Pub Date : 2024-01-30DOI: 10.1007/s11940-024-00781-y
Christine Meadows, Naraharisetty Anita Rau, Warda Faridi, Cindy V. Ly
Purpose of Review
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease causing weakness, respiratory failure, and death within 3 to 5 years. Approximately, 10% of ALS cases have a genetic etiology (familial/fALS). The etiology of the remaining 90% of sporadic ALS (sALS) cases remains unknown. In this review, we provide an overview of approved and investigational therapies for fALS, as well as genetically informed therapeutic advances aimed at the larger sALS population.
Recent Findings
Antisense oligonucleotides (ASOs) are a promising strategy to treat toxic gain-of-function mutations underlying most forms of fALS. We discuss the recent approval of tofersen for ALS caused by mutation in SOD1. We also discuss progress in the development of therapies for fALS associated with C9orf72 hexanucleotide repeat expansions (C9orf72) and fused in sarcoma (FUS) mutations. Finally, we will discuss the rationale and status of molecular therapies for sALS targeting mediators of TDP-43 pathogenesis: ataxin-2 (ATXN2) and stathmin-2 (STMN2).
Summary
Advances in understanding the genetics of ALS have propelled the development of promising gene therapies. Lessons learned from tofersen continue to inform clinical trial design for a growing pipeline of therapies directed towards other fALS subtypes and sALS.
{"title":"Translating the ALS Genetic Revolution into Therapies: A Review","authors":"Christine Meadows, Naraharisetty Anita Rau, Warda Faridi, Cindy V. Ly","doi":"10.1007/s11940-024-00781-y","DOIUrl":"https://doi.org/10.1007/s11940-024-00781-y","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Purpose of Review</h3><p>Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease causing weakness, respiratory failure, and death within 3 to 5 years. Approximately, 10% of ALS cases have a genetic etiology (familial/fALS). The etiology of the remaining 90% of sporadic ALS (sALS) cases remains unknown. In this review, we provide an overview of approved and investigational therapies for fALS, as well as genetically informed therapeutic advances aimed at the larger sALS population.</p><h3 data-test=\"abstract-sub-heading\">Recent Findings</h3><p>Antisense oligonucleotides (ASOs) are a promising strategy to treat toxic gain-of-function mutations underlying most forms of fALS. We discuss the recent approval of tofersen for ALS caused by mutation in <i>SOD1</i>. We also discuss progress in the development of therapies for fALS associated with <i>C9orf72</i> hexanucleotide repeat expansions (<i>C9orf72</i>) and fused in sarcoma (<i>FUS</i>) mutations. Finally, we will discuss the rationale and status of molecular therapies for sALS targeting mediators of TDP-43 pathogenesis: ataxin-2 (<i>ATXN2</i>) and stathmin-2 (<i>STMN2</i>).</p><h3 data-test=\"abstract-sub-heading\">Summary</h3><p>Advances in understanding the genetics of ALS have propelled the development of promising gene therapies. Lessons learned from tofersen continue to inform clinical trial design for a growing pipeline of therapies directed towards other fALS subtypes and sALS.</p>","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"22 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-01-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139579320","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-01-23DOI: 10.1007/s11940-024-00783-w
Ashish D. Patel, Aashin Shah, J. David Avila
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.
{"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":"https://doi.org/10.1007/s11940-024-00783-w","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":2.0,"publicationDate":"2024-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139559993","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-01-18DOI: 10.1007/s11940-024-00784-9
Lauryn Currens, Alexander Pantelyat
Purpose of review
This review describes the current approaches to the diagnosis and management of progressive supranuclear palsy (PSP)
Recent findings
PSP is an atypical parkinsonian disorder associated with the accumulation of abnormal 4-repeat tau protein in the brain. Initially, the recognized clinical phenotype included a progressive disorder with vertical supranuclear gaze palsy and prominent postural instability leading to early falls. However, the current PSP diagnostic criteria recognize a broader range of clinical PSP presentations and define eight clinical PSP variants according to the levels of diagnostic certainty. While definite PSP remains a neuropathological diagnosis, imaging modalities including brain magnetic resonance imaging (MRI), dopamine transporter (DAT), and tau positron emission tomography (PET) scans may aid in the diagnosis. In the future, new tau PET ligands and CSF and genetic biomarkers may improve diagnostic accuracy. There is no disease-modifying therapy currently available for PSP. However, there are many pharmacological and non-pharmacological treatment options for symptomatic management. Because PSP is a multisystem disease, optimal management requires a coordinated multidisciplinary team approach.
