title: Liver Transplantation in Patients With Niemann-Pick Disease--Single-Center Experience.
abstract: Niemann-Pick disease (NPD) is a rare syndrome caused by abnormal intracellular sphingomyelin lipid storage in cells known as "Pick cells." NPD can start in childhood or develop insidiously, with a predilection for reticuloendothelial cells and the nervous system. NPD is a heterogeneous clinical, and biomolecular disorder which has 6 variants. There is no curable treatment for NPD. Generally, the treatment for all types of Niemann-Pick disease is to support. Type B NPD (NPD-B) is mostly characterized by hepatosplenomegaly, which can evolve to hepatic cirrhosis. In patients who progress to liver failure, liver transplantation may be improve liver function. The Transplant Service of Hospital Universitário Walter Cantídio performed its first liver transplants in patients with NPD-B with good results, demonstrating the efficacy of this procedure in selected cases.
authors.affiliation: Department of Surgery, Liver Transplant Unit of Federal University of Ceara, Fortaleza, Brazil. Electronic address: gustavorcoelho@hotmail.com.
authors.forename: G R
authors.initials: GR
authors.lastname: Coelho
authors.affiliation: School of Medicine, Federal University of Ceará, Fortaleza, Brazil.
authors.forename: A M
authors.initials: AM
authors.lastname: Praciano
authors.affiliation: School of Medicine, Federal University of Ceará, Fortaleza, Brazil.
authors.forename: J P C
authors.initials: JP
authors.lastname: Rodrigues
authors.affiliation: Liver Transplant Unit of Federal University of Ceara, Fortaleza, Brazil.
authors.forename: C F G
authors.initials: CF
authors.lastname: Viana
authors.affiliation: Liver Transplant Unit of Federal University of Ceara, Fortaleza, Brazil.
authors.forename: K P
authors.initials: KP
authors.lastname: Brandão
authors.affiliation: Department of Pathology, Liver Transplant Unit of Federal University of Ceara, Fortaleza, Brazil.
authors.forename: J T
authors.initials: JT
authors.lastname: Valenca
authors.affiliation: Department of Surgery, Liver Transplant Unit of Federal University of Ceara, Fortaleza, Brazil.
authors.forename: J H P
authors.initials: JH
authors.lastname: Garcia
mesh_terms: D000328:Adult; D005260:Female; D006529:Hepatomegaly / Q000209:etiology / Q000601:surgery*; D006801:Humans; D017093:Liver Failure / Q000209:etiology / Q000601:surgery*; D016031:Liver Transplantation; D008297:Male; D052537:Niemann-Pick Disease, Type B / Q000150:complications* / Q000601:surgery; D013163:Splenomegaly / Q000209:etiology / Q000601:surgery*; D016896:Treatment Outcome