Biliary atresia management and treatment options
The first-line treatment for biliary atresia, a disorder in which certain bile ducts outside the liver are blocked or absent, typically is a surgical procedure called a Kasai portoenterostomy, which aims to restore the normal flow of bile in the patient’s body.
In biliary atresia, the blocked or absent bile ducts prevent bile from flowing into the small intestine as it should. As such, the goal of biliary atresia treatment is to ensure bile flows properly through the system.
However, there are no specific biliary atresia medications, so surgery often is the only available course of action. Even with surgery, overall biliary disease management is needed, typically involving follow-up therapies, including nutritional support, aimed at keeping the liver healthy.
Outcomes from biliary atresia surgery are generally best when the Kasai procedure is done as early as possible, so an early diagnosis of the disease is key for optimal clinical results.
Following the Kasai procedure, biliary atresia management commonly involves supportive treatment for liver health. In the long term, however, most people with biliary atresia will develop liver damage to the point that a liver transplant becomes necessary.
Bile is a thick, sticky, greenish-yellow fluid that helps the body break down certain fats and proteins during digestion. It also carries certain waste products from the liver into the intestines to be excreted in feces.
Made in the liver, bile flows from the organ to the intestines through a series of tubes called bile ducts. These ducts also transport bile to and from the gallbladder, which stores bile until it’s needed for digestion.
Biliary atresia is characterized by bile duct blockage — either the bile ducts are pinched off or obstructed, or sections of these tubes may be missing entirely. The end result is that bile flow is reduced or even halted, a phenomenon known as cholestasis. As a consequence, bile doesn’t get into the intestines to help with digestion, and excess bile builds up in the liver and causes damage to the organ.
The main symptoms of bile duct blockage in biliary atresia are jaundice, marked by a yellowing of the skin and whites of the eyes, dark urine, and pale stools. The disease can be divided into two types based on when these symptoms become evident. Congenital or fetal biliary atresia is apparent from birth, while perinatal biliary atresia appears in the first weeks of life.
Although it can be fatal in early childhood without treatment, biliary atresia prognosis has improved dramatically with the use of the Kasai procedure and liver transplant. With these modern forms of medical management, most people born with biliary atresia will survive into adulthood, especially if infants are diagnosed and treated in the disease’s early stages. As in most diseases, longer-term outcomes typically are best for patients who are referred to a specialist before starting treatment.
Scientists also are continuously conducting biliary atresia research, aiming to refine these techniques and identify new interventions that can further improve longevity and quality of life for patients.
The Kasai procedure, also called Kasai portoenterostomy or hepatoportoenterostomy, is a surgical operation that aims to reestablish the flow of bile out of the liver and into the intestines.
It essentially works by taking a loop of intestine and attaching it directly to the liver, bypassing the gallbladder and the typical bile duct system. This provides a direct access point for bile to drain out of the liver and into the intestines.
Importantly, this surgery may not work if there are problems obstructing bile flow within the liver itself, as happens in some patients. But for the vast majority of children with biliary atresia, the procedure can successfully restore some amount of bile flow.
The success of the Kasai procedure may depend in part on the timing of surgery. As a general rule, outcomes are better when the procedure is done as early as possible; for example, a 2022 meta-analysis of 14 studies involving more than 3,200 children showed that those who underwent the procedure within the first two months of life lived significantly longer without needing a liver transplant than did children who had the surgery at later ages. Infants treated in the first three months of life also experienced earlier clearance of infant jaundice.
That analysis supports biliary atresia surgery being done as soon as possible once the condition is diagnosed. As the only way to definitively confirm a biliary atresia diagnosis is via surgical exploration, the Kasai procedure is often done in the same surgery where biliary atresia is definitively diagnosed.
The Kasai procedure is named for Morio Kasai, a Japanese surgeon who helped lead the development of the technique in the 1950s. In the decades since, a variety of modifications to the standard procedure have been created. There is no single gold standard for exactly how to do the surgery, so the exact steps taken will depend on the surgeon’s expertise as well as on the specific condition and symptoms of the infant.
Regardless of such factors, it is a major operation; babies typically need to stay in the hospital for at least a week following the Kasai procedure. Regular monitoring is done to check the health of the liver and address any complications of treatment.
During this time, babies often will be given supportive therapies that may include corticosteroids, prophylactic antibiotics, and intravenous (into-the-vein) nutrition to help keep them healthy and comfortable while they recover from the procedure.
