Patient-Patient Centered Outcomes Research最新文献

Objective: We aimed to synthesize the evidence on the construct validity and responsiveness of the EQ-5D and compare them with asthma-specific health-related quality-of-life scales, to guide further research and clinical applications in asthma.

Methods: We searched key databases from inception to 1 June, 2024 and used the COnsensus-based Standards for the Selection of Health Measurement Instruments (COSMIN) method to appraise the evidence. The effect size estimates were aggregated using the inverse variance method to evaluate the relative efficiency of EQ-5D measures against the Asthma Quality of Life Questionnaire (AQLQ) and/or its corresponding preference-based index, Asthma Quality of Life-5 Dimensions (AQL-5D).

Results: There were 493 tests (construct validity: 428; responsiveness: 65) drawn from 37 selected articles (validation: 7; clinical: 30). Overall, 78.4% and 76.9% of the a priori hypotheses for assessing construct validity (convergent validity: 56.4%; known groups: 88.5%) and responsiveness, respectively, were satisfied. The methodological quality was "very good" or "adequate" in 78.2% of construct validity tests and 92.3% of responsiveness tests. The pooled correlation coefficient between EQ-5D index and AQLQ total scores was 0.52 (95% confidence interval 0.43-0.59), and between EQ visual analog scale and AQLQ total scores was 0.53 (95% confidence interval 0.34-0.69). The Cohen's d ratios for the index, level sum scores, and visual analog scale compared to AQLQ were 0.56 (n = 27), 1.16 (n = 16), and 0.75 (n = 37). The EQ-5D index's Cohen's d ratio compared to AQL-5D was 0.49 (n = 5). The standardized response mean ratios for the index and visual analog scale compared to AQLQ were 0.26 (n = 11) and 0.63 (n = 9).

Conclusions: The EQ-5D demonstrated overall good validity and responsiveness in the adult asthma population. However, a comparison against disease-specific instruments suggested scope for improvement in its psychometric performance for this population.

Background and objective: The EQ Health and Wellbeing (EQ-HWB) is a new generic quality-of-life measure for use in evaluating interventions in health, public health and social care. This study aimed to explore proxies' views regarding the appropriateness of the EQ-HWB for measuring residents' quality of life living in residential aged care facilities.

Methods: Qualitative think-aloud and semi-structured interviews were conducted with family members and aged care staff across three facilities in Melbourne, Australia. Proxies completed the 25-item EQ-HWB proxy version 2 (i.e. proxy-person perspective) whilst talking through the reasons for choosing their response. All interviews were audio-recorded and transcribed verbatim. A thematic analysis was used for data analysis.

Results: The sample included 29 proxies; nine family members and 20 aged care staff. The first theme summarised proxies' ability to proxy report residents' health and well-being using the EQ-HWB, which highlighted challenges with adherence to the proxy perspective, proxies' limited knowledge about residents, disagreement with residents' self-evaluation and use of heuristics. The second theme reflected feedback on the suitability of the EQ-HWB for use in residential aged care. Although proxies perceived that the EQ-HWB covered important domains, there were concerns about ambiguity, inappropriate examples, double-barrelled items and perceived repetition. Suggestions were made to improve the response options, comprehensiveness, recall period, layout and instructions of the questionnaire.

Conclusions: While the EQ-HWB captures domains relevant to residential aged care, modifications to item wording and examples are necessary to improve its appropriateness. Use of the proxy-person perspective revealed some challenges that require further consideration.

Background and objectives: Increasing longevity and advances in treatment have increased the cancer burden in the elderly, resulting in complex follow-up care needs; however, in China, little is known about the follow-up care preferences of these patients. This study quantified older cancer patients' preferences for follow-up care and examined the trade-offs they are willing to make to accept an alternative follow-up model.

Methods: A discrete choice experiment was conducted among inpatients aged over 60 years with breast, prostate, or colorectal cancer, at two large tertiary hospitals in Nantong, China. Preference weights for follow-up care were estimated using mixed logit analysis. Subgroup analysis and latent class analysis were used to explore preference heterogeneity.

Results: Complete results were obtained from 422 patients (144 with breast cancer, 133 with prostate cancer, 145 with colorectal cancer), with a mean age of 70.81 years. Older cancer patients stated a preference for follow-up by specialists over primary healthcare (PHC) providers ( $\beta$ = -1.18, 95% confidence interval -1.40 to -0.97). The provider of follow-up care services was the most valued attribute among patients with breast cancer (relative importance [RI] 37.17%), while remote contact services were prioritized by patients with prostate (RI 43.50%) and colorectal cancer (RI 33.01%). The uptake rate of an alternative care model integrating PHC increased compared with the baseline setting when patients were provided with preferred services (continuity of care, individualized care plans, and remote contact services).

