Patient-reported Health Status Research Articles

Functional capacity by cardiopulmonary exercise testing is not related to health status in obstructive hypertrophic cardiomyopathy

Abstract Background In obstructive hypertrophic cardiomyopathy (HCM), the primary goal of therapy is to improve patient quality of life by decreasing symptom burden. Increasingly, cardiopulmonary exercise testing (CPET), with peak oxygen consumption (pVO2) and ventilator efficacy (VE/VCO2), is used to assess efficacy of novel therapies in clinical studies and selectively in clinical practice to determine treatments. However, the relationship of CPET variables to symptom burden in HCM is not well established. Therefore, we examined the relationship between CPET variables and health status measured by Kansas City Cardiomyopathy Questionnaire (KCCQ) and NYHA functional classification in a cohort of obstructive HCM patients. Methods Consecutive patients with HCM and resting left ventricular outflow tract (LVOT) obstruction (≥50 mmHg) underwent CPET between October 2022 to January 2024. Patients with a peak RER <1.0 were excluded (n=4). To investigate distribution of patient reported health status (KCCQ) and NYHA functional classification, patients were stratified into groups for pVO2 (<14, 14-20, and >20 mL/kg/min) to reflect moderate to severe, mild to moderate, and little to no functional limitation, respectively. Results Of 58 patients, clinical evaluation was at 59 ± 13 years of age, 55% male, with LVOT gradient of 83 ± 28 mmHg at rest with 84% NYHA class II or III and 76% with KCCQ overall summary (OS) <75. Both pVO2 and VE/VCO2 had significant but weak correlation with NYHA class and KCCQ scores (Table 1), with the strongest correlation observed with pVO2 and KCCQ-OS (R2= 0.13, p=0.004). In contrast, % predicted pVO2 was not significantly correlated with NYHA or KCCQ. Notably, despite 14 patients being classified as having little to no limitations by pVO2, 29% of these patients had mild to moderate disability and 29% had moderate to severe disability by KCCQ-OS (Figure 1). Conclusion In patients with symptomatic obstructive HCM, objective measures of exercise performance, including pVO2 and VE/VCO2, correlate poorly with measurements of health status. These data suggest that CPET results should not be considered a surrogate marker of heart failure symptom burden in obstructive HCM.

Quality of Life After Transcatheter Tricuspid Valve Replacement: 1-Year Results From TRISCEND II Pivotal Trial.

Severe tricuspid regurgitation (TR) often causes substantial impairment in patient-reported health status (ie, symptoms, physical and social function, and quality of life), which may improve with transcatheter tricuspid valve replacement (TTVR). We performed an in-depth analysis of health status of patients enrolled in the TRISCEND (Edwards EVOQUE Transcatheter Tricuspid Valve Replacement: Pivotal Clinical Investigation of Safety and Clinical Efficacy using a Novel Device) II pivotal trial to help quantify the benefit of intervention to patients. The TRISCEND II pivotal trial randomized 400 patients with symptomatic and severe or greater TR 2:1 to TTVR with the EVOQUE tricuspid valve replacement system plus optimal medical therapy (OMT) or OMT alone. Health status was assessed with the Kansas City Cardiomyopathy Questionnaire and the 36-Item Short Form Health Survey. Changes in health status over 1 year were compared between treatment groups using mixed-effects repeated-measures models. The analysis cohort included 392 patients, of whom 259 underwent attempted TTVR and 133 received OMT alone (mean age 79.2 ± 7.6 years, 75.5% women, 56.1% with massive or torrential TR). Patients had substantially impaired health status at baseline (mean Kansas City Cardiomyopathy Questionnaire Overall Summary Score [KCCQ-OS] 52.1 ± 22.8; mean 36-Item Short Form Health Survey physical component summary score 35.2 ± 8.4). TTVR+OMT patients reported significantly greater improvement in both disease-specific and generic health status at each follow-up time point. Mean between-group differences in the KCCQ-OS favored TTVR+OMT at each time point: 11.8 points (95%CI: 7.4-16.3 points) at 30days, 20.8 points (95%CI: 16.1-25.5 points) at 6months, and 17.8 points (95%CI: 13.0-22.5 points) at 1 year. In subgroup analyses, TTVR+OMT improved health status to a greater extent among patients with torrential or massive TR vs severe TR (treatment effect 23.3 vs 22.6 vs 11.3; interaction P=0.049). At 1 year, 64.6% of TTVR+OMT patients were alive and well (KCCQ-OS≥60 points and no decline of≥10 points from baseline) compared with 31.0% with OMT alone. Compared with OMT alone, treatment of patients with symptomatic and severe or greater TR with TTVR+OMT resulted in substantial improvement in patients' symptoms, function, and quality of life. These benefits were evident 30days after TTVR, continued to increase through 6months, and remained durable through 1 year. (TRISCEND II Pivotal Trial [Edwards EVOQUE Transcatheter Tricuspid Valve Replacement: Pivotal Clinical Investigation of Safety and Clinical Efficacy using a Novel Device]; NCT04482062).

