EQ-5D-5L 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.

Оценка эффективности и безопасности применения медицинского изделия «МАВИТ» у пациентов с хроническим простатитом и синдромом хронической тазовой боли

Abstract. Recent studies have shown that chronic prostatitis is one of the most common clinically and socially significant diseases. With chronic prostatitis, mental health suffers no less than with other severe somatic diseases, which sharply reduces the quality of life of men. This was a serious incentive for this study. In outpatient conditions, 50 patients with a confirmed diagnosis of N41 were examined. 1 Chronic prostatitis; R10.2 Pelvic and perineal pain. At the stage of inclusion in the study and treatment, the patient is examined: 1) Study of anamnesis. (NIH-CPS; IPSS; IIEF-5; PHQ-9; GAD-7; EQ–5D–3L) 2) Physical examination (determination of the somatic status of the study subjects) 3) Laboratory examinations were carried out in the laboratories of the clinical bases of the Consultative and Diagnostic Center No2 of the Moscow Health Department, Moscow, 8 Igralnaya Street; Clinical base Central Medical Clinic CMD, Moscow, Perovo metro station, Novogireevskaya st., 3A; Clinical base Clinic «New Medicine», Moscow region, Orekhovo-Zuevo, Lenin st., 44. (urine culture for microflora or urine assessment with a test strip once - before the start of therapy, four-cup test, PSA, CRP, STI screening) 4). Specialized diagnostic tests (ultrasound of the bladder with determination of the volume of residual urine, uroflowmetry, TRUS of the prostate with Doppler mapping). GE LOGIQ V2 ultrasound machine with convex and rectal probe. The obtained research data and interpretation of statistical data allow us to conclude that it is possible to use the MAVIT device according to GIKS.941549.102 TU, manufactured by Yelatma Instrument Plant JSC, according to the selected methods, 7 procedures every 2 months in urological patients with chronic prostatitis and the presence of chronic pelvic pain syndrome in order to relieve pain symptoms, lower urinary tract symptoms. The study also found positive effects on men’s sexual function and a reduction in anxiety disorders. All these positive outcomes in relation to the treatment of the main group of patients allow us to conclude that the MAVIT device may be widely used in the work of a practitioner both on an outpatient basis and in a day hospital.

YOUNGER HYPERTENSIVE PATIENTS HAVE LOWER HEALTH-RELATED QUALITY OF LIFE AND DIFFERENT HYPERTENSION CORRELATES. EVIDENCE FROM BULGARIA

The purpose of this study was to determine hypertension correlates among the patients' survey characteristics after splitting the sample into two groups based on their age and perform this assessment separately for each of the groups. Materials and methods. A cross-sectional retrospective study among 647 patients was conducted using self-administered EQ-5D 3L, as well as demographic, economic and health questions. Results. The overall self-reported arterial hypertension (AH) prevalence was 36.9%. The sample was split into two groups: 18-54 and 55+ years, with AH prevalence of 23.5% and 51%, respectively. The differences between AH and non-AH patients in each age group were explored. Younger AH patients significantly more often reported fatigue, headache, chest pain and having at least 1 acute condition for the last year than non-hypertensive ones at the same age. Diabetes, asthma/chronic bronchitis and joint pain/arthritis were also more common among younger AH patients. The overall HRQol and self-perceived health were significantly lower among young hypertensive patients, and their median severity index was higher compared to non-hypertensive adults of the same age. They more often experienced moderate pain/discomfort and moderate anxiety/depression. Despite of this, older AH patients reported similar HRQoL compared to non-AH patients. Conclusions. Different comorbidities were found amongst younger and older hypertensive patients. Most of the already known AH correlates were confirmed only among younger adults (18-54 years old). Arterial hypertension leads to significantly lower health-related quality of life, self-perceived health and higher severity index only among the younger group, mainly due to the depression and pain they experience.

Sedentary behavior and associated factors on admissions to internal medicine wards.

