EQ-5D Visual Analogue Scale Scores Research Articles

Health-related quality of life among patients after cardiac resynchronization therapy: a comparison between biventricular and left bundle branch area pacing

Abstract Background Cardiac resynchronization therapy (CRT) with biventricular pacing (BiVP) has the potential to improve cardiac function in patients with heart failure (HF). However, a portion of patients do not respond to therapy. Recently, left bundle branch area pacing (LBBAP) has emerged as a more physiological alternative for CRT in selected cases (1,2). Quality of life (QoL) tools measures subjective experience and its results are relevant as they may help to assessed the impact that medical interventions have in patient's health. Purpose The authors sought to measure and compare the quality of life in a cohort of patients after the implantation of either a BiVP or a LBBaP CRT device. Methods A multicenter prospective quasi-experimental study in which consecutive patients underwent CRT was conducted. According to the study protocol, the four centers would perform their usual clinical practice: two out of them would perform conventional BiVP-CRT while the other half LBBAP-CRT. The health related QoL was measured by the Spanish version of the EuroQol Group’s five-level EuroQol five-dimensional questionnaire (EQ-5D-5L), which the patients were asked to complete at baseline and 12 weeks after the CRT device implantation. The descriptive system section of the EQ-5D-5L questionnaire produces a 5-digit health state profile that can be converted into a single index value. The EQ-5D-5L Spanish index values range from -0.225 to 1.0 (full health). The EQ-5D visual analogue scale (EQ-VAS) is a vertical scale numbered from 0 to 100 (the worst and the best health you can imagine). Both groups (BiVP and LBBaP) were compared with data from the general population of Spain for the EQ-5D-5L and EQ-VAS (n=20.587) according to the national survey (3). Results The study included 44 consecutive patients (31 males, mean age 66 ± 11 years, LVEF 28.9% ± 6.9%). The LBBAP procedure was finally performed in 27 patients, while BiVP was the therapy performed in the other 17 patients. Crossover rate was less than 12% (1 patient underwent rescue BiVP and 2 patients switched to LBBAP). The basal characteristics of included patients are presented in Table 1. Among the individuals with BiVP the mean EQ-5D-5L index score and the EQ-VAS score were 0.74±0.24 and 65.71±17.90 respectively at baseline while they were 0.83±0.19 [+0.08±0.16, p 0.054, CI 95%] and 76.59±15.76 three months later [+10.80±15.38, p 0.01, CI 95%]. In the LBBAP group, the mean EQ-5D-5L index score and the EQ-VAS score were 0.71±0.25 and 50.04±25.49 respectively at baseline while they were 0.80±0.19 [+0.09±0.22, p 0.044, CI 95%] and 69.30±23.53 [+19.26±22.83, p 0.0001, CI 95%] three months later. Conclusion It was found that the EQ-5D-5L index value and EQ-VAS improved in the LBBAP group. In the BiVP group only EQ-VAS improved although there was also a non-statistically significant trend in EQ-5D-5L index score. The main limitation of the study was the small sample size.Table 1Figure 1

Real-world treatment patterns and quality of life among patients with locally advanced or metastatic urothelial carcinoma living in Saudi Arabia, South Korea, Taiwan, and Turkey.

To evaluate demographic and clinical characteristics, treatment patterns, and quality of life in patients with locally advanced or metastatic urothelial carcinoma in Asia. Data were drawn from the Adelphi Real World Metastatic Urothelial Carcinoma Disease Specific Programme™, a cross-sectional survey of medical oncologists/urologists and their adult patients in Saudi Arabia, South Korea, Taiwan, and Turkey. Exploratory patient-reported outcomes included the EQ-5D visual analog scale, European Organisation for Research and Treatment of Cancer Quality of Life of Patient Questionnaire global health, and Brief Pain Inventory. Analyses were descriptive. Overall, 175 physicians reported data for 988 patients. Mean (standard deviation) patient age was 66.3 (10.8) years, 77% were men, and 82% had bladder tumors at diagnosis. Of patients receiving first- (n = 988), second- (n = 290), and third-line (n = 87) treatments, 81%, 35%, and 59% received chemotherapy, respectively, and 17%, 63%, and 34% received programmed cell death protein 1/ligand 1 inhibitors, respectively. Patient-reported (n = 319) mean (standard deviation) EQ-5D visual analog scale score was 51.8 (15.6), European Organisation for Research and Treatment of Cancer Quality of Life of Patient Questionnaire global health status score was 44.6 (19.9), and Brief Pain Inventory score was 6.5 (1.9; n = 315). The most common first- and second-line treatments for locally advanced or metastatic urothelial carcinoma were chemotherapy and programmed cell death protein 1/ligand inhibitors, respectively. At third line, 10% of patients received best supportive care alone, underscoring an unmet need for effective third-line treatment options. Patients in all regions reported quality-of-life impairment.

Health Outcomes Following Engagement With a Digital Health Tool Among People With Prediabetes and Type 2 Diabetes: Prospective Evaluation Study.

