Quality of Life and Burden of Disease in Italian Patients with Transfusion-Dependent Beta-Thalassemia

Lifespan treatment in transfusion-dependent β-thalassemia (TDT) is expected to impact quality of life. This study aimed at evaluating health-related quality of life (HRQoL), well-being, and the burden of TDT on Italian patients. Patients (≥14 years) were invited to complete a cross-sectional, online volunteer survey. HRQoL was measured by the 36-item short-form health survey (SF-36) and wellbeing was measured by the Italian version of the Psychological General-Well-Being-Index (PGWBI). A total of 105/167 completed questionnaires were analyzed (46% males; median age 44, (IQR = 11)). Patients reported lower HRQoL compared with the general population in all SF-36 domains (except for emotional well-being (p = 0.7024) and role limitations due to emotional problems (p = 0.1389)). PGWBI domains general health and vitality and the total PGWBI score were all significantly lower (p = 0.0001) compared with the general population. On average, patients spent 16.62 h/month engaged in care activities that were additional to the time required for completing transfusions. Of the 16.62 h/month, 11.7 h/month were required for therapy management and 4.92 h/month for family management. This study found lower HRQoL and well-being in physical and psychological domains compared with the general population. Further, patients were found to have a high perceived burden of disease.

Health-related quality of life of individuals sharing a household with persons with dementia

Introduction Previous research has found a negative effect of dementia on the health-related quality of life (HrQoL) of persons with dementia (PWD) and their primary informal caregivers. However, the impact of dementia on HrQoL of other individuals sharing a household with PWD has not been investigated to date. The current study therefore aimed to determine differences in the HrQoL between those sharing a household with PWD and those not living with PWD. In addition, factors related to the HrQoL of those sharing a household with PWD were evaluated. Methods The analyses were based on data from the German Socio-Economic Panel, using the SF-12 to measure HrQoL. Mixed-effects models were calculated to compare the HrQoL of those sharing a household with PWD and persons not living with PWD, as well as to determine factors related to the HrQoL of those sharing a household with PWD. Bootstrapping was used where residuals were not normally distributed. Results Mixed-effect models showed a significantly lower HrQoL among those sharing a household with PWD, compared to those not living with PWD. Number of diseases, number of persons in the household, marital status and educational level were significantly related to HrQoL among those sharing a household with PWD. Discussion The HrQoL of those sharing a household with PWD was reduced compared to persons not living with PWD. Further, those living with PWD in small households, or those with multi-morbidities had a lower HrQoL. Further research focusing on HrQoL in the social environment of PWD is needed.

Healthcare Utilization and the Quality of Life of Children and Adolescents with Sickle Cell Disease

Introduction: Youth diagnosed with sickle cell disease (SCD) often utilize the healthcare setting to manage disease-related symptoms, yet the connection between healthcare use and their overall wellbeing has been understudied. This study investigates whether healthcare utilization predicts the health-related quality of life (HRQOL) in youth with SCD. It is hypothesized that increased healthcare utilization will predict lower HRQOL in pediatric SCD. Methods: A total of 150 patients, ages 8-17 years old, were enrolled in this cross-sectional quantitative study. Patients completed the Pediatric Quality of Life (PedsQL) 3.0 SCD module, while the researcher conducted a retrospective chart review to gather patient characteristics such as emergency room (ER) and hospitalization occurrences over the past 12 months. Results: A higher frequency of ER visits (p < .05) and hospitalizations (p < .01) predicted lower HRQOL scores. Age (p < .05) also emerged as a significant predictor for both regression models. Discussion: As youth with SCD require ER treatment and/or hospital admission, they are at increased risk for lower HRQOL, specifically as they get older.

