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 |
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• 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 |