Journal of Patient-Reported Outcomes最新文献

Purpose: To identify available patient-reported outcome measures (PROMs) used to evaluate central sensitization (CS) manifestations in chronic pain conditions and evaluate the quality of psychometric properties of those instruments.

Methods: A comprehensive search across multiple electronic databases was conducted for relevant studies following the specification of eligibility criteria and development of key search terms. After screening and full-text review, the methodological quality of studies and psychometric properties of PROMs were assessed and summarized using the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist and scoring manual. The results were statistically pooled in a meta-analysis, specifically test-retest reliability, based on data availability and consistency of findings across studies.

Results: A total of fifty-eight studies evaluating eight instruments in adult patients with chronic pain were included. The methodological quality of the included studies was varied. Most identified PROMs have limited evidence regarding their measurement properties. The Central Sensitization Inventory (CSI) received the highest overall ratings for most measurement properties among all the instruments, followed by Pain Sensitivity Questionnaire (PSQ) and Fibromyalgia Survey Questionnaire (FSQ). Based on pooled data from available studies, the test-retest reliability of the CSI was found to be excellent, with an intra-class correlation coefficient (ICC) of 0.93 (95% CI: 0.91-0.95) for overall chronic pain, 0.90 (95% CI: 0.87-0.93) for chronic musculoskeletal pain and 0.93 (95% CI: 0.88-0.99) for chronic neck pain. PSQ also demonstrated excellent test-retest reliability, showing an ICC of 0.86 (95% CI: 0.72-0.99) for chronic pain.

Conclusion: Although not all properties have been studied, the CSI, which received the highest overall ratings, could serve as a reliable PROM assessing CS in chronic pain. More studies should be performed to comprehensively evaluate all measurement properties of all included instruments.

Background: Communicative participation is the most important outcome of speech and language therapy, but there are no measurement instruments for children, adolescents, and young adults. This paper describes the development of MyCommunication-Youth: an item bank to measure self-reported communicative participation in children, adolescents and young adults with various communication disorders.

Aims: 1) To develop a comprehensive, comprehensible item bank for measuring communicative participation in children, adolescents, and young adults with communication problems. 2) To assess the content validity of the item bank in a sample of these groups. 3) To define criteria for the minimal age at which children with communication difficulties can self-report their communicative participation.

Method: Based on a literature review and two concept elicitation studies three initial versions of item pools were developed: one for children, one for adolescents and one for young adults. These pools were pilot tested, using cognitive debriefing interviews, on comprehensibility and comprehensiveness in a diverse group of participants with communication difficulties, whereafter a second version of the item pools were created. Hereafter, the content validity was assessed in the target population and in a group of speech and language therapists.

Results: Initially, three item pools were created for children (58 items), adolescents (78 items), and young adults (84 items). In the pilot test with 33 children adolescents and young adults with communication difficulties, items were revised for comprehensibility, some items were added for comprehensiveness, and some items were deleted because they appeared irrelevant, resulting in updated pools of 50, 69, and 72 items. In the content validity study, cognitive interviews with 27 participants and a focus group with 8 professionals identified additional revisions. Most items were comprehensible, but some were irrelevant for specific subpopulations of communication difficulties. Two new items were added after the input of professionals, whereafter the item bank was found comprehensive.

Conclusions: MyCommunication-Youth is an item bank for measuring communicative participation in children, adolescents and young adults with various communication difficulties. Three versions of the instrument were created: MyCommunication-Children of 49 items, MyCommunication-Adolescents of 70 items and MyCommunication-YoungAdults of 73 items. The item bank is comprehensible, relevant and comprehensive according to the target population and target professionals.

Purpose: The Australian and New Zealand Bariatric Surgery Registry is developing a bariatric-specific patient-reported outcome measure (PROM) to capture patient outcomes. This study aimed to establish an item bank and questionnaire to assess which outcomes are considered the most important by pre- & post-surgical patients and healthcare practitioners.

Methods: Initial qualitative studies were undertaken to provide an in-depth understanding of patients' lived experiences, and a targeted literature search was conducted to identify appropriate PROMs. Items from identified PROMs were pooled and categorised to form the basis of a questionnaire developed to interrogate bariatric patients' and healthcare practitioners' opinions on the importance of the various outcomes.

Results: 1,867 items from 76 instruments were extracted and pooled to form the item bank. Items were categorised and refined to generate an Outcome Importance Questionnaire containing 68 items across 10 domains. 313 participants completed the survey, including 48 pre-surgical patients, 180 post-surgical patients, and 85 Healthcare Practitioners. 52 outcomes (of 68; 76.5%) were prioritised by at least 1 group with 'Overall mental health', 'Co-morbidities', 'Satisfaction with surgery' and 'Satisfaction with quality of life' rated as the most important outcomes.

