November 19, 2014
by marfmom


The Losartan Trial Results Are Out…Now What?!

By now you’ve likely read the synopsis of the atenolol vs losartan trial results. Perhaps you’ve read them from multiple sources and are a little confused. Maybe the results are what you expected and you’re happy, or maybe you’re feeling disappointed. Was the trial a success or a failure? Where do we go from here?

Here are my thoughts!

Does losartan work? Yes! The trial showed that both losartan and atenolol work. It seems like people are disappointed because the trial showed that they work the same, and their expectation was that losartan would work better. However, there was a dosing issue. Losartan was compared to a very high dose of atenolol (up to 2x the FDA recommended dose commonly used for people with Marfan syndrome). The losartan was dosed at up to the FDA maximum dose for treating people with hypertension (so not everyone in the study received the maximum dose). In the past, most people with Marfan were prescribed much less atenolol than was used in the trial. And, the amount of losartan prescribed to patients in clinics over the last few years has varied greatly.

Why is this important? If the atenolol had been dosed at a more clinical level, and losartan where it is, or at a higher level (more in line with what some doctors are using clinically), the results might have been different. All we can say from this trial is that losartan and atenolol work equally well at the particular doses from the trial. The results can’t be generalized to other doses. Another important takeaway is that for those on beta blockers, this higher dose is potentially necessary for the best effects.

Why was the atenolol dose so high? My understanding is that because we’d never had a double blind trial (where patients and the trial site administrators don’t know which drug the patients are taking) to prove the effectiveness of beta blockers before (though there were other studies!), these researchers also wanted to prove beyond a shadow of a doubt whether beta blockers were effective at managing aortic growth in Marfan patients. They definitely accomplished this, though the remaining question is whether this might have muddied the comparative effects of losartan at all.

Kids vs adults: The researchers discovered that optimum results occurred in young kids. This provides evidence for what a lot of us have been thinking: it’s important to start our kids on medication early. This makes me feel even more confident in our decision to start J on medication when he was 2.5 years old.

Placebo: The New York Times article (which, I want to point out, neglected to explain the dosing issue with this study) talked about the fact there wasn’t a placebo in this trial and seemed to question the efficacy of the results because of this. In light of the article, I’ve seen a few people asking why there wasn’t a placebo. There was no placebo because it wouldn’t have been ethical. Prior to this study, there was a standard of care for treatment: beta blockers. It’s generally frowned upon to do a high-risk study (in terms of risk to the participants; in this case, the risk of dissection) with a placebo when a standard of care exists already. And let’s be honest: this is a trial looking mostly at children. Would you have taken your child off treatment to enter a study where there was a 50% chance they’d have had NO treatment for their existing aortic aneurysm for 3 years, thereby increasing the risk of needing surgery or having a dissection during that time? No way! This study was designed to be as safe as possible for the patients enrolled and therefore it was set up to compare two drugs against each other. While some researchers might have argued a placebo would have been “better science,” it wouldn’t have been in the best interest for our community.

This study doesn’t have all the answers. It was never going to have all the answers…no single study can! But because of this, other studies are going on all over the world. Some have already been published. These studies ask other questions. They look at whether other ARBs, like irbersartan, might be better than losartan. They see whether losartan + atenolol is better than either drug alone. They study different age groups. There are also ancillary studies to the US trial, which are looking at whether or not losartan affects other body systems.

I am glad to know that losartan is a valid “tool in the Marfan toolbox.” This is an option for people who can’t tolerate beta blockers for whatever reason, and now we know a little more about dosing beta blockers. Over the next few years, we’ll learn even more about what ARBs can and can not do for us, and I am confident that other drug options will be brought forward to be tested as well. Every step forward in finding preventive treatments for aortic aneurysms is a success!

If you haven’t already, consider signing up for the interactive webinar with the study’s principal investigator, Dr. Ron Lacro of Boston Children’s Hospital. He’ll explain more about the study and take your questions. It’s a great opportunity! In addition, the Marfan Foundation will be planning another webinar with Dr. Hal Dietz shortly. The Foundation has also put together a Q&A, which you can find here.


The opinions offered at Musings of a Marfan Mom are for informational purposes only and are not intended to be a substitute for professional medical advice, diagnosis or treatment. Always seek the advice of a qualified healthcare provider with any questions you may have regarding Marfan syndrome and any medical condition. Never disregard professional medical advice or delay in seeking care because of something you have read here.

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September 10, 2014
by marfmom


Approaching Strangers: Do You, or Don’t You?

In continuing my series on conversations we have about Marfan syndrome with our kids, today’s topic is on approaching strangers who look like they might have Marfan.

