EQ-5D-5L Research Articles

Value of PASI90 Versus Merit-Based Incentive Payment System Efficacy Measures.

Achieving ≥90% improvement in Psoriasis Area and Severity Index (PASI90) is achievable with newer biologic therapies, such as ixekizumab. Standard of care payment systems such as the Merit-based Incentive Payment System (MIPS) responder criteria could lead to under treatment and lower quality of life (QoL) outcomes compared with PASI90. Show PASI90 is a higher standard than MIPS and is associated with greater improvements in QoL and other PRO outcomes. Patients with moderate-to-severe psoriasis meeting PASI90 and MIPS criteria were compared in 3 phase 3 clinical trials of the interleukin-17A inhibitor ixekizumab (pooled UNCOVER-2/3 and IXORA-S). Patients satisfying MIPS criteria met either static Physician Global Assessment score ≤2, body surface area <3%, PASI <3, or Dermatology Life Quality Index ≤5. Improvements in QoL were compared between patients meeting PASI90 and MIPS criteria. All PASI90 responders were also MIPS responders (PASI90 responders). Not all MIPS responders met PASI90 (MIPS-only responders). Significantly larger change from baseline improvements for all health (skin pain, Itch NRS, DLQI, PtGA, WPAI-PsO work productivity loss, and WPAI-PsO activity impairment) and quality of life (EQ-5D 5L VAS and acute SF-36 PCS/MCS) outcome measures were observed in the PASI90 responders vs the MIPS-only responders. PASI90 is a higher standard of response than MIPS and is associated with greater improvements in health and quality of life outcome measures.

Comparing the psychometric properties of the EQ-5D-3L and EQ-5D-5L descriptive systems and utilities in atopic dermatitis

BackgroundAtopic dermatitis (AD) is a common chronic inflammatory skin disorder affecting up to 10% of adults. The EQ-5D is the most commonly used generic preference-accompanied measure to generate quality-adjusted life years (QALYs) for economic evaluations.ObjectivesWe aimed to compare psychometric properties of the three-level and five-level EQ-5D (EQ-5D-3L and EQ-5D-5L) in adult patients with AD.MethodsIn a multicentre cross-sectional study, 218 AD patients with a broad range of severity completed the EQ-5D-3L, EQ-5D-5L, Dermatology Life Quality Index (DLQI) and Skindex-16. Disease severity outcomes included the Investigator Global Assessment, Eczema Area and Severity Index and the objective SCORing Atopic Dermatitis.ResultsA good agreement was established between the two EQ-5D versions with an intraclass correlation coefficient of 0.815 (95% CI 0.758–0.859, p < 0.001). Overall, 33 different health state profiles occurred in the EQ-5D-3L and 84 in the EQ-5D-5L. Compared to the EQ-5D-3L, ceiling effect was reduced for the mobility, self-care, usual activities and pain/discomfort dimensions by 4.6–11.5%. EQ-5D-5L showed higher average relative informativity (Shannon’s evenness index: 0.64 vs. 0.59). EQ-5D-5L demonstrated better convergent validity with EQ VAS, DLQI and Skindex-16. The two measures were similar in distinguishing between groups of patients based on disease severity and skin-specific quality of life with a moderate or large effect size (η2 = 0.083–0.489).ConclusionBoth instruments exhibited good psychometric properties in AD; however, the EQ-5D-5L was superior in terms of ceiling effects, informativity and convergent validity. We recommend the use of the EQ-5D-5L to measure health outcomes in clinical settings and for QALY calculations in AD.

Validity, reliability, and responsiveness of the Swedish version of Western Ontario Osteoarthritis of the Shoulder index

