Objectives: Progressive familial intrahepatic cholestasis type 1 (PFIC-1), an autosomal recessive disorder, is characterized by cholestasis, jaundice, and refractory pruritus. In some patients with PFIC-1, liver cirrhosis and end-stage liver disease develop and lead to liver transplantation (LT). In this observational study, we sought to clarify the long-term outcomes of LT for PFIC-1 and predictors of favorable outcomes.
Methods: The study cohort comprised 12 patients with PFIC-1 who had undergone living donor liver transplantation (LDLT) during the previous three decades (1990-2019). We compared the clinical manifestations and type of ATP8B1 mutations between patients in whom LDLT had been successful and those in whom it had been unsuccessful.
Results: LDLT failed in 5 of the 12 patients and the 25-year survival rate was 58%. Comparison of physical growth after LDLT revealed significant retardation of stature in patients in whom LDLT had been unsuccessful; these patients developed severe and persistent diarrhea. ATP8B1 genotypic analysis revealed t
Objectives: Paediatric studies on the role of antibiotic prophylaxis in the prevention of postoperative infections in children undergoing percutaneous endoscopic gastrostomy (PEG) are lacking. The aim of this study was to assess if a single dose of co-amoxiclav before PEG can decrease the rate of peristomal wound and systemic infection in children.
Methods: In this prospective, randomised, double blind, multicenter trial, children undergoing PEG were randomized to antibiotic prophylaxis with co-amoxiclav versus placebo and the rate of local and systemic infections were assessed.
Results: Of the 106 patients considered for inclusion, 49 patients were randomized. In the per protocol analysis, the occurrence of wound infection was 5% (1/20) in the antibiotic group and 21% (4/19) in the placebo group [p = 0.13, 16% difference in proportions, OR 0.19, 95% CI 0.02-1.9]. The occurrence of systemic infection was 9% (2/22) in the antibiotic group and 27.2% (6/25) in the placebo group [p = 0.17, 18% difference in proportions, OR 0.32, 95% CI 0.06-1.80%]. Similar results were obtained in intention to treat analysis. Interestingly, the overall infection rate was significantly higher in the placebo group as compared to the antibiotic group (40% vs 13,6%; p = 0.04) and the duration of hospital stay was significantly longer in the placebo group as compared to the antibiotic group (4.4 ± 1.6 vs 3.5 ± 1.05; p = 0.02). The number-needed-to-treat (NTT) to prevent one peristomal infection on average are 6.7 patients.
Conclusions: A preoperative dose of co-amoxiclav reduces the overall infection rate and the duration of hospital stay. Our data suggest that antibiotic prophylaxis should be recommended in every children undergoing PEG placement.
hat frameshifting, splicing, and large deletion mutations occurred more commonly in successful cases, whereas missense mutations occurred more frequently in unsuccessful cases. No mutations were identical in the two groups.
Conclusions: These results suggest an association between post-LT outcomes and extrahepatic manifestations, especially intestinal function. Further investigation of correlations between ATP8B1 genotypes and intestinal function could help to identify patients with PFIC-1 who will achieve favorable post-LT outcomes.
DOI: 10.1097/MPG.0000000000002981
