Health-related Quality Of Life Impact Research Articles

Empirical Development of a Behavioral Intervention for African American/Black and Latino Persons with Unsuppressed HIV Viral Load Levels: An Application of the Multiphase Optimization Strategy (MOST) Using Cost-Effectiveness as an Optimization Objective.

We used results from an optimization randomized controlled trial which tested five behavioral intervention components to support HIV antiretroviral adherence/HIV viral suppression, grounded in the multiphase optimization strategy and using a fractional factorial design to identify intervention components with cost-effectiveness sufficiently favorable for scalability. Results were incorporated into a validated HIV computer simulation to simulate longer-term effects of combinations of components on health and costs. We simulated the 32 corresponding long-term trajectories for viral load suppression, health related quality of life (HRQoL), and costs. The components were designed to be culturally and structurally salient. They were: motivational interviewing counseling sessions (MI), pre-adherence skill building (SB), peer mentorship (PM), focused support groups (SG), and patient navigation (short version [NS], long version [NL]. All participants also received health education on HIV treatment. We examined four scenarios: one-time intervention with and without discounting and continuous interventions with and without discounting. In all four scenarios, interventions that comprise or include SB and NL (and including health education) were cost effective (< $100,000/quality-adjusted life year). Further, with consideration of HRQoL impact, maximal intervention became cost-effective enough to be scalable. Thus, a fractional factorial experiment coupled with cost-effectiveness analysis is a promising approach to optimize multi-component interventions for scalability. The present study can guide service planning efforts for HIV care settings and health departments.

Development of Patient and Caregiver Conceptual Models Investigating the Health-Related Quality of Life Impacts of Malignant Pleural Mesothelioma.

Malignant pleural mesothelioma (MPM) is a rare and usually fatal malignancy frequently linked to occupational asbestos exposures and associated with poor prognosis and considerable humanistic burden. The study aimed to develop conceptual models of the health-related quality of life (HRQoL) impact on patients with and receiving treatment for MPM, and the burden on their caregivers. This multi-country study (Australia and United Kingdom) adopted a qualitative methodology to conduct semi-structured, independent interviews with people with MPM (n = 26), current caregivers (n = 20), and caregivers of people who had recently died because of MPM (n = 4). Participants were recruited using a purposive sampling approach and interviews conducted via telephone between January 2021 and January 2022. Transcripts were analysed using thematic analysis and used to construct conceptual models. Patient analysis yielded four overarching themes: (1) debilitating burden of breathlessness and fatigue; (2) physical mesothelioma symptoms experienced by patients; (3) distress of MPM on the self and family; and (4) treatment is worth 'having a go' despite the potential impact on symptoms. Caregiver analysis yielded five core themes: (1) daily life limited by caregiving duties; (2) emotional well-being and the need for support; (3) the relational role shift to caregiver; (4) time spent providing care negatively impacts work and productivity; and (5) positive aspects and outcomes of caregiving. This study highlights the substantial daily and emotional HRQoL impact that MPM symptoms have on patients and caregivers. Both groups reduced work, productivity, and social and leisure activities. There was evidence of positive HRQoL impacts as a result of immunotherapy and radiotherapy, but less for chemotherapy. Caregiver impacts were intensified during the end-of-life period and persisted following patient death. Evident is a need for increased psychological support, information, and advice for caregivers, increased during the end-of-life period.

A quantitative cross-sectional study of the burden of caring for patients with Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex-associated epilepsy in Japan

IntroductionLennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC)-associated epilepsy are rare conditions associated with severe childhood-onset epilepsy. Caregivers play a critical role in the patients’ care and may experience significant psychosocial and socioeconomic burden. This cross-sectional study determined the burden of caring for patients with these rare epilepsy conditions in Japan. MethodsA quantitative online survey was used to assess patients’ and caregivers’ characteristics and the caregivers’ emotional state, among others. Several validated questionnaires were used: the Hospital Anxiety and Depression Scale (HADS; 0–21 score) assessed the caregivers’ emotional wellbeing, the Pediatric Quality of Life Inventory Family Impact Module (PedsQL FIM; 0–100 score) assessed the health-related quality of life (HRQoL) of the caregivers and their families, and the Work Productivity and Activity Impairment General Health (WPAI:GH; 0–100 % score) questionnaire assessed work productivity. ResultsA total of 36 caregivers responded (median [interquartile range (IQR)] age 43.5 [39.5, 48.3] years; 33/36 [92 %] female; 13/36 [36 %] working part-time and 13/36 [36 %] not working). Participants cared for 7/36 (19 %), 19/36 (53 %), and 10/36 (28 %) patients with LGS, DS, and TSC, respectively (median [IQR] age, 11.0 [6.8, 16.3] years; age at first seizure, 0 [0, 0] years). Patients received a median (IQR) of 4 (3, 5) treatment drug types. Patients experienced median (IQR) 3.0 (0, 21.0) epileptic seizures in the previous week; 28/36 (78 %) had severe intellectual disabilities, and 34/36 (94 %) had developmental delays. Caregivers reported stress (17/36 [47 %]), sleep problems (13/36 [36 %]), and anxiety (12/36 [33 %]). They spent a median (IQR) of 50.0 (17.5, 70.0) hours caregiving in the previous week, with 3.0 (1.0, 11.0) hours of seizure-specific care. Caregivers reported that their lives would be easier with a median (IQR) of 1.5 (0, 5.0) hours fewer per week caring for patients during/following seizures. Median HADS scores were 9.5 (‘suspected anxiety diagnosis’) and 7.5 (‘no depression’) for caregivers, and PedsQL FIM Total median score was 60.1, indicating HRQoL impairment for the caregiver and their family. WPAI:GH scores for paid workers indicated important work impairment. Higher caregiving hours (≥ 21 h vs. < 21 h in the previous week) resulted in higher caregiver burden as indicated by the HADS Total score (p = 0.0062) and PedsQL FIM Total score (p = 0.0007). ConclusionsCaregivers of patients with LGS, DS, or TSC in Japan experience a significant time burden, reduced HRQoL, and high level of work/activity impairment. Caregivers provide round-the-clock care to patients and rely on family and specialized caring services to help manage the increased caregiving time, which tends to be associated with greater emotional burden and HRQoL impact.