BackgroundThe Western Ontario of the Shoulder index (WOOS) is a patient-reported, disease-specific instrument, designed to measure quality of life in patients with osteoarthritis of the shoulder. The Swedish Shoulder Arthroplasty Registry (SSAR) uses WOOS and EuroQoL 5-dimensions 3 levels (EQ-5D-3L) as patient reported outcome measures. The purpose of this study was to test the validity, responsiveness, and reliability of the Swedish translation of WOOS for patients with osteoarthritis of the shoulder.MethodsData was collected from three shoulder arthroplasty studies performed during 2005–2013, with 23, 21, and 19 patients respectively. Forms were collected preoperatively, and postoperatively between 12 and 24 months. WOOS and EQ-5D-3L were used in all three studies. Additionally, the Oxford Shoulder Score (OSS) (n = 23) was used in one study, and the Constant-Murley score (CMS) (n = 40) in two of the studies. Validity was analysed by calculating Pearson’s correlation coefficient (PCC). Cronbach’s alpha (CA) was used to estimate internal consistency and reliability. The responsiveness of WOOS was measured by calculating effect size and standardized response mean. To assess the performance of WOOS over time, we present repeated measures of WOOS in the registry over a 10-year period.ResultsThe validity analysis showed excellent correlations of WOOS to CMS, OSS and EQ-5D 3L, with Pearson’s correlation coefficient of 0.72, 0.83, and 0.62 respectively (P < 0.001). There were adequate floor effects in the sport and lifestyle domains preoperatively, and adequate ceiling effects in all domains postoperatively. There were no floor effects and adequate ceiling effects for total WOOS. Analyzing reliability, Cronbach’s alpha was 0.95 for the pre- and postoperative WOOS scores combined. The analysis of responsiveness for WOOS showed an effect size of 2.52 and a standardized response mean of 1.43.The individual results measured by WOOS within the registry shows stable levels from 1 to 10 years.ConclusionThe Swedish translation of WOOS is valid, reliable, and responsive for use in a clinical setting for patients with glenohumeral osteoarthritis treated with shoulder arthroplasty, and we regard it as an appropriate instrument for use in the Swedish Shoulder Arthroplasty Registry.

Functional Outcomes among Geriatric Fragility Hip Fracture Patients in a Developing Country: A Comparative Study between Complete and Incomplete Post-operative Rehabilitation

Objective. The study compared functional outcomes among post-operative geriatric fragility hip fracture patients who received complete and incomplete rehabilitation.&#x0D; Methods. This is an ambispective cohort study of 50 acute fragility hip fractures over a 40-month period (October 2017 to November 2020) treated with either arthroplasty or internal fixation under the UP-PGH Orthogeriatric Fracture Liaison Service (FLS). Patients were contacted and interviewed through Telemedicine. They were asked to answer two questionnaires – the Modified Harris Hip Score (MHHS) and the EuroQol-5D-5L (EQ-5D-5L). The scores were tallied and used to describe and compare the post-operative functional outcomes between the two rehabilitation pathways.&#x0D; Results. Among the 50 patients included in this study, twenty-three (46%) patients underwent complete rehabilitation, while 27 (54%) underwent incomplete rehabilitation. The average corrected MHHS was at 82.5 suggesting good outcomes among all patients, with a higher-than-average outcome of 83.6 among patients who underwent complete rehabilitation, and an outcome of 75.9 among patients who underwent incomplete rehabilitation. Results to the EQ-5D—5L survey showed that a majority of patients who underwent complete rehabilitation reported having ‘no problems’ in terms of self-care, and anxiety or depression. However, the same group had more patients reporting ‘any problems’ in terms of mobility. On the other hand, a bigger proportion of patients from the incomplete rehabilitation group presented with ‘any problems’ in terms of usual activities. Proportions were similar for both groups in terms of pain or discomfort, with neither group having patients who reported extreme pain or discomfort.&#x0D; Conclusion. In spite of the heterogenous nature of the hip fracture population, functional outcome measures show generally good outcomes of patients under the UP-PGH Orthogeriatric FLS, with no significant difference among patients who receive complete rehabilitation from those who undergo incomplete rehabilitation. Continuing this study may better describe and differentiate the functional outcomes in order to pave the way for evidence-based protocols dedicated to providing the highest quality of care for acute fragility fracture patients.

An Analysis of 5-Level Version of EQ-5D Adjusting for Treatment Switching: The Case of Patients With Epidermal Growth Factor Receptor T790M-Positive Nonsmall Cell Lung Cancer Treated With Osimertinib

ObjectivesTreatment switching from control to treatment after disease progression is common in oncology trials. Analyses of survival data typically adjust for this bias, but such adjustments are rarely performed in analyses of patient-reported outcomes. This analysis aimed to examine the impact of adjusting for treatment switching on estimated treatment effects on 5-level version of EQ-5D (EQ-5D-5L) utilities and quality-adjusted life-years (QALYs). The AURA3 trial (NCT02151981) was a randomized controlled trial comparing osimertinib with platinum-based doublet chemotherapy (standard care) in patients with locally advanced or metastatic epidermal growth factor receptor mutant- and T790M-positive nonsmall cell lung cancer whose disease has progressed with previous epidermal growth factor receptor tyrosine kinase inhibitor therapy. MethodsDescriptive analyses were used to compare treatment arms. The primary analysis used a 2-stage least squares instrumental variable regression to estimate treatment effect adjusting for treatment crossover. Time to deterioration, defined from baseline to minimally important deterioration in EQ-5D-5L utility, was assessed using a rank preserving structural failure time model. ResultsIntention-to-treat analysis of imputed data showed incremental QALYs for osimertinib of 0.23 at 60 weeks. Accounting for treatment switching increased this to 0.52 in the primary analysis and to 0.63 QALYs in sensitivity analysis at 150 weeks. Time to deterioration analysis showed longer health-related quality of life maintenance with osimertinib, of 12.76 weeks, although this was at the borderline of statistical significance (acceleration factor, ψ = −0.275; 95% confidence interval −0.50 to 0.00). ConclusionsThis analysis demonstrates methods to adjust for treatment switching in the analysis of EQ-5D-5L from clinical trials. Failure to account for crossover substantially underestimated the QALY gain for osimertinib.

