EQ-5D Utility Values Research Articles

Prognostic nutritional index (PNI) is an independent predictor for functional outcome after hip fracture in the elderly: a prospective cohort study

SummaryThe prognostic nutritional index (PNI) is a useful tool for assessing nutritional status using serum albumin and lymphocyte count. This study indicates that a higher preoperative PNI correlates with improved mobility and health-related quality of life during the initial postoperative period in elderly patients with hip fractures.PurposeTo investigate the prognostic value of the prognostic nutritional index (PNI) in predicting mobility and health-related quality of life (HRQoL) in elderly hip fracture patients after surgery.MethodsWe prospectively involved patients aged 65 and above, who could walk freely before injury and underwent surgery between 2018 and 2019. Admission PNI was calculated as serum albumin (g/L) + 5 × total lymphocyte count (× 109/L). Patients were classified into two groups based on PNI median value. All patients were followed up by telephone for four times (30-day, 120-day, 1-year, and 3-year after surgery). The Fracture Mobility Score (FMS) and EuroQol 5-Dimension 5-Level (EQ-5D 5L) were used to evaluate mobility and HRQoL, respectively.ResultsOf 705 eligible patients, 487 completed all assessments. Patients in the higher PNI group had a significantly increased possibility of achieving unrestricted mobility at the 120-day follow-up (OR 1.69, 95% CI 1.10–2.61, P.adj = 0.017), while no significant differences were observed at other follow-ups. Additionally, patients in the higher PNI group had a significantly higher EQ-5D utility value at the 30-day follow-up (P.adj = 0.015). A linear regression model with adjusting for all confounders showed that admission PNI value was positively associated with EQ-5D utility values at 30-day, 120-day, and 1-year follow-up assessments (P.adj = 0.011, P.adj = 0.001, and P.adj = 0.030, respectively). However, this correlation was not observed at the 3-year time point (P.adj = 0.079).ConclusionThe PNI is a valuable predictor of functional outcomes in elderly patients with hip fractures following surgery.

P153 Exploring the relationships between objective and subjective clinical measures in Sjogren's Syndrome

Abstract Background/Aims Patient Reported Outcome Measures (PROMs) are standardised, validated questionnaires designed to measure the patient’s perception of their disease. Discordance between the patient’s perspective and objective clinical measures (clinical scores, laboratory data) has led to increased interest in PROMs in Sjogren’s syndrome (SS). Initial concern about the subjectivity of PROMs has given way to methodological rigour and clear guidance for the development of PROMs. Several studies have now used PROMs for symptom-based stratification, and recently researchers have developed composite disease assessment tools incorporating PROMs and objective clinical measures to create more clinically meaningful endpoints for SS trials. With the advent of PROM-based stratification tools and composite endpoints, it is vital to better understand the relationship between clinical measures in SS. In this project we performed network analysis with data from a large cohort of well-characterised SS patient to improve our understanding of the relationship between objectively measured parameters and PROMs. Methods The UK Primary Sjogren’s Syndrome Registry (UKPSSR) is a nationwide observational cohort of primary SS patients who fulfil the 2002 American European Consensus Group classification criteria. The UKPSSR holds detailed clinical and laboratory data that is collected prospectively, including patient-reported outcomes collected using standardized questionnaires. To assess concordance between clinical measures we used the ARACNE algorithm, which calculates mutual information between the variables of a multidimensional dataset to construct a network of shared information. Statistical analysis was performed in R-v4.1.3 and JMP Pro-v15 and network visualisation in Cytoscape-v3.8.2. Results The ARACNE network highlights key areas of concordance and discordance between PROMs, clinician-based and laboratory-based measurements. Providing assurance to this approach, multi-domain PROMs and ESSDAI (EULAR Sjögren's syndrome disease activity index) subdomains are strongly associated with their component scores and different PROMs measuring the same symptoms were also correlated. In contrast, there is a clear separation between PROMs, clinician-based and laboratory-based measurements each forming their own sub-networks. Furthermore, most laboratory measurements do not have strong relationship with clinician-based disease activity score or PROMs. EQ-5D utility and HADS (Hospital Anxiety and Depression Scale) values provide the primary link between ESSPRI (EULAR Sjogren's Syndrome Patient Reported Index) and ESSDAI subdomain scores and both ESSDAI and ESSPRI are important contributors to quality-of-life. Conclusion The dissociation between PROMs and objective clinical measures is important for clinical management and trial design. Whilst it complicates clinical management of SS including any treat-to-target strategy; it highlights the importance of PROMs in supporting clinician assessment and laboratory measurements to better capture all aspects of SS and give a more individualised, meaningful assessment. Finally, it supports the dual-target strategy of systemically pursuing two targets in the design of clinical trials: one focused on the disease activity and another focused on the symptoms and impact. Disclosure J.S. Berry: None. J. Tarn: None. D. Lendrem: None. J. Casement: None. W. Ng: Consultancies; Expert advice in the area of Sjogren’s syndrome to GlaxoSmithKline, MedImmune, UCB, Abbvie, Roche, Eli Lilly, Takeda, Resolves Therapeutics, Sanofi, Novartis and BMS.

