Health-related Quality Of Life Values Research Articles

Exploring health-related quality of life in Portuguese Informal Caregivers

Abstract Background Caregiving can be associated with several adverse health impacts, such as in terms of mental health, health services use, or health-related quality of life (HRQoL). This study assessed HRQoL levels in Portuguese Informal Caregivers, and their relationship with caregiver psychosocial characteristics (e.g., perceived general and mental health; perceived level of support) and caregiving circumstances (e.g., caregiving duration, caregiving hours). Methods A cross-sectional study was conducted through an online survey directed to informal caregivers. HRQoL was measured using the Portuguese translation of the EQ-5D-5L. This instrument yields two values for HRQoL: a health profile (EQvalue) covering five dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), and a visual analogue scale (EQvas). Bivariate analyses were performed and results with p-values below 0.05 were considered statistically significant. Results A total of 760 informal caregivers were surveyed (mean age 57 years (±9.9), 92.5% female, 41.5% at least university level, 44.4% works full or part-time). The mean total score for EQvalue (n = 733) was 0.8 (SD = 0.2). The mean total score for EQvas (n = 760) was 63.1 (SD = 21.5). The perceived general (p < 0.001) and mental (p < 0.001) health status, and the perceived access to healthcare (p < 0.001) were associated with EQvalue score. Also, EQvalue score was found to be directly correlated with the perceived level of support (r = 0.142; p < 0.001) and competence (r = 0,237; p < 0.001), and inversely correlated with the number of caregiving hours per day (r= -0,150; p = < 0,001) and the caregiving duration (r= -0,109; p = 0.008). Conclusions A significant relationship between caregiver HRQoL and several caregiver psychosocial variables was identified. Investing in and promoting the use of respite care services, as well as ensuring better access to healthcare for caregivers, appears important for improving caregivers’ HRQoL. Key messages • Improving caregivers’ access to healthcare and education or training programs (promoting a greater sense of competence) can be important in helping them maintain their quality of life. • Increasing the availability and promoting the use of respite care services by caregivers can be important in helping them maintain their quality of life.

A systematic review and meta-analysis of health-related quality of life (HRQoL) outcomes in randomised controlled trials for pulmonary arterial hypertension (PAH)

Abstract Introduction ESC guidelines recognise the under use of disease-specific health-related quality of life (HRQoL) instruments. These include CAMPHOR, PAH-SYMPACT, Emphasis-10 and Living with Pulmonary Hypertension (LPH). Patients highly value HRQoL as a treatment outcome in PAH. This is the first meta-analysis to evaluate HRQoL instruments and outcomes in RCTs in pulmonary arterial hypertension (PAH). Purpose To evaluate which PAH-RCT interventions improve HRQoL outcomes and compare the suitability of HRQoL instruments used. Method We searched MEDLINE (Jan 1970 to Dec 2023) and Cochrane Library from Jan 1990 to Dec 2023 (EMBASE, CT.gov, ICTRP, CINAHL) for RCTs in PAH. Abstracts and unpublished studies were excluded and PRISMA reporting structure followed. Trials meeting final selection criteria included HRQoL instruments sufficiently powered to detect a minimal clinically important difference (MCID). Effective sample sizes were calculated in GPower3.1 from a two independent means model for 80% power, 5% significance, one-tailed test. Meta-analysis was performed in SPSSv28.1. Full search criteria are registered on PROSPERO (ID: CRD42024484021). Results After removal of duplicates, 896 records were screened, 43 studies included a HRQoL endpoint. 8 studies were included in the meta-analysis after exclusion for insufficient data (n=10) or power (n=25). 3 of these did not meet the MCID (>33m) for six-minute walk distance (6MWD) and evaluated separately to discriminate accuracy of HRQoL instrument. Instruments available for comparison were Short Form-36(SF-36), EuroQol (EQ-5D-5L) and Living with Pulmonary Hypertension (LPH). Studies not reaching MCID for 6MWD also showed no change in HRQoL (figure 1). Ambrisentan and exercise training demonstrate a significant improvement (p<0.05) in SF-36 physical(P) component (figure 2). This component was anchored to determine 6MWD MCID; other HRQoL values responded variably. Utility weighting to account for geographical variation was not reported with EQ-5D-5L and SF-36 in multicentre studies however AIR 2002 (Iloprost) was significant for both EQ-5D-5L instruments. The total component score of the disease-specific LPH was more sensitive to functional improvements and overall change in HRQoL compared to EQ-5D-5L (p<0.05). Conclusion Fewer than 10% of all RCTs in PAH have adequately considered a HRQoL outcome measure. 8 RCTs were powered to assess HRQoL. Iloprost, ambrisentan, riociguat and exercise training interventions demonstrate significant improvement in HRQoL. LPH was the only PAH-instrument powered to detect change. LPH was more sensitive to HRQoL than generic instruments and is multidimensional. Geographical indexing should be considered for generic instruments such as EQ-5D-5L and SF-36 in multicentre trials. As a highly valued outcome for patients, future studies should prioritise HRQoL as an endpoint.Figure 1Figure 2

