Journal of Patient-Reported Outcomes最新文献

{"title":"Mapping immune checkpoint inhibitor side effects to item libraries for use in real-time side effect monitoring systems.","authors":"Julia Lai-Kwon, Michael Jefford, Stephanie Best, Iris Zhang, David Cella, Claire Piccinin, Bryce B Reeve, Claudia Rutherford","doi":"10.1186/s41687-025-00855-8","DOIUrl":"10.1186/s41687-025-00855-8","url":null,"abstract":"<p><strong>Background: </strong>Monitoring for the side effects of novel therapies using patient-reported outcomes (PROs) is critical for ensuring patient safety. Existing static patient-reported outcome measures may not provide adequate coverage of novel side effects. Item libraries provide a flexible approach to monitoring for side effects using customized item lists, but the ideal process for matching side effects to items sourced from multiple item libraries is yet to be established. We sought to develop a pragmatic process for mapping side effects to items from three major item libraries using immune checkpoint inhibitor (ICI) side effects as an example.</p><p><strong>Methods: </strong>Using a consumer- and clinician-driven list of 36 ICI side effects, two authors independently mapped side effects to Common Terminology Criteria for Adverse Event (CTCAE) terms, and then to three item libraries: the Patient-Reported Outcome version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE), the European Organisation for Research and Treatment of Cancer (EORTC) Item Library, and the Functional Assessment of Chronic Illness Therapy (FACIT) searchable library. The rates of inter-rater agreement were recorded. Following item collation from the item libraries, we devised criteria for selecting the optimal item for each side effect for inclusion in a future electronic PRO system based on guidance from the above groups.</p><p><strong>Results: </strong>All 36 side effects mapped to at least one CTCAE term, with eight mapping to more than one term. Twenty-three side effects mapped to at least one PRO-CTCAE term, 35 side effects mapped to at least one EORTC item, and 31 side effects mapped to at least one FACIT item. The inter-rater agreement rate was 100% (PRO-CTCAE), 83% (EORTC) and 75% (FACIT). Pre-determined criteria were applied to select the optimal item for each side effect from the three item libraries, producing a final 61-item list.</p><p><strong>Conclusion: </strong>Using ICI side effects as an example, we developed a pragmatic approach to creating customized item lists from three major item libraries to monitor for side effects of novel therapies in routine care. This process highlighted the challenges of using item libraries and priorities for future work to improve their usability.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"27"},"PeriodicalIF":2.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11885683/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143568471","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}

{"title":"Principles of good practice for translation of electronic clinical outcome assessments.","authors":"Huda Shalhoub, M Turner, A Bradley-Gilbride, S Eremenco, H Muehlan, E Parks-Vernizzi, B Arnold, D Kuliś, C Anfray, J E Chaplin, J P Repo","doi":"10.1186/s41687-025-00859-4","DOIUrl":"10.1186/s41687-025-00859-4","url":null,"abstract":"<p><strong>Background: </strong>While many publications have outlined good practice recommendations for translation and electronic implementation of clinical outcome assessments (COAs), they are often treated as independent processes. The scientific literature currently lacks recommended guidelines on the process of concurrent translation, cultural adaptation and electronic implementation of COAs for clinical research. In response to this need, the ISOQOL Translation and Cultural Adaptation Special Interest Group (TCA-SIG) sought to identify actionable steps for addressing the scientific and operational intricacies in this concurrent process.</p><p><strong>Methods: </strong>Using snowball sampling, semi-structured questions were sent to language service providers (LSPs), electronic clinical outcome assessment (eCOA) providers, and developers/copyright holders. The TCA-SIG workgroup, consisting of 13 members, then led the methodological groundwork for the disseminated surveys and established a cohesive set of recommendations.</p><p><strong>Results: </strong>The collective feedback that led to the recommendations included a total of 30 experts who responded to the surveys. Most of the respondents worked in companies or represented organizations based in the US and Europe.