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Omaveloxolone for the treatment of Friedreich ataxia: clinical trial results and practical considerations. 治疗弗里德里希共济失调的奥马韦洛酮:临床试验结果和实际考虑因素。
IF 4.3 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-03-01 Epub Date: 2024-01-30 DOI: 10.1080/14737175.2024.2310617
David R Lynch, Susan Perlman, Kim Schadt

Introduction: Omavaloxolone, an NRF2 activator, recently became the first drug approved specifically for the treatment of Friedreich ataxia (FRDA). This landmark achievement provides a background for a review of the detailed data leading to the approval.

Areas covered: The authors review the data from the 4 major articles on FRDA in the context of the authors' considerable (>1000 patients) experience in treating individuals with FRDA. The data is presented in the context not only of its scientific meaning but also in the practical context of therapy in FRDA.

Expert opinion: Omaveloxolone provides a significant advance in the treatment of FRDA that is likely to be beneficial in a majority of the FRDA population. The data suggesting a benefit is consistent, and adverse issues are relatively modest. The major remaining questions are the subgroups that are most responsive and how long the beneficial effects will remain significant in FRDA patients.

简介奥马伐洛隆是一种NRF2激活剂,它最近成为首个获准用于治疗弗里德里希共济失调症(FRDA)的药物。这一里程碑式的成就为我们回顾导致该药物获批的详细数据提供了背景:作者结合自己治疗弗里德里希共济失调(FRDA)患者的丰富经验(超过 1000 名患者),回顾了有关 FRDA 的 4 篇主要文章中的数据。这些数据不仅具有科学意义,还结合了 FRDA 治疗的实际情况:Omaveloxolone 在治疗 FRDA 方面取得了重大进展,可能对大多数 FRDA 患者有益。表明该药有益的数据是一致的,不良反应相对较少。剩下的主要问题是对哪些亚组反应最强烈,以及 FRDA 患者的获益效应将在多长时间内保持显著。
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引用次数: 0
Are pharmacotherapeutics effective for treating aphasia? 药物治疗对治疗失语症有效吗?
IF 4.3 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-03-01 Epub Date: 2024-02-07 DOI: 10.1080/14737175.2024.2313557
Guadalupe Dávila, Marcelo L Berthier

Introduction: Aphasia is a communication disorder resulting from stroke and/or neurodegenerative conditions which involve the left cerebral hemisphere. It is a debilitating disorder affecting a person's ability to speak, understand, read, and write. Its impact on daily life necessitates therapeutic strategies to aid patients with aphasia.

Areas covered: In this special report, the authors speculate whether current pharmacotherapeutic strategies are effective in treating aphasia. The authors look at aphasia caused by different conditions and how this could impact therapy before providing the reader with their expert perspectives. The aim of this paper is for the reader to gain a clearer understanding of the efficacy of the current pharmacotherapeutic treatment paradigms as well as potential future developments.

Expert opinion: The exploration of pharmacotherapy for aphasia in vascular brain disorders and neurodegenerative diseases has received much attention in recent years with various therapeutic strategies having been put forward. In terms of whether pharmacotherapy is effective for the treatment of aphasia, there is still no clear-cut answer. Further research is needed with more studies requiring a greater emphasis on language and communication deficits. Biomarkers may also help clinicians provide their patients with a more personalized treatment plan.

