scholarly journals Estimation of the Quality-of-Life Impact of X-Linked Myotubular Myopathy

2021 ◽  
pp. 1-15
Author(s):  
Andrew Lloyd ◽  
Daniel Aggio ◽  
Ted L. Slocomb ◽  
Jun Lee ◽  
Alan H. Beggs ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trial Data ◽  
Neuromuscular Disorder ◽  
The State ◽  
Health State ◽  
Invasive Ventilation ◽  
Substantial Impact ◽  
Myotubular Myopathy ◽  
Novel Treatments

X-linked myotubular myopathy (XLMTM) is a rare, severe, neuromuscular disorder for which novel treatments are under investigation. This study estimated quality-of-life weights (or utilities) for children with XLMTM. The state that was rated the worst described a child unable to sit and requiring invasive ventilation for≥16 hours a day (utility = –0.07 or –0.27 depending on method used). The state describing a child who can stand and walk and does not require invasive ventilation was the most highly rated state and had a utility of 0.91 or 0.77 (depending on method used). Nine health state vignettes were developed for XLMTM defined in terms of respiratory and motor function based on clinical trial data from parents completing the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND) Domain 1 scale assessing mobility, transfers, sitting, playing, eating, grooming and dressing. These data were supplemented with qualitative data from parent interviews on the daily impact of XLMTM, especially in terms of psychological wellbeing, pain and discomfort, and communication. Seven clinical experts reviewed the draft vignettes for accuracy. Vignettes were rated by members of the UK general public using a time trade-off (TTO) interview and an EQ-5D-5L assessment. This study demonstrated a substantial impact of XLMTM on utility weights.

scholarly journals Estimation of impact of RPE65-mediated inherited retinal disease on quality of life and the potential benefits of gene therapy

2019 ◽  
Vol 103 (11) ◽  
pp. 1610-1614 ◽  
Author(s):  
Andrew Lloyd ◽  
Natalia Piglowska ◽  
Thomas Ciulla ◽  
Sarah Pitluck ◽  
Scott Johnson ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Vision Loss ◽  
Health Utility Index ◽  
Retinal Disease ◽  
Health State ◽  
Health States ◽  
Substantial Impact ◽  
Hand Motion ◽  
Inherited Retinal Disease

Background/aimsIn rare diseases, health-related quality of life (HRQL) data can be difficult to capture. Given the ultrarare nature of RPE65-mediated inherited retinal disease (IRD), it was not feasible to recruit a patient sample and collect HRQL data prospectively. The objectives of this study were to develop health state descriptions of RPE65-mediated IRD, and to estimate associated patient utilities.MethodsVignette descriptions of IRD states were developed and then assessed to elicit utilities. The vignettes ranged from moderate vision loss through to hand motion to no light perception (NLP). Six retina specialists with additional expertise in IRDs provided a proxy valuation of the vignettes using generic measures of health—the 5-level version of EQ-5D-5L and Health Utility Index 3 (HUI3). The data were then scored using standard methods for each instrument.ResultsWeights from both HRQL measures revealed a large decline in scores with vision loss. The EQ-5D-5L weights ranged from 0.709 for moderate vision loss to 0.152 for hand motion to NLP. The HUI3 weights ranged from 0.519 to − 0.039, respectively. A decline was seen on both measures, and the degree of decline from moderate vision loss to NLP was identical on both (−0.56).ConclusionThis is the first study to report HRQL weights (or utilities) for health states describing different levels of vision loss in patients with IRD, specifically those with RPE65-mediated disease. The parallel decline in scores from the EQ-5D and HUI3 corroborates the substantial impact of progressive vision loss on HRQL.

United States Utility Algorithm for the EORTC QLU-C10D, a Multiattribute Utility Instrument Based on a Cancer-Specific Quality-of-Life Instrument

2021 ◽  
pp. 0272989X2110035
Author(s):  
Dennis A. Revicki ◽  
Madeleine T. King ◽  
Rosalie Viney ◽  
A. Simon Pickard ◽  
Rebecca Mercieca-Bebber ◽  
...  
Keyword(s):  
Quality Of Life ◽  
United States ◽  
Emotional Functioning ◽  
Health State ◽  
Life Questionnaire ◽  
Multiattribute Utility ◽  
Value Set ◽  
Eortc Qlq C30 ◽  
Eortc Qlq

