Patient-reported Outcomes In Adults Research Articles

Patient-reported outcomes in adults with tyrosine kinase inhibitor-resistant chronic myeloid leukemia receiving olverembatinib therapy.

The objective of this study was to assess patient-reported outcomes (PROs), including health-related quality of life (HRQoL) and anxiety and depression symptoms, and to identify associated variables in patients with tyrosine kinase inhibitor (TKI)-resistant chronic myeloid leukemia (CML) in chronic-phase (CP) or accelerated-phase (AP) who were receiving olverembatinib. Patients in multicenter studies were invited to complete the European Organization for Research and Treatment of Cancer Quality-of-Life Core 30 Questionnaire, the Self-Rating Anxiety Scale, and the Self-Rating Depression Scale at baseline and regularly during olverembatinib therapy. The time trends in PROs were estimated with a linear model using a generalized estimating equation based on an independent working correlation matrix. A generalized estimating equation model was used to assess the variables associated with PROs. In total, 146 patients with CML-CP or CML-AP were included in this study. Scores on seven items from the European Organization for Research and Treatment of Cancer Quality-of-Life Core 30 Questionnaire, including global health, physical functioning, emotional functioning, fatigue, dyspnea, diarrhea, and financial difficulties, improved significantly over time during olverembatinib therapy. In multivariate analysis, age younger than 40 years was significantly associated with greater improvement in social functioning (p=.033), and CML-CP (vs. CML-AP) was associated with greater improvements in dyspnea (p=.031) and diarrhea (p=.031) over time. Scores on the Self-Rating Anxiety Scale and the Self-Rating Depression Scale decreased significantly over time during olverembatinib treatment (p<.001). The authors concluded that HRQoL significantly improved over time during olverembatinib therapy in heavily treated patients with TKI-resistant CML, especially among those who were younger and those who had CML-CP. Anxiety symptoms also significantly decreased over time.

Rocatinlimab Improves Patient-Reported Outcomes in Adults with Moderate-to-Severe Atopic Dermatitis: Results from a Double-Blind Placebo-Controlled Phase2b Study.

In adults with moderate-to-severe atopic dermatitis (AD), rocatinlimab demonstrated significant and progressive improvement in clinical measures of disease severity compared with placebo. This post hoc analysis of a phase2b study was undertaken to understand the disease burden and to assess the impact of rocatinlimab on patient-reported outcomes (PROs). This analysis used baseline data from a multicenter, randomized, double-blind study of adults with moderate-to-severe AD, who completed a Worst Pruritus numerical rating scale (NRS), Sleep Disturbance NRS, and the Dermatology Life Quality Index (DLQI). A mixed model for repeated measures was used to estimate changes in PRO scores from baseline; scores were also compared with clinically meaningful change benchmarks. The analysis included 267 subjects, mean (SD) age 37.9 (14.7) years, 40.8% female; 55.1% grade3 and 44.9% grade4 Investigator Global Assessment for AD. Mean (SD) scores were: Worst Pruritus NRS 7.5 (1.9), Sleep Disturbance NRS 5.5 (2.9), DLQI total score 12.6 (7.1). Worst Pruritus and Sleep NRS scores had low positive correlations with SCORing AD (SCORAD) score (r = 0.44, r = 0.45 respectively) and negligible correlations with Eczema Area and Severity Index (EASI) score and area affected (r < 0.30). DLQI score varied by sex, study country, race, age, longer disease duration, disease severity (EASI and SCORAD), presence of asthma, and Worst Pruritus NRS, Sleep disturbance NRS, and DLQI scores. Rocatinlimab showed benefit on all three PROs, with significant improvements from baseline at the end of the double-blind period (week18) and active treatment extension (week36). Benefits were maintained over 20weeks' post-treatment follow-up. The benefit of rocatinlimab treatment on PROs is rapid and maintained for at least 20weeks following treatment completion. This analysis demonstrates the importance of characterizing the burden of moderate-to-severe AD from the patient's perspective, alongside clinical disease measures, to develop a fuller picture of treatment benefit. ClinicalTrials.gov identifier, NCT03703102.

