Transplanting Hope, Transforming Lives 2012-2016

Transplanting Hope,Transforming Lives2012-2016

inside When Clyde Horton (right) needed a kidney in 2012, his good friend and former neighbor Joe Wolken was one of many whoFrom the Desk 1 volunteered to be his living donor. “I was thrilled to find outof Thomas Fishbein, MD 2 that Joe was the best match for me,” the 66-year-old says. 6 “Previously, I thought I had to have an African-AmericanKidney and Pancreas 10 donor.” Thanks to advances in apheresis and otherTransplant Program 14 desensitizing technologies, that is no longer the 18 case. At the MedStar Georgetown TransplantCenter for Liver Disease 21 Institute, nearly 36 percent of living donorand Transplant 22 recipients in 2015 were African American, 26 more than three times the national average.*Center for Liver 28and Pancreas Surgery Center for Intestinal Careand Transplant Transplant Centerfor Children Facilities andAmenities Research Publications Faculty *Scientific Registry of Transplant Recipients, June 2016

from the desk of...Dear Colleague,I am honored to present the MedStar Georgetown Transplant Institute progress report, summarizing some of our work overthe last few years. It tells the story of the Institute through the caliber of our faculty and outcomes, the breadth and depthof our programs and, especially, the lives of the patients and families we touch.In 2014, MGTI moved into 20,000 square feet of new outpatient space at its MedStar Georgetown headquarters. For the firsttime, physician offices, waiting rooms, clinics, and a 17-room outpatient facility second to none are housed together in oneintegrated space, enhancing communication and collaboration among colleagues. As an integral component of the Institute,MedStar Washington Hospital Center underwent a similar transformation.Beyond our walls, the Institute has grown into a regional network of three hospitals and three outpatient clinics stretching fromour metropolitan D.C. hub north to Frederick, east to Baltimore and Annapolis, and down into Northern Virginia. Now patientscan be seen and evaluated for treatment, and many times receive it, closer to where they live. The move also allows us toaccommodate patients more quickly, often within 24 hours if necessary. Today, only complex surgical care and early follow-upmust be delivered in the hospital setting.Over the last few years, we devised innovative techniques pioneering domino and even three-way split liver transplantsto maximize the use of donated organs. We also established an islet cell transplant program, one of the few in the UnitedStates today.These cumulative efforts helped establish MGTI as the 22nd-highest volume center within the nation for all organs for 2016;when thoracic transplant is excluded, we rise to number 11.* During 2015, our pediatric liver transplant program performed32 procedures, a volume matched by only one other program nationwide; our one-year pediatric liver patient survival was100 percent. And the intestinal transplant program is a nationally recognized leader in terms of both volume and outcomes.Underlying our clinical advances is a greatly expanded research portfolio, embodied by the new Center for TranslationalTransplant Medicine (CTTM). A joint effort with the Georgetown University School of Medicine, CTTM brings together diseasegroups and specialties, laboratory scientists and transplant physicians, academicians and practitioners to promote the cross-fertilization of ideas for new and better clinical protocols, treatment algorithms and, ultimately, patient care.As you review the pages that follow, I hope you see evidence of progress everywhere—from MGTI’s own institutional growthto its contributions to the broader fields of immunology, oncology, liver diseases, nutrition and transplantation.As always, I invite you to contact me for further information.Sincerely,Thomas M. Fishbein, MD *Organ Procurement and Transplant NetworkExecutive Director, MedStar Georgetown Transplant Institute

kidney and pancreas transplant prograAmong the top 10 percent of the highest volume centers Expanding Paired Kidney Exchangesin the nation, MGTI's kidney transplant program has more than 40 years of experience and • Area-wide incompatible pairs program.5,000 transplants on record. A collaboration between MedStar Washington Hospital Center In 2011, MGTI—site of 2010’s record-breakingand MedStar Georgetown University Hospital, along with Children's National Medical Center, paired kidney exchange, the largest in theit is the most established and busiest kidney transplant center in the Washington, D.C., area. world at the time—started the metropolitan area’s first coordinated program forThe June 2016 report from the Scientific Registry of Transplant Recipients also shows incompatible donor pairs. Today it actsthat MGTI’s deceased donor kidney transplant rate exceeds the national average. Among as the area’s incompatibles repository,transplant centers in the Washington/Baltimore region, MGTI’s one-year patient survival rates coordinating paired exchanges amongfor the period are the area's best. Such accomplishments are directly related to the program’s transplant centers to expand the pool ofcontinuing quest to help more patients get the transplants they need, more quickly, through a locally available organs.variety of innovative approaches. • P articipation in multiple national PairedPromoting Living Donation kidney transplant across the board, a trend Kidney Exchange (PKE) programs. expected to grow as MGTI invests additional MGTI is the only area program that activelyMGTI has performed more than 80 percent of resources in the LD program. participates in regional and national pairedthe more than 2,000 living donor (LD) kidney kidney exchange programs. As a result,transplants in the Washington, D.C., Desensitization Protocols for it has transplanted many highly sensitizedarea on record, making it a regional and Incompatible Pairs patients, including some previouslynational leader. classified as non-transplantable, with patient While many living donor/recipient pairs are and graft survival outcomes that are amongFurther cementing this position is an extensive viable matches, one out of three is not. For them, the best in the country.physician outreach and community education MGTI is one of the few programs in the nationprogram, along with other efforts to raise that offers desensitization protocols for living • Volunteer PKE program for compatibleliving donor awareness and promote paired donor/recipient pairs with ABO blood group or matched pairs. In 2014 MGTI startedkidney exchanges. As a result, the number HLA incompatibilities. Through careful patient approaching otherwise healthy, qualified,of LD transplants doubled over the last four selection and team experience, the program compatible living donor pairs about joiningyears, easing local demand for deceased donor produces outcomes among this population that the paired kidney exchange. To date, sevenkidneys. The overall effect boosts access to exceed local and national averages. pairs have volunteered, multiplying each donor’s impact.2

ogramAdvancing the Field One-Year Patient Survival Among Regional Hospitals*In the face of an escalating local and national demand for kidneys and %a stagnant supply, the program’s research is devoted to developing 99new, better and safer techniques and approaches to improveorgan utilization, reduce graft failure and carefully manage a scarce 98.22resource. 98• Improving patient outcomes following deceased, expanded criteria donor transplant. Nationwide, between 25 and 30 97 96.7 percent of transplants via kidneys from older donors or from 96.17 donors after cardiac death result in delayed graft function (DGF). Consequently, the national discard rate for expanded criteria 96 donor organs is currently about 55 percent. MGTI’s researchers are working on reproducible and successful interventions for DGF 95 so that more expanded criteria kidneys will be considered for transplant, increasing organ availability. 93 University Johns Hopkins MedStar Georgetown of Maryland University• Reducing delayed graft function, rejection and organ failure. Transplant Institute Other current clinical and basic research activities aim to prolong graft function and survival by finding: * Performing more than 100 kidney transplants annually. Source: SRTR Center Specific Reports, June 2016 o New treatments for antibody-mediated graft rejection Observed and Expected Deceased Donor o Strategies to minimize post-transplant CMV infections Transplant Rates 01/01/2015-12/31/2015* o Techniques to reduce post-transplant wound infections o Novel immunosuppressive strategies to avoid organ injury and MedStar 15.4 Georgetown increase graft survival times 12.0 Observed o Increased opportunities for transplant for patients with Transplant Expected Institute 12.7 Hepatitis C and HIV OPO/DSA 11.9 20 o A non-invasive method to detect early DGF 3 Region 11.5 11.6 U.S. 11.5 11.5 05 10 15 Rates per 100 Person Years * SRTR, June 2016

Case StudyKidney Transplant for the Non-TransplantableA 45-year-old mother of five, on dialysis for With only a small window of opportunity, MGTI’s Survival Benefit With Kidneymore than five years, was referred to MedStar team desensitized the patient and proceeded to Transplants from HLA-Georgetown Transplant Institute for a kidney transplant. Two weeks later, her immune system Incompatible Live Donorstransplant evaluation in 2014. The initial work- mounted a robust response, producing a multi-up revealed multiple pre-formed anti-HLA fold increase of the previously known anti-HLA MGTI was one of 22 centers participatingantibodies, leaving her highly sensitized and antibodies along with four new donor-specific in a large, NIDDK-funded research studygreatly limiting her chances of finding an organ anti-HLA antibodies. comparing the outcomes of three groups:her body wouldn’t reject. A combination of plasmapheresis and immune patients who received a kidney from anWhen a potentially acceptable, albeit ABO- modulatory therapies greatly reduced the HLA-incompatible live donor, patients whoincompatible, deceased donor kidney became patient’s level of already-identified antibodies waited for an acceptable deceased organ foravailable, further analysis unearthed an even within days. By week three, the production transplant and patients who remained on themore challenging situation: a positive T- and of new antibodies had practically ceased. A waiting list.B-cell flow cytometric crossmatch with donor- little less than two months after transplant, thespecific anti-HLA antibodies against HLA class patient was totally antibody-free with a normal As reported in the March 10, 2016 issue of the1 and class 2 antigens. transplant kidney biopsy and function. New England Journal of Medicine, the findings were significant. Across the board, the 1,025 Kidney Transplant Program Highlights HLA-incompatible live donor recipients fared better than either of the two control groups • Among the nation's top 10% transplant programs by volume at the 1-, 3-, 5- and 8-year survival markers. Dr. Matthew Cooper, director of MGTI’s kidney • Welcomed Matthew Cooper, MD, as Director, Kidney and Pancreas Transplantation and pancreas transplant program, was a contributing author. • Transplanted 205 kidneys in 2016, the highest annual volume in the program’s history and more than all other programs in the metropolitan D.C. area combined • Increased living donation rates by 35% • Launched the 1st repository for incompatible pairs in the D.C. area • Conducted 18 clinical trials • Achieved a higher transplant rate than the national average and the region’s highest outcomes.4

