Functional impairment

功能损害
  • 文章类型: Journal Article
    人格解体/脱实障碍(DPD)是一种普遍但未充分理解的临床疾病,其特征是反复或持续的不现实感。这项研究旨在通过涉及大量中国参与者的描述性和比较分析来提供对DPD的见解。社会人口统计细节(年龄,性别比例,教育,职业状况,婚姻状况),去个性化和分离症状特征(剑桥去个性化量表和分离体验量表的症状因素或分量表),发展轨迹(发病年龄,潜在的诱发因素,课程特点),治疗史(延迟就诊的持续时间,延迟诊断的持续时间,以前的诊断),并介绍了DPD患者的不良童年经历。比较焦虑和抑郁症状,除了心理社会功能,在DPD参与者和被诊断为广泛性焦虑症的参与者之间,双相情感障碍,并进行了重度抑郁症。分析强调了男性较高的优势和DPD的早期发作,以失实为标志的症状学,心理社会功能明显受损,以及与症状严重程度相关的长时间延迟就诊和诊断。此外,我们发现了不良儿童经历与症状水平之间值得注意的关系.研究结果证实了DPD是一种严重但被忽视的精神障碍的观点,敦促采取措施改善DPD患者的现状。
    Depersonalization/derealization disorder (DPD) is a prevalent yet inadequately understood clinical condition characterized by a recurrent or persistent sense of unreality. This study aims to provide insight into DPD through descriptive and comparative analyses involving a large group of Chinese participants. The socio-demographic details (age, gender proportion, education, occupational status, marital status), depersonalized and dissociative symptom characteristics (symptomatic factors or subscales of the Cambridge Depersonalization Scale and the Dissociative Experiences Scale), development trajectory (age of onset, potential precipitating factors, course characteristics), treatment history (duration of delayed healthcare attendance, duration of delayed diagnosis, previous diagnoses), and adverse childhood experiences of the DPD patients are presented. Comparisons of anxiety and depressive symptoms, alongside psychosocial functioning, between DPD participants and those diagnosed with generalized anxiety disorder, bipolar disorders, and major depressive disorder were conducted. The analysis highlights a higher male preponderance and early onset of DPD, symptomatology marked by derealization, notable impairment in psychosocial functioning, and prolonged periods of delayed healthcare attendance and diagnosis associated with symptom severity. Furthermore, noteworthy relationships between adverse childhood experiences and symptom levels were identified. The findings substantiate the view that DPD is a serious but neglected mental disorder, urging initiatives to improve the current condition of DPD patients.
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  • 文章类型: Journal Article
    背景和目的:众所周知,抑郁症,焦虑,和冲动是相互关联的;然而,评估它们与冠状病毒爆发关联的研究很少。因此,我们的研究旨在评估2022年11月至2023年6月COVID-19感染后的冲动发生率及其与焦虑和抑郁的相关性.材料与方法:201名参与者完成了汉密尔顿抑郁量表(HDRS),汉密尔顿焦虑量表(HAM-A),Sheehan残疾量表(SDS),和短UPPS-P量表(紧迫性,预谋,毅力,寻求感觉,和积极的紧迫性),使我们能够确定他们的焦虑和抑郁症状,功能损害,和冲动,分别。结果:在我们的受访者中,22%,26.7%有中度至重度焦虑和抑郁。短UPPS-P量表与HAM-A和HDRS量表显著相关。COVID-19感染阳性的参与者表现出明显更高的功能障碍评分,特别是在工作/学习领域(平均值(SD):3.12(2.2)与2.43(2.3);p=0.037)。与COVID-19相关的中断与正和负的紧迫性显着相关,HAM-A,HDRS,以及SDS总数和分量表。结论:我们的研究结果显示焦虑显著增加,抑郁症,和COVID-19感染后人群的功能障碍。我们的研究强调了冲动与大流行后经历的心理困扰之间的相关性。