First population norms for the EQ-5D-3L in the Russian Federation.

PurposeThe EQ–5D survey instrument is routinely applied to general and patient specific populations in many countries, as a means of measuring Health Related Quality of Life (HRQOL) and/or informing Health Technology Assessment. The instrument is the subject of growing interest in the Russian Federation, as too is Health Technology Assessment. This research is the first to systematically present the EQ–5D–3L nationally representative population norms and to examine the socioeconomic and socio-demographic characteristics of the instrument among a representative sample of the Russian population.MethodsBased on a nationally representative health and well-being survey of the Russian population, conducted in November 2017, we establish the descriptive results, including the EQ-VAS and the EQ-5D Index, by age and gender, examine the correspondence between the EQ–5D health classifications and the separate EQ-VAS scores, and draw on a set of augmented logistic regressions to evaluate the association between the presence of problems in each dimension and various socio-economic and health-related characteristics.ResultsWe find strong evidence that the EQ-5D instrument is sensitive to underlying observed and latent health experiences, that it mirrors many of the characteristics familiar from other settings but that there are Russian specificities which merit further research, particularly with respect to the anxiety/depression dimension of the instrument.ConclusionThis research represents an important landmark for HRQOL studies in Russia as well as for the prospects of continuing to develop the scholarly and practical infrastructure necessary for Russian Health Technology Assessment to advance.

Impact of remission and low disease activity on health-related quality of life in patients with systemic lupus erythematosus.

To investigate the impact of remission and lupus low disease activity state (LLDAS) on health-related quality of life (HRQoL) in systemic lupus erythematosus. Short-Form 36 (SF-36), three-level EQ-5D (EQ-5D-3L)and Functional Assessment of Chronic Illness Therapy (FACIT)-Fatiguedata from the BLISS-52 (NCT00424476) and BLISS-76 (NCT00410384) trials were used. Duration in remission/LLDAS required to reach a HRQoL benefit ≥ minimal clinically important differences (MCIDs) during and post-treatment was determined using quantile regression and generalized estimating equations. Patients (n = 1684) were assessed every fourth week (15 visits). Four cumulative (β = 0.60) or four consecutive (β = 0.66) visits in remission were required to achieve a benefit ≥MCID in SF-36 physical component summary (PCS) scores, and six cumulative (β = 0.44) or five consecutive (β = 0.49) for a benefit ≥MCID in mental component summary (MCS) scores. Eight cumulative (β = 0.30 for both) or eight consecutive (β = 0.32 for both) visits in LLDAS were required for a benefit in PCS/MCS ≥MCID, respectively. For EQ-5D-3L index scores ≥MCID, six cumulative (β = 0.007) or five consecutive (β = 0.008) visits in remission were required, and eight cumulative (β = 0.005) or six consecutive (β = 0.006) visits in LLDAS. For FACIT-Fatigue scores ≥MCID, 12 cumulative (β = 0.34) or 10 consecutive (β = 0.39) visits in remission were required, and 17 cumulative (β = 0.24) or 16 consecutive (β = 0.25) visits in LLDAS. Remission and LLDAS contribute to a HRQoL benefit in a time-dependent manner. Shorter time in remission than in LLDAS was required for a clinically important benefit in HRQoL, and longer time in remission for a benefit in mental compared with physical HRQoL aspects. When remission/LLDAS was sustained, the same benefit was achieved in a shorter time.

