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Objective: To perform a comparison of the measurement properties of two cancer-specific Multi-Attribute Utility Instruments (MAUIs), EORTC QLU-C10D and FACT-8D, in Chinese patients with hematologic malignancies (HM). Methods: We conducted a longitudinal study on patients with HM in China, using QLU-C10D and FACT-8D at baseline and follow-up (3–4 months from baseline). We assessed: (i) convergent validity using Spearman's rank correlation test (r) with EQ-5D-5L; (ii) clinical-groups validity by differentiating cancer stages, overall health assessment (OHA), Eastern Cancer Oncology Group (ECOG) performance status, and mental health status. We also examined clinical validity with effect size (ES) and relative efficiency (RE); (iii) responsiveness to changes in patient self-perception using receiver operating characteristics (ROC) curves and area under the curves (AUC); and (iv) agreement using intraclass correlation coefficients (ICC) and visualized with Bland-Altman plot. Results: Among the 308 patients with HM at baseline, 131 completed the follow-up survey. Agreement between the two measures was high (ICC = 0.76). Both measures were highly correlated with EQ-5D-5 L and significantly differentiated (p < 0.001) among groups categorized by cancer stage, OHA performance status, and mental health. ESs for QLU-C10D were numerically higher for cancer stage, OHA, and performance status (ES = 0.53–1.49), whereas ES was higher for FACT-8D and mental health status (ES = 1.35). Responsiveness was higher for QLU-C10D (AUC = 0.84) compared to FACT-8D (AUC = 0.78). Conclusion: Both QLU-C10D and FACT-8D are valid cancer-specific MAUIs for evaluating patients with HM. However, scholars should consider their slight differences in focus when choosing between the two measures. [ABSTRACT FROM AUTHOR]

Purpose: Non-response (NR) to patient-reported outcome (PRO) questionnaires may cause bias if not handled appropriately. Collecting reasons for NR is recommended, but how reasons for NR are related to missing data mechanisms remains unexplored. We aimed to explore this relationship for intermittent NRs. Methods: Patients with multiple myeloma completed validated PRO questionnaires at enrolment and 12 follow-up time-points. NR was defined as non-completion of a follow-up assessment within seven days, which triggered contact with the patient, recording the reason for missingness and an invitation to complete the questionnaire (denoted "salvage response"). Mean differences between salvage and previous on-time scores were estimated for groups defined by reasons for NR using linear regression with clustered standard errors. Statistically significant mean differences larger than minimal important difference thresholds were interpreted as "missing not at random" (MNAR) mechanism (i.e. assumed to be related to declining health), and the remainder interpreted as aligned with "missing completely at random" (MCAR) mechanism (i.e. assumed unrelated to changes in health). Results: Most (7228/7534 (96%)) follow-up questionnaires were completed; 11% (802/7534) were salvage responses. Mean salvage scores were compared to previous on-time scores by reason: those due to hospital admission, mental or physical reasons were worse in 10/22 PRO domains; those due to technical difficulties/procedural errors were no different in 21/22 PRO domains; and those due to overlooked/forgotten or other/unspecified reasons were no different in any domains. Conclusion: Intermittent NRs due to hospital admission, mental or physical reasons were aligned with MNAR mechanism for nearly half of PRO domains, while intermittent NRs due to technical difficulties/procedural errors or other/unspecified reasons generally were aligned with MCAR mechanism. [ABSTRACT FROM AUTHOR]

Background: Cost-utility analysis typically relies on preference-based measures (PBMs). While generic PBMs are widely used, disease-specific PBMs can capture aspects relevant for certain patient populations. Here the EORTC QLU-C10D, a cancer-specific PBM based on the QLQ-C30, is validated using Dutch trial data with the EQ-5D-3L as a generic comparator measure. Methods: We retrospectively analysed data from four Dutch randomised controlled trials (RCTs) comprising the EORTC QLQ-C30 and the EQ-5D-3L. Respective Dutch value sets were applied. Correlations between the instruments were calculated for domains and index scores. Bland–Altman plots and intra-class correlations (ICC) displayed agreement between the measures. Independent and paired t-tests, effect sizes and relative validity indices were used to determine the instruments' performance in detecting clinically known-group differences and health changes over time. Results: We analysed data from 602 cancer patients from four different trials. In overall, the EORTC QLU-C10D showed good relative validity with the EQ-5D-3L as a comparator (correlations of index scores r = 0.53–0.75, ICCs 0.686–0.808, conceptually similar domains showed higher correlations than dissimilar domains). Most importantly, it detected 63% of expected clinical group differences and 50% of changes over time in patients undergoing treatment. Both instruments showed poor performance in survivors. Detection rate and measurement efficiency were clearly higher for the QLU-C10D than for the EQ-5D-3L. Conclusions: The Dutch EORTC QLU-C10D showed good comparative validity in patients undergoing treatment. Our results underline the benefit that can be achieved by using a cancer-specific PBM for generating health utilities for cancer patients from a measurement perspective. [ABSTRACT FROM AUTHOR]

Objective: The EORTC QLU-C10D is a new preference-based measure derived from the EORTC QLQ-C30. Country-specific value sets are required to support the cost-utility analysis of cancer-related interventions. This study aimed to generate an EORTC QLU-C10 value set for Hong Kong (HK). Methods: A HK online panel was quota-sampled to achieve an adult general population sample representative by sex and age. Participants were invited to complete an online discrete choice experiment survey. Each participant was asked to complete 16 choice-pairs, randomly assigned from a total of 960 choice-pairs, each comprising two QLU-C10D health states and a duration attribute. Conditional and mixed logistic regression analyses were used to analyse the data. Results: The analysis included data from 1041 respondents who had successfully completed the online survey. The distribution of sex did not differ from that of the general population, but a significant difference was found among age groups. A weighting analysis for non-representative variable (age) was used. Utility decrements were generally monotonic, with the largest decrements for physical functioning (− 0.308), role functioning (− 0.165), and pain (− 0.161). The mean QLU-C10D utility score of the participants was 0.804 (median = 0.838, worst to best = − 0.169 to 1). The value of the worst health state was − 0.223, which was sufficiently lower than 0 (being dead). Conclusions: This study established HK utility weights for the QLU-C10D, which can facilitate cost-utility analyses across cancer-related health programmes and technologies. [ABSTRACT FROM AUTHOR]

Background: Cost-utility analysis generally requires valid preference-based measures (PBMs) to assess the utility of patient health. While generic PBMs are widely used, disease-specific PBMs may capture additional aspects of health relevant for certain patient populations. This study investigates the construct and concurrent criterion validity of the cancer-specific European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Utility-Core 10 dimensions (QLU-C10D) in non-small-cell lung cancer patients. Methods: We retrospectively analysed data from four multicentre LUX-Lung trials, all of which had administered the EORTC Quality of Life Questionnaire (QLQ-C30) and the EQ-5D-3L. We applied six country-specific value sets (Australia, Canada, Italy, the Netherlands, Poland, and the United Kingdom) to both instruments. Criterion validity was assessed via correlations between the instruments' utility scores. Correlations of divergent and convergent domains and Bland-Altman plots investigated construct validity. Floor and ceiling effects were assessed. Results: The comparison of the EORTC QLU-C10D and EQ-5D-3L produced homogenous results for five of the six country tariffs. High correlations of utilities (r > 0.7) were found for all country tariffs except for the Netherlands. Moderate to high correlations of converging domain pairs (r from 0.472 to 0.718) were found with few exceptions, such as the Social Functioning–Usual Activities domain pair (max. r = 0.376). For all but the Dutch tariff, the EORTC QLU-C10D produced consistently lower utility values compared to the EQ-5D-3L (x̄ difference from − 0.082 to 0.033). Floor and ceiling effects were consistently lower for the EORTC QLU-C10D (max. 4.67% for utilities). Conclusions: The six country tariffs showed good psychometric properties for the EORTC QLU-C10D in lung cancer patients. Criterion and construct validity was established. The QLU-C10D showed superior measurement precision towards the upper and lower end of the scale compared to the EQ-5D-3L, which is important when cost-utility analysis seeks to measure health change across the severity spectrum. [ABSTRACT FROM AUTHOR]