Health-related quality of life and prognosis in cardiac sarcoidosis

Abstract Background Cardiac sarcoidosis (CS) is characterized by inflammatory myocardial disease, often leading to conduction disturbances, ventricular tachycardias, and heart failure. While factors like initial symptoms, LVEF, and troponin levels are known prognostic indicators, the impact of CS on health-related quality of life (HRQoL) is not well-understood. Additionally, limited data exists on the predictive value of FDG-PET in CS. Purpose This study aimed to assess HRQoL in CS patients and investigate its prognostic relevance concerning cardiac events. Methods Patients diagnosed with CS completed the RAND-36 general health-related questionnaire, covering eight dimensions. Clinical data were obtained from the Finnish myocardial inflammatory diseases registry. Follow-up data on cardiac events, including life-threatening ventricular tachyarrhythmias (VT/VF), heart transplant, left ventricular assist device (LVAD) implantation, pericardial tamponade, or death, were collected over 36 months. Results Of the 240 CS patients surveyed, 179 (75%) were female, with a median age of 56 [IQR 48 – 64]. CS patients reported significantly lower RAND-36 scores across all dimensions compared to the general Finnish population. Over the 3-year follow-up, 38 (16%) patients experienced cardiac-related adverse events, including VT/VF (26 cases), deaths (8 cases), heart transplants (2 cases), LVAD implantation (1 case), and pericardial tamponade (1 case). We divided CS patients into those without adverse event (N=202) and with adverse events (N=38). Interestingly, in four dimensions of PF 70.0 [50.0-90.0] vs 60.0 [33.8-80.0], p=0.019, RP 50.0 [0.0-100.0] vs 0.00 [0-56.3], p=0.002, MH 76.0 [59.0-88.0] vs 66.0 [51.0-84.0], p=0.036, and SF 75.0 [50-100] vs 62.5 [37.5-78.1], p=0.037 were significantly lower scores in the event group. Univariate Cox regression analysis (Table 1) revealed that physical functioning (PF) and role-physical (RP) dimensions, along with certain clinical factors, such as younger age, higher NYHA classification, elevated proBNP levels, lower hemoglobin levels, and the main manifestation of the disease as ventricular tachyarrhythmia, independently predicted adverse cardiac events. In the multivariate analysis, lower role-physical scores and younger age remained independent prognostic factors for adverse cardiac events. Conclusions Patients with CS demonstrated lower HRQoL compared to the general Finnish population. This study enhances our understanding of prognosis in CS and underscores the predictive value of HRQoL in identifying future adverse cardiac events, highlighting its relevance in the clinical management of these patients.

Impact of colonoscopy on health-related quality of life: findings from the RECEDE study

BackgroundColonoscopy is a valuable diagnostic tool but the procedure and the preparation for it cause anxiety and discomfort that impacts on patients’ health-related quality of life (HRQoL). The ‘disutility’ of undergoing an invasive colonoscopy needs to be considered and accounted for in comprehensive cost-utility analyses that compare different diagnostic strategies, yet there is little empirical evidence that can be used in such studies. To fill this gap, we collected and analysed data on the effect of a colonoscopy examination on patients’ HRQoL that can be used in economic evaluations.MethodsPatients scheduled to undergo a colonoscopy at a large NHS hospital were asked to complete the EuroQol EQ-5D-5 L instrument: (i) before the procedure, at the time of consent (T1), (ii) while undergoing bowel preparation (T2) and (iii) within 24 h after the procedure (T3). Complete responses were translated into preference-based HRQoL (utility) values using a UK-specific value set and were analysed using descriptive and inferential statistical analyses.ResultsTwo-hundred and seventy-one patients with gastrointestinal symptoms referred for a colonoscopy provided complete EQ-5D-5 L questionnaires at all three assessment points. At T1, the mean EQ-5D-5 L value was 0.76 (95%CI: 0.734–0.786). This value dropped to 0.727 at T2 (95%CI: 0.7–0.754, before increasing again to 0.794 (95%CI: 0.768–0.819) at T3. Both changes were statistically significant (p-value < 0.001).ConclusionsPreference-based HRQoL (utility) values reported by patients undergoing a colonoscopy dropped during bowel preparation and rose again shortly after the colonoscopy. This pattern was largely consistent across patients with different characteristics, symptoms and diagnoses.

