Abstract:
:Currently, there are no approved treatments for NMO. All therapeutic studies in NMO have been either small, retrospective case series or uncontrolled prospective studies. Such studies are susceptible to inherent biases. As a consequence, conclusions regarding efficacy and safety from these studies may be erroneous. The optimal method for assessing therapeutic efficacy is the prospective, controlled trial with random treatment assignment that has the potential to control for multiple sources of bias. There is a significant unmet need for well-designed clinical trials in NMO. Successfully conducted, well-controlled NMO trials that show proof of benefit will lead to regulatory approval and subsequent acceptance by payers resulting in broad therapeutic availability. The most direct method to prove efficacy is to compare an active treatment vs. no treatment or placebo control. However, because of the devastating nature of the disease some clinicians are reluctant to expose potential study patients to the risk of no treatment. The primary ethical concern in the case of placebo-control in NMO clinical trials rests on the relative merits of answering the scientific question regarding efficacy compared to the relative risk of exposure to harm in the placebo-control group. This article outlines the case for clinical equipoise in NMO by addressing the uncertainty regarding the relative scientific and clinical merits of current empirically used treatments and showing that a placebo arm is consistent with competent medical care. Because no currently available treatment has proven benefit, and because all therapies are known to potentially cause harm, placebo-control is not only ethical but is in some ways preferable to active comparator or add-on study designs. Without well-designed, placebo-controlled trials, NMO patients may not have access to new treatments and will never know whether the therapies that they may be currently taking have risk to benefit profiles that clearly favor their use.
journal_name
Mult Scler Relat Disordjournal_title
Multiple sclerosis and related disordersauthors
Cree BAdoi
10.1016/j.msard.2015.07.017subject
Has Abstractpub_date
2015-11-01 00:00:00pages
536-45issue
6eissn
2211-0348issn
2211-0356pii
S2211-0348(15)00116-9journal_volume
4pub_type
杂志文章,评审abstract:BACKGROUND:We characterize the variations in availability and affordability of NMO diagnostic testing and treatment by geographic region and country-level income group. METHODS:A structured survey was distributed in English, French, and Spanish in late 2018 to neurologists and other physicians who encounter NMO patien...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2019.05.013
更新日期:2019-08-01 00:00:00
abstract:BACKGROUND:We explored health-related quality of life (HRQoL) and psychosocial aspects in a cohort of patients with a history of longstanding benign MS (BMS). METHODS:Patients with BMS (EDSS≤3 after 20 years disease duration) were re-assessed 25-30 years post-MS symptom onset for: EDSS, HRQoL (MSQoL-54), depression (B...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2014.09.211
更新日期:2015-01-01 00:00:00
abstract:BACKGROUND:The prevalence of multiple sclerosis (MS) varies throughout the world, and available epidemiological data suggest a progressively increasing prevalence of MS in Spain. The objective of this study was to calculate MS prevalence in Health District III of the autonomous community of Murcia in Spain. METHODS:Th...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2016.06.003
更新日期:2016-09-01 00:00:00
abstract:BACKGROUND:Severe relapses that required treatment were important outcomes in the sentinel trials of disease-modifying therapy (DMT). Identifying such relapses in administrative data would allow comparative-effectiveness studies of DMTs to be conducted in real-world clinical settings. METHODS:All relapsing-remitting (...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2017.11.022
更新日期:2018-01-01 00:00:00
abstract:BACKGROUND:Photophobia has never been investigated in MS. METHODS:In this pilot study we used photosensitivity questionnaire assessment (PAQ) to evaluate tolerability to light in 73 MS patients and 62 healthy controls. RESULTS:We identified a lower PAQ score and a higher number of photophobic subjects in MS than in c...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2018.09.005
更新日期:2018-11-01 00:00:00
abstract:OBJECTIVE:Using spectral domain optical coherence tomography (SD-OCT), to compare the choroidal thickness in a healthy population (group 1), with newly diagnosed multiple sclerosis (MS) patients (group 2), with MS patients who underwent β-interferon monotherapy (group 3) and MS patients who underwent fingolimod therapy...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2017.03.005
更新日期:2017-05-01 00:00:00
abstract::Acquired demyelinating CNS syndromes include a broad spectrum of clinical phenotypes and different entities can overlap. Therefore, differential diagnosis is still challenging. A humoral immune reaction against myelin oligodendrocyte glycoprotein (MOG) is present in a subgroup of these patients, particularly in childr...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章,评审