Diabetes is a worldwide chronic condition causing morbidity and mortality, with a growing economic burden on health care systems. Complications from poorly controlled diabetes are associated with increased socioeconomic costs and reduced quality of life. Smartphones have become an influential platform, providing feasible tools such as health apps to deliver tailored support to enhance the ability of patients with diabetes for self-management. Gro Health is a National Health Service division X-certified digital health tool used to deliver educational and monitoring support to facilitate the development of skills and practices for maintaining good health. This study aims to assess self-reported outcomes of the Gro Health app among users with diabetes and prediabetes and identify the factors that determine engagement with the digital health tool. This was a service evaluation of self-reported data collected prospectively by the developers of the Gro Health app. The EQ-5D questionnaire is a standardized tool used to measure health status for clinical and economic appraisal. Gro Health users completed the EQ-5D at baseline and 6 months after using the app. Users provided informed consent for the use of their anonymized data for research purposes. EQ-5D index scores and visual analogue scale (VAS) scores were calculated at baseline and 6 months for individuals with prediabetes and type 2 diabetes. Descriptive statistics and multiple-regression models were used to assess changes in the outcome measures and determine factors that affected engagement with the digital tool. A total of 84% (1767/2114) of Gro Health participants completed EQ-5D at baseline and 6 months. EQ-5D index scores are average values that reflect people's preferences about their health state (1=full health and 0=moribund). There was a significant and clinically meaningful increase in mean EQ-5D index scores among app users between baseline (0.746, SD 0.23) and follow-up (0.792, SD 0.22; P<.001). The greatest change was observed in the mean VAS score, with a percentage change of 18.3% improvement (61.7, SD 18.1 at baseline;73.0, SD 18.8 at follow-up; P<.001). Baseline EQ-5D index scores, age, and completion of educational modules were associated with significant changes in the follow-up EQ-5D index scores, with baseline EQ-5D index scores, race and ethnicity, and completion of educational modules being significantly associated with app engagement (P<.001). This study provides evidence of a significant positive effect on self-reported quality of life among people living with type 2 diabetes engaging with a digital health intervention. The improvements, as demonstrated by the EQ-5D questionnaire, are facilitated through access to education and monitoring support tools within the app. This provides an opportunity for health care professionals to incorporate National Health Service-certified digital tools, such as Gro Health, as part of the holistic management of people living with diabetes.

Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L

BackgroundThe progression of Duchenne muscular dystrophy (DMD) is characterized by loss of ambulation, respiratory insufficiency, cardiomyopathy, and early mortality. DMD profoundly impacts health-related quality-of-life (HRQoL). However, few health state utility data exist; published utilities tend to be derived from small samples for a limited number of health states and are often based on caregiver-reported patient health status. This study estimated utility values for varied clinical and functional health states in DMD, based on patient-reported health status.MethodsIndividuals with DMD in the US aged 12–40 years completed the EQ-5D (5-level) and Health Utilities Index (HUI) preference-based instruments. Based on responses to a clinical questionnaire, participants self-classified into functional health states according to level of lower and upper limb function, use of respiratory support, and presence of cardiomyopathy. Mean [standard deviation (SD)] utility and EQ-5D visual analogue scale (VAS) scores were estimated according to health state; and median (interquartile range) attribute levels calculated to understand which domains of health are most severely affected in DMD.ResultsOf 63 males with DMD, mean (SD) age was 19.8 (6.1) years and 11 (17.5%) were ambulatory. Mean (SD) utility values were 0.92 (0.08; HUI2), 0.84 (0.20; HUI3), and 0.84 (0.13; EQ-5D) for ambulatory patients without cardiomyopathy (n = 10). For non-ambulatory patients with moderately impaired upper limb function, night and daytime ventilation without cardiomyopathy, mean (SD) utilities were 0.49 (0.07) for the HUI2, 0.16 (0.15) for the HUI3 and 025 (0.14) for the EQ-5D. Mean (SD) VAS scores for the same health states were 91 (9) and 83 (21), respectively. In addition to impairments in mobility/ambulation, and self-care, attributes like usual activities and pain also showed notable effects of DMD.ConclusionsIn DMD, although a relationship between disease progression and HRQoL is observed, there is large variability in utility within functional health states, and across instruments. Utility values for less severe non-ambulatory health states described by level of upper limb function are novel. These utility values, derived based on direct patient feedback rather than from caregiver report, are relevant to individuals of varying functional statuses and augment scarce DMD-specific utility data.

Experience with Emicizumab Among People with Hemophilia A in the Canadian Hemophilia Bleeding Disorders Registry