Summary
PSP is a fatal multisystem disease that can be challenging to diagnose and manage. However, improved clinical diagnostic criteria, emerging biomarkers, and availability of useful therapeutic approaches provide cause for optimism.
综述目的本综述介绍了目前诊断和治疗进行性核上性麻痹(PSP)的方法。最初,公认的临床表型包括伴有垂直核上凝视麻痹和突出的姿势不稳导致早期跌倒的进行性障碍。然而,目前的 PSP 诊断标准认识到 PSP 的临床表现范围更广,并根据诊断的确定程度定义了八种临床 PSP 变体。虽然明确的 PSP 仍属于神经病理学诊断,但包括脑磁共振成像(MRI)、多巴胺转运体(DAT)和 tau 正电子发射断层扫描(PET)在内的成像模式可能有助于诊断。未来,新的 tau PET 配体以及 CSF 和基因生物标记物可能会提高诊断的准确性。目前还没有针对 PSP 的疾病改变疗法。不过,有许多药物和非药物治疗方法可用于对症治疗。由于 PSP 是一种多系统疾病,因此最佳治疗需要多学科团队的协调配合。然而,临床诊断标准的改进、新出现的生物标记物以及有用的治疗方法的出现,都让人有理由感到乐观。
{"title":"Progressive Supranuclear Palsy Diagnosis and Treatment","authors":"Lauryn Currens, Alexander Pantelyat","doi":"10.1007/s11940-024-00784-9","DOIUrl":"https://doi.org/10.1007/s11940-024-00784-9","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Purpose of review</h3><p>This review describes the current approaches to the diagnosis and management of progressive supranuclear palsy (PSP)</p><h3 data-test=\"abstract-sub-heading\">Recent findings</h3><p>PSP is an atypical parkinsonian disorder associated with the accumulation of abnormal 4-repeat tau protein in the brain. Initially, the recognized clinical phenotype included a progressive disorder with vertical supranuclear gaze palsy and prominent postural instability leading to early falls. However, the current PSP diagnostic criteria recognize a broader range of clinical PSP presentations and define eight clinical PSP variants according to the levels of diagnostic certainty. While definite PSP remains a neuropathological diagnosis, imaging modalities including brain magnetic resonance imaging (MRI), dopamine transporter (DAT), and tau positron emission tomography (PET) scans may aid in the diagnosis. In the future, new tau PET ligands and CSF and genetic biomarkers may improve diagnostic accuracy. There is no disease-modifying therapy currently available for PSP. However, there are many pharmacological and non-pharmacological treatment options for symptomatic management. Because PSP is a multisystem disease, optimal management requires a coordinated multidisciplinary team approach.</p><h3 data-test=\"abstract-sub-heading\">Summary</h3><p>PSP is a fatal multisystem disease that can be challenging to diagnose and manage. However, improved clinical diagnostic criteria, emerging biomarkers, and availability of useful therapeutic approaches provide cause for optimism.</p>","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"34 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-01-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139501526","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-01-11DOI: 10.1007/s11940-024-00782-x
Avi Landman, Mark Levine, Andrés M. De León
Purpose of review
To describe the clinical presentation, diagnosis, and treatment options of vasculitic neuropathies. Overall, vasculitic neuropathies can be divided into systemic and nonsystemic. Systemic vasculitic neuropathies form part of rheumatological diseases, where in some cases, the vasculitic is the predominant feature, and these are called primary vasculitis, while in others, the vasculitis is secondary to a connective tissue disease. Nonsystemic vasculitic neuropathies involve only the peripheral nervous system, and thus, diagnosis is more challenging as it requires a high degree of suspicion and confirmation with nerve biopsy results.
Recent findings
Over the past few years, rituximab has been increasingly used for induction and maintenance treatment of vasculitis due to a more favorable side effect profile and similar efficacy when compared to cyclophosphamide.
Summary
Herein, we describe the different types of vasculitic neuropathies. Regarding their treatment, have focused on the indications, dosing, and side effects for the most common options for primary systemic vasculitic neuropathies, as these tend to be similar for the classical nonsystemic vasculitic neuropathy phenotype.