Burden of disease in myasthenia gravis: taking the patient’s perspective

Background Myasthenia gravis (MG) leads to exertion-dependent muscle weakness, but also psychological and social well-being are limited. We aim to describe the burden of disease in MG including sociodemographic, economical, psychosocial as well as clinical aspects, to compare health-related quality of life (HRQoL) of patients with MG to the general population (genP) and to explore risk factors for a lower HRQoL. Methods This case–control study was conducted with MG patients of the German Myasthenia Association. A questionnaire-based survey included sociodemographic and clinical data as well as standardized questionnaires, e.g. the Short Form Health (SF-36). HRQoL was compared to genP in a matched-pairs analysis. Participants of the German Health Interview and Examination Survey for Adults (DEGS1) served as control group. Results In our study, 1660 MG patients participated and were compared to 2556 controls from the genP. Patients with MG showed lower levels of physical functioning (SF-36 mean 56.0, SD 30.3) compared to the genP (mean 81.8, SD 22.1, adjusted difference: 25, 95% CI 22–29) and lower mental health sub-score (SF-36 mean 67.3, SD 19.8, vs. 74.1, SD 16.7, adjusted difference: 5, 95% CI 2–8). Female gender, higher age, low income, partnership status, lower activities of daily life, symptoms of depression, anxiety and fatigue and self-perceived low social support were associated with a lower HRQoL in MG patients. Discussion HRQoL is lower in patients with MG compared to genP. The burden of MG on patients includes economic and social aspects as well as their emotional well-being. New therapies must achieve improvements for patients in these areas. Trial registration information Clinicaltrials.gov, NCT03979521, submitted: June 7, 2019, first patient enrolled: May 1, 2019, https://clinicaltrials.gov/ct2/show/NCT03979521

Fatigue after acquired brain injury impacts health-related quality of life: an exploratory cohort study

This study aimed to identify the consequences of fatigue, fatigability, cognitive and executive functioning, and emotional state on health-related quality of life (HRQoL) in a clinical group of outpatients after acquired brain injury (ABI). This cross-sectional retrospective study included assessing outpatients at a rehabilitation clinic with WAIS-III working memory and coding subtests, and self-rating scales (Fatigue Impact Scale, Dysexecutive Questionnaire, Hospital Anxiety and Depression Scale, and the dimension of health-related quality of life from EQ-5D-3L). The predictive variables were investigated using a binary logistic regression with HRQoL as the dependent variable. Descriptive statistics and correlations were analyzed. Participants reported a lower than average HRQoL (95%), fatigue (90%), and executive dysfunction (75%). Fatigue had a significant impact and explained 20–33% of the variance in HRQoL with a moderate significance on depression (p = 0.579) and executive dysfunction (p = 0.555). Cognitive and executive function and emotional state showed no association with HRQoL. A lower HRQoL, as well as fatigue and cognitive and executive dysfunctions, are common after ABI, with fatigue is a partial explanation of a lower HRQoL.

Frequency of Vaso-Occlusive Crises Is Associated with Health-Related Quality of Life in Pediatric Patients with Sickle Cell Disease: US Cross-Sectional Surveys of Adolescents and Caregivers

