一个第三阶段,随机,控制试验的resmetirom在NASH与肝纤维化
Stephen A Harrison1, Pierre Bedossa1, Cynthia D Guy1
1From the University of Oxford, Oxford (S.A.H.), the Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle University, Newcastle upon Tyne (Q.M.A.), and the National Institute for Health Research, Biomedical Research Centre at University Hospitals Birmingham NHS Foundation Trust and the University of Birmingham, Birmingham (P.N.N.) - all in the United Kingdom; Pinnacle Clinical Research, San Antonio (S.A.H., M.R.), South Texas Research Institute, Edinburg (R.P.), and Houston Methodist Hospital, Houston Research Institute, Houston (M.N.) - all in Texas; Liverpat and University of Paris (P.B.), INSERM, Unité Mixte de Recherche Scientifique (UMRS) 1139, Centre de Recherche sur l'Inflammation (L.C.), and Sorbonne Université, ICAN Institute for Cardiometabolism and Nutrition, Assistance Publique-Hôpitaux de Paris (APHP), INSERM, UMRS 1138, Centre de Recherche des Cordeliers (V.R.), Paris, and Université Paris-Cité, Department of Hepatology, Beaujon Hospital, APHP, Clichy (L.C.) - all in France; Duke University Health System, Durham, NC (C.D.G.); the Metabolic Liver Research Program, I. Department of Medicine, University Medical Center of Johannes Gutenburg University Mainz, Mainz (J.M.S.), the Department of Internal Medicine II, Saarland University Medical Center, Homburg (J.M.S.), and Klinikum St. Georg Leipzig, Leipzig (I.S.) - all in Germany; MASLD Research Center, the Division of Gastroenterology and Hepatology, University of California, San Diego, La Jolla (R.L.); Madrigal Pharmaceuticals, West Conshohocken, PA (R.T., D.L.); University of Arizona for Medical Sciences (S.E.M.) and Arizona Liver Health (N.A.) - both in Tucson; Covenant Metabolic Specialists, Sarasota (G.W.N.), and Flourish Research, Boca Raton (S.J.B.) - both in Florida; the Transplant Institute, Department of Medicine, University of Chicago Pritzker School of Medicine, and the Transplant Institute, Center for Liver Diseases, University of Chicago Biological Sciences - both in Chicago (M.E.R., M.R.C.); the Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, MN (M.F.A.); the Department of Medicine, Inova Fairfax Medical Campus, Falls Church (Z.Y.), and Virginia Commonwealth University, Richmond (A.J.S.) - both in Virginia; the Department of Gastroenterology and Hepatology, Antwerp University Hospital, Edegem (S.F.), and Cliniques Universitaires Saint-Luc, Service d'Hépato-gastroentérologie, UCLouvain, Brussels (N.L.) - both in Belgium; the Liver Unit at the IRCCS "Casa Sollievo della Sofferenza" Hospital, San Giovanni Rotondo, Italy (A.M.); the Liver Unit, Vall d'Hebron University Hospital, Vall d'Hebron Institut de Recerca, Universitat Autònoma de Barcelona, Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas, Barcelona (J.M.P.); and the Division of Liver Diseases, Icahn School of Medicine at Mt. Sinai, New York (M.B.B.).
在成年人中,resmetirom显著改善了非酒精性脂肪肝炎 (NASH) 和肝纤维化. 这种口服治疗在3期试验中与安慰剂相比,显示出更高的疗效.
科学领域:
- 肝病学 肝病学是一种肝病学.
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
背景情况:
- 非酒精性脂肪肝炎 (NASH) 是一种进展性肝病,具有显著的未满足的医疗需求.
- 目前,没有批准的药理疗法存在于NASH.
- 选择性甲状腺激素受体βagonist的resmetirom正在NASH治疗中进行研究.
研究的目的:
- 为了评估resmetirom在活检确认的NASH和肝纤维化的成年人中的疗效和安全性.
- 评估resmetirom对NASH解决和纤维化改善的影响.
主要方法:
- 进行了3期,随机,双盲,安慰剂对照试验.
- 患有NASH和纤维化 (F1B-F3) 的成年人每天接受resmetirom (80 mg或100 mg) 或安慰剂.
- 主要终点包括NASH解决和52周纤维化改善.
主要成果:
- 与安慰剂相比,resmetirom (80 mg和100 mg) 显著增加了NASH解决率 (25.9%和29.9%对比9.7%,P<0.001).
- 使用resmetirom,纤维化改善显著增加 (24.2%和25.9%对比14.2%,P<0.001).
- 药物还导致LDL胆固醇的显著降低;腹和恶心更频繁.
结论:
- 在80mg和100mg两种剂量中,resmetirom在统计学上显著地比安慰剂优越.
- 该药物有效地实现了NASH解决和改善肝纤维化阶段.
- 对于患有NASH和肝纤维化患者来说,Resmetirom是一个有前途的治疗选择.
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