Quality Of Life Measures Research Articles

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.

Quality of life among obstructive sleep apnoea patients: a systematic review and meta-analysis of EQ-5D studies.

Obstructive Sleep Apnoea (OSA) is a chronic condition which is associated with extreme daytime sleepiness, cognitive impairment, and other comorbidities and, hence, decreased quality of life (QoL). EQ-5D is one of the most widely used preference-based measures of QoL in patients with different diseases worldwide. This study aims to review the EQ-5D utility value of OSA across primary studies and perform a meta-analysis to derive pooled utility scores for OSA. A systematic review and meta-analysis of the EQ-5D scores of OSA patients adhering to PRISMA guidelines was conducted. Studies reporting EQ-5D utility scores among adult OSA patients were systematically searched across PubMed-Medline, Scopus, and Embase. Selected studies were systematically reviewed and we have assessed the studies for their quality. Meta-analysis was performed using a random-effect model with subgroup analysis to explore heterogeneity. The search yielded 9,121 articles, of which twenty-eight studies were included in the synthesis. The pooled mean EQ-5D utility score was 0.73 (0.69 to 0.78) with high heterogeneity (I2 = 99.6%). The pooled EQ-5D VAS score was 67.14 (63.97 to 70.32) with high heterogeneity (I2 = 87.04%). Subgroup analyses indicated significant variability in utility scores across different countries, age groups, and disease severities. The variation of utility values for OSA was influenced by the characteristics of patients, the living environment, and the EQ-5D value set. The synthesis revealed that the QoL is significantly lower in OSA, and the aggregated quantitative estimates of EQ-5D utility scores are essential inputs for economic evaluations.

A post-hoc analysis of risk factors for poor quality of life after surgical treatment of spondylodiscitis.

Patient-reported outcome measures for quality of life (QoL) like the Oswestry-Disability-Index (ODI) are useful for a quick, comprehensive and comparable evaluation of spondylodiscitis treatment. While it is known that even after successful surgical treatment, the QoL of spondylodiscitis patients remains below that of the general population, risk factors for a poor outcome have not been described so far. We did a retrospective post-hoc analysis of 130 surgically treated spondylodiscitis patients from 2008 to 2022. Biographical, clinical und QoL data were prospectively collected before (T0) and one year (T1) after surgery. The primary outcome was QoL at T1. Patients were grouped according to their ODI score at T1 (Group 1: ODI < 35, favorable QoL; Group 2: ODI ≥ 35, poor QoL) and analyzed for risk factors present at T0. Poor QoL at T1 occurred in 51 patients (39%). Multivariate analysis identified a higher preoperative leg pain (odds ratio: 1.2) and the presence of a malignant disease (odds ratio: 1.9) as independent preoperative risk factors for a poor QoL at T1. Spondylodiscitis patients with malignancy and/or preoperative leg pain may be at increased risk for poor QoL after surgical treatment. These results can be used to individualize patient information and provide a better assessment of prognosis before surgery.

Estimating Meaningful Differences in Measures of Neuropathic Impairment, Health‐Related Quality of Life, and Nutritional Status in Patients With Hereditary Transthyretin Amyloidosis

ABSTRACTIntroduction/AimsThe degree of change in neuropathic impairment and quality of life (QoL) that is clinically meaningful to patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv‐PN) is not established. This study aimed to estimate the magnitude of treatment differences that are meaningful to patients in measures of neuropathy and QoL and to determine whether eplontersen achieved a meaningful improvement versus placebo.MethodsData from the NEURO‐TTRansform trial on patients with ATTRv‐PN treated with eplontersen (n = 141) or historical placebo (n = 59) were used. Anchor‐based approaches were used to estimate thresholds for meaningful differences in the modified Neuropathy Impairment Score +7 (mNIS+7) composite score, Norfolk QoL‐Diabetic Neuropathy (Norfolk QoL‐DN) total score, Neuropathy Symptoms and Change (NSC) total score, and modified body mass index (mBMI). Differences between the least squares means of the treatment groups were analyzed.ResultsMeaningful improvement in mNIS+7 was estimated as −4.0 points and deterioration as 1.8 points. The estimated ranges of meaningful improvement and deterioration in Norfolk QoL‐DN were −12.8 to −4.0 points, and 5.9 to 14.7 points, respectively. For NSC, ranges were −2.4 to −1.3 points for meaningful improvement, and 0.6 to 5.8 points for deterioration. The estimated meaningful improvement in mBMI was 9.8 kg/m2 × g/L and deterioration was −40.9 kg/m2 × g/L. Improvements in each measure with eplontersen versus placebo were greater than the estimates of meaningful differences.DiscussionEplontersen demonstrated a clinically meaningful effect on neuropathic impairment, QoL, and nutritional status. Such estimates have implications for clinical practice and trials.