The relationship between sedentary lifestyle and chronic diseases is well known. This study examined the prevalence and factors associated with reduced physical activity (PA) among internal medicine inpatients on admission. In this single-center, cross-sectional study, inpatients aged 50years or older were prospectively enrolled at a tertiary care facility in Ankara, Türkiye. PA was assessed using the International Physical Activity Questionnaire (IPAQ). Care and performance indicators, quality of life (EQ-5D 3L), nutritional status, timed up-and-go test, muscle strength, and cognitive status were assessed. Participants were classified into 3 groups of PA levels as low, moderate, and high. Study end points were the prevalence of low PA level and associated factors. Of the 240 participants (mean age: 62.7 ± 8.0years; women: 50%), 47.1% (n = 113), 40.8% (n = 98) and 12.1% (n = 29) had low, moderate, and high PA, respectively. Type 2 diabetes mellitus (45.1%), hypertension (66.4%), coronary artery disease (41.6%), dementia (8.8%), and multimorbidity (53.1%) were more common in the low PA group. Outdoor walking < 3days per week (OR: 4.44, 95% CI 1.55 to 12.74, p = 0.006, functional dependence in and outside home (OR: 4.25, 95% CI 1.13 to 15.92, p = 0.032) and EQ-5D VAS score (OR: 0.97, 95% CI 0.95 to 0.99, p = 0.011) were independently associated with low PA level on multivariable logistic regression analysis. This study found low or medium levels of PA in almost nine out of ten admissions to an internal medicine clinic. On the other hand, low PA level was not associated with most classical comorbidities but with altered performance and care indicators.

Pain control and functional recovery as therapeutic goals in patients with chronic musculoskeletal pain: two experiences with tapentadol hydrochloride.

This study aimed to evaluate pain control, functioning, and quality of life (QoL) recovery in patients with chronic low back pain (cLBP) or post-traumatic osteoarthritis (OA) pain in the ankle/foot area, treated with tapentadol prolonged release and unresponsive to other treatments. Two observational retrospective studies were conducted using clinical practice datasets of patients with chronic pain in cLBP and OA foot/ankle at different time points (total follow-up=60-90 days). The studies assessed pain intensity by the Numerical Rating Scale (NRS) pain scale (patients were classified as responder in case of ≥30% pain reduction), QoL by the 5-level EQ-5D (EQ-5D-5L) questionnaire, patient satisfaction by the 7-point Patients' Global Impression of Change (PGIC) scale; cLBP health status by the Roland Morris Disability Questionnaire (RMDQ); foot and ankle functional status by European Foot and Ankle Society (EFAS) score; and treatment-related AEs. For the cLBP setting, 37 patients were enrolled, of which 86.50% were classified as responders (n=32; CI: 75.5% ÷ 97.5%). For the foot/ankle OA pain setting, 21 patients were enrolled. Pain assessment at final follow-up was available only for 11 patients, of which 72.73% (n=8; CI: 39.0% ÷ 94.0%) were classified as responders. Statistically significant improvements were seen in the RMDQ, EQ-5D-5L, and PGIC scores in cLBP. Improvements in the EFAS, EQ-5D-5L, and PGIC scores were seen in OA as well. The incidence of treatment-related adverse reactions was low in both studies. In the study population, tapentadol prolonged release was effective and well tolerated in treating cLBP and post-traumatic foot/ankle OA chronic pain when used in a multimodal manner. The reduction in pain was accompanied by clinically relevant improvements in patients' functionality and QoL.

Suicidal ideation in Chinese patients with advanced breast cancer: a multi-center mediation model study

PurposeThe pathways underpinning suicide ideation (SI) and certain physical and psychological factors in patients with advanced breast cancer remain unclear. This study develops and validates a mediation model that delineates the associations between several multidimensional variables and SI in Chinese patients with advanced breast cancer.MethodsPatients with advanced breast cancer (n = 509) were recruited as study participants from 10 regional cancer centers across China from August 2019 to December 2020. Participants were required to complete five questionnaires using an electronic patient-reported outcomes (ePRO) system: 9 item- Patient Health Questionnaire (PHQ-9), Hospital Anxiety and Depression Scale (HADS), Insomnia Severity Index (ISI), 5-level EQ-5D (EQ-5D-5L), and MD Anderson Symptom Inventory (MDASI). Risk factors for SI were identified using multivariable logistic regression, and inputted into serial multiple mediation models to elucidate the pathways linking the risk factors to SI.ResultsSI prevalence was 22.8% (116/509). After adjusting for covariates, depression (odds ratio [OR] = 1.384), emotional distress (OR = 1.107), upset (OR = 0.842), and forgetfulness (OR = 1.236) were identified as significant independent risk factors (all p < 0.05). The ORs indicate that depression and distress have the strongest associations with SI. Health status has a significant indirect effect (OR=-0.044, p = 0.005) and a strong total effect (OR=-0.485, p < 0.001) on SI, mediated by insomnia severity and emotional distress.ConclusionsThere is a high SI prevalence among Chinese patients with advanced breast cancer. Our analysis revealed predictive pathways from poor health to heightened SI, mediated by emotional distress and insomnia. Regular management of distress and insomnia can decrease suicide risk in this vulnerable population.

Measurement properties of the EQ-5D-3L, EQ-5D-5L, and SF-6Dv2 in patients with late-onset Pompe disease.

