Bob Underwood, MD (Host): Cystic fibrosis is a genetic disorder that causes damage to the lungs, digestive tract, and other organs of the body. The Children's Health Team believes that effectively treating cystic fibrosis means focusing on both the pulmonary aspects of the disease and the resulting digestive tract complications.

This is Pediatric Insights: Advances and Innovations with Children's Health, where we explore the latest in pediatric care and research. I'm your host, Dr. Bob Underwood. And today, we'll hear about the Cystic Fibrosis Program at Children's Health and how it's providing comprehensive care under one roof for children with cystic fibrosis.

With us today are three experts, Dr. Preeti Sharma, pediatric pulmonologist and Co-Director of the Cystic Fibrosis Care Center at Children's Health; Dr. Meghana Sathe, pediatric gastroenterologist and Co-Director of the Cystic Fibrosis Care Center at Children's Health. And Dr. Melissa Ham, pediatric endocrinologist at Children's Health. Each of them also serves as faculty at UT Southwestern. Thank you all for being here.

So, Dr. Sharma, can you tell us about the Claude Prestidge Cystic Fibrosis Center, what it is, and what services does it provide?

Preeti Sharma, MD: So, our Cystic Fibrosis Center cares for about 300 children and young adults with cystic fibrosis in North Texas, providing care in a multidisciplinary fashion to help them manage all different aspects of their chronic disease.

Host: That's phenomenal. You have 300 kids with cystic fibrosis all being treated right there in the same center. I mean, that makes you all experts in this particular kind of care. So, Dr. Sharma and Dr. Sathe, as the only Pulmonary and GI co-directors in the country for cystic fibrosis programs, what unique challenges and opportunities does this present? How do you provide cohesive collaboration between the different specialties at the clinic?

Preeti Sharma, MD: So, I think that it really starts with teamwork, and we are very much able to work together collaboratively, not only the physician providers, but with the remainder of our staff, our nurses, respiratory therapists, dieticians. So, I think that that kind of having everybody in one place provides team cohesiveness, that's very helpful. Like any chronic illness, sometimes things overlap. And so, being able to speak to my colleagues, who have more expertise in, you know, Gastroenterology and Endocrinology in real time about our patients is unique to our center, and it's a wonderful opportunity to make sure that we're taking care of our patients in a more robust manner. And so, I think it's really been beneficial for us too, because it allows us to get kind of an immediate consultation in areas that we need assistance with in real time.

Meghana Sathe, MD: In addition, I think our team is great at collaborating together. We meet every week and plan for the visits in the following week. And so we've discussed the patients and come up with a plan on who the patients need to see, not just including Pulmonology, GI or Endocrinology, but also who needs to see our nurse educator, our dieticians, our physical therapist. A lot of the visits in terms of GI are done in collaboration with the dieticians, so we aren't repeating ourselves in terms of the questions we ask the families, and the families are able to understand that not only is it truly collaborative, but they can see us having those conversations together and with them and making plans that are really patient and family-centered.

Host: Yeah, I think when you talk about that, that's what I think about is really focused on the patient and the family, and having that multidisciplinary approach and that coordination of care is just phenomenal and really shows advances in cystic fibrosis care. So, Dr. Ham, can you elaborate on the role Endocrinology plays, especially in managing cystic fibrosis related diabetes?

Melissa Ham, MD: Sure. Patients with CF can be affected by several complications that might need to involve an endocrinologist As you mentioned cystic fibrosis related diabetes or CFRD is the main one that most people think about. CFRD is unique in that there are features of both type 1 and type 2 diabetes. There's a component of insulin deficiency as in type 1, but also insulin resistance which is the cornerstone of type 2 diabetes. This means the approach to treatment also has to be unique. Typically, with type 1 and type 2, we are measuring and often limiting carbohydrates in an effort to improve blood glucose levels. But restricting carbohydrates and calories in cystic fibrosis is clearly not desired since these patients have higher caloric needs, and low weight is often a concern as well. We allow these patients to continue their high-calorie and high-carb diets and match this with insulin to control their blood glucoses, working closely with the patient's gastroenterologist and CF dietician.

The other issue with CFRD is that CF patients experience great fluctuation in their glucose control when they get sick from a pulmonary standpoint. So, understanding this and working closely with their pulmonary treatment team to make insulin adjustment is critical during those times.

Host: I think that's phenomenal. I mean, the work that you guys are doing, I did my training a while ago, and a long time ago. We really thought of CF as a pulmonary disease, and we did not have this multidisciplinary approach that your team is bringing to the table here, and that's phenomenal to me.

So Dr. Sharma, receiving comprehensive care at one clinic is really beneficial for patients. Can you share why and discuss how a multidisciplinary approach is especially essential for managing complex cases?

