General population normative values for the EORTC QLQ-C30 by age, sex, and health condition for the French general population.

BACKGROUND: General population normative values for the widely used health-related quality of life (HRQoL) measure EORTC QLQ-C30 support the interpretation of trial results and HRQoL of patients in clinical practice. Here, we provide sex-, age- and health condition-specific normative values for the EORTC QLQ-C30 in the French general population. METHODS: French general population data was collected in an international EORTC project. Online panels with quota samples were used to recruit sex and age groups. Number and type of comorbidities were assessed. Descriptive statistics were used to calculate general population values for each QLQ-C30 scale, separately for sex, age, and presence of one- and more chronic health conditions. A multivariate linear regression model has been developed to allow estimating the effect of sex, age, and the presence for one- and more chronic health conditions on EORTC QLQ-C30 scores. Data was weighted according to United Nation statistics adjusting for the proportion of sex and age groups. RESULTS: In total, 1001 French respondents were included in our analyses. The weighted mean age was 47.9 years, 514 (51.3%) participants were women, and 497 (52.2%) participants reported at least one health condition. Men reported statistically significant better scores for Emotional Functioning (+9.6 points, p = 0.006) and Fatigue (-7.8 point; p = 0.04); women reported better profiles for Role Functioning (+8.7 points; p = 0.008) and Financial Difficulty (-7.8 points, p = 0.011). According to the regression model, the sex effect was statistically significant in eight scales; the effect of increasing age had a statistically significant effect on seven of the 15 EORTC QLQ-C30 scales. The sex- and age effect varied in its direction across the various scales. The presence of health conditions showed a strong negative effect on all scales. CONCLUSION: This is the first publication of detailed French normative values for the EORTC QLQ-C30. It aims to support the interpretation of HRQoL profiles in French cancer populations. The strong impact of health conditions on QLQ-C30 scores highlights the importance of considering the impact of comorbidities in cancer patients when interpreting HRQoL data.

EORTC QLQ-C30 general population normative data for the United States.

OBJECTIVE: The European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 is a frequently used cancer-specific health-related quality of life (HRQoL) questionnaire. To aid interpretation of data obtained via EORTC QLQ-C30, general population norm data have been published for many countries. However, despite its frequent use in the United States, no normative data by sex and age exist to date. Therefore, this study aimed to generate sex- and age-specific EORTC QLQ-C30 normative data for the United States. METHODS: Recruitment and data collection were carried out via online panels as part of a larger cross-sectional study. For the recruitment, the sample was stratified by sex and age (18-39, 40-49, 50-59, 60-69, ≥ 70 years) to achieve a balanced distribution, with n = 100 per subgroup. Descriptive statistics are presented by age and age/sex. RESULTS: A total of N = 1009 respondents completed the survey (n = 508 females, n = 501 males). More than two thirds of participants (72.5%) reported at least one health condition, e.g., arthritis (26%). Across EORTC QLQ-C30 scales, women and men 60 years and older reported generally better/higher functioning and better/lower symptom scores compared to the younger age groups. CONCLUSION: To date, no specific EORTC QLQ-C30 general population normative data have been published for the United States. This paper provides these important normative data, which will greatly support the interpretation of EORTC QLQ-C30 scale scores obtained from US cancer patients, and also enable comparison with European norms.

EORTC QLQ-C30 normative data for the United Kingdom: Results of a cross-sectional survey of the general population.

