transplant digest - medstar health · 2019-01-21 · fall 2018 transplant digest a bi-annual...

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TRANSPLANTdigestA B I - A N N U A L P U B L I C AT I O N O F T H E M E D S TA R G E O R G E T O W N T R A N S P L A N T I N S T I T U T E

3 Letter from the Executive Director

5 After Lifelong Struggle With Bowel Disease, Expertise and Insight Help Patient Get Closer to Diagnosis

6 Meet New Our Physicians

7 New Living Will App

8 CASE STUDY: Pancreas Transplantation for Diabetes Mellitus

9 Earlier Referral and Living Donor Options Can Expand Kidney Transplantation and Improve Outcomes

10 Pediatric Liver Specialists Improve Health and Quality of Life for Children With Metabolic Diseases

11 By the Numbers: MedStar Georgetown’s Transplant Programs

For the estimated 40,000 adults and children in the U.S. living with irreversible intestinal failure, intestinal transplantation has the potential to not only save lives, but also to improve quality of life by obviating the need for ongoing total parenteral nutrition (TPN). However, only 109 intestinal transplants were performed in 2017 (the most recent year for which statistics are available), making the procedure the lowest volume solid organ transplant in the U.S. The underuse of intestinal transplantation

has been driven by poor survival rates for both patients and allografts and the need for long-term general immunosuppression, which increases patients’ risk for infection, tumor, kidney failure, neurological complications, and metabolic side effects, such as diabetes and high blood pressure.

“Essentially, intestinal transplantation is caught in a Catch-22,” explains Alexander Kroemer, MD, PhD, a transplant surgeon and immunologist

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New research at MedStar Georgetown Transplant Institute could markedly expand the number of patients who receive intestinal transplants.

Research Team Works to Make Intestinal Transplantation an Option for More Patients

Complications from Pancreatic Cancer Surgery Bring Patient to MedStar Georgetown for Lifesaving TransplantWhile still a high school student in Saudi Arabia, Mohammed Almujel got news that would change his life: he had a rare form of pancreatic cancer. The diagnosis sent the teenager and his family on a medical journey spanning more than 6,000 miles, four years, and numerous surgeries. That journey ended with a successful multivisceral transplant at MedStar Georgetown Transplant Institute, and a new lease on life for Mohammed.

To make matters more complicated, Mohammed first underwent pancreatic surgery at another U.S. hospital to remove his cancerous tumor. That surgery unexpectedly damaged his small bowel and left him with an irreparable foregut and midgut. The only solution was a multivisceral transplant.

“My parents asked for a referral to the best small bowel transplant center in the United States,” Mohammed recalls. “The answer was MedStar Georgetown Transplant Institute.” continued on page 4

IN THIS ISSUE

2 TO SCHEDULE A PATIENT CONSULT, CALL 202-444-3700 .

Research Team Works to Make Intestinal Transplantation an Option for More Patients continued from page 1

Lead investigator, Alexander Kroemer, MD, PhD

year for five years from the National Institutes of Health’s National Institute of Allergy and Infectious Diseases. The study has the potential to help four groups of patients with intestinal conditions:• Patients on total parenteral

nutrition (TPN) and in stable condition: Approximately 40,000 patients in the U.S. are currently undergoing TPN at home without any of the potential life-threatening complications. If the risks of intestinal transplantation were significantly decreased, these patients could undergo transplantation, obviating the need for TPN and improving quality of life.

• Patients on TPN who need an intestinal or multivisceral transplant: With a lower risk of allograft rejection, these patients would be able to undergo transplantation sooner, before TPN-related complications, such as liver failure, deep vein thrombosis, and infections, worsen. Earlier transplantation reduces the risk of post-transplant complications and improves outcomes.

• Patients who have undergone intestinal transplantation: The study goal of developing targeted immunotherapy means that patients who have undergone intestinal transplantation would be able to transition to more targeted therapy, eliminating the potential life-threatening complications that come with general immunosuppression. In addition, patients would no longer have to undergo regular screening endoscopy, which would improve

at the MedStar Georgetown Transplant Institute and director of the Institute’s Transplant Laboratory. “The risk of rejection is high, which leads to over-immunosuppression. This results in complications for the patient and high treatment costs, which in turn dissuade surgeons from offering this treatment to their patients.”

