M A Escobar1, R Tehranchi2, F A Karim3, U Caliskan4, P Chowdary5, T Colberg2, P Giangrande6, A Giermasz7, M E Mancuso8, M Serban9, W Tsay10, J N Mahlangu11. 1. University of Texas Health Science Center and the Gulf States Hemophilia and Thrombophilia Center, Houston, TX, USA. 2. Medical and Science, Haemophilia R&D Portfolio, Novo Nordisk A/S, Søborg, Denmark. 3. National Blood Centre, Kuala Lumpur, Malaysia. 4. Department of Pediatric Hematology, NEU Meram Faculty of Medicine, Konya, Turkey. 5. Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free Hospital, London, UK. 6. Oxford Haemophilia Centre and Thrombosis Unit, Churchill Hospital, Oxford, UK. 7. Division of Hematology Oncology, University of California, San Francisco, CA, USA. 8. Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy. 9. Spitalul Clinic de Urgenta pentru Copii Louis Turcanu, IIIrd Paediatric Clinic, Timisoara, Romania. 10. Department of Hematology, National Taiwan University Hospital, Taipei, Taiwan. 11. Department of Molecular Medicine and Haematology, Faculty of Health Sciences, University of the Witwatersrand, NHLS and Haemophilia Comprehensive Care Centre, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa.
Abstract
INTRODUCTION: Surgery in patients with haemophilia B carries a high risk of excessive bleeding and requires adequate haemostatic control until wound healing. Nonacog beta pegol, a long-acting recombinant glycoPEGylated factor IX (FIX), was used in the perioperative management of patients undergoing major surgery. AIM: To evaluate the efficacy and safety of nonacog beta pegol in patients with haemophilia B who undergo major surgery. METHODS: This was an open-label, multicentre, non-controlled surgery trial aimed at assessing peri- and postoperative efficacy and safety of nonacog beta pegol in 13 previously treated patients with haemophilia B. All patients received a preoperative nonacog beta pegol bolus injection of 80 IU kg-1 . Postoperatively, the patients received fixed nonacog beta pegol doses of 40 IU kg-1 , repeated at the investigator's discretion. Safety assessments included monitoring of immunogenicity and adverse events. RESULTS: Intraoperative haemostatic effect was rated 'excellent' or 'good' in all 13 cases. Apart from the preoperative injection, none of the patients needed additional doses of nonacog beta pegol on the day of surgery. The median number of postoperative doses of nonacog beta pegol was 2.0 from days 1 to 6 and 1.5 from days 7 to 13. No unexpected intra- or postoperative complications were observed including deaths or thromboembolic events. No patients developed inhibitors. CONCLUSIONS: These results indicated that nonacog beta pegol was safe and effective in the perioperative setting, allowing major surgical interventions in patients with haemophilia B with minimal peri- and postoperative concentrate consumption and infrequent injections as reported with standard FIX products.
INTRODUCTION: Surgery in patients with haemophilia B carries a high risk of excessive bleeding and requires adequate haemostatic control until wound healing. Nonacog beta pegol, a long-acting recombinant glycoPEGylated factor IX (FIX), was used in the perioperative management of patients undergoing major surgery. AIM: To evaluate the efficacy and safety of nonacog beta pegol in patients with haemophilia B who undergo major surgery. METHODS: This was an open-label, multicentre, non-controlled surgery trial aimed at assessing peri- and postoperative efficacy and safety of nonacog beta pegol in 13 previously treated patients with haemophilia B. All patients received a preoperative nonacog beta pegol bolus injection of 80 IU kg-1 . Postoperatively, the patients received fixed nonacog beta pegol doses of 40 IU kg-1 , repeated at the investigator's discretion. Safety assessments included monitoring of immunogenicity and adverse events. RESULTS: Intraoperative haemostatic effect was rated 'excellent' or 'good' in all 13 cases. Apart from the preoperative injection, none of the patients needed additional doses of nonacog beta pegol on the day of surgery. The median number of postoperative doses of nonacog beta pegol was 2.0 from days 1 to 6 and 1.5 from days 7 to 13. No unexpected intra- or postoperative complications were observed including deaths or thromboembolic events. No patients developed inhibitors. CONCLUSIONS: These results indicated that nonacog beta pegol was safe and effective in the perioperative setting, allowing major surgical interventions in patients with haemophilia B with minimal peri- and postoperative concentrate consumption and infrequent injections as reported with standard FIX products.
Authors: Mindy L Simpson; Roshni Kulkarni; Carmen Escuriola Ettingshausen; Rikke Medom Meldgaard; David L Cooper; Robert Klamroth Journal: J Blood Med Date: 2019-11-13
Authors: Davide Matino; Alfonso Iorio; Arun Keepanasseril; Federico Germini; Alexandre Caillaud; Manuel Carcao; Julia Hews-Girard; Emma Iserman; Paula James; Adrienne Lee; Chai W Phua; Haowei Linda Sun; Jerome Teitel; Man-Chiu Poon Journal: Res Pract Thromb Haemost Date: 2022-03-31
Authors: Ola Sternebring; Charlotte Gabel-Jensen; Helene Jacobsen; Andrew James Benie; Inga Bjørnsdottir Journal: BioDrugs Date: 2019-12 Impact factor: 5.807