Elevated plasma glucosylsphingosine in Gaucher disease: relation to phenotype, storage cell markers, and therapeutic response

Blood. 2011 Oct 20;118(16):e118-27. doi: 10.1182/blood-2011-05-352971. Epub 2011 Aug 25.

Abstract

Gaucher disease, caused by a deficiency of the lysosomal enzyme glucocerebrosidase, leads to prominent glucosylceramide accumulation in lysosomes of tissue macrophages (Gaucher cells). Here we show glucosylsphingosine, the deacylated form of glucosylceramide, to be markedly increased in plasma of symptomatic nonneuronopathic (type 1) Gaucher patients (n = 64, median = 230.7 nM, range 15.6-1035.2 nM; normal (n = 28): median 1.3 nM, range 0.8-2.7 nM). The method developed for mass spectrometric quantification of plasma glucosylsphingosine is sensitive and robust. Plasma glucosylsphingosine levels correlate with established plasma markers of Gaucher cells, chitotriosidase (ρ = 0.66) and CCL18 (ρ = 0.40). Treatment of Gaucher disease patients by supplementing macrophages with mannose-receptor targeted recombinant glucocerebrosidase results in glucosylsphingosine reduction, similar to protein markers of Gaucher cells. Since macrophages prominently accumulate the lysoglycosphingolipid on glucocerebrosidase inactivation, Gaucher cells seem a major source of the elevated plasma glucosylsphingosine. Our findings show that plasma glucosylsphingosine can qualify as a biomarker for type 1 Gaucher disease, but that further investigations are warranted regarding its relationship with clinical manifestations of Gaucher disease.

MeSH terms

  • Chemokines, CC / blood
  • Enzyme Replacement Therapy
  • Enzyme Therapy
  • Female
  • Gaucher Disease / blood*
  • Gaucher Disease / drug therapy*
  • Gaucher Disease / enzymology
  • Gaucher Disease / genetics
  • Genotype
  • Glucosylceramidase / genetics
  • Glucosylceramidase / therapeutic use*
  • Hexosaminidases / blood
  • Humans
  • Macrophages / cytology
  • Male
  • Phenotype
  • Psychosine / analogs & derivatives*
  • Psychosine / blood
  • Recombinant Proteins / genetics
  • Recombinant Proteins / therapeutic use
  • Spectrometry, Mass, Electrospray Ionization

Substances

  • CCL18 protein, human
  • Chemokines, CC
  • Recombinant Proteins
  • Psychosine
  • sphingosyl beta-glucoside
  • Hexosaminidases
  • chitotriosidase
  • Glucosylceramidase