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Joellen Brown - phaware® interview 465

Joellen Brown shares her experience with pulmonary hypertension (PH) on the 15th anniversary of her diagnosis. Joellen was born with a hole in her heart and had her first open heart surgery at the age of two and a half. She believes she may have had PH since birth, but was not aware of it due to limited research in the 1950s. Joellen emphasizes the importance of having a supportive medical team and family, as well as being proactive in advocating for one’s own health. She encourages newly diagnosed individuals to seek out support groups and emphasizes the need for early and appropriate treatment.

My name is Joellen Brown. I am in Newport Richey, Florida now. I was diagnosed 15 years in May with pulmonary hypertension. I was born in 1956 with a hole in my heart. Back then, there wasn't much information on correcting it or how they were going to go about doing it. I was about two and a half when I had my first open heart surgery and they put a patch on my heart. I went through the majority of high school, not being able to take gym, because they really didn't know if that patch was going to be pliable to exercise and all that. I was also told not to have any kids, which thankfully, I didn't listen to that. I ended up with three girls and no issues, and they were healthy. I've got three older sisters. I was the only one with the heart issues, so it wasn't anything like hereditary or anything like that. When I was about 40 years old, they told me that I needed to have another open heart surgery, because I had a tear in my aortic valve. So they went in and I have a cadaver valve. In my early '50s, I started noticing major weight gain and really breathlessness, tired all the time. I had been seeing a cardiologist all my life and nobody had ever mentioned that it was any kind of a lung or heart issue, it always had to do with the heart. I finally went to my regular doctor and she had me walk around the hall. At that time, my oxygen was 67%. I was sent home on oxygen. That's how my journey with pulmonary hypertension went. They first thought I had asthma, so I was put on an inhaler with the oxygen. That didn't work. I went to another cardiologist who was also a PH specialist. He's the one that diagnosed me with PH. But he had told me too, that I might have been born with it. But then again, back in the 1950s, there wasn't a lot of research on PH. So who knows? I might've had it all this life and not even realized it. So all that time, it's always been the heart thing. I'd always gone to the doctors. I didn't really have to worry about my weight, until after I had my kids and I had my second open heart surgery. That's when my weight started creeping up on me, which might've been back then, part of PH, I don't know. So now 15 years later, they're saying that my PH is from congestive heart failure and scoliosis, which is what I was born with. I was born with scoliosis, with the hole in my heart, so I have that against me as well. I think at the very beginning, I had a wonderful PH doctor. My team is what really got me through everything. But then again, that's well and good, but you also have to have that family support as well. Great, the treatment is helping you and everything, but you really need that mindset of having the support of your family to be there. Everything kind of fell into place when I was diagnosed. I want to think that I was one of the lucky ones. I didn't have to go through years and years and years of doctors. I was a special case, because of my open heart surgery at such a young age, on top of having the pulmonary hypertension. My cardiologist at the time, was also a pediatric cardiologist, and that kind of helped. Whenever I went in for any kind of test, they always had to work with my body and my issues. It wasn't like the same thing that they would do with somebody else. I want to think that it was all to do with the doctor I had at the very beginning. I am divorced. I'm pretty lucky that I'm able to still take care of myself. I'm a mother that I tell my kids everything. I don't want to keep it secret, because I want them to know that if anything ever happened to me that I can't speak for myself, I want them to know what I would want. So I know there are some other people out there that are like, "My family has no idea what I'm going through." I tell my kids pretty much everything. They've gone to doctor's appointments with me. They've been there when I've been in the hospital, and that has been wonderful. Right now since I'm in Florida, I have two of my daughters that live very close. That's been really helpful. But I'm not driving now too, so that's been a challenge in itself. But moving to Florida, my health has gotten so much better. My sister and I live together now. My lifestyle is much better because I do feel better. I think this was a great move for me health wise. I've lost weight finally after a year and a half. I have a great team. That's been probably the hardest part, moving. I look back at several years ago and how my health was at that time, which was not very good, and how much it is today. It's really has improved. I'm really thankful for that. I think that's a lot to do with your mindset and the team and fighting for what you need. It's definitely a journey I don't wish on anybody. The one thing that I always tell people that are newly diagnosed, because I've been on several sites on Facebook, is everybody is different. What's going to work for me might not work for you, but getting the right doctors is the best thing. But my biggest thing that I tell people is, everybody is different. You need to get that treatment right away. It's not a wait and see type of disease. It's not going to go away on its own. You need to get that right treatment. You have to be your own advocate. You have to be out there and ask questions. There's no right and wrong answer, but you need to know, because you only have this body once. You need to figure out how it's going to affect your everyday life. I had some great mentors when I first got PH, on Facebook, that are no longer here, so I try to remember what they told me to tell other people. I was born sick and I had to overcome a lot of stuff when I was little. I think that has gone on to me as an adult that is sick. I'm on oxygen now 24/7. I will always be on oxygen, so I try to be an advocate for getting the right type of oxygen I need. I don't know what would've happened if I didn't have the hole in my heart when I was little and all of a sudden, I'm 52 years old and I've got this life-threatening disease. I don't know if things would be different. It's still a shock, but I've always had people telling me, especially when I was younger, they were cruel to me at school, telling me I was going to die when I was 13. You live through that and you're like, "Okay, well, I'm 14 and I'm still here." I have more appreciation for my mother as I'm older, because I can't even imagine what she went through in the 1950s, taking me on the bus to go to the hospital and all that stuff. I can't even imagine that. What brought me here was my grandson. He lives across the street. That's my motivation to be well enough to see him grow up. That's me doing what needs to be done to keep me well. If it means staying in a lot more to stay healthy, then I'll do that. That's my motivation and just to be in spite of everybody that said, you can't do something. Just moving from Ohio to here was a trip and a half, having a friend of ours that had a horse trailer that moved all of our stuff here. Of course, I had to fly because I couldn't do the long trip. I still think we've been here two years and I'm like, "Did we really do this?" A couple years ago, I probably wouldn't have. But it's one of those that you wake up and you're like, "I'm ready. It's time." Where I used to live in Ohio, it was kind of an independent living senior place. Out of all those people, there was one lady in there that had PH at that time. I'm like, "I can't believe that I lived with somebody in this building that has the same thing that I do." She was on oxygen as well. Well, she ended up passing away. She was older than I was. One thing I would like to tell the people that are newly diagnosed, the best thing is to get into a support group. That really helped me, being around other people that have the same disease. I went for many, many years, took my mom with me. She enjoyed it. Learned a lot about pulmonary hypertension. I have not been able to find one here in Florida, so that's going to be on my bucket list for this year is to find a support group near me, or at least someplace I can get to. My name is Joellen Brown, and I'm aware that I'm rare.

Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected]

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Episode 465 - Joellen Brown

In this episode, Joellen Brown shares her experience with pulmonary hypertension (PH) on the 15th anniversary of her diagnosis. Joellen was born with a hole in her heart and had her first open heart surgery at the age of two and a half. She believes she may have had PH since birth, but was not aware of it due to limited research in the 1950s. Joellen emphasizes the importance of having a supportive medical team and family, as well as being proactive in advocating for one's own health. She encourages newly diagnosed individuals to seek out support groups and emphasizes the need for early and appropriate treatment. Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected]

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Chelsea Price - phaware® interview 464

Chelsea Price shares her experience living with pulmonary arterial hypertension (PAH). She emphasizes the importance of having a good support system, including her church community and connecting with other friends with PAH. Chelsea is grateful for the good days she has and strives to be an active mom for her children.

Learn even more about Chelsea and PAH at www.OutnumberPAH.com.  My name is Chelsea Price. I live in St. Louis, Missouri, and I have pulmonary arterial hypertension (PAH). Shortly after I had my second son, I noticed that my endurance had decreased. I grew up playing soccer, competitive cheerleading, and I was always very, very active. But after he was born, I had trouble going up steps, just couldn't catch my breath. We'd be out in the yard and we'd be playing. The next thing you know, my husband turns around and I'm laying on the ground just gasping for air and trying to catch my breath.

I kind of pawned off on being a new mom, not having the time to exercise. My doctors thought I had anxiety. It wasn't until September of 2019 when my husband and I were on a trip to New York that our view of something being wrong kind of came to fruition. We were biking through Manhattan and we came to this hill and I remember being so frustrated, because he just took off right up the hill and I couldn't keep up and I couldn't catch my breath. I just threw the bike down and went and sat and felt like I was so out of shape.

When we returned, I just kind of brushed it all aside. Again, well, my doctor said, I have anxiety. There's nothing else wrong with me. I was 32 years old at the time, and 32 year-olds don't get sick. After going back to the agency that I was at previously, I started having some chest pain and some back pain. Every day when I would go up the steps at work, I would be rushing to my desk to sit down because I would be seeing spots. Again, this all went back to me feeling like I'm a new mom, I'm out of shape and I just can't keep up. I started doing yoga and my back was still starting to hurt more. I thought I was having a panic attack, one day at work, and had asked one of my good friends to just go on a walk with me.

There wasn't anything to be anxious about at work. It was just again, like you have anxiety, so obviously this is a panic attack. I went to the doctor the following week, which was February of 2020. They said, "Oh yeah, you know what? It sounds like it was just a panic attack." To me that didn't feel right in my gut. Something felt more wrong than just being anxious, so I urged my doctor to do an EKG in office just to ease my mind. My family has a history of heart issues, so that's of course kind of where my head went. They had spotted an abnormality on the EKG.

The following week, my doctor had scheduled an appointment for me to do a stress test at our hospital, but that was also right in the midst of COVID shutting down here in St. Louis. Monday, I stayed home from work not thinking anything of it. I mean, it was COVID, but I was working from home. Tuesday, we kept our boys home from school. They were two and four at the time. Wednesday, my mother-in-law came to stay with us for the two weeks that we were going to be hunkered down. But Thursday morning I woke up and I was in so much pain, my chest hurt so bad, my back hurt so bad, and I'd never felt any pain like that before. So my husband drove me to the emergency room where they started running tests. They noticed that my pulmonary artery was three times the size that it should have been. At the time I did not know what that meant. They ran a ton of blood work and saw that I was in heart failure.

They took me up to the critical decision unit, so I was going to stay for 24 hours for them to figure out what was going on. They took me in to do my stress test and had done the echo cardiogram first. They were like, "You're not doing the stress test. You're in heart failure. You have a hole in your heart," which I didn't know I had. It was about the size of a quarter, so 80% of my blood was just circulating in my heart and lungs, and only 20% was going to my body. Then, obviously compounded all of that onto having pulmonary arterial hypertension, which was diagnosed the next day during a right-heart cath.