Predicting Patient-Level 3-Level Version of EQ-5D Index Scores From a Large International Database Using Machine Learning and Regression Methods

ObjectivesThis study aimed to evaluate the performance of machine learning and regression methods in the prediction of 3-level version of EQ-5D (EQ-5D-3L) index scores from a large diverse data set. MethodsA total of 30 studies from 3 countries were combined. Predictions were performed via eXtreme Gradient Boosting classification (XGBC), eXtreme Gradient Boosting regression (XGBR) and ordinary least squares (OLS) regression using 10-fold cross-validation and 80%/20% partition for training and testing. We evaluated 6 prediction scenarios using 3 samples (general population, patients, total) and 2 predictor sets: demographic and disease-related variables with/without patient-reported outcomes. Model performance was evaluated by mean absolute error and percent of predictions within clinically irrelevant error range and within correct health severity group (EQ-5D-3L index <0.45, 0.45-0.926, >0.926). ResultsThe data set involved 26 318 individuals (clinical settings n = 6214, general population n = 20 104) and 26 predictor variables plus diagnoses. Using all predictors and the total sample, mean absolute error values were 0.153, 0.126, and 0.131, percent of predictions within clinically irrelevant error range were 47.6%, 39.5%, and 37.4%, and within the correct health severity group were 56.3%, 64.9%, and 63.3% by XGBC, XGBR, and OLS, respectively. The performance of models depended on the applied evaluation criteria, the target population, the included predictors, and the EQ-5D-3L index score range. ConclusionsRegression models (XGBR and OLS) outperformed XGBC, yet prediction errors were outside the clinically irrelevant error range for most respondents. Our results highlight the importance of systematic patient-reported outcome (EQ-5D) data collection. Dialogs between artificial intelligence and outcomes research experts are encouraged to enhance the value of accumulating data in health systems.

A Comparison of a Preliminary Version of the EQ-HWB Short and the 5-Level Version EQ-5D

ObjectivesThe EQ Health and Wellbeing Short (EQ-HWB-S) is a new broad generic measure of health and wellbeing for use in economic evaluations of interventions across healthcare, social care, and public health. This measure conceptually overlaps with the 5-level version EQ-5D (EQ-5D-5L), while expanding on the coverage of health and social care related dimensions. This study aims to examine the extent to which the EQ-HWB-S and EQ-5D-5L overlap and are different. MethodsA sample of US-based respondents (n = 903; n = 400 cancer survivors and n = 503 general population) completed a survey administered via an online panel. The survey included the EQ-HWB item pool (62 items, including 11 items used in this analysis), EQ-5D-5L, and questions about sociodemographic and health characteristics. The analysis included (Spearman’s) correlations, the comparison of patterns of response (distributions and ceiling effects), and the ability to discriminate between known groups. ResultsModerate to strong associations were found between conceptually overlapping dimensions of the EQ-5D-5L and the EQ-HWB-S (rs > 0.5, P < .001). Among respondents reporting full health on the EQ-5D-5L (n = 161, 18.23%), the EQ-HWB-S identified ceiling effects, particularly with the item “feeling exhausted.” Most EQ-5D-5L and EQ-HWB-S items demonstrated discriminative ability among those with and without physical and mental conditions, yielding medium (> 0.5) to large effect sizes (> 0.8). Nevertheless, only EQ-HWB-S items distinguished between caregivers and noncaregivers and those with low and high caregiver burden, albeit with small effect sizes (0.2-0.5). ConclusionsResults indicate a convergence between the measures, especially between overlapping dimensions, lending support to the validity of the EQ-HWB-S. The EQ-HWB-S performed similarly or better than the EQ-5D-5L among patient groups and is better able to differentiate among caregivers and respondents closer to full health.

Suicidality and Quality of Life in Treatment-Resistant Depression Patients in Latin America: Secondary Interim Analysis of the TRAL Study

BackgroundA large proportion of patients with major depressive disorder (MDD) have treatment-resistant depression (TRD). The TRAL study examines the impact of TRD on suicidality and health-related quality of life (HRQoL) among MDD patients in 4 Latin American countries.MethodsIn this multicenter, prospective, observational study, MDD patients were recruited from 33 sites in Mexico, Colombia, Brazil, and Argentina. Patients were assessed for TRD, defined as failure to respond to ≥2 antidepressant medications of adequate dose and duration. Other assessments included current disease status, Mini International Neuropsychiatric Interview (MINI), Columbia-Suicide Severity Rating Scale (C-SSRS), 5 Level EQ-5D (EQ-5D-5L), Patient Health Questionnaire-9 (PHQ-9), and Sheehan Disability Scale (SDS).Results1,475 MDD patients were included in the analysis (mean age, 45.6 years; 78% women), and 429 met criteria for TRD. Thoughts of suicide and suicide attempts were more common among TRD patients (38.7%) compared with non-TRD patients (24.9%; P < 0.0001), according to the current disease status questionnaire. The C-SSRS showed that lifetime suicidal behavior was significantly more common among TRD patients than non-TRD patients (13.8 vs. 10.0%; P = 0.0384). Compared with non-TRD patients, TRD patients showed significantly greater adverse impacts on QoL (EQ-5D-5L), more severe depression (PHQ-9), and greater functional impairment (SDS).ConclusionTRD patients in clinical sites from Mexico, Colombia, Brazil, and Argentina were more likely to experience suicidality and negative effects on HRQoL than non-TRD patients.

