Health-related Quality Of Life Utility Research Articles

Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial

Background: Pompe disease is a rare lysosomal storage disorder, leading to accumulation of glycogen characterized by muscle weakness, fatigue, pain, and, in the longer term, a requirement for ventilatory and ambulatory support, and early mortality if untreated. Clinical evidence suggests that enzyme replacement therapy improves health outcomes for adults with late-onset Pompe disease (LOPD). PROPEL was a Phase 3, double-blind, randomized controlled trial, which evaluated cipaglucosidase alfa plus miglustat, vs alglucosidase alfa plus placebo in 123 adult patients with LOPD (clinicaltrials.gov: NCT03729362). Objectives: To analyze EQ-5D health-related quality of life (HRQoL) utility data from PROPEL. Methods: Multilevel modeling techniques (mixed regression methods) were used to analyze PROPEL EQ-5D-3L estimates and predict utility values for 7 health states previously identified in an economic evaluation for LOPD. In PROPEL, EQ-5D-5L values were assessed at screening and at weeks 12, 26, 38, and 52. EQ-5D-5L utility values were mapped to EQ-5D-3L values using the van Hout algorithm as recommended by the EuroQoL and the National Institute of Health and Care Excellence position statement at time of analysis. UK population tariffs were applied for all EQ-5D utility valuations. Utility values were predicted according to 6-minute walk distance (6MWD) and percent predicted sitting forced vital capacity. Results: The mixed model predicted that EQ-5D-3L utility values for patients who could walk >75 m with LOPD ranged between 0.55 and 0.67 according to patient 6MWD and respiratory function. In this analysis, patients with a 6MWD ≤75 m, consistent with a health state requiring wheelchair support in the economic analysis, had a predicted utility value of 0.49. There were few patients in PROPEL who could walk ≤75 m at any time point in the study, hence, these utility estimates should be interpreted with caution. EQ-5D-3L utility estimates from PROPEL were consistent with previously reported EQ-5D-3L values in LOPD. Conclusions: Overall, the results from our analysis indicate that important HRQoL losses are associated with reductions in mobility and respiratory function for patients with Pompe disease. The study provides important evidence of HRQoL utility values for patients with advanced LOPD, a population for whom published data are limited.

Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial.

Background: Pompe disease is a rare lysosomal storage disorder, leading to accumulation of glycogen characterized by muscle weakness, fatigue, pain, and, in the longer term, a requirement for ventilatory and ambulatory support, and early mortality if untreated. Clinical evidence suggests that enzyme replacement therapy improves health outcomes for adults with late-onset Pompe disease (LOPD). PROPEL was a Phase 3, double-blind, randomized controlled trial, which evaluated cipaglucosidase alfa plus miglustat, vs alglucosidase alfa plus placebo in 123 adult patients with LOPD (clinicaltrials.gov: NCT03729362). Objectives: To analyze EQ-5D health-related quality of life (HRQoL) utility data from PROPEL. Methods: Multilevel modeling techniques (mixed regression methods) were used to analyze PROPEL EQ-5D-3L estimates and predict utility values for 7 health states previously identified in an economic evaluation for LOPD. In PROPEL, EQ-5D-5L values were assessed at screening and at weeks 12, 26, 38, and 52. EQ-5D-5L utility values were mapped to EQ-5D-3L values using the van Hout algorithm as recommended by the EuroQoL and the National Institute of Health and Care Excellence position statement at time of analysis. UK population tariffs were applied for all EQ-5D utility valuations. Utility values were predicted according to 6-minute walk distance (6MWD) and percent predicted sitting forced vital capacity. Results: The mixed model predicted that EQ-5D-3L utility values for patients who could walk >75 m with LOPD ranged between 0.55 and 0.67 according to patient 6MWD and respiratory function. In this analysis, patients with a 6MWD ≤75 m, consistent with a health state requiring wheelchair support in the economic analysis, had a predicted utility value of 0.49. There were few patients in PROPEL who could walk ≤75 m at any time point in the study, hence, these utility estimates should be interpreted with caution. EQ-5D-3L utility estimates from PROPEL were consistent with previously reported EQ-5D-3L values in LOPD. Conclusions: Overall, the results from our analysis indicate that important HRQoL losses are associated with reductions in mobility and respiratory function for patients with Pompe disease. The study provides important evidence of HRQoL utility values for patients with advanced LOPD, a population for whom published data are limited.

