Abstract:
OBJECTIVE:To analyze the cumulative efficacy and safety of everolimus in treating subependymal giant cell astrocytomas (SEGA) associated with tuberous sclerosis complex (TSC) from an open-label phase II study (NCT00411619). Updated data became available from the conclusion of the extension phase and are presented in this ≥5-year analysis. METHODS:Patients aged ≥ 3 years with a definite diagnosis of TSC and increasing SEGA lesion size (≥2 magnetic resonance imaging scans) received everolimus starting at 3mg/m(2) /day (titrated to target blood trough levels of 5-15ng/ml). The primary efficacy endpoint was reduction from baseline in primary SEGA volume. RESULTS:As of the study completion date (January 28, 2014), 22 of 28 (78.6%) initially enrolled patients finished the study per protocol. Median (range) duration of exposure to everolimus was 67.8 (4.7-83.2) months; 12 (52.2%) and 14 (60.9%) of 23 patients experienced SEGA volume reductions of ≥50% and ≥30% relative to baseline, respectively, after 60 months of treatment. The proportion of patients experiencing daily seizures was reduced from 7 of 26 (26.9%) patients at baseline to 2 of 18 (11.1%) patients at month 60. Most commonly reported adverse events (AEs) were upper respiratory tract infection and stomatitis of mostly grade 1 or 2 severity. No patient discontinued treatment due to AEs. The frequency of emergence of most AEs decreased over the course of the study. INTERPRETATION:Everolimus continues to demonstrate a sustained effect on SEGA tumor reduction over ≥5 years of treatment. Everolimus remained well-tolerated, and no new safety concerns were noted.
journal_name
Ann Neuroljournal_title
Annals of neurologyauthors
Franz DN,Agricola K,Mays M,Tudor C,Care MM,Holland-Bouley K,Berkowitz N,Miao S,Peyrard S,Krueger DAdoi
10.1002/ana.24523subject
Has Abstractpub_date
2015-12-01 00:00:00pages
929-38issue
6eissn
0364-5134issn
1531-8249journal_volume
78pub_type
杂志文章abstract::We describe the cases of eight patients with chronic idiopathic anhidrosis. These patients were heat intolerant and became hot, flushed, dizzy, dyspneic, and weak but did not sweat when the ambient temperature was high or when they exercised. Four patients had preganglionic sudomotor lesions and in the remaining 4 the...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410180312
更新日期:1985-09-01 00:00:00
abstract:OBJECTIVE:Normal aging is often associated with a decline in learning and memory functions. This decline is manifested to a much greater extent in Alzheimer's disease. Recent studies have indicated statins, a class of cholesterol-lowering drugs, as a potential therapy for Alzheimer's disease. Our objective was to deter...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.21053
更新日期:2006-12-01 00:00:00
abstract::We studied 23 patients with clinically defined mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS), 25 oligosymptomatic or asymptomatic maternal relatives, and 50 mitochondrial disease control subjects for the presence of a previously reported heteroplasmic point mutation at nt 3,243 in ...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410310408
更新日期:1992-04-01 00:00:00
abstract::We describe a 33-year-old homosexual man with a steroid-responsive, remitting and relapsing leukoencephalopathy associated with recent human immunodeficiency virus type 1 (HIV-1) seroconversion. Biopsy of a parieto-occipital lesion revealed demyelination and astrogliosis with focal necrosis. Detailed investigations de...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410310107
更新日期:1992-01-01 00:00:00
abstract::Mutations in AbetaPP cause deposition of Abeta amyloid fibrils in brain parenchyma and cerebral vessels, resulting in Alzheimer's disease (AD) and/or cerebral amyloid angiopathy (CAA). We report a novel mutation (L705V) within the Abeta sequence of AbetaPP in a family with autosomal dominant, recurrent intracerebral h...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.20571
更新日期:2005-10-01 00:00:00
abstract::Cerebrospinal fluid (CSF) lymphocytes from three young patients undergoing acute exacerbations of multiple sclerosis were studied by flow cytometry. Using a new method that simultaneously measures cell-surface antigens and the cell-cycle phase, we determined that the CSF lymphocytes in these patients were activated. T...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410180617
更新日期:1985-12-01 00:00:00
abstract::Interpretation of biochemical measurements in the human brain after death is complicated by a variety of premortem, perimortem, and postmortem factors. The activity of glutamic acid decarboxylase (GAD) in particular has been found to vary considerably among human brains. In contrast to neurotransmitter-associated enzy...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410100410
