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.
3.
Single dose of anti-D immune globulin at 75 microg/kg is as effective as intravenous immune globulin at rapidly raising the platelet count in newly diagnosed immune thrombocytopenic purpura in children
Tarantino MD, Young G, Bertolone SJ, Kalinyak KA, Shafer FE, Kulkarni R, Weber LC, Davis ML, Lynn H, Nugent DJ, et al
The Journal of Pediatrics. 2006;148((4):):489-94.
Abstract
OBJECTIVE To conduct a randomized prospective trial of immune globulin treatment for 105 Rh+ children with newly-diagnosed immune thrombocytopenic purpura and a platelet count<20,000/microL, to determine whether anti-D immune globulin (anti-D) is as effective as intravenous immune globulin (IVIg). STUDY DESIGN Eligible patients received either a single intravenous dose of 50 microg/kg anti-D (anti-D50), 75 microg/kg anti-D, (anti-D75), or 0. 8 g/kg IVIg, (IVIg). Patients were monitored for response to treatment and adverse events. RESULTS By 24 hours after treatment 50%, 72%, and 77% of patients in the anti-D50, anti-D75, and IVIg groups, respectively, had achieved a platelet count>20,000/microL (P=. 03). By day 7, hemoglobin concentrations decreased by 1. 6 g/dL, 2 g/dL, and 0. 3 g/dL in the anti-D50, anti-D75, and IVIg groups, respectively. Headache, fever, or chills occurred least often in the anti-D50 group. CONCLUSIONS A single 75 microg/kg dose of Anti-D raised the platelet count in children with newly diagnosed immune thrombocytopenic purpura more rapidly than standard-dose anti-D and as effectively as IVIg, with an acceptable safety profile.
4.
High-dose anti-D immune globulin raises the platelet count in newly diagnosed childhood ITP more rapidly than standard-dose anti-D and higher than IVIg
Tarantino MD, Young G, Bertolone SJ, Kalinyak K, Shafer FE, Kulkarni R, Weber L, Nugent D
Pediatric Research. 2003;: Abstract No. LB3-15.
5.
High-dose anti-D immune globulin is as effective as IVIg in the treatment of newly diagnosed immune thrombocytopenic purpura (ITP) in children
Tarantino MD, Young G, Bertolone SJ, Shafer FE, Kulkarni R, Kalinyak K,, et al.,
Blood. 2003;102((11, Pt 2):):299a-300a.. Abstract No. 1072.