Preventing bleeds.

The right care and treatment can help you live an active, healthy life with a bleeding disorder. The main treatment for bleeding disorders is called factor replacement therapy, which provides the clotting factor that is missing or too low. It can be given in two ways:

• On-demand therapy is giving factor to stop bleeding when it starts. With on-demand therapy there is a risk that bleeds will cause damage before you are treated.
• Prophylactic therapy is giving factor on an ongoing basis to prevent bleeding before it starts. It’s also called prophylaxis.

Benefits of prophylactic therapy.

Prophylactic therapy was created because doctors saw that, compared to severe bleeding disorders, people with a mild or moderate bleeding disorder (factor levels of 1 percent or more) had less sudden bleeding and joint damage.¹

Studies have confirmed that children who receive prophylaxis have fewer bleeds and healthier joints. But breakthrough bleeds may still happen with prophylactic therapy. It also won’t fix the joint damage you already have but it can help slow it down. Prophylaxis also helps you:

• Have fewer bleeds
• Prevent damage to other joints and body parts
• Stay active and able to live a full life

Types.

The types of prophylactic therapy are based on when the treatment is given and why.

Continuous therapy is given over many months or years to help prevent bleeds and damage to joints and other body parts.

Periodic (intermittent) therapy is given for a few weeks or months to prevent bleeding from certain activities such as surgery, physical therapy and sports.

Candidates.

Not everyone needs prophylaxis and prophylactic treatment depends on many things, such as your activities, your need for surgery, and how severe your bleeding disorder is:

• Mild bleeding disorders: Not usually needed
• Moderate bleeding disorders: May be needed periodically to prevent bleeding during certain activities
• Severe bleeding disorders: Needed continuously to prevent bleeding that could damage joints and other body parts

Dosing schedules.

Most of the time, prophylaxis is factor given on a regular basis to keep levels at 1 percent or more. Prophylactic therapy dosing schedules explain when, how much, how often, how long, and what type of factor is given.

Not everyone needs prophylaxis.

There are many dosing schedules and the right dosing schedule for you, including how much factor you receive, should be based on your:

• Age
• Own clotting factor levels
• Bleeding pattern
• Joint health
• Level and types of activity
• Ability to stick to the schedule
• Body and how it handles factor

Starting and stopping.

People with severe bleeding disorders may start prophylaxis at a young age and some need to be treated for life. Most doctors and bleeding disorder experts believe that if factor is available, prophylaxis should start prior to joint damage as prophylaxis therapy will not repair joints that are already damaged. More research is needed to learn about how long prophylaxis should continue.

Treatment issues.

Prophylaxis is a common treatment goal but some issues, such as those listed below, may keep people from getting the best results:

• Inhibitors are antibodies to factor the body makes. Inhibitors destroy the factor before it can work. People given prophylaxis need to be watched closely for inhibitors.
• Adherence means how well you stick to a dosing schedule. Sticking to a dosing schedule can be hard and skipping doses can cause factor levels to fall and increase bleeding risk. Giving factor at home, longer-acting factor products and working with your CVS Specialty CareTeam or Bleeding Disorders Treatment Center (HTC) can help you stay on track.
• Vein access is needed to give factor. It can be hard to find the right veins in children. A port can be implanted to help, but there are health risks. Your child’s doctor will talk to you about these risks before starting treatment.

Another type of prophylaxis.

Bleeding disorders therapy has evolved over the past few years. One advancement is the development of other ways to provide prophylaxis to some bleeding disorder patients other than factor replacement therapy.

Hemlibra (emicizumab) is not a factor replacement therapy. This drug works by replacing the function of factor VIII (8) as opposed to replacing the missing clotting factor VIII directly. This therapy is only available for Hemophilia A patients and can reduce or prevent the frequency of bleeding episodes. Keep in mind that patients who are using Hemlibra as prophylactic therapy must always have factor replacement available to treat an acute bleed if and when one occurs.

Prophylactic therapy helps prevent bleeds to help you live a fuller life. Talk to your doctor or HTC about which prophylactic therapy is best for you or your child. If you or your child is receiving prophylaxis, work with your doctor, HTC or contact the CVS Specialty Bleeding Disorders CareTeam at 1-866-RxCare-1 (1-866-792-2731). Our Bleeding Disorders CareTeam representatives are ready to help.