To overcome these obstacles, Dr. Kroemer is leading a team of researchers that includes Thomas Fishbein, MD, executive director of the Transplant Institute; Michael Zasloff, MD, PhD, professor of surgery and pediatrics at Georgetown University School of Medicine and scientific director of the MedStar Georgetown Transplant Institute; Jill Smith, MD, gastroenterologist; and Simon Robson, MD, researcher with the Liver, Transplantation, and Vascular Biology Centers at the Harvard-affiliated Beth Israel Deaconess Medical Center, Boston. The team is undertaking a translational study using human tissue samples from patients who have undergone intestinal transplantation. The study is designed to explore the role that the adaptive and innate immune systems play in intestinal transplant enteropathy and immunological graft loss.

“By identifying the key cells that play a role in rejection of the allograft, we can work towards developing precision diagnostics and therapeutic targets so that patients no longer have to undergo general immunosuppression with all its risks. Our goal is to develop immunodiagnostic and immunomonitoring tools that are tailored to each patient’s specific immune system response,” says Dr. Kroemer.

Unleashing the Potential of Intestinal TransplantationThe study, Unleash the Potential of Intestinal Transplantation, is receiving a half million dollars in funding per

“This research is an essential part of our drive to take transplant medicine to the next level and develop precision diagnostics and treatments tailored to each patient.”

-Alexander Kroemer, MD, PhD

MedStarGeorgetown.org/Transplant 3

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LETTER FROM THE EXECUTIVE DIRECTOR

Dear colleagues,

In our role as leaders and innovators in transplant medicine, there is one thing that never changes—our dedication to putting our patients at the center of everything we do. The care we provide and the research we conduct is focused on one goal—helping our patients live longer, healthier, higher quality lives. In this issue of Transplant Digest, we highlight several of our physicians and patients to give you a feel for how we do that.

In the Center for Translational Transplant Medicine at Georgetown University School of Medicine, Alexander Kroemer, MD, PhD, is working to make intestinal transplantation a safer option for a wider range of patients. Our studies have the potential to significantly increase the number of candidates for this procedure, and to transform immunotherapy for this transplant. Newer treatments could make long-term generalized immunosuppression a thing of the past for intestinal transplant recipients. Implications of this research for the treatment of inflammatory bowel disease reflect the cross-fertilization between transplantation and our other studies of how the gut microbiome impacts intestinal homeostasis.

In the patient stories and case studies shared in this issue, you’ll see firsthand the impact that careful diagnosis and expert care have not only on patients’ health, but also their quality of life. We highlight a foray into the technology world—working with the MedStar Institute for Innovation—with whom we

designed a new app that provides a convenient way for you to speak to and see our transplant experts at any time. It’s like “HIPAA-secure FaceTime®” to discuss inpatient or outpatient problems with an expert at your convenience. We hope it will make it easier to get questions answered and refer or follow up on your patients.

As always, we invite you, our partners in patient care, to provide us with your feedback and input on how we can impact your practice and your patients positively. Let us know what you’d like to read about in future issues and how we can provide you with the information you need as you care for your patients with organ dysfunction, tumors, or those in need of transplantation.

Thomas M. Fishbein, MDExecutive DirectorMedStar GeorgetownTransplant Institute

quality of life and outcomes, especially for pediatric patients transplanted during their first years of life.

• Patients with inflammatory bowel disease (IBD): The researchers hypothesize that IBD is influenced by factors similar to those that cause transplant enteropathy, so new therapies could benefit these patients as well.

Finding Clues to the Role of the Immune Systems in Allograft RejectionDr. Kroemer and his fellow researchers are focusing on the role that both the innate and adaptive immune systems play in preventing or causing allograft enteropathy.

In their preliminary research, they have identified IL-17-producing Th17 cells as the proinflammatory effector T cells in intestinal transplant immune responses. They also discovered that, in newly transplanted patients who are at highest risk for allograft rejection or enteropathy, proinflammatory ILC1s predominate and protective ILC3s are lacking. Conversely, in healthy transplant recipients showing no signs of rejection, protective ILC3s are abundant. The team and other researchers also have identified the key upstream master regulators in the intestinal immune responses in transplant patients as the antimicrobial sensor NOD2 and the ectonucleotidase CD39.

