Abstract Number: PB0875
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: In Canada, factor IX (FIX) replacement concentrates for patients with hemophilia B (PwHB) are subject to the national competitive procurement process. In April 2018, the introduction of nonacog beta pegol (N9-GP), in all provinces except Quebec, for once-weekly prophylactic therapy in adult PwHB resulted in a switch of products for many patients on prophylaxis.
Aims: To qualitatively evaluate the disease burden among PwHB and the experiences among patients who have switched to prophylactic N9-GP therapy in Canada.
Methods: A semi-structured telephone interview was conducted between January and September 2019 in adult male PwHB, who contacted the study organizers to participate and were receiving prophylactic N9-GP for ≥3 months. Interview transcripts were analyzed thematically to identify key concepts.
Results: Ten PwHB participated in the interviews, representing approximately 10-15% of PwHB receiving
N9-GP in Canada. The median age was 45 years, and 8/10 patients had severe hemophilia. The median annual number of on-demand infusions decreased from 10 to 1.5 infusions following switch to N9-GP (Table). All patients described previous hemarthroses and impaired quality of life (QOL) during their lifetime, including impacts on physical function, daily function, self-care, social life and emotional wellbeing. Based on responses to open-ended questions, when considering the experiences after switching to N9-GP, common themes (described by >50% of patients) included advantages from the reduced infusion frequency and fewer spontaneous bleeds. Most patients spontaneously reported a positive experience with the transition to N9-GP and described increased confidence compared with previous therapy (Figure). However, patients suggested that changes could be made to improve the convenience of N9-GP medication packaging.
Conclusions: Understanding patient experiences with new therapies is pertinent as hemophilia treatment options expand. Overall, patients reported favorable experiences after switching to prophylactic N9-GP and decreased need for on-demand treatment. It is likely that the extended half-life of N9-GP contributes to QOL improvements.
| Patient characteristic | Median (range) (N = 10) |
| Age at study inclusion/age at diagnosis, years | 45 (22-67) / 1.3 (0-16) |
| Length of time on prophylaxis treatment with N9-GP, months | 12 (6-13) |
| Number of on-demand infusions of factor treatment before N9-GP, infusions per year | 10 (0-44) |
| Number of on-demand infusions of N9-GP, infusions per year | 1.5 (0-28) |
| Hemophilia severity | n patients |
| Mild (> 5%-40% FIX levels) | 1 |
| Moderate (1-5% FIX levels) | 1 |
| Severe (< 1% FIX levels) | 8 |
| Prior treatment before switching to N9-GP | Extended half-life FIX: 6. Standard half-life FIX: 4 |
[Table. Patient characteristics]
[Figure. Spontaneously-reported themes captured relating to switching to N9-GP, based on responses to open-ended questions]
To cite this abstract in AMA style:
Phua C, Faurby M, Macdonald V, Lambert J, Teitel J. A Real-World Qualitative Analysis of the Burden of Hemophilia B and Patient Experiences with Prophylactic Nonacog Beta Pegol [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/a-real-world-qualitative-analysis-of-the-burden-of-hemophilia-b-and-patient-experiences-with-prophylactic-nonacog-beta-pegol/. Accessed September 29, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/a-real-world-qualitative-analysis-of-the-burden-of-hemophilia-b-and-patient-experiences-with-prophylactic-nonacog-beta-pegol/