title: Safety surveillance in haemophilia and allied disorders.
abstract: The introduction of clotting factor concentrates has transformed the lives of persons with inherited bleeding disorders. With the use of prophylactic treatment, it is now possible to prevent bleeding in these individuals. The early concentrates were contaminated with the HIV and hepatitis C viruses (HCV) and resulted in major morbidity and mortality in the recipients. Current products are much safer, especially in terms of infectious agents, but other adverse events such as alloantibodies (inhibitors), allergic reactions and thrombotic risks remain of concern. Approximately 30% of previously untreated patients with severe haemophilia A develop inhibitors, making this the most important issue in haemophilia care today. Recently, it was suggested that one of the most commonly used concentrates was associated with a higher inhibitor risk, but this was not supported by the evidence from all studies. Good safety surveillance systems are essential for all diseases and products but are particularly so in the group of individuals with inherited bleeding disorders treated with clotting factor concentrates who have suffered disproportionately from the adverse effects of their treatment. National and multinational systems are now in place to allow reporting of adverse events in patients with inherited bleeding disorders. All clinicians treating individuals with inherited bleeding disorders should prospectively report adverse events to treatment even if they are believed to be common and well recognized.
authors.affiliation: Helsinki University Hospital, Coagulation Disorders, Hematology and Comprehensive Cancer Center, University of Helsinki, Helsinki, Finland.
authors.forename: R
authors.initials: R
authors.lastname: Lassila
authors.affiliation: Department of Cardiovascular Science, Medical School, University of Sheffield, Sheffield, UK.|Sheffield Haemophilia and Thrombosis Centre, Royal Hallamshire Hospital, Sheffield, UK.
authors.forename: M
authors.initials: M
authors.lastname: Makris
mesh_terms: D001779:Blood Coagulation Factors / Q000009:adverse effects* / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy*; D002836:Hemophilia B / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D060735:Pharmacovigilance; D011446:Prospective Studies; D012042:Registries
chemical_list: D001779:Blood Coagulation Factors
keywords: clotting factor concentrate; haemophilia; pharmacovigilance; safety surveillance