An examination of anxiety and its influence on health-related quality of life in Parkinson’s disease using the geriatric anxiety scale: a cross-sectional study

BackgroundAnxiety is one of the most common but often overlooked mood-related nonmotor symptoms in people with Parkinson’s disease (PD). To improve the well-being of people with PD, it is important to understand the impact of anxiety in PD, especially its association with depressive and motor symptoms and its impact on health-related quality of life (HRQoL).Methods91 people with PD were assessed between June 2017 and June 2018. Anxiety was measured using the Geriatric Anxiety Scale (GAS) and its cognitive, somatic, and affective subscales. HRQoL was assessed using the Parkinson’s Disease Questionnaire 39 (PDQ-39). Moreover, sociodemographic information, depressive symptoms, cognition, motor and nonmotor symptoms were assessed. Descriptive statistics, regression analyses, and path analyses were performed to understand predictors of anxiety and its influence on HRQoL.ResultsOf the 91 people with PD, 35 (38.5%) experienced anxiety. Anxiety symptoms in these individuals primarily manifest as somatic sensations. Anxiety, motor, and depressive symptoms are interlinked but contribute individually to HRQoL. Beyond motor symptoms, cognitive and affective aspects of anxiety impact HRQoL. While anxiety and depression overlap, the somatic and cognitive aspects of anxiety play a significant role in determining HRQoL in addition to depressive symptoms.ConclusionOur study used the GAS and its three subscales to shed light on the connections between anxiety, depression, and motor impairment in people with PD. Although anxiety is linked to depression and motor symptoms, it independently affects the HRQoL of people with PD. Thus, it is crucial to adopt a comprehensive diagnostic approach that detects and considers the impact of anxiety on HRQoL in PD.

Health-Related Quality-of-Life Impacts Associated with Transfusion-Dependent β-Thalassemia in the USA and UK: A Qualitative Assessment.

Individuals living with transfusion-dependent β-thalassemia (TDT) experience reduced health-related quality of life due to fatigue and chronic pain, which cause disruptions to daily life. Currently, limited qualitative data exist that describe these impacts. This study aimed to examine the ways in which symptoms and current treatments of TDT impact health-related quality of life, to holistically describe the humanistic burden of TDT, and to identify the unmet needs of individuals living with TDT. Adults (aged ≥18 years) with TDT and caregivers of adolescents (aged 12‒17 years) with TDT participated in semi-structured one-on-one virtual interviews and focus group discussions. Interviews were conducted in the USA and UK and lasted approximately 60 minutes. After transcription, the interviews were analyzed thematically using a framework approach. A total of ten interviews/focus group discussions (six interviews and four focus group discussions) were conducted with 14 adults with TDT and two caregivers of adolescents with TDT. A framework analysis revealed five themes describing health-related quality of life (negative impacts on daily activities, social life, family life, work and education, and psychological well-being) and three themes describing the lived experience of TDT (impact of red blood cell transfusions and iron chelation therapy, treatment, and stigma). Physical, psychological, and treatment-related factors contributed to negative impacts on daily activities, social and family life, and work and education. Concerns about reduced lifespan, relationships and family planning, and financial independence were detrimental to participants' mental well-being. Participants reported having high resilience to the many physical and psychological challenges of living with TDT. A lack of TDT-specific knowledge among healthcare professionals, particularly regarding chronic pain associated with the disease, left some participants feeling ignored or undermined. Additionally, many participants experienced stigma and were reluctant to disclose their disease to others. Individuals living with TDT experience substantial negative impacts on health-related quality of life that disrupt their daily lives, disruptions that are intensified by inadequate healthcare interactions, demanding treatment schedules, and stigma. Our study highlights the unmet needs of individuals living with TDT, especially for alternative treatments that reduce or eliminate the need for red blood cell transfusions and iron chelation therapy.

The Impact of Non-Seizure Symptoms in Dravet Syndrome and Lennox–Gastaut Syndrome

Dravet syndrome (DS) and Lennox–Gastaut syndrome (LGS) are developmental and epileptic encephalopathies (DEE) that onset in childhood, and persist lifelong. In both, non-seizure symptoms (NSS) include intellectual disability, psychiatric symptoms, speech and communication difficulties, motor and gait difficulties, appetite and eating difficulties, autism spectrum characteristics, attention deficit hyperactivity disorder, and sleep disorders. The NSS impact health-related quality of life (HRQoL) for the affected individual and the caregiver, considering personal time, sleep, finances, energy, and family and social relationship. In this industry-sponsored symposium, three leading ex-perts in DEEs discussed NSS, and how properly assessing and tracking these can lead to more informed understanding of an individual’s needs. This can help to guide treat-ment for NSS and, subsequently, increase HRQoL for both the individual and their caregivers.

Social determinants of health rather than race impact health-related quality of life in 10-year-old children born extremely preterm.

