EuroQol-5 Dimensions-5 Levels Research Articles

Health-related quality of life with enzalutamide versus flutamide in castration-resistant prostate cancer from the AFTERCAB study

BackgroundPatient-reported outcome (PRO) measures can provide valuable information in evaluating patients’ health-related quality of life (HRQoL). Post hoc analysis of the AFTERCAB study was conducted to evaluate the HRQoL benefit of enzalutamide plus androgen deprivation therapy (ADT) compared to flutamide plus ADT for the treatment of patients with castration-resistant prostate cancer (CRPC) in Japan.MethodsThe open-label AFTERCAB study was conducted from November 2016 to March 2020 in Japanese men aged ≥ 20 years with asymptomatic or mildly symptomatic CRPC. Patients received enzalutamide plus ADT or flutamide plus ADT, respectively, as first-line alternative androgen therapy (AAT). HRQoL was analyzed through the Functional Assessment of Cancer Therapy–Prostate, EuroQoL 5-Dimension 5-Level instruments, Brief Pain Inventory–Short Form, and Brief Fatigue Inventory. The longitudinal changes in HRQoL, HRQoL deterioration based on minimally important difference (MID), and time to HRQoL deterioration were evaluated for first-line AAT.ResultsOverall, HRQoL between the enzalutamide and flutamide groups was similar during first-line treatment. No statistically significant HRQoL difference in change from baseline to week 61 (least square mean difference; p value) was observed. Furthermore, proportions of pain progression, symptom worsening, and HRQoL deterioration based on MID, were not significantly different between groups.ConclusionsThe results were similar in all subscales of each PRO, demonstrating similar HRQoL deterioration based on MID criteria between the enzalutamide and flutamide groups.

The quality-adjusted life-years in the oncological patients’ health-related quality of life

The oncological treatment can significantly affect patients’ health-related quality of life (HRQoL), which should be monitored to ensure our patients’ well-being. The often-used HRQoL measurer is the quality-adjusted life-year (QALY) indicator of the disease burden, describing both quality and quantity of life lived. The main aim of the study was to discuss the methodology and usefulness of evaluating QALYs using the HRQoL questionnaires: EuroQoL (EQ)-5 dimensions-3 levels (EQ-5D-3L) and EQ visual analogue scale (EQ-VAS) in 32 cervical cancer patients. We obtained the questionnaire and calculated QALYs based on the Gross Domestic Product (GDP) method. In our study, the total scoring of the EQ-Index, EQ-VAS evaluation was 2620 and 2409 points, respectively, which corresponds with the QALYs value of 26.2 and 24.9, respectively. We expressed the QALYs outcome into the economic equivalent of nearly 900,000 US dollars (USD) as the total health profit for both the patients and the healthcare system. Obtaining the QALY factor can help establish the medical management’s influence on the patients’ HRQoL and improve the healthcare services to ensure the best health outcomes.

Economic evaluation of sintilimab plus chemotherapy vs. pembrolizumab plus chemotherapy for the treatment of first-line advanced or metastatic squamous NSCLC.

Background and objectiveSintilimab has superior efficacy and safety in patients with advanced or metastatic squamous non-small cell lung cancer (NSCLC), but its cost-effectiveness in China is unclear. This study is to evaluate the cost-effectiveness of sintilimab plus chemotherapy vs. pembrolizumab plus chemotherapy for locally advanced or metastatic squamous NSCLC in China.MethodsFrom the perspective of the Chinese health system, the partitioned survival model with three health states was established in a 3-week cycle and a lifetime time horizon. The two-stage method was used to estimate the overall survival hazard ratios to avoid the bias by crossover design in ORIENT-12 and KEYNOTE-407 studies. The anchored matching adjusted indirect comparison method (MAIC) was used for indirect comparison based on the individual patient data from ORIENT-12 and the publicly published KEYNOTE-407 study due to the lack of head-to-head clinical trials. Only direct medical costs were included, and utilities were derived from the published literature in the base case analysis. Sensitivity analysis was also performed to verify the robustness of the model results. In addition, the scenario analysis where the utilities were derived from the Quality of Life Questionnaire-Core 30 (QLQ-C30) scale in the ORIENT-12 by mapping to the EuroQol-5-dimension 5-level (EQ-5D-5L) was carried out to explore the uncertainty of the results.ResultsCompared with pembrolizumab + chemotherapy, sintilimab + chemotherapy incurred a lower lifetime cost ($12,321 vs. 36,371) and yielded fewer quality-adjusted life-years (QALYs) (0.9902 vs. 1.0085), which resulted in an incremental cost-effectiveness ratio (ICER) of $1,314,208/QALY. A sintilimab strategy is a cost-effectiveness option under the WTP of 1–3 times the GDP per capita in China ($11,250/QALY~$33,749/QALY). The utility value of the post-progression, the unit cost of albumin paclitaxel, and the utility value of the progression-free state were the main drivers in the deterministic sensitivity analysis (DSA). According to the probabilistic sensitivity analysis (PSA), sintilimab + chemotherapy was 100% cost-effective when the WTP was 1–3 times China's per capita GDP. The results of the scenario analysis showed that sintilimab + chemotherapy obtained more QALYs (1.2319 vs. 1.1815) and lower costs ($12,321 vs. 36,371), which implied that sintilimab + chemotherapy may dominate the pembrolizumab + chemotherapy.ConclusionCompared with pembrolizumab + chemotherapy, sintilimab + chemotherapy is more cost-effective for first-line treatment in Chinese patients with locally advanced or metastatic squamous NSCLC.

