Abstract:
:For patients with relapsing-remitting multiple sclerosis (RRMS), there are currently six approved medications that have been shown to alter the natural course of the disease. The approved medications include three beta interferon formulations, glatiramer acetate, natalizumab and mitoxantrone. Treating aggressive forms of RRMS and progressive disease forms of MS still presents a great challenge to neurologists. Intense immunosuppression has long been thought to be the only feasible therapeutic option. In patients with progressive forms of MS, lymphoid tissues have been detected in the central nervous system (CNS) that may play a critical role in perpetuating local inflammation. Agents that are currently approved for patients with MS have no or very limited bioavailability in the brain and spinal cord. In contrast, cyclophosphamide (CYC), an alkylating agent, penetrates the blood-brain barrier and CNS parenchyma well. However, while CYC has been used in clinical trials and off-label in clinical practice in patients with MS for over three decades, data on its efficacy in very heterogeneous groups of study patients have been conflicting. New myeloablative treatment paradigms with CYC may provide a therapeutic option in patients that do not respond to other agents. In this article we review the scientific rationale that led to the initial clinical trials with CYC. We will also outline the safety, tolerability and efficacy of CYC and provide neurologists with guidelines for its use in patients with MS and other inflammatory disorders of the CNS, including neuromyelitis optica (NMO). Finally, an outlook into relatively novel treatment approaches is provided.
journal_name
Ther Adv Neurol Disordjournal_title
Therapeutic advances in neurological disordersauthors
Awad A,Stüve Odoi
10.1177/1756285609344375subject
Has Abstractpub_date
2009-11-01 00:00:00pages
50-61issue
6eissn
1756-2856issn
1756-2864journal_volume
2pub_type
杂志文章abstract::Neuroinflammation is one of the key components contributing to the devastating outcome of ischemic stroke. Starting with stroke onset, inflammatory processes contribute both to cell damage and tissue remodeling. The early release of alarmins triggers the upregulation of multiple proinflammatory cytokines, resulting in...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756286418818092
更新日期:2018-12-17 00:00:00
abstract::Autoimmune glial fibrillary acidic protein (GFAP) astrocytopathy has been considered a novel central nervous system autoimmune disease characterized by relapse and responsiveness to corticosteroid with a specific GFAP-Immunoglobulin G (IgG) being noted in cerebrospinal fluid. We report the case of a 21-year-old girl p...
journal_title:Therapeutic advances in neurological disorders
pub_type:
doi:10.1177/1756286420909973
更新日期:2020-06-03 00:00:00
abstract::Brain atrophy, white matter abnormalities, and ventricular enlargement have been described in different neuromuscular diseases (NMDs). We aimed to provide a comprehensive overview of the substantial advancement of brain imaging in neuromuscular diseases by consulting the main libraries (Pubmed, Scopus and Google Schol...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756286419845567
更新日期:2019-05-06 00:00:00
abstract::Spinal muscular atrophy (SMA) is a progressive, recessively inherited neuromuscular disease, characterized by the degeneration of lower motor neurons in the spinal cord and brainstem, which leads to weakness and muscle atrophy. SMA currently represents the most common genetic cause of infant death. SMA is caused by th...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756285618754501
更新日期:2018-02-05 00:00:00
abstract:Background:Lennox-Gastaut syndrome (LGS) is a typical intractable form of epilepsy that most often occurs between the second and sixth year of life. This study aimed to evaluate the clinical efficacy and safety of ketogenic diet therapies (DTs) for LGS with mitochondrial dysfunction. Methods:This was a retrospective s...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756286419897813
更新日期:2020-02-06 00:00:00
abstract::Blepharospasm is a focal (although usually bilateral) dystonia of the orbicularis oculi muscles, producing excessive eye closure. This produces significant disability through functional blindness. Botulinum neurotoxins (BoNT) have become the treatment of choice for blepharospasm; the impressive response rate and the t...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756285614557475
更新日期:2015-03-01 00:00:00
abstract::Essential tremor (ET) is the most common adult movement disorder. Traditionally considered as a benign disease, it can cause an important physical and psychosocial disability. Drug treatment for ET remains poor and often unsatisfactory. Current therapeutic strategies for ET are reviewed according to the level of disco...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756285609104791
更新日期:2009-07-01 00:00:00
abstract::Ehlers-Danlos syndrome (EDS) is a heterogeneous heritable connective tissue disorder with various neurological manifestations, including chronic pain. The neurological manifestations in EDS are often regarded as being caused by the associated musculoskeletal disorders or polyneuropathy. Here, we present two patients w...