Background: The use of emicizumab as prophylaxis to prevent bleeding or reduce the frequency of bleeding episodes in congenital hemophilia A (HA) was approved by Health Canada for the treatment of people living with HA (PwHA) with FVIII inhibitors in 2018, and without inhibitors in 2019. Here we present the results of a non-interventional, cohort study, using data routinely collected by the Canadian Bleeding Disorders Registry (CBDR) to evaluate treatment patterns of emicizumab in Canada and the associated safety and effectiveness outcomes, including quality of life. Methods: De-identified data were extracted from the CBDR database for all registered PwHA who had received emicizumab at least once prior to December 31, 2022. Baseline demographic characteristics were stratified by disease severity (defined by the level of endogenous FVIII activity) and age. Effectiveness outcomes were measured by the proportion of patients with zero, traumatic, spontaneous, or joint bleeds as well as annualized bleeding rates (ABR) for any treated bleed. Intra-patient comparisons of bleeding were performed for all patients and for those with at least six months follow-up in both pre- and post-emicizumab periods as a sensitivity analysis. Effectiveness outcomes also included patients' Hemophilia Joint Health Score (HJHS), Patient Reported Outcomes, Burdens and Experiences (PROBE), and EQ-5D index and visual analog scale (VAS) scores. We performed a complete data analysis, without imputation. This study was approved by the research ethics board of McMaster University and other participating centers, and abides by the guiding principles of the Declaration of Helsinki. Results: In total, 533 PwHA received at least one dose of emicizumab (PwHA E) in the CBDR database. Overall, 498 (93.4%) PwHA E had severe disease, 28 (5.3%) had moderate disease, and 7 (1.3%) had mild disease. At start of emicizumab exposure, 220 (41.3%) PwHA E were &amp;lt;18 years old; 78 (14.6%) PwHA E had current FVIII inhibitors, 82 (15.4%) had a history of FVIII inhibitors, and 373 (70%) had no history of inhibitors ( Table 1). A total of 13 adverse events were reported in the entire observation period, including one event of thrombosis (occurring in one person with COVID-19), seven allergic reactions, two FVIII Inhibitor development, two neurological events (headache; and headache, nausea, and vomiting) and one case of large hematoma developed at operative site following removal of port-a-cath. Moreover, nine COVID-19 cases were reported as adverse events. Over a median (first quartile, third quartile) follow-up time of 249 (136, 395) days, 388 (72.8%) PwHA E had no recorded bleeds. Out of 145 PwHA E with recorded bleeds, 106 had joint bleeds, 87 had traumatic bleeds, 69 had spontaneous bleeds and 46 had other bleeds (Table 2). An intra-patient comparison in all PwHA E (n=533) showed a decrease in mean (95% confidence interval [CI]) annualized bleeding rate (ABR) from 1.22 (1.06, 1.42), pre-emicizumab (2018, prior to first dose) to 0.50 (0.41, 0.60) post-emicizumab (rate ratio (95% CI) 0.41 (0.33, 0.50), p-value &amp;lt;0.001). The Intra-patient comparison in PwHA E without current inhibitor (n=455) showed a decrease in mean ABR (95% CI) from 1.02 (0.87, 1.19) to 0.54 (0.44, 0.66) (rate ratio (95% CI) 0.53 (0.43, 0.65), p-value &amp;lt;0.001). The Intra-patient comparison in PwHA E with current inhibitor (n=78) showed a decrease in mean ABR (95% CI) from 5.03 (3.30, 7.66) to 0.41 (0.26, 0.65) (rate ratio (95% CI) 0.08 (0.04, 0.15), p-value &amp;lt;0.001). Conclusions: This analysis describes the baseline characteristics, safety and effectiveness outcomes of Canadian PwHA treated with emicizumab before December 31, 2022. A total of 387 new patients were included in this analysis compared to the previous one (data cut: December 31, 2021, n=146). The data shows that 72.8% PwHA E had no recorded bleeds since they started emicizumab and that there was a substantial decrease in bleeds post-emicizumab in both PwHA with and without current FVIII inhibitor. The safety profile of emicizumab was consistent with previous reports. The CBDR allows for a longitudinal follow-up of the Canadian hemophilia A population, which can inform healthcare practitioners and regulatory authorities of the safety and efficacy outcomes of treatments in routine clinical practice.

Extreme Lateral Interbody Fusion: An Observational Study of Functional and Radiological Outcome

Introduction: Extreme lateral interbody fusion (XLIF) constitutes a minimally invasive procedure employed to address a spectrum of conditions including degenerative disc disorders, trauma, infections, and deformities. Despite its potential, there exists a scarcity of studies exploring perioperative functional and radiological outcomes. This prospective observational study seeks to address this gap by reporting these outcomes in patients treated at the King Faisal Specialist Hospital &amp; Research Centre (KFSHRC) over a mean follow-up period of 5 years.&#x0D; Methods This case series amassed baseline patient data, encompassing gender, age, body mass index (BMI), American Society of Anesthesiology (ASA) classification, operated level, and posterior fixation type. Primary outcome measures encompassed the Oswestry Disability Index (ODI), Roland-Morris Disability Index (RMDI), Euro-Qol (EQ)-5D, visual analog scale (VAS), and EQ-5D index scores. Additionally, the study delved into secondary outcomes encompassing radiological parameters such as sagittal balance, lumbar lordosis, sacral slope, pelvic tilt, and pelvic incidence.&#x0D; Results: The study embraced 51 patients, comprising 9 females and 42 males, with a mean age of 58.6 years and a mean BMI of 29.3. The patients were followed for an average of 5.2 years. Stratification according to ASA classification revealed that 11.8% of patients were classified as ASA I, 52.9% as ASA II, and 35.3% as ASA III. The levels of operation included 60.8% at L4-L5, 33.3% at L3-L4, and 5.9% at L2-L3. The fixation techniques employed encompassed 58.8% bilateral and 41.2% unilateral approaches. The analysis of the primary clinical outcomes unveiled statistically significant enhancements in ODI, RMDI, EQ-5D VAS, and EQ-5D index scores. While lumbar lordosis registered a significant decrease, radiological parameters indicated non-significant shifts in pelvic incidence, pelvic tilt, and sacral slope.&#x0D; Conclusion The XLIF procedure emerges as an efficacious and minimally invasive avenue for managing degenerative disc disorders. The reported functional and radiological outcomes prove satisfactory. However, while demonstrably effective, the generation of clinical recommendations necessitates the compilation of results from randomized clinical trials.

Shoulder problems after percutaneous antegrade intramedullary nailing in humeral diaphyseal fractures using contemporary straight third-generation nail.

