoconferencing (i.e., via telerehabilitation) is not clinically worse than in-clinic therapy for individuals with chronic post-stroke aphasia to provide an opportunity to move to a definitive phase III trial.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101406"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142757564","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Clinical study of Jianpi Qingchang decoction in the treatment of ulcerative colitis patients with spleen deficiency and dampness-heat syndrome accompanied by fatigue: Study protocol for a randomized controlled trial 健脾清肠汤治疗溃疡性结肠炎脾虚湿热证伴疲劳的临床研究：随机对照试验研究方案

IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL

Contemporary Clinical Trials Communications

Pub Date : 2024-12-01 DOI: 10.1016/j.conctc.2024.101409

Zi-Xuan Liu , Xiao-Yan Liu , Wei-wei Tan , Wei-Bing Zhang , Ya-Li Zhang , Lie Zheng , Yan-Cheng Dai

Background

Ulcerative colitis (UC) is a chronic non-specific inflammatory intestinal disease, categoried under "dysentery" and "intestinal bleeding" in Traditional Chinese Medicine (TCM). Jianpi Qingchang decoction (JPQC) is a combination formula specifically designed for the treatment of UC. The primary objective of this study is to examine the clinical efficacy of JPQC in individuals diagnosed with UC who exhibit both spleen deficiency and dampness-heat syndrome, along with the presence of fatigue. The investigation will focus on assessing the impact of JPQC on the gut microbiota and metabolites in these patients, aiming to elucidate the regulatory mechanism that JPQC exerts on the gut microbiota and metabolites in the context of UC-related fatigue.

Methods

In this randomized clinical trial, 140 subjects diagnosed with UC will be recruited and randomized into two groups. They will receive either JPQC combined with mesalazine or mesalazine alone for 12 weeks. Follow-up visits will be conducted every four weeks, with a post-treatment visit scheduled at 6 months. The primary outcome measures include the Inflammatory bowel disease fatigue scale(IBD-F). Secondary efficacy indicators comprise the assessment of TCM syndrome and individual syndrome efficacy before and after treatment, Modified Mayo score, Simple clinical colitis activity index (SCCAI), as well as the Inflammatory Bowel Disease Questionnaire (IBDQ) for each group. The other outcomes are the Intestinal microbial diversity and non-targeted metabonomics, which will be measured at baseline and 12 weeks after randomization.

Discussion

If effective, JPQC will provide substantial clinical evidence concerning the effectiveness and safety in the treatment of patients with UC experiencing spleen deficiency and dampness-heat syndrome accompanied by fatigue.

Trial registration

ChiCTR2300068348.

背景：溃疡性结肠炎（UC）是一种慢性非特异性炎症性肠道疾病，中医分类为“痢疾”和“肠出血”。健脾清肠汤（JPQC）是专门为治疗UC而设计的复方。本研究的主要目的是研究JPQC对脾虚湿热证合并疲劳的UC患者的临床疗效。研究将重点评估JPQC对这些患者肠道微生物群和代谢物的影响，旨在阐明JPQC在uc相关性疲劳背景下对肠道微生物群和代谢物的调节机制。方法：在这项随机临床试验中，140名诊断为UC的受试者将被招募并随机分为两组。他们将接受JPQC联合美沙拉嗪或美沙拉嗪单独治疗，为期12周。每四周进行一次随访，治疗后6个月进行一次随访。主要结局指标包括炎症性肠病疲劳量表（IBD-F）。次要疗效指标包括治疗前后中医证候及个体证候疗效评估、改良梅奥评分、单纯临床结肠炎活动性指数（SCCAI）、炎症性肠病问卷（IBDQ）。其他结果是肠道微生物多样性和非靶向代谢组学，将在基线和随机分组后12周进行测量。讨论：如果有效，JPQC将为UC脾虚湿热证伴疲劳的有效性和安全性提供大量的临床证据。试验注册：ChiCTR2300068348。

