Quality of Life Research

Purpose Chronic pruritus significantly impairs hemodialysis patients’ health status and quality of life (QOL) and it is associated with higher mortality rate, more frequent hospitalizations, poorer dialysis and medication adherence, and deteriorated mental status. However, pruritus is still underestimated, underdiagnosed, and undertreated in the real-life clinical scenario. We investigated prevalence, clinical characteristics, clinical correlates, severity as well as physical and psychological burden of chronic pruritus among adult hemodialysis patients in a large international real-world cohort. Methods We conducted a retrospective cross-sectional study of patients registered in 152 Fresenius Medical Care (FMC) NephroCare clinics located in Italy, France, Ireland, United Kingdom, and Spain. Demographic and medical data were retrieved from the EuCliD® (European Clinical) database, while information on pruritus and QoL were abstracted from KDQOL™-36 and 5-D Itch questionnaire scores. Results A total of 6221 patients were included, of which 1238 were from France, 163 Ireland, 1469 Italy, 2633 Spain, and 718 UK. The prevalence of mild-to-severe pruritus was 47.9% (n = 2977 patients). Increased pruritus severity was associated with increased use of antidepressants, antihistamines, and gabapentin. Patients with severe pruritus more likely suffered from diabetes, more frequently missed dialysis sessions, and underwent more hospitalizations due to infections. Both mental and physical QOL scores were progressively lower as the severity of pruritus increased; this association was robust to adjustment for potential confounders. Conclusion This international real-world analysis confirms that chronic pruritus is a highly prevalent condition among dialysis patients and highlights its considerable burden on several dimensions of patients’ life.

Purpose The burden of different skin diseases may vary leading individuals to have different sensitivity to stress. Therefore, we compared the health-related quality of life (HRQoL) and stress before and during the universal stress from the severe acute respiratory syndrome coronavirus-2-pandemic in individuals with and without hyperhidrosis, hidradenitis suppurativa, or psoriasis. Methods The study cohort was the Danish Blood Donor Study. Overall, 12,798 participants completed a baseline questionnaire before the pandemic, in 2018–2019, and a follow-up questionnaire during the pandemic, in 2020. Regression determined the association between the skin diseases and outcomes. Outcomes were the physical and mental component summary (MCS, PCS, respectively), which assess the mental and physical HRQoL, and the perceived stress scale, which assesses stress in the past four weeks. Results Overall, 1168 (9.1%) participants had hyperhidrosis, 363 (2.8%) had hidradenitis suppurativa, and 402 (3.1%) had psoriasis. At follow-up, the participants with hyperhidrosis had worse MCS (coefficient −0.59 [95% confidence interval (CI) −1.05, −0.13]) and higher odds of moderate-to-severe stress (odds ratio 1.37 [95% CI 1.13, 1.65]) and the participants with hidradenitis suppurativa worse PCS (coefficient −0.74 [95% CI −1.21, −0.27]) than the control groups. The associations were independent of baseline HRQoL, stress, the Connor-Davidson Resilience scale, and other covariables. Psoriasis was not associated with the outcomes. Conclusion Individuals with hyperhidrosis or hidradenitis suppurativa experienced worse mental or physical well-being and individuals with hyperhidrosis also had higher stress during the pandemic compared to healthy individuals. This suggests that individuals with these skin diseases are particularly susceptible to external stress.

Purpose To calibrate the item parameters of the German PROMIS® Pain interference (PROMIS PI) items using an item-response theory (IRT) model and investigate psychometric properties of the item bank. Methods Forty items of the PROMIS PI item bank were collected in a convenience sample of 660 patients, which were recruited during inpatient rheumatological treatment or outpatient psychosomatic medicine visits in Germany. Unidimensionality, monotonicity, and local independence were tested as required for IRT analyses. Unidimensionality was examined using confirmatory factor analyses (CFA) and exploratory factor analysis (EFA). Unidimensional and bifactor graded-response IRT models were fitted to the data. Bifactor indices were used to investigate whether multidimensionality would lead to biased scores. To evaluate convergent and discriminant validity, the item bank was correlated with legacy pain instruments. Potential differential item functioning (DIF) was examined for gender, age, and subsample. To investigate whether U.S. item parameters may be used to derive T-scores in German patients, T-scores based on previously published U.S. and newly estimated German item parameters were compared with each other after adjusting for sample specific differences. Results All items were sufficiently unidimensional, locally independent, and monotonic. Whereas the fit of the unidimensional IRT model was not acceptable, a bifactor IRT model demonstrated acceptable fit. Explained common variance and Omega hierarchical suggested that using the unidimensional model would not lead to biased scores. One item demonstrated DIF between subsamples. High correlations with legacy pain instruments supported construct validity of the item bank. T-scores based on U.S. and German item parameters were similar suggesting that U.S. parameters could be used in German samples. Conclusion The German PROMIS PI item bank proved to be a clinically valid and precise instrument for assessing pain interference in patients with chronic conditions.

Purpose During counseling and management of patients with vestibular schwannoma (VS), the emphasis is shifting from tumour control and nerve preservation towards maintaining or improving health-related quality of life (HRQoL). Understanding the patients’ perspective and impact of VS is, therefore, of utmost importance. The current study aimed to identify treatment outcomes preferred by patients and to explore the patient-reported VS symptoms and management-related side effects and their impact on HRQoL. Methods Patients with VS were contacted through the Dutch VS association Stichting Hoormij and questioned using a semi-structured, cross-sectional online survey. Patients were asked to report and rank symptoms and side effects, with their impact on HRQoL and frequency of occurrence. Results were structured through qualitative content analysis. Coded symptoms, side effects, impacts, frequencies, and patient-preferred outcomes were analysed and summarized with descriptive statistics. Results Of the 231 respondents, 71% were actively treated. Hearing (symptoms vs. side effects: 78.8% vs. 63.6%), balance (62.3%; 48.8%), and energy issues (33.8%; 32.6%) were the most frequently mentioned symptoms and management-related side effects. Fatigue, deafness, headaches, and hearing loss had the highest impact on HRQoL. The majority of patients identified hearing preservation (61%), balance preservation (38.5%), and reduced tinnitus (34.6%) to be the patient-preferred outcomes. Conclusion This qualitative study demonstrates that in this population many patients with VS encounter participation difficulties in their daily physical and social activities and value hearing and balance preservation, reduced tinnitus, and restored energy as preferred outcomes as they are hampered by symptoms and side effects related to hearing, balance, and energy. Healthcare professionals should consider these key points and use these and the patient-preferred outcomes in consultation, shared decision making, treatment, and follow-up to optimize patient-centred care.

Aims: Having a child with congenital heart disease (CHD) can affect parental health-related quality of life (HR-QoL). We investigated the long-term trajectories of mental HRQoL (m-HRQoL) in mothers of children with CHD and examined risk factors for persistent low m-HRQoL. Methods: One hundred twenty-five mothers of children with CHD completed a standardized questionnaire on m-HRQoL (mental subscale SF-12) after the children's first open-heart surgery and subsequently when the children were 1, 4, 6, 10, and 13 years old. A z-score for m-HRQoL was calculated with national norms. Latent class growth analysis (LCGA) was used to identify subgroups of mothers with regards to their m-HRQoL trajectories over time. Regression analysis investigated predictors for chronically low m-HRQoL. Results: Compared to norms, mothers of children with CHD had significantly lower m-HRQoL immediately after open-heart surgery (β = -0.30 (CI-95: -0.44, -0.15)). Subsequently, m-HRQoL increased to a normal level (m-HRQoL compared to the norm from 1 to 13 years: β ranges between 0.05 and 0.27). LCGA revealed two distinct groups of m-HRQoL trajectories: A group with normal m-HRQoL (75% of mothers, means z-scores range between - 0.76 and 0.62) and a group with chronically low m-HRQoL (25% of mothers, mean z-scores range between -1.32 and -0.10). Chronically, low m-HRQoL was associated with poorer social support (OR = 3.39 (CI-95: 1.40, 8.49), p = 0.008) but not with parental education, migration background, number of open-heart surgeries, diagnosis of a univentricular CHD, or low IQ. Conclusion: A quarter of mothers of children with CHD have chronically low m-HRQoL throughout their child's development, especially those mothers with poor social support. Further studies of family-oriented approaches are needed to identify and support these mothers and reinforce parental well-being.

