Table 6.

Therapeutic Goals for Children (Modified from Pastores et al⁷⁴).

Anemia	•Increase Hb levels within 1–2 years to >11.0 g/dl and maintain Hb •Eliminate blood transfusion dependency •Reduce fatigue and dyspnea
Thrombocytopenia	•Increase platelet counts during the 1st year of treatment >60 × 103 cells/mm3 •Moderate thrombocytopenia: Count should increase by 1.5- to 2.0-fold by 1 year and low-normal level by 2 years •Severe thrombocytopenia: Counts should increase by 1.5- fold by 1 year and double by 2 years but normalization is not expected •Avoid splenectomy
Hepatomegaly	•Reduce and maintain the liver volume to <1.5 fold. •Reduce the liver volume by 20–30% within 1–2 years and by 30–40% by 5 years
Splenomegaly	•Reduce and maintain spleen volume to <2–8-fold normal •Reduce the spleen volume by 30% within 1 year and 60% by 2–5 years •Alleviate symptoms due to splenomegaly
Bones	•Lessen or eliminate bone pain within 2 years of treatment •Prevent bone crises •Prevent osteonecrosis and joint collapse •Improve bone mineral density by 2 years of treatment •Attain normal or ideal peak skeletal mass
Growth	•Achieve normal height within 3 years of treatment •Achieve normal onset of puberty