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Epidemiology and etiology of community-acquired CNS infections in Iran: a narrative review 伊朗社区获得性中枢神经系统感染的流行病学和病因:叙述性综述
IF 1.3 Q4 CLINICAL NEUROLOGY Pub Date : 2024-01-02 DOI: 10.2217/fnl-2023-0017
F. Sheybani, Mahboubeh Haddad, Matin Shirazinia
CNS infections are associated with significant morbidity and mortality. The epidemiology of CNS infections shows striking differences in geographic regions. We reviewed the literature on clinico-epidemiological features of community-acquired CNS infections in Iran. Our review highlighted that the causes of CNS infections in Iran are diverse but information regarding the epidemiology and precise estimates of the burden of disease are lacking for most neuroinfections. Enteroviruses, S. pneumoniae, and Klebsiella species are the most commonly reported causes of viral, bacterial, and neonatal meningitis, respectively, whereas neurotuberculosis and neurobrucellosis place a huge burden. Improving the national surveillance system and implementing a nationwide data registry system for CNS infections are necessary to provide practically useful information regarding the microbial spectrum and the burden of CNS infections to suggest optimal preventive, diagnostic, and therapeutic strategies.
中枢神经系统感染与严重的发病率和死亡率有关。中枢神经系统感染的流行病学在不同的地理区域有着显著的差异。我们回顾了有关伊朗社区获得性中枢神经系统感染的临床流行病学特征的文献。我们的综述强调,伊朗中枢神经系统感染的病因多种多样,但大多数神经感染缺乏流行病学信息和疾病负担的精确估计。肠道病毒、肺炎双球菌和克雷伯氏菌分别是病毒性、细菌性和新生儿脑膜炎最常见的病因,而神经结核病和神经布鲁氏菌病则造成了巨大的负担。要提供有关中枢神经系统感染的微生物谱和负担的实用信息,从而提出最佳的预防、诊断和治疗策略,就必须改进国家监测系统并实施全国性的中枢神经系统感染数据登记系统。
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引用次数: 0
The effects of lemborexant on insomnia-related fatigue: a plain language summary 利眠宁对失眠相关疲劳的影响:通俗易懂的摘要
IF 1.3 Q4 CLINICAL NEUROLOGY Pub Date : 2023-11-16 DOI: 10.2217/fnl-2023-0018
C. Chepke, Rakesh Jain, Russell Rosenberg, M. Moline, J. Yardley, K. Pinner, Dinesh Kumar, Carlos Perdomo, G. Filippov, N. Atkins, Manoj Malhotra
This is a summary of an article originally published in the journal Postgraduate Medicine. Lemborexant is a type of medication called a dual orexin receptor antagonist (often abbreviated to DORA) that is approved for treating people with insomnia, a sleep disorder in which people have trouble falling asleep, staying asleep or both. Two studies, one called SUNRISE-1 (Study 304), which lasted one month, and SUNRISE-2 (Study 303), which lasted 12 months, looked at the effects of lemborexant on sleep compared to placebo in people with insomnia. People with insomnia often experience fatigue (or tiredness) during the daytime, which affects their daily lives and health, and often have other conditions which can also cause fatigue. This summary reports an analysis that looked at whether lemborexant improves fatigue during the daytime as well as nighttime symptoms of insomnia in all participants and in participants with clinically significant fatigue before treatment. In both studies, participants completed a questionnaire, called the Fatigue Severity Scale, that measured how significant their fatigue was. The researchers looked at the change in fatigue over time in participants treated with lemborexant compared with those given placebo. They also looked at the change in a group of participants who reported clinically important levels of fatigue at the beginning of the study. People in these studies also completed diaries about how long it took them to fall asleep and how long they slept. The researchers compared improvements in fatigue with changes in how participants slept over time. Overall, lemborexant reduced daytime fatigue compared with placebo in all study participants. It also reduced daytime fatigue in the smaller group of participants who had clinically significant fatigue at the beginning of the study. The reductions in fatigue seen with lemborexant remained during 1 year of treatment. There was also a relationship between reduced fatigue, falling asleep faster and not waking up as much. Researchers concluded that in addition to improving sleep in people with insomnia, lemborexant can reduce daytime fatigue.