{"title":"Clinical study of Jianpi Qingchang decoction in the treatment of ulcerative colitis patients with spleen deficiency and dampness-heat syndrome accompanied by fatigue: Study protocol for a randomized controlled trial","authors":"Zi-Xuan Liu , Xiao-Yan Liu , Wei-wei Tan , Wei-Bing Zhang , Ya-Li Zhang , Lie Zheng , Yan-Cheng Dai","doi":"10.1016/j.conctc.2024.101409","DOIUrl":"10.1016/j.conctc.2024.101409","url":null,"abstract":"<div><h3>Background</h3><div>Ulcerative colitis (UC) is a chronic non-specific inflammatory intestinal disease, categoried under \"dysentery\" and \"intestinal bleeding\" in Traditional Chinese Medicine (TCM). Jianpi Qingchang decoction (JPQC) is a combination formula specifically designed for the treatment of UC. The primary objective of this study is to examine the clinical efficacy of JPQC in individuals diagnosed with UC who exhibit both spleen deficiency and dampness-heat syndrome, along with the presence of fatigue. The investigation will focus on assessing the impact of JPQC on the gut microbiota and metabolites in these patients, aiming to elucidate the regulatory mechanism that JPQC exerts on the gut microbiota and metabolites in the context of UC-related fatigue.</div></div><div><h3>Methods</h3><div>In this randomized clinical trial, 140 subjects diagnosed with UC will be recruited and randomized into two groups. They will receive either JPQC combined with mesalazine or mesalazine alone for 12 weeks. Follow-up visits will be conducted every four weeks, with a post-treatment visit scheduled at 6 months. The primary outcome measures include the Inflammatory bowel disease fatigue scale(IBD-F). Secondary efficacy indicators comprise the assessment of TCM syndrome and individual syndrome efficacy before and after treatment, Modified Mayo score, Simple clinical colitis activity index (SCCAI), as well as the Inflammatory Bowel Disease Questionnaire (IBDQ) for each group. The other outcomes are the Intestinal microbial diversity and non-targeted metabonomics, which will be measured at baseline and 12 weeks after randomization.</div></div><div><h3>Discussion</h3><div>If effective, JPQC will provide substantial clinical evidence concerning the effectiveness and safety in the treatment of patients with UC experiencing spleen deficiency and dampness-heat syndrome accompanied by fatigue.</div></div><div><h3>Trial registration</h3><div>ChiCTR2300068348.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101409"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11681875/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142903724","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Involving youth with intellectual and/or developmental disabilities as collaborators in a comparative effectiveness trial: A community-engaged research approach 让有智力和/或发育障碍的青年作为合作者参与比较有效性试验：一种社区参与的研究方法

IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL

Contemporary Clinical Trials Communications

Pub Date : 2024-12-01 DOI: 10.1016/j.conctc.2024.101395

K.L. Berg , D Herrman , L Bernard , C.S Shiu , I Mihaila , C Arnold , K Acharya , T.R.G Gladstone , C Danguilan , H Gussin , P Perez , A Herrman , S Aaron , A Thornton , M Gerges , C Patriarca , J.J Pak , B.W Van Voorhees

Background

Practices to include youth with intellectual and/or developmental disabilities (IDD) are necessary to design and implement research that specifically meets the behavioral health needs of this population. This article describes a protocol for engaging youth with IDD as collaborators in a comparative effectiveness clinical trial using a community-engaged research (CEnR) approach.

Methods

Our engagement protocol, guided by the Community Engaged Research (CEnR) Framework, emphasized harm avoidance, accessibility, demonstrated value, capacity bridging and co-learning, shared power and equity in decision-making, accountability and respect, and transparent communication. We involved seven youth with IDD in a Youth Advisory Committee (YAC) and four youth with IDD in a Summer Scholars program, ensuring consistent and structured engagement throughout the study.

Results

Youth with IDD maintained high levels of engagement in both the YAC and Summer Scholars Program with 100 % retention across two years. Youth used multiple modalities to provide feedback on aspects of the research project, resulting in study modifications, the co-development of products, and tangible improvements in the accessibility and relevance of the study for youth with IDD.