Post-Migration Stressors and Health-Related Quality of Life in Refugees from Syria Resettled in Sweden.

The link between post-migration stressors and mental ill health is well documented in refugees resettled in high-income host countries, but the consequences of these stressors on refugees’ health-related quality of life (HRQoL) are less known. This study examined the association between post-migration stressors and HRQoL among Syrian adult refugees resettled in Sweden using a preference-based value set obtained from the general Swedish population. A total of 1215 Syrian adults, ages 18–64 years, granted residency in Sweden, responded to a postal questionnaire in 2016 regarding various aspects of their resettlement. The European Quality of Life Five Dimensions Five Level (EQ–5D–5L) questionnaire was used to assess HRQoL through an EQ–5D–5L index score (range; 0=dead to 1=full health). The index score was preference weighted using a Swedish population value set. Predictors were four self-reported post-migration stressors related to daily living in the host country: financial strain, social strain, competency strain and perceived discrimination divided into low, medium and high levels of experienced stress. Multivariable linear regression models were employed to assess the association between post-migration stressors and HRQoL index score, adjusting for potentially traumatic events in the pre- and peri-migration phase as well as sociodemographic confounders/covariates (sex, age, education, civil status, immigration year). The Syrian refugees had a mean EQ–5D–5L index score of 0.863 (SD = 0.145). There was strong evidence of a negative dose-response association in both unadjusted and adjusted models between HRQoL and the post-migration stressors financial strain and social strain—i.e., there was a stepwise, and statistically significant, decrease in HRQoL when going from low to medium to high strain. Competency strain and discrimination were only associated with lower HRQoL when experienced at high levels in fully adjusted models. High exposure to potentially traumatic experiences before or during flight was also associated with lower HRQoL. Syrian refugees resettled in Sweden reported a lower HRQoL than the general Swedish population and lower than age-matched Swedish adults. The present study results point to the possible adverse effects of post-migration stressors on HRQoL.

The Development of a New International Generic Measure (EQ-HWB): Face Validity and Psychometric Stages in Argentina

ObjectivesThis study aimed to present the face validity and psychometric stages performed in Spanish in Argentina, the only Spanish-speaking country of an international collaboration that undertook the construction of a new measure that can be used in economic evaluation across health, social care, and public health, the EQ EQ-HWB (EQ Health and Wellbeing). We also explored the relationship among 3-level version EQ-5D (EQ-5D-3L), 5-level version EQ-5D (EQ-5D-5L), and EQ-HWB. MethodsFace validity was based on semistructured face to face interviews of a purposive sample to explore translatability of language and concepts of 97 candidate items, translated into Argentina Spanish. The psychometric evaluation using an online panel assessed the psychometric properties of 64 items that were carried forward (floor and ceiling effects, item correlations, known-group differences in relevant prespecified subgroups by the international and local teams, exploratory and confirmatory factor analysis, and item response theory). EQ-5D-3L, EQ-5D-5L, and EQ-HWB correlations were explored. ResultsIn the face validity stage, 24 interviews with carers, general public, patients, and users of social services were included. Most items showed adequate face validity. In the psychometric assessment, 497 participants were recruited (64% reporting a long-term health condition). Most of the items showed adequate psychometrics in an Argentinian context. EQ-5D-3L and EQ-5D-5L had strong correlations, and EQ-HWB was moderately correlated to EQ visual analog scale. The Argentina team recommended 23 of the final 25 items. ConclusionsThe assessment of Spanish items contributed to the overall development of EQ-HWB and helped inform the design of an internationally relevant 25-item and a short 9-item measure intended to be used in economic evaluations.

Health-Related Quality of Life and Utility Scores of Lung Cancer Patients Treated with Traditional Chinese Medicine in China.

