Liver transplantation in children: Indications and outcomes

Historic overview

Thomas Starzl attempted the first human liver transplantation (LT) in 1963 in a 3-year-old child with biliary atresia (BA; see Chapter 125 ). Unfortunately, the child died in the operating room from uncontrollable hemorrhage. Just a few years later, however, Starzl successfully performed eight LTs in eight children. In contrast to his first attempt, all survived the operation and half survived for more than 1 year postoperatively. Since those heroic beginnings 5 decades ago, advances in immunosuppression, surgical technique, anesthesia, and critical care have made LT an effective, reliable, and lifesaving procedure that is performed every day at LT centers throughout the world. In children with end-stage liver disease (ESLD), LT has become the accepted therapy, and its use is limited only by the availability of suitable grafts.

In 2018, 8250 adult and 563 pediatric LTs were performed in the United States alone. Currently, approximately 13,046 registrations exist on the LT waiting list, and 1046 of the candidates are younger than 17 years of age. Potential recipients such as infants, toddlers, and young children are limited in graft choice partly because of the narrow options of available graft sizes as they face unique challenges unbeknownst to an adult population. Historically, the lack of size-matched organ availability, coupled with the disproportionate number of adults on the waiting list, has significantly disadvantaged children awaiting transplantation. This challenge still remains in the modern era with 30% of organs refused because of size mismatch. Recent evidence reported that nearly half of pediatric deceased-donor organs are allocated to adult patients. Despite this obvious disadvantage, children with ESLD can deteriorate quickly and cannot afford to spend too much time awaiting transplantation when compared with adults. An infant with decompensated liver disease from cirrhosis typically cannot survive on a waiting list for more than a few months or years. Currently, children aged 1 to 5 years make up the largest age group on the waiting list (31%) and patients younger than 1 year constitute the single age group majority (23%). In addition, the shortage of pediatric donors and size-matched organs contributes to longer waiting times, which translates to an increased mortality in children on the transplant waiting list. In the earlier days of LT, as many as 50% of children on the waiting list would die before they could receive a transplant. Encouragingly, pre-transplant mortality has decreased in all age groups and has since improved to a 10% mortality on the pediatric LT waiting list. ^, Despite this progress, the waiting list mortality for children younger than 1 year continues to exceed that of any other adult or pediatric age group, estimated at 17.1 deaths per 100 waitlist-years. ^{, ,}

Indications (see Chapter 105 )

Box 110.1 lists common indications for pediatric LT, largely falling into two categories: cholestatic and noncholestatic. The classic and most common indication for LT is still ESLD; however, the rates of multiorgan transplants inclusive of LT have also increased. Liver-kidney and liver-pancreas-intestine candidates accounted for 2.5% and 12.6% of pediatric LT candidates in 2018 (see Chapter 112 ).