Frailty, cognition and patient-reported outcomes in middle-aged and older adults with congenital heart disease: insights from an international multicentric study

Abstract Background Prior research has demonstrated that a significant proportion of older patients with congenital heart disease (CHD) are frail or pre-frail and experience cognitive impairment. However, it remains unclear whether frailty and cognition are related to patient-reported outcomes (PROs) such as perceived health, anxiety and depression symptoms or quality of life in CHD. Purpose The objective of this study was to explore the relationships between frailty, cognitive function, and PROs in middle-aged and older adults with CHD ranging from mild to severe complexity. Methods As part of the cross-sectional ‘Assessment of Patterns of Patient-Reported Outcomes in Adults with Congenital Heart disease – International Study II’ (APPROACH-IS II), we enrolled 814 individuals aged 40 years and older (median age: 52.0 years [45.0-61.0]; 51.4% female; 12.7% mild, 70.1% moderate, 17.2% complex CHD; 41.9% pre-frail, 5.8% frail; 38.7% cognitively impaired) across 17 centers in 11 countries. Frailty status was assessed using the Fried criteria, which categorize individuals into robust, pre-frail, and frail based on five factors: weakness, slow gait, unintentional weight loss, exhaustion, and low physical activity levels. Cognitive function was determined using the Montréal Cognitive Assessment. Participants completed questionnaires to evaluate perceived health (RAND-12 Health Survey, yielding both Physical and Mental Component Summaries, and EuroQOL-5D Visual Analog Scale), depressive symptoms (Patient Health Questionnaire-8), anxiety symptoms (General Anxiety Disorder scale-7), and quality of life (QoL Linear Analog Scale). Results Frailty was significantly associated with all PROs, with frail and pre-frail individuals reporting less favorable outcomes (see Figure). Cognitive function was linked to perceived health and anxiety, with patients exhibiting cognitive impairments reporting poorer perceived health and more anxiety symptoms. Conclusion This study indicates that concepts of frailty, cognitive functioning and PROs are closely related in adults with CHD. Frailty and cognition thus represent important domains when developing strategies to optimize PROs in adults with CHD. Moreover, when working with patients who are frail or cognitively impaired, healthcare workers are encouraged to consider and inquire about potential impact on PROs. Longitudinal research is needed to explore the direction of the association.

Variability in patient-reported outcomes among different congenital heart defects: a comparative analysis of 7,498 patients from 32 countries

Abstract Background Patient-reported outcomes (PROs) may not always correlate with the complexity of congenital heart defects (CHDs), as significant variation has been observed between CHDs within complexity levels. Accurate comparisons across various types of CHDs necessitate large sample sizes. Purpose The objective of this study was to compare PROs across different CHDs in a large international sample. Methods ‘Assessment of Patterns of Patient-Reported Outcomes in Adults with Congenital Heart disease – International Study II’ (APPROACH-IS II) was an international cross-sectional investigation, in which 8,415 patients from 32 countries in 6 continents were enrolled (20 high; 7 upper-middle; 4 lower-middle; and 1 low income). For the comparison across different CHDs, conditions with at least 50 patients were included in the analysis. Hence, the sample consisted of 7,498 patients (median age=33y; 53.1% women). Patients completed questionnaires to measure perceived health status (RAND-12 Health Survey; EuroQOL-5D Visual Analog Scale); depressive symptoms (Patient Health Questionnaire-8); anxiety (General Anxiety Disorder-7) and quality of life (Linear Analog Scale). Generalized linear mixed models (GLMMs) were performed with center as random effect. Results Cyanotic heart defects were associated with the lowest scores for physical functioning, mental health, self-rated health, and quality of life (see Figure). Other CHDs found to be associated with poor PRO scores encompassed unrepaired secundum atrial septal defect, Ebstein anomaly, single ventricle, and truncus arteriosus. Conversely, the most favorable scores for all PROs, except for anxiety, were observed with isolated pulmonic stenosis. Other CHDs with favorable PRO scores comprised coarctation of the aorta, isolated small ventricular septal defect, congenital aortic valve disease, and repaired ventricular septal defect. Adjusted for sociodemographic and physiological factors, GLMMs revealed significant differences across defects, using the best score for each PRO as the reference (see Figure). Conclusion Substantial variation in PROs was observed among different types of CHDs. Interestingly, some complex heart defects demonstrated relatively good PRO scores, comparable to some simple defects. On the other hand, unrepaired simple defects or those with residual lesions, were associated with worse PROs.