Structural equation model of the relationship between functional ability, mental health, and quality of life in older adults living alone.

AimsLiving alone, a reality in an increasing number of older adults recently, is a risk factor for low quality of life. This study identified the predictors of quality of life in older adults living alone based on mental health and the International Classification of Functioning, Disability, and Health.MethodsThis secondary data analysis used information from the 2018 Assessing the Requirements of Customized Health Care and Daily Living Support Services survey (N = 1,022), collected from adults aged ≥ 65 living alone in Siheung City, South Korea, from August to October 2018. The exogenous variables were body functions (hand grip strength, timed “up and go” test score, and body mass index), daily living activities (Korean Instrumental Activities of the Daily Living Scale), social activity participation (social activity engagement, neighbor contacts, and family contacts), and participation in economic activity (frequency). The endogenous variables were mental health (Geriatric Depression Scale Short Form—Korean Version and UCLA Loneliness Scale) and quality of life (EuroQoL-5 Dimension-3 Level and EuroQoL-Visual Analog Scale).ResultsAfter modifying the hypothetical model, which had failed to satisfy the recommended fitness level, the (modified) model had good fitness indices Q (CMIN / df) 2.90, GFI 1, AGFI 1, RMSEA 0.04, CFI 0.90 and PCFI 0.53. Of the nine pathways of the modified model, five were statistically significant. Quality of life was affected by body functions, daily living activities, social activity participation, and mental health. These variables explained 68.2% of the factors affecting quality of life.ConclusionsBy highlighting the role of mental health, this model provides a useful framework for improving the quality of life of older adults who live alone and function at various levels in the community. Focusing on advancing mental health through body functions, daily living activities, and social activity participation can improve quality of life.

Effectiveness of Vortioxetine in Patients with Major Depressive Disorder in Real-World Clinical Practice in Italy: Results from the RELIEVE Study.

PurposeVortioxetine has demonstrated efficacy in randomized controlled trials and is approved for the treatment of major depressive disorder (MDD); however, data are limited concerning its effectiveness when used in routine clinical care. The Real-Life Effectiveness of Vortioxetine in Depression (RELIEVE) study aimed to assess the effectiveness and tolerability of vortioxetine for the treatment of MDD in routine clinical practice in Canada, France, Italy, and the USA. This paper presents findings for the patient cohort in Italy.Patients and MethodsRELIEVE was a 6-month, international, observational, prospective cohort study in outpatients initiating vortioxetine treatment for MDD in routine care settings at their physician’s discretion (NCT03555136). Patient functioning was assessed using the Sheehan Disability Scale (SDS). Secondary outcomes included depression severity (9-item Patient Health Questionnaire [PHQ-9]), cognitive symptoms (5-item Perceived Deficits Questionnaire-Depression [PDQ-D-5]), and quality of life (EuroQol 5-Dimensions 5-Levels questionnaire [EQ-5D-5L]). Changes from baseline to month 6 were assessed using mixed models for repeated measures, adjusted for relevant confounders.ResultsData are available for 231 patients enrolled in Italy (mean age, 55.5 years; 27% >65 years). Overall, 69% of patients reported at least one comorbidity, 55% were overweight/obese, and 47% had current anxiety symptoms. Adjusted least-squares mean (standard error) change in SDS score from baseline to week 24 was −6.6 (0.6) points (P < 0.001). Respective changes in PHQ-9, PDQ-D-5, and EQ-5D-5L scores were −5.9 (0.5), −3.6 (0.4), and +0.13 (0.01) points (all P < 0.0001). Adverse events were reported by 29 patients (13%), most commonly nausea (n = 14, 6%). Eleven patients (5%) discontinued treatment due to adverse events.ConclusionClinically relevant and sustained improvements in overall functioning, symptoms of depression, cognitive symptoms, and health-related quality of life were observed in patients with MDD treated with vortioxetine over a period of 6 months in routine care in Italy, including a high proportion of elderly patients.

The Burden of Nocturnal Symptoms in Patients with Chronic Obstructive Pulmonary Disease: Results of a Real-World Survey in the USA.

