Safety and efficacy of viltolarsen treatment in patients with Duchenne muscular dystrophy: A retrospective study with 3-year follow-up

IF 1.3 4区医学 Q4 CLINICAL NEUROLOGY

Brain & Development Pub Date : 2024-11-15 DOI:10.1016/j.braindev.2024.10.005

Michinori Funato , Reina Iwata , Toru Ando , Motohiro Takeuchi , Yohei Horibe , Takashi Funato , Junko Oota , Ryoya Uno , Misaki Yumioka , Emi Kondo , Miho Katoyama , Chie Ito , Yoshiko Kato , Yuji Yamada , Toshifumi Kusukawa

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引用次数: 0

Abstract

Background

Duchenne muscular dystrophy (DMD) is a hereditary neuromuscular disorder characterized by severe, progressive muscle wasting. Viltolarsen, a formulation consisting of exon 53-skipping antisense oligonucleotides of the dystrophin gene, has been indicated for some patients with DMD. However, reports describing the efficacy and safety of viltolarsen treatment in patients with DMD, particularly those comparing patients receiving viltolarsen with age- and time-period-matched controls, are limited. This study aimed to confirm the safety and efficacy of viltolarsen treatment in patients with DMD.

Methods

We retrospectively reviewed real-world data of patients with DMD aged 6–19 years who started viltolarsen treatment between July 2020 and February 2021 and compared them with age- and time-period-matched patients with DMD who did not receive viltolarsen.

Results

Five patients received viltolarsen treatment for over 3 years. Three of the four patients who received viltolarsen once weekly maintained motor function, while one ambulatory patient showed remarkably slow motor function decline. Conversely, eight of nine patients who did not receive viltolarsen and one patient who underwent intermittent viltolarsen treatment showed motor function deterioration. Two patients showed significant treatment-related adverse events, namely swelling of the dorsal surface of the right hand due to extravasation of viltolarsen in one patient and axillary lymph node enlargement due to frequent intravenous infusion in another patient. None of the patients discontinued viltolarsen.

Conclusions

Viltolarsen was a safe and effective treatment for patients with DMD, and the findings highlighted the importance of once-weekly and uninterrupted viltolarsen treatment. The distinct safety and efficacy of viltolarsen require further investigation using a large number of cases and a long follow-up period.

查看原文本刊更多论文

维托拉生治疗杜氏肌营养不良症患者的安全性和有效性：一项为期三年的回顾性研究。

背景：杜氏肌营养不良症（DMD）是一种遗传性神经肌肉疾病，以严重的进行性肌肉萎缩为特征。Viltolarsen是一种由dystrophin基因第53外显子缺失反义寡核苷酸组成的制剂，已被用于治疗部分DMD患者。然而，有关DMD患者接受维尔托拉生治疗的疗效和安全性的报道，尤其是将接受维尔托拉生治疗的患者与年龄和时间匹配的对照组进行比较的报道十分有限。本研究旨在证实DMD患者接受维尔托拉生治疗的安全性和有效性：我们回顾性审查了2020年7月至2021年2月期间开始接受维尔托拉生治疗的6-19岁DMD患者的真实世界数据，并将其与未接受维尔托拉生治疗的年龄和时间段匹配的DMD患者进行了比较：结果：5名患者接受了3年以上的维尔托拉生治疗。在每周接受一次维尔托拉生治疗的四名患者中，有三名患者的运动功能得以维持，而一名卧床患者的运动功能下降速度明显较慢。相反，9名未接受维尔托拉生治疗的患者中有8名和1名接受间歇性维尔托拉生治疗的患者出现了运动功能衰退。两名患者出现了明显的治疗相关不良反应，即一名患者因维尔多拉生外渗导致右手背肿胀，另一名患者因频繁静脉输液导致腋窝淋巴结肿大。所有患者均未停用维尔多拉生：结论：维托拉生对DMD患者是一种安全有效的治疗方法，研究结果强调了每周一次不间断维托拉生治疗的重要性。维尔托拉生的独特安全性和有效性需要通过大量病例和长期随访进行进一步研究。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Brain & Development 医学-临床神经学

CiteScore

3.60

自引率

0.00%

发文量

153

审稿时长

50 days

期刊介绍： Brain and Development (ISSN 0387-7604) is the Official Journal of the Japanese Society of Child Neurology, and is aimed to promote clinical child neurology and developmental neuroscience. The journal is devoted to publishing Review Articles, Full Length Original Papers, Case Reports and Letters to the Editor in the field of Child Neurology and related sciences. Proceedings of meetings, and professional announcements will be published at the Editor''s discretion. Letters concerning articles published in Brain and Development and other relevant issues are also welcome.