Mean Health Utility Scores Research Articles

Quality of life of the Canadian population using the VR-12: population norms for health utility values, summary component scores and domain scores

ObjectivesTo estimate Canadian population norms (health utility values, summary component scores and domain scores) for the VR-12.MethodsEnglish and French speaking Canadians aged 18 and older completed an online survey that included sociodemographic questions and standardized health status instruments, including the VR-12. Responses to the VR-12 were summarized as: (i) a health utility value; (ii) mental and physical component summary scores (MCS and PCS, respectively), and (iii) eight domain scores. Norms were calculated for the full sample and by gender, age group, and province/territory (univariate), and for several multivariate stratifications (e.g., age group and gender). Results were summarized using descriptive statistics, including number of respondents, mean and standard deviation (SD), median and percentiles (25th and 75th), and minimum and maximum.ResultsA total of 6761 people who clicked on the survey link completed the survey (83.4% completion rate), of whom 6741 (99.7%) were included in the analysis. The mean health utility score was 0.698 (SD = 0.216). Mean health utility scores tended to be higher in older age groups, ranging from 0.661 (SD = 0.214) in those aged 18–29 to 0.728 (SD = 0.310) in those aged 80+. Average MCS scores were higher in older age groups, while PCS scores were lower. Females consistently reported lower mean health utility values, summary component scores and domain scores compared with males.ConclusionsThis is the first study to present Canadian norms for the VR-12. Health utility norms can serve as a valuable input for Canadian economic models, while summary component and domain norms can help interpret routinely-collected data.

Health-related quality of life and economic burden of childhood pneumonia in China: a multiregion study

ObjectiveTo systematically investigate the health-related quality of life (HRQOL) and economic burden of children with pneumonia in different regions of China.Study designThe study recruited a series of children under 5...

Lorlatinib Effectiveness and Quality-of-Life in Patients with ALK-Positive NSCLC Who Had Failed Second-Generation ALK Inhibitors: Canadian Real-World Experience.

Lorlatinib is the only targeted therapy approved in Canada to treat patients with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) whose tumor has progressed despite treatment with second-generation ALK tyrosine kinase inhibitor (TKI), a patient population with high unmet need and lack of publicly reimbursed targeted treatments in Canada. We prospectively examined the real-world effectiveness and impact of lorlatinib on quality-of-life in 59 lorlatinib-treated patients, characterized as: median age of 62.0 years; 47.5% were female; 32.2% had central nervous system metastases; 50.8% had 2+ prior ALK TKI lines; and alectinib was the most common ALK TKI (72.9%) administered before lorlatinib, including 44.1% who received first-line alectinib. With a median follow-up of 15.3 months (IQR: 6.2-19.2), median time-to-treatment discontinuation of lorlatinib was 15.3 months (95% CI: 7.9-not reached), with 54.2% (95% CI: 40.8-65.9%) of patients without treatment discontinuation at 12 months. At baseline, the mean health utility score (HUS) was 0.744 (SD: 0.200). At 3 months, patients receiving lorlatinib demonstrated a 0.069 (95% CI: 0.020-0.118; p = 0.007) average HUS increase over baseline; HUS was maintained at 6 and 12 months. Thus, patients with ALK-positive NSCLC post second-generation ALK TKI remained on lorlatinib for a meaningful duration of time while their quality-of-life was preserved.

Health utility of children with acute lymphoblastic leukemia in China.

Acute lymphoblastic leukemia is the most common cancer in children. As the 5-year survival rate has been improved to over 80%, more emphasis is now placed on reducing therapy toxicities and enhancing health-related quality of life (HRQoL) of patients during treatment. Our objective was to measure health utility of pediatric acute lymphoblastic leukemia (pALL) patients in China, examine utility weights of different treatment phases and influencing factors of health utility, as well as identify which aspects of HRQoL were most impaired. A cross-sectional study was conducted in Shanghai Children's Medical Center (SCMC) Affiliated to Shanghai Jiao Tong University School of Medicine in China from April to November 2021. Primary caregivers of 247 patients completed the assessment by CHU9D-CHN and health utility scores were computed for all the patients and stratified by treatment phases. Various multivariable models were constructed and the best was chosen to identify independent factors associated with utility scores. Factors affecting the most impaired dimensions were also examined. The overall mean (SD) health utility score was 0.79 (±0.17) and significantly increased from induction (0.73 [±0.19], P < 0.001) to consolidation (0.74 [±0.18]), and to maintenance (0.82 [±0.16]). After adjusting for potentially influencing factors, utility scores in induction (Beta = -0.086, P = 0.005) and consolidation (Beta = -0.074, P = 0.043) were constantly lower than those during maintenance. In item-level analysis, lower age and induction phase were found to be significantly associated with high severity reported on the "school work/homework" dimension. Additionally, only the induction phase (vs. maintenance, OR = 2.24, P = 0.016) was independently associated with the high severity level reported on the "able to join in activities" dimension. This is the first study that measured health utility of children with pALL in China. Mean health utility scores increased from induction to maintenance. These provided important utility estimates that help inform future health economic models. The phrasing of "School work/homework" in CHU9D-CHN could be further improved. More efforts are needed to design and implement specific interventions targeting at the dimension "able to join in activities" for enhancing HRQoL of children with pALL in China.

