Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease: A Single-Center Experience

Table 3

Percent of patients achieving therapeutic goals in 4 years.

Therapeutic goal	Current study (𝑛=164)	Change in 4 years
Anemia	9.1% to 81.7%	72.6%
Thrombocytopenia	20.7% to 68.9%	48.2%
Splenomegaly	60.0% at 4 years	ND
Hepatomegaly	53.1% at 4 years	ND
No bone pain	42.1% to 59.9%	17.8%
No bone crises	91.5% to 93.8%	2.3%
No osteonecrosis	78.7% to 92.6%	13.9%
LS Z-score	69.9% at 4 years	ND
FN Z-score	70.6% at 4 years	ND

∗ND: not done.