1.
Evaluation of two secondary prophylaxis regimens of recombinant factor IX (r-IX) in moderately severe to severe (FIX <=2%) hemophilia B patients
Rendo P, Barrette-Grischow M-K, Smith L, Korth-Bradley JM, Charnigo R, Shafer FE
Blood. 2012;120((21):): Abstract No. 4628.
2.
Single 270 microg kg(-1)-dose rFVIIa vs. standard 90 microg kg(-1)-dose rFVIIa and APCC for home treatment of joint bleeds in haemophilia patients with inhibitors: a randomized comparison
Young G, Shafer FE, Rojas P, Seremetis S
Haemophilia. 2008;14((2):):287-94.
Abstract
Evidence suggests greater doses of recombinant activated factor VII (rFVIIa; NovoSeven, Novo Nordisk A/S, Bagsvaerd, 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 microg kg(-1) rFVIIa at hour 0 + placebo at hours 3 and 6, 90 microg 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 microg 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 x 3 microg 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 microg kg(-1) and rFVIIa 90 x 3 microg 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 microg kg(-1) is as safe and effective as rFVIIa 90 x 3 microg kg(-1) dosing, and may be considered a potentially more effective alternative to APCCs for the management of joint bleeding in this population.