Effect of Aficamten on Health Status Outcomes in Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM

BackgroundA primary goal in treating obstructive hypertrophic cardiomyopathy (oHCM) is to improve patients’ health status: their symptoms, function, and quality of life. The health status benefits of aficamten, a novel cardiac myosin inhibitor, have not been comprehensively described. ObjectivesThis study sought to determine the effect of aficamten on patient-reported health status, including symptoms of fatigue, shortness of breath, chest pain, physical and social limitations, and quality of life. MethodsSEQUOIA-HCM (Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM) randomized symptomatic adults with oHCM to 24 weeks of aficamten (n = 142) or placebo (n = 140), followed by a 4-week washout. The Kansas City Cardiomyopathy Questionnaire (KCCQ) and Seattle Angina Questionnaire 7-item (SAQ7) were serially administered. Changes in mean KCCQ—Overall Summary Score (KCCQ-OSS) and SAQ7—Summary Score (SAQ7-SS) from baseline to 24 weeks and following treatment withdrawal were compared using linear regression adjusted for baseline scores and randomization strata. Proportions of patients with clinically important changes were compared. ResultsAmong 282 participants, the mean age was 59 ± 13 years, 115 (41%) were female, and 223 (79%) were White. Baseline KCCQ-OSS (69.3 ± 20.1 vs 67.3 ± 18.8) and SAQ7-SS (72.0 ± 21.0 vs 72.4 ± 18.3) were similar between aficamten and placebo groups. Treatment with aficamten, compared with placebo, improved both the mean KCCQ-OSS (13.3 ± 16.3 vs 6.1 ± 12.6; mean difference: 7.9; 95% CI: 4.8-11.0; P < 0.001) and SAQ7-SS (11.6 ± 17.4 vs 3.8 ± 14.4; mean difference: 7.8; 95% CI: 4.7-11.0; P < 0.001) at 24 weeks, with benefits emerging within 4 weeks. No heterogeneity in treatment effect was found across subgroups. A much larger proportion of participants experienced a very large health status improvement (≥20 points) with aficamten vs placebo (KCCQ-OSS: 29.7% vs 12.4%, number needed to treat: 5.8; SAQ7-SS: 31.2% vs 13.9%, number needed to treat: 5.8). Participants’ health status worsened significantly more after withdrawal from aficamten than placebo (KCCQ-OSS: −16.2 ± 19.0 vs −3.0 ± 9.6; P < 0.001; SAQ7-SS: −17.4 ± 21.4 vs −2.5 ± 13.3), further confirming a causal effect of aficamten. ConclusionsIn patients with symptomatic oHCM, treatment with aficamten resulted in markedly improved health status, including significant improvement in chest pain-related health status, than placebo. (Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM [SEQUOIA-HCM]; NCT05186818)

Accelerometer-measured physical activity in patients with heart failure and reduced ejection fraction: Determinants and relationship with patient-reported health status

Background: Accelerometer-measured physical activity is an increasingly used endpoint in heart failure (HF) trials. We investigated the determinants of accelerometer-measured physical activity and the relationship with patient-reported health status. Methods: Post-hoc analysis of the Empire HF trial, including outpatients with HF with reduced ejection fraction (HFrEF). Physical activity was quantified as average accelerometer counts per minute (CPM) with higher values representing higher activity. We investigated associations between activity level and clinical variables, including age, sex, and body mass index, as well as patient-reported health status assessed by Kansas City Cardiomyopathy Questionnaire (KCCQ). Results: Complete data were available in 180 (95%) patients (86% male, mean age 65 year). Baseline median physical activity level was 1318 CPM (Q1-Q3 1111–1585). Age and anemia were independently associated with activity level (β-coefficients: -10 CPM per year age increase [95% CI -16 to -5.1], p=0.00015, and -126 CPM for anemia [95% CI -9.1 to -244], p=0.035). Significant independent associations were observed between activity level and all KCCQ summary scores (β-coefficient point estimates of 3.7, 4.6, and 4.9 CPM, all p<0.02). For 12-week changes in KCCQ-summary scores, only the KCCQ-CSS was associated with activity level; mean increase of 17.5 CPM [95% CI 1.5 to 34.0], p=0.032, per 5-point increase in KCCQ-CSS. Associations were not modified by treatment allocation (interaction p-values>0.05). Conclusions: In patients with HFrEF, older age and anemia were independently associated with lower activity. Moreover, physical activity only weakly increased with better health status, suggesting that changes in physical activity reflect improvements in patients’ health status to a limited degree. This highlights the need to better understand the endpoint with regards to all other health parameters to ease interpretation in future HF trials.

Navigating the transition: the crucial shift from pediatric to adult care for individuals living with spina bifida.

