Treatment of type 3 von Willebrand disease by infusion of von Willebrand factor (VWF) and factor VIII (FVIII) concentrates may lead to the development of anti-VWF antibodies, challenging haemostasis management. The systematic review of the literature presented here retrieved 15 such cases (surgery n = 11, bleeding n = 4). The heterogeneous patient management mostly involved continuous infusion of FVIII, or recombinant FVIIa together with various other strategies. Off-label infusion of the bispecific monoclonal antibody emicizumab was prescribed in three cases and in a complex local case, ultimately well-controlled with emicizumab. This illustrates the fact that emicizumab appears as a therapeutic option in this context of allo-immunisation.
机构:
Univ Med Ctr Ljubljana, Univ Childrens Hosp, Dept Haematol & Oncol, Bohorrceva 20, Ljubljana 1000, SloveniaUniv Med Ctr Ljubljana, Univ Childrens Hosp, Dept Haematol & Oncol, Bohorrceva 20, Ljubljana 1000, Slovenia
Kotnik, Barbara Faganel
Strandberg, Karin
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Malmo Univ Hosp, Dept Clin Chem, Malmo, SwedenUniv Med Ctr Ljubljana, Univ Childrens Hosp, Dept Haematol & Oncol, Bohorrceva 20, Ljubljana 1000, Slovenia
Strandberg, Karin
Debeljak, Marusa
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Univ Childrens Hosp, Univ Med Ctr Ljubljana, Unity Special Lab Diagnost, Ljubljana, SloveniaUniv Med Ctr Ljubljana, Univ Childrens Hosp, Dept Haematol & Oncol, Bohorrceva 20, Ljubljana 1000, Slovenia
Debeljak, Marusa
Kitanovski, Lidija
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Univ Med Ctr Ljubljana, Univ Childrens Hosp, Dept Haematol & Oncol, Bohorrceva 20, Ljubljana 1000, SloveniaUniv Med Ctr Ljubljana, Univ Childrens Hosp, Dept Haematol & Oncol, Bohorrceva 20, Ljubljana 1000, Slovenia
Kitanovski, Lidija
Jazbec, Janez
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Univ Med Ctr Ljubljana, Univ Childrens Hosp, Dept Haematol & Oncol, Bohorrceva 20, Ljubljana 1000, SloveniaUniv Med Ctr Ljubljana, Univ Childrens Hosp, Dept Haematol & Oncol, Bohorrceva 20, Ljubljana 1000, Slovenia
Jazbec, Janez
Benedik-Dolnicar, Majda
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机构:
Natl Haemophilia Ctr, Ljubljana, SloveniaUniv Med Ctr Ljubljana, Univ Childrens Hosp, Dept Haematol & Oncol, Bohorrceva 20, Ljubljana 1000, Slovenia
Benedik-Dolnicar, Majda
Bakija, Alenka Trampus
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Univ Childrens Hosp, Univ Med Ctr Ljubljana, Unity Special Lab Diagnost, Ljubljana, SloveniaUniv Med Ctr Ljubljana, Univ Childrens Hosp, Dept Haematol & Oncol, Bohorrceva 20, Ljubljana 1000, Slovenia
机构:
Univ Pittsburgh, Dept Med, Div Hematol Oncol, Med Ctr, Pittsburgh, PA 15213 USA
Hemophilia Ctr Western Penn, Pittsburgh, PA USAUniv Pittsburgh, Dept Med, Div Hematol Oncol, Med Ctr, Pittsburgh, PA 15213 USA
Seaman, Craig D.
Ragni, Margaret, V
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Univ Pittsburgh, Dept Med, Div Hematol Oncol, Med Ctr, Pittsburgh, PA 15213 USA
Hemophilia Ctr Western Penn, Pittsburgh, PA USAUniv Pittsburgh, Dept Med, Div Hematol Oncol, Med Ctr, Pittsburgh, PA 15213 USA
机构:
Washington Univ, Sch Med, Dept Pediat, Div Hematol Oncol, 660 S Euclid Ave,Campus Box 8208, St Louis, MO 63110 USAWashington Univ, Sch Med, Dept Pediat, Div Hematol Oncol, 660 S Euclid Ave,Campus Box 8208, St Louis, MO 63110 USA
Sadler, Brooke
Castaman, Giancarlo
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Careggi Univ Hosp, Dept Oncol, Ctr Bleeding Disorders & Coagulat, Florence, ItalyWashington Univ, Sch Med, Dept Pediat, Div Hematol Oncol, 660 S Euclid Ave,Campus Box 8208, St Louis, MO 63110 USA
Castaman, Giancarlo
O'Donnell, James S.
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机构:
Royal Coll Surgeons Ireland, Sch Pharm & Biomol Sci, Irish Ctr Vasc Biol, Dublin 2, Ireland
St James Hosp, Natl Coagulat Ctr, Dublin, IrelandWashington Univ, Sch Med, Dept Pediat, Div Hematol Oncol, 660 S Euclid Ave,Campus Box 8208, St Louis, MO 63110 USA