title: [Enzyme therapy in children with severe forms of Gaucher's disease].
abstract: The enzyme therapy with Ceredase in patients with Gaucher's disease is at present probably the most expensive treatment in the whole world. One-year treatment of an adult patient with Gaucher's disease costs more than 7 million crowns. Indications for treatment in individual patients as well as financial provisions are so far problematic in the Czech Republic. From a total of 28 patients of varying age with Gaucher's disease diagnosed by the authors Ceredase was administered to two boys with a severe course of the disease. Within one year of treatment the health status of both children improved, growth became normal, the spleen diminished in size by 20-35%, haematological manifestations of hypersplenism are receding, there was a 32-46% decline of the activity of serum chitotriosidase and biochemical parameters of the disease improved.
authors.affiliation: Klinika dĕtského a dorostového lékarství 1. LF UK a VFN, Praha.
authors.forename: J
authors.initials: J
authors.lastname: Zeman
authors.forename: M
authors.initials: M
authors.lastname: Hrebícek
authors.forename: L
authors.initials: L
authors.lastname: Vepreková
authors.forename: J
authors.initials: J
authors.lastname: Kytnarová
authors.forename: H
authors.initials: H
authors.lastname: Houstková
authors.forename: V
authors.initials: V
authors.lastname: Malinová
authors.forename: K
authors.initials: K
authors.lastname: Hodanová
authors.forename: H
authors.initials: H
authors.lastname: Poupĕtová
authors.forename: J
authors.initials: J
authors.lastname: Ledvinová
authors.forename: J
authors.initials: J
authors.lastname: Hoza
mesh_terms: D002648:Child; D005776:Gaucher Disease / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000627:therapeutic use*; D006801:Humans; D008297:Male
chemical_list: C070292:alglucerase; D005962:Glucosylceramidase