Health-Related Quality of Life

健康相关生活质量
  • 文章类型: Journal Article
    UNASSIGNED: Older adults with multimorbidity experience impaired health-related quality of life and treatment burden. Yoga has the potential to improve several aspects of health and well-being. The British Wheel of Yoga\'s Gentle Years Yoga© programme was developed specifically for older adults, including those with chronic conditions. A pilot trial demonstrated feasibility of using Gentle Years Yoga in this population, but there was limited evidence of its effectiveness and cost-effectiveness.
    UNASSIGNED: To determine the effectiveness and cost-effectiveness of the Gentle Years Yoga programme in addition to usual care versus usual care alone in older adults with multimorbidity.
    UNASSIGNED: Pragmatic, multisite, individually randomised controlled trial with embedded economic and process evaluations.
    UNASSIGNED: Participants were recruited from 15 general practices in England and Wales from July 2019 with final follow-up in October 2022.
    UNASSIGNED: Community-dwelling adults aged 65 years and over with multimorbidity, defined as two or more chronic health conditions from a predefined list.
    UNASSIGNED: All participants continued with any usual care provided by primary, secondary, community and social services. The intervention group was offered a 12-week programme of Gentle Years Yoga.
    UNASSIGNED: The primary outcome and end point were health-related quality of life measured using the EuroQol-5 Dimensions, five-level version utility index score over 12 months. Secondary outcomes were health-related quality of life, depression, anxiety, loneliness, incidence of falls, adverse events and healthcare resource use.
    UNASSIGNED: The mean age of the 454 randomised participants was 73.5 years; 60.6% were female, and participants had a median of three chronic conditions. The primary analysis included 422 participants (intervention, n = 227 of 240, 94.6%; usual care, n = 195 of 214, 91.1%). There was no statistically or clinically significant difference in the EuroQol-5 Dimensions, five-level version utility index score over 12 months: the predicted mean score for the intervention group was 0.729 (95% confidence interval 0.712 to 0.747) and for usual care it was 0.710 [95% confidence interval (CI) 0.691 to 0.729], with an adjusted mean difference of 0.020 favouring intervention (95% CI -0.006 to 0.045, p = 0.14). No statistically significant differences were observed in secondary outcomes, except for the pain items of the Patient-Reported Outcomes Measurement Information System-29. No serious, related adverse events were reported. The intervention cost £80.85 more per participant (95% CI £76.73 to £84.97) than usual care, generated an additional 0.0178 quality-adjusted life-years per participant (95% CI 0.0175 to 0.0180) and had a 79% probability of being cost-effective at the National Institute for Health and Care Excellence threshold of £20,000 per quality-adjusted life-year gained. The intervention was acceptable to participants, with seven courses delivered face to face and 12 online.
    UNASSIGNED: Self-reported outcome data raise the potential for bias in an unblinded trial. The COVID-19 pandemic affected recruitment, follow-up and the mode of intervention delivery.
    UNASSIGNED: Although the Gentle Years Yoga programme was not associated with any statistically significant benefits in terms of health-related quality of life, mental health, loneliness or falls, the intervention was safe, acceptable to most participants and highly valued by some. The economic evaluation suggests that the intervention could be cost-effective.
    UNASSIGNED: Longer-term cost-effectiveness modelling and identifying subgroups of people who are most likely to benefit from this type of intervention.
    UNASSIGNED: This trial is registered as ISRCTN13567538.
    UNASSIGNED: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/94/36) and is published in full in Health Technology Assessment; Vol. 28, No. 53. See the NIHR Funding and Awards website for further award information.
