Psychometric evaluation of Patient-Reported Outcomes Measurement Information System (PROMIS) in pediatric sickle cell disease in Europe.

UNLABELLED: Sickle cell disease has a profound impact on the physical, mental and social health of affected children. Currently, there is considerable variability among the available patient reported outcomes measures (PROMs) used in children with sickle cell disease, and no consensus has yet been achieved. We aim to assess the psychometric properties of the generic pediatric and proxy Patient-Reported Outcomes Measurement Information System (PROMIS®) measures in children with sickle cell disease living in the Netherlands. Dutch children with sickle cell disease aged 5-17 years old and their caregivers were eligible. The following self-report and proxy-report PROMIS® item banks were evaluated: Anger, Anxiety, Depressive Symptoms, Fatigue, Mobility, Pain Interference, Peer Relationships, Cognitive Functioning, and Global Health. We assessed unidimensionality through confirmatory factory analysis, convergent validity with subscales from the Pediatric Quality of Life Inventory, discriminant validity, reliability, and inter-rater reliability. The study enrolled 102 patients and 102 caregivers, of which 71 were dyads. All item banks displayed sufficient unidimensionality and convergent validity. Discriminant validity was hypothesized and found for "Global Health," "Mobility" (d > 0.3), "Fatigue," and "Pain Interference" (d > 0.3), although some comparisons were non-significant. Reliability was acceptable (a > 0.80, SEM < 0.44) for all PROMIS® measures. Inter-rater reliability was moderately-strong for all item bankss (ICC 0.60-0.78) except for "Peer Relationships" (ICC = 0.47, r = 0.31) and "Global Health" (ICC = 0.26, r = 0.16), which scored lower on correlation.

CONCLUSION: PROMIS® measures displayed sufficient psychometric properties for use in pediatric sickle cell disease care and research. Proxy-reports seem viable as alternative to self-report forms of PROMIS®.

WHAT IS KNOWN: • Sickle cell disease has a profound impact on the physical, mental and social health of affected children and identifying early signs of decline is crucial to intervene before complications arise. • No consensus has yet been achieved among the available patient reported outcomes measures (PROMs) used in children with sickle cell disease living in Europe.

WHAT IS NEW: • This study provides evidence that PROMIS® measures displayed sufficient psychometric properties for use in European pediatric sickle cell disease care and research. • Proxy-reports seem viable as alternative to self-report forms of PROMIS®.