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Transplantation of liver cells may be a promising treatment for Crigler-Najjar syndrome type 1. This disease is caused by a defect in the enzyme that metabolizes bilirubin, a chemical produced by the body. Researchers transplanted liver cells taken from a deceased boy into a 10-year-old girl with this disorder. About one month after the transplant, the level of bilirubin in her blood dropped to half its pre-transplant level. She also had enzyme activity and evidence that the bilirubin was being metabolized. Her transplanted cells lasted for more than 11 months.

Intestinal transplantation, in some cases combined with liver transplantation, may be an effective means of treating children with gastrointestinal disease who need an alternative to long-term intravenous feeding. Researchers performed 16 intestinal transplants, 13 of which were combined with liver transplants due to liver damage caused by intravenous feeding. Most children treated were under 5 years old, and most suffered from short bowel syndrome. The survival rate in the liver and intestine transplant recipients was 76% after an average follow up of 263 days, and 6 of the children no longer needed total intravenous feeding. Graft survival in these children was 61%. All three intestinal transplant recipients were alive after an average follow up of 384 days, their grafts were not removed, and they no longer needed intravenous feeding. The most significant complications were from infections and graft rejection., Objective. This report discusses the preliminary experience with intestinal transplantation in children at the University of Nebraska Medical Center. Patients. During the past 4 years, 16 intestinal transplants have been performed in infants and children. Thirteen have been combined liver and bowel transplants, and the remainder were isolated intestinal transplants. Nearly half of the patients were younger than I year of age at the time of surgery, and the vast majority were younger than 5 years of age. All but one had short bowel syndrome. Results. The 1-year actuarial patient and graft survival rates for recipients of liver and small bowel transplants were 76% and 61%, respectively. Eight of 13 patients who received liver and small bowel transplants remain alive at the time of this writing, with a mean length of follow-up of 263 (range, 7 to 1223) days. Six patients are currently free of total parenteral nutrition. All three patients receiving isolated intestinal transplants are alive and free of parenteral nutrition. The mean length of follow-up is 384 (range, 330 to 450) days. Major complications have included severe infections and rejection. Lymphoproliferative disease, graft-versus-host disease, and chylous ascites have not been major problems. Conclusions. Although intestinal transplantation is in its infancy, these preliminary results suggest combined liver and bowel transplants and isolated intestinal transplantation may be viable options for some patients with intestinal failure caused by short bowel syndrome or other gastrointestinal disease in whom long-term total parenteral nutrition is not an attractive option. Pediatrics 1996;97:443-448; short bowel syndrome, intestinal transplantation., Long-term home parenteral nutrition has been the mainstay of therapy for patients with intestinal failure caused by short bowel syndrome and other chronic intestinal disorders. Although this form of therapy [...]

Interwoven among liver cells are canals that carry bile from the liver, where the bile is produced, to the bile ducts and gallbladder where the bile is stored. Atresia (the absence or closure) of bile canals and ducts may occur within the liver or extrahepatically, outside liver. Extrahepatic biliary atresia (EHBA) occurs in 1 of 10,000 live births, has several causes, and is part of a disease continuum that includes neonatal hepatitis. Cytomegalovirus (CMV) infection is a common congenital (at birth) viral infection, which is usually asymptomatic. This article describes twins infected with CMV in utero, one of whom developed neonatal hepatitis, while the other developed EHBA following birth. The twins were not identical, and differences in immune-related cells likely contributed to the different expression of liver disease in the two infants. The article supports an association between CMV infection and liver disease, but does not prove that the former caused the latter. The first twin had hyperbilirubinemia, high levels of bilirubin resulting in yellow skin color (jaundice), the second day after birth, and studies confirmed hepatitis with inflammation and fibrosis of some liver cells. The patient was normal at one year of age. The second twin required surgical repair of birth defects on the first day of life, and developed hyperbilirubinemia on the second day. Studies showed a normal gall bladder and normal bile ducts, but inflammation of liver cells. However, by day 27, there was poor bile excretion and the extrahepatic bile structures were found to be obliterated. Cholestasis, halted bile flow, resulted in accumulation of bile within liver cells. The patient continued to have high blood bilirubin levels at two months, and at four months had a CMV-related severe lung inflammation. The patient received a liver transplant at two years due to end-stage liver disease. Findings from these cases are discussed. (Consumer Summary produced by Reliance Medical Information, Inc.)

Background: This study evaluated the safety and efficacy of teduglutide in pediatric patients with short bowel syndrome-associated intestinal failure (SBS-IF).Methods: A 24-week, phase III trial with 2 randomized, double-blind teduglutide dose groups and a nonblinded standard of care (SOC) arm was used; patients received 0.025 mg/kg or 0.05 mg/kg teduglutide once daily. Safety end points included treatment-emergent adverse events (TEAEs) and growth parameters. The primary efficacy/pharmacodynamic end point was the number of patients who achieved a ≥20% reduction in parenteral support (PS) from baseline at week 24.Results: All 59 enrolled patients completed the study (0.025 mg/kg, n = 24; 0.05 mg/kg, n = 26; SOC, n = 9). Baseline demographics and disease characteristics were comparable among groups. TEAEs were reported by 98% and 100% of patients in the teduglutide and SOC groups, respectively. The most common TEAEs in the teduglutide-treated groups were pyrexia and vomiting. The primary end point was achieved by 13 (54.2%), 18 (69.2%), and 1 (11.1%) patients who received 0.025 mg/kg teduglutide, 0.05 mg/kg teduglutide, and SOC, respectively (P < 0.05 vs SOC). Both 0.025-mg/kg and 0.05-mg/kg teduglutide groups showed clinically significant reductions in PS volume (P < 0.05 vs SOC), PS calories, days per week and hours per day of PS infusions, and increases in enteral nutrition and plasma citrulline at week 24 compared with baseline. Two (8.3%, 0.025 mg/kg teduglutide) and 3 patients (11.5%, 0.05 mg/kg teduglutide) achieved enteral autonomy.Conclusion: The safety profile of teduglutide was similar to that reported previously in children and adults. Treatment with teduglutide was associated with significant reductions in PS for pediatric patients with SBS-IF over 24 weeks. [ABSTRACT FROM AUTHOR]