首页> 美国卫生研究院文献>Pediatric Health Medicine and Therapeutics >Review Of The Safety Efficacy And Tolerability Of Fingolimod In The Treatment Of Pediatric Patients With Relapsing-Remitting Forms Of Multiple Sclerosis (RRMS)
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Review Of The Safety Efficacy And Tolerability Of Fingolimod In The Treatment Of Pediatric Patients With Relapsing-Remitting Forms Of Multiple Sclerosis (RRMS)

机译:芬戈莫德治疗复发缓解型多发性硬化症(RRMS)小儿的安全性有效性和耐受性综述

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摘要

Pediatric-onset multiple sclerosis (POMS) is an immune-mediated, demyelinating, neurodegenerative disease that accounts for 3–5% of all multiple sclerosis (MS) cases. Although evidence suggests that it has similar risk factors and disease pathophysiology as adult-onset MS (AOMS), there are distinctive features in disease characteristics and patient demographics of POMS that require unique therapeutic development and treatment considerations. Gilenya (Novartis International AG, Basel, Switzerland) (fingolimod) is a sphingosine-1-phosphate (S1P) receptor modulator that prevents lymphocytic outflow from peripheral lymph nodes. It has demonstrated efficacy in AOMS. In POMS, there have been three observational studies and one pivotal clinical trial evaluating the efficacy, safety, and tolerability of fingolimod. Currently, fingolimod is the only Food and Drug Administration and European Medicines Agency approved disease-modifying therapy to treat POMS. This review will critically evaluate the available evidence of fingolimod in the treatment of POMS in detail, as well as discussing its treatment implications.
机译:小儿发作性多发性硬化症(POMS)是一种免疫介导的脱髓鞘性神经退行性疾病,占所有多发性硬化症(MS)病例的3-5%。尽管有证据表明它具有与成人型MS(AOMS)类似的危险因素和疾病病理生理,但在POMS的疾病特征和患者人口统计方面有独特的特征,需要独特的治疗开发和治疗考虑。 Gilenya(Novartis International AG,瑞士巴塞尔)(芬戈莫德)是一种鞘氨醇-1-磷酸(S1P)受体调节剂,可防止淋巴细胞从外周淋巴结流出。在AOMS中已证明其功效。在POMS中,已经进行了三项观察性研究和一项关键临床试验,以评估芬戈莫德的疗效,安全性和耐受性。目前,芬戈莫德是唯一获得美国食品药品监督管理局和欧洲药品管理局批准的疾病改良疗法来治疗POMS。这篇综述将详细评估芬戈莫德在POMS治疗中的可用证据,并讨论其治疗意义。

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