简介失语症是一种由中风和/或涉及左侧大脑半球的神经退行性疾病引起的交流障碍。它是一种影响人说话、理解、阅读和书写能力的衰弱性障碍。由于它对日常生活的影响,有必要制定治疗策略来帮助失语症患者:在这份特别报告中,作者推测目前的药物治疗策略是否能有效治疗失语症。在向读者提供他们的专家观点之前,作者们探讨了由不同病症引起的失语症,以及这可能对治疗产生的影响。本文旨在让读者更清楚地了解当前药物治疗范例的疗效以及未来可能的发展:近年来,脑血管疾病和神经退行性疾病失语症的药物治疗探索备受关注,各种治疗策略层出不穷。至于药物疗法是否能有效治疗失语症,目前还没有明确的答案。还需要进一步的研究,更多的研究需要更加重视语言和交流障碍。生物标志物还可以帮助临床医生为患者提供更加个性化的治疗方案。
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引用次数: 0
The management of Neurofibromatosis type 1 (NF1) in children and adolescents 儿童和青少年神经纤维瘤病 1 型 (NF1) 的管理
IF 4.3 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-02-26 DOI: 10.1080/14737175.2024.2324117
Nino Kerashvili, David H. Gutmann
Neurofibromatosis type 1 (NF1) is a rare neurogenetic disorder characterized by multiple organ system involvement and a predisposition to the development of benign and malignant tumors. With revisi...
神经纤维瘤病 1 型(NF1)是一种罕见的神经遗传性疾病,其特点是累及多个器官系统,并易发展成良性和恶性肿瘤。随着研究的不断深入...
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引用次数: 0
Careful considerations for the treatment of posttraumatic stress disorder during and following pregnancy. 妊娠期间和妊娠后治疗创伤后应激障碍的注意事项。
IF 4.3 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-02-01 Epub Date: 2024-02-06 DOI: 10.1080/14737175.2024.2303430
Casey Oliver, Erika Puiras, Verinder Sharma, Dwight Mazmanian

Introduction: The focus on perinatal mental health has expanded recently, though there is less research on post-traumatic stress disorder (PTSD). Therefore, a review of the literature was undertaken and coupled with expert clinical insights to discuss current clinical practice recommendations for PTSD in the perinatal period.

Areas covered: This review covers considerations for the assessment, prevention, and treatment of PTSD during the perinatal period. Within these sections, evidence-based and promising practices are outlined. Extra attention is afforded to treatment, which includes considerations from both psychotherapeutic and psychopharmacological perspectives. This review closes with coverage of three important and related areas of consideration, including bereavement, intimate partner violence, and childhood sexual abuse.

Expert opinion: Psychotherapeutic interventions for PTSD during pregnancy are limited, and no strong recommendations can be supported at this time while evidence points toward the effectiveness of cognitive behavioral therapies and eye movement desensitization therapy as first-line treatments postpartum though research evidence is also limited. Research on psychopharmacological interventions is similarly scarce, though selective serotonin reuptake inhibitors may be beneficial. Clinicians should also be mindful of additional considerations that may be needed for the treatment of PTSD in the context of bereavement, intimate partner violence, and history of sexual violence.

简介尽管有关创伤后应激障碍(PTSD)的研究较少,但对围产期心理健康的关注近来有所扩大。因此,我们对相关文献进行了综述,并结合专家的临床见解,讨论了目前针对围产期创伤后应激障碍的临床实践建议:本综述涉及围产期创伤后应激障碍的评估、预防和治疗。在这些章节中,概述了以证据为基础的实践和有前途的实践。对治疗给予了特别关注,包括从心理治疗和精神药理学角度的考虑。本综述最后介绍了三个重要的相关领域,包括丧亲之痛、亲密伴侣暴力和儿童性虐待:妊娠期创伤后应激障碍的心理治疗干预措施有限,目前尚无有力的建议支持,但有证据表明认知行为疗法和眼动脱敏疗法作为产后一线治疗的有效性,尽管研究证据也很有限。有关精神药物干预的研究同样很少,但选择性血清素再摄取抑制剂可能有益。临床医生还应该注意在治疗创伤后应激障碍时可能需要额外考虑的丧亲、亲密伴侣暴力和性暴力史等因素。
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引用次数: 0
Postoperative analgesic options after spine surgery: finding the optimal treatment strategies. 脊柱手术后的术后镇痛选择:寻找最佳治疗策略。
IF 4.3 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-02-01 Epub Date: 2024-02-06 DOI: 10.1080/14737175.2023.2298824
Alina Razak, Benjamin Corman, John Servider, Ana Mavarez-Martinez, Zhaosheng Jin, Harry Mushlin, Sergio D Bergese

Introduction: Spine surgery is one of the most common types of surgeries performed in the United States; however, managing postoperative pain following spine surgery has proven to be challenging. Patients with spine pathologies have higher incidences of chronic pain and resultant opioid use and potential for tolerance. Implementing a multimodal plan for postoperative analgesia after spine surgery can lead to enhanced recovery and outcomes.