Background The EORTC QLU-C10D is a multiattribute utility measure derived from the cancer-specific quality-of-life questionnaire, the EORTC QLQ-C30. The QLU-C10D contains 10 dimensions (physical, role, social and emotional functioning, pain, fatigue, sleep, appetite, nausea, bowel problems). The objective of this study was to develop a United States value set for the QLU-C10D. Methods A US online panel was quota recruited to achieve a representative sample for sex, age (≥18 y), race, and ethnicity. Respondents undertook a discrete choice experiment, each completing 16 choice-pairs, randomly assigned from a total of 960 choice-pairs. Each pair included 2 QLU-C10D health states and duration. Data were analyzed using conditional logistic regression, parameterized to fit the quality-adjusted life-year framework. Utility weights were calculated as the ratio of each dimension-level coefficient to the coefficient for life expectancy. Results A total of 2480 panel members opted in, 2333 (94%) completed at least 1 choice-pair, and 2273 (92%) completed all choice-pairs. Within dimensions, weights were generally monotonic. Physical functioning, role functioning, and pain were associated with the largest utility weights. Cancer-specific dimensions, such as nausea and bowel problems, were associated with moderate utility decrements, as were general issues such as problems with emotional functioning and social functioning. Sleep problems and fatigue were associated with smaller utility decrements. The value of the worst health state was 0.032, which was slightly greater than 0 (equivalent to being dead). Conclusions This study provides the US-specific value set for the QLU-C10D. These estimated health state scores, based on responses to the EORTC QLQ-C30 questionnaire, can be used to evaluate the cost-utility of oncology treatments.

scholarly journals Nutraceutical Screening in a Zebrafish Model of Muscular Dystrophy: Gingerol as a Possible Food Aid

Nutrients ◽  
2021 ◽  
Vol 13 (3) ◽  
pp. 998
Author(s):  
Rosario Licitra ◽  
Maria Marchese ◽  
Letizia Brogi ◽  
Baldassare Fronte ◽  
Letizia Pitto ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Muscular Dystrophy ◽  
Genomic Medicine ◽  
Neuromuscular Diseases ◽  
Dystrophin Gene ◽  
Neuromuscular Disorder ◽  
Heme Oxygenase 1 ◽  
Zebrafish Model ◽  
Screening Study

Duchenne muscular dystrophy (DMD), caused by mutations in the dystrophin gene, is an inherited neuromuscular disorder that causes loss of muscle mass and motor skills. In the era of genomic medicine, there is still no known cure for DMD. In clinical practice, there is a growing awareness of the possible importance of nutrition in neuromuscular diseases. This is mostly the result of patients’ or caregivers’ empirical reports of how active substances derived from food have led to improved muscle strength and, thus, better quality of life. In this report, we investigate several nutraceutical principles in the sapje strain of zebrafish, a validated model of DMD, in order to identify possible natural products that, if supplemented in the diet, might improve the quality of life of DMD patients. Gingerol, a constituent of fresh ginger, statistically increased the locomotion of mutant larvae and upregulated the expression of heme oxygenase 1, a target gene for therapy aimed at improving dystrophic symptoms. Although three other compounds showed a partial positive effect on locomotor and muscle structure phenotypes, our nutraceutical screening study lent preliminary support to the efficacy and safety only of gingerol. Gingerol could easily be proposed as a dietary supplement in DMD.

scholarly journals Prospective cohort study of surgical outcome for spinal metastases in patients aged 70 years or older

2020 ◽  
Vol 102-B (12) ◽  
pp. 1709-1716
Author(s):  
Yutaro Kanda ◽  
Kenichiro Kakutani ◽  
Yoshitada Sakai ◽  
Takashi Yurube ◽  
Shingo Miyazaki ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Performance Status ◽  
Eastern Cooperative Oncology Group ◽  
Spinal Metastases ◽  
Palliative Surgery ◽  
Advanced Age ◽  
Health State ◽  
Survival Times ◽  
The Mean

