Journal of Patient-Reported Outcomes最新文献

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.

Background: Mirikizumab has demonstrated efficacy in moderately to severely active ulcerative colitis. A 1-2-point change in Urgency Numeric Rating Scale (NRS) score can be meaningful for patients. In these post-hoc analyses, we evaluated the efficacy of mirikizumab compared to placebo by baseline Urgency NRS score groups (0-3, 4-6, and 7-10) and its effect on bowel urgency severity over time.

Methodology: Urgency NRS was measured as a secondary outcome at baseline, week 12, and week 52. Bowel urgency improvement was assessed for patients who achieved and did not achieve multiple efficacy endpoints. Data were analyzed using Fisher's exact test with nonresponder imputation.

Results: At weeks 12 and 52, a significantly higher percentage of mirikizumab-treated patients achieved clinical response as well as clinical, endoscopic, and symptomatic remission compared to placebo-treated patients, regardless of baseline Urgency NRS score category (higher proportions versus placebo, delta 9%-45%). Improvement in Urgency NRS score category at weeks 12 and 52 for mirikizumab-treated patients was observed when other efficacy outcomes were achieved (13%-90%) and not achieved (12%-75%).

Conclusions: A greater proportion of mirikizumab-treated patients with ulcerative colitis achieved symptomatic, clinical, and endoscopic remission endpoints compared to placebo-treated patients, regardless of baseline bowel urgency severity. After one year, bowel urgency was improved to a greater extent with mirikizumab than with placebo, even for patients who did not achieve other clinical outcomes. Small improvements in bowel urgency are associated with significant health-related quality-of-life improvements. Monitoring shifts in urgency severity over time using the Urgency NRS can aid in understanding patients' treatment outcomes.

Trial registration: LUCENT-1 (NCT03518086) Registered 04 May 2018 https://clinicaltrials.gov/study/NCT03518086 . LUCENT-2 (NCT03524092) Registered 10 May 2018 https://clinicaltrials.gov/study/NCT03524092 .

Background: NASH-CHECK is a patient-reported outcome measure (PROM) that assesses symptoms and broader health-related quality of life (HRQOL) impacts of metabolic dysfunction-associated steatohepatitis (MASH), previously called non-alcoholic steatohepatitis (NASH). The measure was initially developed and validated for patients with non-cirrhotic MASH. This study describes an evaluation of the suitability of NASH-CHECK for patients with compensated cirrhotic MASH.

Methodology: Concept elicitation (CE) interviews were conducted with patients with clinically confirmed compensated cirrhotic MASH in the United States (US) and United Kingdom (UK) to determine the symptom burden and broader HRQOL impact of MASH. Symptoms and broader HRQOL impacts identified during analysis of the CE data were compared with the key concepts included in NASH-CHECK; any symptoms reported in the CE interviews but not reflected in NASH-CHECK were reviewed for relevance by clinical experts. The content validity of NASH-CHECK was evaluated further via cognitive debriefing (CD) interviews conducted with patients with compensated cirrhotic MASH in the US and UK.

Results: CE interviews were conducted with 33 patients with compensated cirrhotic MASH (US = 9, UK = 24; 60.6% female; mean age, 64.3 years). Key symptoms described were similar to those reported by patients with non-cirrhotic MASH identified during the development of NASH-CHECK; these included abdominal pain, abdominal bloating, itch, fatigue, sleeping difficulties, and cognitive symptoms. Other key HRQOL impacts included activity limitations and emotional, social, relationship, and work impacts. All key symptom and broader HRQOL impacts reported by patients with compensated cirrhotic MASH are currently included in NASH-CHECK, and no additional symptoms or HRQOL impacts reported during the CE interviews were deemed relevant for inclusion. CD interviews were conducted with 17 patients with compensated cirrhotic MASH (US = 8, UK = 9; 47.1% female; mean age, 62.8 years). Patient feedback on NASH-CHECK content confirmed that the concepts captured by the items were considered important, relevant, and comprehensive for addressing the impact of compensated cirrhotic MASH.

Conclusions: The results support the content validity of NASH-CHECK for patients with compensated cirrhotic MASH, demonstrating that NASH-CHECK is a suitable PROM for use in clinical trials, studies, and practice for this patient population.

Background: The Glasgow Hearing Aid Benefit Profile (GHABP) is a patient-reported outcome measure (PROM) that was developed for the assessment of hearing aid efficacy using standard goals and user-nominated goals. The objective of this study was to describe user-nominated hearing goals to determine themes that are not currently being captured by the standard goals and that could improve comprehensive assessment of hearing quality of life with the GHABP for use in clinical trials.

Methodology: We conducted a secondary analysis of a clinical trial at two tertiary care institutions. Adults ≥ 50 years of age with hearing loss completed the GHABP before treatment, including the portion where they provided up to two user-nominated goals for situations where hearing was personally challenging to them. We then categorized these goals into themes.

Results: A total of 262 participants completed the standard GHABP and provided a total of 501 user-nominated goals. Common themes were having a conversation with several people in a group (80/501, 16.0%), hearing in background noise (73/501, 14.6%), and listening when unable to see the speaker's mouth (57/501, 11.4%).

Conclusions: Themes of listening in background noise and listening when unable to see the speaker's mouth are very important to many individuals with hearing loss. Expanding PROMs to include these goals may improve patient-centeredness of clinical trial and clinical care outcomes tracking.

Background: Recent advances in cancer treatment have extended patient survival and improved quality of life (QOL), often enabling home-based chemotherapy. However, this shift places a growing burden on informal caregivers, impacting their own well-being. This study aims to explore changes in caregiver QOL over the course of treatment and identify contributing factors.

Methods: We conducted a single-institution, prospective observational study involving patients receiving chemotherapy for unresectable or recurrent solid tumors and their primary caregivers. QOL was assessed using the EORTC QLQ-C30 before each treatment line. Scores were stratified by treatment duration and line. Paired t-tests and multiple linear regression analyses were performed.

Results: Among 378 patient-caregiver pairs, caregiver emotional and cognitive functioning declined over time, particularly with longer treatment durations and later treatment lines. Fatigue prevalence increased to 100% by the fourth-line treatment. Regression analyses revealed that caregiver QOL was affected by patient symptoms (e.g., insomnia, appetite loss), patient QOL scores, treatment duration, and caregiver age and gender.

Conclusions: Caregivers experienced progressive emotional and cognitive declines paralleling the patient's clinical trajectory. These findings highlight the necessity of early and comprehensive support systems for caregivers, including psychological and social support, to maintain their QOL throughout cancer treatment.