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Review
. 2022 Feb;48(1):15-29.
doi: 10.1016/j.rdc.2021.09.008.

Assessing Patient-Reported Outcomes in Pediatric Rheumatic Diseases: Considerations and Future Directions

Christina K Zigler  1 , Rachel L Randell  2 , Bryce B Reeve  3
Affiliations
Review

Assessing Patient-Reported Outcomes in Pediatric Rheumatic Diseases: Considerations and Future Directions

Christina K Zigler et al. Rheum Dis Clin North Am. 2022 Feb.

Abstract

For children with pediatric rheumatic diseases (PRDs), the inclusion of patient-reported outcomes (PROs) is critical to inform decision making in health care delivery and research settings. PROs are direct reports from a child on their health status, without interpretation by anyone else. PROs improve understanding of the patient experience, allow clinicians to provide patient-centered care, and add value to clinical trials. When PROs cannot be collected directly from the patient, caregiver-proxy reports can provide important information on the child's more observable symptoms and functioning. In this article, we describe the current use of PROs in specific PRDs, align current research with best practice recommendations for both clinical care and research settings, highlight exciting new developments, and identify areas for future research.

Keywords: Clinical care; Clinical trials; Future directions; Patient-reported outcome measures; Patient-reported outcomes; Pediatric rheumatic diseases.

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Conflict of interest statement

Conflicts of interest statements Dr C.K. Zigler reports funding via her institution from the National Institutes of Health, the U.S. Food & Drug Administration and the Childhood Arthritis and Rheumatology Research Alliance (CARRA). Dr R.L. Randell receives support from the National Institute of General Medical Sciences and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) of the National Institutes of Health (NIH) under Award Number T32GM086330. Dr R.L. Randell’s spouse has current or prior employment and/or stock ownership in Merck & Co and Biogen. Dr B.B. Reeve received funding via his institution from the NIH/NIAMS grant U19AR069522.

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