本文是最初发表在《研究生医学》(Postgraduate Medicine)杂志上的一篇文章的摘要。Lemborexant是一种被称为双奥曲肽受体拮抗剂(通常缩写为DORA)的药物,已被批准用于治疗失眠症患者。有两项研究,一项名为 "SUNRISE-1"(研究 304),为期一个月;另一项名为 "SUNRISE-2"(研究 303),为期 12 个月。失眠症患者在白天经常会感到疲劳(或疲倦),这影响了他们的日常生活和健康,而且他们通常还患有其他也会导致疲劳的疾病。本摘要报告了一项分析,该分析考察了伦博雷沙坦是否能改善所有参与者的白天疲劳以及夜间失眠症状,以及治疗前有临床明显疲劳症状的参与者的白天疲劳和夜间失眠症状。在这两项研究中,参与者都填写了一份名为 "疲劳严重程度量表 "的调查问卷,以测量他们的疲劳程度有多严重。研究人员观察了与服用安慰剂的患者相比,接受伦博雷沙坦治疗的患者随着时间的推移疲劳程度的变化。他们还观察了一组在研究开始时报告了临床重要疲劳程度的参与者的变化。参加这些研究的人还填写了入睡时间和睡眠时间的日记。研究人员将疲劳程度的改善情况与参与者睡眠时间的变化进行了比较。总体而言,与安慰剂相比,所有研究参与者的lemborexant都能减轻白天的疲劳感。在研究开始时有明显临床疲劳感的一小部分参与者中,它也能减轻白天的疲劳感。在一年的治疗过程中,伦博雷沙坦缓解疲劳的效果依然存在。疲劳减轻、入睡更快和醒来次数减少之间也有关系。研究人员得出结论,除了改善失眠症患者的睡眠,lemborexant 还能减轻白天的疲劳。
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引用次数: 0
Why should ‘psychogenic’ be parenthetical in functional (psychogenic) neurological disorder? 为什么“心因性”在功能性(心因性)神经障碍中要加括号?
Q4 CLINICAL NEUROLOGY Pub Date : 2023-09-01 DOI: 10.2217/fnl-2022-0018
Laurent Vercueil, Christo Bratanov
Future NeurologyAhead of Print CommentaryOpen AccessWhy should ‘psychogenic’ be parenthetical in functional (psychogenic) neurological disorder?Laurent Vercueil & Christo BratanovLaurent Vercueil *Author for correspondence: Tel.: +33670316120; E-mail Address: lvercueil@chu-grenoble.frhttps://orcid.org/0000-0003-3323-0091Laboratory of Psychology and Neurocognition, Université Grenoble Alpes, 38000 Grenoble, FranceNeurology Department, CHU Grenoble Alpes, Grenoble, FranceSearch for more papers by this author & Christo BratanovNeurology Department, CHU Grenoble Alpes, Grenoble, FranceSearch for more papers by this authorPublished Online:2 Nov 2023https://doi.org/10.2217/fnl-2022-0018AboutSectionsPDF/EPUB ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareShare onFacebookTwitterLinkedInRedditEmail Keywords: functional neurological disorderpsychogenicFunctional neurologic disorder (FND) refers to a group of common neurological symptoms arising from the voluntary and conscious motor or somatosensory nervous system but experienced as involuntary. In FND, there is no significant structural damage in the brain, or the damage is unrelated to the symptoms. The exact cause of FND is unknown. A substantial amount of debate has risen in the recent literature about adequate naming of the formerly so-called ‘hysteria' [1,2]. Actually, terms, such as ‘conversion’, ‘somatoform’, ‘medically unexplained’, ‘psychogenic’, ‘dissociation’, ‘somatoform’ and ‘functional disorders’ have been concurrently and successively used over the past decades [3]. More recently, a consensus has emerged to promote the use of 'functional neurological disorder' in medical publications, leading to the foundation of the FND Society in 2018 [4]. However, since the beginning of the 2010s, the use of 'functional (psychogenic) neurological disorder' (‘psychogenic’ being put into brackets after ‘functional’) appeared consistently in titles, keywords or abstracts of articles, with a growing frequency, although no consensus or recommendation for using this labelling have been found by the authors. By conducting a bibliographic research in medical and scientific online database (MEDLINE PubMed – end 2022), we found a total of 145 occurrences of ‘functional [psychogenic]’ (F[P]) associated terms since 1960, either in the title of the reference, within the abstract or as keyword (details in the Supplementary Material). First occurrence of the association ‘functional psychogenic’ were retrieved in the English translation from Russian [5] and Romanian [6] articles titles in 1978, respectively, on ‘functional psychogenic disorders of the cardiovascular system’ and ‘functional psychogenic dysphonia’. However, these titles did not use brackets for the term ‘psychogenic’. Seven occurrences from the 145 were not real parenthetical brackets (as in ‘functional (psychogenic)’) but forward slashes (‘functional/psychogenic’), thus suggesting a kind of equivalence between ‘functional’
