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Diagnosis of Lennox-Gastaut syndrome and strategies for early recognition. Lennox-Gastaut 综合征的诊断和早期识别策略。
IF 4.3 2区 医学 Q1 Medicine Pub Date : 2024-04-01 Epub Date: 2024-02-28 DOI: 10.1080/14737175.2024.2323568
Suresh Pujar, J Helen Cross

Introduction: Lennox Gastaut syndrome (LGS) as an electroclinical diagnosis has been utilized as a clinical entity for more than 70 years. However, with the recognition of other distinct electroclinical epilepsy syndromes, no consistent single etiology, and the variability of criteria used in clinical trials, the clinical utility of such a diagnosis has been questioned. Recently, the International League Against Epilepsy for the first time defined diagnostic criteria for epilepsy syndromes, thereby allowing consistent language and inclusion criteria to be utilized.

Areas covered: Recent diagnostic criteria for syndrome diagnosis are explored as defined by the International League Against Epilepsy, with further literature reviewed to highlight relevant features, and differential diagnosis explored.

Expert opinion: Developmental and Epileptic Encephalopathy (DEE) is an overall term that may be descriptive of many different epilepsies, most of early onset, whether electroclinically or etiologically defined, of which LGS is one. Although we have moved forward in defining an increasing number of etiologically specific syndromes, this to date remains a minority of the DEEs. Although there is progress with precision medicine targeted at specific causes, the term LGS still remains useful as a diagnosis in defining treatment options, as well as overall prognosis.

导言:伦诺克斯-加斯托综合征(Lennox Gastaut syndrome,LGS)作为一种临床电临床诊断已使用了 70 多年。然而,随着人们认识到其他不同的癫痫电临床综合征,没有一致的单一病因,以及临床试验中使用的标准的多变性,这种诊断的临床实用性受到了质疑。最近,国际抗癫痫联盟首次定义了癫痫综合征的诊断标准,从而可以使用一致的语言和纳入标准:探讨国际抗癫痫联盟最新定义的综合征诊断标准,进一步回顾文献以突出相关特征,并探讨鉴别诊断:发育性和癫痫性脑病(DEE)是一个总称,可描述多种不同的癫痫,多数为早发性癫痫,无论是电clinically还是病因学定义的癫痫,LGS是其中之一。尽管我们已经定义了越来越多的病因特异性综合征,但迄今为止,这些综合征在 DEEs 中仍占少数。虽然针对特定病因的精准医疗取得了进展,但 LGS 作为一种诊断方法,在确定治疗方案和总体预后方面仍然非常有用。
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引用次数: 0
A critical review of the dextroamphetamine transdermal system for the treatment of ADHD in adults and pediatric patients 右旋安非他明透皮系统治疗成人和儿童多动症的重要综述
IF 4.3 2区 医学 Q1 Medicine Pub Date : 2024-03-14 DOI: 10.1080/14737175.2024.2329306
Ann Childress, Nicolas Vaughn
The dextroamphetamine transdermal system (d-ATS) is a stimulant patch recently approved by the United States (U.S.) Food and Drug Administration for the treatment of attention-deficit/hyperactivity...
右旋安非他明透皮系统(d-ATS)是一种兴奋剂贴片,最近获得了美国食品和药物管理局的批准,用于治疗注意力缺陷/多动症。
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引用次数: 0
Is internet-based psychological therapy effective for treating major depressive disorder? 网络心理疗法对治疗重度抑郁障碍有效吗?
IF 4.3 2区 医学 Q1 Medicine Pub Date : 2024-03-01 Epub Date: 2023-12-19 DOI: 10.1080/14737175.2023.2295417
Daniel Fatori, Ives C Passos, André R Brunoni
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引用次数: 0
Emerging antibody-based therapies for Huntington's disease: current status and perspectives for future development. 亨廷顿氏症的新兴抗体疗法:现状与未来发展前景。
IF 4.3 2区 医学 Q1 Medicine Pub Date : 2024-03-01 Epub Date: 2024-02-07 DOI: 10.1080/14737175.2024.2314183
Anamaria Jurcau, Aurel Simion, Maria Carolina Jurcau

Introduction: Being an inherited neurodegenerative disease with an identifiable genetic defect, Huntington's disease (HD) is a suitable candidate for early intervention, possibly even in the pre-symptomatic stage. Our recent advances in elucidating the pathogenesis of HD have revealed a series of novel potential therapeutic targets, among which immunotherapies are actively pursued in preclinical experiments.

