Pastores et al outlined therapeutic goals for a child with Gaucher Disease. Some of the goals involve hematologic parameters such as correction of anemia and thrombocytopenia.


Time frame: varies from 12 to 24 months (1-2 years)


Goals if the patient is anemic (severe if hemoglobin < 8 g/dL):

(1) Eliminate dependency on blood transfusion.

(2) Increase and maintain the hemoglobin to > 11 g/dL

(3) Reduce symptoms of anemia (fatigue, dyspnea, etc).


Goals if the patient is thrombocytopenic (severe if < 60,000 per µL)

(1) Avoid spontaneous and surgical bleeding related to thrombocytopenia.

(2) Increase and maintain the platelet count by 1.5 times or more (may be able to reach normal range if mild to moderate).


Goals for leucopenia were not listed but it would seem appropriate to avoid absolute neutropenia.


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