Pancreas Transplant Diabetes nearly killed Jeffrey Edwards.Over the last three fiscal years, MGTI’stransplant surgeons performed 50 In 1999, Jeffrey fell into a life-pancreas transplants. Our transplant threatening, two-week-long diabeticrate in pancreas is more than double what coma. His disease led to congestivewas expected for our center in the June heart failure and heart surgery, kidney2016 SRTR report, with excellent outcomes. failure and dialysis.At the same time, the total number of “My life was so horrible, knowing I hadpatients on our pancreas transplant waiting to go through dialysis three times alist also doubled. Based on such volume week to survive,” says the 53-year-old.and outcomes, MGTI is on track to become “Insulin wasn’t controlling my diabetes.a national leader in pancreas transplant. I never knew what complication would happen next.”Much of that growth is due to the pancreas But in February 2016, a kidney/program’s ongoing work to cure diabetes pancreas transplant at MGTI broughtin patients suffering from life-threatening Jeffrey’s 17-year battle to an end.complications. For a subset of patients “I’m no longer doing finger-sticks towith type 2 disease (i.e., those who check my blood sugar,” he says. “I canrequire significant amounts of insulin for eat what I want, when I want andcoverage and have a BMI <30), pancreas I feel great. It’s as though I nevertransplantation offers an improved quality had diabetes.”of life and a reduction in complicationsfrom long-standing diabetes. 5MGTI has also expanded its pancreas-after-kidney (PAK) transplant program.This two-phased strategy encouragespatients to undergo a living donor kidneytransplant when presented with theopportunity. Subsequently, the patient willhave a deceased donor pancreastransplant to reduce the complicationsof diabetes and prevent diabetic injuryto the transplanted kidney.For more information about the MedStar GeorgetownTransplant Institute, visit MedStarGeorgetown.org/Transplant or call 800-442-4200.

center for liver disease and transplantHome to the area’s largest team of hepatologists, MedStarGeorgetown Transplant Institute is the only academic hepatology program and the only fullyintegrated hepatobiliary program in the Washington metropolitan region.With a full multidisciplinary team of specialists and sub-specialists—and a coordinated, Community Department ofcollaborative, collegial approach—MGTI assures the fastest and most accurate diagnosis and Screening Veterans Affairstreatment for advanced and complicated liver disease. Its specialists are equally adept and Medical Centerexperienced with pediatric and adult patients for the highest level of medical, surgical and Clinictransplant care. Community MGTI American Physicians LiverMedically Managing Liver Disease Reducing Transplants and Deaths Foundation From HCVMGTI’s liver disease program has a twofold Unitymission: Hepatitis C virus is the single leading reason Health Care for liver transplant and the cause of more• to circumvent liver transplant through the deaths in the United States today than HIV. MGTI collaborates with multiple stakeholders to identify expert medical management of patients Sofosbuvir and simeprevir were the first drugs those most at risk for HCV infection and reduce the with viral hepatitis, liver tumors, genetic ever with the ability to cure patients infected burden of liver disease throughout the area. Most disorders and other complex conditions with the virus, which can lead to cirrhosis or recently, it opened a community hepatitis screening hepatocellular carcinoma. A whole new arsenal clinic in Fairfax, Va., funded by a grateful patient. and of direct-acting, interferon-free, HCV antiviral agents, including pan-genotypic drugs, have MGTI’s early adoption of the new therapies,• to optimize patient and graft outcomes emerged in the interim, with more in the combined with the liver program’s reputation for those destined for transplant, through pipeline. and outreach, produced an unprecedented meticulous pre- and post-transplant care. three-fold growth in referrals in just 15 months. These new therapies can stop the end-stage In the last two years alone, MGTI treated moreThe advent of the first Hepatitis C antiviral liver disease caused by HCV, leading to than 360 HCV-infected pre-transplant or otheragents in 2013 was a game-changer for the improved health, diminished demand for hepatology patients, and 120 infected patientsfield, affecting both aspects of the program. transplant and reduced mortality. Administered after transplant, curing the majority. post-transplant, the drugs can even improve both graft and patient survival in patients still infected with the virus.6

lant On her 1st birthday, little Natalia Walker Advancing the Field received the ultimate gift— MGTI’s specialists are actively conducting a second chance at life—from a virtual basic and clinical studies to improve stranger, Diana Rotter. The 30-year-old treatments for those with liver disease, now had learned that the baby wouldn’t and in the future. Current research involves: survive without a new liver and selflessly volunteered to contribute • Treating early stage and advanced part of hers. hepatocelluar carcinoma with Y90 and complex resection techniques MGTI physicians successfully removed 20 percent of Diana’s liver • Using adjuvant therapy to reduce and transplanted it into Natalia on the risk of hepatocellular carcinoma November 17, 2015. recurrence after liver transplantation “Within the first two weeks, Natalia • Supporting patients with advanced liver was a completely different baby— disease with a bioartificial liver she came back to life,” says Diana Walker, Natalia’s mother. “We are so • Treating acute liver failure due to grateful there are heroes out there.” amatoxin The story was featured on the November • Advancing HCV treatment through: 23, 2015 broadcast of the “Today” show. o New, all-oral therapies o Shortening the duration of HCV 7 treatment in liver transplant patients o Antifibrotic therapies • Understanding Hepatitis E in organ transplant recipients

Liver Transplant Transplant Volume*Thanks to an aggressive and innovative Number of Transplant Centers 20 MGTI *The dashed blue linesapproach to care, MGTI has the region’s shortest 15 25% represent the 25th, 50thtime-to-transplant for waitlisted patients and is and 75th percentiles oftwice as fast as the national average. 10 50% liver transplant volume performed from November 1,The program has typically performed around 5 75% 2015, to October 31, 2016,100 or more adult and pediatric transplants by 140 transplant centers.annually since FY12. As such, it falls within the MGTI’s transplant volume fortop 25 percent of active liver transplant centers the period is indicated by thebased upon volume. In 2015, it shared the top red line.spot for the most pediatric livers transplanted inthe United States (OPTN). 0 50 100 150 Source: UNOS Benchmark Report 0 Number of Transplants 200A pioneer in advanced liver transplanttechniques, MGTI performed more than 50 World’s First Combined Split Liver and Domino Transplantsplit liver procedures for the reporting period.According to the January 2017 SRTR report, Deceased Donor Livernearly 17 percent of all MGTI transplantsinvolved partial/split livers, compared to anational average of slightly more than 3 percent.It also led the way in developing the domino livertransplant approach for metabolic liver disease,with exceptional results. Patient 1: Patient 2: Adult Recipient Baby With MSUD Patient 3: Baby With Biliary Atresia In October 2015, MGTI became one of the first to perform a combined split liver and domino transplant, effectively saving three lives with two organs. A 36-year-old received the lion’s share of the deceased donor liver, while the smaller portion was reserved for a 1-year-old with MSUD. That infant’s liver was then transplanted into another baby born with biliary atresia. The combined8 procedures took a team of more than 20 people 12 hours to complete.