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引用次数: 0
An overview of reviews with network meta-analyses comparing disease-modifying therapies for relapsing multiple sclerosis 网络荟萃分析比较复发性多发性硬化症疾病改善疗法的综述
IF 1.3 Q4 CLINICAL NEUROLOGY Pub Date : 2023-08-22 DOI: 10.2217/fnl-2023-0009
Christopher Drudge, I. Samjoo, R. Brennan, L. Badgujar, V. Khurana, Santosh Tiwari, N. Adlard, J. Bánházi
Aim: An overview of published systematic reviews (SRs) with integrated network meta-analyses (NMAs) comparing disease-modifying therapies (DMTs) for relapsing multiple sclerosis (RMS) was conducted to help inform healthcare decision-making. Methods: We searched Embase, MEDLINE® and Cochrane Database of Systematic Reviews from inception to May 2023. Full-text studies evaluating annualized relapse rate (ARR) and/or confirmed disability progression (CDP) were qualitatively compared. Methodological quality was assessed using SR and NMA questionnaires. Results: Twenty-one SRs with integrated NMAs were included. Studies varied in their conduct and reporting of the SR, the included primary evidence, treatments, and cross-trial heterogeneity assessment and their conduct and reporting of NMAs. The quality of the studies was variable. Monoclonal antibody therapies were determined to be the most efficacious DMTs for reducing ARR and delaying CDP. Conclusion: Future analyses should carefully consider and clearly report methods and results to permit accurate interpretation of NMA findings and better inform decision-making.
目的:对已发表的系统评价(SRs)和综合网络荟萃分析(nma)进行综述,比较复发性多发性硬化症(RMS)的疾病改善疗法(dmt),以帮助告知医疗保健决策。方法:检索Embase、MEDLINE®和Cochrane系统评价数据库,检索时间为成立至2023年5月。评估年复发率(ARR)和/或确认残疾进展(CDP)的全文研究进行了定性比较。采用SR问卷和NMA问卷评估方法学质量。结果:纳入21例合并nma的SRs。研究在SR的实施和报告、纳入的主要证据、治疗方法、跨试验异质性评估以及nma的实施和报告方面各不相同。研究的质量参差不齐。单克隆抗体治疗被认为是降低ARR和延缓CDP最有效的dmt。结论:未来的分析应仔细考虑并清楚地报告方法和结果,以便准确解释NMA发现并更好地为决策提供信息。
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引用次数: 0
Proton Beam Therapy for Intrahepatic Cholangiocarcinoma: A Multicenter Prospective Registry Study in Japan. 质子束疗法治疗肝内胆管癌:日本多中心前瞻性登记研究。
IF 13.8 Q4 CLINICAL NEUROLOGY Pub Date : 2023-07-24 eCollection Date: 2024-04-01 DOI: 10.1159/000531376
Masashi Mizumoto, Kazuki Terashima, Hirokazu Makishima, Motohisa Suzuki, Takashi Ogino, Takahiro Waki, Hiromitsu Iwata, Hiroyasu Tamamura, Yusuke Uchinami, Tetsuo Akimoto, Tomoaki Okimoto, Takashi Iizumi, Masao Murakami, Norio Katoh, Kazushi Maruo, Kei Shibuya, Hideyuki Sakurai

Introduction: Intrahepatic cholangiocarcinoma (ICC) can be treated with chemotherapy in unresectable cases, but outcomes are poor. Proton beam therapy (PBT) may provide an alternative treatment and has good dose concentration that may improve local control.

Methods: Fifty-nine patients who received initial PBT for ICC from May 2016 to June 2018 at nine centers were included in the study. The treatment protocol was based on the policy of the Japanese Society for Radiation Oncology. Forty patients received 72.6-76 Gy (RBE) in 20-22 fr, 13 received 74.0-76.0 Gy (RBE) in 37-38 fr, and 6 received 60-70.2 Gy (RBE) in 20-30 fr. Overall survival (OS) and progression-free survival (PFS) were estimated by Kaplan-Meier analysis.