Areas covered: This review focuses on the potential of antibody-based treatments targeting various epitopes (of mutant huntingtin as well as phosphorylated tau) that are currently evaluated in vitro and in animal experiments. The references used in this review were retrieved from the PubMed database, searching for immunotherapies in HD, and clinical trial registries were reviewed for molecules already evaluated in clinical trials.

Expert opinion: Antibody-based therapies have raised considerable interest in a series of neurodegenerative diseases characterized by deposition of aggregated of aberrantly folded proteins, HD included. Intrabodies and nanobodies can interact with mutant huntingtin inside the nervous cells. However, the conflicting results obtained with some of these intrabodies highlight the need for proper choice of epitopes and for developing animal models more closely mimicking human disease. Approval of these strategies will require a considerable financial and logistic effort on behalf of healthcare systems.

导言:亨廷顿氏病(Huntington's disease,HD)是一种遗传性神经退行性疾病,具有可识别的基因缺陷,适合早期干预,甚至可能在症状出现前就进行干预。我们最近在阐明亨廷顿氏病发病机制方面取得的进展揭示了一系列新的潜在治疗靶点,其中免疫疗法正在临床前实验中积极探索:本综述重点关注目前在体外和动物实验中评估的针对各种表位(突变亨廷蛋白和磷酸化 tau)的抗体疗法的潜力。本综述中使用的参考文献是从PubMed数据库中检索HD免疫疗法的,并对临床试验登记册中已在临床试验中进行评估的分子进行了审查:基于抗体的疗法在一系列以异常折叠蛋白聚集沉积为特征的神经退行性疾病(包括HD)中引起了广泛关注。体内抗体和纳米抗体可与神经细胞内的突变杭汀蛋白相互作用。然而,使用其中一些内抗体所获得的结果相互矛盾,这凸显了正确选择表位和开发更接近人类疾病的动物模型的必要性。这些策略的批准将需要医疗保健系统在财政和后勤方面做出巨大努力。
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引用次数: 0
Beyond pain catastrophizing: rationale and recommendations for targeting trauma in the assessment and treatment of chronic pain. 超越疼痛灾难化:在评估和治疗慢性疼痛时针对创伤提出的理由和建议。
IF 4.3 2区 医学 Q1 Medicine Pub Date : 2024-03-01 Epub Date: 2024-01-30 DOI: 10.1080/14737175.2024.2311275
Jolin B Yamin, Samantha M Meints, Robert R Edwards
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引用次数: 0
Evaluating fenfluramine hydrochloride as an oral solution for the treatment of seizures associated with Lennox-Gastaut syndrome. 评估盐酸芬氟拉明口服溶液用于治疗与伦诺克斯-加斯塔特综合征相关的癫痫发作。
IF 4.3 2区 医学 Q1 Medicine Pub Date : 2024-03-01 Epub Date: 2024-02-07 DOI: 10.1080/14737175.2024.2313548
Frank M C Besag, Michael J Vasey, Richard F M Chin

Introduction: Lennox-Gastaut syndrome (LGS) is a severe childhood-onset developmental and epileptic encephalopathy characterized by treatment-refractory seizures, including tonic/atonic 'drop' seizures, and intellectual impairment and slow spike-wave discharges on the electroencephalogram. Fenfluramine, previously prescribed as a weight-loss drug but then withdrawn, has recently been approved in the US, EU, and UK for the adjunct treatment of seizures associated with LGS.

Areas covered: The authors review the efficacy and safety findings from clinical trials of fenfluramine in LGS. The authors then discuss the evidence for adverse effects that may be of particular concern to fenfluramine, namely cardiac abnormalities, and weight loss, in the context of the use of fenfluramine for the treatment of the refractory seizures in LGS.

Expert opinion: Fenfluramine has demonstrated efficacy in reducing the frequency of seizures in LGS, notably drop seizures, in short-term and long-term clinical trials. Valvular heart disease and pulmonary hypertension have not been reported at the low doses (≤26 mg/day) used in these studies, however, data are limited. Due to its novel mechanism of action, fenfluramine may be of benefit in LGS which has not responded adequately to other antiseizure medications. However, none of these medications, including fenfluramine, achieves the ultimate goal of seizure freedom in most cases.