Liver Program From UK to US:highlights Baby Sent Stateside for• Performed 32 pediatric liver Life-Saving Liver Transplant transplants in 2015, the highest Six-month-old Ashlynn Beutler was living number in the U.S. in England with her family when she was diagnosed with biliary atresia, a rare congenital• An average of 100 pediatric and defect of the liver ducts. Doctors there adult liver transplants each year performed a Kasai procedure, hoping to delay disease progression, buy time for the baby, and• Expanded eligibility forestall the inevitable—a liver transplant, the thresholds to accept high-risk only definitive cure. patients turned down by other It didn’t work. centers, including those with: In short order, Ashlynn and her mother and o HIV positivity in appropriate father, a physician in the U.S. military, were candidates shipped stateside to MedStar Georgetown Transplant Institute. o Hepatocellular carcinoma within and outside of Milan criteria Ashlynn was immediately put on the national organ donor waiting list. But she continued to o Hilar cholangiocarcinoma under get sicker by the day. a special protocol “You can’t believe it’s happening,” says her• Pioneered split liver, 3-way mother Arielle. “All of a sudden, your beautiful splits, domino transplants and baby needs a new liver before time runs out.” other advanced techniques Tests showed that Arielle was a match for Ashlynn. So in April 2013, she donated a portionFor more information about the MedStar Georgetown of her liver to save her then-9-month-old baby.Transplant Institute, visit MedStarGeorgetown.org/Transplant or call 800-442-4200. Today Ashlynn is a happy, healthy preschooler who “likes dressing up and thinks unicorns are the best thing ever,” Arielle says. “Being treated at MedStar Georgetown was a blessing. Nobody looking at Ashlynn today would ever know she had been so sick.” 9

center for liver and pancreas surgeryMGTI’s depth of experience and expertise in complex liver Area’s First Islet Cell Transplant Programand pancreas transplantation translates into superb surgical care for other advanced orcomplicated hepatobiliary conditions as well. In addition to surgeons and hepatologists, With a newly designed, newly designatedthe team features gastroenterologists, medical oncologists, radiation oncologists, FDA-compliant clean lab in place—and aninterventional radiologists and other specialists as needed, making it the area’s only equal investment in highly experienced lab andfully integrated hepatobiliary program. Combined with its focus on tumors and other transplant specialists—MGTI became one of theabnormalities of the liver, bile duct and pancreas, MGTI’s hepatobiliary program has the very few institutions in the nation to offer isletflexibility, resources and talent to attack any stage disease from any angle. cell transplant for chronic pancreatitis in 2015. To date, the team has performed four of theState-of-the-Art Therapies for Every is compared against known markers for highly specialized procedures, including two inCondition pancreatic cancer—produces the most accurate adolescents. By comparison, the total of all islet diagnoses available, contributing to fewer cell transplant cases in the United States eachFor benign and malignant liver masses, unnecessary surgeries for benign disease. year is only slightly over 100.the program offers a full range of openand minimally invasive surgical techniques, However, when surgery is the answer, Autologous islet cell transplantation is anincluding laparoscopic ablation which may, in MGTI is among the most experienced effective therapy for a specific subset ofsome instances, obviate the need for surgery in the nation with the highly complex patients with irreversible chronic pancreatitis:altogether. It was also the first to use the pancreaticoduodenectomies (Whipple those whose condition is unrelated to a blockedda Vinci robotic surgical system for liver procedures) for the most successful outcomes. gland. Ideal candidates have refractory chronicresection in the Washington, D.C., area. pancreatitis, preserved endogenous insulin Other surgical and non-surgical production and chronic pain. The procedurePatients with pancreatic and bile duct disease techniques, technologies and procedures involves a total pancreatectomy, followedalso benefit from MGTI’s state-of-the-art found in the hepatobiliary program by extraction and purification of islet cells.capabilities, high volume and expertise. include ERCP (endoscopic retrograde Retrieved cells are then infused back into theThe program’s use of molecular profiling—in cholangiopancreatography), chemotherapy, patient’s liver through the portal vein, wherewhich the genetic coding in biopsied tissues ablation, portal vein embolization and more. they engraft and ideally begin to produce insulin again on their own. The benefits of autologous islet cell transplant are multiple. With the malfunctioning pancreas gone, pain vanishes or is significantly10

ry reduced for more than 90 percent of all patients post-procedure. Autologous transplant also eliminates the need for lifelong immunosuppressive medication. Furthermore, one-third of all patients will neither develop diabetes nor require insulin after the procedure, although some may need to regulate blood sugar. For those who do require insulin, their diabetes will be less severe than it would have been without the islet cell transplant. And among those at risk for brittle diabetes, the newly functioning islet cells reduce the possibility of developing the unstable disease and its complications. Because the overall incidence of chronic pancreatitis is relatively low, the number of candidates for islet cell transplant is even lower. As a result, few medical institutions commit to establishing and maintaining the highly specialized programs. Centers treating both adult and pediatric patients are even harder to find, making MGTI’s new islet cell transplant program a life-transforming service for patients, and a major contribution to the system’s established pancreatic disease and disorders portfolio. In MedStar Georgetown Transplant Institute’s designated FDA-compliant clean lab, islet cells are prepared for infusion. 11

First Islet Cell Transplant Patient Back in the Saddle At least once a week, chronic pancreatitis would land Seana Frost-Barnes in the Emergency Room; at least once a month, she’d be admitted. It was the story of the 30-year-old equestrian’s life as her symptoms—severe pain, nausea and vomiting—escalated, reflecting the steady decline of her pancreas. Then MedStar Georgetown Transplant Institute launched the first and only islet cell transplantation center in the region, and Seana and her husband Steve had their first glimpse of hope. “I was sick of being sick,” says Seana. “On my worst days, even the thought of leaving the house to buy a loaf of bread was unbearable. I wasn’t happy and I wasn’t healthy.” So in May 2015—after a terrible attack kept Seana hospitalized for two weeks and in fear for her life—Seana’s diseased pancreas was removed. The islet cells were extracted and then infused back into her liver. Three months later, she was insulin-free, doing well and looking forward to getting back on her beloved horse once again. “I can’t believe I lived like I did for so long,” Seana says today. “I feel amazing—and only wish I had had the transplant sooner!”12

Better Outcomes Through Research Patient With PancreatitisChronic pancreatitis is uncommon and difficultto diagnose, often eluding detection until its Isolatedadvanced stages. By then, unresolved disease Nativemay have consumed much of the organ’s Islet ofinsulin-producing islet cells, leaving some Langerhanspatients with few to isolate and transplant. IsletThat scenario reduces the number of patients Isolationwho could benefit from the procedure,prompting the physician/scientists at MGTI to Remove Pancreassearch for novel solutions on multiple fronts. Pancreas• E ffect of Liver Histopathology on Islet Engraftment Post-Autologous The Auto Islet Cell Transplant Procedure Transplant. With support from the National Pancreas Foundation, MGTI After surgical removal of the whole pancreas, the pancreas is taken to a highly specialized lab where the islet cells are extracted from the diseased organ. studied how pre-existing degrees of fat and inflammation in the liver may affect the These cells are then delivered to the liver by re-injecting through the portal vein. organ’s reaction to engraftment, possibly leading to new ways to optimize the liver’s A similar MGTI-sponsored study substitutes parts, stimulated to grow and then acceptance of transplanted islet cells. human placenta-derived amniotic epithelial reassembled to create a pseudo islet cell. cells for MSC. To date, MGTI’s researchers have proven• A pplication of Human Bone Marrow- that the pseudo cells produce insulin in vitro Derived Mesenchymal Stem Cells to • Pseudo Islet Generation from Human and are now testing how the pseudo cells Human Islet Transplantation. In this U.S. Islet Cells. In this joint study with the perform in an animal model. Department of Defense-funded study, biotechnology firm Propagenix, human islet collaborators from the MGTI islet cell cells are broken down into their component transplant lab and MedStar Georgetown University Hospital’s Blood and Marrow Collection Program—the world’s largest— are examining the effect of transplanting mesenchymal stem cells (MSC) and islet cells simultaneously to improve islet cell engraftment, promote immunomodulation and convert MSC into insulin-producing cells.For more information about the MedStar Georgetown Transplant Institute, visit MedStarGeorgetown.org/Transplant or call 800-442-4200. 13

This is the center spreadcenter for intestinal care and transplantNationally and internationally recognized as a leader in both MGTI’s intense focus on optimizing pre- and post-transplant care is a contributing factor tovolume and outcomes, MedStar Georgetown Transplant Institute’s Center for Intestinal such results. Its use of Virtual Crossmatching, which MGTI first described in 2008, improvesCare and Transplant program regularly surpasses the national average for patient and graft patient and graft survival through enhanced matching of donors to recipients. Long-termsurvival for adults and children alike. immunological monitoring helps detect the possible development of antibodies for MGTI One-Year Intestinal Transplant Survival Rates Compared early intervention with established treatment to National Average for Transplants Between 1/1/2013 and 6/30/2015* protocols for antibody eradication. MGTI Survival U.S. Average Such achievements have produced a steady growth in size and stature. Since opening its Adult 89.47 doors in 2003, the program has performed Patient 76.32 more than 234 intestinal and multivisceral transplants altogether. It is also the sole site Adult 89.47 in the nation in equal volume, expertise and Graft experience in both pediatric and adult intestinalPediatric 69.35 transplant—a distinction that may contribute to Patient its outstanding results with both populations. 100.00 86.46Pediatric 95.00 Graft 75.74 0 20 40 60 80 100 Percent * SRTR, June 201614

nt Gutsy Maneuvers For years, Jarrod Taylor was the picture of health. “It’s part of my job to be in good physical condition and keep up with the soldiers,” says the Sergeant First Class, a paratrooper and veteran of deployments to Iraq and Afghanistan. But on the night of February 22, 2014, Jarrod began vomiting violently, landing him in his local hospital where he immediately went into cardiac arrest. The cause: septic shock. Unbeknownst to the 40-year-old—and undetected during his routine medical exams—a loop of his bowel had twisted, causing an obstruction but none of the condition’s typical warning symptoms. By the time Jarrod was hospitalized, his intestine had died, its toxins poisoning his body. The deceased organ was removed. But Jarrod then spent the better part of a year in hospitals as specialists fought to stabilize his health and rebuild his strength in preparation for an intestinal transplant at the MGTI—his goal. “I wanted to be around for my family,” he says. “I had a lot more life to live.” On May 12, 2016—the one-year anniversary of his transplant— Jarrod says there’s nothing he can’t do. His schedule backs him up: He's on active duty in the Army Cyber Command at Fort Belvoir in Virginia, participates in regular physical training and plans to run in the Army Ten-Miler this fall. In his free time, he also volunteers as a resource to others considering the major procedure. “I wanted to give something back to MedStar Georgetown,” Jarrod says. “They’re terrific.” 15