更新日期:1981-10-01 00:00:00
abstract::Paired-helical filaments (PHFs) are an important diagnostic criterion of the inclusion-body myositis (IBM) muscle biopsy; but, until now, their presence could be identified only by electronmicroscopy. In this report, we describe an easy immunocytochemical procedure, utilizing commercially available antibody, that enab...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410390318
更新日期:1996-03-01 00:00:00
abstract::Seven patients with postfacial palsy contracture and mass contractions were investigated electrophysiologically. In 3 patients the early blink reflex showed an unusually high amplitude, which can be attributed to enhanced excitability of facial motor neurons. In 5 patients the early blink reflex had acquired a crossed...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410020114
更新日期:1977-07-01 00:00:00
abstract::Axonal transport was studied in sciatic motor neurons of rats with neuropathy induced by p-bromophenylacetylurea (BPAU) in dimethylsulfoxide solution. Control rats were treated with the vehicle alone. To label rapidly transported proteins, the rats received an injection of 35S-methionine into the ventral horn of the s...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410190506
更新日期:1986-05-01 00:00:00
abstract:OBJECTIVE:T2-weighted magnetic resonance imaging is a sensitive tool for monitoring progression of multiple sclerosis, but it does not provide information on the severity of the underlying tissue damage. Measurement of T1 hypointensities and magnetization transfer ratio (MTR) can potentially distinguish lesions with mo...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.21113
更新日期:2007-09-01 00:00:00
abstract::B cells have long played an enigmatic role in the scenario of multiple sclerosis pathogenesis. This review summarizes recent progress in our understanding of B-cell trafficking, survival, and differentiation in the central nervous system (CNS). We propose four possible routes of intrathecal immunoglobulin-producing ce...
journal_title:Annals of neurology
pub_type: 杂志文章,评审
doi:10.1002/ana.20890
更新日期:2006-06-01 00:00:00
abstract::Residual tumor cells remain beyond the margins of every glioblastoma (GBM) resection. Their resistance to postsurgical therapy is considered a major driving force of mortality, but their biology remains largely uncharacterized. In this study, residual tumor cells were derived via experimental biopsy of the resection m...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.22036
更新日期:2010-08-01 00:00:00
abstract:OBJECTIVE:Infantile spasms are seizures associated with a severe epileptic encephalopathy presenting in the first 2 years of life, and optimal treatment continues to be debated. This study evaluates early and sustained response to initial treatments and addresses both clinical remission and electrographic resolution of...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.24594
更新日期:2016-03-01 00:00:00
abstract::Patients with multiple sclerosis (MS) frequently have selective depletion of the CD45R+CD4+ T-cell subset during active phases of disease. To study the relationship between changes in this subset and the onset of objective clinical exacerbations of disease, a longitudinal study was undertaken. Two CD4+ T-cell subsets ...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410240204
更新日期:1988-08-01 00:00:00
abstract::Patients with senile dementia of the Alzheimer type frequently have difficulty performing visual tasks. These difficulties may be due, at least partially, to degenerative changes in both the primary visual pathway and the visual association areas. To determine whether retinal ganglion cell dysfunction contributes to v...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410260208
更新日期:1989-08-01 00:00:00
abstract::Results of case-control studies and of a prospective investigation in men suggest that consumption of coffee could protect against the risk of Parkinson's disease, but the active constituent is not clear. To address the hypothesis that caffeine is protective against Parkinson's disease, we examined the relationship of...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.1052
更新日期:2001-07-01 00:00:00
abstract:OBJECTIVE:To examine changes in the response properties of meningeal nociceptors that might lead to migraine pain and examine endogenous processes that could play a role in mediating them using a clinically relevant model of migraine triggering, namely infusion of the nitric oxide (NO) donor nitroglycerin (NTG). METHO...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.23873
更新日期:2013-06-01 00:00:00
