Liver Transplant for Children with Metabolic Disorders

Some children are born with genetic metabolic disorders. These conditions are caused by changes in genes that affect how the body uses food for energy. The genetic changes can be inherited from one parent or from both parents, depending on the condition. Many of these disorders affect the liver, which plays an important role in maintaining overall health.

When the liver does not function properly, toxic substances can build up in the body. This can lead to serious illness, especially during infections or times when a child cannot eat. Families work extremely hard to manage these conditions with very strict diets, special medical formulas, and costly medications and supplements. Even with excellent care at home, some children continue to have frequent medical emergencies.

For selected patients, a liver transplant can offer a safer and more stable future, with better metabolic control, fewer medical emergencies, and for some children, the ability to relax dietary restrictions or discontinue certain medications and supplements.

Daily Life for Families

Living with a metabolic disorder can be demanding and overwhelming. Many children must follow a carefully timed eating schedule with specialized diet, including during overnight hours. Special medical formulas are often required, but these formulas can have strong or unpleasant tastes that make them hard for children to drink. Some children need feeding tubes for short- or long-term use to ensure they receive appropriate nutrition and medications.

Despite careful planning and constant attention, hospital stays may still occur, often frequently, even when families do everything right. These repeated hospitalizations can be physically and mentally exhausting for both children and their caregivers.

Disorders We Support at Nationwide Children’s Hospital

Urea Cycle Disorders
Urea cycle disorders prevent the body from removing ammonia, a waste product that can be very harmful to the brain. High ammonia levels can be life threatening. Liver transplant can provide a working urea cycle and help prevent future ammonia crises. After liver transplant, patients no longer experience metabolic crises and can eat a typical diet.

Maple Syrup Urine Disease
Maple syrup urine disease affects how the body breaks down certain amino acids. Children often depend on very strict diets with special formula and supplements. After liver transplant, patients no longer experience metabolic crises and can eat a typical diet.

Organic Acidemias
Organic acidemias, such as propionic acidemia or methylmalonic acidemia, cause harmful acids to build up in the blood. Even mild illness can trigger repeated hospital stays. While liver transplant does not cure these disorders, it can significantly reduce the frequency and severity of metabolic emergencies.

Glycogen Storage Disease
Glycogen storage diseases affect how the body stores and releases sugar for energy. Some types can lead to liver failure or the development of liver tumors. For selected patients, liver transplant can improve blood sugar control and prevent or treat serious liver complications.

Our Team and Our Commitment to Families

Caring for children with metabolic disorders requires far more than surgery alone. Our program brings together an experienced, multidisciplinary team of metabolic geneticists, transplant surgeons, anesthesiologists, hepatologists, nurses, dietitians, social workers, psychologists and transplant coordinators. This team works closely with families from early evaluation through long-term follow up.

We carefully consider whether liver transplant is the right option for each child and take time to ensure families understand every step of the process and every decision involved. For some children, transplant can mean fewer medical emergencies, fewer hospital stays, and a more predictable daily life. Families come to our program for experienced care, clear communication, and steady support as they make important decisions and plan for the future.