Patient-Reported Outcomes in Cardiovascular Trials.
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ABSTRACT:
SUBMITTER: Masterson Creber R
PROVIDER: S-EPMC8487900 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:ObjectivesTo systematically assess the type of outcomes selected and the prevalence of patient reported outcomes in contemporary cardiovascular trials and to quantify any misuse or underuse of patient reported outcomes using a specially developed tool that would allow estimation of the relevance of such outcomes to clinical decision making.DesignSystematic review.Data sourcesMedline and Embase.Study selectionRandomised controlled trials of the treatment for or prevention of cardiovascular disease published in 10 leading general medical and cardiology journals from January 2005 to December 2008.ResultsPrimary outcomes were patient important (death, morbidity, or patient reported outcomes) in only 93 of 413 trials (23%, SE 2%), whereas another 92 (22%, SE 2%) combined these outcomes with other less important ones into a composite. Sixty five trials (16%; SE 2%) used at least one instrument to measure patient reported outcomes, mostly in trials where such information would have been important or crucial for clinical decision making (52 trials). Patient reported outcomes were judged to be of little incremental value to a large number of, mostly explanatory, cardiovascular trials (152 trials). However, many trials in which patient reported outcomes would have been important or crucial for clinical decision making did not report such outcomes (122 of 174 trials, 70%). These included several trials that primarily aimed to improve symptoms or functional status, trials that tested interventions with a considerable potential for causing harm (mainly bleeding) that were not meaningfully measured, and trials with composite outcomes that were dominated by outcomes of questionable importance to patients.ConclusionsDespite a continued rise in the reporting of patient reported outcomes with no evidence for their misuse in more recent cardiovascular trials, they seem to be still underused once their relevance to clinical decision making has been taken into account. This was largely explained by inappropriate use of composite outcomes and inadequate measurement of harms.
| S-EPMC2967478 | biostudies-literature
Project description:We describe the protocol, design, and methodology of the Prediction, Risk, and Evaluation of Major Adverse Cardiac Events (PRE-MACE) study as a multicomponent remote patient monitoring in cardiology. Using biosensor, biomarkers, and patient-reported outcomes in participants with stable ischemic heart disease, the PRE-MACE study is designed to measure cross-sectional correlations and establish the ability of remote monitoring to predict major adverse cardiovascular event (MACE) biomarkers and incident MACE at baseline and 12-month follow-up. It will further assess the adherence and cost-effectiveness of remote monitoring and blood sampling over the initial months. Despite medication and lifestyle changes, patients with cardiovascular disease can experience MACE due to undertreatment, poor adherence, or failure to recognize clinical or biochemical changes that presage MACE. Identifying patients using remote monitoring to detect MACE forerunners has potential to improve outcomes, avoid MACE, and reduce resource utilization. Data collection will include: (1) continuous remote monitoring using wearable biosensors; (2) biomarker measurements using plasma and at-home micro-sampling blood collection; and (3) patient-reported outcomes to monitor perceived stress, anxiety, depression, and health-related quality of life. Two hundred participants will be followed for 90 days with a subset (n = 80) monitored for 180 days. All participants will be followed up for MACE at 12 months.The PRE-MACE study will utilize remote monitoring with biosensors, biomarkers, and patient-reported outcomes to identify intermediate biomarkers of MACE in patients with stable ischemic heart disease. If shown to be effective, this intervention can be utilized between health visits to predict MACE and reduce financial impact of MACE.
| S-EPMC6722086 | biostudies-literature
Project description:BackgroundLymphoma treatment can lead to long-term consequences such as fatigue, infertility and organ damage. In clinical trials, survival outcomes, clinical response and toxicity are extensively reported while the assessment of treatment on quality of life (QoL) and symptoms is often lacking.ObjectiveWe evaluated the use and frequency of patient-reported outcome (PRO) instruments used in randomized controlled trials (RCTs) for Hodgkin lymphoma (HL) and their consistency of reporting.MethodsMEDLINE, CENTRAL and trial registries for RCTs investigating HL were systematically searched from 01/01/2016 to 31/05/2022. Following trial selection, trial, patient characteristics and outcome data on the use of PRO measures (PROMs) and reporting of PROs using a pre-defined extraction form were extracted. To assess reporting consistency, trial registries, protocols and publications were compared.ResultsWe identified 4,222 records. Following screening, a total of 317 reports were eligible for full-text evaluation. One hundred sixty-six reports of 51 ongoing/completed trials were included, of which 41% of trials were completed and 49% were ongoing based on registry entries. Full-text or abstract were available for 33 trials. Seventy percent of trials were conducted in the newly diagnosed disease setting, the majority with advanced HL. In 32 trials with published follow-up data, the median follow-up was 5.2 years. Eighteen (35%) completed/ongoing trials had mentioned PRO assessment in registry entries, protocol or publications. Twelve trials (67%) had published results and only 6 trials (50%) reported on PROs in part with the exception of 1 trial where PROs were evaluated as secondary/exploratory outcome. The most referenced global PROM was the EORTC-QLQ-C30 (12 studies), the EQ-5D (3 studies) and the FACT-Neurotoxicity (3 studies). FACT-Lymphoma, a disease-specific PROM for non-HL was mentioned in one ongoing trial. None of the trials referenced the EORTC QLQ-HL27, another disease-specific PROM developed specifically for HL patient's QoL assessment.DiscussionsOnly one-third of RCTs in HL report PROs as an outcome and only half present the outcome in subsequent publications, showcasing the underreporting of PROs in trials. Disease-specific PROMs are underutilized in the assessment of QoL in HL patients. Guidance on the assessment of PROs is needed to inform on comprehensive outcomes important to patients.Systematic review registrationhttps://www.crd.york.ac.uk/PROSPERO/display_record.php?RecordID=391552, identifier CRD42023391552.