</p><p><strong>Recommendations: </strong>The recommendations fall into two main categories: namely, operational and scientific. The operational recommendations consist of active involvement from all stakeholders, the communication of clear expectations from the start, and better clarification of timelines of LSPs involved. Examples of scientific recommendations are electronic language feasibility assessment (ELFA), screenshot proofreading, as well as COA-specific developer and copyright holder guidelines for electronic implementation. COA-specific guidelines and instructions for electronic implementation and evaluation were seen to be needed and key recommendations are discussed in detail in this paper.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"26"},"PeriodicalIF":2.4,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11880485/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143543809","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}

{"title":"Psychological indicators for healthy aging: validation of the German short version of Ryff's scales of psychological well-being (SPWB).","authors":"Ana N Tibubos, Anna C Reinwarth, Iris Reiner, Antonia M Werner, Philipp S Wild, Thomas Münzel, Jochem König, Karl J Lackner, Norbert Pfeiffer, Manfred E Beutel","doi":"10.1186/s41687-025-00854-9","DOIUrl":"10.1186/s41687-025-00854-9","url":null,"abstract":"<p><strong>Background: </strong>The important roles of well-being as realization of one's true potential for healthy aging have been highlighted by literature of the recent decades. The Scales of Psychological Well-being (SPWB) are an internationally recognized measurement tool for psychological well-being. Yet, sound validation of the German SPWB 18-item version has been lacking to date. Therefore, the present study aims to (1) test the psychometric properties of the German SPWB 18-item version in terms of factorial validity and (2) determine construct validity by investigating its correlations with psychosocial variables, mental and physical health. (3) Sociodemographic characteristics of the SPWB in a middle to old age German population was explored.</p><p><strong>Methodology: </strong>Data of N = 3,374 participants 45-85 years old of the 10-year follow-up (2017-2022) of the Gutenberg Health Study (GHS) were analyzed. Descriptive analyses and inference statistical analyses were performed to assess construct validity. In order to determine the psychometric properties, item characteristics and reliability coefficients were analyzed. Confirmatory factor analyses tested the proposed theoretical factorial structure.</p><p><strong>Results: </strong>Construct validity of the SPWB was established with respect to sociodemographic, psychosocial (social support and resilient coping), and health variables (sleeping problems, depression and anxiety symptoms, stress, loneliness, and somatic diseases). Analysis of the psychometric properties of the German SPWB 18-item version rendered support for the theoretically proposed multidimensional structure of psychological well-being in our sample rather than a one factorial structure. Bi-factor models that take the method effects of positively and negatively formulated items into account are highly recommended.</p><p><strong>Conclusions: </strong>The German SPWB 18-item version shows comparable psychometric properties to previous large-scale studies from other countries. The SPWB provides psychological indicators for healthy aging.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"25"},"PeriodicalIF":2.4,"publicationDate":"2025-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11861829/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143492570","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}

{"title":"Development and refinement of the Clinical Global Impression of Improvement for Non-seizure Symptoms measure in Dravet syndrome and Lennox-Gastaut syndrome.","authors":"J Scott Andrews, Drishti Shah, Alise Nacson, Tara Symonds, Sophie Hughes, Mahnaz Asgharnejad, Arturo Benitez, Lara Sams","doi":"10.1186/s41687-024-00829-2","DOIUrl":"10.1186/s41687-024-00829-2","url":null,"abstract":"<p><strong>Background: </strong>Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) are rare, severe, childhood-onset developmental and epileptic encephalopathies characterized by treatment-resistant epilepsy and varying intellectual disability levels. Clinical outcome assessments (COAs) describe how patients feel, function, or survive, thus providing valuable information on a therapy's efficacy and impact. Individuals with DS or LGS are heterogeneous, and many have limited verbal abilities and intellectual disability. Existing epilepsy-specific COA measures are unsuitable for DS and LGS clinical trials as many items demonstrate floor effects in these populations. As patients often cannot self-report symptoms, caregiver feedback on the measures' relevance and understandability is critical when developing COAs to ensure their suitability for the intended population, and that caregivers can help clinicians complete the measures when necessary.