Pub Date : 2024-10-01DOI: 10.1016/S1474-4422(24)00322-3
Ali M Alawieh, Hassan Saad, Jonathan A Grossberg, Daniel L Barrow
{"title":"Considerations for future trials in cerebral cavernous malformations.","authors":"Ali M Alawieh, Hassan Saad, Jonathan A Grossberg, Daniel L Barrow","doi":"10.1016/S1474-4422(24)00322-3","DOIUrl":"10.1016/S1474-4422(24)00322-3","url":null,"abstract":"","PeriodicalId":17989,"journal":{"name":"Lancet Neurology","volume":"23 10","pages":"964-965"},"PeriodicalIF":46.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142290351","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-10-01DOI: 10.1016/S1474-4422(24)00375-2
Peter Ranscombe
{"title":"On the trail of Edinburgh's neuroscience past and present.","authors":"Peter Ranscombe","doi":"10.1016/S1474-4422(24)00375-2","DOIUrl":"10.1016/S1474-4422(24)00375-2","url":null,"abstract":"","PeriodicalId":17989,"journal":{"name":"Lancet Neurology","volume":"23 10","pages":"972"},"PeriodicalIF":46.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142290363","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-10-01DOI: 10.1016/S1474-4422(24)00256-4
Jorge Correale, Andrew J Solomon, Jeffrey A Cohen, Brenda L Banwell, Fernando Gracia, Tirisham V Gyang, Fernando Hamuy Diaz de Bedoya, Mary P Harnegie, Bernhard Hemmer, Anu Jacob, Ho Jin Kim, Ruth Ann Marrie, Farrah J Mateen, Scott D Newsome, Lekha Pandit, Naraporn Prayoonwiwat, Mohammad A Sahraian, Douglas K Sato, Deanna Saylor, Fu-Dong Shi, Aksel Siva, Kevin Tan, Shanthi Viswanathan, Mike P Wattjes, Brian Weinshenker, Bassem Yamout, Kazuo Fujihara
The differential diagnosis of multiple sclerosis can present specific challenges in patients from Latin America, Africa, the Middle East, eastern Europe, southeast Asia, and the Western Pacific. In these areas, environmental factors, genetic background, and access to medical care can differ substantially from those in North America and western Europe, where multiple sclerosis is most common. Furthermore, multiple sclerosis diagnostic criteria have been developed primarily using data from North America and western Europe. Although some diagnoses mistaken for multiple sclerosis are common regardless of location, a comprehensive approach to the differential diagnosis of multiple sclerosis in Latin America, Africa, the Middle East, eastern Europe, southeast Asia, and the Western Pacific regions requires special consideration of diseases that are prevalent in those locations. A collaborative effort has therefore assessed global differences in multiple sclerosis differential diagnoses and proposed recommendations for evaluating patients with suspected multiple sclerosis in regions beyond North America and western Europe.
{"title":"Differential diagnosis of suspected multiple sclerosis: global health considerations.","authors":"Jorge Correale, Andrew J Solomon, Jeffrey A Cohen, Brenda L Banwell, Fernando Gracia, Tirisham V Gyang, Fernando Hamuy Diaz de Bedoya, Mary P Harnegie, Bernhard Hemmer, Anu Jacob, Ho Jin Kim, Ruth Ann Marrie, Farrah J Mateen, Scott D Newsome, Lekha Pandit, Naraporn Prayoonwiwat, Mohammad A Sahraian, Douglas K Sato, Deanna Saylor, Fu-Dong Shi, Aksel Siva, Kevin Tan, Shanthi Viswanathan, Mike P Wattjes, Brian Weinshenker, Bassem Yamout, Kazuo Fujihara","doi":"10.1016/S1474-4422(24)00256-4","DOIUrl":"10.1016/S1474-4422(24)00256-4","url":null,"abstract":"<p><p>The differential diagnosis of multiple sclerosis can present specific challenges in patients from Latin America, Africa, the Middle East, eastern Europe, southeast Asia, and the Western Pacific. In these areas, environmental factors, genetic background, and access to medical care can