Health-Related Quality of Life and Economic Analysis of Olanzapine Versus Aprepitant in Preventing Chemotherapy-Induced Nausea and Vomiting in Patients Receiving Highly Emetogenic Chemotherapy in Malaysia

ObjectivesOlanzapine has been shown to be effective in preventing chemotherapy-induced nausea and vomiting (CINV) after highly emetogenic chemotherapy (HEC); however, there is limited work on the impact of CINV on health-related quality of life (HRQoL) and the comparative cost-effectiveness of CINV prophylaxis in the Malaysian context. Therefore, this study was conducted to determine the HRQoL using EQ-5D-5L and the cost-effectiveness of olanzapine compared with aprepitant for CINV prophylaxis in Malaysia using data from a local study. MethodsFifty-nine chemo-naive patients receiving either olanzapine or aprepitant were randomly recruited and completed the EQ-5D-5L before and day 5 after HEC. HRQoL utility scores were analyzed according to the Malaysian valuation set. The economic evaluation was conducted from a healthcare payer perspective with a 5-day time horizon. Quality-adjusted life days (QALD) and the rate of successfully treated patients were used to measure health effects. The incremental cost-effectiveness ratio is assessed as the mean difference between groups’ costs per mean difference in health effects. A one-way sensitivity analysis was performed to assess variations that might affect outcomes. ResultsAprepitant and olanzapine arms’ patients had comparable baseline mean HRQoL utility scores of 0.920 (SD = 0.097) and 0.930 (SD = 0.117), respectively; however, on day 5, a significant difference (P value = .006) was observed with mean score of 0.778 (SD = 0.168) for aprepitant and 0.889 (SD = 0.133) for olanzapine. The cost per successfully treated patient in the aprepitant arm was 60 times greater than in the olanzapine arm (Malaysian Ringgit [MYR] 927 vs MYR 14.83). Likewise, the cost per QALD gain in the aprepitant arm was 36 times higher than in the olanzapine arm (MYR 57.05 vs MYR 1.57). Incremental cost-effectiveness ratio of MYR −937.00 (USD −200.98) per successfully treated patient and MYR −391.84 (USD −85.43) per QALD gained for olanzapine compared with the aprepitant-based regimen. ConclusionsAn olanzapine-based regimen is a cost-effective therapeutic substitution in patients receiving HEC in Malaysia.

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.

The short-term impact of Schistosoma mansoni infection on health-related quality of life: implications for current elimination policies.

The WHO aims to eliminate schistosomiasis as a public health problem by 2030. However, standard morbidity measures poorly correlate to infection intensities, hindering disease monitoring and evaluation. This is exacerbated by insufficient evidence on Schistosoma's impact on health-related quality of life (HRQoL). We conducted community-based cross-sectional surveys and parasitological examinations in moderate-to-high Schistosoma mansoni endemic communities in Uganda. We calculated parasitic infections and used EQ-5D instruments to estimate and compare HRQoL utilities in these populations. We further employed Tobit/linear regression models to predict HRQoL determinants. Two-thirds of the 560 participants were diagnosed with parasitic infection(s), 49% having S. mansoni. No significant negative association was observed between HRQoL and S. mansoni infection status/intensity. However, severity of pain urinating (β = -0.106; s.e. = 0.043) and body swelling (β = -0.326; s.e. = 0.005), increasing age (β = -0.016; s.e. = 0.033), reduced socio-economic status (β = 0.128; s.e. = 0.032), and being unemployed predicted lower HRQoL. Symptom severity and socio-economic status were better predictors of short-term HRQoL than current S. mansoni infection status/intensity. This is key to disentangling the link between infection(s) and short-term health outcomes, and highlights the complexity of correlating current infection(s) with long-term morbidity. Further evidence is needed on long-term schistosomiasis-associated HRQoL, health and economic outcomes to inform the case for upfront investments in schistosomiasis interventions.

Association Between Quality of Life Defined by EuroQol Group 5 Dimension and Composite Inferior Outcome Among Inpatients with Cirrhosis.