IntroductionPatients with chronic obstructive pulmonary disease (COPD) often have poor sleep quality and report a worsening of respiratory symptoms during night-time. However, current clinical guidelines for COPD management do not specifically consider nocturnal symptoms when recommending pharmacological treatment. This study aimed to better understand the burden of nocturnal symptoms in patients with COPD, and to evaluate the importance of nocturnal symptom control compared with daytime and overall symptom control.MethodsData were analyzed from the Adelphi Respiratory Disease Specific Programme, a point-in-time survey of physicians and their patients, conducted in the USA in 2019. Primary care physicians and pulmonologists who managed three or more patients with COPD per month were eligible for inclusion; eligible patients were ≥ 18 years old, with a physician-confirmed diagnosis of COPD.ResultsSurveys from 171 physicians and 800 patients were analyzed. Everyday symptoms were reported in 14% of patients. In total, 88% of patients reported daytime symptoms, and 74% of patients experienced nocturnal symptoms, with 7% reporting daily nocturnal symptoms. Patients experiencing nocturnal symptoms every day had the greatest impairment in their activity as per the Work Productivity and Activity Impairment questionnaire (mean total activity impairment, 66.9%; nocturnal symptoms once or twice a week, 41.1%; no nocturnal symptoms, 26.4%). Patients experiencing daily nocturnal symptoms also had the lowest quality of life (QoL) as per the EuroQoL 5-Dimension 3-Level score. Physicians reported prescribing therapy based on sustained 24-h symptomatic relief for the majority of patients (78%). They reported nocturnal symptom control as a factor in their choice of therapy for 38% of patients, and daytime symptom control as a reason for 61% of patients.ConclusionDaytime and nocturnal symptoms are common among patients with COPD. Frequency of nocturnal symptoms is related to a significant impairment in activity and health-related QoL.Supplementary InformationThe online version contains supplementary material available at 10.1007/s41030-022-00196-7.

Psychometric properties of the Spanish version of the EuroQol-5D-5L in previously hospitalized COVID-19 survivors with long COVID

The EuroQol 5-dimensions 5-levels (EQ-5D-5L) is a generic patient-reported outcome measures (PROM) used for evaluating health-related quality of life (HRQoL). No data on its psychometric properties in COVID-19 survivors is available. We aimed to describe internal consistency, test–retest reliability, and construct validity of the EQ-5D-5L in people with long-COVID. Ninety-three (n = 93) individuals previously hospitalized due to COVID-19 with post-COVID symptoms completed the EQ-5D-5L questionnaire twice one year after hospital discharge in a three-week interval. Internal consistency (Cronbach alpha and Omega value), test–retest reliability (kappa and ICC2,1) and construct validity (factor analysis), and floor/ceiling effects were calculated. No ceiling effect was observed in any dimension whereas the floor effect ranged from 53.76 to 94.62%. The overall Cronbach’s α value was 0.75 (95%CI 0.64–0.83) and the Omega ω value was 0.77 (95%CI 0.66–0.84), showing good internal consistency of the questionnaire. Further, Cronbach’s alpha values the of each dimension ranged from 0.63 to 0.77 whereas those for Omega values ranged from 0.70 to 0.79. The test–retest reliability of the total score was excellent (ICC2,1 0.86, 95%CI 0.798–0.911). The agreement percentage ranged from 85.13 to 96.77%; but kappa coefficients ranged from fair (κ: 0.37) to good (κ: 0.61). The factor analysis showed factor loadings from 0.585 to 0.813 supporting good construct validity. The EQ-5D-5L has good psychometric properties to be used as a PROM to assess HRQoL in hospitalized COVID-19 survivors with long-COVID.

The influence of adverse drug effects on health-related quality of life in chronic obstructive pulmonary disease patients.

Chronic obstructive pulmonary disease (COPD) is a leading cause of morbidity and mortality worldwide. Concerns have been raised about the influence of adverse drug effects on patient's health-related quality of life (HRQoL) in COPD patients. This study aimed to evaluate the impact of COPD treatment-related adverse effects on HRQoL in COPD patients. In a cross-sectional study, COPD patients aged 40 years or older were identified and interviewed during their hospital visits. The EuroQol 5 Dimension 5 Level (EQ-5D-5L) questionnaire was used for evaluating HRQoL. Potential treatment adverse effects were evaluated as experienced by participants during the last 2 weeks preceding the interview. The intensity of adverse effects was reported in the following categories: never, mild, moderate and severe. Multivariable linear regression model was performed to evaluate the influence of adverse drug effects on utility scores as an indicator of HRQoL. A total of 203 patients diagnosed with COPD were recruited in the current study. The mean utility score of the study sample was 0.68 (SD = 0.36). Moderate-severe constipation, moderate-severe confusion, mild urinary hesitation, moderate-severe urinary hesitation, moderate-severe dry eyes and moderate-severe drowsiness were significant predictors/determinants for the average utility scores (coefficients were -0.099, -0.191, -0.111, -0.157 and -0.144, respectively). In addition, having higher COPD Assessment Test scores and severe disease was negatively associated with average utility scores (coefficients were -0.287 and -0.124, respectively). Higher intensity of COPD treatment-related adverse effects has a negative influence on HRQoL in COPD patients. Anticholinergic drug effects are of concern in COPD adults' population.

Effectiveness and safety of Korean medicine treatment based on the clinical practice guidelines in patients with acute peripheral facial palsy: A protocol for a multicenter, prospective, observational study.

