Changes In Health-related Quality Of Life Research Articles

Estimating individual health-related quality of life changes in low back pain patients

BackgroundThere is a need to evaluate different options for estimating individual change in health-related quality of life for patients with low back pain.MethodsSecondary analysis of data collected at baseline and 6 weeks later in a randomized trial of 749 adults with low back pain receiving usual medical care (UMC) or UMC plus chiropractic care at a small hospital at a military training site or two large military medical centers. The mean age was 31; 76% were male and 67% were White. The study participants completed the Patient-Reported Outcomes Measurement Information System (PROMIS®)-29 v 1.0 physical function, pain interference, pain intensity, fatigue, sleep disturbance, depression, anxiety, satisfaction with participation in social roles, physical summary, and mental health summary scores (T-scored with mean = 50 and standard deviation (SD) = 10 in the U.S. general population).ResultsReliability estimates at the baseline ranged from 0.700 to 0.969. Six-week test–retest intraclass correlation estimates were substantially lower than these estimates: the median test–retest intraclass correlation for the two-way mixed-effects model was 0. 532. Restricting the test–retest reliability estimates to the subset who reported they were about the same as at baseline on a retrospective rating of change item increased the median test–retest reliability to 0.686. The amount of individual change that was statistically significant varied by how reliability was estimated, and which SD was used. The smallest change needed was found when internal consistency reliability and the SD at baseline were used. When these values were used, the amount of change needed to be statistically significant (p < .05) at the individual level ranged from 3.33 (mental health summary scale) to 12.30 (pain intensity item) T-score points.ConclusionsWe recommend that in research studies estimates of the magnitude of individual change needed for statistical significance be provided for multiple reliability and standard deviation estimates. Whenever possible, patients should be classified based on whether they 1) improved significantly and perceived they got better, 2) improved significantly but did not perceive they were better, 3) did not improve significantly but felt they got better, or 4) did not improve significantly or report getting better.

Family Caregivers of Children With Medical Complexity: Changes in Health-Related Quality of Life and Experiences of Care Coordination

ObjectiveExamine the longitudinal association between family experiences of care coordination (FECC) and health-related quality of life (HR-QOL) for family caregivers of children with medical complexity (CMC). MethodsA longitudinal survey of family caregivers of CMC was completed between July 2018 and June 2020. Baseline data were collected at initial contact with a regional complex care center; follow-up data were collected 12 to 16 months later. Assessed receipt of care coordination and caregiver HR-QOL via FECC questionnaire and Center for Disease Control’s HR-QOL-14 measure, respectively. Baseline and follow-up results were compared via McNemar’s and Wilcoxon signed-rank tests. Relationships between changes in FECC and changes in HR-QOL were examined using multivariable logistic regression. ResultsOf 185 eligible, 136 caregivers enrolled and completed baseline surveys (74%) and 103 (76% initial sample) follow-up surveys. Caregivers reported significant improvements in 8 of 9 FECC measures after 1 year of care within a complex care center (all P < .05). In contrast, caregiver HR-QOL (general health status, unhealthy days, symptom days) remained stable over the study period (all P > .05) except for monthly days of poor sleep (baseline vs follow-up median; 16 vs 15 [P = .05]). At both timepoints, >20% participants rated their general health status as fair-to-poor, and >50% reported frequent poor sleep and fatigue. No significant associations were observed between changes in FECC and changes in HR-QOL. ConclusionsAfter receiving 1 year of care through a complex care center, CMC family caregivers report improvement in care coordination but not in HR-QOL. Caregivers’ continued mentally unhealthy days and negative mental symptom days highlight the need for a directed intervention.

P26: Self-reported health-related quality of life for people with dementia in long-term care facilities in Taiwan: a nationwide survey

Background:Quality of life is an essential outcome parameter in geriatric research; however, research to date offers mixed evidence about the factors associated with health-related quality of life (HRQOL) among people with dementia (PWDs). We aimed to identify factors relating to HRQOL among PWDs living in long-term care (LTC) facilities.Methods:A total of 299 from 1,607 registered long-term care facilities were randomly selected in every administrative region of Taiwan. A cross-sectional two-phase survey, which included the demographic data, comorbidities, EuroQol-5 dimensions-5 levels (EQ-5D-5L), mini-mental state examination (MMSE) score, clinical dementia rating scores (CDR), behavioral and psychological symptoms of dementia, and activities of daily living (ADL) of PWDs, was then conducted from 2019 to 2020.Results:A total of 1,313 PWDs who self-completed EQ-5D-5L were enrolled in this analysis with a mean age of 76.43 ± 12.7 years. The mean utility and visual analogue scale (VAS) scores of EQ-5D-5L were 0.10 (standard deviation, SD = 0.48) and 66.57 (SD = 20.67), respectively. Multivariate linear regression analysis showed that higher scores in ADL, instrumental ADL, and lower CDR sum scores were associated with higher EQ-5D-5L utility scores. Higher ADL scores and higher MMSE scores were associated with higher scores in an EQ-5D-5L-VAS. More depressive symptoms were related to both lower utility score and lower EQ-5D-5L-VAS score.Conclusion:Findings highlight the importance to recognize and to treat depression to maintain HRQOL of PWDs in LTC facilities. Longitudinal studies are needed to better understand the long-term changes in HRQOL of PWDs.