Aims This paper aims to systematically identify reported health state utility values (HSUVs) in children and adolescents with mental health problems (MHPs) aged less than 25 years; to summarise the techniques used to elicit HSUVs; and to examine the psychometric performance of the identified multi-attribute utility instruments (MAUIs) used in this space. Methods A systematic review was conducted following PRISMA guidelines. Peer-reviewed studies published in English, reporting HSUVs for children and adolescents with MHPs using direct or indirect valuation methods were searched in six databases. Results We found 38 studies reporting HSUVs for 12 types of MHPs across 12 countries between 2005 and October 2021. Attention deficit hyperactivity disorder (ADHD) and depression are the most explored MHPs. Disruptive Behaviour Disorder was associated with the lowest reported HSUVs of 0.06 while cannabis use disorder was associated with the highest HSUVs of 0.88. Indirect valuation method through the use of MAUIs (95% of included studies) was the most frequently used approach, while direct valuation methods (Standard Gamble, Time Trade-Off) were only used to derive HSUVs in ADHD. This review found limited evidence of the psychometric performance of MAUIs used in children and adolescents with MHPs. Conclusion This review provides an overview of HSUVs of various MHPs, the current practice to generate HSUVs, and the psychometric performance of MAUIs used in children and adolescents with MHPs. It highlights the need for more rigorous and extensive psychometric assessments to produce evidence on the suitability of MAUIs used in this area.

Purpose The pain beliefs and perceptions inventory (PBPI) and the pain catastrophizing scales (PCS) characterize beliefs or distress dimensions of the pain experience. It is relatively unknown, however, to what degree the PBPI and the PCS are well suited to classifying pain intensity. Methods This study applied a receiver operating characteristic (ROC) approach to these instruments against the criterion of a visual analogue scale (VAS) of pain intensity with fibromyalgia and chronic back pain patients (n = 419). Results The largest areas under the curve (AUC) were moderate and limited to the constancy subscale (71%) and total score (70%) of the PBPI and to the helplessness subscale (75%) and total score (72%) of the PCS. The best cut-off scores for the PBPI and PCS were better off at detecting true negatives than true positives, with larger specificity than sensitivity values. Conclusion Whereas, the PBPI and PCS are certainly useful instruments to evaluate diverse pain experiences, they may be inappropriate to classify intensity. The PCS performs marginally better than the PBPI for classifying pain intensity.

Objective The objective of this study was to determine the patient-reported outcome measure (PROM) score ranges associated with descriptive labels (i.e., within normal limits, mild, moderate, severe) by using bookmarking methods with orthopedic clinicians and patients who have experienced a bone fracture. Study design and setting We created vignettes comprised of six items and responses from the Patient-Reported Outcomes Measurement Information System (PROMIS) Upper Extremity Function, Physical Function, and Pain Interference item banks reflecting different levels of severity. Two groups of patients with fractures (n = 11) and two groups of orthopedic clinicians (n = 16) reviewed the vignettes and assigned descriptive labels independently and then discussed as a group until reaching consensus via a videoconference platform. Results PROMIS Physical Function and Pain Interference thresholds (T = 50, 40, 25/30 and T = 50/55, 60, 65/70, respectively) for patients with bone fractures were consistent with the results from other patient populations. Upper Extremity thresholds were about 10 points (1 SD) more severe (T = 40, 30, 25/20) compared to the other measures. Patient and clinician perspectives were similar. Conclusion Bookmarking methods generated meaningful score thresholds for PROMIS measures. These thresholds between severity categories varied by domain. Threshold values for severity represent important supplemental information to interpret PROMIS scores clinically.

Purpose This unselected, population-based cohort study aimed to determine the level of health-related quality of life (HRQoL) in patients with Crohn’s disease (CD) and ulcerative colitis (UC) at the time of diagnosis compared with a reference population and identify the demographic factors, psychosocial measures, and disease activity markers associated with HRQoL. Methods Adult patients newly diagnosed with CD or UC were prospectively enrolled. HRQoL was measured using the Short Form 36 (SF-36) and Norwegian Inflammatory Bowel Disease Questionnaires. Clinical significance was assessed using Cohen’s d effect size and further compared with a Norwegian reference population. Associations between HRQoL and symptom scores, demographic factors, psychosocial measures, and disease activity markers were analyzed. Results Compared with the Norwegian reference population, patients with CD and UC reported significantly lower scores in all SF-36 dimensions, except for physical functioning. Cohen’s d effect sizes for men and women in all SF-36 dimensions were at least moderate, except for bodily pain and emotional role for men with UC and physical functioning for both sexes and diagnoses. In the multivariate regression analysis, depression subscale scores ≥ 8 on the Hospital Anxiety and Depression Scale, substantial fatigue, and high symptom scores were associated with reduced HRQoL. Conclusion Patients newly diagnosed with CD and UC reported statistically and clinically significantly lower scores in seven of the eight SF-36 dimensions than the reference population. Symptoms of depression, fatigue, and elevated symptom scores were associated with poorer HRQoL.

In June 2021, the US Food and Drug Administration (FDA) released a draft guidance for industry on core patient-reported outcomes (PROs) and related considerations for instrument selection and trial design in registrational cancer clinical trials, building on prior communications about the use of PROs to assess efficacy and tolerability in oncology drug development. The International Society for Quality of Life Research (ISOQOL) Standards and Best Practices Committee led an initiative to draft a commentary about the guidance, focusing on its positive aspects and areas that would benefit from additional clarification and consideration. For comprehensiveness, the authors reviewed existing public comments on the draft guidance, and the commentary underwent a thorough review process through three ISOQOL Special Interest Groups (Psychometrics, Clinical Practice, and Regulatory and Health Technology Assessment Engagement) followed by the ISOQOL Board. The goal of this commentary is to situate this new and relevant guidance document within the context of recent regulatory efforts on PROs and highlight areas in which further work may ultimately benefit the field.

Purpose The aim of this study was to generate evidence supporting the development and content validity of a new PRO instrument, the Small Intestinal Bacterial Overgrowth (SIBO) Symptom Measure (SSM) daily diary. The SSM assesses symptom severity in SIBO patients, with the ultimate goal of providing a fit for purpose PRO for endpoint measurement. Methods Qualitative research included 35 SIBO patients in three study stages, using a hybrid concept elicitation (CE)/cognitive interview (CI) method with US patients, ≥ 18 years. Stage 1 included a literature review, clinician interviews, and initial CE interviews with SIBO patients to identify symptoms important to patients for inclusion in the SSM. Stage 2 included hybrid CE/CI to learn more about patients’ SIBO experience and test the draft SSM. Finally, stage 3 used CIs to refine the instrument and test its content validity. Results In stage 1 (n = 8), 15 relevant concepts were identified, with items drafted based on the literature review/clinician interviews and elicitation work. Within stage 2 (n = 15), the SSM was refined to include 11 items; with wording revised for three items. Stage 3 (n = 12) confirmed the comprehensiveness of the SSM, as well as appropriateness of the item wording, recall period, and response scale. The resulting 11-item SSM assesses the severity of bloating, abdominal distention, abdominal discomfort, abdominal pain, flatulence, physical tiredness, nausea, diarrhea, constipation, appetite loss, and belching. Conclusions This study provides evidence supporting the content validity of the new PRO. Comprehensive patient input ensures that the SSM is a well-defined measure of SIBO, ready for psychometric validation studies.

Purpose The structure of well-being has been debated for millennia. Dominant conceptualisations, such as the hedonic and eudaimonic models, emphasise different constituents of the well-being construct. Some previous studies have suggested that the underlying structure of well-being may consist of one or a few general well-being factors. We conducted three studies to advance knowledge on the structure of well-being comprising more than 21,500 individuals, including a genetically informative twin sample. Methods In Study 1, we used hierarchical exploratory factor analysis to identify well-being factors in a population-based sample of Norwegian adults. In Study 2, we used confirmatory factor analysis to examine the model fit of the identified factor model in an independent sample. In Study 3, we used biometric models to examine genetic and environmental influences on general well-being factors. Results We identified six well-being factors which all loaded on a single higher-order factor. This higher-order factor may represent a general “happiness factor”, i.e. an h-factor, akin to the p-factor in psychopathology research. The identified factor model had excellent fit in an independent sample. All well-being factors showed moderate genetic and substantial non-shared environmental influence, with heritability estimates ranging from 26% to 40%. Heritability was highest for the higher-order general happiness factor. Conclusion Our findings yield novel insights into the structure of well-being and genetic and environmental influences on general well-being factors, with implications for well-being and mental health research, including genetically informative studies.