The utility of the EuroQol Group 5 Dimension (EQ-5D) measuring health-related quality of life (HRQoL) has been verified; however, knowledge gaps remain concerning predictive performance in cirrhosis. We aimed to identify the optimal threshold for risk stratification and the pronounced domain in the EQ-5D linked to inferior outcomes. The X-tile project was used to obtain a threshold, considering the composite outcome of 1-year all-cause mortality or readmission. A restricted cubic spline (RCS) was performed to test the non-linear relationship between the EQ-5D utility value and the primary outcome. Six multivariate Cox regression models incorporating EQ-5D utility value and each of the five domains were constructed. Totally, 420 patients with cirrhosis were recruited. The median utility value of the study population was 0.77 and 59.8% reported impairment in minimal one EQ-5D domain. RCS indicated a linear relationship between the utility value and composite inferior outcome. X-tile pinpointed a utility value of 0.59 stratifying populations into high- and low-risk groups based on the outcome. Inpatients with cirrhosis and deteriorated HRQoL (utility value ≤0.59) were at higher risk of death or readmission (adjusted HR: 2.18, P < 0.001). Furthermore, mobility and usual activities were the most pronounced domains associated with composite inferior outcome. A utility value ≤0.59 can identify cirrhotic inpatients exhibiting compromised HRQoL and mortality/readmission risk. It is tempting to reverse the decreased HRQoL by applying longitudinal measurements and keeping surveillance on utility value, while interventions appear to mainly focus on improving mobility and usual activities.

Health-related quality of life in people with different diabetes-related foot ulcer health states: A cross-sectional study of healed, non-infected, infected, hospitalised and amputated ulcer states

AimsDiabetes-related foot ulcers (DFU) are a leading cause of infection, hospitalisation and amputation. However, to our knowledge no studies have compared the health-related quality of life (HRQoL) of people with DFU that were infected, hospitalised or amputated. This study aimed to investigate and compare the HRQoL of different groups of people with healed, non-infected, infected, hospitalised, or amputated DFU. MethodsThis was a multi-centre cross-sectional study measuring the HRQoL of patients, attending one of 18 Diabetic Foot Services across Queensland, Australia, with one of five DFU health states: healed, non-infected, infected, hospitalised, amputated. HRQoL was measured using the EQ-5D-5L to estimate age-sex adjusted utility values. ResultsOf 376 included patients (mean age 63 (12) years, 75% male), age-sex adjusted HRQoL utility estimates (95% CIs) were: healed DFU 0.57 (0.51–0.64), non-infected DFU 0.55 (0.49–0.62), infected DFU 0.45 (0.36–0.55), hospitalised DFU 0.53 (0.42–0.64), and amputated DFU 0.55 (0.46–0.63). ConclusionPeople in any DFU health state have considerably reduced HRQoL, with greatest reductions in those with infected DFU. These findings provide valuable HRQoL estimates and comparisons for several different important DFU health states, adding to our understanding of the impact of DFU on HRQoL and facilitating future economic evaluations.

Temporal associations of diabetes-related complications with health-related quality of life decrements in Chinese patients with type 2 diabetes: A prospective study among 19 322 adults-Joint Asia Diabetes Evaluation (JADE) register (2007-2018).

Patients with type 2 diabetes (T2D) are at high risk of developing multiple complications, affecting their health-related quality of life (HRQoL). Existing studies only considered impact of complication on HRQoL in the year of occurrence but not its residual impacts in subsequent years. We investigated temporal impacts of diabetes-related complications on HRQoL in a 12-year prospective cohort of ambulatory Chinese patients with T2D enrolled in the clinic-based Joint Asia Diabetes Evaluation (JADE) Register. HRQoL utility measures were derived from EuroQol five-dimensional three-level questionnaire (EQ-5D-3L) questionnaires completed by 19 322 patients with T2D in Hong Kong (2007-2018). Temporal EQ-5D utility decrements associated with subtypes of cardiovascular-renal events were estimated using generalized linear regression model after stepwise selection of covariates with p < .01 as cutoff. In this cohort (mean ± SD age:61.2 ± 11.5 years, 55.3% men, median [interquartile range] duration of diabetes:10.1 [3.0-15.0] years, glycated hemoglobin [HbA1C] 7.5 ± 1.5%), EQ-5D utility was 0.860 ± 0.163. The largest HRQoL decrements were observed in year of occurrence of hemorrhagic stroke (-0.230), followed by ischemic stroke (-0.165), peripheral vascular disease (-0.117), lower extremity amputation (-0.093), chronic kidney disease (CKD) G5 without renal replacement therapy (RRT) (-0.079), congestive heart failure (CHF) (-0.061), and CKD G3-G4 without RRT (-0.042). Residual impacts on HRQoL persisted for 2 years after occurrence of CHF or ischemic stroke and 1 year after hemorrhagic stroke or CKD G3-G4 without RRT. This is the first comprehensive report on temporal associations of HRQoL decrements with subtypes of diabetes-related complications in ambulatory Asian patients with T2D. These data will improve the accuracy of cost-effectiveness analysis of diabetes interventions at an individual level in an Asian setting.

