儿童多发性硬化症:通过高效疗法改善预后。

IF 6.9 2区 医学 Q1 CLINICAL NEUROLOGY
Lama Saleh Aljomah, E. Ann Yeh
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引用次数: 0

摘要

小儿发病多发性硬化症(POMS)是指发生在18岁以下个体的多发性硬化症。与成人发病的多发性硬化症(AOMS)相比,其特点是认知结果差,炎症过程更严重,临床复发更频繁,MRI病变更多。在这一人群中,及时识别多发性硬化症是至关重要的,因为早期干预治疗可能会改变疾病进展的轨迹。在本文中,我们将回顾小儿多发性硬化症的诊断标准,鉴别诊断,以及目前和新兴的治疗方法。虽然许多dmt被批准用于成人多发性硬化症,但很少被批准用于儿科。这些dmt中有许多是在标签外使用的,有真实的证据证明了它们的有效性和安全性。该综述评估了在儿科人群中使用这些疗法的现有证据,重点是支持其使用的现有临床试验和实际数据。此外,我们将简要介绍POMS正在进行的临床试验和新兴疗法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Pediatric multiple sclerosis: Improving outcome through high-efficacy therapies
Pediatric-onset multiple sclerosis (POMS) refers to multiple sclerosis occurring in individuals under 18 years of age. It is characterized by poor cognitive outcomes and a more inflammatory course, more frequent clinical relapses, and a greater number of MRI lesions compared to adult-onset MS (AOMS). Prompt recognition of multiple sclerosis in this population is essential, as early intervention with disease-modifying therapies may change the trajectory of disease progression. In this paper, we will review diagnostic criteria for pediatric multiple sclerosis, differential diagnosis, and current and emerging therapeutic approaches. While a number of DMTs are approved for adult MS, few are approved for pediatric use. Many of these DMTs are used off-label, with real-world evidence demonstrating their effectiveness and safety. The review evaluates existing evidence for the use of these therapies in pediatric populations, with an emphasis on both existing clinical trials and real-world data that supports their use. In addition, we will briefly highlight ongoing clinical trials and emerging therapies for POMS.
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来源期刊
Neurotherapeutics
Neurotherapeutics 医学-神经科学
CiteScore
11.00
自引率
3.50%
发文量
154
审稿时长
6-12 weeks
期刊介绍: Neurotherapeutics® is the journal of the American Society for Experimental Neurotherapeutics (ASENT). Each issue provides critical reviews of an important topic relating to the treatment of neurological disorders written by international authorities. The Journal also publishes original research articles in translational neuroscience including descriptions of cutting edge therapies that cross disciplinary lines and represent important contributions to neurotherapeutics for medical practitioners and other researchers in the field. Neurotherapeutics ® delivers a multidisciplinary perspective on the frontiers of translational neuroscience, provides perspectives on current research and practice, and covers social and ethical as well as scientific issues.
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