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

Table 2

Long-term goals (2–5 years) for adults (Pastores et al., 2004 [7]).

Anemia	Women: increasing hemoglobin concentration to ≥11 g/dL;
	Men: increasing hemoglobin concentration to ≥12 g/dL;
	Maintaining improved hemoglobin value achieved in first 1-2 years
Thrombocytopenia	Moderate thrombocytopenia at baseline (60–120 × 10^9/L): Increase platelet count 1.5 to 2 times by 1 Year, and approach low normal platelet count values by 2 Year.
	Severe thrombocytopenia at baseline (<60 × 10^9/L): increasing platelet count 1.5 times by 1 Year and continuing to improve platelet counts slightly (2 times by 2 Year)
Splenomegaly	Decreasing spleen volume by 30–50% within 1 Year; reducing and maintaining spleen volume to ≤2–8 times normal or decreasing volume by 50–60% by 2–5 Years
Hepatomegaly	Decreasing liver volume by 20–30% within 1-2 Years; reducing and maintaining liver volume to 1.0 to 1.5 times normal or reducing liver volume by 30–40% by 3–5 Years
Bone pain	Lessening or eliminating bone pain in 1-2 years
Bone crises	Preventing bone crises
Osteonecrosis	Preventing osteonecrosis and joint collapse
Skeletal pathology	Improving trabecular BMD by 3–5 years