PRT-060318, a novel Syk inhibitor, prevents heparin-induced thrombocytopenia and thrombosis in a transgenic mouse model

Blood. 2011 Feb 17;117(7):2241-6. doi: 10.1182/blood-2010-03-274969. Epub 2010 Nov 18.

Abstract

Heparin-induced thrombocytopenia (HIT) is a major cause of morbidity and mortality resulting from the associated thrombosis. Extensive studies using our transgenic mouse model of HIT have shown that antibodies reactive with heparin-platelet factor 4 complexes lead to FcγRIIA-mediated platelet activation in vitro as well as thrombocytopenia and thrombosis in vivo. We tested PRT-060318 (PRT318), a novel selective inhibitor of the tyrosine kinase Syk, as an approach to HIT treatment. PRT318 completely inhibited HIT immune complex-induced aggregation of both human and transgenic HIT mouse platelets. Transgenic HIT model mice were treated with KKO, a mouse monoclonal HIT-like antibody, and heparin. The experimental group received orally dosed PRT318, whereas the control group received vehicle. Nadir platelet counts of PRT318-treated mice were significantly higher than those of control mice. When examined with a novel thrombosis visualization technique, mice treated with PRT318 had significantly reduced thrombosis. The Syk inhibitor PRT318 thus prevented both HIT immune complex-induced thrombocytopenia and thrombosis in vivo, demonstrating its activity in HIT.

MeSH terms

  • Animals
  • Antigen-Antibody Complex / blood
  • Antigen-Antibody Complex / drug effects
  • Blood Platelets / drug effects
  • Blood Platelets / enzymology
  • Blood Platelets / immunology
  • Heparin / adverse effects*
  • Humans
  • In Vitro Techniques
  • Intracellular Signaling Peptides and Proteins / antagonists & inhibitors*
  • Intracellular Signaling Peptides and Proteins / blood
  • Mice
  • Mice, Transgenic
  • Platelet Activation / drug effects
  • Platelet Activation / immunology
  • Protein Kinase Inhibitors / pharmacology*
  • Protein-Tyrosine Kinases / antagonists & inhibitors*
  • Protein-Tyrosine Kinases / blood
  • Receptors, IgG / antagonists & inhibitors
  • Syk Kinase
  • Thrombocytopenia / chemically induced
  • Thrombocytopenia / immunology
  • Thrombocytopenia / prevention & control*
  • Thrombosis / chemically induced
  • Thrombosis / immunology
  • Thrombosis / prevention & control*

Substances

  • Antigen-Antibody Complex
  • Intracellular Signaling Peptides and Proteins
  • Protein Kinase Inhibitors
  • Receptors, IgG
  • Heparin
  • Protein-Tyrosine Kinases
  • SYK protein, human
  • Syk Kinase
  • Syk protein, mouse