Pub Date : 2022-06-27eCollection Date: 2022-01-01DOI: 10.2147/PROM.S353895
Eva Zwaan, Elena Cheung, Alexander IJsselmuiden, Carlo Holtzer, Ton Schreuders, Marcel Kofflard, Marco Alings, J Henk Coert
Purpose: The use of the Disability of the Arm, Shoulder and Hand (DASH) questionnaire and its shortened version, the QuickDASH, have been used to assess upper extremity function following a transradial percutaneous coronary intervention (TR-PCI). However, the use of these scores has not yet been validated for TR-PCI induced complications in the upper extremity. The aim of this study was to establish the validity of the DASH and the QuickDASH, for the assessment of upper extremity dysfunction following a transradial percutaneous coronary intervention (TR-PCI).
Patients and methods: This study was a diagnostic retrospective analysis of the ARCUS study, of whom 440 underwent TR-PCI and 62 control patients were treated via the transfemoral approach. All participants completed the DASH and QuickDASH questionnaire prior to the procedure and at each follow-up visit up to six months of follow-up. Receiver operating characteristics (ROC) were constructed to determine the validity of the questionnaires, using physical examinations to determine the occurrence of upper extremity dysfunction, according to the ARCUS study.
Results: At each follow-up moment, the area under the curve (AUC) showed a poor ability of the DASH and QuickDASH to discriminate between patients with and without upper extremity dysfunction (AUC: 0.565-0.586). There was no significant difference between the questionnaires (p > 0.05).
Conclusion: The DASH and QuickDASH questionnaires are both equally incapable of discriminating between patients with and without upper extremity dysfunction following a TR-PCI. Study results suggest that the DASH and QuickDASH questionnaires are incapable of discerning changes in upper extremity function as a result of procedural complications following a TR-PCI vs cardiac induced activity cessation.
{"title":"Predictive Value of the <i>(Quick)</i>DASH Tool for Upper Extremity Dysfunction Following Percutaneous Coronary Intervention.","authors":"Eva Zwaan, Elena Cheung, Alexander IJsselmuiden, Carlo Holtzer, Ton Schreuders, Marcel Kofflard, Marco Alings, J Henk Coert","doi":"10.2147/PROM.S353895","DOIUrl":"https://doi.org/10.2147/PROM.S353895","url":null,"abstract":"<p><strong>Purpose: </strong>The use of the Disability of the Arm, Shoulder and Hand (DASH) questionnaire and its shortened version, the <i>Quick</i>DASH, have been used to assess upper extremity function following a transradial percutaneous coronary intervention (TR-PCI). However, the use of these scores has not yet been validated for TR-PCI induced complications in the upper extremity. The aim of this study was to establish the validity of the DASH and the <i>Quick</i>DASH, for the assessment of upper extremity dysfunction following a transradial percutaneous coronary intervention (TR-PCI).</p><p><strong>Patients and methods: </strong>This study was a diagnostic retrospective analysis of the ARCUS study, of whom 440 underwent TR-PCI and 62 control patients were treated via the transfemoral approach. All participants completed the DASH and <i>Quick</i>DASH questionnaire prior to the procedure and at each follow-up visit up to six months of follow-up. Receiver operating characteristics (ROC) were constructed to determine the validity of the questionnaires, using physical examinations to determine the occurrence of upper extremity dysfunction, according to the ARCUS study.</p><p><strong>Results: </strong>At each follow-up moment, the area under the curve (AUC) showed a poor ability of the DASH and <i>Quick</i>DASH to discriminate between patients with and without upper extremity dysfunction (AUC: 0.565-0.586). There was no significant difference between the questionnaires (p > 0.05).</p><p><strong>Conclusion: </strong>The DASH and <i>Quick</i>DASH questionnaires are both equally incapable of discriminating between patients with and without upper extremity dysfunction following a TR-PCI. Study results suggest that the DASH and <i>Quick</i>DASH questionnaires are incapable of discerning changes in upper extremity function as a result of procedural complications following a TR-PCI vs cardiac induced activity cessation.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-06-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/06/83/prom-13-145.PMC9249092.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40559418","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}
Oral health problems are associated with poor quality of life, with the potential to cause functional, aesthetic, nutritional, and psychological difficulties, in addition to pain and suffering. Traditionally, dental treatment outcomes are measured using purely clinical parameters; however, this may be ineffective as these parameters cannot adequately capture the full impact of poor oral health on the patient, or their respective coping strategies. From this perspective, there are significant benefits when the patient's perception of their care is considered, and included in treatment planning and delivery. The impacts perceived by the patient on their treatment outcomes can be measured using patient-reported outcomes (PROS), or more specifically with dPROS, focused on dental patient-reported outcomes. Although there are some instruments available for measuring these outcomes in clinical trials, very little information is available for explaining the context in which these outcomes are considered, and also how to capture this information using appropriate instruments, specially in evidence-based dental practice. This article aims to review the literature, seeking to describe what has been considered about assessing patient's outcomes, as well as how to measure them, and explore the potential benefits of using dPROS in evidence-based prosthodontics and clinical care of partially and fully edentulous patients.