Conclusion: To encourage older cancer patients to use PHC-integrated follow-up care, alternative follow-up care models need to be based on patients' preferences before introducing them as a routine option.

Background: Treatment preference research can support shared and informed decision making for currently available atopic dermatitis (AD) treatments, and simultaneously guide research and development for future therapies. In this systematic literature review, we aimed to provide an overview of preferences for AD treatments.

Methods: This systematic literature review was conducted in the Medline and Embase (via Ovid) databases, supplemented by manual searching. Quantitative research published from 2010 to September 2023 that investigated preferences for AD treatments were included. Quality assessment was conducted by using the purpose, respondents, explanation, findings, significance checklist, and a checklist developed by the Professional Society for Health Economics and Outcomes Research.

Results: In total, 207 references were screened after removing duplicates and 15 studies were included. Most studies were conducted in the US, followed by European countries. On average, people directly or indirectly affected by AD rate efficacy and treatment-related risk as the most important criteria when choosing an AD therapy. Participants are willing to increase risks in order to have a higher chance of achieving a certain benefit, e.g. reduction in itch or clearer skin. Participants have preferences for different modes of administration. On average, 68% (all full-text studies) and 87% (only discrete choice experiments [DCEs]) of quality criteria per reference were rated as fulfilled. DCEs received generally higher quality assessment scores than non-DCEs.

Conclusions: This review revealed that AD treatment preference research is limited. Diverse study designs hampered comparison and synthesis of the results. We recommend conducting more DCEs in this field to increase the likelihood of AD patients receiving the therapy that best fits their individual needs and preferences.

Clinical trials registration: This protocol was published in PROSPERO (ID: CRD42023468757).

Background: The intent of plain-language resources (PLRs) reporting medical research information is to advance health literacy among the general public and enable them to participate in shared decision-making (SDM). Regulatory mandates coupled with academic and industry initiatives have given rise to an increasing volume of PLRs summarizing medical research information. However, there is significant variability in the quality, format, readability, and dissemination channels for PLRs. In this scoping review, we identify current practices, guidance, and barriers in developing and disseminating PLRs reporting medical research information to the general public including patients and caregivers. We also report on the PLR preferences of these intended audiences.

Methods: A literature search of three bibliographic databases (PubMed, EMBASE, Web of Science) and three clinical trial registries (NIH, EMA, ISRCTN registry) was performed. Snowball searches within reference lists of primary articles were added. Articles with PLRs or reporting topics related to PLRs use and development available between January 2017 and June 2023 were identified. Evidence mapping and synthesis were used to make qualitative observations. Identified PLRs were quantitatively assessed, including temporal annual trends, availability by field of medicine, language, and publisher types.

Results: A total of 9116 PLRs were identified, 9041 from the databases and 75 from clinical trial registries. The final analysis included 6590 PLRs from databases and 72 from registries. Reported barriers to PLR development included ambiguity in guidance, lack of incentives, and concerns of researchers writing for the general public. Available guidance recommendations called for greater dissemination, increased readability, and varied content formats. Patients preferred visual PLRs formats (e.g., videos, comics), which were easy to access on the internet and used short jargon-free text. In some instances, older audiences and more educated readers preferred text-only PLRs. Preferences among the general public were mostly similar to those of patients. Psychology, followed by oncology, showed the highest number of PLRs, predominantly from academia-sponsored research. Text-only PLRs were most commonly available, while graphical, digital, or online formats were less available. Preferred dissemination channels included paywall-free journal websites, indexing on PubMed, third-party websites, via email to research participants, and social media.

Conclusions: This scoping review maps current practices, recommendations, and patients' and the general public's preferences for PLR development and dissemination. The results suggest that making PLRs available to a wider audience by improving nomenclature, accessibility, and providing translations may contribute to empowerment and SDM. Minimizing variability among available guida

Objectives: Patient preferences have the potential to influence the development of new treatments for locally advanced/metastatic urothelial carcinoma (la/mUC), and therefore we explored how patients with la/mUC value different attributes of first-line treatments.

Methods: An online preference survey and multidimensional thresholding (MDT) exercise were developed following a targeted literature review and qualitative interviews with physicians, patients with la/mUC, and their caregivers. Treatment attributes included two benefits (overall response rate [ORR], pain related to bladder cancer [scored 0-100; 100 being the worst pain possible]) and four treatment-related risks (peripheral neuropathy, severe side effects, mild to moderate nausea, mild to moderate skin reactions). A Dirichlet regression was used to estimate average preference weights. Marginal utility and the reduction in ORR that patients would accept in exchange for a 10-point decrease or a 10% decrease in other attributes were calculated.