The objective of this study was to evaluate the psychometric properties of the EQ-5D (3L and 5L) and SF-6Dv2 in a group of Chinese patients with late-on Pompe disease (PD), and compare their performance in this patient group. The data used in this study were obtained from a web-based and cross-sectional survey conducted in China. All participants completed the 3L, 5L, and SF-6Dv2. Information about their sociodemographic status and health conditions was also collected. The measurement properties were assessed by examining ceiling and floor effects, evaluating convergent validity, known-group validity, and test-retest reliability (Intraclass correlation coefficient [ICC] and Gwet's AC). A total of 117 PD patients completed the questionnaire. All dimensions of the 3L showed strong ceiling effects, ranging between 17.1 and 42.7%. All three measures showed good test-retest reliability, with ICC values ranging from 0.85 to 0.87. The Gwet's AC values showed that four out of five dimensions of the 3L showed very good agreement. All hypothesized correlations between the 3L, 5L, SF-6Dv2, and items of WHODAS were supported, indicating satisfactory convergent validity. The 5L showed stronger correlations (|r|= 0.53-0.84) with WHODAS than the other two measures. The outcomes of ANOVA indicated that the 5L had higher F-statistics than the 3L and SF-6Dv2, indicating a stronger discriminant ability to differentiate most condition groups. The 5L demonstrates lower ceiling and floor effects, higher discriminant ability, and better convergent validity than the SF-6Dv2 and 3L in patients with PD. In addition, the 5L may generate a larger utility gain compared to the other two instruments when conducting cost-effectiveness analysis.

Multimorbidity patterns and health-related quality of life among community-dwelling older adults: evidence from a rural town in Suzhou, China.

The high prevalence of multimorbidity in aging societies has posed tremendous challenges to the healthcare system. The aim of our study was to comprehensively assess the association of multimorbidity patterns and health-related quality of life (HRQOL) among rural Chinese older adults. This was a cross-sectional study. Data from 4,579 community-dwelling older adults aged 60years and above was collected by the clinical examination and questionnaire survey. Information on 10 chronic conditions was collected and the 3-Level EQ-5D (EQ-5D-3L) was adopted to measure the HRQOL of older adults. An exploratory factor analysis was performed to determine multimorbidity patterns. Regression models were fitted to explore the associations of multimorbidity patterns with specific health dimensions and overall HRQOL. A total of 2,503 (54.7%) participants suffered from multimorbidity, and they reported lower HRQOL compared to those without multimorbidity. Three kinds of multimorbidity patterns were identified including cardiovascular-metabolic diseases, psycho-cognitive diseases and organic diseases. The associations between psycho-cognitive diseases/organic diseases and overall HRQOL assessed by EQ-5D-3L index score were found to be significant (β = -0.097, 95% CI -0.110, -0.084; β = -0.030, 95% CI -0.038, -0.021, respectively), and psycho-cognitive diseases affected more health dimensions. The impact of cardiovascular-metabolic diseases on HRQOL was largely non-significant. Multimorbidity was negatively associated with HRQOL among older adults from rural China. The presence of the psycho-cognitive diseases pattern or the organic diseases pattern contributed to worse HRQOL. The remarkable negative impact of psycho-cognitive diseases on HRQOL necessiates more attention and relevant medical assistance to older rural adults.

Quality of Life and Treatment Modalities in Patients with Interstitial Cystitis: The Patients' Perspective.

Quality of life (QoL)-based outcomes are hardly incorporated into interstitial cystitis/bladder pain syndrome (IC/BPS) guidelines, because studies are limited and outdated. Therefore, guidelines might not reflect the current clinical situation accurately. Secondly, guidelines suggest using a multimodal approach for BPS/IC management, but data on the patient-perceived efficacy of these therapies are limited. The aim of this study is to investigate the perception of IC/BPS patients of their QoL, to determine which treatments they have received, and to examine how they evaluate the efficacy of these various (alternative) therapies. A quantitative retrospective database evaluation was performed, with data from an existing IC/BPS patient survey (n = 217) that was conducted in 2021. This survey contained QoL data based on validated questionnaires such as EQ-5D 5L. The QoL of patients is affected significantly by IC/BPS. This is evident from the various affected domains on the EQ-5D 5L. The symptom severity was negatively affected by a delay in diagnosis, and there were clear differences in QoL domains between females and males. Secondly, coagulation therapy and intravesical glycosaminoglycan (GAG) therapy were most appreciated by patients. Other (alternative) treatments were commonly utilized, although some had doubtful results and high discontinuation rates. QoL is considerably impaired in IC/BPS patients. The diverse responses and adherence to various treatments warrant a personalized approach (phenotype-oriented therapy). To achieve QoL improvement, it is important to incorporate the patient's perspective in treatment guidelines.