Longitudinal Differences By Treatment Arm in Health-Related Quality of Life Among High Risk Pediatric Hodgkin's Lymphoma Patients Treated on the Children's Oncology Group Ahod 1331 Study

Introduction: Brentuximab vendotin (BV) with AVE-PC demonstrated superior efficacy to ABVE-PC for pediatric patients with high-risk HL in the AHOD 1331 trial. However, there are no data regarding the impact on health-related quality of life (HRQoL) with the addition of BV into the treatment of pediatric HL. Methods: Eligibility for AHOD 1331 (NCT 02166463) included previously untreated HL with high risk disease, defined as stage IIB +bulk, IIIB, IVA, or IVB classic HL. Participants were randomized to either BV-AVE-PC (“BV” arm) or ABVE-PC (standard arm). Among the first 309 participants enrolled in a prespecified longitudinal patient-reported outcomes sub study, 280 were age 11+ years and eligible to self-report HRQoL using the seven-item Child Health Ratings Inventories (CHRIs)-Global scale. HRQoL was assessed prior to treatment (T1), at cycle 2 (T2), at cycle 5 (T3), and at the end of treatment (T4). A clinically meaningful increase in HRQoL was considered a change of 7 points in the CHRIs-Global score (CHRIs), translating to a 1/3 standard deviation. Multivariable linear regression estimated the association between demographic/clinical variables and HRQoL at T1. Linear mixed models estimated the change in HRQoL relative to T1 with a time*treatment arm interaction to estimate differences in the change in HRQoL across treatment arm. This model was adjusted for age, sex, race/ethnicity, stage, large mediastinal adenopathy (LMA), B-symptoms at diagnosis, and a time-varying covariate for involved site radiation prior to T4. Results: Participant and disease characteristics were balanced by treatment arm; mean age at enrollment was 15.6 years (SD= 1.9), 52% were female, 60.4% had LMA, and 58% were stage IV. 93% of participants completed the CHRIs at T1, 92% at T2, 89% at T3, and 74% at T4. At T1, female sex (mean (SD) 64.1 (20.2) vs 71.3 (23.3) p=0.004) and fever (60.8 (23.0) vs. 71.9 (20.4) p=0.024) were associated with worse HRQoL. At each time point after pre-treatment (T1), HRQoL was higher, on average, in the BV arm compared to the standard arm (Figure 1). By T2, participants in the BV arm experienced a statistically and clinically significant increase in HRQoL compared to pre-treatment (T1) (Table 1; β=7.3 95%CI 3.2, 11.4; p≤0.001), which was greater than the change experienced in the standard arm (difference in change β=5.1 95%CI -0.2, 10.3; p=0.057). At T3 and T4 the BV arm continued to experience statistically significant improvements in HRQoL relative to T1 (T3 β=5.3 95%CI 0.8, 9.9; p=0.022; T4 β=14.6 95%CI 10.3, 18.9; p≤0.001). The standard arm did not experience a statistically or clinically significant increase in HRQoL from T1 until T4 (β=9.3 95%CI 4.7, 11.5; p&amp;lt;0.001). Conclusion: Patients with high-risk pediatric HL treated with BV-AVE-PC experienced more rapid and sustained improvement in HRQoL compared to patients treated with ABVE-PC. These data also demonstrate the feasibility and importance of incorporating HRQoL assessments into pediatric clinical trials.

A Phase 3 Study of Eltrombopag Vs. Standard First-Line Management for Newly Diagnosed Immune Thrombocytopenia in Children: Trial in Progress Update

Background: Immune thrombocytopenia (ITP) causes low platelet counts, variable bleeding symptoms, and reductions in health-related quality of life (HRQoL). Despite this, novel therapies for newly diagnosed ITP have not been introduced in over 30 years. Eltrombopag, a thrombopoietin receptor agonist (TPO-RA), was FDA approved for children with chronic ITP in 2015. The use of eltrombopag for adults with newly diagnosed ITP has been described in two small single-center trials (Tripathi et al, Int J Hematol, 2014; Gόmez-Almaguer et al, Blood, 2014), and pediatric hematologists do use TPO-RAs off-label in some cases of newly diagnosed ITP (Neunert et al, Pediatr Blood Cancer, 2016). TPO-RAs may be an efficacious and more durable first-line therapy for children with newly diagnosed ITP who require treatment. This abstract provides a 4-year interval update on the Pediatric ITP Newly diagnosed patients Eltrombopag vs Standard therapy (PINES) trial in progress. Study Design and Methods: The PINES trial, NCT03939637, is an investigator-initiated prospective, open label, randomized, multi-center trial sponsored by the ITP Consortium of North America (ICON) and funded by Novartis. The primary objective is to determine if the proportion of patients with a platelet response, defined as ≥3 of 4 platelet counts &amp;gt;50 x10 9/L during weeks 6-12 without rescue treatment, is significantly greater in patients with newly diagnosed ITP treated with eltrombopag compared to those treated with standard first-line therapy. Patients (n=162) from 30 ICON centers (Figure) are randomized 2:1, stratified by age and treatment status, to receive the experimental treatment, eltrombopag, or investigator's choice of 3 standard first-line therapies: prednisone, intravenous immune globulin or anti-D globulin at standardized doses. Eligible patients are ages 1 to &amp;lt;18 with primary ITP, within 3 months of diagnosis, with platelet count &amp;lt;30 x10 9/L who require pharmacologic treatment from the perspective of the treating clinician. Baseline treatment status is 1) upfront treatment, or patients within 10 days of ITP diagnosis with no prior treatment, and 2) treatment failure, or patients previously managed with observation or a first-line standard agent. Patients are ineligible if they can be observed without medication or have severe bleeding. A one-sided z-test, at alpha=0.025, will be used to compare the proportion of patients who have a platelet response between the two treatment arms. All randomized patients will be analyzed in the intention-to-treat analysis. Secondary objectives include treatment arm comparison of bleeding scores (WHO Bleeding Scale and Modified Buchanan Score), changes in HRQoL (measured by the Kids ITP Tool, Hockenberry Fatigue Scale, PROMIS, and Global Change Scale), and changes in percentage of CD4 +25 +Foxp3 + regulatory T (Treg) cells. Due to challenges related to recruitment and the logistics and safety of obtaining frequent assessments during the COVID-19 pandemic, the primary endpoint was re-defined in 2021 to what is considered a clinically equivalent definition of response, from ≥6 of 8 weekly to ≥3 of 4 biweekly platelet counts &amp;gt;50 x10 9/L during weeks 6-12. Baseline Characteristics: Since first site activation in 2019, 100 patients have been enrolled (Table). Subjects include 42 patients aged 1-&amp;lt;6 years, 34 patients aged 6-&amp;lt;12 years, and 24 patients aged 12-&amp;lt;18 years. Thirty-six patients received upfront treatment and 64 patients had treatment failure prior to enrollment. Median baseline platelet count at enrollment was 5 x10 9/L (range 1 - 28 x10 9/L). Median WHO Bleeding Scale at enrollment was 1 (range 0 - 3) and median modified Buchanan Overall Score was 3 (range 0 - 3). Baseline HRQoL surveys have been obtained for &amp;gt;80% of eligible subjects. Treg samples have been obtained for 95% of subjects, and 74% of subjects have consented to optional biology studies. Conclusion: Despite historical difficulty completing interventional trials in pediatric ITP and pandemic-related enrollment challenges, PINES trial enrollment is progressing successfully and will ultimately provide definitive data regarding the efficacy of eltrombopag in children with newly diagnosed ITP. There are no current data supporting the use of TPO-RAs for newly diagnosed ITP in children, or as monotherapy for newly diagnosed ITP in adults. Results from this trial therefore carry the potential to change management practices in ITP.

