von Willebrand factor to the rescue

被引:115
作者
De Meyer, Simon F. [1 ]
Deckmyn, Hans [1 ]
Vanhoorelbeke, Karen [1 ]
机构
[1] Katholieke Univ Leuven, Interdisciplinary Res Ctr, Lab Thrombosis Res, B-8500 Kortrijk, Belgium
关键词
PLATELET GLYCOPROTEIN IB; RECOMBINANT HUMAN INTERLEUKIN-11; SEVERE VONWILLEBRAND DISEASE; FACTOR-CLEAVING PROTEASE; HUMAN ENDOTHELIAL-CELLS; FACTOR-DEFICIENT MICE; FACTOR-VIII; GENE-THERAPY; HEMOPHILIA-A; DESMOPRESSIN DDAVP;
D O I
10.1182/blood-2008-10-165621
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
von Willebrand factor (VWF) is a large multimeric adhesive glycoprotein with complex roles in thrombosis and hemostasis. Abnormalities in VWF give rise to a variety of bleeding complications, known as von Willebrand disease (VWD), the most common inherited bleeding disorder in humans. Current treatment of VWD is based on the replacement of the deficient or dysfunctional protein either by endogenous release from endothelial Weibel-Palade bodies or by administration of plasma-derived VWF concentrates. During the last years, several efforts have been made to optimize existing therapies for VWD, but also to devise new approaches, such as inducing endogenous expression with interleukin-11, administering exogenous recombinant VWF, or introducing the protein via gene delivery. Clearly, the efficacy of any strategy will depend on several factors, including, for example, the quantity, activity, and stability of the delivered VWF. The inherent complexity of VWF biosynthesis, which involves extensive posttranslational processing, may be limiting in terms of producing active VWF outside of its native cellular sources. This review summarizes recent progress in the development of different treatment strategies for VWD, including those that are established and those that are at the experimental stage. Potential pitfalls and benefits of each strategy are discussed. (Blood. 2009; 113: 5049-5057)
引用
收藏
页码:5049 / 5057
页数:9
相关论文
共 104 条
[1]   Living with haemophilia and von Willebrand's: A descriptive qualitative study [J].
Barlow, Julie H. ;
Stapley, Jacqueline ;
Ellard, David R. .
PATIENT EDUCATION AND COUNSELING, 2007, 68 (03) :235-242
[2]  
Bernik R, 2008, IRISH J AGR FOOD RES, V47, P53
[3]   Long-term prophylaxis in von Willebrand disease [J].
Berntorp, E ;
Petrini, P .
BLOOD COAGULATION & FIBRINOLYSIS, 2005, 16 :S23-S26
[4]   The von Willebrand factor A3 domain does not contain a metal ion-dependent adhesion site motif [J].
Bienkowska, J ;
Cruz, M ;
Atiemo, A ;
Handin, R ;
Liddington, R .
JOURNAL OF BIOLOGICAL CHEMISTRY, 1997, 272 (40) :25162-25167
[5]   TREATMENT OF THE SEVERE BLEEDING EPISODE IN TYPE-III VONWILLEBRANDS DISEASE BY SIMULTANEOUS ADMINISTRATION OF CRYOPRECIPITATE AND PLATELET CONCENTRATE [J].
BODA, Z ;
PFLIEGLER, G ;
HARSFALVI, J ;
RAK, K .
BLOOD COAGULATION & FIBRINOLYSIS, 1991, 2 (06) :775-777
[6]  
BOND L, 1988, NEW ENGL J MED, V318, P121
[7]  
Brown SA, 2002, THROMB HAEMOSTASIS, V87, P990
[8]   Comparative analysis and classification of von Willebrand factor/factor VIII concentrates:: Impact on treatment of patients with von Willebrand disease [J].
Budde, Ulrich ;
Metzner, Hubert J. ;
Mueller, Heinz-Georg .
SEMINARS IN THROMBOSIS AND HEMOSTASIS, 2006, 32 (06) :626-635
[9]   THROMBOSIS FOLLOWING DESMOPRESSIN FOR UREMIC BLEEDING [J].
BYRNES, JJ ;
LARCADA, A ;
MOAKE, JL .
AMERICAN JOURNAL OF HEMATOLOGY, 1988, 28 (01) :63-65
[10]  
CASH JD, 1974, BRIT J HAEMATOL, V27, P363