Impaired Health-related Quality Of Life Research Articles

Muscle Loss During First-Line Chemotherapy Impairs Survival in Advanced Pancreatic Cancer Despite Adapted Physical Activity.

Advanced pancreatic ductal adenocarcinoma (aPDAC) is often accompanied by significant muscle mass loss, contributing to poor prognosis. SarcAPACaP, an ancillary study of the GERCOR-APACaP phase III trial, evaluated the role of adapted physical activity (APA) in aPDAC Western patients receiving first-line chemotherapy. The study aimed to assess (1) the potential impact of computed tomography (CT)-quantified muscle mass before and during treatments on health-related quality of life (HRQoL) and overall survival (OS) and (2) the role of APA in mitigating muscle mass loss. In the APACaP trial, aPDAC patients with ECOG performance status (PS) 0-2 were randomized 1:1 to usual care including first-line chemotherapy or usual care plus a 16-week home-based APA program. In the SarcAPACaP study, the surface muscular index (SMI) was determined from L3 CT scan slices. Two patient populations were analysed: those with CT scan available at baseline (modified[m] intent-to-treat [ITT]1-W0) and those with CT scans available at both W0 and W16 (mITT2 W0-W16). Low muscle mass was defined by low SMI with SMI < 41 cm2/m2 for women and < 43 and < 53 cm2/m2 for men with body max index < 25.0 and ≥ 25.0 kg/m2, respectively. Muscle loss was defined by the relative difference of SMI between W0 and W16 (100*[SMI W16-SMI W0]/SMI W0). In mITT2 W0-W16, patients were stratified into three groups based on the severity of muscle loss: none, moderate (0%-10%) and high (≥ 10%). Associations between muscle mass loss and OS, time until definitive deterioration (TUDD) of HRQoL and the effect of APA on loss of muscle mass were assessed. Between October 2014 and May 2020, 313 patients were prospectively enrolled, with 225 in mITT1 W0 and 128 in mITT2 W0-W16, with 65 assigned to the APA arm. Both groups had similar baseline characteristics with comparable OS and TUDD. A low SMI at W0 was not associated with OS and TUDD of HRQoL in either group. Among mITT2 W0-W16 patients, high muscle mass loss (n = 27) independently predicted OS (p = 0.012) and showed a trend toward negatively affecting TUDD of HRQoL. Notably, APA did not mitigate muscle loss in our study population. Longitudinal muscle mass loss emerged as a predictive factor for both OS and HRQoL in aPDAC patients undergoing chemotherapy, while a low SMI at diagnosis did not provide prognostic value. APA did not impact muscle mass loss in this population.

The burden of Alagille syndrome: uncovering the potential of emerging therapeutics - a comprehensive systematic literature review.

Aim: Alagille syndrome (ALGS) is a rare, cholestatic multiorgan disease associated with bile duct paucity, leading to cholestasis. Clinical symptoms of cholestasis include debilitating pruritus, xanthomas, fat-soluble vitamin deficiencies, growth failure, renal disease and impaired health-related quality of life (HRQoL). The main objective was to review the current literature on the epidemiological, clinical, psychosocial and economic burden of ALGS in view of the development of ileal bile acid transporter (IBAT) inhibitors. Methods: Electronic literature databases were searched in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses checklist. Results: 330 publications were screened, 119 were relevant: 11 randomized controlled trials (RCTs), 21 non-RCTs, 10 HRQoL studies, two studies assessing cost/resource use and 77 epidemiological studies across several databases through 31 July 2024. Studies confirm that patients with ALGS experience cardiac anomalies, impaired growth, renal disease, poor HRQoL, fat-soluble vitamin deficiencies and debilitating pruritus; until the approval of IBAT inhibitors for the treatment of cholestatic pruritus in patients with ALGS, supportive management was the standard of care. Conclusion: This review confirms the substantial clinical, economic and HRQoL burden associated with ALGS and consolidates current treatment evidence. Data from recent trials in ALGS demonstrate the potential impact of IBAT inhibitors to transform lives by improving cholestatic pruritus symptoms, HRQoL and native liver survival.

Association Between Diagnostic Delay and Economic and Clinical Burden in Axial Spondyloarthritis: A Multicentre Retrospective Observational Study.