Quality of Life and Global Response Score in Cutaneous T-Cell Lymphoma

Background: Cutaneous T-cell lymphoma (CTCL) encompasses a heterogenous group of conditions that are typically incurable and managed as chronic, life-long diseases. Clinically, CTCL is associated with pruritus, and skin lesions that can cause pain and cosmetic discomfort, resulting in reduced health-related quality of life (HRQoL) for patients. As these patients often receive life-long therapy, any proposed therapy should maximize therapeutic safety and efficacy as well as patient HRQoL. Improvement in HRQoL may provide an indication to continue therapy, even for patients with partial response. Clinical trial response assessments for CTCL utilize a global response score, which includes skin, nodal and blood assessments (Olsen et al, JCO 2011). Dermatology-specific instruments, such as Skindex-29 (Chren 2012, Dermatol Clin 30), and oncology-specific instruments such as the Functional Assessment of Cancer Therapy - General (FACT-G) (Cella et al, JCO 1993), have been used to assess quality of life in this population. Disease entity, stage, age, sex, education, and treatment regimen are related to the extent of HRQOL impairment in patients with CTCL (Porkert et al, EJC 2018). Correlation between clinical response and HRQoL response has been reported in other cancers, such as renal cell carcinoma (Grimm Oncol Ther 2017) and other dermatologic conditions such as psoriasis (Acta Derm Venereol 2017), but has not been reported in CTCL. We evaluated changes in HRQoL in relation to the global response score in CTCL. Methods: Global response data, as well as Skindex-29 and FACT-G scores, were obtained from the phase III MAVORIC study (Kim et al, Lancet Oncol 2018). Patients with stage IB-IVB mycosis fungoides or S ézary syndrome were prospectively enrolled. Global response score was assessed every 8 weeks. Skindex-29 and FACT-G questionnaires were administered every 8 weeks. Patients with at least a baseline value for Skindex-29 or FACT-G score, at least one subsequent visit with both Skindex-29 or FACT-G value, and an overall response value were included. Changes in scores were calculated from baseline to the first visit with best overall response. A higher score in Skindex-29 indicated worse HRQoL. A higher score on FACT-G indicated improvement in HRQoL. Wilcoxon rank-sum test was performed. Correlation was calculated by Pearson coefficient. Results: 324 patients were evaluable for total Skindex-29 analysis. First visit with best overall response for patients with PD/SD occurred at a median of 1.26 cycles (IQR; 1.26, 14.26); 5.98 cycles (IQR; 1.26, 48.98) for PR; and 10.98 cycles (IQR; 3.98, 36.98) for CR. Patients with CR/PR as best response (n=121) had median change in Skindex-29 of -16 (IQR; -31, -8) and patients with SD/PD as best response rate had median change in Skindex-29 of -4 (IQR; -12, 3) (p &amp;lt;0.001) (Table 1). 333 patients were evaluable for total FACT-G analysis. Patients with CR/PR as best response rate had median change in FACT-G of 6 (IQR; -2, 16) and patients with SD/PD as best response rate had median change in FACT-G of 2 (IQR; -6, 8) (p &amp;lt;0.001) (Table 1). Skindex-29 correlates with global response score overall and across all domains. FACT-G correlates with global response score overall and across all domains, with the exception of social well-being. There was a moderate reverse correlation between change in total Skindex-29 and change in total FACT-G score (R=-0.61, p&amp;lt;0.001) (Figure 1). Conclusion: HRQoL as measured by the Skindex-29 and FACT-G correlate with complete or partial response to therapy in the global response score in patients with CTCL. There is moderate correlation between skin-specific quality of life and general quality of life in this population. Further exploration of factors affecting social well-being in patients with CTCL is warranted. Incorporation of QOL scales in global response score criteria should be considered.

Survival Outcomes and Health-Related Quality of Life in Older Adults Diagnosed with Acute Myeloid Leukemia Receiving Frontline Therapy in Daily Practice.