Health state utility and health-related quality of life measures in patients with advanced ovarian cancer

PurposeMeasuring health-related quality of life (HRQoL) in ovarian cancer patients is critical to understand the impact of disease and treatment. Preference-based HRQoL measures, called health state utilities, are used specifically in health economic evaluations. Real-world patient-reported data on HRQoL and health state utilities over the long-term course of ovarian cancer are limited. This study aims to determine HRQoL and health state utilities in different health states of ovarian cancer. MethodsThis cross-sectional, multicenter study included patients with stage III-IV ovarian cancer in six health states: at diagnosis, during chemotherapy, after cytoreductive surgery (CRS), after chemotherapy, in remission, and at first recurrence. HRQoL was measured using the European Organization for Research and Treatment of Cancer Core Quality of Life Questionnaire C30, and the ovarian cancer-specific module OV28. Health state utilities were assessed using the EuroQol five-dimension five-level (EQ-5D-5L) questionnaire. Descriptive analyses were performed for each health state. ResultsTwo hundred thirty-two patients participated, resulting in 319 questionnaires. Median age was 66 years. The lowest HRQoL was observed during chemotherapy and shortly after CRS. Physical and role functioning were most affected and the highest symptom prevalence was observed in the fatigue, nausea, pain, dyspnea, gastrointestinal, neuropathy, attitude, and sexuality domains. Patients in remission had the best HRQoL. Mean utility values ranged from 0.709 (±0.253) at diagnosis to 0.804 (±0.185) after chemotherapy. ConclusionsThis study provides clinicians with a valuable resource to aid in patient counseling and clinical decision-making. The utilities, in particular, are crucial for researchers conducting economic analyses to inform policy decisions.

Association of body mass index from childhood to mid-adulthood with health-related quality of life in mid-adulthood

PurposeMost studies regarding the association of obesity with health-related quality of life (HRQoL) have assessed obesity at only one or two time points. We aimed to examine the associations of life course body mass index (BMI) from childhood with health-related quality of life (HRQoL) in mid-adulthood.MethodsData were from a cohort study of Australian children (n = 2254, mean baseline age 12.0 (2.0) years in 1985, 46.8% male). Weight and height were measured at baseline and measured or self-reported on average 20, 25, and 30 years later. Age and sex-standardised BMI-z score was calculated at each time point. Physical and mental HRQoL and health state utilities (HSUs) were measured by SF-12 and SF-6D at the last adult follow-up. Linear regression was used to examine the associations adjusting for age, sex, and childhood health status.ResultsHigher BMI-z score in childhood (βadjusted − 1.39, 95% CI − 1.73 to − 1.05) and increasing BMI-z score from childhood to young adulthood (βadjusted − 1.82, 95% CI − 2.17 to − 1.46) and from young to mid-adulthood (βadjusted − 1.77, 95% CI − 2.28 to − 1.26) were associated with lower physical HRQoL in mid-adulthood. Similar results were found for mid-adulthood HSUs (βadjusted ranged − 0.006 to − 0.014, all P < 0.05). Only increasing BMI-z score from young to mid-adulthood significantly related to poorer mental HRQoL (βadjusted − 0.74, 95% CI − 1.29 to − 0.19) in mid-adulthood.ConclusionHigh BMI from childhood to mid-adulthood had only modest associations with HRQoL and HSUs, with effects on physical HRQoL most apparent.

