Publication: Recombinant long-acting glycoPEGylated factor IX in hemophilia B: A multinational randomized phase 3 trial
1
Issued Date
2014-12-18
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ISSN
15280020
00064971
00064971
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2-s2.0-84919491430
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Mahidol University
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SCOPUS
Bibliographic Citation
Blood. Vol.124, No.26 (2014), 3880-3886
Suggested Citation
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 Recombinant long-acting glycoPEGylated factor IX in hemophilia B: A multinational randomized phase 3 trial. Blood. Vol.124, No.26 (2014), 3880-3886. doi:10.1182/blood-2014-05-573055 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/33196
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Title
Recombinant long-acting glycoPEGylated factor IX in hemophilia B: A multinational randomized phase 3 trial
Other Contributor(s)
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
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
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 www.clinicaltrials.gov as #NCT01333111.