Conclusion: The item bank and outcome importance questionnaire demonstrated good coverage of patient-reported outcomes considered important to all stakeholders. Initial results identified distinct differences in preference votes by patient and healthcare practitioner groups, with sufficient variation to identify those outcomes considered the most important. Additional rounds of testing, including participant-suggested outcomes and forced-choice questions, will facilitate consensus on the most important outcomes for future inclusion in a Registry-based bariatric-specific PROM.

Background: Chronic hepatitis D (CHD) is the most severe form of viral hepatitis, which results in accelerated progression to cirrhosis and poor prognosis compared with other hepatitis infections, impacting patients' health-related quality of life (HRQoL). To adequately capture patient perspectives of new hepatitis D virus (HDV) treatments in clinical trials, patient-reported outcome (PRO) measures that are valid and assess key concepts relevant to the patient are needed. This study aimed to explore the patient experience of CHD and evaluate the content validity of the Hepatitis Quality of Life Questionnaire (HQLQv2) and the Fatigue Severity Scale (FSS) for use in an HDV population.

Methods: Combined qualitative concept elicitation (CE) and cognitive debriefing (CD) interviews were conducted with 39 patients in Germany, Italy, Spain, and the US with a clinician-confirmed diagnosis of CHD. Participants described their experience of CHD, informing the development of a conceptual model, and then completed the HQLQv2 and FSS using a think-aloud technique to assess understanding, relevance, and comprehensiveness of items, instructions, response scales, and recall periods. Interviews were conducted in the principal language of each country; official translations of the instruments were used, and all patient-facing study documents and the interview guide were translated by certified translators.

Results: The sample included participants with a range of liver fibrosis stages, including 11 with compensated (n = 9) and decompensated (n = 2) cirrhosis. Fatigue, loss of appetite, nausea, joint pain, and pain over the liver were the most frequently reported signs/symptoms. Fatigue was most commonly mentioned and was described as a severe and particularly burdensome symptom, that impacted several aspects of patients' daily lives. Participants reported that CHD impacted their emotional wellbeing (low mood, anxiety), physical functioning (difficulty walking), social functioning (attending social events), activities of daily living (household chores), and work. Participants demonstrated a good understanding of the HQLQv2 and FSS items, instructions, response scales and recall periods, and the concepts assessed were considered relevant to CHD by most participants.

Conclusion: Findings contribute to the understanding of the patient experience of CHD and support content validity of the HQLQv2 and FSS as outcome assessments for use in an HDV population.

Purpose: For future health technology assessment, an assessment of the utility value of influenza-like illnesses (ILIs) is crucial. Therefore, the objective of this study was to evaluate the impact of ILIs on utility value in a Japanese population.

Methods: We conducted an online survey between March and June 2024 to evaluate the impacts of ILIs on health-related quality of life, using a Japanese version of the EuroQol 5 Dimensions 5 Levels (EQ-5D-5 L) and EuroQol visual analog scale (EQ-VAS). Participants were children and adults aged < 80 years who experienced ILI symptoms or required home isolation due to a respiratory infection. A follow-up survey was conducted 2-3 weeks after the first survey to assess recovery. For children, we asked their parents or guardians to answer as the child's proxy. A generalized linear model was used to assess the impact of patient demographics, type and onset of symptoms and diagnosis on disutility.

Results: In total, 392 participants answered the first survey, and 264 participants answered the follow-up survey (134 adult participants and 130 parents or guardians). Compared with those who only answered the first survey, those who also answered the follow-up survey were older and more likely to be male in adult participants. The mean differences in the utility value and VAS scores between the first and follow-up surveys were - 0.055 and - 10.6 in the adult samples and - 0.079 and - 17.9 in the pediatric samples, respectively. In the generalized linear model, symptom onset within 7 days in the first survey was significantly associated with disutility value (coefficient - 0.049 [95% confidence interval [CI] - 0.086 to - 0.012], p = 0.010). However, none of the patient demographics were significantly associated with disutility value.

Conclusion: Utility values were lower during the symptomatic phase compared with the recovery phase. Our results are useful for disease burden assessment, health technology assessment, and cost-effectiveness analysis, which can support decision-making on the preventive and therapeutic management of respiratory infections.

Background: LIMB-Q Kids is a new patient-reported outcome measure (PROM) for children and adolescents with Lower limb differences (LLDs). This international field test study aimed to evaluate the psychometric properties of LIMB-Q Kids.