    It is common for older adults to have two or more long-term health conditions. These conditions affect quality of life differently, with some people feeling well and others needing healthcare support. The Gentle Years Yoga programme was developed to improve quality of life for older adults, including those with long-term health conditions. We wanted to see how well the programme worked and if it offered good value for money for the NHS. We tested whether offering a 12-week course of Gentle Years Yoga improved the quality of life and reduced anxiety, depression, loneliness and falls for people aged 65 years and over who had two or more long-term health conditions. We recruited 454 people through general practices across England and Wales, with 240 people selected at random to be invited to take part in the Gentle Years Yoga programme and the other 214 to continue with their usual care and not be offered Gentle Years Yoga. The average age of participants was 74 years, nearly two-thirds were female and the number of long-term health conditions participants had ranged from two to nine (average was three). They completed four questionnaires over a 12-month period. We also interviewed some of the participants and the yoga teachers to find out how the approach worked in practice. The yoga was delivered either face to face or online. We did not find any significant benefits in terms of quality of life, anxiety, depression, loneliness or falls. At interview, some yoga participants noted no or a modest impact on their health or lifestyle, while others described Gentle Years Yoga as transformative, having substantial impacts and improvements on their physical health and emotional well-being. Because running the yoga classes was relatively inexpensive and some insignificant benefits were seen, the Gentle Years Yoga programme may be good value for money.
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  • 文章类型: Journal Article
    UNASSIGNED: Although the negative impact of smoking and health-related quality of life (HRQoL) on depression has been confirmed in various studies, There has been little exploration of how HRQoL mediates the relationship between smoking and depression. The purpose of the current study was to examine the relationship between smoking and depression in the Chinese current smokers with nicotine dependence and the mediating role of HRQoL.
    UNASSIGNED: A cross-sectional study named \"Psychology and Behavior Investigation of Chinese Residents\" was conducted from July 10 to September 15, 2021 in China. Nicotine dependence, HRQoL and depression were measured by Fagerstrom Test for Nicotine Dependence (FTND), the European Five Dimensional Five Level Health scale (EQ-5D-5L) and the 9-item Patient Health Questionnaire (PHQ-9) respectively. Information on age, gender, place of residence, household registration, education level, marital status, employment status, average family monthly income, drinking frequency, living status, BMI, multiple chronic conditions were also collected. Pearson\'s correlation test and logistic regression analysis were conducted to explore the association between nicotine dependence, HRQoL and depression and a mediation analysis was applied to explore the mediating effect of the HRQoL on this relationship.
    UNASSIGNED: A total of 1,381 current smokers were included in the study. The participants showed a moderate level of nicotine dependence with a mean of 1.36(SD=1.50), a relatively high level of HRQoL scores (Mean=0.94, SD=0.13), and a depression score with a mean of 6.48(SD=6.09). Approximately 22.74% (314/1,381) of the participants were considered to indicate depression. In the univariable regression model, it was found that nicotine dependence was positively associated with depression (OR:1.094, 95%CI: 1.008-1.187), while HRQoL was negatively associated with depression (OR:0.011, 95%CI: 0.004-0.033). In the multivariable regression model, HRQoL was still notably associated with depression (OR:0.008, 95%CI: 0.002-0.027), however, the positive association was not observed between nicotine dependence and depression. The Pearson\'s correlation test demonstrated that nicotine dependence was negatively correlated with HRQoL(rs= -0.147, P<0.001) and HRQoL was negatively correlated with depression(rs= -0.275, P<0.001). In contrast, nicotine dependence was positively correlated with depression(rs= 0.136, P<0.001). Mediation analysis found that HRQoL moderated the relationship between nicotine dependence and depression with a mediating effect of 26.49%.
    UNASSIGNED: The findings support that nicotine dependence is positively associated with depression and HRQoL is negatively associated with depression in current smokers. HRQoL mediated the relationship between nicotine dependence and depression. The well-established imperative interventions aimed at promoting smoking cessation and improving quality of life may benefit for alleviation of depression in current smokers.
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  • 文章类型: Journal Article
    BACKGROUND: The psychosocial impact of medical marijuana use is not yet known. This study evaluated short-term changes in health-related quality of life (HRQoL) over the first three months of medical marijuana use.
    METHODS: This prospective, observational, longitudinal study followed adults newly recommended for medical marijuana by a physician for any of the more than 20 qualifying medical conditions in Pennsylvania. Participants (N = 438) provided their clinical status and demographic information, and completed semi-structured interviews prior to medical marijuana initiation (baseline) and at three months. HRQoL was assessed by the Short Form-36 (SF-36). Paired-samples t-tests evaluated changes in HRQoL over time.
