在意大利接受RISDIPLAM治疗的患者:解释现实数据的挑战
Emilio Albamonte1, Adrea Lizio2, Giorgia Coratti3,4
1The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy. emilio.albamonte@centrocliniconemo.it.
概括
这项研究分析了在RISDIPLAM治疗脊髓肌缩症 (SMA) 的意大利患者,在现实环境中发现了良好的粘附性和安全性,即使在更严重的残疾人中也是如此.
科学领域:
- 神经学 神经学
- 罕见疾病 罕见疾病
- 药理学 药理学是指药理学的学科.
背景情况:
- 脊髓肌肉缩 (SMA) 是一种罕见的遗传疾病.
- 里斯迪普拉姆是一种治疗SMA的药物.
- 关于risdiplam使用的现实数据对于了解其在不同患者群体中的有效性和安全性至关重要.
研究的目的:
- 为提供与risdiplam治疗的意大利SMA患者的现实世界快照.
- 为了比较商业药物批准之前和之后的患者队列,考虑准入标准.
- 描述治疗的坚持和从nusinersen切换到risdiplam的原因.
主要方法:
- 对接受risdiplam的患者的回顾性图表审查.
- 两个队列的比较:同情使用 (队列1) 和商业批准 (队列2).
- 记录副作用,守和患者人口统计数据,包括那些从 nusinersen 转换的人.
主要成果:
- 包括283名意大利SMA患者 (平均年龄22岁).
- 第1队列的呼吸道/气囊管并发症 (58%的非坐着者) 比第2队列 (52%的坐着者) 更严重.
- 队列1和队列2中的35%和46%的患者从努辛森切换到RISDIPLAM,在两者中都观察到良好的坚持和安全性.
结论:
- 这是使用risdiplam的SMA患者中最大的现实世界队列.
- 患者的特征 (残疾,年龄) 与临床试验有所不同,这可能解释了现实世界结果的变化.
- 在严重残疾和老年患者中证实了risdiplam的安全性.
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