Purpose: In this study, we developed Danish utility weights for the European Organisation for Research and Treatment of Cancer (EORTC) QLU-C10D, a cancer-specific utility instrument based on the EORTC QLQ-C30. Methods: Following a standardized methodology, 1001 adult participants from the Danish general population were quota-sampled and completed a cross-sectional web-based survey and discrete choice experiment (DCE). In the DCE, participants considered 16 choice sets constructed from the key 10 dimensions of the QLU-C10D and chose their preferred health state for each one. Utility weights were calculated using conditional logistic regression with correction for non-monotonicity. Results: The sample (n = 1001) was representative of the Danish general population with regard to age and gender. The domains with the largest utility decrements, i.e., the domains with the biggest impact on health utility, were physical functioning (− 0.224), pain (− 0.160), and role functioning (− 0.136). The smallest utility decrements were observed for the domains lack of appetite (− 0.024), sleep disorders (− 0.057), and fatigue (− 0.064). Non-monotonicity of severity levels was observed for the domains sleep disturbances, lack of appetite, and bowel problems. Deviations from monotonicity were not statistically significant. Conclusion: The EORTC QLU-C10D is a relatively new multi-attribute utility instrument and is a promising cancer-specific health technology assessment candidate measure. The country-specific Danish utility weights from this study can be used for cost-utility analyses in Danish patients and for comparison with other country-specific utility data. [ABSTRACT FROM AUTHOR]

Background: Decisions regarding maintenance therapy in patients with multiple myeloma should be based on both treatment efficacy and health-related quality of life (HRQL) consequences. In the CARFI trial, patients with first relapse of multiple myeloma underwent salvage autologous stem cell transplantation (salvage ASCT) before randomization to carfilzomib-dexamethasone maintenance therapy (Kd) or observation. The primary clinical endpoint was time to progression, which was extended by 8 months by Kd. The aim of this paper is to present the all HRQL endpoints of the CARFI trial including the HRQL effect of Kd maintenance therapy relative to observation. The primary HRQL endpoint was assessed by EORTC QLQ-C30 Summary score (QLQ-C30-sum) at 8 months follow-up. A key secondary HRQL endpoint was quality-adjusted progression-free-survival (QAPFS). Methods: HRQL was assessed with EORTC QLQ-C30, EORTC QLQ-MY20 and FACT/GOG-Ntx at randomization and every second month during follow-up. HRQL data were analyzed with linear mixed effect models until 8 months follow-up. QAPFS per individual was calculated by multiplying progression-free survival (PFS) by two quality-adjustment metrics, the QLQ-C30-sum and EORTC Quality of Life Utility Measure-Core 10 dimensions (QLU-C10D). The QAPFS per treatment group was estimated with the Kaplan-Meier method. P < 0.05 was used for statistical significance, and a between-group minimal important difference of 10 points was interpreted as clinically relevant for the QLQ-C30-sum. Results: 168 patients were randomized. HRQL questionnaire compliance was 93%. For the QLQ-C30-sum, the difference of 4.62 points (95% confidence interval (CI) -8.9: -0.4, p = 0.032) was not clinically relevant. PFS was 19.3 months for the Kd maintenance group and 16.8 months for the observation group; difference = 2.5 months (95% CI 0.5; 4.5). QAPFS based on the QLQ-C30-sum for the Kd maintenance group was 18.0 months (95% CI 16.4; 19.6) and for the observation group 15.0 months (95% CI 13.5; 16.5); difference = 3.0 months (95% CI 0.8–5.3). QAPFS based on the QLU-C10D for the Kd maintenance group was 17.5 months (95% CI 15.9; 19.2) and 14.0 months (95% CI 12.4; 15.5) for the observation group; difference = 3.5 months (95% CI 1.1–5.9). Conclusions: Kd maintenance therapy after salvage ASCT did not adversely affect overall HRQL, but adjustment for HRQL reduced the PFS compared to unadjusted PFS. PFS of maintenance therapy should be quality-adjusted to balance the benefits and HRQL impact. [ABSTRACT FROM AUTHOR]

Objectives: To develop a value set reflecting the United States (US) general population's preferences for health states described by the Functional Assessment of Cancer Therapy (FACT) eight-dimensions preference-based multi-attribute utility instrument (FACT-8D), derived from the FACT-General cancer-specific health-related quality-of-life (HRQL) questionnaire. Methods: A US online panel was quota-sampled to achieve a general population sample representative by sex, age (≥ 18 years), race and ethnicity. A discrete choice experiment (DCE) was used to value health states. The valuation task involved choosing between pairs of health states (choice-sets) described by varying levels of the FACT-8D HRQL dimensions and survival (life-years). The DCE included 100 choice-sets; each respondent was randomly allocated 16 choice-sets. Data were analysed using conditional logit regression parameterized to fit the quality-adjusted life-year framework, weighted for sociodemographic variables that were non-representative of the US general population. Preference weights were calculated as the ratio of HRQL-level coefficients to the survival coefficient. Results: 2562 panel members opted in, 2462 (96%) completed at least one choice-set and 2357 (92%) completed 16 choice-sets. Pain and nausea were associated with the largest utility weights, work and sleep had more moderate utility weights, and sadness, worry and support had the smallest utility weights. Within dimensions, more severe HRQL levels were generally associated with larger weights. A preference-weighting algorithm to estimate US utilities from responses to the FACT-General questionnaire was generated. The worst health state's value was −0.33. Conclusions: This value set provides US population utilities for health states defined by the FACT-8D for use in evaluating oncology treatments. [ABSTRACT FROM AUTHOR]

Background: Patient-reported experience measures (PREMs) assess quality-of-care from patients' perspectives. PREMs can be used to enhance patient-centered care and facilitate patient engagement in care. With increasing quality improvement studies in clinical practice, the use of PREMs has surged. As a result, knowledge about stakeholder experiences of using PREMs to assess quality of care across diverse clinical settings is needed to inform PREM implementation efforts. To address this, this review examines the qualitative literature on patient and healthcare provider experiences of using PREMs in clinical practice. Methods: Medline, Embase and PsycInfo were systematically searched from inception to May 2021. Additional searching of reference lists for all included articles and relevant review articles were performed. Retrieved articles were screened for eligibility by one reviewer and 25% cross-checked by a second reviewer across all stages of the review. Full texts meeting eligibility criteria were appraised against the COREQ checklist for quality assessment and thematic analysis was used to analyze textual data extracted from the results. Results: Electronic searches identified 2683 records, of which 20 studies met eligibility criteria. Extracted data were synthesized into six themes: facilitators to PREM implementation; barriers to PREM implementation; healthcare providers' perspectives towards using PREMs; patients' perspectives towards using PREMs; advantages of using PREMs in clinical practice; limitations and practical considerations to reduce resistance of PREM usage. The primary factors facilitating and impeding the use of PREMs include organizational-, staff- and patient-related factors. Conclusion: Results can be used to guide the usage and implementation of PREMs in clinical settings by addressing the identified barriers and building on the perceived benefits to encourage adoption of PREMs. Results around facilitators to PREM implementation and practical considerations could also promote appropriate use of PREMs by healthcare providers, helping to improve practice and the quality of care based on patient feedback. [ABSTRACT FROM AUTHOR]

Purpose: Chemotherapy-induced peripheral neuropathy (CIPN) is a common toxicity of cancer treatment, with potential to significantly impact cancer survivors' long-term quality of life. Patient reported outcome measures (PROMs) are increasingly utilised to evaluate CIPN. However, guidance remains lacking on how to identify fit for purpose PROMs with considerations necessarily differing when used in various research and in-clinic contexts. This study aimed to evaluate evidence about CIPN PROMs measurement properties and propose considerations to optimize CIPN PROM selection for each purpose. Methods: A systematic review was conducted to identify literature assessing measurement properties of CIPN PROMs. These were evaluated against Consensus-based Standards for the selection of health Measurement Instruments (COSMIN) criteria and International Society for Quality of Life minimum standards. Risk of Bias (RoB) was assessed using the COSMIN RoB checklist. Results: Thirty-nine papers evaluating measurement properties of 13 PROMs were included. The European Organization for Research and Treatment of Cancer Quality of Life Chemotherapy-Induced Peripheral Neuropathy Questionnaire (QLQ-CIPN20) and Functional Assessment of Cancer Therapy/Gynecologic Oncology Group-Neurotoxicity (FACT/GOG-Ntx) were the most commonly investigated PROMs and had the most measurement properties meeting established criteria. Conclusion: The use of the QLQ-CIPN20 and FACT/GOG-Ntx to assess CIPN in research settings has the most supporting evidence. However other considerations including study aims, endpoints and target population also factor into PROM selection and need to be considered more often when determining the most suitable outcome measure. Evidence of CIPN PROMs use in clinical practice is limited and their adoption to individual-patient level management requires more evaluation. [ABSTRACT FROM AUTHOR]

