Category | Management goals |
Anemia-related symptoms | Maintain improved hemoglobin values achieved after the first 12 to 24 months of therapy |
Bleeding tendency | Maintain platelet count of ≥100,000/mm3 |
Reduce increased bleeding tendency, whether caused by low platelet numbers, platelet defects, or coagulation abnormalities |
Mobility | Prevent bone complications – Avascular necrosis, bone crises, bone infarcts, and pathological fractures |
Prevent osteopenia and osteoporosis (ie, maintain BMD T-scores [DXA] of >–1) |
Prevent chronic use of analgesic medication for bone pain |
Maintain normal mobility or, if impaired at diagnosis, improve mobility |
Increase physical activity |
Visceral complications | Maintain spleen volume of <2 to 8 times normal after year 1 to 2 |
Maintain (near) normal liver volume after year 1 to 2 |
Prevent liver fibrosis, cirrhosis, and portal hypertension |
Pulmonary complications | Prevent or improve pulmonary disease, such as pulmonary hypertension and hepatopulmonary syndrome |
General well-being | Maintain good quality of life as measured by a validated instrument |
Maintain normal participation in school and work activities |
Minimize psychosocial burdens of lifelong treatment |
Achieve normal onset of puberty |
Normalize life expectancy |
Pregnancy and delivery | Prevent GD-related complications during pregnancy and delivery |