Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A

Author:

Mahlangu Johnny1,Kuliczkowski Kazimierz2,Karim Faraizah Abdul3,Stasyshyn Oleksandra4,Kosinova Marina V.5,Lepatan Lynda Mae6,Skotnicki Aleksander7,Boggio Lisa N.8,Klamroth Robert9,Oldenburg Johannes10,Hellmann Andrzej11,Santagostino Elena12,Baker Ross I.13,Fischer Kathelijn14,Gill Joan C.15,P’Ng Stephanie16,Chowdary Pratima17,Escobar Miguel A.18,Khayat Claudia Djambas19,Rusen Luminita20,Bensen-Kennedy Debra21,Blackman Nicole21,Limsakun Tharin21,Veldman Alex22,St. Ledger Katie21,Pabinger Ingrid23,

Affiliation:

1. Department of Molecular Medicine and Haematology, Faculty of Health Sciences, University of the Witwatersrand, National Health Laboratory Service, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa;

2. Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland;

3. Hemophilia Clinic, National Blood Centre, Kuala Lumpur, Malaysia;

4. Institute of Blood Pathology and Transfusion Medicine, National Academy of Medical Sciences of Ukraine, Lviv, Ukraine;

5. Kemerovo Regional Clinical Hospital, Kemerovo, Russia;

6. Perpetual Succour Hospital, Cebu, Philippines;

7. Krakow Medical Centre, Krakow, Poland;

8. Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, IL;

9. Vivantes Clinic Friedrichshain, Berlin, Germany;

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

11. Department of Hematology and Transplantation, Medical University of Gdansk, Gdansk, Poland;

12. A. Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca’ Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy;

13. Western Australian Centre for Thrombosis and Haemostasis, Murdoch University, Perth, Australia;

14. Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands;

15. Blood Center of Wisconsin and Medical College of Wisconsin, Milwaukee, WI;

16. Royal Perth Hospital, Perth, Australia;

17. Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free Hospital, London, United Kingdom;

18. University of Texas Health Science Center and The Gulf States Hemophilia and Thrombophilia Center, Houston, TX;

19. Lebanon Department of Pediatrics, Hotel Dieu de France Hospital, Beirut, Lebanon;

20. Sanador SRL, Bucharest, Romania;

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

22. Clinical Research and Development, CSL Behring, Marburg, Germany; and

23. Clinical Division of Haematology and Haemostaseology, Medical Clinic I, Medical University Vienna, Vienna, Austria

Abstract

Key Points rVIII-SingleChain is a novel rFVIII, designed to have high stability and high binding affinity for VWF. In severe hemophilia A patients, rVIII-SingleChain was well tolerated and resulted in low bleeding rates, when dosed twice per week.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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