Recommended end points for brain outcomes in SCD trials
| Timeframe for outcome assessment . | Physiology . | Imaging . | Function . |
|---|---|---|---|
| Immediate <1 d | TCD, cerebral blood flow, oxygen extraction fraction, cerebrovascular reactivity, MR velocity | ||
| Short-term <1 y | TCD, cerebral blood flow, oxygen extraction fraction, cerebrovascular reactivity, MR velocity | Thrombosis, infarction | School or job absences, functional outcome measures (COPM) |
| 1-2 y | TCD, cerebral blood flow, oxygen extraction fraction, cerebrovascular reactivity, MR velocity | Thrombosis, infarction | School or job absences, cognition, functional outcome measures |
| Long-term 3 y or more | TCD, cerebral blood flow, oxygen extraction fraction, cerebrovascular reactivity, MR velocity | Thrombosis, infarction, iron deposition, white matter structural connectivity | School or job absences, cognition, functional outcome measures |
| Irreversible/degenerative | Stroke, SCI, microbleeds, atrophy, iron deposition, white matter structural connectivity, stenosis, vasculopathy including moya-moya | Held back a grade level in school, cognition, functional outcome measures |
| Timeframe for outcome assessment . | Physiology . | Imaging . | Function . |
|---|---|---|---|
| Immediate <1 d | TCD, cerebral blood flow, oxygen extraction fraction, cerebrovascular reactivity, MR velocity | ||
| Short-term <1 y | TCD, cerebral blood flow, oxygen extraction fraction, cerebrovascular reactivity, MR velocity | Thrombosis, infarction | School or job absences, functional outcome measures (COPM) |
| 1-2 y | TCD, cerebral blood flow, oxygen extraction fraction, cerebrovascular reactivity, MR velocity | Thrombosis, infarction | School or job absences, cognition, functional outcome measures |
| Long-term 3 y or more | TCD, cerebral blood flow, oxygen extraction fraction, cerebrovascular reactivity, MR velocity | Thrombosis, infarction, iron deposition, white matter structural connectivity | School or job absences, cognition, functional outcome measures |
| Irreversible/degenerative | Stroke, SCI, microbleeds, atrophy, iron deposition, white matter structural connectivity, stenosis, vasculopathy including moya-moya | Held back a grade level in school, cognition, functional outcome measures |
Outcomes in bold can be measured with standard measures. As a reminder, for TCD, the greatest benefit would be to determine whether a participant decreased from the abnormal (meeting the threshold for transfusion) to the normal range. There are no data to suggest that simply decreasing the TAMMV from a normal to slightly lower velocity is an added benefit.
COPM, Canadian Occupational Performance Measure.