Investigators were stunned to find that reducing bleeding in the brain by just one teaspoon of blood improved the chances of patient survival by nearly 40 percent.

The multi-center, international study, led by Columbia University Medical Center researchers at NewYork-Presbyterian Hospital/Columbia, shows that recombinant activated factor VII (rFVIIa) has the potential to be a significant advance in treating bleeding stroke (acute intracerebral hemorrhage, or ICH).

The work is published in the February 24 issue of the New England Journal of Medicine.

Current Treatments Not Effective

ICH is the deadliest and most disabling type of stroke -- more than one out of three ICH patients die within one month of onset and only 20% regain functional independence. Physicians have long been frustrated by a lack of effective therapies to improve survival outcomes or recovery; current options are only supportive.

RFVIIa currently is entering phase III trials as an investigational treatment for ICH. If approved by the U.S. Food and Drug Administration, it will become only the second emergency treatment for stroke in more than three decades. The first was TPA, approved by the FDA in 1996.

“Current medical and surgical options for ICH are not effective. Thirty-five to 50 percent of patients die within one month, and if they survive, they live with serious neurological deficits. Current data suggest a possible change in our paradigm for the treatment of ICH,” says Stephan A. Mayer, M.D., the study's principal investigator. Dr. Mayer is an associate professor of neurology and neurosurgery at Columbia University College of Physicians and Surgeons, and director of the Neurological Intensive Care Unit at NewYork-Presbyterian Hospital/Columbia.

Stunning Results

“I approached this study with modest expectations, expecting to see some trends toward improved outcomes,” notes Dr. Mayer. “I was stunned to find that by preventing just 5 ml of additional blood -- about one teaspoon -- of bleeding in the brain, we were able to increase chances of patient survival by nearly 40%.”

The third highest cause of death in the United States, stroke is the leading source of permanent disability, resulting in significant worsening of quality of life for patients and costing billions of dollars to society. ICH affects 15% of all stroke victims in the U.S. -- about 70,000 cases a year. In other parts of the world, the incidence of bleeding stroke is even higher. In Asia, ICH affects 30% of all stroke victims.

With 73 sites in 20 countries, this study represents the largest clinical trial ever conducted with a pharmacologic agent for the treatment of ICH. As a physician treating patients over many years with this deadly form of stroke, Dr. Mayer decided to investigate rFVIIa as a treatment for ICH after reading about its coagulation effects. With the brand name "NovoSeven," rFVIIa has been on the U.S. market since 1999 as a treatment for hemophilia, and is manufactured by Novo Nordisk.