OBJECTIVE: The cancer-specific health-related quality of life (HRQoL) questionnaire of the European Organisation for Research and Treatment of Cancer (EORTC), the EORTC QLQ-C30, is a frequently applied questionnaire to assess cancer patients' self-reported health used as part of research and clinical practice. Normative data obtained from the general population can facilitate the interpretation of these data. Despite its frequent application, no detailed EORTC QLQ-C30 normative data have yet been published for the United Kingdom (UK). This study presents detailed EORTC QLQ-C30 normative data for the United Kingdom overall and by sex and age. METHODS: The data are drawn from a larger published, international, cross-sectional online survey. For the recruitment, the sample was stratified by sex (males, females) and age in five age groups with a sample size of n = 100 per subgroup. RESULTS: A total of N = 1026 UK respondents completed the survey (n = 517 females, n = 509 males). There were no clear subgroup patterns by sex or age; however, older patients tended to show higher (i.e., better) scores in emotional and social functioning; they also reported some of the lowest (i.e., best) scores for symptoms, such as insomnia, appetite loss, diarrhoea, nausea/vomiting or financial difficulties. CONCLUSION: This paper provides EORTC QLQ-C30 general population normative data for the UK, further stratified by sex and age. These data will greatly support the interpretation of EORTC QLQ-C30 scale scores obtained from UK cancer patients, and also enable comparison with other detailed national normative datasets collected in the same project, across several other European countries and the US.

The use of youth healthcare services and its association with health-related quality of life, physical and mental health and over-the-counter analgesics use in 13-19-year-old adolescents: a cross-sectional study.

BACKGROUND: Youth healthcare services in Norway include a public health nurse (PHN) at school and local youth health centres (YHCs). They provide health services for all adolescents free of charge, focusing on health promotion and disease prevention. The present study aimed to assess possible associations between health-related quality of life (HRQoL), physical and mental health, over-the-counter analgesics (OTCA) use and use of youth healthcare services among 13-19-year-old adolescents. METHODS: This study was based on national, cross-sectional data from the Ungdata Survey conducted in 2022. The sample was comprised of 16 482 adolescents. Multiple logistic regression was used to analyse the associations between HRQoL, headaches, selected physical symptoms, psychological distress, use of OTCA, PHN availability, sociodemographic variables, and use of the PHN at school or at a YHC. The KIDSCREEN-10 was used to measure HRQoL, and the Hopkins Symptoms Checklist 10 was used to measure symptoms of psychological distress. RESULTS: Girls used the youth healthcare services more frequently than boys. Better HRQoL was significantly associated with fewer visits to the PHN at school. Girls reported lower HRQoL and mental health, and more pain and frequent OTCA use than boys. When having symptoms of psychological distress, boys had greater odds of visiting the PHN at school than girls. For girls in senior high school, headaches and OTCA use were strongly associated with visiting the PHN at school and the YHC. In senior high school, boys with an immigration background had greater odds of visiting the YHC than native Norwegian boys, while girls with an immigration background were less likely to visit the YHC than native Norwegian girls. CONCLUSIONS: Our results show that more girls than boys use youth healthcare services. When adolescents experience pain, have mental problems, use OTCA, or report low levels of HRQoL, they have greater odds of using youth healthcare services. Youth healthcare services offer excellent opportunities to support and follow up with adolescents. The findings provide important insights into youth healthcare services used by adolescents for various stakeholders, including PHNs and policy makers, with potential implications for future public health efforts.

Comparison between Swedish EORTC QLQ-C30 general population norm data published in 2000 and 2019.

BACKGROUND: Normative health-related quality of life (HRQoL) data from the general population are regularly used to facilitate the interpretation of HRQoL as reported by cancer patients participating in cancer clinical trials, especially when conducting long-term follow-up studies after treatment. The aim of the present study is to compare two Swedish normative data sets, published in 2000 and 2019 respectively, and explore whether HRQoL as reported by the Swedish general population has changed over time. MATERIAL AND METHODS: 'Sample 2000' was comprised of normative data from the Swedish general population who responded to the EORTC QLQ-C30 in a Swedish mail survey in 1999 (n = 3069). 'Sample 2019' consisted of data from the Swedish general population collected as part of a European norm data study using online panels, published in 2019 (n = 1027). Data were analyzed stratified by sex and age (40-49; 50-59; 60-69; 70-79 years). RESULTS: For most of the subscales and single items, no age group differences between the two samples were found, with the exception of the oldest age group (70-79 years), where Sample 2019 generally showed better HRQoL as compared to Sample 2000. Lower (worse) levels of Global quality of life and higher (worse) levels of Dyspnoea were found in Sample 2019 for most age groups. CONCLUSION: There were no differences found between the samples for most EORTC QLQ-C30 subscales and single items, with the exception of the oldest age group of both sexes in Sample 2019 who reported better HRQoL on many variables. When deciding which normative dataset to use, the mode of data collection and age group have to be considered.