Reducing healthcare disparities among children is extremely important given the potential impact of these disparities on long-term health-related quality of life (HRQL). Race and parental socioeconomic status (SES) are associated with child HRQL, but these associations have not been studied in infants born extremely preterm (EP), a population at increased risk for physical, cognitive, and psychosocial impairments. Achieving health equity for infants born EP across their life course requires identifying the impact of racism and SES on HRQL. We aimed to evaluate the association between self-reported maternal race, SES factors, and HRQL among 10-year-old children born EP. Participants were identified from an ongoing multicenter prospective longitudinal study of Extremely Low Gestational Age Newborns (ELGAN Study), born between 2002 and 2004, and evaluated at 10 years of age using the Pediatric quality of life (QoL) Inventory completed by their parent or guardian, assessing physical, emotional, social, school, and total (composite) QoL domains. Multivariable regression models were used to evaluate the relationship between QoL scores and self-identified maternal race, adjusting for SES factors (education level, marital status, and public insurance). Of 1,198 study participants who were alive at 10 years of age, 863 (72.0%) were evaluated at 10 years of age. Differences in mean 10-year QoL scores across racial groups were observed and were significant on univariate analysis. However, these associations attenuated when adjusted for the marital status, public insurance status, and education status of mothers. A comparison of children with English as the primary language spoken at home vs. any other language revealed a significant difference only in school QoL, in which non-English language was associated with more favorable school QoL scores. Among 10-year-old children born EP, differences in parent-reported QoL were associated with maternal SES factors but not with race. Our results suggest that interventions designed to improve the SES of mothers may enhance the QoL of children born EP. Furthermore, these results underscore that race is a social construct, rather than a biological variable, as we work toward greater equity in care provision.

Developing a multivariable prediction model of global health-related quality of life in patients treated for rectal cancer: a prospective study in five countries

PurposeRectal cancer and its treatment have a negative impact on health-related quality of life (HRQoL). If risk factors for sustained low HRQoL could be identified early, ideally before the start of treatment, individualised interventions could be identified and implemented to maintain or improve HRQoL. The study aimed to develop a multivariable prediction model for global HRQoL 12 months after rectal cancer treatment.MethodsWithin COLOR II, a randomised, multicentre, international trial of laparoscopic and open surgery for rectal cancer, a sub-study on HRQoL included 385 patients in 12 hospitals and five countries. The HRQoL study was optional for hospitals in the COLOR II trial. EORTC QLQ-C30 and EORTC QLQ-CR38 were analysed preoperatively and at 1 and 12 months postoperatively. In exploratory analyses, correlations between age, sex, fatigue, pain, ASA classification, complications, and symptoms after surgery to HRQoL were studied. Bivariate initial analyses were followed by multivariate regression models.ResultsPatient characteristics and clinical factors explained 4–10% of the variation in global HRQoL. The patient-reported outcomes from EORTC QLQ-C30 explained 55–65% of the variation in global HRQoL. The predominant predictors were fatigue and pain, which significantly impacted global HRQoL at all time points measured.ConclusionWe found that fatigue and pain were two significant factors associated with posttreatment global HRQoL in patients treated for rectal cancer T1-T3 Nx. Interventions to reduce fatigue and pain could enhance global HRQoL after rectal cancer treatment.Trial registrationThis trial is registered with ClinicalTrials.gov No. NCT00297791

Health-related quality of life and quality-adjusted progression free survival for carfilzomib and dexamethasone maintenance following salvage autologous stem-cell transplantation in patients with multiple myeloma: a randomized phase 2 trial by the Nordic Myeloma Study Group

BackgroundDecisions regarding maintenance therapy in patients with multiple myeloma should be based on both treatment efficacy and health-related quality of life (HRQL) consequences. In the CARFI trial, patients with first relapse of multiple myeloma underwent salvage autologous stem cell transplantation (salvage ASCT) before randomization to carfilzomib-dexamethasone maintenance therapy (Kd) or observation. The primary clinical endpoint was time to progression, which was extended by 8 months by Kd. The aim of this paper is to present the all HRQL endpoints of the CARFI trial including the HRQL effect of Kd maintenance therapy relative to observation. The primary HRQL endpoint was assessed by EORTC QLQ-C30 Summary score (QLQ-C30-sum) at 8 months follow-up. A key secondary HRQL endpoint was quality-adjusted progression-free-survival (QAPFS).MethodsHRQL was assessed with EORTC QLQ-C30, EORTC QLQ-MY20 and FACT/GOG-Ntx at randomization and every second month during follow-up. HRQL data were analyzed with linear mixed effect models until 8 months follow-up. QAPFS per individual was calculated by multiplying progression-free survival (PFS) by two quality-adjustment metrics, the QLQ-C30-sum and EORTC Quality of Life Utility Measure-Core 10 dimensions (QLU-C10D). The QAPFS per treatment group was estimated with the Kaplan-Meier method. P < 0.05 was used for statistical significance, and a between-group minimal important difference of 10 points was interpreted as clinically relevant for the QLQ-C30-sum.Results168 patients were randomized. HRQL questionnaire compliance was 93%. For the QLQ-C30-sum, the difference of 4.62 points (95% confidence interval (CI) -8.9: -0.4, p = 0.032) was not clinically relevant. PFS was 19.3 months for the Kd maintenance group and 16.8 months for the observation group; difference = 2.5 months (95% CI 0.5; 4.5). QAPFS based on the QLQ-C30-sum for the Kd maintenance group was 18.0 months (95% CI 16.4; 19.6) and for the observation group 15.0 months (95% CI 13.5; 16.5); difference = 3.0 months (95% CI 0.8–5.3). QAPFS based on the QLU-C10D for the Kd maintenance group was 17.5 months (95% CI 15.9; 19.2) and 14.0 months (95% CI 12.4; 15.5) for the observation group; difference = 3.5 months (95% CI 1.1–5.9).ConclusionsKd maintenance therapy after salvage ASCT did not adversely affect overall HRQL, but adjustment for HRQL reduced the PFS compared to unadjusted PFS. PFS of maintenance therapy should be quality-adjusted to balance the benefits and HRQL impact.

