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Psychometric evaluation of Patient-Reported Outcomes Measurement Information System (PROMIS) in pediatric sickle cell
Maite E Houwing1, Michiel A J Luijten2,3,4,5, Madieke J Muntendam1
1Department of Paediatric Haematology, Erasmus MC Sophia Children's Hospital, University Medical Centre Rotterdam, Wytemaweg 80, 3015 CN, Rotterdam, The Netherlands.
Insights
Patient-Reported Outcomes Measurement Information System (PROMIS®) measures show good reliability and validity for children with sickle cell disease. Proxy reports are a viable alternative to self-reports in pediatric sickle cell disease research.
Area of Science:
- Pediatric Health
- Rheumatology
- Psychometrics
Background:
- Sickle cell disease significantly impacts children's physical, mental, and social well-being.
- Early identification of health decline is crucial for timely intervention in pediatric sickle cell disease.
- A lack of consensus exists regarding patient-reported outcomes measures (PROMs) for children with sickle cell disease in Europe.
Purpose of the Study:
- To evaluate the psychometric properties of generic pediatric and proxy Patient-Reported Outcomes Measurement Information System (PROMIS®) measures.
- To assess the suitability of PROMIS® measures for children with sickle cell disease in the Netherlands.
- To determine if proxy reports can serve as a viable alternative to self-reports for PROMIS® in this population.
Main Methods:
- A study involving 102 Dutch children (ages 5-17) with sickle cell disease and their caregivers.
- Evaluation of PROMIS® item banks: Anger, Anxiety, Depressive Symptoms, Fatigue, Mobility, Pain Interference, Peer Relationships, Cognitive Functioning, and Global Health.
- Assessment of unidimensionality, convergent and discriminant validity, reliability, and inter-rater reliability using confirmatory factor analysis and the Pediatric Quality of Life Inventory.
Main Results:
- All PROMIS® item banks demonstrated sufficient unidimensionality and convergent validity.
- Acceptable reliability (alpha > 0.80) and low standard error of measurement (SEM < 0.44) were found for all measures.
- Moderately-strong inter-rater reliability was observed, with lower correlations for "Peer Relationships" and "Global Health".
Conclusions:
- PROMIS® measures exhibit adequate psychometric properties for application in pediatric sickle cell disease care and research.
- Proxy-report PROMIS® measures are a feasible alternative to self-report measures in this population.
- These findings support the use of PROMIS® for comprehensive assessment in pediatric sickle cell disease.
Abstract:
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®.
