Psychometrics of patient-reported bother from side effects of treatment single-items in industry-sponsored oncology trials

• Published in: Quality of life research Vol. 32; no. 9; pp. 2601 - 2615
• Main Authors: Trask, Peter C., Holt, Tracy, Pearman, Timothy
• Format: Journal Article
• Language: English
• Published: Cham Springer International Publishing 01-09-2023; Springer Nature B.V
• Subjects: Adolescent; Adult; Aged; Aged, 80 and over; Cancer therapies; Clinical trials; Drug-Related Side Effects and Adverse Reactions; Female; Hematology; Humans; Male; Medicine; Medicine & Public Health; Middle Aged; Neoplasms - drug therapy; Oncology; Patient Reported Outcome Measures; Patients; Psychometrics; Public Health; Quality of life; Quality of Life - psychology; Quality of Life Research; Quantitative psychology; Reproducibility of Results; Sociology; Surveys and Questionnaires; Validity; Working groups; Young Adult; Single item measure; Clinical trials; Oncology; Symptom bother; Quality of life
• ISSN: 0962-9343; 1573-2649
• DOI: 10.1007/s11136-023-03426-w

Background
Improvements in cancer treatment have increased the number of cancer survivors, but also increased the long-term and late effects from cancer therapy. Patient reported “side effect bother” could be used to measure the burden of treatment, and the risk for negative outcomes such as dose reduction, treatment delay or discontinuation. The current study addresses the psychometric properties of a single item, determines what represents a “meaningful change”, and evaluates the correlation to safety endpoints and functioning.
Methods
Results from 5911 patients enrolled in 8 clinical trials representing 5 disease types in oncology and hematology who completed either the Functional Assessment of Cancer Therapy (FACT) GP5 item or a modified bother item (MBI) were assessed.
Results
Patients ranged in age from 18 to 93 years, with all cancer stages represented and approximately equal numbers of males and females. Test–retest reliability was acceptable, as were convergent and known groups validity. The GP5 and MBI effectively demonstrated sensitivity to change over time and established meaningful thresholds.
Conclusions
The results indicate that these single-items are psychometrically sound, capable of distinguishing known groups, responsive to change and can identify meaningful change over time in terms of treatment-related symptoms. It extends the findings of recent scientific groups by providing analyses not included in prior studies, and further supports the FDA’s recommendation to include a single item question in clinical trials.