Low-factor consumption for major surgery in haemophilia B with long-acting recombinant glycoPEGylated factor IX

M. A. Escobar, R. Tehranchi, F. A. Karim, U. Caliskan, P. Chowdary, T. Colberg, P. Giangrande, Adam Giermasz, M. E. Mancuso, M. Serban, W. Tsay, J. N. Mahlangu

Research output: Contribution to journalArticle

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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.

Original languageEnglish (US)
Pages (from-to)67-76
Number of pages10
JournalHaemophilia
Volume23
Issue number1
DOIs
StatePublished - Jan 1 2017

Fingerprint

Hemophilia B
Factor IX
Hemostatics
Safety
Injections
nonacog beta pegol
Intraoperative Complications
Ambulatory Surgical Procedures
Wound Healing
Research Personnel
Hemorrhage

Keywords

  • factor IX
  • haemophilia B
  • long-acting recombinant factor IX
  • nonacog beta pegol
  • Phase III
  • surgery

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

Escobar, M. A., Tehranchi, R., Karim, F. A., Caliskan, U., Chowdary, P., Colberg, T., ... Mahlangu, J. N. (2017). Low-factor consumption for major surgery in haemophilia B with long-acting recombinant glycoPEGylated factor IX. Haemophilia, 23(1), 67-76. https://doi.org/10.1111/hae.13041

Low-factor consumption for major surgery in haemophilia B with long-acting recombinant glycoPEGylated factor IX. / Escobar, M. A.; Tehranchi, R.; Karim, F. A.; Caliskan, U.; Chowdary, P.; Colberg, T.; Giangrande, P.; Giermasz, Adam; Mancuso, M. E.; Serban, M.; Tsay, W.; Mahlangu, J. N.

In: Haemophilia, Vol. 23, No. 1, 01.01.2017, p. 67-76.

Research output: Contribution to journalArticle

Escobar, MA, Tehranchi, R, Karim, FA, Caliskan, U, Chowdary, P, Colberg, T, Giangrande, P, Giermasz, A, Mancuso, ME, Serban, M, Tsay, W & Mahlangu, JN 2017, 'Low-factor consumption for major surgery in haemophilia B with long-acting recombinant glycoPEGylated factor IX', Haemophilia, vol. 23, no. 1, pp. 67-76. https://doi.org/10.1111/hae.13041
Escobar MA, Tehranchi R, Karim FA, Caliskan U, Chowdary P, Colberg T et al. Low-factor consumption for major surgery in haemophilia B with long-acting recombinant glycoPEGylated factor IX. Haemophilia. 2017 Jan 1;23(1):67-76. https://doi.org/10.1111/hae.13041
Escobar, M. A. ; Tehranchi, R. ; Karim, F. A. ; Caliskan, U. ; Chowdary, P. ; Colberg, T. ; Giangrande, P. ; Giermasz, Adam ; Mancuso, M. E. ; Serban, M. ; Tsay, W. ; Mahlangu, J. N. / Low-factor consumption for major surgery in haemophilia B with long-acting recombinant glycoPEGylated factor IX. In: Haemophilia. 2017 ; Vol. 23, No. 1. pp. 67-76.
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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.",
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AU - Tehranchi, R.

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AU - Caliskan, U.

AU - Chowdary, P.

AU - Colberg, T.

AU - Giangrande, P.

AU - Giermasz, Adam

AU - Mancuso, M. E.

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AU - Tsay, W.

AU - Mahlangu, J. N.

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N2 - 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.

AB - 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.

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