Journal article
Conscientious use of patient-reported outcome measures in supportive care
Supportive care in cancer, Vol.31(4), p.226
04/01/2023
DOI: 10.1007/s00520-023-07681-y
PMCID: PMC10033615
PMID: 36947341
Abstract
Purpose Patient-reported outcome measures (PRO) are critical tools to developing an understanding of cancer patients' experience. This paper presents some of the lesser-understood implications of using patient-reported outcome measures in clinical research. Methods This study uses a combination of literature sources, real-world examples from supportive care studies, and statistical simulations to demonstrate the operating characteristics of patient-reported measures. Results It is demonstrated that care must be taken in the analysis of PROs as the assumptions of the most common mean-based approaches are often violated including linearity, normally distributed errors, interference with asymptotic convergence via boundary values, and more. Further, the implications of subjective discretization are shown to reduce the apparent statistical power of PRO-based studies. Conclusions PRO-based studies must be designed conscientiously as each PRO item will demonstrate a varying degree of subjectivity in a given population. Sample sizes of randomized studies using PROs must be inflated to account for this. Analyses should consider using ordinal statistical models until such time as the assumptions of mean-based models can be verified.
Details
- Title: Subtitle
- Conscientious use of patient-reported outcome measures in supportive care
- Creators
- Derek K. Smith - American Dental Association
- Resource Type
- Journal article
- Publication Details
- Supportive care in cancer, Vol.31(4), p.226
- DOI
- 10.1007/s00520-023-07681-y
- PMID
- 36947341
- PMCID
- PMC10033615
- NLM abbreviation
- Support Care Cancer
- ISSN
- 0941-4355
- eISSN
- 1433-7339
- Publisher
- Springer Nature
- Number of pages
- 6
- Grant note
- K23 DE028010 / National Institute for Dental and Craniofacial Research; United States Department of Health & Human Services; National Institutes of Health (NIH) - USA; NIH National Institute of Dental & Craniofacial Research (NIDCR)
- Language
- English
- Date published
- 04/01/2023
- Academic Unit
- Preventive and Community Dentistry; Dental Research
- Record Identifier
- 9984966752002771
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