Cost-Effectiveness of Pharmacist Prescribing for Managing Hypertension in the United States

Pharmacist-led interventions can significantly improve blood pressure (BP) control. The long-term cost-effectiveness of pharmacist-prescribing interventions implemented on a large scale in the US remains unclear. To estimate the cost-effectiveness of implementing a pharmacist-prescribing intervention to improve BP control in the US. This economic evaluation included a 5-state Markov model based on the pharmacist-prescribing intervention used in The Alberta Clinical Trial in Optimizing Hypertension (or RxACTION) (2009 to 2013). In the trial, control group patients received an active intervention, including a BP wallet card, education, and usual care. Data were analyzed from January to June 2023. Cardiovascular (CV) events, end-stage kidney disease events, life years, quality-adjusted life years (QALYs), lifetime costs, and lifetime incremental cost-effectiveness ratio (ICER). CV risk was calculated using Framingham risk equations. Costs were based on the reimbursement rate for level 1 encounters, medication costs from published literature, and event costs from national surveys and pricing data sets. Quality of life was determined using a published catalog of EQ-5D utility values. One-way sensitivity analyses were used to assess alternative reimbursement values, a reduced time horizon of 5 years, alternative assumptions for BP reduction, and the assumption of no benefit to the intervention after 10 years. The model was expanded to the US population to estimate population-level cost and health impacts. Assumed demographics were mean (SD) age, 64 (12.5) years, 121 (49%) male, and a mean (SD) baseline BP of 150/84 (13.9/11.5) mm Hg. Over a 30-year time horizon, the pharmacist-prescribing intervention yielded 2100 fewer cases of CV disease and 8 fewer cases of kidney disease per 10 000 patients. The intervention was also associated with 0.34 (2.5th-97.5th percentiles, 0.23-0.45) additional life years and 0.62 (2.5th-97.5th percentiles, 0.53-0.73) additional QALYs. The cost savings were $10 162 (2.5th-97.5th percentiles, $6636-$13 581) per person due to fewer CV events with the pharmacist-prescribing intervention, even after the cost of the visits and medication adjustments. The intervention continued to produce benefits in more conservative analyses despite increased costs as the ICER ranged from $2093 to $24 076. At the population level, a 50% intervention uptake was associated with a $1.137 trillion in cost savings and would save an estimated 30.2 million life years over 30 years. These findings suggest that a pharmacist-prescribing intervention to improve BP control may provide high economic value. The necessary tools and resources are readily available to implement pharmacist-prescribing interventions across the US; however, reimbursement limitations remain a barrier.

#5510 CKD-ASSOCIATED PRURITUS SEVERITY AND ITS ASSOCIATION WITH BODY DISTRIBUTION AND EQ-5D HEALTH-RELATED QUALITY OF LIFE IN HAEMODIALYSIS PATIENTS

Abstract Background and Aims Chronic Kidney Disease associated pruritus (CKD-aP) is common affecting 40% of people with kidney disease receiving dialysis. It is associated with decreased health-related quality of life (HRQoL) as assessed using disease-specific instruments. A better understanding of how the severity of CKD-aP is related to the distribution of affected body parts and generic of HRQoL assessed using the EQ-5D instrument could improve the identification, assessment, and treatment of CKD-aP including advocating for access to new therapies. Method Prevalent in-centre haemodialysis patients from five centres prospectively completed the EQ-5D-5L HRQoL questionnaires, the severity-based worst itch numeric rating scale (WI-NRS) and multi-dimensional 5-D itch pruritus disease specific quality of life instruments. Latent class mixture models were used to identify clusters of patients with similarly affected body parts as assessed through the 5-D itch and map the pruritus measures to the EQ-5D utility value (1 being perfect health and 0 being dead, heavily skewed). Patient demographics, comorbidities, dialysis prescription and anti-pruritus medications were collected. Results Pruritus data on 485 respondents were obtained. No pruritus was reported in 164 (33.8%), with 117 (24.1%) reporting mild, 123 (25.4%) reporting moderate and 81 (16.7%) reporting severe pruritus. Commonly affected body parts included groin (22%), upper arms (21%), forearms (11%), and back (10%). Anti-pruritus medication use across CKD-aP severity (none to severe) was 40.6%, 38.5%, 36.6% and 55.6%, and varied by body part with 38.8%, 21.0% and 14.5% use in those affected in the upper limbs, groin and lower legs respectively. Latent class analysis identified three groups of patients who had progressively worsening severity and number of body parts affected, but the distribution of affected body parts was relatively constant (left-hand figure) and reduction in EQ-5D by affected body part was similar. Although the WI-NRS and 5-D itch instruments correlated with each other, only the 5-D itch had a strong relationship with EQ-5D-3L: Controlling for age, sex, diabetes, and years receiving dialysis, predicted EQ-5D utility dropped linearly from 0.69 to 0.41 (right-hand figure). Conclusion Contemporary UK data shows CKD-aP remains highly prevalent amongst people with kidney failure on dialysis. Severe CKD-aP was commonly reported despite half of the patients with severe CKD-aP receiving antipruritic medication, illustrating a high unmet need and likely undertreated. Although there were similar reductions in HRQoL, medication use varied by body part and those whose groin is affected may be reporting other body parts to access therapies. High use of CKD-aP medications in none or mild severity may represent more severely affected individuals benefiting from these drugs as the cross-sectional nature of the study means those who reported no pruritus may have had pruritus in the past which resolved in response to the medications prescribed and reported in these analyses. Overall, as it worsens CKD-aP appears to affect a similar distribution of body parts. Pruritus instruments that include domains that are broader than just pruritus severity more closely approximate the EQ-5D generic HRQoL measure and therefore more strongly advocate for the value of treating this unpleasant condition. Funded by CSL Vifor.

Mapping Oswestry Disability Index Responses to EQ-5D-3L Utility Values: Are Cost-Utility Results Valid?