简介伦诺克斯-加斯托特综合征(Lennox-Gastaut Syndrome,LGS)是一种严重的儿童期发育性癫痫脑病,其特征是难治性癫痫发作,包括强直/失张力 "跌倒 "发作、智力障碍和脑电图上的慢尖波放电。芬氟拉明以前曾作为减肥药被处方使用,但后来被撤消,最近在美国、欧盟和英国被批准用于LGS相关癫痫发作的辅助治疗:作者回顾了芬氟拉明治疗 LGS 临床试验的疗效和安全性。然后,作者结合芬氟拉明用于治疗 LGS 难治性癫痫发作的情况,讨论了芬氟拉明可能引起特别关注的不良反应的证据,即心脏异常和体重减轻:专家意见:在短期和长期临床试验中,芬氟拉明对降低LGS患者的癫痫发作频率,尤其是减少跌倒发作具有疗效。在这些研究中使用低剂量(≤26 毫克/天)时,尚未发现瓣膜性心脏病和肺动脉高压的报道,但数据有限。由于芬氟拉明的作用机制新颖,它可能对其他抗癫痫药物反应不佳的 LGS 患者有益。然而,在大多数病例中,包括芬氟拉明在内的所有这些药物都无法实现摆脱癫痫发作的最终目标。
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引用次数: 0
Treatment of dissociative identity disorder: leveraging neurobiology to optimize success. 分离性身份识别障碍的治疗:利用神经生物学优化成功率。
IF 3.4 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-03-01 Epub Date: 2024-02-15 DOI: 10.1080/14737175.2024.2316153
Juliann B Purcell, Bethany Brand, Heidi A Browne, Richard A Chefetz, Meghan Shanahan, Zoe A Bair, Kim A Baranowski, Vona Davis, Patricia Mangones, Rebecca L Modell, Cori A Palermo, Emma C Robertson, Matthew A Robinson, Laura Ward, Sherry Winternitz, Milissa L Kaufman, Lauren A M Lebois

Introduction: Dissociative identity disorder (DID) is a treatable mental health condition that is associated with a range of psychobiological manifestations. However, historical controversy, modern day misunderstanding, and lack of professional education have prevented accurate treatment information from reaching most clinicians and patients. These obstacles also have slowed empirical efforts to improve treatment outcomes for people with DID. Emerging neurobiological findings in DID provide essential information that can be used to improve treatment outcomes.

Areas covered: In this narrative review, the authors discuss symptom characteristics of DID, including dissociative self-states. Current treatment approaches are described, focusing on empirically supported psychotherapeutic interventions for DID and pharmacological agents targeting dissociative symptoms in other conditions. Neurobiological correlates of DID are reviewed, including recent research aimed at identifying a neural signature of DID.

Expert opinion: Now is the time to move beyond historical controversy and focus on improving DID treatment availability and efficacy. Neurobiological findings could optimize treatment by reducing shame, aiding assessment, providing novel interventional brain targets and guiding novel pharmacologic and psychotherapeutic interventions. The inclusion of those with lived experience in the design, planning and interpretation of research investigations is another powerful way to improve health outcomes for those with DID.

简介分离性身份识别障碍(DID)是一种可治疗的精神疾病,与一系列心理生物学表现相关。然而,由于历史上的争议、现代人的误解以及缺乏专业教育,大多数临床医生和患者无法获得准确的治疗信息。这些障碍也延缓了改善 DID 患者治疗效果的经验努力。新出现的 DID 神经生物学发现提供了可用于改善治疗效果的重要信息:在这篇叙述性综述中,作者讨论了 DID 的症状特征,包括分离性自我状态。文中介绍了当前的治疗方法,重点是针对 DID 的经验性心理治疗干预措施和针对其他情况下分离症状的药物治疗。回顾了 DID 的神经生物学相关性,包括旨在确定 DID 神经特征的最新研究:专家观点:现在是超越历史争议,专注于提高 DID 治疗可用性和疗效的时候了。神经生物学研究成果可以通过减少羞耻感、帮助评估、提供新的大脑干预目标以及指导新的药物和心理治疗干预来优化治疗。让有生活经验的人参与研究调查的设计、规划和解释,是改善 DID 患者健康状况的另一种有效方法。
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引用次数: 0
Evaluating the efficacy and safety of the currently available once-every-two months long-acting injectable formulations of aripiprazole for the treatment of schizophrenia or as a maintenance monotherapy for bipolar I disorder in adults. 评估阿立哌唑目前每两个月一次的长效注射制剂治疗成人精神分裂症或作为双相情感障碍 I 的维持性单一疗法的疗效和安全性。
IF 4.3 2区 医学 Q1 Medicine Pub Date : 2024-03-01 Epub Date: 2024-02-05 DOI: 10.1080/14737175.2024.2313550
Ludovic Samalin, Ludivine Boudieu, Pierre Michel Llorca