Acute myeloid leukemia has a poor prognosis in older adults, and its management is often unclear due to its underrepresentation in clinical trials. Both overall survival (OS) and health-related quality-of-life (HRQoL) are key outcomes in this population, and patient-reported outcomes may contribute to patient stratification and treatment assignment. This prospective study included 138 consecutive patients treated in daily practice with the currently available non-targeted therapies (intensive chemotherapy [IC], attenuated chemotherapy [AC], hypomethylating agents [HMA], or palliative care [PC]). We evaluated patients' condition at diagnosis (Life expectancy [Lee Index for Older Adults], Geriatric Assessment in Hematology [GAH scale], HRQoL [EQ-5D-5L questionnaire], and fatigue [fatigue items of the QLQ-C30 scale]), OS, early death (ED), treatment tolerability (TT) and change in HRQoL over 12 months follow-up. The median OS was 7.1 months (IC not reached, AC 5.9, HMA 8.8, and PC 1.0). Poor risk AML category and receiving just palliative care, as well as a higher Lee index score in the patients receiving active therapy, independently predicted a shorter OS. The Lee Index and GAH scale were not useful for predicting TT. The white blood cell count was a valid predictor for ED. Patients' HRQoL remained stable during follow-up.

Fatigue and health-related quality of life in patients with immune thrombocytopenia: a longitudinal assessment in China

ABSTRACT Background There is lacking studies of longitudinally assessment of fatigue and health-related quality of life (HRQoL) among Chinese immune thrombocytopenia (ITP) adults. We aimed to evaluate changes in fatigue and HRQoL and identify the associated factors. Methods Patients’ characteristics, Functional Assessment of Chronic Illness Therapy (FACIT-F) and the ITP-specific Patient Assessment Questionnaire (ITP-PAQ) scores at admission (T0), at discharge (T1), and three months after discharge (T2) were collected. Linear mixed effects models were used to examine changes over time. Results We included 175 patients. The mean score of FACIT-F at T0 was 37.2 and increased at T1 (39.0), while then decreased at T2 (34.7). Patients who were single, retired, had persistent ITP, splenomegaly had more severe fatigue, whereas those who had not received any prior treatment and had a bleeding score of 0 at admission had milder fatigue. The mean score of ITP-PAQ was 57.7 at T0, then gradually increased to 60.3 at T1 and 62.8 at T2. Patients with persistent ITP and those who have never received treatment for ITP have better HRQoL. Conclusion ITP adults’ fatigue and HRQoL were impaired. Patients’ fatigue improved at discharge but worsened at three months after discharge, while HRQoL gradually improved over time.

Health-related Quality of Life During the First 4 Years After Non–Muscle-invasive Bladder Cancer Diagnosis: Results of a Large Multicentre Prospective Cohort

BackgroundThe health-related quality of life (HRQoL) of patients with non–muscle-invasive bladder cancer (NMIBC) may be impaired due to the chronic and burdensome disease course, but longitudinal data are limited. ObjectiveTo evaluate HRQoL outcomes during the first 4 yr after NMIBC diagnosis, and to compare HRQoL across patient characteristics and with a normative population. Design, setting, and participantsPatients with NMIBC (n = 1019) were included from the multicentre prospective cohort UroLife. Data were collected at 6 wk (baseline), and 3, 15, and 51 mo after diagnosis. Longitudinal reference data were obtained from an age- and sex-matched normative population (n = 490). Outcome measurements and statistical analysisCancer- and NMIBC-specific HRQoL outcomes (range 0–100) were evaluated by the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 and EORTC QLQ-NMIBC24 questionnaires, respectively. Linear mixed modelling was used to analyse within-group changes and between-group differences. Results and limitationsThe majority of HRQoL outcomes remained stable over time. Observed changes were only of small clinical relevance. Improvements were noted in insomnia, social functioning, and three NMIBC-specific symptoms, while minor deteriorations in appetite and diarrhoea lasted until 51 mo. HRQoL in some domains was worse for high-grade NMIBC, high European Association of Urology (EAU) risk group, initial Bacillus Calmette-Guérin (BCG) treatment, being female, and being younger (<65 yr); yet differences were few, small, and temporary. No differences were observed across recurrence status. Compared with a normative population, clinically relevant worse scores were observed for six of 15 outcomes, which mostly recovered at 51 mo, except for minor symptoms of appetite loss and diarrhoea. ConclusionsNo remarkable changes in HRQoL were observed during the first 4 yr after NMIBC diagnosis. Grade, EAU risk group, initial treatment, recurrence, sex, and age did not importantly affect HRQoL. HRQoL was largely comparable with that of the general population, especially after 4 yr. Patient summaryQuality of life is not largely affected during the first 4 yr after non–muscle-invasive bladder cancer diagnosis.

Effect of telemedicine on health-related quality of life in patients with heart failure: insights from the iCOR randomised controlled trial

