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Short term real-world Fingolimod efficacy and safety in Emirati patients with multiple sclerosis.

阿联酋多发性硬化患者的短期真实世界芬戈莫德疗效和安全性。

  • 影响因子:1.63
  • DOI:10.1016/j.jocn.2019.11.010
  • 作者列表:"Ceccarelli A","Mifsud V","Abusamra E","Hussain SI
  • 发表时间:2020-01-01
Abstract

:In clinical trials, Fingolimod was an efficacious and safe treatment for multiple sclerosis (MS). Despite this, few studies have explored its real-world efficacy and safety in the Middle East. The aim of this study was to describe our clinical experience with Fingolimod at Cleveland Clinic Abu Dhabi in Emirati patients with MS. We retrospectively collected clinical and brain and spinal cord MRI activity over time in 30 Emirati MS patients [F/M = 22/8, mean (SD) disease duration at treatment initiation = 3.3 (3.6) years, age at onset = 25.9 (6.9) years] who were commenced on Fingolimod from 2015 and followed for 18 months. The proportion of MS patients clinically and radiologically silent after Fingolimod initiation over time was assessed together with annualized relapse rate (ARR) reduction and adverse events (AEs) occurrence. 70% of MS patients who started Fingolimod were naïve. The baseline ARR [mean (SD; range)] was [1.2 (0.8; 0-4)] and 23.3% of MS patients had Gadolinium enhancing lesions at baseline MRI. Overall, the ARR was reduced by 72% and, relapses and MRI activity were found in only 24% and 38% of MS patients respectively. Mild to moderate AEs were observed in 33% of MS patients. No severe AEs were recorded. In Emirati MS patients, Fingolimod is safe and efficacious as an early treatment choice.

摘要

: 在临床试验中,芬戈莫德是多发性硬化症 (MS) 的有效和安全的治疗方法。尽管如此,很少有研究探讨其在中东的真实世界的疗效和安全性。本研究的目的是描述我们在阿布扎比克利夫兰诊所使用芬戈莫德治疗阿联酋MS患者的临床经验。我们回顾性收集了30例阿联酋MS患者的临床和脑和脊髓MRI随时间的活动 [F/m   =   22/8,治疗开始时的平均 (SD) 病程   =   3.3 (3.6) 年,Onset   的年龄 =   25.9 (6.9) 岁],从2015年开始使用Fingolimod,随访18个月。随着时间的推移,评估了芬戈莫德开始后临床和放射学上沉默的MS患者的比例,以及年复发率 (ARR) 降低和不良事件 (ae) 发生。70% 开始使用芬戈莫德的MS患者是幼稚的。基线ARR [平均值 (SD; 范围)] 为 [1.2 (0.8; 0-4)],23.3% 的MS患者在基线MRI时有钆增强病变。总体而言,ARR降低了72%,并且分别仅在24% 和38% 的MS患者中发现复发和MRI活性。在33% 的MS患者中观察到轻度至中度ae。未记录到严重不良事件。在阿联酋MS患者中,芬戈莫德作为早期治疗选择是安全有效的。

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影响因子:1.55
发表时间:2020-01-01
DOI:10.1097/PHM.0000000000001264
作者列表:["Gutiérrez-Cruz C","Rojas Ruiz FJ","De La Cruz Marquez JC","Gutiérrez-Davila M"]

METHODS:PURPOSE:The aim of the study was to assess dual-task cost to spatio-temporal gait parameters in people with multiple sclerosis and a matched control group. METHOD:The multiple sclerosis group was composed of 17 participants with a diagnosis of multiple sclerosis and an Expanded Disability Status Scale score of less than 6. A total of 17 healthy participants were allocated to the control group by stratification. Controls were matched on the basis of age, sex, sociocultural habits, and body structure. Dual-task cost was determined by within-group repeated-measures analysis of variance. Participants were instructed to ambulate under normal conditions and perform a discrimination and decision-making task concurrently. Then, between-group analysis of variance was used to assess differences in mean dual-task cost between groups and determine dual-task cost differential. Testing was performed using three-dimensional photogrammetry and an electronic walkway. RESULTS:Based on dual-task cost differential, gait cycle time increase (-5.8%) and gait speed decrease (6.3%) because of multiple sclerosis-induced impairment. CONCLUSIONS:During single- and dual-task conditions, gait speed was lower in multiple sclerosis participants, because of a shorter step length and increased swing time. Increased gait time might be the result of compensatory mechanisms adopted to maintain stability while walking specially during the double-support phases.

