title: The evolution of comprehensive haemophilia care in the United States: perspectives from the frontline.
abstract: The establishment of dedicated comprehensive treatment centres more than a half century ago transformed the management of haemophilia in the United States. Formerly, a disease associated with crippling disability and premature death, today, persons with haemophilia who are treated appropriately from infancy and do not develop inhibitors can expect a normal life expectancy and relatively few bleeding episodes. The evolution of the comprehensive haemophilia care, while chastened by the viral epidemics of the 1980s, has been marked by ongoing advances, including prophylaxis, immune tolerance induction, new drugs and gene therapy research. Current challenges include sustaining the comprehensive care model despite decreased funding and expanding the delivery and affordability of comprehensive haemophilia care.
authors.affiliation: Mount Sinai School of Medicine, New York, NY, USA. louis.aledort@mountsinai.org.
authors.forename: L M
authors.initials: LM
authors.lastname: Aledort
mesh_terms: D001779:Blood Coagulation Factors / Q000235:genetics / Q000378:metabolism / Q000627:therapeutic use; D003191:Comprehensive Health Care / Q000592:standards*; D003695:Delivery of Health Care / Q000458:organization & administration; D015316:Genetic Therapy; D006467:Hemophilia A / Q000175:diagnosis* / Q000628:therapy; D002836:Hemophilia B / Q000175:diagnosis* / Q000628:therapy; D006801:Humans; D015202:Protein Engineering; D011994:Recombinant Proteins / Q000627:therapeutic use; D014481:United States
chemical_list: D001779:Blood Coagulation Factors; D011994:Recombinant Proteins
keywords: clotting factor concentrate; comprehensive care; haemophilia; haemophilia treatment centres; inhibitors