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Patient-reported system for measuring adverse events appears reliable
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A reporting system that integrates patient perspectives regarding adverse events related to cancer drugs appeared valid and reliable, according to findings from NCI.
The side effects patients experience during clinical trials often are reported by physicians rather than patients. However, several studies over the past decade have indicated physicians tend to under-report these symptoms.
This prompted NCI to create the patient-reported outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) in 2011.
Amylou C. Dueck, PhD, head of the section of biostatistics in the division of health sciences research for Mayo Clinic in Scottsdale, Ariz., and colleagues designed a study to evaluate the measurement properties of PRO-CTCAE, as well as its reliability and responsiveness.
Amylou C. Dueck
“A major achievement of this study is the testing of over 100 distinct questions about adverse events, which is unprecedented in a validation study,” Dueck told HemOnc Today. “[Another] great achievement is the successful recruitment of over 1,000 patients across the U.S., including many with impaired physical functioning, racial diversity and myriad cancer types. This wide representation of patients reflects the spectrum of people enrolled in cancer clinical trials.”
Dueck and colleagues identified 975 adults with cancer undergoing outpatient chemotherapy and/or radiation therapy and enrolled them into this questionnaire-based study between January 2011 and February 2012.
To evaluate the measurement properties of PRO-CTCAE, the researchers needed a set of comparators, so they used clinician-reported ECOG performance status and the European Organisation for Research and Treatment of Cancer Core Quality of Life Questionnaire (QLQ-C30).
Overall, 96.4% of the patients completed the PRO-CTCAE at their first visit, and 90.6% of those patients completed it again during a second visit 1 to 6 weeks later.
The median age of participants was 59 years, and 57.3% were women. Additionally, 32.4% of the patients had a high school education or less, and 17.1% had an ECOG performance status of 2 to 4.
At least one symptom was reported by 99.8% of the patients who completed at least one PRO-CTCAE questionnaire.
Every PRO-CTCAE item had at least one correlation in the expected direction with a QLQ-C30 scale (111 of 124; P < .05). Researchers reported stronger correlations between PRO-CTCAE items and conceptually related domains from QLQ-C30.
Results showed scores for 94 of 124 PRO-CTCAE items were higher among patients with ECOG performance status of 2 to 4, whereas scores for 58 of 124 PRO-CTCAE items were higher among patients with ECOG status of 0 or 1 (P < .05 for both).
Overall, 119 of the 124 items met at least one construct validity criterion, and test–retest reliability was 0.7 or more for 36 of the 49 pre-specified items (median range intraclass correlation coefficient = 0.76; 95% CI, 0.53-0.96).
Researchers reported statistically significant correlations between PRO-CTCAE item changes and corresponding QLQ-C30 scale changes for 27 prespecified items (median range = 0.43; 95% CI, 0.10-0.56).
“This is a landmark study demonstrating that meaningful information about adverse events can be elicited from patients themselves, which is a major step for advancing the patient-centeredness of clinical trials,” Ethan Basch, MD, MSc, director of University of North Carolina Lineberger Cancer Outcomes Research Program and associate professor of medicine and public health at UNC School of Medicine, told HemOnc Today. “The PRO-CTCAE initiative has involved a large multidisciplinary group of investigators, patients, clinicians and other stakeholders to develop a tool with the greatest possible rigor and meaning to patients. The NCI was visionary to support this research, which was formative to the current focus on patient-centered drug development.”
In an accompanying editorial, Benjamin Movsas, MD, radiation oncologist at Henry Ford Hospital in Detroit, noted that fewer than 4% of evaluated patients underwent cancer surgery, which indicates more research is necessary in that population.
Also, the study only included English-speaking patients, meaning future studies would be needed to focus on linguistic and cultural adaptations of the PRO-CTCAE.
Still, Movsas said the PRO-CTCAE is a monumental step toward improved communication between clinicians and their patients, as well as better selection of treatment regimens.
“In the future, the PRO-CTCAE may be used as a strategy to provide real-time information about patients’ symptoms so that clinicians can enhance their communication with patients regarding symptom management,” Movsas wrote. “One thing is reasonably clear: When it comes to optimally understanding and appreciating the patient experience, our patients want us to ‘PROceed with PROs.” – by Anthony SanFilippo
For more information:
Amylou C. Dueck, PhD, can be reached at Mayo Clinic, 13400 East Shea Blvd., Scottsdale, AZ 85259; email: dueck.amylou@mayo.edu.
Ethan Basch, MD, MSc, can be reached at UNC Lineberger Comprehensive Cancer Center, 101 Manning Drive, Chapel Hill, NC 27514; email: ebasch@email.unc.edu.
Disclosure: Dueck, Basch and several other researchers served as contracted investigators or study managers to develop the PRO-CTCAE for NCI. Movsas is co-chair of the NCI NRG patient-centered outcomes research committee. His department receives research funding from Philips Inc. and Varian Inc.
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