Introduction:Peripheral facial palsy (PFP) results in weakness or paralysis of the affected side of the face. In Korea, there is a high demand for Korean medicine treatment for PFP. The clinical practice guidelines (CPGs) of Korean medicine for facial palsy were developed; however, there remains insufficient evidence to support the effectiveness and safety of Korean medicine treatment. Thus, this study aimed to evaluate the effectiveness and safety of Korean medicine treatment based on the CPGs in patients with acute PFP.Methods:This is a multicenter, prospective, observational study. The participants will be recruited from one Korean medicine hospital and eight Korean medicine clinics. The participants will receive Korean medicine treatments based on the CPGs, fill in survey questionnaires, and undergo electrophysiologic testing. The changes in House-Brackmann (H-B) grade, movement of the lip and eye, symptoms related to or accompanied by facial palsy, Facial Disability Index, EuroQol 5-dimension 5-level (EQ-5D-5L), and EuroQol Visual Analogue Scale (EQ-VAS), and the results of electromyography (EMG), electroneurography (ENoG), and Blink Reflex test will be analyzed. For the safety analysis, adverse events will be recorded, and for the feasibility analysis, the results of the Was It Worth It questionnaire will be assessed.Conclusion:We expect to draw real-world clinical data on the effectiveness and safety of Korean medicine treatment based on the CPGs in patients with acute PFP from this study. It would be the basis for complementing and improving the CPGs and provide the basis of clinical and policy decision-making.Trial registration:This study was approved by the Institutional Review Board of Kyung Hee University Korean Medicine Hospital (2021-06-005-001), and registered with the Korean Clinical Trial Registry (CRIS), Republic of Korea (KCT0006562).

Health Inequalities by Sexual Orientation: Results from the 2016-2017 Barcelona Health Survey.

Purpose: The aim of this study was to assess health inequalities by sexual attraction in the 2016-2017 Barcelona population, stratifying by sex. Methods: Data came from the 2016-2017 Barcelona Health Survey, where 3362 adults answered among other instruments the EuroQol-5 dimensions-5 levels (EQ-5D-5L), which measures five dimensions and summarizes health-related quality of life into a single utility index score. To assess health differences by sexual attraction, we constructed Tobit models for the EQ-5D index score and Poisson regression models for the EQ-5D dimensions. Nested models were constructed to examine the mediating role of discrimination and health-related variables. Results: After adjusting for sociodemographic variables, women feeling attraction to more than one sex showed a lower EQ-5D index score (worse health) than those with only other sex attraction (-0.042, p = 0.012), and higher prevalence of problems with mobility, usual activities, and anxiety/depression with the following adjusted prevalence ratios (aPR) and confidence intervals (CIs): 1.79 (95% CI 1.05-3.05), 1.84 (95% CI 1.05-3.21), and 1.76 (95% CI 1.27-2.43). Women feeling attraction only to their same sex also presented higher prevalence of anxiety/depression (aPR = 1.46, CI 95% 1.10-1.92). In contrast, differences were not observed for men. Conclusion: Women, but not men, feeling attraction to more than one sex and only same-sex attraction in Barcelona in 2016-2017 presented worse health than those feeling only other sex attraction, with discrimination playing a mediating role in explaining such inequalities. These results among women indicate the need to develop public health strategies in Barcelona addressed to lesbian and bisexual women, considering the intersection of gender and sexual orientation.

Correlation of patient-reported outcome measures to performance-based function in critical care survivors: PREDICTABLE

BackgroundEstablishing sequela following critical illness is a public health priority; however, recruitment and retention of this cohort make assessing functional outcomes difficult. Completing patient-reported outcome measures (PROMs) via telephone may improve participant and researcher involvement; however, there is little evidence regarding the correlation of PROMs to performance-based outcome measures in critical care survivors. ObjectivesThe objective of this study was to assess the relationship between self-reported and performance-based measures of function in survivors of critical illness. MethodsThis was a nested cohort study of patients enrolled within a previously published study determining predictors of disability-free survival. Spearman's correlation (rs) was calculated between four performance-based outcomes (the Functional Independence Measure [FIM], 6-min walk distance [6MWD], Functional Reach Test [FRT], and grip strength) that were collected during a home visit 6 months following their intensive care unit admission, with two commonly used PROMs (World Health Organization Disability Assessment Scale 2.0 12 Level [WHODAS 2.0] and EuroQol-5 Dimension-5 Level [EQ-5D-5L]) obtained via phone interview (via the PREDICT study) at the same time point. ResultsThere were 38 PROMs obtained from 40 recruited patients (mean age = 59.8 ± 16 yrs, M:F = 24:16). All 40 completed the FIM and grip strength, 37 the 6MWD, and 39 the FRT. A strong correlation was found between the primary outcome of the WHODAS 2.0 with all performance-based outcomes apart from grip strength where a moderate correlation was identified. Although strong correlations were also established between the EQ-5D-5L utility score and the FIM, 6MWD, and FRT, it only correlated weakly with grip strength. The EQ-5D overall global health rating only had very weak to moderate correlations with the performance-based outcomes. ConclusionThe WHODAS 2.0 correlated stronger across multiple performance-based outcome measures of functional recovery and is recommended for use in survivors of critical illness.

Working Conditions, Work Productivity, Quality of Life, and Depressive Symptoms in Undiagnosed Adults with and without Attention-Deficit/Hyperactivity Disorder (ADHD) Symptoms During the COVID-19 Pandemic.