Following the Kasai operation, it’s commonplace at many centers, especially in Europe and Japan, to treat children with ursodeoxycholic acid (UDCA), a synthetic bile acid that can help promote the flow of bile. UDCA is standard treatment for other bile duct disorders. Given that it can promote bile flow, there’s a theoretical basis that it could be helpful in biliary atresia, although there’s not much high-quality data on UDCA in this indication specifically. There also are no specific biliary atresia medications available, though many are under investigation for biliary atresia treatment.
Most people with biliary atresia will eventually require a liver transplant, in which a healthy liver from a donor is used to replace the patient’s diseased organ. The Kasai procedure can delay the need for liver transplant by years or even decades. But over time the liver acquires too much damage and starts to fail, necessitating transplant. In some cases where biliary atresia is diagnosed late or there’s already significant liver damage, transplant may be the first line of treatment.
It’s estimated that at least 1 in 3 children with biliary atresia will need a liver transplant before age 5, and roughly 80% will need a new liver by the time they reach age 20. The remaining 20% survive into adulthood with their native liver, although these patients usually still have liver problems that require medical management in adulthood; they also may eventually need a transplant.
In liver transplants for children, surgeons may use a whole liver from a deceased child donor, or they may use part of a healthy adult liver. The liver is one of the few organs in the body that can regenerate itself, so the transplanted liver usually is able to grow to support the needs of the child. In the U.S., there are more than 100 pediatric liver transplant centers where these procedures are performed.
When the decision is made to pursue a liver transplant, the patient is placed on a waiting list. Children with pediatric liver disease are ranked according to a score that determines how ill patients are and how urgently they’ll need a liver. When a liver becomes available, computers will automatically identify children who are a potential match, and the highest-ranked patient will be selected to receive the liver. However, because any given liver will only be a match for some children but not others, it’s never possible to say exactly what supposed place in line a child has.
It’s estimated that biliary atresia patients in the U.S. wait a median of 90 days, or about three months, before getting a transplant — but getting a new liver can take anywhere from days to years. While waiting for a liver transplant, it’s important for patients and their families to be proactive about creating a biliary atresia management plan to keep the individual as healthy as possible while waiting.
As with any major surgical procedure, a liver transplant carries risks, including the possibility that the surgery will not be successful or the organ will be rejected. But with modern techniques, estimated success rates for liver transplant in children with biliary atresia are upward of 90%.
Still, for patients who have received a liver transplant, lifelong monitoring will be required to make sure the transplanted organ is functioning appropriately. Individuals who’ve had a transplant also may be at higher risk of complications such as high blood pressure, high cholesterol, kidney problems, certain infections, some types of cancer, and heart problems.
Biliary atresia is defined by problems restricting bile flow into the intestines. Because bile is needed for digestion, children with biliary atresia often have difficulty getting all the nutrients they need from the food they eat. Because of that, management of biliary atresia commonly involves nutritional support, such as:
- supplements of fat-soluble vitamins, including vitamins A, D, E, and K
- supplements of medium-chain triglycerides, a kind of fat molecules naturally found in coconut and palm kernel oils, which can be used as an energy source but don’t require bile to be digested
- delivery of nutrients directly into the stomach via a tube inserted through the nose, called nasogastric feeding, or directly into the bloodstream, known as total parenteral nutrition.
People with biliary atresia have liver disease by definition, so it’s generally recommended that the patients avoid substances that can put a lot of stress on the liver. This includes alcohol and other recreational drugs, as well as high doses of some medications such as ibuprofen or aspirin.
Patients should talk with their healthcare provider about their specific situation to determine whether there are any other substances they should limit or avoid.
Although there hasn’t been a lot of formal research into quality of life for people with biliary atresia, the majority of studies suggest that patients tend to report life quality scores that are within normal ranges compared with their healthy peers. Studies also suggest that most adults with biliary atresia are able to successfully pursue education and career goals.
Living with biliary atresia can be daunting, but a number of support groups are available to help patients and caregivers connect with people facing similar challenges. Such groups allow members to share life experiences, give and receive support, and become more educated about their disease. Some groups are the Facebook-based Biliary Atresia Network, which is associated with the Children’s Liver Association for Support Services, known as C.L.A.S.S., and Life with Biliary Atresia and Beyond, from the American Liver Foundation.
People affected by biliary atresia also may find support in organizations such as the Childhood Liver Disease Research Network, Children’s Liver Disease Foundation, and Children’s Organ Transplant Association. Other local or online patient organizations also may provide community and connection, as well as resources for helping with disease management and dealing with the cost of treatment.
Liver Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
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