differ substantially from those in North America and western Europe, where multiple sclerosis is most common. Furthermore, multiple sclerosis diagnostic criteria have been developed primarily using data from North America and western Europe. Although some diagnoses mistaken for multiple sclerosis are common regardless of location, a comprehensive approach to the differential diagnosis of multiple sclerosis in Latin America, Africa, the Middle East, eastern Europe, southeast Asia, and the Western Pacific regions requires special consideration of diseases that are prevalent in those locations. A collaborative effort has therefore assessed global differences in multiple sclerosis differential diagnoses and proposed recommendations for evaluating patients with suspected multiple sclerosis in regions beyond North America and western Europe.</p>","PeriodicalId":17989,"journal":{"name":"Lancet Neurology","volume":"23 10","pages":"1035-1049"},"PeriodicalIF":46.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142290354","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-10-01DOI: 10.1016/S1474-4422(24)00171-6
Carlos M Madrid-Casado
{"title":"Health as a philosophical idea.","authors":"Carlos M Madrid-Casado","doi":"10.1016/S1474-4422(24)00171-6","DOIUrl":"10.1016/S1474-4422(24)00171-6","url":null,"abstract":"","PeriodicalId":17989,"journal":{"name":"Lancet Neurology","volume":"23 10","pages":"969"},"PeriodicalIF":46.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142290357","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-10-01DOI: 10.1016/S1474-4422(24)00309-0
Jeffrey A Allen, Jie Lin, Ivana Basta, Tina Dysgaard, Christian Eggers, Jeffrey T Guptill, Kelly G Gwathmey, Channa Hewamadduma, Erik Hofman, Yessar M Hussain, Satoshi Kuwabara, Gwendal Le Masson, Frank Leypoldt, Ting Chang, Marta Lipowska, Murray Lowe, Giuseppe Lauria, Luis Querol, Mihaela-Adriana Simu, Niraja Suresh, Anissa Tse, Peter Ulrichts, Benjamin Van Hoorick, Ryo Yamasaki, Richard A Lewis, Pieter A van Doorn
<p><strong>Background: </strong>Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is an autoimmune disease of the peripheral nervous system that can lead to severe disability from muscle weakness and sensory disturbances. Around a third of patients do not respond to currently available treatments, and many patients with a partial response have residual neurological impairment, highlighting the need for effective alternatives. Efgartigimod alfa, a human IgG1 antibody Fc fragment, has demonstrated efficacy and safety in patients with generalised myasthenia gravis. We evaluated the safety, tolerability, and efficacy of subcutaneous efgartigimod PH20 in adults with CIDP.</p><p><strong>Methods: </strong>ADHERE, a multistage, double-blind, placebo-controlled trial, enrolled participants with CIDP from 146 clinical sites from Asia-Pacific, Europe, and North America. Participants with evidence of clinically meaningful deterioration entered an open-label phase of weekly 1000 mg subcutaneous efgartigimod PH20 for no longer than 12 weeks (stage A). Those with confirmed evidence of clinical improvement (ECI; treatment responders) entered a randomised-withdrawal phase of 1000 mg subcutaneous efgartigimod PH20 weekly treatment versus placebo for a maximum of 48 weeks (stage B). Participants were randomised (1:1) through interactive response technology and stratified by their adjusted Inflammatory Neuropathy Cause and Treatment (aINCAT) score change during stage A and their most recent CIDP medication within 6 months before screening. Investigators, the clinical research organisation, and participants were masked to the treatment. The primary endpoint in stage A, evaluated in the stage A safety population, was confirmed ECI (≥1 points aINCAT decrease, ≥4 points [centile metric] Inflammatory Rasch-built Overall Disability Scale increase, or ≥8 kPa grip strength increase after four injections and two consecutive visits). The primary endpoint in stage B, evaluated in the modified intention-to-treat population, was the risk of relapse (time to first aINCAT increase of ≥1 points). ADHERE is registered with ClinicalTrials.gov (NCT04281472) and EudraCT (2019-003076-39) and is completed.