Health-related quality of life instrument with 8 items to measure health-related quality of life among family caregivers of people with dementia: A pilot validation study.

The health-related quality of life instrument with 8 items (HINT-8) was developed to measure health-related quality of life (HRQoL) in Korea. However, the HINT-8 has not yet been validated among the family caregivers of people with dementia (PwD). A cross-sectional pilot study. The study aimed to examine the convergent and discriminant validity of the HINT-8 among family caregivers of individuals with dementia. Forty-seven family caregivers of PwD. HINT-8 was compared with the 5-level EQ-5D (EQ-5D-5L) to assess its convergent and discriminant validity. Additionally, the association between the two instruments assessing HRQoL was examined using the short-form Bédard-Zarit Burden Interview (SZBI). The HINT-8 was a promising and valid HRQoL instrument for family caregivers of PwD. There was a significantly high correlation between the overall HINT-8 and EQ-5D-5L indices (r=0.85, p<.001). The HINT-8 had acceptable psychometric properties compared to the commonly used EQ-5D-5L, as indicated by the subdomains associated with family caregivers' burden measured by the SZBI. Future studies should compare the HINT-8 with existing dementia carer-specific QoL instruments among a larger study sample to enhance its statistical power and confirm its reliability and structural validity.

A quality-adjusted time without symptoms and toxicity (Q-TWiST) analysis comparing nivolumab plus ipilimumab (N+I) or nivolumab plus chemotherapy (N+CT) versus CT in patients (pts) with advanced esophageal squamous cell carcinoma (ESCC): CheckMate 648.

251 Background: In CheckMate 648, pts with advanced ESCC treated with first-line N+I or N+CT had significantly longer overall survival (OS) vs CT and maintained quality of life (QoL) with no new safety signals. Here we compare between-treatment differences in quality-adjusted survival using a Q-TWiST analysis in all randomized pts (29-month minimum follow-up). Methods: Survival time was partitioned into 3 health states: toxicity (TOX), time without symptoms of disease progression or toxicity (TWiST), and relapse (REL). TOX was defined as time spent with all-cause grade 3/4 adverse events after randomization, prior to disease progression. TWiST was defined as time prior to REL where pts did not experience TOX. REL was defined as time between disease progression and death, or the last known date alive. Mean duration of time in each state was calculated for each treatment group using descriptive statistics and Kaplan-Meier analysis. Utility values from the 3-level EQ-5D (EQ-5D-3L) questionnaire were used to adjust the time spent within each health state for QoL and to calculate Q-TWiST as the utility-weighted sum of the mean health state durations. Bootstrapping (500 samples) was used to estimate time in each health state and to compare mean Q-TWiST estimates between treatments. A between-group difference in Q-TWiST of 5 weeks was prespecified as being clinically important (defined as 10% of OS; based on a median OS in CT group of 10.7 months). Sensitivity analyses were conducted using different combinations of prespecified utility weights for each health state. Results: 970 pts were randomized 1:1:1 to N+I (n = 325), N+CT (n = 321), or CT (n = 324) and included in the Q-TWiST analysis. Median duration of follow-up for survivors was 155 weeks. Mean time in each health state was significantly longer for pts treated with N+I or N+CT compared to CT (Table). Between-group differences in Q-TWiST score were considered clinically important: 5.7 weeks (95% CI 5.5–6.0) favoring N+I vs CT and 7.6 weeks (95% CI 7.4–7.9) favoring N+CT vs CT. Sensitivity analyses supported these findings. Conclusions: In CheckMate 648, pts with unresectable advanced ESCC treated with N+I or N+CT had significantly longer OS compared with those treated with CT. Quality-adjusted survival using Q-TWiST also significantly favored N+I and N+CT compared to CT alone. Pts treated with N+I and N+CT not only experienced increased survival time, but also better QoL during their survival time. Clinical trial information: NCT03143153 . [Table: see text]

Using the Online Elicitation of Personal Utility Functions Approach to Derive a Patient-Based 5-Level Version of EQ-5D Value Set: A Study in 122 Patients With Rheumatic Diseases From Germany