Antegrade intramedullary nailing in humeral shaft fracture has been abandoned by certain orthopedic surgeons because of rotator cuff injury caused by first- and second-generation intramedullary nails (IMNs). However, only a few studies have specifically addressed the results of antegrade nailing for the treatment of humeral shaft fractures with a straight third-generation IMN; thus, complications need to be re-evaluated. We hypothesized that fixation of displaced humeral shaft fractures with a straight third-generation antegrade IMN with the percutaneous technique avoid shoulder problems (stiffness and pain) incurred by first- and second-generation IMNs. This was a retrospective, single-center, nonrandomized study of 110 patients with a displaced humeral shaft fracture between 2012 and 2019 treated surgically with a long third-generation straight IMN. Mean follow-up was 35.6 months (range, 15-44 months). There were 73 women and 37 men with a mean age of 64.7±19 years. All fractures were closed (37.3% 12A1, 13.6% 12B2, and 13.6% 12B3 AO/OTA classification). Mean Constant score was 82±19, Mayo Elbow Performance Score 96±11 and the mean EQ-5D visual analog scale score was 69.7±21.5. Mean forward elevation 150°±40°, abduction 148°±45°, and external rotation 38°±15°. Symptoms associated with rotator cuff disease were present in 6.4%. Evidence of radiographic fracture healing was detected in all but 1 case. One postoperative nerve injury and 1 adhesive capsulitis were present. Overall, 6.3% underwent second surgeries (4.5% were minor surgeries like hardware removal). Percutaneous antegrade intramedullary nailing of humeral shaft fractures with a straight third-generation nail considerably reduced complications related to shoulder problems and achieved good functional results.

The Subglottic Stenosis 6 Questionnaire: A Novel Quality-of-Life Survey Validated to Predict Need for Intervention in Patients With Idiopathic Subglottic Stenosis

Idiopathic subglottic stenosis (iSGS) is a recurrent, chronic disease defined by fibroinflammatory narrowing of the subglottic airway. A key challenge in treatment is monitoring disease progression, which may be debilitating and unpredictable in its timing. Can the Subglottic Stenosis 6 (SGS-6) questionnaire be validated as a novel quality-of-life (QOL) instrument to monitor breathing, disease progression, and disease severity proactively in patients with iSGS? Panel data from 51 patients with iSGS were collected from January 2012 through June 2022, representing 1,684 patient encounters including routine office visits and treatment encounters. Subjective QOL scores (including the novel SGS-6 and established RAND-36 and EuroQol Five Dimensions [EQ-5D] Visual Analog Scale) and objective pulmonary function test (PFT) results were collected at each visit. Subjective SGS-6 QOL scores were repeated within 1week of initial reporting. Panel regression analyses were performed to assess the relationship between SGS-6 scores, PFT results, and a patient's need for intervention. Minimal clinically important differences (MCIDs) for SGS-6 and peak expiratory flow percentage (PEF%) were assessed using receiver operating characteristic (ROC) curve analysis and a patient's need for intervention as the external anchor. Each one-point increase in SGS-6 score (of a maximum of 27) was associated with a 3.26%decrease in PEF%, a 1.93-point decrease in RAND-36 Physical Health composite score, a 1.27-point decrease in RAND-36 Mental Health composite score, and a 0.88-point decrease in EQ-5D Visual Analog Scale score. The intracorrelation coefficient for the SGS-6 composite score is 0.838 (95%CI, 0.770-0.888). Compared with patient baselines, SGS-6 scores were 4.66 points greater at the time of intervention with an MCID of 2.25 from a patient's baseline. The area under the ROC curve for SGS-6 and a patient's intervention point was 0.81. iSGS disease severity can be modeled using the SGS-6 questionnaire, offering physicians and patients a potentially new method of tracking disease progression and need for intervention remotely.

Impact of recurrent ischaemic and bleeding events on quality of life in patients with acute coronary syndrome: Insights from the FORCE-ACS registry

ObjectivePatients with acute coronary syndrome (ACS) remain at high risk for recurrent ischaemic and bleeding events during follow-up. Our study aimed to quantify and compare the impact of these adverse...

Quality-adjusted survival in women with gynecologic malignancies receiving IMRT after surgery: A Ppatient Rreported Ooutcome study of NRG oncology's RTOG 1203

IntroductionNRG/RTOG 1203 compared 3-D conformal radiotherapy (3D CRT) to intensity-modulated radiotherapy (IMRT) in patients with endometrial or cervical cancer requiring post-operative radiotherapy after hysterectomy. The purpose of this study was to report the first quality-adjusted survival analysis comparing the two treatments. MethodsNRG/RTOG 1203 randomized patients having undergone hysterectomy to either 3DCRT or IMRT. Stratification factors included RT dose, chemotherapy, and disease site. The EQ-5D, both index and visual analog scale (VAS), were obtained at baseline, 5 weeks after the start of RT, 4–6 weeks post RT and 1 and 3-years post RT. EQ-5D index and VAS scores along with quality-adjusted survival (QAS) were compared between treatment arms using the t-test at a two-sided significance level of 0.05. ResultsNRG/RTOG 1203 enrolled 289 patients of which 236 consented to participate in the patient reported outcome (PRO) assessments. QAS was higher in women treated with IMRT, 1374 vs 1333 days (p = 0.5) compared to patients treated with 3DCRT, but this difference was not statistically different. Patients treated with IMRT had less of a decline in VAS score 5 weeks post RT, −5.04, compared to patients treated with 3DCRT, −7.48, although not statistically significant (p = 0.38). ConclusionThis is the first report of the use of the EQ-5D comparing two radiotherapy techniques in the treatment of gynecologic malignancies after surgery. While there were no significant differences in QAS and VAS scores between patients who received IMRT vs. 3DCRT, RTOG 1203 was not powered to show statistical differences in these secondary endpoints.

Quality of Life and Associated Factors of HIV Patients Under Treatment with First Line Regimens in Public Hospitals in Amhara Region, North-West Ethiopia.