ObjectivesTo develop and validate approaches for mapping Oswestry Disability Index responses to 3-level version of EQ-5D utility values and to evaluate the impact of using mapped utility values on cost-utility results compared with published regression models. MethodsThree response mapping approaches were developed in a random sample of 70% of 18 692 patients with low back pain: nonparametric approach (Non-p), nonparametric approach excluding logical inconsistencies (Non-peLI), and ordinal logistic regression (OLR). Performance was assessed in the remaining 30% using R-square (R2), root mean square error (RMSE), and mean absolute error (MAE). To evaluate whether MAEs and their 95% limits of agreement (LA) were clinically relevant, a minimally clinically important difference of 0.074 was used. Probabilities of cost-effectiveness estimated using observed and mapped utility values were compared in 2 economic evaluations. ResultsThe Non-p performed the best (R2 = 0.43; RMSE = 0.22; MAE = 0.03; 95% LA = −0.40 to 0.47) compared with the Non-peLI (R2 = 0.07; RMSE = 0.29; MAE = −0.15; 95% LA = −0.63 to 0.34) and OLR (R2 = 0.22; RMSE = 0.26; MAE = 0.02; 95% LA = −0.49 to 0.53). MAEs were lower than the minimally clinically important difference for the Non-p and OLR but not for the Non-peLI. Differences in probabilities of cost-effectiveness ranged from 1% to 4% (Non-p), 0.1% to 9% (Non-peLI), and 0.1% to 20% (OLR). ConclusionsResults suggest that the developed response mapping approaches are not valid for estimating individual patients’ 3-level version of EQ-5D utility values, and—depending on the approach—may considerably affect cost-utility results. The developed approaches did not perform better than previously published regression-based models and are therefore not recommended for use in economic evaluations.

Impact of medication therapy management (MTM) service model on multi-morbidity (MMD) patients with hypertension: a pilot RCT

BackgroundThis study explored the impact of MTM service on MMD patients with hypertension.MethodsA total of 120 MMD inpatients from September to November 2019 were received and randomly divided into intervention group and control group. General services for noninfectious chronic diseases were given to the control group, while a standard MTM service was given to the intervention group. Patients’ blood pressure, EQ-5D utility value, readmission rate, drug-related problems, and average daily medication therapy cost were compared between the two groups and within the groups. This was done at the initial admission phase and in the first, third, sixth, and twelfth months after discharge.ResultsThe intervention group had significantly lower blood pressure and average daily medication therapy cost 12 months after discharge compared to the control group (systolic blood pressure: P = 0.023, diastolic blood pressure: P < 0.001, average daily medication therapy cost: P = 0.049); the number of DRPs decreased in both groups 12 months after discharge; the number of DRPs solved in the intervention group in the third, sixth and twelfth months after discharge were statistically higher compared with that in the control group (P = 0.013, P = 0.012, P = 0.001); there was no significant difference in the EQ-5D utility value and readmission rate between the two groups (P > 0.05).ConclusionsMTM implementation in MMD patients can improve health outcomes and reduce healthcare-related costs among MMD patients.Trial registrationChinese Clinical Trial Register ChiCTR2200065111, date of registration: October 28, 2022.

54MO Quality of life (QoL) and value of health (V-He) in advanced biliary cancers (ABC) treated with second-line active-symptom-control (ASC) alone or ASC with oxaliplatin/5-FU chemotherapy (ASC+FOLFOX) in the randomised phase III, multi-centre, open-label ABC-06 trial

The ABC-06 clinical trial stablished ASC+mFOLFOX as the standard of care treatment after Cisplatin and Gemcitabine (CisGem) for ABC but its impact on QoL has not been reported. Within the ABC-06 study, patients (pts) diagnosed with ABC with progression after CisGem were randomised (1:1) to ASC+mFOLFOX or ASC. QoL (European Organisation for Research and Treatment of Cancer [EORTC] QLQ-30, QLQ-BIL21) and V-He (Euroqol EQ5D) questionnaires were completed at baseline and 12-weekly thereafter. Pateints who completed all or part of the EORTC or EQ-5D measure at baseline (BSL) were elegible. Follow-up data is reported for month 4 (M4) (follow-up visit that occurred up to 120 days post screening). Descriptive analyses,of the QoL measures by study arm are reported here; T-test was used for comparison between time-points (two-tailed p-value <0.05 was considered statistically significant). Out of 162 pts randomised, 138 were eligible for this available case analysis (65 ASC arm, 73 ASC+FOLFOX arm). Baseline characteristics and time from BSL to M4 visit were well balanced between both study arms. Addition of FOLFOX to ASC did not appear to induce worsening of the QOL parameters assessed (Table). In contrast, pts in the ASC-alone arm appeared to experience worsening of the EQ-5D utility values, and most of the QLQ-30 scales (including global, physical, social and role scales). There also appeared to be worsening of nausea and pain which remained stable in the ASC+FOLFOX arm.Table: 54MOMeasureTime-pointASCASC+mFOLFOXMeanp-valueInterpretationMeanp-valueInterpretationEQ-5D utility valueBSL0.750.0309↓0.770.6183↔M40.620.70QLQ-30 Summary scoreBSL770.0446↓790.1846↔M46471QLQ-30 Global health scaleBSL670.0447↓660.8295↔M45358QLQ-30 Physical health scaleBSL740.0221↓780.1238↔M45966QLQ-30 Social function scaleBSL770.0272↓760.1307↔M46165QLQ-30 Emotional scaleBSL790.4630↔811.000↔M47379QLQ-30 Role scaleBSL710.0283↓760.2713↔M44957QLQ-30 Cognitive scaleBSL870.3009↔850.5075↔M47580QLQ-30 NauseaBSL100.0009↑100.4495↔M42516QLQ-30 PainBSL290.0323↑280.2089↔M44421QLQ-30 FatigueBSL400.1129↔350.4662↔M45648 Open table in a new tab For ABC patients, treatment with second-line ASC+mFOLFOX may allow stabilisation of QoL scales and avoid worsening of nausea and pain at 4 months after baseline assessment. Full analyses of QoL to explore this potentially positive impact of FOLFOX is ongoing, along with an assessment of whether FOLFOX may be cost-effective.