Psychometric properties of the Alzheimer's Disease Cooperative Study - Activities of Daily Living for Mild Cognitive Impairment (ADCS-MCI-ADL) scale: a post hoc analysis of the ADCS ADC-008 trial.

BACKGROUND: The Alzheimer's Disease Cooperative Study - Activities of Daily Living Scale for use in Mild Cognitive Impairment (MCI), the ADCS-ADL-MCI, is an evaluation scale with information provided by an informant/caregiver to describe the functional impairment of patients with MCI. As the ADCS-ADL-MCI has yet to undergo a full psychometric evaluation, this study aimed to evaluate the measurement properties of the ADCS-ADL-MCI in subjects with amnestic MCI. METHODS: Measurement properties, including item-level analysis, internal consistency reliability, test-retest reliability, construct validity (convergent/discriminant, known-groups validity), and responsiveness were evaluated using data from the ADCS ADC-008 trial, a 36-month, multicenter, placebo-controlled study in 769 subjects with amnestic MCI (defined by clinical criteria and a global clinical dementia rating, CDR, score of 0.5). Due to most subjects' mild condition at baseline and resulting low variance in scores, psychometric properties were assessed using both baseline and 36-month data. RESULTS: Ceiling effects were not apparent at the total score level, with 3% of the cohort reaching the maximum score of 53, despite most subjects having a relatively high score at baseline (mean score = 46.0 [standard deviation = 4.8]). Item-total correlations were overall weak at baseline, most likely due to low variability in responses; however, at month 36, good item homogeneity was found. Cronbach's alpha values ranged from acceptable (0.64 at baseline) to good (0.87 at month 36), indicating overall very good internal consistency reliability. Further, moderate to good test-retest reliability was found (intraclass correlation coefficients ranging from 0.62-0.73). The analyses also largely supported convergent/discriminant validity, especially at month 36. Finally, the ADCS-ADL-MCI discriminated well between groups showing good known-groups validity, and was responsive in patients who indicated a longitudinal change in other instruments. CONCLUSIONS: This study provides a comprehensive psychometric evaluation of the ADCS-ADL-MCI. Findings suggest that the ADCS-ADL-MCI is a reliable, valid and responsive measure capable of capturing functional abilities in patients with amnestic MCI. TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT00000173.

Differences in Quality of Life between German and Dutch Patients with Prostate Cancer Treated with Robot-assisted Radical Prostatectomy: Implications for International Multicenter Randomized Controlled Trials.

BACKGROUND: As a local treatment for prostate cancer (PCa), robot-assisted radical prostatectomy (RARP) may have a quality of life (QoL) benefit over open surgery. Recent analyses revealed substantial between-country differences in the function and symptom scale scores for the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30), which is typically used to assess patient-reported QoL. Such differences could have implications for multinational studies in PCa. OBJECTIVE: To examine whether nationality is significantly associated with patient-reported QoL. DESIGN, SETTING, AND PARTICIPANTS: The study cohort comprised Dutch and German patients with PCa treated with RARP in a single high-volume prostate center from 2006 to 2018. Analyses were restricted to patients who were preoperatively continent with at least one follow-up time point. OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS: QoL was measured in terms of the global Quality of Life (QL) scale score and the overall summary score for the EORTC QLQ-C30. Linear mixed models for repeated-measures multivariable analyses (MVAs) were used to examine the association between nationality and both the global QL score and the summary score. MVAs were further adjusted for QLQ-C30 baseline values, age, Charlson comorbidity index, preoperative prostate-specific antigen, surgical expertise, pathological tumor and nodal stage, Gleason grade, degree of nerve-sparing, surgical margin status, 30-d Clavien-Dindo grade complications, urinary continence recovery, and biochemical recurrence/postoperative radiotherapy. RESULTS AND LIMITATIONS: For Dutch (n = 1938) versus German (n = 6410) men, the mean baseline scores were 82.8 versus 71.9 for the global QL scale score and 93.4 versus 89.7 for the QLQ-C30 summary score. Urinary continence recovery (QL: +8.9, 95% confidence interval [CI] 8.1-9.8; p < 0001) and Dutch nationality (QL: +6.9, 95% CI 6.1-7.6; p < 0001) were the strongest positive contributors to the global QL and summary scores, respectively. The main limitation is the retrospective study design. In addition, our Dutch cohort may not be representative of the general Dutch population and reporting bias cannot be ruled out. CONCLUSIONS: Our findings provide observational evidence under specific conditions involving the same setting for patients of two different nationalities suggesting that cross-national patient-reported QoL differences appear to be real and may need to be taken into consideration in multinational studies. PATIENT SUMMARY: We observed differences in the quality-of-life scores reported by Dutch and German patients with prostate cancer after they underwent robot-assisted removal of the prostate. These findings should be taken into consideration in cross-national studies.

