Ghezzi Angelo
Dipartimento di Scienze della Salute, Università Piemonte Orientale A. Avogadro, Via Solaroli 17, 28100, Novara, Italy.
Neurol Ther. 2024 Aug;13(4):949-963. doi: 10.1007/s40120-024-00633-6. Epub 2024 Jun 1.
Up to 10 years ago the most common approach to the treatment of pediatric MS (ped-MS) was to start with IFNB or GA (so-called first-line therapies or moderate-efficacy disease-modifying therapies [ME-DMTs]) and to switch to more aggressive treatments (or high-efficacy disease-modifying therapies [HE-DMTs]) in non-responder patients. The use of HE-DMTs as first choice was recommended in selected cases with an active, aggressive form of MS. Indications for the treatment of ped-MS were essentially derived from data of observational studies. Recently, results of three randomized clinical trials have been published as well as data from many observational studies evaluating the effect of new and more active DMTs, with clear evidence that HE-DMTs are more effective than ME-DMTs. Therefore, the paradigm of treatment for patients with MS onset before 18 years of age should be changed, offering treatment with HE-DMTs as first option, because of their superior effectiveness to prevent relapses and disease progression. HE-DMTs present an overall reassuring safety profile and obtain better adherence to treatment.
直到10年前,治疗儿童多发性硬化症(ped-MS)最常见的方法是先使用干扰素β(IFNB)或醋酸格拉替雷(GA)(所谓的一线疗法或中等疗效疾病修正疗法[ME-DMTs]),对于无反应的患者再改用更积极的治疗方法(或高效疾病修正疗法[HE-DMTs])。在某些患有活动性、侵袭性多发性硬化症的病例中,建议将HE-DMTs作为首选。儿童多发性硬化症的治疗指征基本上来自观察性研究的数据。最近,三项随机临床试验的结果已经发表,还有许多观察性研究的数据评估了新型且更有效的疾病修正疗法(DMTs)的效果,有明确证据表明HE-DMTs比ME-DMTs更有效。因此,18岁之前发病的多发性硬化症患者的治疗模式应该改变,将HE-DMTs作为首选治疗方法,因为它们在预防复发和疾病进展方面效果更优。HE-DMTs总体安全性令人放心,并且患者对治疗的依从性更好。