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Pharmacology · Cardiovascular drugs: emicizumab
Sample itemA boy with severe haemophilia A and high-titre factor VIII inhibitors has recurrent joint bleeds despite bypassing agents. Weekly subcutaneous emicizumab is started. How does it restore haemostasis?
Original practice item, not a recalled exam question. Nothing here is recorded.
The answer and the reasoning
C. A bispecific antibody that bridges factors IXa and X in place of factor VIIIa
Emicizumab is a humanised bispecific antibody that binds activated factor IX and factor X, mimicking the cofactor role of factor VIII. Because it does not resemble factor VIII, inhibitors do not neutralise it. It is given subcutaneously as prophylaxis, and combining it with activated prothrombin complex concentrate has caused thrombotic microangiopathy.
Option by option
A. Inhibition of fibrinolysis by blocking plasminogen binding
Blocking plasminogen binding is the action of tranexamic acid.
B. Inhibition of antithrombin synthesis in the liver
Reducing antithrombin synthesis is the action of the siRNA drug fitusiran.
C. A bispecific antibody that bridges factors IXa and X in place of factor VIIIa (correct)
Correct. Emicizumab bridges IXa and X and so substitutes for factor VIIIa.
D. Replacement of factor VIII in a form the inhibitors cannot neutralise
It is not a factor VIII replacement, and inhibitors are why replacement fails.
Source note: Original NextPrep sample item — pending expert review. This item is original practice material awaiting independent expert review, so confirm important facts in a standard textbook.
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