Long-Term Air Pollution Exposure and Severity of Idiopathic Pulmonary Fibrosis: Data from the IPF-PRO Registry.

While exposure to air pollution is a known risk factor for adverse pulmonary outcomes, its impact in individuals with idiopathic pulmonary fibrosis (IPF) is less well understood. To investigate the effects of long-term exposure to air pollution on disease severity and progression in patients with IPF and to determine whether genomic factors, such as MUC5B promoter polymorphism or telomere length, modify these associations. We performed analyses at enrollment and after one year of follow-up in the IPF-PRO Registry, a prospective observational registry that enrolled individuals with IPF at 46 US sites from June 2014 to October 2018. Five-year average pollution exposures (PM2.5, NO2, O3) prior to enrollment date were estimated at participants' residential addresses with validated national spatio-temporal models. Multivariable regression models estimated associations between pollution exposure and physiologic measurements (FVC, DLCO, supplemental oxygen use at rest) and quality of life measurements (St. George's Respiratory Questionnaire [SGRQ], EuroQoL, Cough and Sputum Assessment Questionnaire) at enrollment. Cox proportional hazard models estimated associations between pollutants and a composite outcome of death, lung transplant, or >10% absolute decline in FVC % predicted in the year after enrollment. Models were adjusted for individual-level and spatial confounders, including proxies for disease onset. Gene-environment interactions with MUC5B and telomere length were assessed. Of 835 participants, 94% were non-Hispanic Whites, 76% were male, mean (SD) age was 70 (7.7) years. In fully adjusted analyses, higher PM2.5 exposure was associated with worse quality of life per SGRQ activity score (3.48 [95% confidence interval (CI) 0.64, 6.32] per 2µg/m3 PM2.5) and EuroQoL scores (-0.04 [95%CI -0.06, -0.01] per 2µg/m3 PM2.5), and lower FVC % predicted and lower DLCO% predicted at enrollment. Each 3 parts per billion difference in O3 exposure was associated with a 1.57% [95% CI 0.15, 2.98] higher FVC % predicted at enrollment, although this effect was attenuated in multi-pollutant models. There was no association between NO2 and enrollment measures, between pollution exposure and one-year outcomes, or evidence for gene-environment interactions. In the IPF-PRO Registry, long-term exposure to PM2.5 was associated with worse quality of life and lung function at enrollment, but not with short-term disease progression or mortality. There was no evidence of effect modification by interaction of genomic factors with pollution. The reason for the unexpected relationship between O3 exposure and higher FVC is unclear.

Examining the interplay between mental health indicators and quality of life measures among first-year law students: a cross-sectional study.

