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从Nusinersen切换到Risdiplam:克罗地亚在有效性和安全性方面的现实世界经验

Andrej Belančić^1,2, Tea Strbad³, Marta Kučan Štiglić⁴

¹Department of Clinical Pharmacology, Clinical Hospital Centre Rijeka, 51000 Rijeka, Croatia.

Journal of personalized medicine

|March 28, 2024

概括

在现实研究中,从nusinersen切换到risdiplam治疗脊柱肌肉缩 (SMA) 显示了非劣质的有效性和良好安全性. 这一发现支持risdiplam作为SMA患者可行的治疗选择.

科学领域:

神经学神经学
遗传学遗传学是一个
药理学药理学是指药理学的学科.

背景情况:

关于使用疾病修饰药物 (DMDs) 顺序治疗脊柱肌缩 (SMA) 的数据有限.
之前关于从努辛森切换到RISDIPLAM的研究主要集中在安全方面.
需要在 nusinersen 治疗后对 risdiplam 的有效性和安全性的真实数据.

研究的目的:

调查risdiplam在儿童和成人SMA患者中从nusinersen转换后的现实世界的有效性和安全性.
在现实世界"切换"队列中,假设和评估与nusinersen相比risdiplam的非劣势性.

主要方法:

从克罗地亚的SMA患者收集回顾性,匿名的数据,这些患者从nusinersen转换为risdiplam.
纳入标准:对risdiplam的CHIF补偿,至少六次nusinersen剂量之前,治疗之间没有显著的暂停,和完整的数据.
在一年的随访期间,对17名符合条件的患者进行了有效性和安全性分析.

主要成果:

根据相关的临床评分,在SMA亚群 (SMA 1, 3p, 3a) 中,risdiplam与nusinersen的非劣势证明.
没有报告呼吸或食功能恶化,也没有报告致命事件.
在两名患者中,体重增加被确定为潜在的新药不良反应信号;没有其他新的安全问题出现.

结论:

这项研究提供了关键的现实世界的证据,支持将SMA患者从nusinersen切换到risdiplam的有效性 (非劣等性),安全性和耐受性.
这些发现提高了SMA患者的护理质量和安全标准,特别是那些试图转换DMD的患者.
支持risdiplam作为异质儿科和成人SMA群体的可行选择,从nusinersen过渡.

关键词:

有效性有效性.没有任何的 nusinersenersen.现实世界的数据.里斯迪普拉姆的风险脊柱肌肉缩脊柱肌肉缩脊柱肌肉缩

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