Aims With recent progress in cancer treatment, the number of advanced-age patients with spinal metastases has been increasing. It is important to clarify the influence of advanced age on outcomes following surgery for spinal metastases, especially with a focus on subjective health state values. Methods We prospectively analyzed 101 patients with spinal metastases who underwent palliative surgery from 2013 to 2016. These patients were divided into two groups based on age (< 70 years and ≥ 70 years). The Eastern Cooperative Oncology Group (ECOG) performance status (PS), Barthel index (BI), and EuroQol-5 dimension (EQ-5D) score were assessed at study enrolment and at one, three, and six months after surgery. The survival times and complications were also collected. Results In total, 65 patients were aged < 70 years (mean 59.6 years; 32 to 69) and 36 patients were aged ≥ 70 years (mean 75.9 years; 70 to 90). In both groups, the PS improved from PS3 to PS1 by spine surgery, the mean BI improved from < 60 to > 80 points, and the mean EQ-5D score improved from 0.0 to > 0.7 points. However, no significant differences were found in the improvement rates and values of the PS, BI, and EQ-5D score at any time points between the two groups. The PS, BI, and EQ-5D score improved throughout the follow-up period in approximately 90% of patients in each group. However, the improved PS, BI, and EQ-5D scores subsequently deteriorated in some patients, and the redeterioration rate of the EQ-5D was significantly higher in patients aged ≥ 70 than < 70 years (p = 0.027). Conclusion Palliative surgery for spinal metastases improved the PS, activities of daily living, and quality of life, regardless of age. However, clinicians should be aware of the higher risk of redeterioration of the quality of life in advanced-age patients. Cite this article: Bone Joint J 2020;102-B(12):1709–1716.

Changes in weight and weight-related quality of life in a multicentre, randomized trial of aripiprazole versus standard of care

2008 ◽  
Vol 23 (8) ◽  
pp. 561-566 ◽  
Author(s):  
Ronette L. Kolotkin ◽  
Patricia K. Corey-Lisle ◽  
Ross D. Crosby ◽  
Hong J. Kan ◽  
Robert D. McQuade
Keyword(s):  
Quality Of Life ◽  
Clinical Symptoms ◽  
Clinical Trial Data ◽  
Standard Of Care ◽  
Random Regression ◽  
Sexual Life ◽  
Baseline Body Mass Index ◽  
Meaningful Change ◽  
Related Quality

AbstractBackgroundThis is a secondary analysis of clinical trial data collected in 12 European countries. We examined changes in weight and weight-related quality of life among community patients with schizophrenia treated with aripiprazole (ARI) versus standard of care (SOC), consisting of other marketed atypical antipsychotics (olanzapine, quetiapine, and risperidone).MethodFive-hundred and fifty-five patients whose clinical symptoms were not optimally controlled and/or experienced tolerability problems with current medication were randomized to ARI (10–30 mg/day) or SOC. Weight and weight-related quality of life (using the IWQOL-Lite) were assessed at baseline, and weeks 8, 18 and 26. Random regression analysis across all time points using all available data was used to compare groups on changes in weight and IWQOL-Lite. Meaningful change from baseline was also assessed.ResultsParticipants were 59.7% male, with a mean age of 38.5 years (SD 10.9) and mean baseline body mass index of 27.2 (SD 5.1). ARI participants lost an average of 1.7% of baseline weight in comparison to a gain of 2.1% by SOC participants (p < 0.0001) at 26 weeks. ARI participants experienced significantly greater increases in physical function, self-esteem, sexual life, and IWQOL-Lite total score. At 26 weeks, 20.7% of ARI participants experienced meaningful improvements in IWQOL-Lite score, versus 13.5% of SOC participants. A clinically meaningful change in weight was also associated with a meaningful change in quality of life (p < 0.001). A potential limitation of this study was its funding by a pharmaceutical company.ConclusionsCompared to standard of care, patients with schizophrenia treated with aripiprazole experienced decreased weight and improved weight-related quality of life over 26 weeks. These changes were both statistically and clinically significant.

Analysis of public safety in European countries

2015 ◽  
Vol 60 (8) ◽  
pp. 66-80
Author(s):  
Anna Wierzbicka ◽  
Agata Żółtaszek
Keyword(s):  
Quality Of Life ◽  
Public Safety ◽  
The State ◽  
Point Of View ◽  
European Countries ◽  
Effective Strategy ◽  
Sense Of Security ◽  
The Subject

Maintaining security is one of public tasks that determine the quality of life of the population. This issue is the subject of much debate both social and political. An in-depth assessment of the situation requires a variety of analyzes, significant from the point of view of the implementation of appropriate, effective strategy to increase the sense of security among citizens. The aim of the paper is to compare the state of public safety in selected European countries. The study was conducted based on Eurostat data from the years 2005–2011.

