Quality of Life Research最新文献

Purpose: Outcome-based pricing models which consider domains of value not previously considered in healthcare, such as societal outcomes, are of increasing interest for healthcare systems. Societal outcomes can include family-reported outcome measures (FROMs), which measure the impact of disease upon the patient's family members. The FROM-16 is a generic and easy-to-use family quality of life tool, but it has never been used in the context of patients undergoing advanced therapy medicinal product (ATMP) treatment. The use of potentially curative ATMPs is limited due their high cost and the low number of eligible patients. Using the FROM-16 to collect the impact on family of disease and treatment in ATMP patients may demonstrate additional value created by an ATMP intervention and strengthen the case for its use.

Methods: This feasibility study aimed to test the validity of the FROM-16 in family members of ATMP patients as a prelude for its use in ATMP value estimation. Patients and family members (n = 24) were recruited from ATMP treatment centres in England and Wales. Family members completed the FROM-16 and were invited to a short debriefing interview.

Results: The FROM-16 showed high validity demonstrated by strong internal consistency (Cronbach's alpha = 0.917) and intraclass correlation (0.803, 95%). Interviews identified that whilst the FROM-16 covered most areas of quality-of-life impact experienced by the participants, some explained that they also experienced other impacts upon their personal health and future outlook.

Conclusion: This feasibility study provides evidence that the FROM-16 could be used as part of a structured systematic approach to measure family quality of life impact in ATMP patients.

Purpose: Meaningful change thresholds are important to help interpret patient-reported outcome scores. To date, meaningful within-patient change (MWPC) thresholds have only been proposed for NSCLC-SAQ total score. This study proposed clinically MWPC thresholds, and group-level minimal important change/difference (MIC/MID) thresholds for both improvement and worsening for the Non-Small Cell Lung Cancer- Symptom Assessment Questionnaire (NSCLC-SAQ) total and symptom scores.

Methods: Blinded patient data (N = 246) from the Phase 3 LIBRETTO-431 clinical trial were used in a pre-specified meaningful change analysis. A combination of anchor- and supportive distribution-based methods were used to estimate the MWPC, MIC, and MID thresholds. Triangulation across anchor estimates was then performed using a correlation-weighted average to provide a single MWPC, MIC, and MID estimate for improvement and worsening.

Results: NSCLC-SAQ total score and symptom scores showed moderate to high correlations with various anchors (ranging from 0.306 to 0.890), with threshold estimates being provided from multiple anchors (except for cough). Triangulation suggested MWPC, MIC, and MID thresholds for improved total score were - 2.5, -3.5, and - 2.0, respectively. For worsening, the proposed thresholds were 2.0, 0.5, and 2.0, respectively. The MWPC, MIC, and MID thresholds for improved symptom scores ranged from - 0.5 to -1.5, and the worsening thresholds for symptom scores ranged from 0.5 to 1.0.

Conclusion: This study provides the first worsening and improvement estimates of MWPC, MIC, and MID for NSCLC-SAQ total and symptom scores. The thresholds proposed in this study can be used to inform interpretation of NSCLC-SAQ scores in clinical trials.

Purpose: To systematically review qualitative studies on outcomes, needs, experiences, preferences, concerns and health-related quality of life (HRQoL) of people surviving cancer in Europe in the last decade.

Methods: Protocol registered ( https://www.crd.york.ac.uk/PROSPERO , ID575065).

Inclusion criteria: studies with qualitative methods, constructs related to HRQoL, and adults surviving cancer in Europe. The search was conducted in PubMed and Scopus since 2013. Abstracts and full text were revised, data extracted and study risk of bias assessed independently by two researchers. The primary outcomes were the themes arising from each study. A thematic analysis stratified according to the study objective was undertaken by grouping themes into categories.

Results: Of 18,256 articles identified, 43 fulfilled the inclusion criteria: 16 studies with a generic objective and 27 with specific objectives. Seven categories (57 themes) emerged from the studies with a generic focus: Clinical Management (n = 16), Symptoms and Physical Function (n = 5), Psychological Function (n = 21), Social Function (n = 18), HRQoL (n = 3), Life Disruption (n = 6), and Individual Factors (n = 1). The 12 studies focused on treatment and care experiences stand out among those with specific objectives, with most themes fitting into the same seven categories.