Spina bifida (SB) is a complex congenital condition characterized by incomplete closure of the neural tube, resulting in varying degrees of physical and neurological impairment. Although commonly managed by multidisciplinary pediatric clinics, a substantial proportion of SB patients are now living into adulthood, necessitating the transition from pediatric to adult healthcare. This transition introduces a myriad of challenges for individuals living with SB and their families. Prior research on SB transition programs has demonstrated anecdotal success; however, minimal research has been published on early posttransition health outcomes and compliance with medical recommendations. This quality improvement study assessed early posttransition compliance with medical recommendations, adverse health events, access to medical supplies/equipment, and patient-reported health outcome and confidence in medical providers. Adult participants in the Spina Bifida Transition Clinic at the authors' pediatric institution were invited to complete a telephone survey after transition to adult care. The mean (SEM) elapsed time since transition was 1.21 (0.11) years. The survey evaluated adult provider utilization, accessibility of medical supplies and equipment, adverse medical events, compliance with sleep study acquisition, patient-reported health status, and satisfaction with providers. Of 52 eligible participants, 49 (94%) completed a telephone survey. Within the cohort, 82% had open SB (myelomeningocele), with the remaining having occult SB (lipomyelomeningocele). The mean age at transition was 26.0 years. Since transition, 78% have attended at least one primary care visit, with 76% seeking care from at least one adult care specialist (69% sought care with urologists). Forty-five percent reported an adverse medical event: 31% required an emergency department visit, 22% were hospitalized, 18% underwent surgery, and 24% had skin breakdown. Access to medical supplies varied, with patients experiencing the most difficulty obtaining wheelchairs and assistive walking devices. Patients rated pediatric provider engagement and knowledge of SB significantly higher than adult providers (mean 3.92 vs 3.32, p < 0.001). This quality improvement study evaluated the effectiveness of our Spina Bifida Transition Clinic in the early post transition period. While patients have used primary and specialty care (urology), they have experienced many adverse events and low compliance with sleep study acquisition. Continued evaluation of transition programs is required to optimize the outcome of those living with SB.

Effects of Pycnogenol® in people with post-COVID-19 condition (PYCNOVID): study protocol for a single-center, placebo controlled, quadruple-blind, randomized trial

BackgroundA significant proportion of the global population has been infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at some point since the onset of the pandemic. Although most individuals who develop coronavirus disease 2019 (COVID-19) recover without complications, about 6% have persistent symptoms, referred to as post-COVID-19 condition (PCC). Intervention studies investigating treatments that potentially alleviate PCC-related symptoms and thus aim to mitigate the global public health burden and healthcare costs linked to PCC are desperately needed. The PYCNOVID trial investigates the effects of Pycnogenol®, a French maritime pine bark extract with anti-inflammatory and antioxidative properties, versus placebo on patient-reported health status in people with PCC.MethodsThis is a single-center, placebo-controlled, quadruple blind, randomized trial. We aim to randomly assign 150 individuals with PCC (1:1 ratio) to receive either 200 mg Pycnogenol® or placebo daily for 12 weeks. Randomization is stratified for duration of PCC symptoms (≤ 6 months versus > 6 months) and presence of symptomatic chronic disease(s). The primary endpoint is perceived health status at 12 weeks (EuroQol—Visual Analogue Scale) adjusted for baseline values and stratification factors. Secondary endpoints include change in self-reported PCC symptoms, health-related quality of life, symptoms of depression and anxiety, cognitive function, functional exercise capacity, physical activity measured with accelerometry, and blood biomarkers for endothelial health, inflammation, coagulation, platelet function, and oxidative stress. Investigators, study participants, outcome assessors, and data analysts are blinded regarding the intervention assignment. Individuals with PCC were involved in the design of this study.DiscussionThis is the first trial to investigate the effects of Pycnogenol® versus placebo on patient-reported health status in people with PCC. Should the trial proof clinical effectiveness, Pycnogenol® may serve as a therapeutic approach to mitigate symptoms associated with PCC.Trial registrationThe study is registered at ClinicalTrials.gov. :NCT05890534, June 6, 2023.

Risk perception, well-being, depression and anxiety in children and adolescents with rheumatic diseases during the COVID-19 pandemic - results from the prospective multicenter KICK-COVID study in Germany

ObjectiveTo investigate the psychosocial burden in children and adolescents with juvenile rheumatic diseases during the COVID-19 pandemic.MethodsAs part of the multicentre observational KICK-COVID study linked to the National Pediatric Rheumatology Database, adolescents < 21 years and parents of children < 12 years with rheumatic diseases answered questions on perceptions of health risk (PHR) due to SARS-CoV2, stress, well-being (WHO-5) and symptoms of depression (PHQ-9) and anxiety (GAD-7). Data were collected at routine visits from June to December 2021 and assessed for association with demographic and clinical parameters, treatment and patient-reported outcomes by multivariable regression analyses.ResultsData from 1356 individuals (69% female, 50% adolescents) were included. Median PHR on a numeric rating scale (NRS, 0–10) was 4 (IQR 2–6), median perceived stress was 3 (IQR 1–6). Adolescents reported a worse well-being with a significantly lower median WHO-5-score (60, IQR 40–76) than parents reported for their children < 12 years (80, IQR 68–84). Moderate to severe symptoms of depression and anxiety were reported by 14.3% and 12.3% of the adolescents, respectively. PHR was significantly higher in patients with systemic lupus erythematosus, methotrexate or biologic disease-modifying anti-rheumatic drug therapy than in patients without these characteristics, whereas lower WHO-5 or higher PHQ-9 or GAD-7 scores were only associated with poorer patient-reported health status and physical functioning.ConclusionThe perception of health risk due to SARS-CoV2 infection was not paralleled by an impairment of mental health, which were, however, significantly correlated with self-rated health status and functional capacity, highlighting the importance of patient-reported outcome assessment.Trial registrationGerman Clinical Trials Register (DRKS), no. DRKS00027974. Registered on 27th of January 2022.

Clinical Impact of Routine Assessment of Patient-Reported Health Status in Heart Failure Clinic: The PRO-HF Trial.