journal_title:Therapeutic advances in neurological disorders
pub_type:
doi:10.1177/1756286418793766
更新日期:2018-08-18 00:00:00
abstract::The treatment of neuropathic pain is difficult. Oral pharmaceuticals have significant side effects, and treatment efficacy tends to be modest. The use of topical analgesics reduces the potential for systemic side effects and allows direct application of medications to the area of pain. The natural spicy substance, cap...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756285613501576
更新日期:2014-01-01 00:00:00
abstract:Background:The humanized anti-α4 integrin monoclonal antibody natalizumab has proven to be very effective in patients with highly active relapsing-remitting multiple sclerosis (MS), but harbors the risk of progressive multifocal leukoencephalopathy (PML). Recently, new therapeutic options have become available for pati...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756286419887596
更新日期:2019-12-02 00:00:00
abstract::Multiple sclerosis (MS) is a potentially disabling chronic autoimmune neurological disease that mainly affects young adults. Our understanding of the pathophysiology of MS has significantly advanced in the past quarter of a century. This has led to the development of many disease-modifying therapies (DMTs) that preven...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756285612450936
更新日期:2012-07-01 00:00:00
abstract::B cells play a central role in the pathogenesis in multiple sclerosis (MS), being involved in the activation of proinflammatory T cells, secretion of proinflammatory cytokines, and production of autoantibodies directed against myelin. Hence, the usage of B-cell-depleting monoclonal antibodies as therapy for autoimmune...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756285615601933
更新日期:2016-01-01 00:00:00
abstract::Over 320 years after Thomas Sydenham described the condition labelled Sydenham's chorea, it remains poorly understood. The disorder is an antineuronal antibody-mediated neuropsychiatric disorder caused by a poststreptococcal, autoimmune condition affecting control of movement, mood, behaviour and potentially the heart...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756285610382063
更新日期:2010-09-01 00:00:00
abstract:BACKGROUND:The aim of this study was to analyse registry data of seizure outcome and adverse events (AEs) for perampanel as add-on therapy in patients with focal epilepsy since its approval in 2012 for adjunctive treatment of focal epilepsy in patients ⩾12 years. METHOD:A retrospective 2-year chart review of all patie...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756285616661115
更新日期:2016-11-01 00:00:00
abstract::Stroke is the fifth leading cause of death and the most frequent cause of disability worldwide. Currently, stroke diagnosis is based on neuroimaging; therefore, the lack of a rapid tool to diagnose stroke is still a major concern. In addition, therapeutic approaches to combat ischemic stroke are still scarce, since th...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756286418789340
更新日期:2018-08-06 00:00:00
abstract::Since the approval in 2010 of fingolimod 0.5 mg (Gilenya; Novartis Pharma AG, Basel, Switzerland) in the USA as an oral therapy for relapsing forms of multiple sclerosis, long-term clinical experience with this therapy has been increasing. This review provides a summary of the cumulative dataset from clinical trials a...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756285616628766
更新日期:2016-03-01 00:00:00
abstract:Background:Although the pathogenesis of myasthenia gravis (MG) is well known, prognostic markers are not yet available. We assessed the utility of anti-acetylcholine receptor (AChR) antibody (AChR-ab) titer and concentration of C3, C4, and C5a as potential severity biomarkers in MG. Methods:Levels of C3, C4, C5a, and ...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756286420935697
更新日期:2020-08-10 00:00:00
abstract:Background:To compare the long-term clinical outcomes of different antihypertensive drugs in stable patients after acute hemorrhagic stroke (HS). Methods:From January 2001 to December 2013, patients with first-ever primary HS were identified in the National Health Insurance Research Database, Taiwan. Patients with tra...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756286418802688
更新日期:2018-09-28 00:00:00
abstract::Experimental models of Parkinson's disease (PD) are of great importance for improving the design of future clinical trials. Various neurotoxic models are available, including 6-hydroxydopamine (6-OHDA), 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine (MPTP), paraquat and rotenone. However, no model is considered perfect;...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756285611413004