Intestinal Program Intestinal Rehabilitation: Comprehensive, Coordinated Medical and Surgical Highlights Solutions • One of the most experienced, While transplant may be the ultimate therapy for certain patients, the majority of intestinal failure accomplished and established centers in patients can avoid transplant altogether in the hands of experts. That’s often the case at MGTI’s the U.S. Center for Intestinal Care and Transplant, where medical and non-transplant surgical procedures account for approximately 50 percent of its annual patient volume. • 100% 1-year survival rates for The integrated program offers the full continuum of care to patients with intestinal failure, short gut pediatric patients transplanted between syndrome, fistulas and other conditions. For short bowel, surgical interventions can restore enteral January 1, 2013 and June 30, 2015* continuity and nutritional autonomy, particularly important for pediatric patients. Other therapies developed from clinical trials, many involving MGTI, include using the glucagon-like peptide-2 • Celebrated 10th anniversary in 2013 growth hormone to enhance gut absorption and novel lipid preparations like omegaven to reduce • One of only 5 intestinal/ the risk of liver damage from total parenteral nutrition, or TPN. Results of an MGTI study on the latter appeared in the May 2014 issue of the Journal of Pediatrics. multivisceral transplant training Research • Studying the role of the microbiome and programs accredited by the American its therapeutic relevance in intestinal Society of Transplant Surgeons The Center for Intestinal Care and Transplant transplantation has an active lab and research program, • One of only 7 CMS-approved along with one of the nation’s few intestinal • Examining the interactions between innate centers for small bowel transplant tissue repositories which supports scientific and adaptive immune responses and investigation here and elsewhere. intestinal stem cells, and their respective nationwide roles in intestinal regeneration after small A major current focus is determining how key bowel transplantation • 234 intestinal and multivisceral immunologic factors affect long-term transplant cases to date outcomes in intestinal transplant recipients by: • Translating integrative knowledge into novel diagnostic and therapeutic strategies such * SRTR, June 2016 • Advancing understanding of critical as immunomonitoring and immunotherapies interactions between host innate and that will benefit intestinal transplant patients16 adaptive immune cells such as antigen in the future presenting cells and T cells For more information about the MedStar Georgetown • Investigating how recently discovered innate Transplant Institute, visit MedStarGeorgetown.org/ effector cells such as innate lymphoid cells Transplant or call 800-442-4200. (ILCs) critically determine allograft rejection or acceptance

36th Time’s the CharmBorn with short gut syndrome, Karlene Novotny ofsoutheastern Pennsylvania was 25 when surgeonsfirst attempted to fix her intestinal tract. Over thesubsequent decades, she endured illness, pain anda total of 35 surgeries as her diseased bowels werefurther whittled away. Finally, what remained of hercolon had to come out completely.For the next 15 years, Karlene survived on totalparenteral nutrition—TPN or intravenous feedings—but long-term TPN actually destroys the liver. Sheand her husband Steve decided transplant was theonly way out of their living nightmare.A suitable donor organ became available, and onNovember 22, 2015, Karlene received a new colonand intestine during an 11-hour operation at theMedStar Georgetown Transplant Institute. Fivemonths later, MGTI’s specialists connected thetwo organs, giving Karlene a fully intact, workingintestinal tract for the first time in years.“I feel like I’m all together and in one piece again,”the 54-year-old says today. To celebrate the firstanniversary of the end of her surgeries and herreturn to health, Karlene hopes to run a 5K with herdaughter Ava next Mother’s Day.“My donor gave me the gift of life,” she says.“Once again, I have a ‘tomorrow’ to lookforward to.” 17 Jen Lebo Photography

transplant center for childrenOne of the largest and most experienced pediatric liver One-Year Pediatric Liver Transplant Patient Survivaltransplant programs in the United States, MedStar Georgetown’s Transplant Center forChildren is nationally and internationally known for its pediatric small bowel transplant 01/01/13-06/30/15*volume and outcomes. In 2015, it performed 32 pediatric liver transplants, a numbermatched by only one other program in the nation. Through a unique 12-year-old partnership, % 100.0its specialists perform all liver and small bowel transplants for Children’s National Medical 100Center, contributing to MGTI’s position as the largest program in the Washington, D.C., area. 95.72 96.45Providing complex medical, surgical and transplant care for pediatric patients from birthonward, the Transplant Center for Children benefits from being part of the larger adult 95program through the advantages of shared resources, depth of disciplines, years of 95experience and dedicated expertise. As a consequence, MGTI can offer patients across theage spectrum the highest, most sophisticated and effective level of care—before, during and 90after, or even instead of, transplant—while still providing the same services, family-centered 90approach and kid-friendly environment found within a children’s hospital. 85Pediatric Liver Disease Specialists from Children’s, home to one of the foremost metabolic disease programs in the 85In 2013, MGTI became one of only two centers world, and MGTI jointly manage pre- and post- 80nationwide to embrace a novel concept: transplant care.Prevent potential brain damage from Maple 80 MGTISyrup Urine Disease, propionic acidemia and In 2015 alone, MGTI performed 32 liver MGTI Actual U.S. Actual Expectedcertain other organic acidurias through a pre- transplants on pediatric patients includingemptive liver transplant. infants—a population very few transplant *SRTR, June 2016 centers nationwide are willing to tackle. In fact,The approach has worked. To date, more babies less than one year old account for morethan 30 pediatric patients with metabolic than 50 percent of the center’s pediatric livergenetic disorders have undergone liver transplants. Even more impressive: A full 25transplant at MGTI, a group that now comprises percent of the program’s patients are <6mo/between 40 and 45 percent of the program’s 6 kg at the time of transplant.overall pediatric liver transplant recipients.18

A Tale of Two Transplants:Liver Lethal to One SavesAnotherFirst-grader Korey Scott of Maryland was born with MapleSyrup Urine Disease—a rare metabolic disorder that cancause coma, seizures, brain damage, even death. The onlycure is a liver transplant.Meanwhile, middle-aged Isobel Short of Virginia Beach knewshe would die if she didn’t soon get a new liver to replace herfailing one.Both got what they needed on the same day, in the sameplace, thanks to the MedStar Georgetown TransplantInstitute.A pioneer in using transplantation as a precautionarytreatment for children with MSUD, the Institute is alsoat the forefront of transplanting those MSUD livers intopatients who desperately need them. In effect, the liver thatcontributes to life-threatening conditions in one person cansometimes be used to save the life of another.That’s exactly what took place in January 2016. In acoordinated, domino procedure, Korey received a liverfrom a deceased donor, then his liver was transplantedinto Isobel. After the operations, both flourished.“It’s just remarkable,” says Korey’s mother Andrea.“We’re now looking forward because Korey’s best quality oflife is yet to come. And I cried when I learned his liver wasnow working in someone else.”Adds Isobel, “Thanks to this beautiful little boy,I have a second chance at life.” 19

One-Year Pediatric Intestinal Pediatric Intestinal Disease ResearchTransplant Patient Survival Over the last few years, the pediatric small Such accomplishments notwithstanding, the01/01/13-06/30/15* bowel transplant program achieved a level of Center’s physician-scientists continue to look excellence unequalled by any other program for more and better ways to improve care and % 100.0 in the nation, regularly attaining a 100 percent outcomes. Current areas of inquiry include100 patient survival rate, as reported by the research into intravenous lipid therapy for Scientific Registry of Transplant Recipients TPN-associated liver disease, the use of a95 (SRTR). During FY 2015 alone, the Transplant genetically engineered hormone (GLP-2) to Center for Children performed 13 of the improve intestinal rehabilitation and prevent complex procedures. (See page 14 for more the need for intestinal transplant in children, information.) and understanding and reducing opportunistic infections.90 Pediatric Transplant Program Highlights 86.46 • Site of the first-ever double domino liver transplants involving two pediatric patients85 with Maple Syrup Urine Disease and two adults (see story on page 19) 80 U.S. MGTI • One of the highest performing and largest pediatric small bowel programs*SRTR, June 2016 in volume and outcomes • Achieved 100% 1-year patient survival for both pediatric liver and pediatric intestinal transplants* • Expanded medical staff *SRTR, June 2016For more information about the MedStar Georgetown Transplant Institute, visit MedStarGeorgetown.org/Transplant or call 800-442-4200.20