Purpose Despite advances in cancer treatment, there is a prevalence of pediatric childhood cancer survivors still at risk of developing adverse disease and treatment outcomes, even after the end of treatment. The present study aimed to (1) explore how mothers and fathers assess the health-related quality of life (HRQoL) of their surviving child and (2) evaluate risk factors for poor parent-reported HRQoL in childhood cancer survivors about 2.5 years after diagnosis. Methods We assessed parent-reported HRQoL of 305 child and adolescent survivors < 18 years diagnosed with leukemia or tumors of central nervous system (CNS) with the KINDL-R questionnaire in a prospective observational study with a longitudinal mixed-methods design. Results In agreement with our hypotheses, our results show that fathers rate their children’s HRQoL total score as well as the condition-specific domains family (p = .013, d = 0.3), friends (p = .027, d = 0.27), and disease (p = .035, d = 0.26) higher than mothers about 2.5 years after diagnosis. Taking variance of inter-individual differences due to family affiliation into account, the mixed model regression revealed significant associations between the diagnosis of CNS tumors (p = .018, 95% CI [− 7.78, − 0.75]), an older age at diagnosis, (p = .011, 95% CI [− 0.96, − 0.12]), and non-participation in rehabilitation (p = .013, 95% CI [− 10.85, − 1.28]) with poor HRQoL in children more than 2 years after being diagnosed with cancer. Conclusion Based on the results, it is necessary for health care professionals to consider the differences in parental perceptions regarding children’s aftercare after surviving childhood cancer. High risk patients for poor HRQoL should be detected early, and families should be offered support post-cancer diagnosis to protect survivors’ HRQoL during aftercare. Further research should focus on characteristics of pediatric childhood cancer survivors and families with low participation in rehabilitation programs.

Purpose Sexual health is an important contributing factor for health-related quality of life, but research in this domain is scarce. Moreover, normative data are needed to interpret patient-reported outcome measures on sexual health. The aim of this study was to collect and describe normative scores of the Female Sexual Distress Scale (FSDS) and the Body Image Scale (BIS) from the Dutch population and assess the effect of important demographic and clinical variables on the outcome. As the FSDS is also validated in men, we refer to it as SDS. Method Dutch respondents completed the SDS and BIS between May and August 2022. Sexual distress was defined as a SDS score > 15. Descriptive statistics were calculated to present normative data per age group per gender after post-stratification weighting was applied. Multiple logistic and linear regression analyses were conducted to assess the effect of age, gender, education, relationship status, history of cancer and (psychological) comorbidities on SDS and BIS. Results For the SDS 768 respondents were included with a weighted mean score of 14.41 (SD 10.98). Being female (OR 1.77, 95% CI [1.32; 2.39]), having a low educational level (OR 2.02, CI [1.37; 2.39]) and psychological comorbidities (OR: 4.86, 95% CI [2.17; 10.88]) were associated with sexual distress. For the BIS, 696 respondents were included. Female gender (β: 2.63, 95% CI [2.13; 3.13]), psychological comorbidities (β: 2.45, 95% CI [1.43; 3.47]), higher age (β: −0.07, 95% CI [−0.09; −0.05]), and a high educational level (β:−1.21, CI: −1.79 to −0.64) were associated with the non-disease related questions of the Body Image Scale. Conclusion This study provides age- and gender-dependent normative values for the SDS and the non-disease related questions of the BIS. Sexual distress and body image are influenced by gender, education level, relationship status and psychological comorbidities. Moreover, age is positively associated with Body Image.

Purpose There is limited research exploring the usefulness of generic preference-based quality of life (GPQoL) measures used to facilitate economic evaluation in the context of posttraumatic stress disorder (PTSD). The aim of the current study was to explore the validity and responsiveness of a common GPQoL measure (Assessment of Quality of Life 8 Dimension [AQoL-8D]) in relation to a PTSD condition-specific outcome measure (Posttraumatic Stress Disorder Checklist for the DSM-5 [PCL-5]). Method This aim was investigated in a sample of individuals (N = 147) who received trauma-focused cognitive-behavioural therapies for posttraumatic stress disorder. Convergent validity was investigated using spearman’s correlations, and the level of agreement was investigated using Bland–Altman plots. Responsiveness was investigated by exploring the standardised response means (SRM) from pre-post-treatment across the two measures, which allow the comparison of the magnitude of change between the measures over time. Results Correlations between the AQoL-8D (dimensions, utility and summary total scores) and the PCL-5 total score ranged from small to large and agreement between the measures was considered moderate to good. While SRMs were large for the AQoL-8D and PCL-5 total scores, the SRM for the PCL-5 was nearly double that of the AQoL-8D. Conclusion Our findings demonstrate that the AQoL-8D has good construct validity but present preliminary evidence that economic evaluations using only GPQoL measures may not fully capture the effectiveness of PTSD treatments.

Purpose The aim of this study was to develop and validate a brief personalized instrument that (1) defines patients’ priorities for improvement, (2) measures progress in prioritized quality of life (QoL) and self-management outcomes, and (3) is applicable in both clinical practice and clinical trials. Methods The instrument was developed based on the literature on personalized assessment and patient priorities, feedback by clinicians, and six cognitive interviews with patients with chronic kidney disease. The resulting questionnaire, the Personalized Priority and Progress Questionnaire (PPPQ), contains a baseline and follow-op measurement. The baseline measurement assesses functioning on QoL (8 items) and self-management (5 items). The final item evaluates patients’ priorities for improvement. The follow-up measurement assesses progress in QoL and self-management. A personalized progress score can be calculated indicating the amount of progress on the QoL or self-management domain that is prioritized by the individual patient. Psychometric properties of the PPPQ were evaluated among patients with chronic kidney disease (n = 121) and patients with kidney failure treated with dialysis (n = 22). Results The PPPQ showed to be a feasible instrument that is easy and quick to complete. Regarding the construct validity, small to large correlations were found between the items and existing validated questionnaires measuring related constructs. Conclusion The PPPQ proved to be a feasible and valid instrument. The PPPQ can be adapted to match diverse populations and could be a useful tool both in clinical practice (e.g., to identify priorities and tailor treatment) and clinical trials (e.g., to evaluate the effectiveness of personalized interventions).

Purpose The objective of this study was to quantitatively evaluate psychological and quality of life-related complications at three months following discharge in hospitalized coronavirus disease 2019 (COVID-19) patients during the pandemic in Iran. Methods In this time-point analysis of prospective cohort study data, adult patients hospitalized with symptoms suggestive of COVID-19 were enrolled. Patients were stratified in analyses based on severity. The primary outcomes consisted of psychological problems and pulmonary function tests (PFTs) in the three months following discharge, with Health-related quality of life (HRQoL) as the secondary outcome. Exploratory predictors were determined for both primary and secondary outcomes. Results 283 out of 900 (30%) eligible patients were accessible for the follow-up assessment and included in the study. The mean age was 53.65 ± 13.43 years, with 68% experiencing a severe disease course. At the time of the final follow-up, participants still reported persistent symptoms, among which fatigue, shortness of breath, and cough were the most common. Based on the regression-adjusted analysis, lower levels of forced expiratory volume in one second (FEV1) to forced vital capacity (FVC) ratio was associated with higher levels of depression (standardized β = − 0.161 (SE = 0.042), P = 0.017) and stress levels (standardized β =− 0.110 (SE = 0.047), P = 0.015). Furthermore, higher levels of anti-SARS-CoV-2 immunoglobulin-M (IgM) were associated with significantly lower levels of depression (standardized β = − 0.139 (SE = 0.135), P = 0.031). Conclusions There is an association between lung damage during COVID-19 and the reduction of pulmonary function for up to three months from acute infection in hospitalized patients. Varying degrees of anxiety, depression, stress, and low HRQoL frequently occur in patients with COVID-19. More severe lung damage and lower COVID-19 antibodies were associated with lower levels of psychological health.

Purpose Mental health and well-being trajectories are not expected to be homogeneous in diverse clinical populations. This exploratory study aims to identify subgroups of patients with cancer receiving radiation therapy who have different mental health and well-being trajectories, and examine which socio-demographic, physical symptoms, and clinical variables are associated with such trajectories. Methods Retrospective analysis of radiation therapy patients diagnosed with cancer in 2017 was conducted using data from the Ontario Cancer Registry (Canada) and linked with administrative health data. Mental health and well-being were measured using items from the Edmonton Symptom Assessment System—revised questionnaire. Patients completed up to 6 repeated measurements. We used latent class growth mixture models to identify heterogeneous mental health trajectories of anxiety, depression, and well-being. Bivariate multinomial logistic regressions were conducted to explore variables associated with the latent classes (subgroups). Results The cohort (N = 3416) with a mean age of 64.5 years consisted of 51.7% females. Respiratory cancer was the most common diagnosis (30.4%) with moderate to severe comorbidity burden. Four latent classes with distinct anxiety, depression, and well-being trajectories were identified. Decreasing mental health and well-being trajectories are associated with being female; living in neighborhoods with lower income, greater population density, and higher proportion of foreign-born individuals; and having higher comorbidity burden. Conclusions The findings highlight the importance of considering social determinants of mental health and well-being, in addition to symptoms and clinical variables, when providing care for patients undergoing radiation therapy.