Quality of life assessment of cabozantinib in patients with advanced hepatocellular carcinoma in the CELESTIAL trial

BackgroundThe CELESTIAL trial (NCT01908426) demonstrated overall survival benefit for cabozantinib versus placebo in patients with advanced hepatocellular carcinoma (aHCC) who had received prior sorafenib treatment. This analysis of CELESTIAL compared the impact of cabozantinib versus placebo on health-related quality of life (HRQoL). Materials and methodsHealth status was assessed using the EuroQol five-dimension five-level (EQ-5D-5L) questionnaire over the 800-day follow-up period. EQ-5D-5L health states were mapped to health utility scores using reference values for the UK population. Quality-adjusted life years (QALYs) were calculated for each treatment group as the area under the curve for the plot of health utility score over time. The between-treatment group difference in restricted mean QALYs was calculated by generalized linear models and adjusted for baseline differences. A difference of 0.08 in health utility score (or in QALY) was deemed a minimally important difference and to be clinically significant. ResultsAt week 5, the difference in mean health utility score between cabozantinib and placebo was −0.097 (95% confidence interval [95% CI]: −0.126, −0.067; p ≤ 0.001). Between-group differences in health utility scores diminished over time and were generally non-significant. The cabozantinib group accrued more QALYs than the placebo group over follow-up. Differences in mean QALYs (cabozantinib minus placebo) were statistically and clinically significant, ranging from +0.092 (95% CI: 0.016, 0.169) to +0.185 (95% CI: 0.126, 0.243) in favour of cabozantinib, depending on the reference value set used. ConclusionsThese HRQoL findings support a positive benefit–risk profile for cabozantinib in previously treated patients with aHCC.

Valuing health states of people with type2 diabetes: Analyses of the nationwide representative linked databases.

Aims/IntroductionTo estimate preference‐based measures of health‐related quality of life associated with sociodemographic and clinical characteristics in type 2 diabetes patients.Materials and MethodsIndividuals with EuroQol‐5 dimensions‐3 levels data were identified from Taiwan’s National Health Interview Survey in 2009 and 2013. Status of diabetes, comorbidities, complications and treatments were ascertained through data linkage to Taiwan’s National Health Insurance Research Database. Multivariable ordinary least squares, Tobit and median regression analyses were used to estimate the coefficients that represented independent impacts of patients’ characteristics on health‐related quality of life.ResultsThe mean health utility score for 2,104 participants was 0.838. Being female, aging, divorced/widowed, never worked or underweight, or having a lower monthly household income, injectable glucose‐lowering therapy, comorbid connective tissue disease or depression were associated with lower health utilities. Having an amputation led to the largest reduction by 0.288 in health utilities, followed by debilitating stroke (0.266), heart failure (0.237), other coronary heart disease (0.185), kidney dialysis/transplant (0.148), coronary revascularizations (0.093), transient ischemic attack/stroke (0.078), diabetic neuropathy (0.062), polyneuropathy (0.055) and other neuropathy (0.043).ConclusionsMajor vascular complications, connective tissue disease and depression are associated with considerably worse health‐related quality of life. These health utility estimates can facilitate health economic evaluations to determine cost‐effective strategies for diabetes management.

Temporal changes in quality of life amongst European atrial fibrillation patients: relationship to all-cause mortality. A report from the ESC-EHRA EORP-AF General Long-Term Registry