When I was in the hospital, during COVID, it was a very scary time because, one, COVID being a new thing, but also now having this condition that I didn't know much about, but the looks on the doctor's faces we're definitely saying that it's something serious. I was by myself because my husband wasn't able to come back in because of COVID protocols.

I had an amazing team that sort of walked me through everything. I was put on oxygen, because my oxygen saturation was dropping into the 70s when I was in the hospital walking just over to the bathroom and back to my bed. They started treatments right away. I was discharged a few days after. I sort of stepped back into the world of now having this rare condition alongside navigating a COVID world.

With having a diagnosis, it was something for me to take action forward, which isn't something that happened initially in terms of being an advocate for myself, speaking out about what had happened. I immediately hopped on Instagram, because I was so scared because the doctors tell you don't Google, and of course you do, and then you see all of the stats of things that may or may not happen in terms of life expectancy. I had two little boys at home and a husband that I wanted to see them grow up. I was so scared that I wasn't going to see that. Sometimes those fears still come up, but at the time it was just, "Am I going to see them go to kindergarten? Am I going to see them play soccer? Am I going to see them turn into little boys?" They were babies at the time.

So having a plan for treatment definitely eased my mind a little bit. Once I was able to get up on the treatments and I started feeling better and I could walk around and I was able to get off the oxygen, there was so much more hope in those moments knowing, "Okay, it's going to be a battle and it's going to be hard some days. Some days I'm not going to feel good, but I'm going to be here and I'm going to have a good life and I'm going to be there to see them grow up and to experience everything with them." That's one thing that just keeps me going on the days that I don't feel good, is that I have these two amazing little boys and a husband of 10 years now that we can live a good happy life even in the midst of something that's scary, because I've responded well to the treatments.

Having gone through it during COVID was scary. But honestly, part of me is happy that it happened when it did, because I was working from home at the time at a place that was extremely understanding of what I was going through. So when I was starting up on the medications and having some really tough days with symptoms and side effects, I was able to lay in bed and to rest and to recover and to hop online for meetings if I was feeling up to it. So I'm glad COVID happened when it did. I'm glad I got diagnosed when I did, because I could not imagine trying to go into an office while battling the initial stages of the disease and getting up on treatments.

For the most part, I tend to try to live in the moment, and that's been a blessing with this disease, because I am 100% type A - go, go, go. Things have to be perfect and all of that. I always wanted everything to be controlled and I wanted to be in control of everything. I think the one thing that this disease has shown me is that you can think about what may or may not happen in the future. Most times, our minds tend to wander further than things are actually going to go, or at least in my case. So I tend to focus, and actually have for the past almost I guess four years, focused on day to day, doing what I can and focusing on what I can control, taking my medications, eating good, walking when I'm feeling up to it and things like that.

I don't really try to linger too much on the future, because it scares me. I think there's a lot of hope coming, hopefully with some new medications that are coming out. I really honestly, in the past couple of years, haven't thought to the future too much. There's been one time since diagnosis that I've really thought about that, and that was a few weeks ago I had some symptoms and things like that after traveling, and that's kind of where my mind went is, "Oh gosh, is it progressing? Am I going to be here?" But everything is looking good and I'm doing everything that I can to control what I can and enjoying every single moment that I have with my family and my friends.

Even before I was diagnosed, we were somewhat involved with church, just different things in our past that we've been through, we've lost a child. So getting a diagnosis, I leaned into my church family, I leaned into my faith, because it's not something that we can carry on our own. So I'm thankful that I have my faith, that I have an amazing church family. My friends and my family have been incredible post-diagnosis, dropping off food. My mother-in-law ended up staying with us for months to help me and help with the kids and my husband. We just have such a good network that without them, I think it'd be a lot darker.

I also, post-diagnosis, found a ton of other friends that have PH that I text with on a daily basis that when I'm having a rough day, we can commiserate. One of my friends and I actually titrate up at the same time on a medication that was really challenging to get up onto. So while I don't love that we both were going through that, I'm glad that we had each other, because I think it made it a little bit easier. We were able to cheer each other on. Community is so important, and having those friendships and people that will be around you that may not understand exactly what you're going through, but can see that you're in a moment of need and can step in and help hold you up is so important.

Day-to-day now, I think sometimes I forget how sick I am. I'm able to go play with my kids and cook dinner and play soccer with them, which has always been a huge passion of mine. That was one of the big goals that I had post-diagnosis and talking to my doctor, is I want to be able to be the active mom, I want to be able to go play with my kids, because my parents were always out there playing with me and I don't want to sit on the sidelines. It took some time and I did have to change my mindset a lot when I couldn't do that initially. So again, it just goes back to just trying to focus on the day-to-day and enjoying every single moment. Right now, I'm thankful that I have way more good days than challenging days.

My name is Chelsea Price, and I'm aware that I am rare. Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected] 

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Episode 464 - Chelsea Price

In this episode, Chelsea Price shares her experience living with pulmonary arterial hypertension (PAH). She emphasizes the importance of having a good support system, including her church community and connecting with other friends with PAH. Chelsea is grateful for the good days she has and strives to be an active mom for her children.

Discover even more about Chelsea and PAH at www.OutnumberPAH.com. 

Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected] 

  Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Rajan Saggar, MD - phaware® interview 463

Dr. Rajan Saggar, a pulmonologist at the University of California in Los Angeles, discusses the complications of pulmonary hypertension in various lung diseases. He explains that pulmonary hypertension can either be its own disease or can complicate other conditions such as heart disease or lung tissue diseases like emphysema or fibrosis. Dr. Saggar mentions a recent FDA-approved medication for pulmonary hypertension complicating lung tissue diseases, and ongoing research to develop more treatments.

My name is Rajan Saggar. I'm a pulmonologist. I work out of the University of California in Los Angeles, where I actually did my training. I also span the advanced lung disease area of medicine, so I'm also heavily active in the transplant world, as well. I co-direct the program at UCLA with Dr. Richard Channick. I've been there now since the early 2000s, so it's been a good ride. One of the topics we will address today would be specifically pulmonary hypertension complicating various types of lung disease. I get to see the natural history of those types of diseases as well as just regular PAH. All the different kinds of pulmonary hypertension, all groups and how some of those patients unfortunately do progress over time. Some of these unfortunate patients regardless of all the medications we try to use or get on board, eventually need a lung transplant. I have a perspective, which I think is a little bit different from others. As all of us know with this pulmonary hypertension business, high blood pressure in the lung is a final common pathway for a lot of different diseases. It either comes by itself as its own disease or it can complicate other types of diseases. So, for instance, it can complicate heart disease. In other words, where the pulmonary hypertension is a consequence of the heart disease. Then, what we're talking about here is pulmonary hypertension complicating an underlying lung disease. Pulmonary hypertension is a lung disease. It involves the blood vessels, but it can actually be the consequence or it can complicate what we call parenchymal lung disease. The parenchyma means the actual lung tissue itself. So if you have a lung tissue problem, that lung tissue problem sometimes can be complicated by pulmonary hypertension. The lung tissue problems that we're talking about, generally speaking, most common one is emphysema, or sometimes we call it COPD. Chronic obstructive pulmonary disease comes in many flavors, but emphysema is one type of COPD. It turns out that pulmonary hypertension can complicate the course of patients who have COPD or emphysema. Not all patients who have COPD or emphysema develop pulmonary hypertension. There's a select group that develop it, and we're always trying to figure out who is more predisposed compared to others. Another kind of lung disease of the lung tissue, if you will, are diseases that scar the lung. The word in medicine for scarring is we call it fibrosis. That's a fancy word for scarring. Then sometimes, you may hear the term interstitial lung disease. Interstitium is one part of the lung where you tend to develop this scarring process or fibrosis. Interstitial lung disease and fibrosis are on a spectrum. It's a type of lung tissue disease. Just like the COPD emphysema type of lung tissue disease, this particular type, the interstitial lung disease fibrosis lung tissue problem can also be complicated by pulmonary hypertension. It turns out that for years we've tried to use different medications to try to treat that type of pulmonary hypertension, specifically complicating the lung tissue diseases. In our attempts have failed several times over; however, there is a recent arrival of a medication that we also use in regular pulmonary hypertension, where you don't have a lung tissue problem, where we were able to use the same medicine. Luckily, it actually did work in patients who specifically have this lung tissue problem, this interstitial lung disease or fibrosis or scarring of the lung complicated by pulmonary hypertension. We used a pulmonary hypertension medication to try to work on the pulmonary hypertension complicating this lung tissue problem. Now, that's FDA approved. It's the first medication that's now approved in this particular type of pulmonary hypertension, which is cool. At this point now, since there's one medication that's hit the market specifically for this fibrosis, scarring of the lung disease complicated by pulmonary hypertension, there's a lot of interest by other companies in drug development who are trying to look into other ways or other pathways to help folks with the pulmonary hypertension piece complicating this scarring of the lung disease. What's interesting is some of the medications may actually work on not just the pulmonary hypertension part, but you might get a twofer. In other words, the medication actually helps with preventing progression of the scarring. There's some thoughts that there's certain medications that might be able to hit both problems, because the patients we're talking about here have two lung problems. They have a scarring of the lung problem and they have a high blood pressure in the lung problem. It would be ideal to have a medication that either reverses or helps to improve or doesn't allow progression of the scarring, as well as helps the pulmonary hypertension piece, the high blood pressure piece, which would be really great for patients. Right now, the medication that's approved that I mentioned earlier, the one single medication we have is inhaled. There's a big push to use something that's inhaled, so it has direct delivery to the lung. A lot of companies are interested in looking at this not just from a new medication standpoint, but using something that's inhaled, which the medical community feels may be the best way to deliver such a medication even though some of these medications can be given orally or through the veins or a lot of different ways to give medications as one might predict. But for lung diseases involving the actual lung tissue, the inhalational route seems to have gained a lot of momentum, and that's where the field's going. The scarring of the lung diseases are considered to be an orphan lung disease. The idea is you have less than 200,000 cases in the US at any given time, the definition of an orphan lung disease. Then, you're talking about that disease being complicated by the high blood pressure or pulmonary hypertension, which affects a fraction of those patients. But what's interesting is some of the work that's been looked at in terms of following patients with the scarring disease to see if they develop pulmonary hypertension, if they don't have it on the initial evaluation, the overwhelming majority will go on to develop some degree of the high blood pressure problem. Then, the question is, does it matter how high your blood pressure is before one of these medications can actually help you? In other words, a lot of people will have a mild variety, but then even a smaller group will have more of the moderate severe variety. That's where we've seen most of the medications work for the high blood pressure piece complicating the scarring. But now, there's a push to see, well, maybe if we treat these patients earlier, even when they have a mild problem. Or, for instance, if they just have scarring, we just mentioned earlier maybe a medication can prevent both. So if someone just has scarring and no high blood pressure and you use one of these medicines, maybe you could prevent the development of the high blood pressure problems. None of this is in stone. It's all evolving. We're looking and thinking out of the box with all this stuff. I've always said the patients who we see who have lung tissue diseases, particularly when it's progressive, and even if it's not, pulmonary hypertension patients too, high blood pressure patients, when you combine the two, the double whammy I call it, where you have two lung diseases, generally speaking, are patients with advanced lung disease, they're just a special group of patients in the sense that they're more than happy to sort of give to the scientific endeavor. They understand that they have diseases that don't have obvious cures. They are interested, obviously, in helping themselves, but also in helping move the scientific community forward. You can imagine to do any of these studies, you need willing patients. You need patients who are willing to put themselves into a study where they might not get an active medication. It's disconcerting when you think about it. If you're going to enter a clinical study, and remember a lot of these studies we do in medicine, they're so-called blinded. The doctors don't know what the patient's getting and the patients don't know what they're getting. But we all know that they could be getting one of two things. One thing could be the active drug that's being studied, where we're trying to get some experience and some data so that we can prove that something works or doesn't work. But the other arm of the study is where they're getting not the real drug. They're getting some what we call placebo, which they're going to get for some amount of time. You have to be a special person to enroll in a study and to want to put yourself on the line, so to say. Now granted, there's not a lot out there as it were, but still some people feel very uncomfortable with the concept. But the studies are designed in a way such that if someone has a worsening of their status while they're in the study, of course, we don't know if they're getting the active drug or placebo, but while they're in the study, the studies are designed, and folks should know this, such that if they are to progress and meet criteria for progression or worsening, and those are preset criteria. They're usually pretty forgiving in the sense that we will pull them out of this study, and then they can go into what's called an open label extension, where they would get the active drug regardless of what they were getting to begin with because no one knows. We would assume that they're not necessarily getting the drug. They may have been getting the drug, but the point is after that point that they got worsening of their condition, everyone would get the drug. They would continue to be followed as part of what they call an open label extension to the main study. My only point in all this is that we as the medical community in this setting want to move the field forward. We can't do that without patients who are willing to enroll in such studies. I want to give props to the patients. The majority of the patients we deal with are more than willing to help. They're really moving the needle to help folks in the future and hopefully help themselves, but it's a selfless act. I think at the end of the day, we just want the listeners to know, we're very much committed to trying to prevent and decrease the need for lung transplantation from any lung disease. There's so many different ones that can progress and eventually require a lung transplant, because when we have no medical options, then we take the surgical option, which is the last thing we want to do. No one actually says, "Oh, I wish I could have a lung transplant," obviously, but at the end of the day, it is some light at the end of the tunnel. Having said that, our job and goal all the time is to get medical options to, number one, help people, improve people, et cetera, get people better, having full lives and getting back to their lives, knowing that they have a chronic disease. Of course, a cure would be amazing, but even just to slow down the process or keep what you got in terms of your lung function would be amazing. But what we also really want to do is really minimize the folks who need a lung transplant or hopefully obliterate it. As we sit here today, unfortunately, lung transplant still remains the last option. That whole process of transitioning to we have no medical therapy, we need to commit to a lung transplant, that's a whole other discussion and a very difficult one for patients, because it's scary. It's really scary. Our job is to help people not miss the boat and basically realize that their options may be dwindling sometimes. Some of these patients have to go to lung transplant. But our overall overarching goal is to decrease the number of transplants. Actually, that's what's happened as the pulmonary hypertension, high blood pressure medications in the lung have rolled out over the years, the need for lung transplant, for pulmonary hypertension, the pure disease of high blood pressure in the lung has gone down. That's a nice thing to see. My name is Rajan Saggar, and I'm aware my patients are rare.

Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected] #phawareMD @UCLAHealth

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Episode 463 - Rajan Saggar, MD

In this episode, Dr. Rajan Saggar, a pulmonologist at the University of California in Los Angeles, discusses the complications of pulmonary hypertension in various lung diseases. He explains that pulmonary hypertension can either be its own disease or can complicate other conditions such as heart disease or lung tissue diseases like emphysema or fibrosis. Dr. Saggar mentions a recent FDA-approved medication for pulmonary hypertension complicating lung tissue diseases, and ongoing research to develop more treatments. 

Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected] #phawareMD @UCLAHealth

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

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Sandeep Sahay, MD - phaware® interview 462

Dr. Sandeep Sahay, a pulmonologist at Houston Methodist Hospital, discusses the concept of disease modification in the treatment of pulmonary hypertension (PH). He explains how traditional PH therapies focus on vasodilation, reducing pressure in the pulmonary artery by dilating blood vessels. However, newer therapies, such as sotatercept, work in different ways to reduce smooth muscle proliferation and thickness of the artery walls. Dr. Sahay suggests that if a drug is labeled as disease-modifying, it may be used in combination with existing PH medications from the beginning of treatment. However, he notes that further research, regulatory approval, and payer considerations are necessary for this to happen. @MethodistHosp @SandeepSahayMD

Hi, I am Sandeep Sahay. I'm a pulmonologist. I'm at Houston Methodist Hospital, Houston, Texas. I've been involved with pulmonary hypertension patients and the PH Center at our place for the last 10 years. Clinically, I see patients with PH, or chronic thromboembolic pulmonary hypertension (CTEPH), or different types of pulmonary hypertension. I'm actively involved in research. We do a lot of clinical trials with the disease state. And also, our own academic stuff with grants and coming up with more thought-provoking ideas in this disease state, so that we can help our patients. 

In the last three decades, we have made a lot of progress in this particular disease state. I would like to thanks to all my colleagues, and researchers, and my friends, mentors for the last two to three decades who invested their whole lives and careers into this disease state and made tremendous progress. But still there’s a lot more work to do. We are here to further the science and advance in this disease state. On that note, the new thing, which is sort of I can say talk of the town is about disease modification. Many may wonder what this really means, and why we are even talking about it, and what's so new. I think this all started with us looking beyond our traditional PH therapies, which were generally considered as vasodilators. When I say that term in simple words, it means that we tend to reduce the pressure in the pulmonary artery by dilating the blood vessel. So most of our current therapies sort of classify into that category. But now with newer therapies, specifically sotatercept, which I think many patients are excited about. I would say clinicians, researchers, physicians, we are all excited about this therapy. This is not in a way our traditional PH therapy. When I say that term, what I really mean is that it doesn't work the way our traditional drugs work. It doesn't dilate the pulmonary artery, which we historically understand with our currently approved therapies. This drug kind of works in a different mechanism. When we look at that mechanism, the way it works, I'm going to be putting it in a very simplistic way that it just sort of tries to reduce that smooth muscle proliferation. Or, I should say that in PH, the thickness of the artery. It tries to reduce that by different mechanisms. I'm not going into detail scientifically that what it does, but it tries to restore the cellular level dysfunction in the endothelial cells, and then reduces the thickness of the vessel wall, so that it increases the size of your pulmonary artery. Now, since this drug works in a little different mechanism, we started thinking that, how do we describe how this drug works? When we started thinking about that, one of the things that naturally came to our mind was, should we call this drug as disease-modifying agent? Now, what that really means is that by definition, which could modify the course of the disease. When we use this term as physicians and researchers, we generally tend to refer it in a positive connotation. It means we believe that the drugs which can affect the pathophysiology or the underlying process of the disease in a positive way, it means improving the disease. However, it's a generic English term disease-modification. Last week, I got surprised when one of the patients, actually, heard this term, she actually was thinking this can mean anything. Actually, she was right. Because disease-modification, could mean positive or could mean negative. But in the scientific world, in our research world, when we started using this term, we thought of it only in the positive way. We didn't really think in the negative way, because we were talking about the treatment agent. This term actually comes from other disease states like rheumatological conditions. For example, rheumatoid arthritis, where you may have drugs to relieve the joint pain like your regular pain medications. But at the same time, there may be drugs which will work on the underlying mechanism, what causes rheumatoid arthritis and tries to prevent further damage to your joints. So, I'm just trying to make it a little different. I'm going to take one example in the PH world also, when someone has pulmonary hypertension and is symptomatic, more short of breath. You start noticing that you develop fluid swelling, or leg fluid retention, or leg swelling, or fluid retention on your body. Then, you take a water pill. Those water pills can get your fluid off, can make you feel better, but they do not do anything to your pulmonary hypertension. But then your doctors will, after the water pill, they will say, "Okay. Well, you need to take PH medications also," which are whatever… you know all those names. So then, you are started on those PH medications, in addition to the water pill. So try to understand the water pill doesn't really do anything. It doesn't change anything about your pulmonary artery pressure or the mechanism by which you develop that disease. But then your PH medications, on the other hand, I’ll take an example of those infusion pumps or those prostacyclin drugs. They definitely do help to relieve the pressure on your right side of the heart by dilating the pulmonary artery. So, that basically changes something about your pulmonary hypertension, which you do not see with the water pill. So, this is how the distinction is. To further advance our understanding in these PH medications, we have this newer drug, sotatercept. In the future, you will see many more such drugs will come out. There is a lot of exciting work being done currently. So, all these different drugs, which are going to come out, they affect your disease process itself. That's why they're called disease-modification. Now, this term is generally used by the regulatory bodies, like FDA or maybe the European drug approving agencies. It's not something which we commonly use in our day-to-day practice when we are talking to patients. The relevance of using this drug could be if, let's say, hypothetically speaking a drug gets a label from a regulatory authority that this drug is disease-modifying. Then, there may be a possibility that this drug may get inducted into the treatment algorithms along with your currently existing medication. There may be a possibility. And again, this will need a lot of research, regulatory body approval, and then with payers, etc. We're not going into all that. But in very simplistic way, what I'm trying to explain, if a drug becomes disease-modifying labeled along with the other properties, you could use that drug from the very beginning in addition to your other PH medications. There is a possibility. Maybe we will be using it this way. I don't know right now, because we don't have the drug available right now. We are waiting for FDA's call how and what all the recommendations when the drug gets approved. This is why this whole new terminology is being discussed. To be honest, it's a good thing to do. It's a good scientific exercise. We all are learning through this process. So, I believe that yes, we should be discussing this, because that's how we advance new ideas come. That will be very helpful for everyone, for the scientific community, for our patients, and for everyone around us. So, I tried to explain this in a very simplistic way. I hope this is helpful for our patients and their care providers. But again right now, there is no gold standard way for us to define this term. It's a work in progress. So with that, I would like to end. If you have any questions, please reach out to us at www.phaware.global.   I am Dr. Sandeep Sahay, and I'm aware that my patients are rare.

Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected] #phawareMD

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Episode 462 - Sandeep Sahay, MD

In this episode, Dr. Sandeep Sahay, a pulmonologist at Houston Methodist Hospital, discusses the concept of disease modification in the treatment of pulmonary hypertension (PH). He explains how traditional PH therapies focus on vasodilation, reducing pressure in the pulmonary artery by dilating blood vessels. However, newer therapies, such as sotatercept, work in different ways to reduce smooth muscle proliferation and thickness of the artery walls. Dr. Sahay suggests that if a drug is labeled as disease-modifying, it may be used in combination with existing PH medications from the beginning of treatment. However, he notes that further research, regulatory approval, and payer considerations are necessary for this to happen. @MethodistHosp @SandeepSahayMD

Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected] #phawareMD

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

UNISUS PAH Clinical Trial - phaware® interview 461

  SPECIAL EDITION Jonathan Hudson is a Global Trial Leader in Pulmonary Hypertension at Janssen Research & Development and is accompanied by colleague Josephine Garcia-Ferrer, Ph.D and Medical Manager. Jonathan and Josephine discuss Pulmonary Arterial Hypertension (PAH), the importance of clinical trials, what they entail, and share information on Janssen’s UNISUS Study for adults with PAH. Learn more about UNISUS Study with the Janssen Global Trail Finder.

Steve Van Wormer: Hello, and welcome to, I’m Aware That I’m Rare, the phaware® podcast. I’m your host, Steve Van Wormer, from Phaware Global Association. Today, I am joined by two Study Team Members from Janssen Research & Development, a pharmaceutical company dedicated to Pulmonary Hypertension research. Jonathan Hudson is a Global Trial Leader and Josephine Garcia-Ferrer is a Medical Manager. They are overseeing the UNISUS clinical trial — evaluating an investigational medicine in patients with Pulmonary Arterial Hypertension, or PAH. Jonathan and Josephine... welcome...

Jonathan Hudson: Thank you for the introduction, Steve. Great to be here today speaking with an organization dedicated to patients in the PH community.

Josephine Garcia-Ferrer: I feel the same, Jonathan.

Steve: First off, Jonathan, could you tell our listeners a bit more about Janssen Research & Development and Janssen’s commitment to the PH space?

Jonathan: Sure, for nearly a century, we have fearlessly confronted the world’s most devastating and complex diseases. We are proud to be a part of Johnson & Johnson, one of the largest and most broadly-based healthcare companies. Backed by the size and reach of Johnson & Johnson, we are transforming lives and reinventing healthcare. As part of Johnson & Johnson, we tackle society’s most pressing health challenges, connecting big ideas to the resources it takes to make them a reality and advancing access to good health for all. We seek medical breakthroughs wherever they occur, leveraging internal expertise and embracing external science to bring solutions to people who need them. We envision a world where people in every community, of all walks of life, have access to necessary medicines and health solutions. We are a leader across our medical areas of focus with a pioneering history of firsts. Expanding and enhancing the quality of people’s lives remains our greatest reward. We are committed to transforming pulmonary arterial hypertension into a long-term, manageable condition so that patients can live a full life. We have made PH a ‘therapeutic area of focus’ and are here to help advance research for those affected by PH.

Steve: Well, thanks for that background. Now, before we discuss clinical research and the UNISUS study, would you be able to tell us a bit more about Pulmonary Arterial Hypertension?

Jonathan: Certainly. There are five groups of PH, as defined by the World Health Organization (WHO). All are severe diseases characterized by high blood pressure in the lungs. Our focus has been on a specific, rare form of PH (WHO Group 1) called pulmonary arterial hypertension (PAH), but our research covers all groups of PH. PAH (WHO Group 1) is a rare, progressive, and relentless disease, where the pressure in the blood vessels of the lungs is elevated, resulting in stress on the heart. Despite recent advances, PAH remains a severely debilitating condition that leads to heart disease and early death. Early diagnosis and treatment are critical to helping improve life expectancy, but PAH is difficult to diagnose and there is currently no cure. PAH causes significant physical, social, and emotional burdens amongst affected patients and their caregivers. Over the last 20 years, the number of medications for the management of PAH has increased, which has helped to improve the lives of patients. However, even with these new advancements, we still have a lot of work to do to improve the lives of our patients. We’re working to transform PAH into a long- term, manageable condition to help improve the lives of patients. Janssen’s aim is to help increase the mainstream awareness around PAH so that no patient goes undiagnosed and there’s no delay in diagnosis.

Steve: Thanks, Jonathan. We are very familiar with PAH here at phaware®. I’d love to hear more about clinical trials in general, and their role in bringing a therapy to market. Could you elaborate on that a little bit?

Jonathan: Sure, I’d be happy to. The complex, global healthcare landscape continues to present new challenges, new diseases, and new dynamics. Healthcare systems are shifting toward delivering improved outcomes, enhancing the patient experience, and providing value-based care. Clinical research is medical research involving human participation. So, clinical trials are the primary way that researchers find out if a new medication, which we refer to as investigational medicine, is safe and effective. Clinical trials are conducted by doctors and researchers. Using what they learn from studies, regulatory agencies can decide if an investigational medication for a specific condition could one day be approved and made available to the public. Before the regulatory agencies, like the U.S. Food and Drug Administration, or FDA, can approve a clinical trial to begin, potential treatments must be studied in laboratory animals first.

Steve: I see. And for those who might be unfamiliar, could you breakdown the different phases of clinical research, and let us know what phase the UNISUS study is in?

Jonathan: Yes. We have a deep responsibility to the people we serve; we work tirelessly to advance and improve how medicines are discovered, developed, and made. Phase I is the first study in humans. These are small studies with 20–100 participants, mostly healthy volunteers. The main purpose for phase I studies is to evaluate the safety of the study medication at various doses. During phase I, researchers evaluate how the study medication is absorbed by the body and removed from the body, while also determining any potential side effects of the study medication. If a study meets its endpoints in phase 1, it will then move to phase II with the doses selected. Phase II studies are small studies with around 100-500 participants, who are patients with a disease being investigated. Phase II studies typically assess safety and efficacy, which is whether the study medication works for a particular disease. Then we get to phase III studies, which is what the UNISUS Study is. Phase III studies are large studies with around 500 or more participants. These are the main studies for final approval by health authorities. The main objectives for phase III studies to investigate are as follows: to further evaluate safety and efficacy (whether the study medication works for a particular disease), and how the study medication compares to already existing standard therapies. The next phase is phase IV, which take place AFTER a study medication has been approved by regulatory authorities for prescription use or public use. The goal of phase IV studies is to evaluate safety and side effects during real world use and assess risks and benefits over a longer period of time.

Steve: Could you go over the benefits and risks associated with trial participation?

Jonathan: While it is possible that a patient’s PAH could improve as a participant in the study, that cannot be guaranteed. However, participation in research may help people with PAH in the future. Your consent to participate in the UNISUS Study is optional, and you can withdraw your consent at any time. It is possible that patients could experience one or more side effects during this study. Before a patient begins, however, the study staff will review any potential risks or side effects and ensure that the patient is a good fit for the clinical trial. In clinical trials, patients are closely monitored throughout. Study researchers design protocols that explain the study in detail, and the protocol must be followed exactly as written. An independent ethics committee or institutional review board carefully reviews study protocols before approving studies at a clinical site.

Steve: I see. Could you share a bit more about why the study is being conducted?

Jonathan: The UNISUS study is a research study for adults with Pulmonary Arterial Hypertension. While there has been progress in the development of medications for PAH, there is still a need for medications that can help treat and slow the progression of the disease. In this study, doctors want to learn more about a higher dose of an investigational medication for adults with PAH when compared to a lower dose. If a patient is determined to be eligible to participate, they will be randomly assigned to receive either the higher dose investigational medication, or lower dose. The patient, the study doctor, and the study staff will not know what medication the patient is assigned. However, in the event of an emergency, that information can be provided to your study doctor so he or she may take the right actions for the participant. Additionally, an independent committee, including PAH experts, is aware of what medication the patients receive during the study and monitor their safety. Patients will take their assigned medication once a day while in the study, which could last between 3–6 years. During the study, patients are asked to attend regular study visits to evaluate their health, and also to participate in phone calls from the study doctor.

Steve: What else can a study participant expect during clinical trials for PAH?

Jonathan: Clinical trials aim to show how a study medication affects the participant’s health condition over a given timeframe. To assess how the study medication is working, the clinical trial team evaluates the participant through various assessments. For PAH, teams can monitor the change in PAH symptoms, like shortness of breath, swollen extremities, chest pain, tiredness, and dizziness, along with PAH tests like heart rate, blood pressure, WHO Functional Class, and 6-minute walk test values. These are also known as risk assessments. These evaluations are used alongside the patient reported data, like how you feel and how your symptoms are, to provide healthcare professionals with a better understanding of your PAH. We also conduct standard safety lab assessments to ensure the well-being of our patients. I’m sure the PAH community is familiar with these tests, as they’re fairly standard diagnostic tests for PAH, but I would like to take a moment to have Josephine elaborate on them.

Steve: Sure, Jonathan, I’m sure we’d all love a refresher. Thank you for explaining, Josephine.

Josephine: Not a problem at all, Steve. The World Health Organization, abbreviated as WHO, standardized a test to measure how severe a patient’s pulmonary hypertension symptoms are. This is called the WHO Functional Class. They created 4 classes, class I, class II, class III, and class IV. Class I is defined as symptom free when physically active or resting, more generally speaking, the least progressed version of pulmonary hypertension. Class II individuals have no symptoms at rest, but normal activities such as grocery shopping or going up stairs can cause some discomfort and shortness of breath. Class III may also have no symptoms at rest, but are more limited when performing and completing normal chores around the house. And finally, class IV will have symptoms at rest and severe symptoms with activity. Normally, a patient’s doctor can assign them to one of these classes and monitor their PAH through the different definitions.

Steve: Can you also explain a bit about the methodology behind the 6-minute walk tests? Why are they such an important piece of a patient’s treatment regimen?

Josephine: Yes, of course. The 6-minute walk test was developed by the American Thoracic Society. To keep it simple, the test is designed exactly as it sounds. It works to measure the distance an individual can walk in 6 minutes. A site staff member will explain how this is set up and performed during an individual’s site visit. As an individual’s PH progresses, the distance they can walk for 6 minutes will decrease. This is one of the tests we use to measure disease progression, we also use other assessments like Jonathan had mentioned. A patient’s heart rate, blood pressure, WHO Functional Class, among other things, are considered when evaluating disease progression.

Steve: Very straight forward. Thanks!