</p><p><strong>Methodology: </strong>We aimed to develop a novel clinician-reported outcomes measure, to be completed in consultation with caregivers at clinic visits, to assess non-seizure symptoms in individuals with DS or LGS using a Clinical Global Impression of Improvement (CGI-I) approach: the CGI-I Non-seizure Symptoms measure. A 13-item initial draft measure was reviewed by experts in a three-round Delphi panel to confirm each item's relevance and refine descriptions, reduce overlap, and limit respondent burden.</p><p><strong>Results: </strong>Following panel review, three items reached consensus (≥70% agreement of no revision required) and were included in the final measure: communication, alertness, and disruptive behaviors. To ensure caregivers can help clinicians complete the measure, and to establish levels of change in each item domain considered meaningful from their perspective, the three-item measure was cognitively debriefed with caregivers of individuals with DS or LGS. Caregivers showed that each item was understandable by describing their child using the descriptions provided in the measure and reported that items were relevant or important to assess in DS or LGS. Most caregivers reported that even a minimal change to their child's condition in each domain would be meaningful to them and their child.</p><p><strong>Conclusions: </strong>This CGI-I Non-seizure Symptoms measure represents relevant non-seizure outcomes considered important to individuals with DS or LGS and their families. The systematic development and refinement approach presented here supports its use in DS and LGS clinical trials.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"24"},"PeriodicalIF":2.4,"publicationDate":"2025-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11845656/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143469480","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}

{"title":"The evaluation of clinical outcomes assessments and digital health technologies in clinical trials for obesity.","authors":"Iris A Goetz, Carolyn Sutter, Traci Abraham, Chisom Kanu, Kristina S Boye, Tara Symonds","doi":"10.1186/s41687-025-00841-0","DOIUrl":"10.1186/s41687-025-00841-0","url":null,"abstract":"<p><strong>Background: </strong>Clinical trials for obesity have traditionally focused on weight loss and resolution of comorbidities as primary outcomes. However, secondary outcomes, such as the impact of weight reduction on patient experience, like health-related quality of life (HRQoL), have increasingly been recognized as important. Therefore, a review was conducted to determine the Clinical Outcome Assessments (COAs) and Digital Health Technologies (DHTs) used in clinical trials for obesity to assess the patient experience.</p><p><strong>Methods: </strong>Two clinical trial databases (United States & European Union) were reviewed to identify Phase 2-4 clinical trials for obesity (2018-2023). A targeted literature review was also conducted using the OVID database to identify clinical trial for obesity publications which included COAs/DHTs (2010-2023).</p><p><strong>Result: </strong>Trials from the databases (n = 53) and publications (n = 42) were included in data extraction (N = 73). This resulted in identification of 108 COAs, the majority being patient-reported outcome (PRO) measures (n = 83), but also 24 performance outcomes (PerfO) measures, and 1 composite PRO-clinician-reported outcomes (ClinRO) measure, as well as 2 DHTs. The most frequently identified PRO measures were the Short Form 36 and the Impact of Weight on Quality of Life Lite Clinical Trials. Twenty-four PerfO measures were also identified, with the 6-minute walk test being most common. These measures were most often used to construct secondary endpoints, with physical function (PF) being the most frequently specified domain. PRO measures assessing eating-related thoughts/behaviours, physical activity, and disordered eating were also frequently included, although individual measures varied widely across trials.</p><p><strong>Conclusion: </strong>Review of COAs and DHTs in registered clinical trials and publications for obesity found that PRO measures were the most common type of COA used to develop endpoints with current use of DHTs limited. Specifically, the physical function domain of multidimensional patient-reported outcome measures assessing health-related quality of life were often used to construct secondary endpoints. Further work is warranted to assess how the COAs and DHT data collected in clinical trials are viewed by regulators and payers.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"23"},"PeriodicalIF":2.4,"publicationDate":"2025-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11842654/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143459688","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}