This research explores the intricate relationships between mental health indicators (depression, stress, and anxiety) and various dimensions of quality of life among first-year law students. The study aims to understand how affective valence, mood states, physical activity, body image perception, and social relations influence mental health outcomes. Data were collected from 75 first-year law students (46 females, 29 males), a group predominantly composed of young adults with limited financial means, living in various housing situations, primarily within urban environments, and generally reporting low levels of physical activity. Standardized questionnaires were used to assess mental health and quality of life, including the Depression, Anxiety, and Stress Scale-21 Items (DASS-21), Feeling Scale (FS), Exercise-Induced Feeling Inventory (EIFI), Modified Baecke Physical Activity Questionnaire (MBPAQ), World Health Organization Quality of Life-BREF (WHOQOL-BREF), and Contour Drawing Rating Scale (CDRS). Descriptive statistics, Pearson correlation, and regression analysis were employed to analyze the data. The analysis revealed significant correlations between depression (mean = 5.97, SD = 4.21), stress (mean = 7.81, SD = 4.80), and anxiety (mean = 6.17, SD = 4.58) with affective valence (p < 0.05), mood states (p < 0.05), physical activity (p < 0.05), body image perception (p < 0.05), and social relations quality (p < 0.05). Additionally, mood states (mean = 20.73, SD = 10.60), physical activity (mean = 8.43, SD = 1.35), body image perception (mean = 4.21, SD = 1.91), and social relations quality (mean = 12.46, SD = 2.33) were identified as significant predictors of mental health outcomes (p < 0.05). These findings underscore the complex interplay between mental health indicators and various dimensions of quality of life, emphasizing the necessity for a comprehensive approach to mental health care. By identifying these predictors, we have gained a clearer understanding of the factors that impact mental health in this specific population. The insights gained highlight the value of interventions aimed at improving mood, increasing physical activity, enhancing body image, and strengthening social connections. These targeted strategies could effectively address mental health issues and promote well-being among law students. Future research should further investigate these relationships and develop tailored interventions to better support students' mental health. This study contributes to understanding the complex interplay between mental health and quality of life, offering a foundation for both practical interventions and future research.

Improvement in quality of life in MDS patients who become transfusion independent after treatment

Myelodysplastic syndromes (MDSs) treatment focuses on improving quality of life (QOL), affected by anemia and transfusion dependence (TD). Using the MDS-CAN registry, we studied how changes in transfusion status – TD to transfusion independence (TI) (group A), or vice versa (group B), and maintaining TD (group C) or TI (group D) – affected OS and QOL in 1120 MDS patients. Analysis showed superior OS for those remaining TI, poorer for those remaining TD, and intermediate for those with changes. Among 656 treated patients, group A (n = 54) showed improved QOL, with trends toward improved physical and social function scores. Group B (n = 151) experienced declines in global QOL measures after switching to TD, particularly in fatigue and physical, role, and social functioning. Group C had notable fatigue worsening, while group D showed milder declines across multiple QOL aspects. Achieving TI in MDS correlates with improved QOL, whereas reverting to TD more significantly worsens overall QOL and function scores.

Patient-centred composite scores as tools for assesment of response to biological therapy for paediatric and adult severe asthma.

We have previously developed Core Outcome Measures sets for Severe Asthma (COMSA) by multi-stakeholder consensus. There are no patient-centred tools to quantify response to biologics for severe asthma. We aimed to develop paediatric and adult CompOsite iNdexes For Response in asthMa (CONFiRM) incorporating clinical parameters and patient-reported quality of life (QoL). International expert healthcare professionals (HCPs) and patients with severe asthma were invited to: 1) develop consensus levels of clinically relevant changes for each outcome measure within COMSA; 2) use multicriteria decision analysis to develop the CONFiRM scores and 3) assess their internal validity. A separate group of HCPs evaluated CONFiRM's external validity. Five levels of change for each COMSA outcome were agreed. Severe exacerbations and maintenance oral corticosteroids use were rated as most important in determining both paediatric and adult CONFiRM scores. There was strong agreement between HCPs and patients, although patients assigned greater importance to QoL. The CONFiRM score quantified response to a biological from -31 (deterioration) to 69 (best possible response). Paediatric and adult CONFiRMs had good discriminative ability for a sufficient (AUC≥0.92) and a substantial (AUC≥0.95) response to biologics. Both CONFiRMs demonstrated excellent external validity (Spearman correlation coefficients 0.9 and 0.8 for paediatric and adult respectively (p<0.0001)). We have developed novel patient-centred paediatric and adult CONFiRMs which include QoL measures. CONFiRMs should allow a more holistic understanding of response for the patient and a standardised assessment of the effectiveness of biologics between studies. Further research is needed to prospectively validate CONFiRM scores.

How Do Surgical Interventions for Neurogenic Lower Urinary Tract Dysfunction Impact Quality of Life?

