Treatment

How is Haemophilia treated?

The treatment of bleeding disorders has improved over the last decade with new types of products changing the way we think about the treatment of a bleeding disorder. There are two basic types of treatment approaches:

  • On demand – This is treatment received in response to a bleed which may have occurred after an injury or accident or may have been spontaneous. The aim of this is to stop the bleed once it has started. Each bleeding episode must be promptly treated by an intravenous (into a vein) infusion of the clotting factor which is deficient. Once the bleeding stops, pain rapidly diminishes and use of the limb returns. The requirement for rapid treatment has meant that the vast majority of treatment in those with severe haemophilia are treated at home where the person with haemophilia or the parent has been trained to administer the treatment. Planned on-demand treatment may also be used prior to some dental treatment, surgery, or childbirth.
  • As prophylaxis – This is treatment taken regularly, to prevent bleeding from an injury before it requires further interventions. This is where the most advances have been made in the bleeding disorder community to make it easier to protect from bleeding and reduce the impact of even 1 or 2 bleeds a year, on long term joint movement and pain.

Historically, the options for treatment were very simple. It was replacement of the missing clotting factor with a manufactured concentrated version of the factor that was missing. In some products, that are manufactured, the treatments are harder to separate so the treatment may have more than just the one clotting factor in the treatment. Haemophilia A and B now have factor concentrates which stay in the body for longer called extended half-life (EHLs) factor concentrates. Haemophilia A has a non-factor replacement treatment option that uses an antibody to copy the clotting function of the missing factor VIII.

It is an exciting time in the treatment of people with bleeding disorders with many products “in the pipeline” at various stages of clinical trials. This are discussed under each section if you would like more information.

Licenced Therapies

Haemophilia AHaemophilia B
Clotting Factor Concentrates  Clotting Factor Concentrates
Human plasma derived single factor Concentrate Recombinant Standard half-life (SHL)
Recombinant Extended Half -Life (EHL)  

By-Passing Agents (for Inhibitors only) Human plasma Activated Prothrombin Complex (aPCC) Recombinant rFVIIa) Non-Factor replacements (Prophylaxis only) Bi-Specific Antibodies    

Adjunct  (supplementary or other therapies[BO2] )     Adjunct Therapies DDAVP/Desmopressin (Mild Haemophilia) Tranexamic Acid Hormone treatment (Women/Girls)  
Human plasma derived single factor Concentrate Recombinant Standard half-life (SHL)
Recombinant Extended Half -Life (EHL)  

By-Passing Agents (for Inhibitors only) Human plasma Activated Prothrombin Complex (aPCC) Recombinant rFVIIa)     Adjunct Therapies Tranexamic Acid Hormone treatment (Women/Girls)  

 [BO1]Heading

 [BO2]Heading