Purpose Despite the benefits of palliative care (PC) in pancreatic cancer, little is known about patients who access PC. This observational study examines the characteristics of patients with pancreatic cancer at their first episode of PC. Methods First-time, specialist PC episodes captured through the Palliative Care Outcomes Collaboration (PCOC), in Victoria, Australia between 2014 and 2020, for pancreatic cancer, were identified. Multivariable logistic regression analyses examined the impact of patient- and service-level characteristics on symptom burden (measured through patient-reported outcome measures and clinician-rated scores) at first PC episode. Results Of 2890 eligible episodes, 45% began when the patient was deteriorating and 32% ended in death. High fatigue and appetite-related distress were most common. Generally, increasing age, higher performance status and more recent year of diagnosis predicted lower symptom burden. No significant differences were noted between symptom burden of regional/remote versus major city dwellers; however, only 11% of episodes recorded the patient as a regional/remote resident. A greater proportion of first episodes for non-English-speaking patients began when the patient was unstable, deteriorating or terminal, ended in death and were more likely to be associated with high family/carer problems. Community PC setting predicted high symptom burden, with the exception of pain. Conclusion A large proportion of first-time specialist PC episodes in pancreatic cancer begin at a deteriorating phase and end in death, suggesting late access to PC. Timely referrals to community-based specialist PC, access in regional/remote areas, as well as development of culturally diverse support systems require further investigation.

Purpose Researchers and service providers typically assess pediatric Health-Related Quality of Life (HRQOL) by collecting independent reports from parents and youth. An emerging body of work indicates that patterns of parent-youth reports yield information germane to understanding youth outcomes. We identified patterns of HRQOL among youth and their parents seeking mental health treatment and examined links between agreement patterns and mental and physical health functioning. Methods Participants included 227 youth (mean age = 14.40 years, SD = 2.42; 63% female) and parent dyads presenting at a mood disorders clinic between 2013 and 2020. We assessed HRQOL using parallel youth and parent forms of the Pediatric Quality of Life Inventory Generic Core Scales. We also assessed youth clinical correlates of depression, suicidal ideation, and impairment, as well as health information via electronic health record (e.g., psychotropic medication usage, BMI). Results Latent class analysis showed three parent-youth reporting patterns: Low–Low (LL), High-High (HH), and Parent Low-Youth High (PL-YH). Relative to youth in the HH group, youth in the LL and PL-YH groups reported significantly greater depressive symptoms and had higher rates of suicidal ideation and psychotropic medication use. In addition, youth in the LL group reported significantly greater levels of impairment. Conclusions Parent-youth patterns of HRQOL reporting can reveal clinically meaningful information and indicate poorer functioning for certain groups (LL, PL-YH) of youth. These findings have implications for improving accuracy of risk assessments that leverage HRQOL data.

PurposeThe TORG0503 study was undertaken to select a preferred platinum-based third-generation regimen for patients with completely resected non-small cell lung cancer (NSCLC). This study aimed to describe the quality of life (QOL) analysis of that study.Methods Patients with completely resected NSCLC were randomized to receive three cycles of docetaxel plus cisplatin (DC) or paclitaxel plus carboplatin (PC) on day 1 every 3 weeks. QOL was assessed at three time points (baseline, after two cycles, and after three cycles) using the Functional Assessment of Cancer Therapy–taxane (FACT-Taxane). The adjusted odds ratio (OR) and 95% confidence interval (CI) were calculated by logistic regression analysis that was adjusted for the baseline score in the FACT-Taxane total score and each subscale to evaluate treatment (PC vs. DC) effectiveness.ResultsQOL data from 104 patients (DC, n = 56 patients; PC, n = 48) were analyzed. In the FACT-Taxane total score, the baseline-adjusted OR (95% CI) of not worse QOL for the DC group was 3.3 (1.4–8.3) compared with the PC group. In the taxane subscale, the baseline-adjusted OR (95% CI) was 6.2 (2.6–16.0).Conclusion Total QOL was maintained better in the DC group than in the PC group, especially the taxane subscale that consists of neurotoxicity and taxane components in spite of no treatment-related death in both arms between DC and PC. We might recommend DC as the control regimen for the next clinical trial from the viewpoint of QOL, similar to the primary outcomes in TORG0503.

Background Improvements in cancer treatment have increased the number of cancer survivors, but also increased the long-term and late effects from cancer therapy. Patient reported “side effect bother” could be used to measure the burden of treatment, and the risk for negative outcomes such as dose reduction, treatment delay or discontinuation. The current study addresses the psychometric properties of a single item, determines what represents a “meaningful change”, and evaluates the correlation to safety endpoints and functioning. Methods Results from 5911 patients enrolled in 8 clinical trials representing 5 disease types in oncology and hematology who completed either the Functional Assessment of Cancer Therapy (FACT) GP5 item or a modified bother item (MBI) were assessed. Results Patients ranged in age from 18 to 93 years, with all cancer stages represented and approximately equal numbers of males and females. Test–retest reliability was acceptable, as were convergent and known groups validity. The GP5 and MBI effectively demonstrated sensitivity to change over time and established meaningful thresholds. Conclusions The results indicate that these single-items are psychometrically sound, capable of distinguishing known groups, responsive to change and can identify meaningful change over time in terms of treatment-related symptoms. It extends the findings of recent scientific groups by providing analyses not included in prior studies, and further supports the FDA’s recommendation to include a single item question in clinical trials.

Purpose Preference-based measures have been increasingly recommended to measure health outcomes for economic evaluation. However, none of existing cardiovascular disease (CVD)-specific health-related quality of life (HRQoL) instruments are preference-based. This study aimed to develop the descriptive system of preference-based HRQoL instrument for Chinese patients with CVDs under the Initiative of China Health Related Outcomes Measures (CHROME). Methods Qualitative face-to-face interviews were conducted with Chinese patients with CVDs. Content analysis was employed to generate candidate items for the instrument. Then expert consultation and cognitive debriefing interviews were conducted to guide further selection and revision of the items. Results We interviewed 127 CVD patients with 67.7% being male and 63.8% living in the urban area. A hierarchical code book comprised of four themes, 20 categories, 62 sub-categories, and 207 codes, was developed. Candidate items were selected based on the criteria set by the Consensus-based Standards for the selection of health Measurement Instruments (COSMIN) methodology and ISPOR PRO guidance. An online survey and meeting with an expert advisory panel (n = 15) followed by cognitive debriefing interviews with 20 patients and 13 physicians were conducted to further select and revise the candidate items. The descriptive system of CHROME-CVD consists of 14 items, namely frequency and severity of chest pain, chest tightness, palpitation, shortness of breath, dizziness, fatigue, appetite, sleeping, mobility, daily activities, depression, worry, and social relationship. Four or five level responses were selected based on cognitive debriefing results to each item. Conclusion The current study developed the descriptive system (items and response options) of CHROME-CVD, the future CVD-specific preference-based HRQoL instrument for Chinese CVD patients.

Purpose To assess the psychometric properties of glaucoma-specific health-related quality of life (HRQoL) item banks (IBs), and explore their efficiency using computerized adaptive testing (CAT) simulations. Methods In this cross-sectional, clinical study, 300 Asian glaucoma patients answered 221 items within seven IBs: Ocular Comfort Symptoms (OS); Activity Limitation (AL); Lighting (LT); Mobility (MB); Glaucoma Management (GM); Psychosocial (PSY); and Work (WK). Rasch analysis was conducted to assess each IB’s psychometric properties (e.g., item “fit” to the construct; unidimensionality) and a set of analytic performance criteria guiding decision making relating to retaining or dropping domains and items was employed. CAT simulations determined the mean number of items for ‘high’ and ‘moderate’ measurement precision (stopping rule: SEM 0.3 and 0.387, respectively). Results Participants’ mean age was 67.2 ± 9.2 years (62% male; 87% Chinese). LT, MB, and GM displayed good psychometric properties overall. To optimize AL’s psychometric properties, 16 items were deleted due to poor “fit”, high missing data, item bias, low discrimination and/or a low clinical/patient importance rating. To resolve multidimensionality in PSY, we rehomed 16 items into a “Concern (CN)” domain. PSY and CN required further amendment, including collapsing of response categories, and removal of poorly functioning items (N = 7). Due to poor measurement precision, low applicability and high ceiling effect, low test information indices, and low item separation index the WK IB was not considered further. In CAT simulations on the final seven IBs (n = 182 items total), an average of 12.1 and 15.7 items per IB were required for moderate and high precision measurement, respectively. Conclusions After reengineering our seven IBs, they displayed robust psychometric properties and good efficiency in CAT simulations. Once finalized, GlauCAT™-Asian may enable comprehensive assessment of the HRQoL impact of glaucoma and associated treatments.

