To Stanch the Flow: Hemophilia drug curbs brain hemorrhage

There’s no effective emergency treatment for a cerebral hemorrhage. Roughly 60 percent of people who experience this so-called bleeding stroke die within a year.

A new international study, however, indicates that a drug that speeds blood clotting can reduce death and disability after a bleeding stroke, provided that the person is treated promptly. The drug limits the amount of brain tissue damaged by blood leakage, a predictor of how damaging the stroke will be.

A cerebral hemorrhage kills neurons and other brain cells at the site of the bleeding and threatens cells on the hemorrhage’s periphery. If a doctor could limit the bleeding, a patient would have a better chance of recovery, says study coauthor Stephan A. Mayer, a neurologist at the Columbia University College of Physicians and Surgeons in New York.

Mayer and a team of physicians in North America, Europe, Asia, and Australia treated 303 bleeding-stroke patients with an intravenous drug called recombinant activated factor VII (rFVIIa), which certain hemophilia patients receive under the brand name NovoSeven. The researchers gave a placebo infusion to 96 other patients with bleeding strokes. Upon admission to a hospital and 24 hours later, each participant underwent computed tomography brain scans to detect bleeding.

Patients receiving the drug had about half as much bleeding in the brain the day after admission as did those getting the placebo. Those receiving the largest of the three doses given in the study had the least bleeding, the researchers report in the Feb. 24 New England Journal of Medicine.

After 3 months, 69 percent of the people getting the placebo had died or were severely disabled, compared with 53 percent of those getting the drug.

“This is really an outstanding finding,” says neurologist Lewis B. Morganstern of the University of Michigan Medical School in Ann Arbor. “This drug holds great promise as a targeted therapy for cerebral hemorrhage.”

The drug had one serious side effect. It caused unwanted blood clots in some patients’ brains and heart arteries. This safety concern led the researchers, midway through the trial, to stop accepting people who had a history of clotting problems, such as a previous heart attack, chest pains, or blocked leg veins.

The success of stroke treatment depends on getting the patient to a hospital quickly, Morganstern says. The study participants all received rFVIIa within 4 hours of their strokes. After that time, natural clotting has already ended most bleeding, and the risks of giving a clotting agent outweigh the benefits, Mayer says.

Some physicians have proposed that surgical drainage of cerebral hemorrhages could reduce damage from bleeding strokes. But an international group of researchers reported in the Jan. 29 Lancet that bleeding-stroke patients randomly assigned to undergo drainage surgery fared no better than did similar patients who didn’t undergo that procedure.