{"title":"The Potential Role of Dental Patient-Reported Outcomes (dPROs) in Evidence-Based Prosthodontics and Clinical Care: A Narrative Review.","authors":"Cláudio Rodrigues Leles, Jésio Rodrigues Silva, Thalita Fernandes Fleury Curado, Martin Schimmel, Gerald McKenna","doi":"10.2147/PROM.S256724","DOIUrl":"10.2147/PROM.S256724","url":null,"abstract":"<p><p>Oral health problems are associated with poor quality of life, with the potential to cause functional, aesthetic, nutritional, and psychological difficulties, in addition to pain and suffering. Traditionally, dental treatment outcomes are measured using purely clinical parameters; however, this may be ineffective as these parameters cannot adequately capture the full impact of poor oral health on the patient, or their respective coping strategies. From this perspective, there are significant benefits when the patient's perception of their care is considered, and included in treatment planning and delivery. The impacts perceived by the patient on their treatment outcomes can be measured using patient-reported outcomes (PROS), or more specifically with dPROS, focused on dental patient-reported outcomes. Although there are some instruments available for measuring these outcomes in clinical trials, very little information is available for explaining the context in which these outcomes are considered, and also how to capture this information using appropriate instruments, specially in evidence-based dental practice. This article aims to review the literature, seeking to describe what has been considered about assessing patient's outcomes, as well as how to measure them, and explore the potential benefits of using dPROS in evidence-based prosthodontics and clinical care of partially and fully edentulous patients.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-06-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9172924/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46972083","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}
Pub Date : 2022-05-30eCollection Date: 2022-01-01DOI: 10.2147/PROM.S297699
Nitika Sanger, Balpreet Panesar, Michael Dennis, Tea Rosic, Myanca Rodrigues, Elizabeth Lovell, Shuling Yang, Mehreen Butt, Lehana Thabane, Zainab Samaan
Introduction: Patient centred care is needed now more than ever in the treatment of opioid use disorder. Trials, policy makers, and service providers have most often used treatment retention and opioid urine screens as measures of treatment effectiveness. However, patients receiving medication for opioid use disorder treatment (MOUD) may prioritise the use of different ways to assess treatment success.
Objective: The aim of this review is to synthesize literature examining the self-reported goals patients would like to achieve in MOUD for opioid use disorder.
Methods: We searched MEDLINE, EMBASE, PsycINFO, Cumulative Index to Nursing and Allied Health Literature, Web of Science, Cochrane Library, Cochrane Clinical Trials Registry, the National Institutes for Health Clinical Trials Registry, and the WHO International Clinical Trials Registry Platform from inception until April 30th, 2021. No restrictions were placed on language, age, or type of MOUD. A qualitative synthesis is presented given that a meta-analysis was not possible.
Results: The search yielded a total of 21,082 records from which 8 met criteria for inclusion in the qualitative synthesis. We identified a total of 43 patient-reported treatment goals from the 8 studies. Twelve domains were created from the 43 goals reported. These domains cover a range of important areas for patients' goals related to living a normal life, physical health, mental health, treatment, and substance use specific areas.
Conclusion: This review highlights several patient goals that they would like to achieve during treatment for opioid use disorder that are not commonly considered as markers of treatment effectiveness. Goals related to health, living a normal life, and overall substance use concerns by patients should be taken into consideration by clinical trialists, researchers, policy makers, service providers, patients, and communities engaged in developing and tailoring treatment plans for opioid use disorder.
