New Finding! Hemophilia Drug Fights Deadliest Stroke

Positive results from a major international trial suggest that a drug used to treat 1 hemophilia may reduce deaths and disability from a hemorrhagic stroke, in which a blood vessel in the brain bursts.

Patients given the drug, called recombinant activator factor VII (rFVIIa), within four hours of a hemorrhagic stroke had a significantly increased chance of survival and less disabling brain damage than those who were not given the treatment.

Burst blood vessels are responsible for just 10% to 15% of strokes; the remainder are caused by clots that block blood vessels in the brain. However, more than 60% of patients who have hemorrhagic strokes die within a year, despite the best treatments currently available. Many of those deaths occur within the first hours following the stroke.

Origins Of The Study

The difficulty of successfully treating people who had hemorrhagic strokes prompted this trial, says Dr. Stephan A. Mayer, an associate professor of neurology and neurosurgery at Columbia University Medical Center in New York City, "I would be called to the emergency room to see a patient with an acute cerebral hemorrhage," he says. "I would sit and watch them go down the drain before my eyes, vomiting and sweating before they went into a coma. It was clear that many were experiencing active bleeding into the brain."

Marketed in the United States for the treatment of hemophilia, rFVIIa, (NovoSeven) caught Mayer's attention. The drug helps combat the blood disorder by improving blood clotting ability, stopping the uncontrolled bleeding that characterizes hemophiliacs.

Mayer thought the drug could take a normal coagulation system and supercharge it, so I saw the potential as an ultra-early treatment to arrest active bleeding in the brain."

He called Novo Nordisk, the Danish pharmaceutical company that markets NovoSeven, to propose a trial, and they flew someone over from Copenhagen right away, Mayer says. He describes what followed as an ideal example of how academia and industry can work together."

The Study

The trial included 399 stroke patients treated at 73 sites in 20 countries. Some were given a placebo, while others received different doses of rFVIla along with their treatment.

By 90 days post-stroke, patients who received rFVila experienced a 38% drop in mortality compared with patients who did not receive the drug. Furthermore, close to three times more patients using rFVIIA survived their stroke with no major neurological damage (24%) compared with those taking a placebo (8%).

In general, patients who got the largest dose of rFVIIa did the best, although there was an increased risk of heart attack and blockage of brain arteries in high-dose patients. Those problems call for more trials before the treatment can be approved for general use, says Dr. Lewis B. Morgenstern, director of the stroke program at the University of Michigan Health System.

However, he adds, "These results are to be celebrated because, with future studies, we are on the brink of the first targeted therapy for cerebral hemorrhages."

Right now, "physicians tend to give up early on patients with intracerebral hemorrhage," Morgenstern says. "Doctors say there is a lot of blood in the brain, and families agree to withdraw care."

A Treatment Revolution

Mayer and Morgenstern agree that stroke treatment is in a revolutionary period, with more medical centers establishing units that are devoted to the problem.

"The whole idea of trying to treat patients with stroke is relatively new," Mayer says. "In 30 years of research trying to find treatments, we had only one success-tissue plasminogen activator (LPA) for ischemic stroke," he adds.

Timely use of tPA has improved the survival of individuals who have strokes caused by ischemia (blocked arteries) by approximately one-third, Mayer notes.

A trial aimed at getting US approval for the routine use of rFVIIa may provide the results these and other experts hope for. But, until then, there is nothing to prevent physicians from using the drug "off label to treat hemorrhagic strokes, Mayer says.