Catalogues of EQ-5D-3L Health-Related Quality of Life Scores for 199 Chronic Conditions and Health Risks for Use in the UK and the USA.

Health-related quality of life (HRQoL) measures are essential in economic evaluation, but sometimes primary sources are unavailable, and information from secondary sources is required. Existing HRQoL UK/US catalogues are based on earlier diagnosis classification systems, amongst other issues. A recently published Danish catalogue merged EQ-5D-3L data from national health surveys with national registers containing patient information on ICD-10 diagnoses, healthcare activities and socio-demographics. To provide (1) UK/US EQ-5D-3L-based HRQoL utility population catalogues for 199 chronic conditions on the basis of ICD-10 codes and health risks and (2) regression models controlling for age, sex, comorbidities and health risks to enable predictions in other populations. UK and US EQ-5D-3L value sets were applied to the EQ-5D-3L responses of the Danish dataset and modelled using adjusted limited dependent variable mixture models (ALDVMMs). Unadjusted mean utilities, percentiles and adjusted disutilities based on two ALDVMMs with different control variables were provided for both countries. Diseases from groups M, G, and F consistently had the smallest utilities and the largest negative disutilities: fibromyalgia (M797), sclerosis (G35), rheumatism (M790), dorsalgia (M54), cerebral palsy (G80-G83), post-traumatic stress disorder (F431), dementia (F00-2), and depression (F32, etc.). Risk factors, including stress, loneliness, and BMI30+, were also associated with lower HRQoL. This study provides comprehensive catalogues of UK/US EQ-5D-3L HRQoL utilities. Results are relevant in cost-effectiveness analysis, for NICE submissions, and for comparing and identifying facets of disease burden.

Abstract WMP11: Health-related Quality Of Life After Thrombolysis In Acute Stroke: Results From The Act Trial

Background and aims: Recent evidence from thrombolysis trials indicate the non-inferiority of the Tenecteplase (TNK) to the Alteplase (TPA) with respect to functional outcomes in acute stroke patients. This study examines the predictors of patient-reported health-related quality of life (HRQOL) in acute stroke patients who received thrombolysis therapy. Methods: Data were used from all acute stroke patients included in AcT, a pragmatic, registry-linked randomized trial comparing TNK with TPA. HRQOL at 90-day post-randomization was assessed using the EuroQol-5D-5L (EQ-5D) visual analogue scale (VAS). Based on the Canadian norms, health state utilities were estimated from the EQ-5D items using the EQ-5D time trade-off approach. Tobit regression model and linear mixed-effects regression were used to evaluate the adjusted effect of type of treatment on health utility scores and VAS score, respectively. Results: Of the 1262 patients included in this analysis, 647(51.3%) were administered the TNK, 584(46.3%) were female, and 380(30.1%) were greater than or equal to 80 years old. There was no significant adjusted effect of the type of thrombolysis on health utility scores (adjusted beta coefficient [95%CI] = 0.029 [-0.001, 0.062]), but patients who received TNK reported higher adjusted VAS scores than those that received TPA [adjusted beta coefficient [95%CI] = 2.1 [-0.29, 4.49]). Older age (p &lt; 0.01), female sex (p = 0.01), higher NIHSS score (p &lt; 0.01) were associated with lower health utilities and VAS scores. Conclusion: There is no differential effect of the type of thrombolysis on patient-reported global HRQOL and health state utilities of acute stroke patients. Disparities in HRQOL were mainly explained by sex, age, and disease severity.

Health-related quality of life of Chinese AIDS patients: a multi-region study.

To assess the health-related quality of life (HRQOL) of Chinese AIDS patients in three regions (Shanghai, Zhejiang, and Henan) and to examine the relationship between region and the HRQOL. A cross-sectional study was conducted from 2017 to 2018. Multistage stratified sampling was employed to recruit 1,273 AIDS patients from local Centers for Disease Control and Prevention. Data were collected by means of face-to-faceinterview using a structured questionnaire including the SF-12 and various rating scales. The multivariate linear and logistic regression models were performed to analyze the relationship of region and a variety of factors with the HRQOL and health utility. The three most affected dimensions were mental health, general health, and vitality as 18.2%, 18.0%, and 16.4% of the patients reported problems respectively. The mean (SD) scores of physical component summary (PCS) and mental component summary (MCS), were 48.19 (8.02) and 46.74 (10.71) respectively. The mean (SD) health utility score was 0.75 (0.13) assessed by the SF-6D derived from the SF-12. Region, age, employment status, individual income, government assistance and stigma significantly affected the patients' HRQOL (P < 0.01). BMI, opportunistic infection, treatment compliance and time of treatment were also found to be significant factors of the HRQOL. This study comprehensively estimated the HRQOL and health utility for the AIDS population in China, their HRQOL was mainly deteriorated in psychological dimensions, and geographical area may be closely related. Hence, close attention needs to be paid on the regional differences in HRQOL and the psychological problems of the patients.

