B Brand1, R Gruppo2, T T Wynn3, L Griskevicius4, M F Lopez Fernandez5, M Chapman6, T Dvorak7, B G Pavlova7, B E Abbuehl7. 1. Universitaetsspital Zuerich, Zuerich, Switzerland. 2. Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. 3. College of Medicine, University of Florida, Gainesville, FL, USA. 4. Vilnius University Hospital Santariskiu Klinikos, Medical Faculty of Vilnius University, Vilnius, Lithuania. 5. Complejo Hospitalario Universitario A Coruña, A Coruña, Spain. 6. Baxalta US Inc., Cambridge, MA, USA. 7. Baxalta Innovations GmbH, Vienna, Austria.
Abstract
INTRODUCTION: 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. AIM: 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. CONCLUSION: These results demonstrate that BAX 855 is safe and haemostatically effective in patients with severe haemophilia A undergoing surgery.
INTRODUCTION: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. AIM: 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. CONCLUSION: These results demonstrate that BAX 855 is safe and haemostatically effective in patients with severe haemophilia A undergoing surgery.
Authors: Pierre Chelle; Cindy H T Yeung; Stacy E Croteau; Jennifer Lissick; Vinod Balasa; Christina Ashburner; Young Shil Park; Santiago Bonanad; Juan Eduardo Megías-Vericat; Azusa Nagao; Tung Wynn; Fernando Corrales-Medina; Huyen Tran; Anjali Sharathkumar; Meera Chitlur; Samuel Sarmiento; Andrea Edginton; Alfonso Iorio Journal: Clin Pharmacokinet Date: 2020-02 Impact factor: 6.447