Conclusions: Results clearly showed the predominance of the social and psychological function domains over physical domains among people surviving cancer, additionally identifying specific needs in clinical management, such as information and communication, and relationship with and support from professionals. Therefore, these aspects should be incorporated into the evaluation of patient-centred initiatives for people surviving cancer.

Limitations: only two databases were searched, and most European countries were not represented.

Purpose: MMQ1 is a Danish-language patient-reported outcome measure (PROM) for quality of life (QOL) in people with multiple long-term conditions (MLTC). It measures needs-based QOL across six scales: Physical ability, Concerns and worries, Limitations in daily life, Social life, Personal finances and Self-image. There is currently no such measure available in English. This study aimed to translate and validate MMQ1 for use in the United Kingdom.

Methods: Translation used a two-panel method (expert panel: n = 5; and lay panel: n = 6). Content validity was assessed via cognitive interviews (n = 6). A postal survey of 2,753 patients with MLTC recruited through eight GP practices in Scotland included EQ-5D-5L and ICE-CAP as comparator measures alongside MMQ1. Classical test theory psychometric analysis of survey responses followed the International Society for Quality of Life Research minimum reporting standards.

Results: Translation resulted in an English-language MMQ1 with good face validity. Cognitive interviews established good content validity. 597 survey responses were received (response rate 22%). Good internal consistency reliability and concurrent validity were demonstrated. Confirmatory factor analysis showed acceptable fit to the six-scales of MMQ1. Five of the six scales also demonstrated good discriminative ability for detecting clinically meaningful differences in QOL. However, inter-item and inter-scale correlations suggested item redundancy and scale overlap.

Conclusion: The English-language MMQ1 demonstrated adequate psychometric properties using classical test theory. Further validation using Rasch analysis is planned, and may help optimise and abbreviate the measure. This PROM has the potential to improve the measurement of QOL in MLTC research, including trials.

Purpose: To examine global and health-related quality of life (QOL) among parents of individuals with Fontan physiology and determine associations with sociodemographic, parent and child-related health, psychological, and relational factors.

Methods: Parents participating in the Australian and New Zealand Fontan Registry (ANZFR) QOL Study (N = 151, Parent Mean age = 47.9 ± 10.2 years, age range: 31.6-79.6 years, 66% women; child Mean age = 16.3 ± 8.8, age range: 6.9-48.7 years, 40% female) completed a series of validated measures. Health-related QOL was assessed using the PedsQL 4.0 Core Generic Scales for adults and global QOL was assessed using a visual analogue scale (0-10).

Results: Most parents (81%) reported good global QOL (≥ 6), consistent with broader population trends. Nearly one-third of parents (28%) reported at-risk health-related QOL (based on total PedsQL scores) with physical functioning most affected (44%). Psychological factors, including psychological stress and sense of coherence, emerged as the strongest correlates of global and health-related QOL, explaining an additional 16 to 30% of the variance (using marginal R²). Final models explained 35 and 57% and of the variance in global and health-related QOL, respectively (marginal R²). Relational factors, including perceived social support and family functioning contributed minimally when analyzed alongside psychological variables.

Conclusion: While parents of individuals with Fontan physiology report good global QOL, challenges in health-related QOL exist. We identified key psychological, sociodemographic, and health-related factors associated with parental QOL outcomes. These data may aid early identification of physical and psychosocial difficulties and guide targeted health resource allocation for this population.

Aim: To identify instruments used to measure patient-reported outcomes after LT, and critically evaluate their measurement properties.

Methods: Five online databases were searched to find English-language LT-specific PROMs from their inception to October 2024. Studies describing the development or validation of PROMs were included. Two reviewers extracted and synthesized information about the characteristics of the studies and instruments. The methodological quality of the included studies was evaluated using the Consensus-Based Standards for the Selection of Health Measurement Instrument (COSMIN). Two reviewers independently evaluated the methodological quality of the included instruments.

