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
Review
. 2023 Nov;29(6):1419-1429.
doi: 10.1111/hae.14866. Epub 2023 Sep 27.

Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review

Lynn Malec  1   2 , Davide Matino  3
Affiliations
Review

Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review

Lynn Malec et al. Haemophilia. 2023 Nov.

Abstract

Introduction: The standard of care in severe haemophilia A is prophylaxis, which has historically aimed for a factor VIII (FVIII) trough level of ≥1%. However, despite prophylactic treatment, people with haemophilia remain at risk of bleeds that have physical and quality of life implications, and that impact everyday life.

Aim: The aim of this review was to evaluate evidence supporting the relationship between targeting higher FVIII activity levels with prophylaxis and improved outcomes in people with haemophilia A.

Methods: We conducted a narrative review that defined the unmet needs and treatment goals in people with haemophilia A, evaluated evidence to support targeting higher FVIII activity levels, and highlighted therapies that may support higher and sustained FVIII activity levels and improved outcomes for people with haemophilia A.

Results: Despite recent advances in treatment, unmet needs remain, and people with haemophilia continue to experience joint and functional impairment, acute and chronic pain, and poor mental health. All these negatively impact their health-related quality of life. Evidence suggests that FVIII activity levels of up to 50% may be needed to achieve a near-zero joint bleed rate. However, achieving high FVIII activity levels with current standard and extended half-life (EHL) FVIII replacement therapies is associated with a high treatment burden. Innovative treatment options may provide high sustained FVIII activity levels and improved patient outcomes.

Conclusion: Evidence suggests that FVIII activity levels in people with haemophilia A should be sustained at higher levels to improve joint and patient outcomes and enable progression towards health equity.

Keywords: arthropathy; factor VIII; gene therapy; haemophilia A; non-factor replacement therapy; prophylaxis; quality of life.

PubMed Disclaimer

References

REFERENCES

    1. Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of Hemophilia. Haemophilia. 2020;26(Suppl 6):1-158.
    1. Olasupo OO, Lowe MS, Krishan A, Collins P, Iorio A, Matino D. Clotting factor concentrates for preventing bleeding and bleeding-related complications in previously treated individuals with haemophilia A or B. Cochrane Database Syst Rev. 2021;8(8):Cd014201.
    1. Collins PW, Blanchette VS, Fischer K, et al. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost. 2009;7(3):413-420.
    1. Iorio A, Iserman E, Blanchette V, et al. Target plasma factor levels for personalized treatment in haemophilia: a Delphi consensus statement. Haemophilia. 2017;23(3):e170-e179.
    1. Martin AP, Burke T, Asghar S, Noone D, Pedra G, O'Hara J. Understanding minimum and ideal factor levels for participation in physical activities by people with haemophilia: an expert elicitation exercise. Haemophilia. 2020;26(4):711-717.

LinkOut - more resources