facilities and amenitiesFrom specially trained, Magnet-designatedtransplant nurses to dedicated facilities, MedStar GeorgetownTransplant Institute takes the needs and comforts of patients andfamilies to heart. The inpatient pediatric transplant unit featureshomelike accommodations with sleep-in rooms, a family lounge,a play room and even laundry facilities. For the convenienceof out-of-town families, the hospital maintains two nearbyapartments and offers special pricing at an on-site hotel andothers. A tasteful waiting area and colorful play room welcomepatients and families to MGTI’s new office and exam space.Free internet access, a variety of eateries and a warm and caringstaff help make each stay the best it can be. 21

researchIn 2013, the Center for Translational Transplant Medicine Intestinal Immunologywas jointly formed by the MedStar Georgetown Transplant Institute and Georgetown Intestinal immunology is a prime example ofUniversity Medical Center with the ultimate goal of transforming patient care. Toward that how CTTM is contributing to the field. Withend, CTTM promotes the cross-fertilization of ideas by bringing together disease groups so few intestinal transplant centers in theand specialties, laboratory scientists and transplant physicians to work together for nation, MGTI fulfills a vital role by collecting,innovative responses to current clinical problems. processing and storing intestinal biospecimens from its well-established center. The largestOverarching areas of interest include immunity modulation, inflammation, cellular such repository in the world, MGTI currentlyreprogramming, tolerance induction and other potential paths to improve graft survival and houses 3,000 samples which it shares withreduce infection and rejection, particularly for complex small bowel transplantations. other institutions conducting intestine-­related scientific investigations nationwide. CombinedSince its debut, approximately 100 clinical trials have been launched under the CTTM with MGTI’s own body of work, these collectiveumbrella, ranging from investigator-initiated basic studies to retrospective chart reviews studies are molding the future of overallto sponsored therapeutic drug and clinical device trials. By comparison, only 85 transplant- gastrointestinal care.related trials were conducted altogether over the previous decade. MGTI’s intestinal research focuses onAs a result of such activities, clinical research enrollment for phase I and phase II surgery delineating the mechanisms that contribute totrials is now double what it was before CTTM. Today, nearly all MGTI physicians are post-transplant infection and rejection of theengaged in research to hasten the clinical adaptation of new and better protocols, treatment small bowel. By identifying specific biomarkers,algorithms, immunosuppressive regimens, surgical techniques and other advancements. translational researchers hope to manipulate the body’s immune response for improvedIn 2015, CTTM expanded into a new 3,000-square-foot lab dedicated to transplant graft survival.immunology and recruited Alexander Kroemer, MD, PhD, to the newly created position ofTransplant Lab Chief, advancing the pace of research and development even further.22

Select Research Activities 2012-2016As part of a major academic medical center, MGTI is actively involved Use of Acthar in CKD Stage V or ESRD A Three-part, Multicentre,in basic, clinical and translational research to improve current care Patients in Preparation for a Kidney Randomised, Double-blind, Placebo-and advance knowledge, practices and techniques for the future. Transplant. controlled, Parallel-group, Sequential Monica Grafals, MD Adaptive, Phase II Study to EvaluateKidney Envarsus XR in African American Renal A Randomized, Parallel-group, Double- the Safety, Tolerability and Efficacy of Transplant Recipients. blind, Placebo-controlled, Multicenter OPN305, a Humanised MonoclonalAn Historical-cohort, Multicenter, Monica Grafals, MD Study of Eculizumab for the Prevention Antibody that Blocks Toll-like ReceptorObservational Study to Identify and A Multicenter, Prospective, Double- of Delayed Graft Function after Kidney 2, in Renal Transplant Patients at HighCharacterize CALCINEURIN Inhibitor blind, Randomized, Placebo- Transplantation in Adult Subjects Risk of Delayed Graft Function.Usage Patterns Post-transplant and controlled, Phase 3 Study of BB3 to at Increased Risk of Delayed Graft Matthew Cooper, MDImpact on Allograft Outcome-registry. Reduce the Severity of Delayed Graft Function. The Use of the Prevena™ IncisionMatthew Cooper, MD Function in Recipients of a Deceased Matthew Cooper, MD Management System on ClosedWellness and Health Outcomes of LivE Donor Kidney. Prospective Observational Trial to Recipient Site Incisions in RenalDonors (WHOLE-Donor). Matthew Cooper, MD Evaluate the Correlation of T-SPOT® Transplant Subjects.Matthew Cooper, MD A Randomized, Placebo-controlled, response to CMV disease and T Cell Matthew Cooper, MDAstagraf XL® to Understand the Prospective, Double-blind, Multicenter Mediated Acute Graft Rejection, as A Randomized, Open-label,Impact of Immunosuppression on Phase 2/3 Study of the Efficacy Amended from Time to Time and Multicenter Trial to Determine SafetyDe Novo DSA Development and and Safety of SANGUINATE™ for Incorporated Herein by Reference. and Efficacy of ECULIZUMAB in theChronic Immune Activation in Kidney Reduction of Delayed Graft Function Matthew Cooper, MD Prevention of Antibody MediatedTransplantation. in Recipients of a Donation after Brain Evaluation of Acute Rejection Rates in Rejection (AMR) in Living DonorMatthew Cooper, MD Death Kidney Transplant. De Novo Renal Transplant Recipients Kidney Transplant Recipients RequiringA Phase 3, Randomized, Double-blind, Matthew Cooper, MD Following Thymoglobulin Induction, Desensitization Therapy.Placebo-controlled Study to Evaluate A Randomized, Double-blind, CNI-free, Nulojix (belatacept) -based Matthew Cooper, MDthe Efficacy and Safety of QPI-1002 for Placebo-controlled Study to Evaluate Immunosuppression. Multicenter Pilot Study of BB3 toPrevention of Delayed Graft Function the Efficacy and Safety of Cinryze® Alexander Gilbert, MD Improve Renal Function in Patientsin Recipients of a Donation after (C1 Esterase Inhibitor [Human]) for Prospective Kidney Bx Collection. with Signs and Symptoms ofBrain Death Older Kidney Transplant the Treatment of Acute Antibody- Monica Grafals, MD Significant Renal Injury after Kidney(ReGIFT). Mediated Rejection in Kidney Retrospective Kidney Bx Collection. Transplantation and at Risk for Dialysis.Matthew Cooper, MD Transplant Patients. Monica Grafals, MD Matthew Cooper, MDImpact of CCR5 Blockade in HIV+ Alexander Gilbert, MDKidney Transplant Recipients.Matthew Cooper, MD 23

A Phase II Randomized, Double- Polaris 1: Phase 3, Global, Multicenter, HCV-Target: Hepatitis C Therapeutic Establishment of a Liver Tissue andblind, Placebo-controlled Trial of Randomized, Double-blind, Placebo- Registry and Research Network— Serum Bank.MCMV5322A/MCMV3068A for controlled Study to Investigate the A Longitudinal, Observational Study. Amol S. Rangnekar, MDthe Prevention of Cytomegalovirus Safety and Efficacy of Sofosbuvir/ Coleman I. Smith, MD A Multicenter, Double-blind, Sponsor-Disease in High-risk Kidney Allograft Velpatasvir/GS-9857 Fixed-dose A Phase 3, Double-blind, Randomized, open Trial of IDN-6556 in Subjects whoRecipients. Combination for 12 Weeks in Direct- Long-term, Placebo-controlled, had Hepatitis C Virus (HCV) ReinfectionMatthew Cooper, MD acting Antiviral-experienced Subjects Multicenter Study Evaluating the and Liver Fibrosis Following with Chronic HCV Infection. Safety and Efficacy of Obeticholic Orthotopic Liver TransplantationLiver Coleman I. Smith, MD Acid in Subjects with Nonalcoholic for Chronic HCV Infection and who Polaris 2: A Phase 3, Global, Steatohepatitis. Subsequently Achieved a SustainedA Pilot Study to Assess Safety, Multicenter, Randomized, Open-label Coleman I. Smith, MD Virologic Response Following Anti-Tolerability, and Efficacy of Sofosbuvir Study to Investigate the Safety and A Multicenter, Randomized, Double- HCV Therapy.and Ledipasvir for Hepatitis C Efficacy of Sofosbuvir/Velpatasvir/ blind, Placebo-controlled Phase III Coleman I. Smith, MDTreatment in HIV/HCV Co-infected GS-9857 Fixed-dose Combination for Study to Evaluate the Efficacy and An Open-Label, Multicenter,Subjects Pre- or Post-liver Transplant. 8 Weeks Compared to Sofosbuvir/ Safety of Elafibranor in Patients with Historically-controlled Study to AssessThomas M. Fishbein, MD Velpatasvir for 12 Weeks in Direct- Nonalcoholic Steatohepatitis (NASH) Safety and Efficacy of ELAD® in acting Antiviral-naïve Subjects with and fibrosis. Subjects with Acute Liver Failure (ALF).A Randomized, Open-label, Chronic HCV Infection. Coleman I. Smith, MD Rohit S. Satoskar, MDMulticenter, Controlled, Pivotal Study Coleman I. Smith, MD Seroprevalence of Hepatitis E in Organ A Phase III Double Blind Clinical Trial toto Assess Safety and Efficacy of ELAD® Polaris 3: A Phase 3, Global, Transplant Subjects. Study the Efficacy and Safety of thein Subjects with Alcohol-induced Liver Multicenter, Randomized, Open-label Amol S. Rangnekar, MD Combination Regimen of MK-5172 andDecompensation (AILD). Study to Investigate the Safety and A Phase 2, Randomized, Double-blind, MK-8742 in Subjects with Chronic HCVRohit S. Satoskar, MD Efficacy of Sofosbuvir/Velpatasvir/ Placebo-controlled, Parallel Group, GT1, GT4 and GT6 Infection with GS-9857 Fixed-dose Combination for Multiple Center Study to Evaluate the Inherited Blood Disorders with andA Multicenter, Randomized, Placebo- 8 Weeks and Sofosbuvir/Velpatasvir Safety, Tolerability, and Efficacy of without HIV Co-infection.controlled, Double-blind Study for 12 Weeks in Subjects with Chronic NGM282 Administered for 12 Weeks Rohit S. Satoskar, MDto Confirm Efficacy and Safety Genotype 3 HCV Infection and in Patients with Primary Sclerosing Standardized Treatments forof Terlipressin in Subjects with Cirrhosis. Cholangitis (PSC). Subjects with Alcohol-inducedHepatorenal Syndrome Type 1. Coleman I. Smith, MD Coleman I. Smith, MD Liver Decompensation (AILD) plusRohit S. Satoskar, MD Polaris 4: A Phase 3, Global, HCV-TARGET: Hepatitis C Therapeutic Associated Co-morbidities for the VTI- Multicenter, Randomized, Open-label Registry. 210 Study.A Phase 2, Multicenter, Open-label Study to Investigate the Safety and Coleman I. Smith, MD Rohit S. Satoskar, MDStudy to Investigate the Safety and Efficacy of Sofosbuvir/Velpatasvir/ Prevention and Treatment of AmatoxinEfficacy of Sofosbuvir/Ledipasvir GS-9857 Fixed-dose Combination for Induced Hepatic Failure withFixed-dose Combination + Ribavirin 12 Weeks and Sofosbuvir/Velpatasvir Intravenous Silibinin (Legalon® SIL): AnAdministered in Subjects Infected with for 12 Weeks in Direct-acting Antiviral- Open Multicenter Clinical Trial (PhaseChronic HCV who have Advanced Liver experienced Subjects with Chronic II/III).Disease or are Post-liver Transplant. HCV Infection who Have Not Received Rohit S. Satoskar, MDRohit S. Satoskar, MD an NS5A Inhibitor. Coleman I. Smith, MD24