The Impact of Symptom Severity on Health-Related Quality of Life in People with Narcolepsy Type 1

ABSTRACT Objective To assess the impact of symptom severity on health-related quality of life (HRQoL) in people with narcolepsy type 1 (NT1). Methods A total of 174 people with NT1 were enrolled. They completed the Narcolepsy Severity Scale (NSS) and EQ-5D-3L consisting of five dimensions (EQ-5D utility values) and a visual analog scale (EQ-5D VAS). The relationship between severity of symptoms and HRQoL dimensions was evaluated by Pearson correlation analyses. Logistic regression was used to identify significant predictors of HRQoL. Nomogram was established based on results of independent predictors of factors on logistic regression analyses. Results The mean score for NSS, EQ-5D utility values, and EQ-5D VAS were 29.8 (10.08), 0.78 (0.09), and 64.30 (19.84) in people with NT1, respectively. NSS score showed a significant correlation with self-care (r = 0.157, p < .05), usual activities (r = 0.236, p < .01), pain/discomfort (r = 0.174, p < .05), anxiety/depression (r = 0.2, p < .01), and EQ-5D utility values (r = −.261, p < .01). EDS (excessive daytime sleep), cataplexy, hallucinations, paralysis, and disrupted nocturnal sleep (DNS) were significantly associated to EQ-5D VAS (r ranged from −0.154 to −0.354, p < .05). EDS (OR = −0.297) and DNS (OR = −0.16) were predictors of HRQoL. NSS score (OR = −0.360) and treatment (OR = 0.215) were predictors of the metrics of HRQoL. The C-indices of the nomogram were 0.726. Conclusion The severity of symptoms could disrupt self-care and usual activities, and increase pain/discomfort and anxiety/depression. HRQoL might be improved by alleviating symptom severity.

Health-related quality of life using EQ-5D among chronic myeloid leukaemia patients in health centres in Klang Valley, Malaysia.

Chronic Myeloid Leukaemia (CML) responds well with the targeted therapy drugs, Tyrosine Kinase Inhibitors (TKI), that give potentially long-term disease control for the patients. The objective of this study was to determine the disease burden and factors influencing the health-related quality of life (HRQoL) and health status of CML patients in Klang Valley, Malaysia. CML patients were recruited from haematological outpatient clinics in health centres in Klang Valley, Malaysia. A semi-guided self-administered questionnaire was used. HRQoL was measured by EQ-5D utility value and health status was by visual analogue score (VAS). Logistic regression analysis was conducted to determine the factors influencing HRQoL and health status. A total of 221 respondents participated, where more than half were Malay (56.6%), male (53.4%), and an Imatinib user (68.8%). Majority were diagnosed at the chronic phase (89.5%). The mean age of diagnosis was 41 years old. Significant determinant associated with HRQoL was age of diagnosis. These factors had no significant effect on the HRQoL of these patients regardless of types of TKI used and initial phase of CML. The overall HRQoL of CML patients were comparable to, if not higher, than the general population. Any TKI that was good enough to eliminate disease symptoms and erase patient’s worries, can possibly make CML patients have a better quality of life than typical cancer patients and even the general population.

Economic Evaluation for the US of Venetoclax Versus Allogeneic Hematopoietic Stem-Cell Transplantation for Patients with Relapsed or Refractory Chronic Lymphocytic Leukemia with 17p Deletion