HOUSING AFFORDABILITY IN THE STATE OF MELAKA

2017 ◽  
Vol 15 ◽  
Author(s):  
Mariana Mohamed Osman ◽  
Noor Suzilawati Rabe ◽  
Muhammad Faris Abdullah ◽  
Nur Farhanah Rosli ◽  
Farah Eleena Zainudin
Keyword(s):  
Quality Of Life ◽  
Secondary Data ◽  
Housing Affordability ◽  
The State ◽  
Improve Quality ◽  
Governmental Agencies ◽  
Income Ratio ◽  
Basic Need

Housing is a basic need to man. It provides shelter and comfort from the elements and improve quality of life of the residents. Hence, housing should be affordable to all. This paper assesses the housing affordability for the districts in Melaka. This was achieved by calculating the median multiple of the price income ratio and comparing the score to the housing affordability index. Secondary data were obtained from reports published by the governmental agencies. The results show that housing is mostly moderately unaffordable in the districts of Melaka. Additionally, housing affordability has improved from 2012 to 2014 in majority of the districts.

scholarly journals La Repercusión en la calidad de vida, salud y práctica de actividad física del confinamiento por Covid-19 en España (Effects on quality of life, health and practice of physical activity of Covid-19 confinement in Spain)

Retos ◽  
2021 ◽  
Vol 42 ◽  
pp. 684-695
Author(s):  
Marta García-Tascón ◽  
Cristina Mendaña-Cuervo ◽  
César Sahelices-Pinto ◽  
Ana-Mª Magaz-González
Keyword(s):  
Quality Of Life ◽  
Physical Activity ◽  
Paradigm Shift ◽  
Ad Hoc ◽  
The State ◽  
Muscular Fitness ◽  
Innovative Strategies ◽  
Calidad De Vida ◽  
Total Percentage

En España, la declaración del estado de alarma por la pandemia Covid-19, supuso que entre el 14 de marzo y el 2 de mayo de 2020, 47 millones de habitantes estuvieran confinados y sólo pudieran salir a cubrir necesidades básicas. Ante este hecho es oportuno analizar su impacto sobre la salud, tomando en consideración cómo afectó a la práctica de la actividad física. Se administró un cuestionario ad hoc. Los participantes fueron 1046 personas (hombre 48.57% y mujer 51.43%) y con una edad media de 40 (± DT 13.35 años). Respecto a la práctica de actividad física, destaca una significativa disminución de intensidad y cantidad (p < .001), y una modificación del tipo de actividad física realizada, pasando de deportes cardiovasculares y de aptitud muscular a prácticas de gimnasias suaves y flexibilidad. Aumentó el porcentaje total de personas que no realizaron nada de actividad física. Aun disponiendo de más tiempo libre, las personas en situación de ERTE, disminuyeron significativamente su cantidad de actividad física realizada (p < .001). Estos resultados permiten concluir que el confinamiento supuso una modificación de hábitos de práctica de actividad física, una disminución de los valores recomendados como saludables, lo que conllevó implicaciones sobre la salud y la calidad de vida. Se ofrece información que ayudará a entender estos nuevos comportamientos y que facilitará a los gestores estrategias innovadoras para activar la vuelta a niveles anteriores de actividad física, su incremento y, en definitiva, la reactivación del sector deportivo.  Abstract: In Spain, the declaration of the state of alarm due to the Covid-19 pandemic, meant that between 14th of March and 2nd of May 2020, 47 million inhabitants were confined at home and could only go out to cover basic needs. This unprecedented event has led to a paradigm shift, and it is appropriate to analyze its impact on health, considering how it has affected the practice of physical activity. An ad hoc questionnaire was administered. The participants were 1046 people (48.57% male and 51.43% female) and the average age of 40 (± DT 13.35 years). Regarding the practice of physical activity, a significant decrease in intensity and quantity stands out (p < .001), and a modification of the type of physical activity performed, going from cardiovascular sports and muscular fitness to soft gymnastics and flexibility practices. The total percentage of people who did no physical activity at all, increased. In addition, although more free time was available, people in ERTE situation significantly decreased their amount of physical activity performed (p < .001). These results allow us to conclude that confinement has meant a modification of physical activity practice habits and a decrease in the recommended values ​​of healthy healthy, and therefore has had implications on health and quality of life. This information will help to understand these new behaviors in order for managers to implement innovative strategies to reactivate the return to previous levels of physical activity, its increase and, in short, the reactivation of the sports sector.

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