Outcomes | Definitiona |
---|---|
Primary outcome | The primary outcome is a composite of one or more individual components among the following: |
- Liver volume >1.25 MN | |
- Spleen volume >5 MN | |
- Hemoglobin concentration <11.0 g/dL (<10.0 g/dL for patients 6 to 59 months of age) | |
- Platelet count <100 × 109/mL | |
Secondary outcomes | 1. Composite of one or more individual components among the following: |
- Liver volume >2.5 MN | |
- Spleen volume >15 MN | |
- Hemoglobin concentration <8.0 g/dL (<7.0 g/dL for patients 6 to 59 months of age) | |
- Platelet count <50 × 109/mL | |
2. Symptomatic bone manifestations with imaging confirmation including the following: | |
- Osteonecrosis | |
- Fracture |