Introduction: Pediatric patients with sickle cell disease (SCD) experience considerable disease-related burden. SCD-related morbidity is characterized by vaso-occlusive crises (VOCs), which are acutely painful events that may cause chronic and potentially life-threatening complications, contributing to the early mortality associated with SCD. Two cross-sectional observational surveys were fielded to evaluate the relationship between VOC frequency and health-related quality of life (HRQoL) in pediatric patients with SCD in the United States (US). Methods: Surveys were administered in 2 populations: adolescents with SCD ages 12-17 years (n=247) and caregivers (n=167) of children with SCD ages 2 months-11 years in the US. Adolescent participants first provided assent to participate, and their guardians provided permission. Adolescents then completed an online survey that included the Child Health Questionnaire-Child Form 45 (CHQ-CF45) and the Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) pain and sleep impact domains. After providing informed consent, caregivers completed an online survey assessing the HRQoL of their child with SCD; survey content was tailored to the age of their child. Caregivers of children ages 5-11 completed the CHQ-Parent Form 50 (CHQ-PF50) and the Patient-Reported Outcomes Measurement Information System (PROMIS) pain interference and sleep disturbance short forms (8a and 4a, respectively). Caregivers of children &lt;5 completed the Infant/Toddler Quality of Life Questionnaire-Short Form 47 (ITQOL-SF47). Children were stratified according to the number of VOCs they experienced in the past 12 months (0-2 VOCs; ≥3 VOCs), and a series of general linear models were conducted to examine the relationship between VOC frequency and HRQoL domain scores, with children's age included as covariate. Statistical significance was evaluated using a significance level of 0.05; p-values were also compared to a Benjamini-Hochberg adjusted critical value. Results: In adolescents ages 12-17, higher frequencies of VOCs were associated with lower HRQoL: adolescents with more frequent VOCs reported greater impacts than adolescents with less frequent VOCs across all domains of HRQoL measured by the CHQ-CF45, including general health, physical functioning, role/social limitations, mental health, and family activities (p&lt;0.05 for all) (Table 1). Pain and sleep (ASCQ-Me domains) were also associated with VOC frequency: greater impacts were observed among adolescents with more frequent VOCs (p&lt;0.001 for both). Similar to adolescents, children &lt; 12 who experienced more frequent VOCs exhibited lower HRQoL as reported by caregivers on most CHQ-PF50 and ITQOL-SF47 domains except those related to behavior and the ITQOL-SF47 change in health domain (Table 2). Children with more frequent VOCs also experienced greater pain interference and sleep disturbance than those with less frequent VOCs (p&lt;0.01 for both PROMIS domains; Table 2). Among caregivers of children ages 5-11, those whose children experienced more frequent VOCs reported greater impacts on CHQ-PF50 parental time/emotions and family activity domains than those whose children experienced less frequent VOCs (p&lt;0.01 for all; Table 2). Caregivers of children ages 2 months-4 years also reported a similar association between VOC frequency and the ITQOL-SF47 parental time domain (p&lt;0.01; Table 2). ITQOL-SF47 parental emotional impact scores also differed when compared to a significance level of 0.05, but were not statistically different after adjusting for multiple comparisons. Conclusions: Via both self- and caregiver-reports, these surveys demonstrated significant detrimental impact of VOCs on pediatric patients with SCD. Decrements were observed across various domains of HRQoL, including general health, physical functioning, pain, sleep, role/social limitations, and mental health. VOC frequency negatively impacted family activities and caregiver time and emotions, confirming anecdotal evidence that the effects of VOCs extend beyond the child to the caregiver. Given the cross-sectional study design, these analyses did not establish a temporal or causal relationship between VOCs and HRQoL; however, it is plausible that treatments aimed at reducing the frequency of VOCs may alleviate some of the burden experienced by pediatric patients with SCD and their families. Figure 1 Figure 1. Disclosures Campbell: Novartis Pharmaceuticals Corporation: Consultancy, Research Funding. Rizio: Novartis Pharmaceuticals Corporation: Other: Avery Rizio is employed by QualityMetric Incorporated, LLC, which received funding from Novartis to conduct this research. McCausland: Novartis Pharmaceuticals Corporation: Other: Kristen McCausland is an employee of QualityMetric Incorporated, LLC, which received funding from Novartis to conduct this research. Iorga: Novartis Pharmaceuticals Corporation: Current Employment, Current equity holder in publicly-traded company. Yen: Novartis Pharmaceuticals: Current Employment; Merck & Co., Inc.: Current holder of stock options in a privately-held company, Ended employment in the past 24 months. Paulose: Novartis Pharmaceuticals Corporation: Current Employment. Lee: Novartis Pharmaceuticals Corporation: Current Employment.

The impact of demographic and clinical characteristics on the trajectories of health-related quality of life among patients with Fabry disease

Background Fabry disease (FD) is an X-linked lysosomal storage disorder characterized by multiorgan dysfunction. Since individuals with FD usually experience progressive clinical disease manifestations, their health-related quality of life (HRQOL) is expected to change over time. However, there is limited longitudinal research examining HRQOL outcomes in individuals with FD. We aimed to: assess longitudinal outcomes in HRQOL in adults with FD; examine the physical- and mental HRQOL trajectories at the initial registration (baseline), 3–5 year, and 7–13 year follow-ups; and evaluate the possible associations of age, sex and medical complications with the physical- and mental HRQOL trajectories. Methods Forty-three individuals with FD (53% female) who were aged 18 to 81 years at baseline attended clinical follow-up visits between 2006 and 2020. Medical records were extracted retrospectively. Demographics and the 36-item Short-Form Health Survey (SF-36) were recorded at scheduled visits, except for the last data collection which was prospectively obtained in 2020. The physical (PCS) and mental (MCS) composite scores (SF-36) were chosen as outcome measures. Results The eight SF-36 domain scores were stable over a span of 13 years, and only physical- and social functioning domains worsened clinically over this follow-up period. Mean baseline SF-36 domain scores were all significantly lower (decreased HRQOL) in the FD sample compared with Norwegian population norms. Two hierarchical linear models were run to examine whether demographics and medical complications (measured at the last clinical visit) predicted physical and mental HRQOL trajectories. Age above 47 years (p < 0.001), male sex (p = 0.027), small fibre neuropathy (p < 0.001), renal dysfunction (p < 0.001), and cerebrovascular events (p = 0.003) were associated with lower HRQOL over time. No significant interactions were found between the time of follow up and the abovementioned predictors of HRQOL. Conclusions Overall HRQOL trajectories remained stable between baseline, 3–5 year, and 7–13 year follow-ups, with the majority of individuals reporting decreased physical and mental HRQOL. Medical complications in combination with older age and male sex are important predictors of lower HRQOL in FD. Awareness of this relationship is valuable both for health care providers and for patients. The findings provide indicators that can guide treatment decisions to improve physical and mental HRQOL outcomes.

