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Title: Recombinant long-acting glycoPEGylated factor IX in hemophilia B: A multinational randomized phase 3 trial
Authors: Peter W. Collins
Guy Young
Karin Knobe
Faraizah Abdul Karim
Pantep Angchaisuksiri
Claus Banner
Türkiz Gürsel
Johnny Mahlangu
Tadashi Matsushita
Eveline P. Mauser-Bunschoten
Johannes Oldenburg
Christopher E. Walsh
Claude Negrier
Cardiff University
Keck School of Medicine of USC
Novo Nordisk AS
National Blood Centre
Mahidol University
Gazi University, Faculty of Medicine
University of Witwatersrand
Nagoya University Hospital
Van Creveld Haemophilia Clinic
Universitats-Klinikum Bonn und Medizinische Fakultat
The Mount Sinai Medical Center
Universite Claude Bernard Lyon 1
Keywords: Biochemistry, Genetics and Molecular Biology;Immunology and Microbiology;Medicine
Issue Date: 18-Dec-2014
Citation: Blood. Vol.124, No.26 (2014), 3880-3886
Abstract: © 2014 by The American Society of Hematology. This multinational, randomized, single-blind trial investigated the safety and efficacy of nonacog beta pegol, a recombinant glycoPEGylated factor IX (FIX) with extended half-life, in 74 previously treated patients with hemophilia B (FIX activity ≤2 IU/dL). Patients received prophylaxis for 52 weeks, randomized to either 10 IU/kg or 40 IU/kg once weekly or to on-demand treatment of 28 weeks. No patients developed inhibitors, and no safety concerns were identified. Three hundred forty-five bleeding episodeswere treated,with an estimated success rate of 92.2%. The median annualized bleeding rates (ABRs) were 1.04 in the 40 IU/kg prophylaxis group, 2.93 in the 10 IU/kg prophylaxis group, and 15.58 in the on-demand treatment group. In the 40 IU/kg group, 10 (66.7%) of 15 patients experienced no bleeding episodes into target joints compared with 1 (7.7%) of 13 patients in the 10 IU/kg group. Health-related quality of life (HR-QoL) assessed with the EuroQoL-5 Dimensions visual analog scale score improved from a median of 75 to 90 in the 40 IU/kg prophylaxis group. Nonacog beta pegol was well tolerated and efficacious for the treatment of bleeding episodes and was associatedwith low ABRs in patients receiving prophylaxis.Once-weekly prophylaxiswith 40 IU/kg resolved target joint bleeds in 66.7% of the affected patients and improved HR-QoL. This trial was registered at as #NCT01333111.
ISSN: 15280020
Appears in Collections:Scopus 2011-2015

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