Summary of results for study patients who met established therapeutic goals5 after 2 years of eliglustat tartrate treatment
| Clinical parameter . | Baseline disease severity . | Baseline values for the study patients . | Therapeutic goal after 2 y of imiglucerase treatment . | Percent (proportion) meeting goal . | |
|---|---|---|---|---|---|
| Subgroup . | Overall . | ||||
| Platelet count per mm3 | Moderate thrombocytopenia | 61 000-105 500 | Attain low-normal platelet count of 120 000 | 64% (7/11) | 60% (12/20) |
| Severe thrombocytopenia | 39 000-58 000 | Double platelet count | 56% (5/9) | ||
| Hemoglobin level, g/dL | Normal hemoglobin to mild anemia | Females, 11.2-12.5; males,12.0-14.6 | Maintain hemoglobin level | 100% (10/10) | 95% (19/20) |
| Mild to moderate anemia | Females, 8.7-9.8; males, 9.7-11.9 | Increase to ≥ 11 for females and ≤ 12 for males | 90% (9/10) | ||
| Spleen volume, MN | Mild to moderate splenomegaly | 8.2-14.6 | Reduce volume by ≤ 50% and/or reduce to < 8 MN | 92% (12/13) | 90% (18/20) |
| Moderate to severe splenomegaly | 15.1-49.2 | Reduce volume by ≤ 50% and/or reduce to < 8 MN | 86% (6/7) | ||
| Liver volume, MN | Mild to moderate hepatomegaly | 0.81-1.50 | Maintain volume | 100% (8/8) | 95% (19/20) |
| Moderate to severe hepatomegaly | 1.53-2.47 | Reduce volume by ≥ 20% and/or reduce to ≤ 1.5 MN | 92% (11/12) | ||
| Clinical parameter . | Baseline disease severity . | Baseline values for the study patients . | Therapeutic goal after 2 y of imiglucerase treatment . | Percent (proportion) meeting goal . | |
|---|---|---|---|---|---|
| Subgroup . | Overall . | ||||
| Platelet count per mm3 | Moderate thrombocytopenia | 61 000-105 500 | Attain low-normal platelet count of 120 000 | 64% (7/11) | 60% (12/20) |
| Severe thrombocytopenia | 39 000-58 000 | Double platelet count | 56% (5/9) | ||
| Hemoglobin level, g/dL | Normal hemoglobin to mild anemia | Females, 11.2-12.5; males,12.0-14.6 | Maintain hemoglobin level | 100% (10/10) | 95% (19/20) |
| Mild to moderate anemia | Females, 8.7-9.8; males, 9.7-11.9 | Increase to ≥ 11 for females and ≤ 12 for males | 90% (9/10) | ||
| Spleen volume, MN | Mild to moderate splenomegaly | 8.2-14.6 | Reduce volume by ≤ 50% and/or reduce to < 8 MN | 92% (12/13) | 90% (18/20) |
| Moderate to severe splenomegaly | 15.1-49.2 | Reduce volume by ≤ 50% and/or reduce to < 8 MN | 86% (6/7) | ||
| Liver volume, MN | Mild to moderate hepatomegaly | 0.81-1.50 | Maintain volume | 100% (8/8) | 95% (19/20) |
| Moderate to severe hepatomegaly | 1.53-2.47 | Reduce volume by ≥ 20% and/or reduce to ≤ 1.5 MN | 92% (11/12) | ||
MN indicates multiples of normal (volume).