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Complications from Pancreatic Cancer Surgery Bring Patient to MedStar Georgetown for Lifesaving Transplant continued from page 1

But by the time suitable donor organs became available, Mohammed’s condition had deteriorated badly. Explains Transplant Surgeon Cal Matsumoto, MD, who, with Dr. Fishbein, treated Mohammed, “His pancreatic tumor had vascular involvement. After it was removed, he developed a thrombosis in his superior mesenteric artery, which handles all blood outflow for the small bowel. By the time we saw Mohammed, he had no foregut because of the original Whipple procedure performed to remove the pancreatic tumor. The blood clot had caused him to lose all of his small bowel. He was on total parenteral nutrition (TPN) and had developed TPN-associated liver disease, leaving his liver badly scarred. His liver and stomach secretions were drained through a tube, and none of his organs were salvageable. In addition, he had developed end-stage renal disease.”

To regain his health, Mohammed now needed a new stomach, liver, pancreas, duodenum, small bowel, colon, and kidney, so he was listed for a multivisceral transplant. Dr. Matsumoto and his team followed Mohammed carefully while he was on the waiting list, ensuring he was cancer-free before transplant. Drs. Matsumoto and Fishbein performed the complex procedure with a superior outcome.

“This was a very difficult case and few centers have the ability to handle this type of complicated transplant,” says Dr. Matsumoto. “At the Transplant Institute, we’re among the top two centers based on the volume of bowel transplants performed, and we’ve been doing these transplants for many years, working with the same team of experienced surgeons. Thanks to having all those key pieces in place, we have the highest survival rate for intestinal transplantation in the country.”

Mohammed is grateful not only for the medical care that saved his life, but also for the support he and his family received from the

“I am very grateful to have had a second chance in this life. Now I’m helping people with the same severe needs that I had, giving them hope.”

-Mohammed Almujel

Transplant Institute’s staff. “Through a very long hospitalization and recovery, the doctors and nurses helped me remain strong,” Mohammed recalls. “Every step of the way, we were treated like family members, with mental, emotional, and physical support.”

These days, Mohammed returns the favor as a volunteer at MedStar Georgetown. “I am very grateful to have had a second chance in this life. Now I’m helping people with the same severe needs that I had, giving them hope.” n



intestinal insufficiency and nutrient deficiencies, before the patient becomes debilitated.

The patient underwent a full thickness biopsy of the small intestine which showed a deficiency in the interstitial cells of Cajal. Colonoscopy showed no evidence of inflammatory bowel disease, so her abnormality was not felt to be secondary. Her work-up also included a gastric emptying study and anorectal manometry.

“Our goal is to develop an accurate diagnosis and effective treatment plan in a streamlined way. We are a comprehensive multidisciplinary intestinal rehabilitation center, so we are able to offer our patients a full range of treatments, including transplantation when necessary, for all small bowel conditions,” says Dr. Subramanian. n

After Lifelong Struggle With Bowel Disease, Expertise and Insight Help Patient Get Closer to DiagnosisAll her life, the 25-year-old woman had lived with bloating, distention, and chronic constipation. She had seen many doctors, but the only diagnosis she had received was irritable bowel syndrome (IBS). For years, her condition was managed by her primary care physician and a gastroenterologist who prescribed medications to promote relief, but she still lived with the uncomfortable, troubling symptoms.

The turning point in her story came when she developed a small bowel obstruction. A surgeon at a Washington, D.C.-area hospital operated emergently to provide symptomatic relief. After surgery, the patient was referred to MedStar Georgetown Transplant Institute for further work-up and assessment, where she was seen by Sukanya Subramanian, MD, a small bowel transplant gastroenterologist specializing in intestinal failure and intestinal rehabilitation.

“There is often a bias, especially for young female patients, toward a diagnosis of IBS,” Dr. Subramanian explains. “Exploratory surgery had revealed marked global dilatation, an abnormality in her bowel that was difficult to appreciate on imaging studies. Taking a more accurate, in-depth history from the patient brought to light that her episodes of bloating and distention were quite precipitous and resolved spontaneously, which raised suspicion that the underlying cause of these symptoms may be something other than IBS.”

A range of motility disorders can masquerade as more benign conditions like IBS, and progress insidiously. Further, derangement in motility can be associated with mesenteric laxity, so this presentation of volvulus prompted a detailed investigation of motility. Abnormalities in motility can impact nutrient assimilation, so it’s important to evaluate these patients early on for

To be explored as part of the study are the roles and mechanisms of proinflammatory Th17 and protective Treg cell responses, as well as the roles and mechanisms of proinflammatory ILC1 and protective ILC3 responses in intestinal transplant patients and what effect these have on allograft stability or rejection. The study also aims to explain the role of master regulators NOD2 and CD39 and the innate and

adaptive immune responses in patients with stable allograft function compared to those with enteropathy.

