Mellen Center for Multiple Sclerosis Treatment and Research, Cleveland Clinic, 9500 Euclid Avenue U10, Cleveland, OH, 44195, USA.
NeuroDevelopmental Science Center, Akron Children's Hospital, One Perkins Square, Akron, OH, 44308, USA.
Neurotherapeutics. 2017 Oct;14(4):985-998. doi: 10.1007/s13311-017-0568-1.
Pediatric-onset multiple sclerosis (POMS) is rarer than adult-onset disease, and represents a different diagnostic and treatment challenge to clinicians. We review POMS clinical and radiographic presentations, and explore important differences between POMS and adult-onset MS natural histories and long-term outcomes. Despite having more active disease, current treatment guidelines for patients with POMS endorse the off-label use of lower-efficacy disease-modifying therapies (DMTs) as first line. We review the available MS DMTs, their evidence for use in POMS, and the contrasting treatment strategies of high-efficacy early treatment and escalation therapy. We introduce a new treatment approach, the "high-efficacy early treatment", or HEET strategy, based on using directly observed, high-efficacy intravenously infused DMTs as first-line therapies. Like other proposed POMS treatment strategies, HEET will need to be prospectively studied, and all treatment decisions should be determined by an experienced neurologist, the patient, and his/her parents.
儿科发病型多发性硬化症（POMS）比成人发病型疾病更为罕见，对临床医生来说是一个不同的诊断和治疗挑战。我们回顾了 POMS 的临床和影像学表现，并探讨了 POMS 与成人发病型多发性硬化症自然病史和长期预后之间的重要差异。尽管 POMS 患者的疾病更为活跃，但目前的治疗指南仍支持将低疗效的疾病修正治疗（DMT）作为一线治疗药物进行标签外使用。我们回顾了现有的多发性硬化症 DMT 药物，它们在 POMS 中的应用证据，以及高疗效早期治疗和升级治疗的对比治疗策略。我们提出了一种新的治疗方法，即“高疗效早期治疗”（HEET）策略，该策略基于使用直接观察、高疗效的静脉输注 DMT 作为一线治疗药物。与其他提出的 POMS 治疗策略一样，HEET 需要进行前瞻性研究，所有的治疗决策都应由经验丰富的神经科医生、患者及其父母共同决定。