    RESULTS: Participants (M age = 46.4 years [15.6]; 66.4% female) were mostly commonly referred for medical marijuana to treat anxiety disorders (61.9%) or severe chronic or intractable pain (53.6%). Participants reported rapid and significant improvements in all of the domains of HRQoL from baseline to three months after initiating medical marijuana use (physical functioning, role limitations due to physical health problems, emotional well-being, role limitations due to emotional problems, bodily pain, social functioning, energy/fatigue and general health, P < .001 for all). Age was negatively predictive of level of improvement over time for the physical functioning (P < .0001), role limitations due to physical health problems (P < .001), and pain (P < .0001) domains after controlling for baseline, with older participants displaying less improvement than younger participants.
    CONCLUSIONS: Gains were observed in all HRQoL domains assessed after three months of medical marijuana use. In several domains, age was a significant predictor of degree of improvement.
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  • 文章类型: Journal Article
    目的:意识到作为腹主动脉瘤患者的一级亲属发生腹主动脉瘤的风险增加的心理后果迄今尚未被研究。这项研究调查了与对照组相比,腹主动脉瘤患者的男性和女性成年后代对遗传性和焦虑的认识。将主动脉病理参与者与正常主动脉直径参与者的健康相关生活质量进行比较。
    方法:这是一项横断面患病率研究,基于在一级亲属中检测腹主动脉瘤试验中检查的参与者(DAAAD;752成年后代,756个匹配的控件),2020-2022年。在主动脉超声检查之前,收集了有关健康相关生活质量的问卷调查以及有关遗传度意识的特定研究问题。
    结果:与对照组相比,有遗传的个体的出勤率更高(67%vs.52%,p<0.001)。在检查的1508个成年后代中,65%的人报告与腹主动脉瘤有近亲(对照组为6%)。成年雌性后代报告的遗传力意识高于对照组(38%vs.12%,p<0.001),男性也是如此(32%vs.8%,p<0.001)。略大多数有意识的参与者报告了焦虑(54%的女性后代;51%的男性)。当使用标准的健康相关生活质量工具时,两组之间的健康相关生活质量没有测量差异。
    结论:具有遗传性意识和对此类风险的焦虑的成年后代比例高于预期,这表明我们未能通过医疗保健系统内当前的信息渠道适当地向该群体传达风险。这需要制定专门的策略,以改善腹主动脉瘤风险与患者及其近亲的沟通。
    OBJECTIVE: The psychological consequences of being aware of an increased risk of developing abdominal aortic aneurysm as a first-degree relative of a person with abdominal aortic aneurysm are hitherto unexplored. This study investigates the awareness of heritability and anxiety in male and female adult offspring of abdominal aortic aneurysm patients compared to controls. Health-related quality of life among participants with aortic pathology was compared to participants with normal aortic diameters.
    METHODS: This was a cross-sectional point prevalence study based on the participants examined in the Detecting Abdominal Aortic Aneurysm in First Degree Relatives Trial (DAAAD; 752 adult offspring, 756 matched controls), 2020-2022. Questionnaires about health-related quality of life and study-specific questions regarding awareness of heritability were collected prior to the aortic ultrasound.
    RESULTS: Attendance rate was higher among individuals with heredity compared to controls (67% vs. 52%, p < 0.001). Of 1508 adult offspring examined, 65% reported having a close relative with abdominal aortic aneurysm (6% in controls). Female adult offspring reported higher awareness of heritability than controls (38% vs. 12%, p < 0.001), as did males (32% vs. 8%, p < 0.001). A slight majority of participants with awareness reported anxiety (54% of female offspring; 51% of male). There were no measured differences in health-related quality of life between the groups when standard health-related quality of life instruments were used.
    CONCLUSIONS: The higher-than-expected proportion of adult offspring with awareness of heritability and anxiety about such risk indicates that we fail to communicate risk to this group appropriately via the current channels of information within the healthcare system. This calls for the development of dedicated strategies for improved communication of abdominal aortic aneurysm risk to patients and their next of kin.
