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The Efficiency of Established Diagnostic and Therapeutic Approaches in the Treatment of Trigeminal Neuralgia 治疗三叉神经痛的既有诊断和治疗方法的效率
IF 2 4区 医学 Q1 Medicine Pub Date : 2024-05-03 DOI: 10.1007/s11940-024-00795-6
Fazladin T. Temurov, Asel A. Biseytova, Bakhytkul A. Ernazarova, Bibifatima D. Mukhambetova, Asan S. Ubaydullaev

Purpose of Review

The research aims to determine the effectiveness of the main approaches in the diagnosis and treatment of trigeminal neuralgia. Analysis, synthesis, comparison, and generalisation were used as methods of studying the object of research.

Recent Findings

According to the results of the study, the most effective method of diagnosis was found to be examination and history taking. Correct performance of these actions allows to find out whether the nature of the patient’s pain is a symptom of trigeminal neuralgia. For this purpose, the location, nature, and triggering factors of the pain are determined. At the same time, pain characteristics are compared to exclude other diseases that are also accompanied by pain in the face. Among the instrumental diagnostic methods, magnetic resonance imaging has proven to be the most effective, allowing to determine the form of neuralgia and its causes. To determine the effectiveness of treatment methods, the parameters of pain relief rate, duration of the therapeutic effect and the presence of side effects were used. The most effective approach in the conservative treatment of trigeminal neuralgia was determined to be a complex drug therapy, including anticonvulsants, muscle relaxants, antispasmodics, and physiotherapy procedures. The most effective method of surgical intervention is microvascular decompression, which provides immediate pain relief in most cases and has the longest-lasting effect compared to other methods.

Summary

Rhizotomy was defined as a less effective procedure, but its advantage was its minimally invasive nature. Even though trigeminal neuralgia is a recurrent disease, the right treatment approach can maximise the duration of remission and return the patient to a normal lifestyle.

综述目的该研究旨在确定诊断和治疗三叉神经痛的主要方法的有效性。研究结果表明,最有效的诊断方法是检查和病史采集。正确执行这些操作可以确定患者疼痛的性质是否为三叉神经痛的症状。为此,要确定疼痛的部位、性质和诱发因素。同时,还要对疼痛特征进行比较,以排除同样伴有面部疼痛的其他疾病。在工具性诊断方法中,磁共振成像被证明是最有效的,可以确定神经痛的形式及其原因。为了确定治疗方法的有效性,使用了疼痛缓解率、疗效持续时间和是否存在副作用等参数。在三叉神经痛的保守治疗中,最有效的方法被确定为复合药物疗法,包括抗惊厥药、肌肉松弛药、解痉药和理疗程序。最有效的手术干预方法是微血管减压术,在大多数病例中,这种方法能立即缓解疼痛,而且与其他方法相比,效果最持久。尽管三叉神经痛是一种复发性疾病,但正确的治疗方法可以最大限度地延长缓解时间,使患者恢复正常的生活方式。
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引用次数: 0
Treatment of Malignant Cerebral Edema in Acute Ischemic Stroke 急性缺血性脑卒中恶性脑水肿的治疗
IF 2 4区 医学 Q1 Medicine Pub Date : 2024-04-19 DOI: 10.1007/s11940-024-00793-8
Maximiliano A. Hawkes, Alejandro A. Rabinstein

Purpose of review

To provide an updated summary on the diagnosis and treatment of patients with malignant cerebral edema after ischemic stroke.

Recent findings

The risk of malignant middle cerebral artery (MCA) stroke is highest in young patients with large vessel occlusion and unsuccessful revascularization. Several scores are available for risk stratification. Treatment includes supportive care, close neurologic monitoring, and hyperosmolar therapy. Yet, the main therapeutic decision is whether to proceed with decompressive craniectomy. Multiple randomized clinical trials and several meta-analyses have demonstrated that decompressive hemicraniectomy is the single most important intervention associated with survival. Survivors may face severe disability regardless of surgical treatment, and the definition of acceptable outcome in this context remains elusive.

Summary

Malignant MCA infarcts are life-threatening and invariably cause disability, most often severe. Neurologic deterioration requires airway management and hyperosmolar therapy. Decompressive hemicraniectomy is a lifesaving procedure; approximately 50% of surgically treated patients younger than 60 years can regain independent ambulation, and one nearly in five may become functionally independent at 1 year. Older patients face a much worse functional prognosis; surgical decisions in these patients should be assessed case by case.