“The data we gather will move us closer to developing a more tailored immunotherapeutic approach to prevent and treat allograft rejection so that more patients can benefit from intestinal transplantation,” says Dr. Kroemer. “Our goal is to finalize this immunotherapy approach within

10 years. As one of the largest intestinal and multivisceral transplant centers on the East Coast, this research is an essential part of our drive to take transplant medicine to the next level and develop precision diagnostics and treatments tailored to each patient.” n

Research Team Works to Make Intestinal Transplantation an Option for More Patients continued from page 3


Meet Our New Physicians

Christine Hsu, MDBoard certified in transplant hepatology, gastroenterology, and internal medicine, Dr. Christine Hsu specializes in advanced liver disease and transplant hepatology. Previously an assistant professor in the Division of Gastroenterology at the University of Pennsylvania, Dr. Hsu’s research has been published in Gastroenterology, Clinical Liver Disease, Clinical and Translational Gastroenterology, Oncology, Journal of Hepatology, and other peer-reviewed publications. Dr. Hsu is fluent in English, Mandarin, and Taiwanese.

Dr. Hsu’s areas of expertise include: • Hepatocellular carcinoma• Cirrhosis• Alcoholic liver disease• Non-alcoholic fatty liver disease• Chronic liver disease• Hepatitis B and C• Autoimmune liver disease

She received her medical degree from Thomas Jefferson University and completed her internship and residency at Boston University Medical Center. Dr. Hsu completed fellowships in transplant hepatology and gastroenterology at Columbia University College of Physicians and Surgeons.

Soo Youn Yi, MD Dr. Soo Youn Yi comes to MedStar Georgetown Transplant Institute from the University of Maryland, where she performed pancreas and kidney transplants and was an assistant professor in the Department of Surgery, Transplant Division. She is also a former clinical surgery instructor at Columbia University’s College of Physicians and Surgeons.

Dr. Yi’s areas of clinical expertise and interest include: • Kidney, pancreas, and liver

transplantation• Single port laparoscopic donor

nephrectomy• Dialysis access surgery

Dr. Yi received her medical degree and completed her internship and residency at the Robert Wood Johnson Medical School in New Jersey. She then completed a fellowship in abdominal multi-organ transplant and hepatobiliary surgery at the Columbia University College of Physicians and Surgeons. She is board certified in general surgery.

Beje Thomas, MDKidney Transplant Nephrologist Dr. Beje Thomas comes to the Transplant Institute from the University of Maryland Medical System, where he was a faculty member.

Dr. Thomas has contributed chapters to several nephrology textbooks, and been published in the Annals of Surgery, American Journal of Nephrology, and other peer-reviewed publications.

His areas of clinical expertise and interest include:• Living donor kidney transplantation• Medically complicated transplant

candidates and recipients• Chronic kidney disease following heart,

lung, and liver transplant• Simultaneous kidney and pancreas

transplant

Dr. Thomas received his medical degree from University of Debrecen Medical and Health Sciences Centre in Hungary. He completed his internal medicine residency and general nephrology fellowship at the University of Connecticut Medical Center, followed by a transplant nephrology fellowship at the Medical University of South Carolina. He is board certified in nephrology.

Pejman Radkani, MDDr. Pejman Radkani specializes in the treatment of liver disease and lesions, bile duct disorders, and pancreas tumors. Previously, Dr. Radkani was assistant professor of surgery and a hepatopancreato-

biliary and abdominal multi-organ transplant surgeon at Loyola University Medical Center, Stritch School of Medicine in Chicago.

Dr. Radkani’s areas of clinical expertise include:• Liver cancer, including primary and

secondary liver tumors• Liver transplantation, including living

donor transplant• Bile duct disease and tumors• Benign and malignant pancreatic

tumors• Minimally invasive

hepatopancreatobiliary surgery, including laparoscopic and robotic liver and pancreas resection and surgery

His research interests include benign and malignant lesions of the liver, including intrahepatic cholangiocarcinoma and cholangitis, and neuroendocrine tumors

and adenocarcinoma of the pancreas.Dr. Radkani received his medical degree from Tabriz University of Medical Sciences, Iran and completed his residency in general surgery at Mount Sinai Medical Center, Miami. His fellowships include surgical oncology at Mount Sinai Medical Center, Miami; hepatopancreatobiliary surgery and abdominal multi-organ transplantation at Recanati/Miller Transplantation Institute at Icahn School of Medicine, Mount Sinai, New York; minimally invasive hepatopancreatobiliary surgery and living donor liver transplantation at Severance Hospital, Yonsei University Health System, South Korea; and minimally invasive hepatopancreatobiliary surgery at Institut Mutualiste Montsouris, France.