Abstract Background Atrial fibrillation (AF) significantly impacts on patients' quality of life (QoL). An impaired QoL has been associated with worse outcomes even in AF patients, but contemporary data in a large-scale pan-European population are limited. Purpose We aimed to assess temporal changes in AF patients' QoL across 2 years follow-up observation, and the relationship of QoL changes with all-cause death. Methods We analyzed patients enrolled in the ESC-EHRA EORP-AF General Long-Term Registry. The EQ-5D-5L questionnaire was used to assess QoL. A Health Utility Score (HUS), indicating the overall health state (1 equals perfect health), was derived. Differences throughout the follow-up (Baseline, 1-Y FU, 2-Y FU) observation were assessed. The study outcome was all-cause mortality. Results Out of a total of 11906 patients, 8097 (73.0%) were available for this analysis. Mean (SD) age was 69.1 (11.5) years; 60.8% males; median CHA2DS2-VASc and HASBLED scores were 3 (IQR 2–4) and 1 (1–2), respectively. The mean (SD) HUS at baseline was 0.815 (0.200) and 0.834 (0.196), 0.829 (0.195) at 1-year follow-up and 2-year follow-up, respectively (p&amp;lt;0.0001 for changes over time). Patients with a higher CHA2DS2-VASc score (CHA2DS2-VASc 6–9) reported a significant reduction in the quality of life, compared to the other CHA2DS2-VASc strata, with a mean (SD) HUS decreasing from 0.754 (0.214) at baseline to 0.727 (0.238) at 2-year follow-up (F=6.538, p&amp;lt;0.0001) (Figure). Multivariate analysis demonstrated that age [−0.001 (95% CI [−0.002, −0.121]) and coronary artery disease (CAD) [−0.016 (95% CI [−0.029, −0.004] were independently inversely associated with increasing QoL. Positive changes in HUS over time were inversely associated with an increase in the risk of all-cause death, even after adjusting for chronic kidney disease, liver disease, chronic obstructive pulmonary disease, oral anticoagulants and type of AF (OR:0.24, 95% CI: 0.13–0.45 for increasing HUS difference, as a continuous variable). Conclusions In a contemporary European-wide cohort of AF patients, significant temporal changes in QoL were found. Patients at higher stroke risk according to CHA2DS2-VASc score showed a significant reduction in the QoL. Age and CAD were independently associated with changes in QoL. A greater reduction in HUS (i.e. worsening QoL) over time was associated with a higher risk of all-cause death. Temporal changes in HUS Funding Acknowledgement Type of funding source: Private company. Main funding source(s): Since the start of EORP, several companies have supported the programme with unrestricted grants

The impact of symptoms and comorbidity on health utility scores and health-related quality of life in small cell lung cancer using real world data.

Small cell lung cancer (SCLC) is a highly fatal disease associated with significant morbidity, with a need for real-world symptom and health utility score (HUS) data. HUS can be measured using an EQ-5D-5L questionnaire, however most captured data is available in non-SCLC (NSCLC) only. As new treatment regimens become available in SCLC it becomes important to understand factors which influence health-related quality of life and health utility. A prospective observational cohort study (2012-2017) of ambulatory histologically confirmed SCLC evaluated patient-reported EQ-5D-5L-derived HUS, toxicity and symptoms. A set of NSCLC patients was used to compare differential factors affecting HUS. Clinical and demographic factors were evaluated for differential interactions between lung cancer types. Comorbidity scores were documented for each patient. In 75 SCLC and 150 NSCLC patients, those with SCLC had lower mean HUS ((SCLC vs NSCLC: mean 0.69 vs 0.79); (p < 0.001)) when clinically stable and with progressive disease: ((SCLC mean HUS = 0.60 vs NSCLC mean HUS = 0.77), (p = 0.04)). SCLC patients also had higher comorbidity scores ((1.11 vs 0.73); (p < 0.015)). In multivariable analyses, increased symptom severity and comorbidity scores decreased HUS in both SCLC and NSCLC (p < 0.001); however, only comorbidity scores differentially affected HUS (p < 0.0001), with a greater reduction of HUS adjusted per unit of comorbidity in SCLC. Patients with advanced SCLC had significantly lower HUS than NSCLC. Both patient cohorts are impacted by symptoms and comorbidity, however, comorbidity had a greater negative effect in SCLC patients.

An evaluation of EQ-5D-3L health utility scores using five country-specific tariffs in a rural population aged 45\u201369\u2009years in Hua county, Henan province, China

BackgroundThis study aims to compare the performance of the recently developed Chinese (city) tariff of the EQ-5D-3L against the UK, US, Japanese and Korean tariffs in a general rural population in China.MethodsFrom November 2015 to September 2016, 12,085 permanent residents aged 45–69 from 257 villages randomly selected from Hua County, Henan Province, China, were interviewed using EQ-5D-3L, and a one-on-one questionnaire investigation was used to collect data on factors associated with HRQOL. The health utility scores were calculated using the UK, US, Japanese, Korean and Chinese (city) tariffs. The agreement, known-groups validity and sensitivity of these five tariffs were evaluated. Transition scores for pairs of observed EQ-5D-3L health states were calculated and compared.ResultsThe Korean tariff yielded the highest mean health utility score (0.963), followed by the Chinese (city) (0.948), US (0.943), UK (0.930) and Japanese (0.921) tariffs, but the differences in the scores of any two tariffs did not exceed the MCID. The Chinese (city) tariff showed higher ICC values (ICCs> 0.89, 95% CI:0.755–0.964) and narrower limits of agreement (0.099–0.167) than the Korean tariff [(ICCs> 0.71, 95% CI:0.451–0.955); (0.146–0.253)]. The Chinese (city) tariff had a higher relative efficiency and effect size statistics in 10 out of 11 variables as compared to the UK, US and Japanese tariffs. The Chinese (city) tariff (0.215) was associated with moderate mean absolute transition scores compared with the UK (0.342), US (0.230), Japanese (0.149) and Korean (0.189) tariffs for 1485 observed pairs of the EQ-5D-3L health states.ConclusionsHealth utility scores derived from the five tariffs differed. The Chinese (city) tariff was the most suitable of these tariffs and was without obvious weakness. We recommend adopting the Chinese (city) tariff when applying EQ-5D-3L to assess quality of life among the elderly in China’s agricultural region with socio-economic status similar to Hua County. Results of this study had provided a crucial basis for health surveys, health promotion projects, health intervention trials, and health economic evaluation taking HRQOL as a target in rural areas of China.