PurposeThis study aims to evaluate the impact of the COVID-19 pandemic on employment status, work productivity, quality of life (QOL), and depressive symptoms in undiagnosed adults with and without attention-deficit/hyperactivity disorder (ADHD) symptoms in Japan.MethodsAn observational study used baseline data from a Japanese Medilead Healthcare Panel before the COVID-19 pandemic (October–December 2019). Eligible panel participants were then surveyed during the pandemic (March 2021). ADHD symptoms were screened using the Adult ADHD Self-Report Scale. Changes in QOL (EuroQol 5-Dimensions 5-Levels; EQ-5D-5L) and productivity impairment (Work Productivity and Activity Impairment scale; WPAI) from before to during the pandemic were analyzed in undiagnosed adults with and without ADHD symptoms. Unemployment rate and depressive symptoms (Patient Health Questionnaire; PHQ-9) during the pandemic were compared between groups.ResultsParticipants with (N=949) and without (N=942) ADHD symptoms completed the survey. The unemployment rate was not significantly different between participants with and without ADHD symptoms. Participants with ADHD symptoms were more likely to change jobs or employers during the pandemic. PHQ-9 scores in participants with ADHD symptoms were significantly higher than in those without ADHD symptoms (8.96 vs 3.57, respectively) during the pandemic. Before the pandemic, WPAI scores were significantly higher and EQ-5D-5L scores lower in participants with ADHD symptoms than in those without. Productivity improved and QOL was not altered during the pandemic in both groups, but productivity and QOL remained poorer among participants with ADHD symptoms than in those without.ConclusionProductivity was improved among all participants during the COVID-19 pandemic, contrary to expectations. However, adults with ADHD symptoms consistently had lower productivity, poorer QOL, and more depressive symptoms than those without ADHD symptoms.

Health utilities in Chinese patients with coronary heart disease and impaired glucose tolerance (ACE): A longitudinal analysis of a randomized, double-blind, placebo-controlled trial.

BackgroundWe estimate health‐related quality of life and the impact of four cardiovascular events (myocardial infarction [MI], stroke, congestive heart failure, angina) and gastrointestinal events in 6522 Chinese patients with coronary heart disease (CHD) and impaired glucose tolerance (IGT) participating in the Acarbose Cardiovascular Evaluation (ACE) trial.MethodsHealth‐related quality of life was captured using the EuroQol‐5 Dimension‐3 Level (EQ‐5D‐3L), with data collected at baseline and throughout the trial. Multilevel mixed‐effects linear regression with random effects estimated health‐related quality of life over time, capturing variation between hospital sites and individuals, and a fixed‐effects linear model estimated the impact of cardiovascular and gastrointestinal events.ResultsPatients were followed for a median of 5 years (interquartile range 3.4‐6.0). The average baseline EQ‐5D score of 0.930 (SD 0.104) remained relatively unchanged over the trial period with no evidence of statistically significant differences in EQ‐5D score between randomized treatment groups. The largest decrement in the year of an event was estimated for stroke (−0.107, P < .001), followed by heart failure (−0.039, P = .022), MI (−0.021, P = .047), angina (−0.012, P = .047), and gastrointestinal events (−0.005, P = .430). MI and stroke reduced health‐related quality of life beyond the year in which the event occurred (−0.031, P = .006, and −0.067, P < .001, respectively).ConclusionsAcarbose treatment had no impact on health‐related quality of life in ACE trial participants with CHD and IGT. Events such as MI, stroke, heart failure, and angina reduce health‐related quality of life around the time they occurred, but only MI and stroke impacted on longer‐term health‐related quality of life.

Cohort profile: the Liyang cohort study on chronic diseases and risk factors monitoring in China (Liyang Study)

PurposeThe Liyang cohort study on chronic diseases and risk factors monitoring in China (Liyang Study) is a prospective population-based study which aims to investigate and identify the determinants of the...

Cooled radiofrequency ablation of genicular nerves provides 24-Month durability in the management of osteoarthritic knee pain: Outcomes from a prospective, multicenter, randomized trial.

ObjectiveTo assess long‐term outcomes of cooled radiofrequency ablation (CRFA) of genicular nerves for chronic knee pain due to osteoarthritis (OA).MethodsA prospective, observational extension of a randomized, controlled trial was conducted on adults randomized to CRFA. Subjects were part of a 12‐month clinical trial comparing CRFA of genicular nerves to a single hyaluronic injection for treatment of chronic OA knee pain, who then agreed to visits at 18‐ and 24‐months post CRFA and had not undergone another knee procedure since. The subjects were evaluated for pain using the Numeric Rating Scale (NRS) function using the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC), subjective benefit using the Global Perceived Effect (GPE) scale, quality of life using the EuroQol‐5‐Dimensions‐5 Level (EQ‐5D‐5L) questionnaire, and safety.ResultsOf 57 subjects eligible, 36 enrolled; 32 completed the 18‐month visit with a mean NRS score of 2.4 and 22 (69%) reporting ≥50% reduction in pain from baseline (primary endpoint); 27 completed the 24‐month visit, with a mean NRS of 3.4 and 17 (63%) reporting ≥50% pain relief. Functional and quality of life improvements persisted similarly, with mean changes from baseline of 53.5% and 34.9% in WOMAC total scores, and 24.8% and 10.7% in EQ‐5D‐5L Index scores, at 18‐ and 24‐months, respectively. There were no identified safety concerns in this patient cohort.ConclusionIn this subset of subjects, CRFA of genicular nerves provided durable pain relief, improved function, and improved quality of life extending to 24 months post procedure, with no significant safety concerns.