Abstract Background/introduction Heart failure (HF) can negatively affect health-related quality of life (HRQoL) in HF patients by reducing their independence and limiting their daily activities. Patient-reported outcome measures (PROMs), such as HRQoL, are useful indicators to health status and have good correlation with prognosis. In the past years the addition of telemedicine (TM) to HF programmes has emerged as a promising strategy for the management of chronic HF patients. However, there is little information about the impact of TM on HRQoL. Purpose To determine the effect of a TM-based intervention on HRQoL of high-risk chronic HF patients compared to usual care (UC). Methods We performed a sub-analysis of the "Insuficiència Cardíaca Optimització Remota" (iCOR) study focusing on HRQoL. iCOR was a single centre, randomised, controlled trial, designed to evaluate a TM intervention added to an existing nurse-based, hospital/primary care multidisciplinary, integrated programme for high-risk chronic HF patients. Participants were randomised to TM or UC after hospital discharge ("vulnerable-phase") and were followed for 6 months. The primary study endpoint for the current analysis was the delta change of HRQoL after 6 months of follow-up, assessed using Minnesota Living with Heart failure Questionnaire (MLHFQ). Impaired HRQoL was considered when patient scored &amp;gt; 55 points. To assess the change in HRQoL between the 2 groups, a repeated-measures ANOVA analysis was performed, adjusted for baseline HRQoL. Results 178 patients with HF were analysed (81 TM vs 97 UC). The median age of the patients was 74±11 years, 41% were female and 57% had preserved ejection fraction. As shown in figure 1, HRQoL was poor at baseline (median score of 55±20 points in TM vs 59±19 points in UC) and was not different between both groups (p-value &amp;gt; 0.05). Although both groups showed an improvement in HRQoL after 6 months of follow-up (p-value 0.001 for time effect), HRQoL improved significantly more in the TM-based intervention group (from 55±20 to 14±15, mean Delta change= -37.9±26.4) than in UC group (from 59±19 to 23±21, mean delta change = -29.7±29.1, p-value 0.008) (Figure 1). We designed several multivariate linear regression models exploring the determinants of HRQoL at baseline and the determinants of change in HRQoL over time. As shown in Table 1, HRQoL at baseline, cognitive function (MMSE [mini-mental state examination] and Pfeiffer), affective status and randomisation arm were the only independent predictors of improvement of HRQoL over time. Conclusions The addition of TM to a standard HF programme is effective in improving HRQoL in high risk patients with chronic HF regardless cognitive function, affective status or frailty.HRQoL status changeDeterminants of HRQoL

Factors associated with health-related quality of life in patients undergoing percutaneous coronary intervention: Thai PCI registry.

Percutaneous coronary intervention (PCI) has been shown to improve health-related quality of life (HRQoL) in patients with coronary artery disease (CAD). The objectives of this study were to assess the changes in HRQoL and factors influencing these changes in CAD patients after undergoing PCI. Data from a nationwide PCI registry across 39 hospitals in Thailand were collected in 2018-2019, including baseline characteristics, comorbid diseases, angiographic CAD severity, procedural details, and type of health insurance. HRQoL, as measured by utility scores, was determined in all patients using the Thai version of EQ-5D-5l at admission, discharge, and 6 and 12 months after discharge. The effects of time after PCI procedure and various factors on mean utility scores were assessed using a mixed-effect linear regression model. A total of 19,701 patients were included in the analysis; they had a mean age of 64.2 ± 11.7 years and were predominantly (69.1%) male. Following PCI, the mean utility scores increased from 66.6 ± 19.6 at admission to 81.9 ± 13.8 at discharge, and remained stable at 6 and 12 months (86.1 ± 12.3 and 88.0 ± 11.7, respectively). After adjusting for potential confounding variables, several factors were found to be independently associated with improved HRQoL, including angiographic success, male gender, overweight status, dyslipidemia, and radial access. Six other factors were associated with less improved HRQoLs, including cardiogenic shock/IABP support, old age, CKD, clinical presentation (STEMI and NSTEMI), prior cerebrovascular disease, and heart failure. There were no associations of CAD severity and procedural details with HRQoL. No differences were found related to type of health insurance, except that patients who were uninsured or self-pay tended to have less improvement in HRQoL. HRQoL improved significantly after PCI in these subjects, as observed through 1 year of follow-up. Identifying the factors influencing these improvements may assist clinicians in tailoring patient interventions to optimise quality of life after PCI.

Abstract 12112: Establishing a Minimally Clinically Important Difference Threshold for Health-Related Quality of Life in Atrial Fibrillation: The SAGE-AF Study

Introduction: Health related quality of life (HRQoL) is increasingly recognized as an important patient reported outcome but few research has focused on HRQoL meaningful changes overtime. Using data from Systematic Assessment of Geriatric Elements in Atrial Fibrillation, we identified clinically meaningful change over 1-year in older patients with AF and examined sociodemographic and geriatric factors associated with change. Methods and Results: Between 2016-18, 1,244 patients aged 65+ with atrial fibrillation (AF) were recruited from medical centers in Massachusetts and Georgia. At baseline and 1-year follow-up, patients completed validated HRQoL measure. We used a distribution-based approach to calculate a clinically meaningful threshold (9 points) and to define three groups: improve (N=154, 14%), stable, (N=617, 56%), and decline (N=326, 30%). Logistic regression was used to examine factors associated with clinically meaningful improvement and decline in HRQoL. CHA2DS2-VASC score was associated with an increased odds of HRQoL decline (adjusted odds ratio [AOR]= 1.16, 95% CI: 1.03, 1.32). Frailty (AOR= 1.89, 95% CI: 1.20, 2.92) and depression, (AOR=1.62, 95% CI: 1.17, 2.25) were associated with an increased odds of HRQoL improvement, while cognitive impairment (AOR= 0.72, 95% CI: 0.52, 0.99) was associated with a decreased odds of improvement in HRQoL. Conclusions: A change in HRQoL of 9-points was identified as clinically meaningful over 1-year in our cohort. The majority of patients remained stable in HRQoL over a year of follow-up. Understanding meaningful change in HRQoL can assist providers with knowing how to best treat their patients and develop strategies to improved or stabilize HRQoL.

