| 1. |
- Astermark, Jan, et al.
(author)
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Efficacy of rfixfc versus rix-fp for the treatment of patients with hemophilia b : Matching-adjusted indirect comparison of b-long and prolong-9fp trials
- 2021
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In: Journal of Blood Medicine. - 1179-2736. ; 12, s. 613-621
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Journal article (peer-reviewed)abstract
- Purpose: In patients with hemophilia B, treatment with extended half-life (EHL) recombi-nant factor IX allows for longer dosing intervals while providing equal or superior bleeding protection compared with standard half-life products. This enables flexible, individualized treatment schedules, which reduce the burden of prophylaxis and improve patient outcomes. This analysis compared the efficacy of recombinant factor IX Fc fusion protein (rFIXFc) and recombinant factor IX albumin fusion protein (rIX-FP), two EHL therapies approved for prophylaxis and treatment of bleeding in hemophilia B. Patients and Methods: Matching-adjusted indirect treatment comparison (MAIC) was used to adjust the between-treatment differences in baseline characteristics. Individual patient data for rFIXFc (B-LONG) were matched to aggregated data for rIX-FP (PROLONG-9FP) followed by statistical comparison for estimated annualized bleeding rate (ABR) using a Poisson regression model with adjustment for over dispersion. Data were analyzed according to treatment regimen prior to study entry: prior prophylaxis (rFIXFc, n=48; rIX-FP, n=40) or prior episodic treatment (n=43 and n=19, respectively). Relative treatment effects are presented as incidence rate ratios (IRR) with 95% confidence intervals (CI). Results: After adjustment for baseline characteristics, estimated ABR observed for rFIXFc and rIX-FP was not significantly different in patients on prior prophylaxis (1.87 versus 1.58; IRR 1.18, 95% CI 0.67–2.10) or prior episodic (2.25 versus 2.22; IRR 1.01 95% CI 0.40–2.57) regimens. Conclusion: This MAIC analysis shows that the estimated ABR for rFIXFc-treated patients from B-LONG was similar to that of rIX-FP-treated patients from PROLONG-9FP and, therefore, indicates that the two EHL therapies provide similar efficacy when used as prophylaxis for patients with hemophilia B. Trough levels differ between the two products (1–3% [targeted] versus 20% [observed], respectively), suggesting that trough level is not a surrogate indicator when ABR is used as a criterion for clinical efficacy when comparing these FIX products in hemophilia B.
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| 2. |
- Astermark, Jan, et al.
(author)
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rFIXFc prophylaxis improves pain and levels of physical activity in haemophilia B : Post hoc analysis of B-LONG using haemophilia-specific quality of life questionnaires
- 2022
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In: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 28:1, s. 18-26
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Journal article (peer-reviewed)abstract
- Introduction: Recurrent bleeding in severe haemophilia B causes painful hemarthroses and reduces capacity for physical activity. Recombinant factor IX Fc fusion protein (rFIXFc) prophylaxis results in low annualised bleeding rates, with the potential to improve patients’ health-related quality of life (HRQoL). Aim: To present a post hoc analysis of data from B-LONG describing change over time in patient-reported outcomes associated with pain and physical activity. Methods: Patients (≥12 years) who received weekly dose-adjusted or interval-adjusted rFIXFc prophylaxis and completed the Haemophilia-Specific QoL questionnaire for adolescents (Haemo-QoL) or adults (Haem-A-QoL) at baseline (BL) and end of study (EoS). Individual level changes in items of the ‘Physical Health’ and ‘Sports and Leisure’ domains, categorised as ‘never/rarely/seldom’ or ‘sometimes/often/all the time’, were analysed using McNemar's test to compare distribution of responses at EoS versus BL. Results: At EoS versus BL, a significantly greater proportion of patients did not experience painful swellings (64% vs. 44%; P =.004), painful joints (44% vs. 28%; P =.003) or pain when moving (54% vs. 41%; P =.026). Additionally, at EoS versus BL, patients were less likely to avoid participating in sports like football (30% vs. 8%; P =.002), avoid sports due to their haemophilia (47% vs. 27%; P =.007), or experience difficulty walking as far as they wanted (63% vs. 43%; P =.001). The proportion of patients who played sports as much as the general population was numerically increased (52% vs. 37%; P =.033) at EoS versus BL. Conclusion: Results of the analysis suggest that over time, rFIXFc prophylaxis is associated with significant improvements in pain and physical functioning. This contributes to previous evidence of overall HRQoL improvements in patients with haemophilia B treated with rFIXFc.
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