http://www.nytimes.com/2014/11/19/health/study-tempers-promise-of-drug-for-marfan-syndrome.html 2014-11-18 19:02:54 Study Tempers Promise of Drug for Marfan Syndrome The new study found there was no difference in the aortic growth rate between those taking losartan and those taking the standard treatment. === The idea seemed compelling: a Now the more objective human evidence is in – results from a large clinical trial testing the experimental drug against the standard treatment. There was no difference in outcomes. The three-year Half the participants got the test drug, losartan, and the others got a different type of blood pressure drug, atenolol, which slows the heart rate. Atenolol had never been tested against a placebo but many Marfan’s specialists are convinced that it or similar drugs in its class, beta blockers, slow aortic growth. The reason, they say, is that comparisons of Marfan’s patients who took a beta blocker to patients outside of studies who did not, indicated aortic growth was slower in people taking the drug. That, though, is considered a weak form of evidence because it is hard to know, without randomizing people to a drug or placebo, if the two groups are comparable. The new study comparing losartan and atenolol found there was no difference in the aortic growth rate between those taking losartan and those taking atenolol. Study investigators believe it was slower in both groups than it would have been without the drugs because the rate that the aorta was growing declined in both groups. But without a control group taking a placebo – something parents who agreed to have their children participate adamantly opposed – it is impossible to know if the drugs were responsible for that effect. Studies like this are difficult said Dr. Harlan M. Krumholz, a Yale cardiologist who was not associated with it. Growth of the aorta was assessed with scans but those measurements can be imprecise and it can be easy to miss an effect. “This study is most relevant to people who have decided to take drugs and are deciding which one is best,” he said. In Dr. Hal C. Dietz, a principal investigator “I won’t pretend I’m not disappointed that the results of this trial are much more nuanced than I expected they would be,” Dr. Dietz said. “But hanging my head low is not the right approach.” He said he is encouraged because, in both groups, growth of the aorta slowed and because the effect was more pronounced in younger children. “That should provide incentive to provide drugs as early as possible in the course of the disease,” he said. For now, he added, “losartan has emerged as a viable treatment option.” A co-author, Dr. Reed E. Pyeritz of the University of Pennsylvania, was more circumspect. “I am left with a good news and not so good news perspective,” he said. He is encouraged that beta blockers, used for decades, appeared to protect the aorta because its growth slowed down, and that losartan might be just as good. But the not so good news, he added, is that the “perhaps irrational expectations” that losartan would be the answer for Marfan syndrome “were incorrect.”