BACKGROUND: Hemophilia A is an X-linked bleeding disorder that results from insufficient levels of factor VIII (FVIII) coagulant activity. OBJECTIVE: To evaluate the efficacy and safety of ADVATE rAHF-PFM (Baxter Healthcare Corporation), a recombinant FVIII concentrate manufactured without human or bovine blood-derived additives, and to assess the effect of compliance with prophylactic use in preventing bleeding episodes (BEs). METHODS: Clinical data were integrated from six prospective studies. Two hundred thirty-four hemophilia A subjects (FVIII levels < or = 2%) (median age 14.7 (range: 0.02 - 72.7) years) were included. RESULTS: BEs were managed with one or two infusions and nearly all (1953/1956) responded to treatment. Compliance with a prophylactic treatment regimen significantly reduced the incidence of BEs (p = 0.0061) and prevented non-traumatic joint BEs (median annualized BE rate was 0). One previously treated subject developed an inhibitor; no other safety concerns were observed. CONCLUSIONS: These results reinforce the efficacy and safety of rAHF-PFM and suggest that compliance is an essential contributor to the effectiveness of prophylaxis in the treatment of hemophilia A.
BACKGROUND:Hemophilia A is an X-linked bleeding disorder that results from insufficient levels of factor VIII (FVIII) coagulant activity. OBJECTIVE: To evaluate the efficacy and safety of ADVATE rAHF-PFM (Baxter Healthcare Corporation), a recombinant FVIII concentrate manufactured without human or bovine blood-derived additives, and to assess the effect of compliance with prophylactic use in preventing bleeding episodes (BEs). METHODS: Clinical data were integrated from six prospective studies. Two hundred thirty-four hemophilia A subjects (FVIII levels < or = 2%) (median age 14.7 (range: 0.02 - 72.7) years) were included. RESULTS:BEs were managed with one or two infusions and nearly all (1953/1956) responded to treatment. Compliance with a prophylactic treatment regimen significantly reduced the incidence of BEs (p = 0.0061) and prevented non-traumatic joint BEs (median annualized BE rate was 0). One previously treated subject developed an inhibitor; no other safety concerns were observed. CONCLUSIONS: These results reinforce the efficacy and safety of rAHF-PFM and suggest that compliance is an essential contributor to the effectiveness of prophylaxis in the treatment of hemophilia A.
Authors: Barbara A Konkle; Oleksandra Stasyshyn; Pratima Chowdary; David H Bevan; Tim Mant; Midori Shima; Werner Engl; Jacqueline Dyck-Jones; Monika Fuerlinger; Lisa Patrone; Bruce Ewenstein; Brigitt Abbuehl Journal: Blood Date: 2015-07-08 Impact factor: 22.113
Authors: L A Valentino; V Mamonov; A Hellmann; D V Quon; A Chybicka; P Schroth; L Patrone; W-Y Wong Journal: J Thromb Haemost Date: 2012-03 Impact factor: 5.824
Authors: S V Antunes; S Tangada; O Stasyshyn; V Mamonov; J Phillips; N Guzman-Becerra; A Grigorian; B Ewenstein; W-Y Wong Journal: Haemophilia Date: 2013-08-01 Impact factor: 4.287