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Patient-reported outcomes in the evaluation of toxicity of anticancer treatments

Nature Reviews Clinical Oncology volume 13, pages 319–325 (2016)Cite this article

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Abstract

Symptomatic toxicities associated with anticancer treatments, such as nausea and vomiting, are frequently underreported by clinicians, even when data are prospectively collected within clinical trials. Such underreporting can result in an underestimation of the absolute rate of toxicity, which is highly relevant information for patients and their physicians in clinical practice, and for regulatory authorities. Systematic collection of patient-reported outcomes (PROs) has been demonstrated to be a valid, reliable, feasible and precise approach to tabulating symptomatic toxicities and enables symptoms that are missed by clinicians to be detected. In this Perspectives, the barriers and challenges that should be addressed when considering broad integration of PRO toxicity monitoring in oncology clinical trials are discussed, including challenges related to data collection logistics, analytical approaches, and resource utilization. Instruments conceived to enable description of treatment-related adverse effects, from the patient perspective, bring the potential to improve risk-versus-benefit analyses in clinical research, and to provide patients with accurate information, on the basis of previous experiences of their peers.

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Figure 1: Underreporting of treatment-related toxicities by physicians, relative to patients with either advanced-stage lung cancer, or early-stage breast cancer.
Figure 2: Underreporting by physicians of specific treatment-associated symptoms by physicians in the TORCH trial.
Figure 3: Possible modalities for reconciliation of patient's and physician's report of symptomatic treatment-associated toxicities.

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  • 27 January 2016

    In the original version of this article published online, the title and doi of reference 43 was incorrect. These errors have been corrected in the online and print versions of the article.

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Acknowledgements

M.D.M. has received research funding from the CRT Foundation (Torino, Italy) for a research project studying the impact on the quality of life of cancer patients of a systematic collection of treatment toxicities by dedicated questionnaires.

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Authors and Affiliations

  1. Massimo Di Maio is at the Department of Oncology, San Luigi Gonzaga Hospital, University of Turin, Regione Gonzole 10, 10043 Orbassano (TO), Italy.,

    Massimo Di Maio

  2. Ethan Basch is at the Cancer Outcomes Research Program, Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, North Carolina 27599, USA.,

    Ethan Basch

  3. Jane Bryce and Francesco Perrone are at the Clinical Trials Unit, Istituto Nazionale per lo Studio e la Cura dei Tumori - Fondazione G. Pascale IRCCS, via Mariano Semmola, 80131 Napoli, Italy.,

    Jane Bryce & Francesco Perrone

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All authors researched data for this article, made a substantial contribution to discussions of content, wrote the manuscript and reviewed and/or edited the manuscript prior to submission.

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Correspondence to Massimo Di Maio.

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Competing interests

E.B. has received research funding from the US National Cancer Institute for developing the PRO–CTCAE. The other authors declare no competing interests.

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Di Maio, M., Basch, E., Bryce, J. et al. Patient-reported outcomes in the evaluation of toxicity of anticancer treatments. Nat Rev Clin Oncol 13, 319–325 (2016). https://doi.org/10.1038/nrclinonc.2015.222

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