Patient-reported Health Status Research Articles

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. 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 in order to avoid bias in clinical care, outcomes research and health policy.

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]

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.

Interleukin-6 in Patients With Heart Failure and Preserved Ejection Fraction

BackgroundInterleukin (IL)-6 is a central inflammatory mediator and potential therapeutic target in heart failure (HF). Prior studies have shown that IL-6 concentrations are elevated in patients with HF, but much fewer data are available in heart failure with preserved ejection fraction (HFpEF). ObjectivesThis study aims to determine how IL-6 relates to changes in cardiac function, congestion, body composition, and exercise tolerance in HFpEF. MethodsClinical, laboratory, body composition, exercise capacity, physiologic and health status data across 4 National Heart, Lung, and Blood Institute–sponsored trials were analyzed according to the tertiles of IL-6. ResultsIL-6 was measured in 374 patients with HFpEF. Patients with highest IL-6 levels had greater body mass index; higher N-terminal pro–B-type natriuretic peptide, C-reactive protein, and tumor necrosis factor-α levels; worse renal function; and lower hemoglobin levels, and were more likely to have diabetes. Although cardiac structure and function measured at rest were similar, patients with HFpEF and highest IL-6 concentrations had more severely impaired peak oxygen consumption (12.3 ± 3.3 mL/kg/min 13.1 ± 3.1 mL/kg/min 14.4 ± 3.9 mL/kg/min, P < 0.0001) as well as 6-minute walk distance (276 ± 107 m vs 332 ± 106 m vs 352 ± 116 m, P < 0.0001), even after accounting for increases in IL-6 related to excess body mass. IL-6 concentrations were associated with increases in total body fat and trunk fat, more severe symptoms during submaximal exercise, and poorer patient-reported health status. ConclusionsIL-6 levels are commonly elevated in HFpEF, and are associated with greater symptom severity, poorer exercise capacity, and more upper body fat accumulation. These findings support testing the hypothesis that therapies that inhibit IL-6 in patients with HFpEF may improve clinical status. (Clinical Trial Registrations: Phosphodiesterase-5 Inhibition to Improve Clinical Status and Exercise Capacity in Diastolic Heart Failure [RELAX], NCT00763867; Nitrate’s Effect on Activity Tolerance in Heart Failure With Preserved Ejection Fraction, NCT02053493; Inorganic Nitrite Delivery to Improve Exercise Capacity in HFpEF, NCT02742129; Inorganic Nitrite to Enhance Benefits From Exercise Training in Heart Failure With Preserved Ejection Fraction [HFpEF], NCT02713126)

Healthcare utilization and unmet needs of patients with antisynthetase syndrome: An international patient survey

Antisynthease syndrome (ASSD) is a rare, complex and understudied autoimmune disease. Internet-based studies can overcome barriers of traditional on-site research and are therefore very appealing for rare diseases. The aim of this study was to investigate patient-reported symptoms, diagnostic delay, symptoms, medical care, health status, working status, disease knowledge and willingness to participate in research of ASSD patients by conducting an international web-based survey. The multilingual questionnaire was created by an international group of rheumatologists and patients and distributed online. 236 participants from 22 countries completed the survey. 184/236 (78.0%) were female, mean age (SD) was 49.6 years (11.3) and most common antisynthetase antibody was Jo-1 (169/236, 71.6%). 79/236 (33.5%) reported to work full-time. Median diagnostic delay was one year. The most common symptom at disease onset was fatigue 159/236 (67.4%), followed by myalgia 130/236 (55.1%). The complete triad of myositis, arthritis and lung involvement verified by a clinician was present in 42/236 (17.8%) at disease onset and in 88/236 (37.3%) during the disease course. 36/236 (15.3%) reported to have been diagnosed with fibromyalgia and 40/236 (16.3%) with depression. The most reported immunosuppressive treatments were oral corticosteroids 179/236 (75.9%), followed by rituximab 85/236 (36.0%). 73/236 (30.9%) had received physiotherapy treatment. 71/236 (30.1%) reported to know useful online information sources related to ASSD. 223/236 (94.5%) were willing to share health data for research purposes once a year. Our results reiterate that internet-based research is invaluable for cooperating with patients to foster knowledge in rare diseases.