| S-EPMC10965683 | biostudies-literature
Project description: Not available
| S-EPMC5362349 | biostudies-literature
Project description:BackgroundSymptoms are common among patients enrolled in phase I trials. We assessed the validity of Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) items in relation to previously validated assessments of quality of life and psychological distress. We used data from a randomized trial testing a palliative care support intervention for patients enrolled on phase I trials.MethodsPatients (n?=?479) were accrued to the parent study prior to initiating a phase I clinical trial with data collected at baseline, 4, and 12?weeks. We determined the correlation of PRO-CTCAE with distress level, Functional Assessment of Cancer Therapy - General (FACT-G) total, and subscale domain scores.ResultsPatients were predominantly female (56.8%) and older than age 60 years, and 30.7% were from minority populations. The correlation coefficient for distress level for all PRO-CTCAE items was small to moderate (Pearson r?=?0.33-0.46). Pearson correlation coefficient for FACT-G total was moderate (r = -0.45 to -0.69). Stronger associations were noted for mood items of the PRO-CTCAE only (with distress level, r?=?0.55-0.6; with FACT-G, r = -0.54 to -0.6). PRO-CTCAE symptom interference scores had the strongest correlation with distress level (Pearson r?=?0.46) and FACT-G total (Pearson r = -0.69). Correlations between PRO-CTCAE items and corresponding FACT-G (total and subscales) and distress levels reached statistical significance for all items (P <.001).ConclusionEvidence demonstrates validity of PRO-CTCAE in a heterogeneous US sample of patients undergoing cancer treatment on phase I trials, with small to moderate correlations with distress level for all PRO-CTCAE items and moderate correlations with quality of life as measured by FACT-G total.
| S-EPMC7768928 | biostudies-literature
Project description:Investigators of clinical trials in which the list of outcomes include patient-reported outcomes (PROs) - usually labeled quality of life (QoL) - have a large number of instruments from which to choose. The extent and manner in which PRO instruments are used in clinical trials can be assessed using data from clinical trial registries. Most medical journals now require a clinical trial be registered before its results are considered for publication. This requirement is intended to discourage publication bias, such as the reporting of tests of hypotheses different from those stipulated at the start of the trial and selective reporting of partial results.To assess the usage of PRO instruments in registered trials by various trial characteristics and to determine if the instruments are adequately identified in the registry.A local copy of the ClinicalTrials.gov database was made in September 2007. The outcomes of all interventional trials registered since September 2004 were assessed for usage of a PRO instrument. Odds ratios of PRO usage were estimated by a logistic regression model.Of 17,704 interventional trials, 2481 (14.0%) used at least one PRO instrument. However, less than half of those trials (41.0%) identified the instrument to be used. PRO usage is positively associated with phase (III), randomization type (randomized), intervention type (behavior) and sponsorship type (university/research organization).PRO instruments are used in a significant percentage but minority of clinical trials. Trial registries should require that all PRO instruments be identified, including the concepts or outcomes they are intended to measure.