Adult patients with neurogenic lower urinary tract dysfunction (NLUTD) often have urinary symptoms that impact their quality of life (QOL). Our objective is to identify and summarize studies evaluating QOL changes across different NLUTD surgical interventions. A systematic rapid evidence review was carried using EMBASE and MEDLINE. We included adult patients (> 18 years old) with NLUTD who underwent a relevant surgery and had a measurement of QOL. We included pre-post study designs (primary focus) and cross-sectional studies (secondary focus). Studies were reviewed and data extracted by multiple assessors. Standardized data extraction tables were used, and qualitative synthesis was performed. Of the 1074 screened articles 26 were included. There were 3/15 studies that evaluated reconstructive surgery (augmentation and/or catheterisable channel) pre-post intervention (n = 94 patients); there was a 7%-28% relative improvement in bladder related and overall QOL using validated questionnaires, and a large magnitude of improvement in studies using unvalidated questionnaires. There were 3/7 studies that looked at urinary diversion pre-post intervention (n = 153 patients) and showed an approximately 20%-60% improvement in validated questionnaires assessing bladder specific quality of life, and 0%-25% improvement in overall quality of life. Finally, 3/4 studies were pre-post stress incontinence surgeries (n = 67 patients) and they found an improvement in the ICIQ questionnaire scores and study-specific questionnaires. The literature supporting a change in QOL in adult NLUTD patients undergoing surgical interventions is extremely limited due to a lack of pre-post studies, and the frequent use of unvalidated outcome measures.

Home and Community Participation Patterns, Quality of Life and Family Routines among Preschool Children with ADHD

AbstractAttention deficit hyperactivity disorder (ADHD) symptoms manifest early in childhood and impact participation in daily activities and quality of life (QoL). Family routines may enhance the participation of children with ADHD. However, few studies have examined the correlations linking participation, QoL, and family routines in this population. This study aimed to describe participation patterns, environmental factors, family routines, and QoL of preschool children with ADHD, and examine the relationships of these variables. Parents of 70 children aged 4–6 (79% boys), clinically-evaluated as having ADHD or suspected ADHD, completed demographic, participation and environment, QoL and family routines measures. Frequency of participation and desire for change at home were found to be significantly higher compared to the community, while involvement did not differ between settings. Environmental factors supporting and inhibiting participation were identified. Significant correlations were found between home participation and family routines, and between home participation and QoL. The findings reinforce the need for an in-depth examination of participation patterns, environmental factors, and family routines to determine appropriate intervention goals that promote the participation and QoL of preschool children with ADHD.

Quality of Life in Mild Cognitive Impairment and Mild Dementia Associated with Alzheimer's Disease: A Systematic Review.

Mild cognitive impairment (MCI) and Alzheimer's disease (AD) have a profound impact on patients' quality of life (QoL), with progressive declines occurring as the disease advances. This systematic review aims to summarize the published evidence on patient-reported outcomes (PROs) in individuals with MCI due to AD and mild AD dementia. Comprehensive searches were conducted across five major databases to identify studies reporting on utility values, disutilities, and QoL measures in these patient populations. A total of 23 studies were included that utilized various QoL assessment tools, including EQ-5D (n = 14), SF-36/SF-12 (n = 4), and QOL-AD (n = 11). Reported EQ-5D scores ranged from 0.81 to 0.92 for patients with MCI and from 0.67 to 0.85 for those with mild AD, indicating a noticeable decline in QoL as the disease progresses. QOL-AD scores ranged from 33.8 to 42.5 for MCI and from 32.4 to 38.1 for mild AD, equally reflecting the greater impairment in QoL with disease advancement. Interventions were generally associated with smaller declines in PROs compared to placebo, suggesting a positive impact of treatment in mitigating QoL deterioration. The findings underscore the significant QoL differences between MCI and mild AD, emphasizing the potential benefit of early intervention to preserve QoL and delay disease progression. This review highlights the importance of continued research to better understand QoL in patients with MCI and mild AD dementia, particularly in terms of capturing comprehensive patient-reported outcomes and evaluating the effectiveness of interventions over time. These findings can contribute to a more informed approach in clinical practice and support decision-making in the management of early-stage AD.

Listening to children with lower limb loss: Rationale, design, and protocol for delivery of a novel globally applicable research toolkit-Prosthetic user needs, quality of life, pain, and physical function.