Axial spondyloarthritis (axSpA) is a chronic inflammatory condition associated with considerable pain and impaired health-related quality of life (HRQoL) for affected patients. Despite the documented increase in healthcare resource utilization (HRU) related to axSpA, few studies have explored the impact of diagnostic delays on these outcomes. This study sought to determine the association between diagnostic delay of axial spondyloarthritis (axSpA) and costs in the 3years after diagnosis. This is a retrospective, observational study based on routine follow-up data from adult patients with confirmed axSpA diagnosis in three tertiary Spanish hospitals. Sociodemographic and clinical variables were collected at diagnosis. Direct and indirect healthcare costs were estimated from healthcare resource use (HRU) and productivity losses. The correlation between diagnostic delay and total healthcare costs was analyzed. Eighty-two patients (62.2% men; mean age: 39.3 years at diagnosis) were included, mostly with radiographic axSpA (r-axSpA) (67.1%). The mean (standard deviation, SD) diagnostic delay was 10.1 (9.3) years, with a median (interquartile range, IQR) of 5.4 (2.3, 17.2) years. The mean total healthcare cost per patient accumulated over 3years was €25,812.6 (direct: €16,384.7; indirect: €9427.9). Patients with longer diagnostic delay (> 5.4years) had 57% higher total healthcare cost (€31,717.7 vs. €20,188.7, p = 0.029) and higher disease activity at diagnosis (BASDAI score 4.7 vs. 3.4, p = 0.007) and after 3years (3.9 vs. 2.9, p = 0.042) compared to those with shorter delay (≤ 5.4years). The diagnostic delay in axSpA remains high and is associated with an increase in healthcare costs post-diagnosis. Actions to reduce diagnostic delay should be prioritized by healthcare systems to potentially improve outcomes and reduce long-term costs.

Humanistic and Economic Burden of Atopic Dermatitis in Pediatric Patients in Spain: A Systematic Review

Atopic dermatitis (AD) is a chronic, inflammatory skin disease affecting all age groups, particularly children. This systematic review provides an overview of the humanistic and economic disease burden in the pediatric population with AD in Spain. The evidence, collected from 11 observational studies published over the past 10 years, exhibits the most common characteristics of the patients, disease burden, patient-reported outcomes, use of resources, and treatment patterns. The burden of AD extends beyond physical symptoms, with associated comorbidities such as asthma and impaired health-related quality of life and mental health disorders, particularly in severe cases. Traditional therapies, primarily topical corticosteroids, face adherence and efficacy challenges. Despite promising innovative treatments and available biological therapies, their use is still limited in the pediatric population. The findings of the present review highlight the scarce scientific evidence on the economic burden of pediatric AD, as well as the most updated humanistic evidence on this disease. At the same time, the need for individualized care and innovative therapeutic interventions to address the multifaceted challenges of pediatric AD in Spain is evident.

The effects of long-term exposure to PM2.5 on health-related quality of life in adolescents: an empirical study from China

Health-related quality of life (HRQoL) is a health indicator that indicates the individual’s overall perception of physical, psychological, and social well-being. Air pollution potentially affects the individual’s HRQoL. However, the evidence about the effects of air pollution on HRQoL, particularly in adolescents, remains limited. This study explored the effects of air pollution exposure on adolescents’ HRQoL. Data were collected from a population-based cross-sectional survey conducted in Shandong Province of China. The Tobit regression model (Tobit-CF) combined with the control function (CF) based on instrumental variables was used as the main regression model, and causal mediation analysis was conducted to explore the mechanism with the time spent on outdoor activity (OA) as the mediator. The final analysis included a total of 266,138 adolescents with an average age of 13 years. This study found that long-term exposure to air pollution would impair the adolescent’s HRQoL, with the decrease in outdoor activities potentially serving as a mediator. Compared to males and older adolescents, females, and younger adolescents are more susceptible to experiencing HRQoL impairment induced by air pollution exposure. The empirical strategy used in this study also provides the reference and insight for future environmental health research to infer causality using cross-sectional data.

Qualitative Assessment of Health-Related Quality of Life Impacts Associated with Sickle Cell Disease in the United States and United Kingdom.

