Â鶹ÊÓƵ

For Nanda Kerkar, M.D., performing a liver transplant is a ‘team sport.’

Ever since joining Golisano Children’s Hospital in 2017 as the director of Pediatric Liver Disease and Liver Transplantation, she’s worked with a single goal – to be able to do liver transplants for children living in and around Rochester. She’s collaborated with a group of surgeons, hepatologists, pharmacists, anesthesiologists, intensivists, nurses and physicians to prepare for the first transplant and pave the way for a viable and sustainable program.

“It’s like preparing a dish,” said Kerkar, “your entree may be fabulous, but if everything else isn’t brought together properly, it doesn’t work. And while the medical and surgical teams are key, input from the patient and family is also critical for good outcomes.”

For several years, Kerkar and her team methodically put the pieces into place, and in late 2019, they identified their first patient, 18-year-old Margaret Giordano. Then, after several months of trying to secure a liver, the first surgery came together in a flash.

“They called and asked us, ‘can you and Maggie be in the hospital in 15 minutes,” said Laurie Vahey, Maggie’s mother.

Fifteen minutes was a tight turnaround time, but Maggie’s bag was already packed, and for the family, relief couldn’t come any sooner.

An Unmanageable Condition

Maggie was born with Crigler-Najjar Syndrome (CNS), a genetic disorder in which the liver enzyme that processes bilirubin — an orange-yellow bile pigment that is a byproduct of the natural breakdown of old red blood cells — is not produced in the quantity required. As a result, bilirubin begins to build up in the body resulting in jaundice, which causes yellowing of the eyes and skin. If untreated, this build-up can cross the blood brain barrier and cause excess fatigue, hearing loss, decline of cognitive functioning, and even death.

There are two recognized types of CNS: Type one doesn’t make any of the enzyme, typically necessitating immediate treatment and transplant surgery during infancy. Type two of the syndrome usually makes enough of the enzyme so medical treatment is usually sufficient. Maggie, however, had a unique version that Laurie refers to as “type one-and-a-half:” not needing surgery as an infant but necessitating more extensive treatment. It befuddled many of the pediatricians who saw her.

“They didn’t know what to do with someone like Maggie,” said Laurie. “One doctor told me she would not live to be 2 years old.”

The parents searched for someone who could treat her, eventually finding Holmes Morton, M.D. in Lancaster, Pa. Morton was one of the foremost experts on CNS and also metabolic disorders, having studied and treated the syndrome for patients in Lancaster’s Amish country.

Morton prescribed Maggie a regimen of medications and phototherapy treatment. Every night, Maggie would sleep under a bank of 2-by-2 foot fluorescent lights for 8-to-12 hours. “We did it while she was sleeping so she wouldn’t be blindfolded during the day,” said Laurie.

For several years, Maggie was able to manage with this treatment. As she got older and bigger, however, these lights became less effective, so the  family purchased larger LED lights from a Dutch company. While these lights were more powerful and reduced the amount of needed phototherapy to one hour a day, the therapy was harsh and Maggie’s condition worsened still, with the jaundice increasing. In addition, Maggie was increasingly fatigued.

“Growing up, she would participate in softball, swimming and karate. Around middle school she had to start dropping sports, and by high school, she was sleeping 12-to-18 hours a day,” said Laurie.

Fatigue was only the tip of the iceberg. Sudden illnesses or injuries presented immediate concerns and Maggie started losing hearing in one of her ears, regularly  struggled to think clearly, and had to be vigilant about staying hydrated, as dehydration can cause bilirubin to spike to dangerous levels.

“If you look at 0-10, with 10 being brain damage, Maggie’s buffer was becoming less and less,” said Laurie.

In addition, Maggie — being an outstanding student despite her challenges — was graduating high school, and would potentially face enormous difficulties trying to start a life away from home in college.  The 6-by-4 foot lights she used weighed hundreds of pounds, and Maggie — in her condition –— needed someone to help her when she had episodes where she couldn’t think clearly.

The family began to consider surgery. The first step was transitioning away from UPMC Children’s Hospital of Pittsburgh, where Maggie had received treatment since she was 8. A major staffing transition had left them feeling less comfortable with their care, so they decided to meet with Kerkar at GCH. The family immediately felt at ease, as Kerkar demonstrated both compassion for Maggie and a willingness to study her condition inside-and-out.

“I don’t give praise without it being due, but the woman (Kerkar) is a powerhouse,” said Laurie, “Besides the fact that she’s very intelligent, she is also the most driven and caring doctor for Maggie that I’ve ever met.”

An Uphill Battle – and an Unexpected Break

The family had extensive discussions with Kerkar and surgeon Koji Tomiyama, M.D., Ph.D., a member of the UR Medicine Transplant team and surgical director of pediatric liver transplantation. Both briefed the family on the risks of the surgery.

“Tomiyama is great with patients. He listens and wants to do it right rather than just doing it,” said Laurie.

The family ultimately decided to move forward. There was one significant obstacle, however: in order to get additional points on the waitlist for a deceased donor liver transplant, their case would have to be approved by the National Liver Review Board.

