Table 1.

Recommended end points and measures for PROs in SCD

Outcome/End point	Recommended measurement/tool(s)
Pain
Intensity	For patients at least 8 y of age, an 11-point NRS or appropriately reproduced and administered VAS
Interference/impact	PROMIS Pain Interference measures for studies of adult, pediatric, or mixed age groups
	ASCQ-ME Pain Impact for adults or the Brief Pain Impact for adults
	PedsQL Pain Impact and Pain and Hurt for children
Behavior	PROMIS Pain Behavior domain for children and adults
Affect
Depression/negative emotional impact	PedsQL SCD Module Emotions measure for children
	ASCQ-ME Emotional Impact measure for adults
	PROMIS Depression and Anxiety measures for more general measures of negative affect, particularly when mixed age samples are used
Fatigue	PROMIS or PedsQL Fatigue measures in children
	PROMIS Fatigue measure in adults
Function
Emotional/social, physical, cognitive domains	Relevant domains of the PROMIS and ASCQ-ME in adults and PROMIS and PedsQL in children to measure emotional/social, physical, and cognitive function PRO domains
	The Canadian Occupational Performance Measure may be used to measure functional capacity that integrates self-report and expert interview
Occupational status	One item in PhenX for assessment of employment status and the more detailed WHO’s Health and Work Performance Questionnaire to assess multiple factors related to work performance
Self-efficacy	Sickle Cell Efficiency Scale to measure self-efficacy in adolescents and adults with SCD