Patient-Reported Outcomes in Adults with FSGS: Sparsentan vs. Irbesartan

Patient-Reported Outcomes in Adults with FSGS: Sparsentan vs. Irbesartan

Patient-Reported Outcomes in Adults With Newly Diagnosed Ph+ Acute Lymphoblastic Leukemia Treated With Ponatinib or Imatinib in Combination With Reduced-Intensity Chemotherapy: Results From the Phase III PhALLCON Study

Patient-Reported Outcomes in Adults With Newly Diagnosed Ph+ Acute Lymphoblastic Leukemia Treated With Ponatinib or Imatinib in Combination With Reduced-Intensity Chemotherapy: Results From the Phase III PhALLCON Study

ALL-442 Patient-reported Outcomes in Adults With Newly Diagnosed Ph+ Acute Lymphoblastic Leukemia Treated With Ponatinib or Imatinib in Combination With Reduced-Intensity Chemotherapy: Results From the Phase III PhALLCON Study

ALL-442 Patient-reported Outcomes in Adults With Newly Diagnosed Ph+ Acute Lymphoblastic Leukemia Treated With Ponatinib or Imatinib in Combination With Reduced-Intensity Chemotherapy: Results From the Phase III PhALLCON Study

Item Response Theory Quantifies the Relationship Between Improvements in Serum Phosphate and Patient-Reported Outcomes in Adults With X-Linked Hypophosphatemia.

Burosumab is indicated for treatment of a rare bone disease, X-linked hypophosphatemia (XLH). The aim of this analysis was to evaluate the relationship between a treatment response biomarker and patient-reported outcomes (PROs). Longitudinal data for PROs were obtained from adults with XLH from a phase III study. Individual rich time profiles of the biomarker, serum phosphate were simulated using a prior population pharmacokinetic-pharmacodynamic model to calculate serum phosphate exposure metrics for each 28-day treatment cycle, which were then merged with PROs data. Item response theory parameters were first estimated to map a latent variable, ψ, that is, disability score, relative to baseline. Next, the relationships between serum phosphate exposures and ψ were modeled using a nonlinear mixed-effect (NLME) modeling approach. A combined item response theory-NLME model with average serum phosphate as a predictor of ψ described PROs data well. The model estimates suggested 28%, 31%, and 25% reduction in Western Ontario and McMaster Universities Osteoarthritis Index, brief pain inventory, and brief fatigue inventory scores, respectively, with every unit increase in average serum phosphate from the lower limit of normal (2.5 mg/dL). Additionally, a time effect of ~ 0.08% improvements each week was estimated. The analysis suggested that burosumab treatment-induced improvements in serum phosphate levels are associated with improvements in PROs in adults with X-linked hypophosphatemia. The analyses confirmed the importance of prolonged serum phosphate level correction in adult patients with XLH. These results can be useful to guide the design of further studies and to design treatment optimization strategies.

Construct validity of PROMIS pain interference, fatigue, and physical function as patient-reported outcomes in adults with idiopathic inflammatory myopathies: An international study from the OMERACT myositis working group

BackgroundValidated patient-reported outcome measures to assess disease impact in patients with adult idiopathic inflammatory myopathies (IIMs) are needed. The objective of this study was to assess the construct validity of PROMIS Pain Interference, Fatigue, and Physical Function measures in comparison with core disease activity measures. MethodsAdults with IIM, excluding inclusion body myositis, from OMERACT Myositis Working Group (MWG) clinic sites completed PROMIS Short Form v1.0—Pain Interference 6a, PROMIS Short Form v1.0—Fatigue 7a, and PROMIS Short Form v2.0—Physical Function 8b measures. Core disease activity measures including patient and physician global disease activity assessments, manual muscle testing, serum creatine kinase activity, and Health Assessment Questionnaire Disability Index (HAQ-DI) were simultaneously assessed. To evaluate construct validity, a priori hypotheses for the expected correlations between PROMIS measures, age, and core disease measures were determined by >70 % agreement among MWG members and were compared against observed Pearson's correlations. Internal consistency of items and floor or ceiling effects for the PROMIS measures were also assessed. Subgroup analysis according to IIM subtype (dermatomyositis vs. non-dermatomyositis IIM) was performed. Results135 adults with IIM from 5 countries across North America, Europe, Asia, and Australia were included. For construct validity, a priori hypotheses were confirmed for 5 of 6 (83 %) PROMIS Pain Interference, 4 of 5 (80 %) PROMIS Fatigue, and 3 of 4 (75 %) PROMIS Physical Function correlations. Internal consistency was high for each PROMIS measure (Cronbach's alpha >0.9). Ceiling effects were observed only for PROMIS Pain Interference, with low/no pain in 29 % of patients. Subgroup analysis between dermatomyositis (n = 65) and non-dermatomyositis (n = 70) subtypes demonstrated similar correlations between PROMIS measures and disease activity measures. ConclusionsPROMIS Short Form v1.0—Pain Interference 6a, PROMIS Short Form v1.0—Fatigue 7a, and PROMIS Short Form v2.0—Physical Function 8b measures demonstrate strong construct validity when compared to core disease activity measures in IIM, with consistent results across IIM subtypes. These findings support the use of these selected PROMIS measures to assess core domains of interest for measuring life impact in IIMs.