Purpose Although the use of Patient-Reported Outcomes Measurement Information System (PROMIS) measures is widely advocated, little is known on their use in patients with inflammatory arthritis. We systematically describe the use and outcomes of PROMIS measures in clinical studies involving people with rheumatoid arthritis (RA) or axial spondyloarthritis (axSpA). Methods A systematic review was conducted according to the PRISMA guidelines. Through a systematic search of nine electronic databases, clinical studies including patients with RA or axSpA and reporting the use of PROMIS measure were selected. Study characteristics, details of PROMIS measures and their outcomes, if available, were extracted. Results In total, 29 studies described in 40 articles met the inclusion criteria, of which 25 studies included RA patients, three studies included axSpA patients and one study included both RA and axSpA patients. The use of two general PROMIS measures (PROMIS Global Health, PROMIS-29) and 13 different domain-specific PROMIS measures was reported, of which the PROMIS Pain Interference (n = 17), Physical Function (n = 14), Fatigue (n = 13), and Depression (n = 12) measures were most frequently used. Twenty-one studies reported their results in terms of T-scores. Most T-scores were worse than the general population mean, indicating impairments of health status. Eight studies did not report actual data but rather measurement properties of the PROMIS measures. Conclusion There was considerable variety regarding the different PROMIS measures used, with the PROMIS Pain interference, Physical function, Fatigue, and Depression measures being the most frequently used. In order to facilitate the comparisons across studies, more standardization of the selection of PROMIS measures is needed.

Purpose There are limited data on the impact of caregiving for patients with malignant pleural mesothelioma (MPM) on the caregiver. We aimed to identify the demographic characteristics of these caregivers, the caregiving activities they perform and how caregiving burden impacts their work productivity and overall activity. Methods This cross-sectional study collected data from caregivers of patients with MPM across France, Italy, Spain and the United Kingdom January-June 2019. Caregiver demographics, daily caregiving tasks and the impact of caregiving on physical health was collected via questionnaire. The Zarit Burden Interview (ZBI) was used to assess caregiver burden and the Work Productivity and Activity Impairment questionnaire (WPAI) assessed impairment at work and during daily activities. Analyses were descriptive. Results Overall, 291 caregivers provided data. Caregivers were mostly female (83%), living with the patient (82%) and their partner/spouse (71%). Caregivers provided over five hours of daily emotional/physical support to patients. ZBI scores indicated 74% of caregivers were at risk of developing depression. Employed caregivers had missed 12% of work in the past seven days, with considerable presenteeism (25%) and overall work impairment (33%) observed. Overall, the mean activity impairment was 40%. Conclusion Caregivers provide essential care for those with MPM. We show caregiving for patients with MPM involves a range of burdensome tasks that impact caregivers’ emotional health and work reflected in ZBI and WPAI scores. Innovations in the management of MPM must account for how caregivers may be impacted and can be supported to carry out this important role.

Aim To explore the perspectives, experience, and concerns of patients with irritable bowel syndrome (IBS) in China. Methods We used data mining to investigate posts shared in Baidu Tieba concerned with IBS; we collected the data through the crawler code, and mined the cleaned data’s themes based on Latent Dirichlet allocation (LDA) and the Grounded theory. Results We found 5746 network posts related to IBS. LDA analysis generated 20 topics, and grounded theory analysis established eight topics. Combining the two methods, we finally arranged the topics according to five concepts: difficulty in obtaining disease information; serious psychosocial problems; dissatisfied with the treatment; lack of social support; and low quality of life. Conclusion Social media research improved patient-centric understanding of patients’ experiences and perceptions. Our study may facilitate doctor-patient communication and assist in the formulation of medical policies.

Purposes The study intended to (1) assess changes of health-related quality of life (HRQoL) between early treatment-related time points and 10 years post-treatment in a cohort of breast cancer (BC) patients who received radiotherapy (RT), (2) to evaluate differences in HRQoL between long-term BC survivors and unaffected women from the same geographical region and (3) to identify determinants of long-term HRQoL in the survivor cohort. Methods 292 BC patients were recruited prior to RT after breast-conserving surgery between 1998 and 2001 in Germany and prospectively followed up for a median of 11.4 years (range 10.3–12.8 years). HRQoL was assessed using EORTC QLQ-C30 at pre-RT (baseline), during RT, 6 weeks after RT, and at the 10-year follow-up. Changes in mean HRQoL scores over time were assessed using linear-mixed models. HRQoL in long-term survivors and controls was compared using Wilcoxon rank-sum test, stratified by age groups. Multivariable linear regression models were used to identify determinants for HRQoL in long-term BC survivors. Results Compared to baseline level (mean summary score of 64.9), global health status/quality of life (GHS/QoL) declined during RT (62.4) and improved 6 weeks after RT (69.9) before decreasing to baseline level at the 10-year follow-up (66.7). Most functional domains deteriorated or remained stable at 10 years post-diagnosis compared to post-RT scores, except for role functioning which improved, while dyspnea and diarrhea significantly deteriorated between those two time points. There were no significant differences in long-term GHS/QoL between BC survivors 10 years post-RT and controls for all age groups (p > 0.05). However, deficits in specific HRQoL domains such as emotional burden, sleep problems or fatigue were found to more strongly affect survivors, in particular those younger than 65 years, compared to controls. In the determinant analysis, being overweight was associated with lower GHS/QoL and physical functioning, while living with others was found to be associated with better physical functioning, and decreased dyspnea and pain levels. Certain comorbidities such as depression had a strong association with multiple HRQoL domains, including lower GHS/QoL and functioning as well as a higher level of fatigue, pain, sleep/intestinal problems, and financial difficulties. Side effects such as lymphedema/pain and fibrosis were associated with worse physical and social functioning, respectively. Conclusion The long-term GHS/QoL remained comparable when compared with the control population while restrictions in certain functional and symptoms domains in long-term BC survivors persisted over 10 years, in particular among younger survivors. Targeted screening to identify cancer survivors at risk for psychosocial/other impairment accounting also for comorbidities and treatment side effects may be warranted in long-term aftercare to address unmet health needs.

Introduction The COVID-19 pandemic forced people to give up their daily routines and adjust to new circumstances. This might have affected health-related quality of life (HRQOL). We aimed to compare HRQOL during the first COVID-19 wave in 2020 to HRQOL before the pandemic and to identify determinants of HRQOL during the pandemic in Switzerland. Methods We conducted a cross-sectional online survey during the pandemic (between May and July 2020; CoWELL sample; convenience sample). Before the pandemic (2015–2016), we had conducted a cross-sectional paper-based survey among a representative random sample of the Swiss general population (SGP sample). In both samples, we assessed physical and mental HRQOL (Short Form-36) and socio-demographic characteristics. In the CoWELL sample, we additionally assessed health- and COVID-19-related characteristics. Data were analysed using linear regressions. Results The CoWELL sample included 1581 participants (76% women; mean age = 43 years, SD = 14 years) and the SGP sample 1209 participants (58% women, mean age = 49 years, SD = 15 years). Adjusted for sex, age, and education, the CoWELL sample reported higher physical HRQOL (PCS, +5.8 (95% CI: 5.1, 6.6), p < 0.001) and lower mental HRQOL (MCS, −6.9 (−7.8, −6.0), p < 0.001) than the SGP sample. In the CoWELL sample, especially persons with lower health literacy, who had no support network or who have had COVID-19, reported lower HRQOL. Discussion Aspects unique to the COVID-19 pandemic affected HRQOL. Vulnerable persons such as those having had COVID-19, less support opportunities, and with lower health literacy are especially prone to impaired HRQOL during the COVID-19 pandemic.

Background Checkpoint inhibitors have been shown to substantially improve the survival of patients with advanced melanoma. With this growing group of survivors treated with immunotherapies, assessing their health-state utilities is essential and can be used for the calculation of quality-adjusted life years and for cost-effectiveness analyses. Therefore, we evaluated the health-state utilities in long-term advanced melanoma survivors. Methods Health-state utilities were evaluated in a cohort of advanced melanoma survivors 24–36 months (N = 37) and 36-plus months (N = 47) post-ipilimumab monotherapy. In addition, the health-state utilities of the 24–36 months survivor group were assessed longitudinally, and utilities of the combined survival groups (N = 84) were compared with a matched control population (N = 168). The EQ-5D was used to generate health-state utility values, and quality-of-life questionnaires were used to establish correlations and influencing factors of utility scores. Results Health-state utility scores were similar between the 24–36 months’- and the 36-plus months’ survival group (0.81 vs 0.86; p = .22). In survivors, lower utility scores were associated with symptoms of depression (β = − .82, p = .022) and fatigue burden (β = − .29, p = .007). Utility scores did not significantly change after 24–36 months of survival, and the utilities of survivors were comparable to the matched control population (0.84 vs 0.87; p = .07). Discussion Our results show that long-term advanced melanoma survivors treated with ipilimumab monotherapy experience relatively stable and high health-state utility scores.

