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

This article will cover:  

• 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.
   
• 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.
   
• 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.
   
• 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.

Personal Treatment Goals in Hemophilia Care

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. 

Treatment goals should be revisited and adjusted as patients age, since their needs and priorities related to their factor VIII levels may change. 

Quality-of-Life Questionnaires and Treatment Goals

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. 

These assessments allow patients to voice their preferences and concerns while providing valuable insights that inform personalized treatment decisions. 

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. 

Setting a higher goal for factor VIII levels

When establishing factor VIII activity-level goals, consider:

• The frequency of infusions: Some patients might prefer to maintain higher factor VIII levels, which may reduce the risk of bleeds. 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.

Discover a high sustained factor therapy for your patients.

• 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.

Pursuing Physical Activity Goals in Hemophilia Management
 

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:

• Swimming
• Walking
• Cycling

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 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.

Strategies for achieving pain management goals

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.

Helping Patients Understand Factor VIII Activity Assays

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 (>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.

Interpreting Factor VIII Assay Results: What Do Higher Factor VIII Levels Mean?

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.

Assays Measuring Factor VIII Deficiency: aPTT

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.

Other hemophilia laboratory tests: what patients need to know

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.

Learn more about the risk of inhibitors.

CONCLUSION

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.