Introduction: Failure to incorporate key patient-reported outcome (PRO) content in trial protocols affects the quality and interpretability of the collected data, contributing to research waste. Our group developed evidence-based training specifically addressing PRO components of protocols. We aimed to assess whether 2-day educational workshops improved the PRO completeness of protocols against consensus-based minimum standards provided in the SPIRIT-PRO Extension in 2018. Method: Annual workshops were conducted 2011–2017. Participants were investigators/trialists from cancer clinical trials groups. Although developed before 2018, workshops covered 15/16 SPIRIT-PRO items. Participant feedback immediately post-workshop and, retrospectively, in November 2017 was summarised descriptively. Protocols were evaluated against SPIRIT-PRO by two independent raters for workshop protocols (developed post-workshop by participants) and control protocols (contemporaneous non-workshop protocols). SPIRIT-PRO items were assessed for completeness (0 = not addressed, 10 = fully addressed). Mann–Whitney U tests assessed whether workshop protocols scored higher than controls by item and overall. Results: Participants (n = 107) evaluated the workshop positively. In 2017, 16/41 survey responders (39%) reported never applying in practice; barriers included role restrictions (14/41, 34%) and lack of time (5/41, 12%). SPIRIT-PRO overall scores did not differ between workshop (n = 13, median = 3.81/10, interquartile range = 3.24) and control protocols (n = 9, 3.51/10 (2.14)), (p = 0.35). Workshop protocols scored higher than controls on two items: 'specify PRO concepts/domains' (p = 0.05); 'methods for handling missing data' (p = 0.044). Conclusion: Although participants were highly satisfied with these workshops, the completeness of PRO protocol content generally did not improve. Additional knowledge translation efforts are needed to assist protocol writers address SPIRIT-PRO guidance and avoid research waste that may eventuate from sub-optimal PRO protocol content. [ABSTRACT FROM AUTHOR]

This review of reviews aimed to appraise the use of the CONSORT-PRO Extension as an evaluation tool for assessing the reporting of patient-reported outcome (PROs) in publications, and to describe the reporting of PRO research across reviews. We also outlined how variation in such evaluations impacts knowledge translation and may lead to potential misuse of the CONSORT-PRO Extension. We systematically searched Medline, Pubmed and CINAHL from 2013 to 2025 March 2021 for reviews of the completeness of reporting of PRO endpoints according to CONSORT-PRO criteria. Two reviewers extracted details of each review, the percentage of included studies that addressed each CONSORT-PRO item, and key recommendations from each review. Fourteen reviews met inclusion criteria, and only six of these used the full CONSORT-PRO checklist with minimal justified modifications. The remaining eight studies made significant or unjustified adjustments to the CONSORT-PRO Extension. Review studies also varied in how they scored multi-component CONSORT-PRO items. CONSORT-PRO items were often unreported in trial reports, and certain CONSORT-PRO items were reported less often than others. The reporting of statistical approaches to dealing with missing PRO data were poor in RCTs included in all 14 review articles. Studies reviewing PRO publications often omitted recommended CONSORT-PRO items from their evaluations, which may cause confusion among readers regarding how best to report their PRO research according to the CONSORT-PRO extension. Many trials published since CONSORT-PRO's release did not report recommended CONSORT-PRO items, which may lead to misinterpretation and consequently to research waste. [ABSTRACT FROM AUTHOR]

Background: Detecting anxiety in oncology patients is important, requiring valid yet brief measures. One increasingly popular approach is the Patient Reported Outcomes Measurement Information System (PROMIS); however, its validity is not well established in oncology. We assessed the convergent and criterion validity of PROMIS anxiety measures in an oncology sample. Methods: 132 oncology/haematology outpatients completed the PROMIS Anxiety Computer Adaptive Test (PROMIS-A-CAT) and the 7 item (original) PROMIS Anxiety Short Form (PROMIS-A-SF) along with six well-established measures: Hospital Anxiety and Depression Scale-Anxiety (HADS-A); Generalised Anxiety Disorder-7 (GAD-7); Depression, Anxiety and Stress Scale-Anxiety (DASS-A) and Stress (DASS-S); Distress Thermometer (DT) and PSYCH-6. Correlations, area under the curve (AUC) and diagnostic accuracy statistics were calculated with Structured Clinical Interview as the reference standard. Results: Both PROMIS measures correlated with all legacy measures at p <.001 (Rho =.56–.83). AUCs (>.80) were good for both PROMIS measures and comparable to or better than all legacy measures. At the recommended mild cut-point (55), PROMIS-A-SF had sensitivity (.67) comparable to or better than all the legacy measures, whereas PROMIS-A-CAT sensitivity (.59) was lower than GAD-7 (.67) and HADS-A (.62), but comparable to PSYCH-6 and higher than DASS-A, DASS-S and DT. Sensitivity for both was.79. A reduced cut-point of 51 on both PROMIS measures improved sensitivity (.83–.84) although specificity was only adequate (.61–.62). Conclusions: The convergent and criterion validity of the PROMIS anxiety measures in cancer populations was confirmed as equivalent, but not superior to, established measures (GAD-7 and HADS-A). The PROMIS-A-CAT did not demonstrate clear advantages over PROMIS-A-SF. [ABSTRACT FROM AUTHOR]

A range of anxiety measures is used in oncology but their comparability is unknown. We examined variations in measurement across three commonly used instruments: Hospital Anxiety and Depression-Anxiety subscale (HADS-A); Depression, Anxiety, Stress Scale - Anxiety subscale (DASS-A); and Generalised Anxiety Disorder scale (GAD-7). Participants (n = 164) completed the self-report measures and the Generalised Anxiety Disorder module of the Structured Clinical Interview for DSM-IV (SCID). We performed Rasch analysis and calculated diagnostic accuracy statistics. Instruments measured similar constructs of anxiety, but had different ranges of measurement, with the HADS-A including lower severity symptoms than the other two measures. Anxiety severity was similar for GAD-7 'mild' and HADS-A 'possible' categories, but 'mild' anxiety on the DASS-A represented more severe symptoms. Conversely, DASS-A 'severe' anxiety represented less intense symptoms than GAD-7 'severe' anxiety. Co-calibration indicated a score of eight on the HADS-A was equivalent in anxiety severity to scores of three on the DASS-A and six on the GAD-7. Area under the curve (AUC) was just acceptable for HADS-A and GAD-7 but not DASS-A. The HADS-A, DASS-A and GAD-7 displayed important differences in how they measured anxiety. In particular, categorical classifications of anxiety severity (mild/moderate/severe) were not equivalent across instruments. Thus, prevalence estimates of anxiety symptoms will vary as a consequence of the instrument used. The GAD-7 and HADS-A obtained more similar results and better AUC than the DASS-A. Our co-calibration could be used in future studies and meta-analyses of individual participant data to set cut-off points that provide more consistent classification of anxiety severity. [ABSTRACT FROM AUTHOR]

Purpose: Patient-reported outcome measures (PROMs) are increasingly used in clinical settings to inform individual patient care. In-depth understanding of end-users' experiences may help identify factors that promote or hinder their use in clinical decision-making. We aimed to examine stakeholder perceptions of the utility of using PROMs in clinical practice based on real-life experience. Methods: Systematic review searching Medline, Embase and PsychINFO from inception to May 2021. Qualitative studies examining patients' and/or clinicians' experiences of using PROMs in clinical settings were included. Study screening and data extraction were performed by two independent reviewers. Qualitative data from included studies was analysed thematically. Results: Of 2388 abstracts retrieved, 52 articles reporting 50 studies met eligibility. Five key benefits were identified: (1) promotes active patient involvement (enables goal setting and discussion of sensitive topics); (2) enhances the focus of consultations (prioritizes patient needs); (3) improves quality of care (enables tailored, holistic care and prompts action); (4) enables standardized monitoring of patient outcomes; and (5) enhances the patient–clinician relationship (provides reassurance). Perceived limitations included the capacity of PROMs to shift the focus of consultations; inaccurately estimate problems; raise unrealistic expectations for care; inhibit patient–clinician interaction; lack clinically meaningful information; and not be suitable for all patients. Conclusion: Both patients and clinicians reported benefits of using PROMs across diverse health conditions and clinical settings, but also highlighted several limitations. These limitations shed some light on why PROM use may not always improve patient outcomes and provide considerations for the design and implementation of future PROM initiatives. [ABSTRACT FROM AUTHOR]