</p><p><strong>Findings: </strong>Between April 15, 2020, and May 11, 2023, 629 participants were screened; 322 (114 female, 208 male) entered stage A, of whom 214 (66%, 95% CI 61·0-71·6) had confirmed ECI. In stage B, 221 participants were randomised (79 female, 142 male; 111 to subcutaneous efgartigimod PH20, 110 to placebo). Subcutaneous efgartigimod PH20 significantly reduced the risk of relapse versus placebo (hazard ratio 0·39 [95% CI 0·25-0·61]; p<0·0001). 31 (27·9% [19·6-36·3]) participants given subcutaneous efgartigimod PH20 had a relapse versus 59 (53·6% [44·3-63·0]) given placebo. In stage A, treatment-emergent adverse events (TEAEs) occurred in 204 (63%) participants and serious TEAEs in 21 (7%). In stage B, TEAEs occurred in 71 (64%) participants on subcutan
背景：慢性炎症性脱髓鞘多发性神经病（CIDP）是一种外周神经系统自身免疫性疾病，可导致肌肉无力和感觉障碍等严重残疾。约有三分之一的患者对目前可用的治疗方法没有反应，许多有部分反应的患者会残留神经损伤，这凸显了对有效替代疗法的需求。Efgartigimod alfa 是一种人类 IgG1 抗体 Fc 片段，已在全身性肌无力患者中证明了其有效性和安全性。我们评估了皮下注射依加替莫德 PH20 对成人 CIDP 患者的安全性、耐受性和疗效：ADHERE是一项多阶段、双盲、安慰剂对照试验，从亚太地区、欧洲和北美的146个临床研究机构招募了CIDP患者。有临床意义的病情恶化证据的参与者进入开放标签阶段，每周皮下注射1000毫克依加替莫德PH20，疗程不超过12周（A阶段）。有确凿证据表明临床症状有所改善（ECI；治疗应答者）的患者进入随机撤药阶段，每周皮下注射1000毫克依加替莫德PH20与安慰剂对比治疗，最多持续48周（B阶段）。参与者通过交互响应技术进行随机分配（1:1），并根据他们在A阶段的调整后炎症性神经病病因和治疗（aINCAT）评分变化以及他们在筛查前6个月内最近服用的CIDP药物进行分层。研究人员、临床研究机构和参与者均对治疗进行了蒙蔽。A阶段的主要终点在A阶段的安全人群中进行评估，即确认ECI（在四次注射和连续两次就诊后，aINCAT下降≥1分，炎症性Rasch-built总体残疾量表增加≥4分[百分位数]，或握力增加≥8千帕）。B 阶段的主要终点是复发风险（AINCAT 首次增加≥1 分的时间），该终点在修改后的意向治疗人群中进行评估。ADHERE已在ClinicalTrials.gov（NCT04281472）和EudraCT（2019-003076-39）注册，研究结果已完成：2020年4月15日至2023年5月11日期间，共筛选出629名参与者；322人（女性114人，男性208人）进入A阶段，其中214人（66%，95% CI 61-0-71-6）确诊为ECI。在B阶段，221名参与者接受了随机治疗（79名女性，142名男性；111人接受皮下注射依加替莫德PH20治疗，110人接受安慰剂治疗）。与安慰剂相比，皮下注射依夫加替莫德 PH20 能显著降低复发风险（危险比为 0-39 [95% CI 0-25-0-61]; p解释：ADHERE显示，与安慰剂相比，皮下注射依加替莫德PH20能有效降低对治疗有反应的CIDP患者的复发风险。还需要进一步的研究来提供有关依加替莫德α长期疗效的数据，以及它与现有治疗方案的比较。
{"title":"Safety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE): a multicentre, randomised-withdrawal, double-blind, placebo-controlled, phase 2 trial.","authors":"Jeffrey A Allen, Jie Lin, Ivana Basta, Tina Dysgaard, Christian Eggers, Jeffrey T Guptill, Kelly G Gwathmey, Channa Hewamadduma, Erik Hofman, Yessar M Hussain, Satoshi Kuwabara, Gwendal Le Masson, Frank Leypoldt, Ting Chang, Marta Lipowska, Murray Lowe, Giuseppe Lauria, Luis Querol, Mihaela-Adriana Simu, Niraja Suresh, Anissa Tse, Peter Ulrichts, Benjamin Van Hoorick, Ryo Yamasaki, Richard A Lewis, Pieter A van Doorn","doi":"10.1016/S1474-4422(24)00309-0","DOIUrl":"10.1016/S1474-4422(24)00309-0","url":null,"abstract":"<p><strong>Background: </strong>Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is an autoimmune disease of the peripheral nervous system that can lead to severe disability from muscle weakness and sensory disturbances. Around a third of patients do not respond to currently available treatments, and many patients with a partial response have residual neurological impairment, highlighting the need for effective alternatives. Efgartigimod alfa, a human IgG1 antibody Fc fragment, has demonstrated efficacy and safety in patients with generalised myasthenia gravis. We evaluated the safety, tolerability, and efficacy of subcutaneous efgartigimod PH20 in adults with CIDP.