Role of enhanced half-life factor VIII and IX in the treatment of haemophilia

British Journal of Haematology
Ali J MahdiP W Collins

Abstract

Treatment of congenital haemophilia with factor VIII and IX concentrates often requires frequent infusions. This has obvious implications in establishing effective administration strategies and, in turn, adherence. To overcome these issues, three main technologies--polyethylene-glycol, Fc-neonatal IgG1 and albumin fusion products--have emerged into various stages of clinical development. Published data indicates an approximately 1·5- and fivefold increase in half-life of factor VIII and IX, respectively, compared to standard recombinant concentrates. Studies into efficacy and safety are starting to be published. Monitoring and optimal use of these new concentrates remains unknown. Weekly factor IX prophylaxis appears to be a feasible prophylactic regimen in haemophilia B patients. Weekly longer-acting FVIII is unlikely to provide adequate prophylaxis in most patients with haemophilia A but may reduce the frequency of infusions. Ongoing clinical trials and real life experience will help shape how these products can be used in practice and their cost effectiveness. The drive for convenience however should not overshadow the ultimate goal of prophylaxis, namely, preventing bleeding and arthropathy.

References

Jan 18, 2005·Trends in Cell Biology·Wayne I Lencer, Richard S Blumberg
Apr 12, 2007·Journal of Thrombosis and Haemostasis : JTH·P J LentingC V Denis
Aug 10, 2007·The New England Journal of Medicine·Marilyn J Manco-JohnsonBruce L Evatt
Nov 28, 2007·Journal of Thrombosis and Haemostasis : JTH·J S PowellE Gorina
Jan 16, 2009·Journal of Thrombosis and Haemostasis : JTH·P W CollinsUNKNOWN rAHF-PFM Study Group
Oct 7, 2009·Thrombosis and Haemostasis·Hubert J MetznerStefan Schulte
Jan 9, 2010·Blood·Robert T PetersAlan J Bitonti
Aug 25, 2010·Haemophilia : the Official Journal of the World Federation of Hemophilia·P W CollinsUNKNOWN International Prophylaxis Study Group Pharmacokinetics Expert Working Group
Nov 22, 2011·Blood·Charles R M HayUNKNOWN International Immune Tolerance Study
Jul 21, 2012·Haemophilia : the Official Journal of the World Federation of Hemophilia·H AgersøM Ezban
Dec 5, 2012·Journal of Thrombosis and Haemostasis : JTH·R T PetersG F Pierce
Jun 5, 2013·Haemophilia : the Official Journal of the World Federation of Hemophilia·S Björkman
Nov 15, 2013·Blood·Johnny MahlanguUNKNOWN A-LONG Investigators
Dec 7, 2013·The New England Journal of Medicine·Jerry S PowellUNKNOWN B-LONG Investigators
Dec 3, 2014·Haemophilia : the Official Journal of the World Federation of Hemophilia·L A ValentinoN Hakobyan

❮ Previous
Next ❯

Citations

Feb 13, 2016·Haemophilia : the Official Journal of the World Federation of Hemophilia·E BerntorpS Lethagen
Oct 13, 2015·British Journal of Haematology·Mike Laffan
Jan 26, 2016·Seminars in Hematology·Rajiv K Pruthi
Jun 9, 2016·European Journal of Haematology·Erik BerntorpGianna Franca Rivolta
Jun 18, 2016·Haemophilia : the Official Journal of the World Federation of Hemophilia·P CollinsA Thomas
Jun 30, 2016·Haemophilia : the Official Journal of the World Federation of Hemophilia·W Miesbach, W Kalnins
Aug 27, 2016·American Journal of Hematology·Jan Hartmann, Stacy E Croteau
Oct 21, 2016·Journal of Thrombosis and Haemostasis : JTH·F PeyvandiE Biguzzi
Mar 25, 2017·Journal of Clinical Medicine·Adele GiampaoloHamisa Jane Hassan
Mar 31, 2017·Thrombosis and Haemostasis·Alfonso IorioUNKNOWN Pharmacokinetic (PK) Expert Working Group of the International Prophylaxis Study Group (the IPSG)
Jan 20, 2017·European Journal of Haematology·Erik BerntorpStephanie Taylor
Jul 12, 2017·The New England Journal of Medicine·K John PasiMargaret V Ragni
Jan 10, 2018·Haemophilia : the Official Journal of the World Federation of Hemophilia·J A MasonS McRae
Aug 18, 2017·Haemophilia : the Official Journal of the World Federation of Hemophilia·R J LiesnerE J Neufeld
Oct 12, 2017·Blood·Valder R ArrudaBenjamin J Samelson-Jones
Nov 22, 2017·Drugs & Aging·Massimo Franchini, Pier Mannuccio Mannucci
Apr 1, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Beatrice NolanJohannes Oldenburg
Aug 17, 2019·Haemophilia : the Official Journal of the World Federation of Hemophilia·Flora PeyvandiMichael Makris
Jul 27, 2018·Research and Practice in Thrombosis and Haemostasis·Stacy E CroteauAlfonso Iorio
Jul 12, 2019·Research and Practice in Thrombosis and Haemostasis·Pratima ChowdaryElena Santagostino
Sep 22, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·Maria Elisa MancusoKarin Fijnvandraat

❮ Previous
Next ❯

Related Concepts

Related Feeds

Blood Clotting Disorders

Thrombophilia includes conditions with increased tendency for excessive blood clotting. Blood clotting occurs when the body has insufficient amounts of specialized proteins that make blood clot and stop bleeding. Here is the latest research on blood clotting disorders.

Related Papers

Haemophilia : the Official Journal of the World Federation of Hemophilia
C D Thornburg, S W Pipe
Haemophilia : the Official Journal of the World Federation of Hemophilia
Pier Mannuccio Mannucci
Expert Opinion on Emerging Drugs
Pier Mannuccio Mannucci, Massimo Franchini
Blood Transfusion = Trasfusione Del Sangue
M MorfiniGiovanni Di Minno
© 2022 Meta ULC. All rights reserved