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. 2018 Nov 29;3(1):109-113.
doi: 10.1002/rth2.12163. eCollection 2019 Jan.

Extending recombinant factor IX Fc fusion protein dosing interval to 14 or more days in patients with hemophilia B

Amy D Shapiro  1 , K John Pasi  2 , Margareth C Ozelo  3 , Roshni Kulkarni  4 , Christopher Barnowski  5 , Bent Winding  6 , Johan Szamosi  6 , Stefan Lethagen  6   7
Affiliations

Extending recombinant factor IX Fc fusion protein dosing interval to 14 or more days in patients with hemophilia B

Amy D Shapiro et al. Res Pract Thromb Haemost. .

Abstract

Background: In the phase 3 B-LONG study (NCT01027364), prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) every 7 to >14 days was associated with low annualized bleed rates (ABRs) in males aged ≥12 years with severe hemophilia B. The long-term safety and efficacy of rFIXFc prophylaxis was confirmed in the B-YOND study (NCT01425723), an extension of the B-LONG clinical trial.

Objective: The aim of this post-hoc analysis was to evaluate the efficacy of a ≥14-day rFIXFc dosing interval in patients treated prophylactically during B-LONG or B-YOND.

Methods: The analysis included 22 patients aged ≥12 years who received prophylactic rFIXFc with a ≥14-day dosing interval at any time during B-LONG or B-YOND up until the second interim analysis of B-YOND (September 2015).

Results: The median (interquartile range [IQR]) rFIXFc exposure on the ≥14-day dosing interval was 3.4 (1.8-4) years. Patients treated with a ≥14-day dosing interval were well controlled with a median (IQR) overall ABR of 1.6 (0.6-2.7) and a median (IQR) spontaneous ABR of 0.7 (0.3-1.1) in 18 evaluable patients. A rFIXFc dosing interval of ≥14 days was well tolerated, with no new safety concerns identified.

Conclusion: Most patients on rFIXFc prophylaxis, with a dosing interval of ≥14 days, remained well controlled; ABRs were consistent with those reported in the overall study population. A ≥14-day dosing interval can be utilized in some well controlled individuals and reduces the burden imposed by frequent prophylactic injections while maintaining adequate bleed suppression.

Keywords: clinical trial; factor IX; hemophilia B; prophylaxis; recombinant fusion proteins.

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Figures

Figure 1
Figure 1
Study flow in B‐LONG and the B‐YOND extension study
See this image and copyright information in PMC

References

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