Nghiên cứu giai đoạn 3 về protein hợp nhất factor VIII tái tổ hợp Fc trong bệnh máu khó đông A nặng

Blood - Tập 123 Số 3 - Trang 317-325 - 2014
Johnny Mahlangu1, Jerry S. Powell2, Margaret V. Ragni3, Pratima Chowdary4, Neil C. Josephson5, Ingrid Pabinger6, Hideji Hanabusa7, Naresh Gupta8, Roshni Kulkarni9, Patrick Fogarty10, David J. Perry11, Amy D. Shapiro12, John Pasi13, Shashikant Apte14, Ivan Nestorov15, Haiyan Jiang15, Shuanglian Li15, Srividya Neelakantan15, Lynda M. Cristiano15, Jaya Goyal15, Jürg M. Sommer15, Jennifer Dumont15, Nigel Dodd15, Karen Nugent15, Gloria Vigliani15, Aoife M. Brennan15, Glenn F. Pierce15
1Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service., Johannesburg, South Africa
2University of California at Davis, Davis, California;
3University of Pittsburgh and the Hemophilia Center of Western Pennsylvania, Pittsburgh, PA;
4Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free Hospital, London, United Kingdom;
5Puget Sound Blood Center, Seattle, WA
6Universitätsklinik für Innere Medizin I, Medizinische Universität Wien, Wien, Austria
7Department of Hematology, Ogikubo Hospital, Suginami-ku, Tokyo, Japan;
8Haemophilia Centre and Department of Medicine, Maulana Azad Medical College & Associated Lok Nayak Jai Prakash Narayan Hospital, New Delhi, India;
9Department of Pediatrics and Human Development, Michigan State University, East Lansing, MI;
10Penn Comprehensive Hemostasis and Thrombosis Program, Hospital of the University of Pennsylvania, Philadelphia, PA;
11Cambridge Haemophilia & Thrombosis Centre, Addenbrooke’s Hospital, Cambridge, United Kingdom;
12Indiana Hemophilia and Thrombosis Center, Department of Hematology, Indianapolis, IN;
13Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, United Kingdom
14Sahyadri Hospital, Department of Hematology, Mahara, India; and
15Biogen Idec Hemophilia, Cambridge, MA

Tóm tắt

Các điểm chính

Factor VIII tái tổ hợp mới có thời gian bán hủy kéo dài, rFVIIIFc, đã được phát triển nhằm giảm tần suất tiêm phòng. rFVIIIFc được dung nạp tốt ở bệnh nhân mắc bệnh máu khó đông A nặng, và dẫn đến tỷ lệ xuất huyết thấp khi được tiêm 1 đến 2 lần mỗi tuần.

Từ khóa


Tài liệu tham khảo

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Blood

Tập 123 Số 3

317-325

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