Health-related quality of life in various health conditions: two consecutive surveys of older Japanese adults.

Measuring health-related quality of life (HRQOL) in various health conditions in different countries is important given the regional differences. This study employed large-scale nationwide data targeting older adults in Japan to estimate the HRQOL in the key health conditions that are the major causes of disability. Our data were derived from two survey waves (2016 and 2019 surveys) of cross-sectional data from the Japan Gerontological Evaluation Study, an ongoing nationwide study targeting functionally independent older adults in Japan. A total of 28,345 individuals from 27 of the 47 Japanese provinces were analyzed. The EuroQoL 5-dimension 5-level instrument (EQ-5D-5L) was employed to assess the HRQOL utility scores. The targeted minimum loss-based estimator with sampling weighting methods was applied to estimate the utility score in eight major health conditions, including sensory organ disease, musculoskeletal disease, oral disorders, and depressive disorders. The estimated HRQOL utility score for those with the poorest health conditions in self-rated health, hearing loss, vision loss, number of remaining teeth (e.g., no teeth with no denture use), oral dysfunction, depressive symptoms, chronic low back pain, and chronic knee pain was 0.576 (95% confidence interval (CI) 0.555-0.598), 0.768 (95% CI 0.737-0.800), 0.680 (95% CI 0.662-0.699), 0.809 (95% CI 0.796-0.821), 0.776 (95% CI 0.764-0.788), 0.723 (95% CI 0.710-0.737), 0.715 (95% CI 0.690-0.739), and 0.742 (95% CI 0.722-0.763), respectively. We successfully provided a catalog for the HRQOL utility score in key health conditions that are the leading causes of disability among older adults.

Health-related quality of life, one-year costs and economic evaluation in extracorporeal membrane oxygenation in critically ill adults

Purpose: This study reports on survival and health related quality of life (HRQOL) after extracorporeal membrane oxygenation (ECMO) treatment and the associated costs in the first year.Materials and Methods: Prospective observational cohort study patients receiving ECMO in the intensive care unit during August 2017 and July 2019. We analyzed all healthcare costs in the first year after index admission. Follow-up included a HRQOL analysis using the EQ-5D-5L at 6 and 12 months.Results: The study enrolled 428 patients with an ECMO run during their critical care admission. The one-year mortality was 50%. Follow up was available for 124 patients at 12 months. Survivors reported a favorable mean HRQOL (utility) of 0.71 (scale 0–1) at 12 months of 0.77. The overall health status (VAS, scale 0–100) was reported as 73.6 at 12 months. Mean total costs during the first year were $204,513 ± 211,590 with hospital costs as the major factor contributing to the total costs. Follow up costs were $53,752 ± 65,051 and costs of absenteeism were $7317 ± 17,036.Conclusions: At one year after hospital admission requiring ECMO the health-related quality of life is favorable with substantial costs but considering the survival might be acceptable. However, our results are limited by loss of follow up. So it may be possible that only the best-recovered patients returned their questionnaires. This potential bias might lead to higher costs and worse HRQOL in a real-life scenario.

The Impact of Hearing Loss and Its Treatment on Health-Related Quality of Life Utility: a Systematic Review with Meta-analysis.

