Experts from the Pediatric Liver Metabolic Transplant Program at Mount Sinai Kravis Children’s Hospital discuss their interdisciplinary collaboration—and why it is so important in treating pediatric patients with inherited disorders of metabolism. The group consists of doctors and specialists from the Department of Genetics and Genomic Sciences, the Recanati/Miller Transplantation Institute, the Division of Pediatric Hepatology, and the Division of Pediatric Nephrology and Hypertension. Close collaboration between physicians, clinicians, nutritionists, and social workers is essential to improve the long-term functional health of the children treated in the program. Weekly team meetings between the physicians facilitate the coordination of care, faster scheduling of appointments, and better decision-making for each patient.
For more information, visit or call: mountsinai.org/care/genetics/services/pediatric-liver-metabolic-transplant 212-541-2387 Hi, I'm George D s. I am the division chief of Medical, the division of medical Genetics and genomics at the Icahn School of Medicine at Mount Sinai. My name is Kimiko. Is she an assistant professor in the Department of Genetics and Genomic Sciences and pediatrics. I'm working for metabolic diseases. I'm John, Book of Alice. I'm, uh, transplant Hepatology ist and professor of pediatrics at the Icahn School of Medicine at Mount Sinai. Um, I work closely with the transplant program with the Recanati Miller Transplant Institute and also very closely with Dr so she and Diaz Thio address the needs of Children with inherited disorders of metabolism. So the program in clinical genetics at Mount Sinai has a very long history. It goes back, really? To the to the 19 sixties, with the recruitment of current personal one who's absolutely recruited Bob Destiny. These Air two giants in the field of, um, medical genetics. They've established programs in prenatal and many other aspects of clinical genetics. Uh, the one that we're gonna focus on today really is the programming inborn errors of metabolism. And so this is a program that has been running continuously since the 19 sixties. The physicians who developed the treatments for maple syrup urine disease on board other international metabolism started at N. Y. U finished. They created Mount Sinai and really have are the ones who trained me and trained Doctor, is she? So We have a very expansive history, Andi. In terms of the importance of all this to pediatrics, Um, all of these disorders are rare, but they're really a medically important. Patients with genetic diagnoses actually occupy a significant fraction of hospital beds in any pediatric institution, and so the medical importance really is quite significant. So now China has always been at the forefront of of innovation in genetics, whether that was in the era of microscopy and sido genetics, whether it was looking at the application of genetics across, um, the population of women who were having Children and wanted more genetic information about those pregnancies, whether it was in the application of novel therapeutics. And really, this is an area where I think we particularly excel the development and application of new therapies for diseases that didn't have treatment in the past. This is particularly true in the case of the disease that affect life simple storage. So, like some storage diseases, But increasingly, we're starting to see new treatments that are applied to the inborn errors of metabolism. Whether they are gene therapies are in a therapies andan the cases where those are not available transplantation. Okay, so let me start that. So we have years of experience of liver transplant for metabolic diseases in collaboration with hip apologists and transplant surgeons. However, we recognize that there is a need of better multi disciplinary approach to manage patients. So therefore, we launched a new collaborative team nearly two years ago, so this is a kind of a very important aspect of our team. And of course, our goal is to reduce the medical and psychosocial burdens on to make make them feel comfortable to continue their ongoing management. I agree with you, Kimmy. I think that's exactly right. You know, it seems like innovation and advances often occurred when you bring two disciplines together. E. I think what's happened here in Montana that really distinguishes us is we bring together the incredible expertise, um, of the genetics and metabolic team. The nutrition is the clinicians and we interface that bring that into direct contact with the expertise and hepatology and leverage while many of the other, um, pediatric institutions across the country have both of those things in their within their institution, I don't think that any can match the collaboration in the way that we work together. And I think that our ability to work together and to care for the patients together really distinguishes us, whether with the ultimate goal being, how to have the best outcomes for the patient. As you said, it's the clinical care, it's the communication of the providers. And when the when the family see what a great job we do working together, I think that increases their trust. They know that communications, but that leads the best outcomes. I think also the other thing that really wanted to emphasize to focus on the long term when transplant started. We're just trying to help people survive, but we know that the outcomes are really good and respected. The inherited disorders of metabolism you're talking about, it really is about improving long term functional health for these kids. And as Dr Diaz said, it's really almost like a gene therapy. We're replacing the apparent, the ingenious abnormal and we're replacing it with a team that is working. There were really our goal is really for long term outcome. And once again it really requires this incredible collaboration with your team and R t people that really not only committed the same thing with people that really like working with each other to Despite being Yankees fans and Red Sox fans on one