Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial

DOI PDF 6 Citations
  • Peter W. Collins
    Arthur Bloom Haemophilia Centre, Institute of Infection and Immunity, School of Medicine, Cardiff University, Cardiff, United Kingdom;
  • Guy Young
    Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA;
  • Karin Knobe
    Haemophilia R&D Portfolio, Novo Nordisk A/S, Soeborg, Denmark;
  • Faraizah Abdul Karim
    Haemophilia Centre, National Blood Centre, Kuala Lumpur, Malaysia;
  • Pantep Angchaisuksiri
    Division of Hematology, Department of Medicine, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand;
  • Claus Banner
    Haemophilia Clinical Operations, Novo Nordisk A/S, Soeborg, Denmark;
  • Türkiz Gürsel
    Gazi University Medical School, Ankara, Turkey;
  • Johnny Mahlangu
    Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, National Health Laboratory Services and University of the Witwatersrand, Johannesburg, South Africa;
  • Tadashi Matsushita
    Department of Transfusion Medicine, Nagoya University Hospital, Nagoya, Japan;
  • Eveline P. Mauser-Bunschoten
    Van Creveldkliniek, University Medical Centre, Utrecht, The Netherlands;
  • Johannes Oldenburg
    Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany;
  • Christopher E. Walsh
    Division of Hematology and Medical Oncology, Mount Sinai Medical Center, New York, NY; and
  • Claude Negrier
    Hôpital Edouard Herriot, University Claude Bernard Lyon 1, Lyon, France

Description

<jats:title>Key Points</jats:title><jats:p>Nonacog beta pegol, a recombinant glycoPEGylated FIX with extended half-life, was developed to improve care for patients with hemophilia B. Weekly prophylaxis with nonacog beta pegol was well tolerated and was associated with low bleeding rates and an improved quality of life.</jats:p>

Journal

  • Blood

    Blood 124 (26), 3880-3886, 2014-12-18

    American Society of Hematology

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