Hearing loss significantly impacts health-related quality of life (QoL), yet the effects of current treatments on QoL utility remain uncertain. Our objective was to describe the impact of untreated and treated hearing loss on QoL utility to inform hearing healthcare policy. We searched databases for articles published through 02/01/2021. Two independent reviewers screened for articles that reported elicitation of general QoL utility values for untreated and treated hearing loss health states. We extracted data and quality indicators from 62 studies that met the inclusion criteria. Included studies predominately used observational pre/post designs (61%), evaluated unilateral cochlear implantation (65%), administered the Health Utilities Index 3 (HUI3; 71%), and were conducted in Europe and North America (84%). In general, treatment of hearing loss improved post-treatment QoL utility when measured by most methods except the Euro-QoL 5 dimension (EQ-5D). In meta-analysis, hearing aids for adult mild-to-moderate hearing loss compared to no treatment significantly improved HUI3-estimated QoL utility (3 studies; mean change=0.11; 95% confidence interval (CI): 0.07 to 0.14) but did not impact EQ-5D-estimated QoL (3 studies; mean change=0.0; 95% CI: -0.03 to 0.04). Cochlear implants improved adult QoL utility 1-year post-implantation when measured by the HUI3 (7 studies; mean change=0.17; 95% CI: 0.11 to 0.23); however, pediatric VAS-estimated QoL utility was non-significant (4 studies; mean change=0.12; 95% CI: -0.02 to 0.25). The quality of included studies was limited by failure to report missingness of data and low survey response rates. Our study was limited by heterogeneous study populations and designs. Treatment of hearing loss significantly improves QoL utility, and the HUI3 and VAS were most sensitive to improvements in hearing. Improved access to hearing healthcare should be prioritized. SYSTEMATIC REVIEWREGISTRATION: PROSPERO: CRD42021253314.

Association Between Cognition, Health Related Quality of Life, and Costs in a Population at Risk for Cognitive Decline.

Background:The association between health-related quality of life (HRQoL) and care costs in people at risk for cognitive decline is not well understood. Studying this association could reveal the potential benefits of increasing HRQoL and reducing care costs by improving cognition.Objective:In this exploratory data analysis we investigated the association between cognition, HRQoL utilities and costs in a well-functioning population at risk for cognitive decline.Methods:An exploratory data analysis was conducted using longitudinal 2-year data from the FINGER study (n = 1,120). A change score analysis was applied using HRQoL utilities and total medical care costs as outcome. HRQoL utilities were derived from the Short Form Health Survey-36 (SF-36). Total care costs comprised visits to a general practitioner, medical specialist, nurse, and days at hospital. Analyses were adjusted for activities of daily living (ADL) and depressive symptoms.Results:Although univariable analysis showed an association between cognition and HRQoL utilities, multivariable analysis showed no association between cognition, HRQoL utilities and total care costs. A one-unit increase in ADL limitations was associated with a -0.006 (p < 0.001) decrease in HRQoL utilities and a one-unit increase in depressive symptoms was associated with a -0.004 (p < 0.001) decrease in HRQoL utilities.Conclusion:The level of cognition in people at-risk for cognitive decline does not seem to be associated with HRQoL utilities. Future research should examine the level at which cognitive decline starts to affect HRQoL and care costs. Ideally, this would be done by means of cross-validation in populations with various stages of cognitive functioning and decline.

Health-related quality of life, fatigue and health utilities in lupus nephritis: A systematic literature review.

BackgroundSystemic lupus erythematosus (SLE) is a chronic, autoimmune disease characterized by abnormal B-cell activation and the presence of autoantibodies, which can result in organ damage. Lupus nephritis (LN) is the most common severe organ manifestation of SLE and may result in impaired kidney function. However, there is limited research on the health-related quality of life (HRQoL) burden amongst patients with LN. The objective of this systematic literature review was to assess the HRQoL, fatigue and health utilities associated with LN.MethodsA structured literature search (GSK Study 212980) of the MEDLINE and Embase databases was conducted in July 2019 and updated September 2021. Relevant international congress abstracts from 2016 to 2021 were searched, and gray literature searches and keyword-based searches in PubMed, Google, and Google Scholar were also conducted. Results were screened according to predefined criteria and data on the outcomes of interest were extracted. A quantitative analysis was conducted to supplement the narrative review, to provide 36-item Short Form survey (SF-36) estimates, and to determine variation by prognostic factors.ResultsOf 1155 articles identified, 26 studies for a total of 3440 patients were included. Patients with LN showed poorer HRQoL and more fatigue than healthy controls/the general population, although these were similar between patients with SLE with and without LN. HRQoL was worse in patients with LN Class III/IV or with active disease. Fatigue was generally reported as the most burdensome symptom and was associated with lower HRQoL and increased treatment dissatisfaction. During induction treatment, HRQoL and fatigue were improved with mycophenolate mofetil versus cyclophosphamide. HRQoL improved over time with treatment amongst patients with active LN. Very limited data were identified assigning utilities to health states for cost-effectiveness analysis. Nine studies were considered for quantitative analysis of baseline SF-36 scores. The analysis suggested that LN has a significant impact across all SF-36 domains, with the lowest scores in the general health perceptions and role-physical domains and physical component summary.ConclusionsThere is a large HRQoL burden in patients with LN, in particular regarding symptoms of fatigue. Future research should focus on investigating fatigue severity and health utilities in LN.

