Reversal of intestinal failure-associated liver disease in infants and children on parenteral nutrition: experience with 93 patients at a referral center for intestinal rehabilitation.

PURPOSE: Intestinal failure (IF)-associated liver disease (IFALD) complicates the treatment of children with IF receiving parenteral nutrition (PN). We hypothesized that prevention or resolution of IFALD was possible in most children and that this would result in improved outcomes. METHODS: We reviewed prospectively gathered data on all children referred to the intestinal rehabilitation and transplantation center at our institution. Total bilirubin level (TB) was used as the marker for IFALD. Patients were grouped based on TB at referral and at subsequent inpatient stays and outpatient visits. Standard treatment consisted of cycling of PN, limiting lipid infusion, enteral stimulation, use of ursodeoxycholic acid, and surgical intervention when necessary. Outcomes such as mortality, dependence on PN, and need for transplantation were assessed. Statistical analyses were performed using Fisher's exact, Mann-Whitney U, and Wilcoxon signed rank tests. RESULTS: Ninety-three patients with intestinal failure and on PN were treated at our center from 2003 to 2009. Median age at referral was 5 months (0.5-264 months). Prematurity was a complicating factor in 63 patients and necrotizing enterocolitis was the most common diagnosis. Eighty-two children had short bowel syndrome, whereas the remaining 11 had extensive motility disorders. 97% of children required significant alteration of their PN administration. At referral, 76 of 93 children had TB 2.0 mg/dL or higher, and 17 had TB below 2.0 mg/dL. TB normalized in 57 of 76 children with elevated TB at referral, and TB remained elevated in 19. Normalization of TB was associated with a mortality of 5.2%, and transplantation was needed in 5.2%. Conversely, when TB remained elevated, mortality was 58% (P = .0002 vs TB normalized), and transplantation occurred in 58% owing to failure of surgical and medical rehabilitation. CONCLUSIONS: Most children referred for treatment of IF have IFALD. A dedicated IF rehabilitation program can reverse IFALD in many children, and this is associated with improved outcome.

Timing of liver transplantation in biliary atresia-results in 71 children managed by a multidisciplinary team.

BACKGROUND: Kasai portoenterostomy (KP) remains the initial surgical therapy for biliary atresia (BA). Liver transplantation (LTx) is offered after a failed KP or if KP is not feasible. The timing of LTx in these children is not well established. We attempted to define factors that may help choose the optimal timing for LTx in children with BA managed by a multidisciplinary team including a pediatric surgeon, hepatologist, and liver transplant surgeon. METHODS: Records of children who underwent LTx for BA at our institution between January 1998 and December 2006 were reviewed. Clinical data such as pre-LTx pediatric end-stage liver disease (PELD) score, location of KP, and outcome were evaluated. RESULTS: Seventy one children underwent 77 liver transplants for BA at an average age of 25 months (range, 3-216 months). Sixty-one had a previous KP, 30 at our institution. Ten had LTx without KP. The overall patient survival was 94.4% and overall graft survival was 87% at median follow-up of 58 months (range, 6-111 months). Four patients died, 1 because of vascular thrombosis despite repeat LTx, 1 because of fungal infection after LTx, and 2 because of causes unrelated to LTx. Six children required retransplantation. Living donor liver transplantation was performed in 32 of these children with 91% patient and graft survival. Fifty-three children had a PELD score of 10 or higher with patient and graft survivals of 92% and 86%, respectively. Eighteen children had a PELD score of less than 10 with patient and graft survivals of 100%. For the 30 children who underwent KP at our institution, the median age at LTx was 9 months (range, 3-168 months), and patient and graft survival were both 93%. CONCLUSIONS: Outcome of LTx for BA is excellent. Children with higher PELD scores (>/=10) at LTx may have worse outcome. Children with a PELD score of less than 10 survived with their original grafts. In children with BA, the PELD score should be monitored and may help stratify patients for eventual LTx. When a child with BA is deemed a candidate for LTx, the PELD score should be determined. A PELD score that approaches 10 should trigger discussion of LTx and living donor liver transplantation with the family.