ObjectivesTraditional preference elicitation methods, such as discrete choice experiments or time trade-off, usually require large sample sizes. This can limit their applicability in patient populations, where recruiting enough participants can be challenging.The objective of this study was to test a new method, called the Online elicitation of Personal Utility Functions (OPUF) approach, to derive an EQ-5D-5L value set from a relatively small sample of patients with rheumatic diseases. MethodsOPUF is a new type of online survey that implements compositional preference elicitation techniques. Central to the method are 3 valuation steps: (1) dimension weighting, (2) level rating, and (3) anchoring. An English demo version of the OPUF survey can be accessed at https://valorem.health/eq5d5l. From the responses, a personal EQ-5D-5L utility function can be constructed for each participant, and a group-level value set can be derived by aggregating model coefficients across participants. ResultsA total of 122 patients with rheumatic disease from Germany completed the OPUF survey. The survey was generally well received; most participants completed the survey in less than 20 minutes and were able to derive a full EQ-5D-5L value set. The precision of mean coefficients was high, despite the small sample size. ConclusionsOur findings demonstrate that OPUF can be used to derive an EQ-5D-5L value set from a relatively small sample of patients. Although the method is still under development, we think that it has the potential to be a valuable preference elicitation tool and to complement traditional methods in several areas.

Australian population norms for health-related quality of life measured using the EQ-5D–5L, and relationships with sociodemographic characteristics

BackgroundMeasuring health related quality-of-life (HRQoL) of the general population is essential to establish a reference for health outcome evaluations. This study sought to establish EQ-5D-5L population norms in Australia and to investigate the heterogeneity of HRQoL between sociodemographic variables.MethodsA cross-sectional study comprising of a representative sample of Australia’s general population (n = 9958) aged 18 or older. Recruitment quotas were set for the Australian census population by age, sex, state/territory of residence and rurality. Participants were recruited by Qualtrics through its database of over 800,000 registered panel members and asked to value their own state of health using the EQ-5D-5L domains and the EuroQol-Visual Analogue Scale (EQ-VAS). An Australian value set developed using Discreet Choice Experiment was used to calculate utility scores.ResultsThe estimated mean EQ-5D-5L index for Australia’s general population was 0.86 (standard deviation [SD] 0.19), and the EQ-VAS score was estimated as 73.2 (SD 21.7). 23.9% of the study population reported being in the best health state (11,111). Younger people, current smokers, people who are unemployed and people with more financial stress reported a lower EQ-5D-5L index score (p < 0.001). Residents in the major cities, inner regional and outer regional Australia reported higher health utility scores than those residing in remote and very remote Australia.ConclusionsThis is the first Australian study to apply the EQ-5D-5L in a nationally representative sample. The EQ-5D-5L Australian population norms obtained can be used as reference scores for future population health evaluations and comparisons. The findings facilitate a national reference for clinical, economic, and policy decision-making processes and provide a fuller understanding of the Australian population’s HRQoL.

Safety profile of pimavanserin therapy in elderly patients with neurodegenerative disease‐related neuropsychiatric symptoms: a Phase 3b study

AbstractBackgroundPatients with neurodegenerative diseases including Parkinson’s disease often experience neuropsychiatric symptoms, including hallucinations and delusions, apathy, and agitation.1,2 These symptoms can greatly impair daily function and lead to reduced quality of life and significant caregiver burden. These patients are generally elderly and often frail. Treatment options are limited, representing a substantial unmet need. Pimavanserin, a 5‐HT2A receptor inverse agonist/antagonist, is FDA‐approved to treat hallucinations and delusions associated with Parkinson’s disease psychosis.3 MethodThis Phase 3b, multicenter, randomized, double‐blind, placebo‐controlled, parallel‐group study evaluated the safety of pimavanserin (34 mg) versus placebo in frail adults and elderly patients with neuropsychiatric symptoms related to neurodegenerative disease. The primary endpoint was safety and tolerability as measured by treatment‐emergent adverse events. Secondary safety endpoints included change from baseline in motor (Extrapyramidal Symptom Rating Scale‐Abbreviated [ESRS‐A]) and cognitive function (Mini‐Mental State Examination [MMSE]) at Week 8. Suicidality was assessed using the Columbia‐Suicide Severity Rating Scale (C‐SSRS) or Global Clinician Assessment of Suicidality (GCAS). Exploratory efficacy endpoints included Clinical Global Impression‐Severity (CGI‐S) and ‐Improvement (CGI‐I), 5‐level version of EQ‐5D (EQ‐5D‐5L), and Sleep Disorders Inventory (SDI).ResultOverall, incidence of TEAEs were similar between pimavanserin (n = 392) and placebo (n = 392); 29.8% reported ≥1 TEAE (pimavanserin: 30.4%; placebo: 29.3%) and 1.8% reported serious TEAEs (pimavanserin: 2.0%; placebo: 1.5%) (Table 1). TEAEs leading to discontinuation or study termination were reported in 2.6% and 2.3% of patients on pimavanserin and placebo, respectively. The most frequently reported TEAEs included urinary tract infection (pimavanserin: 6.4%; placebo: 4.1%) and headache (pimavanserin: 2.0%; placebo: 3.8%). Mortality due to TEAEs was similar between groups (0.5% each). Pimavanserin did not impact motor‐ or cognition‐related function (ESRS‐A and MMSE, respectively); an improvement was seen in CGI‐I (MMRM LSM [SE]: ‐0.2 [0.07], p = 0.0140] and sleep function (SDI) at Week 8 (MMRM LSM [SE]: ‐0.3 [0.06], p&lt;0.0001] compared to placebo (Figures 1, 2); no differences were observed across other measures.ConclusionPimavanserin was well tolerated in patients with neuropsychiatric symptoms related to neurodegenerative disease, consistent with the known safety profile of pimavanserin, informing its use in Parkinson’s disease psychosis. Pimavanserin was not associated with cognitive decline or motor dysfunction.

