Pediatric Liver Transplant Conditions We Treat

Liver damage in children happens for a variety of reasons. Some children are born with a liver condition; others acquire liver disease after birth. At Lucile Packard Children’s Hospital Stanford, we perform transplants on children for the following conditions.

• Pediatric acute liver failure
• Pediatric cholestatic diseases
• Pediatric hepatitis
• Pediatric metabolic liver diseases
• Pediatric liver tumors
• Other liver diseases

Pediatric acute liver failure

Acute liver failure damages a large portion of the liver quickly, causing cells to die. The cause of acute liver failure is unknown in nearly 50% of cases. However, a dysregulated immune system is suspected in most of these cases. The following are known reasons for acute liver failure in children, listed in order of frequency:

• Autoimmune hepatitis and immune deficiencies
• Viral infections, such as hepatitis, herpes simplex, and Epstein-Barr
• Excess acetaminophen or another drug or toxin
• Conditions that disrupt metabolism (how bodies build up or break down substances), such as Wilson’s disease and Reye’s syndrome

Pediatric cholestatic diseases

One of the main liver functions is to produce bile, a fluid that helps digest food and carry waste to the small intestine. Cholestatic liver diseases can cause problems with bile production or flow. Symptoms vary but can include jaundice (yellowing of the skin and eyes), impaired growth, fatigue, and itching.

• Biliary atresia: A rare liver disease in infants that blocks bile ducts and is the most common reason for liver transplant in children, accounting for over one-quarter of the transplants we perform.
• Alagille syndrome: A genetic disorder that affects the heart, the liver, and other parts of the body. In the liver, bile ducts do not form well or become narrow, causing bile to back up. We partner with Lucile Packard Children’s Hospital Stanford’s Alagille Syndrome Center to provide multidisciplinary, holistic care for your child.
• Progressive familial Intrahepatic cholestasis (PFIC): A group of diseases that run in families where the liver cannot make and release bile.
• Sclerosing cholangitis: An inflammation of the bile ducts, causing them to harden and not function.

Pediatric hepatitis

Hepatitis simply means an inflamed liver. If not resolved, hepatitis can damage and destroy liver cells. Viral infections are the most common cause of hepatitis, but it can also be caused by an autoimmune response. Symptoms include jaundice, fever, flulike symptoms, nausea, poor appetite, malaise, and more. Patients can receive treatment for Hepatitis B and C from our Gastroenterology, Hepatology and Nutrition services.

• Autoimmune hepatitis: With this disease, a child’s body attacks its own liver, causing it to swell and be damaged.
• Neonatal hepatitis: This type of hepatitis is identified in the first few months after birth by jaundice, low weight, and slow growth. The liver becomes inflamed, making it hard for an infant’s body to absorb vitamins and nutrients.
• Hepatitis B: This common type of hepatitis destroys liver cells and can be passed from a mother to a baby during pregnancy, or when a child comes in contact with the blood of someone with the disease
• Hepatitis C: Similar to hepatitis B, this infection is passed to a child by blood exchange at birth or the blood of another person who has the disease.

Pediatric metabolic liver diseases

• Alpha-1 antitrypsin deficiency: This genetic disorder affects a protein produced by the liver that protects the lungs. The liver can’t properly release the protein, and it gets stuck in the liver, causing liver damage or liver cancer.
• Cystic fibrosis (CF): A progressive genetic disease that causes ongoing lung infections, making it hard to breathe. CF changes the texture of the bile—the fluid that passes from the liver to the intestine. With CF, bile sticks in the liver’s bile ducts, damaging the liver.
• Glycogen storage disease: A rare condition that affects how the liver stores and uses glycogen, a form of sugar. A missing enzyme causes glycogen to stay in the liver and cause damage.
• Maple syrup urine disease: Named for making urine smell sweet, this inherited disease causes the body to be unable to process amino acids, the building blocks of protein.
• Methylmalonic acidemia: An inherited disorder that leaves the body unable to break down certain fats and proteins, causing a buildup of methylmalonic acid, which can result in an enlarged liver.
• Mitochondrial function defects: Inherited conditions that affect the mitochondria of the cells—the part of the cells that converts food into energy—causing the liver to function improperly.
• Propionic acidemia: An inherited disorder that leaves the body incapable of processing certain parts of fats and proteins, resulting in a buildup of an organic acid.
• Urea cycle defect: With this rare disease, a missing enzyme allows ammonia to build up in the body, causing liver damage.
• Wilson’s disease: A rare genetic disorder that makes it impossible for a child to excrete copper, allowing it to build up in the body and damage the liver.

Pediatric liver tumors

Liver tumors are growths that are benign (harmless) or malignant (cancerous). Among the symptoms are a noticeable mass or swelling in the abdomen, low appetite, weight loss, jaundice, and itchy skin. Some malignant liver tumors, depending on certain factors, are best cured by a liver transplant.

• Hemangioendothelioma: This rare disease includes both benign and malignant tumors caused by blood vessel cells in the liver that don’t grow correctly.
• Hepatoblastoma: The most common early childhood liver cancer. It’s caused by a changed gene and usually occurs in children aged 3 and under.
• Hepatocellular carcinoma: A rare, malignant tumor that often spreads to other places in the body and is usually seen in younger teens ages 10 to 14.
• Sarcoma: A childhood cancer that starts in soft tissue or bones and tends to occur in children ages 6 to 10.

Other liver diseases

• Budd-Chiari syndrome: A rare disease, especially in children, where blood clots occur in the veins of the liver.
• Caroli’s disease: With this rare genetic disease that occurs more frequently in children of Asian descent, bile ducts become widened and don’t function properly.
• Cryptogenic cirrhosis: This is the final stage of chronic liver disease. The cause of the cirrhosis, or scar tissue, is unknown, but it causes the liver to fail.
• Hyperalimentation induced liver disease: Also known as parenteral nutrition–associated liver disease, this injury to the liver occurs in children who need special parenteral nutrition (PN) delivered through an IV or port.
• Neonatal hemochromatosis: Fetal liver injury, as a likely result of gestational alloimmune liver disease (GALD), which can result in liver failure.