Introduction Chronic rhinosinusitis (CRS) is strongly associated with significant impairment of quality of life (QoL) in children. The SN-5 questionnaire is an important assessment tool for pediatric CRS. This study aimed to evaluate potential prognostic factors for treatment of pediatric CRS within the Hebrew version of the SN-5 questionnaire. Methods A prospective study in pediatric otolaryngology unit. Patients were treated either surgically or pharmacologically. Following informed consent, parents of pediatric CRS patients completed the translated and validated Hebrew version (SN-5H) prior to treatment and after three months. We analyzed the results of both treatment arms according to success (achieving minimal clinically important difference; MCID). Results 102 children aged 5–12 years and their caregivers participated (74 CRS patients and 28 controls without CRS). SN-5H items scores were significantly higher in CRS patients compared to controls (p < 0.001). Baseline activity scores were higher, while baseline emotional scores were lower in MCID( +) CRS patients, compared to MCID(-) CRS patients (p < 0.05). High emotional stress and low activity scores at baseline were associated with poorer odds to achieve MCID. Conclusions The SN-5H questionnaire is invaluable tool for assessing pediatric CRS patients. Psychosocial aspects of CRS significantly affect QoL and should be addressed in the office pre-treatment. The SN-5H can aid in highlighting patients in need for further reassurance and psychosocial support to manage expectations, and to improve QoL.

Background Coeliac disease (CD) is a life-long food-related disorder with a global prevalence of approximately 1%. Patients with CD often experience reduced health-related quality of life that could improve with a strict adherence to a gluten-free diet (GFD). Objectives To obtain visual analogue scale (VAS), time trade-off (TTO) and willingness-to-pay (WTP) values amongst patients with CD. Methods In 2020–2021, a cross-sectional online survey was conducted amongst 312 adult CD patients in Hungary. Patients completed the Gastrointestinal Symptom Rating Scale (GSRS) and evaluated their current health and three hypothetical health state vignettes defined based on dietary adherence using VAS, conventional 10-year TTO and WTP. Multivariate regressions were used to explore the effect of patients’ demographic and clinical characteristics on utility and WTP values. Results Mean VAS values for current health and ‘CD with strict adherence to GFD’, ‘CD with loose adherence to GFD’ and ‘CD without GFD’ hypothetical health states were 79.69 ± 18.52, 85.36 ± 16.18, 62.44 ± 19.91 and 36.69 ± 25.83, respectively. Corresponding mean TTO utilities were: 0.90 ± 0.19, 0.91 ± 0.20, 0.87 ± 0.23 and 0.76 ± 0.29. Mean annual WTP values for returning to full health were: €845 ± 1077, €648 ± 1002, €862 ± 1135 and €1251 ± 1496. Older age at diagnosis, male sex, more severe gastrointestinal symptoms (GSRS) and having comorbidities were associated with lower VAS and TTO or higher WTP values for current own health (p < 0.05). Conclusion This is the first study to report TTO utilities for CD health states. Strict adherence to the GFD may result in substantial health gains in symptomatic patients. Utilities and WTP results can be used to estimate benefits of GFD in cost-utility and cost–benefit analyses.

Purpose To generate a normative profile for the EQ-5D-5L dimensions, EQ-5D-5L index, and EQ-VAS scores of the general Thai population and to examine the associations between sociodemographic characteristics and their norm-based scores. Methods Data from 2019 general Thai samples were employed to estimate the norm-based scores elicited using the Thai EQ-5D-5L value set. Descriptive statistics were used to estimate the norm-based scores stratified by gender and six age bands to obtain the normative profile for the general Thai population. Multivariable logistic and Tobit regression models were used to investigate the relationships between sociodemographic characteristics and EQ-5D-5L dimensions, EQ-5D-5L index, and EQ-VAS scores. Results The mean EQ-5D-5L index and EQ-VAS scores were 0.931 and 82.3, respectively. Approximately 88.9% of the participants reported 19 out of 3125 (3%) possible health states. The odds of having problems with mobility was greatest for the sample aged ≥ 65 years and declined with decreasing age. Women, samples with advancing age, and those with a household income of ≤ 10,000 Baht/month and fair and poor health perceptions were more likely to report a lower EQ-5D-5L index. Furthermore, advanced age and fair and poor health perception were significantly associated with lower EQ-VAS scores. Conclusion The EQ-5D-5L population norms were established as the benchmark for both EQ-5D-5L index and EQ-VAS scores for the general Thai population. This is expected to support the health service research and inform policymakers on the allocation of limited healthcare resources.

Purpose Studies have found that many people who sustain an injury can experience adverse outcomes for a considerable time thereafter. Māori, the Indigenous peoples of Aotearoa me Te Waipounamu (New Zealand; NZ), are no exception. The Prospective Outcomes of Injury Study (POIS) found that almost three-quarters of Māori participants were experiencing at least one of a range of poor outcomes at two years post-injury. The aim of this paper was to estimate the prevalence, and identify predictors, of adverse health-related quality of life (HRQoL) outcomes in the POIS-10 Māori cohort, 12 years after participants sustained an injury. Methods Interviewers reached 354 individuals who were eligible to participate in a POIS-10 Māori interview, to be conducted a decade after the last phase of POIS interviews (held 24 months post-injury). The outcomes of interest were responses to each of the five EQ-5D-5L dimensions at 12 years post-injury. Potential predictors (i.e., pre-injury sociodemographic and health measures; injury-related factors) were collected from earlier POIS interviews. Additional injury-related information was collected from administrative datasets proximate to the injury event 12 years prior. Results Predictors of 12-year HRQoL outcomes varied by EQ-5D-5L dimension. The most common predictors across dimensions were pre-injury chronic conditions and pre-injury living arrangements. Conclusion An approach to rehabilitation where health services proactively enquire about, and consider the broader aspects of, patient health and wellbeing throughout the injury recovery process, and effectively coordinate their patients’ care with other health and social services where necessary, may help improve long-term HRQoL outcomes for injured Māori.

Purpose The primary goal of this analysis is to describe the health-related quality of life (HRQoL), medical history, and medication use among adolescents and adults individuals with Angelman syndrome (AS). Methods The analysis uses baseline data collected during the STARS study, a double-blind placebo controlled trial of gaboxadol (OV101) in adolescents and adults with AS. The HRQoL was estimated using EuroQoL 5-Dimension 5-Level (EQ-5D) health questionnaire proxy 1 version, which was completed by the caregivers. EQ-5D consists of two parts, a 5-dimension descriptive and a visual analogue scale (VAS) component. The utility score derived from EQ-5D ranges from 0 to 1 (perfect health) and VAS ranges from 0 to 100 (perfect health). Results 87 individuals with AS were included in the present analysis. The mean utility score was 0.44 ± 0.20 and VAS score was 84 ± 1.5. The EQ-5D data indicated that the self-care, mobility and daily activities were most impacted. All adolescents (100%) and most adults (93%) had at least moderate problems with self-care activities, such as washing or dressing themselves. More than half (55%) of the adolescents and adults had at least moderate issues with mobility and usual activities. Approximately, 30% of adolescents and adults had moderate to extreme problems with anxiety/depression. High baseline concomitant use of medications was observed across both age groups with an average of 5 medications being used per person. Conclusion This study highlights the impact of AS on HRQoL and medication utilization among adolescents and adults individuals with AS.

Purpose To determine factors associated with postpartum quality of life (QOL). Methods An analytic cross-sectional design was used in this study, and data was collected from December 2019 to March 2020. Participants were 252 postpartum women visiting eight governmental primary healthcare centers in Madinah city, Saudi Arabia. Data were collected using a sociodemographic questionnaire, the World Health Organization Quality of Life Assessment-BREF, Multidimensional Scale of Perceived Social Support (MSPSS), and Edinburgh Postnatal Depression Scale. Sleep problems were assessed using an item from the Prime-MD Patient Health Questionnaire. Results Maternal age between 26 and 35 years had a significant independent association with the physical health domain of QOL (p < .01). Postpartum depression was significantly associated with lower QOL in all dimensions (p < .01). In addition, sleep problems were associated with three out of the four QOL domains (p < .05). The significant other subscale of the MSPSS was significantly associated with higher QOL scores in all dimensions (p < .01); additionally, family and friends subscales of the MSPSS were significantly associated with the social domain of QOL (p < .01). Conclusions Maternal QOL, during the postpartum period, showed negative associations with age, postpartum depression, and sleep disturbances. Ultimately, social support appeared to be an essential factor in mothers’ ability to cope with the physical and psychological problems experienced during this period.

