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Japanese longitudinal biomarker study in progressive supranuclear palsy and corticobasal degeneration: Clinical features of the first registered patients and short-term follow-up analysis 日本进行性核上性麻痹和皮质基底变性的纵向生物标志物研究:首批登记患者的临床特征和短期随访分析
IF 1.9 Q3 CLINICAL NEUROLOGY Pub Date : 2024-01-01 DOI: 10.1016/j.prdoa.2024.100279
Hiroshi Takigawa , Ritsuko Hanajima , Ikuko Aiba , Takayoshi Shimohata , Takahiko Tokuda , Mitsuya Morita , Osamu Onodera , Shigeo Murayama , Kazuko Hasegawa , Aya M. Tokumaru , Hisanori Kowa , Masato Kanazawa , Tameto Naoi , Kenji Nakashima , Takeshi Ikeuchi , JALPAC study group

Introduction

Progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD) manifest with variable clinical features. We initiated a multicenter prospective registry study—the Japanese Longitudinal Biomarker Study in PSP and CBD—in November 2014 at 45 Japanese institutions to collect clinical information and biological samples to elucidate the natural courses and diagnostic biomarkers of PSP/CBD.

Methods

Initial symptoms, clinical features, and scores (Progressive Supranuclear Palsy Rating Scale [PSPRS], Barthel Index, Mini-Mental State Examination, and Frontal Assessment Battery) of patients clinically diagnosed with PSP/corticobasal syndrome (CBS) at the first registration were analyzed. PSPRS score progression in the initial 8 years and predictive factors were examined.

Results

As of October 2022, first registration had been conducted for 349 patients—57 with probable/possible Richardson’s syndrome (RS), 133 with possible CBS, 41 with overlapping CBS and PSP criteria (RS/CBS group), 20 with PSP subtypes other than RS, and 98 who did not fulfill the PSP or CBS criteria. Among the RS, CBS, and RS/CBS groups, the RS group exhibited the best scores. Initial symptoms of personality change and asymmetric onset were correlated with the total PSPRS score. The average PSPRS score increment by the second registration (n = 116 patients) was 11.8 in all three groups, and progression was correlated with cognitive dysfunction. Seventy patients died during the study period. The 5-year survival rate from onset was approximately 90 %.

Conclusion

There were fewer severe clinical features in the RS group than in the CBS group. Cognitive dysfunction may be important in predicting clinical severity and disease progression.
导言:进行性核上性麻痹(PSP)和皮质基底变性(CBD)表现出不同的临床特征。我们于 2014 年 11 月在日本 45 家机构启动了一项多中心前瞻性登记研究--日本 PSP 和 CBD 纵向生物标志物研究,收集临床信息和生物样本,以阐明 PSP/CBD 的自然病程和诊断生物标志物。方法分析首次登记时临床诊断为PSP/皮质基底综合征(CBS)患者的初始症状、临床特征和评分(进行性核上麻痹评分量表[PSPRS]、巴特尔指数、迷你精神状态检查和额叶评估电池)。结果 截至2022年10月,已对349名患者进行了首次登记,其中57人可能患有理查森综合征(RS),133人可能患有CBS,41人CBS和PSP标准重叠(RS/CBS组),20人患有RS以外的PSP亚型,98人不符合PSP或CBS标准。在 RS 组、CBS 组和 RS/CBS 组中,RS 组的得分最高。人格改变和不对称发病的初始症状与 PSPRS 总分相关。所有三组患者在第二次登记时(n = 116 名患者)的 PSPRS 平均增量为 11.8 分,其进展与认知功能障碍相关。研究期间有 70 名患者死亡。结论 RS 组的严重临床特征少于 CBS 组。认知功能障碍可能是预测临床严重程度和疾病进展的重要依据。
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引用次数: 0
Dystonia: A novel sign of the Smith-Magenis syndrome – A three-case report 肌张力障碍:史密斯-马盖尼综合征的一种新征兆--三例病例报告
IF 1.9 Q3 CLINICAL NEUROLOGY Pub Date : 2024-01-01 DOI: 10.1016/j.prdoa.2024.100267
Lukáš Kunc , Petra Havránková , Matěj Škorvánek , Iva Příhodová , Kamila Poláková , Lenka Nosková , Markéta Tesařová , Tomáš Honzík , Michael Zech , Robert Jech
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引用次数: 0
Continuous subcutaneous foslevodopa/foscarbidopa infusion for the treatment of motor fluctuations in Parkinson’s disease: Considerations for initiation and maintenance 连续皮下注射磷左多巴/磷卡比多巴治疗帕金森病的运动波动:启动和维持治疗的注意事项
Q3 CLINICAL NEUROLOGY Pub Date : 2024-01-01 DOI: 10.1016/j.prdoa.2024.100239
Victor S.C. Fung , Jason Aldred , Martha P. Arroyo , Filip Bergquist , Agnita J.W. Boon , Manon Bouchard , Sarah Bray , Sara Dhanani , Maurizio F. Facheris , Nahome Fisseha , Eric Freire-Alvarez , Robert A. Hauser , Anna Jeong , Jia Jia , Pavnit Kukreja , Michael J. Soileau , Amy M. Spiegel , Saritha Talapala , Arjun Tarakad , Enrique Urrea-Mendoza , Rajesh Pahwa

