Skip to content
Home
About Us
Resources
Profiles Metrics
Authors Directory
Institutions Directory
Top Authors
Top Institutions
Top Sponsors
AI Digest
Contact Us
Menu
Home
About Us
Resources
Profiles Metrics
Authors Directory
Institutions Directory
Top Authors
Top Institutions
Top Sponsors
AI Digest
Contact Us
Home
About Us
Resources
Profiles Metrics
Authors Directory
Institutions Directory
Top Authors
Top Institutions
Top Sponsors
AI Digest
Contact Us
Menu
Home
About Us
Resources
Profiles Metrics
Authors Directory
Institutions Directory
Top Authors
Top Institutions
Top Sponsors
AI Digest
Contact Us
Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
biochemistry, genetics and molecular biology
Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A
Blood, Volume 123, No. 3, Year 2014
Notification
URL copied to clipboard!
Description
This phase 3 pivotal study evaluated the safety, efficacy, and pharmacokinetics of a recombinant FVIII Fc fusion protein (rFVIIIFc) for prophylaxis, treatment of acute bleeding, and perioperative hemostatic control in 165 previously treated males aged ≥12 years with severe hemophilia A. The study had 3 treatment arms: arm 1, individualized prophylaxis (25-65 IU/kg every 3-5 days, n = 118); arm 2, weekly prophylaxis (65 IU/kg, n = 24); and arm 3, episodic treatment (10-50 IU/kg, n = 23). a subgroup compared recombinant FVIII (rFVIII) and rFVIIIFc pharmacokinetics. End points included annualized bleeding rate (ABR), inhibitor development, and adverse events. The terminal half-life of rFVIIIFc (19.0 hours) was extended 1.5-foldvsrFVIII (12.4 hours; P <.001). Median ABRs observed in arms 1, 2, and 3 were 1.6, 3.6, and 33.6, respectively. In arm 1, the median weekly dose was 77.9 IU/kg; approximately 30% of subjects achieved a 5-day dosing interval (last 3 months on study). Across arms, 87.3% of bleeding episodes resolved with 1 injection. Adverse events were consistent with those expected in this population; no subjects developed inhibitors. rFVIIIFc was well-tolerated, had a prolonged half-life compared with rFVIII, and resulted in low ABRs when dosed prophylactically 1 to 2 times per week. This trial was registered at www.clinicaltrials.gov as #NCT01181128. © 2014 by The American Society of Hematology.
Available Materials
https://efashare.b-cdn.net/share/pmc/articles/PMC3894491/bin/supp_123_3_317__index.html
https://efashare.b-cdn.net/share/pmc/articles/PMC3894491/bin/supp_blood-2013-10-529974_blood-2013-10-529974-1.pdf
Authors & Co-Authors
Mahlangu, Johnny Ndoni
South Africa, Johannesburg
University of the Witwatersrand Faculty of Health Sciences
Powell, Jerry S.
United States, Davis
University of California, Davis
Ragni, Margaret V.
United States, Pittsburgh
University of Pittsburgh
Chowdary, Pratima
United Kingdom, London
The Royal Free Hospital
Josephson, Neil C.
United States, Seattle
Puget Sound Blood Center
Pabinger, Ingrid
Austria, Vienna
Medizinische Universität Wien
Hanabusa, Hideji
Japan, Tokyo
Ogikubo Hospital
Gupta, Naresh K.
India, New Delhi
Maulana Azad Medical College
Kulkarni, Roshni
United States, Grand Rapids
Msu College of Human Medicine
Fogarty, Patrick F.
United States, Philadelphia
Hospital of the University of Pennsylvania
Perry, David James
United Kingdom, Cambridge
Addenbrooke's Hospital
Shapiro, Amy D.
United States, Indianapolis
Indiana Hemophilia and Thrombosis Center
Pasi, K. John
United Kingdom, London
Barts and the London School of Medicine and Dentistry
Apte, Shashikant Janardan
India, Mahara
Sahyadri Hospital
Nestorov, Ivan A.
United States, Cambridge
Biogen Inc.
Jiang, Haiyan
United States, Cambridge
Biogen Inc.
Li, Shuanglian
United States, Cambridge
Biogen Inc.
Neelakantan, Srividya
United States, Cambridge
Biogen Inc.
Cristiano, Lynda M.
United States, Cambridge
Biogen Inc.
Goyal, Jaya
United States, Cambridge
Biogen Inc.
Sommer, Jürg M.
United States, Cambridge
Biogen Inc.
Dumont, Jennifer A.
United States, Cambridge
Biogen Inc.
Dodd, Nigel
United States, Cambridge
Biogen Inc.
Nugent, Karen
United States, Cambridge
Biogen Inc.
Vigliani, Gloria
United States, Cambridge
Biogen Inc.
Luk, Alvin Y.
United States, Cambridge
Biogen Inc.
Brennan, Aoife
United States, Cambridge
Biogen Inc.
Pierce, Glenn F.
United States, Cambridge
Biogen Inc.
Statistics
Citations: 412
Authors: 28
Affiliations: 15
Identifiers
Doi:
10.1182/blood-2013-10-529974
ISSN:
00064971
Study Design
Cross Sectional Study