A Randomized, Open-label, An Observational Study to Assess A 24-Week Double-blind, Safety, Small BowelMulticenter, Controlled Study to Assess Documentation of Hepatic Efficacy, and PharmacodynamicSafety and Efficacy of Elad® in Subjects Encephalopathy in Clinical Practice. Study Investigating Two Doses of Intestinal Transplantion for Short Gutwith Acute Alcoholic Hepatitis (AAH). Rohit S. Satoskar, MD Teduglutide in Pediatric Subjects Syndrome after Bariatric Surgery.Rohit S. Satoskar, MD through 17 Years of Age with Short Cal S. Matsumoto, MDProtocol for Tracking Economic Data A Randomized, Open-label Trial of Bowel Syndrome who are Dependentof Subjects Participating in Protocols the Safety and Efficacy of DEB025/ on Parenteral Support. Immunologic Characteristics of Long-VTI-208, VTI-210, or VTI-212. Alisporivir in Combination with Stuart S. Kaufman, MD term Intestinal Transplant Surivors. Rohit S. Satoskar, MD Pegylated Interferon-a2a and Ribavirin Biomarkers for Post-transplant Cal S. Matsumoto, MDProspective, Observational, Post- (peg-IFNa2a/RBV) and Boceprevir in Lymphoproliferative Disorders inmarketing Renal Safety Surveillance Combination with peg-IFNa2a/RBV Children. Incidence of Thrombophilia in PatientsRegistry in Patients with Chronic in African American Treatment-naïve Thomas M. Fishbein, MD Referred for Intestinal Transplantation.Hepatitis B (HBV) Infection with Patients with Chronic Hepatitis C A Pilot Study of Frailty in Children Cal S. Matsumoto, MDDecompensated Liver Disease Genotype 1. with End-stage Liver Disease: MovingReceiving Nucleotide/side Therapy Rohit S. Satoskar, MD Beyond PELD. Successful Isolated IntestinalWhile on the Orthotopic Liver Nada A. Yazigi, MD Transplantation in SensitizedTransplant (OLT) List. Pancreas Prospective Observational Study to Recipients with the Use of Virtual CrossRohit S. Satoskar, MD Assess Outcomes in Pediatric Liver Matching.A Phase 2b, Randomized, Double- Effect of Liver Histopathology on Islet Transplant Recipients. Cal S. Matsumoto, MDblind, Placebo-controlled Trial Cell Engraftment Post Autologous Nada A. Yazigi, MDEvaluating Response Guided Therapy Transplant after Total Pancreatectomy A Safer Approach to Total Parenteralusing Combinations of Oral Antivirals for Chronic Pancreatitis. Nutrition in Pediatric Short Bowel(GS-5885, tegobuvir,and/or GS- Chirag S. Desai, MD Syndrome Intended to Decrease9451) with Peginterferon Alfa 2a and the Frequency and Severity of LiverRibavirin in Treatment Experienced Collaborative Islet Transplant Registry. Damage.Subjects with Chronic Genotype 1 Wanxing Cui, MD, PhD Stuart S. Kaufman, MDHepatitis C Virus Infection.Rohit S. Satoskar, MD Pediatric Liver and SmallAn Expanded Access Phase 2 Study Bowelof Sofosbuvir with Ribavirin andwith or without Pegylated Interferon A Prospective, Open Label, Long-for 24 weeks in Subjects who have term Safety and Efficacy Study ofUndergone Liver Transplantation and Teduglutide in Pediatric PatientsWho Have Aggressive, Recurrent with Short Bowel Syndrome whoHepatitis C Infection. Participated in TED-C14-006.Rohit S. Satoskar, MD Stuart S. Kaufman, MD Prevalence and Correlates of Post- traumatic Stress Symptoms (PTSS) in Adolescent Solid Organ Transplant Recipients. Thomas M. Fishbein, MD 25

