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Rare Factor Deficiencies

A number of rare bleeding disorders require specialized care
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Clotting factors are proteins in the blood that help control bleeding. When a clotting factor is missing or does not work properly, the coagulation process malfunctions and bleeding continues for longer than it should. Many people are familiar with factor VIII deficiency (hemophilia A) and factor IX deficiency (hemophilia B). But there are a number of other rare bleeding disorders. As with factor VIII and factor IX deficiencies, people with these rare disorders can find treatment at a hemophilia treatment center (HTC) under the care of a hematologist, and can find support at their local bleeding disorders chapter. Below is a brief overview of rare clotting factor deficiencies:

Clotting Factor
I

Other Names
Fibrinogen deficiency

Three variations:

Afibrinogenemia: Fibrinogen is absent.

Hypofibrinogenemia: Some protein with normal structure is present, but it’s below levels needed for effective clotting.

Dysfibrinogenemia: A normal amount of fibrinogen is manufactured by the liver, but it doesn’t clot properly.

Incidence
Afibrinogenemia: 1 in 1 million

Hypofibrinogenemia: not available

Dysfibrinogenemia: 1 in 1 million

• Affects men and women equally

Bleeding Severity
Usually mild, except in afibrinogenemia

Treatment
• RiaSTAP™ fibrinogen concentrate
• Cryoprecipitate
• Fresh frozen plasma


Clotting Factor
II

Other Names
Prothrombin deficiency

Incidence
1 in 2 million

• Affects men and women equally

Bleeding Severity
Usually mild

Treatment
• Prothrombin complex concentrate
• Fresh frozen plasma


Clotting Factor
V

Other Names
Labile factor deficiency, proaccelerin deficiency, Owren’s disease, parahemophilia

Incidence
Fewer than 200 cases have been documented worldwide

• Affects men and women equally

Bleeding Severity
Usually mild

Treatment
Fresh frozen plasma


Clotting Factor
VII

Other Names
Labile factor deficiency, proconvertin deficiency, Alexander’s disease

Incidence
1 in 500,000

• Affects men and women equally

Bleeding Severity
Severe when factor levels are low

Treatment
• Recombinant factor VIIa (rFVIIa)
• Prothrombin complex concentrate
• Fresh frozen plasma


Clotting Factor
X

Other Names
Stuart-Prower factor deficiency

Incidence
1 in 1 million

• Affects men and women equally

Bleeding Severity
Moderate to severe when factor levels are low

Treatment
• Coagadex®
• Prothrombin complex concentrate
• Fresh frozen plasma


Clotting Factor
XI

Other Names
Hemophilia C, plasma thromboplastin antecedent (PTA) deficiency, Rosenthal syndrome

Incidence
1 in 100,000

• Affects men and women equally

Bleeding Severity
Mild to moderate when factor levels are low

Treatment
• Antifibrinolytic drugs
• Fibrin glue
• Fresh frozen plasma


Clotting Factor
XII

Other Names
Hageman factor deficiency

Incidence
1 in 1 million

• Affects men and women equally

• More common in Asians than other ethnic groups

Bleeding Severity
Mild

Treatment
No treatment required


Clotting Factor
XIII

Other Names
Fibrin stabilizing factor deficiency

Incidence
1 in 5 million

• Affects men and women equally

Bleeding Severity
Severe

Treatment
• Corifact®
• Tretten®