S304: SUTIMLIMAB, A COMPLEMENT C1S INHIBITOR, PROVIDES SUSTAINED IMPROVEMENTS IN PATIENT-REPORTED OUTCOMES IN PATIENTS WITH COLD AGGLUTININ DISEASE (CAD): 2 YEAR FOLLOW-UP FROM THE CARDINAL STUDY

Background: CAD is a rare chronic autoimmune hemolytic anemia characterized by classical complement pathway (CP)-mediated hemolysis, anemia, fatigue, and poor quality of life (QOL). Sutimlimab is a first-in-class humanized monoclonal antibody that selectively inhibits C1s of the C1 complex, preventing CP activation, while leaving the alternative and lectin pathways intact. One-year interim follow-up from the CARDINAL study (NCT03347396) have previously demonstrated continuous classical CP inhibition with sutimlimab resulted in rapid, sustained improvements in all patient-reported outcomes (PROs) measures evaluated. Aims: To report sutimlimab effect on PROs at 2 years, from the CARDINAL Part B extension. Methods: CARDINAL was a Phase 3, open-label, single-arm study with a 26-week treatment period (Part A) and a 2-year extension (Part B) after the last patient finishes Part A. Sutimlimab was administered through intravenous infusions on Days 0 and 7, followed by biweekly dosing. PRO data through Week 135, the last data recording within the 2-year Part B time period, are reported here. Efficacy endpoints included hemolytic markers and the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) as a measure of QOL. Exploratory QOL endpoints included mean change from baseline in EuroQol 5-dimension 5-level (EQ-5D-5L) scores, 12-Item Short Form Health Survey (SF-12), Patient Global Impression of Change (PGIC) and Patient Global Impression of Fatigue Severity (PGIS). PRO measures were evaluated every 3 months and conducted in the following order: FACIT-Fatigue, PGIS, PGIC, SF-12 and EQ-5D-5L. Descriptive statistics, frequency, or percentage were used to analyze outcomes. Results: Overall, 24 patients enrolled and 22 finished Part A and entered Part B, with 19 (86.4%) patients completing Part B. The mean (SD) FACIT-Fatigue score at baseline was 32 (11) and improved by 7 (8) within 1 week of sutimlimab treatment; the mean change score from baseline remained ≥5 from Week 1 to Week 135 (minimum score of 38 (9) at Week 123, maximum score of 44 (5) at Week 7), consistent with a clinically meaningful improvement. Efficacy for the EQ-5D-5L visual analogue scale (VAS) was sustained over 2 years; the mean (SD) change from baseline in EQ-5D-5L visual analogue scale (VAS) score (n=15/22) at Week 135 was 17.1 (21.6) (Figure A). The majority of evaluable patients (n=13/15) indicated an improved disease state compared to baseline on the PGIC at Week 135. No or mild fatigue was reported in patients (80%, n=12/15) on completing PGIS at Week 135, compared with one-third at baseline (n=2/6). The mean increase in SF-12 physical and mental component scores (n=6/22) from baseline to Week 123 were 4.7 (6.9) and 3.8 (14.1) (Figure B), consistent with the clinically important changes of 3.9 and 2.8 respectively. Image:Summary/Conclusion: From this CARDINAL follow-up extension study, sutimlimab has shown to produce rapid and sustained improvements in FACIT-Fatigue and other PRO measures evaluated up to 2 years, demonstrating the continued meaningful impact of sutimlimab on patient QOL.

P1739: HEALTH-RELATED QUALITY OF LIFE IN PATIENTS WITH TRIPLE-CLASS EXPOSED RELAPSED/REFRACTORY MULTIPLE MYELOMA TREATED WITH IBERDOMIDE PLUS DEXAMETHASONE IN A PHASE 1B/2A OPEN-LABEL DOSE ESCALATION STUDY

