UNASSIGNED: Arginase 1 deficiency (ARG1-D) is a ultrarare disease with manifestations that cause mobility and cognitive impairment that progress over time and may lead to early mortality. Diseases such as ARG1-D have a major impact also outside of the health care sector and the aim of this study was to estimate the current burden of disease associated with ARG1-D from a societal perspective.
UNASSIGNED: The study was performed as a web-based survey of patients with ARG1-D and their caregivers in four European countries (France, Portugal, Spain, United Kingdom). The survey was distributed at participating clinics and included questions on e.g. symptoms (including the Gross Motor Function Classification System, GMFCS, and cognitive impairment), health care use, medication, ability to work, caregiving, and impact on health-related quality-of-life (HRQoL) using the EQ-5D-5L.
UNASSIGNED: The estimated total mean societal cost per patient and year was £63,775 (SD: £49,944). The cost varied significantly with both mobility impairment (from £49,809 for GMFCS level 1 to £103,639 for GMFCS levels 3-5) and cognitive impairment (from £43,860 for mild level to £99,162 for severe level). The mean utility score on the EQ-5D-5L for patients was 0.498 (SD: 0.352). The utility score also varied significantly with both mobility impairment (from 0.783 for GMFCS level 1 to 0.153 for GMFCS level 3-5) and cognitive impairment (from 0.738 for mild level to 0.364 for severe level).
UNASSIGNED: Similar to other studies of rare diseases, the study is based on a limited number of observations. However, the sample appear to be reasonably representative when comparing to previous studies of ARG1-D. This study shows that ARG1-D is associated with a high societal cost and significant impact on HRQoL. Earlier diagnosis and better treatment options that can postpone or withhold progression may therefore have a potential for improved HRQoL and savings for the patient, caregiver, and society.

OBJECTIVE: Studies reporting patient-centered outcomes, including quality of life and satisfaction, in perforated peptic ulcer (PPU) treatment are insufficient. This study was designed to assess the relative efficacy of laparoscopic repair (LR) as opposed to open surgical repair (OR) in the treatment of and its impact on quality of life.
METHODS: This investigation performed a retrospective review of patients who underwent treatment for PPU at a secondary care hospital between January 2017 and April 2020. Patients were categorized into 2 separate groups according to the type of surgical procedure received: LR and OR. Comparisons were made based on a variety of factors, such as demographic data, intra- and postoperative metrics, pain control, patient contentment, and quality of life indicators.
RESULTS: In the analysis, there were no statistically significant differences in demographic or clinical characteristics between the LR (n = 35) and OR (n = 62) groups (P > .05). Conversely, the rates of incisional hernia and surgical site infection were significantly greater in the OR group (P < .05). Moreover, the LR exhibited benefits such as a shorter length of hospital stay (P < .05), more rapid resumption of a normal diet, and fewer surgical site infections-factors that contributed to a lower rate of overall postoperative complications. According to the quality-of-life questionnaire, the LR group exhibited significantly greater scores for physical function, role, pain, and general health by the 30th postoperative day (P = .003, P < .001, P = .006, and P = .001, respectively), and by the 1-year follow-up, the LR group showed substantial improvements in physical function, physical role, emotional role, pain and general health (P = .047, P = .004, P = .039, P = .001, and P = .021, respectively), indicating its effectiveness in patient recovery and quality of life enhancement after surgery.
CONCLUSIONS: This study showed that LR could provide certain benefits in managing PPUs, such as reduced lengths of hospital stay and lower incidences of surgical site infections. Although LR reported promising directions in patient satisfaction and quality of life indicators, the limited duration of postoperative monitoring necessitates caution in broadly applying these results.

Using cross-sectional data from a representative sample of the French population (the 2008 Disability Health survey), this paper examines whether the SF-6D, a widely used preference-based measure of health-related quality of life in economic evaluations, fully captures the variation in subjective well-being (SWB) due to chronic illnesses. We conduct a mediation analysis to disentangle the direct and indirect, through the SF-6D, effects of various chronic conditions on SWB (happiness). Our results show that the SF-6D reflects changes in happiness due to most illnesses except mental illness. Changes in SWB mediated by the SF-6D account for 74% of the total effect. The variation unexplained by the SF-6D is significant and increases substantially in the presence of multimorbidity when a chronic illness is combined with anxiety or depression. Overall, our results suggest that the SF-6D incompletely captures the subjective experience of chronically ill patients, especially those with comorbid conditions.

