Pediatric Intestinal Failure in the Intestinal Transplant Era: A Retrospective Review of 272 Infants

Abstract

Background. Pediatric intestinal failure (IF) is a rare but devastating condition defined as the inability of the intestine to support growth and development without supplemental parenteral nutrition (PN). A retrospective study was performed to provide the foundation for the first prospective study of IF in North America. Methods. PIFCon includes 14 sites with multi-disciplinary IF management teams; 9 are also intestinal transplant (ITx) centers. Infants 60 continuous days for IF were included. Demographic, clinical, biochemical, nutritional and outcome data were abstracted from medical charts. Values are presented as median (25th, 75th percentiles) or as (n, %). Enteral autonomy (EA) was defined as the discontinuation of PN for > 3 consecutive mo. Results. 272 infants with a gestational age of 34 wks (30, 36) and birth weight of 2.1 kg (1.2, 2.7) were followed for 25.7 mo (11.2, 40.9). The majority were male (156, 57%) and Caucasian (204, 75%). Residual small bowel length in 144 patients was 41 cm (25.0, 65.5). Diagnoses were necrotizing enterocolitis (71, 26%), gastroschisis (44, 16%), intestinal atresia (27, 10%), volvulus (24, 9%), combinations of these diagnoses (46, 17%), intestinal aganglionosis (11, 4%), and other single or multiple diagnoses (48, 18%). The cumulative proportions achieving EA at 1 and 3 yr were 0.31 and 0.43, respectively. Commonly used medications were oral antibiotics (207, 76%), H2 blockers (187, 69%), and PPIs (156, 57%). Breast milk was given to 52 (19%); the cumulative probabilities of oral solid feeding were .67 at 1 yr and .85 at 3 yr. 25 different infant formulas were used as initial diet; 40 different formulas were used overall. 3.3 new catheter related blood stream infections occurred per person year (PPY) on PN and 0.2 intestinal bleeding episodes PPY of observation. 28 bowel lengthening procedures were performed. Cumulative probability of survival (CPS) was 0.84 at 1 yr and 0.74 at 3 yr. 58 died before and 10 died after ITx. Following study entry, time to death without ITx was 8.2 mo (4.8, 12.6). 137 were alive without ITx when the last data were abstracted from the medical record. 60 received ITx, the cumulative incidence of ITx at 1 and 3 years was .087 and .229, respectively. The CPS 1 year after ITx was 0.84. Conclusions. Children with IF suffer a high rate of mortality and morbidity. EA may require years to achieve. Substantial practice variability is noted among sites. Improved medical, nutritional, and surgical management of IF may reduce time on PN, mortality and need for ITx. Grant support: NIH/NIDDK R21 DK081059-01