恩加塞尔蒂布与安慰剂对遗传性出血长管切除症的出血
Hanny Al-Samkari1, Josefien Hessels2, Antoni Riera-Mestre3
1Division of Hematology-Oncology, Massachusetts General Hospital, Harvard Medical School, Boston.
The New England journal of medicine
|November 26, 2025
概括
一种AKT抑制剂Engasertib在一项针对遗传性出血性长长生病 (HHT) 的试验中显示出有前途,减少了表症的频率和持续时间,并具有可控的副作用,如皮疹.
科学领域:
- 药理学 药理学是指药理学的学科.
- 遗传学 是一个遗传学.
- 内部医学 内部医学
背景情况:
- 遗传性出血性长膜炎 (HHT) 是一种遗传性疾病,导致严重的表,贫血和降低生活质量.
- 目前,全球范围内没有获得HHT许可的治疗方法,这突出显示了医疗需求尚未得到满足.
研究的目的:
- 评估口服 engasertib,选择性AKT抑制剂的安全性和疗效,作为对HHT的潜在治疗方法.
- 评估 engasertib 对 HHT 患者表症频率和严重性的影响.
主要方法:
- 这是一项多中心,双盲,安慰剂控制的试验,涉及75名HHT患者.
- 患者被随机分配,每天口服一次,接受12周的恩萨塞蒂布 (30毫克或40毫克) 或安慰剂.
- 主要结局包括不良事件的发生频率和严重程度;次要结局侧重于表的发生频率和持续时间.
主要成果:
- 在一些患者中,Engasertib治疗与轻度至中度的皮疹和可逆性高血糖症有关.
- 严重不良事件的发生率在 engasertib 和安慰剂组中是相似的.
- 与安慰剂 (分别为18.0%和23.8%) 相比,接受恩萨塞蒂布的患者表现出表现频率 (26.5-27.8%) 和持续时间 (29.9-41.4%) 的较大减少.
结论:
- 恩加塞尔蒂布的安全性与安慰剂相似,皮疹是最常见的目标副作用,在大多数情况下都会消失.
- 在HHT患者中,Engasertib治疗导致表症的频率和持续时间显著下降.
- 这些发现支持 engasertib作为管理HHT症状的潜在治疗选择.
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