引言在治疗阿片类药物使用障碍方面,现在比以往任何时候都更需要以患者为中心的护理。试验、政策制定者和服务提供者最常使用治疗保留和阿片类药物尿液筛查作为治疗效果的衡量标准。然而,接受阿片类药物使用障碍治疗的患者可能会优先使用不同的方法来评估治疗成功率。目的本综述的目的是综合文献,研究患者在阿片类药物使用障碍的MOUD中希望实现的自我报告目标。方法从成立到2021年4月30日,我们搜索了MEDLINE、EMBASE、PsycINFO、护理和相关健康文献累积指数、Web of Science、Cochrane图书馆、Cochran临床试验注册中心、美国国立卫生研究院临床试验注册处和世界卫生组织国际临床试验注册平台。对语言、年龄或MOUD类型没有任何限制。在不可能进行荟萃分析的情况下,进行了定性综合。结果检索共产生21082份记录,其中8份符合纳入定性综合的标准。我们从8项研究中确定了总共43名患者报告的治疗目标。根据报告的43个目标创建了12个领域。这些领域涵盖了患者目标的一系列重要领域,涉及正常生活、身体健康、心理健康、治疗和药物使用特定领域。结论这篇综述强调了他们在阿片类药物使用障碍治疗期间希望实现的几个患者目标,这些目标通常不被认为是治疗效果的标志。临床试验人员、研究人员、政策制定者、服务提供商、患者和参与制定和定制阿片类药物使用障碍治疗计划的社区应考虑到与健康、正常生活和患者总体药物使用相关的目标。系统审查注册PROSPERO CRD42018095553。
{"title":"The Inclusion of Patients' Reported Outcomes to Inform Treatment Effectiveness Measures in Opioid Use Disorder. A Systematic Review.","authors":"Nitika Sanger, Balpreet Panesar, Michael Dennis, Tea Rosic, Myanca Rodrigues, Elizabeth Lovell, Shuling Yang, Mehreen Butt, Lehana Thabane, Zainab Samaan","doi":"10.2147/PROM.S297699","DOIUrl":"10.2147/PROM.S297699","url":null,"abstract":"<p><strong>Introduction: </strong>Patient centred care is needed now more than ever in the treatment of opioid use disorder. Trials, policy makers, and service providers have most often used treatment retention and opioid urine screens as measures of treatment effectiveness. However, patients receiving medication for opioid use disorder treatment (MOUD) may prioritise the use of different ways to assess treatment success.</p><p><strong>Objective: </strong>The aim of this review is to synthesize literature examining the self-reported goals patients would like to achieve in MOUD for opioid use disorder.</p><p><strong>Methods: </strong>We searched MEDLINE, EMBASE, PsycINFO, Cumulative Index to Nursing and Allied Health Literature, Web of Science, Cochrane Library, Cochrane Clinical Trials Registry, the National Institutes for Health Clinical Trials Registry, and the WHO International Clinical Trials Registry Platform from inception until April 30th, 2021. No restrictions were placed on language, age, or type of MOUD. A qualitative synthesis is presented given that a meta-analysis was not possible.</p><p><strong>Results: </strong>The search yielded a total of 21,082 records from which 8 met criteria for inclusion in the qualitative synthesis. We identified a total of 43 patient-reported treatment goals from the 8 studies. Twelve domains were created from the 43 goals reported. These domains cover a range of important areas for patients' goals related to living a normal life, physical health, mental health, treatment, and substance use specific areas.</p><p><strong>Conclusion: </strong>This review highlights several patient goals that they would like to achieve during treatment for opioid use disorder that are not commonly considered as markers of treatment effectiveness. Goals related to health, living a normal life, and overall substance use concerns by patients should be taken into consideration by clinical trialists, researchers, policy makers, service providers, patients, and communities engaged in developing and tailoring treatment plans for opioid use disorder.</p><p><strong>Systematic review registration: </strong>PROSPERO CRD42018095553.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-05-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9165704/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49280323","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}
Introduction Treatment-related attributes and process characteristics such as dosing frequency, timing flexibility, ease of use of injection devices and unpleasant side-effects may have small but measurable effects on quality of life (QoL) in people with type 2 diabetes (T2D). A literature review was performed to identify recently published utility values quantifying the effect of treatment-related attributes on QoL. Methods Literature search strategies were designed using high-level medical subject heading (MeSH) terms supplemented with free-text terms and searches were run in March 2020 in the PubMed, Embase and Cochrane Library databases. For inclusion, studies were required to be published in full-text form, in English, since 2010 and report utility values (elicited using either direct or indirect methods) for treatment-related attributes or process characteristics including side effects, change in weight/body mass index (BMI), dosing frequency and timing flexibility, device attributes (e.g. needle handling, requirement for reconstitution) and convenience (e.g. waiting time). Results A total of 30 studies were included in the review, of which all but three were conducted in people with T2D. The EQ-5D was the most commonly used elicitation method (fourteen studies), followed by time tradeoff (TTO) methodology. Treatment-related adverse events and inconveniences such as needle handling in administration devices and waiting time were consistently associated with lower QoL, whereas lower dosing frequency and increased timing flexibility with dosing were consistently associated with utility benefits. The relationship between change in BMI and QoL was non-linear and influenced by baseline BMI. Conclusion Treatment-related attributes and process characteristics are associated with minor changes in QoL, which should be taken into account in long-term health economic modeling of new treatments and administration devices.