Predictors of and longitudinal assessment of health utility scores in patients with small cell lung cancer using real-world data.

e19306 Background: Recent advances in small cell lung cancer (SCLC) treatments necessitate a better understanding of health utility scores (HUS) of patients treated under standard regimens to facilitate robust pharmaco-economic assessments. HUS collected in clinical trials may be inherently skewed due to restrictive eligibility criteria, highlighting the need for real-world data. Methods: In this cohort observational study, HUS were evaluated in SCLC patients through EQ-5D surveys. We also evaluated patient reported (pr) ECOG performance status (PS)), treatment toxicities (modified patient reported (mpr) CTCAE) and symptoms (Edmonton Symptom Assessment System, ESAS). Clinical data were abstracted from electronic medical records. The impact of these variables on HUS was explored using regression. Results: Of 282 clinical encounters (12% newly diagnosed; 37% stable on treatment; 22% progressing; 29% stable off therapy/other) in 111 SCLC patients (58% male; 64% extensive stage), 29% had pr-ECOG PS ≥ 2 at the first encounter. Mean HUS in treatment naïve patients with limited disease was 0.848 (SEM = 0.028); for extensive stage, mean HUS = 0.715 (SEM = 0.046). Extensive stage (β = -0.12; p = 0.03), bone metastases (β = -0.10; p = 0.04), females (β = -0.07; p = 0.006), and pr-ECOG PS ≥ 2 (β = -0.21; p &lt; 0.001) were each associated with decreased HUS in multivariable analyses. When excluding pr-ECOG PS from the model (highly correlated with HUS), progressive disease (β = -0.07; p = 0.02) was also associated with decreased HUS. Longitudinally, in patients with disease stability, HUS were unchanged in limited disease, but slowly decreased for extensive stage over time. HUS were inversely associated with increasing severity of most measured toxicities (mpr-CTCAE rho values ranged from -0.34 to -0.47) and symptoms (rho values -0.27 to -0.54). Conclusions: HUS in SCLC is impacted by the presence of extensive stage and bone metastases, in addition to cancer symptoms and treatment toxicities. The values reported from this real-world sample provides a basis in which to compare with new SCLC therapies in health technology assessments. [Table: see text]

Comparing health utility scores, treatments, and outcomes in lung cancer patients with rare EGFR mutations with those with exon 19 deletion (del) and L858R mutations.

102 Background: Precision oncology divides patients into smaller cohorts each with unique characteristics, treatment and outcomes. Cost-effectiveness assessments that rely on quality-adjusted life years will require mutation-specific health utility scores (HUS). We assessed the impact of having exon 19 del, L858R and rare EGFR mutations on outcomes and HUS. Methods: From a retrospective database of 719 patients with EGFR mutations, specific baseline EGFR mutations, clinicodemographic and treatment characteristics, and outcomes (overall survival, OS; progression-free survival, PFS) were analyzed using Cox models (adjusted hazard ratios, HR). In a subset of 289 patients with metastatic disease, serial HUS data collected through EQ-5D-5L at clinic visits were compared by mutation using t-tests. Results: Of 380 (53%) patients with exon 19 del, 288 (40%) with L858R, and 51 (7%) with rare mutations (mostly G719A/C, Exon 18 or 20 insertion, L861Q, compound mutations): 68% were female; median age was 74 years; 51% were Asian; and 74% were never smokers. In 334 Stage I-III pts, recurrence-free survival was not associated with specific mutations. In contrast, among 365 stage IV pts on TKIs, when compared to a reference of patients with exon 19 del, outcomes were worse in patients with L858R mutations (PFS: 1.35, 95% CI 1.1-1.7; OS: HR 1.39, 95% CI 1.0-1.9) and for rare mutations (PFS: 1.16 95% CI 0.7-1.9; OS: 1.45 95% CI 0.7-2.9). From an analysis of 1064 clinic encounters, different TKIs were used in similar proportions by mutation. In stable disease, mean HUS [SEM] were 0.80 [0.008] (exon 19), 0.81 [0.009] (L858R), and 0.82 [0.02] (rare mutation). Progressive disease led to significant drops in mean HUS for exon 19 (0.76 [0.01]; p = 0.01 compared to stable disease) and L858R, mean HUS = 0.74 [0.02] p &lt; 0.001, but less so for rare mutations, mean HUS = 0.79 [0.05] p = 0.65. Conclusions: Patients in this EGFR mutated cohort had similar exposures of different TKI therapy regardless of specific EGFR mutation. L858R and rare mutations had inferior survival outcomes but similar HUS as patients with exon 19 del mutations.