{"title":"Vasculitic Neuropathies","authors":"Avi Landman, Mark Levine, Andrés M. De León","doi":"10.1007/s11940-024-00782-x","DOIUrl":"https://doi.org/10.1007/s11940-024-00782-x","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Purpose of review</h3><p>To describe the clinical presentation, diagnosis, and treatment options of vasculitic neuropathies. Overall, vasculitic neuropathies can be divided into systemic and nonsystemic. Systemic vasculitic neuropathies form part of rheumatological diseases, where in some cases, the vasculitic is the predominant feature, and these are called primary vasculitis, while in others, the vasculitis is secondary to a connective tissue disease. Nonsystemic vasculitic neuropathies involve only the peripheral nervous system, and thus, diagnosis is more challenging as it requires a high degree of suspicion and confirmation with nerve biopsy results.</p><h3 data-test=\"abstract-sub-heading\">Recent findings</h3><p>Over the past few years, rituximab has been increasingly used for induction and maintenance treatment of vasculitis due to a more favorable side effect profile and similar efficacy when compared to cyclophosphamide.</p><h3 data-test=\"abstract-sub-heading\">Summary</h3><p>Herein, we describe the different types of vasculitic neuropathies. Regarding their treatment, have focused on the indications, dosing, and side effects for the most common options for primary systemic vasculitic neuropathies, as these tend to be similar for the classical nonsystemic vasculitic neuropathy phenotype.</p>","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"4 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-01-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139461100","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-07DOI: 10.1007/s11940-023-00775-2
Alicia O’Hara, Marie Newkirk, Mina Girgis, Carrie Esopenko, David Putrino, Laura Tabacof, Christopher Kellner, Jenna M. Tosto-Mancuso
Purpose of review
This review appraises the existing literature to highlight the benefits, barriers, and role of rehabilitation in the early mobilization of patients in neurological critical care.
Recent findings
While the benefits of early mobilization in patients with critical illness have become more widely acknowledged, there are benefits and challenges to the successful implementation of early mobilization rehabilitation programming in the neurological critical care setting. The imperative role of rehabilitation providers is underscored in successful implementation. Furthermore, the discrete needs of patients and caregivers in neurocritical care necessitate interdisciplinary collaboration among medical and rehabilitation teams for the successful deployment of early mobilization programs.
Summary
This work discusses the expanding role of rehabilitation providers in neurocritical care and the importance of early mobilization for patients with neurological injuries. Experience-dependent neuroplasticity is a primary mechanism in facilitating recovery following neurological injury, and physical rehabilitation plays a critical role in this process. There is, however, a discrepancy in nomenclature regarding the definition of “early mobilization,” which makes it challenging to synthesize and translate evidence into practice. Despite the benefits of early mobilization, applying this evidence to the neurocritically ill patient is controversial given the innate medical complexity associated with these diagnoses. Nonetheless, early mobilization has been shown to decrease ICU and hospital length of stay, increase discharge to home, and reduce medical costs. Furthermore, consideration for those with severe acute brain injury and necessary medical interventions should be accounted for. While extensive literature has demonstrated the feasibility of adopting early mobilization to minimize secondary complications of ICU episodes of care, adoption of this paradigm has progressively expanded in the neurological population. Factors including the timing and frequency of mobilization are unclear and vary among studies, suggesting that more research is needed to better understand the role and benefits of early mobilization in neurocritical care.