{"title":"Evaluating the Impact of Therapy on Quality of Life in Type 2 Diabetes: A Literature Review of Utilities Associated with Treatment-Related Attributes","authors":"W. Valentine, K. Norrbacka, K. Boye","doi":"10.2147/PROM.S322390","DOIUrl":"https://doi.org/10.2147/PROM.S322390","url":null,"abstract":"Introduction Treatment-related attributes and process characteristics such as dosing frequency, timing flexibility, ease of use of injection devices and unpleasant side-effects may have small but measurable effects on quality of life (QoL) in people with type 2 diabetes (T2D). A literature review was performed to identify recently published utility values quantifying the effect of treatment-related attributes on QoL. Methods Literature search strategies were designed using high-level medical subject heading (MeSH) terms supplemented with free-text terms and searches were run in March 2020 in the PubMed, Embase and Cochrane Library databases. For inclusion, studies were required to be published in full-text form, in English, since 2010 and report utility values (elicited using either direct or indirect methods) for treatment-related attributes or process characteristics including side effects, change in weight/body mass index (BMI), dosing frequency and timing flexibility, device attributes (e.g. needle handling, requirement for reconstitution) and convenience (e.g. waiting time). Results A total of 30 studies were included in the review, of which all but three were conducted in people with T2D. The EQ-5D was the most commonly used elicitation method (fourteen studies), followed by time tradeoff (TTO) methodology. Treatment-related adverse events and inconveniences such as needle handling in administration devices and waiting time were consistently associated with lower QoL, whereas lower dosing frequency and increased timing flexibility with dosing were consistently associated with utility benefits. The relationship between change in BMI and QoL was non-linear and influenced by baseline BMI. Conclusion Treatment-related attributes and process characteristics are associated with minor changes in QoL, which should be taken into account in long-term health economic modeling of new treatments and administration devices.","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45521946","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-03-30eCollection Date: 2022-01-01DOI: 10.2147/PROM.S256715
Gabriel Horta-Baas
Purpose: This review aims to provide an overview of the impact of TNFis biosimilars, with marketing authorization, in patient-reported outcome measures (PROMs) scores and explore how PROMs endpoints might add value in biosimilars uptake in RA patients.
Patients and methods: A comprehensive search of Medline, Scopus, Lilacs, and CINAHL databases was performed for papers published between January 2012 and December 2021. For inclusion, studies had to be prospective, published in a peer-reviewed journal, published in English or Spanish language; studies using PROMs as an outcome measure. After screening title and abstracts and assessing the remaining full texts fulfilling the inclusion criteria, 31 papers were used in this narrative review.
Results: PROMs were used as secondary outcomes in included studies. The most frequently employed domains to assess biosimilar efficacy include physical function, patient global assessment (PtGA), health-related quality of life (HRQoL), and fatigue. The results of randomized clinical trials uniformly showed that mean change in PROMs scores is comparable between biosimilar and reference biologic treatment groups. However, open-label and real-world studies revealed high rates of discontinuation of therapy, mainly for subjective worsening of disease activity or non-specific adverse events. Even without objective clinical evidence of inflammation, patients who are considered to have active disease (higher scores on PtGA) have higher discontinuation rates of biosimilars. The available information suggests that the nocebo effect is the most likely cause for the discontinuation of biosimilars.
Conclusion: There is scarce literature surrounding the impact of biosimilars in PROMs, especially in open-label studies. In real-life studies, biosimilars have a higher discontinuation rate than reference products. TNFis biosimilars treatment efficacy in RA depends on disease activity and other factors such as PtGA and fatigue. The nocebo effect is the best explanation for biosimilar's discontinuation.
{"title":"Patient-Reported Outcomes in Rheumatoid Arthritis: A Key Consideration for Evaluating Biosimilar Uptake?","authors":"Gabriel Horta-Baas","doi":"10.2147/PROM.S256715","DOIUrl":"10.2147/PROM.S256715","url":null,"abstract":"<p><strong>Purpose: </strong>This review aims to provide an overview of the impact of TNFis biosimilars, with marketing authorization, in patient-reported outcome measures (PROMs) scores and explore how PROMs endpoints might add value in biosimilars uptake in RA patients.</p><p><strong>Patients and methods: </strong>A comprehensive search of Medline, Scopus, Lilacs, and CINAHL databases was performed for papers published between January 2012 and December 2021. For inclusion, studies had to be prospective, published in a peer-reviewed journal, published in English or Spanish language; studies using PROMs as an outcome measure. After screening title and abstracts and assessing the remaining full texts fulfilling the inclusion criteria, 31 papers were used in this narrative review.</p><p><strong>Results: </strong>PROMs were used as secondary outcomes in included studies. The most frequently employed domains to assess biosimilar efficacy include physical function, patient global assessment (PtGA), health-related quality of life (HRQoL), and fatigue. The results of randomized clinical trials uniformly showed that mean change in PROMs scores is comparable between biosimilar and reference biologic treatment groups. However, open-label and real-world studies revealed high rates of discontinuation of therapy, mainly for subjective worsening of disease activity or non-specific adverse events. Even without objective clinical evidence of inflammation, patients who are considered to have active disease (higher scores on PtGA) have higher discontinuation rates of biosimilars. The available information suggests that the nocebo effect is the most likely cause for the discontinuation of biosimilars.</p><p><strong>Conclusion: </strong>There is scarce literature surrounding the impact of biosimilars in PROMs, especially in open-label studies. In real-life studies, biosimilars have a higher discontinuation rate than reference products. TNFis biosimilars treatment efficacy in RA depends on disease activity and other factors such as PtGA and fatigue. The nocebo effect is the best explanation for biosimilar's discontinuation.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2022-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8977480/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47718087","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}
Abstract Two general classes of concepts measured by patient-reported outcome measures (PROMs) are abilities and feelings. Over the past several decades, there has been a significant progress in measuring both. Nevertheless, current multi-item scales are subject to criticism related to scale length, score dimensionality, interpretability, cultural bias, and insufficient detail in measuring specific domains. To address some of these issues, the author offers an alternative perspective on how questions about abilities and feelings could be formulated. Abilities can be defined in terms of a relationship between the level of performance and the associated perception of difficulty, and represented graphically by an ability curve. For feelings, it may be useful to measure frequency and intensity jointly to determine the proportion of time in each level of intensity. The resultant frequency × intensity matrix can be presented as a bar graph. Empirical data to support the feasibility and validity of these approaches to PROM design are provided, potential advantages and limitations are discussed, and some future research avenues are suggested.
