title: Achievements, challenges and unmet needs for haemophilia patients with inhibitors: Report from a symposium in Paris, France on 20 November 2014.
abstract: 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.
authors.affiliation: Unite d'Hemostase Clinique, Hopital Cardiologique Louis Pradel, Universite Lyon 1, Lyon, France.
authors.forename: Y
authors.initials: Y
authors.lastname: Dargaud
authors.affiliation: Institute of Experimental Haematology and Transfusion Medicine, University Clinic, Bonn, Germany.
authors.forename: A
authors.initials: A
authors.lastname: Pavlova
authors.affiliation: INSERM UMRS 1138, Immunopathologie et immuno-intervention thérapeutique, Centre de Recherche des Cordeliers, Paris, France.
authors.forename: S
authors.initials: S
authors.lastname: Lacroix-Desmazes
authors.affiliation: Van Creveldkliniek (HP C01.425), University Medical Center Utrecht, Utrecht, The Netherlands.
authors.forename: K
authors.initials: K
authors.lastname: Fischer
authors.affiliation: Division of Blood Disorders, National Center for Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA.
authors.forename: M
authors.initials: M
authors.lastname: Soucie
authors.affiliation: Chu Purpan Pav. Centre Hospitalier Lefebvre, Centre Rgal de l'Hemophilie, Toulouse, France.
authors.forename: S
authors.initials: S
authors.lastname: Claeyssens
authors.affiliation: Department of Medicine, Uniformed Services, University for the Health Sciences, Bethesda, MD, USA.
authors.forename: D W
authors.initials: DW
authors.lastname: Scott
authors.affiliation: Centre de Traitement de l'Hémophilie et des Maladies Hémorragiques Constitutionnelles, Hôpitaux Universitaires Paris Sud - Site Bicêtre, Le Kremlin-Bicêtre, France.
authors.forename: R
authors.initials: R
authors.lastname: d'Oiron
authors.affiliation: Laboratoire d'Hématologie et Consultations d'Hématologie Biologique Centre Hospitalier Universitaire de Nîmes, Place du Pr R. Debré Nîmes, France.
authors.forename: G
authors.initials: G
authors.lastname: Lavigne-Lissalde
authors.affiliation: National Hemophilia Institute, Sheba Medical Center, Tel-Hashomer, Tel Aviv University, Tel Aviv, Israel.
authors.forename: G
authors.initials: G
authors.lastname: Kenet
authors.affiliation: Hemophilia Centre Rhein-Main HZRM, Frankfurt-Mörfelden, Germany.
authors.forename: C
authors.initials: C
authors.lastname: Escuriola Ettingshausen
authors.affiliation: Haemophilia and von Willebrand Disease Centre, University Hospital of Caen, Caen.
authors.forename: A
authors.initials: A
authors.lastname: Borel-Derlon
authors.affiliation: Hemophilia Care Center, Bicêtre AP-HP Hospital and Faculté de Médecine Paris XI, Paris, France.
authors.forename: T
authors.initials: T
authors.lastname: Lambert
authors.affiliation: UOSD di Ortopedia e Traumatologia, Centro Emofilia 'Angelo Bianchi Bonomi', Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milano, Italy.
authors.forename: G
authors.initials: G
authors.lastname: Pasta
authors.affiliation: Haematology Department, Director Hemophilia Comprehensive Care Center, Hopital Louis Pradel, Université Lyon 1, Bron Cedex, France.
authors.forename: C
authors.initials: C
authors.lastname: Négrier
mesh_terms: D000906:Antibodies / Q000276:immunology*; D018604:Epitope Mapping; D005169:Factor VIII / Q000235:genetics / Q000276:immunology* / Q000627:therapeutic use*; D006467:Hemophilia A / Q000235:genetics / Q000276:immunology* / Q000601:surgery / Q000628:therapy*; D006801:Humans; D007108:Immune Tolerance; D007111:Immunity, Cellular; D010297:Paris
chemical_list: D000906:Antibodies; D005169:Factor VIII
keywords: FVIII products; genetics; haemophilia; inhibitors; prophylaxis; surgery