Summary
Several biological medicines derived from human and animal plasmas can effectively
improve haemostasis in individuals with inherited or acquired defects in haemostasis.
Factor VIII and factor VIII/vWF and factor IX concentrates are used to treat haemophilia
A, von Willebrand disease and hemophilia B respectively. Cryoprecipitates are used
to treat hypofibrinogenemia and von Willebrand disease where desmopressin (DDAVP)
is ineffective or when plasma-derived factor VIII/vWF concentrates are unavailable.
Thrombin-containing topical haemostatic agents and fibrin sealants are used to control
perioperative bleeding. Intravenous immunoglobulin has several uses, including management
of patients with autoimmune thrombocytopenias and patients with acquired factor VIII
deficiency. Similar to most protein- based biological medicines, all the above products
can elicit some level of antibody response, with clinical consequences that vary from
mild anaphylaxis to loss of product efficacy. An ongoing potential safety concern
with any biological medicine derived from blood/plasma is transmission of blood-borne
pathogens. This safety concern has lessened significantly in the past decade as a
result of the institution of more effective pre- and post-donation screening that
tests for potential pathogens, and institution of pathogen reduction strategies to
which many plasma-derived biological medicines are now routinely subjected. This article
considers the manufacture, standardization, clinical efficacy and adverse event profiles
of the plasma-derived biological medicines currently used to promote haemostasis in
patients with inherited or acquired functional defects in haemostasis. It also considers
approaches employed to minimize infectivity of biological medicines derived from human
and animal plasmas and to manage patients who develop antibodies (inhibitors) to clotting
factor concentrate infusions.
Keywords
Adverse events - biological medicines - cryoprecipitate - factor VIII - factor IX
- haemostasis - IVIG - thrombin