The European Organisation for Research and Treatment of Cancer Quality of Life Utility-Core 10 Dimensions: Development and Investigation of General Population Utility Norms for Canada, France, Germany, Italy, Poland, and the United Kingdom.

OBJECTIVES: The European Organisation for Research and Treatment of Cancer Quality of Life Utility-Core 10 Dimensions (EORTC QLU-C10D) is a cancer-specific preference-based measure, providing health utilities for use in economic evaluations derived from the widely used health-related quality of life measure, EORTC QLQ-C30. Several EORTC QLU-C10D country-specific value sets are available. This article aimed to provide EORTC QLU-C10D general population utility norms for Canada, France, Germany, Italy, Poland, and the United Kingdom, to aid interpretability of obtained utilities in these countries. METHODS: Data were collected in aforementioned countries via a quota-sampled, cross-sectional online survey (n = 100/age-sex group; N = approximately 1000/country). Participants were asked to complete the EORTC QLQ-C30 and provide sociodemographic data. Country-specific utility norms were calculated using the respective country tariff on the country's EORTC QLQ-C30 data after weighting to achieve population representativeness for age and sex. Norm values are provided as means (SDs) by country, age, and sex groups. Tukey's multiple comparison test investigated mean differences among countries. The impact of country, age, and sex on utility values was investigated with a multiple linear regression model. RESULTS: Country-specific mean utilities range from 0.724 (United Kingdom) to 0.843 (Italy). Country-, sex-, and age-specific mean utilities range from 0.664 for 30- to 39-year-old male Canadians to 0.899 for > 70-year-old male Italians. Utilities were lower in females in 4 of 6 countries, and the impact of age differed among countries. Independent of the impact of age and sex, between-country differences were found (P ≤ .05). CONCLUSION: Results showed a varying impact of age and sex on EORTC QLU-C10D utilities and significant between-country differences. Using national utility norms and utility decrements is recommended.

Modelling variance in the multidimensional Health Literacy Questionnaire: Does a General Health Literacy factor account for observed interscale correlations?

Objectives: Self-report measures of health literacy frequently encompass a group of conceptually distinct but related domains scored as either a single scale or separately for each domain. Psychometric studies of the 44-item Health Literacy Questionnaire, scored as nine separate scales, typically report the results of correlated factors confirmatory factor analyses as validity evidence. However, some scales are quite strongly correlated, raising the question of whether there is sufficient discriminant validity to warrant separate scoring. This psychometric study aims to contrast the results of fitting higher-order and bifactor models as alternative options to illuminate the issue. Methods: Correlated factors, higher-order and bifactor confirmatory factor analysis models were fitted to an Australian sample of responses to the Health Literacy Questionnaire (N = 813) using Bayesian confirmatory factor analysis methods. Results: All models representing a nine-factor structure for the Health Literacy Questionnaire fitted well. The correlated factors model replicated previous findings, showing inter-factor correlations between 0.19 and 0.93. A higher-order model showed relatively high loadings of all nine first-order factors on the second-order factor with particularly high loadings (⩾0.97) for three. Two bifactor models showed that the majority of Health Literacy Questionnaire items were multifactorial, each containing systematic variance from both a General Health Literacy factor and a domain-specific factor. Seven items from four scales were identified as strongly associated with the General Health Literacy factor, with item content suggesting that this factor indexes a broad sense of agency and efficacy in interacting with health-related information and healthcare providers. Conclusion: Contrasting correlated factors, higher-order and bifactor models fitted to the Health Literacy Questionnaire suggest that constituent items in self-report health literacy questionnaires might be anticipated to represent at least two sources of reliable and substantive common factor variance: variance associated with General Health Literacy and variance associated with a more specific domain, suggesting that items may be 'irreducibly' heterogeneous. Implications for test development and validation practice are discussed.