    Background and Objectives: It is well known that depression, anxiety, and impulsiveness are interrelated; however, studies that have assessed their association with the coronavirus outbreak are scarce. Hence, our study aimed to evaluate the impulsivity incidence and its correlation with anxiety and depression following COVID-19 infection between November 2022 and June 2023. Materials and Methods: The 201 participants completed the Hamilton Depression Rating Scale (HDRS), Hamilton Anxiety Rating Scale (HAM-A), Sheehan Disability Scale (SDS), and short UPPS-P scales (urgency, premeditation, perseverance, sensation seeking, and positive urgency) to allow us to determine their anxiety and depression symptoms, functional impairment, and impulsivity, respectively. Results: Among our respondents, 22%, and 26.7% had moderate to severe anxiety and depression. The short UPPS-P scale significantly correlated with the HAM-A and HDRS scales. Participants with positive COVID-19 infection showed significantly higher functional impairment scores, especially in the work/study domain (mean (SD): 3.12 (2.2) vs. 2.43 (2.3); p = 0.037). COVID-19-related disruption significantly correlated with negative and positive urgency, HAM-A, HDRS, and the SDS total and subscales. Conclusions: Our findings showed a notable increase in anxiety, depression, and functional impairment among the population following COVID-19 infection. Our research highlights the correlation between impulsivity and the psychological distress experienced following the pandemic.
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  • 文章类型: Journal Article
    残留症状在重度抑郁症(MDD)中普遍存在,包括广泛的症状,如睡眠障碍,体重和食欲的变化,认知障碍,和焦虑。这些症状持续损害日常功能,降低生活质量,并预测疾病复发。尽管它们具有临床意义,残留症状缺乏统一的定义,可能导致与治疗时出现的症状混淆和研究中的歧义,从而阻碍了研究成果的普遍性。虽然一些研究将失眠和情绪障碍确定为关键指标,其他研究强调不同的症状或发现没有显著的相关性。残留症状定义不一致,以及不同研究的方法差异,导致这些相互矛盾的结果。虽然临床医生专注于缓解阴性症状以改善功能状态,患者通常优先考虑获得积极影响和整体健康作为成功治疗的重要组成部分。这需要对抑郁症患者采取全面的护理方法。本文综述了MDD中残留症状的现象,关注定义的模糊性,临床特征,以及它们对长期结果的影响。缺乏对残留症状的标准化监管或学术定义导致临床医生之间的不同解释。强调需要标准化的术语来指导有效的治疗策略和未来的研究。
    Residual symptoms are prevalent in major depressive disorder (MDD), encompassing a wide spectrum of symptoms such as sleep disturbances, changes in weight and appetite, cognitive impairment, and anxiety. These symptoms consistently impair daily functioning, diminish quality of life, and forecast disease relapse. Despite their clinical significance, residual symptoms lack a unified definition, potentially leading to confusion with treatment-emergent symptoms and ambiguity across studies, thereby hindering the generalizability of research findings. While some research identifies insomnia and mood disturbances as critical indicators, other studies emphasize different symptoms or find no significant correlation. Inconsistencies in defining residual symptoms, as well as methodological differences across studies, contribute to these conflicting results. While clinicians focus on alleviating negative symptoms to improve functional status, patients often prioritize achieving positive affect and overall well-being as essential components of successful treatment. It necessitates a comprehensive approach to patient care in depression. This review explores the phenomenon of residual symptoms in MDD, focusing on the ambiguity in definitions, clinical characteristics, and their impact on long-term outcomes. The lack of a standardized regulatory or academic definition for residual symptoms leads to varied interpretations among clinicians, underscoring the need for standardized terminology to guide effective treatment strategies and future research.
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  • 文章类型: Journal Article
    目的:自闭症谱系障碍(ASD)和/或注意力缺陷多动障碍(ADHD)的儿童与通常发育中(TD)的同龄人相比,表现出更多的执行功能(EF)缺陷。EF缺陷与日常功能的各种损害和父母压力的增加有关。本研究的第一个目的是调查ASD和ADHD儿童与TD同龄人相比的EF。第二个目标是探索ASD和ADHD儿童的执行功能概况,最后,确定EF谱与父母压力和儿童功能障碍的差异。
    方法:样本包括30个TD儿童,47名自闭症儿童,和34名患有多动症的儿童,8至12岁。父母填写了育儿压力问卷,和儿童的社会和日常生活功能。家长和老师报告了有关儿童EF的信息。
    结果:结果表明,与TD组相比,临床组的EF损伤明显更大。此外,根据报告的EF困难的严重程度,确定了3个不同的功能簇.重要的发现表明,EF谱更严重的儿童与更大的每日损伤和更高的父母压力水平相关。
    结论:鉴于EF缺陷对ASD和ADHD儿童及其家庭生活的影响,至关重要的是,此类研究能增进我们的理解,并激发未来旨在改善ASD和ADHD儿童执行功能的干预措施.这种干预措施可以帮助减轻父母的压力并改善日常功能。
    OBJECTIVE: Children with autism spectrum disorder (ASD) and/or attention deficit hyperactivity disorder (ADHD) exhibit more executive function (EF) deficits compared to typically developing (TD) peers. EF deficits are linked to various impairments in daily functioning and increased parental stress. The first aim of the present study is to investigate EFs in children with ASD and ADHD compared to their TD peers. The second aim is to explore profiles of executive functions in children with ASD and ADHD and, finally, to determine the differences of EF profiles in relation to parental stress and children\'s functional impairments.
    METHODS: The sample comprised 30 TD children, 47 children with ASD, and 34 children with ADHD, aged 8 to 12 years. Parents completed questionnaires of parenting stress, and children\'s social and daily-life functioning. Parents and teachers reported information about children\'s EF.
    RESULTS: The results indicated significantly greater impairment of EFs in the clinical groups compared to the TD group. Moreover, three distinct clusters of functioning were identified based on the severity of reported EF difficulties. The significant findings showed that children with more severe EF profiles were associated with greater daily impairment and higher levels of perceived parental stress.
    CONCLUSIONS: Given the impact of EF deficits on the lives of children with ASD and ADHD and their families, it is crucial that studies like this enhance our understanding and inspire future interventions aimed at improving executive functions in children with ASD and ADHD. Such interventions could help reduce parental stress and improve daily functioning.
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  • 文章类型: Journal Article
    介绍糖尿病和骨关节炎(OA)是普遍存在的慢性疾病,经常同时发生,使患者管理复杂化。虽然每种情况对功能损害的个人影响是有据可查的,他们的综合效果仍然知之甚少。本研究旨在阐明糖尿病与OA相关功能损害之间的关系。方法这是一项对290名单侧膝关节OA参与者的横断面研究。他们的人口统计,临床,并收集糖尿病数据。使用西安大略省和麦克马斯特大学骨关节炎指数-风湿病中心(WOMAC-CRD)评估功能障碍。统计分析调查了糖尿病之间的关系,OA严重程度,和功能损害。结果糖尿病患者的身体功能和整体残疾均显著恶化,WOMAC-CRD分数较低。糖尿病和非糖尿病组的平均WOMAC-CRD疼痛评分分别为6.46(SD=1.088)和6.48(SD=1.101),分别。糖尿病和非糖尿病组的平均WOMAC-CRD硬度评分分别为6.48(SD=1.101)和6.56(SD=1.083)。糖尿病参与者的平均WOMAC-CRD身体功能评分为55.93(SD=2.484),相比之下,非糖尿病参与者为64.02(SD=2.542)。糖尿病参与者的平均WOMAC总分为68.80(SD=2.857),非糖尿病参与者的平均WOMAC总分为77.06(SD=2.933)。较长的糖尿病病程与身体功能和WOMAC总分呈负相关。讨论研究结果表明,糖尿病会加剧OA患者的功能损害,特别是影响身体功能和整体残疾。慢性炎症和晚期糖基化终产物的积累可能导致观察到的关节功能恶化。结论针对糖尿病和OA的综合管理策略对于优化患者护理至关重要。
    Introduction Diabetes and osteoarthritis (OA) are prevalent chronic conditions, often occurring concurrently and complicating patient management. While the individual impact of each condition on functional impairment is well documented, their combined effect remains poorly understood. This study aims to elucidate the relationship between diabetes and OA-related functional impairment. Methodology This was a cross-sectional study of 290 participants with unilateral knee OA. Their demographic, clinical, and diabetes data were collected. Functional impairment was assessed using the Western Ontario and McMaster Universities Osteoarthritis Index-Center for Rheumatic Diseases (WOMAC-CRD). Statistical analyses investigated the relationships between diabetes, OA severity, and functional impairment. Result Diabetic participants showed significantly worse physical function and overall disability, with lower WOMAC-CRD scores. Mean WOMAC-CRD pain scores were 6.46 (SD = 1.088) and 6.48 (SD = 1.101) for the diabetic and non-diabetic groups, respectively. Mean WOMAC-CRD stiffness scores were 6.48 (SD = 1.101) and 6.56 (SD = 1.083) for diabetic and non-diabetic groups. Diabetic participants had a mean WOMAC-CRD physical function score of 55.93 (SD = 2.484), compared to 64.02 (SD = 2.542) for non-diabetic participants. The mean total WOMAC score was 68.80 (SD = 2.857) for diabetic participants and 77.06 (SD = 2.933) for non-diabetic participants. Longer diabetes duration correlated negatively with physical function and total WOMAC scores. Discussion The findings suggest that diabetes exacerbates functional impairment in OA patients, particularly affecting physical function and overall disability. Chronic inflammation and the accumulation of advanced glycation end-products may contribute to the observed deterioration in joint function. Conclusion Integrated management strategies addressing both diabetes and OA are essential for optimizing patient care.
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  • 文章类型: Journal Article
    目的:我们目前的研究旨在使用来自社区居住和住院个体的代表性样本的纵向数据来调查年龄最大的老年人痴呆的决定因素。
    方法:纵向代表性数据取自“北莱茵-威斯特法伦州老年人(NRW80)的生活质量和主观幸福感调查”,该调查调查了80岁及以上的社区居住和机构化个人(分析样本中n=1,296个观察值),生活在北莱茵-威斯特法伦州(德国人口最多的州)。建立的DemTect用于测量认知障碍(即,可能的痴呆症)。使用逻辑随机效应模型来检查可能的痴呆的决定因素。
    结果:平均年龄为86.3岁(SD:4.2岁)。多重逻辑回归显示,可能的痴呆的可能性较高与受教育程度较低呈正相关(例如,与中等教育相比,教育程度较低:OR:3.31[95%CI:1.10-9.98]),较小的网络大小(OR:0.87[95%CI:0.79-0.96]),健康素养较低(OR:0.29[95%CI:0.14-0.60]),和更高的功能损害(OR:13.45[3.86-46.92]),虽然它与性别没有显著关系,年龄,婚姻状况,孤独,总样本中的抑郁症状。还报告了按性别分层的回归。
    结论:我们的研究确定了与年龄最大的老年人痴呆相关的因素。这项研究通过使用来自最古老的老年人的数据来扩展当前的知识;并通过基于纵向,代表性数据(也包括居住在制度化环境中的个人)。
    结论:努力增加,除其他外,正规教育,网络大小,健康素养在延缓痴呆症方面可能会有成效,尤其是老年妇女。制定健康素养计划,例如,可能有利于减轻与痴呆相关的负担。
    OBJECTIVE: Our current study aimed to investigate the determinants of dementia among the oldest old using longitudinal data from a representative sample covering both community-dwelling and institutionalized individuals.
    METHODS: Longitudinal representative data were taken from the \"Survey on quality of life and subjective well-being of the very old in North Rhine-Westphalia (NRW80+)\" that surveyed community-dwelling and institutionalized individuals aged 80 years and above (n = 1,296 observations in the analytic sample), living in North Rhine-Westphalia (most populous state of Germany). The established DemTect was used to measure cognitive impairment (i.e., probable dementia). A logistic random effects model was used to examine the determinants of probable dementia.
    RESULTS: The mean age was 86.3 years (SD: 4.2 years). Multiple logistic regressions revealed that a higher likelihood of probable dementia was positively associated with lower education (e.g., low education compared to medium education: OR: 3.31 [95% CI: 1.10-9.98]), a smaller network size (OR: 0.87 [95% CI: 0.79-0.96]), lower health literacy (OR: 0.29 [95% CI: 0.14-0.60]), and higher functional impairment (OR: 13.45 [3.86-46.92]), whereas it was not significantly associated with sex, age, marital status, loneliness, and depressive symptoms in the total sample. Regressions stratified by sex were also reported.
    CONCLUSIONS: Our study identified factors associated with dementia among the oldest old. This study extends current knowledge by using data from the oldest old; and by presenting findings based on longitudinal, representative data (also including individuals residing in institutionalized settings).
    CONCLUSIONS: Efforts to increase, among other things, formal education, network size, and health literacy may be fruitful in postponing dementia, particularly among older women. Developing health literacy programs, for example, may be beneficial to reduce the burden associated with dementia.
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  • 文章类型: Journal Article
    目的:临床实践指南建议在乳腺癌治疗后提供康复服务,然而,我们对利用可能因患者水平特征而异知之甚少,我们旨在使用SEER-Medicare数据进行研究.
    方法:来自监测的数据,流行病学,和最终结果(SEER)-Medicare关联数据库用于识别2011年至2016年间诊断的年龄≥66岁的非转移性乳腺癌幸存者.诊断后0-11个月提供的康复服务是通过门诊或医师就诊索赔确定的。使用改进的Poisson模型估计相对风险(RR)和相应的95%置信区间(CI),计算了描述性统计数据以及患者特征与康复服务之间的关联。
    结果:在55,539名乳腺癌幸存者中,33%(n=18,244)接受过任何类型的康复服务。幸存者的平均年龄为75岁(SD6.7),88%白色86%居住在城市,和21%的医疗保险/医疗补助双重登记。在调整后的模型中,年龄>75岁的患者vs.≤75有6%(RR0.94,95%CI0.92-0.96)的患者接受康复服务的可能性较小。在教育程度较高的地区的幸存者与受教育程度较低,白色vs.非白色,或者生活在农村城市面积为26%(1.26,CI1.22-1.30),6%(1.06,CI1.02-1.11),6%(1.06,CI1.02-1.10)更有可能接受康复服务,分别。
    结论:不同教育和治疗状态的幸存者在康复利用方面存在最大差异。
    结论:需要对障碍进行进一步研究,access,以及提供康复服务,专门针对老年乳腺癌幸存者,非白色,或Medicare/Medicaid双重资格。
    OBJECTIVE: Rehabilitation services are recommended by clinical practice guidelines following breast cancer treatment, yet little is known about how utilization may vary by patient-level characteristics which we aimed to study using SEER-Medicare data.
    METHODS: Data from the Surveillance, Epidemiology, and End Results (SEER)-Medicare linked database was used to identify non-metastatic breast cancer survivors aged ≥ 66 years diagnosed between 2011 and 2016. Rehabilitation services delivered 0-11 months post-diagnosis were identified via outpatient or physician visit claims. Descriptive statistics and associations between patient characteristics and rehabilitation services were calculated using modified Poisson models estimating relative risk (RR) and corresponding 95% confidence intervals (CIs).
    RESULTS: Of 55,539 breast cancer survivors, 33% (n = 18,244) had received any type of rehabilitative services. Survivors were a mean age of 75 years (SD 6.7), 88% White, 86% urban-dwelling, and 21% Medicare/Medicaid dually enrolled. In adjusted models, patients aged > 75 vs. ≤ 75 were 6% (RR 0.94, 95% CI 0.92-0.96) less likely to have received rehabilitative services. Survivors in an area with greater educational attainment vs. less educational attainment, White vs. non-White, or living in a rural vs. urban area were 26% (1.26, CI 1.22-1.30), 6% (1.06, CI 1.02-1.11), and 6% (1.06, CI 1.02-1.10) more likely to have received rehabilitative services, respectively.
    CONCLUSIONS: The largest differences in rehabilitation utilization were observed for survivors of differing educational and treatment statuses.
    CONCLUSIONS: Further research is needed on barriers, access, and delivery of rehabilitation services, specifically for breast cancer survivors who are older-aged, non-White, or Medicare/Medicaid dual eligible.
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  • 文章类型: Journal Article
    抗治疗性抑郁症(TRD)定义为在严重抑郁发作期间,至少两种抗抑郁药以足够的剂量和时机失败。鼻内使用Esketamine(ESK-IN)已被食品和药物管理局和欧洲药品管理局批准用于与其他抗抑郁药联合治疗TRD。
    评估接受ESK-IN治疗的TRD患者样本的有效性和耐受性作为同情使用计划的一部分。
    回顾,在9个中心的ESK-IN早期接入计划中纳入的诊断为TRD的患者中进行了观察性研究.在四个时间点使用Montgomery-Asberg抑郁量表(MADRS)评估有效性:基线,28、90和180天的治疗。
    样本包括71名患者(70%的女性),平均基线MADRS评分为38.27±5.9,总或部分工作残疾率为85%。在评估的所有时间点,ESK-IN治疗与抑郁症状严重程度的统计学和临床显着降低相关。副作用的存在很常见,但大多数严重程度较轻,在观察期后缓解。与未接受心理治疗的患者相比,接受心理治疗与ESK-IN联合治疗的患者在90天和180天时的MADRS评分显着降低。
    ESK-IN已被证明在严重TRD患者的临床样本中是有效和安全的。为了优化临床结果,TRD的药物治疗应始终纳入包括心理治疗等策略的综合治疗计划,社会支持,家庭干预。
    UNASSIGNED: Treatment-resistant depression (TRD) is defined as the failure of at least two antidepressants in adequate doses and timing during a major depressive episode. Esketamine intranasal (ESK-IN) has been approved by the Food and Drug Administration and the European Medicines Agency for the treatment of TRD in combination with other antidepressants.
    UNASSIGNED: To assess the effectiveness and tolerability of a sample of TRD patients who received treatment with ESK-IN as part of the compassionate use program.
    UNASSIGNED: A retrospective, observational study was carried out on patients with a diagnosis of TRD enrolled in the early access program of ESK-IN in nine centers. Effectiveness was assessed with the Montgomery-Asberg depression rating scale (MADRS) at four time points: baseline, 28, 90, and 180 days of treatment.
    UNASSIGNED: The sample included 71 patients (70% women) with a mean baseline MADRS score of 38.27 ± 5.9 and total or partial work disability rates of 85%. ESK-IN treatment was associated with a statistically and clinically significant reduction in the severity of depressive symptoms at all time points assessed. The presence of side effects was common but the majority were mild in severity and resolved after the observation period. Those patients who received psychotherapy in combination with ESK-IN showed a significantly lower MADRS score at 90 and 180 days than those patients who did not undergo psychotherapy.
    UNASSIGNED: ESK-IN has proven to be effective and safe in a clinical sample of patients with severe TRD. To optimize clinical outcomes, the pharmacological treatment for TRD should always be integrated into a comprehensive therapeutic plan that encompasses strategies such as psychotherapy, social support, and family interventions.
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  • 文章类型: Journal Article
    随着时间的推移客观地测量帕金森病(PD)的体征和症状对于成功开发旨在阻止PD患者疾病进展的治疗方法至关重要。
    创建一种临床试验模拟工具,该工具可以表征PD进展的自然史,并支持数据驱动的随机对照研究设计,测试早期PD的潜在疾病改善治疗(DMT)。
    使用非线性混合效应建模技术分析了帕金森进展标志物计划(PPMI)的数据,以表征MDS-UPDRS第一部分(日常生活经验的非运动方面)的进展,第二部分(日常生活经验的运动方面),和第三部分(运动标志)。根据这些疾病模型构建临床试验模拟工具,并将其用于根据试验设计预测成功概率。
    MDS-UPDRS第三部分的进展速度比MDS-UPDRS第二部分和第一部分快大约3倍,每年增加3分比1分。较高量的对症治疗与MDS-UPDRS部分II和III的较慢进展相关。建模框架预测,DMT对MDS-UPDRS第三部分的影响可能先于对第二部分的影响大约2至3年。
    我们的临床试验模拟工具预测,在一项为期两年的随机对照试验中,MDS-UPDRS第三部分可用于评估潜在的新型DMT,而第II部分则需要更长的试验,最短持续时间为3~5年,强调需要创新的试验设计方法,包括新的以患者为中心的措施.
    开发能够减缓或阻止帕金森病(PD)进展的有效药物,准确了解疾病如何随着时间的推移而恶化是很重要的。我们使用了一项观察性研究的数据,由迈克尔·J·福克斯基金会领导,称为帕金森进展标志物倡议(PPMI),以了解PD的自然进展。我们使用不同的尺度在计算机上模拟临床试验以测量PD的进展。我们特别研究了医生报告的MDS-UPDRS第三部分,以及在患者报告的MDS-UPDRS第二部分中,PD症状如何随着时间的推移而恶化。使用患者报告的MDS-UPDRS第二部分测量一种新药减缓PD进展的效果,我们估计我们可能需要进行至少3~5年的临床试验.另一方面,使用医生报告的MDS-UPDRS第三部分测量效果,试验持续时间可能短于2年.我们还能够证明,医生报告的MDS-UPDRS第三部分记录的恶化可以预测患者报告的MDS-UPDRS第二部分记录的后期恶化。我们得出的结论是,MDS-UPDRS第III部分可能是合理持续时间的临床试验的良好终点,并且MDS-UPDRS第II部分可以在更长的研究中进行测量,例如,开放标签扩展。
    UNASSIGNED: Objectively measuring Parkinson\'s disease (PD) signs and symptoms over time is critical for the successful development of treatments aimed at halting the disease progression of people with PD.
    UNASSIGNED: To create a clinical trial simulation tool that characterizes the natural history of PD progression and enables a data-driven design of randomized controlled studies testing potential disease-modifying treatments (DMT) in early-stage PD.
    UNASSIGNED: Data from the Parkinson\'s Progression Markers Initiative (PPMI) were analyzed with nonlinear mixed-effect modeling techniques to characterize the progression of MDS-UPDRS part I (non-motor aspects of experiences of daily living), part II (motor aspects of experiences of daily living), and part III (motor signs). A clinical trial simulation tool was built from these disease models and used to predict probability of success as a function of trial design.
    UNASSIGNED: MDS-UPDRS part III progresses approximately 3 times faster than MDS-UPDRS part II and I, with an increase of 3 versus 1 points/year. Higher amounts of symptomatic therapy is associated with slower progression of MDS-UPDRS part II and III. The modeling framework predicts that a DMT effect on MDS-UPDRS part III could precede effect on part II by approximately 2 to 3 years.
    UNASSIGNED: Our clinical trial simulation tool predicted that in a two-year randomized controlled trial, MDS-UPDRS part III could be used to evaluate a potential novel DMT, while part II would require longer trials of a minimum duration of 3 to 5 years underscoring the need for innovative trial design approaches including novel patient-centric measures.
