在患有进展性家族性肝内胆固醇症的儿童中使用odevixibat的现实世界经验
Angelo Di Giorgio1, Marco Sciveres2,3, Maurizio Fuoti4
1Paediatric Hepatology, Gastroenterology and Transplantation, Hospital Papa Giovanni XXIII, Bergamo, Italy.
JHEP reports : innovation in hepatology
|March 27, 2025
概括
在现实环境中,奥德维克西巴特有效地降低了慢性家族性肝内胆固醇症 (PFIC) 的儿童的胆固醇性和血清胆酸. 这种治疗在各种PFIC亚型中是安全和有益的,包括更罕见的形式.
科学领域:
- 肝病学和儿科胃肠道学
- 对遗传性肝脏疾病的药理干预
- 临床试验 现实世界的证据
背景情况:
- 以前的试验表明,奥德维克西巴特在治疗儿科进展性家族性肝内胆固醇症 (PFIC) 中胆固醇性的疗效.
- 现实世界的数据对于验证注册试验的结果与严格的资格标准至关重要.
- 本研究介绍了在各种PFIC亚型中使用odevixibat的现实经验.
研究的目的:
- 为了评估odevixibat在各种PFIC亚型的儿童的现实世界队列中的有效性和安全性.
- 评估奥德维克西巴特对胆固醇性和血清胆汁酸 (sBA) 水平的影响.
- 探索患有经典和较罕见形式的PFIC的患者的治疗反应.
主要方法:
- 一项多中心前性研究,涉及24名患有PFIC的儿科患者,接受了奥德维克西巴特 (40或120μg/kg/天) 治疗.
- 通过医生评估症状的全球印象每月一次,持续6个月.
- 通过≥70%的减少或<70μmol/L定义的sBA响应者;通过症状改善的反应者.
主要成果:
- 6个月后,sBA水平显著下降 (中位数为317.1至45.6μmol/L;p <0.001),75%的sBA反应.
- 气明显改善 (平均变化为-1.7),有73%的气反应者.
- 减少的与减少的sBA相关 (p <0.05);需要30%的剂量升级. 没有报告任何严重的不良事件.
结论:
- 在现实生活环境中,奥德维克西巴特在降低sBA水平和改善皮方面表现出有效性和安全性,用于患有经典和较罕见的PFIC亚型的儿童.
- 这种药物对患有各种PFIC类型的患者有益,包括患有晚期肝病和并发症的患者.
- 在某些患者中,剂量升级可能是必要的,以优化治疗反应.
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