治疗肌无力的新时代:过去 6 年中的六种新药

IF 2 4区 医学 Q1 Medicine
Ashish D. Patel, Aashin Shah, J. David Avila
{"title":"治疗肌无力的新时代:过去 6 年中的六种新药","authors":"Ashish D. Patel, Aashin Shah, J. David Avila","doi":"10.1007/s11940-024-00783-w","DOIUrl":null,"url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Purpose of Review</h3><p>This article presents a summary of the new medications approved for generalized myasthenia gravis (gMG) since 2017. Pivotal clinical trials that led to the approval of these medications and their open-label extension studies are reviewed. We also provide information on healthcare cost when available. Lastly, we propose an approach to selecting therapies.</p><h3 data-test=\"abstract-sub-heading\">Recent Findings</h3><p>Six new medications have been approved for acetylcholine receptor antibody positive gMG. These include the complement inhibitors eculizumab, ravulizumab, and zilucoplan and the neonatal Fc receptor blockers efgartigimod, efgartigimod and hyaluronidase, and rozanolixizumab. The latter is also approved for muscle-specific kinase gMG. The Myasthenia Gravis Activities of Daily Living (MG-ADL) has become the most commonly used primary outcome measure in gMG clinical trials.</p><h3 data-test=\"abstract-sub-heading\">Summary</h3><p>All medications have shown safety and efficacy as measured by clinically meaningful changes in the MG-ADL. The medications differ in the need for meningococcal vaccination, the route and frequency of administration, and timing of treatments. Comparison studies are lacking and therefore there is limited evidence to guide the selection of therapy.</p>","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"195 1","pages":""},"PeriodicalIF":2.0000,"publicationDate":"2024-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"A New Era in the Treatment of Myasthenia Gravis: Six New Medications in The Last 6 Years\",\"authors\":\"Ashish D. Patel, Aashin Shah, J. David Avila\",\"doi\":\"10.1007/s11940-024-00783-w\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<h3 data-test=\\\"abstract-sub-heading\\\">Purpose of Review</h3><p>This article presents a summary of the new medications approved for generalized myasthenia gravis (gMG) since 2017. Pivotal clinical trials that led to the approval of these medications and their open-label extension studies are reviewed. We also provide information on healthcare cost when available. Lastly, we propose an approach to selecting therapies.</p><h3 data-test=\\\"abstract-sub-heading\\\">Recent Findings</h3><p>Six new medications have been approved for acetylcholine receptor antibody positive gMG. These include the complement inhibitors eculizumab, ravulizumab, and zilucoplan and the neonatal Fc receptor blockers efgartigimod, efgartigimod and hyaluronidase, and rozanolixizumab. The latter is also approved for muscle-specific kinase gMG. The Myasthenia Gravis Activities of Daily Living (MG-ADL) has become the most commonly used primary outcome measure in gMG clinical trials.</p><h3 data-test=\\\"abstract-sub-heading\\\">Summary</h3><p>All medications have shown safety and efficacy as measured by clinically meaningful changes in the MG-ADL. The medications differ in the need for meningococcal vaccination, the route and frequency of administration, and timing of treatments. Comparison studies are lacking and therefore there is limited evidence to guide the selection of therapy.</p>\",\"PeriodicalId\":10975,\"journal\":{\"name\":\"Current Treatment Options in Neurology\",\"volume\":\"195 1\",\"pages\":\"\"},\"PeriodicalIF\":2.0000,\"publicationDate\":\"2024-01-23\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Current Treatment Options in Neurology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s11940-024-00783-w\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"Medicine\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Current Treatment Options in Neurology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s11940-024-00783-w","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"Medicine","Score":null,"Total":0}
引用次数: 0

摘要

综述目的本文概述了自 2017 年以来获批治疗全身性肌无力(gMG)的新药。回顾了导致这些药物获批的关键临床试验及其开放标签扩展研究。我们还提供了可用的医疗成本信息。最后,我们提出了一种选择疗法的方法。最近的研究结果六种新药已被批准用于乙酰胆碱受体抗体阳性的 gMG。这些药物包括补体抑制剂 eculizumab、ravulizumab 和 zilucoplan,以及新生儿 Fc 受体阻断剂 efgartigimod、efgartigimod 和透明质酸酶以及 rozanolixizumab。后者还被批准用于治疗肌肉特异性激酶 gMG。摘要所有药物都显示出安全性和疗效,其衡量标准是 MG-ADL 有临床意义的变化。这些药物在接种脑膜炎球菌疫苗的必要性、给药途径和频率以及治疗时机方面存在差异。由于缺乏比较研究,因此指导选择疗法的证据有限。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
A New Era in the Treatment of Myasthenia Gravis: Six New Medications in The Last 6 Years

Purpose of Review

This article presents a summary of the new medications approved for generalized myasthenia gravis (gMG) since 2017. Pivotal clinical trials that led to the approval of these medications and their open-label extension studies are reviewed. We also provide information on healthcare cost when available. Lastly, we propose an approach to selecting therapies.

Recent Findings

Six new medications have been approved for acetylcholine receptor antibody positive gMG. These include the complement inhibitors eculizumab, ravulizumab, and zilucoplan and the neonatal Fc receptor blockers efgartigimod, efgartigimod and hyaluronidase, and rozanolixizumab. The latter is also approved for muscle-specific kinase gMG. The Myasthenia Gravis Activities of Daily Living (MG-ADL) has become the most commonly used primary outcome measure in gMG clinical trials.

Summary

All medications have shown safety and efficacy as measured by clinically meaningful changes in the MG-ADL. The medications differ in the need for meningococcal vaccination, the route and frequency of administration, and timing of treatments. Comparison studies are lacking and therefore there is limited evidence to guide the selection of therapy.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
CiteScore
4.00
自引率
0.00%
发文量
40
审稿时长
6-12 weeks
期刊介绍: This journal aims to review the most important, recently published treatment option advances in the field of neurology. By presenting clear, insightful, balanced contributions by international experts, the journal intends to facilitate worldwide approaches to the treatment of neurologic conditions. We accomplish this aim by appointing international authorities to serve as Section Editors in key subject areas, such as epilepsy, headache, neurologic ophthalmology and otology, neuromuscular disorders, psychiatric manifestations of neurologic disease, and sleep disorders. Section Editors select topics for which leading experts contribute comprehensive review articles that emphasize new developments and recently published papers of major importance, highlighted by annotated reference lists. We also provide commentaries from well-known neurologists, and an international Editorial Board reviews the annual table of contents, suggests articles of special interest to their country/region, and ensures that topics are current and include emerging research.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信