Chronic Disease Outcomes As Predictors of Quality of Life in Patients with Hemophilia Α: Data from the Real-World AHEAD International Study

Background: Hemophilia A (HA), a genetic disorder caused by deficient or defective factor VIII (FVIII) clotting protein, has a significant impact on patients' quality of life (QoL).The international Antihemophilic factor Hemophilia A outcome Database (AHEAD) study (NCT02078427) is an ongoing prospective, non-interventional, multicenter study evaluating the long-term safety and effectiveness of FVIII replacement in patients with HA receiving either octocog alfa or rurioctocog alfa pegol (ADVATE®; ADYNOVATE® [US] / ADYNOVI™ [Europe]; Baxalta US Inc., a Takeda company Lexington, MA, USA) in routine clinical practice. Aim: This analysis aimed to evaluate the association between chronic disease outcomes and QoL in patients with moderate or severe HA without an active inhibitor and treated with octocog alfa. Methods:Male patients aged ≥18 years with moderate (FVIII 1-≤5%) or severe (FVIII &amp;lt;1%) HA without an active inhibitor and receiving octocog alfa were included in the analysis. Data were included across study years 1 to 5 for patients with all model variables reported in at least one study year. Cut off was set at year 5 as smaller sample sizes in later years did not allow for appropriate statistical modelling due to natural attrition and loss to follow up. The association between chronic disease outcomes and QoL was estimated using a Mixed Effects Model with repeated measures. The model assessed the impact of chronic pain (classified as no pain [used as the reference], mild pain, moderate pain, or severe pain) and number of problem joints (PJs; identified through the reporting of relevant components of the hemophilia joint health score, Gilbert Score and/or Petterson score) on EQ5D 3L index score (calculated using UK value set), controlling for clinical and demographic characteristics (annualized bleeding rate [ABR], HA severity, body mass index [BMI], and age at visit), in addition to individual patient effect, via the random effects component. The random effects component allows the model to control and account for individual patient effects that may not be measured by available independent variables but that may influence the output of the analysis. Results:The final analysis included 175 adult male patients with an average (range) of 2.1 (1-5) study years. The majority (70%) of patients had severe HA. At study year 1, the mean ± SD age was 33.6 ± 12.7 years and the mean ± SD number of PJs was 3.8 ± 2.0, while 47% of patients reported some level of chronic pain, with 20% of patients reporting moderate or severe pain levels. Mean ± SD EQ5D 3L index score was 0.77 ± 0.21 at study year 1. Overall, after controlling for demographic and clinical characteristics, and any individual patient effect, chronic outcomes (chronic pain and number of PJs) were found to have a statistically significant negative association with QoL (Table). The negative association of chronic pain with EQ5D 3L index score was greater with increasing pain level (mild, -0.082; moderate, -0.135; severe -0.169; P≤0.001). Number of PJs was also negatively associated with EQ5D 3L index score (P=0.005) with a decrease of -0.018 for each additional problem joint. Age at visit also showed a negative association with EQ5D 3L index score (-0.004; P=0.002). ABR, HA severity, and BMI did not have a statistically significant association with EQ5D 3L index score. Conclusions: In this Mixed Effects Model, chronic pain level and number of PJs both had a statistically significant negative association with QoL, highlighting the importance of managing these chronic outcomes with effective treatment.