Individuals living with sickle cell disease (SCD) commonly report impaired health-related quality of life (HRQoL). However, impacts of SCD on HRQoL and the unmet needs of SCD treatment/management are under-researched. This study characterized the impact of SCD on HRQoL and identified the unmet needs of individuals with SCD. Adults with SCD (aged≥18years) and caregivers of adolescents (aged 12‒17) with SCD in the United States (US) and United Kingdom (UK) participated in one-on-one virtual semi-structured interviews and focus group discussions (hereafter referred to as 'interviews'). Interviews were transcribed and thematically analyzed. Nineteen individuals participated in the study (across five interviews and three focus group discussions), including 18 adults with SCD (United States, n=11; United Kingdom, n=7) and one caregiver of an adolescent with SCD (United States). Most participants were female (n=15). Participants reported negative impacts of SCD on their HRQoL, including the burden of structuring their lives around SCD, due to unpredictable symptoms. They reported negative impacts to psychological health (e.g., depression/low mood and anxiety) and physical health (e.g., chronic pain and fatigue) that affected their social and family life, work, and education, leading to feelings of isolation. Participants expressed concerns about the future, feelings of resentment, and the need for high resilience when facing the barriers/impacts associated with SCD. Many participants reported negative interactions with healthcare professionals, leading to trauma, anxiety, and routine care avoidance. Most participants reported perceived prejudice during routine SCD treatment/management, including being treated as drug-seekers. Individuals with SCD experience negative HRQoL impacts, including impacts to daily activities, social and family life, work and education, psychological health, and prejudice/stigma. Our findings highlight significant unmet needs of individuals living with SCD, including alternative treatment options to reduce vaso-occlusive crisis (VOC) frequency and treat fatigue.

More absolute moderate-to-vigorous physical activity is associated with better health-related quality of life in outpatients with an acquired brain injury.

Health-related quality of life (HRQoL) is a patient-perceived measure of physical, social, and emotional health. Acquired brain injury (ABI) occurs due to damage to the brain after birth. Individuals with an ABI typically present with reduced HRQoL and require additional support to maintain their HRQoL. Although structured exercise training has been shown to improve HRQoL in individuals with ABI, there is little research on habitual, real-world activity. Most activity research characterizes moderate-to-vigorous physical activity (MVPA) in absolute terms; however, relative physical activity levels have been promoted for research in clinical populations. We tested whether longer MVPA durations, measured in absolute/relative levels, are associated with higher HRQoL in outpatients with ABIs. In total, 26 adults (54 ± 13 years, 16 females) with ABI completed the Quality of Life After Brain Injury questionnaire, a 6-min walk test (a measure of aerobic fitness; 490 ± 105 m), and wore an activPAL device 24 h/day for 7 days. Participants had an average HRQoL score of 53.4 ± 15.0 (out of 100), with 20 of 26 showing impaired HRQoL (score <60). Absolute MVPA (74.6 ± 91.0 min/week, b = 0.09, p = 0.03) was associated with HRQoL, whereas total physical activity (565.7 ± 264.8 min/week, p = 0.47), light physical activity (LPA; 491.1 ± 224.3 min/week, p = 0.98), and step count (5,960 ± 3,037 steps/day, p = 0.24) were not. Neither relative LPA (521.4 ± 244.9) nor relative MVPA (33.5 ± 34.9 min/week) were associated with HRQoL (both p values > 0.14). Targeting more absolute MVPA, but not necessarily relative MVPA, may be an effective strategy for interventions aiming to improve HRQoL in individuals with ABI.

P48 Maintenance response of guselkumab in the Chinese population: an interim analysis of an observational study

Abstract Maintenance treatment with biologics is recommended for the treatment of moderate-to-severe psoriasis; however, treatment withdrawal occurs frequently in the real world. In a post-approval commitment (PAC) study (NCT04914429), guselkumab demonstrated efficacy in Chinese patients with moderate-to-severe plaque psoriasis. Here, we report interim analysis results from the observational post-PAC study evaluating health-related quality of life (HRQoL) and psoriasis relapse within 24 weeks following guselkumab withdrawal. This prospective, observational study enrolled patients who achieved a psoriasis area and severity index (PASI) 75 response at Week 44 in the PAC study. In the framework of a decentralized clinical trial design, patients utilized an electronic patient-reported outcome system to report on HRQoL, psoriasis status, and administration of systemic treatment for psoriasis. HRQoL was assessed by the dermatology life quality index (DLQI) at baseline, then every 12 weeks through Week 48. Psoriasis relapse was defined as the initiation of a new systemic treatment for psoriasis. Off-treatment duration (OTD) was defined as the time between the last guselkumab administration at Week 44 in the PAC study and the date of the first dose of any systemic treatment for psoriasis, loss to follow-up, death, or end of the study. A planned interim analysis was conducted when all patients had completed questionnaires 24 weeks after guselkumab withdrawal. The data cut-off (DCO) date was 3 June 2024. A total of 243 patients were enrolled. As of DCO, two patients withdrew from the study due to loss of follow-up. Most patients were male [n = 194 (79.8%)] and mean [standard deviation (SD)] age was 41.2 (12.3) years. At baseline, nearly all patients had PASI &amp;lt;3 [94.2% (229/243)] and Investigator’s Global Assessment 0/1 [91.4% (222/243)]. DLQI scores gradually increased after guselkumab withdrawal, from a mean (SD) of 4.8 (4.4) on the index date to 5.2 (4.6) at Week 12 and 7.2 (6.0) at Week 24 [mean (SD) change: 0.4 (4.5) at Week 12; 2.5 (6.2) at Week 24]. Overall, 5.8% (14/241) of patients had a psoriasis relapse. The mean (SD) OTD was 157.6 (1.1) days; the median OTD was not reached. Patients in this post-PAC study in China experienced gradual deterioration of HRQoL after guselkumab withdrawal. Only a small proportion of patients experienced a psoriasis relapse during 24 weeks of follow-up, suggesting treatment response was generally maintained after guselkumab withdrawal. Results from the planned interim analysis were not conclusive due to the limited duration of follow-up; long-term data analysis will be conducted upon study completion at Week 48.