最新研究结果在大血管闭塞和血管再通不成功的年轻患者中,恶性大脑中动脉(MCA)卒中的风险最高。有几种评分方法可用于风险分层。治疗包括支持性护理、密切的神经监测和高渗治疗。然而,治疗的主要决定因素是是否进行颅骨减压切除术。多项随机临床试验和数项荟萃分析表明,减压性半颅骨切除术是与存活率相关的最重要干预措施。无论手术治疗与否,幸存者都可能面临严重残疾,在这种情况下,可接受结果的定义仍然难以确定。摘要 恶性 MCA 梗死危及生命,必然导致残疾,通常是严重残疾。神经系统恶化需要气道管理和高渗治疗。减压性半颅骨切除术是一种挽救生命的手术;在接受过手术治疗的 60 岁以下患者中,约有 50% 的患者可以重新独立行走,近五分之一的患者可以在 1 年后实现功能独立。高龄患者的功能预后要差得多;对这些患者的手术决定应根据具体情况进行评估。
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引用次数: 0
Updates on Facioscapulohumeral Muscular Dystrophy (FSHD) 面阔肱肌营养不良症 (FSHD) 的最新进展
IF 2 4区 医学 Q1 Medicine Pub Date : 2024-04-02 DOI: 10.1007/s11940-024-00790-x
Amanda X. Y. Chin, Zhi Xuan Quak, Yee Cheun Chan, Amy M. L. Quek, Kay W. P. Ng

Purpose of review

This review aims to provide a summary of the pathophysiology, clinical presentation and management options for facioscapulohumeral dystrophy (FSHD). We discuss current management options and delve into updates about developments in targeted therapy.

Recent findings

New breakthroughs in FSHD research have led to a further understanding of aberrant DUX4 protein expression in the underlying pathophysiology of FSHD. This has paved the way for the development of targeted therapies aimed at targeting DUX4 expression or its downstream effects. Therapeutic strategies for FSHD primarily target DUX4 through three main avenues: small molecules, antisense oligonucleotide therapeutics and CRISPR-based approaches. This review discusses these strategies further. Presently, all prospective targeted therapies are in the pre-clinical phase, except for losmapimod, which is currently undergoing a phase 3 clinical trial.

Summary

Given the absence of approved disease-modifying treatments for FSHD, the primary approach for management currently involves multidisciplinary supportive measures which are limited. Recent developments in the form of targeted therapies and strategies for the definitive treatment of FSHD indicate a promising era.

综述目的本综述旨在概述面岬肱肌营养不良症(FSHD)的病理生理学、临床表现和治疗方案。我们讨论了当前的治疗方案,并深入探讨了靶向治疗的最新进展。最新研究结果FSHD研究的新突破使人们进一步了解了DUX4蛋白的异常表达在FSHD潜在病理生理学中的作用。这为开发针对 DUX4 表达或其下游效应的靶向疗法铺平了道路。前列腺增生症的治疗策略主要通过三种途径靶向 DUX4:小分子药物、反义寡核苷酸疗法和基于 CRISPR 的方法。本综述将进一步讨论这些策略。目前,除了正在进行3期临床试验的losmapimod外,所有前瞻性靶向疗法均处于临床前阶段。摘要鉴于FSHD尚无获批的疾病改变疗法,目前的主要治疗方法包括多学科支持性措施,但这些措施效果有限。靶向疗法和FSHD确切治疗策略的最新进展预示着一个充满希望的时代即将到来。
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引用次数: 0
Update on Treatment of Idiopathic (and Non-Idiopathic) Orbital Inflammation 特发性(和非特发性)眼眶炎症的最新治疗方法
IF 2 4区 医学 Q1 Medicine Pub Date : 2024-03-26 DOI: 10.1007/s11940-024-00788-5
Elana Meer, Michael K. Yoon, Jonathan E. Lu

Purpose of review

This review aims to provide a critical appraisal of current diagnostic and therapeutic strategies for patients with orbital inflammatory disease (OID). We present the reader with a review of clinical, imaging, laboratory, and biopsy based assessment of OID and review the current treatment modalities utilized including corticosteroids, corticosteroid-sparing (immunomodulatory) agents, radiation, antibiotics, and disease specific therapy.