Josephine: I thought so too. There are also other ways we can measure PAH progression, through things that are naturally occurring in everyone’s body, like peptides, prostaglandins, and molecules. PAH can cause imbalances in these compounds, which can lead to strain on the heart and lungs in PAH patients. But, that’s enough about the science behind PAH. I’ll now pass it back to Jonathan to review a bit more about the UNISUS Study.

Steve: Thank you, Josephine. Jonathan, can you tell us where this study is being conducted?

Jonathan: The study is being conducted worldwide in about 40 different countries, at more than 200 study sites. There are currently 42 sites in the US and 4 sites in Canada. The study is looking to enroll around 900 patients with PAH and are WHO FC II to IV. We are driven by our belief that “patients are waiting” and there is no time to waste. We do our best to select sites that best serve the patient population. We take pride in selecting our study sites for our patients.

Steve: Just to be clear... What are study “sites?”

Jonathan: Study sites are institutions where clinical research can take place. We personally vet each possible study site to ensure that they’re a good fit for our clinical trial. We advocate for sustainable healthcare systems that value innovation and are accessible to people around the world. A lot of research goes into selecting and opening study sites, so we know that they’ll be a good fit for our patients and study goals. We support our participating sites by ensuring healthcare professionals and patients have access to the information, programs, and support they need to achieve the best results possible.

Steve: Can you tell us a little bit more about the investigational medication?

Jonathan: We work to ensure broad and timely access to medicines through the unique collaborations with governments, healthcare agencies, and others. The lower dose of the investigational medication has been approved for other indications by the U.S. Food and Drug Administration and the European Medicines Agency (EMA), as well as by health authorities in other countries. As part of the UNISUS study, a higher dose of the study medication is now being evaluated by doctors in people with PAH to study safety and efficacy.

Steve: And who is eligible to be in the UNISUS study?

Jonathan: To qualify for the UNISUS study, participants must be 18 years of age or older and must have been diagnosed with PAH. More specifically, participants must have a World Health Organization functional class of II, III or IV. Additional criteria apply, but those are the main requirements. The study site would conduct additional tests and assessments to determine if a patient is eligible. Once a potential patient reaches out to a study site, the site will usually ask for medical records if they’re not already established in their care, and work to set up a more detailed consenting and screening visit. Each site will take the time to work with an individual to ensure the clinical trial is a good fit for them. We work with patients through the entire process to ensure the best possible experience.

Steve: Are there costs for patients when joining a clinical trial?

Jonathan: All study-required visits, tests, and medications will be provided at no cost. Reimbursement for study- required travel is also available.

Steve: What else can you tell us about the study design of UNISUS?

Jonathan: The study was designed with 2 parts. The first part is the double-blind period which we referenced earlier where neither the patent nor doctor know which dose of the study medication they are receiving. If the double-blind part of the study meets its endpoints, then patients that complete the 1st part will be enrolled into the 2nd part, which is an open-labelled part of the study that allows for patients to take the higher dose of the study medication for up to 2 years. If study endpoints are met, and if the study medication is approved for use, participants would be allowed to take the study medication following their participation in the study.

Steve: So, how can patients learn more about the study?

Jonathan: If interested in learning more about the UNISUS study or perhaps participating, patents can search for the UNISUS study on clinicaltrials.gov or visit Janssen’s own Global Trial Finder on the Janssen website. We suggest using “PAH” as a keyword when searching, and then select the correct study for your age group. Please talk to your treating physician before considering if participation is right for you.

Steve: Great, a big thank you to you and Josephine for taking the time to review the UNISUS Study. It’s nice to provide our listeners with some more information on PAH in clinical trials. It was great to hear from you today. Thanks a lot, Jonathan and Josephine!

Jonathan: Thank you for having us, Steve. It was great to share information on the UNISUS Study and help educate on clinical trials for PAH too.

Josephine: Thank you for having us, Steve. 

Steve: And thank you, listener, for joining us here today on I'm Aware That I'm Rare, the phaware® podcast. You can also learn more about pulmonary hypertension clinical trials on our website at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast with our podcast app available on the app stores and be sure to listen wherever you get your podcasts. Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected] 

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Episode 461 - UNISUS PAH Clinical Trial

SPECIAL EDITION: Jonathan Hudson is a Global Trial Leader in Pulmonary Hypertension at Janssen Research & Development and is accompanied by colleague Josephine Garcia-Ferrer, Ph.D and Medical Manager.

Jonathan and Josephine discuss Pulmonary Arterial Hypertension (PAH), the importance of clinical trials, what they entail, and share information on Janssen’s UNISUS Study for adults with PAH.

Learn more about UNISUS Study with the Janssen Global Trial Finder. Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected] 

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Dr. Lewis Romer - phaware® interview 460

Dr. Lewis Romer discusses his work on the PPHNet's Kids MoD PAH Trial: Mono- vs. Duo-Therapy for Pediatric PAH patients.

The Kids Mod PAH study is a research project funded by the NIH that aims to determine the best initial therapy for children with newly diagnosed pulmonary hypertension. The study compares the effectiveness of one medication (sildenafil) versus a combination of two medications (sildenafil and bosentan) in improving the quality of life and functional class of these children. 

The study team aims to provide enhanced care and support to the participants and their families throughout the study. The ultimate goal is to contribute to improving the care and outcomes for all children with pulmonary hypertension.

I'm Lewis Romer. I'm a pediatric intensive care doctor at Johns Hopkins University Children's Center in Baltimore, Maryland. I also take care of children with pulmonary hypertension there. Together with Erika Rosenzweig and Steve Abman, I’m the principal investigator on an NIH-funded study, looking at what is the best initial therapy to get kids with new diagnosis pulmonary hypertension started on. This is called the Kids Mod PAH Study. That stands for kids, children mono or duo, one or two medication therapies for pulmonary arterial hypertension, (PAH). We are, as I mentioned, trying to decide whether kids with a new diagnosis of pulmonary hypertension who have symptoms but are not in heart failure should be started on one medicine, sildenafil alone, or two medicines, sildenafil and bosentan. Both of these medications are approved for use in children by the FDA. They are out there in wide distribution in the community. People have probably heard the names of these medicines.

Our study is to try to answer a really important question. We want to be able to start children on a therapeutic regimen that helps them to be as healthy as they can be to maintain as high a level of function as they can going forward. The study incorporates some innovative ways of looking at that, actigraphy and quality of life surveys that parents and children fill out together, and other ways of looking at the biology of how kids are doing, various blood tests, biomarkers. We're trying to understand, as I say, how best to maintain and improve, if possible, the quality of life for kids with pulmonary hypertension from the time of their diagnosis. Although we know a lot about pulmonary hypertension in children, there has been a terrific effort by the Pediatric Pulmonary Hypertension Network of North America, the PPHNet of which I am a member, that has created a, relatively speaking, very large registry of close to 1,500 children, looking at all aspects of how they presented, what their studies showed, how they were treated and so forth. Still, we don't know the answer to this major question. What's the best first drug regimen to start kids on with pulmonary hypertension? 

Why is that important? Because we want to get it right. We want to give kids the best shot that they can have to maintain their level of functioning and their quality of life and improve it, if possible. We're looking at what's called the World Health Organization, (WHO Functional Class), and where that functional class, which measures how well they're feeling, how well they're doing at one year is our primary outcome variable. That's the main thing we're focused on in the study. The study does go on for two years, because we're interested in more of a long-term impact of the initial therapy. 

There are challenges to our study. One challenge is that both of these medicines, sildenafil and bosentan are FDA approved, widely available in the community. So pediatricians, pediatric cardiologists, pediatric pulmonologists, neonatologists, intensivists feel that they're safe and approved therapies for kids with pulmonary hypertension. So, if a new diagnosis is made, it's much more common now in 2024 than it was even back in 2022 for one of those first responder type care providers to make a prescription for sildenafil, most commonly. They get kids started on medicine and then send them off to a pulmonary hypertension center. This is a big challenge to us, because we have a very important question to answer - What's the best initial therapy? But if kids are already on medicine when they come to be enrolled in this study, to be looked at by the pulmonary hypertension center, we can't turn back the hands of time. 

So, we’re trying to get the word out as broadly as we can to kids, to their family, their friends. This is a really important question that's trying to be answered. We want all the practitioners, all the different types of physicians that I mentioned that are seeing children who have a new diagnosis of pulmonary hypertension, to help us to identify people who could enroll in this study and help us to follow them with very high quality and very innovative ways of looking at the quality of life. What is the best initial therapy? How do you get started on medication that's going to help you to have a better life? That's the question and that's who we need your help with. 

As an individual, being in a study like this means that you as a child, as an adolescent, and you as a family member, parent of that child or adolescent are going to be part of a group that is looking together. This is a very interactive endeavor at what is the best way to help kids with pulmonary hypertension to have a better life. It is a very interactive process. There are tests and studies that are done, as I mentioned, looking at activity. These are things that are not done commonly. This is a very integrated, creative way of understanding what is the quality of life for kids with pulmonary hypertension. These tools and studies are not part of regular care, so that's a very exciting thing. 

Being part of an endeavor that's trying to answer a question for all the kids in the world with pulmonary hypertension is something that appeals to a lot of people. Many of the children and their families that we take care of really feel a very high level of altruism. They want the best thing for themselves, but they also want the best thing for the next patient, for the next kid, for the next family. This is a way to contribute to that. 

The alternative is that if these medicines are out there and people are prescribing one, it's the first thing that comes to mind or the easiest thing to get, and children are having those medications and on those medications and their situation goes however it goes, they do well, they don't do well. We're missing an opportunity to answer this question. Those are kids that didn't have an opportunity to be part of a solution to this major question. What is the best way forward? Once the world starts to decide, well, it doesn't really matter what you treat kids with first, then our opportunity to answer this question really goes away altogether. So, it's time sensitive. 

We are making available a brochure to patients and their families that actually contains a graphic called The Patient Journey. So, what's different about getting involved in care for your pulmonary hypertension and getting involved in the Kids Mod PAH study? Many of the things are exactly the same. Typically, the standard of care is that you come to the pulmonary hypertension clinic every three months. They're going to be echocardiograms, where they put that microphone on your chest and get a sound picture of the heart. You're going to take some blood work that's going to be done about every three months. There're going to be six minute walk tests for kids that are eight years of age and older, where they're walking for six minutes and we see how far they go. There's some other standard sorts of things occasionally that will come up, as needed. X-rays, EKGs, those sorts of things. If the doc says they want them, that's the standard of care. 