Systematic Literature Review of Clinical and Economic Evidence for Spinal Muscular Atrophy.

IntroductionThe recent advent of disease-modifying therapies (DMTs) has dramatically changed the treatment landscape of spinal muscular atrophy (SMA), and the multifaceted impact of this advancement has not been assessed thoroughly in the growing body of literature. We sought to summarize the literature on the natural history of SMA and the impact of SMA DMTs, including health-related quality of life (HRQOL) and utilities, clinical efficacy and safety, and economic impact.MethodsSystematic literature reviews were conducted following PRISMA guidelines with no inclusive dates. Relevant studies were identified by searching full-text databases on November 12–13, 2020, including MEDLINE, EMBASE, the Cochrane Central Register of Controlled Trials, and EconLit, conference proceedings, health technology assessment databases, and clinical trial registries. All searches used a combination of MeSH and key terms. Studies were screened according to criteria based upon population, intervention, outcomes, and study design structure.ResultsFindings from 17, 23, 32, and 42 studies were included for the evaluation of natural history of SMA, HRQOL and utilities, clinical efficacy and safety, and economic impact of DMTs, respectively. Currently available data indicate that untreated SMA is associated with considerable humanistic and economic burden, with estimates of costs varying by treatment. While a variety of interventions have been evaluated in SMA clinical trials, quantitative synthesis of safety and efficacy findings was not feasible because of inconsistencies in reported outcomes. Data assessing impacts of DMTs on HRQOL were also lacking.ConclusionsOverall, this systematic literature review highlights a clear need for up-to-date and methodologically rigorous clinical, HRQOL, and economic data to support unbiased assessments of the relative clinical and economic effectiveness of SMA treatments. More research is required to extend our understanding of the burden of SMA on HRQOL utility assessments and the impact of new DMTs on HRQOL and utilities for patients with SMA.Supplementary InformationThe online version contains supplementary material available at 10.1007/s12325-022-02089-2.

Health-Related Quality of Life and Health Utilities of Mild, Moderate, and Severe Asthma: Evidence from the Medical Expenditure Panel Survey.

BackgroundLittle information is known about the health-related quality of life (HRQOL) and the patient’s preference values by the severity of asthma. We evaluated the HRQOL and health utility impairment associated with asthma severity using the SF-12 and SF-6D.MethodsWe conducted a cross-sectional analysis of 2010–2016 Medical Expenditure Panel Survey database of asthma patients aged ≥18 years and categorized them into mild, moderate, and severe asthma. Study outcomes included the SF-12 physical component summary (PCS) and mental component summary (MCS) for measuring HRQOL and SF-6D for health utility. Survey regression models were used to estimate HRQOL and utilities for mild, moderate, and severe asthma.ResultsOf 10,222 patients with asthma, 75.4%, 23.9%, and 0.8% had mild, moderate and severe asthma. We observed that the greater the severity, the lower the SF-6D scores: 0.731 in mild, 0.723 in moderate, and 0.659 in severe asthma (P < 0.001). Patients with severe asthma had a significantly lower PCS compared to those with mild asthma (−5.3; P < 0.001) but there was no significant difference in MCS (−1.9; P = 0.309) controlling for socioeconomic and clinical variables. Asthma severity, women, older age, and having a lower level education and public insurance were significantly associated with lower PCS (P < 0.01).ConclusionAsthma patients had worse physical HRQOL than mental health, especially patients with severe asthma. These data suggest that the management of physical health of female, older aged, and low education patients with asthma should be focused on improving HRQOL.