Experimental Stroke Drug Slows Bleeding, But Doesn’t Improve Recovery

A global clinical trial has found that an experimental drug designed to rapidly slow bleeding in the brain after a hemorrhagic stroke does not improve long-term recovery for patients, according to research published in The Lancet. While the drug demonstrably reduced bleeding, it didn’t translate into better functional outcomes.

What is a Hemorrhagic Stroke?

Hemorrhagic strokes occur when a blood vessel in the brain ruptures and bleeds. This bleeding can cause damage to brain tissue and lead to long-term disability or death. Spontaneous intracerebral hemorrhage (ICH) is a common type of hemorrhagic stroke.

The Trial and Recombinant Factor VIIa

The trial, conducted across 93 hospitals in six countries, investigated recombinant factor VIIa (rFVIIa). RFVIIa is a medication designed to accelerate blood clotting. Researchers tested whether administering rFVIIa within two hours of a spontaneous ICH could improve patient outcomes after 180 days. More than 600 patients participated, receiving either rFVIIa or a placebo.

Key Findings of the Study

• Reduced Bleeding: Imaging showed that rFVIIa significantly slowed the growth of both intracerebral and intraventricular hemorrhages.
• No Functional Improvement: The modified Rankin Scale, a standard measure of stroke disability, showed no difference in outcomes between the rFVIIa and placebo groups at 180 days.
• Thromboembolic Risks: Life-threatening thromboembolic events (blood clots) occurred in 15 patients in the treatment group, compared to four in the placebo group.

Expert Perspective

“We were able to reduce bleeding, but that wasn’t enough to improve patients’ long-term outcomes,” said Andrew M. Naidech, MD, MSPH, professor in the Ken & Ruth Davee Department of Neurology, Division of Neurocritical Care, and a co-author of the study. Northwestern News He emphasized that stopping the hemorrhage is only one aspect of a complex recovery process.

Naidech explained that brain damage often occurs very quickly, even before treatment can be administered. He also noted that the location of the hematoma (blood clot) and preventing secondary brain injury—such as swelling and inflammation—are crucial factors in recovery.

Future Research

Despite the trial’s overall findings, researchers believe rFVIIa may still benefit a specific subgroup of patients: those with extremely rapid, ongoing bleeding. Further research will focus on identifying these patients and determining if a targeted approach could improve outcomes.

Funding

The trial was funded by the National Institute of Neurological Disorders and Stroke, the Japan Agency for Medical Research and Development, and Novo Nordisk, the manufacturer of recombinant factor VIIa.