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  • 文章类型: Journal Article
    改善高血压个体的健康相关生活质量(HRQoL)已成为一个重要的公共卫生问题。然而,目前的研究忽视了高血压患者感知社会支持(PSS)的个体异质性。延迟贴现(DD)的潜在机制,独自生活,关于HRQoL的PSS仍不清楚,需要进一步探索。
    这项研究旨在确定高血压患者中的PSS概况,并检验以下假设:DD介导PSS和HRQoL之间的关系,并且这种介导过程通过单独生活在高血压患者中而受到调节。
    在江苏进行了一项横断面研究,中国。总的来说,1815名高血压患者完成了社会人口统计学和HRQoL问卷,PSS量表,和DD任务。数据分析包括潜在的轮廓分析,χ2检验,Spearman相关分析,和过程宏进行回归分析。
    确定了四个潜在的PSS配置文件:最低(3.2%),中低(26.6%),中高(42.4%),最高(27.8%)。DD介导了PSS与HRQoL之间的关联。这种调解过程的前半部分是通过独居来缓和的。
    我们的研究结果表明,PSS,DD,独居对高血压患者的HRQoL有显著影响。医疗保健专业人员应考虑高血压患者中PSS的变化,并实施干预措施以通过增强PSS来降低DD。以提高这一人群的HRQoL。
    UNASSIGNED: Improving health-related quality of life (HRQoL) among hypertensive individuals has emerged as a significant public health issue. However, current research has ignored the individual heterogeneity of perceived social support (PSS) among hypertensive patients. The potential mechanism of delay discounting (DD), living alone, and PSS on HRQoL remains unclear, and further exploration is required.
    UNASSIGNED: This study aimed to ascertain PSS profiles among hypertensive patients and examine the hypotheses that DD mediates the relationship between PSS and HRQoL and that this mediating process is moderated by living alone in hypertensive patients.
    UNASSIGNED: A cross-sectional study was carried out in Jiangsu, China. In total, 1815 hypertensive patients completed socio-demographic and HRQoL questionnaires, a PSS scale, and a DD task. Data analyses included a latent profile analysis, χ2-test, Spearman correlation analysis, and PROCESS macro for regression analysis.
    UNASSIGNED: Four potential PSS profiles were identified: lowest (3.2%), moderate-low (26.6%), moderate-high (42.4%), and highest (27.8%). DD mediated the association between PSS and HRQoL. The first half of this mediating process was moderated by living alone.
    UNASSIGNED: Our findings indicated that PSS, DD, and living alone significantly influence the HRQoL of individuals with hypertension. Healthcare professionals should consider variations in PSS among hypertensive patients and implement interventions to reduce DD by enhancing PSS, in order to improve the HRQoL of this population.
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  • 文章类型: Journal Article
    背景:EQ-5D是获取与健康相关的生活质量(HRQoL)的推荐指标,认可用于卫生技术评估机构。为了评估是否适合使用EQ-5D来决定囊性纤维化(CF)治疗的成本效用,这项研究评估了EQ-5D-5L在患有CF的成人和青少年中的表现。
    方法:这是一项横断面观察性调查研究,研究对象是在一个大型CF中心就诊的CF患者。参与者被要求完成一项调查,其中包括两个HRQoL测量;EQ-5D-5L和CF生活质量(CFQoL)问卷。
    结果:在213名参与者中,EQ-5D-5L指数的中位数评分为0.76(IQR0.66~0.84),视觉模拟(EQ-VAS)评分为70(60~80).EQ-5D指数和EQ-VAS均根据肺功能区分疾病严重程度(分别为p=0.01和p<0.01)和肺加重(分别为p=0.02和p<0.01);然而,与EQ-5D指数相比,EQ-VAS在肺功能严重程度更高的组之间进行了区分。EQ-5D-5L证明了其维度的收敛有效性,指数得分,EQ-VAS与大多数CFQoL域有显著相关性。不过,与EQ-5D指数(仅1个域)相比,EQ-VAS显著预测更多CFQoL域(4个域)。
    结论:通用EQ-5D-5L在区分CF疾病严重程度方面表现良好,指数评分和EQ-VAS与CFQoL呈中等相关性。然而,与EQ-5D-5L一起使用互补的特定条件措施可以更好地了解CF中的HRQoL,并有利于成本效用分析过程。
    BACKGROUND: The EQ-5D is the recommended measure to capture health-related quality of life (HRQoL), recognised for use in health technology appraisal bodies. In order to assess whether it is appropriate to use the EQ-5D for making decisions about the cost-utility of treatments in cystic fibrosis (CF), this study assesses the performance of the EQ-5D-5L in adults and adolescents with CF.
