Efficacy and safety of pegylated full-length recombinant factor VIII with extended half-life for perioperative haemostasis in haemophilia A patients.
BACKGROUND
BAX 855 is a pegylated full-length recombinant factor VIII (rFVIII) with an extended half-life, built on a licensed rFVIII (ADVATE(®) ). BAX 855 demonstrated efficacy and safety in prophylaxis and the treatment of bleeding episodes in previously treated patients (PTPs) with severe haemophilia A.


OBJECTIVE
This phase 3 surgery study evaluates the haemostatic efficacy and safety of BAX 855 for perioperative haemostasis in PTPs with severe haemophilia A undergoing surgery.


METHODS
Elective procedures were prospectively classified as major or minor. The dose and frequency of BAX 855 administered perioperatively were to be guided by each patient's pharmacokinetic profile for major procedures or BAX 855 incremental recovery for minor procedures. Haemostatic efficacy was evaluated using a predefined scale. Blood loss was compared to the expected average and maximum blood loss predicted preoperatively.


RESULTS
A total of 15 male patients (aged 19-52 years) underwent 15 procedures (11 major and four minor). The overall intra- and perioperative haemostatic efficacy of BAX 855 was 'excellent' in all 15 subjects (100%). Postoperatively, evaluated at postoperative Day 1, all treatments were 'excellent' except for one minor (dental) procedure which was rated 'good'. No related adverse events, allergic reactions, thrombotic events, nor signs of immunogenicity in terms of induction of binding antibodies to FVIII, PEG or PEG-VIII, or FVIII inhibitors were observed.


CONCLUSIONS
These results demonstrate that BAX 855 is safe and haemostatically effective in patients with severe haemophilia A undergoing surgery.
Universitaetsspital Zuerich, Zuerich, Switzerland.
B
B
Brand
Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
R
R
Gruppo
College of Medicine, University of Florida, Gainesville, FL, USA.
T T
TT
Wynn
Vilnius University Hospital Santariskiu Klinikos, Medical Faculty of Vilnius University, Vilnius, Lithuania.
L
L
Griskevicius
Complejo Hospitalario Universitario A Coruña, A Coruña, Spain.
M F
MF
Lopez Fernandez
Baxalta US Inc., Cambridge, MA, USA.
M
M
Chapman
Baxalta Innovations GmbH, Vienna, Austria.
T
T
Dvorak
Baxalta Innovations GmbH, Vienna, Austria.
B G
BG
Pavlova
Baxalta Innovations GmbH, Vienna, Austria.
B E
BE
Abbuehl
D000328:Adult; D057134:Antibodies, Neutralizing / Q000097:blood; D003029:Coagulants / Q000737:chemistry / Q000493:pharmacokinetics / Q000627:therapeutic use*; D005169:Factor VIII / Q000235:genetics / Q000378:metabolism / Q000627:therapeutic use*; D006207:Half-Life; D006467:Hemophilia A / Q000097:blood / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control; D006487:Hemostasis; D006801:Humans; D008297:Male; D008875:Middle Aged; D019990:Perioperative Care; D011092:Polyethylene Glycols / Q000737:chemistry; D011994:Recombinant Proteins / Q000096:biosynthesis / Q000737:chemistry / Q000493:pharmacokinetics / Q000627:therapeutic use; D013514:Surgical Procedures, Operative; D016896:Treatment Outcome; D055815:Young Adult
D057134:Antibodies, Neutralizing; D003029:Coagulants; D011994:Recombinant Proteins; D011092:Polyethylene Glycols; D005169:Factor VIII
extended half-life; haemophilia A; pegylated recombinant factor VIII; perioperative haemostasis