The influence of patient-reported adverse events on Health Utility Score (HUS) and Health-Related Quality of Life (HRQoL) in small cell lung cancer (SCLC) and non-small cell lung cancer (NSCLC) patients.

185 Background: The Patient Reported Outcome version of Common-Terminology-Criteria-for-Adverse-Events (PRO-CTCAE) and Edmonton Symptom Assessment System (ESAS) are validated tools that measure toxicities and symptoms in cancer patients. We compared the extent in which the presence and severity of toxicities and symptoms affected HUS in SCLC and NSCLC patients. Methods: Adult SCLC and NSCLC patients were recruited from the Princess Margaret Cancer Centre and surveyed cross-sectionally for clinico-demographic variables, EQ5D-5L, PRO-CTCAE and ESAS. HUS were estimated using EQ5D-5L. PRO-CTCAE toxicities include diarrhea, constipation, decreased appetite, nausea, vomiting, fatigue, neuropathy and rash. ESAS symptoms include pain, tiredness, drowsiness, appetite loss, nausea, shortness of breath, depression, anxiety and lack of well-being. These were combined to show frequency and average severity of toxicities/symptoms per patient. Univariable and multivariable linear regression analyses identified toxicity/symptom influencing HUS. Results: Of 75 SCLC and 150 NSCLC, 52% were male with median age of 65 years. The mean HUS was 0.76 (SCLC = 0.69; NSCLC = 0.79; p = 0.001). Compared to NSCLC, SCLC patients had a significantly higher number of toxicities (3.14 versus 1.33 using PRO-CTCAE, p &lt; 0.0001); symptoms (6.75 vs 5.45 using ESAS, p = 0.0003) and severity of toxicities/symptoms (PRO-CTCAE 0-4: 0.95 versus 0.35, p &lt; 0.0001; ESAS 0-10: 3.37 versus 2.04. p &lt; 0.0001). There were significant correlations between the average severity of toxicities/symptoms and HUS (p &lt; 0.0001) adjusted for age, histology, smoking pack years and performance status. For each increase in the average severity of toxicities, there was a corresponding mean drop of 0.03 in the HUS; for every increase in the average severity of symptoms, the drop was 0.04. These relationships were similar for both SCLC and NSCLC patients. Conclusions: Patient reported toxicities and symptoms have a significant impact on HUS in both SCLC and NSCLC patients. Early and aggressive management of such adverse events may be necessary to improve patients’ HRQoL.

Quality of Life and Health Utility Scores Among Canadians Living With Traumatic Spinal Cord Injury - A National Cross-Sectional Study.

National, multicenter, cross-sectional study. The goal of this study was to provide overall quality of life (QOL) and health utility (HU) values for patients with traumatic spinal cord injury (SCI) stratified by injury level and neurological status. Traumatic SCI is a leading cause of disability. Varying injury level and severity generate a spectrum of neurological dysfunction and reduction in long-term QOL. The Canadian SCI Community Survey was sent to Canadians living in the community after SCI. The impact of demographics, complications, and SCI classification on QOL was assessed using Analysis of variance, multiple linear regressions and ordinal logistic regression analyses. There were 1109 respondents with traumatic SCI. american spinal injury association impairment scale (AIS) grade was reported to be cervical motor complete in 20%, cervical motor incomplete in 28%, thoracolumbar motor complete in 32%, thoracolumbar motor incomplete in 16%, and normal (any level) in 1%. Injury level or AIS grade had no impact on either HU or QOL. The physical component of health-related quality of life (HRQOL) was significantly affected by the neurological level, but not the social or mental components. With a mean health utility score of 0.64 ± 0.12, SCI patients living in the community reported having HRQOL similar to patients after total knee arthroplasty or lumbar spinal stenosis decompression. QOL or HU measured by generic HRQOL outcome tools should not be used as outcomes to assess the effectiveness of interventions targeting neurological function in traumatic SCI. A disease-specific instrument that captures the nuances specific to spinal cord injury patients is required. 1.