    METHODS: This was a cross-sectional observational survey study of patients with CF attending a single large CF centre. Participants were asked to complete a survey that included two HRQoL measures; the EQ-5D-5L and CF Quality of Life (CFQoL) questionnaires.
    RESULTS: Among 213 participants, the median EQ-5D-5L index score was 0.76 (IQR 0.66 - 0.84) and the visual analogue (EQ-VAS) was 70 (60 - 80). Both the EQ-5D index and EQ-VAS discriminated between disease severity based on lung function (p = 0.01 and p < 0.01, respectively) and pulmonary exacerbation (p = 0.02 and p < 0.01, respectively); however, EQ-VAS differentiated between more lung function severity groups compared to EQ-5D index. The EQ-5D-5L demonstrated convergent validity as its dimensions, index score, and EQ-VAS had significant correlations with most CFQoL domains. Though, EQ-VAS significantly predicted more domains of CFQoL (4 domains) compared to EQ-5D index (only 1 domain).
    CONCLUSIONS: The generic EQ-5D-5L performed adequately in discriminating between CF disease severity, and its index score and EQ-VAS had moderate correlations with CFQoL. However, using a complementary condition-specific measure alongside the EQ-5D-5L can provide better insight of HRQoL in CF and benefit the process of cost-utility analysis.
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  • 文章类型: Journal Article
    目标:供养老年人的专业寄养家庭可以替代疗养院。参与家庭生活并与单个参考人密切接触可以提高他们的生活质量(QOL)。这项研究旨在比较寄养家庭与疗养院老年人的健康相关生活质量(HrQOL)和主观生活质量。
    方法:在寄养家庭(KASAF研究)和疗养院(KASEHPAD研究)进行的双生子研究的横断面分析。
    方法:法属加勒比群岛的老年人(60岁或以上)住在寄养家庭或疗养院。
    方法:使用EuroQol-五维(EQ5D-3L)测量HrQOL,并使用视觉模拟量表(QOL-VAS)评估QOL。对于无法完成这些量表的老年人,代理EQ-5D-3L评估由辅助医务人员或寄养护理人员进行.
    结果:共有439名老年人,其中包括107个寄养家庭和332个疗养院。居住在寄养家庭的参与者很少是男性,很少有高血压,依赖性更强或身体受损,认知评分较低。在多变量分析中,与自我报告的低HRQoL(n=240)相关的因素是简易精神状态检查(MMSE)评分(β:-0.011;p=0.003)和日常生活活动能力(ADL)评分(β:0.014;p<0.001)。较低的QOL-VAS评分(n=150)与生活在寄养家庭(β:-19.48分;p<0.001)和ADL评分(2.94分;p=0.019)有关。在患有严重认知障碍的老年人中,与低代理EQ-5D代理指数得分(n=136)相关的唯一因素是依赖性(β:0.167;p<0.001)。
    结论:生活在养老院和寄养家庭中的老年人的生活质量相似。此外,居住在寄养家庭时,老年人的主观生活质量较好。这些发现表明,寄养家庭模式可以满足不适合疗养院的依赖老年人的社会和环境需求。
    OBJECTIVE: Professional foster families for dependent older adults could be an alternative to nursing homes. Engagement in the family life and close contact with a single reference person could enhance their quality of life (QOL). This study aimed to compare the Health-Related Quality of Life (HrQOL) and subjective QOL among older adults living in foster families versus those in nursing homes.