更新日期:2011-11-01 00:00:00
abstract:Background:No evidence of disease activity (NEDA) is a composite measurement, incorporating clinical and magnetic resonance imaging (MRI) elements of disease activity to sensitively evaluate the therapeutic efficacy of treatments for relapsing-remitting multiple sclerosis (RRMS). Objective:To assess the NEDA status of...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756286418795085
更新日期:2018-08-28 00:00:00
abstract::The World Wide Web is increasingly used in therapeutic settings. In this regard, internet-based interventions have proven effective in ameliorating several health behaviors, amongst them physical activity behavior. Internet-delivered interventions have shown positive effects on physical activity and physical function ...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756285616640684
更新日期:2016-07-01 00:00:00
abstract::More than 30% of epilepsy patients remain refractory despite the advent of new antiepileptic drugs (AEDs) over two decades. Although a small percentage of these refractory patients may become seizure free when a new AED is added, combined administration of AEDs or the application of novel AEDs is the most common thera...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756285609355850
更新日期:2010-03-01 00:00:00
abstract::Prolonged-release (PR) fampridine is the only approved medication to improve walking in multiple sclerosis (MS), having been shown to produce a clinically meaningful improvement in walking ability in the subset of MS patients with Expanded Disability Status Scale 4-7. Recent responder subgroup analyses in the phase II...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756286418803248
更新日期:2018-10-05 00:00:00
abstract::The development of neutralizing antibodies (NAbs) is a major problem in multiple sclerosis (MS) patients treated with interferon-beta (IFN-ß). Whereas binding antibodies (BAbs) can be demonstrated in the vast majority of patients, only a smaller proportion of patients develop NAbs. The principle in NAb in vitro assays...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756285608095144
更新日期:2008-09-01 00:00:00
abstract::Brain imaging is increasingly used to support clinicians in diagnosing multiple sclerosis (MS) and monitoring its progression. However, the role of magnetic resonance imaging (MRI) in MS goes far beyond its clinical application. Indeed, advanced imaging techniques have helped to detect different components of MS patho...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756286419859722
更新日期:2019-06-27 00:00:00
abstract::Until the early 1990s, a limited number of antiepileptic drugs (AEDs) were available. Since then, a large variety of new AEDs have been developed and introduced, several of them offering new modes of action. One of these new AED families is described and reviewed in this article. Levetiracetam (LEV) and brivaracetam (...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756286419873518
更新日期:2019-09-09 00:00:00
abstract::Cluster headache (CH) is a short-lasting unilateral headache associated with ipsilateral craniofacial autonomic manifestations. A positron emission tomography (PET) study has shown that the posterior hypothalamus is activated during CH attacks, suggesting that hypothalamic hyperactivity plays a key role in CH pathophy...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756285610370722
更新日期:2010-05-01 00:00:00
abstract::The BEtaferon®/BEtaseron® in Newly Emerging MS For Initial Treatment (BENEFIT) trial assessed the efficacy of early versus delayed treatment with interferon beta-1b for patients with clinically isolated syndrome (CIS). Patients were randomly assigned to receive either interferon beta-1b 250 μg every other day (early t...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章,评审
doi:10.1177/1756285614549554
更新日期:2014-11-01 00:00:00
abstract:Introduction:Wernicke encephalopathy (WE) and Wernicke-Korsakoff syndrome (WKS) are well-known disorders caused by thiamine deficiency. In addition to the classical concept of these diseases, some literature data suggest a connection between mitochondrial dysfunction and WE/WKS. Psychotic disorders and WKS seem to run ...
journal_title:Therapeutic advances in neurological disorders
pub_type:
doi:10.1177/1756286420938972
更新日期:2020-07-30 00:00:00
abstract:Background:Gastrointestinal (GI) events are common adverse events (AEs) associated with delayed-release dimethyl fumarate (DMF), an approved treatment for relapsing-remitting multiple sclerosis (RRMS). The objective of the TOLERATE study was to evaluate GI tolerability and GI mitigation via symptomatic therapies in pat...
journal_title:Therapeutic advances in neurological disorders
pub_type: 杂志文章
doi:10.1177/1756286418768775
更新日期:2018-04-18 00:00:00