Recent findings

Two major developments and trends have emerged in the management of orbital inflammation. First, improved understanding and distinction of inflammation subtypes (myositis, dacryoadenitis, or infiltrative) allows for more nuanced workup and treatment. Second, immunomodulatory agents have shown promise in achieving disease control in cases of truly idiopathic or corticosteroid-resistant OID. Together, these advances have led to fewer adverse effects and better efficacy.

Summary

The optimal treatment of OID depends on distinguishing between nonspecific and specific inflammation. Nonspecific inflammation tends to respond to corticosteroid therapy with a lower chance of relapse, while specific orbital inflammation often requires targeting the underlying disease with steroid-sparing therapy and immunomodulatory agents.

综述目的 本综述旨在对眼眶炎症性疾病(OID)患者目前的诊断和治疗策略进行批判性评估。我们向读者介绍了眼眶炎症的临床、影像学、实验室和活组织检查评估,并回顾了目前使用的治疗方法,包括皮质类固醇、皮质类固醇辅助(免疫调节)剂、放射线、抗生素和疾病特异性疗法。首先,人们对炎症亚型(肌炎、泪腺炎或浸润性炎症)的认识和区分有所提高,从而可以进行更细致的检查和治疗。其次,免疫调节药物在真正的特发性或皮质类固醇耐药的 OID 病例中有望实现疾病控制。总结OID的最佳治疗取决于区分非特异性炎症和特异性炎症。非特异性炎症往往对皮质类固醇治疗有反应,复发几率较低,而特异性眼眶炎症往往需要针对潜在疾病使用类固醇节省疗法和免疫调节药物。
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引用次数: 0
Distinguishing Benign Rashes From Severe Skin Reactions From Anti-Seizure Medications 区分良性皮疹和抗癫痫药物引起的严重皮肤反应
IF 2 4区 医学 Q1 Medicine Pub Date : 2024-03-21 DOI: 10.1007/s11940-024-00785-8
Ram Mani, Ahmad Almelegy, Thu Minh Truong, Gaurav N. Pathak, Mary L. Wagner, Cindy Wassef

Purpose of review

This review describes risk factors for severe skin reactions to antiseizure medications (ASMs), the usage of updated tests to predict those with increased risk of a severe cutaneous reaction, and guides how to choose specific ASMs and dosing to lower the risk for these reactions. Information is given regarding specific mild versus severe reactions, initial diagnostic evaluation, and treatment. A table listing the risk of mild and severe cutaneous reaction risks as well as the management of potential seizures that may occur while stopping the culprit ASM are provided.

Recent findings

Five new ASMs have joined the total of 26 FDA-approved ASMs since 2018. Cenobamate had three patients develop a drug reaction with eosinophilia and systemic symptoms. A lower starting dosing and slower titration have resulted in no further published cases. Based on limited data, rash risk is low for fenfluramine, ganaxalone, and stiripentol. It is low-moderate for Epidiolex. Molecular tests can predict severe reactions.

Summary

Skin reactions are a relatively common side effect of ASMs with aromatic ASMs having the greatest risk. Identifying and informing high-risk patients when to seek medical attention, stopping the culprit ASM when a severe reaction looks possible, and providing appropriate medical triage can reduce morbidity and mortality from severe skin and systemic reactions.

综述目的 本综述介绍了抗癫痫药物(ASMs)引起严重皮肤反应的风险因素、使用最新检测方法预测严重皮肤反应风险增加的患者,并指导如何选择特定的 ASMs 和剂量以降低这些反应的风险。还提供了有关特定轻度反应与严重反应、初步诊断评估和治疗的信息。提供了一份表格,列出了轻度和严重皮肤反应风险,以及在停用罪魁祸首 ASM 时可能出现的潜在癫痫发作的处理方法。近期发现自 2018 年以来,已有五种新的 ASM 加入了 FDA 批准的 26 种 ASM 的行列。塞诺巴马特有3名患者出现药物反应,伴有嗜酸性粒细胞增多和全身症状。由于起始剂量较低,滴定速度较慢,因此未再出现已公布的病例。根据有限的数据,芬氟拉明、甘纳沙隆和司替潘托的皮疹风险较低。Epidiolex的皮疹风险为中低水平。总结皮肤反应是 ASMs 比较常见的副作用,其中芳香族 ASMs 的风险最大。识别并告知高危患者何时就医,在可能出现严重反应时停止使用致病的 ASM,并提供适当的医疗分流可降低严重皮肤和全身反应的发病率和死亡率。
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引用次数: 0
Management Strategies for Atypical Parkinsonism 非典型帕金森病的管理策略
IF 2 4区 医学 Q1 Medicine Pub Date : 2024-03-21 DOI: 10.1007/s11940-024-00787-6
Vasilios C. Constantinides, Nikolaos Giagkou, Maria-Evgenia Brinia, Christos Koros, Leonidas Stefanis, Maria Stamelou

