I think one, there a couple of things that I think, are important that is I need to emphasize that when we looked specifically at beta blockers we found that patients with beta blockers had exactly the same, we found no evidence for difference in patients who are on beta blockers. Now that strongly implies to us that the effects of these medications are similar in patients who take beta blockers to, to those who don't take beta blockers implying that the effects of angiotensin receptor blockers are similar to those who are taking beta blockers to are not taking beta blockers, so that implies that the effects are likely to be additive, so in other words the effects are likely to be independent of beta blockers.
And the other thing is that we, we, we, the one thing that we did find that was, that was, that was different is that the, the effects of angiotensin receptor blockers were, were different in, in patients who were on, who had FBN1 mutations, so there were some patients included in the trials who did not have FBN1 mutations and when we looked at those patients we found that we did find evidence for heterogenity for the FBN1 mutations, so patients who had FBN1 mutations at baseline were more likely to have an effect of angiotensin receptor blockers. Angiotensin receptor blockers were, you know, were particularly likely to be effective in those patients. Now that's not saying if you don't have an FBN1 mutation the ARBs won't work. They may well work but, but the ARBs had a particularly strong effect in FBN1 mutations and what that means is that, that strongly reinforces our, our belief that the angiotensin receptor blockers are effective in, in Marfan syndrome because you would really expect the drug to be most effective in people with ARB with FBN1 mutations.
The Marfan Foundation did not participate in the translation of these materials and does not in any way endorse them. If you are interested in this topic, please refer to our website, Marfan.org, for materials approved by our Professional Advisory Board.
The Marfan Foundation は、当翻訳には関与しておらず、翻訳内容に関してはいかなる承認も行っておりません。このトピックに興味をお持ちの方は、Marfan.org にアクセスし、当協会の専門家から成る諮問委員会が承認した内容をご参照ください。
So a number of trials now were, were, were then undertaken and we then organized with the, with the investigators who, who did these trials. We put these trials all together to try to give a definitive answer because the trials were confusing, you know, when, when as the trials were coming out they, they started to say kind of slightly conflicting things and it was a confusing picture and it was only really when we put the data together that a, a clear picture at least to my, to my mind emerged and I think I can hopefully explain it in a way that at least makes sense to you and I want to share it with you in a way that hopefully will make sense to to you as well. Let me, let me take you through it.
So let's, yeah, first of all I want to talk, talk you through these four trials, so there were four trials that essentially asked the same question. There was a trial from France, there was a trial from the Netherlands, there was the AIMS trial, which child was heavily involved with from the Marfan Trust and then there was the trial from Belgium, which, were all fundamentally trying to ask the same question, very similar question. What these trials were doing is they were comparing angiotensin receptor blockers with a placebo medication, so they were, they were comparing the same thing but is it better, you know, essentially they are asking the question, "Are angiotensin receptors blockers better than a, a sugar pill?" effectively.
And they, they differed by size, so this trial had 299 patients, this trial had 233 patients, this trial had 192 and this trial had 22 patients and so you might say, "Okay, well, it's not fair to compare to, to include this trial with this trial because this trial was much bigger than this trial," and if you and, if you thought that then you understand meta-analysis because we didn't add those trials together in a, in a lump and just say, "Well, okay that's one trial, that's two trials, three trials, four trials." We weighted the trials according to the amount of information that they contained and this trial contained an awful lot more information than this trial, so this trial got given more emphasis in the meta-analysis than this trial and a lot of people didn't understand that, okay?
There were certain trials that did very, very precise measurements of the aorta and there were other trials that did slightly less precise measurements. They just kind of, "Yeah, it's roughly there," and other trials really measured, it very precisely and some of the trialists said, "Well, I don't want my trial being mixed in with the guys who just kind of went, 'yeah it's roughly there'," and I said don't worry because we're going to weight the trials by the amount of information available, so if you did really precise measurements we know that and we're going to put more emphasis on your trials so that's the beauty of meta-analysis. We know which trials provide the most information and we can weight those trials accordingly, okay?