The impact of routine clinic use of patient-reported outcome (PRO) measures on clinical outcomes in patients with heart failure (HF) has not been well-characterized. We tested if clinic-based use of a disease-specific PRO improves patient-reported quality of life at 1 year. The PRO-HF trial (Patient-Reported Outcome Measurement in Heart Failure Clinic) was an open-label, parallel, patient-level randomized clinical trial of routine PRO assessment or usual care at an academic HF clinic between August 30, 2021, and June 30, 2022, with 1 year of follow-up. In the PRO assessment arm, participants completed the Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12) at each HF clinic visit, and results were shared with their treating clinician. The usual care arm completed the KCCQ-12 at randomization and 1 year later, which was not shared with the treating clinician. The primary outcome was the KCCQ-12 overall summary score (OSS) between 12 and 15 months after randomization. Secondary outcomes included domains of the KCCQ-12, hospitalization and emergency department visit rates, HF medication therapy, clinic visit frequency, and testing rates. Across 17 clinicians, 1248 participants were enrolled and randomized to PRO assessment (n=624) or usual care (n=624). The median age was 63.9 years (interquartile range [IQR], 51.8-72.8), 38.9% were women, and the median baseline KCCQ-12 OSS was 82.3 (IQR, 58.3-94.8). Final KCCQ-12 (available in 87.9% of the PRO arm and 85.1% in usual care; P=0.16) median OSS were 87.5 (IQR, 68.8-96.9) in the PRO arm and 87.6 (IQR, 69.7-96.9) in the usual care arm with a baseline-adjusted mean difference of 0.2 ([95% CI, -1.7 to 2.0]; P=0.85). The results were consistent across prespecified subgroups. A post hoc analysis demonstrated a significant interaction with greater benefit among participants with a baseline KCCQ-12 OSS of 60 to 80 but not in less or more symptomatic participants. No significant differences were found in 1-year mortality, hospitalizations, emergency department visits, medication therapy, clinic follow-up, or testing rates between arms. Routine PRO assessment in HF clinic visits did not impact patient-reported quality of life or other clinical outcomes. Alternate strategies and settings for embedding PROs into routine clinical care should be tested. URL: https://www.clinicaltrials.gov; Unique identifier: NCT04164004.

Disparities in Patient-reported Outcome Measure Completion Rates and Baseline Function in Newly Presenting Spine Patients.

Retrospective review. The purpose of this study was to evaluate disparities in patient-reported outcome measures (PROM) completion rates and baseline function scores among newly presenting spine patients. Prior studies have demonstrated that minority patients and those of low socioeconomic status may present with worse physical and mental health on PROMs. As PROMs are increasingly used in clinical care, research, and health policy, accurate assessment of health status among populations relies on the successful completion of PROM instruments. A retrospective review of 10,803 consecutive new patients presenting to a single multidisciplinary spine clinic from June 2020 to September 2022 was performed. Univariate statistics were performed to compare demographics between patients who did and did not complete PROMs. Multivariable analysis was used to compare PROM instrument completion rates by race, ethnicity, and Social Vulnerability Index (SVI) and baseline scores among responders. A total of 68.4% of patients completed PROMs at the first clinic visit. After adjusting for age, sex, body mass index, and diagnosis type, patients of non-White race (OR=0.661, 95% CI=0.599-0.729, P <0.001), Hispanic ethnicity (OR=0.569, 95% CI=0.448-0.721, P <0.001), and increased social vulnerability (OR=0.608, 95% CI=0.511-0.723, P <0.001) were less likely to complete PROMs. In the multivariable models, patients of non-White race reported lower levels of physical function (β=-6.5, 95% CI=-12.4 to -0.6, P =0.032) and higher levels of pain intensity (β=0.6, 95% CI=0.2-1.0, P =0.005). Hispanic ethnicity (β=1.5, 95% CI=0.5-2.5, P =0.004) and increased social vulnerability (β=1.1, 95% CI=0.4-1.8, P =0.002) were each associated with increased pain intensity. Among newly presenting spine patients, those of non-White race, Hispanic ethnicity, and with increased social vulnerability were less likely to complete PROMs. As these subpopulations also reported worse physical function or pain intensity, additional strategies are needed to better capture patient-reported health status to avoid bias in clinical care, outcomes research, and health policy. 4.