Abstract WP97: Disparities in Health-Related Quality of Life Among Adults With Heart Disease, Stroke, and Both Conditions

Background: Notable racial and ethnic disparities exist in cardiovascular disease (CVD) and stroke epidemiology; however, few studies have assessed the disparate quality of life (QOL) among individuals living with CVD, stroke, or both. This study evaluated the impact of these conditions on health related QOL (HRQOL) and quantified demographic disparities. Methods: Using data from the 2021 National Health Interview Survey (NHIS), we calculated the Health and Activity Limitation Index (HALex)—a generic HRQOL measure comprising perceived health and activity limitations—for 29,482 adults. Stratified analysis and ordinary least-squares regressions were used to adjust for sex, age, income, education, urbanicity, marital status, household size, region of residence, and insurance status. Differences in HRQOL were estimated across racial and ethnic groups with and without CVD, stroke, or both conditions. To test the robustness of study findings, analyses were repeated using the EQ-5D. Results: Adjusted HRQOL was lower for Blacks (-0.014, SE=0.004) and Hispanics (-0.012, SE=0.003) compared to Whites. Compared those with neither CVD nor stroke, individuals with CVD only (-0.21, SE=0.015), stroke only (-0.29, SE=0.028), and both conditions (-0.44, SE=0.040) had 20% to 44% lower HRQOL. Even after for age, income, education, and income, diagnosis with stroke only (-0.19, SE=0.081), CVD only (-0.12, SE=0.051), or both (-0.44, SE=0.040) lowered the HRQOL more for Blacks than Whites, while other racial/ethnic groups showed no statistically significant difference HRQOL. In general, geographic location, sex, and household composition had little impact on HRQOL, but higher age, lower educational attainment, and low/middle income was associated with significant reductions in HRQOL. Conclusion: CVD and stroke were independently associated with significantly low HRQOL, their co-occurrence has a substantially negative impact on HRQOL for all individuals particularly Blacks who had the largest HRQOL impact in all three condition groups. Findings suggest that public health interventions should be aimed at improving HRQOL among individuals diagnosed with these conditions, especially the most vulnerable groups.

Efficacy and Health-Related Quality of Life Impact of Fecal Microbiota, Live-jslm: A Post Hoc Analysis of PUNCH CD3 Patients at First Recurrence of Clostridioides difficile Infection.

Clostridioides difficile infection (CDI) causes symptoms of varying severity and negatively impacts patients' health-related quality of life (HRQL). Despite antibiotic treatment, recurrence of CDI (rCDI) is common and imposes clinical and economic burdens on patients. Fecal microbiota, live-jslm (REBYOTA [RBL]) is newly approved in the USA for prevention of rCDI following antibiotic treatments. We analyzed efficacy and HRQL impact of RBL vs. placebo in patients at first rCDI using data from the phase3 randomized, double-blind placebo-controlled clinical trial, PUNCH CD3. This post hoc analysis included patients at first rCDI fromPUNCH CD3. Treatment success (i.e., absence of diarrhea within 8weeks post-treatment) was analyzed adjusting for baseline patient characteristics. HRQL was measured using the Clostridioides difficile Quality of Life Survey (Cdiff32); absolute scores and change from baseline in total and domain (physical, mental, and social) scores were summarized and compared between arms. Analyses were conducted for the trial's blinded phase only. Among 86 eligible patients (32.8% of the overall trial population, RBL 53 [61.6%], placebo 33 [38.4%]), RBL-treated patients had significantly lower odds of recurrence (i.e., greater probability of treatment success) at week8 vs. placebo (odds ratio 0.35 [95% confidence interval 0.13, 0.98]). Probability of treatment success at week8 was 81% for RBL and 60% for placebo, representing 21% absolute and 35% relative increases for RBL (crude proportions 79.2% vs. 60.6%; relative risk 0.53, p = 0.06). Additionally, RBL was associated with significantly higher Cdiff32 total (change score difference 13.5 [standard deviation 5.7], p < 0.05) and mental domain (16.2 [6.0], p < 0.01) scores vs. placebo from baseline to week8. Compared to placebo, RBL demonstrated a significantly higher treatment success in preventing further rCDI and enhanced HRQL among patients at first recurrence, establishing RBL as an effective treatment to prevent further recurrences in these patients. ClinicalTrials.gov Identifier NCT03244644.

PCR179 The Socio-Economic and Health-Related Quality of Life Impact of Myasthenia Gravis (MG) in Greece

PCR179 The Socio-Economic and Health-Related Quality of Life Impact of Myasthenia Gravis (MG) in Greece

Title: The Impact of Recent Vaso-Occlusive Crisis on Health-Related Quality of Life in Adults with Sickle Cell Disease