    METHODS: Cross-sectional analysis from twin studies conducted in foster families (the KASAF study) and nursing homes (the KASEHPAD study).
    METHODS: Older adults (aged 60 years or older) in French Caribbean Islands living in foster families or nursing homes.
    METHODS: HrQOL was measured using the EuroQol-five dimensions (EQ5D-3L) and QOL was assessed using a Visual Analog Scale (QOL-VAS). For older adults unable to complete these scales, proxy EQ-5D-3L assessments were conducted by paramedical staff or foster caregivers.
    RESULTS: A total of 439 older adults, with 107 in foster families and 332 in nursing homes were included. Participants living in foster families were less often male, had less often hypertension, were more dependent or physical impaired and had lower score of cognition. In multivariate analyses, factors associated with low self-reported HRQoL (n = 240) were Mini Mental State Examination (MMSE) score (β: -0.011; p = 0.003) and Activities of Daily Living (ADL) score (β: 0.014; p < 0.001). A lower QOL-VAS score (n = 150) was associated with living in a nursing home compared to living in a foster family (β: -19.48 points; p < 0.001) and with the ADL score (2.94 points; p = 0.019). In older adults with major cognitive disorders, the only factor associated with low proxy EQ-5D proxy index score (n = 136) was dependency (β: 0.167; p < 0.001).
    CONCLUSIONS: HrQOL was similar between older adults living in nursing homes and foster families. Additionally, older adults reported a better subjective quality of life when residing in foster families. These findings suggest that the foster family model may meet the social and environmental needs of dependent older adults for whom nursing homes are not suitable.
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  • 文章类型: Journal Article
    目的:本研究旨在开发妊娠生活质量量表(PREG-QOL),作为评估妊娠期间生活质量并测试其心理测量特性的新工具。
    方法:对内容和结构效度进行仪器开发研究和心理测量测试,因素结构和可靠性。
    方法:该研究分三个阶段进行:(1)创建项目池,(2)项目的初步评估,(3)细化量表并评估心理测量学特性。仪器开发指南用于评估内容效度,构造效度,随着时间的推移,仪器的内部一致性和稳定性。在2021年4月至8月之间收集了评估PREG-QOL心理测量特性的数据。
    结果:项目是通过对孕妇的深入访谈和广泛的文献综述得出的。量表内容效度指数为0.98。探索性因素分析揭示了26项包含6个因素的工具,这解释了%56.2的差异。验证性因素分析(CFA)表明,因素3和5应合并到物理领域的因素中,因为它们包括有关相同主题的项目。CFA获得的拟合指数处于足够的水平。采用平行测试方法来评估PREG-QOL与SF-36的相关性。结果表明,随着时间的推移,PREG-QOL具有较高的内部不一致性和稳定性。
    结论:就其心理测量特征而言,PREG-QOL是一种有效且可靠的工具。26项工具由总体满意度感知的五个因素组成,情感领域,物理域,健康支持系统和社会领域。
    结论:显示良好的心理测量特性,PREG-QOL可用于评估妊娠期生活质量的多个维度.
    OBJECTIVE: This study aims to develop the Quality of Life in Pregnancy Scale (PREG-QOL) as a new instrument to evaluate the quality of life during pregnancy and test its psychometric properties.
    METHODS: An instrument development study and psychometric testing of the content and construct validity, factor structure and reliability.
    METHODS: The study was conducted in three stages: (1) creating an item pool, (2) preliminary evaluation of items, and (3) refining the scale and evaluating psychometric properties. Instrument development guidelines were used to evaluate content validity, construct validity, internal consistency and stability of the instrument over time. Data to evaluate psychometric properties of the PREG-QOL were collected between April and August 2021.