PurposeTo assess health-related quality of life (HRQoL) and utility scores of lung cancer patients treated with traditional Chinese medicine (TCM) in China.MethodsThis cross-sectional study included lung cancer patients treated with TCM in seven tertiary hospitals in Shanghai, China. The HRQoL and utility scores of these patients were measured using the five-level EQ-5D (EQ-5D-5L). The EQ-5D-5L utility scores were derived from the Chinese EQ-5D-5L Value Set. The relationships between HRQoL and the socio-demographic and clinical characteristics of these patients were further explored by Tobit regression.ResultsThis study included a total of 347 patients. Their mean ± SD and median EQ-5D-5L utility scores were 0.851 ± 0.198 and 0.893, respectively. The highest proportion of participants reporting problems was observed in pain/discomfort dimension (57.9%) and anxiety/depression (45.5%). Lung cancer patients treated with TCM had poor HRQoL, influenced by cancer clinical stage.ConclusionLung cancer patients treated with TCM have poor HRQoL, with many patients reporting problems in the pain/discomfort and anxiety/depression dimensions. The information on health utility scores and HRQoL of lung cancer patients treated with TCM could be useful for future supportive care, economic evaluations and decision-making in China.

Development of an EQ-5D Value Set for India Using an Extended Design (DEVINE) Study: The Indian 5-Level Version EQ-5D Value Set

ObjectivesThis study aimed to develop the Indian 5-level version EQ-5D (EQ-5D-5L) value set, which is a key input in health technology assessment for resource allocation in healthcare. MethodsA cross-sectional survey using the EuroQol Group’s Valuation Technology was undertaken in a representative sample of 3548 adult respondents, selected from 5 different states of India using a multistage stratified random sampling technique. The participants were interviewed using a computer-assisted personal interviewing technique. This study adopted a novel extended EuroQol Group’s Valuation Technology design that included 18 blocks of 10 composite time trade-off (c-TTO) tasks, comprising 150 unique health states, and 36 blocks of 7 discrete choice experiment (DCE) tasks, comprising 252 DCE pairs. Different models were explored for their predictive performance. Hybrid modeling approach using both c-TTO and DCE data was used to estimate the value set. ResultsA total of 2409 interviews were included in the analysis. The hybrid heteroscedastic model with censoring at −1 combining c-TTO and DCE data yielded the most consistent results and was used for the generation of the value set. The predicted values for all 3125 health states ranged from −0.923 to 1. The preference values were most affected by the pain/discomfort dimension. ConclusionsThis is the largest EQ-5D-5L valuation study conducted so far in the world. The Indian EQ-5D-5L value set will promote the effective conduct of health technology assessment studies in India, thereby generating credible evidence for efficient resource use in healthcare.

Clinical outcome analysis of patients with autism spectrum disorder: analysis from the UK Medical Cannabis Registry.

Introduction:Cannabis-based medicinal products (CBMPs) have been identified as a promising novel therapeutic for symptoms and comorbidities related to autism spectrum disorder (ASD). However, there is a paucity of clinical evidence of their efficacy and safety. Objective: This case series aims to assess changes to health-related quality of life and the incidence of adverse events in patients treated with CBMPs for associated symptoms of ASD enrolled on the UK Medical Cannabis Registry (UKMCR).Methods:Patients treated with CBMPs for ASD-related symptoms for a minimum of 1 month were identified from the UKMCR. Primary outcomes were changes in validated patient-reported outcome measures [Generalised Anxiety Disorder-7 (GAD-7), Single-Item Sleep Quality Scale (SQS), 5-level version of the EQ-5D (EQ-5D-5L) index values] at 1, 3 and 6 months compared with baseline. Adverse events were recorded and analysed. Statistical significance was determined by p < 0.050.Results:Seventy-four patients with ASD were included in the analysis. The mean age of participants was 32.7 (±11.6) years. There were significant improvements in general health-related quality of life and sleep as assessed by the EQ-5D-5L, SQS and GAD-7 at 1 and 3 months, with sustained changes in EQ-5D-5L and SQS at 6 months (p < 0.010). There were 180 (243.2%) adverse events reported by 14 (18.9%) participants. If present, adverse events were commonly mild (n = 58; 78.4%) or moderate (n = 81; 109.5%), rather than severe (n = 41; 55.4%).Conclusion:This study demonstrated an associated improvement in general health-related quality of life, and anxiety- and sleep-specific symptoms following initiation of treatment with CBMPs in patients with ASD. These findings, while promising, are limited by the confines of the study which lacks a control arm and is subject to attrition bias. Therefore, further evaluation is required with randomised controlled trials.