Conclusion

Researchers and clinicians seeking to engage the historically underserved population of disabled youth in clinical trial research can leverage our findings to enhance the accessibility and inclusivity of their studies.

背景实践包括青少年智力和/或发育障碍（IDD）是必要的设计和实施研究，专门满足这一人群的行为健康需求。本文描述了一种利用社区参与研究（CEnR）方法让患有IDD的青年作为合作者参与比较有效性临床试验的方案。方法以社区参与研究（CEnR）框架为指导，我们的参与协议强调避免伤害、可及性、展示价值、能力衔接和共同学习、共享权力和决策公平、问责制和尊重，以及透明的沟通。我们让7名患有IDD的青年参加青年咨询委员会（YAC），让4名患有IDD的青年参加暑期学者项目，确保整个研究过程中始终有组织地参与。结果IDD青年在YAC和夏季学者项目中保持了很高的参与度，两年内保持了100%的参与度。青年使用多种方式对研究项目的各个方面提供反馈，从而修改了研究，共同开发了产品，并切实改善了研究对缺碘症青年的可及性和相关性。研究人员和临床医生可以利用我们的研究结果来提高他们研究的可及性和包容性，以吸引历史上服务不足的残疾青年人群参与临床试验研究。

{"title":"Involving youth with intellectual and/or developmental disabilities as collaborators in a comparative effectiveness trial: A community-engaged research approach","authors":"K.L. Berg , D Herrman , L Bernard , C.S Shiu , I Mihaila , C Arnold , K Acharya , T.R.G Gladstone , C Danguilan , H Gussin , P Perez , A Herrman , S Aaron , A Thornton , M Gerges , C Patriarca , J.J Pak , B.W Van Voorhees","doi":"10.1016/j.conctc.2024.101395","DOIUrl":"10.1016/j.conctc.2024.101395","url":null,"abstract":"<div><h3>Background</h3><div>Practices to include youth with intellectual and/or developmental disabilities (IDD) are necessary to design and implement research that specifically meets the behavioral health needs of this population. This article describes a protocol for engaging youth with IDD as collaborators in a comparative effectiveness clinical trial using a community-engaged research (CEnR) approach.</div></div><div><h3>Methods</h3><div>Our engagement protocol, guided by the Community Engaged Research (CEnR) Framework, emphasized harm avoidance, accessibility, demonstrated value, capacity bridging and co-learning, shared power and equity in decision-making, accountability and respect, and transparent communication. We involved seven youth with IDD in a Youth Advisory Committee (YAC) and four youth with IDD in a Summer Scholars program, ensuring consistent and structured engagement throughout the study.</div></div><div><h3>Results</h3><div>Youth with IDD maintained high levels of engagement in both the YAC and Summer Scholars Program with 100 % retention across two years. Youth used multiple modalities to provide feedback on aspects of the research project, resulting in study modifications, the co-development of products, and tangible improvements in the accessibility and relevance of the study for youth with IDD.</div></div><div><h3>Conclusion</h3><div>Researchers and clinicians seeking to engage the historically underserved population of disabled youth in clinical trial research can leverage our findings to enhance the accessibility and inclusivity of their studies.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101395"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142744572","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Comparison of crisis response planning and treatment as usual for active duty service members at risk for suicide: Study protocol for a stepped-wedge cluster randomized trial in a military treatment facility 有自杀风险的现役军人危机应对计划与常规治疗的比较：在军事治疗设施中进行的阶梯形随机试验的研究方案。

IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL

Contemporary Clinical Trials Communications

Pub Date : 2024-12-01 DOI: 10.1016/j.conctc.2024.101407

Kristen H. Walter , Pia R. Khandekar , Alexander C. Kline , Erin L. Miggantz , Nicholas P. Otis , Lisa H. Glassman , Cynthia J. Thomsen , Guy Brock , Craig J. Bryan

Background

Suicide is one of the leading causes of death among U.S. service members, and rates of suicide among military personnel have increased over the past two decades. To address this serious issue, effective preventive treatments are needed in settings where at-risk service members are frequently seen, such as emergency departments and inpatient psychiatric units. This study will compare the longitudinal effects of crisis response planning (CRP) and treatment as usual (TAU) on suicidal thoughts and behaviors among active duty service members seeking emergent care for suicidality at a military treatment facility.