A trial-based EuroQol EQ-5D health utility analysis in patients with classical Hodgkin lymphoma.

e19011 Background: Pembrolizumab has shown a high response in patients classic Hodgkin lymphoma (cHL) patients who have experienced disease progression after brentuximab vedotin in KEYNOTE (KN)-087 and the results have been presented. This study aimed to evaluate the health-related quality of life (HRQoL) of the trial patients in KN087. Methods: KN-087 is an ongoing single-arm multi-center, non-randomized Phase II trial evaluating pembrolizumab 200mg Q3W IV in patients with relapsed or refractory cHL. In KN-087, HRQoL data were collected at baseline and every drug administration over the 18 months of follow-up. HRQoL was assessed using both the EQ-5D and EORTC QLQ-C30 instruments. The generic health statuses assessed from both instrument were converted to population-based utility values using published algorithms. More specifically, US-based scoring was applied to US patients, UK-based scoring for UK patients and EU-based scoring for all other patients. HRQoL was reported by status of respond and disease progression. Response was defined based upon IWG criteria. Furthermore, stratified analyses were conducted to examine the health disabilities of the patients who experienced grade 3+ adverse events (AEs), and by ECOG performance and the number of prior therapies. Results: Among 210 trial patients, HRQoL data were collected for 205 patients at baseline and the mean health utility score was 0.759 (95% CI 0.730-0.788). Mean health utility score among responders and non-responders was 0.826 (95% CI 0.811-0.842) and 0.760 (95% CI 0.718-0.801), respectively. The difference is considered clinically significant. Mean utility decreased from 0.820 (95% CI 0.807-0.833) for time spent prior to progression to 0.806 (95% CI 0.780-0.832) post disease progression. Progression-free patients who experienced grade 3+ AEs (N=17) had a mean health utility of 0.736 (95% CI 0.662-0.811), compared with 0.825 (95% CI 0.811-0.838) among those did not. Conclusions: The results showed a substantial HRQoL impact of R/R cHL. Treatment response was associated with significant clinically meaningful improvement in HRQoL. The utility estimates from the study are important for economic evaluations of treatments in R/R cHL patients. Clinical trial information: NCT02453594.

Neurocognitive function testing and health related quality of life in stage IV lung cancer patients with and without brain metastases.

2073 Background: Survival in stage IV lung cancer (S4LC) patients (pts) continues to improve, highlighting the importance of assessing health related quality of life (HRQoL). Lung cancer, involvement with brain metastases (BM), and systemic or brain-specific treatment can all impact neurocognitive function (NCF) and HRQoL. We evaluated the relationship between NCF and HRQoL in S4LC pts by BM status. Methods: S4LC pts with BM (BM+) were frequency distributionally-matched to pts without BM (NBM). NCF was measured using the Hopkins Verbal Learning Test – Revised (HVLT-R), the Controlled Oral Word Association Test (COWAT) and Trail Making Tests (TMT-A/B); scores were correlated with health utility score (HUS) data from EQ5D-3L surveys (Pearson Coefficient, R). Results: BM+ (n = 54) and matched NBM (n = 40) pts had similar demographics. The overall median age was 61years; 59% were female; of 89% that were adenocarcinomas, half had EGFR/ALK alterations; mean time since diagnosis was 2.6 years; mean time since BM were diagnosed in BM+ pts was 0.5 years. Mean HUS (mHUS) were similar between groups: 0.77 for BM+ vs. 0.78 for NBM; p = 0.86. However, pts with stable BM had higher HUS than those with progressive BM (mHUS: 0.80 vs 0.69; p = 0.045). Of BM+ pts, 44% had received whole brain radiation (WBRT). Correlations of NCF and HUS specific for BM+ pts were observed for several HLVT scores, including Total Recall (TR), which was correlated with HUS in BM+ (R = 0.35, p = 0.01) but not in NBM (R = 0.04, p = 0.84) and Recognition Discrimination Index (BM+: R = 0.32, p = 0.03 vs NBM: R = 0.13, p = 0.51). In contrast, TMT-A/B NCF test results had slightly stronger associations with HUS in NBM pts. COWAT was least associated with HUS in BM+ or NBM pts. In BM+ pts treated vs untreated with WBRT, HLVT scores were better in untreated patients (TR, p &lt; 0.0001; delayed recall, p = 0.006; retention, p = 0.089), associations not seen with either TMT-A/B or COWAT. Mutation status had no bearing on these associations. Conclusions: NCF impacts HUS in S4LC pts and should be considered in treatment planning. HVLT scores are useful to assess specifically the impact of BM and WBRT in S4LC pts, and is reflected in associations with HRQoL.

The impact of neurocognitive function on health utility scores (HUS) in Stage 4 (S4) lung cancer patients (pts) with and without brain metastases (BrM).

