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Hemophilia A: Moving Beyond Bleeds

Higher Factor VIII (FVIII) Levels Make a Difference1,2
FVIII levels are used to determine the severity of hemophilia and also correlate with bleed risk. The higher a person with hemophilia’s FVIII levels are, the lower their risk for bleeds. 

Still Bleeding3,4
Despite advances in treatment, many people with hemophilia A are still bleeding while on an extended half-life (EHL), standard half-life (SHL), or factor mimetic therapy. 

Enhancing Treatment Goals 
This means aiming for more protection from bleeds and a more individualized treatment plan. How can your patients benefit from aiming for more? 

Learn more about hemophilia A and some treatment strategies in the tabs, articles, and resources below.

Symptoms may include5

Bleeding into jointsBruising or hematomaBleeding that is hard to stopBlood in the urine or stool

Additionally, it is common for people with hemophilia A to experience:

  • Depression and anxiety around the possibility of bleeds6
  • Challenges with daily life due to a diminishing range of motion, relative difficulty moving, and increased pain and swelling from even 1 joint bleed2,4
  • Chronic pain that impacts their physical ability and interferes with their daily life7

The severity of hemophilia A is determined by how much factor VIII is in the blood.

MildModerateSevere
Factor levels between >5% to <40%1Factor levels between 1% to 5%1Factor levels of <1%1
Severe bleeding can occur in the cases of major trauma and surgery2Can result in occasional spontaneous bleeding, with prolonged or severe bleeding associated with minor trauma or surgery2Can result in spontaneous bleeding into the joints or muscles, mostly in the absence of an identifiable hemostatic challenge2

FVIII levels correlate with the risk of bleeds2

FVIII levels are correlated with the ability to perform activities with lower levels of risk.

Normal8,9*

  • FVIII activity levels of 50% to 150%
  • High-impact activity is possible

Mild9*: 

  • FVIII activity levels of 5% to 40%
  • High-risk activity is possible
  • Intensive sport activity should be limited to those with FVIII levels between 15% to 30%
  • Appropriate level of physical activity should be evaluated on a case-by-case basis

Moderate9*: 

  • FVIII activity levels of 1% to 5%
  • FVIII levels between 1% to 3% may be sufficient for those with a sedentary lifestyle, but higher levels between 3% to 5% are recommended if engaging in mild physical activity
  • Individuals should refrain from high-risk activity

Severe9*:

  • FVIII activity levels of <1%
  • High risk of spontaneous bleeds–even with low activity there is pain in target joints

How often should FVIII levels be tested?

WFH guidelines recommend2:

  • Adults should test once per year
  • Children should test every 6 months

WFH=World Federation of Hemophilia.

* Based on the consensus of a panel of 11 physicians–selected on their expertise in the treatment of patients with hemophilia–who used a modified Delphi approach to define a range of target factor levels for use in clinical practices.

Treatment ClassFactor replacement therapy2

Factor mimetic therapy2

Gene therapy10

Mechanism of Action

Temporarily replaces the missing coagulation FVIII

Bridges activated factor IX and factor X to restore the function of missing activated factor VIII

Introduces a functional transgene encoding for human coagulation factor FVIII into the liver to replace the missing coagulation FVIII

Route of Administration

Intravenous infusion

Subcutaneous injection

Intravenous infusion

Frequency of Use

Routine and/or as needed

Routine

One-time

Indication(s)

Prophylaxis

Yes ✓

Yes ✓2

Yes ✓

On-demand

Yes ✓

No 🅧

Exogenous FVIII or other hemostatic products may be required for breakthrough bleeds2

No 🅧

Exogenous FVIII or other hemostatic products may be required for an active bleed

Perioperative management

Yes ✓

No 🅧

Exogenous FVIII or other hemostatic products may be required for perioperative management11

No 🅧

Exogenous FVIII or other hemostatic products may be required for perioperative management

Monitoring
 Yes ✓

No 🅧2,11

Does not impact factor activity levels, but it is possible to quantify the factor equivalency, which provides an estimate of hemostatic activity

Yes ✓

Factor replacement therapies

  • Include standard half-life (SHL) and extended half-life (EHL) therapies
  • Current limitations include length of half-life and frequency of infusions2
    • Half-life up to ~12 hours for SHL therapies12
    • Half-life up to ~19 hours for EHL therapies2

Factor mimetic therapies

  • Hemophilia treatment that is independent of factor replacement13
  • Factor-mimetic prophylaxis treatment is estimated to maintain factor-equivalency levels at ~9% to 20%, which is consistent with a mild hemophilia phenotype14,15
  • Factor levels of 15% are not sufficient to control acute bleeding events and may not be sufficient to protect from all bleeds, particularly subclinical bleeds and those associated with traumas, surgery, and/or intense physical activity11

Learn more about the different available hemophilia treatments in Exploring Factor VIII Replacement Therapies: A Guide to Choosing the Right Treatment for Patients With Hemophilia A

Despite advancements in treatment, patients are still at risk for bleeds and microbleeds.3,4

SHL

EHL

FVIII mimetic

50% to 65%

of adults and adolescents receiving standard half-life (SHL) therapies are still experiencing bleeds16-20*

55% to 63%

of adults and adolescents receiving extended half-life (EHL) therapies are still experiencing bleeds21-24†

50% to 72%

of adults and adolescents receiving factor mimetic therapy are still experiencing bleeds25‡

* The data included are derived from the Prescribing Information of multiple SHL therapies for hemophilia A. These data are specific to patients that received prophylaxis with various dosing regimens of SHL therapy. 

