Stimate for Hemophilia A

Stimate is a nasal spray marketed by CSL Behring to control bleeding in patients with mild forms of hemophilia. It contains a man-made form of desmopressin acetate — also called antidiuretic hormone — which is produced by the pituitary gland. Desmopression’s primary role in the body is to conserve water by keeping the kidneys from releasing too much water into the urine. Doctors began using desmopressin to treat hemophilia when they discovered that it increases the levels of several factors involved in blood clotting.

How Stimate works

Blood levels of factor VIII and von Willebrand factor are essential players in the coagulation cascade, which is the series of chain reactions, set off by blood vessel injury, that culminates in the formation of fibrin, the protein that clumps together to form a blood clot. Upon receiving desmopressin administration, levels of both factor VIII and von Willebrand factor rise dramatically.

Scientists recognized desmopressin’s ability to increase clotting factors as early as the 1980s, but they still do not understand exactly how this happens. Desmopressin is a vasodilator, which means it widens the blood vessels, in part by stimulating reactions in the cells that line the inside of blood vessels (epithelial cells). One theory is that desmopressin triggers the release of blood clotting factors that are stored in these cells.

Stimate in clinical trials 

Doctors first used desmopressin intravenously to treat hemophilia in 1977.  A 1991 clinical trial showed it was just as safe and effective in treating bleeding episodes and preventing bleeds during surgery when administered as a nasal spray.

In 2001, an open-label multicenter trial showed that high-dose Stimate safely and effectively controlled bleeding in patients with mild to moderate hemophilia, results that led the U.S. Food and Drug Administration to eventually approve the drug.

Additional information

Stimate is prescribed for patients with mild to moderate hemophilia A or von Willebrand’s disease whose factor VIII levels exceed 5 percent. Because the response to Stimate varies from one patient to the next, hemophiliacs should be given a test dose to make sure the levels of clotting factors in their blood rise enough to effectively control bleeding.

Stimate can reduce sodium levels in the blood — a condition known as hyponatremia — because of its action on the kidneys, so it should be used cautiously in people already at risk for hyponatremia.

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