Abstract:
OBJECTIVES:To estimate patient-acceptable symptom state (PASS) cut points for myasthenia gravis (MG) health scales. METHODS:We conducted an electronic survey that included the Myasthenia Gravis Impairment Index (MGII), EuroQol 5-Dimension (EQ5D), and a simple PASS question. PASS-anchored thresholds were estimated for the MGII questionnaire through receiver operating characteristic curves. We used the MGII PASS cut point in a validation cohort of 257 patients to estimate PASS thresholds for other clinically relevant health scales such as the Quantitative Myasthenia Gravis Scale (QMGS), Myasthenia Gravis Activities of Daily Living (MG-ADL), Myasthenia Gravis Composite (MGC), and Myasthenia Quality of Life (MG-QoL15). RESULTS:One hundred twenty-four of ≈250 invited patients answered the electronic survey (49% response rate), and 80 considered their current symptom state acceptable (PASS-positive). They had lower MGII scores than PASS-negative patients (7.76 ± 9.37 vs 25.0 ± 13.7, p < 0.0001) and better EQ5D scores (0.86 ± 0.17 vs 0.69 ± 0.18, p < 0.0001). The MGII questionnaire threshold for PASS was ≤10 points. With the use of this threshold in an independent dataset of 257 patients, all patients in remission or minimal manifestation status were PASS-positive. In addition, some patients in Classes I, II, and IIIA also achieved PASS status. PASS thresholds for the QMGS, MG-ADL, MGC, and MG-QoL15 were ≤7, 2, 3, and 8 points, respectively. CONCLUSIONS:We have estimated thresholds for commonly used myasthenia health scales reflecting patient-acceptable states in patients with MG. These thresholds indicate a global state of well being, rather than a change in scores, or being better. Therefore, PASS thresholds can be used as secondary endpoints for myasthenia research.
journal_name
Neurologyjournal_title
Neurologyauthors
Mendoza M,Tran C,Bril V,Katzberg HD,Barnett Cdoi
10.1212/WNL.0000000000010574subject
Has Abstractpub_date
2020-09-22 00:00:00pages
e1617-e1628issue
12eissn
0028-3878issn
1526-632Xpii
WNL.0000000000010574journal_volume
95pub_type
杂志文章相关文献
NEUROLOGY文献大全abstract::We report a Japanese boy with muscular dystrophy whose clinical symptoms were intermediate between those usually considered typical of Duchenne and Becker muscular dystrophies. The patient had a large inframe deletion extending from exons 3 to 41 of the dystrophin gene, which would be expected to cause the production ...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/wnl.44.9.1648
更新日期:1994-09-01 00:00:00
abstract::Neurologists are prescribing intravenous immunoglobulin (IVIg) with increasing frequency to treat many neurologic conditions that have a proven or presumed autoimmune or inflammatory pathogenesis. Although IVIg is not FDA approved for any neurologic condition, physicians can safely prescribe it for several disorders w...
journal_title:Neurology
pub_type: 杂志文章,评审
doi:10.1212/wnl.59.12_suppl_6.s41
更新日期:2002-12-24 00:00:00
abstract:OBJECTIVE:To determine the clinical value of JC virus (JCV) detection in various anatomic compartments for the diagnosis of progressive multifocal leukoencephalopathy (PML). METHODS:CSF, peripheral blood mononuclear cells (PBMC), plasma, and urine samples were evaluated from HIV-infected and uninfected individuals. JC...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/wnl.52.2.253
更新日期:1999-01-15 00:00:00
abstract:OBJECTIVE:Chronic inflammatory demyelinating polyneuropathy (CIDP) is characterized by immune-mediated peripheral demyelination. Although corticosteroid, IV immunoglobulin (IVIg) and plasma exchange have been established as the most effective therapeutics, subpopulations of patients show little or no response to either...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/WNL.0b013e3181bd1139
更新日期:2009-10-27 00:00:00
abstract::Levodopa is the most effective drug treatment for Parkinson's disease (PD). However, its chronic use is associated with dyskinesia, motor fluctuations, and hallucinations. There are various approaches to the management of levodopa complications. Motor fluctuations can be treated with a long-acting levodopa preparation...