Purpose of Review

Atypical parkinsonism is a term usually used to describe three neurodegenerative parkinsonian disorders: progressive supranuclear palsy (PSP), corticobasal degeneration (CBD), and multiple system atrophy (MSA). In contrast to Parkinson’s disease, these disorders have a poor prognosis and present with a multitude of diverse symptoms, including parkinsonism, dystonia, myoclonus, gait disorders, dysarthria, dysphagia, sleep, cognitive, and behavioral disorders. In the absence of an approved disease-modifying treatment, symptomatic treatment is the mainstay of management. The purpose of this review is to present an overview of the management of these complex disorders, with a particular focus on a holistic and multidisciplinary approach.

Recent Findings

In addition to presenting the most significant pharmacological interventions for symptom management, data regarding non-pharmacological interventions are analyzed. Important non-pharmacological clinical practice questions such as breaking the news, palliative care, and end-of-life issues are discussed, in an effort to present an overview of the management of atypical parkinsonism from diagnosis to the most advanced stages of these diseases.

Summary

Management of atypical parkinsonian disorders includes symptomatic pharmacological and non-pharmacological interventions, in addition to addressing issues such as informing the patient of the diagnosis, palliative care, and end-of-life issues, which require a multidisciplinary approach.

综述目的 非典型帕金森病通常用于描述三种神经退行性帕金森病:进行性核上性麻痹(PSP)、皮质基底变性(CBD)和多系统萎缩(MSA)。与帕金森病相比,这些疾病的预后较差,并表现出多种多样的症状,包括帕金森病、肌张力障碍、肌阵挛、步态障碍、构音障碍、吞咽困难、睡眠、认知和行为障碍。在没有获得批准的改变病情的治疗方法的情况下,对症治疗是主要的治疗手段。本综述旨在概述这些复杂疾病的治疗方法,并特别关注整体和多学科治疗方法。小结非典型帕金森病的治疗包括对症药物治疗和非药物治疗干预,此外还包括告知患者诊断、姑息治疗和临终关怀等问题,这些都需要采用多学科方法来解决。
{"title":"Management Strategies for Atypical Parkinsonism","authors":"Vasilios C. Constantinides, Nikolaos Giagkou, Maria-Evgenia Brinia, Christos Koros, Leonidas Stefanis, Maria Stamelou","doi":"10.1007/s11940-024-00787-6","DOIUrl":"https://doi.org/10.1007/s11940-024-00787-6","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Purpose of Review</h3><p>Atypical parkinsonism is a term usually used to describe three neurodegenerative parkinsonian disorders: progressive supranuclear palsy (PSP), corticobasal degeneration (CBD), and multiple system atrophy (MSA). In contrast to Parkinson’s disease, these disorders have a poor prognosis and present with a multitude of diverse symptoms, including parkinsonism, dystonia, myoclonus, gait disorders, dysarthria, dysphagia, sleep, cognitive, and behavioral disorders. In the absence of an approved disease-modifying treatment, symptomatic treatment is the mainstay of management. The purpose of this review is to present an overview of the management of these complex disorders, with a particular focus on a holistic and multidisciplinary approach.</p><h3 data-test=\"abstract-sub-heading\">Recent Findings</h3><p>In addition to presenting the most significant pharmacological interventions for symptom management, data regarding non-pharmacological interventions are analyzed. Important non-pharmacological clinical practice questions such as breaking the news, palliative care, and end-of-life issues are discussed, in an effort to present an overview of the management of atypical parkinsonism from diagnosis to the most advanced stages of these diseases.</p><h3 data-test=\"abstract-sub-heading\">Summary</h3><p>Management of atypical parkinsonian disorders includes symptomatic pharmacological and non-pharmacological interventions, in addition to addressing issues such as informing the patient of the diagnosis, palliative care, and end-of-life issues, which require a multidisciplinary approach.</p>","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"25 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140201950","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Treatment Approaches in Pediatric Relapsing Autoimmune Encephalitis 小儿复发性自身免疫性脑炎的治疗方法
IF 2 4区 医学 Q1 Medicine Pub Date : 2024-03-21 DOI: 10.1007/s11940-024-00786-7

