Turoctocog alfa pegol provides effective management for major and minor surgical procedures in patients across all age groups with severe haemophilia A: Full data set from the pathfinder 3 and 5 phase III trials
Open Access
- 1 May 2020
- journal article
- research article
- Published by Wiley in Haemophilia
- Vol. 26 (3), 450-458
- https://doi.org/10.1111/hae.13980
Abstract
Introduction Turoctocog alfa pegol is a glycoPEGylated recombinant factor VIII (FVIII) with an extended half-life developed for prophylaxis, treatment of bleeds and perioperative management in patients with haemophilia A. Aim Evaluate the efficacy and safety of turoctocog alfa pegol treatment for major and minor surgeries in the pathfinder 3 and 5 phase III trials. Methods Adults/adolescents aged >= 12 years with severe haemophilia A (FVIII 80% during major surgery (pathfinder 3). The primary end point was haemostatic efficacy during surgery; secondary end points were blood loss, haemostatic effect postsurgery, consumption, transfusions, safety and health economics. Children (0-11 years) undergoing minor surgeries received 20-75 IU/kg turoctocog alfa pegol at Investigator's discretion (pathfinder 5). Results pathfinder 3 included 35 patients undergoing 49 major surgeries. Haemostasis was successful in 47/49 (95.9%) surgeries; two had moderate haemostatic responses. Median (mean) blood loss during major surgery was 75 (322.6) mL. Four bleeds were reported postsurgery; three were successfully treated with turoctocog alfa pegol (one was not evaluated). On the day of surgery, overall mean (median) dose was 75.5 (74.5) IU/kg and mean (median) number of doses was 1.7 (2.0). Five procedures required 11 transfusions on the day of surgery or days 1-6. No safety concerns or inhibitors were identified. Forty-five minor surgeries in 23 children were performed without complications. Conclusion Turoctocog alfa pegol was effective for perioperative haemostatic management of major and minor surgeries in patients across age groups with severe haemophilia A.Funding Information
- Novo Nordisk A/S
This publication has 22 references indexed in Scilit:
- Long-acting recombinant factor VIII Fc fusion protein (rFVIIIFc) for perioperative haemostatic management in severe haemophilia AThrombosis and Haemostasis, 2016
- Surgery in patients with inherited bleeding disordersAnaesthesia, 2014
- World Medical Association Declaration of HelsinkiJAMA, 2013
- Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia AJournal of Thrombosis and Haemostasis, 2013
- Perioperative clotting factor replacement and infection in total knee arthroplastyHaemophilia, 2011
- Surgery and inhibitor development in hemophilia A: a systematic reviewJournal of Thrombosis and Haemostasis, 2011
- BDDrFVIII (Moroctocog alfa [AF-CC]) for surgical haemostasis in patients with haemophilia A: results of a pivotal studyHaemophilia, 2010
- Total knee replacement in patients with end-stage haemophilic arthropathyThe Journal of Bone and Joint Surgery. British volume, 2010
- Surgical evaluation of a recombinant factor VIII prepared using a plasma/albumin-free method: Efficacy and safety of Advate in previously treated patientsThrombosis and Haemostasis, 2008
- Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin‐free method: pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A1Haemophilia, 2004