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Real-world patient characteristics, treatment patterns and costs in relapsing multiple sclerosis patients treated with glatiramer acetate, dimethyl fumarate or teriflunomide in Germany. 在德国使用醋酸格拉替默、富马酸二甲酯或特立氟米特治疗的多发性硬化症复发患者的真实世界患者特征、治疗模式和成本
IF 2.6 Q3 CLINICAL NEUROLOGY Pub Date : 2022-04-01 Epub Date: 2021-12-21 DOI: 10.2217/nmt-2021-0031
Tjalf Ziemssen, Anna Kurzeja, Bogdan Muresan, Jennifer S Haas, Jessica Alexander, Maurice T Driessen

Aim: To evaluate adherence, healthcare resource utilization (HRU) and costs for glatiramer acetate (GA; injectable), dimethyl fumarate (oral) and teriflunomide (oral) in relapsing multiple sclerosis. Patients & methods: Retrospective analyses of a claims database. Results: Teriflunomide patients were older with more co-morbidities and fewer relapses versus GA and dimethyl fumarate. GA patients were mostly disease-modifying therapies (DMTs)-treatment naive. Treatment adherence was 61-70%. All DMTs reduced HRU versus pre-index. Costs were comparable across cohorts. High adherence reduced hospitalizations and several costs versus low adherers. Conclusion: Adherence rates were high and comparable with all DMTs. Similar (and high) reductions in HRU and costs occurred with all DMTs. High adherence improved economic outcomes versus low adherence. Thus, investing in adherence improvement is beneficial to improve outcomes in relapsing multiple sclerosis.

目的:评价醋酸格拉替默(GA)的依从性、医疗资源利用率(HRU)和成本;可注射),富马酸二甲酯(口服)和特立氟米特(口服)复发多发性硬化症。患者与方法:回顾性分析索赔数据库。结果:与GA和富马酸二甲酯相比,特立氟米特患者年龄更大,合并症更多,复发更少。GA患者大多采用疾病修饰疗法(dmt)-初始治疗。治疗依从性为61-70%。与索引前相比,所有dmt均降低了HRU。不同队列的成本具有可比性。与低依从者相比,高依从性降低了住院率和多项费用。结论:依从率高,与所有dmt相当。在所有的dmt中,HRU和成本都出现了类似的(并且很高的)降低。与低依从性相比,高依从性改善了经济结果。因此,投资于依从性改善有利于改善复发性多发性硬化症的预后。
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引用次数: 1
Accuracy of cognitive screening instruments reconsidered: overall, balanced or unbiased accuracy? 重新考虑认知筛查工具的准确性:总体、平衡还是无偏准确性?
IF 2.6 Q3 CLINICAL NEUROLOGY Pub Date : 2022-04-01 Epub Date: 2022-02-18 DOI: 10.2217/nmt-2021-0049
Andrew J Larner

Aim: To examine three different accuracy metrics for evaluation of cognitive screening instruments: overall correct classification accuracy (Acc), the sum of true positives and negatives divided by the total number tested; balanced accuracy (balanced Acc), half of the sum of sensitivity and specificity; and unbiased accuracy (unbiased Acc), removing biasing effects of random associations between test results and disease prevalence. Materials & methods: Data from a prospective test accuracy study of Mini-Addenbrooke's Cognitive Examination were used to calculate and plot the Acc measures. Results: Each Acc metric resulted in a similar pattern of results across the range of Mini-Addenbrooke's Cognitive Examination cut-offs for diagnosis of both dementia and mild cognitive impairment. Acc and balanced Acc gave more optimistic outcomes (closer to possible maximum value of 1) than unbiased Acc. Conclusion: Unbiased Acc may have advantages over Acc and balanced Acc by removing biasing effects of random associations between test result and disease prevalence.

目的:探讨认知筛查工具评估的三种不同准确性指标:总正确分类准确率(Acc),真阳性和阴性的总和除以测试总数;平衡准确度(balanced Acc),敏感性和特异性之和的一半;和无偏准确性(unbiased Acc),消除了测试结果和疾病流行之间随机关联的偏倚效应。材料与方法:数据来自Mini-Addenbrooke认知测验的前瞻性测试准确性研究,用于计算和绘制Acc测量值。结果:每个Acc指标在Mini-Addenbrooke认知检查的范围内诊断痴呆和轻度认知障碍的结果模式相似。Acc和平衡Acc比无偏Acc给出更乐观的结果(更接近可能的最大值1)。结论:通过消除检测结果与疾病患病率之间随机关联的偏倚效应,无偏Acc可能比Acc和平衡Acc具有优势。
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引用次数: 7
Effects of an low carbohydrate/healthy fat/ketogenic diet on biomarkers of health and symptoms, anxiety and depression in Parkinson's disease: a pilot study. 低碳水化合物/健康脂肪/生酮饮食对帕金森病健康和症状、焦虑和抑郁生物标志物的影响:一项初步研究
IF 2.6 Q3 CLINICAL NEUROLOGY Pub Date : 2022-04-01 Epub Date: 2022-02-18 DOI: 10.2217/nmt-2021-0033
Melanie M Tidman, Dawn White, Timothy White

