TABLE 2.
The Primary Endpoint Response to Eliglustat Tartrate in Type 1 Gaucher Patients Participating in the Phase 2 Trial
| 12 Months | 48 Months | |
|---|---|---|
| Hemoglobin | +1.62 g/dL | +2.3 g/dL |
| Platelet count | +40.3% | +95% |
| Spleen volume | −38.5% | −63% |
| Liver volume | −27.0% | −28% |
Nineteen patients completed 4 years of treatment with eliglustat. The overall change in hemoglobin was from 11.3 ± 1.5 to 13.6 ± 1.2 g/dL and in platelet count from 68,700 ± 21,200 to 125,400 ± 51,100/mm3. Spleen and liver volumes were measured as multiples of normal (MN). The spleen volumes decreased from 17.3 ± 9.5 to 6.1 ± 3.4 MN and the liver volumes decreased from 1.7 ± 0.4 to 1.2 ± 0.3 MN.