Rehabilitation after childhood lower limb loss is complex and dependent on multiple stakeholders and environmental factors. While research with adults underscores the importance of involving prosthetic limb users and caregivers in discussions to drive innovation, children are often excluded or not effectively engaged. This protocol lays out the development and implementation protocol for an internationally applicable research toolkit which has been designed and evaluated around the essential presence of the child. Research domains span their unique prosthetic needs, quality of life, pain, and mobility. Cohorts of children in contrasting environments were identified (Cambodia, Gaza Strip, and the UK) to provide a comprehensive global understanding of the child with lower limb loss. A literature review revealed a lack of appropriate tools for identifying paediatric prosthetic user requirements leading to the development of novel interview guides for each key stakeholder: child, caregiver, and prosthetist. The child's guide centred around enjoyment and engagement using card games and activities. A panel of experts in paediatric limb loss and mental health rigorously reviewed the guides. Guides were integrated with existing validated measures for quality of life, pain, and mobility to form a comprehensive toolkit. The toolkit was successfully piloted with 5 children, their families, and 2 prosthetists. This protocol lays out the toolkit rationale and implementation plan (Jan 2023 to Dec 2025). This work offers the opportunity for this cohort to enjoyably engage with research that seeks to radically improve prospects for all children living with limb loss. The outlined best practices ensure ethical considerations when working with vulnerable cohorts. This study is approved to cover implementation at all geographical locations as well as the researcher institutions. Results will be disseminated through national and international conferences, as well as through manuscripts in leading peer-reviewed journals.

Adding Metastasis-Directed Therapy to Standard-of-Care Systemic Therapy for Oligometastatic Breast Cancer (EXTEND): a Multicenter, Randomized Phase II Trial

PurposePrior evidence suggests a progression-free survival (PFS) benefit from adding metastasis-directed therapy (MDT) to standard-of-care (SOC) systemic therapy for patients with some oligometastatic solid tumors. Randomized trials testing this hypothesis in breast cancer have yet to be published. We sought to determine whether adding MDT to SOC systemic therapy improves PFS in oligometastatic breast cancer. MethodsEXTEND is a multicenter phase II randomized basket trial testing the addition of MDT to SOC systemic therapy in patients with ≤5 metastases (NCT03599765). Patients were randomized 1:1 to MDT (definitive local treatment to all sites of disease, plus SOC systemic therapy) or to SOC systemic therapy only. Primary endpoint was PFS, and secondary endpoints included overall survival (OS), time to subsequent line of systemic therapy, and time to the appearance of new metastases. Exploratory analyses included quality of life (QOL) and systemic immune response measures. ResultsFrom September 2018 through July 2022, 22 and 21 patients were randomized to the MDT and no-MDT arms, respectively. At a median follow-up of 24.8 months, PFS was not improved with the addition of MDT to SOC systemic therapy (median PFS 15.6 months MDT vs 24.9 months no-MDT [hazard ratio {HR} 0.91; 95% CI 0.34–2.48, p=0.86]). Similarly, MDT did not improve OS, time to subsequent line of systemic therapy, or time to the appearance of new metastases (all p>0.05). No significant differences were found in QOL measures, systemic T-cell activation, or T-cell stimulatory cytokine concentration. ConclusionAmong patients with oligometastatic breast cancer, the addition of MDT to SOC systemic therapy did not improve PFS. These findings suggest that MDT may have no systemic benefit in otherwise unselected oligometastatic breast cancer patients, although this trial was limited by a heterogenous and small sample size and overperformance of both treatment arms.

