Design and validation of the biliary atresia research consortium histologic assessment system for cholestasis in infancy.
Russo P, Magee JC, Boitnott J, Bove KE, Raghunathan T, Finegold M, Haas J, Jaffe R, Kim GE, Magid M, Melin-Aldana H, White F, Whitington PF, Sokol RJ; Biliary Atresia Research Consortium. Clin Gastroenterol Hepatol. 2011 Apr;9(4):357-362. (CUMC Full Text PDF)
In this study, BARC researchers evaluated 97 liver biopsy specimens from 49 cases of BA, 17 cases of idiopathic neonatal hepatitis, and 31 cases of other neonatal cholestatic diseases (including TPN-induced liver disease, alpha1-antitrypsin deficiency, Alagille's syndrome, PFIC and others). Numerous important observations were reported in this study that are of practical utility for those who evaluate liver biopsies from cholestatic newborns. The overall sensitivity for the diagnosis of BA (including the "favor BA" and "favor obstruction other than BA" groups) was 86% (among 464 biopsies from 49 patients) . Inadequate samples (small and fragmented specimens) and very early biopsies (one patient was biopsied at 2-weeks after birth, an age at which the features of BA may not be sufficiently developed for diagnosis) were identified as causes for false negative interpretations. The investigators, therefore, suggest that samples should be at least 2 cm long, 0.2 cm wide, and contain at least 10 portal tracts to be considered adequate. Among patients with idiopathic neonatal hepatitis, on the other hand, 21% of biopsies (among 156 samples from 17 patients) were incorrectly interpreted as either "favor BA" or "favor obstruction other than BA". The error rate, therefore is was approximately 14% for BA cases and 21% fo INH cases. Biopsies from the third category of patients ("other") were classified as BA or "obstruction other than BA" 50% of the time, including nearly all cases of TPN liver disease and alpha1-antitrypsin deficiency.
Among the 16 individual histologic features investigated, the most useful ones in distinguishing BA from non-BA cases on logistic regression were 1- ductular reaction, 2- portal fibrosis, and 3- lack of sinusoidal fibrosis. Bile duct and canalicular cholestasis and portal edema were also seen more commonly in BA. Interestingly, several features considered useful by many pathologists in this setting, including hepatocellular changes (swelling, necrosis) giant-cell transformation/multinucleation, pseudorosette formation, and EMH were seen as frequently in BA as in non-BA cases.

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