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重组ADAMTS13在先天性血栓性血栓性缺血紫外线中
Marie Scully1, Ana Antun1, Spero R Cataland1
1From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).
再组合ADAMTS13预防疗法有效地预防了先天性TTP患者的血栓性血栓性紫外线 (TTP) 事件,显示出比标准治疗更好的安全性和有效性.
科学领域:
- 血液学 血液学 血液学
- 遗传学 是一个遗传学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 先天性血栓性血栓塞性紫外线 (TTP) 源于严重的遗传ADAMTS13缺乏症.
- 再组合ADAMTS13与用于先天性TTP预防的标准血衍生疗法的比较疗效和安全性仍未确立.
研究的目的:
- 评估与先天性TTP预防的标准治疗相比,复合ADAMTS13的疗效和安全性.
- 评估接受任何一种治疗的患者的TTP事件率,疾病表现和不良事件.
主要方法:
- 一个第三期,开放标签,交叉试验,涉及48名患有先天性TTP的患者.
- 患者被随机分配到6个月的复合ADAMTS13或标准治疗预防,然后进行交叉治疗.
- 主要结局是急性TTP事件的发生.
主要成果:
- 在复合ADAMTS13预防期间没有发生急性TTP事件,而在标准治疗期间发生了一次事件.
- 再组合ADAMTS13实现了~100%的正常ADAMTS13活性,明显高于标准治疗 (19%).
- 与标准疗法相比,复合ADAMTS13的不良事件发生频率较低,严重程度也较低,并且由于AE而没有停止试验药物治疗.
结论:
- 重组ADAMTS13预防对先天性TTP是有效和安全的,达到正常的ADAMTS13活性水平.
- 与标准治疗相比,这种治疗显著减少了TTP事件和表现.
- 再组合ADAMTS13为治疗先天性TTP提供了一个有前途的治疗选择.