Abstract

Purpose of review

Autoimmune encephalitis (AE) is increasingly recognized as a treatable cause of encephalitis in children. While prior observational studies demonstrate improved motor outcomes with early immunotherapy, less is known about long-term management and treatment for relapsing disease. In this review, we present current treatment approaches to pediatric AE, in particular relapse risk and treatment for relapsing AE in children.

Recent findings

A recent meta-analysis of anti-NMDAR encephalitis demonstrated that disease onset in adolescence was associated with an increased odds of relapse whereas treatment with rituximab and IVIG for 6 months or longer were associated with a non-relapsing course. However, no specific pediatric sub-analyses were reported. A single-center study on adult and pediatric AE showed that rituximab use was associated with a reduction in time to relapse and recurring relapses although the data for the pediatric cohort did not achieve statistical significance.

Summary

The use of second-line immunotherapy during the initial attack may reduce the risk for relapsing disease in pediatric AE. Larger studies are needed to investigate relapse risk and treatment in both anti-NMDAR and non-NMDAR encephalitis in children.

摘要 综述目的 越来越多的人认识到,自身免疫性脑炎(AE)是一种可治疗的儿童脑炎病因。先前的观察性研究表明,早期免疫治疗可改善运动能力,但对复发疾病的长期管理和治疗却知之甚少。在本综述中,我们将介绍目前治疗小儿脑炎的方法,尤其是复发风险和儿童复发性脑炎的治疗方法。 最近的研究结果 最近一项关于抗 NMDAR 脑炎的荟萃分析表明,青少年时期发病与复发几率增加有关,而使用利妥昔单抗和 IVIG 治疗 6 个月或更长时间与病程不复发有关。不过,没有具体的儿科子分析报告。一项针对成人和儿童 AE 的单中心研究显示,使用利妥昔单抗与复发时间缩短和复发次数减少有关,但儿童队列的数据未达到统计学意义。 总结 在初次发病时使用二线免疫疗法可能会降低小儿 AE 复发的风险。需要进行更大规模的研究,以调查儿童抗NMDAR和非NMDAR脑炎的复发风险和治疗方法。
{"title":"Treatment Approaches in Pediatric Relapsing Autoimmune Encephalitis","authors":"","doi":"10.1007/s11940-024-00786-7","DOIUrl":"https://doi.org/10.1007/s11940-024-00786-7","url":null,"abstract":"<h3>Abstract</h3> <span> <h3>Purpose of review</h3> <p>Autoimmune encephalitis (AE) is increasingly recognized as a treatable cause of encephalitis in children. While prior observational studies demonstrate improved motor outcomes with early immunotherapy, less is known about long-term management and treatment for relapsing disease. In this review, we present current treatment approaches to pediatric AE, in particular relapse risk and treatment for relapsing AE in children.</p> </span> <span> <h3>Recent findings</h3> <p>A recent meta-analysis of anti-NMDAR encephalitis demonstrated that disease onset in adolescence was associated with an increased odds of relapse whereas treatment with rituximab and IVIG for 6 months or longer were associated with a non-relapsing course. However, no specific pediatric sub-analyses were reported. A single-center study on adult and pediatric AE showed that rituximab use was associated with a reduction in time to relapse and recurring relapses although the data for the pediatric cohort did not achieve statistical significance.</p> </span> <span> <h3>Summary</h3> <p>The use of second-line immunotherapy during the initial attack may reduce the risk for relapsing disease in pediatric AE. Larger studies are needed to investigate relapse risk and treatment in both anti-NMDAR and non-NMDAR encephalitis in children.</p> </span>","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"12 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140201959","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Ethical Considerations in the Treatment of Cerebrovascular Disease 治疗脑血管疾病的伦理考量
IF 2 4区 医学 Q1 Medicine Pub Date : 2024-03-15 DOI: 10.1007/s11940-024-00789-4
Nathan A. Shlobin

Purpose of Review

To care for patients with cerebrovascular disease (CVD), neurointerventionalists, intensivists, and other healthcare providers must be equipped to address associated ethical challenges. This review aims to delineate the applicability of fundamental bioethical approaches to CVD, highlight key ethical issues in CVD care, and delineate an ethical framework to streamline ethical decision-making for people with CVD.