The availability of medication related to HIV treatment in the world is one of the substantial improvements for reaching USAID's 90-90 targets. Among the 90% of patients who have awareness about their disease, 90% are accessing their treatment and patients who received appropriate treatment have a suppressed viral load and improved CD4 cell count. Therefore, the main objective of the current study was to investigate the quality of life and associated factors of people living with HIV receiving first-line regimens at public hospitals in the Amhara region, Ethiopia. A retrospective cohort study was conducted on 700 adult HIV-infected patients under treatment with first-line regimens, who were followed-up in 17 public hospitals in the Amhara region. A multivariate linear regression analysis was used for the current study. Of the 700 patients included in the current analyses, 59.5% (n=358) reported no impairment in self-care, while 63.1% (n=380) were extremely anxious/depressed. The overall expected EQ-5D utility score and visual analog scale (EQ-VAS) scores were 0.3880.41 and 66.2017.22 respectively. The current study indicated that the covariates sex, age of patient, level of education, appointment frequency, disclosure status of the disease, and substance use significantly affected the quality of life of people living with HIV and under treatment with first-line regimens. Hence, higher CD4 cell count and less detectable viral load lead to good quality of life of people living with HIV. This study indicates that certain covariates have been identified as statistically significant predictors of the study variable "quality of life" of HIV-positive people. The findings obtained in the current investigation can help policy-makers to revise the current directives. The result obtained in this study can also help health staff to conduct health-related education during the treatment of HIV patients.

Targeted Hypoglossal Nerve Stimulation for Patients With Obstructive Sleep Apnea

Evidence is lacking from randomized clinical trials of hypoglossal nerve stimulation in obstructive sleep apnea (OSA). To evaluate the safety and effectiveness of targeted hypoglossal nerve stimulation (THN) of the proximal hypoglossal nerve in patients with OSA. This randomized clinical trial (THN3) was conducted at 20 centers and included 138 patients with moderate to severe OSA with an apnea-hypopnea index (AHI) of 20 to 65 events per hour and body mass index (calculated as weight in kilograms divided by height in meters squared) of 35 or less. The trial was conducted from May 2015 through June 2018. Data were analyzed from January 2022 through January 2023. Implant with THN system; randomized 2:1 to activation at month 1 (treatment) or month 4 (control). All received 11 months of THN with follow-up at months 12 and 15, respectively. Primary effectiveness end points comprised AHI and oxygen desaturation index (ODI) responder rates (RRs). Treatment responses at months 4 and 12/15 were defined as a 50% or greater reduction in AHI to 20 or less per hour and an ODI decrease of 25% or greater. Coprimary end points comprised (1) month 4 AHI and ODI RR in the treatment greater than the control group and (2) month 12/15 AHI and ODI RR in the entire cohort exceeding 50%. Secondary end points included sleep apnea severity (AHI and ODI) and patient-reported outcomes (Epworth Sleepiness Scale, Functional Outcomes of Sleep Questionnaire, and EQ-5D visual analog scale). Among 138 participants, the mean (SD) age was 56 (9) years, and 19 (13.8%) were women. Month 4 THN RRs were substantially greater in those in the treatment vs control group (AHI, 52.3% vs 19.6%; ODI, 62.5% vs 41.3%, respectively) with treatment-control standardized mean differences of 0.725 (95% CI, 0.360-1.163) and 0.434 (95% CI, 0.070-0.843) for AHI and ODI RRs, respectively. Months 12/15 RRs were 42.5% and 60.4% for AHI and ODI, respectively. Improvements in AHI, ODI, Epworth Sleepiness Scale, Functional Outcomes of Sleep Questionnaire, and EQ-5D visual analog scale scores were all clinically meaningful (medium to large effect size). Two serious adverse events and 100 nonserious related adverse events were observed from the implant procedure or study protocol. This randomized clinical trial found that THN demonstrated improvements in sleep apnea, sleepiness, and quality of life in patients with OSAs over an extended AHI and body mass index range without prior knowledge of pharyngeal collapse pattern. Clinically meaningful improvements in AHI and patient-reported responses compared favorably with those of distal hypoglossal nerve stimulation trials, although clinically meaningful differences were not definitive for ODI. ClinicalTrials.gov Identifier: NCT02263859.

Predictors of quality of life in patients within the first year of commencing haemodialysis based on baseline data from the PIVOTAL trial and associations with the study outcomes.

Impaired quality of life is common in patients with end-stage kidney disease. We report the baseline quality of life measures in participants from the PIVOTAL randomized controlled trial and the potential relationship with the primary outcome (all-cause mortality, myocardial infarction, stroke, and heart failure hospitalisation), and associations with key baseline characteristics. This was a post hoc analysis of 2141 patients enrolled in the PIVOTAL trial. Quality of life was measured using EQ5D index, Visual Analogue Scale, and the KD-QoL [Physical Component Score and Mental Component Score]. Mean baseline EQ5D index and visual analogue scale scores were 0.68 and 60.7 and 33.7 (Physical Component Score) and 46.0 (Mental Component Score), respectively. Female sex, higher Body Mass Index, diabetes mellitus, history of myocardial infarction, stroke or heart failure were associated with significantly worse EQ5D index and visual analogue scale. Higher C-reactive protein levels and lower transferrin saturation were associated with worse quality of life. Haemoglobin was not an independent predictor of quality of life. A lower transferrin saturation was an independent predictor of worse physical component score. A higher C-reactive protein level was associated with most aspects of worse quality of life. Impaired functional status was associated with mortality. Quality of life was impaired in patients starting haemodialysis. A higher C-reactive protein level level was a consistent independent predictor of the majority of worse quality of life. Transferrin saturation ≤ 20% was associated with worse physical component score of quality of life. Baseline quality of life was predictive of all-cause mortality and the primary outcome measure. 2013-002267-25.