Select Representative Publications 2012 to 2016Kidney Andrews P, Cooper M, Verbesey J, Pomposelli JJ, Akoad M, Khwaja K, Khalili M, Lombardero M, Chung RT, Ghasemian S, Rogalsky D, Moody P, Lewis WD, Cheah YL, Verbesey J, Terrault NA, Ghany MG, Kim WR, LauMassie A, Luo X, Lonze B, Cooper M, Chen A, Alexandrov P, Wang H, Chen Jenkins RL, Pomfret EA. Evolution of D, Lisker-Melman M, Sanyal A, Lok ASSegev D.  Early Changes in the Kidney Y.  Mannitol Infusion Within 15 Min of Anterior Segment Reconstruction After for the Hepatitis B Research Network,Distribution under the New Allocation Cross-Clamp Improves Living Donor Live Donor Adult Liver Transplantation: Roberts LR, Smith CI, et al. DiabetesSystem. J Am Soc Nephrol 2016 Aug Kidney Preservation. Transplantation  A Single-center Experience. Clin and Prediabetes in Patients with27. 2014;98(8):893-897. Transplant 2012 May-Jun;26(3):470-5. Hepatitis B Residing in North America.Orandi B, Luo X, Massie A, Cooper Andrews P, Wang, HW, Wierwille J, Hepatology 2015;62:1364-1374.M, Montgomery R, Segev D.  Survival Gong W, Verbesey J, Cooper M, Chen, Liver Ghany MG, Perrillo R, Li R, Belle SB,Benefit in HLA-Incompatible Live Donor Y.  Optical Coherence Tomography of Janssen HLA, Terrault NA, Shuhart MC,Kidney Transplantation.  NEJM 2016; the Living Human Kidney.  J Innov Opt Kwok RW, Ahn J, Schiano TD, Te HS, Lau DT, Kim WR, Fried MW, Sterling RK,374:940-950. Health Sci 2014;7(4):130064. Potosky DR, Tierney A, Satoskar R, Di Bisceglie AM, Han SB, Ginoya-Racya Sosin M, Lumeh W, Cooper M.  Robertazzi S, Rodigas C, Wiegel J, Patel LM, Chang K, Lok AS for the HepatitisMoore D, Serur D, LaPointe Rudow Torsion of the Retroperitoneal Kidney: N, Gripshover J, Hassan MA, Branch A, B Research Network, Chung RT, SmithD, Rodrigue J, Hays R, Cooper M.  Uncommon or Underreported?  A Case Smith CI. Sofosbuvir + Ledipasvir for CI, et al. Characteristics of Adults in theLiving Donor Kidney Transplantation: Report and Review of the Literature.  Recurrent Hepatitis C in Liver Transplant Hepatitis B Research Network in NorthImproving the Efficiencies of Case Reports Trans 2014. Recipients: A Real-world Multicenter America Reflect Their Country of OriginLive Kidney Donor Evaluation — Orandi B, Massie A, Zachary A, Ratner Experience. Liver Transpl 2016 and Hepatitis B Virus Genotype. ClinicalRecommendations from a Consensus L, Cooper M. Montgomery R, Segev D.  Nov;22(11):1536-1543. doi: 10.1002/ Gastroenterology and HepatologyConference.  CJASN 2015;10(9):1678- Quantifying the Risk of Incompatible lt.24614. 2015;13:183-192.1686. Kidney Transplantation: A Multicenter Fontana RJ, Ellerbe C, Durkalski Study Am J Transplant 2014;14(7): Hsueh W, Shope TR, Koch TR, Smith VE, Rangnekar AS, et al. Two-YearRudow D, Hays R, Baliga P, Cohen D, 1573-1580. CI. Bariatric Surgery for the Treatment Outcomes in Initial Survivors with AcuteCooper M, Rodrigue J. Consensus Verbesey J, Cooper M. \"Simultaneous of Nonalcoholic Fatty Liver Disease: Liver Failure: Results from a Prospective,Conference on Best Practices in Live Paired Kidney Exchange.\" Kidney Is Vertical Sleeve Gastrectomy the Multicentre Study. Liver Int 2015Kidney Donation: Recommendations to Transplantation: Practical Guide to Best Future Option? Journal of Feb;35(2):370-80.Optimize Education, Access and Care.  Management, Weir MR, Lerma EV (Eds). Gastroenterology and Hepatology Chapman KA, Collado MS, Figler RA,Am J Transplant 2015;15(4):914-922. 2014. Research 2016;5:1880-1884. Hoang SA, Armstrong AJ, Cui W, PurdyScalea J, Barth R, Philosophe B, Cooper Wang D, Melancon JK, Verbesey J, Hu Rudnick MR, Demarchi L, Plotkin JS. M, Simmers MB, Yazigi NA, SummarM, LaMattina J.  Shorter Waitlist Time H, Liu C, Aslam S, Young M, Wilcox CS. Hemodynamic Monitoring During ML, Wamhoff BR, Dash A.for HCV+ Renal Allografts Improves Microvascular Endothelial Dysfunction Liver Transplantation: A State of the Recapitulation of Metabolic Defects in aLong-term Graft Survival Without and Enhanced Thromboxane and Art Review. World J Hepatol 2015 Jun Model of Propionic Acidemia UsingAssociated Progression of Clinical Liver Endothelial Contractility in Patients 8;7(10):1302-11. Patient-derived Primary Hepatocytes.Disease.  Transplantation 2015;99(6): with HIV. J AIDS Clin Res 2013 Dec Khan R, Albugeary M, Rangnekar Mol Genet Metab 2015 Dec 24. pii:1192-1196. 1;4(12):267. AS. Oral Vancomycin Effect in S1096-7192(15)30092-5. doi: Primary Sclerosing Cholangitis. Am J 10.1016/j.ymgme. 2015.12.008. [Epub Gastroenterol 2015 Oct;110(S1):S393. ahead of print] PubMed PMID: (Winner of ACG Presidential Poster 26740382. Award)26 Javaid AI, Hagn KJ, Kwok RM, Albugeaey M, Rangnekar AS, et al. A Fatal Cause of Fever and Pancytopenia in a Patient with Ulcerative Colitis Treated with Azathioprine.  Am J Gastroenterol 2015 Oct;110(S1):S389.

Rangnekar AS and Fontana RJ. Gabrielson A, Wu Y, Wang H, Jiang J, Satoskar R, Reau N. Potential Matsumoto CS, Kaufman SS, IslandIL-28B Polymorphisms and the Kallakury B, Gatalica Z, Reddy S, Kleiner Consequences of Healthcare ER, Kallakury B, Yazigi NA, KhanResponse to Antiviral Therapy in HCV D, Fishbein T, Johnson L, Island E, Recommendations: A Focus on the KM, Fishbein TM. Hepatic ExplantGenotype 2 and 3 Varies by Ethnicity: Satoskar R, Banovac F, Jha R, Kachhela U.S. Preventive Services Task Force. Pathology of Pediatric IntestinalA Meta-analysis. J Viral Hepat 2013 J, Feng P, Zhang T, Tesfaye A, Prins P, Hepatology 2013 Jul;58(1):422-7. Transplant Recipients PreviouslyJun;20(6):377-84. Loffredo C, Marshall J, Weiner L, Atkins Treated with Omega-3 Fatty Acid LipidRangnekar AS, Ellerbe C, Durkalski M, He AR. Intratumoral CD3 and CD8 Small Bowel and Intestine Emulsion. J Pediatr 2014 Jul;165(1):59-V, McGuire BM, et al. Quality of Life T cell Densities Associated with 64. doi: 10.1016/j.jpeds.2014.03.034.is Significantly Impaired in Long- Relapse-Free Survival in HCC. Cancer Kroemer A, Elsabbagh AM, Epub 2014 May 3. PubMedterm Survivors of Acute Liver Failure Immunol Res 2016 May;4(5):419-30. Matsumoto CM, Zasloff M, Fishbein PMID:24793206.and Particularly in Acetaminophen- Laster J, Satoskar R. Aspirin-induced TM. The Microbiome and its Yazigi NA. Long-term Outcomesoverdose Patients. Liver Transpl 2013 Acute Liver Injury. ACG Case Rep J Implication in Intestinal Transplantation. after Pediatric Liver Transplantation.Sept;19(9):991-1000. 2014 Oct 10;2(1):48-9. Curr Opin Organ Transplant 2016 Pediatr Gastroenterol Hepatol NutrRangnekar AS and Fontana RJ. Desai AP, Satoskar R, Appannagari A, Apr;21(2):135-9. doi: 10.1097/ 2013 Dec;16(4):207-18. doi: 10.5223/Managing Drug-Drug Interactions with Reddy KG, Te HS, Reau N, Meltzer DO, MOT.0000000000000278. pghn.2013.16.4.207. Epub 2013 DecBoceprevir and Telaprevir. Clinical Liver Jensen D. Co-management Between 31. Review. PubMed PMID: 24511516;Disease 2012 Apr;1(2):36-40. Hospitalist and Hepatologist Improves Diaz JL, Wanta SM, Fishbein TM, PubMed Central PMCID: PMC3915734.Rangnekar AS and Fontana RJ. the Quality of Care of Inpatients Kroemer A. Developments in McDaniel JD, Kukreja K, RistagnoMeta-analysis: IL-28B Genotype and with Chronic Liver Disease. J Clin Immunotherapy for Gastrointestinal RL, Yazigi NA, Nathan JD, Tiao G.Sustained Viral Clearance in HCV Gastroenterol 2014 Apr;48(4):e30-6. Cancer. Minerva Chir 2015 Radiofrequency Ablation of a LargeGenotype 1 Patients. Aliment Pharmacol Nguyen P, Plotkin JS, Fishbein Aug;70(4):217-30. Epub 2015 Apr 28. Hepatic Adenoma in a Child. J PediatrTher 2012 Jul;36(2):104-14. TM, Laurin JM, Satoskar R, Shetty PubMed PMID: 25916195. Surg 2013 Jun;48(6):E19-22. doi:Morales AL, Liriano-Ward L, Tierney K, Taylor AJ. Dobutamine Stress 10.1016/j.jpedsurg.2013.04.007.A, Sang M, Lalos A, Hassan M, Nair Echocardiography in Patients Pediatric Transplant PubMed PMID:23845652.V, Schiano T, Satoskar R, Smith C. Undergoing Orthotopic Liver Bucuvalas J, Filipovich L, Yazigi NA,Ledipasvir/sofosbuvir is Effective and Transplantation: A Pooled Analysis Schwarz KB, Dell Olio D, Lobritto SJ, Narkewicz MR, Ng V, Belle SH, Zhang S,Well Tolerated in Post-kidney Transplant of Accuracy, Perioperative and Long Lopez MJ, Rodriguez-Baez N, Yazigi Squires RH. Immunophenotype PredictsPatients with Chronic Hepatitis C Virus. Term Cardiovascular Prognosis NA, Belle SH, Zhang S, Squires RH; Outcome in Pediatric Acute LiverClin Transplant 2017 Feb 26. Int J Cardiovasc Imaging 2013 Pediatric Acute Liver Failure Study Failure. J Pediatr Gastroenterol NutrKwok RM, Ahn J, Schiano TD, Te HS, Dec;29(8):1741-8. Group. Analysis of Viral Testing in Non- 2013 Mar;56(3):311-5. doi: 10.1097/Potosky DR, Tierney A, Satoskar R, Juneja M, Euliano R, Satoskar R, Lewis acetaminophen Pediatric Acute Liver MPG.0b013e31827a78b2. PubMedRobertazzi S, Rodigas C, Lee Sang M, JH. Polypharmacy and Comorbidity are Failure. J Pediatr Gastroenterol Nutr. PMID: 23111765; PubMed CentralWiegel J, Patel N, Gripshover J, Hassan Associated with a Lower Early Virologic 2014 Nov;59(5):616-23. doi:10.1097/ PMCID: PMC3582763.MA, Branch A, Smith CI. Sofosbuvir Response in Hepatitis C Patients Treated MPG.0000000000000512. PubMedPlus Ledipasvir for Recurrent Hepatitis with First Generation Protease Inhibitor PMID: 25340974. Epub 2014 Jul 24. 27C in Liver Transplant Recipients. Liver Triple Therapy: A Preliminary Analysis. PubMed PMID: 25079486; PubMedTransplant 2016 Nov;22(11):1536-1543. Dig Dis Sci 2013 Nov;58(11):3348-58. Central PMCID: PMC4305349.For more information about the MedStar Georgetown Transplant Institute, Ng V, Nicholas D, Dhawan A, Yazigi NA,visit MedStarGeorgetown.org/Transplant or call 800-442-4200. Ee L, Stormon M, Gilmour S, Schreiber R,Taylor R, Otley A; PeLTQL study group. Development and Validation of the Pediatric Liver Transplantation Quality of Life: A Disease-specific Quality of Life Measure for Pediatric Liver Transplant Recipients. J Pediatr 2014 Sep;165(3):547-55.e7. doi: 10.1016/j. jpeds.2014.05.024. Epub 2014 Jun 26. PubMed PMID: 24976329.

