Treatment


Hemophilia is treatable. Missing factor can be replaced by infusing clotting factor concentrates, elevating factor levels enough to allow the body to form a clot and stop the bleeding. Many parents and caregivers learn how to infuse their child at home to prevent or treat bleeds. As a child matures, they will learn how to self-infuse and manage their infusions as they move into adulthood.

Clotting factor concentrate comes as a freeze-dried powder that is mixed with sterile water or a diluent (an agent to dilute medications) prior to infusion. It is injected into the body through a vein or a central venous access device. There are two forms of clotting factor concentrates, depending on the source from which it is made: plasma-derived or recombinant.


Treatment by factor

Plasma-derived factor Plasma-derived factor is made from donated human blood plasma. Plasma contains clotting proteins. The clotting proteins are separated from other blood components, purified, and made into a freeze-dried product. All plasma donated for production of clotting factor concentrates is tested for the presence of viruses.
Recombinant factor

Recombinant factor is made in a laboratory and most do not use blood plasma in the manufacturing process. Because recombinant products contain few or no proteins from human blood, the risk of contracting viruses is minimal.

All factor products — both plasma-derived and recombinant — are treated for purification and to prevent infection. 

Episodic regimen Episodic treatment (also called on-demand or as-needed) occurs when factor is given only when a bleeding episode occurs.
Prophylaxis regimen Prophylaxis is the practice of giving factor on a regularly scheduled basis (up to several times a week). The goal of prophylaxis is to raise factor levels to prevent bleeds. Primary prophylaxis is the type of long-term continuous prophylaxis started before a pattern of bleeding has occurred, usually at a young age. Secondary prophylaxis is also long-term continuous prophylaxis but is started after a pattern of bleeding has been demonstrated.
Planned preventive treatment Planned preventive treatment involves infusing factor before an event or activity that may potentially cause a bleed (for example, infusing before practicing or playing a sport. This type of preventive treatment regimen can also be prescribed for those that already infuse on a regular prophylaxis scheduled.

Rest, Ice, Compression, and Elevation (RICE)

No – not the stuff you eat. RICE is another treatment option for people with hemophilia. It is often used in conjunction with factor replacement therapy. This treatment can be used when experiencing a bleed away from home until factor is accessible (although it is advised to have factor on-hand whenever possible). The damaging effects of a bleed in a muscle or a joint can be reduced when RICE is used appropriately and when factor infusions are initiated within 3 hours of the first signs of a bleed. Your hematologist will work with you to develop a treatment plan for you or your child.
View Accredo’s RICE guide for more information.

R = REST Stop the activity, infuse clotting factor or medication and protect the body part. To rest the injured area, a sling, splint, crutches or a wheelchair may be needed for several days to several weeks after an injury.
I = ICE Ice or cold packs are useful in slowing the bleeding, reducing inflammation and reducing pain. Heat should not be applied to an area that is bleeding, hot or swollen.
C = COMPRESSION Compression with soft elastic supports helps reduce swelling and pain.
E = ELEVATION Elevation above the level of the heart reduces swelling.

Ports and Venous Access

Factor must be infused into veins. Infants have a high percentage of body fat, which may make finding a vein difficult. For children or adults who require frequent infusions, or those with difficulty accessing a vein, your hematologist may recommend a central venous access device (also known as a port) that is implanted under the skin. Factor is infused into the port, which has a tube connected to a large vein. Proper sterile technique when accessing ports is the most important thing to do to avoid infections in your blood. Other types of central venous access devices are visible outside of the skin.

For Children: As a parent or caregiver, whether using a port or peripheral veins, it is important to involve your child in the infusion process. This can start at an early age. A positive approach is important. Talk to your child during the infusion and speak in terms he or she can understand. Although toddlers cannot comprehend that their blood lacks a protein necessary for coagulation, they can be taught about the various supplies needed for an infusion: factor, sterile water, needles, gloves, alcohol wipes and so on. These are the seeds you plant for the future, as there will come a time when your child will need to learn to self-infuse. In the early years, keep it simple. Start by making it the child’s job just to get the factor out of the refrigerator or to hold the bandage. These simple actions can be built upon, adding more complex steps as the child matures and can handle more responsibility.

For Adults: As an adult learning to manage your hemophilia, your hematologist and specialty pharmacy can help train and educate you on the infusion process. You can learn to self-infuse as well.


Treatment logs

It is important that you always maintain infusion/treatment logs. They are much more accurate than trying to recall bleed information from memory. They will also serve as a record of infusions and bleeds in the event your factor therapy needs to be modified by your hematologist. In the event of a product recall, treatment logs will let you know exactly which lot numbers were infused. More and more, insurance companies are also requiring logs before they will approve a refill of factor products.
View Accredo’s Bleeding Disorders Treatment Log.

 

1. National Heart, Lung and Blood Institute. What is hemophilia? https://www.nhlbi.nih.gov/health/health-topics/topics/hemophilia. Accessed Aug. 4, 2017.

2. McDaniel M. "Treatment of Hemophilia A and B." Nurses' Guide to Bleeding Disorders. National Hemophilia Foundation. 2013.