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Single 270 μg kg −1 ‐dose rFVIIa vs. standard 90 μg kg −1 ‐dose rFVIIa and APCC for home treatment of joint bleeds in haemophilia patients with inhibitors: a randomized comparison
Author(s) -
YOUNG G.,
SHAFER F. E.,
ROJAS P.,
SEREMETIS S.
Publication year - 2008
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.1365-2516.2007.01601.x
Subject(s) - medicine , haemophilia , recombinant factor viia , haemophilia a , placebo , anesthesia , haemophilia b , surgery , alternative medicine , pathology
Summary. Evidence suggests greater doses of recombinant activated factor VII (rFVIIa; NovoSeven ® , Novo Nordisk A/S, Bagsværd, Denmark) than currently administered may result in enhanced haemostasis and convenience for patients with haemophilia A and B with inhibitors. This study evaluated efficacy and safety of rFVIIa and an activated prothrombin complex concentrate (APCC; Factor Eight Inhibitor Bypassing Activity [FEIBA] ® , Baxter AG, Vienna, Austria) for controlling joint bleeds in a home‐treatment setting. Patients received each of three treatments in one of six possible sequences: 270 μg kg −1 rFVIIa at hour 0 + placebo at hours 3 and 6, 90 μg kg −1 rFVIIa at hours 0, 3 and 6, and 75 U kg −1 APCC at hour 0. Efficacy was assessed by the requirement for additional haemostatics within 9 h and by a novel global response algorithm. The percentage of rFVIIa 270 μg kg −1 group patients requiring additional haemostatics within 9 h (8.3%) was significantly lower than that for the APCC group (36.4%, P = 0.032). The percentage of rFVIIa 90 × 3 μg kg −1 group patients requiring such rescue medication (9.1%) was also lower compared to the APCC group. This result approached, but did not reach statistical significance ( P = 0.069). Both rFVIIa treatment groups showed similar use of rescue medication (8.3% and 9.1% of episodes for rFVIIa 270 μg kg −1 and rFVIIa 90 × 3 μg kg −1 groups respectively). No significant differences in treatment response were observed with the global response algorithm ( P = 0.173). No safety issues were identified. A single dose of rFVIIa 270 μg kg −1 is as safe and effective as rFVIIa 90 × 3 μg kg −1 dosing, and may be considered a potentially more effective alternative to APCCs for the management of joint bleeding in this population.