Aim: To evaluate a low carbohydrate/healthy fat/ketogenic diet (LCHF/KD) on symptoms, depression, anxiety and biomarkers in adults with Parkinson's disease (PD). Patients & methods: 16 adults ages 36-80 with PD participated in the intervention for 12 weeks. The study provided pre-post-study comparisons of biomarkers, weight, waist measurement, united Parkinson's Disease Rating Scale (UPDRS), Parkinson's Anxiety Scale (PAS) and Center for Epidemiologic Studies Depression Scale Revised-20 (CESD-R-20) Depression Scale. Results: Although LCHF/KD improves blood glucose in diabetes and seizure control in epilepsy, research gaps exist in this dietary intervention in PD. Statistically, significant improvements occurred in several measurements, PAS scores and Part I of the UPDRS. Conclusion: The LCHF/KD shows positive trends with improvements in biomarkers and anxiety symptoms. Further research is needed to evaluate dietary interventions for PD.

目的:评价低碳水化合物/健康脂肪/生酮饮食(LCHF/KD)对成年帕金森病(PD)患者的症状、抑郁、焦虑和生物标志物的影响。患者和方法:16名年龄在36-80岁的PD患者参与了为期12周的干预。该研究提供了生物标志物、体重、腰围测量、联合帕金森病评定量表(UPDRS)、帕金森焦虑量表(PAS)和流行病学研究中心抑郁量表修订版-20 (CESD-R-20)抑郁量表的研究前后比较。结果:虽然LCHF/KD可以改善糖尿病患者的血糖和癫痫患者的癫痫发作控制,但这种饮食干预在PD中的研究仍存在空白。统计上,在几个测量、PAS分数和UPDRS的第一部分中发生了显著的改善。结论:LCHF/KD随生物标志物和焦虑症状的改善呈阳性趋势。需要进一步的研究来评估饮食干预对PD的影响。
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引用次数: 11
Levodopa-induced dyskinesia: a brief review of the ongoing clinical trials. 左旋多巴诱发的运动障碍:正在进行的临床试验简评。
IF 2.6 Q3 CLINICAL NEUROLOGY Pub Date : 2022-04-01 Epub Date: 2021-12-23 DOI: 10.2217/nmt-2021-0051
Philippe Huot, Woojin Kang, Esther Kim, Dominique Bédard, Sébastien Belliveau, Imane Frouni, Cynthia Kwan
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引用次数: 0
Evaluating binary classifiers: extending the Efficiency Index 评估二元分类器:扩展效率指数
IF 2.6 Q3 CLINICAL NEUROLOGY Pub Date : 2022-02-04 DOI: 10.1101/2022.02.02.22270139
A. Larner
An efficiency index (EI) for the evaluation of binary classifiers was recently characterised, where EI is the ratio of classifier accuracy to inaccuracy. The purpose of this study was to further develop EI by substituting balanced accuracy and unbiased accuracy in place of accuracy, and their respective complements in place of inaccuracy, to construct balanced EI and unbiased EI measures. Additional investigations, using the dataset of a prospective pragmatic test accuracy study of a cognitive screening instrument, explored use of the log method to calculate confidence intervals for the various EI formulations; the dependence of EI formulations on prevalence; and comparison of EI formulations with analogous formulations based on the Identification Index (II), a previously described metric which is also based on accuracy and inaccuracy, where II is accuracy minus inaccuracy. EI formulations are shown to have advantages over II formulations, in particular their boundary values (0 and infinity) mean that negative values never occur, unlike the case for II, and the inflection point of 1 demarcates likelihood of correct versus incorrect classification.
最近描述了用于评估二元分类器的效率指数(EI),其中EI是分类器准确性与不准确性的比率。本研究的目的是进一步发展EI,以平衡准确度和无偏准确度代替准确性,并以它们各自的互补代替不准确性,构建平衡EI和无偏EI测度。其他调查,使用认知筛选工具的前瞻性语用测试准确性研究数据集,探索使用对数方法计算各种EI公式的置信区间;EI公式对患病率的依赖性;以及EI公式与基于识别指数(II)的类似公式的比较,识别指数(II)是先前描述的度量,也是基于准确性和不准确性,其中II是准确性减去不准确性。EI公式被证明比II公式有优势,特别是它们的边界值(0和无穷大)意味着负值永远不会出现,不像II的情况,并且1的拐点划定了正确与不正确分类的可能性。
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引用次数: 1
Evidence base for investigative and therapeutic modalities in chronic inflammatory demyelinating polyneuropathy and multifocal motor neuropathy. 慢性炎症性脱髓鞘性多神经病变和多灶性运动神经病变的调查和治疗方法的证据基础。
IF 2.6 Q3 CLINICAL NEUROLOGY Pub Date : 2022-02-01 Epub Date: 2022-01-10 DOI: 10.2217/nmt-2021-0015
Hendrik Stephan Goedee, Yusuf A Rajabally

