Immune tolerance by intermittent factor VIII boluses in two high responder hemophilia A patients
Istituto di Semeiotica Medica, Università Cattolica del S. Cuore, Rome, Italy. Haematologica
(Impact Factor: 5.81).
10/2000; 85(10 Suppl):73-4.
Immune tolerance (IT) in hemophilia A patients with anti-factor VIII antibodies is generally based on daily factor VIII administrations. Here we report the preliminary results of an immune tolerance regimen based on recombinant high-dose (400 U/kg) factor VIII boluses administered at 48-hour intervals. Two high responder hemophilia A patients aged 2 and 3 years received this treatment without the need of permanent venous access. In both cases the IT regimen caused an anamnestic response of less than three weeks' duration and an antibody reduction to less than 5 Bethesda units was achieved in about 8-10 weeks. In the child with a more prolonged follow-up the inhibitor became undetectable after four months and factor VIII recovery at 6 months was > 85%. This intermittent high-dose regimen seems to be effective in rapidly inducing immune tolerance and seems particularly suitable for very young children in whom it may be useful to avoid the risks and to reduce the psychological burden of permanent venous access.
Available from: Guglielmo Mariani
Haematologica 04/2001; 86(3):225-6. · 5.81 Impact Factor
Available from: Marco Cattaneo
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ABSTRACT: Platelet aggregation is initiated by receptor activation coupled to intracellular signaling leading to activation of integrin alphaIIbbeta3. Recent advances in the study of platelet receptors for collagen, von Willebrand factor, thrombin, and adenosine diphosphate are providing new insights into the mechanisms of platelet aggregation.
Current Opinion in Hematology 10/2001; 8(5):270-6. DOI:10.1097/00062752-200109000-00002 · 3.97 Impact Factor
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ABSTRACT: Adenosine diphosphate (ADP) plays a crucial role in hemostasis and thrombosis, and its receptors are potential targets for antithrombotic drugs. Two G-protein-coupled P2 receptors contribute to platelet aggregation: the P2Y1 receptor initiates aggregation through mobilization of calcium stores, whereas the P2Y12 receptor coupled to adenylyl cyclase inhibition is essential for a full aggregation response to ADP and the stabilization of aggregates. The latter is defective in certain patients with a selective congenital deficiency of aggregation to ADP. It is also the target of the antithrombotic drug clopidogrel and of adenosine triphosphate analogues and other compounds currently under evaluation. In addition, the P2X1 ionotropic receptor is present in platelets, but its role is not yet completely known. Studies in P2Y1-knockout mice and experimental thrombosis models using selective P2Y1 antagonists have shown that the P2Y1 receptor, like the P2Y12 receptor, is a potential target for new antithrombotic drugs.
International Journal of Hematology 01/2002; 74(4):375-81. DOI:10.1007/BF02982079 · 1.92 Impact Factor
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