journal_title:Neurology
pub_type: 杂志文章,评审
doi:
更新日期:2000-01-01 00:00:00
abstract:OBJECTIVES:To study the effects of head injury on disease progression and on neuropathologic outcomes in amyotrophic lateral sclerosis (ALS). METHODS:Patients with ALS were surveyed to obtain head injury history, and medical records were reviewed. Linear regression was performed to determine if head injury was a predi...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/WNL.0000000000001522
更新日期:2015-04-28 00:00:00
abstract:OBJECTIVE:To determine whether a simple small vessel disease (SVD) score, which uses information available on rapid visual assessment of clinical MRI scans, predicts risk of cognitive decline and dementia, above that provided by simple clinical measures. METHODS:Three prospective longitudinal cohort studies (SCANS [St...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/WNL.0000000000009141
更新日期:2020-03-24 00:00:00
abstract:OBJECTIVE:To determine the cumulative incidence of epilepsy in a population-based cohort of patients with multiple sclerosis (MS) and to investigate the association between epilepsy and clinical features of MS. METHODS:All available patients in the Swedish MS register (n = 14,545) and 3 age- and sex-matched controls p...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/WNL.0000000000004740
更新日期:2017-12-12 00:00:00
abstract::Nodoparanodopathy is a recent concept in the field of peripheral neuropathy, corresponding to peripheral nerve disorders stemming from an autoimmune attack directed and limited to the nodal region. This concept was identified using modern techniques of electrophysiology, immunology, and pathology (including electron m...
journal_title:Neurology
pub_type: 历史文章,杂志文章
doi:10.1212/WNL.0000000000008399
更新日期:2019-10-29 00:00:00
abstract::No uniformly accepted hypothesis explains the genesis and rupture of intracranial aneurysms. We followed 5,184 men and women prospectively for 26 years; 36 cases of aneurysmal subarachnoid hemorrhage (SAH) accounted for 62% of all intracranial hemorrhages. Blood pressure before SAH was higher in these patients than in...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/wnl.34.7.847
更新日期:1984-07-01 00:00:00
abstract:OBJECTIVE:To investigate evidence of the potential role of early cortical vulnerability in the development of primary progressive aphasia (PPA). METHOD:A woman with a diagnosis of PPA and her 9 adult siblings, 7 with developmental language disabilities, underwent neuropsychological testing, structural MRI, and resting...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/WNL.0000000000009842
更新日期:2020-08-18 00:00:00
abstract::Serum ratios of monoclonal immunoglobulin (Ig) to total protein, monoclonal immunoglobulin to albumin, CSF/serum Ig-albumin index, and CSF to serum monoclonal immunoglobulin were obtained in 21 patients with multiple myeloma and 2 with Waldenström's macroglobulinemia. Twelve patients (3 with CNS complications) showed ...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/wnl.33.11.1411
更新日期:1983-11-01 00:00:00
abstract:OBJECTIVES:Functional neuroimaging has shown that the absence of externally observable signs of consciousness and cognition in severely brain-injured patients does not necessarily indicate the true absence of such abilities. However, relative to traumatic brain injury, nontraumatic injury is known to be associated with...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/WNL.0b013e318249f764
更新日期:2012-03-13 00:00:00
abstract::Zinc is essential for normal embryogenesis and may have particular importance for closure of the human neural tube. Compared to 258 controls, we found increased zinc content in umbilical cord serum in eight of nine newborn anencephalics (p less than 0.02) and three infants with spina bifida (p less than 0.001). Increa...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/wnl.34.4.443
更新日期:1984-04-01 00:00:00
abstract::The excitability of the corticospinal system was studied in 23 healthy subjects in sleep stages NREM2, NREM4, REM, and wakefulness using transcranial magnetic stimulation. Assessment of motor thresholds, stimulus-response curves, and latencies of motor evoked potentials shows activation of the fast-conducting corticos...
journal_title:Neurology
pub_type: 临床试验,杂志文章
doi:10.1212/01.wnl.0000038762.11894.da
更新日期:2002-12-24 00:00:00
abstract:OBJECTIVE:To assess functional changes in lymphocyte repertoire and subsequent clinical implications during delayed-release dimethyl fumarate (DMF) treatment in patients with multiple sclerosis. METHODS:Using peripheral blood from several clinical trials of DMF, immune cell subsets were quantified using flow cytometry...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/WNL.0000000000007262
更新日期:2019-04-09 00:00:00
abstract::The mechanism by which prednisone improves muscle strength and function in Duchenne muscular dystrophy (DMD) is unknown. We addressed the possibility that clinical improvement was related to prednisone-induced alterations in skeletal muscle dystrophin. We performed muscle biopsies on patients at the conclusion of a ra...
journal_title:Neurology
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1212/wnl.41.5.661
更新日期:1991-05-01 00:00:00
abstract:OBJECTIVE:To compare in the Vertebral Artery Ischaemia Stenting Trial (VIST) the risks and benefits of vertebral angioplasty and stenting with best medical treatment (BMT) alone for symptomatic vertebral artery stenosis. METHODS:VIST was a prospective, randomized, open-blinded endpoint clinical trial performed in 14 h...