225 Background: As targeted therapies for S4LC pts have improved survival, the impact of health-related quality of life (HRQoL) in these pts grows in importance. In pts with BrM, loss of NCF may affect HRQoL significantly. We evaluated the relationship between NCF and HUS as a measure of HRQoL in S4LC pts. Methods: Self-reported HUS data from EQ5D-3L were obtained cross-sectionally from S4LC pts with (BrM) or without BrM (non-BrM). NCF was measured using the Hopkins Verbal Learning Test – Revised (HVLT-R), the Controlled Oral Word Association Test (COWAT) and Trail Making Tests (TMT-A/B). NCF scores were correlated with HUS (Pearson Coefficient, R). Results: BrM (n = 54) and non-BrM (n = 40) patients had similar demographics- overall median age was 61 (range 33-89) years; 59% were female; 45% had EGFR/ALK alterations; BrM pts were treated with whole brain (n = 20), stereotactic radiation (n = 19), both (n = 6), or sole use of systemic agents (n = 6); 3 were observed; 7 had BrM resection. Overall HUS were similar between BrM and non-BrM groups (mean HUS (mHUS): 0.77 vs. 0.78; p = 0.86). However, pts with stable brain disease had higher HUS than those with progressive brain disease (mHUS: 0.80 (n = 36) vs 0.69 (n = 17); p = 0.045). There was a trend towards lower HUS in symptomatic vs. asymptomatic BrM (mHUS: 0.70 (n = 10) vs. 0.78 (n = 43); p = 0.07). Multiple correlations between NCF scores and HUS were found. HLVT-Total Recall correlated with HUS in BrM but not non-BrM (R=0.35, p = 0.01; vs. non- R=0.04, p = 0.84 respectively) as did the HLVT-Recognition (BrM: R=0.32, p = 0.03 vs. non-BrM: R=0.13, p = 0.51). In contrast, TMT-A/B were associated with HUS in both BrM (p = 0.03, 0.06) and non-BrM pts (p = 0.001, 0.03). COWAT was associated with HUS only when all pts were analyzed together (p = 0.04). Conclusions: In S4LC pts, HUS were correlated with multiple measures of NCF. The impact of uncontrolled brain disease and poor NCF on HUS demonstrates that this measure has clinical utility, with important HRQoL implications as metastatic lung cancer pts live longer.

P2.03b-008 The Impact of Brain Metastases and Their Treatment on Health Utility Scores in Molecular Subsets of Lung Cancer Patients: Topic: Brain Meta

New therapies, particularly in advanced patients with EGFR-mutated and ALK-rearranged tumors, result in prolonged survival. Brain metastases and/or their treatment, may have a negative impact on health-related quality of life. Technological assessment of the cost-effectiveness of various treatments for brain metastases will benefit from measurements of health-related qualify of life and health utility scores (HUS). This study evaluated the impact of brain metastases on HUS across multiple health states defined on the basis on disease stability, brain-specific therapies, and molecularly-defined subsets of NSCLC. A longitudinal cohort study at Princess Margaret Cancer Centre evaluated 1571 EQ5D-3L-derived HUS in 476 Stage IV lung cancer outpatients, from Dec, 2014 through May, 2016: EGFR+ (n=183), ALK+ (n=38), wild-type (WT) non-squamous (n=171), squamous (n=29), and small cell lung cancer (SCLC) (n=30). Patients were stratified according to presence or absence of brain metastases at the time of assessment; mean HUS (± standard error of the mean, SEM) by presence of brain metastases and various health states and disease subtypes were reported. For patients with repeated measures, only the earliest time point was analyzed. 172 patients had brain metastases, median age 62 (range 32-86) years and 304 patients did not have brain metastases, median age 66 (29-96) years. Overall HUS was related to disease subtype but not presence of brain metastases: EGFR/ALK+ patients with (0.78±0.02) or without brain metastases (0.79±0.01) versus WT/SCC/SCLC with (0.74±0.02) and without brain metastases (0.73±0.01) (p=0.01 by subtype; p>0.10 by presence of brain metastases). However, symptomatic CNS disease (0.69±0.04) had lower HUS (versus asymptomatic disease (0.77±0.02)) (p=0.03). Patients achieving intracranial stability or response to treatment had significantly higher HUS (0.81±0.05) than patients with progressive CNS metastases (0.72±0.02) (p=0.03). Extra-cranial control also correlated with higher HUS (0.81±0.02 versus 0.69±0.03, p<0.0001). When local treatment for brain metastases was delivered within 6 months, HUS was lower (0.71±0.02 versus 0.82±0.02, p=0.0005). CNS disease treated only with systemic therapy or on no active therapy had mean HUS of 0.81±0.03, while patients treated only with stereotactic radiosurgery (SRS) had values of 0.80±0.04; there was a trend for lower HUS with whole brain radiation (WBRT) only (0.72±0.03) or WBRT+SRS (0.74±0.03) (p=0.11). Brain metastasis stability has significant impact on HUS in lung cancer patients. Treatment modalities of brain metastases may also impact HUS. Data collection is ongoing; updated HUS data including longitudinal assessments and multivariable analyses will be presented.