Impact of major age-related eye disorders on health-related quality of life assessed by EQ-5D: a systematic review and meta-analysis.

This study is to quantitatively estimate the health-related quality of life (HRQOL) impact of major age-related eye diseases (AREDs) including cataract, glaucoma, age-related macular degeneration (AMD), and diabetic retinopathy (DR) assessed by the EuroQoL Five-Dimensional Questionnaire (EQ-5D). PubMed, Embase, Cochrane Library, and CINAHL were searched until October 20, 2022. Studies were included if they reported the EQ-5D health utility score (HUS) or visual analogue scale (VAS) score of both AREDs patients and healthy controls. The mean difference (MD) in HUS or VAS score between cases and controls and its 95% confidence interval (95%CI) were pooled using the random-effects model. We also performed sensitivity analysis using the leaving-one-out method and subgroup analyses by sample size and race. The prevalence in reporting any problems in the five EQ-5D dimensions was summarized and compared between cases and controls using the Chi-square test. Fifteen articles involving 30,491 participants were included in this review. Pooled estimates indicated reduced HUS in AMD patients (MD = -0.04, 95%CI -0.07, -0.01; P = 0.009), DR patients (MD = -0.03, 95%CI -0.05, -0.01; P = 0.01), and glaucoma patients (MD = -0.06, 95%CI -0.10, -0.01; P = 0.01), compared with the controls. Significantly lower EQ-5D VAS score was also observed in cataract patients (MD = -11.33, 95%CI -13.47, -9.18; P < 0.001) and DR patients (MD = -6.41, 95%CI -10.64, -2.18; P = 0.003). AREDs patients reported usual activities and anxiety/depression problems more frequently than the control group. Our findings confirmed the HRQOL impairment caused by major AREDs including AMD, cataract, DR, and glaucoma. High-quality studies with large sample sizes are warranted to further verify our results.

Abstract P175: Shared Medical Appointments in Heart Failure for Post-Hospitalization Follow-Up

Introduction: Shared medical appointments (SMAs) are medical visits where several patients with a common disease have clinic at the same time with medical provider(s). SMAs in heart failure (HF) is a unique opportunity where providers of different disciplines, one of which is a prescriber with HF expertise, can improve the health of patients recently discharged with HF. Hypothesis: Compared with usual care, patients discharged with HF randomized to a HF-SMA will experience better cardiac health status as measured by Kansas City Cardiomyopathy Questionnaire (KCCQ) and overall health status as measured by the EQ-5D Visual Analog Scale (EQ5D-VAS) Methods: We conducted a 3-site open label randomized-controlled trial of parallel design. Participants within 12 weeks of a HF hospitalization were randomized to receive either HF-SMA versus usual care. The HF-SMA team consisted of a nurse, nutritionist, psychologist, nurse practitioner and/or a clinical pharmacist and included four 2 hour sessions that met every other week for 8 weeks. Primary outcomes are overall health status measured by EQ5D-VAS (range 0-100 with 0 = worst health and 100 = Best health) and cardiac health status by KCCQ (range 0-100, higher score is better cardiac health), after 180 days post randomization. Results: Of the 242 patients enrolled (mean age 69.3±9.4 years, 71.5% white, 94.6% male), 83.8% HF-SMA and 84.8% usual care participants completed the study. Although both groups had significant improvements from baseline in their overall health status, patients in the HF-SMA intervention experienced a greater improvement in EQ5D-VAS scores than usual care (change = 7.2 ± 15.8 versus -0.4 ± 19.0 points, p&lt;0.001, respectively). There were no significant differences between the groups in the cardiac health status KCCQ Summary or Clinical Scores. Conclusion: HF-SMA achieved higher Improvements in overall health status than usual care but not in cardiac health status. Future studies should power to compare data on event outcomes to better understand impact.

Health-Related Quality of Life (HRQoL) in Tuberculosis Patients: a Cross-Sectional Study Using the EuroQoL EQ-5D-3L Scale

The measurement of health-related quality of life (HRQoL) of pulmonary tuberculosis (TB) patients is important since TB significantly affects the patient’s health and sufferings worldwide. HRQoL indicates the consciousness of patients regarding their physical and mental health. It is, therefore, very relevant in comprehending and measuring the exact impact of the disease state. The current study assessed the HRQoL of TB patients and the correlation between the EQ-5D index score and visual analogue scale (VAS) score. A prospective, cross-sectional study was conducted in February–March, 2022, at Rawalpindi Leprosy Hospital. A total of 400 patients with TB were included in the study. HRQoL was measured by using a validated Urdu version of EuroQol 5 dimensions 3 level (EQ-5D-3L) and visual analogue scale (EQ-VAS). The overall mean score of EQ-5D and the visual analogue scale (VAS) score were (0.56 ± 0.43) and (65.56 ± 17.02), respectively. The correlation coefficient of r = 0.521 between VAS and EQ-5D indicates that our results are statistically significant. Multivariate linear regression analysis implied that gender and education have a significant impact on the patient quality of life. The current study indicated that tuberculosis negatively affects the mental health, social function, and emotional aspects of the patient’s quality of life. The HRQoL data from this study could be used to design future interventions and help prescribers to gain insight into the factors in which patient’s health-related quality of life can be improved.

Toward the Definition of Patient-Reported Outcome Measurements in Hereditary Spastic Paraplegia.