Background: Until the advent of targeted agents, the primary treatment for patients with relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL) with 17p deletion (del(17p)) was allogeneic hematopoietic stem cell transplantation (HSCT). Venetoclax is a targeted therapy that has been shown to effect significant progression free (PFS) and overall survival (OS) with an acceptable safety profile and the convenience of oral administration. HSCT is potentially curative but is associated with potential transplant-associated morbidity and mortality (incl. graft-versus-host disease) and relapse. Both treatments are expensive, HSCT largely up-front and venetoclax long term, with relative differences in cost of treatment and supportive care management. Considering these different efficacy, safety, and procedural profiles, we evaluated the cost-effectiveness and cost-utility of venetoclax versus HSCT in R/R CLL del(17p) from a US payer perspective.Methods: A life time horizon Markov model was constructed specifying 3 health states: PFS with embedded sub-states specifying whether patient is on or off therapy, progression, and death. Data sources to simulate the R/R CLL del(17p) patients included Stilgenbauer et al (Lancet Oncol 2016) for venetoclax and Schetelig et al (J Clin Oncol 2008) for HSCT. Kaplan-Meier PFS and OS curves were digitized, PFS and OS data extracted, and Weibull distributions fitted to these data for both venetoclax and HSCT. The wholesale acquisition cost of venetoclax was sourced from RedBook. Costs of the HSCT procedure, pre-conditioning treatment, and post-procedural side effects were estimated from published prediction equations and national private claims database. EQ-5D utility values were sourced from published literature. Disutility values for side effects were assumed same for both interventions. A discount rate of 3% was applied when survival exceeded one year. Following extrapolation to a life time horizon, the life years (LY) and quality adjusted LY (QALY) for each treatment were estimated, and the incremental LY and QALY gained with venetoclax over HSCT calculated. From the respective total treatment costs, cost differentials and the incremental cost effectiveness (ICER) and cost utility ratios (ICUR) were determined. Base case analyses were validated by probabilistic sensitivity analyses (PSA) of 2000 iterations.Results: Venetoclax prevailed in PFS over HSCT until ~190 weeks (3.7 years) when curves crossed and HSCT prevailed. Venetoclax prevailed in OS over HSCT over the full life time horizon. As shown in the Table, in the life time Markov model, compared to HSCT, venetoclax showed cost savings of -$360,995 (PSA: -$350,350) and incremental gains of 0.64 (PSA: 0.62) LY and 0.39 (PSA: 0.37) QALY. This yielded an ICER of savings of -$564,054 (PSA: -$565,080) per LY gained and an ICUR of savings of -$925,628 (PSA: -$946,891) per QALY gained.Conclusion: Against a life time horizon, venetoclax prevails in OS and for ~3.7 years in PFS over HSCT in the management of patients with R/R CLL del(17p). Yielding a cost saving by ~$565,000 if LYs gained are concerned but by ~$945,000 in the case of QALYs gained, it has the life time benefit of being clinically superior in combined OS and safety at markedly lower cost. While surviving HSCT-treated patients had improved outcomes with a slightly higher likelihood of subsequent PFS than venetoclax-treated patients, this marginal benefit is dominated by the significantly higher probability of OS for patients with venetoclax therapy. Venetoclax provides significantly greater life time clinicoeconomic value in the treatment of patients with R/R CLL del(17p) versus HSCT. [Display omitted] DisclosuresPersky:Genentech: Consultancy; MorphoSys: Other: Independent Data Monitoring Committee member ; Verastem: Consultancy; Spectrum Pharmaceuticals: Research Funding. Abraham:Janssen Oncology (Johnson & Johnson, LLC): Consultancy.

Health state utility values derived from EQ-5D in psoriatic patients: a systematic review and meta-analysis

Background To determine pooled EQ-5D utility scores for psoriasis as a general condition, plaque psoriasis, and psoriatic arthritis. Methods A systematic review and meta-analysis of EQ-5D utility scores for psoriatic patients was conducted. Univariate meta-regression was used to explore the degree of heterogeneity. Results Seventy-five studies were included in the systematic review. The EQ-5D in psoriatic patients demonstrated decent convergent, known-groups validity, and a degree of responsiveness with a ceiling effect. Among the five EQ-5D dimensions, ‘self-care’ showed the lowest and ‘pain/discomfort’ showed the highest percentages of reporting any problems. For meta-analysis, we identified 70 utility scores from 59 studies: 22 for plaque psoriasis, 26 for psoriasis as a general condition, and 22 for psoriatic arthritis. The mean (95% CIs; I2) of the EQ-5D utility scores for psoriasis as a general condition, plaque psoriasis, and psoriatic arthritis was 0.748 (0.718, 0.777; 98.8%), 0.755 (0.727, 0.783; 98.6%), and 0.585 (0.538, 0.632; 98.2%), respectively. For psoriasis as a general condition and plaque psoriasis, factors such as country, psoriasis area and severity index (PASI), dermatology life quality index (DLQI) and questionnaire version (EQ-5D-3L or EQ-5D-5L) all significantly influenced the utility scores. Conclusion Psoriasis imposes a substantial impairment on patients’ quality of life, especially the pain/discomfort dimension. Heterogeneity exists among different EQ-5D utility values found in the literature.

Utility-Weighted Modified Rankin Scale Scores for the Assessment of Stroke Outcome: Pooled Analysis of 20 000+ Patients.

Patient-centered care prioritizes patient beliefs and values towards wellbeing. We aimed to map functional status (modified Rankin Scale [mRS] scores) and health-related quality of life on the European Quality of Life 5-dimensional questionnaire (EQ-5D) to derive utility-weighted (UW) stroke outcome measures and test their statistical properties and construct validity. UW-mRS scores were derived using linear regression, with mRS as a discrete ordinal explanatory response variable in 8 large international acute stroke trials. Linear regression models were used to validate UW-mRS scores by assessing differences in mean UW-mRS scores between the treatment groups of each trial. To explore the variability in EQ-5D between individual mRS categories, we generated receiver operator characteristic curves for EQ-5D to differentiate between sequential mRS categories and misclassification matrix to classify individual patients into a matched mRS category based on the closest UW-mRS value to their observed individual EQ-5D value. Among 22 946 acute stroke patients, derived UW-mRS across mRS scores 0 to 6 were 0.96, 0.83, 0.72, 0.54, 0.22, -0.18, and 0, respectively. Both UW-mRS and ordinal mRS scores captured divergent treatment effects across all 8 acute stroke trials. The sample sizes required to detect the treatment effects using UW-mRS scores as a continuous variable were almost half that required in trials for a binary cut point on the mRS. Area under receiver operator characteristic curves based on EQ-5D utility values varied from 0.66 to 0.81. Misclassification matrix showed moderate agreement between actual and matched mRS scores (kappa, 0.68 [95% CI, 0.67-0.68]). Medical strategies that target avoiding dependency may provide maximum benefit in terms of poststroke health-related quality of life. Despite variable differences with mRS scores, the UW-mRS provides efficiency gains as a smaller sample size is required to detect a treatment effect in acute stroke trials through use of continuous scores. Registration: URL: https://www.clinicaltrials.gov; Unique identifiers: NCT00226096, NCT00716079, NCT01422616, NCT02162017, NCT00120003, NCT02123875. URL: http://ctri.nic.in; Unique identifier: CTRI/2013/04/003557. URL: https://www.isrctn.com; Unique identifier: ISRCTN89712435.