    RESULTS: Items were developed using in-depth interviews with pregnant women and extensive literature review. Scale-content validity index was 0.98. Exploratory factor analysis revealed a 26-item instrument with 6 factors, which explained % 56.2 of variance. Confirmatory factor analysis (CFA) showed that factors 3 and 5 should be combined into the factor of physical domain since they included items about the same theme. Fit indices obtained by CFA were at sufficient levels. Parallel test method was employed to evaluate the correlation of the PREG-QOL with the SF-36. The findings indicated that the PREG-QOL had high internal inconsistency and stability over time.
    CONCLUSIONS: The PREG-QOL is a valid and reliable instrument in terms of its psychometric characteristics. The 26-item instrument was composed of the five factors of perception of general satisfaction, emotional domain, physical domain, health support systems and social domain.
    CONCLUSIONS: Displaying good psychometric properties, the PREG-QOL may be used to evaluate multiple dimensions of the quality of life during pregnancy.
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  • 文章类型: Journal Article
    背景:粘多糖贮积症(MPS)患者通常面临延迟诊断,有限的治疗选择和高昂的医疗费用,这可能会显著影响患者的生活质量。这项研究的目的是了解与诊断和治疗相关的医疗服务利用情况,诊断期间的经济负担,中国MPS患者的健康相关生活质量。
    方法:从2019年5月至7月招募了一系列在国家患者组织注册的诊断为MPS的患者进行横断面调查。通过电话采访从患者或其父母那里收集信息,包括人口统计数据,利用与诊断和治疗有关的服务,MPS诊断期间的总费用和健康相关生活质量(HRQoL)。根据MPS患者的年龄,通过PedsQL4.0通用核心量表(PedsQL)和36项简短健康调查(SF-36)评估HRQoL,并与中国普通人群进行比较。
    结果:总共180名MPS患者(I型为50、67、15、46、1和1,II,III,IV,VI和VII),平均年龄9.54岁,男性137人(76.11%),包括在分析中。因MPS相关症状首次就诊医生的平均年龄为3.65±2.58岁,而首次就诊时确诊的患者只有12例(6.67%)。平均诊断延迟,这被定义为从第一次去看医生的MPS相关症状到最终诊断之间的时间,是9.42个月,类型之间没有显著差异。平均误诊为4.56例。在确诊之前,患者平均访问6.31次,访问了4.3家医院。诊断期间,平均81,086.72日元的直接医疗费用占总费用的63.75%。只有32.78%的患者曾经接受过特定的治疗。患者的PedsQL和SF-36平均得分明显低于中国标准。家庭人均年收入,特定治疗的使用和MPS亚型与患者的HRQoL显著相关.
    结论:结果突出了MPS患者在诊断方面面临的挑战,获得特定治疗,经济负担和较低的HRQoL。迫切需要改进早期发现和诊断,建立公平和一致的机制,以增加获得专门治疗的机会,减轻中国MPS患者的经济负担。
    BACKGROUND: Patients with mucopolysaccharidosis (MPS) often face delayed diagnoses, limited treatment options and high healthcare costs, that may significantly affect patients\' quality of life. The objective of this study was to understand medical service utilization related to diagnosis and treatment, economic burden during diagnosis period, and health-related quality of life among MPS patients in China.
    METHODS: A series of patients diagnosed with MPS registered in the national patient organization were recruited for a cross-sectional survey from May to July 2019. Information were collected from patients or their parents via phone interview, including demographic data, utilization of services related to diagnosis and treatment, total cost during the period of MPS diagnosis and health-related quality of life (HRQoL). HRQoL was assessed by PedsQL 4.0 Generic Core Scale (PedsQL) and 36-item short-form health survey (SF-36) depending on the age of patients with MPS and compared with the general Chinese population.
