Review Article
Achievements, challenges and unmet needs for haemophilia patients with inhibitors
Report from a symposium in Paris, France on 20 November 2014
Y. Dargaud,
A. Pavlova,
S. Lacroix-Desmazes,
K. Fischer,
M. Soucie,
S. Claeyssens,
D.w. Scott,
R. d'Oiron,
G. Lavigne-Lissalde,
G. Kenet,
C. Escuriola Ettingshausen,
A. Borel-Derlon,
T. Lambert,
G. Pasta,
C. Négrier,
Corresponding Author
Y. Dargaud
Unite d'Hemostase Clinique, Hopital Cardiologique Louis Pradel, Universite Lyon 1, Lyon, France
Correspondence: Yesim Dargaud, Unite d'Hemostase Clinique, Hopital Cardiologique Louis Pradel, Universite Lyon 1, Lyon 69008 France.
Tel.: +33472118810; fax: +33472118817;
e-mail: [email protected]
Search for more papers by this authorA. Pavlova
Institute of Experimental Haematology and Transfusion Medicine, University Clinic, Bonn, Germany
Search for more papers by this authorS. Lacroix-Desmazes
INSERM UMRS 1138, Immunopathologie et immuno-intervention thérapeutique, Centre de Recherche des Cordeliers, Paris, France
Search for more papers by this authorK. Fischer
Van Creveldkliniek (HP C01.425), University Medical Center Utrecht, Utrecht, The Netherlands
Search for more papers by this authorM. Soucie
Division of Blood Disorders, National Center for Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA
The findings and conclusions in this report are those of the authors and do not necessarily represent the official position of the Centers for Disease Control and Prevention.Search for more papers by this authorS. Claeyssens
Chu Purpan Pav. Centre Hospitalier Lefebvre, Centre Rgal de l'Hemophilie, Toulouse, France
Search for more papers by this authorD.w. Scott
Department of Medicine, Uniformed Services, University for the Health Sciences, Bethesda, MD, USA
Search for more papers by this authorR. d'Oiron
Centre de Traitement de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles, Hôpitaux Universitaires Paris Sud - Site Bicêtre, Le Kremlin-Bicêtre, France
Search for more papers by this authorG. Lavigne-Lissalde
Laboratoire d'Hématologie et Consultations d'Hématologie Biologique Centre Hospitalier Universitaire de Nîmes, Place du Pr R. Debré Nîmes, France
Search for more papers by this authorG. Kenet
National Hemophilia Institute, Sheba Medical Center, Tel-Hashomer, Tel Aviv University, Tel Aviv, Israel
Search for more papers by this authorC. Escuriola Ettingshausen
Hemophilia Centre Rhein-Main HZRM, Frankfurt-Mörfelden, Germany
Search for more papers by this authorA. Borel-Derlon
Haemophilia and von Willebrand Disease Centre, University Hospital of Caen, Caen
Search for more papers by this authorT. Lambert
Hemophilia Care Center, Bicêtre AP-HP Hospital and Faculté de Médecine Paris XI, Paris, France
Search for more papers by this authorG. Pasta
UOSD di Ortopedia e Traumatologia, Centro Emofilia ‘Angelo Bianchi Bonomi’, Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milano, Italy
Search for more papers by this authorC. Négrier
Haematology Department, Director Hemophilia Comprehensive Care Center, Hopital Louis Pradel, Université Lyon 1, Bron Cedex, France
Search for more papers by this authorY. Dargaud,
A. Pavlova,
S. Lacroix-Desmazes,
K. Fischer,
M. Soucie,
S. Claeyssens,
D.w. Scott,
R. d'Oiron,
G. Lavigne-Lissalde,
G. Kenet,
C. Escuriola Ettingshausen,
A. Borel-Derlon,
T. Lambert,
G. Pasta,
C. Négrier,
Corresponding Author
Y. Dargaud
Unite d'Hemostase Clinique, Hopital Cardiologique Louis Pradel, Universite Lyon 1, Lyon, France
Correspondence: Yesim Dargaud, Unite d'Hemostase Clinique, Hopital Cardiologique Louis Pradel, Universite Lyon 1, Lyon 69008 France.