Recent Findings

Three introductory cases are presented. The four key principles of principalism and the approach of narrative ethics are described with reference to CVD. Key ethical considerations include decision-making capacity and informed consent, uncertainty, and resource allocation. A categorization of CVD as emergent/nonemergent and the recommended management as intervention/no intervention helps frame the spectrum of CVD. A different six-pathway may then be taken based on which category the patient case corresponds to.

Summary

Physicians involved in the care of people with cerebrovascular disease must understand how the ethical issues manifest in individual patient cases to ensure appropriate care. The aforementioned ethical framework may aid physicians in providing ethically sound care. All decisions must involve a balance between clinical expertise and patient values and preferences or those articulated by a surrogate to properly respect the wishes of patients with CVD.

综述目的为了护理脑血管疾病(CVD)患者,神经介入医生、重症监护医生和其他医疗服务提供者必须具备应对相关伦理挑战的能力。本综述旨在阐明基本生物伦理方法对 CVD 的适用性,强调 CVD 护理中的关键伦理问题,并勾勒出一个伦理框架,以简化 CVD 患者的伦理决策。结合心血管疾病介绍了主体论的四项关键原则和叙事伦理学的方法。主要的伦理考虑因素包括决策能力和知情同意、不确定性和资源分配。将心血管疾病分为急症/非急症,建议的管理分为干预/不干预,有助于确定心血管疾病的范围。小结参与护理脑血管疾病患者的医生必须了解伦理问题在个别患者病例中的表现形式,以确保提供适当的护理。上述伦理框架可帮助医生提供符合伦理的护理。所有决定都必须兼顾临床专业知识与患者的价值观和偏好,或由代理人阐明的价值观和偏好,以适当尊重心血管疾病患者的意愿。
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引用次数: 0
Early Mobilization in Neurocritical Care 神经重症监护中的早期动员
IF 2 4区 医学 Q1 Medicine Pub Date : 2024-02-06 DOI: 10.1007/s11940-023-00779-y
Kristen Nobles, Kiersten Norby, Kristina Small, Monisha A. Kumar

Purpose of Review

Bed rest was a treatment recommended for critically ill patients admitted to the intensive care unit (ICU) that aimed to minimize energy expenditure, permit wound healing and minimize somatic stressors. However, evidence demonstrates that bed rest leads to disuse atrophy, which may be compounded by premorbid sarcopenia and ICU-acquired weakness (ICUAW). ICUAW partly results from muscle breakdown and systemic inflammation and may exacerbate critical illness. Coupled with analgosedation, ICUAW may prolong mechanical ventilation (Kho et al. in BMJ Open Respir Res. 2019;6(1) 2019; Maffei et al. in Arch Phys Med Rehabil. 2017;98 2017; McWilliams et al. in J Crit Care. 2018;44 2018; Sarfati et al. in J Crit Care. 2018;46 2018), increase risk of venous thromboembolism (Denehy et al. in Intensive Care Med. 2017;43(1) 2017; Lyles in J Am Geriatr Soc. 1988;36(11) 1988) create dependence on vasopressor agents (Lyles in J Am Geriatr Soc. 1988;36(11) 1988; Fortney et al. in Comprehen Physiol. 1996) restrict joint mobility, and induce pressure injuries. Neurologically injured patients may be at a higher risk of ICUAW than other critically-ill patients, given that neurological injury itself results in weakness, which may be focal or generalized. Early mobilization (EM), typically defined as physical therapy within 72 h of ICU (Cumming et al. in Neurology. 2019;93(7) 2019), may preempt or mitigate these deleterious consequences of critical care.