Abstract 15211: Age and Improvement in Health- Related Quality of Life After Percutaneous Coronary Intervention

Background: The purpose of percutaneous coronary intervention (PCI) is to relieve chest pain, improve health-related quality of life (HRQoL), or reduce the risk of cardiac adverse events and death among patients with coronary artery disease. The aim of this study was to investigate whether age is related to HRQoL improvement in patients undergoing PCI and which clinical characteristics explain the changes in HRQoL during 12 months. Methods: Altogether 301 patients undergoing PCI were divided into three age groups: ≤65 (n=94), 66-74 (n=117), and ≥75 (n=90). Patients responded to the generic 15D and disease-specific SAQ-7 HRQoL instruments at baseline and 1 and 12 months after PCI. Results: The median ages in groups were 60, 69, and 79 years, and the proportions of women were 19.1%, 32.5%, and 35.6%, respectively. Baseline instrument scores were lower in the oldest group compared to the youngest groups (15D p &lt;0.001, SAQ-7 p =0.006). 15D and SAQ-7 improved statistically ( p &lt;0.001 for all) and clinically significantly in all groups at 1 month. At 12 months, the 15D score decreased in all groups but remained statistically and clinically higher compared to baseline in the two youngest groups, and in the oldest group, it was statistically ( p =0.005) and nearly clinically higher (threshold 0.015, Δ score 0.013, 95% CI -0.003-0.030). In the oldest group, deterioration in the 15D between 1 and 12 months was due to dimensions “mobility”, “vision”, and “excretion” but not in “cardiac-related” dimensions. Significant improvement in SAQ-7 score at one-year follow-up was observed in all groups ( p &lt;0.001 for all). The improvements in 15D and SAQ-7 scores at 12 months showed no difference between the age groups ( p =0.381). Conclusions: There was no difference in the improvement of generic or disease-specific HRQoL after PCI between the age groups. Importantly, both 15D and SAQ-7 improved equally in the oldest groups as in the younger patients.

Abstract 15228: Impact of Gender and Left Ventricular Ejection Fraction on Health-Related Quality of Life and Its Change in Patients With Heart Failure

Introduction: Health-related quality of life (HRQoL) is an important part in managing patients with heart failure (HF). However, the influence of clinical factors on HRQoL in HF remains limited. Hypothesis: This study aimed to examine HRQoL changes based on gender and left ventricular ejection fraction (LVEF) among patients with HF. Methods: A total of 199 patients hospitalized for acute HF (mean age, 71±14 years; men:women=119:80; mean LVEF=42±16) were enrolled in a prospective way from Jan 2022 to Jan 2023. Baseline and 4-month follow-up Kansas City Cardiomyopathy Questionnaire (KCCQ) scores were assessed. Patients were categorized into 4 subgroups: Group 1, women with LVEF≥50% (n=37); Group 2, men with LVEF≥50% (n=26); Group 3, women with LVEF&lt;50% (n=43); Group 4, and men with LVEF&lt;50% (n=93). Results: Women demonstrated lower KCCQ clinical and overall summary scores than men at baseline (p=0.003, 0.004, respectively) and 4-month follow-up (p=0.029, 0.038, respectively) after adjusting for age and body mass index. In addition, these scores tended to be lower in patients with LVEF ≥50% than those with LVEF &lt;50% at both baseline and follow-up (all p&lt;0.05). When we assessed KCCQ scores according to group, group 1 showed the lowest KCCQ overall summary score (KCCQ-OS: 36.9 [IQR 22.9-49.8], p=0.004) and clinical summary score (KCCQ-CS: 37.8 [IQR: 24.5-51.1], p=0.001). Also, the scores for physical limitation, symptom frequency, symptom burden, social limitation, self-efficacy scale, and total symptom score were lowest in Group 1 (all p&lt;0.05). Although HRQoL improved across all groups, the follow-up KCCQ-OS and KCCQ-CS scores remained lowest in Group 1 (KCCQ-OS: 54.0 [IQR 44.3-64.4], p=0.026; KCCQ-CS: 56.5 [IQR: 45.0-63.9], p=0.031). Conclusions: Among patients with HF, women with LVEF≥50% reported significantly worst HRQoL even after HF treatment, suggesting more attention should be paid to HRQoL in managing this specific population during HF treatment.

Measurement of Health-Related Quality of Life in Individuals With Rare Diseases in China: Nation-Wide Online Survey.

Rare diseases (RDs) affect millions of people worldwide, and these diseases can severely impact the health-related quality of life (HRQoL) of those affected. Despite this, there is a lack of research measuring HRQoL using the EQ-5D-5L, which is one of the most widely used generic preference-based instruments to measure HRQoL in populations living with RDs. This study aimed to measure HRQoL using the EQ-5D-5L in a large number of patients with various types of RDs in China, and to examine the relationship between respondents' socioeconomic characteristics and preference-based health utility scores. The data used in this study were obtained from a web-based survey conducted in China. The survey aimed to explore and understand the health and socioeconomic status of patients with RDs in China. We recruited registered and eligible members, including patients or their primary caregivers, from 33 RD patient associations to complete the questionnaires via their internal social networks. HRQoL was measured using the EQ-5D-5L utility score, which was calculated based on an established Chinese value set. Utility scores have been presented based on demographics and disease-related information. Univariate linear regression analysis was used to assess the differences in the EQ-5D-5L utility scores between subgroups. A total of 12,502 respondents completed the questionnaire and provided valid responses, including 6919 self-completed respondents and 5583 proxy-completed respondents. Data from 10,102 participants over the age of 12 years were elicited for analysis. Among patients with RDs, 65.3% (6599/10,102), 47.5% (4799/10,102), 47.0% (4746/10,102), 24.8% (2506/10,102), and 18.4% (1855/10,102) reported no problems for "self-care," "usual activities," "mobility," "pain/discomfort," and "anxiety/depression," respectively. A full health state was reported by 6.0% (413/6902) and 9.2% (295/3200) of self- and proxy-completed patients, respectively. Among self-completed patients, 69.9% (4826/6902) and 50.4% (3478/6902) reported no problems for "self-care" and "usual activities," respectively, whereas only 17.7% (1223/6902) reported problems for "anxiety/depression." Proxy-completed respondents showed a higher proportion of reporting extreme problems than self-completed respondents in all 5 dimensions. The mean utility scores reported by self- and proxy-completed respondents were 0.691 and 0.590, respectively. Different types of caregivers reported different utility scores, and among them, proxy-completed (mother) respondents reported the highest mean utility score. The establishment of a normative profile for RD patients can facilitate patients' adaptation and assess the effectiveness of interventions to improve the HRQoL and well-being of this population. Differences between self- and proxy-completed HRQoL assessed by the EQ-5D-5L have been identified in this study. This finding highlights the importance of incorporating perspectives from both patients and their proxies in clinical practice. Further development of the patient cohort is necessary to assess long-term changes in HRQoL in the RD population.

