A real-world evidence analysis of the impact of switching from factor VIII to emicizumab prophylaxis in patients with hemophilia A without inhibitors

ABSTRACT

Background

This study retrospectively compared annualized billed bleed rates (ABR_b) in people with hemophilia A (PwHA) without inhibitors who switched from factor VIII (FVIII) prophylaxis to emicizumab.

Research design and methods

A real-world comparison study was performed on the effect of switching from FVIII to emicizumab prophylaxis in male, non-inhibitor patients on ABR_b using an all-payer claims database (APCD) dataset from 1 January 2014, to 31 March 2021. The identification period was from 1 November 2017, to 30 September 2020.

Results

One hundred and thirty-one patients were included with a total of 82 and 45 bleeds in the pre- and post-switch periods, respectively. The average follow-up period pre-switch was 978.37 days (SD 555.03), whereas the average follow-up period post-switch was 522.26 days (SD 191.36). No significant differences in mean ABR_b were observed pre-/post-switch (0.25 and 0.20, respectively; P = 0.4456).

Conclusions

The results of this study demonstrate no significant reduction in ABR_b, suggesting that switching from FVIII to emicizumab may not deliver incremental benefits to PwHA receiving prophylactic care.

KEYWORDS:

• Hemophilia A
• billed annualized bleed rate
• factor VIII
• emicizumab
• real-world evidence
• retrospective analysis
• prophylaxis
• treatment switching

Acknowledgments

Writing and editorial assistance for the development of this manuscript was provided by Ari Simenauer and Zela Keuylian (employees of Parexel), funded by Takeda Pharmaceuticals U.S.A., Inc., Lexington, MA.

Declaration of Interests

M Escobar receives honoraria from Bayer, BioMarin, CSL Behring, Genentech/Roche, Hemabiologics/LFB, Kedrion, NHF, Novo Nordisk, Pfizer, Sanofi, Takeda, and uniQure. The University of Texas participates in clinical trials sponsored by CSL Behring, Bayer, Genentech/Roche, Novo Nordisk, Sanofi, Takeda, and uniQure. M Bullano, AG Mokdad, J Caicedo, BG Schultz, and Q Fan are employees and stockholders of Takeda Pharmaceuticals U.S.A., Inc., Lexington, MA. S Verma is an employees of STATinMED, which was contracted by Takeda for work on this study. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer Disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contributions

Conception and design: M Bullano, J Caicedo, AG Mokdad, BG Schultz, S Verma. Analysis/interpretation of data: J Caicedo, M Bullano, BG Schultz, Q Fan, AG Mokdad, S Verma, M Escobar. Drafting the manuscript: Q Fan, AG Mokdad, S Verma. Revising the manuscript critically for intellectual content: J Caicedo, M Bullano, BG Schultz, Q Fan, AG Mokdad, S Verma, M Escobar.

Data availability statement

The data underlying this article were provided by STATinMED via confidential license agreement, which has legally enforceable usability restrictions that preclude our making the data available publicly.

Additional information

Funding

This work was supported by Takeda Pharmaceuticals U.S.A., Inc.