{"title":"Changes in healthcare engagement during the COVID-19 pandemic.","authors":"McKenzie Lockett, Gisselle C Tamayo, Benjamin D Schalet, Steven P Reise, Rachel Kimerling","doi":"10.1186/s41687-025-00850-z","DOIUrl":"10.1186/s41687-025-00850-z","url":null,"abstract":"<p><strong>Background: </strong>Healthcare engagement, defined as the self-efficacy to enact the behaviors needed to obtain optimal benefit from health services, is an important aspect of healthcare quality. Measuring changes to healthcare engagement is essential to informing current and ongoing adaptations to health service delivery. The objective of the present study was to explore the responsiveness of the recently developed PROMIS^® Healthcare Engagement measure (PHE), a patient-reported outcome, through investigating the impact of COVID and COVID-related healthcare disruptions on healthcare engagement from pre- to peri-pandemic.</p><p><strong>Methods: </strong>Baseline data (2018-2019) were collected via a national mail survey of Veterans receiving VA care. For follow-up data, a subset of participants was randomly selected to be invited to a follow-up survey. Administrative data was used from the VA's Corporate Data Warehouse (CDW). We used mixed effects linear modeling to compare changes in healthcare engagement from baseline to follow-up between Veterans who reported healthcare disruptions and Veterans who did not report healthcare disruptions, adjusting for covariates.</p><p><strong>Results: </strong>From baseline to follow-up, healthcare engagement scores increased on average by 2.84 points. Compared to Veterans who reported no disruptions, Veterans who experienced COVID-related healthcare disruptions demonstrated greater decreases to healthcare engagement (difference scores ≥ - 1.98, ps ≤ 0.002) Further, Veterans with more healthcare disruptions showed greater decreases in healthcare engagement relative to those with fewer healthcare disruptions, such that Veterans with 2 healthcare disruptions (difference score = -4.20) significantly differed from Veterans reporting only 1 healthcare disruption, and Veterans reporting 3 or more disruptions (difference score = -3.75) significantly differed from those with 2 disruptions.</p><p><strong>Conclusion: </strong>Our results provide preliminary evidence of the PHE's responsiveness through demonstrating that environmental factors, such as pandemic-related factors, influence healthcare engagement. The COVID-19 pandemic had a complex effect on healthcare engagement, with healthcare engagement scores increasing overall during the pandemic but Veterans reporting COVID-related healthcare disruptions showing decreased changes in healthcare engagement. These findings support the utility of the PHE as a measure of healthcare engagement.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"21"},"PeriodicalIF":2.4,"publicationDate":"2025-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11842638/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143459686","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}

{"title":"A social return on investment analysis of patient-reported outcome measures in value-based healthcare.","authors":"Ellie Crane, Jane Noyes, Mayara S Bianchim, Leah Mclaughlin, Adele Cahill, Gareth Roberts, Carys Stringer","doi":"10.1186/s41687-025-00853-w","DOIUrl":"10.1186/s41687-025-00853-w","url":null,"abstract":"<p><strong>Objectives: </strong>There is growing interest in the use of Patient Reported Outcome Measures (PROMs) to improve patient and healthcare service outcomes. This study aimed to measure the social and economic value of PROMs implemented within a VBHC framework.</p><p><strong>Methods: </strong>We conducted a Social-Return on Investment (SROI) analysis in Epilepsy, Heart Failure, and Parkinson's Disease services, to measure the value generated by PROMs for patients and the healthcare provider.</p><p><strong>Results: </strong>The SROI analysis revealed substantial variation in the value derived from the PROMs intervention across different services. The highest value was observed in Heart Failure with an SROI ratio of 5.55:1, which represents a substantial return on investment for patients and services. In contrast, the Parkinson's Disease service had small return on investment from PROMs with an SROI ratio of 1.29:1. In Epilepsy, the social value derived from PROMs was proportionally less than the investment made, with an SROI ratio of 0.85:1.