{"title":"The Role of Rehabilitation in Neurological Critical Care: Innovations in Early Mobilization","authors":"Alicia O’Hara, Marie Newkirk, Mina Girgis, Carrie Esopenko, David Putrino, Laura Tabacof, Christopher Kellner, Jenna M. Tosto-Mancuso","doi":"10.1007/s11940-023-00775-2","DOIUrl":"https://doi.org/10.1007/s11940-023-00775-2","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Purpose of review</h3><p>This review appraises the existing literature to highlight the benefits, barriers, and role of rehabilitation in the early mobilization of patients in neurological critical care.</p><h3 data-test=\"abstract-sub-heading\">Recent findings</h3><p>While the benefits of early mobilization in patients with critical illness have become more widely acknowledged, there are benefits and challenges to the successful implementation of early mobilization rehabilitation programming in the neurological critical care setting. The imperative role of rehabilitation providers is underscored in successful implementation. Furthermore, the discrete needs of patients and caregivers in neurocritical care necessitate interdisciplinary collaboration among medical and rehabilitation teams for the successful deployment of early mobilization programs.</p><h3 data-test=\"abstract-sub-heading\">Summary</h3><p>This work discusses the expanding role of rehabilitation providers in neurocritical care and the importance of early mobilization for patients with neurological injuries. Experience-dependent neuroplasticity is a primary mechanism in facilitating recovery following neurological injury, and physical rehabilitation plays a critical role in this process. There is, however, a discrepancy in nomenclature regarding the definition of “early mobilization,” which makes it challenging to synthesize and translate evidence into practice. Despite the benefits of early mobilization, applying this evidence to the neurocritically ill patient is controversial given the innate medical complexity associated with these diagnoses. Nonetheless, early mobilization has been shown to decrease ICU and hospital length of stay, increase discharge to home, and reduce medical costs. Furthermore, consideration for those with severe acute brain injury and necessary medical interventions should be accounted for. While extensive literature has demonstrated the feasibility of adopting early mobilization to minimize secondary complications of ICU episodes of care, adoption of this paradigm has progressively expanded in the neurological population. Factors including the timing and frequency of mobilization are unclear and vary among studies, suggesting that more research is needed to better understand the role and benefits of early mobilization in neurocritical care.</p>","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"108 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2023-12-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138548342","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-12-05DOI: 10.1007/s11940-023-00778-z
Kristopher A. Hendershot, Maya N. Elias, Breana L. Taylor, Sarah Wahlster, Claire J. Creutzfeldt
Purpose of review
We investigate the complexities and interplay between the concepts of prognostic uncertainty and patient preferences as they relate to the delivery of goal-concordant care to patients with severe acute brain injuries (SABI) in the Neurological Intensive Care Unit (Neuro-ICU).
Recent findings
Patients with SABI in the Neuro-ICU have unique palliative care needs due to sudden, often unexpected changes in personhood and quality of life. A substantial amount of uncertainty is inherent and poses a challenge to both the patient’s prognosis and treatment preferences. The delivery of goal-concordant care can be difficult to achieve.
Summary
The uncertainty inherent to both prognosis and patient preferences challenges the provision of goal-concordant care to patients with SABI. The best case/worst case/most likely case scenario is a communication strategy that can aid clinicians when discussing the patient’s uncertain prognosis. A time-limited trial may provide a framework for families and clinicians to pursue aggressive life-sustaining treatment for a certain amount of time, in which prognosis may become more defined, patient goals may become clearer, and clinicians and families may establish a common ground. Although the delivery of goal-concordant care in the Neuro-ICU may be an unachievable, lofty goal, it is a level of care that we should continue to strive for and discuss.
{"title":"An Update on Palliative Care in Neurocritical Care: Providing Goal-Concordant Care in the Face of Prognostic Uncertainty","authors":"Kristopher A. Hendershot, Maya N. Elias, Breana L. Taylor, Sarah Wahlster, Claire J. Creutzfeldt","doi":"10.1007/s11940-023-00778-z","DOIUrl":"https://doi.org/10.1007/s11940-023-00778-z","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Purpose of review</h3><p>We investigate the complexities and interplay between the concepts of prognostic uncertainty and patient preferences as they relate to the delivery of goal-concordant care to patients with severe acute brain injuries (SABI) in the Neurological Intensive Care Unit (Neuro-ICU).</p><h3 data-test=\"abstract-sub-heading\">Recent findings</h3><p>Patients with SABI in the Neuro-ICU have unique palliative care needs due to sudden, often unexpected changes in personhood and quality of life. A substantial amount of uncertainty is inherent and poses a challenge to both the patient’s prognosis and treatment preferences. The delivery of goal-concordant care can be difficult to achieve.</p><h3 data-test=\"abstract-sub-heading\">Summary</h3><p>The uncertainty inherent to both prognosis and patient preferences challenges the provision of goal-concordant care to patients with SABI. The best case/worst case/most likely case scenario is a communication strategy that can aid clinicians when discussing the patient’s uncertain prognosis. A time-limited trial may provide a framework for families and clinicians to pursue aggressive life-sustaining treatment for a certain amount of time, in which prognosis may become more defined, patient goals may become clearer, and clinicians and families may establish a common ground. Although the delivery of goal-concordant care in the Neuro-ICU may be an unachievable, lofty goal, it is a level of care that we should continue to strive for and discuss.</p>","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"20 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2023-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138516181","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-22DOI: 10.1007/s11940-023-00773-4
Allison L. Nogi, Daniel Ludwig, Jennifer Millar
{"title":"Correction to: Clinical Practice Update Part I: Diagnosis and Treatment for Benign Paroxysmal Positional Vertigo","authors":"Allison L. Nogi, Daniel Ludwig, Jennifer Millar","doi":"10.1007/s11940-023-00773-4","DOIUrl":"https://doi.org/10.1007/s11940-023-00773-4","url":null,"abstract":"","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"5 2","pages":"1"},"PeriodicalIF":2.0,"publicationDate":"2023-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139247970","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-22DOI: 10.1007/s11940-023-00776-1
Andrew B. Wolf, Jacqueline Palace, Jeffrey L. Bennett
Purpose of review
Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) is a rare inflammatory disorder of the central nervous system that affects both adults and children. Neurologic disability is relapse-driven; therefore, early diagnosis and targeted treatment are critical for effective care. We review the new MOGAD diagnostic criteria and evidence for current acute and preventative therapies.
