Version May 2024
| 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 |
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