Disease burden concerning hepatitis E-infected inpatients in Jiangsu province, China

ObjectiveThis study aimed to determine the disease burden of hepatitis E virus (HEV)-infected inpatients in Jiangsu province, China. MethodsBetween July 1, 2016 and October 31, 2018, 1152 HEV-infected inpatients were identified from four cities in Jiangsu province, namely, Nanjing, Suzhou, Yancheng, and Zhenjiang. The disease burden comprised the economic burden and loss of health due to HEV infection. Factors influencing the disease burden were analyzed using univariate and multivariate analyses. ResultsThe average direct, indirect, and total economic burden for 1152 HEV-infected inpatients was US$ 4,986.40, US$ 1,507.28, and US$ 6,493.68, respectively, accounting for 46.66%, 14.11%, and 60.77% of per capita disposable income (PCDI) in Jiangsu province, respectively. The disease burden for HEV-infected inpatients with hepatitis B was significantly higher than that for other inpatients. The average EQ-5D utility value of 1152 HEV-infected inpatients was 0.72 ± 0.18 quality-adjusted life years (QALYs) and the average EQ-visual analogue score (EQ-VAS) was 0.66 ± 0.17 points. Multivariate analysis showed that the direct economic burden and the total economic burden were influenced by variables such as hospitalization days, outcomes, past history of other diseases, and regions (P < 0.05). It was estimated the direct economic burden, the indirect economic burden, and the total economic burden for all HEV-infected inpatients in Jiangsu province in 2018 was approximately US$ 9.2 million, US$ 2.8 million and US$ 12.0 million, respectively. ConclusionThe disease burden of HEV infection in Jiangsu province is severe, and more attention should be paid to the prevention of hepatitis E and the treatment of comorbidities.

Measurement of health-related quality by multimorbidity groups in primary health care

BackgroundIncreased life expectancy in Western societies does not necessarily mean better quality of life. To improve resources management, management systems have been set up in health systems to stratify patients according to morbidity, such as Clinical Risk Groups (CRG). The main objective of this study was to evaluate the effect of multimorbidity on health-related quality of life (HRQL) in primary care.MethodsAn observational cross-sectional study, based on a representative random sample (n = 306) of adults from a health district (N = 32,667) in east Spain (Valencian Community), was conducted in 2013. Multimorbidity was measured by stratifying the population with the CRG system into nine mean health statuses (MHS). HRQL was assessed by EQ-5D dimensions and the EQ Visual Analogue Scale (EQ VAS). The effect of the CRG system, age and gender on the utility value and VAS was analysed by multiple linear regression. A predictive analysis was run by binary logistic regression with all the sample groups classified according to the CRG system into the five HRQL dimensions by taking the “healthy” group as a reference. Multivariate logistic regression studied the joint influence of the nine CRG system MHS, age and gender on the five EQ-5D dimensions.ResultsOf the 306 subjects, 165 were female (mean age of 53). The most affected dimension was pain/discomfort (53%), followed by anxiety/depression (42%). The EQ-5D utility value and EQ VAS progressively lowered for the MHS with higher morbidity, except for MHS 6, more affected in the five dimensions, save self-care, which exceeded MHS 7 patients who were older, and MHS 8 and 9 patients, whose condition was more serious. The CRG system alone was the variable that best explained health problems in HRQL with 17%, which rose to 21% when associated with female gender. Age explained only 4%.ConclusionsThis work demonstrates that the multimorbidity groups obtained by the CRG classification system can be used as an overall indicator of HRQL. These utility values can be employed for health policy decisions based on cost-effectiveness to estimate incremental quality-adjusted life years (QALY) with routinely e-health data.Patients under 65 years with multimorbidity perceived worse HRQL than older patients or disease severity. Knowledge of multimorbidity with a stronger impact can help primary healthcare doctors to pay attention to these population groups.

Economic Evaluation for the US of Ibrutinib Versus Acalabrutinib for Patients with Relapsed or Refractory Mantle Cell Lymphoma