Physician Estimates and Patient-Reported Health Status in Atrial Fibrillation

A primary objective in managing atrial fibrillation (AF) is to optimize patients' health status, which can be done only if physicians accurately quantify the outcomes associated with AF in patients' lives. To explore physicians' estimation of the health status of patients with AF and its association with subsequent care and outcomes. A multicenter, prospective cohort study was conducted in 2 outpatient practices in Tokyo, Japan. Participants included patients with newly diagnosed AF or those referred for initial treatment of AF at outpatient practices and treating physicians from November 8, 2018, to April 1, 2020. Data analysis was performed from December 22, 2022, to July 7, 2023. Participating patients completed the Atrial Fibrillation Effect on Quality-of-Life (AFEQT) questionnaire, a 20-item tool covering 4 domains with a 7-point Likert scale; 3 domains (symptoms, daily activities, and treatment concerns) were used in this study. Blinded to patients' responses, treating physicians answered a 3-item questionnaire quantifying each patient's AFEQT domain with a single item. Patients' mean Likert scale responses within each AFEQT domain were subtracted from the physicians' assessments so that higher scores (≥0.5 points) indicate physician underestimation, while lower scores (≤0.5 points) indicate physician overestimation of the health status of patients with AF. The independent association of physician-patient concordance with treatment escalation (alteration or initiation of antiarrhythmic drugs, cardioversion, or catheter ablation) and 1-year adjusted changes in AFEQT scores. Among 330 patients (238 [72.1%] men; mean [SD] age, 67.9 [11.9] years; 163 [49.4%] with paroxysmal AF), physicians correctly estimated health status in 112 patients (33.9%), underestimated it in 42 patients (12.7%), and overestimated it in 176 patients (53.3%). Treatment escalation occurred in 63.6% of patients whose health status was correctly estimated, 47.6% of those whose health status was underestimated, and 66.3% of patients whose health status was overestimated. After multivariable adjustment, underestimation of health status was independently associated with less treatment escalation (adjusted odds ratio, 0.43; 95% CI, 0.20-0.90) and less frequent AFEQT overall summary score improvement at 1 year (underestimated, 2.5 [95% CI, -1.6 to 6.7] vs correctly and overestimated health status, 8.4 [95% CI, 7.0-9.9] points; P = .01). In this cohort study, physician underestimation of the health status of patients with AF was common and associated with less aggressive treatment and less health status improvement at 1 year.

Work Productivity and General Health Through 2 Years of Guselkumab Treatment in a Phase 3 Randomized Trial of Patients With Active Psoriatic Arthritis.

To evaluate the effect of guselkumab on work productivity and nonwork daily activity impairment and general health status through 2years in patients who were biologic-naïve with active psoriatic arthritis (PsA) in the phase 3 DISCOVER-2 clinical trial. Adult patients with PsA were randomized to subcutaneous injections of guselkumab 100mg every 4weeks (Q4W); at weeks 0, 4, then every 8 weeks (Q8W); or placebo (throughweek 24 with crossoverto guselkumab Q4W). Work productivity and nonwork daily activity impairment were assessed using the Work Productivity and Activity Impairment Questionnaire for PsA (WPAI-PsA) and patient-reported general health status using the EuroQol 5-Dimension 5-Level (EQ-5D-5L) Index and EQ-Visual Analog Scale (EQ-VAS). Least-squares (LS) mean changes from baseline in WPAI-PsA domains and EQ-5D-5L/EQ-VAS were assessed through week 100. Changes in employment status were utilized to estimate potential indirect savings from improved work productivity. Of 739 randomized patients, 738 had available baseline data for the analyses (Q4W 245; Q8W 248; placebo 245). At week 24, greater improvements in work productivity, nonwork daily activity, and EQ-5D-5L/EQ-VAS were observed in the Q4W and Q8W groups versus the placebo group. At week 100, LS mean reductions in work productivity impairment (- 23.8% to - 28.0%) and nonwork daily activity impairment (- 26.6% to - 29.2%) and improvements in EQ-5D-5L/EQ-VAS (0.14 to 0.15/21.2 to 25.0) were maintained in patients receiving guselkumab. Among patients employed at baseline, 12.1-16.4% were not employed at week 100, and 20.0-25.3% shifted from not employed at baseline to employed at week 100. Potential yearly indirect cost savings (USD) from improved work productivity at week 100 ranged from $16,529 to $19,409. Patients with active PsA treated with guselkumab demonstrated reduced impairment in work productivity and nonwork daily activity, together with improvement in general health status and substantial potential cost savings, over a 2-year period. Clinicaltrials.gov identifier: NCT03158285.

Maintenance of patient-reported quality of life (EQ-5D-3L) post neoadjuvant relugolix prior to the initiation of prostate radiation therapy.

117 Background: Injectable GnRH receptor agonists have been shown to improve cancer control when combined with radiotherapy (RT) for unfavorable intermediate- to high-risk prostate cancer. Relugolix is a new oral GnRH receptor antagonist that achieves high rates of rapid profound castration (total testosterone &lt; 20 ng/dL), which may impact health-related quality of life. This prospective study sought to evaluate the impact of neoadjuvant relugolix on health-related quality of life in prostate cancer patients prior to the start of prostate RT. Methods: Patients treated at Georgetown between May 2021 and August 2023 with neoadjuvant relugolix (at least 2 months prior to RT) per an institutional protocol were included in the study (IRB 12-1775). The 5-item EQ-5D-3L, a validated questionnaire that assesses the patient-reported health status outcome, was collected for each patient at baseline (before relugolix initiation) and one hour prior to RT initiation. EQ VAS Score Overall ranges from 0-100 with a higher score indicating better quality of life. Individual items including mobility, self-care, usual activities, pain/discomfort, and anxiety/depression were scored on a three-point scale from 1 (No problems) to 3 (Extreme). To examine changes before and after relugolix treatment, McNemar’s test and paired samples t-test were used. Minimally important difference (MID) calculated by 0.5 of standard deviation at baseline was used to examine clinical significance. Results: Of 79 patients, the median age was 73 years, and 42% were non-white. Patients initiated relugolix at a median of 4 months prior to questionnaire administration (IQR: 3.9-5.4), at which most (95%) patients achieved castration (≤ 50 ng/dL). The VAS Score Overall was significantly higher at baseline (Mean ± SD: 83 ± 10) than the paired score at the start of RT (80 ± 14, p = 0.01). This change was not clinically significant. There were no statistically or clinically significant differences in any of the 5 individual items. Conclusions: Neoadjuvant relugolix prior to prostate radiation therapy did not clinically significantly affect patient-reported quality of life. Also, there was no statistically significant decrease in each of the 5 individualized items evaluating health-related quality of life. Future studies should focus on comparing it to GnRH agonist-induced change in quality of life. [Table: see text]

Psychometric Properties of the Instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosapatient score (iscorEB-p): a patient-reported outcome measure.