Introduction: Sickle cell disease (SCD) is characterized by vaso-occlusive crises (VOCs) and a progressive clinical course, often leading to end organ damage and mortality. Individuals living with SCD with recurrent VOCs experience daily life disruptions due to recurrent pain crises, debilitating chronic pain and fatigue, which substantially impair health-related quality of life (HRQoL). The impact of a recent VOC on HRQoL has not been quantified. We investigated HRQoL impacts in adults with SCD who experienced a recent VOC (&amp;lt; 1 week) in comparison to overall adults with SCD with recurrent VOCs. Methods: A mixed-methods study was conducted to quantify the HRQoL, work impacts, and perceived health inequalities of individuals with SCD with recurrent VOCs. The study consisted of qualitative interviews and an online longitudinal survey administered at 3 timepoints (month 0, 3, and 6) in the US, UK, France, Germany, and Italy. Patient advocacy groups used multi-channel outreach to invite participants, who were required to meet the eligibility criteria and provide consent. Key eligibility criteria included recurrent VOCs defined as ≥ 2 VOCs/year in each of the 2 years prior to enrollment requiring a medical facility interaction and administration of pain medication or red blood cell transfusion. The survey included multiple patient-reported outcome (PROs) measures: the Functional Assessment of Cancer Therapy - Bone Marrow Transplant (FACT-BMT) inclusive of the FACT-General (FACT-G), EuroQol- 5 Dimension 5-Level (EQ-5D-5L), 11-point pain numerical rating scale (NRS), Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me), and the Work Productivity and Activity Impairment (WPAI) questionnaire. In this post-hoc analysis study participants with a current or recent VOC (&amp;lt; 1 week ago) were identified based on ASCQ-Me responses (herein referred to as recent VOC). The HRQoL impact of a recent VOC was assessed by descriptively comparing PRO scores between those with a recent VOC and the overall population of adults with SCD with recurrent VOCs (herein referred to as overall population). Results: The survey was completed by 142 participants with SCD (≥ 18 years) with 139 providing time since last VOC living in the US (n=82) and UK, Germany, France, and Italy (n=57). Fifty-six (40.3%) reported a recent VOC and had a similar mean age (35.7 vs. 35.3 years) and hydroxyurea use in the past month (58.9% vs. 61.9%) compared with the overall population. A higher proportion of those with a recent VOC were unemployed or unable to work due to SCD (76.8% vs. 64.7%). The mean EQ-5D VAS (0-100 scale, 100=best health) among those with a recent VOC was 49.2 (SD: 14.2), which was 16.2% and 38.8% lower relative to the overall study population (58.7 [SD: 19.4]) and the US general population (80.4 [SD: 15.6]), respectively. The overall mean EQ-5D health utility US index score (0.59 [SD: 0.27]) was much lower than the US general population (0.85 [SD: 0.21]) and further reduced among those with a recent VOC by 0.11 (0.48 [SD: 0.25]). The FACT-G total mean score was 53.6 (SD: 14.3) (0-108 scale, 108=best HRQoL) among those with a recent VOC representing a clinically meaningful reduction compared with the overall population (62.5 [SD:18.1]); with all FACT-G domain scores negatively impacted by a recent VOC. Based on the WPAI, those employed with a recent VOC (n=15) reported a mean of 35.4% (SD: 35.8%) absenteeism and 62.5% (SD: 21.8%) presenteeism, which were higher than the overall study population (24.7% [SD: 27.1%] and 42.0% [SD: 27.8%)], respectively). In the past 12 months, those with a recent VOC had a greater mean number of emergency room visits (9.6 [SD: 12.6] vs 6.2 [SD: 8.8]), hospitalizations with overnight stays (6.0 [SD: 5.7] vs. 4.0 [SD: 4.5]), and days overnight in the hospital (21.8 days [SD: 32.9] vs. 14.7 [SD: 24.7]) compared to the overall population. Conclusions: Despite current treatments, nearly 40% of adults with SCD in the surveyed population experienced a recent VOC (&amp;lt; 1 week), which has significant negative impacts on HRQoL and functional status, including the ability to work. These results demonstrate the substantial humanistic burden of those living with SCD with recurrent VOCs and the additional impairment of a recent VOC. Novel treatments that reduce or eliminate VOCs may provide an opportunity to improve health inequalities, HRQoL, and address unmet needs in this patient population.

Health-Related Quality of Life (HRQoL) in Patients with Genetic Standard and High-Risk Disease at Trial Entry: A Cross-Sectional Analysis of RADAR (UK-MRA Myeloma XV)

Introduction Clinical outcomes in newly diagnosed multiple myeloma (MM) are independently influenced by genetic risk as determined by FISH and molecular profiling. However, little is known about the impact of these characteristics on HRQoL of MM patients at diagnosis, and if this is independent of other diagnostic criteria such as International Staging System (ISS) and CRAB. Similarly, there is little quantitative information on HRQoL of MM patients in contrast to other cancer patients and healthy individuals. We examined patient-reported outcomes from participants enrolling in the RADAR study to understand the HRQoL of MM patients in different subgroups prior to treatment. Methods The RADAR study (Eudract: 2019-001258-25) is a national, multi-centre, risk-adapted, response-guided multi-arm, multi-stage phase II/III trial for newly diagnosed MM eligible for ASCT. RADAR treats patients with induction, consolidation and maintenance options using combinations of lenalidomide, cyclophosphamide, bortezomib, dexamethasone and isatuximab. Genetic risk was defined using centrally reviewed standard-of-care NHS testing and participants enter the standard or high-risk pathway based on the presence of at least two of the markers: t(4;14), t(14;16), t(14:20), del(17p), gain(1q) and del(1p). CRAB diagnostic criteria at baseline were defined as hypercalcaemia (&amp;gt;2.6 mmol/l), renal impairment as indicated by eGFR by MDRD (30-60, &amp;gt;60 mL/min/1.73m 2), anaemia (Hb&amp;lt;100g/L) and the number of lytic lesions at trial entry (0, &amp;gt;=1). HRQoL was measured by EORTC QLQ-C30 and MY20 completed at trial entry. We used one-way ANOVA and two-sample unequal variance t-tests to compare subscales in standard and high-risk groups and CRAB criteria groups. We sought minimally important differences that were at least medium by accepted definitions, and adjusted p-values using the Bonferonni correction for the 19 subscales (adjusted P = 0.05/19 = 0.0026). We undertook similar comparisons with normative scores (NORM) for cancer patients and UK healthy controls. Results Baseline HR-QoL was available for 301 participants enrolled in RADAR. Median age at trial entry was 61 (range, 34-74), 165 (57.5%) were male and 249 (88.3%) ECOG PS0-1. 235 (78.1%) patients were classified as standard risk and 52 (21.9%) as high-risk. 111 (38.7%) patients were ISS1, 116 (40.4%) were ISS1 and 50 (17.4%) were ISS3. 24 (8.6%) patients had hypercalcaemia, 48 (16.7%) patients had eGFR &amp;lt; 60 mL/min/1.73m 2, 67 patients (23.8%) had anaemia and 287 (65.9%) patients had at least one bone lesion. Comparing standard and high-risk genetics patients, there were no significant differences in any C30 or MY20 subscales (Figure A). Increasing ISS was significantly associated with worse Physical and Role Functioning, increased Fatigue and Dyspnoea. Renal impairment (eGFR&amp;lt; 60 mL/min/1.73m 2) was not associated with differences in any C30 or MY20 subscales. Anaemia was significantly associated with worse Global health status/QoL, Physical Functioning and Role Functioning, and Fatigue was significantly increased. The presence of bone lesions was significantly associated with worse Global health status/QoL, Physical Functioning, Role Functioning and Social Functioning, and Fatigue, Pain, Disease Symptoms, Side Effects and Body Image worries were significantly increased (Figure B). Comparing RADAR to the C30 Cancer NORM, 7 of 15 subscales were significantly worse. Small negative differences were observed for Physical and Role Functioning, Fatigue, Insomnia and Constipation and medium negative differences were observed for Social Functioning and Pain. Similarly, in contrast to UK C30 Healthy NORM, 8 of 15 subscales were significantly different. There were small detrimental differences observed for Physical Functioning, Role Functioning, Appetite Loss, Constipation and Financial Problems, medium differences for Fatigue and Pain and large differences for Social Functioning. Conclusion Prior to commencing treatment, patients with genetic high-risk MM did not have significantly different HRQoL to standard risk MM whereas ISS, an established measure of disease burden, did impact HRQoL. Similarly, CRAB criteria of anaemia and bone disease significantly impact several HRQoL domains. Compared to the general Cancer and UK healthy population HRQoL in patients entering RADAR was significantly worse in some subscales, notably Pain and Fatigue.