Frequently Used Patient-Reported Outcome Measures of General Physical Function Were Highly Correlated With a Multitask Performance Outcome Test Battery.

OBJECTIVES: This study aimed to determine the relationship between frequently used patient-reported outcome (PRO) measures and a multitask performance outcome (PerfO) measure of general physical function (PF) and to examine the association of these measures with depressive mood, pain, and age. METHODS: Frequently used PRO measures of general PF (Patient-Reported Outcomes Measurement Information System [PROMIS] PF item bank, PROMIS PF Short Form 20a, Short Form 36 Physical Function Scale) and a PerfO test battery, namely, the Physical Performance Test (PPT), were administered to 78 adult patients from 3 inpatient clinics (cardiology and angiology, rheumatology and clinical immunology, and psychosomatic medicine) at Charité - Universitätsmedizin Berlin. Pearson correlations were used to investigate the associations between PRO measures and the PPT. To explore the predictive value of age, depressive symptoms, and pain intensity, we conducted multiple linear regression analysis for each PF measure. RESULTS: We found strong linear relationships between PRO measures and PPT sum scores. Correlations between PPT sum scores and PROMIS PF T-scores were r > 0.75. For all PRO and PerfO measures, age was a predictor of general PF whereas depressive mood was not found to be a relevant predictor. Moreover, pain intensity was found to be a significant predictor of PRO measures but not for PPT sum scores. CONCLUSIONS: Our findings suggest that frequently used PRO measures and a multitask PerfO measure of general PF can be used to measure a common PF construct. Nevertheless, PF scores based on PRO measures should ideally be controlled for self-rated pain intensity.

EORTC QLQ-C30 general population normative data for Italy by sex, age and health condition: an analysis of 1,036 individuals.

BACKGROUND: General population normative values for the widely used health-related quality of life (HRQoL) measure, European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire - Core 30 (EORTC QLQ-C30), are available for a range of countries. These are mostly countries in northern Europe. However, there is still a lack of such normative values for southern Europe. Therefore, this study aims to provide sex-, age- and health condition-specific normative values for the general Italian population for the EORTC QLQ-C30. MATERIAL AND METHODS: This study is based on Italian EORTC QLQ-C30 general population data previously collected in an international EORTC project comprising over 15,000 respondents across 15 countries. Recruitment and assessment were carried out via online panels. Quota sampling was used for sex and age groups (18|-|39, 40-49, 50-59, 60-69 and ≥ 70 years), separately for each country. We applied weights to match the age and sex distribution in our sample with UN statistics for Italy. Along with descriptive statistics, linear regression models were estimated to describe the associations of sex, age and health condition with the EORTC QLQ-C30 scores. RESULTS: A total of 1,036 respondents from Italy were included in our analyses. The weighted mean age was 49.3 years, and 536 (51.7%) participants were female. Having at least one health condition was reported by 60.7% of the participants. Men reported better scores than women on all EORTC QLQ-C30 scales but diarrhoea. While the impact of age differed across scales, older age was overall associated with better HRQoL as shown by the summary score. For all scales, differences were in favour of participants who did not report any health condition, compared to those who reported at least one. CONCLUSION: The Italian normative values for the EORTC QLQ-C30 scales support the interpretation of HRQoL profiles in Italian cancer populations. The strong impact of health conditions on EORTC QLQ-C30 scores highlights the importance of adjusting for the impact of comorbidities in cancer patients when interpreting HRQoL data.