Immunotherapy related patient-reported outcomes from five randomized controlled trials in patients with triple negative breast cancer: A systematic review.

e23167 Background: Immunotherapy (IO) has improved clinical outcomes in patients with early-stage Triple Negative Breast Cancer (TNBC) receiving neoadjuvant therapy and certain patient subsets in the metastatic setting. This study aims to evaluate IO-related Patient-Reported Outcomes (PRO) in patients older than 65 years. Methods: We searched Medline ALL (Ovid), Embase (Elsevier), Cochrane Central (Wiley), ClinicalTrials.gov, and the WHO International Clinical Trials Registry Platform. The searches combined terms reflecting (1) the immune checkpoint inhibitors atezolizumab, durvalumab, and pembrolizumab; (2) breast cancer; and (3) randomized controlled trials. Results were limited to items published in English from 2013 through September 2023. We excluded review articles and clinical trial records without results. Two independent reviewers (C.S. and M.L.) screened the studies using the systematic review management tool Covidence, and conflicts were resolved by a third reviewer (W.I.Z.). We included studies that reported on PRO in adults with TNBC receiving any of the three immunotherapies of interest. Results: Five publications composed of two peer-reviewed papers and three abstracts were identified, including 3878 total patients and 3184 with PRO. Three pembrolizumab studies had 2643 total patients (2056 with PRO) and two atezolizumab studies (IMPASSION 130, IMPASSION 031) had 1235 total patients (1128 with PRO). The PRO analysis in KEYNOTE 522 included 1145 patients in the neoadjuvant phase and 847 in the adjuvant phase. The PRO analysis in KEYNOTE 355 included total 847 patients and 317 with Combined Positive Score (CPS) ≥10. No study specified PRO data for patients older than 65 years. Baseline completion rates for all studies were &gt; 80%. All studies used standardized PRO instruments, including the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC- QLQ C30) and its Breast Cancer Module (QLQ-BR23), the Functional Assessment of Cancer-Therapy-General (FACT-G GP5), and the EuroQol-5 Dimension (EQ-D5). All studies showed no statistical difference, or clinical meaningful decline in PRO endpoints between patients who received chemo with IO versus chemo with placebo, during and post treatment follow up. The PRO analysis from KEYNOTE 119, which included total 594 patients of which 187 had CPS ≥10, demonstrated that patients receiving single agent chemotherapy had worse PROs related to systemic therapy side effects and hair loss while the pembrolizumab group had decreased worry about future health. None included subgroup analysis by age, including patients older than 65 years. Conclusions: Our systematic review demonstrates that in both early and advanced TNBC, IO did not worsen PRO. Prospective and retrospective data in IO-related PRO in older patient population are needed.

Dupilumab Improves Disease Control and Patient-Reported Outcomes in Adults with Atopic Dermatitis in Clinical Practice: Subgroup Analysis of Black/African American Population from RELIEVE-AD

Dupilumab Improves Disease Control and Patient-Reported Outcomes in Adults with Atopic Dermatitis in Clinical Practice: Subgroup Analysis of Black/African American Population from RELIEVE-AD