Multicomponent Training Improves the Quality of Life of Older Adults at Risk of Frailty.

Achieving a high quality of life in older adults can be difficult if they have limited physical function. The aims of this study were to evaluate the relationship between baseline values and variations in body composition, fitness, and nutritional status on health-related quality of life (HRQoL) and to describe the effects of a 6-month multicomponent training (MCT) programme and a 4-month detraining period on HRQoL. A total of 106 participants with limited physical function were included in this study (age: 80.8 ± 5.9 years; 74 females) and were divided into two groups: control (CON) and intervention (TRAIN). HRQoL was measured using the EQ-5D-3L questionnaire and a visual analogue scale (EQ-VAS). Information on body composition, physical fitness, Mediterranean diet adherence, and nutritional status were obtained. Healthier baseline values for body composition, fitness and nutritional status were associated with better HRQoL (explaining 23.7-55.4%). The TRAIN group showed increased HRQoL during this 6-month MCT, showing group-by-time interaction (p < 0.05) and a deleterious effect of detraining. Changes in weight, arm strength, and aerobic capacity contributed to explaining 36% of the HRQoL changes obtained with MCT (all p < 0.05). This MCT improved HRQoL in older adults with limited physical function. However, HRQoL returned to baseline values after detraining. This study highlights the importance of performing ongoing programs in this population.

"not one size fits all” The challenges of measuring paediatric health-related quality of life and the potential role of digital ecological momentary assessment: a qualitative study

PurposeTo explore the views of clinicians and researchers about the challenges of measuring health-related quality of life (HRQoL) in children (5–11 years) and to explore whether digital ecological momentary assessment (EMA) could enhance HRQoL measurement.MethodsSemi-structured qualitative interviews with 18 professionals (10 academics/researchers, four clinicians, four with both professional backgrounds) experienced in child HRQoL measurement. We analysed data thematically.ResultsTheme One describes the uncertainty around conceptualising HRQoL for children and which domains to include; the greater immediacy and sensitivity of children’s reflections on their HRQoL, leading to high variability of the construct; and the wide individual differences across childhood, incongruent with fixed HRQoL measures. Theme Two describes the challenges of proxy reporting, questioning whether proxies can meaningfully report a child’s HRQoL and reflecting on discrepancies between child and proxy reporting. Theme Three covers the challenge of interpreting change in HRQoL over time; does a change in HRQoL reflect a change in health, or does this reflect developmental changes in how children report HRQoL. Theme Four discusses digital EMA for HRQoL data capture. In-the-moment, repeated measurement could provide rich data and address challenges of recall, ecological validity and variability; passive data could provide objective markers to supplement subjective responses; and technology could enable personalisation and child-centred design. However, participants also raised methodological, practical and ethical challenges of digital approaches.ConclusionDigital EMA may address some of the challenges of HRQoL data collection with children. We conclude by discussing potential future research to explore and develop this approach.

Health-Related Quality of Life and Sleep Habits of Children with Overweight or Obesity

Abstract Background Obesity is a major public health challenge affecting children of all ages worldwide. Being overweight or obese can have severe negative psychological impacts and a significant reduction in health-related functioning. To improve health-related quality of life (HRQoL) and sleep habits for children with overweight/obesity, it is important to design and implement effective interventions. Few studies have included parents as behavioral agents to improve sleep habits and quality of life in children and adolescents with overweight/obesity. Methods This 6-month, non-randomized, cluster-controlled trial included 166 Norwegian children aged 5-13 years with overweight/obesity and their parents. The intervention group received individual family counselling and participated in physical activity groups and nutrition courses. A frequency questionnaire was filled out by parents, and the sleep habits questionnaire (CSHQ) and Kidsscreen-10 index (proxy version) were used to assess HRQoL. Results The results indicate that, overall, the children had good HRQoL. The mean average scores for HRQoL were 50.0 (standard deviation (SD): 8.1) for the intervention group and 49.0 (SD: 10.1) for the control group, which are broadly in line with normative data. For sleep habits, the mean average score was 43.8 (SD: 11.6) for the intervention group and 45.1 (SD: 11.7) for the control group. No significant changes in HRQoL and sleep habits were reported as a result of the intervention. Conclusions The children with overweight/obesity reported overall good HRQol at baseline and somewhat worse for sleep habits than the general population. No increase in quality of life or sleep quality was found in the intervention group compared to the control group in children with overweight/obesity. Key messages • The HRQoL among the children with overweight/obesity was good, but there were clinically relevant challenges related to sleep. • No increase in HRQoL and sleep habits were found after the intervention.