Real-World EQ5D Health Utility Scores for Patients With Metastatic Lung Cancer by Molecular Alteration and Response to Therapy

IntroductionEconomic analyses of upcoming treatments for lung cancer benefit from real-world health utility scores (HUSs) in an era of targeted therapy. MethodsA longitudinal cohort study at Princess Margaret Cancer Centre evaluated 1571 EQ5D-3L-derived HUSs in 475 outpatients with metastatic lung cancer across various disease states. Patients with epidermal growth factor receptor (EGFR) (n = 183) and anaplastic lymphoma kinase (ALK) (n = 38) driver alterations were enriched through targeted enrolment; patients with wild-type non–small-cell lung cancer (WT NSCLC) (n = 224) and small-cell lung cancer (SCLC) (n = 30) were sampled randomly. ResultsFor patients stable on most appropriate treatment, the mean HUSs were 0.81 and 0.82 in patients receiving EGFR and ALK tyrosine kinase inhibitors (TKIs) respectively (with similar HUSs across agents), which were higher than patients with WT NSCLC (0.78; P = .04) and SCLC receiving chemotherapy (0.72; P = .06). In mutation-specific comparisons, disease stability on appropriate therapy resulted in significantly higher mean HUSs (P < .002-.02) than when disease was progressing (mean HUS: EGFR, 0.70; ALK, 0.69; WT NSCLC, 0.66; SCLC, 0.52). When evaluating treatment-related toxicities, significant inverse relationships were observed between HUS and the severity of fatigue and decreased appetite in the EGFR group. There was also a significant inverse relationship between the total number of clinically significant symptoms and HUS, both in patients who were EGFR-mutated and patients with WT NSCLC. ConclusionsIn a North American setting, HUSs generated from patients with metastatic lung cancer are higher in treated, stable patients carrying driver mutations. This is partially explainable by treatment toxicity and patient symptom differences. Such differences in scores should be considered in economic analyses.

Can the general public use vignettes to discriminate between Alzheimer's disease health states?

BackgroundValid estimates of health-related quality-of-life (HRQoL) are often difficult to obtain from persons with Alzheimer’s disease (AD) and family caregiver proxies. To help assess whether the general public can serve as an alternate source of proxy HRQoL estimates in AD, we examined whether the general public can use vignettes to discriminate between AD health states.MethodsWe administered a telephone survey to randomly recruited participants from the general public who were aged 18 years or older. Interviewers read vignettes describing the mild, moderate, and severe AD health states to the participants, who answered the EQ-5D-5L and Quality of Life-Alzheimer’s Disease (QoL-AD) scales as if they had AD based on the vignette descriptions. Participants also answered the EQ-5D-5L for their current health states. We converted EQ-5D-5L responses into health utility scores using Canadian preference weights. We employed the Wilcoxon signed rank test to examine whether mean health utility scores and mean QoL-AD scores differed between health states. We used Pearson’s r to assess correlations between health utility and QoL-AD scores.ResultsForty-eight participants (median age = 53 years; 25 female) completed the telephone interview; health utility and QoL-AD scores decreased as AD severity increased (p <0.0001). Mean health utility scores were 0.65 (mild), 0.51 (moderate), and 0.25 (severe). Mean QoL-AD scores were 26.7 (mild), 23.0 (moderate), and 17.4 (severe). The correlations between health utility and QoL-AD scores were moderate to strong (r ≥ 0.62).ConclusionsUsing the vignettes, the general public provided HRQoL estimates that discriminated between the three AD health states. This finding suggests the general public may be a promising source of proxy HRQoL estimates in place of persons with AD.Electronic supplementary materialThe online version of this article (doi:10.1186/s12877-016-0207-4) contains supplementary material, which is available to authorized users.

Patient-reported symptoms and Canadian Health Utility scores in esophageal cancer patients.

6607 Background: Health utility scores (HUS) are an increasingly important tool to help determine the cost-effectiveness of therapies worldwide. The EQ-5D-3L is a validated tool for deriving HUS, with reference data in numerous populations, but there is limited information on the role that individual symptom complexes (SC) play in determining HUS in Esophageal cancer (EC) patients. Methods: A cross-sectional survey of EC patients at Princess Margaret Cancer Centre (2012-2014) assessed EQ-5D-3L and FACT-E. EQ-5D scores were converted to HUS using Canadian references. SC were developed: domains from FACT-E were identified and grouped to represent symptoms such as pain, dysphagia, fatigue, dyspnea, nausea and loss of appetite. Multivariable regression analysis was performed to assess associations between SC and HUS, adjusting for age, sex, and stage of disease. Results: Of 197 patients, median age was 67 (range 32-93) years, 76% were male, with stage I/II-IVA/IVB (6%/66%/28%) disease. Mean (SE) HUS were 0.90...