Older Adults with Acute Myeloid Leukemia (AML) Experience Improvement in Health-Related Quality of Life (HRQoL) Scores with Intensive Therapy: Prospective Study from ECOG-ACRIN (EA) E2906 Phase 3 Trial

Background There is limited prospective or comparative data evaluating the HRQoL impact of intensive chemotherapy (IC) in older adults (age ≥60 years) with AML. In the recent EA E2906 phase 3 study of '7&amp;3' (daunorubicin &amp; cytarabine) vs. clofarabine (CLO), a putative lower intensity therapy, we incorporated a prospective patient-reported outcomes (PRO) assessment of HRQoL and fatigue to understand treatment impact from the patients' (pts) perspective. Methods E2906 study design and results have been presented previously [n=727, med. age 68 years (range 60-86), randomized 1:1 to ‘standard’ ‘7&amp;3‘ &amp; high dose cytarabine (Arm A) vs. single agent CLO (Arm B), as remission induction (Step 1) and consolidation (Step 2), respectively]. There was no difference in composite complete remission (CCR, 50%) or 30-day mortality rates (8.5%), and in primary analysis CLO was inferior for Overall Survival. HRQoL was a key secondary protocol endpoint. PROs assessed HRQoL and leukemia-specific symptoms and concerns using Functional Assessment of Cancer Therapy-Leukemia scale (FACT-Leu), &amp; Fatigue using FACT-Fatigue subscale. PROs were administered at prespecified time points: (1) Baseline; (2) Cycle 1 at nadir (approx. Day 15); (3) End of Step 1 restaging (approx. D 40); (4) Beginning of Step 2 (1 st consolidation, approx. D 45); and (5) 2 nd cycle consolidation/End Step 2 (approx. D 60). The FACT-Leu Trial Outcome Index ( TOI) was calculated by summing physical well-being, functional well-being, and leukemia-specific concerns subscales to create a single composite score. Higher TOI scores correspond to better HRQoL. The primary endpoint was to evaluate TOI difference from randomization to day 30 after induction therapy between arms, evaluated using Wilcoxon rank sum test. We also compared Clinically Meaningful differences (CMiD) in TOI (defined as patient change ≥½ standard deviation from baseline TOI) between arms, using the X2 test. Pts not completing at least 2 assessment forms (including baseline) were excluded. Testing of constructs of Physical Well-being (PWB), Functional, Leukemia &amp; Fatigue scales demonstrated high Cronbach's a values (a &amp;gt;0.7) except for PWB (a=0.672) at a single time point (End of Step 2). Results Baseline TOI was available for n=489, and 364 pts (n=182 each Arm) were evaluable in this planned analysis. Evaluating CMiD during Cycle 1 induction, pts in Arm B were less likely to experience ‘Worse’ TOI score (32% vs. 40.7%, Arm A), more likely to maintain stable TOI scores (49.1% vs. 39%, Arm A), and had a similar proportion of ‘Better’ TOI scores (20.3%, A vs. 18.9%, B) although differences were not significant (p=0.1389). Conversely in cycle 1 consolidation, more patients in Arm A experienced Better TOI (45.1% vs. 29.2%, Arm B), fewer had stable TOI (45.1% vs. 58.3%, Arm B), and rates of ‘Worse’ TOI were similar (9.8%, A vs 12.5%, Arm B), again differences were not significant (p=0.2616). There was no difference in Fatigue scores between Arms (p=0.49). We evaluated whether TOI and Fatigue scores could predict the ability to complete protocol therapy. In univariate analysis baseline TOI (p=0.0059) and Fatigue (p=0.0057) were associated with Step 1 completion, however on multivariate analysis (adjusting for age, sex, and Arm) were no longer significant [TOI, Odds Ratio (OR) 1.03 (0.98-1.08, p=0.27); Fatigue, OR 1.03 (0.96-1.11, p=0.42)]. There was a borderline association of TOI with Step 2 completion [TOI OR 1.10 (1.00-1.23, p=0.06)]. Using a multivariable linear mixed effects model, we observed similar and significant changes in TOI scores in both Arms, demonstrating the effect of IC &amp; CLO on HRQoL (Figure). Comparing to baseline, TOI scores decreased significantly at C1 induction nadir (-4.0873, p&amp;lt;0.0001), and increased by 5.0762 (p=0.0004) at end Step 1, by 6.9047 at cycle 1 consolidation (p&amp;lt;0.0001), and by 11.4601 (p&amp;lt;0.0001) at end Step 2. Differences were mainly apparent in those who achieved CCR, and there was significant attrition in PRO responses in non-CCR pts. Conclusions Older adults experienced significant improvement in HRQoL after nadir regardless of therapy, and those achieving CCR report significantly higher scores. We did not observe differences in leukemia specific HRQoL or fatigue between treatment Arms (7&amp;3 vs. CLO). Patient-reported HRQoL &amp; Fatigue was not predictive of completion of protocol therapy. These results serve as a benchmark for expectations with IC in older adults.

