title: Haemophilia patients' unmet needs and their expectations of the new extended half-life factor concentrates.
abstract: BACKGROUND
  National Member Organisations (NMO) of persons with haemophilia (PWH) from the DACH Region (D = Germany, A = Austria, CH = Switzerland) were interested to better understand PWH's expectations and concerns of extended half-life (EHL) factor concentrates (FC) before availability in these countries.
  
  
  METHODS
  Based on an expert meeting and focus groups conducted across Germany a survey for haemophilia patients and their parents was developed and was sent out to 2,644 PWH.
  
  
  RESULTS
  One thousand and seven questionnaires were sent back (38.1%); 743 adults and 262 parents. Most patients had haemophilia A (84.5%), were severely affected (73.7%), received prophylaxis (57%) and used recombinant FC (60.2%). One-quarter did not know the correct half-life of their FC [HA/FVIII: 26%, HB/FIX: 31.1%]. Four percent were unsatisfied with their current FC, mainly with short half-life of FC and difficult manageability. They expected from new EHL products less frequent injections (55.2%), better efficacy (32.1%) and safety/no side effects (15.7%); 59.5% would be willing to switch to new products if they have a prolonged half-life and the same safety of the current FC. They wish more information about half-life (84.4%), possible side-effects (81.3%) and efficacy (77%) and wanted to receive information about new products from their haemophilia treater (76.3%) and the newsletter of their NMO (74.3%). Significant differences across countries were found.
  
  
  CONCLUSIONS
  The representative survey could show that although PWH were generally satisfied with their current FC, the majority would be willing to switch to EHL products assuming half-life is prolonged and has the same safety of the current FC.
authors.affiliation: Institute of Medical Psychology, University Medical Centre, Hamburg, Germany.
authors.forename: S
authors.identifier: http://orcid.org/0000-0002-5926-0478
authors.initials: S
authors.lastname: von Mackensen
authors.affiliation: German Haemophilia Society (DHG), Hamburg, Germany.
authors.forename: W
authors.initials: W
authors.lastname: Kalnins
authors.affiliation: Swiss Haemophilia Society (SHG), Altstätten, Switzerland.
authors.forename: J
authors.initials: J
authors.lastname: Krucker
authors.affiliation: Austrian Haemophilia Society (ÖHG), Vienna, Austria.
authors.forename: J
authors.initials: J
authors.lastname: Weiss
authors.affiliation: Haemophilia Centre, Medical Clinic II, Institute of Transfusion Medicine, Goethe University Hospital Frankfurt, Frankfurt am Main, Germany.
authors.forename: W
authors.initials: W
authors.lastname: Miesbach
authors.affiliation: University Children's Hospital Zürich, Zürich, Switzerland.|Swiss Hemophilia Network, AltstÃ Tten, Switzerland.
authors.forename: M
authors.initials: M
authors.lastname: Albisetti
authors.affiliation: Haemophilia Treatment Centre, Medical University of Vienna, Vienna, Austria.
authors.forename: I
authors.initials: I
authors.lastname: Pabinger
authors.affiliation: Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn AöR, Bonn, Germany.
authors.forename: J
authors.initials: J
authors.lastname: Oldenburg
mesh_terms: D000328:Adult; D002648:Child; D005164:Factor IX / Q000493:pharmacokinetics* / Q000627:therapeutic use*; D005169:Factor VIII / Q000493:pharmacokinetics* / Q000627:therapeutic use*; D005260:Female; D017144:Focus Groups; D006207:Half-Life; D006467:Hemophilia A / Q000188:drug therapy*; D002836:Hemophilia B / Q000188:drug therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D017060:Patient Satisfaction / Q000706:statistics & numerical data*
chemical_list: D005169:Factor VIII; D005164:Factor IX
keywords: DACH Region; expectations; extended half-life; haemophilia; long-acting factor concentrates; patients’ needs; survey