A drug designed to rapidly slow bleeding in the brain after a hemorrhagic stroke does not improve patients’ long-term recovery, a global clinical trial found, according to research. published In Lancet magazine.
The trial, conducted at 93 hospitals in six countries, tested whether administering recombinant factor VIIa within two hours of spontaneous intracerebral hemorrhage (ICH) improved patient outcomes after 180 days.
Despite measurable biological effects, the researchers found no improvement in functional recovery compared to placebo.
“While we were able to reduce bleeding, it was not enough to improve long-term outcomes for patients.” Andrew M. Neidek, MD, MSc Ph.D.Ken and Ruth Davey Undergraduate Professor Neurologydivision neurocritical carewas a co-author of the study. “This confirms that stopping the bleeding is only part of a more complex problem.”
More than 600 patients participated in the study and were randomly assigned to receive either recombinant factor VIIa or an identical placebo. All doses were administered within 2 hours of symptom onset. This is one of the earliest treatment durations attempted in the ICH study.
There were no differences in outcomes between groups at 180 days on the modified Rankin Scale, the gold standard measure of stroke disability.
Although functional outcome did not improve, imaging studies showed that recombinant factor VIIa significantly slowed the growth of both intracerebral and intraventricular hemorrhages.
Still, the treatment came with risks. Life-threatening thromboembolic events occurred in 15 patients in the treatment group compared with 4 in the placebo group.
“Drugs that promote clotting carry a risk of blood clots,” says Dr. Neidek. “What we still need to determine is whether there are subgroups where the risk of bleeding is so high that the potential benefit outweighs the risk.”
Neidek said several factors could explain why the drug’s biological effects did not translate into improved recovery. Brain injury from ICH often occurs within minutes before treatment is possible, and the location of the hematoma may be more important than its size. Additionally, treatments beyond coagulation control are required to prevent secondary damage (swelling, inflammation, tissue toxicity).
“ICH is not just about blood volume,” says Naidek. “What matters is where that blood is, how the brain responds, and how quickly irreversible damage develops.”
Although the trial did not show broad clinical benefit, Neidek said the drug could still help a narrow range of patients.
“We believe that some patients who are bleeding very rapidly may still benefit,” he said. “That’s where the research is going next.”
The study 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.