Methodology: Patients from pediatric orthopaedic clinics with a diagnosis of LLDs were recruited. Participants completed LIMB-Q Kids and 2 generic quality of life questionnaires, i.e., PROMIS Pediatric Short Form v2.0 - Mobility 8a, and PedsQL. Demographic and clinical data were collected including the LLRS AIM Index, a measure of clinical severity for LLDs. Rasch measurement theory (RMT) analysis was used to examine the psychometric properties of LIMB-Q Kids. Test-retest (TRT) reliability was examined and tests of construct validity were performed.

Results: Participants (N = 800) were recruited from 16 sites in 7 countries. Participants were aged 8 years and older (mean = 13, standard deviation = 3.2, range 8-25 years) and had a broad range of LLDs (e.g., Leg Length Discrepancy, Fibular Hemimelia, Skeletal Dysplasia, Blount's disease, Posteromedial Tibial Bowing, Osteogenesis Imperfecta, Congenital Pseudarthrosis of Tibia, Tibial Hemimelia and Amputations). RMT analysis provided evidence of the reliability and validity of 9 independently functioning scales that measure leg appearance, physical function, symptoms (hip, leg, knee, ankle, and foot), leg-related distress, and social, and psychological function. In addition, TRT reliability based on a sample of 46 participants was high for all 9 scales (Intraclass correlation coefficient ranges from 0.76-0.95). LIMB-Q Kids Physical Function scale correlated strongly with the PROMIS Pediatric Short Form v2.0 - Mobility 8a (Pearson correlation 0.82) and the PedsQL Physical Function total score (Pearson correlation 0.77). As hypothesized, participants with more severe LLDs based on the LLRS AIM index scores reported lower scores on all LIMB-Q Kids scales, indicating more impact on the patients.

Conclusions: This study provided evidence for the validity and reliability of LIMB-Q Kids. This new PROM can be used to inform research, quality improvement efforts, and clinical care. By measuring outcomes that matter most to children and adolescents with LLDs, LIMB-Q Kids can provide information to support evidence-based decisions.

Level of evidence: Level III.

Introduction: The use of electronic patient reported outcome measures (ePROMs) is increasing in routine cancer care, with benefit demonstrated in improving patient survival, satisfaction and response time. ePROMs represent a complex intervention, with successful implementation reliant upon a range of questionnaires, platform, patient and clinician characteristics alongside the wider organisational readiness and environment. Key performance indicators (KPIs) assess the performance of a system. A KPI framework would offer value in assessing ePROM implementation projects, however the outcomes and indicators of importance are not clear.

Method: A modified Delphi methodology was used to define a framework of KPIs for assessing the deployment of ePROMs in routine cancer care. Potential KPIs were identified through literature searches, de-duplicated and allocated to a matrix of domains. Delphi participants were identified through a literature review and study team networks. KPIs were presented to participants for prioritisation using an online platform. A final set of KPIs was identified through two rounds of consensus with participants rating each KPI for relevance.

Results: The literature search generated a list of 196 potential KPIs of which 48 were considered by 15 experts in the Delphi process. Consensus was reached to include 12 KPIs in the first round and a further 2 KPIs in the second round. Participant's open text responses were analysed, suggesting a number of areas of debate regarding which KPIs are most pertinent.

Discussion: This work provides a framework of 14 KPIs, covering those of relevance to patients, clinicians and health services and recognising the acceptability, feasibility and impact of ePROMs. This framework offers a means to appraise the implementation of ePROMs, supporting teams as they implement ePROMs in routine cancer care and other healthcare settings.

Background: Frequency and severity of vasomotor symptoms (VMS; hot flashes) associated with menopause significantly impact women's health-related quality of life (HRQoL). Treatment benefit in VMS clinical trials is assessed using patient-reported outcome (PRO) measures, which must demonstrate evidence of content validity. This research aimed to establish a conceptual model in VMS and evaluate content validity of the Hot Flash Daily Diary (HFDD), PROMIS Sleep Disturbance Short Form 8b (PROMIS SD SF 8b), and Menopause-Specific Quality of Life (MENQOL) questionnaire for use in VMS clinical trials.

Methods: Targeted searches were conducted to identify qualitative literature documenting women's VMS experiences. Qualitative concept elicitation (CE) and cognitive interviews (CI) were then conducted with 20 US participants experiencing VMS (n = 10 postmenopause; n = 10 AET-treated). Literature and CE findings were used to develop a conceptual model and confirm conceptual coverage of PRO measures selected for assessing efficacy in VMS clinical trials. CIs assessed the content validity of PRO measures.

