{"title":"实现重症肌无力表型的个性化管理:从多组学的作用到新兴的生物标记物和治疗靶点","authors":"Carmela Rita Balistreri , Claudia Vinciguerra , Daniele Magro , Vincenzo Di Stefano , Roberto Monastero","doi":"10.1016/j.autrev.2024.103669","DOIUrl":null,"url":null,"abstract":"<div><div>Predicting the onset, progression, and outcome of rare and chronic neurological diseases, i.e. neuromuscular diseases, is an important goal for both clinicians and researchers and should guide clinical decision-making and personalized treatment plans. A prime example is myasthenia gravis (MG), an antibody-mediated disease that affects multiple components of the postsynaptic membrane, impairing neuromuscular transmission and producing fatigable muscle weakness. MG is characterized by several clinical phenotypes, defined by a broad spectrum of factors, which have contributed to the current lack of consensus on the optimal management and treatments of this disease and its related phenotypes (subtypes). This represents a crucial challenge in MG and encourages a revolutionary change in diagnostic, prognostic and therapeutic guidelines. Emerging factors, such as demographic, clinical and pathophysiological factors, must also be considered. Consequently, the different MG phenotypes are characterized by precise biological signatures, which could represent appropriate biomarkers and targets. Here we describe and discuss these new concepts, highlighting that, thanks to multi-omics technologies, the identification of emerging diagnostic/prognostic biomarkers, such as miRNAs, and the subsequent development of new diagnostic/therapeutic algorithms could be facilitated. The latter, in turn, could facilitate the management of different MG phenotypes also in a personalized manner. Limitations and advantages are also reported.</div></div>","PeriodicalId":8664,"journal":{"name":"Autoimmunity reviews","volume":null,"pages":null},"PeriodicalIF":9.2000,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Towards personalized management of myasthenia gravis phenotypes: From the role of multi-omics to the emerging biomarkers and therapeutic targets\",\"authors\":\"Carmela Rita Balistreri , Claudia Vinciguerra , Daniele Magro , Vincenzo Di Stefano , Roberto Monastero\",\"doi\":\"10.1016/j.autrev.2024.103669\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>Predicting the onset, progression, and outcome of rare and chronic neurological diseases, i.e. neuromuscular diseases, is an important goal for both clinicians and researchers and should guide clinical decision-making and personalized treatment plans. A prime example is myasthenia gravis (MG), an antibody-mediated disease that affects multiple components of the postsynaptic membrane, impairing neuromuscular transmission and producing fatigable muscle weakness. MG is characterized by several clinical phenotypes, defined by a broad spectrum of factors, which have contributed to the current lack of consensus on the optimal management and treatments of this disease and its related phenotypes (subtypes). This represents a crucial challenge in MG and encourages a revolutionary change in diagnostic, prognostic and therapeutic guidelines. Emerging factors, such as demographic, clinical and pathophysiological factors, must also be considered. Consequently, the different MG phenotypes are characterized by precise biological signatures, which could represent appropriate biomarkers and targets. Here we describe and discuss these new concepts, highlighting that, thanks to multi-omics technologies, the identification of emerging diagnostic/prognostic biomarkers, such as miRNAs, and the subsequent development of new diagnostic/therapeutic algorithms could be facilitated. The latter, in turn, could facilitate the management of different MG phenotypes also in a personalized manner. Limitations and advantages are also reported.</div></div>\",\"PeriodicalId\":8664,\"journal\":{\"name\":\"Autoimmunity reviews\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":9.2000,\"publicationDate\":\"2024-10-18\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Autoimmunity reviews\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S1568997224001605\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"IMMUNOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Autoimmunity reviews","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S1568997224001605","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"IMMUNOLOGY","Score":null,"Total":0}
Towards personalized management of myasthenia gravis phenotypes: From the role of multi-omics to the emerging biomarkers and therapeutic targets
Predicting the onset, progression, and outcome of rare and chronic neurological diseases, i.e. neuromuscular diseases, is an important goal for both clinicians and researchers and should guide clinical decision-making and personalized treatment plans. A prime example is myasthenia gravis (MG), an antibody-mediated disease that affects multiple components of the postsynaptic membrane, impairing neuromuscular transmission and producing fatigable muscle weakness. MG is characterized by several clinical phenotypes, defined by a broad spectrum of factors, which have contributed to the current lack of consensus on the optimal management and treatments of this disease and its related phenotypes (subtypes). This represents a crucial challenge in MG and encourages a revolutionary change in diagnostic, prognostic and therapeutic guidelines. Emerging factors, such as demographic, clinical and pathophysiological factors, must also be considered. Consequently, the different MG phenotypes are characterized by precise biological signatures, which could represent appropriate biomarkers and targets. Here we describe and discuss these new concepts, highlighting that, thanks to multi-omics technologies, the identification of emerging diagnostic/prognostic biomarkers, such as miRNAs, and the subsequent development of new diagnostic/therapeutic algorithms could be facilitated. The latter, in turn, could facilitate the management of different MG phenotypes also in a personalized manner. Limitations and advantages are also reported.
期刊介绍:
Autoimmunity Reviews is a publication that features up-to-date, structured reviews on various topics in the field of autoimmunity. These reviews are written by renowned experts and include demonstrative illustrations and tables. Each article will have a clear "take-home" message for readers.
The selection of articles is primarily done by the Editors-in-Chief, based on recommendations from the international Editorial Board. The topics covered in the articles span all areas of autoimmunology, aiming to bridge the gap between basic and clinical sciences.
In terms of content, the contributions in basic sciences delve into the pathophysiology and mechanisms of autoimmune disorders, as well as genomics and proteomics. On the other hand, clinical contributions focus on diseases related to autoimmunity, novel therapies, and clinical associations.
Autoimmunity Reviews is internationally recognized, and its articles are indexed and abstracted in prestigious databases such as PubMed/Medline, Science Citation Index Expanded, Biosciences Information Services, and Chemical Abstracts.