doi:10.1016/j.msard.2018.02.023
更新日期:2018-05-01 00:00:00
abstract::Neurosarcoidosis is a rare disease with various clinical phenotypes. Thus, case reports and series broaden the understanding of this entity (Ibitoye et al., 2017). We present a case of isolated intramedullary spinal cord sarcoidosis, an exceedingly rare phenotype, which needs to be distinguished especially from neurom...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2017.09.010
更新日期:2017-11-01 00:00:00
abstract:BACKGROUND:Relapsing-Remitting MS (RRMS) Deep Grey Matter (DGM) 5 year changes were examined using MRI measures of volume, transverse relaxation rate (R2*) and quantitative magnetic susceptibility (QS). By applying Discriminative Analysis of Regional Evolution (DARE), R2* and QS changes from iron and non-iron sources w...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2019.05.028
更新日期:2019-08-01 00:00:00
abstract:BACKGROUND:There are limited data on the impact of coronavirus disease 2019 (COVID-19) caused by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) on people with multiple sclerosis (MS). OBJECTIVE:To better understand SARS-CoV-2 infection in ocrelizumab-treated people with MS. METHODS:Internal Roche/Ge...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2020.102725
更新日期:2020-12-30 00:00:00
abstract:BACKGROUND:Cognitive deficits, especially in attention, are common in persons with MS (PwMS) and are associated with clinically meaningful outcomes, such as work disability and lower quality of life (QOL). In this study, we aimed to determine whether Cogmed Working Memory Training (CWMT) improves attention/working memo...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2021.102770
更新日期:2021-01-17 00:00:00
abstract::Neuromyelitis optica spectrum disorder (NMOSD) is a common neuroinflammatory demyelinating disease associated with aquaporin-4 (AQP4) antibody in the central nervous system. Neurosyphilis is a neurological disease caused by Treponema pallidum infection. NMOSD commonly occurs concurrently with autoimmune diseases. Howe...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2019.06.028
更新日期:2019-09-01 00:00:00
abstract:BACKGROUND:Cerebrospinal fluid (CSF) levels of neurofilament light (NFL), a biomarker of axonal damage, and CXCL13, a chemokine involved in B-cell regulation, are both associated with disease activity in multiple sclerosis (MS). OBJECTIVE:To explore the potential of NFL and CXCL13 to detect residual disease activity i...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2020.102463
更新日期:2020-11-01 00:00:00
abstract:BACKGROUND:Daytime sleepiness is a common symptom of multiple sclerosis (MS) that may jeopardize safe driving. Our aim was to compare daytime sleepiness, recorded in real-time through eyelid tracking, in a simulated drive between individuals with MS (iwMS) and healthy controls. METHODS:Fifteen iwMS (age = median (Q1 -...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2020.102607
更新日期:2020-10-31 00:00:00
abstract:UNLABELLED:Both female and male persons with MS are at increased risk for various forms of physical, sexual, and disability-specific abuse. An ongoing study revealed a subset of respondents in which the caregiver acknowledged mistreatment of the person with MS, but that person either denied or minimized mistreatment M...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2012.09.006
更新日期:2013-04-01 00:00:00
abstract:BACKGROUND:People with multiple sclerosis (MS) experience symptoms in multiple domains. High-quality patient-reported outcomes (PROs) that assess multiple domains can aid healthcare providers in assessing these symptoms and may support remote disease monitoring. The "SymptoMScreen" PRO correlates with other PROs in MS;...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2019.01.031
更新日期:2019-04-01 00:00:00
abstract:OBJECTIVE:To investigate the predictive factors associated with good outcomes of plasma exchange in severe attacks through neuromyelitis optica spectrum disorder (NMOSD) and long extensive transverse myelitis (LETM). In addition, to review the literature of predictive factors associated with the good outcomes of plasma...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2017.02.015
更新日期:2017-04-01 00:00:00
abstract:BACKGROUND:Anti-CD20 monoclonal antibodies such as ocrelizumab, rituximab, and ofatumumab target B-cell lineage. Clinical trials have demonstrated their effect on reducing both magnetic resonance imaging (MRI) active lesion burden as well as clinical activity. Zytux™ (Rituximab, AryoGen Pharmed) used in the present stu...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2019.101419
更新日期:2019-11-01 00:00:00