Tel.: +33472118810; fax: +33472118817;
e-mail: [email protected]
Search for more papers by this authorA. Pavlova
Institute of Experimental Haematology and Transfusion Medicine, University Clinic, Bonn, Germany
Search for more papers by this authorS. Lacroix-Desmazes
INSERM UMRS 1138, Immunopathologie et immuno-intervention thérapeutique, Centre de Recherche des Cordeliers, Paris, France
Search for more papers by this authorK. Fischer
Van Creveldkliniek (HP C01.425), University Medical Center Utrecht, Utrecht, The Netherlands
Search for more papers by this authorM. Soucie
Division of Blood Disorders, National Center for Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA
The findings and conclusions in this report are those of the authors and do not necessarily represent the official position of the Centers for Disease Control and Prevention.Search for more papers by this authorS. Claeyssens
Chu Purpan Pav. Centre Hospitalier Lefebvre, Centre Rgal de l'Hemophilie, Toulouse, France
Search for more papers by this authorD.w. Scott
Department of Medicine, Uniformed Services, University for the Health Sciences, Bethesda, MD, USA
Search for more papers by this authorR. d'Oiron
Centre de Traitement de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles, Hôpitaux Universitaires Paris Sud - Site Bicêtre, Le Kremlin-Bicêtre, France
Search for more papers by this authorG. Lavigne-Lissalde
Laboratoire d'Hématologie et Consultations d'Hématologie Biologique Centre Hospitalier Universitaire de Nîmes, Place du Pr R. Debré Nîmes, France
Search for more papers by this authorG. Kenet
National Hemophilia Institute, Sheba Medical Center, Tel-Hashomer, Tel Aviv University, Tel Aviv, Israel
Search for more papers by this authorC. Escuriola Ettingshausen
Hemophilia Centre Rhein-Main HZRM, Frankfurt-Mörfelden, Germany
Search for more papers by this authorA. Borel-Derlon
Haemophilia and von Willebrand Disease Centre, University Hospital of Caen, Caen
Search for more papers by this authorT. Lambert
Hemophilia Care Center, Bicêtre AP-HP Hospital and Faculté de Médecine Paris XI, Paris, France
Search for more papers by this authorG. Pasta
UOSD di Ortopedia e Traumatologia, Centro Emofilia ‘Angelo Bianchi Bonomi’, Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milano, Italy
Search for more papers by this authorC. Négrier
Haematology Department, Director Hemophilia Comprehensive Care Center, Hopital Louis Pradel, Université Lyon 1, Bron Cedex, France
Search for more papers by this authorPublication of this supplement was supported by an unrestricted educational grant from Octapharma.
Summary
Over the past 20 years, there have been many advances in haemophilia treatment that have allowed patients to take greater control of their disease. However, the development of factor VIII (FVIII) inhibitors is the greatest complication of the disease and a challenge in the treatment of haemophilia making management of bleeding episodes difficult and surgical procedures very challenging. A meeting to discuss the unmet needs of haemophilia patients with inhibitors was held in Paris on 20 November 2014. Topics discussed were genetic and non-genetic risk factors for the development of inhibitors, immunological aspects of inhibitor development, FVIII products and inhibitor development, generation and functional properties of engineered antigen-specific T regulatory cells, suppression of immune responses to FVIII, prophylaxis in haemophilia patients with inhibitors, epitope mapping of FVIII inhibitors, current controversies in immune tolerance induction therapy, surgery in haemophilia patients with inhibitors and future perspectives for the treatment of haemophilia patients with inhibitors. A summary of the key points discussed is presented in this paper.
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Citing Literature
January 2016
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