</p><p><strong>Conclusion: </strong>These findings demonstrate the complexities of implementing PROMs within a clinical context, and careful consideration is likely needed in selecting suitable services and tailoring the implementation of PROMs to effectively meet specific service and patient requirements. Where PROMs yielded low or no value, the lack of return-on-investment prompts a strategic re-evaluation regarding how PROMs are funded, implemented, and utilized. As the first economic evaluation of PROMs in clinical practice, this study is a novel contribution to the emergent VBHC and PROMs evidence base. Furthermore, the findings from this study will inform recommendations to improve PROMs delivery across Wales.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"22"},"PeriodicalIF":2.4,"publicationDate":"2025-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11842646/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143459684","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}

{"title":"The outpatient experience questionnaire for child and adolescent mental health services: reliability and validity following a nationwide survey.","authors":"Hilde Hestad Iversen, Mona Haugum, Oyvind Bjertnaes","doi":"10.1186/s41687-025-00852-x","DOIUrl":"10.1186/s41687-025-00852-x","url":null,"abstract":"<p><strong>Background: </strong>The main objective was to evaluate the psychometric properties of the OutPatient Experience Questionnaire for Child and Adolescent Mental Health Services (OPEQ-CAMHS) among patients aged 16 and above, with a secondary objective of developing a parsimonious set of items and a short version of the instrument for this age group.</p><p><strong>Methods: </strong>A national pilot study was conducted with adolescents from outpatient CAMHS in Norway, testing a new digital, continuous measurement approach using a measurement instrument developed through a comprehensive methodological framework. The study assessed missing data, ceiling effects, factor structure, internal consistency, discriminant validity, and construct validity. A shorter version was derived from psychometric results.</p><p><strong>Results: </strong>The pilot study included responses from 555 (46.3%) patients. Low proportions of missing or \"not applicable\" responses were found for 18 of the 20 items, and all items were below the ceiling-effect criterion. Two scales emerged: \"structure and process\", and \"outcome\", both meeting alpha criteria. Each individual item demonstrated a stronger correlation with its expected scale than with any of the other scales. Construct validity was confirmed through associations with relevant variables expected to be associated with patient-reported experiences, including self-reported current state and well-being. The results supported a six-item short version.</p><p><strong>Conclusions: </strong>Psychometric testing confirmed data quality, internal consistency, and construct validity of OPEQ-CAMHS. The short version addresses respondent burden concerns and is now ready for broad implementation in Norwegian CAMHS and potentially in similar healthcare settings worldwide.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"20"},"PeriodicalIF":2.9,"publicationDate":"2025-02-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11836262/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143442029","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}

{"title":"A patient-reported outcome measure comprising the stool frequency and abdominal pain items from the Crohn's Disease Activity Index: psychometric evaluation in adults with Crohn's disease.","authors":"James D Lewis, Aisha Vadhariya, Sylvia Su, Xian Zhou, Frederick Durand, Ariane K Kawata, Larissa Stassek, Claudine Clucas, Stefan Schreiber","doi":"10.1186/s41687-025-00851-y","DOIUrl":"10.1186/s41687-025-00851-y","url":null,"abstract":"<p><strong>Background: </strong>The Stool Frequency (SF) and Abdominal Pain (AP) items from the Crohn's Disease Activity Index are together referred to as the \"Patient Reported Outcome\" (PRO). The SF item measures the number of very soft/liquid stools and the AP item measures abdominal pain severity, which are common Crohn's disease (CD) symptoms that patients consider important to treat. This study evaluated the psychometric properties of both PRO items separately and estimated thresholds for clinical remission in moderately to severely active CD.</p><p><strong>Methods: </strong>The measurement properties of the PRO items were analyzed using pooled data from VIVID-1 (NCT03926130), a Phase 3, randomized, placebo- and active-controlled study in adults with moderately to severely active CD. Analyses used weekly average scores of the SF and AP items at Weeks 0 (Baseline), 4, 12, and 52. Remission thresholds were estimated using the Patient Global Rating of Severity (PGRS) and Patient Global Impression of Change (PGIC) as primary anchors as well as qualitative evidence from exit interviews.