On top of that, what does the Kids Mod PAH study add? It adds an app on your smartphone or your parent's smartphone that helps the family and the child stay in touch with the study team, keep track of the medication diary, keep track of anything unusual that might happen. You get a happy birthday, to know we're thinking about you. It involves filling out a survey about what is the quality of life like when you come to clinic every three months. It's not every time, but it's at those scheduled clinic visits that those surveys are filled out. Then, it's an opportunity to wear a really cool watch, which keeps track of your activity that's worn for about a week at various intervals throughout the two years that one is in the study. Then, that is an opportunity again, to do something new, do something cool. What is my activity like? You can ask questions about that. Those are pieces of information that are going to the study community to try to understand this question. What's the best way to improve and preserve quality of life for children with pulmonary hypertension? 

Am I getting better care if I'm in the study than if I'm not in the study? I think it's a question a lot of people are going to ask. I think the answer is that the standard of care for kids with pulmonary hypertension is that there's a multidisciplinary group at their center that really cares about them and they want to know about any questions they have, any problems that are coming up, any side effects from the medication. All this are looked at very carefully. What we all strive for is a very personable relationship between the care team and the child and their family. At all points, that's the standard. What does the study do to change that? It potentially enhances the ways in which people are in touch. There is this app that I mentioned that keeps the study team in touch with families. There are phone calls from the study team that happen every month, even those months that the kids are not coming to clinic. Those might happen if the family needs to talk, or if the kid has a problem, there's a side effect. Those might happen anyway just for standard of care. Not that the phone doesn't get answered, but being in the study, those are scheduled. So, there is more opportunity if it's needed to interface with the study team. 

The actigraphy is kind of cool. It's not part of standard of care. It's not able to be afforded by standard of care kinds of situations. So, it's an extra added benefit of being in the study. The same goes with the quality of life scales. They're just not part of routine care. That doesn't mean that the study team doesn't care about how the kid is doing and the same kinds of questions generically are going to be asked, but they're not going to be asked as systematic or detailed away. 

We really appreciate this opportunity to reach out, tell you about what we're doing. We hope to make a difference. We hope to be able to answer that question. What is the best drug regimen to get started on as a child or adolescent or your child or adolescent gets a new diagnosis of pulmonary hypertension. I really appreciate your time and your consideration of this study. I also want to say a big thanks to the PPHNet, all the people that have worked so hard to make this opportunity available. It's a really exciting thing. It's a team endeavor. Thanks again. 

I'm Lewis Romer, and I'm aware my patients are very special and very rare.

Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected] #phawareMD #PPHNet @PPHNet @HopkinsMedicine

Learn more about the Kids MoD PAH Trial: https://clinicaltrials.gov/ct2/show/NCT04039464 

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Episode 460 - Lewis Romer, MD

Dr. Lewis Romer discusses his work on the PPHNet's Kids MoD PAH Trial: Mono- vs. Duo-Therapy for Pediatric PAH patients.

The Kids Mod PAH study is a research project funded by the NIH that aims to determine the best initial therapy for children with newly diagnosed pulmonary hypertension. The study compares the effectiveness of one medication (sildenafil) versus a combination of two medications (sildenafil and bosentan) in improving the quality of life and functional class of these children. 

The study team aims to provide enhanced care and support to the participants and their families throughout the study. The ultimate goal is to contribute to improving the care and outcomes for all children with pulmonary hypertension.

Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected] #phawareMD #PPHNet @PPHNet @HopkinsMedicine

Learn more about the Kids MoD PAH Trial: https://clinicaltrials.gov/ct2/show/NCT04039464 

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Morris Salem, MD - phaware® interview 459

In this episode, Dr. Morris Salem, a pediatric cardiologist, discusses his involvement with Camp del Corazon, a camp for children with congenital heart disease. Dr. Salem emphasizes the importance of addressing the emotional well-being of these patients, in addition to their physical health. He believes that the camp helps boost their self-confidence and allows them to have normal experiences. Dr. Salem also mentions the positive impact the camp has on parents, some of whom become involved in planning and fundraising for the organization. Dr. Salem is also a 2024 Gala del Sol Honoree. This gala is a major fundraiser for the camp taking place Saturday, April 13, 2024. Click Here for Tickets. Click Here to take part in the virtual auction.  #galadelsol #campdelcorazon #heartcamp #camplove #supportagoodcause 

Hi, my name is Dr. Morris Salem. I am a board certified pediatric cardiologist and I have an additional board certification in adult congenital heart disease. I work at Kaiser Permanente in Southern California and I am happy to say that I'm one of the honorees at the Gala del Sol, that's going to be occurring on April 13th, 2024. I've been involved with this camp, Camp del Corazon, for over 20 years. I was very close friends with the founders of this camp, Lisa Knight, and I continue to be very close friends with Kevin Shannon, MD. Over the last 20 some odd years, I've been referring lots of patients to this camp. It's truly been an incredible experience when these patients come back and tell me about the best time that they've had in their entire lives and that they want to go back. I have on my desk right in front of me a prescription pad that says, "This is a prescription from me to you to go to camp." I find kids that are 7, 8, 10, 12, teenagers who are shy, who are in a space in their lives where they're not confident at all. They don't have a whole lot of friends and they're not feeling confident about themselves. In the 15, 20, 30 minutes that they're in the office with me, it's pretty obvious. I've known a lot of these patients their entire lives from when there were fetuses all the way up to however old they are, and you can tell. You can tell that they're just not confident. They are very gun shy and perhaps very self-conscious about who they are and what they're able to do and what their role is in life. Every time I see one of these patients, I run to my office, grab a prescription and hand it over to them. I tell them that they have to do this. This is a requirement. This is medication. A fair number of them actually take me up on it and sign up for camp. A lot of these kids are really sheltered by their parents just because of everything they've been through. The parents are just as hesitant as the kids are in terms of letting their little babies go off and do whatever they need to do. Now, obviously they're not babies, they're older kids, but they treat them like they're a piece of ceramic. They're very, very sheltered. It is quite difficult to get both the parents and the kids to agree to go to this, but a lot of times there's a lot of tears shed on both the parents' part and the kids' parts before they leave. But once they get there, it is a transformative experience. It is one that completely changes their outlook on life. They don't want to come back. I had one patient, recently, who fit this scenario precisely. Parents didn't want him to go. He didn't want to go. I forced him to go. He went and due to some weather circumstances, they actually got trapped there for an extra day or two and he did not want to come back. Absolutely did not want to come back. I saw him shortly after his trip and he was just glowing, bubbling. He's going to be a frequent flyer. He's going to keep going back until somebody tells him he cannot go back anymore. It's always very difficult when you get patients who have done nothing wrong. This is not of any fault of themselves. This is something that's just happened to them and unfortunately they have to learn to deal with it, and our job is not just to help them deal with it from a physical standpoint, but also from an emotional standpoint. The physical standpoint, we can take care of. We have medications, we have procedures, we have all sorts of different things that we can do. We can tell you with the ultrasound what the heart function looks like. We can give it numbers, but the emotional aspect is something that's a lot more challenging for us to deal with. Learning how to treat these patients as not just as patients, but as human beings who think like any other child does. They need to experience things that other kids who don't have these limitations and don't have to think about, can I do this? Can I not do this? Should I do it? Should I not do it? That's really what keeps us going and keeps us motivated to keep pushing and treating these patients and helping them to have normal experiences. That's what the point of all of this is, not just the body, but the mind and the soul. I think camp and self-confidence and your ability to see your self-worth as who you truly are and not as a diagnosis really helps open up a lot of opportunities for you. These kids come back and they want to do things. They want to go out and run a little bit faster in PE. They want to join that team. Maybe they're not the fastest, maybe they're not the strongest. It doesn't matter. They just want to be involved. That in and of itself really helps to propel them and open up more and more and more doors and really make them productive members of our society, which is very important. It's very interesting, I think a lot of our parents are also incredibly positively impacted by this camp experience that their children have. One of my parents of a child who actually attended camp has actually become very, very invested and very, very involved in the camp in terms of planning and in terms of fundraising, in terms of all aspects of this camp. It is a sense of belonging. It's a sense of making a difference and taking this experience that you had nothing to do with provoking or bringing onto yourself, but turning it into a positive aspect and turning it into something that you can share with other people who are going through the same experience. Being at Kaiser, we take care of roughly 5 million members in the Southern California region. As you can imagine, that's a huge, huge number of patients that we deal with. I've been at Kaiser close to 25 years, and I've seen and taken care of thousands upon thousands of patients. Part of what I do is really coordinate and take care of all sorts of patients with congenital heart disease and all sorts of associated conditions from San Diego all the way up to Bakersfield. I'm well known and affiliated with all the major institutions in town, including UCLA, Children's Hospital of LA, Loma Linda, everybody in Southern California. It's through my affiliation with all these institutions and my referral to the camp, many, many patients over the last 20 years, that the camp committee took notice and said, "Wait a second. Maybe this guy who does a lot of work and refers a lot of patients should be someone we recognize." I really want to encourage everybody to look into this amazing organization, Camp del Corazon. They are a purely volunteer led organization with one goal and one goal only in mind, and that is to take care of these children and their families with congenital heart disease. They do this out of love. They do this out of commitment. They do this out of a sense of purpose. This Gala that's set for April 13th, 2024 is their major fundraiser. It is where they collect a majority of the funds that they're going to be utilizing over the summertime for this camp that is held in Catalina. This camp is really intended for patients who are special, patients with congenital heart disease, other affiliated conditions like pulmonary hypertension, anything that involves the heart and the lungs as a chronic issue. These are the types of kids that attend this camp in a very safe and very closely monitored fashion so nothing can happen to them. If it does, there are people there who can take care of them and make sure that they're safe. It is free of costs. All expenses are completely paid for, mainly from fundraisers such as this. There is a virtual camp for families that cannot make it to California to make it to this camp. There is both a silent auction and a live auction that will be offered at Gala del Sol on April 13th. That is well, well worth everyone to sign in and look at if you can't physically make it to this Gala. My name is Dr. Morris Salem and I'm aware that my patients are rare.

Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected]

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Episode 459 - Morris Salem, MD

In this episode, Dr. Morris Salem, a pediatric cardiologist, discusses his involvement with Camp del Corazon, a camp for children with congenital heart disease. Dr. Salem emphasizes the importance of addressing the emotional well-being of these patients, in addition to their physical health. He believes that the camp helps boost their self-confidence and allows them to have normal experiences. Dr. Salem also mentions the positive impact the camp has on parents, some of whom become involved in planning and fundraising for the organization. Dr. Salem is also a 2024 Gala del Sol Honoree. This gala is a major fundraiser for the camp taking place Saturday, April 13, 2024. Click Here for Tickets. Click Here to take part in the virtual auction. #galadelsol #campdelcorazon #heartcamp #camplove #supportagoodcause  Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected]

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Brittany Valim - phaware® interview 458

Brittany Valim shares her experience with pulmonary hypertension and her journey towards a heart and double lung transplant. She now lives a fulfilling life as a mother, student, and aspiring nurse. Brittany encourages others to embrace life and ride the wave, appreciating the rarity of their experiences.

