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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
biochemistry, genetics and molecular biology
Evaluation of efficacy and safety of the anti-VWF Nanobody ALX-0681 in a preclinical baboon model of acquired thrombotic thrombocytopenic purpura
Blood, Volume 120, No. 17, Year 2012
Notification
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Description
ALX-0681 is a therapeutic Nanobody targeting the A1-domain of VWF. It inhibits the interaction between ultra-large VWF and platelet GpIb-IX-V, which plays a crucial role in the pathogenesis of thrombotic thrombocytopenic purpura (TTP). In the present study, we report the efficacy and safety profile of ALX-0681 in a baboon model of acquired TTP. In this model, acute episodes of TTP are induced by administration of an ADAMTS13-inhibiting mAb. ALX-0681 completely prevented the rapid onset of severe thrombocytopenia and schistocytic hemolytic anemia. After induction of TTP, platelet counts also rapidly recovered on administration of ALX-0681. This effect was corroborated by the full neutralization of VWF activity. The schistocytic hemolytic anemia was also halted and partially reversed by ALX-0681 treatment. Brain CT scans and post mortem analysis did not reveal any sign of bleeding, suggesting that complete neutralization of VWF by ALX-0681 under conditions of thrombocytopenia was not linked with an excessive bleeding risk. The results obtained in this study demonstrate that ALX-0681 can successfully treat and prevent the most important hallmarks of acquired TTP without evidence of a severe bleeding risk. Therefore, ALX-0681 offers an attractive new therapeutic option for acquired TTP in the clinical setting. © 2012 by The American Society of Hematology.
Authors & Co-Authors
Callewaert, Filip
Belgium, Zwijnaarde
Ablynx nv
Roodt, Jan P.
South Africa, Bloemfontein
University of the Free State
South Africa, Bloemfontein
Universitas Private Hospital
Ulrichts, Hans
Belgium, Zwijnaarde
Ablynx nv
Stöhr, Thomas
Belgium, Zwijnaarde
Ablynx nv
Janse van Rensburg, Walter James
South Africa, Bloemfontein
University of the Free State
Lamprecht, Seb
South Africa, Bloemfontein
University of the Free State
Rossenu, Stefaan
Belgium, Zwijnaarde
Ablynx nv
Priem, Sofie
Belgium, Zwijnaarde
Ablynx nv
Willems, Wouter
Belgium, Zwijnaarde
Ablynx nv
Holz, Josefin Beate
Belgium, Zwijnaarde
Ablynx nv
Statistics
Citations: 113
Authors: 10
Affiliations: 3
Identifiers
Doi:
10.1182/blood-2012-04-420943
ISSN:
00064971