So the trials bit frequent measurements, so we got lots of, we got huge amounts of information for every participant and they were measuring the aorta at exactly the place where the aorta tends to enlarge, okay? So that's the characteristics of those trials. I'm going to skip this trial and come back to it.
We published the protocol before we knew what the trials were going to show so that there was no opportunity for us doing, you know, statistical jiggery-pokery. We, we said what we were going to do, we did it, we found the results. There was no opportunity for clever statistical messing around and we analyzed all eligible randomized trials that were done in the world that provided data to us. We used individual patient data and trials were weighted according to the information that they contain, okay.
The primary aims, that we wanted to estimate the effects of angiotensin receptor blockers and beta blockers on the change in aortic root size in patients with Marfan syndrome who had no prior aortic root surgery. There's no point in including patients with aortic root surgery because they've already had their aortas replaced with a piece of surgical material and, and so that would have affected the results in a way that was unhelpful.
So and then we had various secondary aims as well to it, to look at different subgroups, the children benefit to do, that sort of thing, so we looked at various other secondary outcomes as well, okay.
So let's look at the results, so now this looks a little complex but it's really pretty simple, so I just want to spend a minute talking you through this, so these are the names of the trials that we just talked about here's the French trial, here's the Dutch trial, here's the UK AIMS study, some of you may have been participants in that trial, here's the Belgian trial, okay and these are the names of the trials.
This is the annual change in the size of the aorta, so for each year that the participant was in the trial how much bigger did their aorta get. These are the participants who are on the drug treatment and these are the participants who are taking the sugar pill the placebo medication. If the patient, if the trial found that the angiotensin receptor blocker was better for the patient's aorta then the black dot here would be shifted over to the left hand side, side like this and if the trial found that it was actually better to be on the placebo medication then the black dot would be shifted over to this side.
And then the diamond represents the overall result, so when we looked at the results the first thing my eye went to is, "did this diamond fall on this side or did it fall on this side?" and if there was no difference the diamond would have fallen bang in the center, okay? and the further the diamond is to this side the more the ARBs work and the further to this side the more the, the controls work this side and if the diamond is completely on this side then we are, can be certain or more certain within certain confidence limits, that's a kind of statistical term, that the treatment works and what we can see is that the diamond falls completely on this side of the black line. If the diamond had been falling over the line, so half the diamonds on this side and half the diamonds on this side then it's a, it's a wash and the neither treatment works, you know, that just means the drugs do nothing.
So what we can see is the diamond falls completely on this side of the line and that tells us that the ARB is better than the the no treatment and the fact that the diamond falls completely over the line means we can be 90, well, we can have 95% confidence that, that result is true, more or less, it's not entirely right but that's basically it so, so, so that was, that was the kind of key finding of the meta-analysis.
And then the, and then the next thing to, to look at is well what how big is this effect, you know, are we, are we saying that the angiotensin receptors reduce the rate of aortic enlargement a tiny bit or is it a lot, what does that mean? Well, let, let's look at this, so per year the aorta was enlarging by 0.13 units here and if you were on treatment it was 0.07 units. Here now 0.13 and 0.07, this is about half of that 0.1, you know, if you double, if you double 0.07 you get 0.14, don't you? So .03, .14 it's about the same, so effectively what's happening is the ARB is halving the rate of at which the aorta enlarges. That's huge, okay?
So, you know, like if, if, if there was a, if there was a medication that would give me half my hair back I take it, okay? So, you know, that so, so halving the rate of hair loss would be huge for me, halving the rate of which my, a the aorta was expanding for, for you guys is really really big, okay? So that's a really big effect and I and I'm and, you know, on a statistical level I'm quite certain about it, okay? So that, that's what, that's, what that showed and then there's various different ways of, of, of looking at it in in terms of the statistics which I think I'm just going to jump over for the moment. I'll put that on there, so it's on the, on the YouTube video.