In contrast to clinical diagnosis via external examination, patient-related outcome measures (PROMs) allow access to patients' internal perceptions. In the case of epidermolysis bullosa (EB) - a rare disease characterized by a wide variety of symptoms and individual disease courses - it is important to integrate the patient's perspective into diagnostic processes. The Instrument for Scoring Clinical Outcomes of Research for EB (iscorEB) is an EB-specific measurement tool, combining a clinician score (iscorEB-c) and a patient questionnaire (iscorEB-p). The aim of this study is to establish the iscorEB-p as an independent PROM tool by exploring its psychometric properties. Sample-based psychometric testing and evaluation were performed on data collected via a multinational online cross-sectional study. Data analysis was performed with n = 95 participants across all EB types. The reliability and internal consistency of the iscorEB-p was excellent (α = 0.90). Principal component analysis with a varimax rotation resulted in a two-factor solution, explaining 55.6% of the total variance, with the distinct factors 'everyday life functioning' and 'specific EB symptoms'. Convergent validity was shown by high correlations to the Satisfaction With Life Scale (r = -0.52, P < 0.001), the Quality of Life in Epidermolysis Bullosa questionnaire (r = 0.72, P < 0.001) and the Epidermolysis Bullosa Family Burden of Disease questionnaire (r = -0.73, P < 0.001). The iscorEB-p is a reliable and valid instrument to assess patient-reported health status of people with EB.

Lung cancer registry to promote person-centered care.&amp;nbsp;

Introduction: Lung cancer is a leading cause of death and major burden on healthcare systems. Patient-centered outcome measurements allow the optimization of care organization and quality improvement. The aim of this project was to outline a set of variables and outcomes to evaluate the quality of care provided to lung cancer patients. &#x0D; Methods: This project took place at Hospital Italiano de Buenos Aires, a community-based tertiary care hospital in Argentina; which provides thoracic surgery, pneumology, oncology, pathology, radiology and radiotherapy services. It also has a Lung Cancer Multidisciplinary Board. All patient information is stored in a Data Repository fed by the electronic health record (EHR).&#x0D; We established a team of experts in management, IT, thoracic surgery, pneumology, oncology, and internal medicine. We reviewed care processes and patient trajectory along the usual pathway, through a retrospective EHR analysis of a consecutive sample of lung cancer patients. &#x0D; We performed a literature review to outline possible variables and health outcomes to be collected, instruments, and frequency of measurement. We determined needs, usefulness, and feasibility of collecting variables and outcomes in group meetings. &#x0D; Results: We reviewed 15 cases of lung cancer, 53% male, median age 68 years old (range: 29-85). We detected heterogeneity in the time elapsed between critical points of management (eg first visit to diagnosis 21 to 642 days; biopsy report to chemotherapy 19 to 163). The proportion of use of the healthcare system was also variable (eg: 87% consulted pneumonolory or oncology, only 13% had pain management visits and 7% received respiratory kinesic therapy). Patient-reported outcomes were not systematically evaluated.&#x0D; The selected baseline variables were: age, sex, weight loss, smoking status, comorbidities, patient-reported health status (EORTC European Organization for Research and treatment of cancer, QLC-LC30 Core quality of life questionnaire, QLC-LC13 Lung cancer-specific quality of life questionnaire), performance status (ECOG Eastern Cooperative Oncology Group), pulmonary function (VEF1), histology, clinical and pathological stage (AJCC-TNM 8th Ed), molecular tests and treatment intent. The outcomes (collected annually): clinical staging performed before treatment, time from diagnosis to treatment, performance status, patient-reported health status, major surgical complications, major chemotherapy/radiotherapy complications, time from diagnosis to treatment, completed treatment, overall survival, cause of death, treatment-related mortality, place of death, time spent in hospital at the end of life (last 30 days).&#x0D; Discussion: Cancer registries comprise a set of systematically and prospectively collected data. Quality measurements performed in the context of cancer registries could assist in monitoring and evaluating quality of care. &#x0D; Conclusions: The project determined essential variables and outcomes to be measured, considering their relevance and feasibility.&#x0D; Lessons learnt: Teamwork is essential when developing a registry, as different stakeholders' opinions are reflected. Integrated care is a useful solution to overcome fragmentation.&#x0D; Limitations: Patients did not participate directly in this development. &#x0D; Suggestions for future research: These results will assist in outlining a care pathway for patients with lung cancer, which is currently under development. Evaluation before and after the implementation of care pathways may evidence quality improvement.

Home-based tele-rehabilitation versus hospital-based outpatient rehabilitation for pain and function after initial total knee arthroplasty: A systematic review and meta-analysis.