High throughput multiplex immunoassays stratify patients according to symptom burden across the anti-Ro positive systemic autoimmune rheumatic disease spectrum

Anti-Ro/SS-A antibodies are prevalent across systemic autoimmune rheumatic diseases (SARDs) and may signify a distinctive phenotype. This study aimed to identify protein biomarkers associated with symptom burden and health-related quality of life (HR-QoL), and use protein-based stratification to identify clinically meaningful clusters and inflammatory pathways implicated. Anti-Ro positive SARD patients were enrolled in a 6-month pilot study. HR-QoL was determined using a patient-reported visual analogue scale, and symptom burden was assessed with the EULAR Sjögren’s Syndrome Patient Reported Index (ESSPRI). Proximity extension immunoassays measured normalized protein expression (NPX) across 92 inflammatory proteins. Linear regression identified proteins linked to patient outcomes. Unsupervised hierarchical clustering of baseline NPX identified patient clusters. Functional protein association networks were visualized using String V.12.0. Diagnostic groups showed no differences in HR-QoL or physician global assessment (PhGA). Poor HR-QoL and high symptom burden correlated with downregulated inflammatory proteins, while PhGA correlated with upregulated proteins. Two distinct clusters were identified; Cluster 1, ‘low expression cluster’ exhibited higher symptom burden and more impaired HR-QoL, while Cluster 2, ‘high expression cluster’ correlated with a higher physician global assessment (PhGA). Key hub proteins included TGF-β1, CXCL-8, and CCL-2. This study identified patient clusters across the Ro-positive SARD, linking symptom burden to specific proteomic profiles. Unraveling novel protein networks associated with symptom burden and poor HR-QoL may identify therapeutic targets, which address patient-reported outcome measures (PROMs) across several disease indications.

Global, regional, and national burden of gout in elderly 1990–2021: an analysis for the global burden of disease study 2021

BackgroundGout, an inflammatory arthritis, disproportionately affects the elderly due to hyperuricemia, leading to significant health-related quality of life impairments and escalating healthcare costs. However, a comprehensive global analysis focusing on the elderly population is needed to inform effective interventions.MethodsUtilizing data from the Global Burden of Disease Study 2021, this study assessed the prevalence, incidence, and Years Lived with Disability (YLDs) of gout among individuals aged ≥ 55 years in 204 countries from 1990 to 2021. We also evaluated the impact of high body mass index (BMI) and kidney dysfunction as key risk factors.ResultsThe study identified 37,230,366 cases of gout globally among the elderly, with an age-standardized prevalence rate of 2505.4 per 100,000 population. There was a notable increase in prevalence with an Estimated Annual Percentage Change (EAPC) of 1.08. Similarly, the age-standardized incidence and YLD rates increased, with EAPCs of 0.83 and 1.06, respectively. High-income regions, particularly Australasia and High-income North America, exhibited the highest rates, while Central Latin America and the Caribbean reported the lowest. Males had a higher burden of gout than females. High BMI and kidney dysfunction were significant contributors to YLDs, with their impact more pronounced in regions with higher Socio-Demographic Index (SDI).ConclusionThe study found a growing gout burden among the elderly, with substantial regional and gender disparities. It underscores the urgent need for targeted public health interventions, particularly in high SDI regions, to address modifiable risk factors like high BMI and kidney dysfunction and to curb the rising trend of gout prevalence and disability.

