Abstract
Turoctocog alfa pegol (N8-GP) is a site-specific, 40 kDa glycoPEGylated recombinant factor VIII (FVIII) product with an extended half-life. The comprehensive main phase of the pivotal pathfinder 2 trial showed N8-GP dosed every 4 days (Q4D) provided favourable safety and efficacy for preventing bleeds in 175 patients with haemophilia A. We investigated the safety and efficacy of N8-GP prophylaxis when administered weekly (Q7D) for 24 weeks to patients with low bleeding rates in the pathfinder 2 extension trial. Patients (≥12 years) with ≤2 bleeds during the preceding 6 months of the pathfinder 2 main phase were eligible for randomization to receive N8-GP 50 IU/kg Q4D or 75 IU/kg Q7D. Safety and efficacy endpoints were incidence of FVIII inhibitors and annualized bleeding rate (ABR), respectively. Fifty-five of 143 (38.5%) patients on prophylaxis who continued into the extension phase were randomized to receive 50 IU/kg Q4D (n = 17) or 75 IU/kg Q7D (n = 38). Nine patients in the Q7D cohort reverted to 50 IU/kg Q4D. No inhibitors were detected. In both cohorts, >50% of patients experienced no bleeds. Median ABR for overall, joint, spontaneous, traumatic and muscle was 0.00 for both cohorts. Overall estimated success rate for trea...Continue Reading
References
Jul 10, 2001·Haemophilia : the Official Journal of the World Federation of Hemophilia·M R HackerM Manco-Johnson
Aug 15, 2002·European Journal of Health Law·M HirtleD Sprumont
Feb 26, 2004·Journal of Vascular Nursing : Official Publication of the Society for Peripheral Vascular Nursing·Gail Ann DeLuca Havens
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·M RichardsC Rothschild
Jul 9, 2010·Current Opinion in Hematology·David Lillicrap
Dec 14, 2011·Hematology·Patrick F Fogarty
Jan 4, 2012·Journal of Thrombosis and Haemostasis : JTH·L A ValentinoUNKNOWN Prophylaxis Study Group
Apr 27, 2012·Haemophilia : the Official Journal of the World Federation of Hemophilia·S ZappaG Allen
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
Feb 13, 2013·Journal of Thrombosis and Haemostasis : JTH·A TiedeD Viuff
Nov 15, 2013·Blood·Johnny MahlanguUNKNOWN A-LONG Investigators
Nov 22, 2013·PloS One·Axel Newe, Thomas Ganslandt
Jun 10, 2014·Seminars in Thrombosis and Hemostasis·Massimo Franchini, Pier Mannuccio Mannucci
Apr 29, 2015·Journal of Thrombosis and Haemostasis : JTH·G YoungG Allen
Jul 15, 2015·Blood·Barbara A KonkleBrigitt Abbuehl
Mar 18, 2016·Blood·Marshall A MazepaUNKNOWN US Hemophilia Treatment Center Network
Jan 1, 1995·Haemophilia : the Official Journal of the World Federation of Hemophilia·K A Rickard
Oct 21, 2016·Haemophilia : the Official Journal of the World Federation of Hemophilia·E BerntorpS Lethagen
Dec 3, 2016·Thrombosis and Haemostasis·Paul GiangrandeUNKNOWN Pathfinder™2 Investigators
Dec 20, 2016·Journal of Thrombosis and Haemostasis : JTH·M T RedingL A Michaels
Oct 26, 2018·Circulation·David M ReboussinSuma Vupputuri
Citations
Oct 17, 2019·Journal of Thrombosis and Haemostasis : JTH·Robert KlamrothPratima Chowdary
Jan 29, 2020·Expert Opinion on Investigational Drugs·Pier Mannuccio Mannucci
Feb 6, 2020·Journal of Managed Care & Specialty Pharmacy·Mindy L SimpsonSongkai Yan
Jun 10, 2020·Expert Review of Clinical Pharmacology·Massimo Morfini, Emanuela Marchesini
Mar 11, 2020·Drugs in R&D·Inga BjørnsdottirOla Sternebring
Dec 22, 2020·Seminars in Thrombosis and Hemostasis·Matteo Nicola Dario Di MinnoMassimo Franchini
Jan 28, 2021·ClinicoEconomics and Outcomes Research : CEOR·Gary BensonXin Ying Lee
Feb 5, 2021·Journal of Blood Medicine·Mariasanta NapolitanoHermann Eichler
Apr 6, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·Mark T RedingShadan Lalezari
May 7, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·Pier Mannuccio MannucciGiovanni Di Minno
Jun 23, 2021·Scientific Reports·Cihan AyIngrid Pabinger
Aug 19, 2021·The Cochrane Database of Systematic Reviews·Omotola O OlasupoDavide Matino
Sep 26, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·Andreas TiedePratima Chowdary
Dec 6, 2021·Thrombosis and Haemostasis·Pratima ChowdaryAndreas Tiede