Background: The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Utility-Core 10 Dimensions (QLU-C10D) is a novel cancer-specific preference-based measure (PBM) for which value sets are being developed for an increasing number of countries. This is done by obtaining health preferences from the respective general population. There is an ongoing discussion if instead patients suffering from the disease in question should be asked for their preferences. We used the QLU-C10D valuation survey, originally designed for use in the general population, in a sample of cancer patients in Austria to assess the methodology's acceptability and applicability in this target group before obtaining QLU-C10D patient preferences. Methods: The core of the QLU-C10D valuation survey is a discrete choice experiment in which respondents are asked to give preferences for certain health states (described by a relatively large number of 10 quality of life domains) and an associated survival time. They therewith are asked to trade off quality of life against life time. As this might be a very burdensome task for cancer patients undergoing treatment, a cognitive interview was conducted in a pilot sample to assess burden and potential additional needs for explanation in order to be able to use the DCE for the development of QLU-C10D patient preferences. In addition, responses to general feedback questions on the survey were compared against responses from a matched control group from the already completed Austrian general population valuation survey. Results: We included 48 patients (mean age 59.9 years; 46% female). In the cognitive interview, the majority indicated that their experience with the survey was positive (85%) and overall clarity as good (90%). In response to the general feedback questions, patients rated the presentation of the health states less clear than matched controls (p = 0.008). There was no difference between patients and the general population concerning the difficulty in choosing between the health states (p = 0.344). Conclusion: Despite the relatively large number of DCE domains the survey was manageable for patients and allows going on with the QLU-C10D patient valuation study. [ABSTRACT FROM AUTHOR]

Aims: Proxy reports are often used when patients are unable to self-report. It is unclear how proxy measures are currently in use in adult health care and research settings. We aimed to describe how proxy reports are used in these settings, including the use of measures developed specifically for proxy reporting in adult health populations. Methods: We systematically searched Medline, PsycINFO, PsycTESTS, CINAHL and EMBASE from database inception to February 2018. Search terms included a combination of terms for quality of life and health outcomes, proxy-reporters, and health condition terms. The data extracted included clinical context, the name of the proxy measure(s) used and other descriptive data. We determined whether the measures were developed specifically for proxy use or were existing measures adapted for proxy use. Results: The database search identified 17,677 possible articles, from which 14,098 abstracts were reviewed. Of these, 11,763 were excluded and 2335 articles were reviewed in full, with 880 included for data extraction. The most common clinical settings were dementia (30%), geriatrics (15%) and cancer (13%). A majority of articles (51%) were paired studies with proxy and patient responses for the same person on the same measure. Most paired studies (77%) were concordance studies comparing patient and proxy responses on these measures. Discussion: Most published research using proxies has focused on proxy-patient concordance. Relatively few measures used in research with proxies were specifically developed for proxy use. Future work is needed to examine the performance of measures specifically developed for proxies. Systematic review registration: PROSPERO No. CRD42018103179 [ABSTRACT FROM AUTHOR]

Purpose: To ensure clarity in communication in the field of quality of life research, and meaningful use of 'quality of life' as a research outcome, requires two things: awareness that there is a range of conceptualisations and definitions of 'quality of life', and for any particular study, consistency between the way the term is defined and operationalised in that setting. We aimed to identify how frequently research articles described (HR)QOL as a construct of interest, how frequently they referred to "patient-reported outcome (measures)", which patient-reported outcome measures were used, and how (HR)QOL was defined. Methods: We reviewed all Quality of Life Research articles published in 2017 and recorded whether they described health-related quality of life or quality of life as constructs of interest, and/or mentioned the term(s) patient-reported outcome (measures). We recorded definitions of (HR)QOL stated and questionnaires used. We classified articles according to constructs assessed and instruments used, and examined whether articles citing the same definition used the same questionnaires. Results: We reviewed 300 articles; 65% stated that (HR)QOL was a construct of interest, 27% mentioned patient-reported outcome (measures), and 20% mentioned neither. Fifty-one articles provided definitions of (HR)QOL, citing 66 sources, with 11 definitions cited more than once. PROMIS, SF, EQ-5D, and EORTC instruments were the most commonly used. The only definition and questionnaire consistently used together were the WHO definitions/instruments. Conclusion: These results demonstrate considerable heterogeneity in the definition and operationalisation of (HR)QOL, between and within studies. This limits meaningful interpretation of (HR)QOL scores and complicates literature searches. Investigators should define constructs and select instruments aligned with their definitions. [ABSTRACT FROM AUTHOR]

Objective: Non-muscle invasive bladder cancer (NMIBC) is a chronic condition requiring repeated treatment and endoscopic examinations that can occur life-long. In this context, patient-reported outcomes (PROs) are important considerations to patients and managing clinicians. We undertook a systematic review to synthesise PRO results relevant to NMIBC treatment to explore trajectories overtime and differences between treatment options. Methods: We searched databases AMED, MEDLINE, EMbase, PsycINFO, Web of Knowledge and Scopus (inception to 5th December 2019), reference lists and contacted key authors to identify studies that reported PROs after NMIBC treatment. Two reviewers independently applied inclusion and quality criteria and extracted findings. Results for PROs were synthesised for treatment groups across three time periods: acute/during induction therapy; during maintenance therapy; and long-term follow-up (> 1 year). Results: Of 3193 papers screened, 29 were eligible. These provided evidence about induction treatment effects, but few reported maintenance or long-term evidence, and evidence about differences between NMIBC treatment options was lacking. A range of symptoms (pain in bladder area, urinary frequency and urgency, pain or burning during urination) were commonly experienced during and soon after treatment for NMIBC. Less common symptoms included fatigue, disrupted sleep and gastrointestinal problems. Conclusions: Treatments for NMIBC can cause symptoms and functional impairment during the acute treatment phase and reduce quality of life. Clinicians should be aware of these impairments to prepare patients for short-term sequelae and enable those with treatment options to exercise preferences in choosing among them. However, gaps in current evidence limit our understanding of PRO trajectories from diagnosis through to long-term survivorship and treatment effects. [ABSTRACT FROM AUTHOR]

Background: Colorectal cancer (CRC) is prevalent in the developed world. Favourable survival rates highlight the need to better understand CRC survivors' experiences of long-term impacts of treatment, which can in turn inform decision making. This systematic review aimed to identify and synthesise CRC survivors' experiences of long-term impacts on health-related quality of life. Methods: We searched Medline, Embase and PsychINFO from inception to January 2019. Qualitative studies describing CRC survivors' experiences at least 1-year post-treatment were included. Study eligibility, quality assessment (COREQ guidelines), and data synthesis was performed independently by two reviewers and discussed with the study team. Results: Of 1363 papers retrieved, 20 reporting 15 studies met eligibility. Thematic synthesis produced 12 themes: symptoms, physical, social, psychological and sexual functioning, impact on relationships, informal care needs provided by family/friend, supportive care needs provided by healthcare professional, health care experiences, health behaviour, financial toxicity and occupational experiences. Stoma problems (e.g. leakage, skin irritation) were common in ostomates. Survivors with no/reversed stoma experienced unexpected, long-term altered and unpredictable bowel functioning. Survivors often regulated timing, amount and foods consumed to manage bowel functioning. Less common symptoms included fatigue, impaired sleep and anal pain. Stoma problems and altered bowel functioning impaired survivors' physical, social, sexual and psychological functioning. Cognitive functioning and heredity issues were not reported in any paper. Conclusion: CRC survivors experience ongoing symptoms and functioning impairments more than 1-year post-treatment completion. Many survivors find their own ways to manage symptoms rather than seek professional help. Follow-up care for CRC survivors should integrate screening for long-term effects and provide targeted supportive care. [ABSTRACT FROM AUTHOR]

Purpose: Head-to-head comparison of reliability, validity and responsiveness of four patient-reported outcome measures (PROMS) suitable for assessing health-related quality of life (HRQOL) in palliative care settings: EORTC QLQ-C15-PAL, FACT-G7, FACIT-Pal and short-form FACIT-Pal-14.Methods: Secondary analysis of two phase III randomised trials: ketamine for chronic cancer pain, octreotide for vomiting in inoperable malignant bowel obstruction. Sub-groups were defined by Australia-modified Karnofsky performance status (AKPS) and participants' global impression of change (GIC). Two aspects of reliability were assessed: internal consistency (Cronbach alpha, α); test-retest reliability (intra-class correlation coefficient (ICC)) of patients with stable AKPS and those who self-reported 'no change' on GIC. Construct validity was assessed via pre-determined hypotheses about sensitivity of PROM scores to AKPS groups and responsiveness of PROM change scores to GIC groups using analysis of variance.Results: FACIT-Pal had better internal consistency (α ranged 0.59-0.80, 15/18 ≥ 0.70) than QLQ-C15-PAL (0.51-0.85, 4/8 ≥ 0.70) and FACT-G7 (0.54-0.64, 0/2 ≥ 0.70). FACIT scales had better test-retest reliability (FACIT-Pal 11/27 ICCs ≥ 0.70, FACT-G7 2/3 ICCs ≥ 0.70) than QLQ-C15-PAL (2/30 ICCs ≥ 0.70, 18/30 ≤ 0.5). Four scales demonstrated sensitivity to AKPS: QLQ-PAL-15 Physical Functioning and Global QOL, FACT-G Functional Wellbeing and FACIT-Pal Trial Outcome Index (TOI). Nine scales demonstrated responsiveness: three in the ketamine trial population (QLQ-C15-PAL Pain, FACIT-Pal-14, FACT-G7), six in the octreotide trial population (QLQ-C15-PAL Fatigue; FACIT-Pal PalCare, TOI, Total; FACT-G Physical Wellbeing and Total).Conclusions: No PROM was clearly superior, confirming that choosing the best PROM requires careful consideration of the research goals, patient population and the domains of HRQOL targeted by the intervention being investigated. [ABSTRACT FROM AUTHOR]