journal_title:Neurology
pub_type: 杂志文章,多中心研究,随机对照试验
doi:10.1212/WNL.0000000000004385
更新日期:2017-09-19 00:00:00
abstract:BACKGROUND:Mutations in the early growth response 2 (EGR2) gene have recently been found in patients with congenital hypomyelinating neuropathy and Charcot-Marie-Tooth type 1 (CMT1) disease. OBJECTIVE:To determine the frequency of EGR2 mutations in patients with a diagnosis of CMT1, Dejerine-Sottas syndrome (DSS), or ...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/wnl.52.9.1827
更新日期:1999-06-10 00:00:00
abstract:OBJECTIVE:To compare neurofilament light (NfL) and tau as blood-based biomarkers for acute sports-related concussion (SRC) and determine whether their concentrations at different time points after the injury are associated with prolonged time to return to play (RTP). METHODS:A total of 288 professional hockey players ...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/WNL.0000000000005518
更新日期:2018-05-15 00:00:00
abstract:BACKGROUND:Subjective memory deficit (SMD) is one of few potential presenting symptoms for people with early cognitive impairment. However, associations with underlying brain changes are unclear. METHODS:In a community sample of 1,779 people without dementia, and with neuroimaging (MRI) data, associations were investi...
journal_title:Neurology
pub_type: 杂志文章,多中心研究
doi:10.1212/01.wnl.0000310982.99438.54
更新日期:2008-04-29 00:00:00
abstract::We developed a scheme using routinely available data to estimate the risk of human immunodeficiency virus (HIV) dementia in HIV-infected persons over time. We performed a longitudinal review of medical records from more than 100 medical facilities in 11 U.S. cities. A total of 19,462 HIV-infected persons without histo...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/wnl.50.2.392
更新日期:1998-02-01 00:00:00
abstract::IgM anti-GM1 antibodies are associated with motor neuropathy syndromes, including multifocal motor neuropathy (MMN). We compared the ability of serum IgM from patients with multifocal motor neuropathy to bind to GM1 ganglioside alone and to GM1 as a component of a lipid mixture that also contained galactocerebroside a...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/wnl.48.4.1104
更新日期:1997-04-01 00:00:00
abstract:OBJECTIVE:This study prospectively examined the relationships among late night salivary cortisol (NSC) levels and depressive symptoms, memory performance, and hippocampal volumes in patients with medically intractable temporal lobe epilepsy (TLE) and the potential mediating effects of cortisol in the relationships betw...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/WNL.0b013e31824e8efb
更新日期:2012-04-03 00:00:00
abstract:OBJECTIVE:To report the assessment of a patient exhibiting gustatory agnosia. METHODS:Preoperative and postoperative neuropsychological, neuroimaging, and chemosensory evaluations were performed in a 39-year-old woman undergoing surgical treatment for intractable epilepsy. RESULTS:Preoperative MRIs showed bilateral (...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/01.WNL.0000149515.77718.35
更新日期:2005-01-25 00:00:00
abstract:OBJECTIVE:To determine whether there is a relationship between aortic plaques and intracranial (IC) atherosclerosis. METHODS:We reviewed 922 patients with stroke who had both transesophageal echocardiography and cerebral angiography. The plaques of these patients were classified as either complex aortic plaques (CAP),...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/01.wnl.0000238511.72927.3c
更新日期:2006-10-10 00:00:00
abstract::To learn if limb-kinetic apraxia (LKA) is associated with Parkinson disease (PD), participants with PD (on medications) and control subjects performed finger tapping (FT), measuring movement speed, and performed coin rotation (CR), measuring precise coordinated but independent finger movements and speed. There were no...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/01.wnl.0000250331.35912.a5
更新日期:2007-01-09 00:00:00
abstract::We reviewed the clinical and laboratory features of 81 patients who had trigeminal sensory neuropathy (TSN) and a connective tissue disease (CTD). The neuropathy developed before the symptoms of CTD in 6/81 patients (7%), and in 38/81 patients (47%) TSN and CTD were diagnosed concurrently. The most frequently associat...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/wnl.40.6.891
更新日期:1990-06-01 00:00:00
abstract:OBJECTIVE:To characterize the prevalence and prognostic significance of major driver molecular alterations in adult midline diffuse gliomas (MLG). METHODS:Adults with histologically proven MLG diagnosed between 1996 and 2017 were identified from our tumor bank, systematically reviewed, and reclassified according to WH...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/WNL.0000000000005658
更新日期:2018-06-05 00:00:00
abstract::Human cytomegalovirus and herpes simplex virus DNA probes were labeled in vitro and hybridized to the DNA extracted from brains of patients with multiple sclerosis (MS) to detect the presence of genetic information related to these viruses. We were unable to detect any virus-related genetic information complementary t...
journal_title:Neurology
pub_type: 杂志文章
doi:10.1212/wnl.30.5.530
更新日期:1980-05-01 00:00:00