Association of Health-Related Quality of Life and Work Productivity with Clinical Characteristics in Adults with Sickle Cell Disease

Introduction: Adults with sickle cell disease (SCD) suffer from periodic episodes of severe acute pain, chronic pain, fatigue, acute complications, end-organ damage and early mortality. We explored clinical characteristics associated with health-related quality of life (HRQoL) and work productivity and activity impairment (WPAI) in adults with SCD. Methods: Between January 2022 and June 2023, we recruited 87 adults aged ≥18 years with a diagnosis of SCD from four U.S. NASCC recognized SCD centers participating in the Globin Research Network for Data and Discovery ( GRNDaD) registry. We collected data on patient socio-demographics, fatigue, chronic pain, WPAI, and HRQoL measured by EQ-5D-5L and ASCQ-Me via patient survey. Medical history checklist (MHC) score sums 9 SCD complications and treatment history. Clinical charateristis on SCD subtype and baseline hemoglobin were obtained from the medical record. Clinical characteristics and self-reported MHC, chronic pain associated with HRQoL, and WPAI scores were assessed by Student T-tests or Pearson correlation. Results: Mean age was 38.0±13.4 (standard deviation) years, 58.6% of the sample was female, 64.3% had hemoglobin (Hb) SS disease, 73.6% reported chronic pain (experiencing pain on ≥3 days per week in the past 6 months). Mean EQ visual analogue scale (VAS) was 71.3 (lower than the U.S. 35-44 age mean of 81.8). Mean EQ index score was 0.73 (lower than the U.S. 35-44 age group population norm 0.85). Mean fatigue score was 52.1 (range 33.7-75.8) and was negatively correlated with HRQoL scores, indicating higher fatigue correlated with lower patient's self-rated health, including the EQ VAS (correlation coefficient r=-0.49, p&amp;lt;0.0001), EQ index score (r=-0.53, p&amp;lt;0.0001), and all domain of ASCQ-Me scores including pain (r=-0.52, p&amp;lt;0.0001), stiffness (r=-0.55, p&amp;lt;0.0001), sleep (r=-0.43, p&amp;lt;0.0001), emotion (r=-0.65, p&amp;lt;0.0001), and social functioning score (r=-0.64, p&amp;lt;0.0001). Fatigue score was correlated with pain episode frequency (r=0.29, p=&amp;lt;0.001, indicated more frequent pain episodes correlated with higher fatigue), and pain episode severity score (r=0.34, p=0.002). Fatigue score also correlated with overall activity impairment due to SCD (r=0.47, p&amp;lt;0.0001). MHC score was negatively correlated with EQ VAS (r=-0.32, p=0.003), EQ index score (r=-0.35, p=0.001), ASCQ-Me pain (r=-0.25, p=0.02), stiffness (r=-0.28, p=0.009), emotion (-0.23, p=0.03), social (r=-0.28, p=0.01), pain episode severity score (r=0.26, p=0.02) and fatigue score (r=0.35, p=0.0009). Hemoglobin was not correlated with HRQoL or fatigue scores. MHC score significantly correlated with overall activity impairment (r=0.22, p=0.04). As compared to patients who had MHC≤2, those with score&amp;gt;2 had significantly lower mean EQ-VAS, EQ index score, ASCQ-Me domain impacts in pain, stiffness, sleep, emotion, social funtioning, and pain episode severity, but higher fatigue score and greater absenteeism. Patients who reported chronic pain had significantly lower mean EQ VAS, EQ index score, ASCQ-Me domain impacts in pain, stiffness, sleep, emotion, social functioning, higher pain episode frequency, pain episode severity, and fatigue score, greater presenteeism score (impairment while working), overall work productivity loss and overall activity impairment than those without chronic pain. SCD subtype was not associated with HRQoL and fatigue scores. Conclusions: Hemoglobin and SCD subtype were not significantly associated with HRQoL and fatigue in this sample (possibly due to selection bias, where higher functioning patients were more likely to be selected for study participation). Fatigue in adults with SCD is moderately correlated with EQ-5D-5L scores, ASCQ-Me domains on pain, stiffness, sleep, emotion, and social functioning, as well as overall activity impairment, but weakly correlated with pain episode frequency and severity. MHC had weak correlation with EQ-5D-5L scores, ASCQ-Me domains on pain, stiffness, emotion, and social, and activity impairment. Individuals who had higher fatigue score, MHC score≥2, and reported chronic pain showed lower HRQoL and higher impairment of work productivity and activity. Future analyses will explore how multiplicity problems that SCD patients suffer impact HRQoL and work productivity and activity impairment in longitudinal data, which will identify the opportunity on intervention to improve patient care.

Association of Reaction Symptoms and Eliciting Dose With Health-Related Quality of Life in Children With Peanut Allergy.

Food allergy adversely affects the health-related quality of life (HRQoL) of patients. It is unclear whether factors such as the reaction eliciting dose (ED) and the nature of allergic reaction symptoms affect HRQoL. To explore associations between reaction ED or the nature of allergic symptoms and HRQoL among children with peanut allergy. This study was a secondary analysis of baseline data from the PPOIT-003 randomized trial in 212 children aged 1 to 10 years with challenge-confirmed peanut allergy. Children's past reaction symptoms were collected by clinicians during screening. Associations between variables of interest and parent-reported child-proxy HRQoL were examined by univariable and multivariable linear regression. Mean age of study participants was 5.9 years; 63.2% were male. Children with a low reaction ED of 80 mg peanut protein had significantly poorer HRQoL (β= -0.81; 95% CI, -1.61 to -0.00; P= .049) compared with children with a high ED of 2,500 mg peanut protein. Gastrointestinal symptoms (β= 0.45; 95% CI, 0.03-0.87; P= .037), lower airway symptoms (β= 0.46; 95% CI, 0.05-0.87; P= .030), multisystem involvement (β= 0.71; 95% CI, 0.25-1.16; P=.003), or anaphylaxis (β= 0.46; 95% CI, 0.04-0.87; P= .031) during a previous reaction were associated with worse HRQoL. Peanut-allergic children with a lower allergen reaction threshold experienced a greater negative HRQoL impact compared with children with higher reaction thresholds. In addition, specific past allergic reaction symptoms were associated with comparatively worse HRQoL. Children experiencing these symptoms and those with lower reaction ED require increased clinical support to manage the food allergy and are likely to benefit from interventions that can improve HRQoL.