So now then the next question people always want to say is, "Oh, does it work for older people, does it work for children, does it work equally for men and for women, does it work if my parents had Marfan syndrome, did it work if I've got a big aorta to start with or a small aorta, does it work people have got high and low blood pressure, does it work if I also take a beta blocker?" These, these are all very good questions and the short answer is that we found, I'm gonna get, I'm got to get this right, otherwise my, my statistical colleagues will sort of raise an eyebrow at me and, and the correct way of saying this I think is, is to say that we explored the data for evidence of differences because of these things and we found no evidence that it made any difference at all for any of these things, okay? If I'm being properly statistically correct about it that it, that is the way I should should say it, okay? We found no evidence for any differences according to any of these subgroups. We, we looked for whether people were, you know, for children, young people under the age of 16 compared to over the 16 we found no evidence for any difference. We, we looked for whether it was different for men and for women, we found no difference, we looked for family history, we looked for people with big aorta compared to small aorta, we found no difference, okay?
I, I, I think what people would like me to say is to say that it makes no difference, that is going very slightly beyond what the data, what one can say from these types of data sets but I think I would I, I recommend these medications to men and women. I recommend it for children and for adults. I recommend it for people with high blood pressure, low blood pressure, big aortas, small aortas and if I fell into any of these categories I would be taking these meds. I'd be considering these medications for myself, for my children, for my sister, for me, my mom, my dad whether someone had a big or small aorta. That's how I would be approaching this and I've see no rational reason for denying these medications for certain people.
Some colleagues say, "I like to individualize things, I like to tailor things to the individual patient," and with very limited number of exceptions what I like to ask those colleagues is on what rational basis do you do that because I can find no rational basis for doing it apart from one or two very limited exceptions.
The Marfan Foundation did not participate in the translation of these materials and does not in any way endorse them. If you are interested in this topic, please refer to our website, Marfan.org, for materials approved by our Professional Advisory Board.
The Marfan Foundation は、当翻訳には関与しておらず、翻訳内容に関してはいかなる承認も行っておりません。このトピックに興味をお持ちの方は、Marfan.org にアクセスし、当協会の専門家から成る諮問委員会が承認した内容をご参照ください。
So I want to talk now about the two different classes of medications and we're going to dig into this in a lot of detail and people are asking really sensible questions in the Q&A and I'm going to answer all those questions in, in the slides, I hope and if I haven't we'll take them up in the Q&A, okay?
So the first class, class of drugs I'm going to talk about angiotensin receptor blockers and then we're going to talk about beta blockers and we're going to talk about who benefits from all this kind of stuff, okay?
So let's give the the top level view first of all, so angiotensin receptor blockers in Marfan syndrome, so, so, so I'm going to talk about these things called ARBs, okay? and angiotensin receptor blockers are these medications like Losartan and Irbesartan and Telmisartan and Olmesartan and anything ending in -sartan essentially and I'm just going to call these ARBs to start with, okay? and in a minute I'm going to talk about whether which, which one's best and all that kind of stuff but for the moment I just want you to think about the classes of medications and essentially the, these medications have been around for about 30 years and, and they've been used for hypertension, they've been used for patients who've had heart failure and there are many hundreds of thousands of patients throughout the world who've been prescribed these medications.
And, and the, the, the, the concept essentially is that in Marfan syndrome the problem is here, the fibrillin microfibrils are disrupted and do not work and as a consequence we think that these fibrillin microfibrils cannot effectively bind a medication called TGF-beta that means that this TGF-beta is disregulated. As a consequence there is excessive signaling through TGF-beta and that means that TGF-beta is signaling excessively and so making the, the cells in the TGF-beta in the, in the smooth muscle of the aortic wall do things they shouldn't do and causing the chemicals inside the the cells to signal excessively and this is not good for the way the cell is behaving.
And that can be resolved by giving treatment through this pathway here, which is the angiotensin receptor pathway and that if we block signaling through this pathway it is possible to interfere with this signaling pathway because these pathways here link up inside the cell. That's the concept is a little complex I, I understand.