Background

As Parkinson's disease (PD) advances, management is challenged by an increasingly variable and inconsistent response to oral dopaminergic therapy, requiring special considerations by the provider. Continuous 24 h/day subcutaneous infusion of foslevodopa/foscarbidopa (LDp/CDp) provides steady dopaminergic stimulation that can reduce symptom fluctuation.

Objective

Our aim is to review the initiation, optimization, and maintenance of LDp/CDp therapy, identify possible challenges, and share potential mitigations.

Methods

Review available LDp/CDp clinical trial data for practical considerations regarding the management of patients during LDp/CDp therapy initiation, optimization, and maintenance based on investigator clinical trial experience.

Results

LDp/CDp initiation, optimization, and maintenance can be done without hospitalization in the clinic setting. Continuous 24 h/day LDp/CDp infusion can offer more precise symptom control than oral medications, showing improvements in motor fluctuations during both daytime and nighttime hours. Challenges include infusion-site adverse events for which early detection and prompt management may be required, as well as systemic adverse events (eg, hallucinations) that may require adjustment of the infusion rate or other interventions. A learning curve should be anticipated with initiation of therapy, and expectation setting with patients and care partners is key to successful initiation and maintenance of therapy.

Conclusion

Continuous subcutaneous infusion of LDp/CDp represents a promising therapeutic option for individuals with PD. Individualized dose optimization during both daytime and nighttime hours, coupled with patient education, and early recognition of certain adverse events (plus their appropriate management) are required for the success of this minimally invasive and highly efficacious therapy.

背景随着帕金森病(PD)的发展,口服多巴胺能治疗的反应越来越多变且不一致,这给治疗带来了挑战,需要提供者做出特别考虑。我们的目的是回顾 LDp/CDp 治疗的启动、优化和维持,识别可能存在的挑战,并分享潜在的缓解方法。方法根据研究者的临床试验经验,回顾现有的 LDp/CDp 临床试验数据,了解在 LDp/CDp 治疗启动、优化和维持期间患者管理的实际注意事项。与口服药物相比,每天 24 小时连续输注 LDp/CDp 可以更精确地控制症状,白天和夜间的运动波动均有所改善。面临的挑战包括输注部位不良事件,可能需要及早发现和及时处理,以及全身不良事件(如幻觉),可能需要调整输注速度或采取其他干预措施。开始治疗时应预计到学习曲线,与患者和护理伙伴共同设定期望值是成功开始和维持治疗的关键。日间和夜间的个体化剂量优化、患者教育和某些不良反应的早期识别(以及适当的处理)是这一微创、高效疗法取得成功的必要条件。
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引用次数: 0
Treatment of axial postural abnormalities in parkinsonism disorders: A systematic review of pharmacological, rehabilitative and surgical interventions 帕金森病患者轴向姿势异常的治疗:药物、康复和手术干预的系统回顾
Q3 CLINICAL NEUROLOGY Pub Date : 2024-01-01 DOI: 10.1016/j.prdoa.2024.100240
Marialuisa Gandolfi , Christian Geroin , Gabriele Imbalzano , Serena Camozzi , Zoe Menaspà , Michele Tinazzi , Carlo Alberto Artusi