Areas covered: This review presents several options for analgesia following spine surgery with an emphasis on multimodal techniques to best aid this specific patient population. In addition to traditional therapeutics, such as acetaminophen, non-steroidal anti-inflammatory medications, and opioids, we discuss intrathecal morphine administration and emerging regional anesthesia techniques.

Expert opinion: Several adjuncts to improve analgesia following spine surgery are efficacious in the postoperative period. Intrathecal morphine provides sustained analgesia and can be instilled intraoperatively by the surgical team under direct visualization. Local anesthetics deposited under ultrasound guidance by an anesthesiologist trained in regional techniques also provide the opportunity for single injections or continuous analgesia via an indwelling catheter.

导言:脊柱手术是美国最常见的手术类型之一;然而,脊柱手术后的术后疼痛管理已被证明具有挑战性。脊柱病变患者的慢性疼痛发生率较高,因此需要使用阿片类药物并可能产生耐受性。在脊柱手术后实施多模式术后镇痛计划可促进康复并提高疗效:本综述介绍了脊柱手术后镇痛的几种选择,重点是多模式技术,以便为这一特殊患者群体提供最佳帮助。除了对乙酰氨基酚、非甾体抗炎药物和阿片类药物等传统疗法外,我们还讨论了鞘内吗啡给药和新兴的区域麻醉技术:专家观点:脊柱手术后,有几种改善镇痛的辅助药物在术后具有疗效。鞘内吗啡可提供持续镇痛,并可由手术团队在直视下进行术中灌注。由受过区域技术培训的麻醉师在超声引导下注入局部麻醉剂,也可进行单次注射或通过留置导管进行持续镇痛。
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引用次数: 0
The latest developments with internet-based psychological treatments for depression. 基于互联网的抑郁症心理治疗的最新进展。
IF 4.3 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-02-01 Epub Date: 2024-02-06 DOI: 10.1080/14737175.2024.2309237
Gerhard Andersson

Introduction: Internet-based psychological treatments for depression have been around for more than 20 years. There has been a continuous line of research with new research questions being asked and studies conducted.

Areas covered: In this paper, the author reviews studies with a focus on papers published from 2020 and onwards based on a Medline and Scopus search. Internet-based cognitive behavior therapy (ICBT) programs have been developed and tested for adolescents, older adults, immigrant groups and to handle a societal crisis (e.g. COVID-19). ICBT works in regular clinical settings and long-term effects can be obtained. Studies on different treatment orientations and approaches such as acceptance commitment therapy, unified protocol, and tailored treatments have been conducted. Effects on quality-of-life measures, knowledge acquisition and ecological momentary assessment as a research tool have been reported. Factorial design trials and individual patient data meta-analysis are increasingly used in association with internet intervention research. Finally, prediction studies and recent advances in artificial intelligence are mentioned.

Expert opinion: Internet-delivered treatments are effective, in particular if therapist guidance is provided. More target groups have been covered but there are many remaining challenges including how new tools like artificial intelligence will be used when treating depression.