{"title":"How to Improve Interpretability of Patient-Reported Outcome Measures for Clinical Use: A Perspective on Measuring Abilities and Feelings","authors":"J. Kopec","doi":"10.2147/PROM.S355679","DOIUrl":"https://doi.org/10.2147/PROM.S355679","url":null,"abstract":"Abstract Two general classes of concepts measured by patient-reported outcome measures (PROMs) are abilities and feelings. Over the past several decades, there has been a significant progress in measuring both. Nevertheless, current multi-item scales are subject to criticism related to scale length, score dimensionality, interpretability, cultural bias, and insufficient detail in measuring specific domains. To address some of these issues, the author offers an alternative perspective on how questions about abilities and feelings could be formulated. Abilities can be defined in terms of a relationship between the level of performance and the associated perception of difficulty, and represented graphically by an ability curve. For feelings, it may be useful to measure frequency and intensity jointly to determine the proportion of time in each level of intensity. The resultant frequency × intensity matrix can be presented as a bar graph. Empirical data to support the feasibility and validity of these approaches to PROM design are provided, potential advantages and limitations are discussed, and some future research avenues are suggested.","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43028294","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-02-09eCollection Date: 2022-01-01DOI: 10.2147/PROM.S343302
Brian D Moseley, Shaloo Gupta, Nate Way, Jonathon Wright, John C Rowland, Victoria E Barghout, Feride Frech, Craig Plauschinat
Background: Epilepsy is a complex disorder that can affect patients' medical, psychological, and social well-being. The purpose of this study was to evaluate the patient-reported outcome (PRO) measures of health-related quality of life (HRQoL), satisfaction, and adherence in adult patients diagnosed with epilepsy treated with perampanel in the United States (US).
Methods: A US-based, multicenter, observational cross-sectional survey was completed by 61 patients taking perampanel with or without other antiseizure medications (ASMs). Respondents were ≥18 years old, had a physician-confirmed diagnosis of epilepsy, used perampanel for ≥4 months, and provided informed consent. Patients responded to questions concerning their demographic characteristics, treatment history, experiences before perampanel, experiences while taking perampanel, HRQoL, treatment satisfaction, and medication adherence.
Results: Patients (N=61) were 42.8 years old on average; majority were female (63.9%) and white (75.4%). Mean time on perampanel was 2.5 years, with sodium channel blockers often (55.7%) used concomitantly with perampanel. Patients reported, on average, 5.5 (standard deviation [SD]=13.2) seizures/month after initiating perampanel, whereas these same patients reported experiencing 20.4 (SD=60.0) seizures/month prior to perampanel. When comparing their experience on perampanel with their experience with previous ASMs, more patients "strongly agreed" that perampanel allowed them to live a more normal life (36.1% vs 27.5%) and worked as intended if they missed taking a dose (16.4% vs 7.8%). Average satisfaction scores were high, with ratings of 71.8 for effectiveness, 84.0 for convenience, and 71.9 for global satisfaction (0-100 scores). Perampanel use was associated with improvements in HRQoL and fewer symptoms of depression and anxiety. The majority of patients were adherent (62.3%) to perampanel.
Discussion: Perampanel use was associated with reductions in number of seizures, better HRQoL, and high adherence rates. These results provide initial evidence that perampanel can be an effective, tolerable, and valid option for patients with epilepsy in the real world.