关键词: 暂无
翻译标题与摘要 下载文献
影响因子:1.55
发表时间:2020-01-01
DOI:10.1097/PHM.0000000000001272
作者列表:["Cattaneo D","Gervasoni E","Pupillo E","Bianchi E","Aprile I","Imbimbo I","Russo R","Cruciani A","Jonsdottir J","Agostini M","Beghi E","NEUROFALL Group."]

METHODS:OBJECTIVE:The aims of the study were to compare mobility in multiple sclerosis, Parkinson disease, and stroke, and to quantify the relationship between mobility and participation restrictions. DESIGN:This is a multicenter cross-sectional study. Included were compliant subjects with Parkinson disease, multiple sclerosis, and stroke seen for rehabilitation, with no comorbidities interfering with mobility. Functional scales were applied to each subject to investigate gait speed (10-meter walking test), balance while maintaining body position (Berg Balance Scale), dynamic balance and mobility (Timed Up and Go and Dynamic Gait Index), and participation (Community Integration Questionnaire). RESULTS:Two hundred ninety-nine patients (111 multiple sclerosis, 94 Parkinson disease, and 94 stroke) were enrolled. Stroke had the slowest gait speed (mean gait speed = 0.9 m/sec) compared with Parkinson disease (1.1 m/sec), and multiple sclerosis (1.2 m/sec) (P < 0.001). Multiple sclerosis was more limited than Parkinson disease and stroke in dynamic balance both in the Timed Up and Go Test (multiple sclerosis = 16.7 secs, Parkinson disease = 11.4 secs, stroke = 14.0 secs; P < 0.001) and Dynamic Gait Index (multiple sclerosis = 11.6 points, Parkinson disease = 12.9 points, stroke = 13.6 points; P = 0.03); ability to maintain balance and body position (Berg Balance Scale) was more affected in stroke and Parkinson disease than multiple sclerosis (multiple sclerosis = 42.6 points, Parkinson disease = 39.4 points, stroke = 39.7 points; P = 0.03). Balance disorders were associated with participation restrictions but not gait speed. CONCLUSIONS:Neurological conditions have differing impacts on gait and balance, leading to different levels of participation restriction.

关键词: 暂无
翻译标题与摘要 下载文献
影响因子:2.61
发表时间:2020-01-01
DOI:10.1111/ane.13152
作者列表:["Ferraro D","Guicciardi C","De Biasi S","Pinti M","Bedin R","Camera V","Vitetta F","Nasi M","Meletti S","Sola P"]

METHODS:OBJECTIVES:Cerebrospinal fluid (CSF) and blood neurofilaments (NFLs) are markers of axonal damage and are being investigated, mostly in relapsing-remitting (RR) MS, as a marker of disease activity and of response to treatment, while there are less data in progressive MS patients. Primary aim was to measure NFL in plasma samples of untreated patients with primary (PP) and secondary (SP) progressive MS and to correlate them with disability, disease severity, and prior/subsequent disability progression. MATERIALS AND METHODS:Neurofilament concentrations were measured using SIMOA (Single Molecule Array, Simoa HD-1 Analyzer; Quanterix). RESULTS:Neurofilament concentrations were measured on plasma samples of 70 progressive (27 PP and 43 SP), 21 RRMS patients, and 10 HCs. Longitudinal plasma NFL (pNFL) concentrations (median interval between sampling: 25 months) were available for nine PP/SP patients. PNFL concentrations were significantly higher in PP/SP compared to RRMS patients. They correlated with EDSS and MS Severity Score values. There was no difference in pNFL levels between PP/SP patients with EDSS progression in the preceding year (14% of patients) or during a median follow-up of 27 months (41%). In the longitudinal sub-study, pNFL levels increased in all patients between sampling by a mean value of 23% while EDSS mostly remained stable (77% of cases). CONCLUSION:In PP/SP progressive MS patients, pNFL levels correlate with disability and increase over time, but are not associated with prior/subsequent disability progression, as measured by EDSS, which may not be a sufficiently sensitive tool in this context.

脱髓鞘疾病方向

急性发作或亚急性损害神经中枢的疾病,发病高峰为二到三周,若治疗延误受损神经继发缺血变性则发生多发性硬化,发病严重时可侵犯脊髓前角细胞和脑干神经核以及大脑运动皮质锥体细胞危及生命,多为基因免疫异常或病毒感染所致。

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