Anxiety and Depression in Adults With Congenital Heart Disease

BackgroundA comprehensive understanding of adult congenital heart disease outcomes must include psychological functioning. Our multisite study offered the opportunity to explore depression and anxiety symptoms within a global sample. ObjectivesIn this substudy of the APPROACH-IS (Assessment of Patterns of Patient-Reported Outcomes in Adults With Congenital Heart Disease–International Study), the authors we investigated the prevalence of elevated depression and anxiety symptoms, explored associated sociodemographic and medical factors, and examined how quality of life (QOL) and health status (HS) differ according to the degree of psychological symptoms. MethodsParticipants completed the Hospital Anxiety and Depression Scale, which includes subscales for symptoms of anxiety (HADS-A) and depression (HADS-D). Subscale scores of 8 or higher indicate clinically elevated symptoms and can be further categorized as mild, moderate, or severe. Participants also completed analogue scales on a scale of 0 to 100 for QOL and HS. Analysis of variance was performed to investigate whether QOL and HS differed by symptom category. ResultsOf 3,815 participants from 15 countries (age 34.8 ± 12.9 years; 52.7% female), 1,148 (30.1%) had elevated symptoms in one or both subscales: elevated HADS-A only (18.3%), elevated HADS-D only (2.9%), or elevations on both subscales (8.9%). Percentages varied among countries. Both QOL and HS decreased in accordance with increasing HADS-A and HADS-D symptom categories (P < 0.001). ConclusionsIn this global sample of adults with congenital heart disease, almost one-third reported elevated symptoms of depression and/or anxiety, which in turn were associated with lower QOL and HS. We strongly advocate for the implementation of strategies to recognize and manage psychological distress in clinical settings. (Patient-Reported Outcomes in Adults With Congenital Heart Disease [APPROACH-IS]; NCT02150603)

Association between glycemic control and patient-reported outcomes in adults with type 1 diabetes in Japan: the SAGE study subanalysis.

Psychosocial aspects and the quality of life (QOL) of individuals with diabetes are important for achieving glycemic control and treatment goals. Here, we describe patient-reported outcomes (PROs) of Japanese adults with type 1 diabetes (T1D) and evaluate the association thereof with glycemic control. This subanalysis of a subgroup of 528 Japanese participants in the SAGE study of adults with T1D used data on glycosylated hemoglobin (HbA1c) and PRO scores [Hypoglycemia Fear Survey-II (HFS-II), Problem Areas In Diabetes (PAID), Insulin Treatment Satisfaction Questionnaire (ITSQ), and Audit of Diabetes-Dependent QOL (ADDQoL)] and summarized the score by the predefined age groups (26-44-years: n = 208, 45-64-years: n = 217, and ≥ 65-years: n = 103). The association between PROs, achieving HbA1c < 7.0%, and individualized targets was explored using multivariate logistic regression analysis. The HFS-II and PAID scores were lower, and the ITSQ score was higher in the ≥ 65-years group than in the younger groups with a linear trend of better scores with increasing age (P for trend < 0.05). ADDQoL scores were similar across the age groups, and present QOL (ADDQoL subscale) tended to improve with age (P for trend < 0.05). Achieving HbA1c < 7.0% and individualized targets were associated with satisfaction with insulin treatment regarding glycemic control. In Japanese adults with T1D, the impact on psychosocial aspects and QOL varied across age groups, with a trend of improving scores with age, potentially in relation to the less stringent glycemic control targets adopted in older individuals. Glycemic control was significantly associated with treatment satisfaction. The online version contains supplementary material available at 10.1007/s13340-023-00668-4.

Burosumab Improves Patient-Reported Outcomes in Adults With Tumor-Induced Osteomalacia: Mixed-Methods Analysis.

Tumor-induced osteomalacia (TIO) is an ultra-rare disease caused by tumors that secrete fibroblast growth factor 23, leading to chronic hypophosphatemia, poor skeletal health, and impaired physical function. In a phase 2 trial (UX023T-CL201; NCT02304367; n = 14), 48 weeks of burosumab treatment restored phosphate homeostasis, with improvements in skeletal health, functional mobility, and patient-reported pain, fatigue, and health-related quality of life (HRQL) (SF-36 v2). Here, we report an exploratory mixed-methods analysis of change from baseline after 144 weeks of burosumab treatment alongside qualitative data from exit interviews with 8 of 14 trial participants to evaluate meaningful treatment effects from a patient perspective. The interview subset (n = 8) reported pain and fatigue and compromised HRQL at baseline. In the interviews, participants reported that compromised HRQL and pain were the most important aspects of the disease to treat; both were considered more bothersome than fatigue and compromised physical function and activities of daily living. Improvements in pain and fatigue after treatment were reported, some of which achieved statistically and/or clinically meaningful thresholds. Furthermore, improvements in SF-36 v2 scores were most pronounced in the Physical Component Score and its Physical Function and Bodily Pain domains. Overall, the interview subset provided descriptions of symptomatic improvement and its clinical meaningfulness, including physical function, participation in activities of daily living, and mental well-being. Thus, this exploratory mixed-methods analysis provides deeper understanding of patients' perception of clinical meaningfulness beyond that articulated in validated patient-reported outcome instruments. © 2023 The Authors. Journal of Bone and Mineral Research published by Wiley Periodicals LLC on behalf of American Society for Bone and Mineral Research (ASBMR).