Hello, my name is Brittany Valim. I am 34 years old, and I was diagnosed with pulmonary hypertension in 2010. In 2009, I decided to go on a big hike, they're called the Punch Bowls in Santa Paula, California. I noticed walking up to the trail, which is a really steep hill, I started getting out of breath. I thought it was just because I just had a baby two years ago and I'm overweight. We kept going on this trail. The more and more I kept going, the more and more I started losing breath. I was holding onto tree branches to climb up these hills. Finally, when I got down from the hike, five hours later, I decided that I was going to relax and just figured my body was just not ready for that. I decided that I was going to go to the doctors. I was wrongly diagnosed for a year with high blood pressure. I was rushed to the ER after many other ER visits and I was told I had pulmonary hypertension. They sent me home to go see another doctor who disagreed with the emergency room doctors. A month later, I was rushed to the ER again, and that's when I was kept in ICU and was told that my heart was so bad that I could have possibly died. When I was in the ICU, they were trying many different IV medications. I remember them trying one of them. I remember getting so sick that we could not do it. I think I was in the ICU for about two to three weeks. My hardest thing was not, “Oh my goodness, this disease I have,” it was more, “My son is at home, and I cannot see him,” because the swine flu was pretty big at that time, so no visitors could really come in. There were people passing away all around me, so that was freaking me out and giving me anxiety. Then, at the end of my stay, we finally figured that Letairis and sildenafil would be my treatment from then on out. I have idiopathic pulmonary hypertension. They have no idea how I got it. I believe that it was from my son. I can't quote that, no doctor has said that's what it's from. But when I look at pictures from after me being pregnant until I was sick, my neck was pretty swollen and so was my face. I just took that as maybe my heart had a lot of fluid around it, and no one really saw it before then. I was 21 years old, and I wanted to do what every other 21-year-old wanted to do, and that was going dancing and hanging out with friends. So, those first years, actually, for a long time, I struggled with having this diagnosis because I was in my 20s, and I just wanted to hide everything under the rug, so I acted like everything was fine. I went out and probably did way too much more on my body than I should have, but I was still taking medication. In 2018, I got sent to the ICU again. We used to tell my son that it's just, "Mom needs a tune-up. Mom needs a tune-up." I was put in there, and my doctor and my nurse at Santa Barbara Cottage Hospital sat me down and told me, "Hey, look, we need to try something new." They came up with the IV meds, the sub-q and Tyvaso at the time. I was in my late twenties now, and I said, "Oh, I want Tyvaso." I was going to beauty school, so I didn't want a pump. The thought of having something attached to me all the time just was not for me. I did good on Tyvaso, but I hated taking it. After I started Tyvaso, I was able to go to the gym. I was able to kayak with my family at that time, but I was just not compliant with taking it. It was just too bulky. I had to put it in a bag and everybody always stared at me when I was taking it. I was over it. I decided to tell my doctor that I did not want to do it anymore, and I was no longer compliant with it. He suggested that I do sub-q or IV. I had a serious talk with my nurse, at that time, at Santa Barbara who is amazing, she's my saving grace. She told me that sub-q would be really hard, and that's when I decided I want the intravenous line. From then on out I was taking it and it was making me very sick for a long time. I finally realized that, hey, I need to change my life and what I'm doing. When I got sent home on oxygen that same time, the pump isn't what scared me so much, it was the oxygen that got me shook. That's what caused me to say, this is the real deal now, this could be your life forever being on oxygen and that's when I decided to change my partying habits, my cooking habits, my exercising habits. I was ready to change. IV meds, did save my life for a while. For about five or six years, I was on IV meds, and in 2018 when I moved to San Jose, California from where I had been before, I started noticing that I was just not breathing well. I couldn't work as much. I was getting sick more often. We started talking about the transplant list. So in February, in 2020 is when I officially got put on the transplant list. I had to have my mom come in and help me live, because there's times I couldn't even get out of bed I was so weak. I needed help with my son. Just house cleaning, I have two dogs. But it still didn't hit that, “Oh my goodness, I'm one day going to be on a table and I'm going to get lungs and a heart.” It didn't really hit me yet. When I was first diagnosed with PH, I was really alone. I didn't know that there was these groups online. Every time I went to a support group, they were all older than me. Everybody was older. I just didn't feel connected to anybody, so I felt like very alone. My mom, we were fighting a lot, because she would tell me things that I shouldn't be doing. I was telling her, I'm a grown woman, I know what's right for me. I know when to take a break. It was just really hard at first and towards the end, that's when I finally felt support from my friends, family. Then, I started becoming closer to people on Facebook. Then I started reaching out to people on Instagram. Then, I backed away for a little bit because I was starting to face the realities. I was going through so many things. I thought I was having a heart attack at times when it was just anxiety. I had a lot of support, especially from family. I had family and friends were more supportive than I've ever thought. I had so many cheerleaders of people who I had been friends with for years on Facebook and Instagram. They were just cheering me on every day. That's when I started showing my stages of this is me today, this is how I feel. One weekend, I was with my family, it was my best friend who is my cousin, it was her baby shower. That following Monday I got a call saying, "Brittany, we noticed some of your symptoms and some of your testing, we would like you to come into the hospital. We just want to check you out." I said, "Okay." I went in. They did more testing. They let me go home. I got a call on a Wednesday and they said, "Hey, I want you to enjoy your weekend with your family, but on Monday, I think it's time to put you on ECMO if we don't get a call before then." That following Friday morning, I got woken up by my boyfriend and he's telling me, "Brittany, they're calling you. They're calling you." I said, "Why are you lying to me? Why are you lying to me? Why would you even say something like that?" He said, "No, I'm serious." Because for some reason I turned my ringer off. My mom came running in my room and she's like, "No, Brittany, you need to call them back." I called them back and then that was the reality of me getting a heart and lung transplant. This was at five o'clock in the morning is when I called them back. They told me that I needed to be at the hospital by eight o'clock. Well, when I got the call, I sat there in awe for a second, because my son was still sleeping, and that was the hardest thing for me to do was leave my son. I wanted to wake him up. Well, I tried to, I don't know if anybody's ever tried to wake up a 13-year-old. They refuse to wake up. I tried and he was just kind of like, "Okay, okay, bye." Of course, being a mom and my son is my rock, that was really hard. I decided I wanted to take a shower, because I wanted to be clean. That's when we started packing my bags. But my mom said if there is anything else I needed that she would definitely pack a bag for me. To get to the hospital, it was about 40 minutes. Since it was during COVID, only one person can take me up to the room. My mom chose my boyfriend to do that. I was sad about that, but I was really happy, because me and my mom were very close and I know she was stressing out. So if we had been together, I feel like I would've gone into the OR with all of this anxiety, because I knew that my mom was hurting. I wasn't even thinking of me. I was just thinking about how sad my mom, but happy she was and so scared was. I couldn't even think about how scared I was. When they were ready for me, they allowed my boyfriend to wheel me down in a wheelchair to the OR. I remember talking to all the nurses. My stomach was just in my throat. They were asking me what type of music I wanted. I told them I don't even care what kind of music there was. I remember joking with the nurse, sitting on the table staring at this white ceiling with the big hood light. I remember telling her, "Has anybody ever told you no, nevermind. I need to go." She was like, "No, nobody has." I said, "Well, I might just be your first one to want to leave the OR." We kind of laughed about it and she gave me encouraging words. As soon as that phone rang, I heard one of the nurses in the OR say, "Okay, it's time to go." They told me to count backwards, and I don't even think I remember counting. It was a 12 hour surgery. I got out. I think it was three hours out of the surgery I started bleeding into my lungs and they had to take me back to the OR. They told my mom that it is pretty common for that to happen. After that, everything was pretty well except for waking up and realizing you cannot see. That was the hard part. I had TED's disease, so I woke up with very, very blurred vision. I couldn't see who my nurses were. I couldn't see who my doctors were. I couldn't see where my mom was. I couldn't see anything. My eyes were just so blurry I could not see. In the end, we think that it happened to be with so much trauma on your body that it caused me to have retinal detachment in both eyes. Within these three years, because my transplant was in 2020. My right eye is pretty good. I still can't see very well through my left eye. I think that's just damaged for good. My life has been forever changed. I did have a bump in the road, of course, diabetes. Diabetes comes with the territory of transplant, but mine has stayed with me. I got put in the hospital, I believe it was six months after transplant. I was just not feeling good. I was throwing up and I finally called my team because my mom, of course, made me, and my boyfriend was like, "Yeah, you do need to call the doctors." I did. I went into the ER and my blood sugar was at 650, my glucose. After that, I started realizing, okay, I definitely need to eat a lot better, which I was pretty much doing anyways because you're on so many restrictions out of transplant. You have slow emptying of your stomach. You have all of this stuff, so you can't eat fiber. Your potassium might be too high. So you're trying to figure all this out. I remember being on a high dose of steroids. I actually yelled at my mom, in one of the grocery stores, because I didn't know what to eat anymore. I just told her I wanted a starve and not eat anymore. But diabetes was my only bump in the road. I consider myself very lucky. I am the mom that I've wanted to be. I wish I can go back and be this mom back then with PH, but it was obviously hard. I am going to school now. I am a full-time nanny and I take ballet. I've been a dancer for a very long time. I did it in high school. I just always loved to dance, and I finally took myself to the JC here, and I decided to do ballet for fun. If I could give advice to someone in their twenties or just anybody in general or to my 21-year-old self, I would say ask for help. Find other people who are like you, or people going through the same thing as you, because they really do know what you're going through, emotionally, physically, they do. I know everybody's tolerance level for pain and emotions are always different, but you need someone to talk to. It's really hard to do this alone. I tried it and it wasn't the greatest. With a PH diagnosis, I didn't really live day-to-day. I didn't think of myself as passing away. I never thought about that, because I don't know if it has to do with me being a mom or not, but I just could not fathom not being here. I would fight to stay here just for my son. After having the diagnosis, I still have a great outlook on life. I ride the wave, definitely ride the wave. I now go to school and I'm going to school to be a nurse, and while I'm waiting for the nursing program, I would love to get into med school. I plan to be an endocrinologist. I've never been able to think about school, it was always, "Am I going to have enough money? How am I going to work? Can I work? Am I too sick to work?" Now I don't have that anymore. I don't have that, am I too sick? I don't feel sick anymore. My nurses, I have to thank. There's one in particular, and if she listens to this, she knows who she is. She was not just a nurse, but she did above and beyond. I'm sure there's nurses that are great out there, but she still my cheerleader today. My name is Brittany Scarlet Valim, and I am aware that I am rare.

Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected] 

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Episode 458 - Brittany Valim

In this episode, Brittany Valim shares her experience with pulmonary hypertension and her journey towards a heart and double lung transplant. She now lives a fulfilling life as a mother, student, and aspiring nurse. Brittany encourages others to embrace life and ride the wave, appreciating the rarity of their experiences.

Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us on social @phaware Engage for a cure: www.phaware.global/donate #phaware Share your story: [email protected] 

Listen and View more on the official phaware™ podcast site

#pulmonary hypertension #phaware #rare disease #lung disease #clinical trials #podcast #PH #asthma

[phawareglobal]

Cam Wells - phaware® interview 457

Stroke survivor and journalist, Cam Wells, discusses his mission to change the way disability stories are portrayed in the media.

Cam also discusses his work in the disability community, including his involvement with the Italian Canadian HandiCapable Association, which provides sports and recreation opportunities for people with disabilities.

Cam believes that disabilities and rare conditions affect everyone in some way and advocates for inclusivity and respect. #RareDiseaseDay

My name is Cam Wells. I am a stroke survivor and I hold four diplomas from St. Clair College. After getting the first one, which is journalism, I came to realize there's a great imbalance in the way disability stories are typically portrayed in the media. Portrayed as either really bitter or really inspirational. You don't often see the middle ground. I figured after graduating, I could set myself the task of trying to change all that. I've been actually doing a show [Handi Link].  for 15 years now. As a person with a disability myself, I've come to appreciate that there are so many beautiful shades out there to be appreciated. I've actually been honored both locally and nationally. I'm a nominee for Canada's Disability Hall of Fame. God willing, I'll actually get it sometime. I've interviewed everyone from a cast member of TV's Breaking Bad to a couple from Corner Gas, but really the best stories are the ones that come totally out of the blue. Like during the opening of the pandemic, there's a quadriplegic pilot, a friend of mine who had lost I think it was his home and his vehicle to a fire. The health restrictions didn't allow people to do very much, but this town got together and they said, "We want to rebuild for this guy. He's part of our community." So they took it upon themselves and did what they could to help him out. I was your typical kid. I didn't know much about the world beyond just your typical kid. You go to school, you spend time with your friends and family. That was my world. September 29th, 1995, I was eating a can of frozen juice, I remember. I was at school having lunch and I thought I was having a brain freeze. Must've been the world's biggest brain freeze, because it resulted in a first round of neurosurgery and six days in a coma, which is I might add, the only time in my whole life I got enough sleep, but I couldn't move my left side. For 35 days, I was in the hospital and went through various rehab procedures only to find that part of the anomaly in my brain had been left behind. They couldn't get it. I had what was essentially an experimental procedure at that point. I was the youngest kid they had ever done it on at this hospital. It wasn't available where I was, so thanks to a long and tedious insurance battle, ultimately, we were able to get what was called gamma knife surgery and successfully remove the anomaly. I came back a different person. The truth is, I would not change it for anything. I basically discovered who and what I am spending time among people with disabilities and various rare conditions. In an academic sense, yeah, you can learn the medical model of this condition may be this or that, but you can't learn the strength factor. For every condition out there, there's always that one unaccountable person who's defied the odds. The best way of learning about such people, just spend time with them. Share a meal, share some time, tell them a bad joke, what have you. Probably the biggest moment for me was... and I’m not real proud of this, but I chose entirely the wrong program my first year of college, entirely the wrong thing. I figured because I was working in a job related to the field, I'd be good at the academic side. I was not. I actually found myself academically dismissed from the college. Through various acts of misunderstanding, I was basically forced out and spent an entire summer begging to get back in. But when I found myself doing the correct field of study, something I could do, a journalism scholarship was granted to me by a disability group that I now work for. That's where I met Egidio Novelletto, a man who was advocating for his people. I grew more and more fascinated by the work they were doing, and I started writing articles on the subject and even did a field placement doing public relations materials for them. I realized that so many stories are just not shared. You look at a traditional sports page and you'll see some great athletics, but every night of the week, there's disabilities athletics. There's people who medically complex. They're out there doing it. They're putting on a great show, playing a great game, but where is the representation of that? Now, it was a long gap between my years in elementary school, which I was in when I had the stroke and my years in college, but the funny thing is the reporter who did the story on the experimental surgery I alluded to was actually later one of my professors. As a graduation gift, I gave her what may be the only surviving copy of that footage. I became more and more fascinated by the world of disabilities, and since I was doing PR for them, I wasn't able to do anything with the radio station at the time, but the program director, she left the door open. She basically said, "If ever you want to do anything, come and pitch a show for us." Basically upon graduating, I went to them and I said, "Hey, can I do something on disability issues?" Well, the rest is history. However, I realized that there were others involved in similarly interested stuff. I actually worked with a team in 2013 to release a guide for radio stations on accessibility. Some of the things people don't think about like adapting your physical space, or some of the language you use. Even things as simple as the lighting for people with seizure disorder. I'm coming to realize that I wasn't the only person who actually wanted to see the best in people, wanting to see them living up to their own strengths. It was really an eye-opening experience. One thing I always have maintained, when a person with a disability gets up and they make toast, that is not inspirational per se. That is called breakfast. That is a very human thing. But really people with disabilities, they do what they do just to live the best life they can. That is the principal reason behind my work. As a trained reporter, I love a good story. I love hearing tales of what society doesn't think is possible. I thrive on that. But yeah, in a way, owning it for myself is part of the process. One thing that I've shared any number of times, I had a childhood bully, horrible, horrible experience, I might add. He used to try and stick magnets to my leg to see if it was real, and as I do the show sometimes when I'm interviewing somebody working with younger kids, I think to myself, "I don't want anyone to have those feelings that I experienced in my own youth. I don't want anyone to ever ask the question, am I good enough?" Knowing that you're rare, knowing that you're unique is a way of owning it. I have a friend, Daniel Brenner, great, great guy. He's actually a robbery survivor. He was shot as a child and witnessed several people in his family being essentially murdered in front of him. He was told he'd never walk again, but he picked up a guitar and he used a form of self-healing. He actually gets by just fine now. In fact, I have another friend named Renee who runs a music studio, and she was looking for a teacher, so I introduced them and now they're working together. One of my primary roles in the disability community is working for the Italian Canadian HandiCapable Association, which is an organization that does sports and recreation for persons with disabilities. It's my honor to work alongside them as they provide things like yoga, tae kwon do, soccer, all manner of things for people with disabilities who might not necessarily have any other athletic opportunity. Fact is, when you're out there and you're an athlete with a disability or anything medically complex, you're not thinking about the factors that might hold you down. You're not thinking about somebody telling you you're not good enough or you'll never do that. You're just living in the moment and enjoying the game. The ICHA was founded by a man named Egidio Novelletto, whose son wasn't allowed to participate in a traditional soccer program, which was a very eye-opening experience for him. He decided to found a charity, and working with various levels of government, he was able to build a complex and establish something meaningful for persons with disabilities to be a part of. Normally, that's where it comes from. Having somebody personally connected, it's usually the gateway. Truth is, when it comes to disabilities, rare conditions, they affect us all. There's a term I heard in my work once, not disabled yet, meaning we prepare terms of respect and inclusion. We're only making our own futures better in so much as we're all affected. If you pass 10 people in the street, somebody's got some factor that makes them unique. Somebody has a mental health concern or an emotional disability, or maybe somebody in a wheelchair. One of my primary sources of information is NORD, the National Organization for Rare Disorders. I consult them on an almost weekly basis to reach out to disability and rare disease groups around the world. My thinking is if it's affecting one person here, it's affecting others out there. I figure it's a universal concept, or rather a series of universal concepts. Yeah, you can solve the problem for yourself at home. That's great, but if others around the world don't have the same access to medical information or don't have the same resources or even wherewithal to ask those questions... A few years ago, I actually interviewed an accessible theater in China and I might add I had a very difficult time placing that call due to various satellite connections, but they actually have this great thing where they have a sign language interpreter in stage productions. However, they're an acknowledged character. They're part of the show. Are they providing a service for the audience? Sure they are, but they're actually included. It's shown me what the world really is, if that makes any sense. I've met people and learned things I never would have otherwise. Honestly, one of the greatest people I ever knew, Dr. Marcia Rioux, she was the head of Disability Rights Promotion International, God rest her soul. She introduced me actually to one of the UN Representatives who was involved in writing some of the disability legislation that has now been embraced pretty much the world over. The work I do, there is no end, honestly. It's a stepping stone, but it's incumbent on everyone to try and take that step forward. I always say on the air, it's not about an immediate cure, but it's about the work towards getting there and disability, rare conditions, they affect us all. Maybe you're not the one affected directly. Maybe you're friends with a caregiver, or maybe you just happen to have a friend of a friend. It's the degrees of separation. You can't look at any family. You can't go back all the way or as far back as humanly possible and not find some factor that makes a person unique or rare. As we go, we gain understanding. Look at things like chronic fatigue syndrome. That's a big one. 20 years ago, doctors would argue, "No, that's not a thing. You're just getting old. Just go home, get some sleep. These days, we look at it and we recognize this is a condition. It's about taking the next step forward. It's not about listening to anyone who says, "There is this glass ceiling. This is as far as you can go." Progress is built on the idea that a person will test it for themselves. I had a professor once, a lady I actually have a lot of respect for now who told me it was physically impossible for me to pass some speed drills in my college days, because I only type with one finger. My exact response, "Watch me." And I got through it. There's one story I would like to share as a shout-out to some of the people that I've had the pleasure of meeting over the years. You never know who's going to be connected to who, so keeping an open mind and supporting others. 27 years prior to this, I remember there was a cartoon I loved watching. After the stroke, it was part of my rehab. It was my one half-hour during the week that I didn't have to think about the rehab or the pain. An organization I worked with provided some promotion for Easterseals. They happened to have two of the voice actors from that show speaking at an event in Windsor. So I called in a favor, got photos with them, autographs, and just got to share a little bit of their story. After all those years, I was able to say, "Your show saved my life, so thank you for that." It was actually the 90s X-Men. I was a bit of a comic enthusiast. I actually got to meet Gambit and Rogue, nicest people in the world. They were so kind to me, I cannot stress that enough. But the fact that I had a chance to actually tell them what their work had meant to me. It's important in any condition, any rare disease, just to acknowledge when somebody is there, when somebody does some good. Yeah, there's a lot of hardships people have to endure, but there's always support and if you find the right people and you show them that you appreciate what they do, it just stays with them. My name is Cam Wells and I'm aware that I'm rare.

@RareDiseases @DRPI_global @EasterSeals @CJAMFM 

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