治疗小儿多发性硬化症的新旧策略：新治疗方法的个人观点。

IF 3.9 3区医学 Q1 CLINICAL NEUROLOGY

Neurology and Therapy Pub Date : 2024-08-01 Epub Date: 2024-06-01 DOI:10.1007/s40120-024-00633-6

Angelo Ghezzi

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引用次数: 0

摘要

直到 10 年前，治疗小儿多发性硬化症（ped-MS）最常见的方法还是从 IFNB 或 GA（所谓的一线疗法或中效疾病修饰疗法 [ME-DMTs]）开始，然后在无应答的患者中改用更具侵袭性的疗法（或高效疾病修饰疗法 [HE-DMTs]）。建议在选定的活动性、侵袭性多发性硬化症病例中首选 HE-DMT。小儿多发性硬化症的治疗指征主要来自观察性研究的数据。最近，三项随机临床试验的结果以及许多观察性研究的数据相继发表，这些数据评估了新型和更有效的 DMTs 的效果，有明确证据表明 HE-DMTs 比 ME-DMTs 更为有效。因此，18 岁前发病的多发性硬化症患者的治疗模式应该改变，将 HE-DMTs 作为首选治疗方案，因为 HE-DMTs 在预防复发和疾病进展方面具有更好的疗效。HE-DMTs 的总体安全性令人放心，而且能获得更好的治疗依从性。

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Old and New Strategies in the Treatment of Pediatric Multiple Sclerosis: A Personal View for a New Treatment Approach.

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.

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来源期刊

Neurology and Therapy CLINICAL NEUROLOGY-

CiteScore

5.40

自引率

8.10%

发文量

103

审稿时长

6 weeks

期刊介绍： Aims and Scope Neurology and Therapy aims to provide reliable and inclusive, rapid publication for all therapy related research for neurological indications, supporting the timely dissemination of research with a global reach, to help advance scientific discovery and support clinical practice. Neurology and Therapy is an international, open access, peer reviewed, rapid publication journal dedicated to the publication of high-quality clinical (all phases), observational, real-world and health outcomes research around the discovery, development, and use of neurological and psychiatric therapies, (also covering surgery and devices). Studies relating to diagnosis, pharmacoeconomics, public health, quality of life, and patient care, management, and education are also welcomed. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, case reports, trial designs, communications and letters. 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