Health-related quality of life scores of metastatic pancreatic cancer patients responsive to first line chemotherapy compared to newly derived EORTC QLQ-C30 reference values.

BACKGROUND: Metastatic pancreatic cancer (mPC) and its treatments significantly impact health-related quality of life (HRQoL). POLO, a randomized, double-blind, placebo-controlled phase 3 trial evaluated the efficacy of olaparib as maintenance therapy in germline BRCA mutated mPC patients who had not progressed during ≥16 weeks of first-line platinum-based chemotherapy. HRQoL was assessed using the EORTC QLQ-C30. To enhance score interpretation, we derived reference values for treatment-naïve mPC patients from the literature. METHODS: A targeted literature review identified EORTC QLQ-C30 baseline values in treatment-naïve mPC patients. Reference values were calculated by deriving means from studies meeting inclusion criteria, with scores from 0 to 100 (higher scores indicate better QoL/functioning but worse symptoms). For POLO patients, means were calculated using pooled baseline data across study arms. RESULTS: Four studies met inclusion criteria. Depending on the specific scale, sample sizes ranged from n = 466 to n = 639. Compared to newly derived reference values, POLO patients reported markedly better HRQoL scores at baseline across most scales, with eight scales showing differences of ≥10 points. POLO patients' HRQoL scores were often close to or better than general population norm data. CONCLUSIONS: This is the first study to systematically derive EORTC QLQ-C30 reference values for mPC. POLO patients had better HRQoL scores than those in the literature and similar to general population data. Comparatively high HRQoL of POLO patients are likely due to effects of prior first-line treatment and resolution of chemotherapy-related symptoms, response shift, or a combination. Newly derived reference values can enhance interpretation of mPC patients' HRQoL. TRIAL REGISTRATION: The POLO trial was registered on 9 July 2014 with ClinicalTrials.gov as NCT02184195.

Evaluation of an electronic psycho-oncological adaptive screening program (EPAS) with immediate patient feedback: findings from a German cluster intervention study.

PURPOSE: Distress screening has become mandatory and essential in comprehensive cancer care. We evaluated an electronic psycho-oncological adaptive screening (EPAS) which assesses objective indicators of care needs and subjectively perceived care needs and subsequently provides patient feedback with individualized recommendations about psychosocial care services. METHODS: Patients were assessed within clusters, i.e., different oncological facilities of the competence network of the University Cancer Center Hamburg (UCCH). Patients in the intervention arm underwent the screening, controls received standard care. Patients were assessed at baseline (t0), 3-month (t1), and 6-month (t2) follow-up. Outcomes included information level and use of/access to nine psychosocial services at UCCH, well-being (GAD-7, PHQ-9, SF-8), and treatment satisfaction (SCCC). Conditional linear and logistic regressions were used to identify screening effects at t1 and t2. RESULTS: Of 1320 eligible patients across 11 clusters, 660 were included (50%). The average age was 60 years; 46% were female. The intervention was associated with increased information level for all psychosocial services at t1 and t2 (all p < .001), increased use in some of these services at t1 and t2, respectively (p ≤ .02), and better evaluation of access (e.g., more recommendations for services provided by physicians, p < .01). At t2, the intervention was associated with a lower level of satisfaction with disease-related information (p = .02). CONCLUSIONS: EPAS may improve information about psychosocial services as well as utilization of and access to these services. The effect on information level seems not to be generalizable to other aspects of oncological care. Future studies should incorporate novel technologies and condense the procedure to its core factors. IMPLICATIONS FOR CANCER SURVIVORS: The screening may help to enhance self-management competencies among cancer survivors. TRIAL REGISTRATION: The trial was retrospectively registered (2/2021) at ClinicalTrials.gov (number: NCT04749056).

Investigating the response scale of the EORTC QLQ-C30 in German cancer patients and a population survey.