Mega-OATS of the knee without specialised instrumentation: a low-cost option for large cartilage defects in a resource-restrained environment

BACKGROUND: A 26-year-old patient presented to a specialised knee clinic in a public hospital with ongoing pain after having sustained a soccer injury six years prior. A large osteochondral defect of the distal medial femoral condyle was diagnosed. Due to resource limitations, fresh allograft or a large osteochondral autograft transplantation system (Mega-OATS) workbench was unavailable. CASE REPORT: A Mega-OATS cartilage transplantation was done, using the patient's posteromedial femoral condyle as donor tissue, and transplanted to the defect in the distal femoral condyle, a technique that has been well documented and followed up. At six weeks postoperatively, an MRI showed early incorporation of the graft tissue. Clinical outcomes were excellent at one year follow-up with the EQ-5D 5L score 11111, the Knee Injury and Osteoarthritis Outcome Score (KOOS-PS) 100%, and the Lysholm score also 100%. Radiographs at one year confirmed an unchanged graft position and showed no signs of osteoarthritis. DISCUSSION: Large osteochondral lesions in the knee (&gt; 4 cm2) are challenging to treat, and the most commonly used modalities are fresh osteochondral allograft (OCA) or autologous chondrocyte implantation (ACI). Mega-OATS of the knee has previously been described but is not commonly used due to the requirement of a specialised and expensive workbench, and fear of morbidity at the donor site. CONCLUSION: Mega-OATS of the knee is possible without a specialised workbench or tools and had good clinical outcomes at two-year follow-up of the patient. Level of evidence: Level 5

Classification of Adverse Events Following Surgery in Patients With Diffuse Lower-Grade Gliomas.

BackgroundRecently, the Therapy-Disability-Neurology (TDN) was introduced as a multidimensional reporting system to detect adverse events in neurosurgery. The aim of this study was to compare the novel TDN score with the Landriel–Ibanez classification (LIC) grade in a large cohort of patients with diffuse lower-grade glioma (dLGG). Since the TDN score lacks validation against patient-reported outcomes, we described health-related quality of life (HRQoL) change in relation to TDN scores in a subset of patients.MethodsWe screened adult patients with a surgically treated dLGG World Health Organization (WHO) grade 2 and 3 between 2010 and 2020. Up until 2017, it consists of a retrospective cohort (n = 158). From 2017 and onwards, HRQoL was registered using EuroQoL-5-dimension, three levels of response (EQ-5D 3L) questionnaire at baseline and 3 months follow-up, in a prospectively recruited cohort (n = 102). Both the LIC grade and TDN score were used to classify adverse events.ResultsIn total, 231 patients were included. In 110/231 (47.6%) of the surgical procedures, a postoperative complication was registered. When comparing the TDN score to LIC grades, only a minor shift towards complications of higher order could be observed. EQ-5D 3L was reported for 45 patients. Patients with complications related to surgery had pre- to postoperative changes in EQ-5D 3L index values (n = 27; mean 0.03, 95% CI −0.06 to 0.11) that were comparable to patients without complications (n = 18; mean −0.06, 95% CI −0.21 to 0.08). In contrast, patients with new-onset neurological deficit had a deterioration in HRQoL at follow-up, with a mean change in the EQ-5D 3L index value of 0.11 (n = 13, 95% CI 0.0 to 0.22) compared to −0.06 (n = 32, 95% CI −0.15 to 0.03) for all other patients.ConclusionsIn patients with dLGG, TDN scores compared to the standard LIC tend to capture more adverse events of higher order. There was no clear relation between TDN severity and HRQoL. However, new-onset neurological deficit caused impairment in HRQoL. For the TDN score to better align with patient-reported outcomes, more emphasis on neurological deficit and function should be considered.

Comparison of the PROMIS Preference Score (PROPr) and EQ-5D-5L Index Value in General Population Samples in the United Kingdom, France, and Germany

ObjectivesThe Patient-Reported Outcome Measurement Information System (PROMIS) Preference score (PROPr) can be used to assess health state utility (HSU) and estimate quality-adjusted life-years in cost-effectiveness analyses. It is based on item response theory and promises to overcome limitations of existing HSU scores such as ceiling effects. The PROPr contains 7 PROMIS domains: cognitive abilities, depression, fatigue, pain, physical function, sleep disturbance, and ability to participate in social roles and activities. We aimed to compare the PROPr with the 5-level EQ-5D (EQ-5D-5L) in terms of psychometric properties using data from 3 countries. MethodsWe collected PROMIS-29 profile and EQ-5D-5L data from 3 general population samples (United Kingdom = 1509, France = 1501, Germany = 1502). Given that cognition is not assessed by the PROMIS-29, it was predicted by the recommended linear regression model. We compared the convergent validity, known-groups construct validity, and ceiling and floor effects of the PROPr and EQ-5D-5L. ResultsThe mean PROPr (0.48, 0.53, 0.48; P<.01) and EQ-5D-5L scores (0.82, 0.85, 0.83; P<.01) showed significant differences of similar magnitudes (d = 0.34; d = 0.32; d = 0.35; P<.01) across all samples. The differences were invariant to sex, income, occupation, education, and most conditions but not for age. The Pearson correlation coefficients between both scores were r = 0.74, r = 0.69, and r = 0.72. PROPr’s ceiling and floor effects both were minor to moderate. The EQ-5D-5L’s ceiling (floor) effects were major (negligible). ConclusionsBoth the EQ-5D-5L and the PROPr assessed by the PROMIS-29 show high validity. The PROPr yields considerably lower HSU values than the EQ-5D-5L. Consequences for quality-adjusted life-year measurements should be investigated in future research.

