Table 2.

Pediatric PRO Instruments

			Psychometric Properties
Instrument	Aim	Items and Scoring	Developed for SCD Patients	Content Validity (SCD-specific)	Internal Reliability (Cronbach’s α)	Test-retest Reliability	Construct Validity	Responsiveness
Health-related Quality of Life
PedsQL™ Generic Core Scales [28, 34, 36]	To assess HRQL of children as young as 2 years (proxy report) and children as young as 5 years (self-report)	23 items rated on a 5-point Likert scale; higher scores indicate more problems	No	Unclear	Strong (0.80–0.90 parent-report; 0.93 self-report)	Unclear	Unclear	Unclear
PedsQL™ SCD Module [36]	To assess quality of life that is specific to assessing the health issues relevant to pediatric patients with SCD	43 items rated by a 5-point Likert scale; higher scores represent better quality of life	Yes	Unclear	Strong (0.95–0.97)	Unclear	Unclear	Unclear
PedsQL™ Multidimensional Fatigue Scale [35, 36]	To measure fatigue experienced during the past 1 month across a variety of pediatric populations	18 items rated on a 5-point Likert scale; higher scores represent better quality of life	No	Unclear	Strong (0.95 parent-report; 0.90 self-report)	Unclear	Unclear	Unclear
Patient Reported Outcomes Measurement Information System (PROMIS) [29]	To measure a pediatric patient’s health attributes of depressive symptoms over the previous 7 days	156 items over 8 domains (physical functioning mobility, physical functioning upper extremity, pain interference, fatigue, depressive symptoms, anxiety, peer relationships, and anger) rated on a 5-point Likert scale; higher scores signify worse severity for depression, anxiety, anger, fatigue, and pain interference and better functioning for physical functioning mobility, physical functioning upper extremity, and peer relationships	No	Unclear	Unclear	Unclear	Good	Unclear
Pain
Faces Pain Scale-Revised (FPS-R) [31]	To assess pain intensity in pediatric populations experiencing acute pain	6 faces depicting different levels of increasing pain intensity	No	Good	Unclear	Unclear	Unclear	Unclear
Sickle Cell Disease Pain Burden Interview-Youth (SCPBI-Y) [26]	To assess the pain burden among children and adolescents with SCD over the previous month	7 items ranked on a 5-point Likert scale; higher scores indicate higher pain burden	Yes	Good	Strong (0.89)	Good	Good	Unclear
Coping, Self-efficacy
Coping Strategy Questionnaire (CSQ) [30]	To measure coping with pain in adults, adolescents, and young children	13 subscales, each with 6 items rated on a 7-point Likert-type scale; higher scores indicate greater pain	No	Unclear	Unclear	Unclear	Unclear	Unclear
Sickle Cell Self-efficacy Scale (SCSES) [27]	To assess adults and adolescent’s self-appraisals of their ability to engage in daily functional activities despite having SCD	9 items rated on a 5-point Likert scale; higher scores indicate greater self-efficacy	Yes	Unclear	Strong (0.87)	Unclear	Weak	Unclear
Functioning
Youth Acute Pain Functional Ability Questionnaire (YAPFAQ) [37]	To assess physical function and functional recovery in youth undergoing acute and procedural pain	40 items rated on a 5-point Likert scale; higher scores indicate greater difficulty performing functional activities	No	Unclear	Strong (0.92)	Unclear	Unclear	Unclear
Family Impact
Psychosocial Assessment Tool 2.0 (PAT2.0) [32]	To measure psychosocial risk in families of a child newly diagnosed with cancer	7 subscales; higher scores indicate many stressors	No	Unclear	Strong (0.84)	Unclear	Unclear	Unclear

Note: “Weak” indicates poor performance (e.g., evidence of poor reliability) or a weakness that should be considered within the trial design (e.g., requires significant input by research team to administer, or no availability of language translations); “Good” indicates adequate or moderate performance (e.g., adequate reliability) or only mild limitations (e.g., availability of a small number of language translations, absence of evidence in a minority of adult patients (e.g., older adults)); “Strong” indicates excellent performance on all reported indicators (e.g., all subscales report excellent reliability; evidence) or notable advantages for use within a trial (e.g., freely accessible, wide range of language translations); “Unclear” indicates where no or insufficient evidence was reported to assess, or where evidence reported was conflicting (e.g., some subscales show excellent reliability while others did not)

Abbreviations: HRQL health-related quality of life, SCD sickle cell diseas