Methods

The current study is conducted through a consortium, Augmenting Suicide Prevention Interventions for Service Members. This article details an ongoing stepped-wedge cluster randomized clinical trial that compares rates of suicidal thoughts and behaviors among service members at risk for suicide following care from CRP-trained providers versus untrained providers (i.e., TAU). Participants complete assessments at pretreatment and every 3 months up to 1 year. Primary outcomes include suicide attempts and behaviors, and suicidal ideation is a secondary outcome. Moderators of treatment effects will also be examined. The methodological development of this trial is discussed, along with clinical and ethical considerations for suicide prevention research in emergency, inpatient, and military treatment settings.

Conclusion

Providing evidence-based treatment for suicidality that addresses service members’ unique needs is crucial to reduce suicide rates and facilitate mental health recovery in this population. This study aims to inform future implementation and dissemination of CRP in healthcare systems to ultimately decrease suicide among service members.

Clinical trials identifier

NCT05795764.

背景：自杀是美国军人死亡的主要原因之一，在过去的二十年里，军人的自杀率有所上升。为了解决这一严重问题，需要在经常见到有危险的服务人员的环境中，如急诊科和精神病住院病房，采取有效的预防性治疗。本研究将比较危机应对计划（CRP）和常规治疗（TAU）对在军事治疗机构寻求自杀紧急护理的现役军人自杀念头和行为的纵向影响。方法：目前的研究是通过一个联盟进行的，为服务成员增加自杀预防干预。这篇文章详细介绍了一项正在进行的楔形聚类随机临床试验，该试验比较了在接受过crp培训的提供者和未接受过培训的提供者（即TAU）的护理后，有自杀风险的服务人员的自杀念头和行为比率。参与者在预处理时和每3个月完成一次评估，直至1年。主要结果包括自杀企图和行为，自杀意念是次要结果。还将研究治疗效果的调节因子。讨论了该试验的方法学发展，以及在急诊、住院和军事治疗环境中自杀预防研究的临床和伦理考虑。结论：针对服务人员的独特需求提供基于证据的自杀治疗对于降低自杀率和促进这一人群的心理健康恢复至关重要。本研究旨在为今后在医疗保健系统中实施和传播CRP提供信息，以最终降低服务人员的自杀率。临床试验标识符：NCT05795764。

{"title":"Comparison of crisis response planning and treatment as usual for active duty service members at risk for suicide: Study protocol for a stepped-wedge cluster randomized trial in a military treatment facility","authors":"Kristen H. Walter , Pia R. Khandekar , Alexander C. Kline , Erin L. Miggantz , Nicholas P. Otis , Lisa H. Glassman , Cynthia J. Thomsen , Guy Brock , Craig J. Bryan","doi":"10.1016/j.conctc.2024.101407","DOIUrl":"10.1016/j.conctc.2024.101407","url":null,"abstract":"<div><h3>Background</h3><div>Suicide is one of the leading causes of death among U.S. service members, and rates of suicide among military personnel have increased over the past two decades. To address this serious issue, effective preventive treatments are needed in settings where at-risk service members are frequently seen, such as emergency departments and inpatient psychiatric units. This study will compare the longitudinal effects of crisis response planning (CRP) and treatment as usual (TAU) on suicidal thoughts and behaviors among active duty service members seeking emergent care for suicidality at a military treatment facility.</div></div><div><h3>Methods</h3><div>The current study is conducted through a consortium, Augmenting Suicide Prevention Interventions for Service Members. This article details an ongoing stepped-wedge cluster randomized clinical trial that compares rates of suicidal thoughts and behaviors among service members at risk for suicide following care from CRP-trained providers versus untrained providers (i.e., TAU). Participants complete assessments at pretreatment and every 3 months up to 1 year. Primary outcomes include suicide attempts and behaviors, and suicidal ideation is a secondary outcome. Moderators of treatment effects will also be examined. The methodological development of this trial is discussed, along with clinical and ethical considerations for suicide prevention research in emergency, inpatient, and military treatment settings.</div></div><div><h3>Conclusion</h3><div>Providing evidence-based treatment for suicidality that addresses service members’ unique needs is crucial to reduce suicide rates and facilitate mental health recovery in this population. This study aims to inform future implementation and dissemination of CRP in healthcare systems to ultimately decrease suicide among service members.</div></div><div><h3>Clinical trials identifier</h3><div>NCT05795764.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101407"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11681889/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142903734","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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