OP443 Evaluating The Value Of Endovascular Innovations For Aortic Valve Replacement Through Clinical Benefits, Patient-reported Outcomes And Resource Consumption.

IntroductionThe use of most recent Transcatheter aortic valve implants (TAVI) in the treatment of symptomatic severe aortic stenosis (SAS) is evolving with expanded indications from inoperable/high-risk to intermediate and low risk patients. Consequently, TAVI outcomes must be monitored to highlight its value under real-world conditions. Our aim was to prospectively evaluate TAVI (SAPIEN 3) outcomes in terms of patient's health-related quality of life (HRQoL), clinical outcomes, and healthcare resource utilization (HRU).MethodsAn observational prospective study including all consecutive patients with SAS undergoing a transcatheter valve implantation with Edwards SAPIEN 3 valve (transfemoral access) was conducted in full accordance with clinical guidelines from the European Society of Cardiology. Patients were evaluated before the intervention (baseline), at discharge, and after one, six and twelve months from the implant. A thoughtful and systematic evaluation of patients’ HRQoL (EQ-5D 5L, the Short Form-36 Health Survey -SF-36- and the Kansas City Cardiomyopathy Questionnaire -KCCQ-), clinical endpoints (that is, cardiovascular mortality, and rates of stroke, major bleeding, myocardial infarction, and re-hospitalization), echocardiographic measurements, and HRU (that is, Length of stay-LOS- in ward/intensive care unit -ICU-) was implemented. Multivariate regression models were applied to test outcomes while controlling key risk factors (that is, patient’ severity at baseline).ResultsA total of seventy-six patients (fifty percent female, fifty-five percent of intermediate-high risk) with a mean age of 82.1 ± 4.78 years were included. Implant success was 97.34% and cardiovascular death was 2.6% at one year. Significant reductions in mean and maximum gradients were achieved and maintained during follow-up. Mean LOS in ward (5.2 ± 4.0days) and ICU (0.22 ± 0.64 days) were low. Statistically significant improvements were detected in the KCCQ overall summary scores, EQ-5D, and SF-36 (Physical component summary) - all adjusted - p &lt; 0.05 - after the intervention.ConclusionsTAVI represents a safe and effective innovation for SAS with clinical benefits translated into significant improvements in terms of HRQoL. Besides, the low HRU provides new insights for health-economic modelling and the optimization of limited resources of special importance under current pandemic situation.

Investigating 5-Level EQ-5D (EQ-5D-5L) Values Based on Preferences of Patients With Heart Disease

ObjectivesSeveral studies have shown that patients with heart disease value hypothetical health states differently from the general population. We aimed to investigate the health preferences of patients with heart disease and develop a value set for the 5-level EQ-5D (EQ-5D-5L) based on these patient preferences. MethodsPatients with confirmed heart disease were recruited from 2 hospitals in Singapore. A total of 86 EQ-5D-5L health states (10 per patient) were valued using a composite time trade-off method according to the international valuation protocol for EQ-5D-5L; 20-parameter linear models and 8-parameter cross-attribute level effects models with and without an N45 term (indicating whether any health state dimension at level 4 or 5 existed) were estimated. Each model included patient-specific random intercepts. Model performance was evaluated for out-of-sample and in-sample predictive accuracy in terms of root mean square error. The discriminative ability of the utility values was assessed using heart disease-related functional classes. ResultsA total of 576 patients were included in the analysis. The preferred model, with the lowest out-of-sample root mean square error, was a 20-parameter linear model including N45. Predicted utility values ranged from −0.727 for the worst state to 1 for full health; the value for the second-best state was 0.981. Utility values demonstrated good discriminative ability in differentiating among patients of varied functional classes. ConclusionsAn EQ-5D-5L value set representing the preferences of patients with heart disease was developed. The value set could be used for patient-centric economic evaluation and health-related quality of life assessment for patients with heart disease.