Sukanya Subramanian, MDDr. Sukanya Subramanian comes to MedStar Georgetown Transplant Institute from NewYork-Presbyterian/Columbia University Medical Center, where she was medical director of the adult intestinal transplantation program. Her areas of clinical expertise and interest include:• Management of patients with: – Malabsorptive disorders, including

short bowel syndrome and complicated inflammatory bowel disease (IBD)

– Intestinal failure and dependence on intravenous total parenteral nutrition (TPN)

– Intestinal insufficiency due to global motility disorders

– Complications from TPN• Preoperative and postoperative

evaluation and care of intestinal transplant candidates

• Functional bowel disorders associated with organ transplantation

• Clinical pharmacology (advanced applications in IBD and transplant medicine, novel therapeutics)

She earned her medical degree at Indiana University School of Medicine

and completed her internship and residency in internal medicine at University of Michigan Hospitals. Dr. Subramanian completed fellowships in clinical pharmacology and pharmacogenomics at University of Chicago Medicine. She is board certified in gastroenterology.

Gayle Vranic, MDAfter six years on the faculty of the University of Virginia School of Medicine, Dr. Gayle Vranic has joined MedStar Georgetown Transplant Institute, where she will continue to practice kidney transplant nephrology. Dr. Vranic is a member of the American Society of Nephrology and a reviewer for its journal, Clinical Journal of the American Society of Nephrology (CJASN). The recipient of an NIH research training grant, she has had her original research appear in Pediatric Transplant, American Journal of

Transplantation, and other peer- reviewed publications.

Her research interests include:• Health care disparities in renal

transplantation• Care of the highly sensitized renal

transplant recipient• Apheresis in solid organ transplant

Dr. Vranic received her medical degree from the University of North Carolina-Chapel Hill. She completed her residency and general nephrology fellowship at Vanderbilt University Medical Center and her transplant fellowship at the University of Virginia. She is board certified in nephrology. n

New Living Will App Opens New Avenues to Combat Organ ShortagesIn the search for new ways to encourage organ donation and combat an ongoing shortage of organs, MedStar Georgetown Transplant Institute has a digital solution—a new living will smartphone app. A living will gives individuals the chance to outline how they want to be cared for if they become incapacitated and unable to make decisions about their medical care. It can also serve as an opportunity to become an organ donor and combat the shortage of viable, transplantable organs. Becoming an organ donor can have a significant impact on the lives of people in need of transplants. One donor can save eight lives, restore

sight to two people, and heal up to 75 more through tissue donation.

Unfortunately, a study published in the American Journal of Preventive Medicine found that nearly two-thirds of Americans don’t have a living will. To make the process of creating a living will and becoming an organ donor as easy and convenient as possible, the Transplant Institute has partnered with Donate Life America to offer a free app, available for iOS and Android, that facilitates the process with just a few clicks.

Users can indicate whether they want to be an organ, eye, and/or tissue donor. A deceased organ donor can donate organs (liver, kidney, heart,

lung, intestines, and pancreas), corneas, and tissue. Donor registration is then added to the National Donate Life Registry.

“As two leading organizations addressing the issue of a shortage of viable organs, it was only natural for us to partner to offer a donor registration opportunity in this living will registry app,” says Thomas Fishbein, MD, executive director, MedStar Georgetown Transplant Institute.

For more information, visit MedStarGeorgetown.org/LivingWill or download the app from the App Store or Google Play; search MedStar Living Will. n


CASE STUDY:

Pancreas Transplantation for Diabetes Mellitus at MedStar Georgetown Transplant InstituteOptimizing Access to Transplant, Minimizing Time on Waitlist

ABSTRACT:

A 29-year-old woman with diabetes mellitus (DM) transferred care from a Baltimore transplant center to the Transplant Institute after waiting three years for a simultaneous pancreas-kidney transplant (SPK). She received her SPK in less than three months after being listed at the Transplant Institute. Her transplant function was excellent from day one. She was discharged home in six days, diabetes-free, dialysis-free, to enjoy a normal, active life.

