口服塞伯特拉斯塔特用于按需治疗遗传性血管发作
Marc A Riedl1, Henriette Farkas1, Emel Aygören-Pürsün1
1From the Division of Allergy and Immunology, University of California, San Diego, La Jolla (M.A.R.); the Hungarian Angioedema Center of Reference and Excellence, Department of Internal Medicine and Haematology, Semmelweis University, Budapest, Hungary (H.F.); University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.), and the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universitätsmedizin Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology, Berlin (M.M.) - all in Germany; Naval Hospital of Athens, Athens (F.P.); Asthma and Allergy Associates, Colorado Springs (D.F.S.), and IMMUNOe Research Center, Centennial (I.M.) - both in Colorado; Clinical Center of Allergology, Clinic of Allergy and Asthma, University Hospital Alexandrovska, Medical University of Sofia, Sofia, Bulgaria (M.S.); the Department of Systems Medicine, University of Padua, Padua (M.C.), and Operative Unit of Medicine, Angioedema Center, IRCCS Policlinico San Donato, San Donato Milanese, and Dipartimento di Scienze Biomediche per la salute, University of Milan, Milan (A.Z.) - all in Italy; Tel Aviv Sourasky Medical Center (D.H.) and Sheba Medical Center (N.A.-L.), Tel Aviv, and Bnai Zion Medical Center, Haifa (A.K.) - all in Israel; Chiba University Hospital, Chiba, Japan (D.H.); the Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Leeds (S.S.), and KalVista Pharmaceuticals, Salisbury (P.K.A., J.H., M.I., M.D.S., C.M.Y.) - both in the United Kingdom; Jagiellonian University Medical College, Krakow (M.S.), and Medical University of Lodz, Lodz (M.K.) - both in Poland; Icahn School of Medicine at Mount Sinai, New York (P.J.B.); Allergy and Clinical Immunology Department, Centro Hospitalar Universitário de S. João, and Faculty of Medicine, University of Porto, Porto, Portugal (E.D.); Marycliff Clinical Research, Spokane, WA (R.G.); Hospital Universitario Bellvitge de L'Hospitalet de Llobregat, Allergology Department, Barcelona (R.L.); University Clinic of Dermatology, School of Medicine, University Saints Cyril and Methodus, Skopje, North Macedonia (V.G.P.); Washington University School of Medicine, St. Louis (H.J.W.); KalVista Pharmaceuticals, Cambridge, MA (P.K.A., J.H., M.I., M.D.S., C.M.Y.); AARA Research Center, Dallas (W.R.L.); the University of Cincinnati College of Medicine and Bernstein Clinical Research Center, Cincinnati (J.A.B.); and the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C.).
与安慰剂相比,口服赛伯特拉斯塔特可以更快地缓解遗传性血管发作的症状. 这种口服治疗改善了症状缓解,减少了发作严重程度,并更快地实现了完全的解决.
科学领域:
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
- 罕见疾病 罕见疾病
背景情况:
- 遗传性血管 (HAE) 发作的亲肠道治疗可能会导致治疗延迟.
- 需要口服按需治疗HAE以改善患者的治疗结果.
研究的目的:
- 评估口服赛伯特拉斯塔特在治疗遗传性血管发作的疗效和安全性.
- 为了比较使用sebetralstat与安慰剂缓解症状,减轻严重程度和完全解决的时间.
主要方法:
- 第三阶段,双盲,随机,三向交叉试验.
- 患有HAE的参与者在发作期间接受了口服赛伯特拉斯塔特 (300毫克或600毫克) 或安慰剂.
- 主要终点:症状缓解的时间;次要终点:严重程度降低和完全解决的时间.
主要成果:
- 与安慰剂相比,口服赛伯特拉斯塔特显著减少了症状缓解的时间 (300毫克: 1.61小时; 600毫克: 1.79小时与安慰剂相比: 6.72小时).
- 此外,Sebetralstat还加速了攻击严重程度的降低,并在24小时内完全解决了问题.
- 42.5% (300毫克) 和49.5% (600毫克) 的发作在24小时内通过sebetralstat解决,与安慰剂的27.4%相比.
- 塞贝特拉斯塔显示了与安慰剂相比的可比性安全性.
结论:
- 口服sebetralstat是一种有效的按需治疗HAE发作.
- 与安慰剂相比,Sebetralstat提供了更快的起作用和更好的分辨率.
- 口服sebetralstat可以克服与亲肠道HAE治疗相关的局限性.
相关概念视频
Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
Open Angle Glaucoma: Treatment
Drugs such as carbonic anhydrase inhibitors, α2- and...
Treatment for Pulmonary Arterial Hypertension: Prostacyclin Receptor Agonists
These agonists bind to the IPR receptor situated on the plasma membrane of the pulmonary artery smooth muscle cells. This binding triggers a cascade of reactions known as the GS-AC-cAMP-PKA pathway. This pathway results in the relaxation of smooth muscle...
Drug Delivery: Enteral Route
Adrenergic Agonists: Therapeutic Uses
Emergency and Intensive Care Unit (ICU) applications: Pressor agents increase blood pressure, heart rate, and contractility in shock and organ failure situations. Dopamine can induce vasodilation and stimulate adrenoceptors. Endogenous catecholamines are effective in treating cardiogenic shock. α2-agonists like clonidine can reverse anesthesia-induced hypertension.
Allergies and...
Antiasthma Drugs: Mast Cell Stabilizers and Anti-IgE Drugs
Mast cell stabilizers, such as cromolyn (also known as sodium cromoglycate) and nedocromil (Tilade), are effective drugs in asthma management. These stabilizers hinder histamine release by skillfully obstructing the activation of mast cells and other cellular entities. Notably, they navigate this task without...


