Pediatric hepatologist Emily Perito, MD, and Sang-Mo Kang, MD, surgical director of the Pediatric Liver Transplant Program, describe how their specialized team provides expert care for children with liver diseases who may need transplantation. They present strategies for expediting access, achieving exceptional survival rates and minimizing immunosuppressants. Drs. Perito and Kang emphasize the team’s commitment to collaborating with referring providers and ensuring children are supported throughout the transplant journey.
I'm Emily Prudo. I'm a professor of clinical pediatrics and a pediatric hepatologist or liver doctor here at UCSF Benioff Children's Hospital. I have the honor of being the medical director of our pediatric liver center, and today I'm going to talk to you about pediatric liver transplant. I'm pleased to share the stage today with Seng Mo Kang, who's a professor of surgery and the surgical director of our pediatric liver transplant program at UCSF. Our Pediatric Liver Center of Excellence was set up as a multidisciplinary clinical team who's really designed to take care of any need that our patients and families who have kids with pediatric liver diseases might have. Um, you can see here some key members of our team. That includes me and some of our other hepatologists, including Sue Ri, Kate Chang, Shirad Wadwani, Preta Mohanty, as well as the transplant surgeons, including Seng Mokang, the pediatric surgeons, including Amran Nigal, who's the surgical director of our pediatric liver center, Arum Rangaswamy, who's our oncologist that's really has, um, incredible expertise in managing pediatric liver tumors, as well as our other colleagues in radiology, interventional radiology, and pathology. And together, we really strive to provide multidisciplinary coordinated care for kids with liver diseases or who may be in need of liver transplant, um, and really walk their families through every part of the journey, um, and anything that may come up during it. Here you can see the contact information for me and some of my colleagues who helped lead the pediatric liver Center. One of the Most important things is that we want um for the families that we're taking care of and for the referring providers that we're working with to have um really easy way to contact us. We really wanna be accessible for whenever you need us. Um, you can always contact us directly at the emails you see here. Um, importantly, particularly for patients and family. We have a patient navigator named Lily Greenwald, whose job is really to provide a single point of entry to any specialist or any service that you might need through our pediatric liver Center. And so she's available to consult with you to help you figure out what you need, and to talk to you to help to contact any of us, again, to get you to whatever specialist or service you need within the pediatric liver Center easily. You can always reach out to us, um, 24 hours a day through the UCSF Access Center and talk to the provider on call. We'll put this contact information back up at the end of the presentation, but just again to emphasize the importance of accessibility of being able to contact our team whenever you need us because we're here for you. The UCSF and Children's Hospital Liver Center of Excellence is set up to take care of a very broad array of different, um, liver. Diseases that can present in childhood. We are with patients and families and with their referring providers from the beginning steps of diagnosis through, um, managing all different aspects of these diseases. Here you can see a partial list of some of the rare pediatric liver diseases that our specialists have expertise in. Um, this includes biliary atresia, acute liver failure, malignant liver tumors. Uh, metabolic diseases, several of the rare genetic diseases that can present in childhood, autoimmune liver diseases, and things that are more common like steatohepatitis. We work with different specialists depending on where you are in this spectrum of diseases. For example, in the malignant liver tumor, um, area, our oncologists work really closely. with our transplant team as well as the surgeons. Um, this is a good example of where we're with patients from the very beginning of diagnosis often, and we're thinking of all the different possible treatments that we can offer. Um, if a liver tumor is resectable, then our pediatric surgeons and our transplant surgeons work together to try to, um, resect or take out that tumor instead of transplant. Um, another example is portal hypertension, where sometimes a child could be really helped or treated with interventional radiology instead of needing a surgery. But upfront, we're gonna talk to you as a patient or family or referring provider about all the different options in terms of specialists that you might access through our pediatric liver center, and then really work with the family to try to figure out which is the best option for, for your child at that time for treatment. The liver transplant journey is a complicated one. As you can see on the diagram at the Top. There are many different stages to this journey. There are many different people involved. Our job at UCSF is to keep the patient in the center of that journey. As you can see in the diagram here, the blue line where you follow all the way across through the, um, time of diagnosis of the liver disease, through the transplant, and into life afterwards. Our goal is to accompany the family along this journey and try to keep it as simple and comfortable as possible. for the patient and the family through access to transplant, finding the right liver donor for, for a particular child at a particular stage of liver disease, through the liver transplant itself, and then into the many decades of life after liver transplant as we follow the child through the rest of their younger years into adulthood. I'm gonna go through now several different strategies that we use at UCSF to try to help kids get through that journey again as comfortably and as efficiently as possible. Um, when we have a child with life-threatening complications of liver disease, our goal is to get them access to liver transplant as quickly and safely as possible. And to do that, we really want to do everything we can to prevent waitlist morbidity or kids getting sicker on the waitlist, as well as mortality. Um, mortality of kids on the liver transplant, um, waitlist remains a large problem for the US overall. And one of the ways we can help prevent that is by getting kids access to liver donors more quickly. Here you can see some specific statistics from UCSF that demonstrate our outcomes. On the left side, you can see our transplant rates, particularly for children, at UCSF's Pediatric Liver Transplant Center. You can see from the dark blue bar that our transplant rates are higher. We're getting kids to transplant faster than most other local, even regional and US centers are able to do. And we'll talk um in the coming slides about some specific strategies that we use to help get kids to transplant faster. On the right side, you can see pre-transplant mortality rates. In the light purple is expected rates for our center, and you can see that the dark blue bar is missing because we have not had any pre-transplant mortality rates in the two years depicted here. Um, so we have been able to get kids successfully. To transplant without any wait on any deaths on the waitlist for children in the last couple of years. So again, most important for helping kids through transplant and into life after transplant is getting them successfully to transplant, and we have been really proud of our outcomes at UCSF over the last couple of years for accomplishing that. Hi, my name is Sang Mo Kang. I'm the surgical director of pediatric liver transplantation at UCSF. I'd like to talk to you about how transplants are performed uh for children. The majority of donors in the United States for children come from deceased donors, or 89%, and only about 11% are from living donors. And uh in the case of the living donor, we uh take a a portion of the uh uh donor's liver and we're able to uh transplant into a child who is generally much smaller. For a deceased donor, uh, we can often split the liver if the liver's too big into two different pieces. You can see that on the lower right hand side of the slide, or we actually can take. Uh, liver and split it into two parts and transplant a part into a child and another portion into the adult from the same donor. Uh, some, in some cases the liver fits, uh, perfectly fine and we can just transplant the whole liver, uh, into a child. So how do we improve access to liver transplants at UCSF? We work really proactively to, to help children to access transplant. Uh, by exploring really all types of donors, uh, especially living donors, because we know that living donor transplants have the best outcomes and we can also provide a transplant, uh, when the child needs a transplant instead of when the child finally rises to the top of the transplant list. We also have uh a lot of experience with what we call non-directed or some might call altruistic donors who volunteer to uh donate a portion of their liver to a child, and that can be an incredible resource to get children transplanted uh before they get too ill. We, uh, of course, uh, often have to use, uh, deceased donors. Uh, we use ABO incompatible donors for children under 2 years of age. We use complex, uh, size reduced graphs or split graphs as I showed you on the previous slide. And we also have been using a new technology called machine perfusion that can improve graft quality and also allow us to transport livers across the United States uh in case there's a specific liver that we want to transplant into our child. We also want to work hard to ensure appropriate waitlist, uh, priority and by submitting PEL or uh MELD exceptions to increase, uh, the waitlist priority score. As I showed you before, in the United States, the deceased donor usage is about 89% for kids and 11% living donor. Uh, but at UCSF we actually have a 46% living donor transplant rate. And uh which only leaves us with about 54% that we have to use cadaver donors, so we're very proud of that and. That's part of the reason why we're able to get kids to transplant sooner. So, as Sengmo mentioned in the last slide, reduced size grafts from living donors is a really important way that we help kids um access liver transplant at UCSF. There are different ways to do that, and these are just a couple of examples from some of the amazing families that we've been able to work with over the last few years. On the right side, you can see a picture of two mothers that were able to donate to their own children. That's a living-related living donor transplant. And in that the surgeons take a small piece of the liver, usually from the left side, um, from the mother or from another relative that's usually blood type matched to the child, and they transplant it into the child. Um, on the left side, you can see a