ETMR-09. HEALTH-RELATED QUALITY OF LIFE OF CHILDREN, ADOLESCENTS AND ADULTS AFTER DIAGNOSIS OF CHILDHOOD-ONSET MENINGIOMA

Abstract BACKGROUND Meningiomas are rare intracranial tumors in childhood. Childhood-onset meningiomas differ from their adult counterparts in terms of frequency, molecular features and clinical presentation. There is limited data on health-related quality of life (HRQoL) of pediatric meningioma patients. The aim of this study was to analyze the HRQoL of pediatric or adult patients after completion of treatment for childhood-onset meningioma. Further, the influence of disease-specific characteristics on HRQoL, fatigue or functional capacity was analyzed. METHODS In this cross-sectional study, the HRQoL of 8 pediatric and 27 adult meningioma patients from the clinical trials HIT-ENDO, KRANIOPHARYNGEOM 2000/2007 and KRANIOPHARYNGEOM Registry 2019 was assessed. The validated questionnaires EQ-5D-Y, EQ-5D-3L, PedsQL Generic Core Scale, EORTC QLQ-C30 and QLQ-BN20 were used. Patients’ fatigue and functional capacity were scored using the EORTC QLQ-C30, the PedsQL Multidimensional Fatigue Scale and the FMH questionnaire. Exploratory data analysis was performed using univariate, bivariate and multivariate analyses (α=0.05). RESULTS The HRQoL was assessed in median 10 (IQR: 5-17) years after diagnosis. The mean HRQoL value was 86 (IQR: 79-92) for pediatric and 75 (IQR: 67-92) for adult patients using the PedsQL Generic Core Scale or EORTC QLQ-C30 questionnaire, respectively. Most limitations were reported in emotional function and fatigue. The median functional capacity of patients was at the 90th (IQR: 65-90) percentile. HRQoL was lower in adults with neurofibromatosis type 2 or at older age (p=0.009) and higher HRQoL after progressive disease. Age at diagnosis, WHO grade, residual tumor, use of radiotherapy, tumor location, tumor entity (meningioma vs. craniopharyngioma), degree of disability, or time since diagnosis did not affect HRQoL in adults. CONCLUSION The median HRQoL of childhood-onset meningioma patients indicates that it is comparable to the healthy normal population. Nevertheless, HRQoL is a construct that can be influenced in many ways. Further research on HRQoL is warranted.

A Cost-Utility Analysis of Add-On Cannabidiol Versus Usual Care Alone for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in England and Wales.

The aim of this study was to evaluate the cost effectiveness of plant-derived highly purified cannabidiol (Epidyolex® in the UK; 100mg/mL oral solution) as an add-on treatment to usual care for the management of treatment-refractory seizures associated with tuberous sclerosis complex (TSC) in patients aged ≥2 years. A cohort-based model was developed using a National Health Service perspective and lifetime horizon. Health states were based on weekly seizure frequency and seizure-free days, utilizing patient-level data from the GWPCARE6 trial (ClinicalTrials.gov identifier: NCT02544763). Two independent regression models were applied to individual patient-level data to predict seizure-free days and seizure frequency. Healthcare resource utilization data were sourced from a Delphi panel, and patient and caregiver health-related quality of life values were elicited using vignettes valued by the general public. Outcomes relating to TSC-associated neuropsychiatric disorders were modeled with costs and quality-adjusted life-years sourced from published literature. In the base case, compared with usual care alone, 12mg/kg/day cannabidiol was associated with an incremental cost-effectiveness ratio (ICER) of £23,797. The National Institute for Health and Care Excellence disease severity modifier reduced the ICER to £19,831. Probabilities of cost effectiveness at willingness-to-pay thresholds of £20,000 and £30,000 were 30% and 52%, respectively, for the base case and 39% and 66%, respectively, for the disease severity modifier. Results were robust to sensitivity and scenario analyses. At 12mg/kg/day and an ICER threshold of £20,000-£30,000, we provide evidence for the cost effectiveness of add-on cannabidiol treatment for patients with TSC-associated seizures aged ≥2 years who are refractory to current treatment.

The RETRIEVE Checklist for Studies Reporting the Elicitation of Stated Preferences for Child Health-Related Quality of Life.