Non-Surgical Medical Aesthetics and Patient Quality of Life: An Umbrella Review

Abstract Background Non-surgical cosmetic facial procedures have become popular treatment options for individuals seeking aesthetic improvements. Despite a breadth of literature on patient satisfaction with treatment outcomes, there is a lack of information specific to changes in quality of life outcomes. Objectives The objective of this umbrella review was to report the effectiveness of non-surgical facial aesthetic treatments on reported quality of life in cosmetic treatment seeking patients. We also aimed to identify gaps in the literature on measures of quality of life outside of patient satisfaction. Methods We completed a comprehensive, systematic search of review articles across six databases including Medline, CINAHL Plus, EMBASE, APA PsychINFO, Cochrane Reviews and Google Scholar. We included review-level studies examining the change in quality of life measures following treatment. Critical appraisal was completed for each review article included. Results A total of seven reviews included. One review was of strong quality, two moderate, and four were weak. Several non-surgical procedures were evaluated across reviews including injectable neurotoxins, dermal fillers and laser skin resurfacing. The majority of included studies reported increases in measures of quality of life, post treatment in the same patient or compared to controls. The most commonly reported measure was psychological wellbeing, followed by self-perception. There was a lack of measures outside of improvements to aesthetics, including those specific to mental health (e.g., depression). Conclusions Overall, studies that report on non-surgical treatments report increases in overall quality of life. This conclusion should be interpreted with caution as the majority of reviews included were of moderate to weak quality. A major gap in this literature includes mental health outcomes. Future research should focus on increasing the rigour of reporting for systematic reviews.

Safety and Efficacy of Neoadjuvant Docetaxel and Radiotherapy in Localized High-Risk Prostate Cancer: Results From a Prospective Pilot Study.

Approximately 15% of patients with localized prostate cancer are at high risk for disease recurrence. Many clinical trials have evaluated the impact of neoadjuvant therapy before radical prostatectomy with mixed results (NCT00321698). This phase I/II clinical trial evaluated the tolerability and preliminary efficacy of neoadjuvant radiation therapy and docetaxel before prostatectomy in 25 men with high-risk prostate cancer. The treatment regimen included 45Gy radiotherapy in 25 fractions to the prostate and seminal vesicles over 5 weeks, along with weekly dose-escalated docetaxel up to 30mg/m², followed by prostatectomy and bilateral lymph node dissection. The primary endpoint was the rate of pathologic complete response (pCR). Secondary endpoints included adverse events, symptom and quality of life measures, and prostate-specific antigen metrics. All 25 patients completed the planned treatment. The primary endpoint of pCR was not achieved. Lymphopenia was the most common grade 3 or higher toxicity, with no grade 3 or higher genitourinary or gastrointestinal toxicities observed. With a median follow-up of 11.6 years, the 10-year biochemical recurrence-free survival was 60%, and distant metastasis-free survival was 80%. Prostate cancer-specific survival and overall survival at 10 years were 84% and 60%, respectively. Although pCR was not met, the treatment demonstrated a modest toxicity profile and reasonable long-term outcomes, suggesting feasibility and safety. Further studies are needed to optimize endpoints and assess the efficacy of neoadjuvant treatments compared with standard approaches in high-risk prostate cancer patients.

Vascular occlusion for optimising the functional improvement in patients with knee osteoarthritis: a randomised controlled trial.

Knee osteoarthritis (KOA) is a leading cause of global disability with conventional exercise yielding only modest improvements. Here we aimed to investigate the benefits of integrating blood flow restriction (BFR) into traditional exercise programmes to enhance treatment outcomes. The Vascular Occlusion for optimizing the Functional Improvement in patients with Knee Osteoarthritis randomised controlled trial enrolled 120 patients with KOA at Ghent University Hospital, randomly assigning them to either a traditional exercise programme or a BFR-enhanced programme over 24 sessions in 12 weeks. Assessments were conducted at baseline, 6 weeks, 12 weeks and 3 months postintervention using linear mixed models with Dunn-Sidak corrections for multiple comparisons. Primary outcome was the Knee injury and Osteoarthritis Outcome Score (KOOS) questionnaire at 3 months follow-up with knee strength, Pain Catastrophizing Scale questionnaire and functional tests as secondary outcomes. Analysis followed an intention-to-treat approach (NCT04996680). The BFR group showed greater improvements in KOOS pain subscale (effect size (ES)=0.58; p=0.0009), quadriceps strength (ES=0.81; p<0.0001) and functional tests compared with the control group at 12 weeks. At 3 months follow-up, the BFR group continued to exhibit superior improvements in KOOS pain (ES=0.55; p=0.0008), symptoms (ES=0.59; p=0.0004) and quality of life (QoL) (ES=0.66; p=0.0001) with sustained benefits in secondary outcomes. Drop-out rates were similar in both groups. Incorporating BFR into traditional exercise programmes significantly enhances short-term and long-term outcomes for patients with KOA demonstrating persistent improvements in pain, symptoms, QoL and functional measures compared with conventional exercise alone. These findings suggest that BFR can provide the metabolic stimulus needed to achieve muscle strength and functional gains with lower mechanical loads. Reduced pain and increased strength support a more active lifestyle, potentially maintaining muscle mass, functionality and QoL even beyond the supervised intervention period. NCT04996680.