Introduction. Mantle cell lymphoma (MCL) is a rare subtype of Non-Hodgkin lymphoma (NHL) with a poor prognosis. Ibrutinib is an oral Bruton's tyrosine kinase inhibitor (BTKi) approved in the US in 2013 for, among other indications, MCL patients with at least one prior therapy (i.e., relapsed/refractory [R/R] MCL). The BTKi acalabrutinib was approved in late 2017 specifically for this same indication. No studies have assessed the comparative efficacy and cost-effectiveness and cost-utility of both agents in the management of R/R MCL. We performed an indirect comparison of both agents in terms of progression-free survival (PFS) and overall survival (OS) to subsequently evaluate the relative cost-effectiveness and cost-utility of both treatment options of R/R MCL from a USA payer perspective.Methods. A life time horizon Markov model with 3 health states was specified: PFS, progression, and death. Trials to simulate these health outcomes under the respective treatment regimens included Dreyling et al (Lancet 2016) for ibrutinib and Wang et al (Lancet 2018) for acalabrutinib. Kaplan-Meier PFS and OS curves were digitized, PFS and OS data extracted, Weibull distributions fitted to these data, and extrapolations to the life time horizon estimated for both acalabrutinib and ibrutinib to estimate the life time efficacy of each treatment. Naïve patient simulation was performed to indirectly estimate and compare the OS and PFS estimates of the two agents. Wholesale acquisition costs (WAC) were obtained from RedBook (average sales price not yet available for acalabrutinib): $12,180 for ibrutinib and $14,064 for acalabrutinib. Costs of managing side effects and EQ-5D utility values were sourced from published literature. Costs were inflated based on the medical consumer price index to the first quarter of 2018. Disutility values for side effects were assumed the same for both interventions. A discount rate of 3% was applied when survival exceeded one year. The life years (LY) and quality adjusted LY (QALY) for each treatment, the incremental LY and QALY gained with acalabrutinib over ibrutinib, and the incremental cost-effectiveness (ICER) and cost-utility ratios (ICUR) were estimated in both base and probabilistic sensitivity analyses (PSA; 2000 simulations). The cost-effectiveness plane (CEP) and cost-effectiveness acceptability curves (CEAC) were plotted.Results. In the naïve patient simulation median PFS and OS were estimated to be 14.6 months and 25.1 months for ibrutinib, and 27.6 months and 168 months for acalabrutinib. As shown in the table below, in the life time horizon Markov model acalabrutinib showed incremental cost of $259,200/LY (PSA: $258,780/LY) and incremental gains of 0.48 LY (PSA: 0.48 LY) and 0.36 QALY (PSA: 0.36 QALY) over ibrutinib. This yielded an ICER of $540,000 per LY gained (PSA: $539,125 per LY gained) and an ICUR of $720,000 per QALY gained (PSA: $718,833 per QALY gained). In the CEP scatter plot, all estimates were in the upper-right quadrant indicating greater incremental QALY at higher incremental cost. Per the CEAC plot, the probability that acalabrutinib would be considered cost-effective was 50% at a willingness to pay threshold (WTP) of $718,833 per QALY and 70% at a WTP threshold of $748,000 per QALY.Conclusions. Clinically, treating patients with R/R MCL with acalabrutinib was associated with an absolute survival gain of 0.48 years (~6 months) and a quality-adjusted survival gain of 0.36 years (~4 months) over treatment with ibrutinib but at an additional cost of nearly $260,000 per year of treatment. Treating patients with RR/MCL acalabrutinib requires high WTP thresholds to be considered cost-effective to US payers. DisclosuresPersky:Genentech: Honoraria; Spectrum: Research Funding; Merck: Research Funding; Morphosys (IDMC): Consultancy. Abraham:Sandoz: Consultancy.

PSS11 - A Systematic Review and Meta-Analysis of Health State Utility Values in Psoriasis

To perform a systematic review and meta-analysis of published literature on health state utility values (HSUVs) for psoriasis. A total of 7 databases were searched in November 2017. These were Pubmed, EMBASE, Cochrane Library databases, Web of Science, Wiley online library, Scopus, and EBSCO. A systematic review of literature was conducted to estimate mean EQ-5D utility scores using random-effects meta-analysis, as well as to explore degree of heterogeneity in the HSUVs across a variety of clinical and study characteristics using univariate meta-regression. Fifty studies were selected for meta-analysis. A total of 69 HSUVs were identified: 26 for plaque psoriasis, 22 for psoriasis as a general condition, and 21 for psoriatic arthritis. “Self-care” showed the lowest and “pain/discomfort” showed the highest percentages of reporting any problems in the EQ-5D dimensions. The mean (95% CIs) of the pooled HSUV for psoriasis as a general condition was estimated to be 0.763（0.727, 0.799） , and the I2 statistic in Cochran’s Q test was 0.989. For plaque psoriasis, the mean (95% CIs; I2) of the HSUV estimate was 0.762 (0.737, 0.787；=0.986). For psoriasis arthritis, the mean (95% CIs; I2) of the HSUV estimate was 0.580 (0.533, 0.628；=0.982). HSUV difference between plaque psoriasis and psoriasis as a general condition was not statistically significant. The HSUVs for psoriasis arthritis were significantly lower than that of psoriasis, with a mean difference of 0.181. For psoriasis as a general condition and plaque psoriasis, factors such as countries of the study, different tariffs of EQ-5D, disease severities were all significantly. For psoriasis arthritis, different EQ-5D tariffs were found to have no significant influence on the HSUVs. Psoriasis imposes a substantial impairment on patients’ quality of life, especially in the dimension of pain/discomfort. Heterogeneity exists among different EQ-5D utility values derived from literature.

PSS12 - Cost-Effectivess Analysis of Brodalumab in Moderate-To-Severe Plaque Psoriasis in Canada

To assess the cost-effectiveness of brodalumab versus available biologics and best supportive care (BSC) in adult patients with moderate-to-severe plaque psoriasis from a Canadian public payer perspective. A decision tree followed by a Markov model was developed to compare brodalumab with etanercept, adalimumab, infliximab, ustekinumab 45mg, ustekinumab 90mg, secukinumab, ixekizumab, infliximab subsequent entry biologic, and BSC. Psoriasis Area Severity Index (PASI) response was assessed at the end of the induction period (10-16 weeks) for each comparator. Responders (defined as at least 75% reduction in baseline PASI score) continued initial therapy and maintained their PASI response until treatment discontinuation or death. Non-responders switched to BSC. Comparative efficacy data were informed by a network meta-analysis. EQ-5D utility values associated with each PASI health state were derived from the brodalumab AMAGINE-1 study. Drug costs were obtained from the Ontario Drug Benefit Formulary and unit costs from the Ontario Health Insurance Plan. A 10-year time horizon was employed with 1.5% annual discount rate for costs and outcomes. Probabilistic analyses were conducted for base case and all scenario analyses. The base case analysis demonstrated that brodalumab had an ICER of $118,741 per quality-adjusted life year versus BSC. The ICER for ixekizumab versus BSC is $152,703. All other comparators were either dominated or subject to extended dominance. The results were robust across a range of scenario analyses. The model concluded that brodalumab is the most cost-effective option compared with publicly funded biologics for the treatment of moderate-to-severe plaque psoriasis in Canada. As such, the reimbursement of brodalumab will benefit the Canadian public by introducing a highly effective and most likely costs-savings alternative to the public payer system.