Axial postural abnormalities (PA) are frequent, highly disabling, and drug-refractory motor complications affecting patients with Parkinson’s disease (PD) or atypical parkinsonism. Over the past few years, advances have been reached across diagnosis, assessment, and pathophysiological mechanisms of PA. Nonetheless, their management remains a challenge, and these disturbances are generally overlooked by healthcare professionals, potentially resulting in their worsening and impact on patients’ disabilities. From shared consensus-based assessment and diagnostic criteria, PA calls for interdisciplinary management based on the complexity and multifactorial pathogenesis. In this context, we conducted a systematic literature review to analyze the available pharmacological and non-pharmacological treatment options for PA in PD according to the new expert-based classification of axial PA in Parkinsonism. Different multidisciplinary approaches, including dopaminergic therapy adjustment, physiotherapy, botulinum toxin injection, and deep brain stimulation, can improve PA depending on its type and severity. An early, interdisciplinary approach is recommended in PD patients to manage PA.

轴性姿势异常(PA)是帕金森病(PD)或不典型帕金森病患者经常出现的、致残率高且药物难治的运动并发症。在过去几年中,帕金森病的诊断、评估和病理生理机制都取得了进展。然而,对这些患者的管理仍然是一项挑战,医护人员通常会忽视这些障碍,从而可能导致其恶化并影响患者的残疾。从基于共识的评估和诊断标准来看,PA 需要基于其复杂性和多因素致病机理进行跨学科管理。在此背景下,我们进行了一项系统性文献综述,根据基于专家的帕金森病轴性 PA 新分类,分析了帕金森病 PA 可用的药物和非药物治疗方案。根据PA的类型和严重程度,不同的多学科方法,包括多巴胺能治疗调整、物理治疗、肉毒毒素注射和脑深部刺激,可改善PA。建议帕金森病患者尽早采用跨学科方法来控制 PA。
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引用次数: 0
Dysphagia in progressive supranuclear palsy: A scoping review 进行性核上性麻痹的吞咽困难:范围综述
IF 1.9 Q3 CLINICAL NEUROLOGY Pub Date : 2024-01-01 DOI: 10.1016/j.prdoa.2024.100283
Éadaoin Flynn , Julie Regan , Julia Glinzer , Sean O’Dowd , Margaret Walshe

Introduction

One of the most prevalent types of atypical parkinsonian syndrome is progressive supranuclear palsy (PSP). PSP is associated with early onset of dysphagia which can result in malnutrition, dehydration, and aspiration pneumonia, affecting quality of life and increasing mortality rate. To date, research describing dysphagia in PSP and its impact is scant.

Methods

The objective of this scoping review is to determine the characteristics of dysphagia in PSP, differences in dysphagia presentation according to PSP subtype, principal methods used for identifying and diagnosing dysphagia and the impact dysphagia has on quality of life in individuals with PSP. This review was conducted in accordance with the JBI methodology. Six electronic databases were searched.

Results

Of the 20 studies included, the most frequently reported characteristics of dysphagia were oral preparatory and oral phase difficulties. A variety of methods were used to identify and diagnose dysphagia including instrumental assessment (65%), patient reported scales (45%) and clinical swallow evaluation (20%). The most used instrumental assessment was videofluoroscopy (46%). Limited data was available describing characteristics of dysphagia according to the subtype of PSP. The impact that dysphagia has on quality of life was assessed in only one study.