Acceptability and psychometric properties of four scales assessing the impact of Type 2 diabetes on quality of life-Results of 'YourSAY: Quality of Life'.

To assess and compare the psychometric properties and acceptability of four diabetes-specific quality of life (QoL) scales among adults with Type 2 diabetes (T2D). Adults (≥18 years) with T2D living in the United Kingdom (n = 1465) or Australia (n = 248) completed a cross-sectional, online survey including the following: ADDQoL, DCP, DIDP and Diabetes QoL-Q (presented in randomised order), followed by rating scales to assess clarity, relevance, ease of completion, length and comprehensiveness of each scale. Demographic, clinical and psychosocial characteristics were collected. Acceptability (scale completeness and user ratings), response patterns, structure (exploratory and confirmatory factor analyses) and validity (convergent, confirmatory, divergent and known-groups) were examined. Data were analysed by country to assess cross-country reproducibility. High completion rates (≥89%) and positive user ratings were observed across scales indicating broad acceptability. The DIDP was the strongest performing scale: highest completion rate (97%), user ratings (≥84% positive) and most satisfactory psychometric properties (highest variance explained, consistent factor loadings >0.5 on all items and most permissible model fit parameters). Scale-level floor effects may suggest domain omissions for the brief DIDP. The current study provides novel insights into the acceptability, validity and reliability of diabetes-specific QoL measures for adults with T2D. Consistent with the published Type 1 diabetes cohort findings, the DIDP is recommended as a brief, acceptable and psychometrically sound measure. However, selection needs to be considered in the context of the specific research or clinical aims and further evidence (e.g. responsiveness) may be required before it can be recommended for use in trials or prospective studies.

Quality of life measures with etripamil self-administration for acute episodes of paroxysmal supraventricular tachycardia in a medically unsupervised setting: patient-reported outcomes from NODE-303

Abstract Background Etripamil nasal spray (NS) is a fast-acting, self-administered calcium-channel blocker in development for the conversion of AV-nodal dependent paroxysmal supraventricular tachycardia (PSVT) in a medically unsupervised setting. Prior studies show favorable safety and efficacy in converting single episodes of PSVT to sinus rhythm (SR). Purpose In NODE-303, patient-reported outcome (PRO) instruments assessed quality of life (QoL) over multiple etripamil-treated episodes of PSVT. Methods NODE-303 was an event-driven, multi-center, open-label Phase 3 study in North and South American patients with documented PSVT that evaluated the safety and efficacy of etripamil in PSVT termination, over multiple episodes and without the need for prior test dosing. Enrolled patients who perceived PSVT symptoms applied an ambulatory ECG cardiac monitoring system (CMS), performed a pre-trained vagal maneuver and, if symptoms persisted, self-administered etripamil NS 70 mg. A study amendment allowed a repeat dose (etripamil NS, 70 mg) if symptoms persisted 10 min after the first dose. Each patient could self-treat up to 4 episodes. Safety was assessed through adverse events, clinical data, and ECG CMS recordings. The Treatment Satisfaction Questionnaire for Medication (TSQM; validated for acute episodic assessments) and other PROs were administered, and healthcare utilization data were acquired. Patients completed PROs at baseline (BL), monthly, and per PSVT episode, which collected information on symptoms, episode characteristics, rescue medications, and emergency room visits. Additional PROs were the Brief Illness Perception Questionnaire (BIPQ), Cardiac Anxiety Questionnaire (CAQ), and 36-Item Short Form Health Survey (SF-36). Results There were higher proportions of patients reporting mild, minimal or no disease severity, every 6 months, than at BL; extremely severe, severe, and moderate disease severity was reported by smaller proportions of patients than at BL, though incomplete surveys at later visits may limit this analysis. Anxiety and stress about future PSVT episodes were consistently reduced from BL. Measures of treatment satisfaction, effectiveness, and convenience were consistent across multiple episodes, and were at levels considered favorable for the TSQM instrument (Table 1). Rates of emergency-room visits and hospital admissions across multiple, treated PSVT episodes were similar to those observed in trials assessing single episodes (Table 2). The BIPQ, CAQ or SF-36 had no notable changes from BL. Conclusions Etripamil self-administration was associated with patient-reported improvements in degree of disease severity, decreased anxiety and stress over future PSVT episodes, and levels of treatment satisfaction, effectiveness, and convenience considered favorable for respective PRO instrument. Using a symptom-prompted, self-administered treatment for PSVT could potentially improve QoL measures and reduce healthcare resource utilization.