Hen’s egg white allergy in adults leading to strong impairment of quality of life

In adulthood, hen’s egg white allergy (EWA) is a rare condition and rising in prevalence. Typically, EWA begins in early childhood and resolves at school age. Persistence into adulthood or newly onset of the allergy has been reported, but scientific data is scarce. Symptoms reach from typical gastrointestinal problems to severe systemic reactions. EWA and the fear of allergic reactions lead to drastic restrictions in diet as in social life of the affected individuals. This study aims to assess health related quality of life (HRQoL) in adults with EWA using the validated questionnaire “Food Allergy Quality of Life Questionnaire - adult form (FAQoLQ-AF)” and Food Allergy Independent Measure (FAIM). Between July 2023 and October 2023, 16 adults with hen’s egg white allergy were identified and questioned using the FAQoLQ-AF to evaluate HRQoL. Patients’ characteristics were obtained including age at allergy onset and the most severe allergic symptom. The results were summarized using descriptive statistical analysis. HRQoL was impaired in 16/16 allergic individuals with an overall mean score of 4.64/7 (SD 1.3). Self-assessed emotional impact of the EWA was more problematic than food allergy related health. Food Allergy Independence Measure (FAIM) mean score was 4.64 (SD 1.0) with highest result in product avoidance. The most frequent occurring symptoms were oral allergy syndrome and stomach pain in 7 (44%) patients each. This study shows impaired HRQoL in a small cohort of adults with hen’s egg white allergy using the FAQoLQ and FAIM questionnaire with special emphasis on emotional impact. We identified an urgent need for correct food labelling and research into safe treatment options to improve HRQoL.

Long-term quality of life and surgical outcome of female congenital adrenal hyperplasia patients.

To investigate the long-term quality of life and surgical outcome of female patients with congenital adrenal hyperplasia (CAH). Questionnaires for health-related quality of life (HRQoL) outcome (WHO-5, SF-36), lower urinary tract symptoms (ICIQ-FLUTS) and sexual outcome (ICIQ-FLUTSsex) were administered to adult CAH patients. Paediatric CAH patients and their parents were invited to complete WHO-5, PedsQL-4.0-SF15-Generic Core Scales and ICIQ-CLUTS. Six (46%) adults and 7 (54%) children with 21-hydroxylase-deficiency (age ranged from 8 years to 53 years) were recruited. Eleven (85%) had undergone a feminising genitoplasty. In the adult group, no statistically significant difference was identified in the HRQoL and ICIQ-FLUTS scores. Mean overall score of ICIQ-FLUTSsex was lower but not significantly in adult patients compared to controls (7.3 ± 2.9 vs. 2.9 ± 3.4, P = 0.06). A statistically significant negative correlation (r = -0.98, P = 0.02) was found between age at first operation and role limitations due to emotional problems in SF-36. No statistically significant difference was identified in the self-reported HRQoL and ICIQ-CLUTS scores in the paediatric group compared to controls. However, parent's report showed a statistically significant difference in school functioning (73.8 ± 18.9 vs. 91.2 ± 18.7, P = 0.02) and emotional functioning (80.3 ± 15.9 vs. 94.7 ± 12.7, P = 0.007). Our data did not show an impaired HRQoL nor long-term lower urinary tract symptoms in CAH patients' self-reports. Healthcare team should pay more attention to adult patient's sexual function.

Incidence, disease burden and clinical presentation of patients newly diagnosed with inflammatory bowel disease in a population-based inception cohort.

Emerging data indicate a stabilizing incidence of inflammatory bowel diseases (IBD), including ulcerative colitis (UC), Crohn's disease (CD), and IBD unclassified (IBDU) in Western countries. We aimed to investigate the incidence of IBD, its initial clinical presentation, and patient-reported burden. Copenhagen IBD Inception Cohort is a prospective, population-based cohort of patients with newly diagnosed IBD according to the ECCO guidelines in the period between May 2021 and May 2023, within a catchment area covering 20% of the Danish population. Based on 554 patients (UC: 308, CD: 201, and IBDU: 18), the incidence rates per 100,000 person-years were: IBD: 23.4 (95% confidence interval, 21.5-25.4), UC: 14.0 (12.6-15.6), CD: 8.6 (7.4-9.8), and IBDU: 0.8 (0.5-1.3). The median diagnostic delay was significantly shorter for UC (2.5 months (interquartile range [IQR] 1-6)) than for CD (5 months (IQR 1.5-11), p<0.01). Moderate-to-severe disability was reported by 34% of CD patients and 22% of UC patients (p=0.01), severe fatigue by 30% and 26% (p=0.43), and severely impaired health-related quality of life (HRQoL) by 43% and 30% of patients, respectively (p=0.01). Hospitalization rates (UC: 20%, CD: 34%, p<0.01), and need for immunomodulators, biologics, or surgery within three months of diagnosis, were high in both UC (3%, 7%, and 37%, respectively) and CD (31%, 18%, and 10%, respectively). We found a high incidence of IBD in Copenhagen with a substantial disease burden characterized by early and high requirement for advanced therapies and high rates of fatigue, disability and impaired HRQoL at diagnosis.

