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

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Abstract

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.

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Data availability

For eligible studies, qualified researchers may request access to individual patient level clinical data through a data request platform. At the time of writing this request, the platform is Vivli. https://vivli.org/ourmember/roche/. For up-to-date details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here: https://go.roche.com/data_sharing. Anonymised records for individual patients across more than one data source external to Roche cannot, and should not, be linked due to a potential increase in risk of patient re-identification.

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Acknowledgements

The authors would like to thank all of the patients who participated in the clinical trials used in this paper. Editorial support by Eleanor Porteous, MSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd.

Funding

This work was supported by F. Hoffmann-La Roche/Genentech, Inc. F. Hoffmann-La Roche/Genentech, Inc., sponsored the studies of which the data for this analysis were interpreted. The design of the study; the collection, analysis, and interpretation of the data; and the decision to submit the manuscript for publication was led by the authors. Editorial support by Eleanor Porteous, MSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd.

Author information

Authors and Affiliations

  1. Genentech, 1 DNA Way, South San Francisco, CA, USA

    Peter C. Trask & Tracy Holt

  2. PAREXEL International, Billerica, MA, USA

    Tracy Holt

  3. Supportive Oncology, Robert H. Lurie Comprehensive Cancer Center of Northwestern University, School of Medicine, 625 N. Michigan Avenue, Chicago, IL, USA

    Timothy Pearman

Authors
  1. Peter C. Trask
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  2. Tracy Holt
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Contributions

All authors contributed to the study conception and design. Material preparation, data collection and analysis were performed by PT and TH. The first draft of the manuscript was written by TP and PT, and all authors commented on all versions of the manuscript. All authors reviewed and approved the final manuscript.

Corresponding author

Correspondence to Timothy Pearman.

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Conflict of interests

PCT, is an employee of Genentech. TH is employed as a consultant (contractor) by Genentech. TP is on the speakers’ bureau for Pfizer and Genentech.

Ethical approval

The study was performed in line with the principles of the Declaration of Helsinki. Peter Trask is employed by Genentech and holds stock in Roche. Tracy Holt has no financial interests. Timothy Pearman has received speaker honorarium in the past from Genentech.

Informed consent

Written informed consent was obtained from all participants of the original clinical trials. There is no personally identifiable information in the manuscript, and therefore consent to publish was not required.

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Trask, P.C., Holt, T. & Pearman, T. Psychometrics of patient-reported bother from side effects of treatment single-items in industry-sponsored oncology trials. Qual Life Res 32, 2601–2615 (2023). https://doi.org/10.1007/s11136-023-03426-w

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