Association between healthy lifestyle score changes and quality of life and health-related quality of life: a longitudinal analysis of South Korean panel data

ObjectivesWe aimed to determine the influence of changes in the aggregate score of healthy lifestyle factors on health-related quality of life (HRQOL) and overall quality of life (QOL) in the Korean older adult population.DesignThis study used a longitudinal design.Setting and participantsData on 9474 participants aged 45 years or older were extracted from the Korean Longitudinal Study on Aging for the period 2006–2016. A composite score of four lifestyle factors (smoking, drinking, physical activity and body mass index) was calculated, and biennial changes in aggregate score were computed.Primary and secondary outcome measuresThe primary outcomes were HRQOL and QOL.ResultsGeneralised estimating equation analysis results showed that those with healthy lifestyle score changes from ‘Low–High’ (β =−0.987, p=0.002; β =−1.288, p<0.0001), ‘High–Low’ (β =−1.281, p<0.0001; β =−1.952, p<0.0001) and ‘Low–Low’ (β =−1.552, p<0.0001; β =−2.398, p<0.0001) groups were more likely to be have lower HRQOL and QOL estimates than those in ‘High–High’ group. Female gender, older age and depression had a more negative impact on HRQOL, while male gender and younger age had a more negative impact on QOL, especially in the Low–Low group. The relationship between changes in scores and HRQOL and QOL varied across different elements of healthy lifestyle scores. Changes in physical activity, drinking and smoking status were significantly associated with lower HRQOL and QOL.ConclusionThe findings suggest an association between a low healthy lifestyle score and poor quality of life, in both general and health-related aspects. Strategies targeting the Korean ageing demographic to promote a healthier lifestyle should be encouraged.

Exploring the relationship between functional limitations of the older adults and the health-related quality of life of their spouse in Shaanxi Province, China

Background With trends towards longer life expectancy, lifetime with disability has also been prolonged. It is increasingly recognized that not only the person with disability but also those around them are affected. The relationship between functional limitation (FL) of the older adults and health-related quality of life (HRQoL) of their spouse is of interest. So too is the determination of the factors aside from FL that influence HRQoL. Methods The sample was derived from the 2013 National Health Service Survey conducted in Shaanxi Province in China. Married couples aged ≥ 60 years were selected (n = 3463). The European quality of life five dimensions (EQ-5D) and visual analogue scale were used to measure HRQoL. Results Both wife and husband reported lower HRQoL if either the male or female partner had some or serious FLs (P < 0.001). Other factors associated with lower HRQoL of the spouse included age, lower educational level, presence of chronic disease, and lower household economic status. Family size was associated with wife's HRQoL only when the male had no FL and lived with another 1–2 persons, or when the male had some FLs and lived in a larger family (n ≥ 5). Residential status did not relate to the HRQoL of spouses regardless of FL status. Conclusions Older adults in Shaanxi province who have partners with FLs tend to report poorer EQ-5D, suggesting that couples amongst whom one has FL may be particularly vulnerable to lower HRQoL.

Health-Related Quality of Life in the Spironolactone to Reduce ICD Therapy (SPIRIT) Trial

To describe health related quality of life (HRQOL) and symptoms in the SPIRIT trial and determine effects of implantable cardioverter defibrillator (ICD) shocks on HRQOL over 24 months. Ninety participants aged 66 ± 10 years, 96% men, 75% with NYHA class II, with an ICD were randomized to spironolactone 25 mg ( N = 44) or placebo ( N = 46). HRQOL was measured every 6 months for 24 months using: Patient Concerns Assessment (PCA), Short Form Health Survey-Veterans Version (SF-36V), and Kansas City Cardiomyopathy Questionnaire (KCCQ). Linear mixed modeling compared changes in HRQOL over-time and ANCOVA compared HRQOL between those getting an ICD shock or not. Over 24-months, there were no differences in HRQOL between the spironolactone versus placebo groups. Those with at least one ICD shock reported significantly lower HRQOL and more symptoms at 6- and 24-months. Patients receiving one or more ICD shocks reported significant reductions in HRQOL and higher symptoms.