Table 3.

Plasma concentrates and drug products used for management of inherited bleeding disorders²,⁵⁹

Concentrate drug product	Important effects	Why used	How used
Factor VIII deficiency: recombinant factor VIII concentrate	—	Genetic deficiency	Intravenous transfusion
Factor IX deficiency: recombinant factor IX concentrate
Factor V deficiency: factor concentrate
Factor VII deficiency
Factor XIII deficiency
Factor XI deficiency: plasma-derived factor XI concentrate*
Tranexamic acid	Lysine analogue that binds plasmin, preventing it from adhering to fibrin and thus preventing clot breakdown Adverse effects: dizziness and nausea/thrombosis may occur with other thrombotic risk factors	Inhibits fibrinolysis	Administered intravenously or orally, or suspended in solution for topical or mouthwash use 1000–1300 mg orally or 1000 mg intravenously 3 times per day Weight-based dosing 10 mg/kg 3 times per day
Desmopressin	Vasopressin derivative that releases stores of vWF and factor VIII from endothelial cells; can elevate plasma levels of vWF and factor VIII three- to fivefold Adverse effects: has antidiuretic hormone activity, so fluid restriction is recommended	Used primarily for vWD type 1, as endothelial cells must produce normal molecules Use in type 2 minor bleeding is valid	Administered intravenously or subcutaneously (0.3 μg/kg) for clinic or hospital-based use, or as nasal spray by outpatients (common use for heavy bleeding) A test dose to determine the size and duration of the response is recommended, as the 2 responses may vary substantially; response frequently declines with repeated use at intervals < 24–48 h
vWF concentrate†	—	vWF-containing concentrate with high-molecular-weight vWF multimers is used for treatment if desmopressin is contraindicated or if response is likely to be inadequate Has second role in hemostasis: to bind to factor VIII and protect it from premature clearance and degradation	Intravenous transfusion

^vWD = von Willebrand disease; vWF = von Willebrand factor.

^*If factor XI concentrate is not available, fresh frozen plasma can be used, as it has all the coagulation factors.

^†The size of vWF multimers and therefore their activity are regulated by the plasma enzyme ADAMTS13, which cleaves vWF in the A2 domain.