Table 6.

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

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.