Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A-Reference-Cited by-同舟云学术

Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A

Published:2014-01-16 Issue:3 Volume:123 Page:317-325
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Mahlangu Johnny¹,Powell Jerry S.²,Ragni Margaret V.³,Chowdary Pratima⁴,Josephson Neil C.⁵,Pabinger Ingrid⁶,Hanabusa Hideji⁷,Gupta Naresh⁸,Kulkarni Roshni⁹,Fogarty Patrick¹⁰,Perry David¹¹,Shapiro Amy¹²,Pasi K. John¹³,Apte Shashikant¹⁴,Nestorov Ivan¹⁵,Jiang Haiyan¹⁵,Li Shuanglian¹⁵,Neelakantan Srividya¹⁵,Cristiano Lynda M.¹⁵,Goyal Jaya¹⁵,Sommer Jurg M.¹⁵,Dumont Jennifer A.¹⁵,Dodd Nigel¹⁵,Nugent Karen¹⁵,Vigliani Gloria¹⁵,Luk Alvin¹⁵,Brennan Aoife¹⁵,Pierce Glenn F.¹⁵

Affiliation:

1. Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa;

2. University of California at Davis, Davis, California;

3. University of Pittsburgh and the Hemophilia Center of Western Pennsylvania, Pittsburgh, PA;

4. Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free Hospital, London, United Kingdom;

5. Puget Sound Blood Center, Seattle, WA;

6. Universitätsklinik für Innere Medizin I, Medizinische Universität Wien, Wien, Austria;

7. Department of Hematology, Ogikubo Hospital, Suginami-ku, Tokyo, Japan;

8. Haemophilia Centre and Department of Medicine, Maulana Azad Medical College & Associated Lok Nayak Jai Prakash Narayan Hospital, New Delhi, India;

9. Department of Pediatrics and Human Development, Michigan State University, East Lansing, MI;

10. Penn Comprehensive Hemostasis and Thrombosis Program, Hospital of the University of Pennsylvania, Philadelphia, PA;

11. Cambridge Haemophilia & Thrombosis Centre, Addenbrooke’s Hospital, Cambridge, United Kingdom;

12. Indiana Hemophilia and Thrombosis Center, Department of Hematology, Indianapolis, IN;

13. Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, United Kingdom;

14. Sahyadri Hospital, Department of Hematology, Mahara, India; and

15. Biogen Idec Hemophilia, Cambridge, MA

Abstract

Key Points A novel recombinant factor VIII with prolonged half-life, rFVIIIFc, was developed to reduce prophylactic injection frequency. rFVIIIFc was well-tolerated in patients with severe hemophilia A, and resulted in low bleeding rates when dosed 1 to 2 times per week.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/123/3/317/1377512/317.pdf

Reference29 articles.

1. Fc-fusion proteins and FcRn: structural insights for longer-lasting and more effective therapeutics [published online ahead of print October 24, 2013].;Rath

2. National Hemophilia Foundation MASAC recommendation concerning prophylaxis (regular administration of clotting factor concentrate to prevent bleeding). Available at:http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=57&contentid=1007. Accessed July 18, 2013

3. Improvements in factor concentrates.;Lillicrap;Curr Opin Hematol,2010

4. Barriers to compliance with prophylaxis therapy in haemophilia.;Hacker;Haemophilia,2001

Cited by 404 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Long-term clinical outcomes of prophylaxis with an rFVIIIFc or rFIXFc in adults aged ≥50 years with hemophilia A or B;Blood Advances;2024-09-24

2. Pharmacokinetic model-based assessment of factor IX prophylaxis treatment regimens in severe hemophilia B;Scientific Reports;2024-09-04

3. International Society on Thrombosis and Haemostasis clinical practice guideline for treatment of congenital hemophilia A and B based on the Grading of Recommendations Assessment, Development, and Evaluation methodology;Journal of Thrombosis and Haemostasis;2024-09

4. Joint health and pain in the changing hemophilia treatment landscape;Expert Review of Hematology;2024-07-15

5. Real-world usage and effectiveness of recombinant factor VIII/factor IX Fc in hemophilia A/B: final data from the 24-month, prospective, noninterventional PREVENT study in Germany;Research and Practice in Thrombosis and Haemostasis;2024-07