Abstract Number: PB1115
Meeting: ISTH 2020 Congress
Background: Surgery in patients with hemophilia B carries a high risk of excessive bleeding and requires adequate hemostatic control for extended periods of time. Nonacog beta pegol (N9-GP) is a recombinant FIX molecule and a 40-kilodalton polyethylene glycol (PEG) moiety which is attached to the FIX activation peptide enables to generate native-activated FIX. A single-dose PK trial have shown a five-fold increase in terminal half-life as compared with standard FIX products. Development of N9-GP allowed for not only regular once-weekly prophylaxis but also reduced interval of intra/postoperative infusion in hemophilia B patients.
Aims: We report the experiences of major/minor surgeries in 4 hemophilia B patients and show the reduced consumption FIX concentrates to achieve good hemostasis.
Methods: Four male patients with hemophilia B who were scheduled for surgery were included (Table). There were no history of FIX inhibitors. The selection of the perioperative dosing regimen was based on PK modelling of data obtained in the first human dose trial where a single dose of N9-GP had been injected in a non-bleeding state (Collins, et al., 2012). All patients received total dose from 102.4 to 188.7 IU/kg of N9-GP throughout the perioperative periods of which every dosing was a single bolus injection.
Results: Intraoperative hemostasis was rated as ‘excellent’ as was defined previously (Escobar, et al., 2017). No transfusions were required and all patients discharged safely from the hospital and did not need surgery-related FIX replacement. No patients developed FIX inhibitors and no thromboembolic events occurred. In case 1, total cost of replacement therapy for left knee arthroplasty by using N9-GP was 1/3 of that for the previous right knee arthroplasty by using continuous infusion of a recombinant FIX.
|Case 1||Case 2||Case 3||Case 4|
|Age and weight on surgery||57 and 75kg||1y3m and 8.6kg||1y1m and 9.8kg||68 and 47.7kg|
|History of treatment||rFIX, on demand||rFIX, on demand→ rFIX-FC, qw PPX||PUPS||pdFIX, bw PPX→rFIX, bw PPX→rFIX-FC, qw PPX→rFIX-FP, qw PPX|
|Surgery||Left knee arthroplasty||Port insertion||Port insertion||Release of diaphragmatic hernia incarceration + hernia repair|
|Surgery time (min)||166||43||64||110|
|Bleeding volume (ml)||11||3||Small amount||130|
|Total number of N9-GP infusions||3||2||2||4|
|Total infused units||10000 IU (131.93 IU / kg)||1000 IU (116.55 IU / kg)||1000 IU (102.4 IU / kg)||9000 IU (188.68IU / kg)|
[Patient demographics and results]
Conclusions: Compared with standard FIX products, N9-GP was safe and effective as replacement therapy to manage surgeries in our patients and effectively reduced the consumption of FIX concentrates.
To cite this abstract in AMA style:Okamoto S, Matsushita T, Kanematsu T, Suzuki A, Tamura S, Kojima T, Suzuki N. Low-Factor Consumption and Cost Effectiveness for Surgeries in Hemophilia B Patients with N9-GP [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/low-factor-consumption-and-cost-effectiveness-for-surgeries-in-hemophilia-b-patients-with-n9-gp/. Accessed January 28, 2022.
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