The impact of neighborhood deprivation on mental health and quality of life in children and adolescents during the COVID-19 pandemic: Findings from the COPSY Hamburg study.

Socioeconomic inequalities have been associated with poorer mental health outcomes in children and adolescents during the COVID-19 pandemic. Despite numerous studies on individual risk factors, the impact of societal environment, such as neighborhood characteristics, on changes in mental health has rarely been investigated. This study investigates the effect of neighborhood deprivation on mental health problems and health-related quality of life (HRQoL) in children and adolescents during the COVID-19 pandemic in Hamburg, Germany. Data were derived from the prospective German COPSY Hamburg study. Children and adolescents aged between 11-20 years and their parents participated in the study, which took place in summer 2020 (T1) and summer 2022 (T2). Neighborhood deprivation was assessed by a neighborhood status index. Mental health problems and HRQoL were assessed using internationally validated and established instruments. The prevalence of mental health problems and impaired HRQoL was reported. Analysis of covariance was conducted to examine the effect of neighborhood deprivation of the districts in Hamburg on the (changes in) mental health problems and HRQoL while controlling for social individual-level indicators. The total sample included in the statistical analysis consisted of N = 2,645 families. Children and adolescents living in more deprived areas had higher levels of general mental health problems and depressive symptoms during the COVID-19 pandemic. However, differences in neighborhood deprivation did not relate to the HRQoL and the averaged changes in children and adolescents' mental health problems and HRQoL from summer 2020 to summer 2022. Neighborhood deprivation is associated with impaired mental health in youth during the COVID-19 pandemic. Children and adolescents' mental health and overall well-being should be addressed by health promotion measures to create a health-promoting living environment, including diverse neighborhoods. Future research should focus on uniform assessment methods and addressing additional neighborhood factors.

Cancer-specific utility: clinical validation of the EORTC QLU-C10D in patients with glioblastoma.

Many health economic evaluations rely on the validity of the utility measurement for health-related quality of life (HRQoL). While generic utility measures perform well in HRQoL assessments of many diseases and patient populations, appropriateness for cancer-specific disease burdens needs attention and condition-specific measures could be a viable option. This study assessed the clinical validity of the cancer-specific EORTC QLU-C10D, a utility scoring algorithm for the EORTC QLQ-C30, in patients with glioblastoma. We expect the EORTC QLU-C10D to be sensitive and responsive in glioblastoma patients. Furthermore, we compared its statistical efficiency with the generic utility measure EQ-5D-3L. We used data from a multi-center randomized controlled trial (NCT00689221) with patients from 146 study sites in 25 countries. Both, the QLQ-C30 and the EQ-5D-3L, had been administered at seven assessment points together. Utilities of both measures were calculated for four country value set (Australia, Canada, UK, USA). Ceiling effects, agreement (Bland-Altman plots (BA), intra-class correlation (ICC)), were calculated to analyze construct validity. Sensitivity to known-groups (performance status; global health) and responsiveness to changes (progressive vs. non-progressive; stable vs. improved or deteriorated HRQoL) were investigated for clinical validity. Relative Efficiency (RE) was calculated to compare statistical efficiency of both utility measures. 435 patients were included at baseline and six subsequent time points (median timeframe 497days). QLU-C10D country value set showed negligible ceiling effects (< 6.7%) and high agreement with EQ-5D-3L (ICC > 0.750). BA indicated that differences between both utility measures increased with deteriorating health states. While the QLU-C10D was more sensitive to global health groups (RE > 1.2), the EQ-5D-3L was more sensitive to performance status groups (RE < 0.7) than the other utility measure. Statistical efficiency to detect differences between change groups and within HRQoL deterioration group (RE > 1.4) favored QLU-C10D in 18 of 24 (75%) and 20 of 24 (83%) comparisons with the EQ-5D-3L respectively. Responsiveness to overall HRQoL change (RE > 3.4) also favored the QLU-C10D. Our results indicate that the QLU-C10D is a valid utility measure to assess HRQoL in patients with glioblastoma. This facilitates the investigation of HRQoL profiles and utilities in this patient population by administering a single questionnaire, the EORTC QLQ-C30. Efficiency analyses point to higher statistical power of the QLU-C10D compared to the EQ-5D-3L.