Results: The 59 patients (35 men, 24 women; median age: 71 years; range: 41-91 years) had PS of 0 (n = 47), 1 (n = 10), and 2 (n = 2). Nine patients had hepatitis and all 59 cases were considered inoperable. The Child-Pugh class was A (n = 46), B (n = 7), and unknown (n = 6); the median maximum tumor diameter was 5.0 cm (range 2.0-15.2 cm); and the clinical stage was I (n = 12), II (n = 19), III (n = 10), and IV (n = 18). At the last follow-up, 17 patients were alive (median follow-up: 36.7 months; range: 24.1-49.9 months) and 42 had died. The median OS was 21.7 months (95% CI: 14.8-34.4 months). At the last follow-up, 37 cases had recurrence, including 10 with local recurrence. The median PFS was 7.5 months (95% CI: 6.1-11.3 months). In multivariable analyses, Child-Pugh class was significantly associated with OS and PFS, and Child-Pugh class and hepatitis were significantly associated with local recurrence. Four patients (6.8%) had late adverse events of grade 3 or higher.

Conclusion: PBT gives favorable treatment outcomes for unresectable ICC without distant metastasis and may be particularly effective in cases with large tumors.

导言:肝内胆管癌(ICC)在无法切除的情况下可采用化疗,但疗效不佳。质子束疗法(PBT)是一种替代治疗方法,其剂量浓度高,可改善局部控制:研究纳入了2016年5月至2018年6月在9个中心接受初次PBT治疗的59例ICC患者。治疗方案基于日本放射肿瘤学会的政策。40名患者在20-22 fr接受了72.6-76 Gy(RBE)的治疗,13名患者在37-38 fr接受了74.0-76.0 Gy(RBE)的治疗,6名患者在20-30 fr接受了60-70.2 Gy(RBE)的治疗。总生存期(OS)和无进展生存期(PFS)通过 Kaplan-Meier 分析法进行估算:59 名患者(35 名男性,24 名女性;中位年龄:71 岁;范围:41-91 岁)的 PS 值分别为 0(47 人)、1(10 人)和 2(2 人)。9 名患者患有肝炎,所有 59 例患者均无法手术。Child-Pugh分级为A级(46例)、B级(7例)和未知(6例);肿瘤最大直径中位数为5.0厘米(范围2.0-15.2厘米);临床分期为I期(12例)、II期(19例)、III期(10例)和IV期(18例)。最后一次随访时,17 名患者存活(中位随访时间:36.7 个月;范围:24.1-49.9 个月),42 名患者死亡。中位生存期为 21.7 个月(95% CI:14.8-34.4 个月)。最后一次随访时,37 例患者复发,其中 10 例为局部复发。中位 PFS 为 7.5 个月(95% CI:6.1-11.3 个月)。在多变量分析中,Child-Pugh分级与OS和PFS显著相关,Child-Pugh分级和肝炎与局部复发显著相关。4名患者(6.8%)出现了3级或以上的晚期不良反应:结论:PBT 对不可切除且无远处转移的 ICC 有良好的治疗效果,对肿瘤较大的病例尤为有效。
{"title":"Proton Beam Therapy for Intrahepatic Cholangiocarcinoma: A Multicenter Prospective Registry Study in Japan.","authors":"Masashi Mizumoto, Kazuki Terashima, Hirokazu Makishima, Motohisa Suzuki, Takashi Ogino, Takahiro Waki, Hiromitsu Iwata, Hiroyasu Tamamura, Yusuke Uchinami, Tetsuo Akimoto, Tomoaki Okimoto, Takashi Iizumi, Masao Murakami, Norio Katoh, Kazushi Maruo, Kei Shibuya, Hideyuki Sakurai","doi":"10.1159/000531376","DOIUrl":"10.1159/000531376","url":null,"abstract":"<p><strong>Introduction: </strong>Intrahepatic cholangiocarcinoma (ICC) can be treated with chemotherapy in unresectable cases, but outcomes are poor. Proton beam therapy (PBT) may provide an alternative treatment and has good dose concentration that may improve local control.</p><p><strong>Methods: </strong>Fifty-nine patients who received initial PBT for ICC from May 2016 to June 2018 at nine centers were included in the study. The treatment protocol was based on the policy of the Japanese Society for Radiation Oncology. Forty patients received 72.6-76 Gy (RBE) in 20-22 fr, 13 received 74.0-76.0 Gy (RBE) in 37-38 fr, and 6 received 60-70.2 Gy (RBE) in 20-30 fr. Overall survival (OS) and progression-free survival (PFS) were estimated by Kaplan-Meier analysis.</p><p><strong>Results: </strong>The 59 patients (35 men, 24 women; median age: 71 years; range: 41-91 years) had PS of 0 (<i>n</i> = 47), 1 (<i>n</i> = 10), and 2 (<i>n</i> = 2). Nine patients had hepatitis and all 59 cases were considered inoperable. The Child-Pugh class was A (<i>n</i> = 46), B (<i>n</i> = 7), and unknown (<i>n</i> = 6); the median maximum tumor diameter was 5.0 cm (range 2.0-15.2 cm); and the clinical stage was I (<i>n</i> = 12), II (<i>n</i> = 19), III (<i>n</i> = 10), and IV (<i>n</i> = 18). At the last follow-up, 17 patients were alive (median follow-up: 36.7 months; range: 24.1-49.9 months) and 42 had died. The median OS was 21.7 months (95% CI: 14.8-34.4 months). At the last follow-up, 37 cases had recurrence, including 10 with local recurrence. The median PFS was 7.5 months (95% CI: 6.1-11.3 months). In multivariable analyses, Child-Pugh class was significantly associated with OS and PFS, and Child-Pugh class and hepatitis were significantly associated with local recurrence. Four patients (6.8%) had late adverse events of grade 3 or higher.