Congenital fibrinogen deficiency (CFD) is a very rare, inherited bleeding disorder in which
the body’s ability to form blood clots is impaired. Depending on the severity of the deficiency,
patients might be asymptomatic or might experience uncontrolled bleeding—either spontaneously or
in response to surgery or other trauma.
Fibrinogen deficiency can be characterized as a quantitative or qualitative deficiency.*
Individuals with a quantitative fibrinogen deficiency have abnormally low levels of fibrinogen
(<200 mg/dL) circulating in their bloodstream.
A qualitative fibrinogen deficiency occurs when there is a structural defect in the fibrinogen molecule that results in functional impairment. Individuals with this type of deficiency usually have normal plasma levels of fibrinogen proteins, but those proteins do not function properly.
CFD is caused by a mutation in one of the three genes responsible for producing fibrinogen.
Because CFD is not a sex-linked genetic disorder (ie, it is autosomal), it is equally likely to occur
in males and females.
History
It wasn’t until 1920 that CFD came to the attention of the medical world. Two
German physicians, Fritz Rabe and Eugene Salomon, tested a 9-year-old boy with
lifelong unexplained bleeding problems and found a lack of fibrinogen in his
blood. Nearly a century later, available treatment in the United States was
still limited to fresh frozen plasma, 24-hour plasma, or cryoprecipitate. With
all of these products, the exact amount of fibrinogen is unknown. Not until 2009
was a fibrinogen concentrate approved for use in the US market.
Important Safety Information
RiaSTAP®, Fibrinogen Concentrate (Human), is contraindicated in patients with known anaphylactic or severe systemic reactions to human plasma-derived products.
Monitor patients for early signs of anaphylaxis or hypersensitivity reactions and if necessary, discontinue administration and institute appropriate treatment. Thrombotic events have been reported in patients receiving RiaSTAP; weigh the benefits of administration versus the risks of thrombosis.
RiaSTAP is made from human blood. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent and its variant (vCJD), cannot be completely eliminated.
The most serious adverse reactions observed are thrombotic episodes (pulmonary embolism, myocardial infarction, deep vein thrombosis) and anaphylactic reactions. The most common adverse reactions observed in clinical studies (frequency >1%) were fever and headache.
Indications
RIASTAP is indicated for the treatment of acute bleeding episodes in pediatric and adult patients with congenital fibrinogen deficiency, including afibrinogenemia and hypofibrinogenemia.
Please see full prescribing information for RiaSTAP.
To report SUSPECTED ADVERSE REACTIONS, contact the CSL Behring Pharmacovigilance Department at 1-866-915-6958 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