Purpose The European Organisation for Research and Treatment of Cancer (EORTC) quality of life core questionnaire (QLQ-C30) is a validated and widely-used Patient-Reported Outcome Measure for measuring the health-related quality of life (HRQoL) of cancer patients. To facilitate interpretation of results obtained in studies using the EORTC QLQ-C30, we generated normative data for the Dutch general population, stratified by age and sex. Methods Dutch participants were selected from a larger cross-sectional online panel research study collecting EORTC QLQ-C30 general population normative data across 15 countries. EORTC QLQ-C30 raw scores based on a 4-point response scale were transformed to linear scores ranging from 0 to 100. Transformed scores were weighted based on the United Nations population distribution statistics and presented by age and sex/age. Differences in scale scores of ≥ 10 points in HRQoL were applied to indicate clinical relevance. Results One thousand respondents completed the online survey. Stratified by age, clinically meaningful differences were observed, with worse physical functioning scores and better emotional functioning scores with increased age. Symptom scores remained stable across age groups, except for small age differences observed for fatigue, nausea/vomiting, diarrhoea, and financial difficulties. Stratified by sex/age, men generally scored better for both functioning and symptoms. However, these differences were not clinically meaningful. Conclusions These updated normative EORTC QLQ-C30 for the Dutch general population can be used to better interpret HRQoL data obtained from Dutch cancer patients. Being part of a larger international study, these data can further be used for inter-country comparisons in multi-national studies.

Purpose Using data from a randomized controlled trial for treatment of prescription-type opioid use disorder in Canada, this study examines sensitivity to change in three preference-based instruments [EQ-5D-3L, EQ-5D-5L, and the Health Utilities Index Mark 3 (HUI3)] and explores an oft-overlooked consideration when working with contemporaneous responses for similar questions—data quality. Methods Analyses focused on the relative abilities of three instruments to capture change in health status. Distributional methods were used to categorize individuals as ‘improved’ or ‘not improved’ for eight anchors (seven clinical, one generic). Sensitivity to change was assessed using area under the ROC (receiver operating characteristics) curve (AUC) analysis and comparisons of mean change scores for three time periods. A ‘strict’ data quality criteria, defined a priori, was applied. Analyses were replicated using ‘soft’ and ‘no’ criteria. Results Data from 160 individuals were used in the analysis; 30% had at least one data quality violation at baseline. Despite mean index scores being significantly lower for the HUI3 compared with EQ-5D instruments at each time point, the magnitudes of change scores were similar. No instrument demonstrated superior sensitivity to change. While six of the 10 highest AUC estimates were for the HUI3, ‘moderate’ classifications of discriminative ability were identified in 12 (of 22) analyses for each EQ-5D instrument, compared with eight for the HUI3. Conclusion Negligible differences were observed between the EQ-5D-3L, EQ-5D-5L, and HUI3 regarding the ability to measure change. The prevalence of data quality violations—which differed by ethnicity—requires further investigation.

Objectives The visual analogue scale (VAS) has been used in the context of health and healthcare for various purposes, for example, to measure pain and to provide a single-index measure of health-related quality of life (HRQoL). This scoping review aims to describe how the VAS has been used for health state valuation in the published literature. Methods The search was carried out in Medline, Web of Science and PsycInfo. The findings of the included articles were tabulated and presented descriptively using frequencies and proportions. Results The database search yielded 4856 unique articles, out of these, 308 were included. In 83% of the articles, the main purpose for using a VAS was to value health states. The two most common perspectives when valuing health states with a VAS were hypothetical (44%) and own health (34%). Some (n = 14) articles used the VAS in the context of economic evaluations, including calculating quality-adjusted life years (QALYs). A large variation in the design of the VAS was found, including the description of the lower and upper anchors. Advantages and disadvantages with using a VAS were mentioned in 14% of the included articles. Conclusion The VAS has been a common method for valuing health states, both as a stand-alone method and in combination with other valuation methods. Despite its widespread use, the design of the VAS has been inconsistent which makes comparison of results across studies challenging. Further research on the role of using the VAS in economic evaluations is warranted.

Introduction Generic or condition-specific Patient-reported Outcome Measures (PROMs) are used to measure physical, mental, and social aspects of health to promote patient-centered care. This scoping review aims to identify and summarize generic and condition-specific PRO domains and PROMs that have been assessed and used in liver transplant (LT) candidates and recipients. Methods We searched Medline, Embase, Cochrane Database of Systematic Reviews and Register of Trials, PsychInfo, and CINAHL from inception to 08/26/2020. Included studies addressed a PRO or PROM in LT candidates or recipients. Results After screening, 341 studies yielded 189 unique PRO domains. Mental health domains (depression, anxiety, and guilt) were most frequently assessed, followed by domains of physical and social health. Fifty-one generic and three condition-specific unique PROMs were identified, with only 13% (n = 45) of studies including condition-specific tools. Discussion The most frequent PROMs were the SF-36, Nottingham Health Profile, Hospital Anxiety and Depression Scale, followed by the Liver Disease Quality of Life (LDQoL). Very few studies used transplant-specific PROMs, which may partly be related to the scarcity of LT-specific instruments. We will use these results in future qualitative research to identify PROs and PROMs to build an electronic PROM toolkit to facilitate patient-centered LT care.

Purpose To determine whether treatment of anal high-grade squamous intraepithelial lesions (HSIL), vs active monitoring, is effective in reducing incidence of anal cancer in persons living with HIV, the US National Cancer Institute funded the Phase III ANal Cancer/HSIL Outcomes Research (ANCHOR) clinical trial. As no established patient-reported outcomes (PRO) tool exists for persons with anal HSIL, we sought to estimate the construct validity and responsiveness of the ANCHOR Health-Related Symptom Index (A-HRSI). Methods The construct validity phase enrolled ANCHOR participants who were within two weeks of randomization to complete A-HRSI and legacy PRO questionnaires at a single time point. The responsiveness phase enrolled a separate cohort of ANCHOR participants who were not yet randomized to complete A-HRSI at three time points: prior to randomization (T1), 14–70 (T2), and 71–112 (T3) days following randomization. Results Confirmatory factor analysis techniques established a three-factor model (i.e., physical symptoms, impact on physical functioning, impact on psychological functioning), with moderate evidence of convergent validity and strong evidence of discriminant validity in the construct validity phase (n = 303). We observed a significant moderate effect for changes in A-HRSI impact on physical functioning (standardized response mean = 0.52) and psychological symptoms (standardized response mean = 0.60) from T2 (n = 86) to T3 (n = 92), providing evidence of responsiveness. Conclusion A-HRSI is a brief PRO index that captures health-related symptoms and impacts related to anal HSIL. This instrument may have broad applicability in other contexts assessing individuals with anal HSIL, which may ultimately help improve clinical care and assist providers and patients with medical decision-making.

Purpose During the first SARS-CoV-2-infection wave, a deterioration in emotional well-being and increased need for mental health care were observed among patients treated or being treated for breast cancer. In this follow-up study, we assessed patient-reported quality of life (QoL), physical functioning, and psychosocial well-being during the second SARS-CoV-2-infection wave in a large, representative cohort. Methods This longitudinal cohort study was conducted within the prospective, multicenter UMBRELLA breast cancer cohort. To assess patient-reported QoL, physical functioning and psychosocial well-being, COVID-19-specific surveys were completed by patients during the first and second SARS-CoV-2-infection waves (April and November 2020, respectively). An identical survey was completed by a comparable reference population during the second SARS-CoV-2-infection waves. All surveys included the validated EORTC-QLQ-C30/BR23, HADS and “De Jong-Gierveld Loneliness” questionnaires. Pre-COVID-19 EORTC-QLQ-C30/BR23 and HADS outcomes were available from UMBRELLA. Response rates were 69.3% (n = 1106/1595) during the first SARS-CoV-2-infection wave and 50.9% (n = 822/1614) during the second wave. A total of 696 patients responded during both SARS-CoV-2-infection waves and were included in the analysis comparing patient-reported outcomes (PROs) during the second SARS-CoV-2-infection wave to PROs during the first wave. Moreover, PROs reported by all patients during the second SARS-CoV-2-infection wave (n = 822) were compared to PROs of a similar non-cancer reference population (n = 241) and to their pre-COVID-19 PROs. Results Patient-reported QoL, physical functioning, and psychosocial well-being of patients treated or being treated for breast cancer remained stable or improved from the first to the second SARS-CoV-2-infection wave. The proportion of emotional loneliness reduced from 37.6 to 29.9% of patients. Compared to a similar non-cancer reference population, physical, emotional, and cognitive functioning, future perspectives and symptoms of dyspnea and insomnia were worse in patients treated or being treated for breast cancer during the second SARS-CoV-2-infection wave. PROs in the second wave were similar to pre-COVID-19 PROs. Conclusion Although patients scored overall worse than individuals without breast cancer, QoL, physical functioning, and psychosocial well-being did not deteriorate between the first and second wave. During the second wave, PROs were similar to pre-COVID-19 values. Overall, current findings are cautiously reassuring for future mental health of patients treated or being treated for breast cancer.