{"title":"Patient-Reported Outcome Measures in Adult Patients Diagnosed with Epilepsy Being Treated with Perampanel.","authors":"Brian D Moseley, Shaloo Gupta, Nate Way, Jonathon Wright, John C Rowland, Victoria E Barghout, Feride Frech, Craig Plauschinat","doi":"10.2147/PROM.S343302","DOIUrl":"https://doi.org/10.2147/PROM.S343302","url":null,"abstract":"<p><strong>Background: </strong>Epilepsy is a complex disorder that can affect patients' medical, psychological, and social well-being. The purpose of this study was to evaluate the patient-reported outcome (PRO) measures of health-related quality of life (HRQoL), satisfaction, and adherence in adult patients diagnosed with epilepsy treated with perampanel in the United States (US).</p><p><strong>Methods: </strong>A US-based, multicenter, observational cross-sectional survey was completed by 61 patients taking perampanel with or without other antiseizure medications (ASMs). Respondents were ≥18 years old, had a physician-confirmed diagnosis of epilepsy, used perampanel for ≥4 months, and provided informed consent. Patients responded to questions concerning their demographic characteristics, treatment history, experiences before perampanel, experiences while taking perampanel, HRQoL, treatment satisfaction, and medication adherence.</p><p><strong>Results: </strong>Patients (N=61) were 42.8 years old on average; majority were female (63.9%) and white (75.4%). Mean time on perampanel was 2.5 years, with sodium channel blockers often (55.7%) used concomitantly with perampanel. Patients reported, on average, 5.5 (standard deviation [SD]=13.2) seizures/month after initiating perampanel, whereas these same patients reported experiencing 20.4 (SD=60.0) seizures/month prior to perampanel. When comparing their experience on perampanel with their experience with previous ASMs, more patients \"strongly agreed\" that perampanel allowed them to live a more normal life (36.1% vs 27.5%) and worked as intended if they missed taking a dose (16.4% vs 7.8%). Average satisfaction scores were high, with ratings of 71.8 for effectiveness, 84.0 for convenience, and 71.9 for global satisfaction (0-100 scores). Perampanel use was associated with improvements in HRQoL and fewer symptoms of depression and anxiety. The majority of patients were adherent (62.3%) to perampanel.</p><p><strong>Discussion: </strong>Perampanel use was associated with reductions in number of seizures, better HRQoL, and high adherence rates. These results provide initial evidence that perampanel can be an effective, tolerable, and valid option for patients with epilepsy in the real world.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/fa/6d/prom-13-39.PMC8841652.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39638048","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}
Pub Date : 2022-02-04eCollection Date: 2022-01-01DOI: 10.2147/PROM.S346434
John Soper, Islam Sadek, Alyson Urniasz-Lippel, Deborah Norton, Marina Ness, Ruben Mesa
A diagnosis of myelodysplastic syndrome (MDS) is typically unexpected and can be difficult for patients to grasp. Not only is MDS a complicated disease to understand, which can contribute to stress and anxiety, but it also has an uncertain prognosis, which can be emotionally paralyzing. Not surprisingly, emotional distress and the symptom burden of MDS, including extreme fatigue due to cytopenias, negatively impact a patient's quality of life (QOL). Studies have shown that patient-centered care-including greater physician understanding of the disease burden their patients experience, discussing and establishing agreed-on treatment goals, and including patients in the decision-making process about their care-may help improve patient QOL. To better understand patient and caregiver experiences with MDS and how the disease impacts QOL, a small survey was conducted of patients with MDS or leukemia and their caregivers on an online health network. Among the 30 respondents who completed the survey, four had MDS and one was a caregiver for a patient with MDS. Here we focus on the five MDS respondents and contextualize the findings with personal experiences from a patient and physician perspective. The patient perspective was provided by John Soper, PhD, DABCC, who was diagnosed with MDS in 2019. Dr Soper is a retired board-certified clinical chemist and a member of the MDS Foundation. The physician perspective was provided by Dr Ruben Mesa, Executive Director of the Mays Cancer Center at UT Health San Antonio MD Anderson. The survey responses and the accompanying patient and physician perspectives highlight the importance of open communication between patients and their healthcare provider to better serve those with MDS and improve their QOL.