faculty Thomas Fishbein, MD Executive Director, MedStar Georgetown Transplant Institute Director, Liver Diseases and Transplant Program [email protected] and Small Bowel Hepatologists Coleman Smith, MD Kidney and Pancreas TransplantTransplant Rohit Satoskar, MD Ron Thomas, MD Surgeons Medical DirectorSurgeons Pediatric Hepatologists Matthew Cooper, MD Adult Liver Transplant Stuart Kaufman, MD Director, Kidney and Pancreas Cal Matsumoto, MD Medical Director, Transplantation Director, Intestinal Transplant Pediatric Transplant Thomas Faust, MD Khalid M. Khan, MD Raffaele Girlanda, MD Nada A. Yazigi, MD S. Reza Ghasemian, MD, FACS Alexander Lalos, MD Jason Hawksworth, MD Peter L. Abrams, MD Jennifer Verbesey, MD Amol Rangnekar, MD Alexander H. Kroemer, MD28

Nephrologists Interventional Radiology Oncology Lorenzo DeMarchi, MD Basit Javaid, MD Alexander Kim, MD John L. Marshall, MD Medical Director, Chief, Vascular and Chief, Division of Hematology/ Interventional Radiology Oncology Kidney and Pancreas Transplant Director, Clinical Research, Georgetown’s LombardiJack Moore, MD, FACP Comprehensive Cancer Center ResearchMedical Director, NephrologyDepartment Emil I. Cohen, MD Aiwu “Ruth” He, MD Michael Zasloff, MD, PhDMedStar Washington Scientific DirectorHospital Center Center for Translational Transplant Medicine (CTTM)Alexander Gilbert, MD Michael Jon Pishvaian, MD, PhD Gastroenterology Transplant Anesthesia Children’s National Medical Center Nadim G. Haddad, MD Jeffrey Plotkin, MD Chief of Gastroenterology Director, Transplant Asha Moudgil, MD Anesthesia Director, Kidney TransplantImaging/Radiology Thomas Loughney, MD Children’s National Medical Chief, Endoscope Unit Center Reena C. Jha, MD Director, Magnetic Resonance Parvathi Mohan, MD Imaging Director of HepatologyImaging/Radiation Oncology Sudha Ved, MD Clarivet Torres, MD Director, Pediatric Transplant Medical Director Intestinal Keith Unger, MD Anesthesia Rehabilitation Program 29

Clinical Support MedStar Georgetown Transplant Institute’s clinical support teams include every professional needed throughout the transplant process. From social workers, transplant coordinators, financial assistance personnel, and the HLA lab to psychologists and dietitians, resources are in place to get patients listed and transplanted quickly. Clinical Operations Kidney Transplant Pediatric Liver Director Program Transplant Toby Conlon, RN, BSN Eloida Gonzales, RN, BSN Lauren Anderson, RN, BSN Jaclyn Inman, RN, BSN Aimee Block, LGSW Hepatobiliary/Islet Arlette Jackson, RN, BSN Ashley Dunnan, RN, BSN Cell Alexandra Radomsky, LGSW Gabrielle Rinaldi, RN, BSN Hepatology Erin Meslar, PA-C Denise Ryles-McKoy, RD Leah VanderMark, LICSW Michelle Jenkins, PA-C Inpatient Services Liver Transplant Post Liver Ashley Baird, PA-C Program Courtney Barrett, NP Peymei Wu, PA-C Lauren Chapnick, NP Michelle Domine, RN, BSN Annelise Nolan, PharmD Maggie Green, RN, BSN Post Transplant Jackie O'Rourke, RN, BSN Andrea Keller, PA-C Services Colleen Rodigas, DNP Mercedes Laudano, RD Karen Strenger, PA-C Rose Neugroschel, LGSW Caroline Cannon, NP Bernadette Schwan, NP Cristina Pacheco, RN, BSN Katie Cruz, NP Jessie Wetherby, RN, BSN Jill Fenton, NP Kidney and Marcella Heffelfinger, NP Pancreas Transplant Living Donor Siobhan Hourigan, NP Program Titi Orimogunje, NP Nancy Atouani, RN, BSN Melika Powell, NP Maggie Coakley, RN, BSN Sarah Cady, LGSW Joanne Rapisura, RN, BSN Caitlin Cocopardo, RN, BSN Marisa Gallahan, LPN Suzanne Robertazzi, NP Pamela Grewal, LICSW Ashley Anne Hamby, RN, BSN Hannah Sagedy, PA-C Small Bowel Transplant Program Jill Fenton, NP, a member of the Rebecca Jones, RD post-transplant team at MedStar Rebecca Kahn, RD Georgetown University Hospital, Eryca Kasse, LICSW discusses treatment with a patient. Ashley Voyles, RD30

A Medical Journey Ends WithGratitude and Giving BackWhile still a high school student in Saudi Arabia,Mohammed Almujel got news that would change his life:He had pancreatic cancer. The diagnosis sent the teenagerand his family on a medical journey spanning more than6,000 miles, four years and numerous surgeries, andending with a successful multivisceral transplant atMedStar Georgetown.Mohammed’s ordeal began in earnest at another U.S.hospital when surgery to remove his cancerous tumorunexpectedly and irreparably damaged his small bowel.The only solution was a transplant.“My parents asked for a referral to the best small bowelcenter in the United States,” Mohammed recalls. “And theanswer was MedStar Georgetown Transplant Institute.”But by the time a suitable small bowel was available,Mohammed’s condition had deteriorated badly. To regainhis health, he now needed a new stomach, liver, pancreas,colon and kidneys.“Through a very long hospitalization and recovery,MGTI’s doctors and nurses helped me remain strong,”Mohammed recalls. “Every step of the way, we weretreated like family members, with mental, emotional andphysical support.”These days, Mohammed returns the favor as a volunteerat MedStar Georgetown. “I am very grateful to havehad a second chance in this life. Now I’m helping peoplewith the same severe needs that I had, givingthem hope.” 31

MedStar Georgetown Transplant Institute LocationsThe MedStar Georgetown Transplant Institute is making itmore convenient to be evaluated for transplantation. Our seven locations throughout theBaltimore-Washington region make it easy to access our experienced multidisciplinary team.E 695 G A. MedStar Georgetown University Hospital 3800 Reservoir Rd., NW Frederick 70 Baltimore Washington, DC 20007 270 B. M edStar Washington Hospital Center 110 Irving St., NW 370 95 Washington, DC 20010 495 Montgomery C. MedStar Georgetown Transplant Institute 270 A B County, MD in Fairfax 3301 Woodburn Rd. 495 Annandale, VA 22003 C Washington, D.C. F D. M edStar Southern Maryland Hospital Center Virginia 395 95 7503 Surratts Rd. 295 Clinton, MD 20735 D 495 E. MedStar Georgetown Transplant Institute in Frederick 70 110 Thomas Johnson Dr. Frederick, MD 21702 95 F. MedStar Georgetown Transplant Institute32 in Annapolis 888 Bestgate Rd. Suite 210 Annapolis, MD 21401 G. MedStar Franklin Square Medical Center 9000 Franklin Square Dr. Baltimore, MD 21237 For more information about the MedStar Georgetown Transplant Institute, visit MedStarGeorgetown.org/Transplant or call 800-442-4200. Leonardtown, MD 235 5 234



MedStar Georgetown University Hospital3800 Reservoir Rd., NWWashington, DC 20007202-444-3700 phone800-442-4200 physician access lineMedStarGeorgetown.org/TransplantMedStar Washington Hospital Center110 Irving Street, NWWashington, DC 20010MedStarWashington.org