Preeti Sharma, MD: Yeah. And so, I think, as you pointed out, we've commonly thought of cystic fibrosis as being primarily a pulmonary disease, when in fact, multiple organ systems are affected. There's a lot of interplay. So if I'm seeing a patient who has a decline in lung function, and weight loss, that can be related to having elevated blood sugars, or, you know, the weight loss could be related to their lung disease, but it could also be related to, changes in enzyme dosing, et cetera. And so, it's really helpful for our patients to know that we're kind of approaching all of their issues from that multidisciplinary standpoint, and we're doing it expeditiously. So, you know, if you needed to make a referral to another clinic, you'd get an appointment a week later, versus all of us are in one place at the same time. And so, even if a patient is scheduled, you know, in many instances, just to see one provider or one discipline, we're all there together and we can say, "Hey, you know, I've noticed that this is what's happening for our patient, and I really need assistance with that" or "How can we work together to create a plan for our patient and their family that meets all of their needs, but also takes care of all their medical issues?"

Host: Yeah, I mean the efficiency of coordination like that is such a benefit for the patient and their family. It really, really helps with their time and their understanding of the disease process. So Dr. Sathe, what is the Cystic Fibrosis Therapeutics Development Network, and how is your team involved in it?

Meghana Sathe, MD: The Cystic Fibrosis Therapeutic Development Network is a consortium of CF centers that are part of the CF Foundation who work together to optimize research in the area of cystic fibrosis. You're either born with CF or you're not. And so, there's a limited amount of patients out there in order to further science through studies. And not any one center has enough patients to really conduct effective studies on their own. So, this is a way in which we work together to enroll patients into pivotal studies like the CFTR modulators that recently came out and were life-changing for many of our patients in terms of their lung function, their nutritional status, even many of our patients who were potentially needing a lung transplant had such great improvements that they no longer are on a lung transplant list. So, we are part of this network, and it's a lot of fun to be able to bring those studies to our patients. The patients definitely understand that the improvements we've seen in the last 40 years are because of their involvement in research.

Host: So, you sort of answered this question already, how does the participation in clinical trials through this network benefit current patients? And you talked about people actually coming off the lung transplant list. And how do you see the future for patients in being able to participate in these clinical trials?

Meghana Sathe, MD: Yeah, so I think it's really beneficial for patients and families to participate in these studies. And the Foundation is doing a great job of actually including many patients in these studies. So, there's a lot of observational studies as well as therapeutic studies that we participate in. I think it empowers patients and families to take ownership of this disease, especially being a genetic disease that their children are going to have forever. I think there are some great new studies in terms of genetic therapy that are coming down the pipeline for those patients who are not on modulator therapy or eligible for modulator therapy. And our adult colleagues at UT Southwestern are participating in some of those as those studies usually come out in adults first. And so, we're hoping that we can bring those studies with the success that they see over there to our patients here at Children's Health as well.

Host: That makes the future sound really bright. I appreciate all the work that you all are doing with that. So Dr. Sharma, what are your goals for the future of the Cystic Fibrosis Program and how do you envision further improving outcomes and patient experiences?

Preeti Sharma, MD: So, I think that, you know, just like Dr. Sathe mentioned, bringing some of the new and cutting edge therapies to our patients who are not eligible for the current therapies like modulators, but also making sure that for those of our patients who are taking modulators, that we're still addressing the fact that, while they feel healthier and they are healthier, we are still assessing for their other needs, whether those are psychosocial needs, educational needs, et cetera. And so, by continuing to build our multidisciplinary team and bringing in mental health professionals, you know, we have wonderful social workers and a psychologist, and making sure that our patients have access to those experts as well, I think is going to be part of what our comprehensive care includes in the future.

Host: That's terrific. It really is. And Dr. Sathe, how do you envision the future of the program?

Meghana Sathe, MD: I think that, in addition to research, our center also participates a lot in quality improvement, and we actually have involved patients and families in our quality improvement efforts. So, just as Dr. Sharma is alluding to, they again become empowered by sort of understanding what the new basically future is with modulator therapy and CF and how we can personalize medicine, but we have their input in those quality improvement projects. So, I'm looking forward to doing more of those in the future.

Host: Well, personally, I'm duly impressed with all of the work that you are doing and the whole patient experience that you're providing. Thank you so much for all of your time with us today. And to our audience, thanks for listening to Pediatric Insights: Advances, and Innovations with Children's Health, where we explore the latest in pediatric care and research. To learn more about the Cystic Fibrosis Program, as well as other information, go to childrens.com. And if you found this podcast helpful, please rate and review or share the episode and please follow Children's Health on your social channels.