Results: A total of 100 patients were recruited and self-completed the survey and MDT. Mean patient age was 64.9 years (standard deviation, 7.6), 54% were female, and 38% identified as white. All included treatment attributes had a statistically significant impact on preferences. Changes in ORR had the largest impact, followed by cancer-related pain and treatment-related risks. Patients were willing to accept an 8.4% decrease in ORR to reduce their pain level by 10 points or a 7.8% decrease in ORR to reduce the risk of peripheral neuropathy by 10%. For a 10% decrease in severe side effects, mild to moderate nausea, or skin reaction, patients would accept decreases in ORR of 5.5%, 3.7%, or 3.4%, respectively.

Conclusions: Of the attributes tested, changes in ORR were most important to patients. Patients made tradeoffs between treatment attributes indicating that a lower ORR may be acceptable for an improvement in other attributes such as reduced cancer-related pain or the risk of treatment-related adverse events.

Introduction: Patient-reported outcomes measures (PROMs) are increasingly prevalent in healthcare and used for shared decision-making and healthcare quality evaluation. However, the extent to which patients with varying health literacy levels can complete PROMs is often overlooked. This may lead to biased aggregated data and patients being excluded from studies or other PROM collection initiatives. This cross-sectional study evaluates the comprehensibility of 157 well-known and widely used PROM scales using a comprehensibility checklist.

Methods: Pairs of two independent raters scored 157 PROM scales designed for adults included in the 35 sets of outcome information developed as part of the Dutch Outcome-Based Healthcare Program. The PROM scales were scored on the eight comprehensibility domains of the Pharos Checklist for Questionnaires in Healthcare (PCQH). Interrater agreement of domain ratings was assessed using Intraclass Correlation Coefficients or Cohen's kappa. Subsequently, final ratings were established through discussion and used to evaluate the domain-specific comprehensibility rating for each PROM scale.

Results: Comprehensibility of a large number of PROM scales (n = 157), which cover a wide range of diseases and conditions across Dutch medical specialist care, was assessed. While most PROM scales were written at an accessible language level, with minimal use of medical terms, instruction clarity, number of questions, and response options emerged as significant issues, affecting a substantial proportion of PROM scales. Interrater agreement was high for most domains of the PCQH.

Conclusion: This study highlights the need for greater attention to the comprehensibility of PROMs to ensure their accessibility to all patients, including those with low health literacy. The PCQH can be a valuable tool in PROM development in addition to qualitative methods and in selection processes enabling comparison of comprehensibility between PROMs. However, the PCQH needs further development and validation for these purposes. Enhancing the comprehensibility of PROMs is essential for their effective incorporation in healthcare evaluation and decision-making processes.

This paper provides an introduction to statistical analysis of choice data using example data from a simple discrete-choice experiment (DCE). It describes the layout of the analysis dataset, types of variables contained in the dataset, and how to identify response patterns in the data indicating data quality. Model-specification options include linear models with continuous attribute levels and non-linear continuous and categorical attribute levels. Advantages and disadvantages of conditional logit, mixed logit, and latent-class analysis are discussed and illustrated using the example DCE data. Readers are provided with links to various software programs for analyzing choice data. References are provided on topics for which there currently is limited consensus and on more advanced techniques to guide readers interested in exploring choice-modeling challenges in greater depth. Supplementary materials include the simulated example data used to illustrate modeling approaches, together with R and Matlab code to reproduce the estimates shown.

Background: Kaizen is a Japanese term for continuous improvement (kai ~ change, zen ~ good). In a kaizen task, a respondent makes sequential choices to improve an object's profile, revealing a preference path. Including kaizen tasks in a discrete choice experiment has the advantage of collecting greater preference evidence than pick-one tasks, such as paired comparisons. OBJECTIVE AND METHODS: So far, three online discrete choice experiments have included kaizen tasks: the 2020 US COVID-19 vaccination (CVP) study, the 2021 UK Children's Surgery Outcome Reporting (CSOR) study, and the 2023 US EQ-5D-Y-3L valuation (Y-3L) study. In this evidence synthesis, we describe the performance of the kaizen tasks in terms of response behaviors, conditional logit and Zermelo-Bradley-Terry (ZBT) estimates, and their standard errors in each of the surveys.

Results: Comparing the CVP and Y-3L, including hold-outs (i.e., attributes shared by all alternatives) seems to reduce positional behavior by half. The CVP tasks excluded multi-level improvements; therefore, we could not estimate logit main effects directly. In the CSOR, only 12 of the 21 logit estimates are significantly positive (p < 0.05), possibly due to the fixed attribute order. All Y-3L estimates are significantly positive, and their predictions are highly correlated (Pearson: logit 0.802, ZBT 0.882) and strongly agree (Lin: logit 0.744, ZBT 0.852) with the paired-comparison probabilities.

Conclusions: These discrete choice experiments offer important lessons for future studies: (1) include warm-up tasks, hold-outs, and multi-level improvements; (2) randomize the attribute order (i.e., up-down) at the respondent level; and (3) recruit smaller samples of respondents than traditional discrete choice experiments with only pick-one tasks.