abstract::Fingolimod, taken orally once per day, is approved for the treatment of relapsing-remitting multiple sclerosis (MS). It should be stopped at least two months before conception as it is not considered safe during pregnancy or when breast feeding. In vitro and animal studies have found a possibly increased risk of conge...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2018.02.015
更新日期:2018-04-01 00:00:00
abstract:BACKGROUND:Multiple sclerosis (MS) is an immune-mediated neurodegenerative disease in the central nerve system, in which both innate and adaptive immune cells are involved. BBR3378, an aza-anthrapyrazole prevents experimental autoimmune encephalomyelitis (EAE), an inflammatory condition similar to MS, by antagonizing T...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2020.102190
更新日期:2020-08-01 00:00:00
abstract:BACKGROUND:The acute, fulminant type of Multiple Sclerosis (MS), known as Marburg Disease, has been shown to have poor response to conventional acute treatments typically used for demyelinating diseases. METHODS:We report a 20 y/o postpartum female who was consulted to the Neurology service given findings of subacute ...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2019.03.014
更新日期:2019-06-01 00:00:00
abstract:BACKGROUND:The 2015 International Panel for neuromyelitis optica (NMO) spectrum disorders (NMOSD) diagnosis (IPND) criteria was recently proposed. However, because there are no studies evaluating application of the IPND criteria in Latin American populations, we aimed to assess whether these new criteria improve the di...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2018.01.001
更新日期:2018-02-01 00:00:00
abstract::Multiple sclerosis (MS) is the most common acquired demyelinating disorder of the central nervous system (CNS). Diagnosing MS can be very challenging owing to its variable clinical features and lack of specific tests. Magnetic resonance imaging (MRI) is a key measure in this process. Although white matter lesions on b...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章,评审
doi:10.1016/j.msard.2019.101452
更新日期:2020-01-01 00:00:00
abstract:BACKGROUND:Cognitive dysfunctions are very frequent in people living with multiple sclerosis (MS). Several studies have previously indicated grey matter (GM) atrophy as useful predictor of patients' cognitive impairment. However, considerable uncertainty exists about the possible impact of deep grey matter volumes on c...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2020.102351
更新日期:2020-10-01 00:00:00
abstract:BACKGROUND:Fingolimod (FTY720) has been approved as the first oral representative of the class of sphingosine-1-phosphate (S1P) receptor modulators for the treatment of relapsing-remitting multiple sclerosis (MS). Besides inducing vaso-relaxation, fingolimod can also influence electrical conduction in the myocardium an...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章,评审
doi:10.1016/j.msard.2016.04.002
更新日期:2016-07-01 00:00:00
abstract::Thrombotic microangiopathy (TMA) is a rare but increasingly recognized complication of interferon-beta therapy, which can be associated with serious sequelae. We report on a 53-year-old woman with a longstanding history of relapsing-remitting multiple sclerosis, who developed TMA after 15 years of high-dose treatment ...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2016.09.002
更新日期:2016-11-01 00:00:00
abstract:BACKGROUND:Myelin oligodendrocyte glycoprotein (MOG) is an important marker on the surface of oligodendrocytes and is associated with many demyelinating diseases. Recently, MOG-IgG-associated encephalomyelitis (MOG-EM) has been proposed as a disease entity with a preliminary diagnosis standard. Some patients with lung ...
journal_title:Multiple sclerosis and related disorders
pub_type:
doi:10.1016/j.msard.2020.102064
更新日期:2020-07-01 00:00:00
abstract:OBJECTIVES:To compare the liver safety profile of interferon β (IFN β) and teriflunomide in patients with multiple sclerosis. METHODS:A network meta-analysis was carried out following the Cochrane Collaboration methodology. All trials comparing all types of IFN β with teriflunomide, or disease-modifying drugs, or plac...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章,meta分析
doi:10.1016/j.msard.2018.09.014
更新日期:2018-11-01 00:00:00
abstract:BACKGROUND:Cognitive impairment is highly prevalent in multiple sclerosis (MS). Due to the lack of specialized neuropsychological resources in many MS clinics, a brief cognitive monitoring tool that can be administered by other MS clinic staff is needed. BICAMS (Brief International Cognitive As-sessment for Multiple Sc...
journal_title:Multiple sclerosis and related disorders
pub_type: 杂志文章
doi:10.1016/j.msard.2019.101458
更新日期:2020-01-01 00:00:00
abstract::Multiple sclerosis (MS) is a chronic disease characterized by inflammation, demyelination and neurodegeneration in the central nervous system. Recent studies suggested that patients with MS might have a greater risk of ischaemic stroke (IS). IS treatment with intravenous alteplase (IVA) in MS has rarely been reported....
journal_title:Multiple sclerosis and related disorders
pub_type:
doi:10.1016/j.msard.2020.101962
更新日期:2020-05-01 00:00:00