This systematic review and meta-analysis aims to compare the effectiveness of home-based tele-rehabilitation programs with hospital-based rehabilitation programs in improving pain and function at various time points (≤6 weeks, ≤14 weeks, and ≤ 52 weeks) following the initial total knee arthroplasty. This study used PRISMA and AMSTAR reporting guidelines. We systematically searched 5 databases (PubMed, Embase, Web of Science, Cochrane Library, and Medline) to identify randomized controlled trials published from January 1, 2019, to January 1, 2023. The primary outcomes were pain, knee injury and osteoarthritis outcome score, and mobility (knee range of motion). We included 9 studies involving 1944 patients. Low-quality evidence showed hospital-based rehabilitation was better than home-based tele-rehabilitation in knee injury and osteoarthritis outcome score (mean difference [MD], -2.62; 95% confidence interval [CI], -4.65 to -0.58; P = .01) at ≤ 14 weeks after total knee arthroplasty. Based on low-quality evidence, home-based tele-rehabilitation was better than hospital-based rehabilitation in knee range of motion (MD, 2.00; 95% CI, 0.60 to 3.40; P = .005). There was no significant difference between hospital-based rehabilitation and home-based tele-rehabilitation in knee pain at ≤ 6 weeks (MD, 0.18; 95% CI, -0.07 to 0.42; P = .16), 14 weeks (MD, 0.12; 95% CI, -0.26 to 0.49; P = .54), and ≤ 52 weeks (MD, 0.16; 95% CI, -0.11 to 0.43; P = .24). Home-based tele-rehabilitation and hospital-based rehabilitation programs showed comparable long-term outcomes in pain, mobility, physical function, and patient-reported health status after primary total knee arthroplasty. Considering the economic costs, home-based tele-rehabilitation programs are recommended as a viable alternative to hospital-based rehabilitation programs.

ARE OLDER ADULTS’ PERCEPTIONS OF ELDERSPEAK INFLUENCED BY SELF-REPORTED PSYCHOSOCIAL CHARACTERISTICS?

Abstract PROMIS (Patient-Reported Outcomes Measurement Information System) is a system of highly reliable measures of patient-reported health status. Elderspeak is speech that sounds childish to older adults. Use of elderspeak by nursing staff can result in rejection of care. The Iowa Coding Scheme for Elderspeak (ICodE) contains 11 elderspeak attributes: diminutives, childish terms, collectives, short words, directives, reflectives, minimizers, exaggerated praise, laughter, tag questions and exaggerated prosody. Thirty-one older adults (aged 74.5±7.8 years) responded to short form PROMIS instruments measuring self-efficacy, physical function, isolation, cognitive function, depression, anxiety, and pain interference. Participants listened to 41 audio recordings of nursing staff providing care to hospitalized patients with dementia and were asked to rate each audio recording using a 5-point Emotional Tone Rating Scale for patronizing and respectful (5=very, 1=not at all). Using the ICodE, each audio recording was classified as neutral (no elderspeak), containing a single attribute, or containing an attribute combined with exaggerated prosody. Ratings for elderspeak attributes ranged from 2.1 to 2.9 for patronizing and 2.8 to 3.8 for respectful. Using the SPSS statistical package, a Spearman’s rho correlation was calculated for each elderspeak rating and health status domain identified above. Among this small, homogenous sample, no statistically significant correlation was found between participants’ self-efficacy, isolation, physical or cognitive function, depression, anxiety, or pain interference, as measured by PROMIS tools, and their rating of elderspeak attributes as respectful or patronizing. Future research should continue to explore what factors influence older adults’ perceptions of elderspeak as being respectful or patronizing.

Accelerometer-measured physical activity in patients with heart failure and reduced ejection fraction: determinants and relationship with patient-reported health status

Abstract Background Accelerometer-measured physical activity is an increasingly used endpoint in heart failure (HF) trials. Purpose We aimed to investigate the determinants of accelerometer-measured physical activity and to establish the minimal clinically important difference (MCID). Methods Post-hoc analysis of the Empire HF trial, including stable outpatients with HF with reduced ejection fraction (HFrEF). Physical activity was quantified as average accelerometer counts per minute (CPM) with higher values representing higher activity. Associations between activity level and important clinical variables [age, sex, body mass index, N-terminal pro-brain natriuretic peptide, left ventricular ejection fraction (LVEF), atrial fibrillation, and anemia], as well as patient-reported health status (Kansas City Cardiomyopathy Questionnaire, KCCQ) were assessed. Results Complete data were available in 180 (95%) patients (86% male, mean age 65 years, mean LVEF 30%). Baseline median physical activity level was 1318 CPM (Q1-Q3 1111–1585). At baseline, age and anemia were independently associated with activity level (β-coefficients: -10 CPM per year age increase [95% CI -16 to -5.1], p = 0.00015, and -126 CPM for presence of anemia [95% CI -9.1 to -244], p = 0.035). Moreover, a significant independent association was observed between activity level and all KCCQ summary scores (β-coefficient point estimates of 3.7, 4.6, and 4.9 CPM, all p&amp;lt;0.02). For 12-week changes, only the KCCQ-clinical summary score (CSS) was associated with activity level, with a mean increase of 17.5 CPM [95% CI 1.5 to 34.0], p = 0.032, for every clinically relevant 5-point increase in KCCQ-CSS, corresponding to the MCID. Conclusion In patients with HFrEF, high age and anemia were independently associated with lower accelerometer-measured physical activity. Moreover, clinically relevant 5-point increases in Kansas City Cardiomyopathy Questionnaire clinical summary scores at 12 weeks were associated with an increase of 17.5 activity counts per minute, thus representing the minimal clinically important difference.Table 1.Baseline characteristicsFigure 1