Patient Presentation:• 29-year-old female with DM since

age of 12, end-stage renal disease (ESRD) on hemodialysis. Other diabetic history includes retinopathy and episodes of symptomatic hypoglycemic unawareness.

• Already on waitlist for SPK at Baltimore transplant center for almost three years

• Multiple hospitalizations every year for diabetes-related medical issues

Clinical and Operative Course:• The Transplant Institute received

high-quality SPK organ offer from Midwestern region

• The Transplant Institute transplant team traveled to donor hospital to assess and procure the transplant organs

• Less than three months after transferring waitlist time to the Transplant Institute, patient underwent successful SPK transplant

• Uneventful post-operative course with complete functional recovery

• Discharged home six days after transplant

• Patient diabetes-free with no need to check daily glucose levels

Comments:Lessons learned over previous decades of pancreas transplantation have led to excellent outcomes for well-selected diabetics undergoing SPK currently. The one-year patient survival rate of pancreas-kidney transplant meets or exceeds that of any other type of solid organ transplant.

Long-standing DM profoundly impacts the overall health and life expectancy of patients suffering from this intractable disease. Despite significant advancements in medical therapy, DM stubbornly remains associated with mortality rates up to 18 times higher than the non-diabetic population in the U.S. Given the spectrum of potential diabetic complications, there is often not a single organ system unaffected. When any patient reaches this end-stage of severity in terms of DM secondary complications, life becomes a tedious exercise dominated by clinic appointments, emergency department visits, and hospitalizations, at enormous cost to the patient and family both financially and in terms of quality of life.

Improvements in diabetic education; insulin analogs; and technology, such as insulin pumps and continuous glucose monitoring

(CGM) systems; have successfully reduced the incidence, severity, and duration of hypoglycemic events in hard-to-control diabetic patients. However, as in this case, there remains a 25 to 30 percent rate of symptomatic hypoglycemia despite having access to all the latest medical interventions and following the latest guidelines from the American Diabetes Association (ADA). The quality of life impact of restoration of normal glucose metabolism by pancreas transplant cannot be overstated. The risk of development and progression of secondary complications of DM is not eliminated by any established regimen of intensive insulin therapy. Beta-cell replacement is the only treatment that achieves normal body glucose regulation in insulin-dependent patients. Thanks to improvements in surgical technique, immunosuppression, donor and recipient selection, and allograft surveillance, the durability of all types of pancreas transplants continues to improve. With greater reflection on the significant failure rates of best practice medical therapy and an appreciation of the “real life” complications of DM, pancreas transplantation deserves wider recognition as a viable option available to this precarious patient population. n



With New Transplant Link, Talk to MedStar Georgetown Transplant Experts 24/7It’s 4 p.m. on a Sunday and your patient arrives in the emergency department with symptoms of acute liver failure. You have questions about whether she meets the criteria for transplantation and how you can get your patient on the transplant waitlist. Now you can get those answers immediately through MedStar Georgetown Transplant Institute’s new Transplant Link.

Transplant Link provides referring physicians live access to our liver, kidney, pancreas, HPB, and intestinal transplant medicine specialists 24 hours a day, seven days a week via voice or video call. Our experts are

here to answer any questions you have—from how to get a patient on the transplant waitlist; to what support services are available to guide patients through the transplant process; to medical and surgical treatment options for patients with liver, kidney, pancreas, HPB, and/or small bowel disease.

To reach one of our physicians or care navigators, go to MedStarHealth.org/Access on your computer or smartphone and click either Liver, Kidney, Pancreas, HPB, or Small Bowel to be connected and get the answers to your questions. You can also visit MedStarHealth.org/LinkApp to download the free iPhone app.

“We understand that the field of transplantation is complex. Our goal is to make it faster and easier to reach one of our experts and get the answers you need,” says Thomas Fishbein, MD, executive director of MedStar Georgetown Transplant Institute. n

Visit MedStarHealth.org/Access via smartphone or computer to reach a MedStar Georgetown Transplant Institute expert 24/7.