Purpose: Patient decision-aids (PtDAs) help patients make informed treatment decisions incorporating their values. Health-related quality of life (HRQOL) is sometimes an outcome of PtDA effectiveness trials, but its suitability for this purpose is unclear. We sought to provide insights into this question by critically appraising how randomized controlled trials (RCTs) evaluating PtDA effectiveness measure and report HRQOL.Methods: We conducted a sub-analysis of RCTs included in the 2017 Cochrane review of PtDAs. Trials assessing HRQOL at baseline and post-PtDA, and comparing PtDA with comparison groups were included. Two reviewers independently extracted data and assessed study quality. Analysis was descriptive.Results: Of 105 RCTs, 11 were eligible for inclusion. Patients randomized to PtDAs did not report better HRQOL than those randomized to usual care. While all 11 RCTs adequately described baseline sample characteristics and reported HRQOL results for study groups, few stated a priori HRQOL expectations or hypotheses (36%); made a link between HRQOL and the decision (18%); provided a rationale or justification for HRQOL assessment (18%); provided reason for choice of HRQOL assessment time-points (9%); or adjusted p-values for multiple HRQOL domains and time-points (0%).Discussion: PtDAs did not conclusively impact HRQOL. If this holds generally, then HRQOL is an uninformative endpoint for PtDA effectiveness trials. When planning trials of PtDAs, investigators considering HRQOL endpoints should consider whether and why their PtDA is likely to affect HRQOL in their context, and if so, which specific aspect(s) of HRQOL and at which time-point(s), and ensure HRQOL is assessed accordingly. [ABSTRACT FROM AUTHOR]

Purpose: To assess the convergent validity of the Patient-Reported Outcomes Measurement Information System (PROMIS) depression measures relative to legacy measures and criterion validity against a structured diagnostic interview for depression in an oncology sample.Methods: 132 oncology/haematology outpatients completed the PROMIS Depression Computer Adaptive Test (PROMIS-D-CAT) and PROMIS Depression Short Form (PROMIS-D-SF) along with seven legacy measures: Beck Depression Inventory (BDI); Centre for Epidemiological Studies Depression (CES-D); Depression, Anxiety and Stress Scale; Hospital Anxiety and Depression Scale; Patient Health Questionnaire; Distress Thermometer and PSYCH-6. Correlations, area under the curve (AUC) and diagnostic accuracy statistics were calculated with Structured Clinical Interview as the gold standard.Results: Both PROMIS measures correlated with all legacy measures at p < .001 (ρ = 0.589-0.810) and all AUCs (> 0.800) were comparable. At the cut-off points for mild depression of 53, the PROMIS measures had sensitivity (0.83 for PROMIS-D-CAT and 0.80 for PROMIS-D-SF) similar to or better than 6/7 legacy measures with high negative predictive value (> 90%). At cut-off points of 60 for moderate depression, PROMIS measures had specificity > 90%, similar to or better than all legacy measures and positive predictive value ≥ 0.50 (similar to 5/7 legacy measures).Conclusions: The convergent and criterion validity of the PROMIS depression measures in cancer populations was confirmed, although the optimal cut-off points are not established. PROMIS measures were briefer than BDI-II and CES-D but do not offer any advance in terms of diagnostic accuracy, reduced response burden or cost over other legacy measures of depression in oncology patients. [ABSTRACT FROM AUTHOR]

Background: The EORTC QLU-C10D is a new multi-attribute utility instrument derived from the widely used cancer-specific quality-of-life (QOL) questionnaire, EORTC QLQ-C30. The QLU-C10D contains ten dimensions (Physical, Role, Social and Emotional Functioning; Pain, Fatigue, Sleep, Appetite, Nausea, Bowel Problems), each with four levels. To be used in cost-utility analysis, country-specific valuation sets are required.Objective: The aim of this study was to provide Australian utility weights for the QLU-C10D.Methods: An Australian online panel was quota-sampled to ensure population representativeness by sex and age (≥ 18 years). Participants completed a discrete choice experiment (DCE) consisting of 16 choice-pairs. Each pair comprised two QLU-C10D health states plus life expectancy. Data were analysed using conditional logistic regression, parameterised to fit the quality-adjusted life-year framework. Utility weights were calculated as the ratio of each QOL dimension-level coefficient to the coefficient on life expectancy.Results: A total of 1979 panel members opted in, 1904 (96%) completed at least one choice-pair, and 1846 (93%) completed all 16 choice-pairs. Dimension weights were generally monotonic: poorer levels within each dimension were generally associated with greater utility decrements. The dimensions that impacted most on choice were, in order, Physical Functioning, Pain, Role Functioning and Emotional Functioning. Oncology-relevant dimensions with moderate impact were Nausea and Bowel Problems. Fatigue, Trouble Sleeping and Appetite had relatively small impact. The value of the worst health state was -0.096, somewhat worse than death.Conclusions: This study provides the first country-specific value set for the QLU-C10D, which can facilitate cost-utility analyses when applied to data collected with the EORTC QLQ-C30, prospectively and retrospectively. [ABSTRACT FROM AUTHOR]

Purpose: Gynecologic Cancer Intergroup Symptom Benefit Study (GCIG-SBS) Stage 2 aimed to review, revise, and validate a patient-reported outcome measure (PROM), the Measure of Ovarian Symptoms and Treatment concerns (MOST), developed in GCIG-SBS Stage 1 (MOSTv1, 35 items), and document recurrent ovarian cancer (ROC) symptom burden and benefit.Methods: GCIG-SBS Stage 2 recruited patients with platinum-resistant/refractory ROC (PRR-ROC) or potentially platinum-sensitive ROC with ≥ 3 lines of prior chemotherapy (PPS-ROC ≥ 3). Patients completed MOSTv1, QLQ-C30, QLQ-OV28, and FACT-O/FOSI at baseline and before cycle 3 of chemotherapy (pre-C3), and global assessments of change (MOST-Change) pre-C3. Clinicians rated patients' cancer-related symptoms, performance status, and adverse events. Convergent and divergent validity (Spearman's correlations), discriminative validity (effect sizes between groups classified by clinician-rated characteristics), and responsiveness (paired t tests in patients expected to experience clinically meaningful change) were assessed.Results: Of 948 recruits, 903 completed PROMs at baseline and 685 pre-C3. Baseline symptom burden was substantial for PRR-ROC and PPS-ROC ≥ 3. MOSTv2 has 24 items and five multi-item scales: abdominal symptoms (MOST-Abdo), disease or treatment-related symptoms (MOST-DorT), chemotherapy-related symptoms (MOST-Chemo), psychological symptoms (MOST-Psych), and MOST-Well-being. Correlations confirmed concurrent and divergent validity. Discriminative validity was confirmed by effect sizes that conformed with a priori hypotheses. MOST-Abdo was responsive to improvements in abdominal symptoms and MOST-Chemo detected the adverse effects of chemotherapy.Conclusions: The MOSTv2 validly quantifies patient-reported symptom burden, adverse effects, and symptom benefit in ROC, and as such is fit-for-purpose for clinical trials of palliative chemotherapy in ROC. Further research is required to assess test-retest reliability. [ABSTRACT FROM AUTHOR]