Introduction: An aripiprazole long-acting injectable (LAI) antipsychotic is now available for gluteal administration every 2 months via two different formulations: aripiprazole lauroxil (AL) and aripiprazole monohydrate (Ari 2MRTU). These longer dosing regimens of aripiprazole LAI offer new potential benefits for patients.

Areas covered: The authors review the evidence supporting the efficacy and safety of aripiprazole LAIs given every 2 months for the treatment of schizophrenia or bipolar disorder (BD) in adults. The article culminates with the authors' expert perspectives on the subject.

Expert opinion: AL 1064 mg every 2 months has established efficacy for the treatment of schizophrenia based on pharmacokinetic bridging studies and prospective data for treatment of an acute exacerbation of schizophrenia. In an open-label trial, Ari 2MRTU showed efficacy for the treatment of schizophrenia and BD type I based on pharmacokinetic parameters (comparable to aripiprazole once-monthly 400 mg); it also showed efficacy regarding the secondary endpoints. Multiple doses of AL 1064 mg or Ari 2MRTU 960 mg are generally well tolerated, in line with the safety profile of oral aripiprazole, with the exception of the injection-site reactions. While AL may require a 1-day initiation regimen, Ari 2MRTU 960 covers all the recommended doses of oral aripiprazole (10-20 mg).

简介:阿立哌唑长效注射剂(LAI)是一种抗精神病药物,目前有两种不同的制剂:阿立哌唑月桂锭(AL)和阿立哌唑一水合物(Ari 2MRTU),可每两个月通过臀部给药一次。阿立哌唑LAI的这些较长给药方案为患者带来了新的潜在益处:作者回顾了支持每两个月服用一次阿立哌唑LAI治疗成人精神分裂症或双相情感障碍(BD)的疗效和安全性的证据。文章最后提出了作者的专家观点:根据药代动力学桥接研究和治疗精神分裂症急性加重期的前瞻性数据,AL 1064 毫克每 2 个月一次治疗精神分裂症的疗效已经确立。在一项开放标签试验中,根据药代动力学参数(与阿立哌唑每月一次,每次 400 毫克相当),阿里 2MRTU 对治疗精神分裂症和 BD I 型有疗效;在次要终点方面也显示出疗效。多剂量 AL 1064 毫克或 Ari 2MRTU 960 毫克的耐受性总体良好,与口服阿立哌唑的安全性一致,但注射部位反应除外。AL 可能需要 1 天的起始方案,而 Ari 2MRTU 960 则涵盖了口服阿立哌唑的所有推荐剂量(10-20 毫克)。
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引用次数: 0
Evaluating avalglucosidase alfa for the management of late-onset Pompe disease. 评估用于治疗晚发型庞贝氏症的阿瓦糖苷酶α。
IF 4.3 2区 医学 Q1 Medicine Pub Date : 2024-03-01 Epub Date: 2024-01-23 DOI: 10.1080/14737175.2024.2306855
Corrado Angelini

Introduction: Glycogenosis type II (GSDII) is a rare autosomal disorder that is caused by the deficiency of alpha-glucosidase, a lysosomal enzyme that hydrolyzes glycogen to glucose. Autophagy dysregulation plays a critical role. Importantly, since 2006, both patients with infantile (classic Pompe disease) and adult GSDII (late-onset Pompe disease or LOPD) have been treated with enzyme replacement therapy (ERT). To support this use, several double-blind and observational studies including large cohorts of GSDII patients have been undertaken and have shown ERT to be effective in modifying the natural course of disease. Indeed, most LOPD cases improve in the first 20 months of treatment in a six-minute walk test (6MWT), while those who are untreated do not; instead, their response declines over time.

Areas covered: The author reviews avalglucosidase alpha, a therapy approved by both the FDA and European regulatory agencies. Herein, the author considers the pathophysiological approaches such as the role of enzyme entry, autophagy, and the response to ERT treatment of motor and respiratory components.