Conclusion

A range of assessment methods are used to identify and diagnose dysphagia in patients with PSP. Further research is needed to investigate if particular characteristics are associated with certain PSP subtypes. Future studies should also measure the impact that dysphagia has on quality of life in this population.
进行性核上性麻痹(PSP)是最常见的非典型帕金森综合征之一。PSP与早期发生的吞咽困难有关,吞咽困难可导致营养不良、脱水和吸入性肺炎,影响生活质量并增加死亡率。迄今为止,描述PSP患者吞咽困难及其影响的研究还很少。方法本综述的目的是确定PSP患者的吞咽困难的特征、不同亚型的吞咽困难表现的差异、识别和诊断吞咽困难的主要方法以及吞咽困难对PSP患者生活质量的影响。这项审查是按照JBI方法进行的。检索了六个电子数据库。结果在纳入的20项研究中,最常见的吞咽困难特征是口腔准备和口腔期困难。使用多种方法来识别和诊断吞咽困难,包括仪器评估(65%)、患者报告量表(45%)和临床吞咽评估(20%)。最常用的仪器评估是视频透视(46%)。根据PSP亚型描述吞咽困难特征的资料有限。只有一项研究评估了吞咽困难对生活质量的影响。结论一系列的评估方法可用于识别和诊断PSP患者的吞咽困难。需要进一步的研究来调查特定的特征是否与某些PSP亚型有关。未来的研究还应该测量吞咽困难对这一人群生活质量的影响。
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引用次数: 0
Comparing smell identification ability among different motor subtypes of Parkinson’s disease using the Vietnamese Smell Identification Test and the Brief Smell Identification Test 使用越南嗅觉识别测试和简短嗅觉识别测试比较帕金森病不同运动亚型的嗅觉识别能力
IF 1.9 Q3 CLINICAL NEUROLOGY Pub Date : 2024-01-01 DOI: 10.1016/j.prdoa.2024.100270
Thuong Huyen Thi Dang , Daniel Truong , Khang Vinh Nguyen , Uyen Le Ngoc Ha , Khang Chung Ngoc Vo , Thanh Vinh Nguyen , Hien Thi Le , Tai Ngoc Tran

Introduction

Olfactory dysfunction is one of the most common non-motor symptoms of Parkinson’s disease (PD). The association between smell identification ability and motor subtypes of PD is not uniform in previous studies. This study aimed to compare the odor identification ability among different motor subtypes of PD in Vietnamese participants.

Methods

Patients who were diagnosed with PD according to the International Parkinson’s Disease and Movement Disorder Society 2015 Diagnostic Criteria and had normal cognitive function were recruited. Participants were divided into akinetic-rigid (AR), tremor-dominant (TD), and mixed (MX) motor subgroups using the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) score. Olfactory identification ability was evaluated using the Vietnamese Smell Identification Test (VSIT) and the Brief Smell Identification Test (BSIT). Cognitive status was assessed using the Mini-Mental State Examination (MMSE). Age, age at PD onset, disease duration, smell identification ability, and cognitive function were compared among the three PD motor subtypes.

Results

The AR subgroup was the most common motor subtype (n = 164, 75.2 %), followed by TD (n = 39, 17.9 %), and MX (n = 15, 6.9 %) subtypes. Age, age at PD onset, sex, disease duration, and MMSE score were not significantly different between the three motor subgroups (all p > 0.05). The median (IQR) VSIT scores of AR, TD, and MX subgroups were 5.00 [4.00;7.00], 5.00 [3.50;7.00], and 5.00 [3.00;6.00], respectively. The median (IQR) BSIT scores of AR, TD, and MX subgroups were 6.00 [4.00;7.00], 5.00 [4.00;7.00], and 5.00 [4.50;7.00], respectively. The VSIT and the BSIT scores were not significantly different among the three motor subtypes (all p > 0.05).

Conclusion

Smell identification ability assessed in both the VSIT and BSIT did not differ across the three motor subtypes of PD.