Purpose The animated activity questionnaire (AAQ) is a computer-based measure of activity limitations. To answer a question, patients choose the animation of a person performing an activity that matches their own level of limitation. The AAQ has not yet been tested for suitability to be applied as computer-adaptive test (CAT). Thus, the objective of this study was to develop and evaluate an AAQ-based CAT to facilitate the application of the AAQ in daily clinical care. Methods Patients (n = 1408) with hip/knee osteoarthritis from Brazil, Denmark, France, The Netherlands, Norway, Spain, and the UK responded to all 17 AAQ items. Assumptions of item-response theory (IRT) modelling were investigated. To establish item parameters for the CAT, a graded response model was estimated. To evaluate the performance of post-hoc simulated AAQ-based CATs, precision, test length, and construct validity (correlations with well-established measures of activity limitations) were evaluated. Results Unidimensionality (CFI = 0.95), measurement invariance (R²-change < 2%), and IRT item fit (S-X²p > .003) of the AAQ were supported. Performing simulated CATs, the mean test length was more than halved (≤ 8 items), while the range of precise measurement (standard error ≤ 0.3) was comparable to the full AAQ. The correlations between original AAQ scores and three AAQ-CAT versions were ≥ 0.95. Correlations of AAQ-CAT scores with patient-reported and performance measures of activity limitations were ≥ 0.60. Conclusion The almost non-verbal AAQ-CAT is an innovative and efficient tool in patients with hip/knee osteoarthritis from various countries, measuring activity limitations with lower respondent burden, but similar precision and construct validity compared to the full AAQ.

Purpose To estimate the health-related quality of life (HRQOL) according to glycemic status, and its relationship with sociodemographic and clinical factors in a population at risk of developing type 2 diabetes (T2D). Methods Cross-sectional study, using cluster sampling. Data were collected from 1135 participants over 30 years of age, at risk of developing T2D from the PREDICOL project. Participants' glycemic status was defined using an oral glucose tolerance test (OGTT). Participants were divided into normoglycemic subjects (NGT), prediabetes and diabetics do not know they have diabetes (UT2D). HRQOL was assessed using the EQ-5D-3L questionnaire of the EuroQol group. Logistic regression and Tobit models were used to examine factors associated with EQ-5D scores for each glycemic group. Results The mean age of participants was 55.6 ± 12.1 years, 76.4% were female, and one in four participants had prediabetes or unknown diabetes. Participants reported problems most frequently on the dimensions of Pain/Discomfort and Anxiety/Depression in the different glycemic groups. The mean EQ-5D score in NGT was 0.80 (95% CI 0.79–0.81), in prediabetes, 0.81 (95% CI 0.79–0.83), and in participants with UT2D of 0.79 (95% CI 0.76–0.82), respectively. Female sex, older age, city of residence, lower education, receiving treatment for hypertension, and marital status were significantly associated with lower levels of HRQOL in the Tobit regression analysis. Conclusions HRQOL of NGT, prediabetes, and UT2D participants was statistically similar. However, factors such as gender, age. and place of residence were found to be significant predictors of HRQOL for each glycemic group.

Introduction There is an historical initiative to establish common theoretical ground to support a framework for assessing health-related quality of life (HRQL). Our aim was to add to this effort with an analysis of theoretical/philosophical themes embedded in HRQL questionnaires and patient reports. Methods and Results We reviewed recent developments in HRQL assessment. This included analyzing a representative sample of psychometric measures of HRQL to schematically summarize core theoretical/philosophical themes that are embedded in questionnaire items. This analysis indicated a state-based framework for HRQL that was characterized by themes of hedonic and eudaimonic well-being, and desire-satisfaction. In contrast, a review of patient reports of HRQL indicated a process-based framework where goal-directed activities aimed to secure aspirational life goals while striving to accept the reality of declining health. Given this difference in HRQL themes we used a meta-philosophical approach, based on Hadot’s idea of philosophy as a way of living, to identify a process-based theoretical framework for HRQL assessment that addressed patient-reported themes. The Stoic modification of eudaimonic well-being was examined where HRQL and well-being are viewed as a process (vs. state) aimed at transforming the experience of loss or grief in response to adversity through goal-directed activities/exercises (euroia biou, good flow in life). We then introduced a complementary research agenda for HRQL assessment that incorporates self-reported, goal-directed activities that are initiated or maintained to promote HRQL. Conclusion A process-based approach to HRQL assessment may increase the spectrum of clinically relevant features that currently comprise operational measures of this patient-reported appraisal.

Purpose Health utilities are challenging to ascertain in children and have not been studied in pediatric Crohn’s disease (CD) and ulcerative colitis (UC). The objective was to assess discriminative validity by comparing utilities elicited using the Child Health Utility-9 Dimension (CHU9D) to the Health Utilities Index (HUI) across multiple disease activity scales in pediatric UC and CD. Methods Preference-based instruments were administered to 188 children with CD and 83 children with UC aged 6 to 18 years. Utilities were calculated using CHU9D adult and youth tariffs, and HUI2 and HUI3 algorithms in children with inactive (quiescent) and active (mild, moderate, and severe) disease. Differences between instruments, tariff sets and disease activity categories and were tested statistically. Results In CD and UC, all instruments detected significantly higher utilities for inactive compared to active disease (p < 0.05). Mean utilities for quiescent disease ranged from 0.810 (SD 0.169) to 0.916 (SD 0.121) in CD and from 0.766 (SD 0.208) to 0.871 (SD 0.186) in UC across instruments. Active disease mean utilities ranged from 0.694 (SD 0.212) to 0.837 (SD 0.168) in CD and from 0.654 (SD 0.226) to 0.800 (SD 0.128) in UC. Conclusion CHU9D and HUI discriminated between levels of disease activity in CD and UC regardless of the clinical scale used, with the CHU9D youth tariff most often displaying the lowest utilities for worse health states. Distinct utilities for different IBD disease activity states can be used in health state transition models evaluating the cost-effectiveness of treatments for pediatric CD and UC.

Purpose A significant number of people will experience prolonged symptoms after COVID-19 infection that will greatly impact functional capacity and quality of life. The aim of this study was to identify trajectories of health-related quality of life (HRQOL) and their predictors among adults diagnosed with COVID-19. Methods This is a retrospective analysis of an ongoing prospective cohort study (BQC-19) including adults (≥18y) recruited from April 2020 to March 2022. Our primary outcome is HRQOL using the EQ-5D-5L scale. Sociodemographic, acute disease severity, vaccination status, fatigue, and functional status at onset of the disease were considered as potential predictors. The latent class mixed model was used to identify the trajectories over an 18-month period in the cohort as a whole, as well as in the inpatient and outpatient subgroups. Multivariable and univariable regressions models were undertaken to detect predictors of decline. Results 2163 participants were included. Thirteen percent of the outpatient subgroup (2 classes) and 28% in the inpatient subgroup (3 classes) experienced a more significant decline in HRQOL over time than the rest of the participants. Among all patients, age, sex, disease severity and fatigue, measured on the first assessment visit or on the first day after hospital admission (multivariable models), were identified as the most important predictors of HRQOL decline. Each unit increase in the SARC-F and CFS scores increase the likelihood of belonging to the declining trajectory (univariable models). Conclusion Although to different degrees, similar factors explain the decline in HRQOL over time among the overall population, people who have been hospitalized or not. Clinical functional capacity scales could help to determine the risk of HRQOL decline.

Purpose The collection and use of patient reported outcomes (PROs) in care-based child health research raises challenging ethical and logistical questions. This paper offers an analysis of two questions related to PROs in child health research: (1) Is it ethically obligatory, desirable or preferable to share PRO data collected for research with children, families, and health care providers? And if so, (2) What are the characteristics of a model best suited to guide the collection, monitoring, and sharing of these data? Methods A multidisciplinary team of researchers, providers, patient and family partners, and ethicists examined the literature and identified a need for focus on PRO sharing in pediatric care-based research. We constructed and analyzed three models for managing pediatric PRO data in care-based research, drawing on ethical principles, logistics, and opportunities to engage with children and families. Results We argue that it is preferable to share pediatric PRO data with providers, but to manage expectations and balance the risks and benefits of research, this requires a justifiable data sharing model. We argue that a successful PRO data sharing model will allow children and families to have access to and control over their own PRO data and be engaged in decision-making around how PROs collected for research may be integrated into care, but require support from providers. Conclusion We propose a PRO data sharing model that can be used across diverse research settings and contributes to improved transparency, communication, and patient-centered care and research.