Incremental hemodialysis transition in veterans and nonveterans with kidney failure.

Initiation of hemodialysis treatment with a thrice-weekly prescription is currently the standard of care irrespective of patients' residual kidney function (RKF), comorbidities, and preferences. Each year ∼12 000 Veterans with advanced kidney disease progress to end-stage kidney disease (ESKD) requiring dialysis and comprise greater than 10% of the US incident ESKD population. Dialysis is costly and is associated with impaired health-related quality of life (HRQOL) and high mortality risk, especially in the first year of treatment. Evidence suggests an incremental dialysis transition using twice-weekly hemodialysis provides various benefits, including more dialysis-free time, longer RKF preservation, less vascular access damage, and lower patient burden. Pragmatic studies are needed to inform the efficacy and safety of incremental hemodialysis as a personalized dialysis regimen, and could inform its consideration as a conservation strategy during times of supply shortages. Broadly implementing twice-weekly hemodialysis could also potentially allow more Veterans to receive care within VA-based dialysis units. The VA IncHVets Trial is a pragmatic, multicenter, randomized controlled trial comparing the efficacy and safety of twice-weekly incremental vs. thrice-weekly hemodialysis among Veterans transitioning to ESKD. Further research is needed to determine whether incremental hemodialysis is well tolerated, effective, and facilitates a more favorable transition to dialysis.

Effectiveness of a psychoeducational intervention on myositis patients’ quality of life and well-being: a randomized controlled trial

BackgroundMyositis is a rare disease associated with impaired health-related quality of life. A study evaluating the effectiveness of an intervention to improve the quality of life and well-being of myositis patients is presented.MethodsAll myositis patients in a health district were contacted. Thirty-four eligible patients were randomly assigned to the experimental (n = 17) or control (n = 17) group. A psychoeducational intervention of 5 100-min sessions focusing on the disease as related to daily life was conducted only in experimental patients. Several reliable tools to measure quality of life and well-being were administered twice, before and after the intervention, to both groups.ResultsIn the experimental group, post-test scores were higher than pre-test in quality of life, well-being, and self-efficacy to manage the disease. Improvements were more evident in the experimental group than controls in 70% of the variables studied. Specifically, sedentariness decreased and satisfaction with social relationships increased in the post-test evaluation to a greater degree in the experimental group than in controls.ConclusionsThis randomized controlled trial on a representative sample of myositis patients in an extensive population provides evidence indicating the effectiveness of a psychoeducational intervention for improving HRQoL, well-being, and self-efficacy to manage the disease.Trial registration: NCT06300983.

Better health-related quality of life is associated with prolonged survival and reduced hospitalization risk among dialysis-dependent chronic kidney disease patients: a historical cohort study

Rationale & objectiveEnd-stage kidney disease (ESKD) negatively affects patients’ physical, emotional, and social functioning. Furthermore, adjustment to dialysis require substantial lifestyle changes that may further impact on patients physical and emotional well-being. However, the relationship between Health-Related Quality of life impairment with future adverse outcomes in dialysis is not well characterized. Our study aims to investigate the relationship between Health-Related Quality of Life (HRQoL) and patients’ survival and hospitalization rates within a large European dialysis network.MethodsA historical cohort study was conducted to evaluate association of HRQoL with hospitalization and mortality rates over a 12-month follow-up period. Patients responded to a self-administered survey as part of a Continuous Quality Improvement Program implemented in clinics affiliated with the Spanish FMC-Nephrocare organization. Health-Related Quality of Life (HRQoL) was measured with the KDQOL-36. Potential confounders included socio-demographic characteristics, comorbidities, biochemical parameters, dialysis treatment. We used Cox’s Proportional Hazard regression to assess the hazard of death and Logistic Regression to assess the likelihood of hospital admissions during 12-month follow-up period.ResultsA total of 2280 (51.5%) completed the self-administrated survey, and 1838 patients met the inclusion/exclusion criteria of the study. Higher HRQoL scores were associated with significantly lower mortality and hospitalization risk. Risk estimates were robust to adjustment for potential confounders.ConclusionsSeveral dimensions of HRQoL are associated with patient-centered outcomes (i.e., mortality and hospitalizations at 1 year). Patient-Reported Outcomes contribute unique pieces of information characterizing patients’ health. Residual confounding cannot be fully ruled out; moreover, the high attrition rate could result in selection bias, which may limit the generalizability of the findings to a broader population.