Earlier Referral and Living Donor Options Can Expand Kidney Transplantation and Improve OutcomesWith more than 95,000 people on the waiting list for a new kidney in the U.S. and fewer than 18,000 receiving a donor organ in 2017 according to the United Network for Organ Sharing (UNOS), it is critical that the option of living donation be considered earlier. Living donor kidney transplantation offers patients several important benefits, including:• Bettershort-andlong-term

outcomes compared to deceased donor transplants. Organs from living donors last 15 to 20 years on average, while a deceased donor kidney has an average lifespan of 10 to 15 years.

• Decreased time on the transplant waiting list compared to average waiting times that can range as high as five to ten years for a deceased donor organ

• Lowered risk of post-surgery complications and rejection

• Better early function of the transplanted kidney compared to the timing for deceased donor kidneys to reach full function

• Surgery can be scheduled electively, when most convenient for the donor and recipient

Multiple Living Donor Options There are several living kidney donor options, which means that even if a patient cannot find a donor among friends and family with compatible blood or tissue types, living donor transplantation can still be possible. Options include:• ABO- or HLA- Compatible

Kidney Transplantation: the donor and recipient have the same or compatible blood type, and a compatible immunological profile

• ABO- or HLA- Incompatible Kidney Transplant: The donor and recipient have incompatible blood types or the recipient has antibodies against the donor. In these cases, the recipient often can be treated prior to and following transplantation with plasmapheresis, effectively filtering out antibodies in the recipient that would predispose to rejection of the transplanted organ.

• Paired Kidney Exchange: a program that matches multiple donor and recipient pairs through the “exchange” of kidneys between unrelated pairs

The Importance of Early ReferralPatients who undergo kidney transplantation before starting dialysis have several advantages over those who undergo transplantation after being on dialysis for an extended time period. “We strongly encourage referral for transplant evaluation before patients with evolving kidney disease begin dialysis,” explains Matthew Cooper, MD, director of kidney and pancreas transplantation at the MedStar Georgetown Transplant Institute. “Not only do patients who undergo transplant before dialysis have better outcomes, they also have a much better quality of life when they can avoid the arduous process of dialysis.”

To refer a patient for evaluation for a kidney transplant, please call 202-444-3700. n

Pediatric Liver Specialists Improve Health and Quality of Life for Children With Metabolic DiseasesFor children living with metabolic diseases and their parents, life can be extremely challenging. Not only does their disease increase their risk for a host of serious, often life-threatening complications, but navigating the metabolic treatment can be exceptionally complicated. Depending on the specific disease process, a very restrictive diet can be required, which in turn can negatively affect their growth. Breakthrough metabolic attacks are always “a viral illness away” and threaten to cause severe morbidities and even death. Getting patients to comply with draconian diets and tedious, frequent, physician’s appointments becomes an issue when patients reach adolescence. However, not complying with diet and follow-up care requirements can lead to suboptimal neurodevelopmental and general health outcomes.

The liver specialists at MedStar Georgetown Transplant Institute and their partners at the Children’s National Health System Rare Disease Institute, led by Marshall Summar, MD, chief of Genetics and Metabolism at Children’s National, provide children with genetic metabolic disorders another treatment option: liver transplantation. Home to one of only four integrated metabolic disease management and pediatric liver transplant programs in the nation, the team provides medical and surgical management of a wide range of pediatric metabolic conditions, including:

• Alpha-I Antitrypsin Deficiency (AATD)

• Crigler-Najjar Syndrome I• Cystic fibrosis (CF)

• Familial intrahepatic cholestatic disorders

• Glycogen storage disorders• Maple Syrup Urine Disease

(MSUD)• Propionic acidemia• Neonatal hemochromatosis• Oxalosis• Tyrosinemia• Urea cycle disorders• Wilson’s disease• Familial hypercholesterolemia

Many children with these conditions may benefit from a liver transplant, which can stop the progression of the systemic disease and lessen (or in some cases, eliminate) the need for restrictive diets and frequent diagnostic testing. That’s where MedStar Georgetown Transplant Institute’s pediatric liver transplant specialists come into play.

“We have expertise performing transplants on the smallest, youngest patients, including those who are less than a year old,” explains Nada

Yazigi, MD, a pediatric hepatologist certified in transplant medicine. “This is a special population of patients whose needs are addressed currently by only a handful of integrated pediatric metabolic transplant centers in the U.S. Indeed, in experienced multidisciplinary hands, a liver transplant can stabilize a metabolic patient’s health by affecting physiologic levels of the defective enzyme activity throughout the body or by fully correcting the deficit. This in turn reduces or eliminates systemic and neurologic complications.” Metabolic liver transplantation also lessens the patient’s reliance on special diets and provides a better quality of life and long-term outcome. This focus has been a major change in the treatment paradigm in the Transplant Institute’s program. In fact, 45 percent of the pediatric liver transplants we performed in the last three years have been on children with metabolic disorders, with over half of those patients being under one year of age.