But the evidence is here, so this, this is evidence from Dr. Hal Dietz who many of you will have heard of and many of you I'm sure will have met and listened to at various times. This is the aortic wall of a mouse, a normal mouse, so called wild type. These are mice that, you know, live in the, in the wild I guess and there they have these beautiful architecture to their aortic wall. The, the black lines are the, the lamina, the elastic walls of the aorta and then in between is where the cells live and everything here is beautifully organized.
Here is a mouse with Marfan syndrome who has just been treated with a placebo, just a sugar pill. It's not doing anything to them so this is a Marfan mouse and what you can see is the aortic wall is really thick. You might think thick is good but it's not good because what's making it thick is there's a load of expansion of stuff in between the, the good elastic walls and the elastic walls are all broken down and this is not good tissue. There's holes here, there's gaps there and this is a, you know, could tear, so this is bad aorta. This is not going to keep the aorta looking good for long. This is not good.
Here is what happens if it's treated with beta blockers, Propanolol, which is normal treatment for Marfan syndrome. It's probably a little bit better than the bad aorta but it's no any as good as the good healthy aorta.
Here's what happens when the mouse is, is treated with Losartan, the the angiotensin receptor blocker and this effectively normalize has, has prevented the mouse from developing these, these bad changes, so that, that's what happened in the mouse.
And, and here's the wall thickness. This is the normal mouse. This is the mouse with Marfan syndrome with no treatment. This is the mouse given the angiotensin receptor blocker, okay.
The Marfan Foundation did not participate in the translation of these materials and does not in any way endorse them. If you are interested in this topic, please refer to our website, Marfan.org, for materials approved by our Professional Advisory Board.
The Marfan Foundation は、当翻訳には関与しておらず、翻訳内容に関してはいかなる承認も行っておりません。このトピックに興味をお持ちの方は、Marfan.org にアクセスし、当協会の専門家から成る諮問委員会が承認した内容をご参照ください。
So, so next I just want to change topic before we come on to the, the, the, the real substance of what I want to talk about but I just want to make sure everyone's on the same page with understanding what a randomized controlled trial is. Some people, some of you are going to know all this already, some people maybe not, so I just want to make sure everyone's clear about it.
So in a randomized control, some of you will have been in randomized control trials I know, so the idea of a randomized control trial, it hasn't, you know, it hasn't been around for the whole of human history. This, this concept was invented in the 1950s. It's a British invention, I should say.
ランダム化比較試験の歴史はそれほど長くなく、考案されたのは1950年代のイギリスです。
And the idea was that you, you take participants who, who have got the disease that you want to study, let's say, patients with Marfan syndrome and then you want to test whether the treatment works compared to no treatment and so what you do is you randomly allocate, let's say, half the the patients to having the intervention that you want to test, let's say, the medication and the other half of the patients are randomly allocated to not getting the treatment and this idea that the treatment allocation had to be at random was not obvious to human beings until really about the 1980s and it's kind of amazing that this hadn't really become clear but until the 1980s.
It was extremely common for people not to be allocated at random and what would happen is the doctors would decide which group you would go into and you would think, well and the doctors used to, who did these trials before the 1980s, used to really think that they could decide in an unbiased way but they really, really couldn't and we, we have learned the hard way that, that if you want to get an unbiased group, in other words, if you want the the people getting the, the, the treatment group and, the and, the and, the control group to be similar in terms of their composition you, you must randomly allocate people and so if you ever see a trial a study that's reporting to show that a medication works and there's no randomization there is no random allocation of people to, to treatment or no treatment or to different treatment arms then alarm bells should be ringing and is, you know, one has to treat that with significant amount of skepticism.
And then what usually happens and what happened in the trials of the treatments that I'm going to show you is that measurements were taken of people's aortas at the beginning of the trial and then they continued to take measurements throughout the trial and then they took a final measurement at the end of the trial and so for each patient it was possible to see how that patient aorta was behaving over the course of the trial and, and, and then you could work out whether the patient aorta was growing and if it was growing how fast it was growing and that's how the trials of, of, of Marfan syndrome were conduct, in Marfan syndrome were conducted, so that's, that's how these trials were, were done.