Recent systematic reviews show varying methods for eliciting, modelling, and reporting preference-based values for child health-related quality-of-life (HRQoL) outcomes, thus producing valuesets with different characteristics. Reporting in many of the reviewed studies was found to be incomplete and inconsistent, making them difficult to assess. Checklists can help to improve standards of reporting; however, existing checklists do not address methodological issues for valuing child HRQoL.Existing checklists also focus on reporting methods and processes used in developing HRQoL values, with less focus on reporting of the values' key characteristics and properties. We aimed to develop a checklist for studies generating values for child HRQoL, including for disease-specific states and value sets for generic child HRQoL instruments. A conceptual model provided a structure for grouping items into five modules. Potential items were sourced from an adult HRQoL checklist review, with additional items specific to children developed using recent reviews. Checklist items were reduced by eliminating duplication and overlap, then refined for relevance and clarity via an iterative process. Long and short checklist versions were produced for different user needs. The resulting long RETRIEVE contains 83 items, with modules for reporting methods (A-D) and characteristics of values (E), for researchers planning and reporting child health valuation studies. The short RETRIEVE contains 14 items for decision makers or researchers choosing value sets. Applying the RETRIEVE checklists to relevant studies suggests feasibility. RETRIEVE has the potential to improve completeness in the reporting of preference-based values for child HRQOL outcomes and to improve assessment of preference-based value sets.

Long-term outcomes with haloperidol versus placebo in acutely admitted adult ICU patients with delirium.

We assessed long-term outcomes in acutely admitted adult patients with delirium treated in intensive care unit (ICU) with haloperidol versus placebo. We conducted pre-planned analyses of 1-year outcomes in the Agents Intervening against Delirium in the ICU (AID-ICU) trial, including mortality and health-related quality of life (HRQoL) assessed by Euroqol (EQ) 5-dimension 5-level questionnaire (EQ-5D-5L) index values and EQ visual analogue scale (EQ VAS) (deceased patients were assigned the numeric value zero). Outcomes were analysed using logistic and linear regressions with bootstrapping and G-computation, all with adjustment for the stratification variables (site and delirium motor subtype) and multiple imputations for missing HRQoL values. At 1-year follow-up, we obtained vital status for 96.2% and HRQoL data for 83.3% of the 1000 randomised patients. One-year mortality was 224/501 (44.7%) in the haloperidol group versus 251/486 (51.6%) in the placebo group, with an adjusted absolute risk difference of - 6.4%-points (95% confidence interval [CI] - 12.8%-points to - 0.2%-points; P = 0.045). These results were largely consistent across the secondary analyses. For HRQoL, the adjusted mean differences were 0.04 (95% CI - 0.03 to 0.11; P = 0.091) for EQ-5D-5L-5L index values, and 3.3 (95% CI - 9.3 to 17.5; P = 0.142) for EQ VAS. In acutely admitted adult ICU patients with delirium, haloperidol treatment reduced mortality at 1-year follow-up, but did not statistically significantly improve HRQoL.

Surgical prioritization based on decision model outcomes is not sensitive to differences between the health-related quality of life values estimates of physicians and citizens

PurposeDecision models can be used to support allocation of scarce surgical resources. These models incorporate health-related quality of life (HRQoL) values that can be determined using physician panels. The predominant opinion is that one should use values obtained from citizens. We investigated whether physicians give different HRQoL values to citizens and evaluate whether such differences impact decision model outcomes.MethodsA two-round Delphi study was conducted. Citizens estimated HRQoL of pre- and post-operative health states for ten surgeries using a visual analogue scale. These values were compared using Bland–Altman analysis with HRQoL values previously obtained from physicians. Impact on decision model outcomes was evaluated by calculating the correlation between the rankings of surgeries established using the physicians’ and the citizens’ values.ResultsA total of 71 citizens estimated HRQoL. Citizens’ values on the VAS scale were − 0.07 points (95% CI − 0.12 to − 0.01) lower than the physicians’ values. The correlation between the rankings of surgeries based on citizens’ and physicians’ values was 0.96 (p < 0.001).ConclusionPhysicians put higher values on health states than citizens. However, these differences only result in switches between adjacent entries in the ranking. It would seem that HRQoL values obtained from physicians are adequate to inform decision models during crises.