Hereditary spastic paraplegias (HSPs) are a heterogeneous group of rare neurodegenerative diseases, characterized by a progressive spastic paraparesis. Currently, there is a HSP-specific clinician-reported outcome measure (CROM) called Spastic Paraplegia Rating Scale (SPRS). There are, however, no specific patient-reported outcome measures (PROMs) for HSP. In the present cohort study, we prospectively follow up a well-examined Austrian HSP cohort using validated rating scales and compared PROM with disease-specific and non-disease-specific CROM. Patients were recruited and followed up at the Center for Rare Movement Disorders, Innsbruck, Austria. CROM included the SPRS, Scale for the Assessment and Rating of Ataxia (SARA), Barthel Index (BI), and Mini-Mental State Examination (MMSE). PROM included the EQ-5D questionnaire and the Patient Health Questionnaire 9 (PHQ-9). Standardized response means (SRMs) were calculated for all scales at follow-up (FU) after 1 year. A total of 55 patients (36 males) with HSP were included in the study. FU was performed for 30 patients (21 males). Apart from females reporting more problems in the EQ-5D domain of anxiety and depression (p = 0.008), other clinician-reported outcomes (CROs) or patient-reported outcomes (PROs) did not differ significantly across sex. SPRS showed significant correlations with SARA (p < 0.001), mainly driven by the gait item, as well as the BI. Although SPRS did not correlate with EQ-5D visual analogue scale and PHQ-9 scores, several EQ-5D domains correlated significantly with SPRS. At FU, SPRS showed the highest responsiveness (SRM 1.11), followed by SARA (SRM 0.47). Neither MMSE nor PRO significantly increased at FU. In this study, we present an Austrian cohort of patients with HSP and a prospective study evaluating correlations of CRO and PRO as well as their progression. Demographics from our cohort are comparable with several other European cohort studies. Our data highlight the capabilities of the SPRS to show clinical progression and warrant consideration of ataxia rating scales such as SARA in HSP cohorts. We also show that the generic PROMs are not suitable to detect change in HSP, and thus, we propose to create a disease-specific PROM fully depicting the effect of HSP on the patients' lives.

Real-World Outcomes of Emicizumab in Hemophilia A with or without FVIII Inhibitors from the Canadian Hemophilia Bleeding Disorder Registry

Background: Hemophilia A is a bleeding disorder characterized by deficiency or absence of blood coagulation factor (F)VIII, which leads to lifelong bleeding tendency. The Canadian Bleeding Disorders Registry (CBDR) is a clinical database designed to assist clinicians in Canadian hemophilia treatment centers with the management of people with bleeding disorders and gain a better understanding of their incidence and prevalence, their treatment patterns, outcomes, and safety. Emicizumab is a bispecific monoclonal antibody that bridges activated FIX and FX to substitute for missing activated FVIII in people with hemophilia A (PwHA), thereby restoring hemostasis. Emicizumab was approved by Health Canada for the treatment of PwHA with FVIII inhibitors in 2018, and for PwHA without FVIII inhibitors in 2019. In this non-interventional, secondary data use cohort study, we use data routinely collected by the CBDR to evaluate the effectiveness, safety, and treatment patterns of emicizumab in Canada. The study includes patients treated according to routine clinical practice; therefore, it reflects patterns of care and outcomes in the real world, outside of the experimental setting. Methods: We retrieved de-identified data from the CBDR database of all registered PwHA who had received emicizumab at least once prior to December 31, 2021. Baseline demographic characteristics, including sex, body mass index, FVIII inhibitor status, emicizumab regimen, and immune tolerance induction (ITI) while receiving emicizumab were stratified by disease severity (defined by the level of endogenous FVIII activity) and age. Real-world efficacy outcomes were measured by the proportion of patients with zero traumatic, spontaneous, or joint bleeds as well as annualized bleeding rates (ABR) for any treated bleed. ABRs were calculated as (total number of bleeds/duration of follow-up [days])*365.25. Efficacy outcomes also included patients’ Hemophilia Joint Health Score (HJHS), Patient Reported Outcomes Burdens and Experiences (PROBE), and EQ-5D index and visual analog scale (VAS) scores. We performed a complete data analysis, without imputation. Intra-patient comparisons of bleeding were performed for all patients This study was approved by the research ethics board of McMaster University and other participating centers, and abides by the guiding principles of the Declaration of Helsinki. Results: In total, 146 PwHA received emicizumab at least once up to December 2021 in the CBDR database. The median (Q1, Q3) age at initiation of emicizumab was 13.9 (6.7, 28.7) years; 58.9% (n=86) of PwHA were ≤18 years (Table 1). Overall, 132 PwHA had severe disease, 10 had moderate disease, and 4 had mild disease. In total, 40.4% (n=59) of PwHA had current FVIII inhibitors, 15.1% (n=22) had a history of FVIII inhibitors, and 44.5% (n=65) had no history of inhibitors; 4.1% (n=6, all with severe HA) received ITI while on emicizumab. Three adverse events were reported during 2021, including two allergic reactions and one case of large hematoma at the operative site following removal of a port-a-cath. No thromboembolisms or thrombotic microangiopathies were reported. Over a median follow-up of 389.0 days, 76.7% (n=112) of PwHA had no recorded bleeds (Table 2). For the 34 patients with recorded bleeds on emicizumab, median (Q1, Q3) ABR was 1.0 (0.5, 2.0); 13 of the 34 had joint bleeds, 18 had traumatic bleeds, and 19 had spontaneous bleeds. An intra-patient comparison in all PwHA (n=146) showed a decrease in mean (95% confidence interval [CI]) ABR from 2.38 (1.77-3.22) pre-emicizumab (2018, prior to first dose) to 0.36 (0.24-0.53) post-emicizumab (rate ratio [RR] 0.15, p<0.0001). In PwHA with bleeds before or after initiation of emicizumab (n=97), mean (95% CI) ABR decreased from 5.31 (3.79-7.45) pre-emicizumab to 0.87 (0.58-1.32) post-emicizumab (RR 0.16, p<0.0001). Conclusions: This analysis describes the baseline characteristics, bleed outcomes, and safety of Canadian PwHA treated with emicizumab before December 31, 2021. The majority had severe disease and current or historical FVIII inhibitors. The data show 76.7% had no recorded bleeds and there was a substantial decrease in bleeds post-emicizumab. The CBDR allows for a longitudinal follow-up of the Canadian hemophilia A population, which can inform healthcare practitioners and regulatory authorities of the real-world safety and effectiveness outcomes of treatments. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Evaluating the Utility of EQ5D-5L in Patients with Brain Metastases: Secondary Analysis of NRG-CC001

