Treatment options

Factor concentrates
When they are available, factor concentrates are the ideal and safest treatment for rare bleeding disorders. Unfortunately, individual concentrates are available only for factors I, VII, VIII, XI, and XIII. Factor concentrates for rare bleeding disorders are usually made from human plasma and are treated to eliminate viruses like HIV and hepatitis B and C. Recombinant factor VIII and recombinant factor VIIa are also available. They are made in the laboratory and not from human plasma, so they carry no risk of infectious disease. Factor concentrates are administered intravenously.

Prothrombin complex concentrate (PCC)
This concentrate is made from human plasma and contains a mixture of clotting factors, including factors II, VII, IX, and X (however, some products do not contain all four factors). PCC is suitable for individual deficiencies of factor II and X as well as inherited combined deficiency of the vitamin K-dependent clotting factors (VKCFD). It is treated to eliminate viruses like HIV and hepatitis B and C. Some PCCs have been reported to cause potentially dangerous blood clots (thrombosis). PCC is administered intravenously.

Fresh frozen plasma (FFP)
Plasma is the portion of blood that contains all the clotting factors, as well as other blood proteins. FFP is used to treat rare bleeding disorders when concentrates of the specific factor that is missing are not available. FFP is the usual treatment for factor V deficiency. However, it usually does not undergo viral inactivation, so the risk of transmission of infectious diseases is higher. Viral-inactivated FFP is available in some countries and is preferable. Circulatory overload is a potential problem with this treatment: since the concentration of each coagulation factor in FFP is low, a large volume of it must be given over several hours in order to achieve an adequate rise in factor level. This large amount of FFP needed can overload the circulatory system and stress the heart. Other complications of treatment with FFP can occur, particularly allergic reactions or lung problems (transfusion-related lung injury [TRALI]). These problems are much less common if viral-inactivated pooled FFP is used. FFP is administered intravenously.

Cryoprecipitate
Made from human plasma, cryoprecipitate contains factor VIII, fibrinogen (factor I), and a few other proteins important for blood clotting. Cryoprecipitate does not undergo viral inactivation and should only be used when factor concentrate is not available. It contains higher concentrations than FFP of some (but not all) coagulation factors, so less volume is needed. It is only suitable for a few deficiencies. Cryoprecipitate is administered intravenously.

Desmopressin
Desmopressin is a synthetic hormone that raises the levels of factor VIII in patients with combined factor V and factor VIII deficiency. Since it is man-made, there is no risk of transmission of infectious diseases. Desmopressin has no effect on the levels of any of the other coagulation factors. It can be administered intranasally or intravenously.

Antifibrinolytic drugs
The antifibrinolytic drugs tranexamic acid and aminocaproic acid are used to hold a clot in place in certain parts of the body, such as the mouth, bladder, and uterus. They are also very useful in many situations, such as during dental work, but are not effective for major internal bleeding or surgery. Antifibrinolytic drugs are particularly useful for patients with factor XI deficiency. They are also used to help control excessive menstrual bleeding. Antifibrinolytic drugs can be administered orally or by injection.

Fibrin glue
Fibrin glue can be used to treat external wounds and during dental work, such as a tooth extraction. It is not used for major bleeding or surgery. It is applied to the bleeding site.

Platelet transfusions
Platelets are small blood cells that are involved in the formation of blood clots and the repair of damaged blood vessels. Certain clotting factors, including factor V, are stored in small sacs inside them. Platelet transfusions are sometimes used to treat factor V deficiency.

Vitamin K
Treatment with vitamin K (either in pill form or by injection) can help control symptoms of inherited combined deficiency of the vitamin K-dependent clotting factors (VKCFD). However, not everyone responds to this type of treatment. People who do not respond to vitamin K and have a bleed or need surgery will need factor replacement.

Hormonal Contraceptives
Hormonal contraceptives (birth control pills) help control menstrual bleeding.

Intra-uterine devices (IUDs)
Intra-uterine devices (IUDs) help control menstrual bleeding.

These treatments may have side effects, so people with rare clotting factor deficiencies should talk to their physician about possible side effects of treatment.


Content developed by the WFH von Willebrand Disease and Rare Bleeding Disorders Committee

 

Updated May 2012

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