</p><p><strong>Results: </strong>Data from 1065 participants (mean age: 36.2 years [standard deviation: 13 years]) were analyzed. During the trial, scores improved for both PRO items. Both items demonstrated moderate-to-good test-retest reliability for participants defined as stable based on PGRS and PGIC. Most correlations of related assessments were moderate (0.30≤|ρ| <0.70) with SF and moderate-to-large (0.30≤|ρ| ≤0.90) with AP. By contrast, as anticipated, both items had weak correlations (|ρ| <0.30) with endoscopic and laboratory assessments. The PRO items could discriminate between groups of participants known to differ based on other assessments. The PRO items were able to detect change, as score changes in both items between Baseline and Weeks 12 and 52 differed significantly between most PGRS and PGIC categories. Anchor-based analyses combined with responses from the exit interviews suggested that an SF score of ≤ 3 and an AP score of ≤ 1 could together represent clinical remission.</p><p><strong>Conclusion: </strong>These results support the reliability, construct-validity, and responsiveness of both PRO items in moderately to severely active CD and confirm previously suggested scores for both items that could represent clinical remission.</p><p><strong>Trial registration: </strong>Clinicaltrials.gov, NCT03926130. Registered 23 April 2019, https://clinicaltrials.gov/study/NCT03926130 .</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"19"},"PeriodicalIF":2.4,"publicationDate":"2025-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11833035/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143441958","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}

{"title":"Comparative performance of PROMIS Sleep Disturbance computerized adaptive testing algorithms and static short form in postmenopausal women.","authors":"Andrew Trigg, Claudia Haberland, Huda Shalhoub, Christoph Gerlinger, Christian Seitz","doi":"10.1186/s41687-025-00849-6","DOIUrl":"10.1186/s41687-025-00849-6","url":null,"abstract":"<p><strong>Background: </strong>The Patient-Reported Outcomes Measurement Information System (PROMIS) Sleep Disturbance v1.0 item bank (27 items) measures sleep disturbances. Rather than the full item bank, an 8-item short form (PROMIS SD SF 8b) or computerized adaptive testing (CAT) can be used. This study compares the performance of the PROMIS SD SF 8b with two CAT algorithms in postmenopausal women.</p><p><strong>Methods: </strong>This is a secondary analysis of data collected for the original psychometric testing of the PROMIS Sleep Disturbance item bank, in a sub-sample of women aged ≥55. A graded response model (GRM) was fitted for the item bank, then simulations evaluated the performance of CAT algorithms and the short form, in terms of root mean square error (RMSE) versus the latent trait estimate derived from the full bank. Two CAT algorithms were tested: CAT1 (stop once standard error <0.3 or 12 items administered) and CAT2 (stop once 8 items administered). Convergent and divergent hypotheses for validity were tested through correlations with the Pittsburgh Sleep Quality Index (PSQI) and Epworth Sleepiness Scale (ESS). Known-groups comparisons were made between those with and without self-reported sleep disorder.</p><p><strong>Results: </strong>A sample of 337 women was analyzed. Unidimensionality and item-level fit to the GRM was supported; however, the local independence assumption was violated. The CAT1 algorithm showed 4.18 items on average, with a minor decrease in performance (higher RMSE value) compared to CAT2 or the PROMIS SD SF 8b. Administering 8 items adaptively (CAT2) compared to fixed (PROMIS SD SF 8b) performed similarly (RMSE difference = 0.001). Reliability exceeded 0.90 across most of the latent trait for all approaches. Correlations with the PSQI and ESS were largely as hypothesized, with minor differences in coefficient values between the approaches (all within 0.05). Women reporting a sleep disorder had greater sleep disturbance than those who did not (p < 0.001 for all).</p><p><strong>Conclusions: </strong>The results of this study support using the PROMIS Sleep Disturbance item bank in postmenopausal women. The choice of PROMIS SD SF 8b versus CAT can largely be driven by practical reasons (respondent burden and operational complexity) rather than concerns of differential reliability and validity.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"18"},"PeriodicalIF":2.4,"publicationDate":"2025-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11832987/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143442026","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}