Factor VIII (FVIII) replacement therapies have been a step forward for the prevention and treatment of bleeding episodes in patients with hemophilia A. One remaining challenge is the development of neutralizing antibodies, known as inhibitors, which interfere with patient response to a factor infusion or render it ineffective.

The Risk of Factor VIII Inhibitors in Hemophilia A Treatment 

HEMOPHILIA-A

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.

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

This article will cover: &nbsp;
<ul>
 <li style="margin-left:8px">Patient participation in care plans: The importance of active patient involvement when creating a personalized treatment plan and identifying goals through ongoing quality-of-life (QOL) questionnaires and assay results. 
 &nbsp;</li>
 <li style="margin-left:8px">Discussing factor VIII levels and goals: Engaging patients in discussions about their factor VIII levels and the various assays and blood tests that support these goals. These conversations help patients comprehend the technical aspects and the broader implications of these results. 
 &nbsp;</li>
 <li style="margin-left:8px">Integrating physical activity: Recognizing how physical activity fits into your patient’s treatment goals and its value for their overall physical health and well-being. Providing clear guidelines can help your patients with hemophilia safely engage in physical activity. 
 &nbsp;</li>
 <li style="margin-left:8px">Empowering patients for better outcomes: Keeping patients informed and proactive in managing their condition can lead to better health outcomes and an improved quality of life.</li>
</ul>
<h2>Personal Treatment Goals in Hemophilia Care</h2>
Each patient with hemophilia A may have different values, needs, preferences, and goals based on their disease severity, medical history, lifestyle, and personal experiences with therapy. By actively involving your patients in setting their own treatment goals and priorities, including their goals for factor VIII activity levels, you can help personalize their treatment and ensure their unique needs are being addressed.&nbsp;
Treatment goals should be revisited and adjusted as patients age, since their needs and priorities related to their factor VIII levels may change.&nbsp;
&nbsp;
<h3>Quality-of-Life Questionnaires and Treatment Goals</h3>
How can QOL questionnaires help inform your overall treatment plan? The questionnaires can help your patients identify their treatment priorities and goals, like engaging in sports or physical exercise or maintaining mobility as they age.&nbsp;
These assessments allow patients to voice their preferences and concerns while providing valuable insights that inform personalized treatment decisions.&nbsp;
By regularly completing these questionnaires, patients and healthcare providers can assess progress and collaborate to create a plan that truly reflects the patient’s unique needs.&nbsp;
&nbsp;
<h3>Setting a higher goal for factor VIII levels</h3>
When establishing factor VIII activity-level goals, consider:
<ul>
 <li style="margin-left:8px">
 The frequency of infusions: Some patients might prefer to maintain higher factor VIII levels, which may reduce the risk of bleeds.&nbsp;Other patients might prefer reduced treatment burden and fewer infusions so they can minimize disruption to their daily routines, even if it means accepting a higher risk of bleeds.</li>
</ul>
<a href="https://pro.campus.sanofi/us/products/altuviiio/pharmacokinetics" style="color:blue; text-decoration:underline">Discover a high sustained factor therapy for your patients.</a>
<ul style="margin-bottom:11px">
 <li style="margin-bottom:11px; margin-left:8px">Physical activity levels: Patients who regularly exercise or play sports may need higher factor VIII levels to prevent bleeding episodes during these activities. But even patients who are less active remain at risk of bleeds that can affect long-term joint health and overall mobility, so whatever the activity level of patients, aiming for higher factor VIII levels should be a priority. </li>
</ul>
<h2>Pursuing Physical Activity Goals in Hemophilia Management 
 &nbsp;</h2>

Staying active can improve cardiovascular health, enhance muscle strength, and support mental health. However, engaging in physical activity requires careful planning and adherence to specific guidelines to reduce the risk of a bleeding episode. 
The World Federation of Hemophilia (WFH) Guidelines for the Management of Hemophilia emphasize the importance of choosing low-impact activities that are gentle on the joints and reduce the likelihood of bleeds. Low-impact activities can include: 
<ul>
 <li style="margin-left:8px">Swimming</li>
 <li style="margin-left:8px">Walking</li>
 <li style="margin-left:8px">Cycling</li>
</ul>
For your patients interested in higher-impact sports, it’s important to be on the same page about the risks and precautions that can be taken, like using protective gear to minimize potential injury&nbsp;and being on a prophylaxis regimen.
Recommending regular physiotherapy to your patients can also help strengthen muscles, maintain joint function, and prevent chronic joint damage and the development of hemophilic arthropathy. 
&nbsp;
<h3>Strategies for achieving pain management goals</h3>
Early intervention and proactive pain management are crucial to prevent long-term joint damage and disability. By addressing pain promptly and effectively, patients can maintain mobility and improve their overall quality of life. Managing acute and chronic pain may include strategies like treating and preventing acute bleeds, pain relief medications, and physiotherapy.
<h2>Helping Patients Understand Factor VIII Activity Assays</h2>