简介基于互联网的抑郁症心理治疗已有 20 多年的历史。随着新研究问题的提出和研究的开展,相关研究一直在持续进行:在本文中,作者根据 Medline 和 Scopus 的检索结果,重点回顾了 2020 年及以后发表的研究论文。基于互联网的认知行为疗法(ICBT)项目已针对青少年、老年人、移民群体以及应对社会危机(如 COVID-19)的问题进行了开发和测试。ICBT 可在常规临床环境中运行,并可取得长期效果。对不同的治疗取向和方法,如接受承诺疗法、统一方案和定制治疗,都进行了研究。对生活质量测量、知识获取和作为研究工具的生态学瞬间评估的效果也有报道。因子设计试验和个体患者数据荟萃分析越来越多地被用于互联网干预研究。最后,还提到了预测研究和人工智能的最新进展:专家意见:互联网提供的治疗是有效的,尤其是在提供治疗师指导的情况下。专家观点:互联网提供的治疗方法是有效的,尤其是在有治疗师指导的情况下。已经覆盖了更多的目标群体,但仍存在许多挑战,包括在治疗抑郁症时如何使用人工智能等新工具。
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引用次数: 0
Family accommodation: a diagnostic feature of obsessive-compulsive disorder? 家庭迁就:强迫症的诊断特征?
IF 4.3 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-02-01 Epub Date: 2024-02-06 DOI: 10.1080/14737175.2024.2309239
Rebecca G Etkin, Michael H Bloch, Eli R Lebowitz
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引用次数: 0
An update on the role of magnetic resonance imaging in predicting and monitoring multiple sclerosis progression. 磁共振成像在预测和监测多发性硬化症进展方面的最新作用。
IF 4.3 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-02-01 Epub Date: 2024-02-06 DOI: 10.1080/14737175.2024.2304116
Piriyankan Ananthavarathan, Nitin Sahi, Declan T Chard

Introduction: While magnetic resonance imaging (MRI) is established in diagnosing and monitoring disease activity in multiple sclerosis (MS), its utility in predicting and monitoring disease progression is less clear.

Areas covered: The authors consider changing concepts in the phenotypic classification of MS, including progression independent of relapses; pathological processes underpinning progression; advances in MRI measures to assess them; how well MRI features explain and predict clinical outcomes, including models that assess disease effects on neural networks, and the potential role for machine learning.

Expert opinion: Relapsing-remitting and progressive MS have evolved from being viewed as mutually exclusive to having considerable overlap. Progression is likely the consequence of several pathological elements, each important in building more holistic prognostic models beyond conventional phenotypes. MRI is well placed to assess pathogenic processes underpinning progression, but we need to bridge the gap between MRI measures and clinical outcomes. Mapping pathological effects on specific neural networks may help and machine learning methods may be able to optimize predictive markers while identifying new, or previously overlooked, clinically relevant features. The ever-increasing ability to measure features on MRI raises the dilemma of what to measure and when, and the challenge of translating research methods into clinically useable tools.

导言:虽然磁共振成像(MRI)在诊断和监测多发性硬化症(MS)的疾病活动方面已得到证实,但其在预测和监测疾病进展方面的作用却不太明确:作者考虑了多发性硬化症表型分类中不断变化的概念,包括独立于复发的疾病进展;支撑疾病进展的病理过程;评估这些过程的 MRI 测量方法的进展;MRI 特征对临床结果的解释和预测能力,包括评估疾病对神经网络影响的模型,以及机器学习的潜在作用:专家观点:复发性多发性硬化症和进展性多发性硬化症已从相互排斥发展到相当程度的重叠。病情进展可能是多种病理因素共同作用的结果,每种因素对于建立超越传统表型的更全面的预后模型都非常重要。磁共振成像非常适合评估导致病情进展的病理过程,但我们需要弥合磁共振成像测量与临床结果之间的差距。将病理效应映射到特定的神经网络可能会有所帮助,机器学习方法可能能够优化预测标记,同时识别新的或以前被忽视的临床相关特征。测量磁共振成像特征的能力不断提高,这就提出了一个难题:测量什么、何时测量,以及将研究方法转化为临床可用工具的挑战。
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引用次数: 0
A proposal for a shared therapeutic algorithm in children with prolonged convulsive seizures and status epilepticus. 针对长期惊厥发作和癫痫状态儿童的共同治疗算法建议。
IF 4.3 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-02-01 Epub Date: 2024-02-06 DOI: 10.1080/14737175.2024.2305813
Roberta Roberti, Antonella Riva, Gianluca D'Onofrio, Emanuele Giacheri, Elisabetta Amadori, Maria Stella Vari, Angela La Neve, Federico Vigevano, Alberto Verrotti, Duccio Maria Cordelli, Antonino Romeo, Antonella Palmieri, Maria Margherita Mancardi, Sergio Caglieris, Antonio Varone, Carlo Minetti, Emilio Russo, Silvia Buratti, Pasquale Striano
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引用次数: 0
The promise and challenges of transcranial magnetic stimulation and deep brain stimulation as therapeutic options for obsessive-compulsive disorder. 经颅磁刺激和脑深部刺激作为强迫症治疗方案的前景与挑战。
IF 4.3 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-02-01 Epub Date: 2024-02-06 DOI: 10.1080/14737175.2024.2306875
Nicola Acevedo, David Castle, Susan Rossell

