Bill Klaproth (Host): Welcome to Pediatric Insights: Advances and Innovations with Children's Health, where we explore the latest in pediatric care and research. Today's episode is a special edition featuring our In The Know series, a new addition to our podcast lineup that brings you insightful conversations with our Chief Medical Executive, Dr. Dai Chung, and expert physicians from across Children's Health. We'll be sharing these special episodes regularly to keep you informed on emerging trends and advancements in pediatric care straight from the leaders who are shaping it.

In this episode, Dr. Chung is joined by Dr. Preeti Sharma, pediatric pulmonologist at Children's Health and Associate Professor at UT Southwestern; and Dr. Meg Sathe, pediatric gastroenterologist at Children's Health and Professor at UT Southwestern. Together, they'll discuss the cystic fibrosis program and how it provides patient families with multidisciplinary care in one location.

Dr. Dai Chung (Moderator): I'm Dai Chang. Welcome to the In The Know video series. Good afternoon. I have today two great leaders at Children's Health UT Southwestern Medical Center. First, Dr. Meg Sathe, who's a Professor of Pediatrics and a pediatric gastroenterologist, and Dr. Preeti Sharma, who's a pediatric pulmonologist and a Clinical Associate Professor of Pediatrics at UT Southwestern. Welcome.

Dr. Preeti Sharma: Thank you.

Dr. Meghana Sathe: Thank you. We've been co-directors of the Cystic Fibrosis Center here at Children's Health since 2013. And we have worked really hard to build a national reputation and provide excellence in care to our patients and families. We have an amazing team that allows us to do this.

Dr. Dai Chung (Moderator): I understand we have the only cystic fibrosis program where it's actually co-led by expertise in pulmonary and GI. So, you got to tell us a lot more about it.

Dr. Preeti Sharma: So, it is unusual. The Cystic Fibrosis Foundation accredits CF Care Centers across the country, and we're the only accredited care center with two center directors from different disciplines. And it's really important because cystic fibrosis is very much a multi-organ disease. Patients with CF are equally affected by that gene mutation in their lungs and their gastrointestinal tract. And so, this gives us the opportunity to make sure that we're taking comprehensive care of our patients.

Dr. Dai Chung (Moderator): What is it about the pathogenesis of CF that we should be putting all the resources to really do the great care that you provide?

Dr. Meghana Sathe: One misconception is that CF is only in the Caucasian population. That's kind of what we were taught in medical school. Back then, they really only knew about the F508 homozygous mutation when it was initially discovered. And since then, we have discovered about 2,000 mutations in the gene that can lead to different manifestations of cystic fibrosis. And so, this now is seen in all ethnicities.

Dr. Dai Chung (Moderator): So, with the number of gene mutations, what is it about patients' inability to secrete enzymes or key functionalities?

Dr. Meghana Sathe: On the GI side, I think it all has to do with growth and nutrition. CFTR is, we say, in every tube in the body. So, your gut is one big tube. But it's also in your pancreatic ducts and the ducts in your liver.

And so, patients often present with growth failure or steatorrhea, not gaining weight as much, because their pancreatic enzymes are not able to be delivered to the small intestine, where they help digest food. So, that's one of the most common manifestations that's seen early on. But they can also have problems with constipation and meconium ileus, which you're probably very familiar with.

And in addition, people don't really think about the liver very much with CF, but because you have CFTR in the bile ducts, if you have damage to the bile ducts, then you can also have damage to the hepatocytes. And so, patients can actually end up with cirrhosis, and we have a few CF patients who've needed a liver transplant. Luckily, that's very, very rare, but it can happen.

Dr. Preeti Sharma: Interestingly, you know, young children generally have fewer pulmonary manifestations, and that's something that occurs over time with exposure to bacteria. But things that we see as kids get older, it's not just what we commonly think of as a cough or pneumonia. It's recurrent sinusitis because of the mucus impaction in the sinuses. It's symptoms that can look like asthma, but asthma that doesn't respond to the asthma therapies we commonly use. And so, lots of different pulmonary manifestations that can be seen over time.

Dr. Dai Chung (Moderator): Roughly about how many patients that are established, and how many patients do we get referred in from the region as new patients?

Dr. Meghana Sathe: We have about 280 to 300 patients. I would say we have 20-ish new infants a year. As patients are doing better and living longer lives, our adult colleagues are up to 450 over at UT Southwestern. And we have a robust transition program for our patients that gets them from our clinic to UT Southwestern, or really anywhere else, they're going to go to college now, or, "I'm going to move."

Dr. Dai Chung (Moderator): With all the advances, the lifespan of patients with CF gotten longer, right? How does a transition to adult care take place?

Dr. Preeti Sharma: The transition program in cystic fibrosis is very much driven by our team. We have a phenomenal team. Our social workers, our psychologists. We have a nurse educator who all work together along with the remainder of the staff to really think about how do we build their self-efficacy as they're looking towards becoming young adults and moving on to the adult program?

Dr. Dai Chung (Moderator): What about being as a UT Southwestern physician working at Children's Health?

Dr. Meghana Sathe: I'm also the program director for our fellowship program in GI. One of my favorite things is we have trained about eight or nine CF fellows. So, they've gotten grant funding through the CF Foundation for part of their clinical training. And just being able to bring them into this multidisciplinary clinic and teach them.

Dr. Preeti Sharma: It is a great model for other disease processes, and it's been really interesting to look at that and try to emulate that across different chronic illnesses.

Dr. Meghana Sathe: In smaller centers, they may not have enough of a population to have a full center. So, we actually, with your help, added El Paso as our affiliate center, and Tyler has also been our affiliate center.

So, we can help provide additional expertise and support to these programs and provide care for patients closer to home. But then, if they need our help, we can do a telehealth, or we can have their patients participate in research that is happening over here.

Dr. Dai Chung (Moderator): Love to hear your thoughts on what do you think is the just around the corner or perhaps in the near future biggest advancement in clinical care in CF?

Dr. Meghana Sathe: I've actually been very lucky to be part of some studies that we're doing here at Children's on non-porcine pancreatic enzyme replacement therapy. And so, I think we have a medication that hopefully will really meet the mark. People have been trying to get this to fruition for many a years, but I think we're really close.

Dr. Preeti Sharma: Over the last, you know, five to seven years with the advent of highly effective modulator therapies, we've made a lot of strides in CF care just through that. But there's still about 10% to 15% of the population who have gene mutations that are not responsive to highly effective modulators. And so, I think that's kind of the next area of focus therapies that can provide better, more comprehensive management for those patients who can't take modulators.

Dr. Dai Chung (Moderator): I thought I knew plenty about cystic fibrosis, but I learned quite a bit today. And I really enjoyed speaking with both of you. And thank you so much for all the great work. I mean, it is really amazing.

Bill Klaproth (Host): Thanks for listening to Pediatric Insights: Advances and Innovations with Children's Health, where we explore the latest in pediatric care and research. We'll be back soon with another special edition featuring our In The Know Series with Chief Medical Executive Dr. Dai Chung, where he interviews expert physicians from across Children's Health.

You can find out more at children's.com. And if you found this podcast helpful, please rate and review or share this episode and please follow Children's Health on your social channels.