Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment.

ABSTRACT:

Objective

We studied the effect of long-term alglucerase/imiglucerase (Ceredase®/Cerezyme®, Genzyme, a Sanofi company, Cambridge, MA, USA) treatment on hematological, visceral, and bone manifestations of Gaucher disease type 1 (GD1).

Methods

The International Collaborative Gaucher Group (ICGG) Gaucher Registry identified GD1 patients treated with alglucerase/imiglucerase who had dose and clinical data at first infusion and after 10 years of follow-up. Data for hemoglobin, platelet count, organ volumes, bone pain, and bone crisis were analyzed. Tests of the null hypothesis (no change from first infusion to 10 years) were performed using t tests for within-patient absolute change in continuous measurements and McNemar/chi-square tests for change in distributions using categori

SUBMITTER: Weinreb NJ 

PROVIDER: S-EPMC3648688 | biostudies-literature | 2013 May

REPOSITORIES: biostudies-literature