Deciding whether losartan is right for you and your family

What's the buzz about losartan?

The Marfan community is quite excited about small research studies showing that the lives of people with Marfan syndrome may be dramatically improved by taking an existing blood pressure medication, called losartan, which belongs to a class of drugs known as angiotensin II receptor blockers (ARBs). One study showed that losartan can halt or reverse growth of the aorta in Marfan-affected mice. Another research study showed that losartan can improve muscle function in mice affected with certain forms of muscular dystrophy.

Related Resources:

• Commonly Used Blood Pressure Medication Prevents Aortic Aneurysm in Mice with Marfan Syndrome, from the Johns Hopkins University School of Medicine web site.
• Common Blood Pressure Drug Treats Muscular Dystrophy in Mice, from the Johns Hopkins University School of Medicine web site.
• Studying the rare can help understand the common: summarizes a lecture by Harry "Hal" C. Dietz, October 27, 2006, Vanderbilt University Medical Center. This lecture explains in some detail how researchers discovered that a drug like losartan might be able to create vast improvements in heart, lung, and muscle tissue in Marfan patients.

What's the buzz on "The Losartan Study"?

The early research results have shown so much promise that the National Heart, Lung, and Blood Institute (NHLBI) recently began a larger-scale clinical trial to test losartan in Marfan-affected people. It is titled "Comparison of Two Medications Aimed at Slowing Aortic Root Enlargement in Individuals With Marfan Syndrome".

The trial will only be successful if enough people enroll in the study. To participate, a person must:

• Be between the ages of six months and 25 years at the time of enrollment. (200 of the 600 participants must be between 16 and 25 years of age.)
• Meet the Ghent criteria for diagnosis of Marfan syndrome.
• Have an aortic root measurement with a “z-score” of greater than 3 ( a z-score is a measurement that considers aortic size in relation to a person’s weight, height and body mass.)
• Not be pregnant.
• Not have had any previous aortic surgery.
• Be able to tolerate beta-blockers and losartan.
• Not need to take a beta blocker for some medical problem other than a dilated aorta.
• Commit and be able to have all echocardiograms done at a study site.

You can learn more about the study and find instructions for enrolling at the National Marfan Foundation web page about the losartan vs. atenolol trial. This page includes links to press releases, a Q&A, and a list of study sites. It also includes an option for you to sign up for emailed updates about the study. You may also sign up for the study by viewing the study's web page in ClinicalTrials.gov.

Is losartan right for me, right now?

Because only some of the children and young adults enrolled in the study will receive losartan, deciding whether to enroll is a difficult decision. And all Marfs who cannot or will not participate in the study are facing a difficult decision as well: If losartan is available and my doctor is willing to prescribe it for me, should I start taking it today, rather than waiting 3 to 6 years for the study results to come out?

The decision seems to hinge on two main issues: safety and time. Researchers have not yet fully tested the safety of using losartan in Marfan patients over a period of time. Researchers cannot be sure that the drug will be safe and effective, and deliver long-term results. If you choose to participate in the study, or get losartan right away from your physician, you must be comfortable with this inherent safety risk. The idea of lost time is a second major consideration. If losartan is available now (through the trial or perhaps through your physician), and can potentially prevent abnormal aortic growth, why not use it today, rather than waiting years for the research study to be completed? Anyone who chooses to avoid taking losartan until the study is complete must be comfortable with the possible health costs of waiting.

Anyone who does participate in the study will be doing a great service to the Marfan community. The study is essential to determine the safety of losartan and get it approved for widespread use.

Below are some resources you may find helpful as you decide whether to join the losartan study, get losartan from your physician, or wait it out.

About losartan:

• Medline Plus Drug Information: Losartan
• Patient Package Insert, Losartan 100mg

About clinical trials (in general):

• An Introduction to Clinical Trials, From ClinicalTrials.gov. This web page includes questions to consider before enrolling in a clinical trial ("clinical trial" is the official term used to describe studies like the losartan study). It also includes a general statement about the benefits and risks of participating in a clinical trial.

About "The Losartan Study":

• Information for Marfan Trial Patients and Their Families (children's brochure), from the Pediatric Heart Network.
• Information for Marfan Trial Patients (adults' brochure), from the Pediatric Heart Network.
• The Tough Questions about the Losartan Trial -- Hal Dietz MD Provides Personal Insights - from the National Marfan Foundation's Connective Issues newsletter, Fall 2006, pp. 5. Dr. Dietz (one of the lead researchers involved in the study) answers questions like "If your child had Marfan, would you enroll him/her in the study"?.
• National Marfan Foundation web page about the losartan vs. atenolol trial. This page includes links to press releases, Q&A, list of study sites, and has option for you to sign up for emailed updates about the study.
• ClinicalTrials.gov information about the losartan study.

Curious about Dr. Dietz's latest work? You can view some of his latest papers here.