Literature DB >> 28318950

Cholesteryl Ester Storage Disease: An underdiagnosed cause of cirrhosis in adults.

Mamta Pant1, Kiyoko Oshima2.   

Abstract

Cholesteryl Ester Storage Disease (CESD), is a rare multisystem autosomal recessive disorder and belongs to the broad family of lysosomal storage disorders. It can present anytime from infancy and childhood to even adulthood. The clinical manifestations are generally severe in infants and with milder forms in adults. One of the prominent sites of involvement is liver. Due to low awareness of this condition among physicians including surgical pathologists, majority of the liver biopsies, especially from the adults are often misdiagnosed as non-alcoholic fatty liver disease/non-alcoholic steatohepatitis or cryptogenic cirrhosis. Given the recent availability of safe and effective enzyme replacement therapy that can alter the natural course of CESD, the pathologists signing out adult and pediatric liver biopsies should be aware of this entity, thus contributing to timely patient management. This review discusses the clinical features, pathogenesis, diagnostic approach, differential diagnosis and management of CESD in adults.
Copyright © 2017 Elsevier Inc. All rights reserved.

Entities:  

Keywords:  Cholesteryl Ester Storage Disease; Cirrhosis; Lysosomal acid lipase deficiency; Lysosomal storage disease; Wolman disease

Mesh:

Year:  2017        PMID: 28318950     DOI: 10.1016/j.anndiagpath.2017.02.005

Source DB:  PubMed          Journal:  Ann Diagn Pathol        ISSN: 1092-9134            Impact factor:   2.090


  4 in total

1.  Case Report of Gastrointestinal Bleeding in an Adult with Chronic Visceral Acid Sphingomyelinase Deficiency.

Authors:  David Cassiman; Louis Libbrecht; Wouter Meersseman; Alexander Wilmer
Journal:  Case Rep Gastrointest Med       Date:  2019-04-04

Review 2.  Reduced lysosomal acid lipase activity: A new marker of liver disease severity across the clinical continuum of non-alcoholic fatty liver disease?

Authors:  Francesco Baratta; Daniele Pastori; Domenico Ferro; Giovanna Carluccio; Giulia Tozzi; Francesco Angelico; Francesco Violi; Maria Del Ben
Journal:  World J Gastroenterol       Date:  2019-08-14       Impact factor: 5.742

3.  Carnitine palmitoyl transferase 1A deficiency in an adult with recurrent severe steato hepatitis aggravated by high pathologic or physiologic demands: A roller-coaster for internists.

Authors:  Prasit Phowthongkum; Kanya Suphapeetiporn; Vorasuk Shotelersuk
Journal:  Clin Mol Hepatol       Date:  2019-06-24

Review 4.  Lysosomal acid lipase deficiency in pediatric patients: a scoping review.

Authors:  Camila da Rosa Witeck; Anne Calbusch Schmitz; Júlia Meller Dias de Oliveira; André Luís Porporatti; Graziela De Luca Canto; Maria Marlene de Souza Pires
Journal:  J Pediatr (Rio J)       Date:  2021-05-06       Impact factor: 2.990

  4 in total

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