team, we get along really pretty well. Okay, I really I wish we could have said that about five years ago. Uh, so the inherited metabolic disease program is a program that really grew out of the efforts of two physicians with really, uh, great histories in the borders of metabolism. Selma Sneiderman, who again was one of the pioneers, along with Claude Sansa Rick in the development of treatments for some inborn errors of metabolism that we're starting to identified in the 19 sixties. So when newborn screening became the law of the land in New York state, thes physicians started taking care of patients with inborn errors and follow them throughout their lives. And so, you know, we have a program where we have been following patients from birth into their forties and fifties on dso I think that you know that continuity something you don't see in many places. You know, many Children's hospitals will transfer their patients to other facilities over time. Eso I think you know that is something that is, um, distinguishing for us. And again, you know, we have within our own team a multi disciplinary approach. Eso we have social work, nutrition, clinical coordination That's very integrated. We have child life to our patients. We realized the burden that these patients have coming to clinics so frequently. And so we have, ah, wonderful child live team to kind of help them deal with these traumas and and make things better for them and for their parents. So I think that's kind of what we bring to the game. And, you know, into the partnership with John Steam. I can talk about it s o. It's more about the communication off the physicians basically like, um, patients after the liver transplant needs. Ah, lot of coordination of cares, um, done by many different physicians. However, the difficult things for them was more about the coordination of cares. And occasionally, uh, their providers are not able to communicate each other very well, so we basically have weekly meeting Thio get update each other, and all team members are so engaged. So that's why patients are not, um, having any trouble to get different kind of like service a book or like appointment, for example, our team is able to see patients all together. You know, they don't have to go to one place on another. But ah, patient can come to my clinic and liver doctor. Doctor breakables can come together with us, so that can reduce the any kind of psycho social burden burdens off the patients To re emphasize that, I think that the trust we have among the teams a couple things that really strikes me is how much everybody stresses each other, trust each other, and then also the way that we really are interdisciplinary. So the people it's not. It's a real team working together with different expertise and nutritionists. Social workers are coordinators. Everybody plays a critical role, Um, in in this whole effort with and and we really do, um, are committed to the kids committed to the effort, and it allows us to make really good decisions together, and the communication is so so important. Even when the kids get their transplants, um, they still may have some residual metabolic challenges. And having Kimmy and Georgia on board to help us deal with these is just spectacular. And I think that really improves the outcome for these kids. Well, I think the the way, this way Ask ourselves a question. And Dr D s a doctor Oshawa say this patient might be a candidate. So we asked ourselves the question first. Well, the transplant improve their survival. Their quality of life will really make a difference in their in their lives. And if that if we go forward with that, then they're put on what they call the waiting list. And during that waiting period, our whole goal is to work together with the metabolic team to prevent any other complications to make sure the family is ready for the transplant. Once again, it really depends on communication trust, excellent decision making. And that's on the foundation of the really great content knowledge that the, uh the department the metabolic Injects Group have shared with our group during the peri transplant period. The time of the organ of kids in the hospital want to make sure the new organ works and to make sure they can make that transition to a new way of life trying to manage the new organ keeping healthy. And that effort is really the long term effort. And we work very closely over time with the families, from the transplant team's perspective, with our coordinators part once again with the metabolic teams, nutritionists and their positions say, What's the best path forward? I think I will say that the the there's a couple, but one that really jumps out the size of a child's life is a nutrition support. I think your nutrition team, Um, I'm sure that George and Kimmy can speak to them there calcula early. Um uh, skills and such an integral part of the team. And then they're great people with him Toe work, e Let them speak to their own team. Yes, I mean that John is right that they're uncredible team. I think you know they're the backbone of any metabolic team. The nutritionist really dio you know the brunt of the work. And, uh, they're incredibly dedicated, you know, they just gave themselves thio the patients in a really inspiring way just And additionally, our social workers are working hard to manage patients. Um, so that, um but I can't forget mentioning. Yeah, they they help us deal with the barriers that families are facing. And we once again the partnership between the metabolic team, social worker and the transplanting social workers is really tremendous. They really worked very closely together. So there's multiple different ties among different piece of the team. So I really think it's a situation where, as good as the individual parts are, the whole is greater than the sum of the parts. So I'm very happy to answer that question. So we have a very nice designated website, so please check our website, which also has a video link, and please give us a call to make an appointment with a number below.