[Enzyme therapy in children with severe forms of Gaucher's disease].
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.
Zeman
J
J
Klinika dĕtského a dorostového lékarství 1. LF UK a VFN, Praha.
Hrebícek
M
M
Vepreková
L
L
Kytnarová
J
J
Houstková
H
H
Malinová
V
V
Hodanová
K
K
Poupĕtová
H
H
Ledvinová
J
J
Hoza
J
J
D002648:Child; D005776:Gaucher Disease / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000627:therapeutic use*; D006801:Humans; D008297:Male
C070292:alglucerase; D005962:Glucosylceramidase