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