abstract::Fuel utilization in two adult patients with the myopathic form of very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency and five healthy subjects was investigated with stable isotopes during exercise at 50% of VO2max. The findings indicate that residual VLCAD activity in the patients is sufficient to maintain norm...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.20168
更新日期:2004-08-01 00:00:00
abstract:OBJECTIVE:To determine whether brain activation changes in clinically and neurocognitively normal human immunodeficiency virus (HIV)-infected and in HIV-seronegative control (SN) participants over a 1-year period. METHODS:Functional magnetic resonance imaging (fMRI) was performed in 32 SN and 31 HIV patients (all with...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.21594
更新日期:2009-03-01 00:00:00
abstract::To study the distribution of myelin-associated glycoprotein (MAG) in human nervous tissue and in multiple sclerosis (MS) lesions, we used paraffin sections and our modification of the peroxidase-antiperoxidase technique. Sections of MS lesions also were treated with antiserum to basic protein (BP) and with histologica...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410070212
更新日期:1980-02-01 00:00:00
abstract::As more small peptidergic components of the central nervous system are isolated, their role in disease states is being investigated. Several of these neuropeptides, especially the opioidlike peptides, adrenocorticotropic hormone, and some hypothalamic releasing factors, have been found to alter neuronal excitability. ...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410160707
更新日期:1984-01-01 00:00:00
abstract::Authorship of scientific publications holds great importance for basic and clinical researchers. Academic appointments and promotions, grant funding, and salary support depend to some extent on published recognition through authorship. Peer-recognition and personal satisfaction are additional incentives for authorship...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.22232
更新日期:2010-12-01 00:00:00
abstract::A prospective study was made of changes in the electroencephalogram after the use of metrizamide for myelography and posterior fossa cisternography in 61 patients (62 studies). The EEG changed in 21 patients (34%); 3 of these had had previously abnormal records, and the abnormalities were accentuated on the second rec...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410060308
更新日期:1979-09-01 00:00:00
abstract::Striopallidodentate calcinosis (Fahr's disease) is characterized clinically by seizures, rigidity, and dementia and pathologically by mineral deposition in the basal ganglia, dentate nucleus, and cerebral cortex. Disorders of iron and calcium-phosphate metabolism are thought to play a role in its pathogenesis. We pres...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410260412
更新日期:1989-10-01 00:00:00
abstract::Demonstration of intrathecal IgG production is employed in the diagnosis of various neurological disorders. This pathological IgG fraction in cerebrospinal fluid (CSF) can be visualized directly as oligoclonal bands by electrophoresis or isoelectric focusing or can be calculated as "excess" or "synthesized" IgG accord...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410170105
更新日期:1985-01-01 00:00:00
abstract::This study assessed whether or not levodopa induces dyskinesias in normal (ie, unlesioned) squirrel monkeys. All six animals treated twice daily with levodopa (15 mg/kg with carbidopa by oral gavage) for two weeks developed choreoathetoid dyskinesias, whereas none of the vehicle-treated animals displayed any abnormal ...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.1099
更新日期:2001-08-01 00:00:00
abstract::A patient with an intracranial hemorrhage showed differential impairment among arithmetic types (impaired in multiplication but not in subtraction). A functional magnetic resonance imaging (fMRI) experiment using normal volunteers also revealed a differential activation between the two arithmetic types. The fMRI resul...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:
更新日期:2000-10-01 00:00:00
abstract::Nineteen patients with radiologically confirmed stroke, and varying degrees of hemiparesis, were studied using somatosensory evoked potentials and the recently developed technique of transcutaneous motor cortex stimulation. The functional deficit caused by stroke was assessed at the time of evoked potential testing an...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.410250111
更新日期:1989-01-01 00:00:00
abstract:OBJECTIVE:The Epi4K Consortium recently identified 4 de novo mutations in the γ-aminobutyric acid type A (GABAA ) receptor β3 subunit gene GABRB3 and 1 in the β1 subunit gene GABRB1 in children with one of the epileptic encephalopathies (EEs) Lennox-Gastaut syndrome (LGS) and infantile spasms (IS). Because the etiology...
journal_title:Annals of neurology
pub_type: 杂志文章
doi:10.1002/ana.24631
更新日期:2016-05-01 00:00:00