Health-related quality of life and willingness to pay measurement among patients on warfarin in Thailand

BackgroundComplex dosage regimens and the high incidence of adverse events associated with warfarin therapy can affect the health-related quality of life (HRQoL) and willingness to pay (WTP) among patients on warfarin. No such previous study has been conducted to assess the HRQoL and WTP among Thai patients on warfarin; therefore, this study aimed to measure these parameters and identify some sociodemographic factors associated with those aspects among patients on warfarin in Thailand.MethodsThis cross-sectional survey study involving 260 patients on warfarin between June 2022 and June 2023 used a quantitative method for data collection. Face-to-face interviews with well-trained interviewers were conducted and patients were required to complete the questionnaires of both World Health Organization Quality of Life-BREF (WHOQOL-BREF) and EQ-5D-5L to assess and measure their HRQoL levels. WTP was assessed using a bidding game technique. Descriptive statistics with mean and standard deviations were used to report HRQoL scores and WTP, whereas a generalized linear model was employed to identify factors associated with both HRQoL and WTP.ResultsThe mean EQ-5D index and mean EQ-VAS score were 0.89 ± 0.15 and 76.92 ± 15.95, respectively, whereas the mean WHOQOL-BREF domain scores were 59.18 ± 14.13, 68.56 ± 15.47, 59.13 ± 19.64, and 65.23 ± 14.04 for the physical, psychological, social, and environmental domains, respectively. Elderly participants (age > 60 years) and those with comorbidities had lower HRQoL scores than their counterparts. The mean WTP was 22.25 ± 32.19 USD for one patient’s visit. The presence of comorbidities was the only factor significantly associated with WTP values.ConclusionsThai patients on warfarin have lower mean EQ-5D indexes and EQ-VAS scores than members of the general Thai population. Patients on warfarin with comorbidities have diminished HRQoL and WTP values. Therefore, all healthcare professionals should pay more attention to this group of patients on warfarin to achieve better outcomes.

Health-Related Quality of Life and Mental Health of Children with Embryonal Abdominal Tumors.

(1) Background: Embryonal abdominal tumors are one of the most common entities of solid childhood cancer. The present study investigates the Health-Related Quality of Life (HRQoL) and the mental health of children to obtain a comprehensive picture of their health status and uncover a possible gap in healthcare. (2) Methods: The sample consisted of 54 children who were treated for embryonal abdominal tumors and a control group of 46 children who received uncomplicated outpatient surgery. The HRQoL and the mental health were assessed by the parent proxy reports of the questionnaires Pediatric Quality of Life Inventory (PedsQL) and Strengths and Difficulties Questionnaire (SDQ). (3) Results: Children with embryonal abdominal tumors showed significantly lower HRQoL and mental health values compared to the norm data. The index group showed lower values in the social subscales of HRQoL and mental health compared to the control group. (4) Conclusions: Embryonal abdominal tumors affect the well-being of children. There is still a gap in healthcare due to children's HRQoL and mental health, especially regarding social development. It is essential to further advance the psychological care of children and improve their chances to develop social relationships.

Health-related quality of life trajectories in critical illness: Protocol for a Monte Carlo simulation study.

Health-related quality of life (HRQoL) is a patient-centred outcome increasingly used as a secondary outcome in critical care research. It may cover several important dimensions of clinical status in intensive care unit (ICU) patients that arguably elude other more easily quantified outcomes such as mortality. Poor associations with harder outcomes, conflicting data on HRQoL in critically ill compared to the background population, and paradoxical effects on HRQoL and mortality complicate the current operationalisation in critical care trials. This protocol outlines a simulation study that will gauge if the areas under the HRQoL trajectories could be a viable alternative. We will gauge the behaviour of the proposed HRQoL operationalisation through Monte Carlo simulations, under clinical scenarios that reflect a broad critical care population eligible for inclusion in a large pragmatic trial. We will simulate 15,360 clinical scenarios based on a full factorial design with the following seven simulation parameters: number of patients per arm, relative mortality reduction in the interventional arm, acceleration of HRQoL improvement in the interventional arm, the relative improvement in final HRQoL in the interventional arm, dampening effect of mortality on HRQoL values at discharge from the ICU, proportion of so-called mortality benefiters in the interventional arm and mortality trajectory shape. For each clinical scenario, we will simulate 100,000 two-arm trials with 1:1 randomisation. HRQoL will be sampled fortnightly after ICU discharge. Outcomes will include HRQoL in survivors and all patients at the end of follow-up; mean areas under the HRQoL trajectories in both arms; and mean difference between areas under the HRQoL trajectories and single-sampled HRQoLs at the end of follow-up. In the outlined simulation study, we aim to assess whether the area under the HRQoL trajectory curve could be a candidate for reconciling the seemingly paradoxical effects on improved mortality and reduced HRQoL while remaining sensitive to early or accelerated improvement in patient outcomes. The resultant insights will inform subsequent methodological work on prudent collection and statistical analysis of such data from real critically ill patients.

