A popular blood-pressure medication has shown promise as a therapy for Marfan syndrome, slowing and, in some cases, stopping the enlargement of a major blood vessel that can lead to fatal ruptures, Johns Hopkins researchers reported today.
Although they cautioned that their study was small and the results preliminary, the scientists at Hopkins' School of Medicine say the drug could reduce the need for the open-heart operations that many patients need to stay alive.
"The results are telling me that there is the potential to prevent aortic disease for a lifetime," said Dr. Harry Dietz, the Hopkins geneticist and cardiologist who led the study appearing in today's New England Journal of Medicine. The aorta is the body's largest artery, carrying blood from the heart to other parts of the body.
In a study of 18 patients, the aorta enlarged by an average of half a millimeter a year after the drug, losartan, was given - compared to an average of 3.5 millimeters before.
"Everyone showed some degree of reduction," said Dietz, adding that 10 patients showed no enlargement at all.
Though a much larger trial is under way to compare losartin with conventional therapy, Dietz said he decided to give the drug to a small group of children with severe disease who were not responding to the drugs they were taking. Those children were at grave risk of complications and faced the possibility of numerous heart operations. (Surgeons replace the damaged section of a patient's aorta with a Dacron tube. But as children age, they often need repeated operations to keep up with the aorta's growth.)
Though he called results of the small study "dramatic," Dietz said doctors should not routinely switch patients to losartin until the larger study is complete and its results known. That could be six years away.
"I personally am not telling all my patients that they should switch to losartin," he said. "I think if patients have not responded well to other therapy and are getting into a desperate situation, I might, in consultation with the family, reach the decision that this is worth trying."
Afflicting 40,000 people in the United States, Marfan is an inherited defect of connective tissue that causes people to be unusually tall and lanky and suffer from dislocated lenses and enlarged aortas. Aortic tears and ruptures can be fatal and are the most serious medical complication.