</p><p><strong>Conclusion: </strong>PBT gives favorable treatment outcomes for unresectable ICC without distant metastasis and may be particularly effective in cases with large tumors.</p>","PeriodicalId":12606,"journal":{"name":"Future Neurology","volume":"8 1","pages":"161-168"},"PeriodicalIF":13.8,"publicationDate":"2023-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11095592/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"90168824","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Stroke severity among ischemic stroke patients with elevated diastolic blood pressure 舒张压升高的缺血性脑卒中患者的脑卒中严重程度
IF 1.3 Q4 CLINICAL NEUROLOGY Pub Date : 2023-06-23 DOI: 10.2217/fnl-2022-0016
K. Knisely, Camron Edrissi, Chase Rathfoot, C. Sanders, Samuel I Nathaniel, T. Nathaniel
Aim: Understanding risk factors with elevated diastolic blood pressure (DBP) in acute ischemic stroke (AIS) is significant. Methods: Multivariable logistic regression was used to determine predictors of stroke severity in AIS patients, categorized by their DBP levels >80 mmHg and ≤80 mmHg. Results: AIS patients with DBP >80 mmHg that presents with a history of alcohol use (OR = 2.11, 95% CI: 1.116–3.988, p = 0.022), hypertension (OR = 1.572, 95% CI: 1.01–2.449, p = 0.035), and increasing heart rate (OR = 1.012, 95% CI: 1.012–1.022, p = 0.024) were associated with higher stroke severity Conclusion: Identified factors are independent predictors of higher stroke severity in AIS patients with elevated DBP.
目的:了解急性缺血性脑卒中(AIS)舒张压升高的危险因素具有重要意义。方法:采用多变量逻辑回归确定AIS患者卒中严重程度的预测因素,按DBP水平>80 mmHg和≤80 mmHg进行分类。结果:有饮酒史(OR=2.11,95%CI:1.16–3.988,p=0.022)、高血压(OR=1.572,95%CI:1.01–2.449,p=0.035)的DBP>80 mmHg的AIS患者,和心率增加(OR=1.012,95%CI:1.012–1.022,p=0.024)与较高的卒中严重程度相关结论:已确定的因素是DBP升高的AIS患者卒中严重程度较高的独立预测因素。
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引用次数: 0
Key results from two phase 3 trials on the efficacy and safety of daridorexant in patients with chronic insomnia daridorexant对慢性失眠症患者的疗效和安全性的两项3期试验的关键结果
IF 1.3 Q4 CLINICAL NEUROLOGY Pub Date : 2023-05-04 DOI: 10.2217/fnl-2022-0014
E. Mignot, D. Mayleben, I. Fietze, D. Léger, G. Zammit, Claudio L A Bassetti, D. S. Kinter, T. Roth
This plain language summary describes the main results from two similar research studies, “Study 1” and “Study 2”, which evaluated the use of a medication called daridorexant in patients with chronic insomnia disorder. These two studies were phase 3 clinical trials, which compared different doses of daridorexant to an inactive pill called a placebo, which looked and tasted similar to the daridorexant pill but did not contain daridorexant or any other active ingredient. Adults with chronic insomnia disorder typically find it hard to fall or stay asleep and may wake up too early, leading to sleep dissatisfaction. They also have impaired daytime functioning, which is when a person can feel tired or drowsy, have difficulty concentrating, or experience low mood as a consequence of lack of sleep. Adults with chronic insomnia disorder are also at increased risk for injuries and accidents as a result of that impaired daytime functioning. Researchers found that daridorexant 50 mg and 25 mg improved on average participants' ability to get to sleep and stay asleep, as well as increasing their overall total sleep time. Daridorexant 50 mg also improved daytime functioning without any negative effects the next morning, such as feeling tired or drowsy, on average in the participants included in the studies. While available medications are effective in treating night-time symptoms of insomnia, they have not been shown to improve daytime symptoms in insomnia, and in many cases have been shown to have residual effects the next day due to their nature as sedatives. Therefore there has been a need for new treatments for chronic insomnia disorder that will improve both night-time and daytime symptoms with minimal next morning effects. These findings show the effectiveness of daridorexant 50 mg in improving both night-time and daytime functioning, and that daridorexant is well-tolerated at all doses. The results from these two studies resulted in the approval of the 25 mg and 50 mg doses of daridorexant by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Clinical Trial Registration: NCT03545191 (study 1) and NCT03575104 (study 2) ( ClinicalTrials.gov )
