Table 1.

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)

MN indicates multiples of normal (volume).