Treatment

 

Haemophilia A Haemophilia B Von Willebrands Other clotting factor deficiencies Platelet disorders
Recombinant FVIII X
Recombinant FIX X
Plasma FVIII with vWF X*
Recombinant FVIIa X* X* X X
FEIBA X* X*
HEMLIBRA X*
Other clotting factors X
Platelets X
Fibrin glue X X X X X
Tranexamic acid X X X X X
Desmopressin X X X X
Fresh Frozen Plasma X
Cryoprecipitate X
*May be used if you have an inhibitor to Factor VIII or IX

Factor VIII and IX

Factors VIII and IX were first extracted from human blood in the 1960s and they revolutionised treatment for bleeding in people with haemophilia A and B, even allowing for patients to treat themselves at home. These concentrates were in widespread use from the 1970s until Scotland started to replace them with genetically-engineered forms (known as recombinant product) in the mid 1990s because they have a theoretically zero risk of spreading any infection.

How often to inject prophylaxis treatment
Conventional recombinant/plasma Longer-acting
Factor VIII Normally every 2 or 3 days Every 3–5 days
Factor IX Normally every 3 or 4 days Every 7–14 days


The latest development in clotting factor treatment is the preparation of longer-acting concentrates. If on prophylaxis you infuse these less often than conventional Factor VIII and IX concentrates.
Although ‘recombinant’ is the treatment of choice for severe haemophilia A and B, some people continue to prefer concentrates that have been made from plasma (the liquid part of human blood). If you have von Willebrands you may need to use a plasma-derived Factor VIII that also contains a good amount of von Willebrand factor. This type of Factor VIII can also be useful if you have haemophilia A with an inhibitor. You can use all these treatments on demand (when bleeding), or for prophylaxis.

Treatments for inhibitors to Factor VIII and IX: NovoSeven, FEIBA and HEMLIBRA

Recombinant Factor VIIa treatment (the ‘a’ stands for ‘activated’) is better known by its brand name, NovoSeven®. It is mainly used to treat bleeds or as pre-surgery prophylaxis in people with an inhibitor to Factor VIII or IX. It is also licensed for factor VII deficiency and the platelet disorder, Glanzmann’s thrombasthenia.
FEIBA (pronounced “Feeba”) is a concentrate made from plasma that is mainly used if you have an inhibitor to Factor VIII
HEMLIBRA® is a type of medicine known as a therapeutic antibody. It can bridge activated Factor IX and Factor X in your blood to replace the function of missing activated Factor VIII in people who have an inhibitor to Factor VIII.

Other clotting factor concentrates

A range of other single clotting factor concentrates to treat rarer conditions is available, but their availability can vary over time. Currently two are recombinant – for factor VII and XIII deficiency, and the rest are made from plasma from non-UK donors – for Fibrinogen and factor VII, X, XI and XIII deficiencies. Concentrates containing four clotting factors exist (II, VII, IX and X) and are preferred if you have factor II or X deficiency.

Desmopressin

Desmopressin (also known as DDAVP) is a synthetic hormone that treats bleeds in mild (and sometimes moderate) haemophilia A and most forms of von Willebrands (types 1, 2A, 2M and 2N). Because it is not made from blood, it has always been safe from any blood-borne infections. But it does cause your body to retain water and can make your skin temporarily red and hot (flushing). It can reduce your blood pressure and even cause thrombosis (clotting). You will be given a trial dose to check how well it works and see if there are any side-effects.

Platelets

Platelet transfusions may be used if you have a severe platelet disorder. They are obtained from voluntary donors.

Fresh Frozen Plasma (FFP)

FFP, from voluntary donors, contains all the clotting factors you need and is used when one of them is not manufactured or available, or when your bleeding disorder has not been diagnosed. It is treated to greatly reduce the risk of infection.

Cryoprecipitate

Cryoprecipitate is made from plasma and contains fibrinogen, factor VIII, factor XIII and the von Willebrand factor. It is used if Fibrinogen or Factor XIII concentrates aren’t available.

Tranexamic acid

Tranexamic acid helps prevent clots break up naturally. It can be taken as a tablet, a mouthwash (before dental work) or an infusion. It is often used for mouth bleeds or before dental surgery, and by women with heavy periods. Epsilon-Aminocaproic acid (EACA) is a similar drug.

Fibrin glue

As its name suggests, fibrin glue helps clots form and is used by dentists and surgeons and other people treating bleeds.

The European Haemophilia Consortium (EHC) have produced an informative series on gene therapy. Take a look here.