Factor VIII/factor IX prophylaxis for severe hemophilia

Seminars in Hematology
Manuel D Carcao, Alok Srivastava

Abstract

Experience with clotting factor concentrate (CFC) replacement products over several decades has shown that regular replacement (prophylaxis) is the only way to prevent musculoskeletal damage in hemophilia and impact the natural history of hemophilia. Yet there is a lack of data on the optimal age to start such replacement therapy and the regimens to be used. While very early administration of high doses is certainly more effective in preventing bleeding, cost and compliance are major constraints all over the world. Starting prophylaxis with even lower doses comparable to that used in episodic therapies leads to major reduction in bleeding. Recognition of the clinical heterogeneity of hemophilia even among patients with a label of severe hemophilia in terms of their spontaneous bleeding has led to efforts aimed at individualizing CFC replacement, based on clinical responses or pharmacokinetic data of the CFC. The importance of long-term outcome assessment being combined with CFC replacement therapy cannot be overemphasized.

References

Jul 1, 1992·Journal of Internal Medicine·I M NilssonH Pettersson
Jan 5, 1999·Haemophilia : the Official Journal of the World Federation of Hemophilia·W KreuzB Kornhuber
Dec 21, 2000·Haemophilia : the Official Journal of the World Federation of Hemophilia·R LjungJ Tusell
Jul 10, 2001·Haemophilia : the Official Journal of the World Federation of Hemophilia·M R HackerM Manco-Johnson
Dec 4, 2001·Pediatrics·A D ShapiroUNKNOWN Academic Achievement in Children with Hemophilia Study Group
Nov 9, 2002·Acta Radiologica·K FischerH M van den Berg
Mar 22, 2003·Lancet·Kanjaksha GhoshDipika Mohanty
Apr 24, 2003·Haemophilia : the Official Journal of the World Federation of Hemophilia·V S BlanchetteM J Manco-Johnson
Apr 24, 2003·Haemophilia : the Official Journal of the World Federation of Hemophilia·H M van den BergJ G van der Bom
Jan 18, 2006·Haemophilia : the Official Journal of the World Federation of Hemophilia·K LindvallH Thykjaer
May 19, 2006·Journal of Thrombosis and Haemostasis : JTH·B M FeldmanUNKNOWN Association of Hemophilia Clinic Directors of Canada Prophylaxis Study Group
Nov 25, 2006·Haemophilia : the Official Journal of the World Federation of Hemophilia·P KomvilaisakV Blanchette
Mar 29, 2007·Journal of Thrombosis and Haemostasis : JTH·K van DijkH M van den Berg
Aug 10, 2007·The New England Journal of Medicine·Marilyn J Manco-JohnsonBruce L Evatt
Sep 21, 2007·Haemophilia : the Official Journal of the World Federation of Hemophilia·C TraivareeM D Carcao
Apr 22, 2008·Haemophilia : the Official Journal of the World Federation of Hemophilia·N RisebroughB M Feldman
Aug 8, 2008·Haemophilia : the Official Journal of the World Federation of Hemophilia·P De MoerlooseM Richards
Jan 16, 2009·Journal of Thrombosis and Haemostasis : JTH·P W CollinsUNKNOWN rAHF-PFM Study Group
Oct 23, 2009·International Journal of Technology Assessment in Health Care·Aliasghar Ahmad Kia DaliriJahanara Mamikhani
Feb 13, 2010·Haemophilia : the Official Journal of the World Federation of Hemophilia·S Björkman
Jun 29, 2010·Haemophilia : the Official Journal of the World Federation of Hemophilia·R WuJ Zhang
Jan 25, 2011·Journal of Thrombosis and Haemostasis : JTH·A GringeriUNKNOWN ESPRIT Study Group
Feb 16, 2011·Journal of Thrombosis and Haemostasis : JTH·M X HangUNKNOWN Canadian Hemophilia Primary Prophylaxis Study Group
Jan 4, 2012·Journal of Thrombosis and Haemostasis : JTH·L A ValentinoUNKNOWN Prophylaxis Study Group
May 3, 2012·Haemophilia : the Official Journal of the World Federation of Hemophilia·N DuncanM P Luo
Jun 29, 2012·Haemophilia : the Official Journal of the World Federation of Hemophilia·P W Collins
Jul 7, 2012·Haemophilia : the Official Journal of the World Federation of Hemophilia·B LundinUNKNOWN International Prophylaxis Study Group
Jul 11, 2012·Haemophilia : the Official Journal of the World Federation of Hemophilia·A SrivastavaUNKNOWN Treatment Guidelines Working Group on Behalf of The World Federation Of Hemophilia
Mar 14, 2013·Blood·Manuel D CarcaoUNKNOWN PedNet and the Rodin Study Group
Apr 5, 2013·Blood·Samantha C GouwUNKNOWN PedNet and Research of Determinants of INhibitor development (RODIN) Study Group
Jun 5, 2013·Haemophilia : the Official Journal of the World Federation of Hemophilia·S Björkman
Oct 26, 2013·Critical Reviews in Biotechnology·Timo RathRichard S Blumberg
Apr 26, 2014·Haemophilia : the Official Journal of the World Federation of Hemophilia·M Carcao

❮ Previous
Next ❯

Citations

May 8, 2016·Journal of Thrombosis and Haemostasis : JTH·P M MannucciM Franchini
Aug 20, 2016·Expert Opinion on Drug Metabolism & Toxicology·Alanna McEneny-KingAndrea N Edginton
May 24, 2017·Journal of Clinical Medicine·Marta BertaminoAngelo Claudio Molinari
Jan 5, 2017·Clinical and Applied Thrombosis/hemostasis : Official Journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis·Peyman EshghiRazieh Hantooshzadeh
May 8, 2018·Haemophilia : the Official Journal of the World Federation of Hemophilia·C HermansE Berntorp
Nov 9, 2018·Haemophilia : the Official Journal of the World Federation of Hemophilia·Martin J ScottCharles R M Hay
Oct 20, 2018·Journal of Pediatric Hematology/oncology·Meghan Drayton JacksonBryce A Kerlin
Jun 15, 2018·Therapeutic Advances in Hematology·Massimo Morfini, Stefano Gherardini
Dec 4, 2016·Hematology·Steven W Pipe

❮ 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
Alok Srivastava
Blood Transfusion = Trasfusione Del Sangue
Antonio CoppolaCiro De Simone
Blood Coagulation & Fibrinolysis : an International Journal in Haemostasis and Thrombosis
Anaadriana Zakarija
© 2021 Meta ULC. All rights reserved