Choosing the Best Instrument for Measuring Health Spillover Effect in Caregivers of Patients With Multiple Sclerosis

ObjectivesTo measure the health spillover effect in caregivers of patients with multiple sclerosis (MS), we aimed to select the best instrument from 2 common health-related quality of life (QoL) instruments, the 3-level EQ-5D (EQ-5D-3L) and the Health Utilities Index Mark 3 (HUI-3), by assessing them. MethodsUsing consecutive sampling, 452 primary caregivers of patients with MS were asked to fill out a Care-related QoL instrument (CarerQol-7D), EQ-5D-3L, HUI-3, and the Center for Epidemiologic Studies Depression Scale between October 2019 and May 2020. Convergent and clinical validity were assessed to measure spillover effect in caregivers of patients with MS. ResultsA strong correlation of health-utility scores between EQ-5D-3L and HUI-3 (r = 0.914, P < .01) was observed. The 95% limit of agreement (LoA) for CarerQol-7D and HUI-3 (−10.6 to 8.2) was narrower than the LoA for CarerQol-7D and EQ-5D-3L (−15.1 to 17.1). Both EQ-5D-3L and HUI-3 proved clinical validity for the QoL of caregivers. The CarerQoL-7D score was significantly lower in female (P < .001), single (P < .014), lower-educated (P < .001), parent’s relatives (P < .001), and unemployed (P < .001) caregivers. ConclusionsWe found that both, EQ-5D-3L and HUI-3, were appropriate for measuring caregivers’ QoL, although HUI-3 was a better choice because of its narrower LoA. Our findings suggest researchers should use HUI-3 to measure the quality-adjusted life-year of caregivers to aggregate with the QoL of patients in the denominator of an economic evaluation equation, such as the cost-effective ratio.

Abstract 18246: Determining the Most Important Factors for 1-year Health Status Using a Machine-Learning Algorithm in Patients With Peripheral Artery Disease Undergoing Revascularization for Claudication

Introduction: Over time, patients with peripheral artery disease (PAD) are at high risk of poor cardiovascular outcomes, but most immediately, their health status is impacted due to lifestyle-limiting symptoms. To improve functioning, peripheral vascular intervention (PVI) may be offered. We aimed to understand which factors predict subsequent health status outcomes following PVI. Methods: We used data from the international ILLUMENATE examining the safety of peripheral angioplasty with vs. without Stellarex drug-coated balloon for femoropopliteal artery disease in randomized trial between 2013 and 2019. Treatment arms were combined for this analysis. EQ-5D 3L Index and Visual Analog Scale (VAS) were assessed at baseline and 1 year. Random forest regression, a base-trees machine-learning algorithm, was built in the training sample (80% of the cohort) and evaluated in the validation sample (20% of the cohort). The importance of 58 included pre-procedural variables was ranked. The most important variables were defined based on visual inspection of the relative importance plots to evaluate the information gained value of each included variable. Results: In the 473 included patients, the mean age was 68.2 ± 9.2 years, 32.1% were female and 4.2% were Black. For the 1-year 3L Index, the most important pre-procedural variables were liver disease and the 3L Index (54.1%). For the 1-year VAS, the most important variables were angioplasty/drug eluting stent following by chronic lung disease (94.9%), mechanical thrombectomy (81.0%), anticoagulant (80.1%) and congestive heart failure (76.5%). (Figure) Conclusions: As part of an effort to develop an individualized health status profile with machine learning, a predictable ranking of pre-procedural variables can inform 1-year health status outcomes in patients with PAD undergoing PVI for claudication. These approaches can inform clinical decision-making and pre-habilitation efforts to maximize treatment success.

What is the relationship between health-related quality of life among scoliosis patients and their caregiver burden? A cross-sectional study in China

BackgroundCaregivers play a vital role in the recovery of scoliosis patients, but limited studies evaluate the caregivers’ HRQoL and burden in health care. This study aimed to explore the health-related quality of life (HRQoL) of scoliosis patients and their caregivers, and identify the factors influencing caregiver burden in Eastern China.MethodsThis cross-sectional study was conducted from August 2018 to January 2019 at the Shandong Provincial Hospital, Jinan, China. The HRQoL of scoliosis patients was measured by the Scoliosis Research Society-22r (SRS-22r), five-level EQ-5D (EQ-5D-5L) and Child Health Utility 9D (CHU9D). The caregivers’ questionnaires consist of the EQ-5D-5L, WHO-five wellbeing index (WHO-5), 22-item Zarit Caregiver Burden Interview (ZBI-22) and Social Support Rating Scale (SSRS). Spearman correlation coefficients were used to estimate the relationship among caregivers’ burden, social support, HRQoL, and SWB. Cohen’s effect size (Cohen’s d) was used to assess the ZBI-22 total score between different groups. Multiple stepwise hierarchical linear regression models were conducted to assess the associated factors of caregiver burden.ResultsThere were 59 scoliosis patients and their caregivers (n = 59) included in the analysis. The mean health state utility of adolescent scoliosis patients (n = 39) was 0.718 (95%CI: 0.654, 0.782) based on CHU9D and adult scoliosis patients (n = 20) was 0.663 (95%CI: 0.471, 0.855) based on EQ-5D-5L. The mean health state utility of male scoliosis patients (0.792/0.667) was higher than females (0.681/0.662) based on CHU9D and EQ-5D-5L (p > 0.05), respectively. The ZBI-22 total score of scoliosis patients’ caregivers was 27.86 (SD: 20.59). Scoliosis patients’ HRQoL was significantly inversely correlated with caregiver burden, and the HRQoL and subjective wellbeing (SWB) of caregivers were moderately and inversely correlated with caregiver burden. The regression results showed that the patients’ age and caregivers’ SWB were key characteristics associated with caregiver burden.ConclusionsThe caregiver burden of adolescent patients was higher than that of adult patients, and the satisfaction rate of adolescent scoliosis patients was higher than that of adult scoliosis patients. Improving the functional state of scoliosis patients and providing appropriate nursing practice education from health professionals would be necessary to effectively improve caregivers SWB and alleviate caregiver burden.