BACKGROUND: The European Organization for research and Treatment of Cancer (EORTC) Core Quality of Life Questionnaire (QLQ-C30) scales are scored on a 4-point response scale, ranging from not at all to very much. Previous studies have shown that the German translation of the response option quite a bit as mäßig violates interval scale assumptions, and that ziemlich is a more appropriate translation. The present studies investigated differences between the two questionnaire versions. METHODS: The first study employed a balanced cross-over design and included 450 patients with different types of cancer from three German-speaking countries. The second study was a representative survey in Germany including 2033 respondents. The main analyses included compared the ziemlich and mäßig version of the questionnaire using analyses of covariance adjusted for sex, age, and health burden. RESULTS: In accordance with our hypothesis, the adjusted summary score was lower in the mäßig than in the ziemlich version; Study 1: - 4.5 (95% CI - 7.8 to - 1.3), p = 0.006, Study 2: - 3.1 (95% CI - 4.6 to - 1.5), p < 0.001. In both studies, this effect was pronounced in respondents with a higher health burden; Study 1: - 6.8 (95% CI - 12.2 to - 1.4), p = 0.013; Study 2: - 4.5 (95% CI - 7.3 to - 1.7), p = 0.002. CONCLUSIONS: We found subtle but consistent differences between the two questionnaire versions. We recommend to use the optimized response option for the EORTC QLQ-C30 as well as for all other German modules. TRIAL REGISTRATION: The study was retrospectively registered on the German Registry for Clinical Studies (reference number DRKS00012759, 04th August 2017, https://www.drks.de/DRKS00012759 ).

Norm-based comparison of the quality-of-life impact of ravulizumab and eculizumab in paroxysmal nocturnal hemoglobinuria.

AIMS: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening intravascular hematologic disorder with significant morbidity and premature mortality. Clinical trials (NCT02946463 and NCT03056040) comparing ravulizumab with eculizumab for PNH have supported the non-inferiority of the former and similar safety and tolerability. This secondary analysis compared PNH trial participants after 26 weeks on either treatment (n = 438) to a general-population sample (GenPop) (n = 15,386) and investigated response-shift effects. METHODS: Multivariate analysis of covariance (MANCOVA) investigated function and symptom scores on the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 of people with PNH as compared to GenPop, after covariate adjustment. Risk-factor groups were created based on clinical indicators known to be associated with worse PNH outcomes, and separate MANCOVAs were computed for lower- and higher-risk-factor groups. Differential item functioning (DIF) analyses examined whether item response varied systematically (1) by treatment, (2) compared to GenPop, and (3) over time, the latter two suggesting and reflecting response-shift effects, respectively. DIF analyses examined 24 items from scales with at least two items. Recalibration response shift was operationalized as uniform DIF over time, reflecting the idea that, for a given group, the difficulty of endorsing an item changes over time, after adjusting for the total subscale score. Reprioritization response shift was operationalized as non-uniform DIF over time, i.e., the relative difficulty of endorsing an item over time changes across the total domain score. RESULTS: Across PNH risk-factor levels, people who had been on either treatment for 26 weeks reported better-than-expected functioning and lower symptom burden compared to GenPop. Ravulizumab generally showed larger effect sizes. Results were similar for lower and higher PNH risk factors, with slightly stronger effects in the former. DIF analyses revealed no treatment DIF, but did uncover group DIF (9 items with uniform DIF, and 11 with non-uniform) and DIF over time (7 items with uniform DIF, and 3 with non-uniform). CONCLUSIONS: This study revealed that people with PNH on ravulizumab or eculizumab for 26 weeks reported QOL levels better than those of the general population. Significant effects of DIF by group and DIF over time support recalibration and reprioritization response-shift effects. These findings suggest that the treatments enabled adaptive changes.

General Spanish population normative data analysis for the EORTC QLQ-C30 by sex, age, and health condition.