Atopic dermatitis is a chronic skin condition that has significant psychosocial and quality-of-life impact. The condition causes physical discomfort, emotional distress, embarrassment, social stigma, and daily activity limitation. In an effort to assess these aspects of disease burden, quality-of-life measurement tools were developed. Through use of these tools, we have expanded our knowledge of the psychosocial and quality-of-life burden of this condition. A variety of quality of assessment tools exist, yet there is no consensus on which tool is best suited to assess the quality-of-life impact of atopic dermatitis. Research studies assessing quality-of-life in atopic dermatitis patients utilize a variety of quality-of-life measurement tools; this complicates comparisons across research studies. Though comparison across studies is difficult, the data echoes tremendous overall burden of disease, especially pertaining to psychosocial status and life quality.

Health-related quality-of-life (HRQoL) data are central to capturing the quality of patients\' life, while endpoints like overall survival (OS) focus on the quantity of life. When analyzing HRQoL data gathered from patients in a randomized trial, a key consideration is the completion rate - indicating the proportion of patients remaining in the trial and with completed questionnaires. When completion rates are disproportionately low in one treatment arm, one likely explanation is that patients who did not complete questionnaires suffered more from toxicities, negatively impacting their HRQoL. This is likely the case when low completion rates occur in the more toxic arm within a randomized trial. If the HRQoL analysis is run as a complete-case analysis - only considering patients without missing data - a decrement in HRQoL can be missed. Conversely, when completion rates are high, the HRQoL data are thought to be more reliable, and informative censoring is less likely. We describe why this reasoning can be inadequate. In trials where high and imbalanced rates of early censoring affect progression-free survival or OS endpoints, the completion rates only apply to the fraction of patients remaining in the trial. In those, HRQoL results should be considered with caution, and reasons for censoring in the primary time-to-event analyses should be explored before making definite conclusions about HRQoL. This is even more relevant in trials with non-inferiority design, where a benefit in HRQoL could be used as a justification to modify practice.

BACKGROUND: The EvalUation of goal-diRected activities to prOmote well-beIng and heAlth (EUROIA) scale is a novel patient-reported measure that was administered to individuals with chronic heart failure (CHF). It assesses goal-directed activities that are self-reported as being personally meaningful and commonly utilized to optimize health-related quality of life (HRQL). Our aim was to evaluate psychometric properties of the EUROIA, and to determine if it accounted for novel variance in its association with clinical outcomes.
METHODS: This study was a secondary analysis of the CHF-CePPORT trial, which enrolled 231 CHF patients: median age = 59.5 years, 23% women. Baseline assessments included: EUROIA, Kansas City Cardiomyopathy Questionnaire-Overall Summary (KCCQ-OS), Patient Health Questionnaire-9 for depression (PHQ-9), and the Generalized Anxiety Disorder-7 (GAD-7). 12-month outcomes included health status (composite index of incident hospitalization or emergency department, ED, visit) and mental health (PHQ-9 and GAD-7).
RESULTS: Exploratory Principal Axis Factoring identified four EUROIA factors with satisfactory internal reliability: i.e., activities promoting eudaimonic well-being (McDondald\'s ω = 0.79), social affiliation (⍺=0.69), self-affirmation (⍺=0.73), and fulfillment of social roles/responsibilities (Spearman-Brown coefficient = 0.66). Multivariable logistic regression indicated that not only was the EUROIA inversely associated with incidence of 12-month hospitalization/ED visits independent of the KCCQ-OS (Odds Ratio, OR = 0.95, 95% Confidence Interval, CI, 0.91, 0.98), but it was also associated with 12-month PHQ-9 (OR = 0.91, 95% CI, 0.86, 0.97), and GAD-7 (OR = 0.94, 95% CI, 0.90, 0.99) whereas the KCCQ-OS was not.
CONCLUSIONS: The EUROIA provides a preliminary taxonomy of goal-directed activities that promote HRQL among CHF patients independently from a current gold standard state-based measure.
BACKGROUND: NCT01864369; https://classic.
RESULTS: gov/ct2/show/NCT01864369 .