Each year, there are around 8000 adult liver transplants and 600 pediatric transplants, according to the American Liver Foundation. At any time, there are as many as 14,000 people on the waiting list. Each person in line for a liver transplant gets assigned a score in a particular region, and in the case of Maggie’s  family, they were in competition with a lot of candidates from downstate New York. To determine who gets approved for a transplant during this competitive process, the board allots a certain amount of points per case. Patients are assigned these points depending on their blood group, blood test results, and severity of liver status. Because Maggie’s condition did not seem urgent on the surface, she did not have the necessary points. Kerkar tried appealing to the National Review Board of which she is a member, but could not vote on this case, as it was local.

“If you feel as a physician that the patient is more sick clinically than the calculated points reflect, you can write to the appeal board. I wrote to them but the appeal was turned down, then I modified it with the help of many details provided by Maggie’s mother and re-sent it requesting for lesser points than the earlier appeal, but unfortunately, it was still turned down,” said Kerkar, “I realized she was not going to get an organ by the conventional route.”

Despite Kerkar’s best efforts, Maggie’s family transitioned toward looking for a living donor. They assembled a list of candidates and then began the process of blood testing to determine the right match. Then, COVID-19 hit and upended the whole process.

While New York City and its suburbs got hit hard – necessitating the prolonged cancellation of many elective surgeries in downstate hospitals, Rochester and Monroe County experienced a manageable COVID case load, and Â鶹ÊÓƵcontinued deceased donor transplants through the pandemic and resumed nearly all types of surgeries by May and June.

A long awaited change in liver allocation occurred in February and that change, combined with some shifting organ availability due to covid, benefitted patients in the region.  Shortly after activating Maggie on the list, a liver became available. Kerkar then worked to see if everyone was comfortable and prepared – from the family to the surgery, intensive care and support teams – to conduct the first pediatric liver transplant during a pandemic.

“It required lot of thinking through and making sure we were all on the same page,” said Kerkar, “We were lucky that our surgical team was quite brave and ready to move forward, and they’d done a few adult ones successfully during the pandemic, so I felt comfortable with my patient taking this risk, particularly as I was fortunate enough to have Maggie and her parents compoletely on-board with the plan of moving forward during the COVID environment.”

When the family got the call on June 24 that their surgery was approved, it was hard to believe. “I was in shock, I thought they were fibbing,” said Maggie. But there was no hesitation; they headed to the hospital.

Coming Together as a Team

The core surgery team was ready. Kerkar had spent several years leading pediatric transplant teams at the Mount Sinai Hospital in New York and Children’s Hospital of Los Angeles, and Tomiyama has more than 10 years of experience conducting both adult and pediatric surgeries. In addition, given that Maggie was adult-sized, she wouldn’t present the same challenges as younger kids.

“For little kids, you have to deal with much smaller connections between the vessels and bile duct,” said Tomiyama, “Maggie was a more straightforward case.”

Ensuring success of the surgery and recovery, however, meant extensive collaboration among several units in the hospital. The anesthesia team, for example, had experience operating on previous adult liver transplant cases, but had only ever transferred patients to the Surgical Intensive Care Unit (SICU).

“As an adult ICU physician, I am very familiar with the SICU since I work there and know all of the staff well.  The Pediatric Intensive Care Unit (PICU) staff are excellent, but I was unfamiliar with them and they were unfamiliar with me,” said Joseph Dooley, M.D., professor in the Department of Anesthesia, “There are some differences in approaches to patient management for pediatric patients versus adults – such as sedation and glycemic control  – that we worked together to manage.” 

In turn, the PICU unit worked to educate the nursing and provider teams to implement multi-disciplinary patient management with the Hepatology, Surgical and ICU units.

“During the process, we learned that our team can provide the excellent post-operative intensive care and ICU monitoring that these complex patients require, and do so with a focus on family-centered multi-disciplinary care,” said Jill Cholette, M.D., medical director of the Pediatric Cardiac Care Center.

The preparation of all teams – particularly with the added dimension of the pandemic – was noticeable to Laurie.

“The hospital had procedures in place, and everyone wore masks and was organized,” she said, “the treatment in the PICU was superb, she was able to get the care and medication she needed right away. It was incredible that she was able to go home after only six days”

Recovery – and Future

Shortly after the surgery, Maggie was able to participate in major milestone. On July 25, she was walked across the stage and got her diploma with her cap-and-gown on, something that would have been unthinkable a few months ago with her liver condition and the pandemic.

Maggie plans to take a gap year before looking to enroll in college in the Fall of 2021. In the meantime, she is enjoying life again, and is sharper and more active than she’s ever been.

“Her clarity of thought is awesome, and she now has the ability to go away and do things that she wouldn’t be able to before,” said Laurie.

After this first success, the GCH liver transplant program is looking to build capacity in both surgery unit and supporting teams. One goal will be developing a core group of our pediatric anesthesiologists to subspecialize in pediatric transplantation, according to Dooley. Another will be potentially adding surgeons to increase volume and eventually treat younger children. Donations and support can assist with the hiring of faculty to fill these positions.

Ultimately, this first surgery will have a major impact on GCH’s ability to serve kids in the region, according to Patrick Brophy, M.D., chair in the department of pediatrics.

“Getting the surgical structure in place for kids, and the ability of having pediatric long-term care for kids getting a liver transplant, expands our service capabilities and keeps kids in the region rather than having to go out-of-state for transplants or to NYC.”

For Kerkar, this first surgery will set the foundation for a successful transplant program for kids of all sizes; all the more important considering the risks involved.

“If we hadn’t done it, how long would we have to wait to eventually get the organ for them? This is one of those cases where I’m glad we just decided to move forward; no regrets about doing things different,” she said.