多尼达尔森在遗传性血管的有效性和安全性
Marc A Riedl1, Raffi Tachdjian1, William R Lumry1
1From the Division of Allergy and Immunology, University of California at San Diego, La Jolla (M.A.R.), the Division of Allergy, Immunology, and Rheumatology, University of California at Los Angeles, Los Angeles (R.T.), Allergy and Asthma Clinical Research, Walnut Creek (J.S.J.), and Ionis Pharmaceuticals, Carlsbad (L.B., K.B.N., S.T., S.W., A.Y.) - all in California; Asthma and Allergy Research Associates, Dallas (W.R.L.); the Department of Medicine, Pediatrics, and Biomedical Sciences, Penn State Health Allergy, Asthma, and Immunology, Hershey, PA (T.C.); Vinmec International Hospital, Hanoi (T.C.); the Division of Allergy and Immunology, Department of Chest Diseases, Hacettepe University School of Medicine, Ankara (G.K.), the Division of Immunology and Allergic Diseases, Istanbul Faculty of Medicine, Istanbul University, Istanbul (A.G.), and the Division of Allergy and Clinical Immunology, Department of Internal Medicine, Ege University Faculty of Medicine, Ege University, Izmir (N.M.G.) - all in Turkey; the Department of Clinical and Environmental Allergology, Jagiellonian University Medical College, Krakow, Poland (M.S.); the Angioedema Center, Barzilai University Medical Center, Ashkelon, Israel (A.R.); the Department of Immunology, Southmead Hospital, North Bristol National Health (NHS) Trust, Bristol, United Kingdom (M.M.G.); Medical Research of Arizona, Scottsdale (M.E.M.); and the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, Amsterdam (D.M.C.).
在3期试验中,Donidalorsen显著减少了遗传性血管发作. 这种反感性寡核酸在患有这种罕见的胀疾病的患者中显示出长期预防的前景.
科学领域:
- 遗传学和基因组学 遗传学和基因组学
- 药理学 药理学是指药理学的学科.
- 临床试验中的临床试验.
背景情况:
- 遗传性血管 (HAE) 是一种罕见的遗传性疾病,导致不可预测的胀.
- 它是由kallikrein-kinin系统的失调引起的.
- 目前的治疗方法旨在稳定该系统,预防是关键策略.
研究的目的:
- 评估doniDolorsen作为HAE.AE预防治疗的疗效和安全性.
- 评估降低发作率和改善生活质量的情况.
主要方法:
- 一个第三阶段,双盲,随机试验,涉及90名HAE患者.
- 患者每4周或每8周接受多尼达尔森 (80毫克) 皮下注射,或安慰剂.
- 主要终点是25周内每4周发生HAE发作的时间正常化数量.
主要成果:
- 与安慰剂相比,Donidalorsen显著降低了HAE攻击率.
- 4周的donidalorsen小组显示攻击率减少了81% (P<0.001),而8周的小组显示攻击率减少了55% (P=0.004).
- 在4周的donidalorsen组中观察到生活质量的改善,并出现轻度至中度的不良事件.
结论:
- 多尼达洛森有效地降低了遗传性血管患者的发作率.
- 这些发现支持doniDalorsen作为HAE.AE的长期预防疗法的潜力.
- 多尼达洛森为管理HAE症状提供了一种新的治疗选择.
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