Health related Quality of Life over time in German sarcoma patients. An analysis of associated factors - results of the PROSa study.

Sarcomas are rare cancers and very heterogeneous in their location, histological subtype, and treatment. Health-Related Quality of Life (HRQoL) of sarcoma patients has rarely been investigated in longitudinal studies. Here, we assessed adult sarcoma patients and survivors between September 2017 and February 2020, and followed-up for one year in 39 study centers in Germany. Follow-up time points were 6 (t1) and 12 months (t2) after inclusion. We used a standardized, validated questionnaire (the European Organisation for Research and Treatment of Cancer Quality of Life Core Instrument (EORTC QLQ-C30) and explored predictors of HRQoL in two populations (all patients (Analysis 1), patients in ongoing complete remission (Analysis 2)) using generalized linear mixed models. In total we included up to 1111 patients at baseline (915 at t1, and 847 at t2), thereof 387 participants were in complete remission at baseline (334 at t1, and 200 at t2). When analyzing all patients, HRQoL differed with regard to tumor locations: patients with sarcoma in lower extremities reported lower HRQoL values than patients with sarcomas in the upper extremities. Treatment which included radiotherapy and/or systemic therapy was associated with lower HRQoL. For patients in complete remission, smoking was associated with worse HRQoL-outcomes. In both analyses, bone sarcomas were associated with the worst HRQoL values. Being female, in the age group 55-<65 years, having lower socioeconomic status, and comorbidities were all associated with a lower HRQoL, in both analyses. HRQoL increased partially over time since treatment and with sporting activities. HRQoL improved with time since treatment, although not in all domains, and was associated with lifestyle and socioeconomic factors. Bone sarcomas were the most affected subgroup. Methods to preserve and improve HRQoL should be developed for sarcoma patients.

Using Age-Specific Values for Pediatric HRQoL in Cost-Effectiveness Analysis: Is There a Problem to Be Solved? If So, How?

Value sets for the EQ-5D-Y-3L published to date appear to have distinctive characteristics compared with value sets for corresponding adult instruments: in many cases, the value for the worst health state is higher and there are fewer values < 0. The aim of this paper is to consider how and why values for child and adult health differ; and what the implications of that are for the use of EQ-5D-Y-3L values in economic evaluations to inform healthcare resource allocation decisions. We posit four potential explanations for the differences in values: (a) The wording of severity labels may mean the worst problems on the EQ-5D-Y-3L are descriptively less severe than those on the EQ-5D-5L; (b) Adults may genuinely consider that children are less badly affected than adults by descriptively similar health issues. That is, for any given health problem, adult respondents in valuation studies consider children's overall health-related quality of life (HRQoL) on average to be higher than that for adults; (c) Values are being sought by eliciting adults' stated preferences for HRQoL in another person, rather than in themselves (regardless of whether the 'other person' concerned is a child); and (d) The need to elicit preferences for child HRQoL that are anchored at dead = 0 invokes special considerations regarding children's survival. Existing evidence does not rule out the possibility that (c) and (d) exert an upward bias in values. We consider the implications of that for the interpretation and use of values for pediatric HRQoL. Alternative methods for valuing children's HRQoL in a manner that is not 'age specific' are possible and may help to avoid issues of non-comparability. Use of these methods would place the onus on health technology assessment bodies to reflect any special considerations regarding child quality-adjusted life-year gains.

Validation of baseline health-related quality of life as prognostic indicator for survival outcome in men with intermediate risk prostate cancer undergoing radical prostatectomy.