| S-EPMC2683916 | biostudies-other
Project description:Background Atherosclerotic cardiovascular disease ( ASCVD ) accounts for approximately one third of deaths in women. Although there is an established relationship between positive patient experiences, health-related quality of life, and improved health outcomes, little is known about gender differences in patient-reported outcomes among ASCVD patients. We therefore compared gender differences in patient-centered outcomes among individuals with ASCVD . Methods and Results Data from the 2006 to 2015 Medical Expenditure Panel Survey, a nationally representative US sample, were used for this study. Adults ?18 years with a diagnosis of ASCVD , ascertained by International Classification of Diseases, Ninth Revision ( ICD-9) codes and/or self-reported data, were included. Linear and logistic regression were used to compare self-reported patient experience, perception of health, and health-related quality of life by gender. Models adjusted for demographics, socioeconomic status, and comorbidities. There were 21 353 participants included, with >10 000 (47%-weighted) of the participants being women, representing ?11 million female adults with ASCVD nationwide. Compared with men, women with ASCVD were more likely to experience poor patient-provider communication (odds ratio 1.25 [95% confidence interval 1.11-1.41]), lower healthcare satisfaction (1.12 [1.02-1.24]), poor perception of health status (1.15 [1.04-1.28]), and lower health-related quality of life scores. Women with ASCVD also had lower use of aspirin and statins, and greater odds of ?2 Emergency Department visits/y. Conclusions Women with ASCVD were more likely to report poorer patient experience, lower health-related quality of life, and poorer perception of their health when compared with men. These findings have important public health implications and require more research towards understanding the gender-specific differences in healthcare quality, delivery, and ultimately health outcomes among individuals with ASCVD .
| S-EPMC6405598 | biostudies-literature
Project description:To optimize the use of patient-reported outcomes (PROs) in clinical research, it is first necessary to review the current use of these outcomes in clinical trials to determine under what circumstances they are most useful, and to reveal current limitations.To investigate current patterns of use of PROs in clinical trials.We conducted a systematic literature review of all double-blind, placebo-controlled, randomized clinical trials using one or more PROs as a study outcome from 2004 to 2006. Data were abstracted and analyzed with descriptive statistics and logistic regression to characterize the use of PROs in clinical trials.The 180 clinical trials that met the study inclusion criteria used 173 unique instruments to measure a total of 466 PROs. Most PRO measurements were obtained using relatively few PRO instruments, with one-third of PRO instruments applied in more than 1 trial. In multivariable analysis, tests of statistical significance were more often reported for PROs used as primary trial outcomes. Statistically significant PRO outcomes (P<0.05) were more likely among disease-specific PROs compared with general PROs, PROs with a discussion of minimally important difference, and larger trials.PRO instruments may be improved through efforts to provide centralized electronic administration, cross-validation, and standardized interpretation of clinically relevant outcomes. The majority of PROs used in current clinical trials come from relatively few, commonly used disease-specific PRO instruments within major therapeutic areas.
| S-EPMC3682647 | biostudies-literature
Project description:Patient-reported outcomes (PROs) are playing an increasing role in multiple sclerosis (MS) research and practice, and are essential for understanding the effects that MS and MS treatments have on patients' lives. PROs are captured directly from patients and include symptoms, function, health status, and health-related quality of life. In this article, we review different categories (e.g., generic, targeted, preference-based) of PRO measures and considerations in selecting a measure. The PROs included in MS clinical research have evolved over time, as have the measures used to assess them. We describe findings from recent MS clinical trials that included PROs when evaluating Food and Drug Administration-approved disease-modifying therapies (e.g., daclizumab, teriflunomide). Variation in the measures used in these trials makes it difficult to draw any conclusions from the data. We therefore suggest a standardized approach to PRO assessment in MS research and describe 2 generic, National Institutes of Health-supported measurement systems [Neuro-QoL and the Patient-Reported Outcomes Measurement Information System (PROMIS)] that would facilitate such an approach. The use of PROs in MS care and research is expanding beyond clinical trials, as is demonstrated by examples from comparative effectiveness and other patient-centered research. The importance of PRO assessment is expected to continue to grow in the future.
| S-EPMC5722775 | biostudies-literature
Project description:Incorporation of the patient perspective into research and clinical practice will enrich our understanding of the status and management of patients with glomerular disease and may result in therapies that better address patient needs. In recent years, the importance of the patient experience of glomerular disease has become clear, and significant efforts have been undertaken to systematically capture and describe the patient's disease experience. Patient-reported outcome instruments provide a means to assess the patient's experience in a quantitative manner, thus enabling for comparisons within and between patients. Patient-reported outcome assessments are solely on the basis of a patient report about the status of their health without amendment or interpretation by a clinician or others. Patient-reported outcome assessments provide an opportunity to incorporate the patient perspective into clinical care, research, and clinical trials. Our paper provides an overview of terminology and development methods for patient-reported outcomes and reviews (1) currently available patient-reported outcome instruments appropriate for use in glomerular disease, (2) existing patient-reported outcome data in glomerular disease, and (3) opportunities for incorporating patient-reported outcome instruments into clinical care and research.
| S-EPMC5220669 | biostudies-other