picture of a family with the, um, little girl in braids in the middle, who benefited from what we call a non-directed living donor or an altruistic donor. And we're extremely fortunate at UCSF to have close collaboration with our living donor center, who's able to screen living donors and Figure out who the right matches for for them might be and really guide them through the living donation process. So this little girl was matched with a non-directed living donor who donated again a portion of the left side of his liver to this little girl who was then able to really grow and thrive after that. Um, the Living Donor Center is well equipped to educate donors about what the living donation process looks like for them, and, um, you know, really exciting outcomes for kids after transplant as they grow and thrive. After, uh, any kind of liver transplant, including liver. donor, the liver grows with the child. So even though they receive a small piece of, uh, an adult's liver, that's the right size for them at the time, as they grow into adolescence and adulthood, that liver grows with them and continues to supply the liver that they need to again grow and thrive and do well. We've been really excited, um, on the surgical side for, um, the living donations to start happening by with robotic surgery, which is even less invasive for the living liver donors themselves, really decreases recovery time, allows For shorter times in the hospitals, um, and so provides a nice opportunity for the living liver donors to be able to donate, but then get back to their own lives in an even shorter time. As I mentioned before, getting the child just through liver transplant is just the beginning of the journey. Um, our team is really committed to helping children and families through the entirety of life after liver transplant. Um, and really trying to optimize the health of the liver and the child through that entire journey. The biggest risk time in terms of graft failure or even losing a patient after transplant is in the first year after transplant. So, on the left side, we're sharing again, recent statistics from UCSF and our pediatric liver center. On the left side, we're sharing some recent data from UCSF about the highest risk time after transplant, which is in that first year after pediatric liver transplant. Um, we're monitoring really closely and treating complications that in some cases can lead to graft. Failure or even lead to patient death. There is an expected rate of that, as you can see in the purple, um, but the lack of dark purple bar indicates that our rates of graft failure and patient death have been well below expected, um, and among the best in the US, which is something we're really proud of and really committed to continuing at the UCS. Pediatric Liver Center. Getting through the first year is just the beginning of the journey. You can see on the right side some information about our outcomes in the 3 years after transplant. Um, on the top, you can see rates of graft failure and on the bottoms of patient death. Each dot represents a center, and UCSF is represented in dark blue here. You can see that our rates of graft failure and rates of patient death, also in the 1st 3 years after transplants are among the lowest in the country. And again, I think working really closely with the patients and the family to meet their needs, and then really utilizing and leveraging. Our multidisciplinary expertise in taking care of these very complicated patients, um, is really key to our ability to achieve this. When we get into the longer term, 3 years or more after liver transplant, one of the biggest things that we're thinking about is optimizing immunosuppression. Liver transplant, for most kids, is a lifelong commitment to taking immunosuppression, which helps prevent rejection of the liver and um protect the liver graft so that it stays healthy inside the child. But that immunosuppression, the medications that help prevent rejection, also can create a burden. They increase risk of infections, and in the long term, they can increase risk of malignancy and cause damage to other organs. So this balance of um of really balancing protecting the liver with the burden on the child is something that we're always thinking about and Always looking for better ways to adjust as we get into the, again, years and decades after transplant and are following kids through adolescence into adulthood. One of the important things for us at UCSF is that patients and families are able to access not just our expertise at our center, but really the collaborative expertise of a lot of different pediatric liver transplant experts across the country. So, several years ago, we helped co-found what's called the Starzal Network for Excellence in Pediatric Liver Transplantation. It's a learning health network that's dedicated to improving outcomes, um, and improving health for kids after liver transplant. And here you can see four of the main areas of focus of the Starzal Network. Um, optimizing immunosuppression is one that we help lead here at UCSF, and I'll talk a little bit more about what that looks like going forward. But we've worked together with Centers across the country to really try to come up with consensus about what's the best immunosuppression, not just for kids here, but for kids across the country in terms of optimizing long-term outcomes. We've also worked with the Stars on Network on, on, um, strengthening patient-reported outcomes and quality of life to make sure that the pediatric