Chronic inflammatory demyelinating polyneuropathy, its variants and multifocal motor neuropathy belong to a spectrum of peripheral nerve disorders with complex dysimmune disease mechanisms. Awareness of the unique clinical phenotypes but also heterogeneity between patients is vital to arrive at early suspicion and ordering appropriate tests. This includes requirements for optimal electrodiagnostic protocol, aimed to capture sufficient electrophysiologic evidence for relevant abnormalities, a case-based approach on the eventual need to further expand the diagnostic armamentarium and correct reading of their results. Considerable phenotypical variation, diverse combinations of abnormalities found on diagnostic tests and heterogeneity in disease course and treatment response, all contribute to widespread differences in success rates on timely diagnosis and optimal treatment. We aim to provide a practical overview and guidance on relevant diagnostic and management strategies, including pitfalls and present a summary of the relevant novel developments in this field.

慢性炎症性脱髓鞘性多神经病变及其变体和多灶性运动神经病变属于具有复杂免疫功能障碍疾病机制的周围神经疾病谱。了解独特的临床表型和患者之间的异质性对于早期怀疑和订购适当的测试至关重要。这包括对最佳电诊断方案的要求,旨在为相关异常捕获足够的电生理学证据,最终需要进一步扩大诊断设备并正确读取其结果的基于病例的方法。相当大的表型差异,诊断测试中发现的异常的不同组合以及疾病病程和治疗反应的异质性,都导致了及时诊断和最佳治疗成功率的广泛差异。我们的目标是提供有关诊断和管理策略的实用概述和指导,包括陷阱,并对该领域的相关新发展进行总结。
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引用次数: 0
Family planning in people with multiple sclerosis: a plain language summary. 多发性硬化症患者的计划生育:简单的语言总结。
IF 2.3 Q3 CLINICAL NEUROLOGY Pub Date : 2022-02-01 Epub Date: 2022-02-03 DOI: 10.2217/nmt-2021-0045
Simona Bonavita, Luigi Lavorgna, Hilary Worton, Dominic Jack

What is this summary about?: This is a plain language summary of an article originally published in the journal Frontiers in Neurology. People with multiple sclerosis (often shortened to MS) may have concerns about pregnancy and fertility. To understand more about these concerns, 332 people with MS in the USA, UK, France, Germany, Italy, and Spain took a survey with questions about how they made family planning decisions.

What were the results?: Most of the survey participants (around 82%) were women. The survey found that people with MS were less likely to have children than people without MS. Over half (56%) of people with MS said the disease impacted their family planning decisions in some way, almost one quarter (22%) significantly changed their plans for the timing of their pregnancy or number of children, and 14% decided against having children. For almost 4 out of 5 (81%) people with MS the main source of family planning information was healthcare professionals.

What do the results of the study mean?: Overall, MS significantly impacted patients' decisions about family planning.

