Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial

Author:

Santagostino Elena1,Martinowitz Uri2,Lissitchkov Toshko3,Pan-Petesch Brigitte4,Hanabusa Hideji5,Oldenburg Johannes6,Boggio Lisa7,Negrier Claude8,Pabinger Ingrid9,von Depka Prondzinski Mario10,Altisent Carmen11,Castaman Giancarlo1213,Yamamoto Koji14,Álvarez-Roman Maria-Teresa15,Voigt Christine16,Blackman Nicole16,Jacobs Iris16

Affiliation:

1. Istituto di Ricovero e Cura a Carattere Scientifico Ca’ Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy;

2. The Israeli National Haemophilia Center, Chaim Sheba Medical Center, Tel Hashomer, Israel;

3. Department of Coagulation Disorders and Anemia, Specialized Hospital for Active Treatment Joan Pavel, Sofia, Bulgaria;

4. Centre Hospitalier Régional Universitaire de Brest, Hôpital A. Morvan, Brest, France;

5. Department of Hematology, Ogikubo Hospital, Tokyo, Japan;

6. Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany;

7. Rush Hemophilia and Thrombophilia Center, Rush University, Chicago, IL;

8. Centre Régional de Traitement de l’Hémophilie, Hôpital Edouard Herriot, University Claude Bernard, Lyon, France;

9. Division of Haematology and Haemostaseology, Medical University of Vienna, Vienna, Austria;

10. Werlhof Institut, Hannover, Germany;

11. Unitat Hemofilia, Hospital Vall d’Hebron, Barcelona, Spain;

12. Haemophilia and Thrombosis Centre, Department of Haematology, San Bortolo Hospital, Vicenza, Italy;

13. Center for Bleeding Disorders, Careggi University Hospital, Florence, Italy;

14. Nagoya University Hospital, Nagoya, Japan;

15. Hospital la Paz, Madrid, Spain; and

16. Clinical Research and Development, CSL Behring, King of Prussia, PA

Abstract

Key Points rIX-FP maintains mean trough of 20 and 12 IU/dL FIX activity with 40 IU/kg weekly and 75 IU/kg every 2 weeks prophylaxis, respectively. Weekly and 14-day prophylaxis regimens with rIX-FP were well tolerated and provided low bleeding rates and target joint improvement.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Reference26 articles.

1. Haemophilias A and B.;Bolton-Maggs;Lancet,2003

2. Guidelines for the management of hemophilia.;Srivastava;Haemophilia,2013

3. Baxter Healthcare Corporation. RIXUBIS (package insert). http://www.rixubis.com/pdf/rixubis_pi.pdf. Accessed September 1, 2015

4. Biogen Idec Inc. ALPROLIX (package insert). http://www.alprolix.com/pdfs/PrescribingInformation.pdf. Accessed September 1, 2015

5. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B.;Powell;N Engl J Med,2013

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