Abstract 13085: Sociodemographic Predictors of NYHA Class Assignment: An Analysis From CHAMP-HF

Background: The New York Heart Association (NYHA) class is the cornerstone of health status assessment in heart failure with reduced ejection fraction (HFrEF). However, prior work has shown marked variability compared to patient-reported health status - measured by the Kansas City Cardiomyopathy Questionnaire (KCCQ). As clinicians determine NYHA, systemic biases may impact class selection. It is unknown if patients’ age, race, sex, BMI, or education influence NYHA class assignment. Aims: To determine whether patient factors (age, race, sex, education, and BMI) predict NYHA class when controlling for KCCQ. Methods: Participants in CHAMP-HF, a prospective registry of ambulatory HFrEF patients, were evaluated by a clinician with a simultaneously completed NYHA and KCCQ-12. Using hierarchical multinomial logistic regression models with site-level random effects to account for clustering by site, we modeled NYHA class on age, Hispanic and Black vs White patients, males vs females, education, and BMI separately while adjusting for baseline KCCQ-12 as a restricted cubic spline. Results: 4,742 participants were included. The table shows model summaries. Of all predictor variables, only age was associated with differential NYHA class assignment. Compared with class I, 5-year increases in age increased the odds of being assigned class II (OR 1.11; 95% CI 1.07-1.16), III (OR 1.2; 95% CI 1.15-1.26) and IV (OR 1.18; 95% CI 1.07-1.31). Conclusions: These results suggest clinicians assign higher NYHA classes to older patients irrespective of patient-reported health status, but there were no differences identified by race, sex, education or BMI. Whether this leads to differential treatment in older patients merits investigation.

Abstract 11574: Clinical Changes and Predictors of Health Status in Patients With Atrial Fibrillation

Background: Recent clinical trials have shown the health status benefit of rhythm control, including catheter ablation (CA) over rate control for patients with atrial fibrillation (AF). However, the trials show population-averaged benefits, which complicates clinical interpretation; in reality, some patients may not benefit or even experience worsening health status after treatment. This study aimed to examine changes in patients' health status and identify predictors in a multicenter outpatient registry (KiCS-AF). Methods: KiCS-AF was a multicenter, prospective registry of patients with newly diagnosed AF collecting baseline and 1-year patient-reported health status. We examined changes in the Atrial Fibrillation Effect on QualiTy-of-life overall summary (AFEQT-OS) score over 1 year across different treatment strategies in patients with paroxysmal or persistent AF. We defined clinically important improvement and deterioration of health status as ≥5 points increase and decrease in AFEQT-OS score and no change as -5 to 5 points based on previous reports. Results: Overall, 1,960 patients were enrolled in this study, with mean AFEQT-OS scores of 76.7±17.7 and 85.4±14.8 at baseline and 1-year follow-up, respectively. The majority of patients (53.9%) experienced a clinically important improvement in health status, albeit a substantial number of patients experienced no change (28.7%) or worsening health status (17.4%). The proportion of patients with no change or worsening health status differed by treatment strategy (59.9%, 53.9%, and 32.0% in the rate control, antiarrhythmic drugs, and CA groups, respectively). After adjustment for baseline characteristics and AFEQT-OS scores, preserved baseline health status, older age (≥65 years), female sex, heart failure, and coronary artery disease were independent predictors of worsening health status in the overall cohort and the CA subgroup. Conclusion: In contemporary practice, a large proportion of patients showed improvement in health status, while a considerable number of patients experienced worsening or no change after intervention. Patient-centered care is essential to prevent the worsening of health status even after CA in high cardiovascular-risk, elderly, and female populations.

The efficacy of inspiratory muscle training in patients with coronary artery disease: Protocol for a systematic review and meta-analysis.

Inspiratory muscle training (IMT) has been recognized as an effective form of training in patients with cardiovascular disease and heart failure. However, little is known about the efficacy of IMT in the treatment of patients with coronary artery disease (CAD). The aim of this systematic review will be to evaluate randomized controlled studies to understand the effect of IMT on CAD patients. We will include randomized controlled trials evaluating the efficacy of IMT in patients 18 years and older diagnosed with CAD. Crossover trials, cluster-randomized, quasi-randomized, and non-randomized trials will be excluded. Study search will be conducted in major databases (MEDLINE, the Cochrane Central Register of Controlled Trials, Embase, and PEDro). The study intervention will be IMT independent of the duration, frequency, or intensity of training. The primary outcome will be quality of life, patient-reported health status, and all adverse events related to IMT. Secondary outcomes will include exercise capacity and respiratory muscle strength. The risk of bias will be evaluated based on the Cochrane Risk of Bias tool. Screening, data extraction, and quality assessment will be performed by two independent reviewers. If two or more studies are considered to be clinically homogeneous, a meta-analysis based on the random-effects model will be performed. The quality of evidence will be evaluated based on the GRADE approach. This systematic review will improve our understanding of the effects of IMT on CAD patients and potentially establish IMT as an alternative form of exercise training for the treatment of CAD. Study registration. OSF registries (https://osf.io/3ch7m). Date registered: May 10, 2022. Registration DOI: https://doi.org/10.17605/OSF.IO/GVMY7.