Factor VIII activity levels indicate the clotting ability of the blood, which is essential to creating a 
 personalized treatment plan based on your patients’ needs. By helping patients grasp these concepts, they can better appreciate the importance of maintaining specific factor VIII levels to prevent bleeds and manage their condition effectively.
It's also important for patients to recognize the purpose of factor VIII activity assays. These assays measure how much factor VIII is in the blood, providing a clearer picture of the patient's clotting capacity. Understanding how these tests work and what their results mean can help empower patients to be proactive about their treatment goals. 
 Patients receiving factor replacement therapy should also understand that factor VIII trough levels indicate the lowest concentration of factor VIII in the blood between infusions. While a trough factor level of 1 IU/dL (1%) was considered adequate, current standards suggest levels should be significantly higher (&gt;3%-5%). 
 Doctors should ensure patients understand that achieving higher trough levels may require higher doses or more frequent infusions with standard half-life and extended half-life therapies. An additional option is high-sustained factor therapies, which have a longer half-life.
&nbsp;
<h3>Interpreting Factor VIII Assay Results: What Do Higher Factor VIII Levels Mean?</h3>
Factor VIII assay results can help patients understand their condition's severity and understand their treatment needs. Higher factor VIII levels correlate with a greater capacity to clot efficiently, reducing the risk of bleeding episodes. Lower factor VIII levels can result in bleeding episodes, a longer recovery time, and spontaneous bleeds, which can lead to long-term joint damage.
&nbsp;
<h3>Assays Measuring Factor VIII Deficiency: aPTT</h3>
The activated partial thromboplastin time (aPTT)-based clotting assay is the most commonly used method worldwide for testing factor VIII deficiencies. This assay measures coagulation activity, which helps to determine factor VIII activity levels. Other tests include two-stage assays and chromogenic assays, which are used less frequently.
It’s important for patients with hemophilia A to know that factor VIII assays are not only for diagnosis, but are also used to set unique treatment goals and are vital for monitoring efficacy of potential treatments, such as factor replacement therapies.
&nbsp;
<h3>Other hemophilia laboratory tests: what patients need to know</h3>
Other blood tests and assays patients may become familiar with are tests for hemophilia inhibitors, which identify antibodies that indiscriminately neutralize endogenous and exogenous factors, potentially complicating treatment.
<a href="https://pro.campus.sanofi/us/products/altuviiio/mechanism-of-half-life-extension">Learn more about the risk of inhibitors.</a>
<h2>CONCLUSION</h2>
Effective communication about treatment goals and factor VIII activity assays is crucial in managing hemophilia A. Doctors play a pivotal role in helping their patients understand their treatment plans, the importance of regular monitoring, and how assays impact their care. By fostering a collaborative relationship and providing clear, concise information, HCPs can empower patients to take an active role in their treatment, ultimately improving outcomes and quality of life.

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. 

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

*Factor-replacement therapy is one class of treatment option for hemophilia patients. This article does not examine factor mimetic, gene therapy, or investigational treatments.
<h2 style="margin-bottom: 11px;">Hemophilia A Treatments: Prophylaxis vs On-Demand </h2>
Prophylaxis treatment is recognized by the World Federation of Hemophilia (WFH) Guidelines for the Management of Hemophilia as the standard of care for individuals with severe hemophilia, and for some with moderate hemophilia. The WFH Guidelines recommend starting prophylaxis before the age of 3 years to help prevent musculoskeletal complications due to recurrent joint and muscle bleeds. 
 Individualized dosing regimens are tailored to a patient's lifestyle, physical activity level, and pharmacokinetics, ensuring that each patient receives the correct dose to <a name="_Int_qo2vpFqm">maintain</a> their target factor VIII level while minimizing the risk of bleeds. On-demand treatment is useful for managing acute bleeds, but doesn’t offer the same level of protection as prophylaxis and may lead to more frequent and severe bleeding episodes in the long run. The WFH Guidelines advise that on-demand factor replacement should no longer be considered a long-term treatment option since it’s only a response to a bleed—prevention of bleeds and long-term joint damage should be the priority for patients and their doctors. 
Types of Factor VIII Replacement Therapies, Infusion Rates, and Dosing Frequency Maintaining adequate factor VIII trough levels is crucial in hemophilia A management to ensure continuous protection against spontaneous bleeding, especially for patients on prophylactic treatment. Previously, experts considered a trough factor level of 1 IU/dL (1%) adequate, but current standards recommend significantly higher levels (&gt;3%-5% or higher). 
 &nbsp;

