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Background: Little information is available on contemporary, prospectively collected data on the long-term outcome of national cohorts of children with biliary atresia., Objective: This study aimed to describe the current outcome of a national cohort of children with biliary atresia., Patients and Methods: All 93 cases of biliary atresia in the United Kingdom and Ireland diagnosed between March 1993 and February 1995 were followed up prospectively., Results: A total of 91 children underwent Kasai portoenterostomy in 15 individual centres. Only 2 centres treated more than 5 children annually. Median age at last follow-up was 12 years (range 0.25-14). Fifteen children (16%) have died: 10 after unsuccessful portoenterostomy, 1 of sepsis after successful portoenterostomy, and 4 after liver transplantation. Forty-two (45%) underwent liver transplantation at a median age of 1 year (range 0.5-9), with 90% survival. All 41 children with failed portoenterostomy (and 2 without portoenterostomy) died or underwent liver transplantation at a median age of 0.8 years (range 0.25-6.5). When the portoenterostomy was successful, 40 of 50 patients (80%) are alive without liver transplantation. The 13-year actuarial survival without liver transplantation is 43.8% overall and is better in children treated at centres that treat more than 5 cases yearly (54% vs 27.3%, P = 0.005)., Conclusions: If the portoenterostomy is successful, then few children with biliary atresia will need transplantation before adolescence. Children with biliary atresia should be treated in experienced centres to maximize the chance of successful surgery.

Described are six patients with Alpers syndrome from four unrelated families. Affected individuals harbored the following combinations of POLG mutations: 1) A467T/W1020X, 2) W748S-E1143G/G848S, 3) A467T/A467T, and 4) A467T/G848S. Homozygosity for the A467T allele in one patient was associated with a later age at onset. Mitochondrial respiratory chain studies in skeletal muscle were normal in each case. Nine combinations of mutant POLG alleles that cause Alpers syndrome are summarized.

Background: Adoptive immunotherapy with autologous and donor-derived cytotoxic T lymphocytes (CTL) has recently been used to treat Epstein Barr virus (EBV)-positive posttransplant lymphoproliferative disease (PTLD)., Methods and Results: We report complete regression of EBV-positive PTLD in an 18-month-old small bowel and liver transplant recipient after one infusion of partially human leukocyte antigen (HLA)-matched EBV-specific CTL grown ex vivo from an EBV seropositive unrelated blood donor. No infusion-related toxicity or evidence of graft-versus-host disease was observed. The tumor showed signs of regression within 1 week and EBV load in peripheral blood dropped to undetectable levels. Limiting dilution analyses (LDA) detected no EBV-specific CTL precursor (CTLp) cells before the infusion, and high numbers of CTLp at 4 hr and 24 hr post-CTL infusion. There was a reversal of the CD4/8 ratio in peripheral blood and an increase in HLA-DR positive CD8 cells. The patient has been in complete remission for 24 months., Conclusion: If this success is repeated in more PTLD patients, then stored CTL could be used for antiviral and antitumor therapies in immunocompromised patients.

Background: The critical shortage of size-matched donor organs for infants and small children in need of combined liver and intestinal transplantation has lead to long waiting times and a high risk of dying before transplantation. Utilizing grafts from larger donors could alleviate this problem, but using larger composite grafts in small children has been challenging and unsuccessful in the past., Methods: We conducted a pilot study for evaluating the results of transplanting into small recipients a composite graft (reduced-size liver and whole small bowel, including duodenum and pancreas head) procured from large donors. Liver size reduction was performed ex situ using the extrahilar approach, which leaves the liver hilum untouched. Straightforward implantation of the graft was performed by simple, two-step vascular anastomoses. The preservation of the donor duodenum in continuity with the combined graft avoided the need for biliary reconstruction, thus preserving maximal bowel length for gut continuity restoration in the recipient., Results: Two children, weighing 7.6 and 9.8 kg, respectively, underwent transplantation of a composite graft procured from donors weighing 35 kg. Their waiting time (68 and 97 days, respectively) was shorter compared with our previous experience with conventional techniques. Both are currently alive and well, at home and on full enteral feeds, 15 and 11 months after transplantation, respectively., Conclusion: This new technique has extended the range of possible donors for small candidates waiting for combined grafts and was successful in two patients. It should be considered for small recipients in the future.