340 Background: Accurate prediction of cancer outcomes remains a key component of prostate cancer (PC) treatment decision-making. Intermediate-risk PC patients represent a heterogeneous group with highly variable prognoses and it is challenging to provide uniform treatment recommendations for those patients. Previous studies have shown baseline health-related quality of life (HRQOL) to be a robust prognostic indicator for survival outcomes of different cancer entities in the metastatic setting, with scarce evidence on PC. We therefore sought to validate the predictive value of baseline HRQOL for survival outcomes in men with intermediate risk PC undergoing radical prostatectomy (RP). Methods: We identified 4780 patients with intermediate risk PC according to NCCN risk stratification and prospectively assessed baseline HRQOL prior RP. Patients were stratified by global health status (GHS) domain of the QLQ-C30 questionnaire. Oncologic endpoints were metastasis-free survival (MFS) and overall survival (OS). Multivariable Cox regression models were applied to assess the predictive value of baseline GHS on survival outcomes. Harrell's discrimination C-index was applied to calculate the predictive accuracy of the model. Decision curve analysis (DCA) was performed to validate the clinical net benefit associated with adding GHS to our multivariable model ( P &lt; .05). Results: Median follow up was 51mo. In multivariable analysis, GHS was confirmed as an independent predictor for increased MFS (HR .98, 95% CI .97-.99; P = .028) and OS (HR .97, 95% CI .95-.99; P = .008), indicating a relative risk reduction of 1.7% for MFS and 2.8% for OS per 1-point increase of baseline GHS. Adding GHS to our model improved predictive accuracy of prognosis for MFS by 6% (c-index .75 vs .72) and for OS by 8% (c-index .78 vs .74). Validation with DCA revealed a net benefit over all thresholds in prediction of MFS and OS when adding GHS to our model. Conclusions: Our findings highlight that baseline HRQOL is a valuable and robust prognostic factor for patients with intermediate-risk PC prior RP. Baseline HRQOL data improve prognostic accuracy of MFS and OS and can therefore support treatment decision-making. [Table: see text]

Rapid decline in kidney function is associated with rapid deterioration of health-related quality of life in chronic kidney disease

This study aimed to evaluate changes in health-related quality of life (HRQOL) in patients with chronic kidney disease (CKD) according to decline in kidney function. HRQOL was assessed using the Short Form-36 questionnaire composed of a physical component summary (PCS) and mental component summary (MCS). Rapid decline in kidney function was defined as a decline in the estimated glomerular filtration rate (eGFR) of > 3 mL/min/1.73 m2/year. Rapid deterioration of HRQOL was defined a change in the HRQOL value greater than the median. Among 970 patients, 360 (37.1%) were in the rapid kidney function decline group. In 720 patients who were 1:1 propensity score-matched, the baseline eGFR was not significantly different between the non-rapid and rapid kidney function decline groups. Compared with the baseline PCS score, the 5-year PCS score decreased in the non-rapid and rapid kidney function decline groups. The 5-year MCS score significantly decreased in the rapid kidney function decline group alone. Rapid decline in kidney function was significantly associated with rapid deterioration of the PCS (odds ratio [OR]: 1.48; 95% confidence interval [CI]: 1.07–2.05; P = 0.018) and MCS (OR: 1.89; 95% CI 1.36–2.62; P < 0.001) scores. Rapid decline in kidney function was associated with rapid deterioration of HRQOL in patients with CKD.

Health-related quality of life after curative treatment for muscle-invasive bladder cancer.

Muscle-invasive bladder cancer (MIBC) is an aggressive disease for which the gold-standard treatment is radical cystectomy (RC) in combination with cisplatin-based neoadjuvant chemotherapy. Bladder-sparing strategies such as trimodal therapy (TMT) have also emerged to improve health-related quality of life (HRQoL) of patients. However, an improved understanding of the effect of all these treatment modalities on HRQoL is essential to provide personalized patient care. Different combinations of generic, cancer-specific and bladder cancer-specific questionnaires can be used as instruments for HRQoL evaluation in patients with MIBC before and after curative treatments, which can largely affect multiple domains of HRQoL including general health as well as physical, functional, social and emotional well-being. However, diagnosis of MIBC per se is also likely to affect HRQoL, and the perspective of cure after RC or TMT could induce a return to baseline HRQoL values for most of these domains. A considerable amount of data on HRQoL after RC is available, but conflicting results have been reported regarding the effect of urinary diversion (ileal conduit or orthotopic neobladder) and surgical approach (open or robotic surgery) on patient quality of life. Data on HRQoL after TMT are scarce, and additional comparative studies including patients receiving RC (especially using ileal orthotopic neobladder) are needed.

Characterizing the individual course of health-related quality of life after subarachnoid haemorrhage: Latent growth mixture modelling