    To develop effective medicines that can slow down or stop the progression of Parkinson’s disease (PD), it is important to accurately understand how the disease worsens over time. We used data from an observational study, led by the Michael J. Fox Foundation, called the Parkinson’s Progression Markers Initiative (PPMI) to understand the natural progression of  PD. We simulated clinical trials on a computer using different scales to measure the progression of PD. We specifically looked at a physician-reported measure MDS-UPDRS part III, and at a patient-reported measure MDS-UPDRS part II of how PD symptoms worsen over time. To measure the effect of a new medicine slowing down the progression of PD using patient-reported measure MDS-UPDRS part II, we estimate that we may need to conduct a clinical trial of at least 3 to 5 years. On the other hand, to measure an effect using physician-reported measure MDS-UPDRS part III, the duration of the trial could be shorter than 2 years. We were also able to show that worsening recorded by the physician-reported measure MDS-UPDRS part III could be predictive of a later worsening recorded by the patient-reported measure MDS-UPDRS part II. We concluded that MDS-UPDRS part III may be a good endpoint for a clinical trial of a reasonable duration and that MDS-UPDRS part II could be measured in longer studies, for example, open-label extensions.
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  • 文章类型: Journal Article
    长期禁闭会导致不同程度的个人恶化。我们在马德里Orcasitas社区的功能依赖人群的全国COVID-19封锁期间研究了这一现象,西班牙,通过测量他们进行日常生活基本活动的能力和死亡率。
    共有127例患者纳入Orcasitas队列。在这个队列中,78.7%是女性,21.3%为男性,他们的平均年龄是86岁。所有参与者的Barthel指数≤60。分析了从分娩前到分娩后以及之后3年的变化,并评估了这些变化对生存率的影响(2020-2023年).
    禁闭后功能评估显示,Barthel评分(t=-5.823;p<0.001)和分类水平(z=-2.988;p<0.003)的独立性均优于禁闭前。这种改善在接下来的3年里逐渐消失,该队列中40.9%的患者在此期间死亡。这些结果与Barthel指数(z=-3.646;p<0.001)和依赖水平(风险比2.227;CI1.514-3.276)相关。男性(HR1.745;CI1.045-2.915)和严重依赖者(HR2.169;CI1.469-3.201)的死亡率较高。将Barthel指数的截止点设置为40,可以最好地检测与依赖相关的死亡风险。
    家庭禁闭和COVID-19大流行导致的死亡风险唤醒了功能依赖的成年人群体在逆境中的一种恢复力。Barthel指数是中期和长期死亡率的良好预测指标,并且是在健康计划中检测处于危险中的人群的有用方法。40的截止分数可用于此目的。在某种程度上,非制度化的依赖人口是无形人口。未来的研究应该分析观察到的高死亡率的原因。
    UNASSIGNED: Prolonged confinement can lead to personal deterioration at various levels. We studied this phenomenon during the nationwide COVID-19 lockdown in a functionally dependent population of the Orcasitas neighborhood of Madrid, Spain, by measuring their ability to perform basic activities of daily living and their mortality rate.
    UNASSIGNED: A total of 127 patients were included in the Orcasitas cohort. Of this cohort, 78.7% were female, 21.3% were male, and their mean age was 86 years. All participants had a Barthel index of ≤ 60. Changes from pre- to post-confinement and 3 years afterward were analyzed, and the effect of these changes on survival was assessed (2020-2023).
    UNASSIGNED: The post-confinement functional assessment showed significant improvement in independence over pre-confinement for both the Barthel score (t = -5.823; p < 0.001) and the classification level (z = -2.988; p < 0.003). This improvement progressively disappeared in the following 3 years, and 40.9% of the patients in this cohort died during this period. These outcomes were associated with the Barthel index (z = -3.646; p < 0.001) and the level of dependence (hazard ratio 2.227; CI 1.514-3.276). Higher mortality was observed among men (HR 1.745; CI 1.045-2.915) and those with severe dependence (HR 2.169; CI 1.469-3.201). Setting the cutoff point of the Barthel index at 40 provided the best detection of the risk of death associated with dependence.
    UNASSIGNED: Home confinement and the risk of death due to the COVID-19 pandemic awakened a form of resilience in the face of adversity among the population of functionally dependent adults. The Barthel index is a good predictor of medium- and long-term mortality and is a useful method for detecting populations at risk in health planning. A cutoff score of 40 is useful for this purpose. To a certain extent, the non-institutionalized dependent population is an invisible population. Future studies should analyze the causes of the high mortality observed.
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