Evaluation of high density lipoprotein as a circulating biomarker of XXXX activity.

@entity1 @entity1289 @entity1120 @entity165
Circulating biomarkers are important surrogates for monitoring disease activity in type I @entity1120 (GD1). We and others have reported low high-density lipoprotein (HDL) in GD1. We assessed HDL @entity165 as a biomarker of GD1, with respect to its correlation with indicators of disease severity and its response to imiglucerase enzyme replacement therapy (ERT). In 278 consecutively evaluated GD1 @entity1 , we correlated HDL @entity165 , chitotriosidase, and @entity1289 ( @entity1289 ) with indicators of disease severity. Additionally, we measured the response of these biomarkers to ERT. HDL @entity165 was negatively correlated with spleen volume, liver volume, and GD severity score index; the magnitude of this association of disease severity with HDL @entity165 was similar to that for @entity1289 and for chitotriosidase. Within individual @entity1 monitored over many years, there was a strikingly strong correlation of HDL with liver and spleen volumes; there was a similarly strong correlation of chitotriosidase and @entity1289 with disease severity in individual @entity1 monitored serially over many years (chitotriosidase r = 0.96 to 0.98, @entity1289 r = 0.88 to 0.94, and HDL r =   -0.84 to -0.94, p < 0.001). ERT for 3 years resulted in a striking increase of HDL while serum levels of chitotriosidase and @entity1289 decreased. Our results reveal markedly low HDL @entity165 in untreated GD1, a correlation with indicators of disease severity in GD1, and a rise towards normal after ERT. These findings suggest HDL @entity165 merits inclusion within the "biomarker basket" for monitoring of @entity1 with GD1.
