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Improving Outcomes in Pediatric Multiple Sclerosis: Current and Emerging Treatments

机译：改善儿科多发性硬化症的结果：当前和新兴治疗

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Pediatric-onset multiple sclerosis (MS) comprises 2-5% of MS cases, and is known to be associated with high disease activity and the accumulation of disability at an earlier age than their adult-onset counterparts. Appropriate therapy leading to disease control has the potential to alter the known trajectory of adverse long-term physical, cognitive, and psychosocial outcomes in this population. Thus, optimizing treatment for children and adolescents with MS is of paramount importance. The last decade has seen a growing number of disease-modifying therapies approved for relapsing MS in adults, and available agents now include oral, injectable, and infusion therapies. Recently, the development of randomized controlled MS trials in youth has led to the first agent approved by the US FDA for the treatment of pediatric MSfingolimod. With this, we have entered a new era of knowledge and treatment in this population and ongoing pediatric trials are expected to further inform clinical management. With the emergence of highly effective therapies targeting the inflammatory component of the disease, there has been increased interest in identifying treatment strategies that instead target mechanisms such as remyelination/repair, neuroprotection, or rehabilitation. The potential role for such emerging therapies in the treatment of pediatric MS remains an important area of study. In this review, we discuss current evidence for MS therapies in children including the treatment of acute relapses, disease-modifying therapies, and symptomatic management. We will also discuss evidence for emerging therapies, including remyelinating and neuroprotective agents.
机译：儿科发病多发性硬化（MS）包含2-5％的MS病例，已知与高疾病活动相关，并且在早期的年龄比其成人发作对应物患者的累积。导致疾病控制的适当治疗有可能改变这种人口中有不良长期身体，认知和心理社会结果的已知轨迹。因此，对儿童和MS的青少年的优化优化是至关重要的。过去十年来看，批准用于复发成人MS的疾病修改疗法越来越多，并且现有的药剂现在包括口服，可注射和输液疗法。最近，青年随机控制MS试验的发展导致美国FDA批准的第一代理用于治疗儿科MSFINGOLIMOD。有了这一点，我们已进入新的知识和治疗中的新时代，并且预计正在进行的儿科试验将进一步通知临床管理。随着靶向疾病的炎症组分的高效疗法的出现，鉴定了识别治疗策略的兴趣增加，而是旨在靶向雷髓鞘/修复，神经保护或康复等机制。在治疗儿科MS治疗中这种新出现的疗法的潜在作用仍然是一个重要的研究领域。在本次审查中，我们讨论了儿童疗法的现有证据，包括治疗急性复发，疾病改性疗法和对症管理。我们还将讨论新出现的疗法的证据，包括髓鞘化和神经保护剂。

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  • 来源
    《Pediatric drugs》 |2019年第3期|共16页
  • 作者

    Wilbur Colin; Yeh E. Ann;

  • 作者单位

    Univ Alberta Women &

    Childrens Hlth Res Inst Fac Med &

    Dent Dept Pediat Edmonton AB Canada;

    Hosp Sick Children Dept Pediat Div Neurol 555 Univ Ave Toronto ON M5G 1X8 Canada;

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  • 原文格式 PDF
  • 正文语种 eng
  • 中图分类 药学;
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