{"title":"Patient and Caregiver Insights into the Disease Burden of Myelodysplastic Syndrome.","authors":"John Soper, Islam Sadek, Alyson Urniasz-Lippel, Deborah Norton, Marina Ness, Ruben Mesa","doi":"10.2147/PROM.S346434","DOIUrl":"https://doi.org/10.2147/PROM.S346434","url":null,"abstract":"<p><p>A diagnosis of myelodysplastic syndrome (MDS) is typically unexpected and can be difficult for patients to grasp. Not only is MDS a complicated disease to understand, which can contribute to stress and anxiety, but it also has an uncertain prognosis, which can be emotionally paralyzing. Not surprisingly, emotional distress and the symptom burden of MDS, including extreme fatigue due to cytopenias, negatively impact a patient's quality of life (QOL). Studies have shown that patient-centered care-including greater physician understanding of the disease burden their patients experience, discussing and establishing agreed-on treatment goals, and including patients in the decision-making process about their care-may help improve patient QOL. To better understand patient and caregiver experiences with MDS and how the disease impacts QOL, a small survey was conducted of patients with MDS or leukemia and their caregivers on an online health network. Among the 30 respondents who completed the survey, four had MDS and one was a caregiver for a patient with MDS. Here we focus on the five MDS respondents and contextualize the findings with personal experiences from a patient and physician perspective. The patient perspective was provided by John Soper, PhD, DABCC, who was diagnosed with MDS in 2019. Dr Soper is a retired board-certified clinical chemist and a member of the MDS Foundation. The physician perspective was provided by Dr Ruben Mesa, Executive Director of the Mays Cancer Center at UT Health San Antonio MD Anderson. The survey responses and the accompanying patient and physician perspectives highlight the importance of open communication between patients and their healthcare provider to better serve those with MDS and improve their QOL.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/54/df/prom-13-31.PMC8824781.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39620378","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}
Luis Alves, R. Pullen, J. Hurst, M. Miravitlles, V. Carter, Rongchang Chen, A. Couper, M. Dransfield, Alexander Evans, A. Hardjojo, David Jones, Rupert C Jones, Margee Kerr, K. Kostikas, J. Marshall, F. Martinez, M. V. van Melle, R. Murray, S. Muro, C. Nordon, M. Pollack, C. Price, Anita Sharma, D. Singh, T. Winders, D. Price
: The COllaboration on QUality improvement initiative for achieving Excellence in STandards of COPD care (CONQUEST) aims to improve the quality of COPD care in primary care. The CONQUEST target population includes patients diagnosed with COPD, and those undiagnosed but with COPD-like exacerbations, who are at high but modifiable risk of COPD exacerbations, increased morbidity, and mortality. Timely diagnosis and optimized management to reduce these risks is vital. There is a need for a quality improvement program (QIP) that enables long-term improvement of patient clinical outcomes via integration of the program into routine clinical care. Core to the CONQUEST program is the adoption of four specifically designed, globally applicable, and expert-agreed quality standards (QS) for modifiable high-risk COPD patients. Translation of the QS into clinical practice, and implementation of the QIP, is guided by the CONQUEST global operational protocol provided to sites meeting the minimum healthcare system requirements. Initial analyses of current practices are conducted to generate baseline assessments of need within healthcare systems and sites looking to implement the QIP. Implementation is supported by the provision of CONQUEST resources and tools, such as clinical decision support, that promote prompt identification and treatment of patients. Utilization of electronic medical record (EMR) and patient-reported data are integral components of the QIP. Regular, automated collection and analysis of data, combined with a cyclical review of the implementation process, will be conducted for long-term, continuous improvement and health impact evaluation. The CONQUEST QIP will be an important resource in the identification and management of patients with modifiable high-risk COPD. Embedding the CONQUEST QS into routine clinical practice with regular evaluation and feedback will result in long-term quality of care improvement.
{"title":"CONQUEST: A Quality Improvement Program for Defining and Optimizing Standards of Care for Modifiable High-Risk COPD Patients","authors":"Luis Alves, R. Pullen, J. Hurst, M. Miravitlles, V. Carter, Rongchang Chen, A. Couper, M. Dransfield, Alexander Evans, A. Hardjojo, David Jones, Rupert C Jones, Margee Kerr, K. Kostikas, J. Marshall, F. Martinez, M. V. van Melle, R. Murray, S. Muro, C. Nordon, M. Pollack, C. Price, Anita Sharma, D. Singh, T. Winders, D. Price","doi":"10.2147/prom.s296506","DOIUrl":"https://doi.org/10.2147/prom.s296506","url":null,"abstract":": The COllaboration on QUality improvement initiative for achieving Excellence in STandards of COPD care (CONQUEST) aims to improve the quality of COPD care in primary care. The CONQUEST target population includes patients diagnosed with COPD, and those undiagnosed but with COPD-like exacerbations, who are at high but modifiable risk of COPD exacerbations, increased morbidity, and mortality. Timely diagnosis and optimized management to reduce these risks is vital. There is a need for a quality improvement program (QIP) that enables long-term improvement of patient clinical outcomes via integration of the program into routine clinical care. Core to the CONQUEST program is the adoption of four specifically designed, globally applicable, and expert-agreed quality standards (QS) for modifiable high-risk COPD patients. Translation of the QS into clinical practice, and implementation of the QIP, is guided by the CONQUEST global operational protocol provided to sites meeting the minimum healthcare system requirements. Initial analyses of current practices are conducted to generate baseline assessments of need within healthcare systems and sites looking to implement the QIP. Implementation is supported by the provision of CONQUEST resources and tools, such as clinical decision support, that promote prompt identification and treatment of patients. Utilization of electronic medical record (EMR) and patient-reported data are integral components of the QIP. Regular, automated collection and analysis of data, combined with a cyclical review of the implementation process, will be conducted for long-term, continuous improvement and health impact evaluation. The CONQUEST QIP will be an important resource in the identification and management of patients with modifiable high-risk COPD. Embedding the CONQUEST QS into routine clinical practice with regular evaluation and feedback will result in long-term quality of care improvement.","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49450715","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2022-01-25eCollection Date: 2022-01-01DOI: 10.2147/PROM.S332519
Adam B Smith, Andria Hanbury, Jennifer A Whitty, Igor Beitia Ortiz de Zarate, Florence Hammes, Gérard de Pouvourville, Katharina Buesch
Purpose: Cost-effectiveness evaluations of interventions require health utility data. However, in medical conditions, such as aromatic L-amino acid decarboxylase (AADC) deficiency, this presents problems due to the rarity of the disease. The study aim therefore was to employ a discrete choice experiment (DCE) to generate health utilities for AADC deficiency.
