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Interim data on long‐term efficacy, safety and tolerability of a plasma‐derived factor VIII concentrate in 109 patients with severe haemophilia A
Author(s) -
NEMES L.,
POLLMANN H.,
BECKER T.
Publication year - 2012
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/j.1365-2516.2011.02738.x
Subject(s) - medicine , tolerability , haemophilia , interim analysis , haemophilia a , incidence (geometry) , interim , adverse effect , factor ix , surgery , clinical trial , pediatrics , physics , archaeology , optics , history
Summary. 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.