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Dr. Lewis Romer - phaware® interview 460

Mar 19, 2024

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.

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Learn more about the Kids MoD PAH Trial: