2003; baxter - Supplements - Haematologica

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haematologicahImmunobiology of Tolerance InductionCatalytic antibodies to factor VIIISébastien Lacroix-Desmazes, Michel D. Kazatchkine, Srini Kaveri...........................48Idiotypic control of inhibitorsJean Guy Gilles..................................................................................................52Murine models for the study of factor VIII inhibitorsBirgit M. Reipert, Maria Sasgary, Christina Hausl, Elisabeth Maier,Rafi U. Ahmad, Peter L. Turecek, Hans P. Schwarz ...............................................55T-lymphocytes in the anti-factor VIII immune responseMarc G. Jacquemin, Jean-Marie R. Saint-Remy ....................................................64Prospectives of immonotherapy for inhibitor patientsJean-Marie R. Saint-Remy ..................................................................................66New Aspects in Treatment of Hemophilia B Patients2003; vol. 88; supplement no. 12 - September 2003(indexed by Current Contents/Life Sciences and in Faxon Finder and Faxon XPRESS, also available on diskette with abstracts)http://www.haematologica.org/Immune tolerance induction in hemophilia B. International immune tolerance registryI. Warrier .........................................................................................................69The Malmö immune tolerance experience in hemophilia BErik Berntorp.....................................................................................................71Factor IX mutations and inhibitor development in hemophilia BDavid Lillicrap...................................................................................................75Acquired Inhibitors in Non-hemophiliacsModified Bonn-Malmö Protocol (MBM-P)L. Hess, H. Zeitler, Ch. Unkrig, W. Nettekoven, T. Albert, R. Schwaab,W. Effenberger, J. Oldenburg, H. Vetter, P. Hanfland, H.H. Brackmann .................78Management of severe hemorrhage and inhibitor-elimination in acquired hemophilia:the modified Heidelberg-Malmö protocolA. Huth-Kühne, P. Lages, H. Hampel, R. Zimmermann ........................................86Acquired factor VIII and factor IX inhibitors: a survey of Italian Hemophilia CentersFrancesco Baudo, Gianni Mostarda, Francesco de Cataldo .....................................93Immunological responsiveness of maternal T cells to self antigens during pregnancy:pregnancy as a model to study peripheral T cell tolerance and the role of co-stimulatorymolecules in tolerance inductionMelanie S. Vacchio, Richard J. Hodes .................................................................100Ten years experience with immune tolerance induction therapy in acquired hemophiliaLaszlo Nemes, Ervin Pitlik ................................................................................106Gene TherapyThe host immune response and risk of inhibitor development following adenoviral genetherapy for hemophiliaDavid Lillicrap.................................................................................................111Gene therapy for hemophilia A: immune consequences of viral-vector mediated factorVIII gene transferThierry VandenDriessche, Desire Collen, Marinee K.L. Chuah .............................115Immunologic sequelae and potential for inhibitor development in adeno-associatedviral gene therapy for hemophilia BRoland W. Herzog............................................................................................122Index of authors
eview paperForewordHANS-HERMANN BRACKMANNhaematologica 2003; 88(suppl. n. 12):1http://www.haematologica.org/free/immunotolerance2001.pdfThe 4 th International Workshop on Immune Tolerancewas held in August 2001 in Bonn-Königswinter, Germany and attracted approximately160 participants to share and discuss themost burning and unresolved issues related toinhibitor treatment in hemophilia. To host all participantsin Königswinter was a particular honourbecause the same year the Hemophilia Center inBonn celebrated its 30 th anniversary.Looking back into the history of hemophiliatreatment we have to recognize that it took a verylong time to understand the pathophysiology ofhemophilia and to develop a diagnostic methodsuch as a simple clotting test.With the increasing knowledge in coagulation,treatment could rapidly be developed. Nowadayssafe products are available and treatment scheduleshave been established; however, development ofinhibitors remains a major challenge.We know that genetic defect and factor exposuredays play a role in the development ofinhibitors, we know how to induce immune tolerancein some of our patients using specific treatmentprotocols. Nevertheless many questions stillremain to be answered, in particular as regards ourunderstanding of the mechanism of inhibitordevelopment. We should therefore focus ourefforts on the most important questions, forinstance the question why some patients developinhibitors, why others do not, why immune toleranceis successful in some patients and in somenot. Researchers and hemophilia treaters all overthe world are working hard to find those answers.The agenda of this meeting focused on immunologicaltopics in association with hemophilia andinhibitors in order to provide an understanding ofcurrent concepts and new approaches.The 4th International Workshop was placedunder the same motto that is inscribed at the baseof the Statue of Liberty, welcoming people from allover the world with the promise of freedom, whichwe would also like to give to all our patients: «Giveme your tired, your poor, your huddled masses, yearningto be free».Hans-Hermann BrackmannAcknowledgmentsI am grateful to all speakers and authors whohave contributed to this supplement which providesvaluable information for researchers, healthcareprofessionals and caregivers dedicated tohemophilia issues and hemophilia patients.I wish also to thank all my co-workers who havedone their best to make this workshop successful.Last but not least I would like to thank my friendGuglielmo Mariani for the partnership in thisworkshop and his exceptional friendship.This publication was made possible by an educationalgrant provided by Baxter BioScience.haematologica vol. 88(supplement n. 12):september 2003
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Index of authorsAhmad, R.U., 56Albe
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2003; baxter - Supplements - Haematologica

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