752-P: Improved BMI Category Is Associated with Better Patient-Reported Outcomes (PROs) in Adults with T2D Treated with Tirzepatide

Treatment with tirzepatide, a first-in-class GIP/GLP-1 receptor agonist, resulted in statistically and meaningfully reduced HbA1c, and substantially reduced weight, in adults with T2D in the Phase 3 SURPASS studies. We explored the association between shift in BMI category and PROs in adults treated with tirzepatide 5, 10, or 15mg in SURPASS-1 to -5. Data from all SURPASS-1 to -5 tirzepatide-treated participants with non-missing BMI were pooled, regardless of tirzepatide dose assigned. Participants with ‘improved’ (≥1 category lower vs. baseline), ‘stable’, or ‘worsened’ (≥1 category higher vs. baseline) BMI category at endpoint were assessed using weight-related PRO instruments at baseline and endpoint - Impact of Weight on Self-Perceptions (IW-SP), Impact of Weight on Quality of Life-Lite-Clinical Trials (IWQOL-Lite-CT), and Ability to Perform Physical Activities of Daily Living (APPADL). All PRO scores were greater at endpoint than at baseline for tirzepatide-treated participants with ‘improved’ or ‘stable’ BMI category (Table). Changes were numerically greater across all PROs for participants with ‘improved’ BMI category compared with those whose BMI category remained ‘stable’ or ‘worsened’. Improvements in PRO scores were greater in tirzepatide-treated adults with T2D with improved BMI category than in those with stable or worsened BMI category. Disclosure C.Lee: Employee; Eli Lilly and Company, Stock/Shareholder; Eli Lilly and Company. K.Boye: Employee; Eli Lilly and Company. V.Thieu: None. S.Allen: None. W.Dong: Employee; TechData Service Company, Other Relationship; Eli Lilly and Company, AstraZeneca. H.Sapin: Employee; Eli Lilly and Company, Stock/Shareholder; Eli Lilly and Company. Funding Eli Lilly and Company

0871 Outcomes in Overlap Syndrome of COPD and OSA: A Secondary Data Analysis of STAGES

Abstract Introduction Chronic obstructive pulmonary disease (COPD) and obstructive sleep apnea (OSA) are highly prevalent, and termed Overlap Syndrome (OVS) when coexistent. Patient-reported outcomes (PROs) are impaired in both COPD and OSA alone, but their combined impact is unclear. We examined differences in the impact of COPD on PROs in adults with and without OSA. Methods Analyses included adults aged≥8 years with polysomnographic (PSG) data from the Stanford Technology Analytics and Genomics in Sleep (STAGES) study. We created sets of individuals with and without COPD, matched up to 1:5 on age±5 years, BMI±2.5 kg/m2, AHI±5 events/hour, and sex. Among all matched sets, we estimated the impact of COPD on fatigue (Fatigue Severity Scale), sleepiness (Epworth Sleepiness Scale), anxiety (Generalized Anxiety Disorder Questionnaire), depression (Personal Health Questionnaire-9), insomnia (Insomnia Severity Index), and function (Functional Outcomes of Sleep Questionnaire) using linear mixed effects models. We also examined whether the effect of COPD differed between those with (AHI≥10 events/hour) and without (AHI&amp;lt; 10 events/hour) OSA. Results Overall, 56 patients with COPD were matched to a total of 199 non-COPD controls (51.0% males). The sample was middle aged (55.8±11.8 years), overweight/obese (BMI 29.8±6.9 kg/m2), and had mild/moderate OSA (AHI 11.0±11.5 events/hour) on average; 26 (46.4%) of COPD cases had an AHI≥10. Among all patients, COPD was associated with worse PROs on average (p&amp;lt; 0.05 for all but ESS [p=0.070]). Among those with AHI&amp;lt; 10, there were no differences for any PRO between groups with and without COPD. However, COPD was associated with worse values of all PROs among those with OSA, including fatigue (mean [95% CI] difference = 9.8 [2.9, 16.7], p=0.006), sleepiness (2.8 [0.01, 5.7], p=0.050), anxiety (3.6 [1.1, 6.0], p=0.005), depression (4.4 [1.8, 7.0], p=0.001), insomnia (4.8 [2.0, 7.6], p=0.001) and function (-3.3 [-5.2, -1.5], p=0.001). Conclusion The effect of COPD on PROs is modified by OSA, with worse outcomes among those with OVS compared to OSA alone, but no impact of COPD among those without OSA. Identification of adults with OVS through early screening is crucial to prompt treatment of disease and reduce symptom burden. Support (if any) T32HL07713 P01 HL094307