Evaluating the Short-Term Costs and Benefits of a Nationwide Diabetes Prevention Programme in England: Retrospective Observational Study.

Prevention programmes typically incur short-term costs and uncertain long-term benefits. We use the National Health Service (NHS) England Diabetes Prevention Programme (NHS-DPP) to investigate whether behaviour change programmes may be cost-effective even within the short-term participation period. We analysed 384,611 referrals between June 2016 and March 2019. We estimated NHS costs using implementation costs and provider payments. We used linear regressions to relate utility changes to the number of sessions attended, based onresponses to the five-level EQ-5D (EQ-5D-5L) at baseline and final session for 18,959 participants. We then calculated the corresponding quality-adjusted life year (QALY) change for all 384,611 referrals by combining the estimated regression coefficients with the observed level of attendance, with individuals that did not attend any programme sessions being assumed to experience zero benefit. In secondary analysis, we added weight change, recorded for 18,105 participants to the regression and applied predicted values to all referrals with missing weight change values estimated using multiple imputation with chained equations. We then estimated the cost-per-QALY generated. Average cost per referral was £119 (standard deviation: £118; 2020 price year, UK £ Sterling). Each session attended was associated with a 0.0042 increase in utility (95% confidence interval (CI): 0.0025-0.0059). This generated 1,773 QALYs across all referrals (95% CI: 889-2,656). Cost-per-QALY was £24,929 (95% CI: £16,635-49,720) when implementation costs were excluded. Secondary analysis showed each session attended and kilogram of weight lost were associated with 0.0034 (95% CI: 0.0016-0.0051) and 0.0025 (95% CI: 0.0020-0.0031) increases in utility, respectively. These generated 1,542 QALYs, at a cost-per-QALY of £28,661 when implementation costs were excluded. Participants experienced small utility gains from session attendance and weight loss during their programme participation. These benefits alone made this low-cost behaviour change programme potentially cost-effective in the short-term.

Obesity is associated with pain and impaired mobility despite therapy in systemic lupus erythematosus.

To investigate whether abnormal BMI is associated with health-related quality of life (HRQoL) impairments, defined as patient-reported problems within the different dimensions of the three-level EQ-5D (EQ-5D-3L), before and after treatment for active systemic lupus erythematosus (SLE). We conducted a post-hoc analysis of data from two phase III clinical trials of belimumab in SLE, i.e., BLISS-52 (n = 865) and BLISS-76 (n = 819). Underweight was defined as BMI <18.5 kg/m2, normal weight as BMI ≥18.5 but <25 kg/m2, pre-obesity as BMI ≥25 but <30 kg/m2, and obesity as BMI ≥30 kg/m2. We investigated associations between BMI groups and problems (level 2 or 3) within each one of the five EQ-5D dimensions before treatment initiation and at week 52, using logistic regression analysis adjusting for age, ethnicity, disease activity, and glucocorticoid dose, and for the post-treatment analysis also for belimumab treatment and baseline EQ-5D-3L responses. Of 1,684 patients included, 73 (4%) were classified as underweight, 850 (50%) as normal weight, 438 (26%) as pre-obese, and 323 (19%) as obese. At baseline, obesity was associated with mild to severe problems in all EQ-5D dimensions (p < 0.05 for all), yielding the strongest association with problems in mobility (adjusted odds ratio, aOR: 2.1; 95% confidence interval, CI: 1.6-2.8; p < 0.001). Pre-obesity was also associated with problems in mobility (aOR: 1.4; 95% CI: 1.1-1.8; p = 0.005). Post-intervention, obesity was associated with problems in mobility and pain/discomfort, and pre-obesity with problems in mobility and self-care (p < 0.05 for all). Our study adds to the evidence that high BMI negatively affects SLE patients' HRQoL, with obesity being associated with pain and impaired mobility despite therapy.