Expert opinion: There has been a notable drive toward the research of various aspects of this disease regarding the role of new enzyme penetration and immune adverse events. Consequently, avalglucosidase alpha might be a further step forward.

简介糖原病 II 型(GSDII)是一种罕见的常染色体疾病,由缺乏α-葡萄糖苷酶引起,α-葡萄糖苷酶是一种能将糖原水解为葡萄糖的溶酶体酶。自噬失调起着关键作用。重要的是,自 2006 年以来,婴儿(典型庞贝氏症)和成人 GSDII(晚发庞贝氏症或 LOPD)患者都接受了酶替代疗法(ERT)。为了支持这种疗法,已经开展了多项双盲和观察性研究,其中包括大量的 GSDII 患者,研究结果表明 ERT 能够有效改变疾病的自然病程。事实上,大多数 LOPD 病例在接受治疗的前 20 个月中,六分钟步行测试(6MWT)的情况都有所改善,而那些未接受治疗的患者则没有改善,相反,他们的反应会随着时间的推移而下降:作者回顾了美国食品及药物管理局和欧洲监管机构批准的一种疗法--阿瓦糖苷酶α。在此,作者考虑了病理生理学方法,如酶进入的作用、自噬以及运动和呼吸部分对 ERT 治疗的反应:专家观点:对这种疾病各方面的研究,尤其是对新酶渗透作用和免疫不良事件的研究,已经取得了显著的进展。因此,α-阿瓦糖苷酶可能是向前迈出的又一步。
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引用次数: 0
An analysis of omega-3 clinical trials and a call for personalized supplementation for dementia prevention. 分析欧米茄-3 临床试验,呼吁为预防痴呆症提供个性化补充剂。
IF 4.3 2区 医学 Q1 Medicine Pub Date : 2024-03-01 Epub Date: 2024-02-20 DOI: 10.1080/14737175.2024.2313547
Nicolás Castellanos-Perilla, Miguel Germán Borda, Dag Aarsland, George E Barreto

Introduction: Targeted interventions are needed to delay or prevent the onset of neurodegenerative diseases. Poor dietary habits are associated with cognitive decline, highlighting the benefits of a healthy diet with fish and polyunsaturated fatty acids (PUFAs). Intake of omega-3 PUFAs docosahexaenoic acid (DHA), α-linolenic acid (ALA) and eicosapentaenoic acid (EPA) is linked with healthy aging, cardiovascular benefits, and reduced risk of Alzheimer's disease. Although omega-3 has health benefits, its intake is often inadequate and insufficient in modern diets. Although fish oil supplements offer an alternative source, inconsistent results from clinical trials raise questions about the factors determining their success.

Areas covered: In this this review, the authors discuss the aforementioned determining factors and highlight strategies that could enhance the effectiveness of omega-3 PUFAs interventions for dementia and cognitive decline. Moreover, the authors provide suggestions for potential future research.

Expert opinion: Factors such as diet, lifestyle, and genetic predisposition can all influence the effectiveness of omega-3 supplementation. When implementing clinical trials, it is crucial to consider these factors and recognize their potential impact on the interpretation of results. It is important to study each variable independently and the interactions between them.

导言:需要采取有针对性的干预措施来延缓或预防神经退行性疾病的发生。不良的饮食习惯与认知能力下降有关,这就凸显了鱼类和多不饱和脂肪酸(PUFAs)健康饮食的益处。摄入欧米伽-3 多不饱和脂肪酸二十二碳六烯酸(DHA)、α-亚麻酸(ALA)和二十碳五烯酸(EPA)与健康老龄化、心血管益处和降低阿尔茨海默氏症风险有关。虽然欧米伽-3 对健康有益,但现代饮食中的欧米伽-3 摄入量往往不足。虽然鱼油补充剂提供了一种替代来源,但临床试验的结果不一致,使人们对决定其成功与否的因素产生了疑问:在这篇综述中,作者讨论了上述决定性因素,并强调了可提高ω-3 PUFAs干预痴呆症和认知能力下降的有效性的策略。此外,作者还对未来可能开展的研究提出了建议:饮食、生活方式和遗传倾向等因素都会影响ω-3补充剂的效果。在进行临床试验时,考虑这些因素并认识到它们对结果解释的潜在影响至关重要。重要的是要独立研究每个变量以及它们之间的相互作用。
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引用次数: 0
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Expert Review of Neurotherapeutics
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