PURPOSE: General population normative data for the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 questionnaire facilitates interpretation of data assessed from cancer patients. This study aims to present normative data of the general Spanish population. METHODS/PATIENTS: Data were obtained from a prior larger study collecting EORTC QLQ-C30 norm data across 15 countries. Data were stratified by sex and age groups (18-39, 40-49, 50-59, 60-69 and > 70 years). Sex and age distribution were weighted according to population distribution statistics. Sex- and age-specific normative values were analysed separately, as were participants with versus those without health conditions. Multiple linear regression was used to estimate the association of each of the EORTC QLQ-C30 scales with the determinants age, sex, sex-by-age interaction term, and health condition. RESULTS: In total, 1,165 Spanish individuals participated in the study. Differences were found by sex and age. The largest sex-related differences were seen in fatigue, emotional functioning, and global QOL (Quality of Life), favouring men. The largest age differences were seen in emotional functioning, insomnia, and pain, with middle-aged groups having the worst scores. Those > 60 years old scored better than those < 60 years old on all scales except for physical functioning. Participants with no health conditions scored better in all QLQ-C30 domains. CONCLUSIONS: The present study highlights differences in HRQOL between specific sex/age strata and especially between people with and without a health condition in the general Spanish population. These factors must be considered when comparing general population HRQOL data with that of cancer patients.

Validity arguments for patient-reported outcomes: justifying the intended interpretation and use of data.

BACKGROUND: Contrary to common usage in the health sciences, the term "valid" refers not to the properties of a measurement instrument but to the extent to which data-derived inferences are appropriate, meaningful, and useful for intended decision making. The aim of this study was to determine how validity testing theory (the Standards for Educational and Psychological Testing) and methodology (Kane's argument-based approach to validation) from education and psychology can be applied to validation practices for patient-reported outcomes that are measured by instruments that assess theoretical constructs in health. METHODS: The Health Literacy Questionnaire (HLQ) was used as an example of a theory-based self-report assessment for the purposes of this study. Kane's five inferences (scoring, generalisation, extrapolation, theory-based interpretation, and implications) for theoretical constructs were applied to the general interpretive argument for the HLQ. Existing validity evidence for the HLQ was identified and collated (as per the Standards recommendation) through a literature review and mapped to the five inferences. Evaluation of the evidence was not within the scope of this study. RESULTS: The general HLQ interpretive argument was built to demonstrate Kane's five inferences (and associated warrants and assumptions) for theoretical constructs, and which connect raw data to the intended interpretation and use of the data. The literature review identified 11 HLQ articles from which 57 sources of validity evidence were extracted and mapped to the general interpretive argument. CONCLUSIONS: Kane's five inferences and associated warrants and assumptions were demonstrated in relation to the HLQ. However, the process developed in this study is likely to be suitable for validation planning for other measurement instruments. Systematic and transparent validation planning and the generation (or, as in this study, collation) of relevant validity evidence supports developers and users of PRO instruments to determine the extent to which inferences about data are appropriate, meaningful and useful (i.e., valid) for intended decisions about the health and care of individuals, groups and populations.

Implications of response shift for micro-, meso-, and macro-level healthcare decision-making using results of patient-reported outcome measures.

PURPOSE: Results of patient-reported outcome measures (PROMs) are increasingly used to inform healthcare decision-making. Research has shown that response shift can impact PROM results. As part of an international collaboration, our goal is to provide a framework regarding the implications of response shift at the level of patient care (micro), healthcare institute (meso), and healthcare policy (macro). METHODS: Empirical evidence of response shift that can influence patients' self-reported health and preferences provided the foundation for development of the framework. Measurement validity theory, hermeneutic philosophy, and micro-, meso-, and macro-level healthcare decision-making informed our theoretical analysis. RESULTS: At the micro-level, patients' self-reported health needs to be interpreted via dialogue with the clinician to avoid misinterpretation of PROM data due to response shift. It is also important to consider the potential impact of response shift on study results, when these are used to support decisions. At the meso-level, individual-level data should be examined for response shift before aggregating PROM data for decision-making related to quality improvement, performance monitoring, and accreditation. At the macro-level, critical reflection on the conceptualization of health is required to know whether response shift needs to be controlled for when PROM data are used to inform healthcare coverage. CONCLUSION: Given empirical evidence of response shift, there is a critical need for guidelines and knowledge translation to avoid potential misinterpretations of PROM results and consequential biases in decision-making. Our framework with guiding questions provides a structure for developing strategies to address potential impacts of response shift at micro-, meso-, and macro-levels.