Excellent outcomes over the last 3 decades have made liver transplantation the treatment of choice for many advanced liver disorders. This success also opened liver transplantation to new indications such as liver tumors and metabolic disorders. The emergence of such new indications for liver transplantation is bringing a new stream of patients along with disease-specific challenges. The cumulative number of liver transplant recipients is peaking, requiring novel systems of health care delivery that meet the needs of this special patient population. This article reviews updates and new development in pediatric liver transplantation.
Key points
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Liver transplantation is the standard of treatment for many end-stage pediatric liver disorders.
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Long-term outcomes are excellent for both patient and graft survival.
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Active research is ongoing to optimize immunosuppression protocols to decrease medications side effects and foster immune tolerance.
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Long-term compliance hurdles and transition to adult care are emerging as the new frontiers of pediatric liver transplantation.
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The growing population of pediatric liver transplant recipients begs for new health system streams that meet the complexity of the needed care.
Evolving trends in liver transplantation
Excellent outcomes over the last 3 decades have made liver transplantation the treatment of choice for many advanced liver disorders. This success also opened liver transplantation to new indications such as liver tumors, metabolic disorders, postoncologic liver disease, and cystic fibrosis liver disease. The emergence of such new indications for liver transplantation is bringing on a new stream of patients along with their disease-specific challenges.
The timing of referral to transplantation remains crucial to assure timely access to organs and an optimized postoperative survival. Disparities in organ demand and supply persist, resulting in a growing number of pediatric patients on the transplant wait list. A change in national allocation priority helped decrease mortality for those on the pediatric wait list; still, many pediatric patients require exception points to receive a liver transplant offer in a timely fashion. The number of liver transplant recipients is peaking, requiring novel systems of health care delivery that meet the needs of this special patient population.
Evolving trends in liver transplantation
Excellent outcomes over the last 3 decades have made liver transplantation the treatment of choice for many advanced liver disorders. This success also opened liver transplantation to new indications such as liver tumors, metabolic disorders, postoncologic liver disease, and cystic fibrosis liver disease. The emergence of such new indications for liver transplantation is bringing on a new stream of patients along with their disease-specific challenges.
The timing of referral to transplantation remains crucial to assure timely access to organs and an optimized postoperative survival. Disparities in organ demand and supply persist, resulting in a growing number of pediatric patients on the transplant wait list. A change in national allocation priority helped decrease mortality for those on the pediatric wait list; still, many pediatric patients require exception points to receive a liver transplant offer in a timely fashion. The number of liver transplant recipients is peaking, requiring novel systems of health care delivery that meet the needs of this special patient population.
Liver transplantation indications and epidemiology
Liver Transplantation Indications
The classical indication for liver transplantation is liver failure causing a life-threatening situation resulting in a mortality risk greater than 90% at 1 year. With improving diagnostic tools and treatment support for end-stage liver disorders, the historical indications for liver transplantation are changing. End-stage liver disease from biliary atresia remains the most common cause of liver disease leading to transplantation. With a growing obesity epidemic, nonalcoholic fatty liver disease is becoming one of the leading causes of liver transplantation in adults and young adolescents. Improving liver transplantation outcomes is allowing consideration of transplantation for patients with metabolic disorders in whom liver transplantation proves to be a reliable treatment modality improving their overall outcomes. Indeed, liver transplantation is now indicated in those with metabolic disorders, in whom a healthy liver can bring enough enzymatic activity to stabilize a metabolic disease like seen in some urea cycle defects and organic acidurias. Finally, advances in oncologic care are allowing hepatoblastomas and hepatocellular carcinomas to be considered for primary transplantation in case they present as unresectable liver tumors.
Pediatric transplants account for about 7% to 8% of total number of liver transplants performed in the country and remain relatively steady at about 500 cases a year. Most transplants are done at less than 2 years of age; another pediatric transplantation peak age is seen in adolescence ( Fig. 1 ).
Contraindications to Liver Transplantation
With improved supportive measures and medical expertise, relative contraindications to liver transplantation related to comorbidities are decreasing. Some absolute contraindications, however, remain and consist mostly in systemic disorders that cannot be cured or can be worsened by transplantation, such as ongoing infections, cancers, severe metabolic disorders, or in advanced cardiopulmonary diseases such as seen in severe pulmonary disease or heart failure.
Referral and transplantation evaluation
Referral
A timely referral to transplant evaluation is a crucial step to assure good success in both early and long-term phases. Such a referral does not necessarily always lead to transplantation, rather it empowers the transplant team to rule out any treatable causes that can avoid transplantation and to put in place a detailed and supportive care plan in case transplant is needed.
It is imperative to refer a patient to the transplant center as soon as acute liver failure or an irreversible liver disease is diagnosed. The healthier a patient is at presentation to the transplant center, the better are the chances to optimize their status and get them if needed, to transplantation in a timely fashion.
Transplantation Evaluation and Wait List
The transplant evaluation is carried out by a multidisciplinary team that includes the primary care physician, primary pediatric gastrointestinal doctor, and the pediatric liver transplant team. The goal is to complete liver disease diagnosis; treat all possible causes of liver failure; rule out any absolute indications to transplantation; palliate to any treatable comorbidities; optimize vaccination, nutrition, and education; and define and foster family and patient education and psychosocial and economic support. A complex care plan is created at the end of the evaluation that puts in motion all that is needed to optimize short- and long-term transplantation outcomes. In some instances, such an evaluation identifies a treatable condition and helps avoid transplantation ( Box 1 ).
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Define indications for transplant.
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Define best timing for listing.
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Identify specialized treatments that can avoid or delay the need for transplantation.
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Plan for care and support needed to minimize risk at transplantation.
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Anticipate long-term outcomes and support needed to optimize them.
Once a patient is on the transplant list, close follow-up is carried out in the transplant center along strict pretransplant protocols. This follow-up allows fine tuning of the care and optimizing of health in anticipation of transplantation. The emphasis in this period is on anticipating complications and on prevention and early treatment. Although it is often not possible to reverse deterioration, every effort is placed to get these children as healthy as possible to transplantation. In addition, this approach allows active wait list management that facilitates a timely transplantation ( Box 2 ).