Methods: A previous literature review, clinician and parent interviews had identified six key AADC deficiency attributes: mobility, muscle weakness, oculogyric crises (OCG), feeding ability, cognitive impairment and screaming. A representative sample of the French general population was recruited. Participants rated 5 health state vignettes describing AADC deficiency using time-trade-off (TTO) and standard gamble (SG). Additionally, participants rated the worst health state using the Health Utility Index version 3 (HUI3). Subsequently, participants completed DCE 11 choice sets. Indirect DCE part-worth utilities were converted to health utilities using the anchors from the TTO, SG and HUI3.
Results: The DCE was completed online by 1001 participants (50.9% female; mean age 45.7 years). Most participants (596, 59.5%) provided consistent responses to the repeated choice task. Five models were evaluated, and one preference reversal ("head control"/"sitting unaided") was identified in all models. The rescaled utilities ranged from 0.3891 to 0.5577 (difference of 0.17 utilities) for TTO anchors corresponding to the worst (633233) and best (111111) health states. Health utilities ranged from 0.5534 to 0.7093 for the SG anchors. The disutility associated with a transition from "no problems walking" to "bedridden" was -0.0533, whereas disutility of moving from "constant screaming" relative to "no screaming" was -0.0248. The disutility associated with daily OCG was -0.0167. Disutilities for the other attributes were small although there were exceptions.
Conclusion: A DCE was used to derive health utilities for AADC deficiency. These health utilities will subsequently be used in an economic model evaluating an AADC deficiency intervention.
{"title":"A Discrete Choice Experiment to Derive Health Utilities for Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency in France.","authors":"Adam B Smith, Andria Hanbury, Jennifer A Whitty, Igor Beitia Ortiz de Zarate, Florence Hammes, Gérard de Pouvourville, Katharina Buesch","doi":"10.2147/PROM.S332519","DOIUrl":"https://doi.org/10.2147/PROM.S332519","url":null,"abstract":"<p><strong>Purpose: </strong>Cost-effectiveness evaluations of interventions require health utility data. However, in medical conditions, such as aromatic L-amino acid decarboxylase (AADC) deficiency, this presents problems due to the rarity of the disease. The study aim therefore was to employ a discrete choice experiment (DCE) to generate health utilities for AADC deficiency.</p><p><strong>Methods: </strong>A previous literature review, clinician and parent interviews had identified six key AADC deficiency attributes: mobility, muscle weakness, oculogyric crises (OCG), feeding ability, cognitive impairment and screaming. A representative sample of the French general population was recruited. Participants rated 5 health state vignettes describing AADC deficiency using time-trade-off (TTO) and standard gamble (SG). Additionally, participants rated the worst health state using the Health Utility Index version 3 (HUI3). Subsequently, participants completed DCE 11 choice sets. Indirect DCE part-worth utilities were converted to health utilities using the anchors from the TTO, SG and HUI3.</p><p><strong>Results: </strong>The DCE was completed online by 1001 participants (50.9% female; mean age 45.7 years). Most participants (596, 59.5%) provided consistent responses to the repeated choice task. Five models were evaluated, and one preference reversal (\"head control\"/\"sitting unaided\") was identified in all models. The rescaled utilities ranged from 0.3891 to 0.5577 (difference of 0.17 utilities) for TTO anchors corresponding to the worst (633233) and best (111111) health states. Health utilities ranged from 0.5534 to 0.7093 for the SG anchors. The disutility associated with a transition from \"no problems walking\" to \"bedridden\" was -0.0533, whereas disutility of moving from \"constant screaming\" relative to \"no screaming\" was -0.0248. The disutility associated with daily OCG was -0.0167. Disutilities for the other attributes were small although there were exceptions.</p><p><strong>Conclusion: </strong>A DCE was used to derive health utilities for AADC deficiency. These health utilities will subsequently be used in an economic model evaluating an AADC deficiency intervention.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-01-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/f6/b8/prom-13-21.PMC8800863.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39885812","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}