A paradigm shift in the intention-to-transplant children with biliary atresia: Outcomes of 101 cases and a review of the literature

Pediatr Transplant. 2019 Nov;23(7):e13569. doi: 10.1111/petr.13569. Epub 2019 Aug 13.

Abstract

For children with BA who do not benefit from Kasai surgery, the only therapeutic option is liver replacement and transplantation. The very decision to proceed for transplantation is a crucial point in time because it is the first step toward the preparation for the transplantation. The former time point is defined in this analysis as "intent-to-transplant" care pathway. In the life of every BA candidate for liver replacement, this point in time varies and mostly depends on the decision of their primary caring teams-about when to switch from supportive care to transplant, and thus to refer to a transplant center. This intent-to-transplant analysis of a series of 101 consecutive infants that were referred to a single transplant team showed that excellent overall outcome (97% survival) has been achieved overall. However, three deaths occurred that were clearly related to a late referral. This analysis and recent observations from other centers strongly support that the timing for referring these children to a transplant center and/or deciding to list them on the waiting list is currently too late and should be anticipated to what it is currently. This paradigm shift in the intention-to-transplant children is likely necessary for giving a better chance to an increased number of children and impacts positively on the general outcome. Networking and defining new tools for a rapid recognition of the infants who need early transplantation are necessary; centralization of these children may be helpful to achieve better outcomes than currently observed.

Keywords: Kasai portoenterostomy; biliary atresia; centralization of care; children; innovative techniques; liver transplantation; living related donor; outcome research; referral practice; review.

Publication types

  • Review

MeSH terms

  • Adolescent
  • Adult
  • Biliary Atresia / surgery*
  • Biopsy
  • Child
  • Child, Preschool
  • Female
  • Humans
  • Infant
  • Intention to Treat Analysis
  • Italy
  • Likelihood Functions
  • Liver Transplantation*
  • Male
  • Middle Aged
  • Portoenterostomy, Hepatic
  • Retrospective Studies
  • Treatment Outcome
  • Young Adult