Results: Findings from the literature and CE interviews informed a conceptual model depicting women's experiences of VMS. Thirty-three symptom concepts were identified with key symptoms including sweating, cold sweats/chills and tiredness/fatigue. Impacts of VMS on HRQoL were categorized into seven domains: sleep, emotional wellbeing, activities of daily living, social wellbeing, work/education, cognitive and physical functioning. The concepts assessed by the HFDD (VMS severity and frequency), PROMIS SD SF 8b (sleep disturbance) and MENQOL (menopause-related quality of life) aligned with those endorsed by women as relevant to their VMS experience. Instructions, recall periods and response options of the measures were understood. A reduction of one moderate or one severe hot flash in 24-hours (assessed by the HFDD) was considered a meaningful improvement by participants. Similar observations were made across study samples.

Conclusions: Findings provide detailed insights into women's experience of VMS, supporting the development of a conceptual model and assessment of conceptual coverage of selected PRO measures. Content validity of the HFDD, PROMIS SD SF 8b, and MENQOL for use in VMS clinical trials was supported. CI results suggest that a reduction of one moderate or one severe hot flash in 24-hours is meaningful to women with VMS.

Background: Although the use of patient-reported outcome measures (PROMs) in practice is increasing, successful implementation is contingent on engaging healthcare providers (HCPs). Using Implementation Science (IS), we present the content of two workshops hosted at the International Society for Quality-of-Life annual conferences for individuals seeking to implement PROMs collection and use in their settings. Our goals were to provide workshop participants with knowledge, tools, and resources to prepare HCPs for PROM adoption and to demonstrate tailored strategies to meet context-specific needs.

Methods: An interdisciplinary team with diverse expertise in PROMs implementation delivered two workshops guided by the Capability, Opportunity, Motivation - Behavior (COM-B) model and the Theoretical Domains Framework (TDF). Using dotmocracy, participants were asked to consider, for their local context, the factors most important for changing HCPs' behaviors to adopt PROMs in daily practice.

Results: The workshops incorporated IS theories, models, and frameworks (TMFs) to identify barriers faced by HCPs, support behavior change, and apply tailored theory-informed implementation strategies to prepare HCPs for PROM integration and evaluate adoption success. The factors rated the most important by workshop participants (n = 53) were woven into the discussions to illustrate the most common barriers encountered by HCPs adopting PROMs. Presenters drew on real-world practice and research experiences to identify promising implementation strategies, including education, training, behavioral modeling, persuasion, environmental restructuring, enablement, and audit and feedback to increase the capability, opportunity, and motivation of HCPs.

Conclusions: Given the increasing evidence base supporting the role of PROMs in patient-centered care, it is imperative to understand the mechanisms and best practices for increasing provider adoption of PROMs. This work offers a roadmap for understanding determinants more important to HCPs and systematically selecting theory-informed implementation strategies that may increase the likelihood of HCP adoption of PROMs. Offering tailored HCP training/education programs and implementation strategies can prepare HCPs for timely and effective PROM implementation.

Background: Vedolizumab is a gut-selective, anti-lymphocyte trafficking biologic therapy for inflammatory bowel disease (IBD). Clinical trials have demonstrated the positive impact of vedolizumab on patient quality of life (QoL); however, real-world evidence of its impact is limited. We evaluated vedolizumab impact on QoL of patients with IBD during the first 52 weeks of treatment in real-world practice in the United Kingdom and Ireland.

Methods: In this prospective observational study, patients with IBD initiated on vedolizumab completed 4 validated IBD-specific QoL questionnaires at baseline and weeks 14, 26, and 52. The primary endpoint was change in mean Short Inflammatory Bowel Disease Questionnaire (SIBDQ) score. Secondary endpoints included changes in mean scores and sub-scores for other QoL questionnaires.

Results: Overall, 61 patients were enrolled, including 22 with Crohn's disease (CD) and 39 with ulcerative colitis (UC). At week 52, the mean change in SIBDQ scores from baseline was statistically significant (+ 12.3 [p = 0.0123] for CD and + 10.8 [p = 0.0037] for UC) and clinically meaningful (considered as a ≥ 10-point change). A significant improvement in mean SIBDQ scores was seen as early as week 14 for both CD and UC cohorts (p = 0.0256 and p = 0.0348, respectively).

Conclusions: These real-world findings, using multiple validated tools, demonstrate that vedolizumab treatment for IBD is associated with measurable improvements in QoL from baseline.