BACKGROUND: Cervical cancer is the second most prevalent and the leading cause of cancer related deaths among Ethiopian women; and about three fourth are diagnosed at advanced stages. Cervical cancer can affect the health-related quality of life (HRQOL) in multiple ways. The main aim of this study was to describe the HRQOL of cervical cancer patients and the predictive factors using validated tools.
METHODS: Institution based cross-sectional study was conducted among 264 cervical cancer patients using the validated Amharic version of European Organization for Research and Treatment of Cancer (EORTC) modules; QLQ-C30 and QLQ CX24. Descriptive statistics were used to summarize the raw data. One way ANOVA was used to determine the significance of mean differences between the dependent and independent variables. Binary and multivariable regression analysis were used to measure the association between Global Health Status and independent factors. The level of significance was set at p-value < 0.05.
RESULTS: On EORTC QLQ-C30 scales, the mean Global Health Status (GHS) was 42.57 ± 23.31. The least and highest affected functions were physical and social, mean (SD) = 76.39 ± 23.24 and 50.40 ± 32.19, respectively. The financial difficulty was the most affected among the symptom scales, 57.83 ± 35.34. Only physical function and financial difficulty have shown an independent association with GHS, (AOR = 0.21, 95% CI = 0.05-0.84), (AOR = 0.21 95% CI = 0.07-0.59), respectively. Illiterate, can read and write, were among the predictor factors that showed an independent association with the Global Health Status. Among the EORTC QLQ-CX24 symptom scales, the highest affected score was for sexual worry, mean (SD) = 51.81 + 32.197.
CONCLUSIONS: In an effort to improve the Global Health Status of cervical cancer patients in Ethiopia; physical function and financial difficulty should be the priority areas. The Illiterate and those who lack formal education need due attention in order to improve the health-related quality-of-life.

BACKGROUND: Biologic therapies have demonstrated benefits for individuals with severe asthma, including reduced daily symptoms and severe exacerbations. However, data describing patient perspectives on these treatments are limited. This study sought to understand the preferences and priorities of Canadians with severe asthma in the context of novel biologic treatment options.
METHODS: Semi-structured, qualitative interviews were conducted among Canadians with severe asthma from July to August 2022. Purposeful sampling included individuals with and without biologic therapy experience. All participants described daily life with severe asthma, experiences and priorities related to asthma treatment and their impressions of biologics. Reflexive thematic analysis was used to explore patterns in the data.
RESULTS: Among 18 individuals included, 10 were currently taking or had prior experience with biologic treatment for asthma. Those who had never been treated with biologics were unfamiliar with them, considering treatment, or believed that they may not be eligible. Four themes were developed to convey the perspectives of participants on biologics: (1) life-changing benefits, but not for all; (2) navigating barriers to being prescribed and remaining adherent to biologic treatments; (3) treatment administration preferences are not only about convenience; (4) concerns about safety and the unknown as a source of treatment hesitancy.
CONCLUSIONS: Findings suggest that the clinical benefits of biologics align with patient perceptions of achieving good asthma control. However, treatment gaps persist among individuals who do not experience a meaningful improvement in their asthma symptoms and those who face barriers accessing biologics. People with severe asthma attributed importance to greater availability of at-home treatment options, improved access to financial support to cover treatment costs and support to address safety concerns. This research provides insight into patient-based treatment priorities and preferences for biologics, which may help inform decision-making related to emerging therapies for severe asthma.
For people with severe asthma, biologics are a treatment option that can be taken in addition to their regular medication. In this study, we asked 18 Canadians with severe asthma about how having severe asthma affects their lives, their current and previous asthma treatments, and their views on biologics. Ten people in this study were currently taking or had previously taken biologics for severe asthma. We found that biologics can be life changing. Also, people with severe asthma can find it difficult to get on and stay on biologics. They would like financial and educational support when considering biologics and prefer to take biologics at home, if possible. This study helps us understand the priorities and preferences related to biologics of patients with severe asthma.