Recent findings
The International MOGAD Panel has released the first clinical, laboratory, and radiographic criteria for MOGAD diagnosis. These criteria set the stage for evaluating clinical investigations and designing future randomized clinical trials. Prior retrospective studies have evaluated multiple off-label agents for the acute care or prevention of MOGAD attacks, and prospective randomized clinical trials are now underway.
Summary
Acute MOGAD attacks are generally responsive to high-dose corticosteroids; however, early use of plasma exchange or intravenous immunoglobulin may be beneficial for severe attacks or cases lacking corticosteroid response. A slow corticosteroid taper may lower the risk of relapse. Preventative treatment has been typically limited to patients with a definitive relapsing disease. While there is no consensus on the choice or duration of treatment, multiple therapies have been retrospectively evaluated. Prospective placebo-controlled trials for interleukin-6 receptor inhibition and neonatal Fc receptor inhibition may open new frontiers for patient care.
{"title":"Emerging Principles for Treating Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD)","authors":"Andrew B. Wolf, Jacqueline Palace, Jeffrey L. Bennett","doi":"10.1007/s11940-023-00776-1","DOIUrl":"https://doi.org/10.1007/s11940-023-00776-1","url":null,"abstract":"<h3 data-test=\"abstract-sub-heading\">Purpose of review</h3><p>Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) is a rare inflammatory disorder of the central nervous system that affects both adults and children. Neurologic disability is relapse-driven; therefore, early diagnosis and targeted treatment are critical for effective care. We review the new MOGAD diagnostic criteria and evidence for current acute and preventative therapies.</p><h3 data-test=\"abstract-sub-heading\">Recent findings</h3><p>The International MOGAD Panel has released the first clinical, laboratory, and radiographic criteria for MOGAD diagnosis. These criteria set the stage for evaluating clinical investigations and designing future randomized clinical trials. Prior retrospective studies have evaluated multiple off-label agents for the acute care or prevention of MOGAD attacks, and prospective randomized clinical trials are now underway.</p><h3 data-test=\"abstract-sub-heading\">Summary</h3><p>Acute MOGAD attacks are generally responsive to high-dose corticosteroids; however, early use of plasma exchange or intravenous immunoglobulin may be beneficial for severe attacks or cases lacking corticosteroid response. A slow corticosteroid taper may lower the risk of relapse. Preventative treatment has been typically limited to patients with a definitive relapsing disease. While there is no consensus on the choice or duration of treatment, multiple therapies have been retrospectively evaluated. Prospective placebo-controlled trials for interleukin-6 receptor inhibition and neonatal Fc receptor inhibition may open new frontiers for patient care.</p>","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"7 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2023-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138516189","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-14DOI: 10.1007/s11940-023-00777-0
Nicole Frost, Gavin Yuan, Julia Zhang, Amy Rickard, Erin McGee, Michelle DiMattia, Stephan A. Mayer
{"title":"Correction to: Speech Language Pathology in the Neurocritical Care Unit","authors":"Nicole Frost, Gavin Yuan, Julia Zhang, Amy Rickard, Erin McGee, Michelle DiMattia, Stephan A. Mayer","doi":"10.1007/s11940-023-00777-0","DOIUrl":"https://doi.org/10.1007/s11940-023-00777-0","url":null,"abstract":"","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"28 19","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134991940","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-06DOI: 10.1007/s11940-023-00774-3
Scott Mintzer
{"title":"Epilepsy Treatment in Patients with Heart Disease","authors":"Scott Mintzer","doi":"10.1007/s11940-023-00774-3","DOIUrl":"https://doi.org/10.1007/s11940-023-00774-3","url":null,"abstract":"","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"29 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135585702","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}