What's New
New Life for Children with Liver Failure
1. Dean Balcirak, III with Kara Ventura, DrNP.
2. Penelope Lee.
3. Nathanial Alvarez.
All have had liver transplants and are doing extremely well.
Of all the diseases that may lead to liver transplantation in children, a rare condition called biliary atresia is the most common. It affects approximately one out of 12,000 newborns each year, and is believed to be the result of genetic, immunologic, and viral factors. In this disease, the biliary tract (the system of tubes carrying bile) both inside and outside the liver is destroyed, preventing the flow of bile from the liver to the gallbladder. If the bile flow away from the liver becomes blocked, it will cause inflammation in the liver. Left untreated, this can lead to scar tissue and ultimately cirrhosis, and the liver will lose its ability to function. Without surgery or a liver transplant, babies with biliary atresia will die.
For many children, the Kasai procedure (named for the surgeon who developed it) succeeds in rerouting the bowel and diverting bile away from the liver. Although many centers perform the Kasai procedure, their survival rates do not compare to the 98% survival rate achieved by R. Peter Altman, MD, previous Surgeon-in-Chief at Morgan Stanley Children's Hospital/NewYork-Presbyterian. Since his recent retirement, Dr. Altman's expert methods have been continued with superb results by Dr. Robert Cowles and other colleagues trained by Dr. Altman, who now comprise the core of Columbia's pediatric surgical team.
Though the surgical risk is small, the Kasai operation does not permanently cure the majority of patients. Because the biliary blockage leads to greatly accelerated fibrosis (scar tissue) formation, nearly half the children will need a liver transplant for complications of cirrhosis in the first few years of life. This is the reason why such patients must be very closely managed by gastrointestinal pediatricians specializing in hepatology, explains Dominique M. Jan, MD, Professor of Clinical Surgery. "Although the Kasai procedure permits some patients to live normally into young adulthood, complications relating to scar tissue in the liver can occur. In other cases, the bile flow is clear at first, but then gets blocked again." When the Kasai procedure succeeds, it works very well, according to Dr. Jan. In the long term, about 40% of patients do not need further treatment. "Two things are very important: making the diagnosis and performing the Kasai procedure very early," Dr. Jan emphasizes. According to Steven J. Lobritto, MD, Medical Director, Pediatric Liver Transplantation, "If we can delay transplant surgery by 10 or 20 years, there will likely be other procedures available later, as well as improved methods to prevent rejection."
About 60% of children will eventually need a liver transplant, and determining the optimal time for transplantation is a critical factor in their success. Because the liver is so important in permitting normal growth and development, failure to grow normally is often the earliest sign of the need for transplantation. This can occur in the first six months of life. "If a child exhibits failure to thrive, recurrent infection, and ascites (fluid in the abdomen), it is time to consider transplantation as soon as possible," says Dr. Jan.
Most transplants take place within the first three years of a child's life. At NewYork-Presbyterian/Columbia, Jean C. Emond, MD, Vice Chair and Director of Transplantation, is considered the world's leader in liver transplantation in children. Together, Dr. Jan and Dr. Emond have 50 years of experience in liver transplantation, and they continue to develop innovative solutions to the challenges presented by complex liver and intestinal diseases.
About half of babies receive living donor organs from a relative; the remainder receive organs from deceased donors. Because of expert management before and after transplantation, and a low surgical complication rate, children receiving liver transplants at Columbia have a 10% higher chance of long-term survival than the national average.
Babies with biliary atresia represent about 60% of those requiring liver transplantation at the center. The next largest group of chronic liver diseases, comprising about 15% of patients, require liver transplantation due to autoimmune liver disease, in which dysregulation of the immune system causes rejection of the liver. About 80% of patients with autoimmune liver disease can be treated with medications, but the other 20% may ultimately require organ transplants, according to Dr. Jan. This group, primarily teenagers, benefits heavily from the expertise of the center's noted liver pathologist, Jay Lefkowitch, MD.
Another 15-20% of patients require liver transplantation after acute liver failure following viral infection, hepatitis B or C, toxicity from acetaminophen or another substance, or other unknown causes. "Sometimes the liver can recover in a few days, and transplantation is not necessary. But patients must be in an intensive care unit in a transplant center, and they absolutely must be in a center with considerable experience and expertise in managing liver failure, so that any progression toward liver failure can be recognized early," says Dr. Lobritto. "We strive to help patients recover with regeneration of their own native liver. If patients are not managed properly at the outset, then we don't have that opportunity."
An even smaller group of patients requires surgery to remove small or large tumors of the liver. "Many of these are considered inoperable by other centers," says Dr. Lobritto, "but we operate on many children that other centers reject." In short, he states, "Transplantation is not the only choice. We treat all aspects of liver disease, and we do everything we can to avoid transplantation when appropriate."
According to Dr. Jan, if a child suffers from a serious liver disorder, treatment by a multidisciplinary center with vast medical and surgical experience can make the difference between an excellent or poor quality of life, or between life and death. NewYork-Presbyterian's outcomes far surpass the national average, with 92% of children surviving at one year, 89% surviving at two years, and 88% surviving at three years after liver transplantation. But short-term survival is not the goal, says Dr. Lobritto. "If a child lives only one or two years, that is not a successful transplant. Our goal is for every child to live a normal life well into adulthood."
