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.
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.