456. PHYSICAL ACTIVITY AND FRAILTY AS PART OF HEALTH RELATED QUALITY OF LIFE ASSESSMENT IN ESOPHAGEAL CANCER SURVIVORS AFTER CURATIVE ESOPHAGECTOMY

Abstract Background Survival rates for patients with esophageal cancer are improving, resulting in an increasing population of esophageal cancer survivors. Surgery is the mainstay of treatment but has a high risk of post-operative morbidity. This study aimed to characterise health related quality of life with a specific focus on physical activity and frailty assessment within an esophageal cancer survivor population. Methods This was a cross-sectional cohort study of patients who underwent esophageal cancer surgery between 2010 and 2015. A multicomponent questionnaire was distributed to disease-free patients at least one year post completion of treatment. Linear and logistic regression analyses were utilised to determine the impact of treatment and pathologic variables on physical activity and frailty. Results On multivariable analysis, age was a significant predictor of level of physical activity (P = 0.001). Male sex, critical care length of stay and eating symptoms were independent predictors of frailty in both the PRISMA7 and SARCF tools (all P &amp;lt; 0.001). Inpatient length of stay (P &amp;lt; 0.001) and persistent eating symptoms following treatment (P &amp;lt; 0.001) were both predictive of physical function following completion of treatment. Conclusion Curative esophagectomy can have significant effects on patients physical function, levels of physical activity and risk of frailty. Current methods of assessing health related quality of life in esophageal cancer survivorship may under-investigate the effects of curative esophagectomy on physical activity and frailty. Further research is warranted to investigate how patient pathways can be enhanced to minimise the long-term health-related quality of life impact of curative treatment.

Impact of Health Related QoL and Mediterranean Diet on Liver Fibrosis in Patients with NAFLD.

Patients with non-alcoholic fatty liver disease (NAFLD) display impaired health-related quality of life (HRQoL) that is often linked to an unhealthy dietary pattern. The aim of this work was to investigate the impact of HRQoL and adherence to the Mediterranean diet on the risk of liver fibrosis (LF) in patients with NAFLD. LF was assessed in 244 patients through transient elastography (FibroScan®530. Echosens, Paris, France). Significant LF was defined according to liver stiffness measurements (LSM) values ≥ 7.1 kPa. The Mediterranean diet score and the Short Form-36 questionnaires were also completed. The median age was 54 (44-62) years and 57% of participants were male. A total of 42 (17.2%) participants had LSM ≥ 7.1 kPa and showed increased GGT (p = 0.001), glucose (p < 0.001), and triglycerides levels (p = 0.015) compared to those with LSM ≤7.0 kPa. Moreover, patients with significant LF had significantly lower scores related to Physical Functioning (p < 0.001) and Role Physical (p < 0.001). In the logistic regression analysis, lower role physical and lower adherence to the MedDiet (p = 0.001 and p = 0.009, respectively), after adjusting for age, diabetes, and obstructive sleep apnea, were associated with an increased risk of significant LF. Low adherence to MedDiet and low role physical may influence the risk of significant liver fibrosis in patients with NAFLD.

Menopausal symptoms by HIV status and association with health-related quality of life among women in Zimbabwe: a cross-sectional study

BackgroundThe scale-up of antiretroviral therapy programmes has resulted in increased life expectancy of people with HIV in Africa. Little is known of the menopausal experiences of African women, including those living with HIV. We aimed to determine the prevalence and severity of self-reported menopause symptoms in women at different stages of menopause transition, by HIV status, and evaluate how symptoms are related to health-related quality of life (HRQoL). We further sought to understand factors associated with menopause symptoms.MethodsA cross-sectional study recruited women resident in Harare, Zimbabwe, sampled by age group (40–44/45–49/50–54/55–60 years) and HIV status. Women recruited from public-sector HIV clinics identified two similarly aged female friends (irrespective of HIV status) with phone access. Socio-demographic and medical details were recorded and women staged as pre-, peri- or post-menopause. The Menopausal Rating Scale II (MRS), which classified symptom severity, was compared between those with and without HIV. Linear and logistic regression determined factors associated with menopause symptoms, and associations between symptoms and HRQoL.ResultsThe 378 women recruited (193[51.1%] with HIV), had a mean (SD) age of 49.3 (5.7) years; 173 (45.8%), 51 (13.5%) and 154 (40.7%) were pre-, peri and post-menopausal respectively. Women with HIV reported more moderate (24.9% vs. 18.1%) and severe (9.7% vs. 2.6%) menopause symptoms than women without HIV. Peri-menopausal women with HIV reported higher MRS scores than those pre- and post-menopausal, whereas in HIV negative women menopausal stage was not associated with MRS score (interaction p-value = 0.014). With increasing severity of menopause symptoms, lower mean HRQoL scores were observed. HIV (OR 2.02[95% CI 1.28, 3.21]), mood disorders (8.80[2.77, 28.0]), ≥ 2 falls/year (4.29[1.18, 15.6]), early menarche (2.33[1.22, 4.48]), alcohol consumption (2.16[1.01, 4.62]), food insecurity (1.93[1.14, 3.26]) and unemployment (1.56[0.99, 2.46]), were all associated with moderate/severe menopause symptoms. No woman reported use of menopausal hormone therapy.ConclusionsMenopausal symptoms are common and negatively impact HRQoL. HIV infection is associated with more severe menopause symptoms, as are several modifiable factors, including unemployment, alcohol consumption, and food insecurity. Findings highlight an unmet health need in ageing women in Zimbabwean, especially among those living with HIV.