这份简明的摘要描述了两项类似研究的主要结果,“研究1”和“研究2”,这两项研究评估了一种名为daridorexant的药物在慢性失眠患者中的使用情况。这两项研究是3期临床试验,将不同剂量的达里多沙与一种名为安慰剂的无活性药丸进行了比较,安慰剂的外观和味道与达里多萨药丸相似,但不含达里多西或任何其他活性成分。患有慢性失眠障碍的成年人通常会发现很难入睡或保持睡眠,而且可能醒得太早,导致睡眠不满意。他们的日间功能也会受损,即一个人可能会感到疲劳或嗜睡,难以集中注意力,或因睡眠不足而情绪低落。患有慢性失眠障碍的成年人由于日间功能受损,受伤和事故的风险也会增加。研究人员发现,50毫克和25毫克的daridorexant平均能提高参与者的睡眠和保持睡眠的能力,并增加他们的总睡眠时间。参与研究的参与者平均而言,50 mg Daridorexant也改善了白天的功能,第二天早上没有任何负面影响,比如感到疲劳或嗜睡。虽然现有的药物对治疗失眠的夜间症状有效,但它们并没有被证明能改善失眠的日间症状,而且在许多情况下,由于其作为镇静剂的性质,它们在第二天会产生残留影响。因此,需要一种新的治疗慢性失眠的方法,这种方法可以改善夜间和白天的症状,第二天早上的影响最小。这些发现表明,50 mg的daridorexant在改善夜间和日间功能方面是有效的,并且Daridorexent在所有剂量下都具有良好的耐受性。这两项研究的结果导致美国食品药品监督管理局(FDA)和欧洲药品管理局(EMA)批准了25毫克和50毫克剂量的达里多列。临床试验注册:NCT03545191(研究1)和NCT03575104(研究2)(ClinicalTrials.gov)
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引用次数: 0
Plain language summary: what symptoms should be measured in clinical studies for early-stage Parkinson's? 简单的语言总结:在早期帕金森病的临床研究中应该测量哪些症状?
IF 1.3 Q4 CLINICAL NEUROLOGY Pub Date : 2023-01-06 DOI: 10.2217/fnl-2022-0017
N. Ratcliffe, S. Cleanthous, John Andrejack, R. Barker, G. Blavat, William Brooks, S. Cano, Casey Gallagher, L. Gosden, Carroll Siu, A. Slagle, Kate Trenam, T. Morel, Karlin Schroeder
Clinical studies test whether a new treatment is safe and if the treatment works in people who have a particular condition. Most current questionnaires used in clinical studies investigating Parkinson's measure symptoms in people who have been diagnosed for many years. This means that these existing questionnaires may not be useful for people living with early-stage Parkinson's, where the symptoms experienced can be quite different to later stages, or may not show if a new treatment is helpful for them. The most common symptoms in Parkinson's are involuntary shaking of parts of the body (“tremor”), slow movement (“bradykinesia”) and stiff, inflexible muscles (“rigidity”), which worsen with time. Symptoms specific to early-stage Parkinson's are not fully understood and research is ongoing in this area. New measures are therefore needed to assess the symptoms affecting people living with early-stage Parkinson's, especially the symptoms that they find most troublesome. This study investigated which symptoms are of most importance to people in the earlier stages of their condition and which would be appropriate to measure in future clinical studies. The research team that led the study was made up of people living with Parkinson's, as well as technical experts and representatives from Parkinson's patient organizations (Parkinson's UK and the Parkinson's Foundation). The participants in the study were people living with early-stage Parkinson's and their care partners. Slowness of movement (called “bradykinesia”) was noted as a key symptom. “Functional slowness” was especially noted. This symptom caused people to feel slower during many daily tasks, such as brushing teeth, walking and cooking. The loss of ability to move easily and freely, termed “mobility”, was also a key symptom. It was noticeable in walking abnormalities and difficulties performing “fine motor skills”. These are tasks that require precision, dexterity and coordination. Other impactful symptoms were: tremor, rigidity/stiffness, feelings of exhaustion (fatigue), depression, sleeping problems and pain. The personal views gathered in this study show the wide-ranging effects of early-stage Parkinson's. The study also identifies functional slowness and loss of mobility as key symptoms that would be appropriate to measure in future early-stage Parkinson's clinical studies to test if treatments are working or not.