Sleep optimization to improve glycemic control in adults with type 1 diabetes: study protocol for a randomized controlled parallel intervention trial

BackgroundDespite improvements in treatment regimens and technology, less than 20% of adults with type 1 diabetes (T1D) achieve glycemic targets. Sleep is increasingly recognized as a potentially modifiable target for improving glycemic control. Diabetes distress, poor self-management behaviors, and reduced quality of life have also been linked to sleep variability and insufficient sleep duration. A significant gap of knowledge exists regarding interventions to improve sleep and the effects of sleep optimization on glycemic control in T1D. The purpose of this study is to determine the efficacy of a T1D-specific sleep optimization intervention (Sleep-Opt) on the primary outcomes of sleep variability, sleep duration, and glycemic control (A1C); other glycemic parameters (glycemic variability, time-in-range [TIR]); diabetes distress; self-management behaviors; quality of life; and other patient-reported outcomes in adults with T1D and habitual increased sleep variability or short sleep duration.MethodsA randomized controlled parallel-arm study will be employed in 120 adults (aged 18 to 65 years) with T1D. Participants will be screened for habitual sleep variability (> 1 h/week) or insufficient sleep duration (< 6.5 h per night). Eligible subjects will be randomized to the Sleep-Opt intervention group or healthy living attention control group for 12 weeks. A 1-week run-in period is planned, with baseline measures of sleep by actigraphy (sleep variability and duration), glycemia (A1C and related glycemic measures: glycemic variability and TIR using continuous glucose monitoring), and other secondary outcomes: diabetes distress, self-management behaviors, quality of life, and additional patient-reported outcomes. Sleep-Opt is a technology-assisted behavioral sleep intervention that we recently developed that leverages the rapidly increasing public interest in sleep tracking. Our behavioral intervention employs four elements: a wearable sleep tracker, didactic content, an interactive smartphone application, and brief telephone counseling. The attention control group will participate in a healthy living information program. Baseline measures will be repeated at midpoint, program completion, and post-program (weeks 6, 12, and 24, respectively) to determine differences between the two groups and sustainability of the intervention.DiscussionA better understanding of strategies to improve sleep in persons with T1D has the potential to be an important component of diabetes.Trial registrationClinical Trial Registration: NCT04506151.

Rationale, design and methodology of APPROACH-IS II: International study of patient-reported outcomes and frailty phenotyping in adults with congenital heart disease

BackgroundIn recent years, patient-reported outcomes (PROs) have received increasing prominence in cardiovascular research and clinical care. An understanding of the variability and global experience of PROs in adults with congenital heart disease (CHD), however, is still lacking. Moreover, information on epidemiological characteristics and the frailty phenotype of older adults with CHD is minimal. The APPROACH-IS II study was established to address these knowledge gaps. This paper presents the design and methodology of APPROACH-IS II. Methods/designAPPROACH-IS II is a cross-sectional global multicentric study that includes Part 1 (assessing PROs) and Part 2 (investigating the frailty phenotype of older adults). With 53 participating centers, located in 32 countries across six continents, the aim is to enroll 8000 patients with CHD. In Part 1, self-report surveys are used to collect data on PROs (e.g., quality of life, perceived health, depressive symptoms, autonomy support), and explanatory variables (e.g., social support, stigma, illness identity, empowerment). In Part 2, the cognitive functioning and frailty phenotype of older adults are measured using validated assessments. DiscussionAPPROACH-IS II will generate a rich dataset representing the international experience of individuals in adult CHD care. The results of this project will provide a global view of PROs and the frailty phenotype of adults with CHD and will thereby address important knowledge gaps. Undoubtedly, the project will contribute to the overarching aim of improving optimal living and care provision for adults with CHD.

