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富马酸二甲酯作为一种新型口服免疫调节疗法用于多发性硬化的复发形式:新兴数据的综述。

Diroximel Fumarate as a Novel Oral Immunomodulating Therapy for Relapsing Forms of Multiple Sclerosis: A Review on the Emerging Data.

机构信息

Department of Psychiatry, Psychotherapy and Psychosomatic Medicine, Christian Doppler Medical Center, Paracelsus Medical University, Salzburg, Austria.

Department of Neurology, Landesklinikum Mistelbach-Gänserndorf, Mistelbach, Austria.

出版信息

Drug Des Devel Ther. 2022 Nov 10;16:3915-3927. doi: 10.2147/DDDT.S236926. eCollection 2022.

Abstract

Multiple sclerosis (MS) is a chronic inflammatory, demyelinating and neurodegenerative disorder of the central nervous system. Disease-modifying drugs (DMDs) and subsequent adherence are crucial for preventing reversible episodes of neurological dysfunction and delayed onset of progressive accumulation of irreversible deficits. Yet, side effects may limit their usage in clinical practice. Gastrointestinal (GI) side effects are a significant limitation of the use of dimethyl fumarate (DMF), the most frequently prescribed oral DMD in MS worldwide. Diroximel fumarate (DRF) is a second-generation oral fumaric acid ester (FAE) that was developed as a formulation with better GI tolerability. The improved tolerability is assumed to be related to a lower synthesis of gut-irritating methanol. Other explanations for DRF's lower extent of GI irritation include a more modest off-target activity due to its chemical structure. The superior GI tolerability of DRF compared to DMF could be proven in clinical trials and lead to approval of DRF for the treatment of relapsing forms of MS/relapsing-remitting MS (United States Food and Drug Administration and European Medicines Agency, respectively). Here, we summarize the mode of action of oral FAE and compare the chemical and physiological characteristics of DMF and DRF. Moreover, we discuss the adverse effects of FAE and introduce the emerging preclinical and trial data leading to the approval of DRF in MS. This article additionally reviews our current understanding of coronavirus disease 2019 (COVID-19) and the efficacy of severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) vaccination in people treated with FAE.

摘要

多发性硬化症 (MS) 是一种中枢神经系统的慢性炎症性、脱髓鞘性和神经退行性疾病。疾病修正药物 (DMD) 和随后的坚持使用对于预防可逆性神经功能障碍发作和延迟进行性不可逆转缺陷的累积至关重要。然而,副作用可能会限制它们在临床实践中的应用。胃肠道 (GI) 副作用是广泛应用于多发性硬化症的二甲基富马酸酯 (DMF) 的一个显著限制。二羟甲氧基富马酸酯 (DRF) 是第二代口服富马酸酯 (FAE),作为一种具有更好胃肠道耐受性的配方而开发。人们认为,其更好的胃肠道耐受性与较低的刺激性甲醇合成有关。DRF 胃肠道刺激程度较低的其他解释包括其化学结构导致的非靶向活性较为温和。DRF 与 DMF 相比,其胃肠道耐受性更好,这一点已在临床试验中得到证实,并导致 DRF 被批准用于治疗复发型多发性硬化症/复发缓解型多发性硬化症(美国食品药品监督管理局和欧洲药品管理局,分别)。在这里,我们总结了口服 FAE 的作用模式,并比较了 DMF 和 DRF 的化学和生理特性。此外,我们讨论了 FAE 的不良反应,并介绍了导致 DRF 在多发性硬化症中获得批准的新兴临床前和试验数据。本文还回顾了我们对 2019 年冠状病毒病 (COVID-19) 的当前理解,以及在接受 FAE 治疗的人群中严重急性呼吸综合征冠状病毒 2 (SARS-CoV-2) 疫苗的有效性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5e24/9663167/4372af6a02e7/DDDT-16-3915-g0001.jpg

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