临床研究测试一种新的治疗方法是否安全,以及这种治疗方法是否对患有特定疾病的人有效。目前在临床研究中使用的大多数问卷调查帕金森氏症测量的是被诊断多年的人的症状。这意味着这些现有的问卷可能对早期帕金森氏症患者没有帮助,因为早期帕金森氏症患者的症状可能与晚期帕金森氏症患者截然不同,或者可能无法显示新的治疗方法是否对他们有帮助。帕金森氏症最常见的症状是身体部位不自主地颤抖(“震颤”)、运动缓慢(“运动迟缓”)和肌肉僵硬、不灵活(“僵硬”),这些症状随着时间的推移而恶化。早期帕金森病特有的症状尚不完全清楚,这方面的研究正在进行中。因此,需要新的措施来评估影响早期帕金森氏症患者的症状,特别是他们认为最麻烦的症状。这项研究调查了在病情的早期阶段,哪些症状对人们最重要,哪些症状适合在未来的临床研究中加以衡量。领导这项研究的研究小组由帕金森病患者、技术专家和帕金森患者组织(帕金森英国和帕金森基金会)的代表组成。该研究的参与者是早期帕金森氏症患者及其护理伙伴。运动缓慢(称为“运动迟缓”)被认为是一个关键症状。特别注意到“功能缓慢”。这种症状会导致人们在许多日常活动中感觉迟钝,比如刷牙、走路和做饭。丧失轻松自由活动的能力(称为“活动能力”)也是一个关键症状。在行走异常和执行“精细运动技能”方面的困难是显而易见的。这些都是需要精确、灵巧和协调的任务。其他有影响的症状有:震颤、僵硬/僵硬、疲惫感(疲劳)、抑郁、睡眠问题和疼痛。在这项研究中收集的个人观点显示了早期帕金森症的广泛影响。该研究还确定了功能迟缓和活动能力丧失是未来早期帕金森临床研究中适当测量的关键症状,以测试治疗是否有效。
{"title":"Plain language summary: what symptoms should be measured in clinical studies for early-stage Parkinson's?","authors":"N. Ratcliffe, S. Cleanthous, John Andrejack, R. Barker, G. Blavat, William Brooks, S. Cano, Casey Gallagher, L. Gosden, Carroll Siu, A. Slagle, Kate Trenam, T. Morel, Karlin Schroeder","doi":"10.2217/fnl-2022-0017","DOIUrl":"https://doi.org/10.2217/fnl-2022-0017","url":null,"abstract":"Clinical studies test whether a new treatment is safe and if the treatment works in people who have a particular condition. Most current questionnaires used in clinical studies investigating Parkinson's measure symptoms in people who have been diagnosed for many years. This means that these existing questionnaires may not be useful for people living with early-stage Parkinson's, where the symptoms experienced can be quite different to later stages, or may not show if a new treatment is helpful for them. The most common symptoms in Parkinson's are involuntary shaking of parts of the body (“tremor”), slow movement (“bradykinesia”) and stiff, inflexible muscles (“rigidity”), which worsen with time. Symptoms specific to early-stage Parkinson's are not fully understood and research is ongoing in this area. New measures are therefore needed to assess the symptoms affecting people living with early-stage Parkinson's, especially the symptoms that they find most troublesome. This study investigated which symptoms are of most importance to people in the earlier stages of their condition and which would be appropriate to measure in future clinical studies. The research team that led the study was made up of people living with Parkinson's, as well as technical experts and representatives from Parkinson's patient organizations (Parkinson's UK and the Parkinson's Foundation). The participants in the study were people living with early-stage Parkinson's and their care partners. Slowness of movement (called “bradykinesia”) was noted as a key symptom. “Functional slowness” was especially noted. This symptom caused people to feel slower during many daily tasks, such as brushing teeth, walking and cooking. The loss of ability to move easily and freely, termed “mobility”, was also a key symptom. It was noticeable in walking abnormalities and difficulties performing “fine motor skills”. These are tasks that require precision, dexterity and coordination. Other impactful symptoms were: tremor, rigidity/stiffness, feelings of exhaustion (fatigue), depression, sleeping problems and pain. The personal views gathered in this study show the wide-ranging effects of early-stage Parkinson's. The study also identifies functional slowness and loss of mobility as key symptoms that would be appropriate to measure in future early-stage Parkinson's clinical studies to test if treatments are working or not.","PeriodicalId":12606,"journal":{"name":"Future Neurology","volume":" ","pages":""},"PeriodicalIF":1.3,"publicationDate":"2023-01-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"44410516","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 1
