title: Interim data on long-term efficacy, safety and tolerability of a plasma-derived factor VIII concentrate in 109 patients with severe haemophilia A.
abstract: The aim of this open-label, multicentre and multinational post-marketing surveillance was to investigate clinical effectiveness, safety and tolerability of a plasma-derived and vWF containing factor VIII product (FVIII/VWF) in patients with severe haemophilia A. Long-term effectiveness, safety and tolerability were investigated in a total of 109 haemophilia A patients treated for prophylaxis or on-demand, as required. Interim data collected until June 2010 are presented. Most patients (99/109; 90.8%) were previously treated patients (PTPs). Mean observation period was 82.6 months. Overall, patients received 105 131 425 IU haemoctin SDH during 68 624 administrations. Each patient was given a mean of 635.4 injections, whereby about half of the administrations were given for treatment of bleeding episodes (46.9%) and the other administrations for prophylactic reasons (53.1%). Patients on prophylaxis had a median of 0.8 bleeding episodes per month. The expected therapeutic effect was reached in 99.3% of treatments. The incidence of clinically relevant inhibitor formation in patients with severe haemophilia (FVIII activity ≤ 1%) was 1.2% for PTPs. One previously untreated patient with severe haemophilia had a clinically relevant transient inhibitor. No treatment related transmissions of hepatitis A, B and C and HIV 1/2 were observed. German patients had a higher extent of exposure and experienced less bleeding episodes than Hungarian patients. In conclusion, haemoctin SDH was effective, safe and well tolerated in long-term prophylaxis and treatment on demand.
authors.affiliation: National Haemophilia Centre and Haemostasis Department, State Health Centre, Budapest, Hungary.
authors.forename: L
authors.initials: L
authors.lastname: Nemes
authors.forename: H
authors.initials: H
authors.lastname: Pollmann
authors.forename: T
authors.initials: T
authors.lastname: Becker
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002648:Child; D002675:Child, Preschool; D003029:Coagulants / Q000009:adverse effects / Q000627:therapeutic use*; D005169:Factor VIII / Q000009:adverse effects / Q000378:metabolism / Q000627:therapeutic use*; D006467:Hemophilia A / Q000188:drug therapy* / Q000378:metabolism; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D055815:Young Adult
chemical_list: D003029:Coagulants; D005169:Factor VIII