Purpose: Non-muscle invasive bladder cancer (NMIBC) is a chronic condition requiring repeated treatment and endoscopic examinations that can be life-long. In this context, health-related quality of life (HRQOL) is important to patients and managing clinicians, and integral to treatment recommendations for NMIBC. The aim of this study was to develop a conceptual framework of patient-reported NMIBC symptoms, treatment side effects, and HRQOL impacts from three sources: (1) literature, (2) patients and (3) treating clinicians.Methods: First, we undertook a scoping literature review for studies reporting patient-reported outcomes associated with NMIBC. Outcomes were extracted and grouped conceptually. Then, we conducted semi-structured interviews with patients with NMIBC and treating clinicians. Patients were asked about symptoms and HRQOL impacts experienced from their NMIBC and treatments. Clinicians were asked about commonly reported outcomes, and outcomes they felt were important to assess clinically. Interviews were audio recorded, transcribed and content analysed.Results: A total of 125 symptom- and functioning-related expressions from 18 studies, 26 patients and 20 clinicians were coded into three themes and 18 sub-themes. Patients commonly reported blood in urine and frequent urination. Clinicians considered BCG sepsis and flu-like symptoms important outcomes to assess during treatment for NMIBC.Conclusion: Our empirically derived conceptual framework identifies patient-reported outcomes that are important to people with NMIBC, provides the basis for the development of a new NMIBC-specific symptom index, and guides the design of a comprehensive PRO assessment plan for clinical practice in NMIBC and future clinical trials of treatments for NMIBC. [ABSTRACT FROM AUTHOR]

Purpose: This study assessed the uptake of the CONsolidated Standards of Reporting Trials (CONSORT)-Patient-Reported Outcomes (PRO) statement; determined if use of CONSORT-PRO was associated with more complete reporting of PRO endpoints in randomised controlled trials (RCTs) and identified the extent to which high-impact journals publishing RCTs with PRO endpoints endorse CONSORT-PRO.Methods: CONSORT-PRO citations were identified by systematically searching Medline, EMBASE and Google from 2013 (year CONSORT-PRO released) to 17 December 2015. RCTs that cited CONSORT-PRO (cases) were compared to a comparable control sample of RCTs in terms of adherence to CONSORT-PRO using t tests. General linear models assessed the relationship between CONSORT-PRO score and key, pre-specified variables. The 100 highest-impact journals that published RCTs with PRO endpoints (2014-2015) were identified via a systematic Medline search. Instructions for authors were reviewed to determine whether journals endorsed CONSORT-PRO.Results: Total CONSORT-PRO scores ranged from 47 to 100% for cases and 25-96% for controls. Cases had significantly higher total CONSORT-PRO scores compared to controls: t = 2.64, p = 0.01. 'Citing CONSORT-PRO', 'journal endorsing CONSORT-PRO' and 'dedicated PRO paper' were significant predictors of higher CONSORT-PRO adherence score: R 2 = 0.48, p < 0.001. 11/100 top-ranked journals endorsed CONSORT-PRO in their instructions to authors, seven of these journals published RCTs included as cases in this study.Conclusion: This study demonstrated improved PRO reporting associated with journal endorsement and author use of the CONSORT-PRO extension. Despite growing awareness, more work is needed to promote appropriate use of CONSORT-PRO to improve completeness of reporting; in particular, stronger journal endorsement of CONSORT-PRO. [ABSTRACT FROM AUTHOR]

Purpose: Patient-reported outcomes (PROs) provide the patient's perspective of the impact of treatment. Evidence suggests that PRO content of randomised controlled trials (RCTs) protocols is generally sub-optimal. This study aimed to describe and evaluate the PRO-specific content of ovarian cancer RCT protocols.Methods: Published, phase III, ovarian cancer RCTs with PRO endpoints were identified following a systematic search of Medline and Cochrane databases (Jan 2000 to Feb 2016). Corresponding RCT protocols were downloaded (if published) or obtained by contacting authors. Two investigators independently assessed adherence of PRO-specific content of included protocols to a checklist of 58 recommended PRO protocol items currently being developed by the International Society for Quality of Life Research. Discrepancies were resolved with a third investigator.Results: Of 41 eligible trials identified, 26 protocols were assessed (developed 1995-2010). We were unable to obtain the remaining 15 protocols. Protocols addressed a mean of 28 % PRO checklist items (range 8-66 %). Fifteen (58 % of assessed protocols) provided a rationale for PRO assessment, 8 (31 %) described a PRO objective, 24 (92 %) included a PRO assessment schedule, but only 6 (23 %) justified timing of PRO assessments. Twelve protocols (46 %) provided staff data collection instructions, 4 (15 %) included plans for monitoring PRO compliance, and 16 (62 %) included a PRO analysis plan.Conclusions: On average, protocols addressed less than one-third of PRO protocol checklist items. In some cases, key guidance regarding PRO administration was lacking, which may lead to inconsistent and sub-optimal PRO methodology. Efforts are needed to improve PRO protocol content in cancer trials. [ABSTRACT FROM AUTHOR]

Purpose: This study aimed to establish the prevalence, severity, and correlates of psychological distress and impaired generic health-related quality of life (HRQOL) in testicular cancer (TC) survivors.Methods: Men who had completed active anti-cancer treatment for TC between 6 months and 5 years previously showing no evidence of recurrence were recruited from 14 Australian cancer centers from September 2009 to February 2011. Participants completed a self-report questionnaire measuring demographic, disease, and treatment information, psychological distress (i.e., depression, anxiety, and stress; DASS21), generic health-related quality of life (HRQOL; SF-36v2), TC-specific HRQOL (EORTC QLQ-TC26), coping (MAC), social support (DUFSS), and unmet needs (CaSUN).Results: Of 486 eligible TC survivors, 244 (50.2%) completed the questionnaire. Compared with normative data, TC survivors reported: small but statistically significant increases in mean levels of anxiety and depression; a greater prevalence of moderate to extremely severe anxiety (19%) and depression (20%); and significant deficits to mostly mental aspects of generic HRQOL. The most problematic TC-specific HRQOL issues (e.g., fear of recurrence) were also more mental than physical. In multiple regression analyses, the strongest correlates of psychological distress and impaired generic HRQOL were psychosocial (e.g., helpless/hopeless coping and lower social support) rather than disease or treatment factors.Conclusions: Generally, TC survivors appear to experience mild psychological distress and HRQOL impairments, while a vulnerable subgroup experience more severe morbidity.Implications For Cancer Survivors: There is a need to identify TC survivors at risk of poorer outcomes and for interventions to target the areas of greatest impairment (i.e., psychological distress and mental HRQOL). [ABSTRACT FROM AUTHOR]

In 2014, the European Medicines Agency (EMA) released for comment a draft reflection paper on the use of patient-reported outcome (PRO) measures in oncology studies. A twelve-member International Society for Quality of Life Research (ISOQOL) taskforce was convened to coordinate the ISOQOL response. Twenty-one ISOQOL members provided detailed comments and suggestions on the paper: 81 % from academia and 19 % from industry. Taskforce members consolidated and further refined these comments and shared the recommendations with the wider ISOQOL membership. A final response was submitted to the EMA in November 2014. The impending publication of the EMA reflection paper presents a valuable opportunity for ISOQOL to comment on the current direction of EMA PRO guidance and strategy. The EMA paper, although focused on cancer, could serve as a model for using PROs in other conditions, as it provides a useful update surrounding some of the design issues common to all trial research including PRO endpoints. However, we believe there are a number of additional areas in need of greater consideration. The purpose of this commentary is therefore to highlight the strengths of this timely and potentially useful document, but also to outline areas that may warrant further discussion. [ABSTRACT FROM AUTHOR]

Purpose: Fatigue is one of the most common symptoms associated with cancer and its treatment. To obtain a more precise and flexible measure of fatigue, the EORTC Quality of Life Group has developed a computerized adaptive test (CAT) measure of fatigue. This is part of an ongoing project developing a CAT version of the widely used EORTC QLQ-C30 questionnaire. Methods: Based on the literature search and evaluations by experts and patients, 41 new fatigue items were developed (in addition to the three QLQ-C30 fatigue items). Psychometric properties of the items, including evaluations of dimensionality, fit to item response theory (IRT) model, and differential item functioning (DIF), were assessed in an international sample of cancer patients. Results: Responses were obtained from 1,321 cancer patients coming from eight countries. Factor analysis showed that 37 of the items could be included in a unidimensional model (RMSEA = 0.098, TLI = 0.995, CFI = 0.920). Of the 37 items, two were deleted because of poor fit to the IRT model forming the basis for the CAT, and one because of DIF between cancer sites. Conclusions: We have established a 34-item fatigue bank allowing for more precise and flexible measurement of fatigue, while still being backward compatible with the QLQ-C30 fatigue scale. [ABSTRACT FROM AUTHOR]

