The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B.

Blood
Amy D ShapiroD A Roth

Abstract

This international clinical trial evaluated the safety and efficacy of recombinant factor IX (rFIX) in previously untreated patients (PUPs) with severe or moderately severe hemophilia B (FIX activity, < or = 3 IU/dL). Sixty-three PUPs aged younger than 1 month to 14 years received rFIX (median treatment duration, 37 months; range, 4-64 months). Mean rFIX recovery (0.68 +/- 0.27 IU/dL per IU/kg) remained constant over 5 years and was similar in infants (1 month to < 2 years) and children (2 to < 12 years). Fifty-four PUPs used rFIX (median dose, 62.7 IU/kg per infusion; range, 8.2-292 IU/kg) to treat 997 hemorrhages. Bleeding was well controlled, with 75% of hemorrhages requiring only one rFIX infusion. Response to rFIX was "excellent" or "good" in 94% of cases. Effective hemostasis was achieved in 32 PUPs receiving rFIX for routine prophylaxis, with 91% of prophylaxis responses rated "excellent." rFIX administered for 30 surgical procedures in 23 PUPs achieved hemostasis for all rated procedures. Five patients experienced allergic-type manifestations, including 2 (3%) patients who developed FIX inhibitors (both > 5 BU/dL). rFIX was well tolerated, with no associated thrombotic events or evidence of viral transmission. These dat...Continue Reading

Citations

Sep 26, 2013·International Journal of Hematology·Barbara DietrichEva-Maria Muchitsch
Jun 2, 2011·Indian Journal of Hematology & Blood Transfusion : an Official Journal of Indian Society of Hematology and Blood Transfusion·Kanjaksha Ghosh, Kinjalka Ghosh
Mar 10, 2001·Haemophilia : the Official Journal of the World Federation of Hemophilia·M V Ragni
Feb 23, 2013·Biologics : Targets & Therapy·Massimo FranchiniCarlo Bonfanti
Feb 16, 2011·Drugs·Trisha Wong, Michael Recht
Oct 10, 2013·EMBO Molecular Medicine·David M MarkusicRoland W Herzog
May 13, 2014·Immunotherapy·Leonard A Valentino
Aug 13, 2014·Expert Review of Hematology·Massimo Franchini
Aug 8, 2014·Expert Opinion on Emerging Drugs·Pier Mannuccio Mannucci, Massimo Franchini
Jan 27, 2016·European Journal of Haematology·Gerry DolanRiitta Lassila
May 15, 2012·Journal of Chromatography. B, Analytical Technologies in the Biomedical and Life Sciences·Yu-ling SunChon-ho Yen
Aug 30, 2008·American Journal of Surgery·Marian N SafaouiDaniel R Margulies
Aug 7, 2007·Lancet·Nigel S Key, Claude Negrier
Dec 27, 2005·Best Practice & Research. Clinical Haematology·Cassandra D Josephson, Thomas C Abshire
Aug 13, 2011·Thrombosis and Haemostasis·Adam GaterMartin Strandberg-Larsen
Jul 22, 2015·Blood Coagulation & Fibrinolysis : an International Journal in Haemostasis and Thrombosis·Pablo RendoFrank Shafer
Oct 21, 2005·Haemophilia : the Official Journal of the World Federation of Hemophilia·A D ShapiroM-C Poon
Aug 17, 2005·Journal of Thrombosis and Haemostasis : JTH·S W Pipe
Feb 28, 2009·Journal of Thrombosis and Haemostasis : JTH·F BernardiUNKNOWN International Factor VII Deficiency Study Group
May 15, 2007·Haemophilia : the Official Journal of the World Federation of Hemophilia·T LambertD A Roth
Jun 25, 2008·Haemophilia : the Official Journal of the World Federation of Hemophilia·R LjungD DiMichele
May 26, 2006·Pediatric Blood & Cancer·Leonard A Valentino, Veeral M Oza
Apr 27, 2012·Haemophilia : the Official Journal of the World Federation of Hemophilia·S ZappaG Allen
Jun 22, 2013·Haemophilia : the Official Journal of the World Federation of Hemophilia·S Björkman
Nov 3, 2011·Haemophilia : the Official Journal of the World Federation of Hemophilia·E BerntorpP Rendo
Nov 20, 2012·British Journal of Haematology·Peter W CollinsUNKNOWN UK Haemophilia Centre Doctors
Dec 14, 2011·Haemophilia : the Official Journal of the World Federation of Hemophilia·B SørensenM Makris
Dec 24, 2010·Vox Sanguinis·T Burnouf
Jan 15, 2014·Haemophilia : the Official Journal of the World Federation of Hemophilia·L A ValentinoP Rendo
Jan 12, 2010·Haemophilia : the Official Journal of the World Federation of Hemophilia·P E MonahanD A Roth
Dec 17, 2014·Haemophilia : the Official Journal of the World Federation of Hemophilia·T UrasinskiB E Abbuehl
Dec 18, 2009·Haemophilia : the Official Journal of the World Federation of Hemophilia·T LissitchkovA Paez
Jul 17, 2013·Haemophilia : the Official Journal of the World Federation of Hemophilia·J PuetzUNKNOWN Hemophilia Treatment Center Network (HTCN) Investigators
Sep 6, 2012·Expert Opinion on Pharmacotherapy·Massimo FranchiniCarlo Bonfanti
Aug 10, 2005·Expert Opinion on Pharmacotherapy·Paul Giangrande
Jun 30, 2016·Haemophilia : the Official Journal of the World Federation of Hemophilia·A SuzukiJ M Korth-Bradley
Jun 6, 2006·Molecular Therapy : the Journal of the American Society of Gene Therapy·Lieven ThorrezThierry Vandendriessche

❮ 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
M V RagniRecombinant FIX Surgical Study Group
JAMA : the Journal of the American Medical Association
Kathryn A O'ConnellM Miles Braun
© 2021 Meta ULC. All rights reserved