Introduction: Obsessive compulsive disorder (OCD) represents a complex and often difficult to treat disorder. Pharmacological and psychotherapeutic interventions are often associated with sub-optimal outcomes, and 40-60% of patients are resistant to first line therapies and thus left with few treatment options. OCD is underpinned by aberrant neurocircuitry within cortical, striatal, and thalamic brain networks. Considering the neurocircuitry impairments that underlie OCD symptomology, neurostimulation therapies provide an opportunity to modulate psychopathology in a personalized manner. Also, by probing pathological neural networks, enhanced understanding of disease states can be obtained.

Areas covered: This perspective discusses the clinical efficacy of TMS and DBS therapies, treatment access options, and considerations and challenges in managing patients. Recent scientific progress is discussed, with a focus on neurocircuitry and biopsychosocial aspects. Translational recommendations and suggestions for future research are provided.

Expert opinion: There is robust evidence to support TMS and DBS as an efficacious therapy for treatment resistant OCD patients supported by an excellent safety profile and favorable health economic data. Despite a great need for alternative therapies for chronic and severe OCD patients, resistance toward neurostimulation therapies from regulatory bodies and the psychiatric community remains. The authors contend for greater access to TMS and DBS for treatment resistant OCD patients at specialized sites with appropriate clinical resources, particularly considering adjunct and follow-up care. Also, connectome targeting has shown robust predictive ability of symptom improvements and holds potential in advancing personalized neurostimulation therapies.

导言:强迫症(OCD)是一种复杂且往往难以治疗的疾病。药物和心理治疗干预往往达不到最佳疗效,40%-60%的患者对一线疗法产生抗药性,因此治疗方案寥寥无几。强迫症的基础是大脑皮层、纹状体和丘脑网络中异常的神经环路。考虑到强迫症症状的神经回路障碍,神经刺激疗法提供了一个以个性化方式调节精神病理学的机会。此外,通过探究病理神经网络,还能加深对疾病状态的理解:本视角讨论了 TMS 和 DBS 疗法的临床疗效、治疗途径选择以及患者管理方面的注意事项和挑战。讨论了最新的科学进展,重点关注神经回路和生物心理社会方面。此外,还对未来研究提出了转化建议和意见:有强有力的证据支持 TMS 和 DBS 作为治疗耐药强迫症患者的有效疗法,并辅以出色的安全性和有利的卫生经济数据。尽管慢性严重强迫症患者非常需要替代疗法,但监管机构和精神病学界对神经刺激疗法的抵制依然存在。作者认为,应在拥有适当临床资源的专科医院为有治疗耐药性的强迫症患者提供更多的 TMS 和 DBS 治疗机会,特别是考虑到辅助治疗和后续护理。此外,连接组靶向治疗已显示出对症状改善的强大预测能力,在推进个性化治疗方面具有潜力。
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引用次数: 0
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Expert Review of Neurotherapeutics
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