Smartphone-based behavioral monitoring and patient-reported outcomes in adults with rheumatic and musculoskeletal disease

BackgroundRheumatic and musculoskeletal diseases (RMD) are associated with depression, fatigue, and disturbed sleep – symptoms that often impact behavior and activity. Patient reported outcomes (PROs) are a way of collecting information on the patient symptom experience directly from the individual. The purpose of this study was to measure and compare user smartphone sensor and activity data in adults with RMDs and assess associations with PROs.MethodsWe invited adults with RMDs enrolled in the FORWARD Databank to participate by installing a custom app on their smartphone and answering PROs (pain, global, HAQ-II) questions daily and weekly over 3 years. Passive data collected included mobility distance, unique calls and text messages, call durations, and number of missed calls. Confounders included sociodemographic, clinical, passive phone behavior, and seasonal factors. Kappa statistics between PRO and flares were computed to measure agreement. The agreement between daily and weekly VAS pain was estimated using the intraclass (ICC) correlation of a two-way random effect model. The relationship between the weekly PRO outcomes and the passive phone data was analyzed with a linear mixed-effect model (LMM), including a random intercept for participant and slope for time in the study with an unstructured covariate structure.ResultsOf the 446 participants, the mean (SD) age was 54 (12) years, most (65.5%) had rheumatoid arthritis (RA), the vast majority (91%) were female, and the US Northeast has the least representation (12%). Longer reaction times, interaction diversity, and higher mobility were associated with worse PROs while longer text messages were associated with better PROs. Participants in this study showed good levels of adherence which holds promise for future interventions using passive behavior measures in self-management and clinical follow-up.ConclusionThis is the first study to examine passive smartphone behavior with PROs in RMDs and we found significant associations between these behaviors and important health outcomes of pain and function. As smartphone usage continues to change, future studies should validate and expand on our findings with a goal of finding changes in patient symptoms passively through mobile device monitoring.

Long-term Adherence To Exercise In Adults With Preserved Ejection Fraction Heart Failure

<h3>Background</h3> Despite exercise being one of few strategies to improve outcomes for individuals with heart failure with preserved ejection fraction (HFpEF), exercise clinical trials in HFpEF are plagued by poor interventional adherence. Over the last 2 decades, our research team has developed, tested, and refined Heart failure Exercise And Resistance Training (HEART) Camp, a multi-component behavioral intervention to promote adherence to exercise in HF. We evaluated the effects of this intervention designed to promote adherence to exercise in HF focusing on a subgroup of participants with HFpEF. <h3>Methods and Results</h3> The randomized controlled trial included 204 adults with stable, chronic HF. Of those enrolled, 59 had HFpEF and were the target of this sub-group analysis. We tested adherence to exercise [defined as greater than or equal to 120 minutes of moderate-intensity (40-80% of heart rate reserve) exercise per week validated with a heart rate monitor] at 6, 12, and 18 months. We also tested intervention effects on symptoms (PROMIS-29 and Dyspnea-fatigue index), HF-related health status (Kansas City Cardiomyopathy Questionnaire, KCCQ), and physical function (6-minute walk test, 6MWT). Participants with HFpEF (n=59) were a mean of 64.6 ±9.3 years old, 54% male, 46% non-white with a mean ejection fraction of 55±6. Participants with HFpEF in the HEART Camp intervention group had significantly greater adherence compared to enhanced usual care at both 12 (43% vs. 14%, <i>phi</i>=.32, medium effect) and 18 months (56% vs. 0%, <i>phi</i>=.67, large effect). HEART Camp significantly improved walking distance on the 6MWT (η² = 0.13, large effect) and the KCCQ overall (η²=0.09, medium effect), clinical summary (η²=0.16, large effect), and total symptom (η²=0.14, large effect) scores. <h3>Conclusions</h3> A multi-component, behavioral intervention can be used to improve long-term adherence to exercise, physical function, and patient-reported outcomes in adults with HFpEF. Our results provide strong rationale for a large HFpEF clinical trial to validate these findings and examine interventional mechanisms and delivery modes that may further promote adherence, and improve clinical outcomes, in this population.