The Transplant Institute and Children’s National physicians are also taking the lead in clinical, basic, and translational research focused on metabolic disease in children. The goal is to eventually find targeted cures for metabolic disorders that will preclude transplantation. “Our program and unique partnership with the Rare Disease Institute provide us with the expertise and resources to work towards uncovering the causes of metabolic disease and developing targeted treatments that will transform the way we care for these patients,” adds Dr. Yazigi. n

Nada Yazigi, MD


By the Numbers: MedStar Georgetown’s Transplant ProgramsLiver Transplant Program• Within the top 15 by volume of liver transplants for patients of all ages

in the U.S.

• Lower median time to transplant than all other programs in the region (8.2 months compared to 14 months)

• Superior 1-year adult graft survival compared to University of Maryland Medical Center and the Johns Hopkins Hospital

• Highest adult 1-year patient survival in the Baltimore and Washington, D.C. area

• In 2017, transplanted a higher proportion of the highest severity (Status 1) patients and performed a higher proportion of the most complex (split liver transplants) than any other program in the country

• More pre- and post-transplant experience treating minority patients than other area programs

Pediatric Liver Transplant Program• Within top 5 by volume in the country

• Graft survival better than expected and better than U.S. average

Kidney Transplant Program• Top 10 percent center by volume out of 238 kidney transplant centers

in the U.S.

• Higher than expected transplant rate and shorter median time to transplant than other centers in the region

• Fewer median days in inactive status than other centers in the region and in the country

• Lower than expected mortality rate for patients on the waitlist

• More pre- and post-transplant experience treating minority patients than other area programs

Other Highlights• Largest program by volume for intestinal transplants in the U.S.

• Within the top 5 by volume for pancreas transplants in the U.S.

• Within the top 15 by volume for all abdominal transplants in the U.S.

Source: SRTR.org, http://optn.transplant.hrsa.gov, January, 2018.



A Tale of Two TransplantsKorey Scott was born with Maple Syrup Urine Disease (MSUD)— a rare metabolic disorder that can cause coma, seizures, brain damage, and even death. The only cure is a liver transplant. Isobel Short would die if she didn’t soon get a new liver to replace her cirrhotic one. Both got what they needed on the same day at MedStar Georgetown Transplant Institute.

A pioneer in transplantation as a precautionary treatment for children with MSUD, the Transplant Institute is also at the forefront of transplanting MSUD livers into patients who desperately need them. In effect, the liver that contributes to life-threatening conditions in one person can sometimes be used to save the life of another. That’s exactly what happened with Korey and Isobel.

In a coordinated, domino procedure, Korey received a deceased donor liver, then his liver was transplanted into Isobel. After the operations, both patients flourished.

“Thanks to this beautiful little boy, I have a second chance at life.”

-Isobel Short

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TRANSPLANTdigest A PUBLICATION OF THE MEDSTAR GEORGETOWN TRANSPLANT INSTITUTE

Transplant Digest is a bi-annual publication featuring news of interest to physicians about the MedStar Georgetown Transplant Institute.

Please submit comments or questions to Daphne Torneyat 202-444-6815,or by [email protected].

MICHAEL C. SACHTLEBENPresident, MedStar Georgetown University HospitalSenior Vice President,MedStar Health

JUDSON STARRChairman of the BoardMedStar Georgetown University Hospital

KENNETH A. SAMET, FACHEPresident and CEO, MedStar Health

THOMAS M. FISHBEIN, MDExecutive Director

WriterSusan Walker

DesignerLaura Sobelman

TO FIND A TRANSPLANT SPECIALIST, CALL 202-444-3700. n

Turn to page 9 to learn about our innovative approach to connecting referring physicians with our team of transplant experts. The Transplant Link app gives you live access to our liver, kidney, pancreas, HBP, and/or intestinal transplant medicine specialists 24 hours a day, seven days a week via voice or video call.

With Our New Transplant Link, Talk to MedStar Georgetown Transplant Experts 24/7

transplant digest - medstar health · 2019-01-21 · fall 2018 transplant digest a bi-annual...

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