</p><p><strong>Methods: </strong>ADHERE, a multistage, double-blind, placebo-controlled trial, enrolled participants with CIDP from 146 clinical sites from Asia-Pacific, Europe, and North America. Participants with evidence of clinically meaningful deterioration entered an open-label phase of weekly 1000 mg subcutaneous efgartigimod PH20 for no longer than 12 weeks (stage A). Those with confirmed evidence of clinical improvement (ECI; treatment responders) entered a randomised-withdrawal phase of 1000 mg subcutaneous efgartigimod PH20 weekly treatment versus placebo for a maximum of 48 weeks (stage B). Participants were randomised (1:1) through interactive response technology and stratified by their adjusted Inflammatory Neuropathy Cause and Treatment (aINCAT) score change during stage A and their most recent CIDP medication within 6 months before screening. Investigators, the clinical research organisation, and participants were masked to the treatment. The primary endpoint in stage A, evaluated in the stage A safety population, was confirmed ECI (≥1 points aINCAT decrease, ≥4 points [centile metric] Inflammatory Rasch-built Overall Disability Scale increase, or ≥8 kPa grip strength increase after four injections and two consecutive visits). The primary endpoint in stage B, evaluated in the modified intention-to-treat population, was the risk of relapse (time to first aINCAT increase of ≥1 points). ADHERE is registered with ClinicalTrials.gov (NCT04281472) and EudraCT (2019-003076-39) and is completed.</p><p><strong>Findings: </strong>Between April 15, 2020, and May 11, 2023, 629 participants were screened; 322 (114 female, 208 male) entered stage A, of whom 214 (66%, 95% CI 61·0-71·6) had confirmed ECI. In stage B, 221 participants were randomised (79 female, 142 male; 111 to subcutaneous efgartigimod PH20, 110 to placebo). Subcutaneous efgartigimod PH20 significantly reduced the risk of relapse versus placebo (hazard ratio 0·39 [95% CI 0·25-0·61]; p<0·0001). 31 (27·9% [19·6-36·3]) participants given subcutaneous efgartigimod PH20 had a relapse versus 59 (53·6% [44·3-63·0]) given placebo. In stage A, treatment-emergent adverse events (TEAEs) occurred in 204 (63%) participants and serious TEAEs in 21 (7%). In stage B, TEAEs occurred in 71 (64%) participants on subcutan","PeriodicalId":17989,"journal":{"name":"Lancet Neurology","volume":"23 10","pages":"1013-1024"},"PeriodicalIF":46.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142290369","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-01Epub Date: 2024-07-23DOI: 10.1016/S1474-4422(24)00284-9
Susanne Petri
{"title":"Targeting C9orf72 in people with ALS.","authors":"Susanne Petri","doi":"10.1016/S1474-4422(24)00284-9","DOIUrl":"10.1016/S1474-4422(24)00284-9","url":null,"abstract":"","PeriodicalId":17989,"journal":{"name":"Lancet Neurology","volume":" ","pages":"850-852"},"PeriodicalIF":46.5,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141766472","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-01Epub Date: 2024-06-20DOI: 10.1016/S1474-4422(24)00261-8
Bruce C V Campbell
{"title":"Durable benefit of thrombectomy 6-24 h after stroke onset.","authors":"Bruce C V Campbell","doi":"10.1016/S1474-4422(24)00261-8","DOIUrl":"10.1016/S1474-4422(24)00261-8","url":null,"abstract":"","PeriodicalId":17989,"journal":{"name":"Lancet Neurology","volume":" ","pages":"849-850"},"PeriodicalIF":46.5,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141442985","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
扫码关注我们
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号