[Enzyme therapy in children with severe forms of Gaucher's disease]

Cas Lek Cesk. 1997 Feb 5;136(3):95-7.
[Article in Czech]

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.

Publication types

  • Case Reports

MeSH terms

  • Child
  • Gaucher Disease / drug therapy*
  • Glucosylceramidase / therapeutic use*
  • Humans
  • Male

Substances

  • alglucerase
  • Glucosylceramidase