Hemophilia and factor replacement therapy

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Teens living with hemophilia learn how factor-replacement therapy works.

As you have learned, to treat your hemophilia you take injections of the clotting factor that is missing from your blood. Since you are replacing the missing factor in your blood, this form of treatment is called factor-replacement therapy.

Understanding factor replacement therapy

The factor you take to treat hemophilia can come from two different sources: blood plasma (plasma-derived) or in the lab (recombinant).

Plasma-derived factor

Plasma-derived factor is product that is collected from a large group of human blood donors. Current screening methods eliminate any known viruses from plasma-derived products, making them safe for the patients who use them for treatment.

Recombinant factor

Recombinant factor is clotting factor that scientists and engineers make artificially inside a laboratory; cells that produce factor are grown inside a controlled environment in the lab. Since it is not derived from human blood, there is no risk of infections being transmitted in the product. After collecting the factor, it is processed and then freeze-dried.

In Canada, most people with hemophilia are treated with recombinant factor products. In the USA and other countries, many people use plasma-derived factor.

Factor can come in various forms

There are many brands of factor, which are made by different companies. There are minor differences in how each of these products are made. The type of factor you take depends on your health care needs. You will work together with your comprehensive care team to decide which product is best for you. This decision is based on several criteria, which include:

  • type of hemophilia: A or B
  • the severity of your hemophilia
  • availability of the product (you may have access to only certain products in your area).

Your treatment plan also depends on whether you have an immune complication in which the body produces antibodies that destroy factor as soon as it is infused. Antibodies that target factor are called inhibitors. People who have hemophilia and also have inhibitors take other types of treatments that help stop the bleeding. The treatment depends on your current inhibitor status, medical history and gene mutation causing hemophilia. You will learn more about inhibitors in Module 5.

The table below outlines the options for treating hemophilia A and B. These may be drugs, hormones or different forms of factor that differ in how they are made. It also includes agents that treat inhibitors. Hearing about different types of products and brand names can be confusing. Talk to your doctor to learn more about your specific factor.

Type of hemophiliaTreatment options
Hemophilia A
  • Factor VIII (FVIII) concentrate (this is the standard of care). This can either be recombinant or plasma-derived factor.
  • Cryoprecipitate. This blood product is collected by thawing fresh factor at low temperatures.
  • People with milder forms of hemophilia can take a synthetic hormone, DDAVP, which triggers the body to release its own stored factors (FVIII/VWF).
  • Drugs that help prevent blood clots from breaking apart. These are called antifibrinolytic agents (tranexamic acid).
Hemophilia B
  • Factor IX (FIX) concentrate (this is the standard of care). This can either be recombinant or plasma-derived factor.
  • Fresh Frozen Plasma (FFP). This blood product is usually only given if no concentrated form of factor is available.
  • Drugs that help prevent blood clots from breaking apart. These are called antifibrinolytic agents.
Hemophilia A or B with Inhibitors
  • Recombinant Factor VIIa.
  • A mixture of plasma proteins which help form clots. This mixture is known as activated prothrombin complex concentrates (aPCCs).
Dernières mises à jour: mars 13th 2019