Now we have in the, in, in, in, the sphere of what's called evidence-based medicine, which is the way in which we you try to apply evidence and, and science to patient care. This kind of pyramid of a hierarchy of, of evidence.
And so in, in the, in the past we used to very heavily on experts opinions, so, you know, a professor or a specialist would say, "this is what I think we should do," and it nowadays we put that really at the bottom of of the hierarchy because we've realized that experts got it wrong a lot and, you know, it was very much biased by what the, you know, sort of loudest voice in the room was the most, you know, sort of, sort of most outspoken person sort of felt.
And then over time, you know, ideas tend to get tested in various kinds of studies and then here we have these kind of non-randomized trials where there was, medications were being systematically tested but without randomization and then there was a feeling that randomized controlled trials were probably better.
But then it, we had a situation in which often people would do random, several different randomized control trials. Maybe there'd be a randomized control trial in France, there'd be a randomized control trial in Spain, there'd be a randomized control trial in Canada, then there'd be one in the US and sometimes they would all say the same thing. Well, that's pretty easy and sometimes they would say slightly conflicting things because they tested things in slightly different ways, maybe they'd slightly different medications, maybe the populations they were studying was slightly different and the, then there would be various different ways of looking at overviews of these trials and sometimes experts would get together and say what they thought were the best ways of doing these trials.
But finally people developed systematic ways of putting trials together and there is a special way of putting trials together that is called the meta-analysis and the, the, the correct way of doing a meta-analysis is to bring together the trials and this is clever bit is that you add, you, you essentially add together the information in all those trials and you weight the trials according to the amount of information that is contained within each trial.
Now that is very, very misunderstood and if you understand that what I just said you know more about meta-analysis than most doctors, okay? Meta-analysis has a slightly bad reputation, particularly in the US, I should say, because there are lots of differences, for two reasons: one, most doctors don't understand that sentence that I just said and number two, because there's lots of ways of doing meta-analysis wrong and there's only really one way of doing them right, okay? but if you, if you avoid those two errors you understand what meta-analysis done correctly really is. It is putting together trials in a logical way in which the information, in which the trials are weighted according to the amount of information that each trial contains and that you do the analysis correctly and you don't make the mistakes that are easy to make then the meta-analysis is the best way of making the correct judgments about the the way in which one should proceed. That's the concept, okay, well, that, that's that's my belief anyway, okay.
The Marfan Foundation did not participate in the translation of these materials and does not in any way endorse them. If you are interested in this topic, please refer to our website, Marfan.org, for materials approved by our Professional Advisory Board.
The Marfan Foundation は、当翻訳には関与しておらず、翻訳内容に関してはいかなる承認も行っておりません。このトピックに興味をお持ちの方は、Marfan.org にアクセスし、当協会の専門家から成る諮問委員会が承認した内容をご参照ください。
So what, what issue am I trying to resolve now? Marfan syndrome obviously is a multisystem disorder. It affects the eyes, it affects the spine, it affects the muscular-skeletal system. In fact, it affects many parts of the body. Some might say it affects every organ system to some extent, sometimes just trivially but sometimes quite seriously but I think that the effects on the, the the cardiovascular system are the, you know, by far the most serious in terms of impacting a patients longevity, how long they're going to to live.
Now there are patients with Marfan syndrome whose life expectancy is unaffected by Marfan syndrome. You know, I have at least one patient I can think. I've got one one chap who comes to my clinic, who is 81 years of age, who has definite Marfan syndrome and he has never had any heart surgeries. He's never had any surgeries on his blood vessels. He was treated, you know, for the first 50 years of his life I don't think he had any medical treatment and even in later years he didn't have much medical treatment, probably just a beta blocker, so, you know, we haven't done very much to him. He's, he's lived for 81 years without much treatment, so it's certainly possible to live to very advanced ages with Maran syndrome and not have serious cardiovascular disease but many other patients do need to have quite, you know, either they need medications or they need surgeries or they run into complications and so, so that's what we're trying to deal with.