    RESULTS: A total of 180 MPS patients (50, 67, 15, 46, 1 and 1 for type I, II, III, IV, VI and VII), with a mean age of 9.54 years and 137 (76.11%) males, were included in analysis. The mean age at first visit to a medical doctor for MPS related symptoms was 3.65 ± 2.58 years old, while only 12 patients (6.67%) were diagnosed on their first visit. The mean diagnostic delay, which is defined as the time between the first visit to a medical doctor for MPS related symptoms and the final diagnosis, was 9.42 months, with no significant difference between types. The average number of misdiagnosis was 4.56. Before the confirmed diagnosis, the patients made an average of 6.31 visits and visited 4.3 hospitals. During diagnosis period, the mean of ¥81,086.72 direct medical costs accounted for 63.75% of the total cost. Only 32.78% of the patients had ever received specific treatments. The mean scores of PedsQL and SF-36 of patients were significantly lower than the Chinese norms. Household annual income per person, specific treatment use and MPS subtype were significantly associated HRQoL of patients.
    CONCLUSIONS: The results highlight challenges faced by MPS patients in terms of diagnosis, access to specific treatments, economic burden and low HRQoL. There is an urgent need to improve early detection and diagnosis, create fair and consistent mechanisms to increase access to specialized treatment and reduce the economic burden of MPS patients in China.
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  • 文章类型: Journal Article
    背景:N-甲基-D-天冬氨酸受体(NMDAR)脑炎是一种罕见的神经系统自身免疫性疾病,在急性期具有严重的神经精神症状。尽管神经功能恢复良好,大多数患者继续经历认知,精神病学,心理,和急性期后数年的社会损害。然而,这些长期后果的确切性质和随时间的演变模式仍不清楚,它们对主要是年轻患者的福祉和生活质量的影响尚未得到彻底检查。
    方法:SAPIENCE是一项欧洲多中心(n=3)前瞻性观察性队列研究,研究长期认知,精神病学,心理,NMDAR脑炎患者的社会结果。该研究由三个相互关联的层次组成。1级包括与患者及其护理人员的定性访谈和焦点小组。第2级由面试的浓缩形式组成,标准化问卷,对病人进行详细的神经心理学检查.第3级涉及一项在线调查,该调查将向全球患者开放,并探讨患者报告的结果(PROM)。以及与临床和认知结果相关的患者报告经验(PREM)。1至3级将逐步促进结构化面试的发展,调查问题,和治疗指南,通过相互通知。
    结论:SAPIENCE是对NMDAR脑炎的长期影响的深入研究,并弥合了标准化评估与个体患者体验之间的差距,旨在改善患者护理并提高对该疾病的心理社会长期后果的认识。通过欧洲临床神经病学和社会健康心理学专家的合作,SAPIENCE旨在创建在线评估工具并制定以患者为中心的急性后护理指南,这将有助于提高患者和护理人员的生活质量。
    BACKGROUND: N-methyl-D-aspartate-receptor (NMDAR) encephalitis is a rare neurological autoimmune disease with severe neuropsychiatric symptoms during the acute phase. Despite good functional neurological recovery, most patients continue to experience cognitive, psychiatric, psychological, and social impairments years after the acute phase. However, the precise nature and evolving patterns over time of these long-term consequences remain unclear, and their implications for the well-being and quality of life of predominantly young patients have yet to be thoroughly examined.
    METHODS: SAPIENCE is a European multi-center (n = 3) prospective observational cohort study studying the long-term cognitive, psychiatric, psychological, and social outcome in patients with NMDAR encephalitis. The study consists of three interconnected levels. Level 1 comprises a qualitative interview and focus groups with patients and their caregivers. Level 2 consists of a condensed form of the interview, standardized questionnaires, and a detailed neuropsychological examination of patients. Level 3 involves an online survey that will be open to patients world-wide and explores patient-reported outcomes (PROMs), and patient-reported experiences (PREMs) in association with clinical and cognitive outcomes. Levels 1 to 3 will progressively contribute developing of structured interviews, survey questions, and treatment guidelines by informing one another.
    CONCLUSIONS: SAPIENCE is an in-depth study of the long-term effects of NMDAR encephalitis and bridges the gap between standardized assessments and individual patient experiences, intending to improve patient care and to increase awareness of the psychosocial long-term consequences of the disease. Through collaboration of experts in clinical neurology and social and health psychology across Europe, SAPIENCE aims to create online assessment tools and formulate guidelines for patient-centered post-acute care that will help enhance the quality of life for patients and caregivers.
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