Novel therapeutics for hemophilia and other bleeding disorders.

Blood
Michael U CallaghanSteven W Pipe

Abstract

Hemophilia and von Willebrand disease are the most common congenital bleeding disorders. Treatment of these disorders has focused on replacement of the missing coagulation factor to prevent or treat bleeding. New technologies and insights into hemostasis have driven the development of many promising new therapies for hemophilia and von Willebrand disease. Emerging bypass agents including zymogen-like factor IXa and Xa molecules are in development and a bispecific antibody, emicizumab, demonstrated efficacy in a phase 3 trial in people with hemophilia A and inhibitors. Tissue factor pathway inhibitor, the protein C/S system, and antithrombin are targets of novel compounds in development to alter the hemostatic balance and new approaches using modified factor VIII molecules are being tested for prevention and eradication of inhibitor antibodies in hemophilia A. The first recombinant von Willebrand factor (VWF) product has been approved and has unique VWF multimer content and does not contain factor VIII. These new approaches may offer better routes of administration, improved dosing regimens, and better efficacy for prevention and treatment of bleeding in congenital bleeding disorders.

References

Jul 28, 1978·Biochemical and Biophysical Research Communications·Y AshiharaY Inada
Sep 29, 1999·Journal of Thrombosis and Thrombolysis·B E Fischer
Jun 9, 2001·The New England Journal of Medicine·P M Mannucci, E G Tuddenham
Feb 16, 2006·Drug Discovery Today·Frank Y XiePatrick Y Lu
Aug 8, 2006·Journal of Thrombosis and Haemostasis : JTH·J E SadlerUNKNOWN Working Party on von Willebrand Disease Classification
Feb 23, 2008·Anesthesia and Analgesia·Roman SniecinskiKenichi A Tanaka
Mar 31, 2010·Thrombosis and Haemostasis·Daniel BolligerKenichi A Tanaka
Jul 17, 2010·Seminars in Thrombosis and Hemostasis·Peter L TurecekHans Peter Schwarz
Oct 25, 2011·Nature Biotechnology·Lacramioara IvanciuRodney M Camire
Feb 23, 2012·Proceedings of the National Academy of Sciences of the United States of America·Susan A MaroneyAlan E Mast
Jan 18, 2013·The New England Journal of Medicine·Samantha C GouwUNKNOWN PedNet and RODIN Study Group
Oct 16, 2013·Proceedings of the National Academy of Sciences of the United States of America·Jeremy P WoodAlan E Mast
Nov 28, 2013·The Journal of Biological Chemistry·Michael DockalFriedrich Scheiflinger
Nov 27, 2014·Nature·Cassandra Willyard
Jan 20, 2015·Scientific American·Cassandra Willyard
Jul 21, 2015·Journal of Thrombosis and Haemostasis : JTH·L A GeorgeR M Camire
May 22, 2016·Thrombosis Research·Julie A PetersonAlan E Mast
May 22, 2016·Thrombosis Research·Rodney M Camire
May 26, 2016·The New England Journal of Medicine·Midori ShimaKeiji Nogami
May 26, 2016·The New England Journal of Medicine·Flora PeyvandiFrits R Rosendaal
Jul 19, 2016·Expert Review of Hematology·Massimo Franchini, Pier Mannuccio Mannucci
Aug 17, 2016·Thrombosis and Haemostasis·Christopher E WalshSalvador Grancha
Oct 30, 2016·Blood·Stéphanie G I PolderdijkJames A Huntington
Jan 7, 2017·Blood·David A Lane
Mar 23, 2017·Molecular Therapy. Nucleic Acids·Yuanyu Huang
Jul 12, 2017·The New England Journal of Medicine·Johannes OldenburgMidori Shima

❮ Previous
Next ❯

Citations

Sep 27, 2018·Pediatric Blood & Cancer·Courtney D Thornburg
Nov 16, 2018·Haemophilia : the Official Journal of the World Federation of Hemophilia·E Carlos Rodriguez-Merchan, Leonard A Valentino
Dec 19, 2018·Blood·George Q PerrinDavid M Markusic
May 28, 2019·Haemophilia : the Official Journal of the World Federation of Hemophilia·Szymon JanczarWojciech Mlynarski
Aug 3, 2019·Haemophilia : the Official Journal of the World Federation of Hemophilia·Glaivy BatsuliRobert F Sidonio
Apr 30, 2019·Haemophilia : the Official Journal of the World Federation of Hemophilia·Manuel CarcaoUNKNOWN Future of Immunotolerance Treatment Group
Jun 7, 2019·Blood Advances·Craig D Seaman, Margaret V Ragni
Mar 3, 2020·Journal of Thrombosis and Haemostasis : JTH·Flora PeyvandiMichael Spannagl
May 23, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Danial M IgnasPaul Babyn
Dec 29, 2018·British Journal of Haematology·Susan Shapiro, Mike Makris
Jul 21, 2020·Research and Practice in Thrombosis and Haemostasis·Corinne KarchCourtney D Thornburg
May 20, 2020·Frontiers in Immunology·Benjamin J Samelson-Jones, Valder R Arruda
Mar 27, 2020·EMBO Molecular Medicine·Jamie M O'Sullivan, James S O'Donnell
Jul 21, 2020·British Journal of Haematology·Sarina Levy-Mendelovich
Apr 24, 2019·Research and Practice in Thrombosis and Haemostasis·Barbara A KonkleAlfonso Iorio
Jan 13, 2022·Seminars in Thrombosis and Hemostasis·Amir SamiiAkbar Dorgalaleh

❮ Previous
Next ❯

Related Concepts

Related Feeds

Antibody Engineering

Antibody engineering technologies are constantly advancing to improve the clinical effectiveness of monoclonal and bispecific antibodies. Discover the latest research on Antibody Engineering here.

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

Nihon Ketsueki Gakkai zasshi : journal of Japan Haematological Society
T Ryu
Seminars in Thrombosis and Hemostasis
J F Irwin
Progress in Clinical and Biological Research
Y Nemerson, M Zur
© 2022 Meta ULC. All rights reserved