Development of a novel Patient-Reported Outcome Measure (PROM) for adults with an Inherited Cardiac Condition (ICC)

Abstract Background Research on the quality-of-life (QoL) among patients with inherited cardiac conditions (ICC) is limited. Existing evidence from a systematic review indicates that most studies rely on generic measures of health-related QoL, which may not adequately capture ICC-specific factors. The current study aims to develop a disease-specific patient-reported outcome measure to address this gap. Methods Using a qualitative study design, we first conducted focus groups until data saturation was achieved (n=15). Additionally, an expert panel of healthcare professionals was interviewed to ascertain their perspectives on patient-centered care and to evaluate face validity. Transcripts of all focus groups and interviews were analyzed thematically using Atlas.ti, qualitative analysis software, to identify concepts and formulate survey items for the initial instrument. We used the ‘Centre for Health Promotion’ QoL model, consisting of three domains- Being, Belonging, and Becoming- as the theoretical framework for our analysis. Results Our analysis revealed alignment with specific QoL domains of the overarching framework and identified a gap in genetic-specific domains. A novel overarching theme, genetic well-being, was identified, encompassing specific constructs related to future uncertainty, reproductive concerns, and the anticipation of disease development, thus enriching the conceptualization of QoL in the context of inherited cardiac conditions. The discussion will include an exploration of the face validity of the draft instrument. Conclusion Patients diagnosed with an inherited cardiac condition (ICC) face a spectrum of challenges originating from both disease-related stressors and hereditary factors alike. Recognizing that relying solely on objective clinical assessment may fail to capture the full impact of the disease, there has been an increasing focus on incorporating patient-reported measures into disease evaluation. This qualitative study contributes to developing a comprehensive patient-reported outcome measure tailored to individuals with ICC.PROM development study

Examining Long-Term Influences of Frailty on Outcomes for Adults Undergoing Left Ventricular Assist Device Therapy.

Frailty is common in adults with end-stage heart failure receiving a left ventricular assist device (LVAD). Short-term studies show frailty reversal post-LVAD. Little is known about long-term frailty and how frailty relates to key LVAD outcomes, including depression, quality of life (QoL), and cognition beyond 6 months. The purpose of this study was to examine physical frailty, depression, QoL, and cognition from pre-LVAD implantation to 3, 6, and 12 months post-LVAD implantation. Clinical data and measures of physical frailty, depression, QoL, and cognition pre- and post-LVAD were extracted from an existing institutional database. Descriptive statistics and tests for statistical significance were used to describe and compare changes over time. Frailty trajectories were identified to describe physical frailty from pre-LVAD to 12 months post-LVAD. The sample (n = 46) was predominantly male (76%), with a mean age of 64.7 ± 11 years, and over half (n = 25) were physically frail. Physical frailty was reduced by nearly half at 12 months. Frailty, depression, and QoL significantly improved at all time points (P < .001). Improvements in cognition did not reach statistical significance. Recipients of LVAD without improvement in frailty exhibited worse depression, QoL, and cognition scores at 12 months compared with those not frail pre-LVAD or no longer frail post-LVAD implantation. Some recipients of LVADs experienced physical frailty reversal with benefits sustained long-term. Those without improvement recorded worse long-term patient-reported outcomes. Identifying factors that predict physical frailty response to LVAD implantation is a key area of future research.