New Treatment For Marfan’s Syndrome Shows Promise

An investigational treatment for Marfan’s syndrome is as effective as the standard therapy at slowing enlargment of the aorta, new research shows (read more). The findings indicate a second treatment option for Marfan’s patients, who are at high risk of sudden death from aortic dissection. “For years, standard medical therapy for Marfan’s syndrome consisted of giving patients beta blockers, which lower heart rate and blood pressure, reducing stress on the wall of the aorta,” said study co-author Alan C. Braverman, MD, a cardiologist at Washington University School of Medicine in St. Louis. “The new study suggests that we have a second option for patients that appears to be as effective as standard treatment.”

The second option is Losartan, an angiotensin receptor blocker. Past research in mice and smaller clinical trials suggested that this class of drugs might actually be superior to beta blocker treatment for Marfan’s syndrome. Angiotensin receptor blockers commonly are prescripted to treat high blood pressure. Investigators in the Pediatric Heart Network of the National Institutes of Health (NIH), including Braverman and senior author Ronald V. Lacro, MD, a cardiologist at Harvard Medical School and Boston Children’s Hospital, conducted a clinical trial comparing the beta blocker Atenolol with Losartan. After following participants for three years, the invetigators reported no differences between the two gropus in the rate of dilation of aorta, and observed similar rates of dissection in the aorta, and no difference in the number of deaths between the two groups. “This trial demonstrated that Marfan’s patients treated with either Atenolol or Losartan had very slow rates of aortic grown, and each group tolerated their medications well,” said Braverman, who treats patients at Barnes-Jewish Hospital. “While beta blockers may be the gold standard for this condition, these results suggest we must use effective doses. This is also an important alternative therapy for the smaller number of patients who are intolerant to beta blockers.”

The study appeared online in the New England Journal of Medicine.