To systematically review the literature and summarize the health-related quality-of-life (HRQoL) of patients undergoing treatment for mucopolysaccharidoses (MPS), a rare, hereditary lysosomal storage disorder.
A systematic review was performed in accordance with PRISMA guidelines to identify research studies that describe the humanistic burden of MPS. A comprehensive literature search was conducted in EMBASE, MEDLINE, and eligible conferences were screened to include applicable abstracts.
Of 870 identified articles, 15 studies reported the HRQoL burden of patients with MPS undergoing or with a history of ERT and/or HSCT. These studies include patients of MPS I (n = 2), MPS II (n = 4), MPS IV (n = 6), MPS VI (n = 1), and subtype not mentioned (n = 2). Although the quality-of-life of MPS patients is influenced by time of diagnosis, pain, cognitive involvement, severity of disease, mobility, dependence, and time of treatment initiation, the HRQoL scores of MPS patients across all the scales were below the median reference population scores across all dimensions. This is seen in comparison to healthy participants but also in comparison to patients with other chronic illnesses. The multi-organ involvement, neurological impairment, pain, and morbidity associated with the condition not only affects activity of daily living but also affects social functioning, emotional status, employment status among adults, and school functioning among children.
This systematic literature review revealed the substantial humanistic burden of individuals affected by MPS as well as caregivers. Significant variation in HRQoL scores was observed, however studies indicate that the quality-of-life of MPS patients is influenced primarily by severity of disease (MPS type and phenotype), and then by time of diagnosis, pain, cognitive involvement, mobility, dependence, and time of treatment initiation. Further studies are needed to assess the global humanistic burden of MPS, particularly in MPS III, VI, VII, and IX subtypes, in adults, and for a longer follow-up period. Considering the vast array of HRQoL assessment tools available and used in this study, researchers should also consider using scales with condition-specific measures to ensure appropriate estimates of effectiveness.

The negative effects of loneliness on population health and wellbeing requires interventions that transcend the medical system and leverage social, cultural, and public health system resources. Group-based social interventions are a potential method to alleviate loneliness. Moreover, nature, as part of our social and health infrastructure, may be an important part of the solutions that are needed to address loneliness. The RECETAS European project H2020 (Re-imagining Environments for Connection and Engagement: Testing Actions for Social Prescribing in Natural Spaces) is an international research project aiming to develop and test the effectiveness of nature-based social interventions to reduce loneliness and increase health-related quality of life.
This article describes the three related randomized controlled trials (RCTs) that will be implemented: the RECETAS-BCN Trial in Barcelona (Spain) is targeting people 18+ from low socio-economic urban areas; the RECETAS-PRG Trial in Prague (Czech Republic) is addressing community-dwelling older adults over 60 years of age, and the RECETAS-HLSNK trial is reaching older people in assisted living facilities. Each trial will recruit 316 adults suffering from loneliness at least sometimes and randomize them to nature-based social interventions called \"Friends in Nature\" or to the control group. \"Friends in Nature\" uses modifications of the \"Circle of Friends\" methodology based on group processes of peer support and empowerment but including activities in nature. Participants will be assessed at baseline, at post-intervention (3 months), and at 6- and 12-month follow-up after baseline. Primary outcomes are the health-related quality-of-life according to 15D measure and The De Jong Gierveld 11-item loneliness scale. Secondary outcomes are health and psychosocial variables tailored to the specific target population. Nature exposure will be collected throughout the intervention period. Process evaluation will explore context, implementation, and mechanism of impact. Additionally, health economic evaluations will be performed.
The three RECETAS trials will explore the effectiveness of nature-based social interventions among lonely people from various ages, social, economic, and cultural backgrounds. RECETAS meets the growing need of solid evidence for programs addressing loneliness by harnessing the beneficial impact of nature on enhancing wellbeing and social connections.
Barcelona (Spain) trial: ClinicalTrials.gov, ID: NCT05488496. Registered 29 July 2022. Prague (Czech Republic) trial: ClinicalTrials.gov, ID: NCT05522140. Registered August 25, 2022. Helsinki (Finland) trial: ClinicalTrials.gov, ID: NCT05507684. Registered August 12, 2022.