导言嗅觉功能障碍是帕金森病(PD)最常见的非运动症状之一。在以往的研究中,气味识别能力与帕金森病运动亚型之间的关联并不一致。本研究旨在比较越南参与者对帕金森病不同运动亚型的气味识别能力。方法招募根据国际帕金森病和运动障碍协会2015年诊断标准被诊断为帕金森病且认知功能正常的患者。根据运动障碍协会-统一帕金森病评定量表(MDS-UPDRS)的评分,将参与者分为运动僵直型(AR)、震颤主导型(TD)和混合型(MX)运动亚组。嗅觉识别能力采用越南气味识别测试(VSIT)和简短气味识别测试(BSIT)进行评估。认知状况采用迷你精神状态检查(MMSE)进行评估。结果 AR亚组是最常见的运动亚型(n = 164,75.2%),其次是TD亚型(n = 39,17.9%)和MX亚型(n = 15,6.9%)。年龄、帕金森病发病年龄、性别、病程和 MMSE 评分在三个运动亚型之间无显著差异(均为 p >0.05)。AR、TD和MX亚组的VSIT评分中位数(IQR)分别为5.00 [4.00;7.00]、5.00 [3.50;7.00]和5.00 [3.00;6.00]。AR、TD和MX亚组的BSIT评分中位数(IQR)分别为6.00 [4.00;7.00]、5.00 [4.00;7.00]和5.00 [4.50;7.00]。结论在VSIT和BSIT中评估的气味识别能力在三种运动亚型的帕金森病患者中没有差异。
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引用次数: 0
Orthostatic tremor as possible presenting sign of progressive supranuclear palsy 静止性震颤可能是进行性核上性麻痹的首发症状
IF 1.9 Q3 CLINICAL NEUROLOGY Pub Date : 2024-01-01 DOI: 10.1016/j.prdoa.2024.100275
Emke Maréchal , Sven Dekeyzer , David Crosiers
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引用次数: 0
Single-center experience of utilization and clinical efficacy of segmented leads for subthalamic deep brain stimulation in Parkinson’s disease 帕金森病眼下丘脑深部脑刺激分段导线的使用和临床疗效的单中心经验
IF 1.9 Q3 CLINICAL NEUROLOGY Pub Date : 2024-01-01 DOI: 10.1016/j.prdoa.2024.100273
Ana Luísa de Almeida Marcelino , Viktor Heinz , Melanie Astalosch , Bassam Al-Fatly , Gerd-Helge Schneider , Patricia Krause , Dorothee Kübler-Weller , Andrea A. Kühn

Background

Segmented electrodes for deep brain stimulation (DBS) of the subthalamic nucleus (STN) in Parkinson’s disease (PD) enable directional current steering leading to expanded programming options.

Objective

This retrospective study covering a longitudinal period of up to 7 years compares the efficacy of segmented and non-segmented leads in motor symptom alleviation and reduction of dopaminergic medication in PD patients treated in a specialized center and assesses the long-term use of directional steering in clinical routine.

Methods

Demographic data and clinical scores before surgery and at 12-month follow-up (12MFU) as well as stimulation parameters at 12MFU and last follow-up (LFU) were assessed in all patients implanted with segmented leads between 01/2016 and 12/2019 and non-segmented leads in a corresponding time-period. Patients were classified as very good (>60 %), good (30–60 %) and poor (<30 %) responders according to DBS-induced motor improvement.

Results

Clinical data at 12MFU was available for 61/96 patients with segmented (SEG) and 42/53 with non-segmented leads (N-SEG). Mean DBS-induced motor improvement and reduction of medication at 12MFU did not differ significantly between SEG and N-SEG groups or in a subgroup analysis of steering modes. There was a lower proportion of poor responders in the SEG compared with the N-SEG group (23% vs. 31%), though not statistically significant. At LFU, the percentage of patients set at directional steering increased from 54% to 70%.

Conclusion

Efficacy in reduction of motor symptoms and medication does not differ between electrode types for STN-DBS at 12 months follow-up. The use of directional steering increases over time and may account for a lower proportion of poor responders.
背景用于帕金森病(PD)丘脑下核(STN)深部脑刺激(DBS)的分段式电极可进行定向电流转向,从而扩大编程选择范围。目的这项回顾性研究涵盖长达 7 年的纵向时间,比较了分段式和非分段式导线在缓解运动症状和减少多巴胺能药物用量方面的疗效,这些患者均在一家专科中心接受治疗,研究还评估了定向转向在临床常规中的长期使用情况。方法对2016年1月1日至2019年12月12日期间植入分段导联的所有患者和相应时间段内植入非分段导联的所有患者进行评估,包括手术前和12个月随访(12MFU)时的人口统计学数据和临床评分,以及12MFU和最后一次随访(LFU)时的刺激参数。根据 DBS 诱导的运动改善情况,将患者分为非常好(60%)、好(30%-60%)和差(30%)三类。结果61/96 例植入分段导联(SEG)的患者和 42/53 例植入非分段导联(N-SEG)的患者均获得了 12MFU 的临床数据。12MFU时,DBS诱导的运动改善和药物减少的平均值在SEG组和N-SEG组之间或转向模式的亚组分析中没有显著差异。与 N-SEG 组相比,SEG 组反应不佳者的比例较低(23% 对 31%),但无统计学意义。结论随访 12 个月时,STN-DBS 不同电极类型在减少运动症状和药物治疗方面的疗效并无差异。随着时间的推移,定向转向的使用会增加,这可能是反应不佳者比例降低的原因。
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引用次数: 0
Role of alpha-synuclein seed amplification assay in Parkinson’s disease clinical trials: A case of misdiagnosis α-突触核蛋白种子扩增测定在帕金森病临床试验中的作用:误诊案例
IF 1.9 Q3 CLINICAL NEUROLOGY Pub Date : 2024-01-01 DOI: 10.1016/j.prdoa.2024.100274
Emily Tharp, Juan D. Martinez-Lemus, Mya C. Schiess, Timothy M. Ellmore, Jessika Suescun, Mohammad Shahnawaz
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引用次数: 0
The effects of Baduanjin on fine motor skills in mild and moderate Parkinson’s disease: A randomized controlled trial 八段锦对轻度和中度帕金森病患者精细运动技能的影响:随机对照试验
IF 1.9 Q3 CLINICAL NEUROLOGY Pub Date : 2024-01-01 DOI: 10.1016/j.prdoa.2024.100276
Ke-Fan Li , Jun Li , A-Long Xia , Xiao-Wei Wang , Ai-Ling Wang , Ying Shi , Huai-Zhen Chen