Recent Findings

Retrospective data suggest that EM protocols reduce ventilator days, decrease venous thromboembolism, avert pressure wounds, and reduce the length of stay. EM may reduce the incidence, duration, and severity of delirium (Morris et al. in Crit Care Med. 2008;36(8) 2008; Needham et al. in Arch Phys Med Rehabil. 2010;91(4) 2010). Larger and more rigorous studies have not demonstrated benefit of EM on outcomes after critical care; some have demonstrated harm. Neurologically injured critical care patients have generally been excluded from early mobilization protocols due to safety concerns that stem from the increased potential for falls, disorders of consciousness, cognitive impairment, intracranial hypertension, and potential dislodgment of intracranial devices. Notably, data from patients with ischemic stroke suggest that EM may also be associated with harm in this group.

Summary

EM may benefit neurologically injured patients once acute ischemia, elevated ICP, and seizures are resolved. Targeting moderate acuity patients may be critical to improving outcomes and optimizing resource utilization in this resource-intensive intervention. The duration of mobility session, optimal frequency of mobility session, and timing of session remain to be determined.

综述目的卧床休息是重症监护室(ICU)建议对重症患者采取的一种治疗方法,旨在最大限度地减少能量消耗、促进伤口愈合并减少躯体压力。然而,有证据表明,卧床休息会导致废用性萎缩,而病前肌少症和重症监护室获得性乏力(ICUAW)可能会加重这种情况。ICUAW 的部分原因是肌肉分解和全身炎症,可能会加重危重病人的病情。加上镇痛,ICUAW 可能会延长机械通气时间(Kho 等人,发表于 BMJ Open Respir Res. 2019;6(1) 2019;Maffei 等人,发表于 Arch Phys Med Rehabil.2017;98 2017; McWilliams et al. in J Crit Care.2018;44 2018; Sarfati et al. in J Crit Care.2018;46 2018),增加静脉血栓栓塞的风险(Denehy 等,载于 Intensive Care Med.2017;43(1)2017;Lyles 发表于 J Am Geriatr Soc. 1988;36(11)1988),造成对血管加压药的依赖(Lyles 发表于 J Am Geriatr Soc. 1988;36(11)1988;Fortney 等人发表于 Comprehen Physiol.由于神经损伤本身会导致虚弱,而虚弱可能是局灶性的,也可能是全身性的,因此神经损伤患者发生 ICUAW 的风险可能高于其他重症患者。早期动员(EM)通常是指在进入 ICU 72 小时内进行物理治疗(Cumming 等人,发表于 Neurology.最近的研究结果回顾性数据表明,EM 方案可减少呼吸机天数、减少静脉血栓栓塞、避免压伤并缩短住院时间。急救可降低谵妄的发生率、持续时间和严重程度(Morris 等,发表于《重症监护医学》(Crit Care Med.2008;36(8)2008;Needham 等人在 Arch Phys Med Rehabil.2010;91(4) 2010).更大规模和更严格的研究并未证明电磁疗法对危重症护理后的预后有益;有些研究则证明电磁疗法有害。神经系统受伤的危重症患者通常被排除在早期移动方案之外,这是因为跌倒、意识障碍、认知障碍、颅内高压和颅内装置可能脱落的可能性增加,因而存在安全隐患。值得注意的是,来自缺血性中风患者的数据表明,EM 也可能会对这部分患者造成伤害。摘要一旦急性缺血、ICP 升高和癫痫发作得到缓解,EM 可能会使神经损伤患者受益。在这种资源密集型干预中,针对中等严重程度的患者可能是改善预后和优化资源利用的关键。活动治疗的持续时间、活动治疗的最佳频率以及治疗时机仍有待确定。
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引用次数: 0
Update on Amyloid Polyneuropathy and Treatment 淀粉样蛋白多发性神经病和治疗的最新进展
IF 2 4区 医学 Q1 Medicine Pub Date : 2024-02-01 DOI: 10.1007/s11940-024-00780-z

Abstract

Purpose of Review

The purpose of this review is to summarize currently available and developing diagnostic and treatment options for hereditary transthyretin amyloid polyneuropathy. Transthyretin amyloidosis (ATTR) predominantly manifests with cardiomyopathy and/or peripheral neuropathy, but amyloid deposits may be found in other organs or tissues.