The topic comes from reader Jamie, who writes:

Once you know about Marfan, you can’t help noticing people you meet in public with great height, long fingers, obviously corrected eyesight, thin limbs, etc. The condition is not *that* rare and if you live in a populated area, the odds are good that you’ll encounter someone else with Marfan — and, you might be the only person in their lives who has learned to recognize it.

Of course, a lot of people have these traits without having Marfan. Even so, warning people seems like an ethical no-brainer since their health might be in danger without them knowing it, and the costs of ruling that possibility out seem small in comparison. On the other hand, it really *feels* wrong; health is personal, and you could be stepping into a stranger’s life in a very intimate way to add a serious health concern and possibly life-changing news to whatever else they have to worry about.

I’d be curious about others’ reactions to this situation.

I’ve only asked a stranger once. I was at a party that one of my brother’s friends was hosting. I had never met the guy before, but he was so tall I couldn’t pass up the chance to ask. I followed him into the room to get more snacks, struck up a short conversation, then said something to the effect of “hey, so this is a little weird to ask, but have you heard of Marfan syndrome? I ask because I have it, and you’re tall like me so I wondered if you might have it too,” or something like that….this was when I was pregnant with the Menininho, so it’s been a few years. He ended up not having Marfan, but he wasn’t offended that I’d brought it up, and I was glad that I’d played it safe and asked.

If you have Marfan, have you approached other people you think you might have it too? What about if you’re a sibling/parent/partner? If you are a parent, have you talked to your child about approaching strangers who fit the Marfan or a related disorder phenotype?

I wanna hold your hand

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September 8, 2014
by marfmom


Talking to Your Kids About Their Diagnosis

How to tell your child they have a particular diagnosis, whether that’s Marfan syndrome, Loeys-Dietz syndrome, autism, or something else, is a question I get frequently…multiple times while writing this post, in fact! Do you tell them in pieces, or all at once? Wait till they ask? Till they’re older? Is their diagnosis part of them, or something that they fight against?

We don’t want Marfan, or autism, to be a big deal in our house. Making them into a big deal makes them seem more scary. So, we’ve never had one Big Conversation. Instead, we’ve just talked about Marfan and autism around the house since the boys were babies, in age-appropriate ways.

For example, J knows he takes medicine. We tell him “you have something called Marfan syndrome, just like Mommy! It makes you tall and stretchy! It also can make your heart and lungs more tired, so you take medicine every day to make them stronger.” Since age 3, J’s been able to identify all his medicines by which body system they’re for, and which time of day he takes them, and he reminds me if I forget one. It’s a little part of his care that he takes pride in.

We got the Marfan syndrome A-Z book, and the Menininho in particular likes for me to point out the real-life identities of our friends and doctors in it. This book (geared towards 3-6 year olds) is actually a great way to open the door to a conversation. You can download it from The Marfan Foundation (not sure if they still have print copies). There’s also a book for older kids (like 7-11 year olds) called Marfan Does Not Mean Martian, written by an affected teen.

We talk about how Marfan makes us bendy and tall, and how much fun we have with our Marfriends. M attended the kids program at conference for the first time this summer, and asked the doctors “what problems does Marfan cause and why does it cause those problems?” I don’t know how much of the answer he understood and retained, but I’m glad he felt comfortable asking the questions.

I can’t even remember any initial conversation we had with M about being autistic. I know one happened over breakfast, where we talked about how autism gives him his good memory, and is also the reason why he is sensitive to noise and touch. Now that he’s 5.5 years old, we’ve seen the fruits of our approach pay off, in how he talks about autism to us, and to other people. M is proud to say “My daddy and me both have autism, and my mommy and J both have Marfan!” He wants to find more autism friends, since we have so many Marfriends.

The boys know that they are different, regardless of whether we’d ever said anything. Even at 3.5 years old, J realizes that his older brother doesn’t take most of the same medications that he does, and we’ve already had a conversation about activity restrictions (an upcoming post). We feel it’s important that they have a name for their differences from an early age. Having a name helps them shape an identity around that, whatever they want that identity to be. As I’ve written about before, maybe Marfan or autism will be a big part of their lives, or maybe it will be a very small part. Either is ok with us.

But, knowing what they have and what that means, particularly in the case of Marfan, is the key to managing it. So at 3, for J that’s knowing he’s got heart, lung, blood, and sleepy medicine, and a vitamin. In a few years, that’ll be knowing the proper names of his meds, and a little more about what they do. He knows his echocardiogram takes pictures of his heart, and as he gets older and asks more questions, we’ll talk more in depth. He’ll guide that conversation. By high school I was scheduling my specialist appointments, and so maybe he’ll do the same. I believe that a successful transition to adult care starts early though, and that’s a very gradual process.

How do you talk about your child’s diagnosis with them? Have you?

(video is of M leading J and some Marfriends in a “Marfan cheer” at conference)