Translation and validation of a Finnish version of the Facial Clinimetric Evaluation (FaCE) Scale

Background: The Facial Clinimetric Evaluation (FaCE) scale is a patient-reported health status instrument developed for assessing the health-related quality of life (HRQoL) in patients with facial nerve paralysis. The aim of this study was to translate and validate the FaCE scale for the Finnish-speaking population.Methods: The FaCE scale was translated according to international guidelines. Sixty patients in an outpatient clinic prospectively completed the translated FaCE scale and generic HRQoL instrument (15D). The objective facial paralysis grading was made using the Sunnybrook and House-Brackmann scales. Repeated FaCE and 15D instruments were mailed to patients 2 weeks later. Total scores of the FaCE instrument and subscales were calculated, and floor and ceiling effects were examined. Exploratory factor analysis was made. Internal consistency, reliability, and repeatability were assessed. Convergence with 15D instrument, Sunnybrook, and House-Brackmann scales was examined.Results: The total internal consistency of the FaCE scale was high (Cronbach’s alpha 0.83). There were no statistically significant differences found between mean scores of the subscales in test-retest analysis (p &gt; 0.05). Intra-class correlations coefficients were high, ranging between 0.78 and 0.92, and the correlations were statistically significant (p &lt; 0.001). There were statistically significant correlations observed between the FaCE scale and the 15D, Sunnybrook, and House-Brackmann scores. Conclusion: The FaCE scale was successfully translated and validated in Finnish with good validity and reliability. We also demonstrated statistically significant correlations between the generic HRQoL15D instrument and both the Sunnybrook and House-Brackmann physician-based grading scales. The FaCE scale is now ready for use in Finnish facial paralysis patients.

Quality of life (QoL) in patients with recurrent ovarian clear cell carcinoma (rOCCC) receiving durvalumab (D) versus physician’s choice chemotherapy (PCC) in the randomized phase II MOCCA/APGOT-OV2/GCGS-OV3 trial.

e17564 Background: In the phase II MOCCA/ APGOT-OV2/ GCGS-OV3 trial, D was not superior to PCC in prolonging progression-free survival in patients with rOCCC. Fewer all-grade and high-grade adverse events were observed for patients treated with D compared with PCC. We analysed health-related QoL by treatment arm. Methods: In this multicentre, open-label, randomised phase II trial, eligible patients were randomly assigned (2:1), using dynamic block randomization with block size of 6, and stratification by Eastern Cooperative Oncology Group performance status, to receive D (1500mg on day 1, in 28-day cycles) or PCC until disease progression, intolerable toxicity or withdrawal of consent. Patients treated with PCC were able to receive any agent deemed appropriate by the Investigator, however the addition of anti-angiogenic drugs was prohibited. Patients with disease progression on PCC were allowed to crossover to D. Patients completed European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 and QLQ-OV28 QoL questionnaires at baseline and every 4 weeks until end of study treatment. The minimal important difference for clinically relevant change was fixed at 10 points. Linear mixed-effects models were used to estimate the treatment effect on QoL outcomes, accounting for the intra-subject correlation in QoL scores at repeated visits from treatment cycles 2 to 6, while adjusting for baseline QoL scores (cycle 1 day 1) and time of visit. We included all patients whose QoL scores were available at both baseline and at least one visit from cycles 2 to 6. Results: 47 patients were randomly assigned to treatment with D (31 patients) or PCC (16 patients). Compliance to QoL questionnaires was 81.6 % (D) and 87.5% (PCC) at baseline. QoL data was available for 22.6-81.6% (D) and 31.3-87.5% (PCC) of patients over time. Longitudinal analysis of QoL data by domain revealed no significant differences in global health status, functional scales or most of the symptom scales based on EORTC QLQ-C30 or QLQ-OV28 between patients treated with D compared with PCC, except for the Constipation symptom scale on QLQ-C30 which showed an estimated mean difference of -20.8 points (95% confidence interval (CI), -31.5 to -10.2; p = 0.001) and the Hair loss symptom scale on QLQ-OV28 which showed an estimated mean difference of -19.6 points (95% CI -36.6 to -2.6; p = 0.025). Conclusions: D and PCC did not lead to significant differences in patient-reported global health status or functional scales. However, PCC was associated with increased patient-reported constipation and hair loss compared with D in rOCCC. Clinical trial information: NCT03405454 .

Health Status, Quality of Life, Psychosocial Well-being, and Wearables Data of Patients With Active Ulcerative Colitis Receiving Filgotinib Therapy (FilgoColitis Study): Protocol for a Real-world Observational Study.

Filgotinib was approved in Germany for treating patients with moderate to severe active ulcerative colitis in November 2021. It represents a preferential Janus kinase 1 inhibitor. The FilgoColitis study began recruiting immediately after approval and aims to assess filgotinib effectiveness under real-world conditions with a particular focus on patient-reported outcomes (PROs). The novelty of the study design is the optional inclusion of 2 innovative wearables, which could provide a new layer of patient-derived data. The study investigates quality of life (QoL) and psychosocial well-being of patients with active ulcerative colitis during long-term exposure to filgotinib. PROs related to QoL and psychometric profiles (fatigue and depression) are collected alongside with disease activity symptom scores. We aim to evaluate physical activity patterns collected by wearables as an addition to traditional PROs, patient-reported health status, and QoL in different phases of disease activity. This is a prospective, single-arm, multicentric, noninterventional, observational study with a sample size of 250 patients. QoL is assessed with validated questionnaires: the Short Inflammatory Bowel Disease Questionnaire (sIBDQ) for the disease-specific QoL, the EQ-5D for the general QoL, and the fatigue questionnaire (Inflammatory Bowel Disease-Fatigue [IBD-F]). Physical activity data are collected from patients using wearables (SENS motion leg sensor [accelerometry] and smartwatch, GARMIN vívosmart 4). The enrollment started in December 2021 and was still open at the date of submission. After 6 months of study initiation, 69 patients were enrolled. The study is expected to be completed in June 2026. Real-world data for novel drugs are important to assess effectiveness outside of highly selected populations represented by randomized controlled trials. We examine whether patients' QoL and other PROs can be supplemented with physical activity patterns measured objectively. Use of wearables with newly defined outcomes represents an additional observational tool for monitoring disease activity in patients with inflammatory bowel disease. German Clinical Trials Register DRKS00027327; https://drks.de/search/en/trial/DRKS00027327. DERR1-10.2196/42574.

