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

D. Matino¹, A. Iorio¹, A. Keepanasseril¹, F. Germini², A.C. Birkegård³, A. Caillaud⁴, M. Carcao⁵, J. Hews-Girard⁶, E. Iserman⁷, P. James⁸, A. Lee⁹, C. Phua¹⁰, H. Sun¹¹, J. Teitel¹², M.-C. Poon¹³

¹McMaster University, Department of Medicine, Hamilton, Canada, ²McMaster University, Department of Health Research Methods, Evidence, and Impact, Hamilton, Canada, ³Novo Nordisk A/S, Bagsvaerd, Denmark, ⁴Novo Nordisk Canada Inc., Montreal, Canada, ⁵Hospital for Sick Children, University of Toronto, Department of Pediatrics, Toronto, Canada, ⁶Foothills Medical Centre, Alberta Health Services, Calgary, Canada, ⁷McMaster University, Hamilton, Canada, ⁸Queen's University, Department of Medicine, Kingston, Canada, ⁹University of Calgary, Hematology, Department of Medicine, Calgary, Canada, ¹⁰Western University, Department of Medicine, London, Canada, ¹¹University of Alberta, Department of Medicine, Edmonton, Canada, ¹²St Michael's Hospital, University of Toronto, Toronto, Canada, ¹³Cumming School of Medicine, University of Calgary, Departments of Medicine, Pediatrics and Oncology, Calgary, Canada

Abstract Number: PB0905

Meeting: ISTH 2020 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

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.

	Previous product				N9-GP
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.

[Figure 1. Comparative effectiveness of N9-GP prophylaxis versus previous product by type of bleed and previous product]

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 September 22, 2023.

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