Contemporary Clinical Trials Communications

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。

{"title":"The effects of alpha-lipoic acid transdermal patch for local subcutaneous fat reduction: A randomized, placebo-controlled, clinical trial in overweight volunteers","authors":"Kanidta Sooklert , Sasin Thamakaison , Siwaporn Nilyai , Sarocha Cherdchom , Rojrit Rojanathanes , Amornpun Sereemaspun","doi":"10.1016/j.conctc.2024.101402","DOIUrl":"10.1016/j.conctc.2024.101402","url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Methods</h3><div>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.</div></div><div><h3>Results</h3><div>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.</div></div><div><h3>Conclusion</h3><div>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.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101402"},"PeriodicalIF":1.4,"publicationDate":"2024-11-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142721253","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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

Contemporary Clinical Trials Communications

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。

{"title":"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","authors":"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","doi":"10.1016/j.conctc.2024.101394","DOIUrl":"10.1016/j.conctc.2024.101394","url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Methods</h3><div>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.</div></div><div><h3>Aims</h3><div>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.</div></div><div><h3>Conclusions</h3><div>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.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101394"},"PeriodicalIF":1.4,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142702607","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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

Contemporary Clinical Trials Communications

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。

{"title":"Defining methods to improve eSource site start-up practices","authors":"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","doi":"10.1016/j.conctc.2024.101391","DOIUrl":"10.1016/j.conctc.2024.101391","url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Methods</h3><div>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.</div></div><div><h3>Results</h3><div>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.</div></div><div><h3>Conclusions</h3><div>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.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101391"},"PeriodicalIF":1.4,"publicationDate":"2024-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142702692","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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

Contemporary Clinical Trials Communications

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。

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

Contemporary Clinical Trials Communications

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。

{"title":"Reasons for declining participation in inpatient research among historically minoritized racial and ethnic communities: A scoping review","authors":"Poyani Bavishi , Alyssa A. Grimshaw , Oscar F. Rojas Perez , Brian D. Kiluk , E. Jennifer Edelman","doi":"10.1016/j.conctc.2024.101386","DOIUrl":"10.1016/j.conctc.2024.101386","url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Methods</h3><div>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.</div></div><div><h3>Results</h3><div>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.</div></div><div><h3>Discussion</h3><div>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.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101386"},"PeriodicalIF":1.4,"publicationDate":"2024-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142702608","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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

Contemporary Clinical Trials Communications

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。

{"title":"Modeling severe uncontrolled asthma: Transitioning away from health states","authors":"Tereza Lanitis , Asif H. Khan , Irina Proskorovsky , Ivan Houisse , Andreas Kuznik , Siddhesh Kamat , Conrado Franco-Villalobos , Florence Joulain","doi":"10.1016/j.conctc.2024.101390","DOIUrl":"10.1016/j.conctc.2024.101390","url":null,"abstract":"<div><h3>Background</h3><div>Models developed to date to simulate long-term outcomes of asthma have been criticized for lacking granularity and ignoring disease heterogeneity.</div></div><div><h3>Objective</h3><div>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.</div></div><div><h3>Methods</h3><div>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.</div></div><div><h3>Results</h3><div>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.</div></div><div><h3>Conclusion</h3><div>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.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101390"},"PeriodicalIF":1.4,"publicationDate":"2024-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142702606","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0