Skip to main content

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