Standard half-life (SHL) factor VIII infusions have a half-life of approximately 12 hours in adults. The short half-life of SHLs results in the need for frequent infusions. On average, this is 3 infusions per week for patients to maintain minimal trough levels (&gt;1%). Because of higher clearance rates, half-life is shorter in individuals younger than 18 years, which may result in requiring more frequent infusions that many patients are unwilling or unable to do. Conventional factor prophylaxis with SHLs is classified by intensity: high-dose (25-40 IU/kg every 2 days), intermediate-dose (15-25 IU/kg 3 days per week), and low-dose (10-15 IU/kg 2-3 days per week).

Extended half-life (EHL) factor VIII infusions have a half-life that is 1.4-1.6–fold longer than SHL products. However, the half-life of EHLs is still limited to up to 19 hours. 
 While EHLs require less frequent administration than SHL therapy, patients may still require 2 or more infusions per week to maintain desired trough levels. The criteria for high-, intermediate-, and low-dose prophylaxis with EHLs are not yet established, and these definitions may require revision due to the differing pharmacokinetic profiles of SHLs and EHLs.

High-sustained factor (HSF) therapy was designed to extend half-life longer than EHLs (~19 hours) and SHLs (~12 hours), allowing for less-frequent administration.

Hemophilia home-infusion therapy allows people to self-infuse from the comfort of home. The WFH recommends providing adequate education to the person with hemophilia, as well as a family member or caregiver. This includes learning the factor VIII replacement calculation, understanding the factor VIII replacement formula so they are using an accurate and safe dose every time, and learning to recognize potential complications.
The rate of infusion and frequency of dosing are dependent on an individual’s specific needs. Several factors can impact these, including the patient’s weight, severity of bleeding, and level of comfort with treatment. These decisions are made at the discretion of the healthcare provider, who will consider all relevant factors to ensure optimal treatment that is tailored to the lifestyle and goals of each patient.
The frequency of dosing depends on whether a patient is using a prophylaxis treatment or on-demand therapy and whether the factor VIII replacement therapy is a standard half-life, extended half-life, or high-sustained factor therapy.

<h3 style="margin-bottom: 11px;">Switching Treatments for Hemophilia A Patients</h3>
With newer treatments, it is now possible to aim for higher factor VIII levels. Doctors should ensure patients understand that achieving higher trough levels may require higher doses or more frequent infusions with SHL and EHL therapies. &nbsp;
<h2 style="margin-bottom: 11px;">Potential Complications of Factor VIII Infusion</h2>
<h3 style="margin-bottom: 11px;">For Hemophilia Patients With Inhibitors</h3>
One of the most significant complications that should be considered with factor VIII replacement therapies is the development of inhibitors—antibodies that neutralize the effectiveness of the replacement clotting factor. According to WFH Guidelines, patients who develop inhibitors should have access to appropriate hemostatic agents for bleeding control and surgical interventions, if needed, at experienced centers.&nbsp;
Bypassing agents and other non-factor treatment products may be an option for patients with inhibitors. These treatments can provide effective hemostasis and help manage bleeding episodes effectively.

<h2 style="margin-bottom: 11px;">Conclusion</h2>
Involving patients in developing personalized treatment plans for hemophilia A is essential. By educating patients on their factor VIII activity and different treatment options, healthcare providers can set individualized and achievable goals. This collaborative approach may enhance adherence to treatment, reduce bleeding episodes, and improve overall quality of life. Engaging patients in their care not only fosters better health outcomes, but also strengthens the patient–provider relationship, ultimately leading to more effective hemophilia A management.
<a href="https://pro.campus.sanofi/us/products/altuviiio" style="color:blue; text-decoration:underline">Learn more about a potential treatment option for your patients.</a>

Ensuring optimal care for patients with hemophilia A requires a thorough understanding of available treatment options, prioritization of bleed prevention and long-term joint health, and comprehensive patient education. This article will cover different types of factor VIII replacement therapies* and will compare an on-demand versus prophylactic treatment approach with the goal of helping tailor treatment plans effectively, which can help enhance quality of life.

Exploring Factor VIII Replacement Therapies: A Guide to Choosing the Right Treatment for Patients With Hemophilia A

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Hemophilia A: Moving Beyond Bleeds

Latest Articles

The Risk of Factor VIII Inhibitors in Hemophilia A Treatment

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

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

Exploring Factor VIII Replacement Therapies: A Guide to Choosing the Right Treatment for Patients With Hemophilia A