Development of a systematic referral model to primary care health interventions for patients with atrial fibrillation. a delphi study

Abstract Background The European Society of Cardiology (ESC) guidelines for the management of atrial fibrillation (AF) recommend integrated care in AF, combining medical treatment with lifestyle modifications and psychosocial management in a multi-disciplinary set-up. Research show that patients with AF can benefit from lifestyle interventions and psychosocial management, like patient education. According to World Health Organization recommendations, these health interventions are moving from hospitals to primary healthcare settings. Therefore, patients with AF should be systematically referred to these interventions. Yet, an evidence-based referral model that can support this transition is lacking. Purpose The purpose is to develop a systematic referral model for primary care health interventions for patients with AF. Methods We used the Delphi-technique with three rounds of questionnaires (figure 1). International and national clinical experts in AF across disciplines and health sectors were invited to participate. In the first round, the expert panel evaluated items that could be part of a referral model on relevance, improvability and self-reporting. In the second round, the expert panel evaluated each item as either an inclusion or exclusion criteria, important knowledge to have, or not relevant. In the third round, the expert panel evaluated the items according to data source and outcome status. A final model was drawn based on items reaching consensus. The study was reported according to the guidance on conducting and reporting Delphi studies (CREDES). Results In total, 69 of 139 invited experts accepted to participate (50%). These were mostly academics or clinicians (90%), stemming from primary sector (16%), secondary sector (35%) and research (32%). Most were medical doctors (36%) and nurses (35%). However physiotherapist, psychologists, dieticians, and other disciplines along with six patients with AF were also represented. The expert panel (60% females) had a mean age of 50 years, and 65% were from Denmark. For the final referral model (figure 2), AF symptom burden and impaired health-related quality of life, anxiety and poor medicine compliance combined with a risk factor profile were the main inclusion criteria. Furthermore, it was important to know about the patients’ mental well-being, self-management abilities and comorbidities. Conclusion Consensus was reached for a generic referral model, which can be further adjusted in the appropriate clinical context. The model includes inclusion criteria which closely correlate with guidelines for AF management focusing on reducing the patients’ symptom and risk factor burden. The referral model is expected to increase the number of patients with AF referred to primary care health interventions, which could lead to better quality of life, fewer hospital admissions and lowered health care costs in AF.

Determinants of poor health-related quality of life among outpatients with rheumatoid arthritis in Jordan.

The purpose of this study was to assess the health-related quality of life (HRQOL) and investigate the variables contributing to reduced HRQOL in patients with rheumatoid arthritis. The present cross-sectional study was conducted on patients diagnosed with rheumatoid arthritis at two teaching hospitals in Jordan using a convenience sampling technique. The participants were interviewed face-to-face during the scheduled appointment at the outpatient rheumatology clinic. The HRQOL was evaluated by the validated EuroQol-5 Dimension (EQ-5D) questionnaire, which included the EQ-5D utility index that evaluated HRQOL in terms of 5 domains, including mobility, self-care, usual activities, pain/discomfort, and anxiety/depression, and the EQ-5D visual analogue scale (EQ-5DVAS), which evaluated HRQOL on a vertical scale ranging from 0 (worst imaginable health) to 100 (best imaginable health). The validated short version of the 19-item Compliance Questionnaire for Rheumatology (CQR-5) was used to evaluate medication adherence. The Clinical Disease Activity Index (CDAI) was used to assess disease activity among the study participants. A stepwise quantile regression model (q = 0.5) was conducted to explore the factors associated with the EQ-5DUtility Index and EQ-5DVAS scores. In total, 261 patients with RA participated in the study. The median (interquartile range) of the EQ-5DUtility Index and EQ-VAS scores was 0.552 (0.006-0.726) and 0.506 (0.233-0.690), respectively. Regression analysis results demonstrated that medication non-adherence (regression coefficient (β) = -0.348, P<0.01), not performing regular physical activity (β = -0.209, P<0.01), and higher disease activity as measured by the CDAI score (β = -0.015, P<0.01) were significant predictors of a lower EQ-5DUtility Index score. In addition, medication non-adherence (β = -0.199, P<0.01), not performing regular physical activity (β = -0.117, P<0.01), increased body mass index (BMI) (β = -0.009, P<0.01), and higher CDAI score (β = -0.009, P<0.01) were significant predictors of low EQ-5DVAS score. Patients with RA experienced significantly impaired HRQOL. Medication non-adherence, not performing regular physical activity, increased body weight, and increased disease activity were identified as determinants of poor HRQOL among patients with RA in the present study. Treating physicians should encourage regular physical activity, maintaining a healthy body weight, and controlling disease activity to improve HRQOL in patients with RA.