BackgroundSurvivors of aneurysmal subarachnoid haemorrhage (SAH) show heterogeneous profiles of health-related quality of life (HrQoL). The aim of this study was to characterize individual differences in the course of HrQoL following SAH using latent growth mixture modelling (LGMM). MethodsA longitudinal study with 113 incident cases of aneurysmal SAH was performed in order to evaluate clinical outcome (Hunt and Hess scale, Barthel-Index, Beck Depression Inventory) and HrQoL data (EQ-5D) at baseline, 6 and 12 months. The heterogeneity in HrQoL courses after SAH was analysed using LGMM. ResultsFour subgroups (classes) of different patterns of HrQoL course after SAH were identified. Two of these classes (1 and 3) comprised patients with considerably reduced initial HrQoL, which was associated with more severe symptoms of SAH. Class 1 showing the worst EQ5D-index values during the entire study period. Class 3 experiencing a considerable improvement in HrQoL values. In comparison to classes 1 and 3, class 2 and 4 were characterized by less severe SAH and better functional outcome. An important difference in the disease course between classes 2 and 4 was a temporary increase in depression scores at the 6-month time point in class 4, which was associated with a considerable reduction in HrQoL.The specific clinical parameters characterizing differences between classes, such as severity of SAH, functional outcome, cognitive impairment and post-stroke depression, were identified and the influence of their potential improvement on HrQoL was estimated. ConclusionBy means of LGMM we could classify the course of HrQoL after SAH in four different patterns, which are relevant for the clinical decisions. Clinical parameters, which can be modified in order to improve the course of HrQoL were identified and could help to develop individual therapeutic strategies for the rehabilitation after SAH.

Health-related quality of life variation by socioeconomic status: Evidence from an Iranian population-based study.

Health-Related Quality of Life (HRQoL) values based on the accurate and reliable European Quality of Life Five Dimension (EQ-5D) questionnaire gives health-state utilities as a helpful data set for studying socio-demographic and socio-economic inequalities in health status in the general population. We aimed to do a population-based study to see how HRQoL varies by socio-demographics and socioeconomic status (SES). The study was a cross-sectional population-based study in Shiraz, Iran's southwest. Data was gathered utilizing a personal digital assistant (PDA). A trained interviewer administered the EQ-5D questionnaire to a representative sample of 1036 inhabitants. Principal component analysis (PCA) was used to create SES indices. Because of the skewed distribution, quantile regression was utilized to model the quartiles of HRQoL values. STATA 12.0 was used to perform all statistical analyses. P <0.05 was considered statistically significant. In 1036 study respondents, women had a mean HRQoL of 0.67 ± 0.28, whereas men had a mean HRQoL of 0.78 ± 0.25. Gender and age remained significant in all quartiles of HRQoL value. Participants with insurance showed 0.14 and 0.08 higher HRQoL values in the first and second HRQoL quartiles than those without coverage, respectively. Education [95% CI: 0.034, 0.111)], economy [95% CI: 0.013, 0.077], and assets [95% CI: 0.003, 0.069] all had an impact on HRQoL value in the lowest quintile. In all quartiles of HRQoL value, women had lower reported HRQoL than men. Insurance programs aimed at more disadvantaged groups with poorer HRQoL may help to minimize inequity. Education, economics, and assets all had an impact on the lower quartiles of HRQoL value, emphasizing the importance of general policies in determining public health status.

Health related quality of life, service utilization and costs for patients with Huntington’s disease in Norway

BackgroundHuntington’s disease (HD) is a progressive genetic neurodegenerative disease accompanied by mental and neurocognitive disabilities, which requires long-term and comprehensive treatment and care. Information on the health and economic burden of HD is scarce, but essential for conducting health economic analyses, in light of the prospect of new therapies for HD. In this study, we aim to identify values for Health-Related Quality of Life (HRQoL), describe service utilization and costs, and their associations with clinical and socio-demographic variables across all phases of HD.MethodsA cross-sectional study including 86 patients across all phases of HD. Values of HRQoL were calculated based on EQ-5D-3L index scores. Additionally, health care and societal costs were estimated based on service utilization collected using the Client Service Receipt Inventory (CSRI) and data from the patients’ interviews. Total societal costs included costs of primary and secondary health care services, informal care and productivity loss of the patients. Multiple regression analyses were used to investigate associations between socio-demographic and clinical variables on HRQoL and costs.ResultsHRQoL values declined, while total costs increased across disease severity. Total six-month healthcare costs and total societal costs were € 18,538 and € 66,789 respectively. Healthcare and societal costs doubled from early to middle phase, and tripled from middle to advanced disease phase. Main six-month cost components for the three disease phases were informal care costs (€ 30,605) accounting for approximately half the total societal costs, and costs due to production loss (€ 18,907) being slightly higher than the total healthcare costs. Disease severity and gender were found to have the strongest effect on both values of HRQoL and costs.ConclusionsReported values of HRQoL and costs including costs for production loss may be used in modelling the cost-effectiveness of treatment for HD. Our results highlight the crucial role the informal caregivers play in the care provided to HD patients in all disease phases. Future research should focus on the estimation of productivity loss among informal caregivers.