这个总结是关于什么的?这是最初发表在《神经病学前沿》杂志上的一篇文章的简单语言总结。多发性硬化症(简称MS)患者可能会担心怀孕和生育问题。为了更好地了解这些问题,来自美国、英国、法国、德国、意大利和西班牙的332名多发性硬化症患者接受了一项关于他们如何做出计划生育决定的调查。结果如何?大多数调查参与者(约82%)是女性。调查发现,患有多发性硬化症的人比没有多发性硬化症的人更不可能生孩子。超过一半(56%)的多发性硬化症患者表示,这种疾病在某种程度上影响了他们的计划生育决策,近四分之一(22%)的人显著改变了他们的怀孕时间或孩子数量计划,14%的人决定不生孩子。对于几乎五分之四(81%)的MS患者,计划生育信息的主要来源是医疗保健专业人员。这项研究的结果意味着什么?总体而言,MS显著影响患者的计划生育决策。
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引用次数: 0
Corrigendum. 有待纠正。
IF 2.6 Q3 CLINICAL NEUROLOGY Pub Date : 2022-02-01 Epub Date: 2022-01-10 DOI: 10.2217/nmt-2020-0021c1
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引用次数: 0
GOCOVRI® (amantadine) extended-release capsules in Parkinson's disease. GOCOVRI®(金刚烷胺)缓释胶囊治疗帕金森病
IF 2.6 Q3 CLINICAL NEUROLOGY Pub Date : 2022-02-01 Epub Date: 2021-12-17 DOI: 10.2217/nmt-2021-0028
Thomas Müller

Amantadine is an old, antiviral compound, which moderately improves motor behavior in Parkinson's disease. Its current resurgence results from an innovative, delayed uptake and extended release amantadine hydrochloride capsule, given at bedtime once daily. It is the only approved compound for reduction of involuntary movements, so called dyskinesia, in fluctuating orally levodopa treated patients. It additionally ameliorates 'OFF' intervals characterized by impaired motor behavior. These beneficial effects result from higher and more continuous brain delivery of amantadine. Future clinical research is warranted on preventive effects of this amantadine capsule combined with enzyme blockers of central monoamine oxidase B and peripheral catechol-O-methyltransferase on motor complications in orally levodopa treated patients, as all these pharmacological principles support the concept of continuous dopamine substitution.

金刚烷胺是一种古老的抗病毒化合物,可以适度改善帕金森病患者的运动行为。它目前的复苏源于一种创新的、延迟吸收和缓释的盐酸金刚烷胺胶囊,每天一次在睡前服用。它是唯一被批准的化合物,用于减少波动口服左旋多巴治疗患者的不自主运动,即所谓的运动障碍。它还能改善以运动行为受损为特征的“OFF”间隔。这些有益的效果是由于金刚烷胺的大脑输送量更高、更持续。金刚烷胺胶囊联合中枢单胺氧化酶B和外周儿茶酚- o -甲基转移酶阻断剂对口服左旋多巴患者运动并发症的预防作用有待进一步的临床研究,因为所有这些药理学原理都支持持续多巴胺替代的概念。
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引用次数: 2
COVID-19-associated encephalitis or Creutzfeldt-Jakob disease: a case report. covid -19相关脑炎或克雅氏病1例报告
IF 2.6 Q3 CLINICAL NEUROLOGY Pub Date : 2022-02-01 Epub Date: 2021-12-02 DOI: 10.2217/nmt-2021-0025
Gooya Tayyebi, Seyed Kazem Malakouti, Behnam Shariati, Leila Kamalzadeh

Background: Accurate diagnosis and management of patients with rapidly progressive dementia may be challenging during the COVID-19 pandemic, which has negatively influenced the diagnostic performances, medical resource allocation and routine care for all non-COVID-19 diseases. Case presentation: We herein present a case of a 57-year-old male with rapidly progressive cognitive decline, headache, diplopia, myalgia, unsteady gait, aggression, depression, insomnia, hallucinations and delusions of persecution. COVID-19-associated encephalitis was briefly considered as a differential diagnosis. However, this hypothesis was rejected upon further investigation. A final diagnosis of sporadic Creutzfeldt-Jakob disease was made. Conclusion: A timely and accurate diagnosis of Creutzfeldt-Jakob disease gives patients and their families the chance to receive a good standard of healthcare and avoid extensive evaluations for other conditions.

背景:COVID-19大流行期间,快速进展性痴呆患者的准确诊断和管理可能具有挑战性,这对所有非COVID-19疾病的诊断性能、医疗资源分配和常规护理产生了负面影响。病例介绍:我们在此报告一例57岁男性患者,其表现为认知能力快速进行性下降、头痛、复视、肌痛、步态不稳、攻击性、抑郁、失眠、幻觉和迫害妄想。与covid -19相关的脑炎曾被短暂视为鉴别诊断。然而,经过进一步的调查,这一假设被否定了。最后诊断为散发性克雅氏病。结论:克雅氏病的及时准确诊断使患者及其家属有机会接受良好的医疗保健,避免对其他疾病进行广泛的评估。
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引用次数: 8
期刊
Neurodegenerative disease management
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