Abstract Number: PB0905
Meeting: ISTH 2020 Congress
Background: The Canadian Blood Disorders Registry (CBDR) captures data collected from hemophilia treatment centers and directly from patients. With the recent availability of nonacog beta pegol (N9-GP) in Canada, the CBDR provides a unique opportunity.
Aims: To assess real-world outcomes following N9-GP use in a clinical setting.
Methods: This non-interventional, retrospective study used CBDR data to describe and analyze real-world outcomes in Canadian hemophilia B patients receiving N9-GP for ≥3 months in any setting (prophylaxis, on-demand treatment, treatment of breakthrough bleeding [BTB]). For comparison with previously-used products, only patients for whom data existed in CBDR for the 6-month period pre-switch to N9-GP were included.
Results: At the data cut-off (September 30, 2019), 40 patients were included in the analysis, with a median age of 44 years. Distribution of disease severity was 2.5% mild, 40% moderate, 55% severe, and 2.5% unknown. At study start, 10 target joints were present in 5 patients. Most patients had previously received rFIXFc (55% versus 40% rFIX), with most previously receiving prophylactic treatment (85% versus 15% on-demand). No patients had present or previous inhibitor development.
During a median treatment period of 11.11 months on N9-GP, 106 BTBs were reported in 22 patients; 42% of patients reported zero bleeds. Median time from last recorded prophylactic injection to start of bleeding was 7.1 days and the mean number of injections required to treat a bleed was 1.23.
Figure 1 shows the comparative effectiveness of N9-GP prophylaxis versus previous product by type of bleed and previous product.
Table 1 shows comparative annualized factor consumption data.
|Total annualized consumption [kIU/year]*||Number of patients||Mean||Median||Min, Max||Number of patients||Mean||Median||Min, Max|
|For prophylaxis||32||228.9||185.0||36.0, 684.0||38||150.5||141.9||51.1, 308.8|
|For treatment of BTBs||19||64.9||48.0||8.0, 48.0||22||23.3||9.3||1.7, 107.4|
|For on-demand treatment||6||62.3||43.0||8.0, 200.0||2|
|BTBs, breakthrough bleeds. *Annualized consumption estimate for each patient based on the data available (6 months for pre-N9-GP); Number of patients where information is available; Insufficient data.|
[Table 1. Comparative annualized factor consumption for N9-GP versus previous product]
Conclusions: This is the first study to investigate treatment patterns and clinical outcomes with N9-GP in hemophilia B patients in a real-world setting. Initial data suggest improved bleeding outcomes with lower factor consumption after switching to N9-GP, regardless of whether patients previously received standard or extended half-life products.
To cite this abstract in AMA style:Matino D, Iorio A, Keepanasseril A, Germini F, Birkegård AC, Caillaud A, Carcao M, Hews-Girard J, Iserman E, James P, Lee A, Phua C, Sun H, Teitel J, Poon M-. A Canadian, Multi-Center, Retrospective, Non-Interventional Study of Clinical Outcomes from Early Use of N9-GP Compared with Previous Treatment in Patients with Hemophilia B in a Real-World Setting [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/a-canadian-multi-center-retrospective-non-interventional-study-of-clinical-outcomes-from-early-use-of-n9-gp-compared-with-previous-treatment-in-patients-with-hemophilia-b-in-a-real-world-setting/. Accessed October 26, 2020.
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