Hemophilia A: Moving Beyond Bleeds

Symptoms may include⁵: 

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 bleed^2,4
• 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.

FVIII levels correlate with the risk of bleeds²

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

Normal^8,9*: 

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

Mild^9*: 

• 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

Moderate^9*: 

• 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

Severe^9*:

• 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.

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 phenotype^14,15
• 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

* 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:

* 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