{"title":"肥厚型心肌病患者临床研究的患者报告结果。","authors":"Nosheen Reza, Sharlene M. Day, Anjali Tiku Owens","doi":"10.1016/j.pcad.2023.08.007","DOIUrl":null,"url":null,"abstract":"<div><h3>Objectives</h3><p>This study aimed to characterize patient-reported outcomes (PROs) in registered clinical studies of participants with hypertrophic cardiomyopathy (HCM).</p></div><div><h3>Background</h3><p>Therapy for HCM is primarily targeted toward alleviation of symptoms and improvement in function and quality of life<span>. Yet, the contemporary landscape of PROs in HCM clinical research has not been investigated.</span></p></div><div><h3>Methods</h3><p><span>ClinicalTrials.gov</span><svg><path></path></svg> was queried to identify clinical studies of HCM that reported PROs as outcome measures. All studies of HCM as the disease condition were included, and PROs were identified using specific search terms in the Outcome Measures field. Study characteristics were collected and compared between those that did versus did not report PROs.</p></div><div><h3>Results</h3><p>From November 1987 to February 2022, 181 studies including participants with HCM were registered on <span>ClinicalTrials.gov</span><svg><path></path></svg>. Of these, 35 (19%) included PROs as outcome measures. Studies reporting PROs were more likely to be designated as interventional (85.7% vs. 46.6%; <em>p</em> < 0.001) and to involve randomization (65.7% vs. 24.7%; <em>p</em> = 0.003) compared with those that did not report PROs. Prior to 2007, no clinical studies that reported PROs were registered in <span>ClinicalTrials.gov</span><svg><path></path></svg>; however, PRO reporting has increased over the last 15 years. Of the 66 PRO tools or domains included as outcome measures, the Kansas City Cardiomyopathy Questionnaire was the most often used.</p></div><div><h3>Conclusions</h3><p>Only approximately one in five registered clinical studies of participants with HCM report PROs. As medical, percutaneous, and surgical therapies for HCM continue to advance, HCM-specific PRO tools that assess the impacts of these new treatments on meaningful patient-related endpoints are urgently needed.</p></div>","PeriodicalId":21156,"journal":{"name":"Progress in cardiovascular diseases","volume":null,"pages":null},"PeriodicalIF":5.6000,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10543561/pdf/","citationCount":"0","resultStr":"{\"title\":\"Patient-reported outcomes in clinical studies of patients with hypertrophic cardiomyopathy\",\"authors\":\"Nosheen Reza, Sharlene M. Day, Anjali Tiku Owens\",\"doi\":\"10.1016/j.pcad.2023.08.007\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Objectives</h3><p>This study aimed to characterize patient-reported outcomes (PROs) in registered clinical studies of participants with hypertrophic cardiomyopathy (HCM).</p></div><div><h3>Background</h3><p>Therapy for HCM is primarily targeted toward alleviation of symptoms and improvement in function and quality of life<span>. Yet, the contemporary landscape of PROs in HCM clinical research has not been investigated.</span></p></div><div><h3>Methods</h3><p><span>ClinicalTrials.gov</span><svg><path></path></svg> was queried to identify clinical studies of HCM that reported PROs as outcome measures. All studies of HCM as the disease condition were included, and PROs were identified using specific search terms in the Outcome Measures field. Study characteristics were collected and compared between those that did versus did not report PROs.</p></div><div><h3>Results</h3><p>From November 1987 to February 2022, 181 studies including participants with HCM were registered on <span>ClinicalTrials.gov</span><svg><path></path></svg>. Of these, 35 (19%) included PROs as outcome measures. Studies reporting PROs were more likely to be designated as interventional (85.7% vs. 46.6%; <em>p</em> < 0.001) and to involve randomization (65.7% vs. 24.7%; <em>p</em> = 0.003) compared with those that did not report PROs. Prior to 2007, no clinical studies that reported PROs were registered in <span>ClinicalTrials.gov</span><svg><path></path></svg>; however, PRO reporting has increased over the last 15 years. Of the 66 PRO tools or domains included as outcome measures, the Kansas City Cardiomyopathy Questionnaire was the most often used.</p></div><div><h3>Conclusions</h3><p>Only approximately one in five registered clinical studies of participants with HCM report PROs. As medical, percutaneous, and surgical therapies for HCM continue to advance, HCM-specific PRO tools that assess the impacts of these new treatments on meaningful patient-related endpoints are urgently needed.</p></div>\",\"PeriodicalId\":21156,\"journal\":{\"name\":\"Progress in cardiovascular diseases\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":5.6000,\"publicationDate\":\"2023-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10543561/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Progress in cardiovascular diseases\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S0033062023000919\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"CARDIAC & CARDIOVASCULAR SYSTEMS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Progress in cardiovascular diseases","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0033062023000919","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CARDIAC & CARDIOVASCULAR SYSTEMS","Score":null,"Total":0}
Patient-reported outcomes in clinical studies of patients with hypertrophic cardiomyopathy
Objectives
This study aimed to characterize patient-reported outcomes (PROs) in registered clinical studies of participants with hypertrophic cardiomyopathy (HCM).
Background
Therapy for HCM is primarily targeted toward alleviation of symptoms and improvement in function and quality of life. Yet, the contemporary landscape of PROs in HCM clinical research has not been investigated.
Methods
ClinicalTrials.gov was queried to identify clinical studies of HCM that reported PROs as outcome measures. All studies of HCM as the disease condition were included, and PROs were identified using specific search terms in the Outcome Measures field. Study characteristics were collected and compared between those that did versus did not report PROs.
Results
From November 1987 to February 2022, 181 studies including participants with HCM were registered on ClinicalTrials.gov. Of these, 35 (19%) included PROs as outcome measures. Studies reporting PROs were more likely to be designated as interventional (85.7% vs. 46.6%; p < 0.001) and to involve randomization (65.7% vs. 24.7%; p = 0.003) compared with those that did not report PROs. Prior to 2007, no clinical studies that reported PROs were registered in ClinicalTrials.gov; however, PRO reporting has increased over the last 15 years. Of the 66 PRO tools or domains included as outcome measures, the Kansas City Cardiomyopathy Questionnaire was the most often used.
Conclusions
Only approximately one in five registered clinical studies of participants with HCM report PROs. As medical, percutaneous, and surgical therapies for HCM continue to advance, HCM-specific PRO tools that assess the impacts of these new treatments on meaningful patient-related endpoints are urgently needed.
期刊介绍:
Progress in Cardiovascular Diseases provides comprehensive coverage of a single topic related to heart and circulatory disorders in each issue. Some issues include special articles, definitive reviews that capture the state of the art in the management of particular clinical problems in cardiology.