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In a previous short-term study (ENCORE), eliglustat — an inhibitor of glucosylceramide synthase — decreased spleen and liver volumes and improved hematologic parameters in patients with Gaucher disease type 1 (NEJM JW Oncol Hematol Apr 2015 and JAMA 2015; 313:695).
To examine the long-term outcomes of eliglustat therapy, investigators reviewed and analyzed the records of 157 patients taking the drug for up to 4.5 years.
Results were as follows:
Mean values for hemoglobin concentration, platelet count, spleen volume, and liver volume remained stable among all eliglustat-treated patients.
The 46 patients receiving eliglustat for 4 years achieved significant reductions in least-square mean liver volumes (3%; P=0.03) and spleen volumes (13%; P<0.00…