Recombinant ADAMTS-13: first-in-human pharmacokinetics and safety in congenital thrombotic thrombocytopenic purpura-Reference-Cited by-同舟云学术

Recombinant ADAMTS-13: first-in-human pharmacokinetics and safety in congenital thrombotic thrombocytopenic purpura

Published:2017-11-09 Issue:19 Volume:130 Page:2055-2063
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Scully Marie¹,Knöbl Paul²,Kentouche Karim³,Rice Lawrence⁴,Windyga Jerzy⁵,Schneppenheim Reinhard⁶,Hovinga Johanna A. Kremer⁷,Kajiwara Michiko⁸,Fujimura Yoshihiro⁹,Maggiore Caterina¹⁰,Doralt Jennifer¹¹,Hibbard Christopher¹²,Martell Leah¹²,Ewenstein Bruce¹²

Affiliation:

1. Department of Haematology and Cardiometabolic BRC, University College London Hospitals/University College London, London, United Kingdom;

2. Division of Hematology and Hemostasis, Department of Medicine 1, Medical University of Vienna, Austria;

3. Jena University Hospital, Jena, Germany;

4. Department of Medicine, Houston Methodist Hospital, Weill Cornell Medical College, Houston, TX;

5. Department Disorders of Hemostasis and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland;

6. Department of Pediatric Hematology and Oncology, Universitaetsklinikum Hamburg-Eppendorf, Hamburg, Germany;

7. Inselspital, University Hospital Bern, Bern, Switzerland;

8. Tokyo Medical and Dental University Hospital Faculty of Medicine, Tokyo, Japan;

9. Nara Medical University, Nara, Japan;

10. Quintiles, Milan, Italy;

11. Shire, Vienna, Austria; and

12. Shire, Cambridge, MA

Abstract

Key PointsFirst-in-human, phase 1 study, recombinant ADAMTS-13 was safe, nonimmunogenic, and tolerated in congenital thrombotic thrombocytopenic purpura. Recombinant ADAMTS-13 pharmacokinetic profile was comparable to plasma infusion studies, with evidence of pharmacodynamic activity.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/130/19/2055/1403097/blood788026.pdf

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