在原发性进行性多发性硬化症中使用标签外治疗的理由：文献综述。
Rationale for off-label treatments use in primary progressive multiple sclerosis: A review of the literature.
机构信息
Hospital Center of Périgueux, 80, avenue Georges Pompidou, 24000 Périgueux, France.
Université Clermont Auvergne, CHU de Clermont-Ferrand, Inserm, Neuro-Dol, 63000 Clermont-Ferrand, France.
出版信息
Rev Neurol (Paris). 2022 Nov;178(9):932-938. doi: 10.1016/j.neurol.2022.02.461. Epub 2022 Jul 16.
BACKGROUND
Until recently, few therapeutic options, other than symptomatic treatment, were available for patients with primary progressive multiple sclerosis (PPMS). Ocrelizumab is the only approved treatment in this indication, and only since 2017. However, many patients in France are receiving off-label treatments for PPMS, mainly rituximab, mycophenolate mofetil, methotrexate, cyclophosphamide, and azathioprine.
OBJECTIVE
To evaluate published data concerning the efficacy of these five treatments frequently used as off-label disease-modifying therapies.
METHODS
We reviewed and summarized the studies published in Pubmed since the inception of the database.
RESULTS
Evidence from randomized controlled trials is lacking to support the use of these treatments as disease-modifying therapies in PPMS.
CONCLUSION
The literature lacks dedicated studies to support the off-label use of these disease-modifying therapies in PPMS. However, some limited data are available in the literature suggesting that the use of rituximab and cyclophosphamide could potentially be of some interest in specific subpopulations.
背景
直到最近，除了对症治疗外，原发性进行性多发性硬化症（PPMS）患者几乎没有其他治疗选择。奥瑞珠单抗是该适应症中唯一批准的治疗药物，但直到 2017 年才开始使用。然而，法国许多 PPMS 患者正在接受利妥昔单抗、霉酚酸酯、甲氨蝶呤、环磷酰胺和硫唑嘌呤等药物的超说明书治疗。
目的
评估这五种常用于超说明书治疗的药物在治疗 PPMS 中的疗效的已发表数据。
方法
我们检索了 Pubmed 自数据库建立以来发表的研究，并对其进行了综述和总结。
结果
缺乏随机对照试验证据支持这些药物作为 PPMS 的疾病修正治疗药物。
结论
文献中缺乏专门研究来支持这些疾病修正治疗药物在 PPMS 中的超说明书使用。然而，文献中提供了一些有限的数据，表明利妥昔单抗和环磷酰胺的使用可能对特定亚组人群具有一定的潜在益处。
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