Background

Fine motor impairment is common in Parkinson’s disease (PD), which reduces patients’ quality of life. There are few suitable targeted treatments. We conducted a clinical trial to determine whether Baduanjin Qigong exercise would increase fine motor skills in PD patients.

Methods

Sixty PD patients (Hoehn-Yahr stage 1–4) with hand fine motor impairment were randomly assigned to the Baduanjin group and the physical activity group. Baduanjin group practiced Baduanjin exercise five times weekly for 40 min (warm-up 5 min, Baduanjin 30 min, cool-down 5 min). The usual physical activity groups maintained their habit of usual physical activities. The participants underwent assessments in the “ON” medication state at baseline and 4-week follow-up time points. The Purdue Pegboard Test (PPT) was used as the primary outcome to assess manual dexterity. The secondary outcomes included the Movement Disorders Society-Unified Parkinson’s Disease Rating Scale, part III (MDS-UPDRS III), and the Parkinson’s disease questionnaire (PDQ-39).

Results

The results of PPT revealed the Baduanjin group showed statistically significant improvement in the “non-dominant hand” and “assembly” scores compared to the usual physical activity group (P < 0.05), but with no significant difference in “dominant hand” and “both hands” (P > 0.05). Additionally, the Baduanjin group showed better performance in the PDQ-39 (P < 0.05).

Conclusion

Our study concludes that a 4-week Baduanjin exercise is effective in improving fine motor function and quality of life in patients with mild and moderate PD. The results suggest a promising intervention to be implemented in community or home settings for managing fine motor impairment in PD.
背景细微运动障碍是帕金森病(PD)的常见症状,会降低患者的生活质量。目前几乎没有合适的针对性治疗方法。我们进行了一项临床试验,以确定八段锦气功运动是否能提高帕金森病患者的精细运动技能。方法将 60 名有手部精细运动障碍的帕金森病患者(Hoehn-Yahr 1-4 期)随机分配到八段锦组和体育锻炼组。八段锦组每周练习五次八段锦,每次40分钟(热身5分钟,八段锦30分钟,冷却5分钟)。平时体育活动组则保持平时体育活动的习惯。参与者在 "开药 "状态下接受基线和四周随访时间点的评估。普渡钉板测试(PPT)是评估手部灵活性的主要结果。结果PPT结果显示,与常规体育锻炼组相比,八段锦组在 "非惯用手 "和 "装配 "得分方面有显著改善(P< 0.05),但在 "惯用手 "和 "双手 "得分方面无显著差异(P> 0.05)。此外,八段锦组在 PDQ-39 中的表现更好(P< 0.05)。结论我们的研究得出结论,为期 4 周的八段锦锻炼能有效改善轻度和中度帕金森病患者的精细运动功能和生活质量。研究结果表明,在社区或家庭环境中实施干预措施以控制帕金森病患者的精细运动障碍是很有前景的。
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引用次数: 0
期刊
Clinical Parkinsonism Related Disorders
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