Health-Related Quality of Life for Parents of Infants with Bronchopulmonary Dysplasia

To determine how bronchopulmonary dysplasia (BPD) affects health-related quality of life (HRQL) among infants from NICU hospitalization through 1-year postdischarge. This was a prospective cohort study of infants with BPD and their parents. Parent HRQL was measured with the PedsQL Family Impact Module before NICU discharge and 3- and 12-months post-discharge. At 12months, parent-reported child health outcomes included questions from the Test of Respiratory and Asthma Control in Kids, Warner Initial Developmental Evaluation of Adaptive and Functional Skills, and National Survey of Children with Special Health Care Needs. HRQL change over time was assessed by multivariable linear regression. Of 145 dyads, 129 (89%) completed 3-month follow-up, and 113 (78%) completed 12-month follow-up. In the NICU, lower HRQL was associated with earlier gestational age, postnatal corticosteroids, outborn status, and gastrostomy tubes. At 3months, lower HRQL was associated with readmissions and home oxygen use. At 12months, lower HRQL was associated with parent-reported difficulty breathing, lower developmental scores, and not playing with other children. At 3 and 12months, 81% of parents reported similar or improved HRQL compared with the NICU period. Parents reporting infant respiratory symptoms experienced less improvement. BPD affects parent HRQL over the first year. Most parents report similar or better HRQL after discharge compared with the NICU stay. Less improvement is reported by parents of infants experiencing respiratory symptoms at 12months. Efforts to improve parent HRQL should target respiratory symptoms and social isolation.

OR10-05 Improved Mental And Psychosocial Patient-Reported Outcomes Among People With Obesity Treated With Tirzepatide: Results From SURMOUNT-1 Study

Abstract Disclosure: J. Poon: Employee; Self; Eli Lilly &amp; Company. Stock Owner; Self; Eli Lilly &amp; Company. S. Zhang: Employee; Self; Eli Lilly &amp; Company. Stock Owner; Self; Eli Lilly &amp; Company. H. Kan: Employee; Self; Eli Lilly &amp; Company. Stock Owner; Self; Eli Lilly &amp; Company. M.C. Bunck: Employee; Self; Eli Lilly &amp; Company. Stock Owner; Self; Eli Lilly &amp; Company. J. Dunn: Employee; Self; Eli Lilly &amp; Company. Stock Owner; Self; Eli Lilly &amp; Company. A. Stefanski: Employee; Self; Eli Lilly &amp; Company. Stock Owner; Self; Eli Lilly &amp; Company. M. Murphy: Employee; Self; Eli Lilly &amp; Company. Stock Owner; Self; Eli Lilly &amp; Company. Background: Living with obesity negatively impacts a person’s mental health and psychosocial function, leading to impaired health-related quality of life (HRQoL). Tirzepatide resulted in clinically sustained reductions in body weight among participants with obesity in SURMOUNT-1, a Phase 3 randomized clinical trial. To determine the impact of tirzepatide treatment on mental health and psychosocial function, changes in HRQoL were assessed in SURMOUNT-1 using the Short Form 36 health survey version 2 acute form (SF-36v2) and the Impact of Weight on Quality of Life-Lite Clinical Trials Version (IWQOL-Lite-CT). Methods: A total of 2,539 participants were included in the study (tirzepatide 5 mg, N=630; 10 mg, N=636; 15 mg, N=630; placebo, N=643). The SF-36v2 (36 items) and IWQOL-Lite-CT (20 items) were collected at both baseline and endpoint of primary treatment period (week 72 or early discontinuation, where last observation was carried forward (LOCF)). The SF-36v2 assesses eight domains of HRQoL including role-emotional and mental health, and yields two additional summary scores, including the Mental Component Summary (MCS) score. SF-36v2 scores are norm-based with mean (SD) 50 (10). IWQOL-Lite-CT assesses three primary composites, including 13-item psychosocial composite score. IWQOL-Lite-CT composite scores were transformed to a scale of 0 to 100. Higher scores of both SF-36v2 and IWQOL-Lite-CT indicates better levels of health and psychosocial function. Estimated mean change from baseline to week 72 for SF-36v2 (norm-based) and IWQOL-Lite-CT composite (transformed) scores were analyzed using an analysis of covariance (ANCOVA) model. Results: Mean scores at baseline for tirzepatide 5, 10, 15 mg doses and placebo for SF-36v2 role-emotional were 50.7, 51.0, 50.5, and 50.8, respectively, and for mental health were 53.4, 54.0, 53.4, and 53.4, respectively. Mean MCS scores at baseline for tirzepatide 5, 10, 15 mg doses and placebo were 53.3, 53.8, 53.4, and 53.5, respectively. Mean scores at baseline for tirzepatide 5, 10, 15 mg doses and placebo for IWQOL-Lite-CT psychosocial composite score were 64.3, 62.1, 63.2, and 63.2, respectively. All doses of tirzepatide showed significant improvements at week 72 compared to placebo for both SF-36v2 and IWQOL-Lite-CT scores (p&amp;lt;0.05). Estimated mean change from baseline to week 72 with tirzepatide 5, 10, 15 mg doses versus placebo were as follows: SF-36v2 role-emotional scores, 1.7, 1.3, and 1.8, versus 0.3 (p&amp;lt;0.05); SF-36v2 mental health scores 0.8, 0.8, and 1.1, versus -0.2 (p&amp;lt;0.05); SF-36v2 MCS scores were 0.7, 0.4, and 0.7 versus -0.5 (p&amp;lt;0.05); IWQOL-Lite-CT psychosocial scores 19.6, 22.1, and 23.6 versus 11.0 (p&amp;lt;0.001). Conclusion: In SURMOUNT-1, tirzepatide demonstrated statistically significant improvements in patient-reported mental health and psychosocial function as assessed by SF-36v2 and IWQOL-Lite-CT, compared to placebo. Presentation: Friday, June 16, 2023