Dynamics of clinical and neurological parameters and role of citicoline during therapeutic interventions in children with stroke 脑卒中患儿临床和神经参数的动态变化及胞胆碱在治疗干预中的作用
IF 1.3 Q4 CLINICAL NEUROLOGY Pub Date : 2023-01-01 DOI: 10.2217/fnl-2022-0009
Shaanvar Shamuradovich Shamansurov, S. Saidazizova, N. Tulyaganova, P. Usmanova, S. O. Nazarova
Aim: The rehabilitation of children who experience stroke is hampered by the lack of proven treatments and the choice of drugs and dosages. We compared clinical and neurological parameters in children receiving citicoline. Materials & methods: We assessed 199 children (128 boys, 64.3%) with stroke using the Pediatric Stroke Outcome Measure–Short Neurological Exam. Results: Hemorrhagic infarction was diagnosed more often than ischemic stroke, most often owing to the child’s early age (before and after 3 months). The presence of disorders of consciousness in the most acute and acute periods is noteworthy. Conclusion: The Pediatric Stroke Outcome Measure–Short Neurological Exam scale can be used to predict adverse outcomes. Citicoline can be administered early and is especially effective during the first year of life.
目的:由于缺乏有效的治疗方法以及药物和剂量的选择,经历中风的儿童的康复受到阻碍。我们比较了接受胞胆碱治疗的儿童的临床和神经学参数。材料与方法:我们使用儿童卒中结局测量-短期神经学检查评估了199名卒中儿童(128名男孩,64.3%)。结果:出血性梗死的诊断高于缺血性卒中,多因患儿年龄早(3个月前和3个月后)。意识障碍的存在在最急性和急性期是值得注意的。结论:小儿脑卒中预后测量-短神经学检查量表可用于预测不良预后。胞胆碱可以早期使用,在生命的第一年尤其有效。
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引用次数: 0
Aromatic L-amino acid decarboxylase deficiency: a systematic review 芳香族L-氨基酸脱羧酶缺乏症的系统综述
IF 1.3 Q4 CLINICAL NEUROLOGY Pub Date : 2022-09-23 DOI: 10.2217/fnl-2022-0012
Mats Bergkvist, C. Stephens, T. Schilling, Antonia Wang, Xiao-li Yu, Elizabeth Goodwin, L. Golden, A. Kristensen, Matthew Klein
Aim: To gain greater knowledge regarding the natural history of aromatic L-amino acid decarboxylase (AADC) deficiency, a genetic disorder that causes severe deficits in motor and cognitive development. Materials & methods: A systematic literature review was performed of all case reports and clinical studies published through December 2019 of patients with AADC deficiency. The data were summarized descriptively. Results: The search identified 94 publications that described 237 unique patients. Mean (standard deviation) age at diagnosis was 3.2 (±5.7) years and 16 deaths were reported. Most patients (57%) received the standard of care therapies, which showed limited efficacy in this patient population. Conclusion: AADC deficiency is a devastating disease and prospectively defined natural history studies are warranted to further understand this disease.
目的:了解芳香族L-氨基酸脱羧酶(AADC)缺乏症的自然史,这是一种导致运动和认知发育严重缺陷的遗传性疾病。材料和方法:对截至2019年12月发表的AADC缺乏症患者的所有病例报告和临床研究进行了系统的文献综述。对数据进行了描述性总结。结果:搜索发现了94篇描述237名独特患者的出版物。诊断时的平均(标准差)年龄为3.2(±5.7)岁,报告了16例死亡。大多数患者(57%)接受了标准护理疗法,在这一患者群体中显示出有限的疗效。结论:AADC缺乏症是一种破坏性疾病,有必要进行前瞻性的自然史研究来进一步了解这种疾病。
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引用次数: 5
期刊
Future Neurology
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