里斯迪普拉姆在患有脊髓肌肉缩的日本患者中的安全性:对营销后监测研究进行了12个月的中间分析
Kayoko Saito1, Toshio Saito2, Reiko Arakawa3
1Institute of Medical Genetics, Tokyo Women's Medical University, 8-1 Kawadacho, Shinjuku City, Tokyo, 162-8666, Japan. saito.kayoko@twmu.ac.jp.
Neurology and therapy
|August 9, 2025
概括
里斯迪普拉姆在日本脊柱肌肉缩 (SMA) 患者中显示出良好的安全性,大多数患者继续治疗. 胃肠道问题是最常见的副作用,但严重事件很少发生.
科学领域:
- 药物监督 药物监督 药物监督
- 神经学 神经学
- 遗传学 是一个遗传学.
背景情况:
- 里斯迪普拉姆是SMN2基因的口服剪接修饰剂,已被批准用于脊髓肌肉缩 (SMA).
- 在日本SMA患者中使用risdiplam的真实安全数据有限.
- 这项研究评估了日本risdiplam的安全性和使用模式.
研究的目的:
- 评估日本SMA患者risdiplam的安全性和使用模式.
- 在营销后监测环境中收集真实世界的安全数据.
主要方法:
- 对正在进行的营销后监测 (PMS) 研究进行了12个月的中间分析.
- 包括日本SMA患者接受RISDIPLAM治疗.
- 使用MedDRA/J分类收集和编码药物不良反应 (ADRs).
主要成果:
- 包括来自259所医院的538名SMA患者.
- 20.8%的患者报告了ADR;1.5%报告了严重的ADR.
- 最常见的副作用是胃肠道疾病 (16.0%),包括腹 (8.0%).
结论:
- 里斯迪普拉姆在日本SMA患者中耐受性良好,与全球试验结果一致.
- 胃肠道的副作用是最常见的,探索性分析表明了潜在的危险因素.
- 24个月后的最终分析将提供全面的安全性和疗效评估.
关键词:
在SMN2拼接修改器中使用SMN2拼接修改器临时安全分析 临时安全分析日本 日本 日本 日本 日本.营销后监督研究研究.里斯迪普拉姆的风险安全的安全的安全的安全的安全.脊柱肌肉缩 脊柱肌肉缩 脊柱肌肉缩更多相关视频
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