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Table 2 Final Core Sets

From: coreSCD: multi-stakeholder consensus on core outcomes for sickle cell disease clinical trials

Core Outcome Set for Clinical Trials of
Disease-Modifying Therapies
Core Outcome Set for Clinical Trials of
Acute Interventions
• Acute sickle cell pain frequency
• Acute chest syndrome
• Stroke or cerebrovascular accident
• Neurocognitive function
• Health-related quality of life
• Frequency of hospitalization
• Emergency department/acute care visit
• Need for blood transfusion
• Cause-specific survival/mortality
• Event-free survival
• Acute sickle cell pain frequency
• Acute chest syndrome
• Ability to return to usual activities
• Frequency of hospitalization
• Emergency department/acute care visit
• Cause-specific survival/mortality