<h3>Purpose/Objective(s)</h3> EuroQoL EQ5D has become the preferred preference-based tool used in oncology clinical trials to assess patient health states following different interventions. It allows for an efficient measure of patient quality-of-life (QoL) that can be used to calculate quality-adjusted life years required to perform cost-effectiveness analyses. NRG-CC001 established the neuroprotective effect of hippocampal avoidant-whole brain radiotherapy (HA-WBRT) plus memantine over WBRT plus memantine in patients with brain metastases. EQ5D results, collected as part of patient reported outcomes, demonstrated no differences between study arms. Although EQ5D was used in this trial, it is unknown if this tool is sensitive to QoL changes experienced by patients with brain metastases. The primary objective of this study was to assess the utility of EQ5D in reflecting neurocognitive function (NCF) changes in this patient population. <h3>Materials/Methods</h3> We performed a post hoc analysis of NRG-CC001 trial data, focusing on EuroQoL EQ5D measures. We used independent sample t-tests to assess mean differences of EQ5D index and visual analog scale (VAS) score changes from baseline (at 2, 4, and 6 months) between patients who experienced NCF failure and those who did not. We also compared mean changes in EQ5D for patients who experienced intracranial progression to those without intracranial progression. <h3>Results</h3> EQ5D index score and VAS score changes between those patients who had NCF failure and those that had intact NCF showed no statistically significant differences at 2, 4, and 6 months (Table). When stratifying by age (<=61, >61), education (<= high school, >high school), and KPS (70, >70), there was no significant EQ5D difference by treatment arm or by NCF change. There was a trend towards significance when assessing changes in EQ5D index and VAS score at 4 months (p=0.06 and 0.05, respectively), and a significant correlation at 6 months for VAS score change (p=0.04) based on progression of disease versus not. <h3>Conclusion</h3> EQ5D was not sensitive to changes in NCF following whole brain radiation therapy for patients with brain metastases, but was sensitive to changes in intracranial progression. Given that NCF is well-established as a patient-centric outcome measure following brain radiation, the results of this study challenge the routine use of EQ5D in brain metastases clinical trials that are focused on preventing NCF failure. There is an urgent need to identify preference-based health status measures that are sensitive to changes in NCF and can be utilized in clinical trials for patients with brain metastases.

ABCL-316 Health-Related Quality of Life and Tolerability in Patients With/Without Skin Toxicity During Loncastuximab Tesirine Treatment in a Phase 2 Clinical Trial (LOTIS-2)

Skin toxicity was commonly reported among patients treated with loncastuximab tesirine (loncastuximab tesirine-lpyl; Lonca). Most skin toxicities (~90%) were grade 1 or 2, but their impact on health-related quality of life (HRQOL) was unknown. This post hoc analysis assesses whether skin toxicity is associated with HRQOL and patient tolerability to Lonca. The LOTIS-2 study (NCT03589469) is a single-arm, open-label, phase 2 study of 145 adult patients with relapsed/refractory diffuse large B-cell lymphoma after ≥2 prior treatments. Patients received Lonca as an intravenous infusion on day 1 of each 3-week treatment cycle for up to 1 year. EQ-5D and FACT-Lym scores were collected. Mean changes from baseline were summarized by visit and with/without skin toxicity. The least-squares means of the differences were estimated using the analysis of covariance models to adjust for age, sex, race, baseline score, and tumor response status. Treatment tolerability was measured using FACT-Lym item GP5 ("I am bothered by side effects of treatment"). Patients with an HRQOL baseline score and a post-baseline score (n=130) were included in the analysis (median age, 66 years; 59% male; and 88% White). With a median of 4 cycles of treatment (range, 1-26), 41% of patients experienced skin toxicity during treatment, >80% due to non-photosensitivity cutaneous reactions. Among all 9 evaluated scores (EQ-5D visual analog scale, FACT-Lym subscale, and composite scores) for visits up to cycle 9 (n≥20), there were no significant differences (P>0.05) between patients with and without skin toxicity except for the FACT-Lym total at cycle 9 (worse in patients with skin toxicity). A majority of patients (≥60%) were "not at all" or "a little bit" bothered in both groups at each cycle, although a higher percentage of patients with skin toxicity reported "a little bit" than those without skin toxicity after 2 cycles. Patients with skin toxicity while receiving Lonca did not experience different HRQOL compared with patients without skin toxicity in most visits. Lonca was tolerated well even among patients with skin toxicity. (Sponsored by ADC Therapeutics).