You know, I focus on the cardiovascular manifestations and in particular I focus on problems of the aorta, so I know that many, probably most of you already know this, most of you do but I guess it's just possible someone on the call has just recently been diagnosed and this hasn't come up yet and I think that perhaps something that people don't fully appreciate is that there are, this is, this usually begins and mainly affects two specific parts of the aorta and the most vulnerable part is this part here, so here's the aorta in the chest, it is of course a gigantic blood vessel people often are very shocked to learn how big it is. It's, I always say, it's the biggest part of your body that no one's ever heard of and, you know, it starts here just at the heart. It then loops up around just under the neck and giving off these branches to the arm and to the to the head and then it loops around like this, this is the arch of the aorta and then it loops down through the chest. That's about a foot long or, you know, maybe that kind of thing and then it sort of goes down through the diaphragm into the abdomen then through the pel, and then it sort of halfway down the abdomen, it's splits around about where the the navel or belly button is here and then splits into these two big blood vessels here.
And usually here it is of taken out and this is the aortic valve and then this very first part here is called the aortic root. These two very, very important blood vessels: the coronary arteries come off here. This is what causes, you know, in the generally older people causes coronary artery disease. That's not a feature of Marfan syndrome. That's different and then but this is the part that tends to swell in Marfan syndrome and if it gets too big that's when there is a risk of tearing of the aorta here and this is where the risk of tearing is very serious because it can disrupt the aortic valve, it can disrupt the coronary arteries and it can run back towards the heart, so that's what we want to avoid and then there's another area of vulnerability just here where the aortic arch meets the descending aorta.
And when they have a problem here it tends to cause tearing called a type A dissection and when you have tearing here it tends to cause what's called a type B dissection, generally speaking, so that's the issue that we're trying to prevent and we're trying to slow down the growth of the aorta here and here.
Now we get these things called aneurysms which are sort of swellings of the blood vessel and here's one type of aneurysm and here's another type of aneurysm and you can get aneurysms anywhere along the aorta but this is the kind of aneurysm that you tend to see in Marfan syndrome and, and also other conditions like Loeys-Dietz syndrome and, and it's right down at the root of the aorta. You don't see this, you see this another conditions, you see this like this. It's like a flask that we used to use in school in chemistry lessons like that and that's what it looks like.
And if I go to the next slide this is what the echo cardiogram looks like. Now this is the same aorta but it's just been tilted 90 degrees on its side. This is what a normal aorta looks like. Here's the heart, here's the aortic valve, here's the aorta and here's the little flask. This is what a normal one would look like and here is what one would look like in a patient with Marfan syndrome where the aorta has been allowed to grow far, far too large. Each one of these markings here is one centimeter, so this is probably one, two, three, four, five, six, seven, eight, eight and a half centimeters. That's that's crazy, too big, so and here's the aortic valve and this whole thing has got, got far too big, so that's, that's what a Marfan aorta would look like on an echo scan.
The Marfan Foundation did not participate in the translation of these materials and does not in any way endorse them. If you are interested in this topic, please refer to our website, Marfan.org, for materials approved by our Professional Advisory Board.
The Marfan Foundation は、当翻訳には関与しておらず、翻訳内容に関してはいかなる承認も行っておりません。このトピックに興味をお持ちの方は、Marfan.org にアクセスし、当協会の専門家から成る諮問委員会が承認した内容をご参照ください。
So I've been asked to talk about the, thankfully, the area that I know most about, which is what, what I think of is the medical treatment of Marfan syndrome. What, what do I mean by medical treatment, well that really is the, the medications as opposed to the other aspects of treatment, which I, I think are extremely important, which I think of as the lifestyle measures and also surgical treatment. We could talk a little bit about that later if you want to but they are important, so I'm going to come on to that in a second.