Background: Patients with relapsed/refractory multiple myeloma (RRMM) who are triple-class exposed (TCE) have limited treatment options and experience poor health-related quality of life (HRQoL). Iberdomide (IBER), a novel cereblon E3 ligase modulator (CELMoD®) compound, has shown an overall response rate of 26% in this population when administered with dexamethasone (DEX) in the open-label, single-arm, phase 1b/2a dose-escalation CC-220-MM-001 clinical trial (NCT02773030). Here, we assess the exploratory endpoint of HRQoL for patients enrolled in this clinical trial. Aims: To assess HRQoL in patients with RRMM who received IBER + DEX in cohorts D and I of the CC-220-MM-001 trial. Methods: In this pooled analysis of cohorts D and I, cohort D patients had RRMM; had received ≥3 prior lines of therapy, including lenalidomide, pomalidomide, a proteasome inhibitor, a glucocorticoid, and an anti-CD38 monoclonal antibody; had experienced disease progression within 60 days of last myeloma therapy; and were triple-class refractory. Patients who had received prior anti-B-cell maturation antigen (BCMA) therapy were excluded from cohort D but included in cohort I. In both cohorts, IBER (1.6 mg) was given orally on days 1–21, in combination with DEX (40 mg; 20 mg if >75 years) on day 1, 8, 15, and 22 of each 28-day cycle. The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 (QLQ-C30), EORTC QLQ-Multiple Myeloma Module (QLQ-MY20), and EuroQoL 5 dimensions 5 levels (EQ-5D-5L) were administered before treatment, on day 1 of each treatment cycle, and at end of treatment. The HRQoL-evaluable population included all patients who received IBER + DEX and who had evaluable assessments of a given HRQoL measure at baseline (cycle 1 day 1) and at ≥1 post-baseline time point. Minimally important differences were prespecified for each domain to interpret whether a mean within-group change from baseline was meaningful. Overall, least square mean HRQoL score changes from baseline were estimated from a mixed-effects model for repeated measures. Proportions of patients who experienced clinically meaningful HRQoL deterioration and improvement were calculated using prespecified responder definitions. Results: A total of 133 patients received IBER + DEX; 99 patients were included in the QLQ-C30-evaluable population (TCE n=79, TCE + anti-BCMA n=20). Median (range) age was 63 (44–82) years, 57% were male, and 95% had ≥4 prior MM regimens (median 6 prior MM regimens). Completion rates for most time points were ≥70%. Mean HRQoL scores were generally stable over time during treatment in all the HRQoL domains, including fatigue, pain, disease symptoms, and side effects. However, the EORTC QLQ-MY20 body image showed clinically meaningful improvement from baseline (Table). At individual level, most patients (≥60%) experienced either clinically meaningful improvement or no change while on treatment in all domains. Image:Summary/Conclusion: HRQoL scores among IBER + DEX patients in cohorts D and I were generally stable in all domains across 3 HRQoL measures. Most patients experienced either clinically meaningful improvement or no change in each domain. This suggests the IBER + DEX treatment maintained HRQoL in heavily pretreated patients with TCE RRMM, while demonstrating a promising clinical response.

P960: HEALTH-RELATED QUALITY OF LIFE IN THE LOCOMMOTION STUDY OF REAL-LIFE CURRENT STANDARD OF CARE IN PATIENTS WITH RELAPSED/REFRACTORY MULTIPLE MYELOMA

Background: Assessment of patient-reported outcomes (PRO) can inform how real-life standard of care (SOC) treatments affect health-related quality of life (HRQoL) for patients with relapsed/refractory multiple myeloma (RRMM). We present measures of symptoms, functioning, and overall HRQoL from LocoMMotion (NCT04035226), the first prospective, multinational study of real-life SOC in triple-class exposed patients with RRMM. Aims: To assess HRQoL in patients with RRMM receiving real-life current SOC in the LocoMMotion study. Methods: All patients provided informed consent. LocoMMotion is a noninterventional study across 76 sites (63 Europe, 13 United States) in patients who had received ≥3 prior lines of therapy (LOT) or were refractory to proteasome inhibitor (PI) and immunomodulatory drug (IMiD). Patients received PI, IMiD, and anti-CD38 monoclonal antibody and had disease progression during/after their last LOT. Real-life SOC treatments were defined as those used in local clinical practice. The following questionnaires were used: European Organisation for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30), 4 single items from EORTC QLQ-myeloma-specific module (EORTC QLQ-MY20), and EuroQol 5-Dimension 5-Level (EQ 5D-5L). HRQoL data were collected at baseline (BL), day 1 of each treatment cycle, end of treatment visit, and during follow-up (every 4 weeks). Established thresholds were used to evaluate improvement compared with BL health status. Mixed models for repeated measures were used to assess within-group change. Results: The questionnaire completion rate was 75.6% during SOC treatment in the LocoMMotion study (N=248; male: 54.4%; median age: 68 years; median cycles of SOC: 4.0 [range: 1-20]). Most patients did not achieve meaningful improvement (defined by a literature-based minimally important difference of 10 points in mean score) in PRO scores. This was most pronounced in pain symptoms, with 62% of patients showing no meaningful improvement during the first 3 months of treatment and 55% showing no improvement during the full treatment duration. Least square (LS) mean changes from BL during SOC treatment and subsequent LOT for the overall population are described (Table). Patients with ≥very good partial response during SOC treatment showed greater improvement in PRO scores, including LS mean change for pain score (-14.9 [95% confidence interval: -22.9, -7.0]). Image:Summary/Conclusion: This first prospective study of real-life current SOC in triple-class exposed patients with RRMM reported limited gains in HRQoL, most notably in pain symptoms. There is an urgent and unmet need for therapies that lead to deep responses and delayed disease progression, as these are associated with improvements in HRQoL.