Health state utilities in patients with advanced non-small-cell lung cancer in China.

Non-small-cell lung cancer (NSCLC) is a leading global health threat that impairs patient health outcomes. Health state utilities are fundamental values in economic evaluation and significantly vary across countries. Given the scarce data on the Chinese population, the current study measured utility values in the Chinese patients with NSCLC. This study was conducted as a cross-sectional survey of patients with advanced NSCLC at the Shanghai Chest Hospital. Utility values were assessed using the EuroQol five-dimension (EQ-5D) instrument and scored based on the Chinese-specific value algorithm. Predictors of utility values were examined using a subgroup analysis and a multiple regression model. The mean EQ-5D utility value of recruited patients was 0.814. The regression analysis revealed that tumor stage, treatment regimen and line of therapy were the potential predictors of utility values. This study provides the Chinese-specific health utility data for advanced NSCLC using the EQ-5D.

PRM88 - Estimating Patient Utility in Migraine: Longitudinal Analysis of Erenumab Clinical Trial Data

Migraine is a debilitating disease with substantial impact on patients’ health-related quality of life (HRQoL). Cost-utility analysis of migraine preventives requires the estimation of patient utility values and how these relate to monthly migraine days (MMD). The objective of this analysis is to model utility values in a longitudinal framework, allowing more accurate estimates of expected utility values. Data from two pivotal erenumab studies in episodic migraine (EM; NCT02456740) and chronic migraine (CM; NCT02066415) were pooled to analyze HRQoL outcomes. Migraine Specific Questionnaire (MSQ) version 2.1 and Headache Impact Test (HIT-6) condition-specific instruments were mapped to the EQ-5D using published mapping algorithms (Gillard et al. 2012) to generate generic, preference-based utility estimates, suitable for use in economic models. Longitudinal non-linear beta regressions were fitted between mapped EQ-5D utility values, MMD and treatment group (erenumab 140mg and placebo), adjusting for age, sex, race and baseline MMD in the various time periods considered. Expected utility values by MMD were calculated using the Delta method. Multilevel linear models and fractional response models with probit and logit links were also assessed. The analysis sample included data from 1,113 patients; 83.7% females with a mean age of 41.6 years. The longitudinal beta regression models closely approximated the observed utility values. Patients with 14 MMD were predicted to have MSQ mapped mean utility values of 0.608 (95% CI: 0.595–0.621) for patients treated with erenumab 140mg and 0.590 (0.577–0.602) for patients receiving placebo. The respective predicted HIT-6 mapped mean utility values were 0.698 (0.654–0.741) and 0.659 (0.629–0.689). Mapped utility values for erenumab patients were consistently higher than placebo patients with the same number of MMD. Linking patient QoL to MMD allows utility estimates for different levels of MMD to be predicted, for use in economic evaluations of preventive therapies.

Adherence to COPD treatment in Turkey and Saudi Arabia: results of the ADCARE study.

BackgroundCOPD affects millions of people worldwide. Poor treatment adherence contributes to increased symptom severity, morbidity and mortality. This study was designed to investigate adherence to COPD treatment in Turkey and Saudi Arabia.MethodsAn observational, cross-sectional study in adult COPD patients in Turkey and Saudi Arabia. Through physician-led interviews, data were collected on sociodemographics and disease history, including the impact of COPD on health status using the COPD Assessment Test (CAT); quality of life, using the EuroQol Five-Dimension questionnaire (EQ-5D); and anxiety and depression using the Hospital Anxiety and Depression Scale (HADS). Treatment adherence was measured using the 8-item Morisky Medication Adherence Scale (MMAS-8). Multivariate logistic regression analysis examined the predictors of non-adherence and the impact of adherence on symptom severity.ResultsFour hundred and five COPD patients participated: 199 in Turkey and 206 in Saudi Arabia. Overall, 49.2% reported low adherence (MMAS-8 <6). Of those, 74.7% reported high disease impact (CAT >15) compared to 58.4% reporting medium/high adherence (p=0.0008). Patients with low adherence reported a lower mean 3-level EQ-5D utility value (0.54±0.35) compared to those with medium/high adherence (0.64±0.30; p<0.0001). Depression with HADS score 8–10 or >10 was associated with lower adherence (OR 2.50 [95% CI: 1.43–4.39] and 2.43 [95% CI: 1.39–4.25], respectively; p=0.0008). Being a high school/college graduate was associated with better adherence compared with no high school (OR 0.57 [95% CI: 0.33–0.98] and 0.38 [95% CI: 0.15–1.00], respectively; p=0.0310). After adjusting for age, gender, and country, a significant association between treatment adherence (MMAS-8 score ≥6) and lower disease impact (CAT ≤15) was observed (OR 0.56 [95% CI: 0.33–0.95]; p=0.0314).ConclusionAdherence to COPD treatment is poor in Turkey and Saudi Arabia. Non-adherence to treatment is associated with higher disease impact and reduced quality of life. Depression, age, and level of education were independent determinants of adherence.