Public Release: 

Investigational drug may offer another option to treat Marfan syndrome

Saint Louis University author of New England Journal of Medicine srticle says medication choice depends on patient

Saint Louis University

A new treatment for Marfan syndrome, a rare genetic disease that can lead to heart problems, works as well as the currently recommended medical therapy, beta blockers, according to an article in the New England Journal of Medicine.

Angela Sharkey, M.D., professor of pediatrics at Saint Louis University, and a study author, said researchers found losartan, which had been more effective in an animal model of Marfan syndrome, was equally effective to a high dose of the beta blocker atenolol.

"While there may be certain patients who respond better to one drug or another, we found no evidence that losartan is superior to atenolol, a beta blocker currently prescribed for Marfan syndrome," said Sharkey, who was honored earlier this year as the Marfan Foundation's Hero with a Heart. "Losartan appears to be a reasonable alternative treatment for patients who can't take beta blockers, which could give physicians another option to treat a rare and debilitating genetic disease."

Both medications are designed to relax the blood vessels so the heart doesn't have to work as hard to pump blood through the body. Atenolol slows the heart rate, which decreases blood pressure and losartan prevents certain natural substances in the body from tightening the blood vessel walls.

The multi-site, NIH-funded trial followed 608 patients between ages 6 months and 25 years who had enlarged aortas (the main artery carrying blood to the body) for three years. All received either losartan, the investigational medication, or a higher dose of atenolol than is typically prescribed.

Patients in both treatment groups showed no difference in the rate of growth of their aortas.

Additionally, the incidence of aortic-root surgery, aortic dissection, or death did not differ between treatment groups.

Affecting 1 in 5,000 people, Marfan syndrome is a disease that affects the body's connective tissues. Those who have Marfan syndrome typically are tall and thin, with long limbs. Marfan syndrome can take a toll on many different parts of the body including the heart, blood vessels, bones and eyes.

The most serious cardiac issue, enlargement of the aorta, can be life-threatening as it can lead to dissection or tearing of the aorta. While there is no cure for Marfan syndrome, the cardiac aspects of the disease are managed with medication and prophylactic surgical therapy.


The lead author of the research paper is Ronald Lacro, M.D., Boston Children's Hospital. Other members of the research team include Harry Dietz, M.D., Johns Hopkins University School of Medicine and McCusick-Nathans Institute for Genetic Medicine; Lynn Sleeper, Sc.D. and Shan Chen, New England Research Institutes; Anji Yetman, M.D., Primary Children's Medical Center and the University of Utah; Timothy Bradley, M.B., Ch.B., and Elizabeth Radojewski, The Hospital of Sick Children; Steven Colan, M.D., Children's Hospital Boston; Gail Pearson, M.D., Sc.D., Victoria Pemberton and Mario Stylianou, Ph.D., National Institutes of Health; Tierney Selamet, M.D., Elif Seda, Lucile Salter Packard Children's Hospital; Jami Levine, M.D., and Gloria Klein, Boston Children's Hospital; Andrew Atz, M.D., Medical University of South Carolina; D. Benson, M.D., Children's Hospital of Wisconsin; Alan Braverman, M.D., Washington University School of Medicine; Julie De Backer, M.D., Ph.D., Ghent University Hospital, Centre for Medical Genetics; Bruce Gelb, M.D., Mount Sinai School of Medicine, Child Health and Development Institute; Paul Grossfeld, M.D., Rady Children's Hospital; Wyman Lai, M.D., Children's Hospital of New York; Aimee Liou, M.D., Texas Children's Hospital; Bart Loeys, M.D., Antwerp University Hospital and the University of Antwerp; Larry Markham, M.D., Vanderbilt School of Medicine; Aaron Olson, M.D., Seattle Children's Research Institute; Stephen Paridon, M.D., Children's Hospital of Philadelphia,; Mary Pierpont, M.D., Children's Hospital and Clinics of Minnesota; Reed Pyertiz, M.D., Ph.D., University of Pennsylvania School of Medicine; Mary Roman, M.D., Weill Cornell Medical College; Stephanie Wechsler, M.D., Duke University Medical Center; Luciana Young, M.D., Anne & Robert H. Lurie Children's Hospital of Chicago; and Lynn Mahony, M.D., University of Texas Southwestern Medical Center.

The paper was presented at the American Heart Association meeting on Tuesday, Nov. 18 and is slated to appear in the print edition of the New England Journal of Medicine on Thursday, Nov. 27.

Established in 1836, Saint Louis University School of Medicine has the distinction of awarding the first medical degree west of the Mississippi River. The school educates physicians and biomedical scientists, conducts medical research, and provides health care on a local, national and international level. Research at the school seeks new cures and treatments in five key areas: infectious disease, liver disease, cancer, heart/lung disease, and aging and brain disorders.

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