Objective: The objective of this paper is to inform choice of optimal patient-reported outcome measures (PROMs) of anxiety, depression and general distress for studies evaluating psychosocial interventions for English-speaking adults with heterogenous cancer diagnoses.Methods: A systematic review was conducted to identify all PROMs used to assess anxiety, depression and general distress in randomised controlled trials (RCTs) of psychosocial interventions for people with cancer published between 1999 and May 2009. Candidate PROMs were evaluated for content, evidence of reliability and validity, clinical meaningfulness, comparison data, efficiency, ease of administration, cognitive burden and track record in identifying treatment effects in RCTs of psychosocial interventions. Property ratings were weighted and summed to give an overall score out of 100.Results: The Hospital Anxiety and Depression Scale (HADS) scored highest overall (weighted score = 77.5), followed by the unofficial short-form of the Profile of Mood States (POMS), the POMS-37 (weighted score = 60), and the Centre for Epidemiological Studies Depression Scale (CES-D) and original POMS (weighted score = 55 each).Conclusions: The HADS' efficiency and substantial track record recommend its use where anxiety, mixed affective disorders or general distress are outcomes of interest. However, continuing controversy concerning the HADS depression scale cautions against dependence where depressive disorders are of primary interest. Where cost is a concern, the POMS-37 is recommended to measure anxiety or mixed affective disorders but does not offer a suitable index of general distress and, like the HADS, emphasises anhedonia in measuring depression. Where depression is the sole focus, the CES-D is recommended. [ABSTRACT FROM AUTHOR]

Developments in electronic data collection methods have allowed researchers to generate larger datasets at lower costs, but relatively little is known about the comparative performance of the new methods. This paper considers the comparability of two modes of administration (face-to-face and remote electronic) for the time trade-off. Data were collected from a convenience sample of adults ( n = 135) randomised to either a face-to-face time trade-off or a remote electronic tool. Patterns of responses were considered. For each sample, standard regression analysis was undertaken to generate a valuation set, which were then contrasted. The pattern of responses differed by mode of administration, with the electronic tool yielding larger standard deviations and higher proportions of responses at −1, 0 and 1. The impact of this on the regression was difficult to disentangle from the high variability around individual scores of states, which is a common feature of responses to time trade-off tasks. Under the scoring algorithms generated by mode of administration, the difference between scores exceeded 0.1 for 100 of the 243 EQ-5D health states. This comparison demonstrates that variability arising from mode of administration needs to be considered in developing health state valuations. While electronic administration has considerable cost advantages, particular attention to the design of the task is required. This has wider implications, as all modes of administration may have mode-specific impacts on the distribution of valuation responses. [ABSTRACT FROM AUTHOR]

The article discusses the use and abuse of subgroup analyses. The article focuses on the 1988 Lancet publication of a large randomised clinical trial. Also discussed are the problems of multiple testing when using of patient reported outcomes (PROs) in clinical trials and independent hypothesis tests.

Cross-sectional surveys depend on retrospective health transition questions (HTQ) to estimate recent changes in health status. This paper assesses the validity of the SF-36 HTQ and calibrates its categories against change assessed prospectively on the SF-36 domain scales in a sub-group known to have experienced clinically important changes in health status. Adults ( n = 9,649) from a longitudinal population survey completed the SF-36 in 2001 and 2002. Prospective measures were calculated as mean changes in SF-36 scale scores adjusted for age and gender, and also expressed as standardised response means. Comparison groups were those who had developed a long-term health condition since the last interview and the HTQ response categories for those who had not developed any new conditions. Those with a new condition and those without a new condition but who described their health as “somewhat worse” than a year ago had comparable declines in health status on all domain scales except role physical, where those with a new condition experienced a greater decline. This analysis demonstrates the validity and limitations of the HTQ as a measure of change in population studies. The calibration is useful for interpreting the meaning of the HTQ categories at the group level but not at the individual level. [ABSTRACT FROM AUTHOR]

To further our understanding of the relationships between asthma control and health-related quality of life (HRQOL) and provide insights into the relative usefulness of various measures in different research contexts. We present a conceptual model and test it with longitudinal survey data. Participants recruited via population sampling and hospital Emergency Departments completed questionnaires every 6 months for up to 3 years. Measures included: sleep disturbance, use of short-acting beta agonists (SABA), activity limitation, urgent medical visits, hospital use, Marks’ Asthma Quality of Life Questionnaire (AQLQ-M) and the SF-36 Health Survey. Correlation analysis and multi-level models tested predictions from the conceptual model. A total of 213 people with asthma aged 16–75 years provided 967 observations. Correlations between asthma control and asthma-specific HRQOL were stronger than those between asthma control and generic HRQOL. The asthma control variables explained 54–58% of the variance in asthma-specific HRQOL and 8–25% of the variance in generic HRQOL. Activity limitation was the main contributor to between-person variation, while sleep disturbance and SABA use were the main contributors to within-person variation. Sleep disturbance and SABA use may be most useful in evaluating treatment effectiveness, while activity limitation may be better when monitoring the impact of asthma in populations. [ABSTRACT FROM AUTHOR]

Purpose: To validate two health-related quality of life (HRQOL) measures, the PedsQL Generic Core and Cancer Module adolescent forms (13-18 years), after modification for 16-25-year-old adolescents and young adults (AYA) with cancer or a blood disorder.Methods: AYA patients and nominated proxies were recruited from three Sydney hospitals. Modified forms were administered by telephone or in clinics/wards. Analyses included correlations, factor analysis, and analysis of variance of known-groups (defined by the Memorial Symptom Assessment Scale).Results: Eighty-eight patients and 79 proxies completed questionnaires. Factor structures consistent with those of the unmodified forms confirmed construct validity. Cronbach's alpha ranged 0.81-0.98. Inter-scale correlations were as hypothesized, confirming discriminant validity. Statistically significant differences between groups with mild, moderate, and severe symptoms (P < 0.05) confirmed clinical validity.Conclusion: These modified forms provide reliable and valid measures of HRQOL in AYA with cancer or a blood disorder, suitable for clinical trials, research, and practice. [ABSTRACT FROM AUTHOR]

Objective: Non-muscle invasive bladder cancer (NMIBC) is a chronic condition requiring ongoing treatment and endoscopic examinations that are frequent and can be life-long. To ensure the comprehensive assessment of the benefits and harms of treatments for NMIBC, the impact on important and relevant patient-reported outcomes (PROs) should be determined. We systematically reviewed the NMIBC PRO literature to determine the suitability of available PRO measures (PROMs) for use in evaluating patient outcomes in NMIBC research.Methods: We searched six electronic databases, reference lists, and key authors. Two reviewers independently applied inclusion and quality criteria and extracted findings. PROM domains, item content, and content coverage and relevance were determined for identified PROMs. Content validity was assessed against an empirically derived NMIBC-specific conceptual framework that includes 11 PRO domains and 19 sub-domains.Results: Seventeen studies assessed PROs related to NMIBC and treatment impact. From these studies, 11 PROMs were identified, including three generic, three cancer-specific, and five symptom-specific. None of the PROMs cover all PRO domains important in NMIBC as assessed against our conceptual framework. The EORTC QLQ-C30 plus the NMIBC24 module was best aligned to the conceptual model, but failed to represent six outcomes important to NMIBC patients.Conclusions: Currently, some outcomes important in NMIBC are inadequately covered by generic and cancer-specific measures despite similar conceptual models. This review identified gaps in the literature regarding assessment of symptoms and other PROs considered important by NMIBC patients. Careful consideration of PROM item content is required when selecting outcome measures for use in future NMIBC clinical trials to ensure that appropriate measures are used to assess outcomes that matter to patients. [ABSTRACT FROM AUTHOR]