So I'm going to tell you at the beginning what I want you to take away as the main messages and then I'm going to, you know, go on to talk in more detail about it and at the end I'm going to tell you what the main messages are again, so just I want to, I'm going to tell you three time basically.
So the first message is that the lifestyle measures for patients with Maran syndrome are important and if you're a patient with Marfan syndrome you can control these to a very large extent, so I don't want anything that I say today to detract from that very important message looking after yourself, looking after your body, looking after your aorta is a really important part of Marfan syndrome and you are the main person who's in control of those and you can get lots of good advice from your care providers and, and if you're not getting good advice then you, you need to ask them for it and get help with, with it.
The second thing is that, you know, I think this is a really optimistic message to come out of the meta analysis and the, and the, the studies that everyone has worked so hard on is that there are now two proven to my mind and I'm going to show you the, the reason why I think it's the case and explain the reason that I, I used that word proven, classes of medications that have been shown to reduce the rate of aortic enlargement in Marfan syndrome.
And that these medications or classes of medication are effective when used alone and you can choose either one or you can choose both and I think they are probably most effective when they're used in combination and if you use both medications you probably get an additive effect and the, the two drugs work together to, to give you double the benefits and so I think that's a pretty positive message and if I had, you know, Maran syndrome I would want to be well, I would want to give serious consideration to taking both of these classes of medication together. That's what I'd want to do now. If there was some reason why I couldn't take one or the other then that's okay. I would just take the one that I could take but, but I would want to be thinking about taking both.
There are also some medications that should be avoided. Now most of you are not going to ever need these medications but I think it would be nice for you to know what they are in case the circumstances arose where a physician might want to put you on these medications and you could say, "I think I'll pass on that one," or "do you have an alternative?"
And of course there is the option of surgery to the aorta, the main blood vessel if that ever grows too big and that's a really good treatment option and surgery on the aorta, especially the part of the aorta that is most vulnerable in Marfan syndrome. We'll talk about that, is now an effective and generally low risk option but it's not risk-free and that's why we want to look at things like lifestyle measures and medications to try and delay the need for surgery or in some patients perhaps even remove the need for surgery.
So we want to use all the tool, tools in our toolbox and if we, if, you know, it's my belief that if we use all three treatment options: lifestyle, medications and surgery, you know, we can get the best outcomes for patients. That's the goal, so those, those are the main options. I'm going to repeat all this at the as we go through the talk and, and elaborate on these things and at the end, I'm going to come back and summarize it all again.
The Marfan Foundation did not participate in the translation of these materials and does not in any way endorse them. If you are interested in this topic, please refer to our website, Marfan.org, for materials approved by our Professional Advisory Board.
The Marfan Foundation は、当翻訳には関与しておらず、翻訳内容に関してはいかなる承認も行っておりません。このトピックに興味をお持ちの方は、Marfan.org にアクセスし、当協会の専門家から成る諮問委員会が承認した内容をご参照ください。
知り合ったマルファンの友人が、地元の医科大学で模擬患者をやってみたら？と言ってくれ、MSU Grand Rapidsキャンパスでのオリエンテーションに参加することになりました。自分の体調に合わせてスケジュール管理ができるので、私にはうってつけでしたが、一番大切なことは、大義に貢献していることを実感できたことです。医学生の皆さんにマルファンのことを説明して、実際の患者と診断とを照らし合わせてもらう、そうすれば、少しでもマルファンのことを記憶してもらえるのではないか、そして、その先の医学の道で命が救われたら嬉しい。模擬患者を始めてもうすぐ12年。非常にやりがいを感じ、満たされた気持ちでいます。
The Marfan Foundation did not participate in the translation of these materials and does not in any way endorse them. If you are interested in this topic, please refer to our website, Marfan.org, for materials approved by our Professional Advisory Board.
The Marfan Foundation は、当翻訳には関与しておらず、翻訳内容に関してはいかなる承認も行っておりません。このトピックに興味をお持ちの方は、Marfan.org にアクセスし、当協会の専門家から成る諮問委員会が承認した内容をご参照ください。