Recent Findings

Currently available treatments include transthyretin gene silencers (for hereditary ATTR peripheral neuropathy only) and transthyretin stabilizers (tafamidis for ATTR cardiomyopathy in the USA, and for both hereditary ATTR peripheral neuropathy and ATTR cardiomyopathy in Europe, Japan, Brazil, and some other countries), and liver transplantation. Gene silencers stop the progression of hereditary ATTR peripheral neuropathy in most patients, and transthyretin stabilizers reduce hospitalizations and mortality in patients with ATTR cardiomyopathy. The use of liver transplantation for ATTR has declined with the availability of more effective therapies, and shortage of available allografts. On the horizon are new treatments already in clinical trials including new gene silencers and gene editing agents, new transthyretin stabilizers, and amyloid removal treatments.

Summary

Recently approved treatments for ATTR have changed its natural history, and additional medications may get approved in the near future. Early diagnosis is still essential to improve treatment outcomes. New management strategies may include combinations of gene silencers, transthyretin stabilizers, gene editing, and amyloid removal agents, but the cost may become the limiting factor.

摘要 综述目的 本综述旨在总结遗传性转甲状腺素淀粉样多发性神经病的现有和正在开发的诊断和治疗方案。转甲状腺素淀粉样变性(ATTR)主要表现为心肌病和/或周围神经病变,但在其他器官或组织中也可能发现淀粉样沉积。 最新发现 目前可用的治疗方法包括转甲状腺素基因沉默剂(仅用于遗传性 ATTR 周围神经病变)和转甲状腺素稳定剂(在美国用于 ATTR 心肌病的他法米迪,在欧洲、日本、巴西和其他一些国家用于遗传性 ATTR 周围神经病变和 ATTR 心肌病),以及肝脏移植。基因沉默剂可阻止大多数患者的遗传性ATTR周围神经病变的发展,转甲状腺素稳定剂可减少ATTR心肌病患者的住院时间和死亡率。随着更有效疗法的出现和同种异体移植的短缺,肝移植治疗ATTR的使用率已经下降。新的治疗方法已经进入临床试验阶段,包括新的基因沉默剂和基因编辑剂、新的转甲状腺素稳定剂和淀粉样蛋白清除疗法。 小结 最近获批的 ATTR 治疗方法改变了 ATTR 的自然病史,不久的将来可能还会有更多药物获批。要改善治疗效果,早期诊断仍然至关重要。新的治疗策略可能包括基因沉默剂、转甲状腺素稳定剂、基因编辑和淀粉样蛋白清除剂的组合,但成本可能成为限制因素。
{"title":"Update on Amyloid Polyneuropathy and Treatment","authors":"","doi":"10.1007/s11940-024-00780-z","DOIUrl":"https://doi.org/10.1007/s11940-024-00780-z","url":null,"abstract":"<h3>Abstract</h3> <span> <h3>Purpose of Review</h3> <p>The purpose of this review is to summarize currently available and developing diagnostic and treatment options for hereditary transthyretin amyloid polyneuropathy. Transthyretin amyloidosis (ATTR) predominantly manifests with cardiomyopathy and/or peripheral neuropathy, but amyloid deposits may be found in other organs or tissues.</p> </span> <span> <h3>Recent Findings</h3> <p>Currently available treatments include transthyretin gene silencers (for hereditary ATTR peripheral neuropathy only) and transthyretin stabilizers (tafamidis for ATTR cardiomyopathy in the USA, and for both hereditary ATTR peripheral neuropathy and ATTR cardiomyopathy in Europe, Japan, Brazil, and some other countries), and liver transplantation. Gene silencers stop the progression of hereditary ATTR peripheral neuropathy in most patients, and transthyretin stabilizers reduce hospitalizations and mortality in patients with ATTR cardiomyopathy. The use of liver transplantation for ATTR has declined with the availability of more effective therapies, and shortage of available allografts. On the horizon are new treatments already in clinical trials including new gene silencers and gene editing agents, new transthyretin stabilizers, and amyloid removal treatments.</p> </span> <span> <h3>Summary</h3> <p>Recently approved treatments for ATTR have changed its natural history, and additional medications may get approved in the near future. Early diagnosis is still essential to improve treatment outcomes. New management strategies may include combinations of gene silencers, transthyretin stabilizers, gene editing, and amyloid removal agents, but the cost may become the limiting factor.</p> </span>","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"3 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139662289","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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Current Treatment Options in Neurology
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