Clinical prediction model for prognosis in kidney transplant recipients (KIDMO): study protocol

BackgroundMany potential prognostic factors for predicting kidney transplantation outcomes have been identified. However, in Switzerland, no widely accepted prognostic model or risk score for transplantation outcomes is being routinely used in clinical practice yet. We aim to develop three prediction models for the prognosis of graft survival, quality of life, and graft function following transplantation in Switzerland.MethodsThe clinical kidney prediction models (KIDMO) are developed with data from a national multi-center cohort study (Swiss Transplant Cohort Study; STCS) and the Swiss Organ Allocation System (SOAS). The primary outcome is the kidney graft survival (with death of recipient as competing risk); the secondary outcomes are the quality of life (patient-reported health status) at 12 months and estimated glomerular filtration rate (eGFR) slope. Organ donor, transplantation, and recipient-related clinical information will be used as predictors at the time of organ allocation. We will use a Fine & Gray subdistribution model and linear mixed-effects models for the primary and the two secondary outcomes, respectively. Model optimism, calibration, discrimination, and heterogeneity between transplant centres will be assessed using bootstrapping, internal-external cross-validation, and methods from meta-analysis.DiscussionThorough evaluation of the existing risk scores for the kidney graft survival or patient-reported outcomes has been lacking in the Swiss transplant setting. In order to be useful in clinical practice, a prognostic score needs to be valid, reliable, clinically relevant, and preferably integrated into the decision-making process to improve long-term patient outcomes and support informed decisions for clinicians and their patients. The state-of-the-art methodology by taking into account competing risks and variable selection using expert knowledge is applied to data from a nationwide prospective multi-center cohort study. Ideally, healthcare providers together with patients can predetermine the risk they are willing to accept from a deceased-donor kidney, with graft survival, quality of life, and graft function estimates available for their consideration.Study registrationOpen Science Framework ID: z6mvj

Patient-Reported Versus Physician-Assessed Health Status in Heart Failure With Reduced and Preserved Ejection Fraction From ASIAN-HF Registry.

We aimed to assess if discordance between patient-reported Kansas City Cardiomyopathy Questionnaire (KCCQ)-overall summary (os) score and physician-assessed New York Heart Association (NYHA) class is common among patients with heart failure (HF) with reduced or preserved ejection fraction, and determine its association with outcomes. A total of 4818 patients with HF were classified according to KCCQ-os score (range 0-100, dichotomized by median value 71.9 into high [good] versus low [bad]) and NYHA class (I/II [good] or III/IV [bad]) as concordant good (low NYHA class, high KCCQ-os score), concordant bad (high NYHA class, low KCCQ-os score), discordant worse NYHA class (high NYHA class, high KCCQ-os score), and discordant worse KCCQ-os score (low NYHA class, low-KCCQ-os score). The composite of HF hospitalization or death at 1 year was compared across groups. There were 2070 (43.0%) concordant good, 1099 (22.8%) concordant bad, 331 (6.9%) discordant worse NYHA class, and 1318 (27.4%) discordant worse KCCQ-os score patients. Compared with concordant good, adverse outcomes were the highest in concordant bad (HR, 2.7 [95% CI, 2.2-3.5]) followed by discordant worse KCCQ-os score (HR, 1.8 [95% CI, 1.4-2.2]) and discordant worse NYHA class (HR, 1.5 [95% CI, 1.0-2.3]); with no modification by HF phenotype (preserved versus reduced ejection fraction, Pinteraction=0.52). At 6 months, 1403 (48%) experienced clinically significant improvement in KCCQ-os score (≥5 points increase over 6 months). Patients with improved KCCQ-os at 6 months (HR, 0.65 [95% CI, 0.47-0.92]) had better outcomes and the association was not modified by HF phenotype (Pinteraction=0.40). One-third of patients with HF had discordance between patient-reported and clinician-assessed health status, largely attributable to worse patient-reported outcomes. Such discordance, particularly in those with discordantly worse KCCQ, should alert physicians to an increased risk of HF hospitalization and death, and prompt further assessment for potential drivers of worse patient-reported outcomes relative to physicians' assessment.