Table 1.
Patient (baseline) | Genotype | Age at GD diagnosis | Chitotriosidase (nmol/hr/ml) | GlcSph in serum (ng/ml) | Hb (gm/dl) | Platelets (x1000 u/L) | Liver (x normal) | Spleen (x normal) | Age diagnosis of MG | Treatment Status | Start (Age) for Eliglustat 84 mg BID |
---|---|---|---|---|---|---|---|---|---|---|---|
(NR: <78.5) | (NR: 2–5) | ||||||||||
Pt1 | N370S/L444P | 12 | 10055.8 | 498.9 | 15.1 | 114 | 1.19 | 5.65 | 33 | Naïve | 34 |
Pt2 | N370S/? | 25 | 2607.1 | 139 | 13.8 | 85 | 0.95 | 7.46 | 56 | On ERT | 57 |