Results: A total of 17 studies involving 14 PROMs met the inclusion criteria and were included in this review. The included PROMs assessed a range of outcomes, including health-related quality of life, therapeutic adherence, symptom experience, social participation, feeling for the donor, satisfaction and informational needs. None of the 14 scales reported the ten boxes of measurement properties outlined by the COSMIN. Among all the measurement properties, content validity from expert (64.3%, 9/14) and internal consistency (57.1%, 8/14) were the two most frequently measured attributes. Less than half of the studies evaluated the content validity from the patients' perspective. Four studies evaluated structural validity using exploratory factor analysis, but no study conducted confirmatory factor analysis or used IRT method to measure the structural validity.

Conclusion: This systematic review provided an overall picture and detailed analysis of LT-specific PROMs, and highlighted the paucity of well-developed and validated instruments. Further studies are urgently needed, both in terms of exploring patients' health concerns after LT and validating the instruments.

Reporting method: This study is reported according to the PRISMA Statement.

Purpose: Clinical cut-offs like minimum clinically important improvement (MCII) and patient acceptable symptom state (PASS) increase the interpretability of patient reported outcome measures (PROMs), but cut-off estimates vary considerably, clouding a clear definition of a successful surgical outcome. We report estimates of MCII and PASS following hip- and knee arthroplasty using multiple methods and compare the different estimation methods.

Methods: Elective hip or knee arthroplasty patients who underwent the regular pre- and postoperative assessments 2014-2018 were included. The generic EQ-5D-5L and either the Hip or Knee disability/injury and Osteoarthritis Outcome Score (HOOS/KOOS) were used. MCII and PASS were estimated based on multiple estimation techniques.

Results: Distributions were skewed, with up to 95% being acceptable according to anchor questions. MCII estimates for HOOS/KOOS Pain ranged 21-60/10-47, with fewest in-sample misclassifications for the lowest cut-offs, provided by the 75th percentile approach. PASS estimates for HOOS/KOOS Pain ranged 84-93/78-91, for EQ-5D Index/EQ-VAS 0.87-0.92/66-79 (for hip), and 0.79-0.88/66-76 (for knee), with fewest misclassifications for the 75th percentile approach (hip) and Pythagoras approach (knee). The 75th percentile approach was the approach most often giving MCII estimates below the minimal detectable change (MDC).

Conclusions: We report new one-year estimates of MCII and PASS of HOOS, KOOS and EQ-5D subscales following hip- and knee arthroplasty. Estimates varied considerably when using different anchors and estimation techniques. Overall, the 75th percentile approach had fewest misclassifications, and had the lowest thresholds for the MCII estimations, but which were often below the MDC.

Background: As patient-reported symptoms are increasingly incorporated into routine clinical practice and captured in electronic medical records these data can be used to conduct health-related quality of life research studies. This study compares symptom reports from the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) and its precursor patient reported symptom monitoring (PRSM) (hereafter PRSM/PRO-CTCAE) with multi-item patient-reported outcome (PRO) scales for fatigue (Functional Assessment of Chronic Illness Therapy/FACIT-Fatigue) and depression and anxiety (Mental Health Index/MHI).

Methods: This is a secondary analysis of data collected from women with early breast cancer (Stage I-III) scheduled for chemotherapy who completed PRSM/PRO-CTCAE, FACIT-Fatigue, and MHI scales pre- and post-chemotherapy. Spearman correlation coefficients estimated the magnitude and direction of correlations between measures (convergent validity). For each symptom, patients were then categorized based on who improved, stayed the same, or worsened on the composite PRSM/PRO-CTCAE score, and changes in scores on the PRO scales were compared.

Results: In a sample of 374 women, mean age was 57 years (SD 12.6) with 76% White. PRSM/PRO-CTCAE fatigue measures were strongly correlated with FACIT-Fatigue total scale and had mixed moderate to strong correlation for individual items within the FACIT-Fatigue scale. PRSM/PRO-CTCAE Sad and Anxiety measures were strongly correlated with MHI-Depression and MHI-Anxiety total scales, respectively, and had mixed moderate to strong correlation with individual items within the MHI subscales. PRSM/PRO-CTCAE pre-post changes in symptom scores mirrored pre-post changes in FACIT-Fatigue and MHI subscales.