Recombinant factor IX. Thromb Haemost 1997 Jul;78(1):261-5
Date
07/01/1997Pubmed ID
9198163Scopus ID
2-s2.0-0030857349 (requires institutional sign-in at Scopus site) 188 CitationsAbstract
Despite the introduction of recombinant preparations of factor VIII and recombinant factor VII and VIIa, patients with other forms of hemophilia, especially hemophilia B, have remained at increased risk for blood borne viruses because of a lack of clinically utilizable preparations of recombinant factor IX. This report describes the state of current tests with a recently licensed preparation of recombinant factor IX, BeneFix, from Genetics Institute. Structurally, functionally, and therapeutically, recombinant factor IX is comparable to monoclonal plasma-derived factor IX. The only observed difference between recombinant and plasma factor IX is the recovery in pharmacokinetic studies where recombinant factor IX recovery was approximately 72% that of a plasma factor IX product. This difference is attributed to be due to minor differences in the post-translational modification of recombinant factor IX compared to plasma. These studies demonstrate that recombinant factor IX is effective in the treatment of hemophilia B and has the safety profile expected from a product prepared by recombinant technology.
Author List
White GC 2nd, Beebe A, Nielsen BAuthor
Gilbert C. White MD Professor in the Medicine department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
AnimalsDrug Evaluation, Preclinical
Factor IX
Hemophilia B
Humans
Protein Processing, Post-Translational
Randomized Controlled Trials as Topic
Recombinant Proteins