P556: CHANGES IN HEALTH-RELATED QUALITY OF LIFE IN PATIENTS WITH NEWLY-DIAGNOSED ACUTE MYELOID LEUKEMIA RECEIVING IVOSIDENIB + AZACITIDINE OR PLACEBO + AZACITIDINE

Background: Ivosidenib (IVO) is a potent, targeted inhibitor of mutant isocitrate dehydrogenase 1 (mIDH1) that is approved for acute myeloid leukemia (AML). IVO plus azacitidine (AZA) demonstrated clinical benefit compared with placebo and AZA in the AGILE study (NCT03173248). Aims: To assess the impact of IVO+AZA versus placebo+AZA on health-related quality of life (HRQoL) using data from the AGILE study. Methods: In the double-blind, placebo-controlled phase 3 AGILE study, patients (pts) were randomized 1:1 to IVO 500 mg QD + AZA 75 mg/m2 SC or IV for 7 days in 28-day cycles, or placebo+AZA. HRQoL was a secondary endpoint assessed using two validated questionnaires: the European Organisation of Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30) and the EuroQol 5-dimension 5-level questionnaire (EQ-5D-5L). Questionnaires were administered pre-dose on cycle (C) 1 Day (D) 1, on C1D15, C2D1, C2D15, and on D1 of every odd cycle thereafter until the end of treatment. Score change from baseline across visits for all subscales of EORTC QLQ-C30 was analyzed with mixed models. A 10-point threshold in EORTC QLQ-C30 subscale score was used to evaluate clinically meaningful changes from baseline or differences between arms. Two-sided nominal p-values are reported. All patients gave written informed consent. Results: At baseline, 69 and 68 pts out of 72 receiving IVO+AZA completed the EORTC QLQ-C30 and EQ-5D-5L, respectively, and 66 pts out of 74 receiving placebo+AZA completed both. Mean baseline HRQoL scores were similar between treatment arms (EORTC QLQ-C30 global health status [GHS/QoL] score was 56.3 and 53.2 in the IVO+AZA and placebo+AZA arms, respectively). There was an initial decline in HRQoL (EORTC QLQ-C30 GHS/QoL) in both arms for ~4 months, consistent with time to response, and which was generally not clinically meaningful (Table). IVO+AZA was associated with preserved or improved HRQoL compared to baseline for most subscales of the EORTC QLQ-C30 from C5 to C19 (after which no placebo+AZA HRQoL data were available), and at most timepoints for EQ-5D-5L VAS scores and index values. EORTC QLQ-C30 subscales with clinically meaningful improvements from baseline at most timepoints from C5 to C19 in the IVO+AZA arm included GHS/QoL (Table), fatigue, pain and appetite loss. In contrast, there were few clinically meaningful improvements from baseline in placebo+AZA pts. GHS/QoL scores were significantly improved (p≤0.05) for IVO+AZA versus placebo+AZA at C2D1, C2D15, C7 and C9 (Table), and differences between treatment arms were clinically meaningful at C2D1, C2D15, C7, C9, C13, C15 and C19 (Table). Likewise, improvements in EORTC QLQ-C30 fatigue, appetite loss, nausea and vomiting, diarrhea, cognitive functioning and social functioning favored IVO+AZA over placebo+AZA at multiple timepoints. Image:Summary/Conclusion: Data from the AGILE study show that patients with mIDH1 AML receiving treatment with IVO+AZA tended to report maintenance or improved HRQoL from cycle 5 through to cycle 19 compared with placebo+AZA.

Health-related quality of life following FDG-PET/CT for cytological indeterminate thyroid nodules.

ObjectiveThis study assessed the health-related quality of life (HRQoL) in patients undergoing 2-[18F]fluoro-2-deoxy-D-glucose (FDG)-PET/CT for an indeterminate (Bethesda III/IV) thyroid nodule. FDG-PET/CT accurately rules out malignancy and prevents 40% of futile diagnostic surgeries in these nodules.DesignSecondary analyses of HRQoL data from a randomised controlled multicentre trial (NCT02208544) in 126 patients from 15 hospitals in the Netherlands were done.MethodsLongitudinal HRQoL assessment was performed using the EuroQol 5-dimension 5-level (EQ-5D-5L), the RAND 36-item Health Survey v2.0 (RAND-36), and the Thyroid Patient-Reported Outcome (ThyPRO) questionnaire on baseline, 3, 6, and 12 months, relative to the date of the FDG-PET/CT scan.ResultsPatients who were randomised to active surveillance following an FDG-negative nodule instead of diagnostic surgery reported stable HRQoL scores throughout the year. Univariate analysis indicated better HRQoL for patients undergoing surveillance than surgical patients with benign histopathology on multiple physical and psychosocial domains. Univariate within-group analysis suggested both temporary and continued HRQoL deteriorations in patients with benign histopathology over time. Multivariate within-group analysis demonstrated no significant longitudinal HRQoL changes in patients undergoing active surveillance. In contrast, in patients with benign histopathology, worse HRQoL was observed with regard to ThyPRO cognitive impairment (P = 0.01) and cosmetic complaints (P = 0.02), whereas goitre symptoms (P < 0.001) and anxiety (P = 0.04) improved over time. In patients with malignant histopathology, anxiety also decreased (P = 0.05).ConclusionsThe reassurance of a negative FDG-PET/CT resulted in sustained HRQoL throughout the first year of active surveillance. Diagnostic surgery for a nodule with benign histopathology resulted in more cognitive impairment and physical problems including cosmetic complaints, but improved goitre symptoms and anxiety. Anxiety was also reduced in patients with malignant histopathology.