The data included are from a review of 4 Phase 3 pivotal trials in patients receiving once- or twice-weekly prophylaxis. The primary arm of each trial was examined to extract these data. 

The data included are from 2 Phase 3 pivotal trials in patients ≥12 years old (n=109, n=152) receiving once-weekly prophylaxis with emicizumab.

By prioritizing greater bleed protection through maintaining closer to normal factor VIII levels, people with hemophilia A can live a more active life.4,9

Starting patients on prophylaxis with enhanced treatment goals has the potential for:

Preserving patients’ joint health over the long term, based on a study in which it was estimated that sustaining baseline factor VIII levels ≥15% could be associated with 0 expected joint bleeds26*Reducing chronic pain to allow patients to work more physical jobs and participate in family life and other daily activities4Offering patients the ability to live a less restricted lifestyle and experience improved quality of life4

* These data are from a study that examined a 2001 series of self-reported Dutch surveys that included 433 mild or moderate hemophilia A patients that were treated on-demand.25

Latest Articles

Hemophilia A in Women - Key Points to Discuss With Your Patients

Hemophilia A–a rare bleeding disorder–often goes undiagnosed in women because of the misconception that it only affects men, due to its X-linked recessive inheritance pattern. Although factor levels above 50% are usually considered within the normal range, emerging data suggest that some women and girls with levels between 50% and 60% may still have an increased risk of bleeding symptoms. Additionally, they may have sex-specific symptoms such as heavy menstrual bleeding and postpartum hemorrhage. This highlights the need for a more nuanced understanding of hemophilia A as it pertains to women.

Learn more
Young man speaking to a healthcare professional in an office.

Discussing Treatment Goals and Factor VIII Activity Assays With Hemophilia A Patients

Why is it important to accurately measure factor VIII activity levels when treating patients with hemophilia A? Because the results can help you tailor treatment plans and help inform personalized treatment goals. To aid in shared decision-making, it’s essential for patients to understand the significance of factor VIII activity level assay measurements. This knowledge can empower them to actively participate in their treatment plan, make informed decisions, and improve their day-to-day lives and overall well-being.

Learn more

References: 1. Centers for Disease Control and Prevention. Accessed November 19, 2024. https://www.cdc.gov/hemophilia/testing/index.html 2. Srivastava A, et al. Haemophilia. 2020;26(suppl 6):1-158. 3. Mannucci PM. Haematologica. 2020;105(3):545-553. 4. Skinner M, et al. Haemophilia. 2020;26(1):17-24. 5. Centers for Disease Control and Prevention. Accessed November 11, 2024. https://www.cdc.gov/hemophilia/about/index.html 6. Al‐Huniti A, et al. Haemophilia. 2020;26(3):431-442. 7. Visweshwar N, et al. Blood. 2020;31(6):346-352. 8. National Library of Medicine, National Institutes of Health. Accessed November 21, 2024. https://www.ncbi.nlm.nih.gov/books/NBK470265/?report=printable 9. Iorio A, et al. Haemophilia. 2017;23(3):e170-e179. 10. Roctavain Prescribing Information. BioMarin Pharmaceutical Inc. Novato, CA. 11. Berntorp E, et al. Blood Rev. 2021;50:100852. 12. Chhabra A, et al. Blood. 2020;31(3):186-192. 13. Carcao M and Goudemand J. Inhibitors in Hemophilia: A Primer. 5th ed. World Federation of Hemophilia; 2018. 14. Malec L, et al. Haemophilia. 2023;29(6):1419-1429. 15. Andrade PE, et al. Haematologica. Published online November 2, 2023:0-0. doi:10.3324/haematol.2022.282099 16. Adynovate Prescribing Information. Takeda Pharmaceuticals U.S.A., Inc. Lexington, MA. 17. Eloctate Prescribing Information. Bioverativ Therapeutics Inc. Waltham, MA. 18. Esperoct Prescribing Information. Novo Nordisk Inc. Plainsboro, NJ. 19. Jivi Prescribing Information. Bayer HealthCare LLC. Whippany, NJ. 20. Hemlibra Prescribing Information. Chugai Pharmaceutical Co., Ltd. Tokyo, Japan. 21. Advate Prescribing Information. Takeda Pharmaceuticals U.S.A., Inc. Lexington, MA. 22. Afstyla Prescribing Information. CSL Behring LLC. Kankakee, IL. 23. Kovaltry Prescribing Information. Bayer HealthCare LLC. Whippany, NJ. 24. Nuwiq Prescribing Information. Octapharma USA, Inc. Paramus, NJ. 25. Xyntha Prescribing Information. Wyeth Pharmaceuticals LLC. Philadelphia, PA. 26. den Uijl IE, et al. Haemophilia. 2011;17(1):41-44.

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