Liver transplantation in an infant with cerebrotendinous xanthomatosis, cholestasis, and rapid evolution of liver failure.
Andrea PietrobattistaMarco SpadaManila CandussoSara BoenziCarlo Dionisi-ViciPaola FrancalanciAmelia MorroneLorenzo FerriGiuseppe IndolfiEmanuele AgoliniGiuseppe GiordanoLidia MontiGiuseppe MaggioreA S KniselyPublished in: Pediatric transplantation (2022)
Bile acid synthesis disorders should be routinely included in the NC/"neonatal hepatitis" work-up. The mutually supportive triple approach of BA profiling, immunohistochemical study, and genetic analysis may optimally address diagnosis in CTX, a treatable disease with widely varying presentation.