The Optimal Timing of Intestinal Transplantation for Children With Intestinal Failure

In Brief Objective: Identify an optimal approach to the timing of intestinal transplantation for children dependent on total parenteral nutrition (PN). Summary Background Data: Children with short bowel syndrome are frequently dependent on PN for growth and development. Intestinal transplantation is often considered after PN-related complications occur, but optimal timing of transplantation is controversial. Methods: A Markov analytic model was used to determine life expectancy (LY) and quality-adjusted life years on a theoretical cohort of 4-year-old subjects for two treatment strategies: (1) standard care consisting of PN and referral to transplantation according to accepted guidelines and (2) early listing for isolated small intestine transplantation. Results: Early listing for intestinal transplantation was associated with 0.27 additional life years (13.16 vs. 12.89) and 0.76 additional quality-adjusted life years (10.51 vs. 9.75) as compared with current standard care. The unadjusted analysis was sensitive to the development of PN-associated liver disease, at a threshold of approximately 11% per year, and its related probability of dying at a threshold of 80% 2-year mortality. Early listing for transplantation was the dominant strategy until the probability of late bowel rejection reached 35% per year. Conclusions: Children with short bowel syndrome dependent on PN should be considered for intestinal transplantation earlier than what is current practice. The timing of intestinal transplantation in patients with intestinal failure is controversial. In this study, a Markov model was developed to compare early listing for intestinal transplantation against standard care, consisting of parenteral nutrition (PN) and delayed transplantation if necessary. Intestinal transplantation has reached a state of clinical equipoise with PN, and quality of life should be considered in the timing decision for transplantation.