Therapeutic Goals for a Child with Gaucher Disease: Hepatomegaly and Splenomegaly

 

Goals for a child with splenomegaly:

(1) Reduce symptoms (abdominal distension, discomfort, early satiety).

(2) Avoid splenectomy.

(3) Avoid new splenic infarctions.

(4) Eliminate hypersplenism (with anemia, thrombocytopenia and/or leucopenia).

(5) Reduce the splenic volume to less than 8 times normal (preferable less than 2 times).

 

Goals for a child with hepatomegaly:

(1) Reduce the liver volume to <= 1.5 times normal and maintain this size.

 

A significant reduction in organ size should be achieved over 5 years (50-60% reduction for spleen, 30-40% reduction for liver) with half of the reduction achieved in the first 2 years.