liver transplant centers are really listening to what is important to patients and families as we help balance things like immunosuppression and other burdens. Um, focusing on surgical and perioperative practices and really trying to optimize that early time after transplant to get the, um, kids through as safely as possible. And then thinking all the way through towards adulthood in terms of the transition of care, thinking again with, with, um, at our own center and across centers about how do we help guide patients and families through that transition of care from the pediatric liver centers to the adult centers that will continue to follow them through. I'll just go a little more into an example of optimizing immunosuppression, how we've work on that and work on innovating at UCSF and how we hope that helps patients both at UCSF and at the other liver centers across the country, um, going forward. So the mainstay of immunosuppression long term after transplant for almost all children is tacrolimus. It's a type of calcinerin inhibitor that again helps prevent rejection and protect the liver. But the calcin inurin inhibitors over the long term can cause some damage to the kidneys and cause um increased risk of infection and even malignancy. So the question for kids that we're always thinking about is how low can we go? How, um, how much can we minimize immunosuppression to protect the child, um, over the long term and really optimize their health. This effort at UCSF started more than a decade ago with a small study that focused actually on complete immunosuppression withdrawal, and then watching liver function and child health from kids that had gotten um a liver transplant from their parent. And this was a multi-center study um sponsored by the NIH but um really started at UCSF by um one of the transplant surgeons, where we followed kids and found that in a very select group of kids, almost half of them could actually come all the way off of immunosuppression. Um, and we watched them over time to try to figure out more about which kids is it that we can minimize or even take off of immunosuppression, and how can we predict who those kids might be. So, as a next step in this journey of, again, working at UCSF, um, and really leading innovation for UCSF and other liver transplant centers, this year in 2026, we're gonna start enrolling. And what we're calling the iSynapse trial. It's a multi-centered trial sponsored by the NIH in which we'll be enrolling children that are stable after liver transplant, but earlier post-liver transplant than children in previous trials, and systematically working on reducing their tacrolimus dose by 50%, and really watching carefully to try to understand the biomarkers that we can use to predict who will be able to successfully and safely minimize their immunosuppression. And understand what that immune process looks like, um, in terms of developing tolerance. Over time. So, I think this is just one example of where you have UCSF and our pediatric liver Center really seeks to innovate and to push the boundaries of what we can offer to children and families in the long term after liver transplant. And then also to collaborate to really make sure that innovations and advances that we're offering And developing here are available to kids across the country at other liver transplant centers that we're both, um, you know, learning what we can here and then integrating expertise from other centers to really offer the best possible care to the kids and families we take care of and other kids and families across the US. Well, thank you, Emily. As Doctor Perrito has uh told you, there's a lot of complicated issues in transplantation. As a parent, you're likely confused and trying to figure out where to go, um, and so we're here to help you. We're, we, uh, know that we have a great multidisciplinary team that can take care of almost any kind of liver disease and, uh, we try to avoid transplantation if at all possible. But there we know that there will be some kids who eventually need a transplant, and at that point we want to afford them every opportunity to get a transplant with all the technical innovations that are available. I do believe it's very important for parents and for referring doctors to look up the data on various centers if they have a choice of where to go in for either for liver care or for a liver transplant. The data is actually out there with the SRTR database that can be easily searched on Google. And so we want you to make a, a, a decision based on actual uh outcomes, but also knowing that we take care of patients in a holistic way, not just from a medical perspective, but we have a very large team of social workers, dietitians, etc. that will help uh with all the other things that go along with being a patient and as you all are well aware, when you have a sick child, there's a lot of different. Uh, uh, resources that you need to really get through the process and so we're quite well aware of that and we wanna provide that for you. So just, uh, putting the slide back up of, uh, the key members of our pediatric, uh, liver center of excellence, as well as, uh, again mentioning Lillian Greenwald who is our patient navigator who can help you get to the right person. Uh, and also if you're a referring doctor, you can get a hold of us 24 hours a day through the UCSF Access Center and of course feel free to email us, uh, if you have direct questions for us or if you're having any trouble getting through, uh, to our center.