Objectives: The aim of the study was to develop and compare utility value sets for the EORTC QLU-C10D, a cancer-specific utility instrument based on the EORTC QLQ-C30, using the preferences of the general public and cancer patients in Singapore, and to assess their measurement properties.A total of 600 individuals from the general public were recruited using a multi-stage random sampling, along with 626 cancer patients with clinically confirmed diagnoses from outpatient clinics of the largest tertiary cancer hospital. Each participant valued 16 pairs of EORTC QLU-C10D health states using a discrete choice experiment (DCE). Conditional logit models were used to analyze the DCE responses of the general public and cancer patients separately. Utility values were assessed for known-group validity and responsiveness in the cancer patients by comparing mean values across subgroups of patients and calculating standardized response means using longitudinal EORTC QLQ-C30 data, respectively.Physical functioning and pain had the most impact on utility for both cancer patients and general public groups. Worst health state utility values were −0.821 and −0.463 for the general public and cancer patients, respectively. Cancer patients’ values were lower for mild-to-moderate health states but higher for moderately-to-highly impaired states compared with the general public’s values. Both value sets discriminated between patients with differing characteristics and responded equally well to improved health status, but the cancer patients’ value set was slightly more responsive to deteriorated health.EORTC QLU-C10D value sets based on the preferences of the Singaporean general public and cancer patients exhibited differences in values but similar psychometric properties.Methods: The aim of the study was to develop and compare utility value sets for the EORTC QLU-C10D, a cancer-specific utility instrument based on the EORTC QLQ-C30, using the preferences of the general public and cancer patients in Singapore, and to assess their measurement properties.A total of 600 individuals from the general public were recruited using a multi-stage random sampling, along with 626 cancer patients with clinically confirmed diagnoses from outpatient clinics of the largest tertiary cancer hospital. Each participant valued 16 pairs of EORTC QLU-C10D health states using a discrete choice experiment (DCE). Conditional logit models were used to analyze the DCE responses of the general public and cancer patients separately. Utility values were assessed for known-group validity and responsiveness in the cancer patients by comparing mean values across subgroups of patients and calculating standardized response means using longitudinal EORTC QLQ-C30 data, respectively.Physical functioning and pain had the most impact on utility for both cancer patients and general public groups. Worst health state utility values were −0.821 and −0.463 for the general public and cancer patients, respectively. Cancer patients’ values were lower for mild-to-moderate health states but higher for moderately-to-highly impaired states compared with the general public’s values. Both value sets discriminated between patients with differing characteristics and responded equally well to improved health status, but the cancer patients’ value set was slightly more responsive to deteriorated health.EORTC QLU-C10D value sets based on the preferences of the Singaporean general public and cancer patients exhibited differences in values but similar psychometric properties.Results: The aim of the study was to develop and compare utility value sets for the EORTC QLU-C10D, a cancer-specific utility instrument based on the EORTC QLQ-C30, using the preferences of the general public and cancer patients in Singapore, and to assess their measurement properties.A total of 600 individuals from the general public were recruited using a multi-stage random sampling, along with 626 cancer patients with clinically confirmed diagnoses from outpatient clinics of the largest tertiary cancer hospital. Each participant valued 16 pairs of EORTC QLU-C10D health states using a discrete choice experiment (DCE). Conditional logit models were used to analyze the DCE responses of the general public and cancer patients separately. Utility values were assessed for known-group validity and responsiveness in the cancer patients by comparing mean values across subgroups of patients and calculating standardized response means using longitudinal EORTC QLQ-C30 data, respectively.Physical functioning and pain had the most impact on utility for both cancer patients and general public groups. Worst health state utility values were −0.821 and −0.463 for the general public and cancer patients, respectively. Cancer patients’ values were lower for mild-to-moderate health states but higher for moderately-to-highly impaired states compared with the general public’s values. Both value sets discriminated between patients with differing characteristics and responded equally well to improved health status, but the cancer patients’ value set was slightly more responsive to deteriorated health.EORTC QLU-C10D value sets based on the preferences of the Singaporean general public and cancer patients exhibited differences in values but similar psychometric properties.Conclusions: The aim of the study was to develop and compare utility value sets for the EORTC QLU-C10D, a cancer-specific utility instrument based on the EORTC QLQ-C30, using the preferences of the general public and cancer patients in Singapore, and to assess their measurement properties.A total of 600 individuals from the general public were recruited using a multi-stage random sampling, along with 626 cancer patients with clinically confirmed diagnoses from outpatient clinics of the largest tertiary cancer hospital. Each participant valued 16 pairs of EORTC QLU-C10D health states using a discrete choice experiment (DCE). Conditional logit models were used to analyze the DCE responses of the general public and cancer patients separately. Utility values were assessed for known-group validity and responsiveness in the cancer patients by comparing mean values across subgroups of patients and calculating standardized response means using longitudinal EORTC QLQ-C30 data, respectively.Physical functioning and pain had the most impact on utility for both cancer patients and general public groups. Worst health state utility values were −0.821 and −0.463 for the general public and cancer patients, respectively. Cancer patients’ values were lower for mild-to-moderate health states but higher for moderately-to-highly impaired states compared with the general public’s values. Both value sets discriminated between patients with differing characteristics and responded equally well to improved health status, but the cancer patients’ value set was slightly more responsive to deteriorated health.EORTC QLU-C10D value sets based on the preferences of the Singaporean general public and cancer patients exhibited differences in values but similar psychometric properties. [ABSTRACT FROM AUTHOR]

Objective: The aim of this study is to provide Chinese utility weights for the European Organization for Research and Treatment of Cancer Quality of Life Utility Measure-Core 10 Dimensions (EORTC QLU-C10D) which is a preference-based cancer-specific utility instrument derived from the EORTC QLQ-C30.We conducted an online survey of the general population in China, with quota sampling for age and gender. Each respondent was asked to complete a discrete choice experimental survey consisting of 16 randomly selected choice sets. The conditional logit model and mixed logit model were used to analyze respondents’ preferences, and the goodness of fit of the model was tested.A total of 2003 respondents were included in the analysis. Utility decrements within dimensions were typically monotonic. Monotonic inconsistency issues in the Fatigue, Sleep, and Nausea dimensions were normalized by monotonicity correction. Physical functioning, Pain, and Role functioning were associated with the greatest utility weights, with the smallest decrements being in Bowel problems and Emotional functioning. The utility value for the worst health state was 0.083, i.e. slightly higher than being dead.This study provides the first China-specific set of value for the QLU-C10D based on societal preferences of the Chinese adult general population. The value set can be used as a cancer-specific scoring system for economic evaluations of new oncology therapies and technologies in China.Methods: The aim of this study is to provide Chinese utility weights for the European Organization for Research and Treatment of Cancer Quality of Life Utility Measure-Core 10 Dimensions (EORTC QLU-C10D) which is a preference-based cancer-specific utility instrument derived from the EORTC QLQ-C30.We conducted an online survey of the general population in China, with quota sampling for age and gender. Each respondent was asked to complete a discrete choice experimental survey consisting of 16 randomly selected choice sets. The conditional logit model and mixed logit model were used to analyze respondents’ preferences, and the goodness of fit of the model was tested.A total of 2003 respondents were included in the analysis. Utility decrements within dimensions were typically monotonic. Monotonic inconsistency issues in the Fatigue, Sleep, and Nausea dimensions were normalized by monotonicity correction. Physical functioning, Pain, and Role functioning were associated with the greatest utility weights, with the smallest decrements being in Bowel problems and Emotional functioning. The utility value for the worst health state was 0.083, i.e. slightly higher than being dead.This study provides the first China-specific set of value for the QLU-C10D based on societal preferences of the Chinese adult general population. The value set can be used as a cancer-specific scoring system for economic evaluations of new oncology therapies and technologies in China.Results: The aim of this study is to provide Chinese utility weights for the European Organization for Research and Treatment of Cancer Quality of Life Utility Measure-Core 10 Dimensions (EORTC QLU-C10D) which is a preference-based cancer-specific utility instrument derived from the EORTC QLQ-C30.We conducted an online survey of the general population in China, with quota sampling for age and gender. Each respondent was asked to complete a discrete choice experimental survey consisting of 16 randomly selected choice sets. The conditional logit model and mixed logit model were used to analyze respondents’ preferences, and the goodness of fit of the model was tested.A total of 2003 respondents were included in the analysis. Utility decrements within dimensions were typically monotonic. Monotonic inconsistency issues in the Fatigue, Sleep, and Nausea dimensions were normalized by monotonicity correction. Physical functioning, Pain, and Role functioning were associated with the greatest utility weights, with the smallest decrements being in Bowel problems and Emotional functioning. The utility value for the worst health state was 0.083, i.e. slightly higher than being dead.This study provides the first China-specific set of value for the QLU-C10D based on societal preferences of the Chinese adult general population. The value set can be used as a cancer-specific scoring system for economic evaluations of new oncology therapies and technologies in China.Conclusions: The aim of this study is to provide Chinese utility weights for the European Organization for Research and Treatment of Cancer Quality of Life Utility Measure-Core 10 Dimensions (EORTC QLU-C10D) which is a preference-based cancer-specific utility instrument derived from the EORTC QLQ-C30.We conducted an online survey of the general population in China, with quota sampling for age and gender. Each respondent was asked to complete a discrete choice experimental survey consisting of 16 randomly selected choice sets. The conditional logit model and mixed logit model were used to analyze respondents’ preferences, and the goodness of fit of the model was tested.A total of 2003 respondents were included in the analysis. Utility decrements within dimensions were typically monotonic. Monotonic inconsistency issues in the Fatigue, Sleep, and Nausea dimensions were normalized by monotonicity correction. Physical functioning, Pain, and Role functioning were associated with the greatest utility weights, with the smallest decrements being in Bowel problems and Emotional functioning. The utility value for the worst health state was 0.083, i.e. slightly higher than being dead.This study provides the first China-specific set of value for the QLU-C10D based on societal preferences of the Chinese adult general population. The value set can be used as a cancer-specific scoring system for economic evaluations of new oncology therapies and technologies in China. [ABSTRACT FROM AUTHOR]