Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2022 Sep 24:13:517-524.
doi: 10.2147/JBM.S359510. eCollection 2022.

Comparison of Real-World Dose and Consumption for Two Extended Half-Life Recombinant Factor VIII Products for the Treatment of Hemophilia A in the United States

Yanyu Wu  1 , Shawn X Sun  1 , Tao Fan  2
Affiliations

Comparison of Real-World Dose and Consumption for Two Extended Half-Life Recombinant Factor VIII Products for the Treatment of Hemophilia A in the United States

Yanyu Wu et al. J Blood Med. .

Abstract

Background: US patients with hemophilia A can receive prophylaxis with extended half-life recombinant factor VIII (rFVIII) products, including efmoroctocog alfa (fragment crystallizable fusion protein) and rurioctocog alfa pegol (antihemophilic factor [recombinant], PEGylated).

Objective: To evaluate dosing patterns and weekly consumption of extended half-life rFVIII products in the United States.

Methods: We performed a retrospective analysis using the US Specialty Pharmacy Database (2015-2018). Included patients had a diagnosis of hemophilia A, ≥2 consecutive monthly claims for efmoroctocog alfa or rurioctocog alfa pegol for prophylaxis, and weight data. Outcome measures included weekly dosing frequency and dispensed weekly dose.

Results: The analysis included 774 patients (efmoroctocog alfa, 506; rurioctocog alfa pegol, 268). Mean (SD) age was 24.2 (15.8) and 26.3 (14.9) years for patients receiving efmoroctocog alfa and rurioctocog alfa pegol, respectively; mean (SD) weight was 68.4 (36.8) and 79.8 (37.7) kg, respectively. The most frequent efmoroctocog alfa regimen was twice weekly (45.7%), followed by every 4 days (20.6%), every 3 days (9.1%), and 3 times per week (7.5%). The most frequent rurioctocog alfa pegol regimen was twice weekly (72.4%), followed by 3 times per week (8.7%), every 4 days (7.6%), and every 3 days (5.5%). The proportion of efmoroctocog alfa twice-weekly dispensing records increased from 31.5% to 50.9%, and every 4 days dispensing records decreased from 31.3% to 14.5% (2015-2018). The proportion of rurioctocog alfa pegol dispensing records remained broadly stable (2016-2018). Overall, mean (SD; median) weekly prophylactic dose was 105.4 (77.9; 92.6) IU/kg with efmoroctocog alfa, and 96.8 (41.9; 90.9) IU/kg with rurioctocog alfa pegol.

Conclusion: In this database study, the most frequently observed dosing frequency was twice weekly for patients receiving efmoroctocog alfa or rurioctocog alfa pegol. The observed mean weekly consumption was slightly higher, and variation was greater, in patients receiving efmoroctocog alfa versus rurioctocog alfa pegol.

Keywords: PEGylated; antihemophilic factor (recombinant); dosing patterns; efmoroctocog alfa; fragment crystallizable fusion protein; hemophilia A; rurioctocog alfa pegol.

PubMed Disclaimer

Conflict of interest statement

Y. Wu and S.X. Sun are employees of Takeda Development Center Americas, Inc., Cambridge, MA, USA, and Takeda stock owners. T. Fan is an employee of Takeda Pharmaceuticals USA, Inc., Lexington, MA, USA, and Takeda stock owner. The authors report no other conflicts of interest in this work.

Figures

Figure 1
Figure 1
Mean (SD) Weekly Consumption (2015–2018), by age category.
See this image and copyright information in PMC

References

    1. Mannucci PM, Tuddenham EG. The hemophilias — from royal genes to gene therapy. N Engl J Med. 2001;344(23):1773–1779. doi: 10.1056/NEJM200106073442307 - DOI - PubMed
    1. Iorio A, Stonebraker JS, Chambost H, et al.; Data Demographics Committee of the World Federation of Hemophilia. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med. 2019;171(8):540–546. doi: 10.7326/M19-1208 - DOI - PubMed
    1. Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood. 2015;125(13):2038–2044. doi: 10.1182/blood-2015-01-528414 - DOI - PubMed
    1. Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia panelists and co-authors. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020;26(Suppl6):1–158. doi: 10.1111/hae.14046 - DOI - PubMed
    1. Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al.; Treatment Guidelines Working Group on Behalf of The World Federation Of Hemophilia. Guidelines for the management of hemophilia. Haemophilia. 2013;19(1):e1–47. doi: 10.1111/j.1365-2516.2012.02909.x - DOI - PubMed