Landmark study validates method for patient reporting of cancer drug-related adverse events

In an article published in JAMA Oncology today, a study led by UNC Lineberger's Ethan Basch and colleagues shows that a system they developed accurately and reliably captures the patient experience with cancer drug side effects.

Landmark study validates method for patient reporting of cancer drug-related adverse events click to enlarge Ethan Basch, MD, MSc, is director of the UNC Lineberger Cancer Outcomes Research Program and associate professor of medicine and public health at the UNC School of Medicine.

Media Contact: Laura Oleniacz, 919-445-4219, laura_oleniacz@med.unc.edu

In cancer clinical trials, symptom side effects patients experience, like nausea, are typically reported by doctors, and not directly by patients. Previous research has shown that doctors under-report these symptoms.

Ethan Basch, MD, MSc, director of the UNC Lineberger Cancer Outcomes Research Program and associate professor of medicine and public health at the UNC School of Medicine, has led studies and national initiatives to enable patients to directly report their own symptoms during cancer drug development. And in an article published in JAMA Oncology today, a study led by Basch and colleagues for the National Cancer Institute shows that a system they developed accurately and reliably captures the patient experience with cancer drug side effects.

“This is a landmark study because patient-reported information has not been used for measuring side effects in cancer research,” said Basch, the paper’s senior author. “This study shows that these patient-reported measures perform well, and are ready for implementation in cancer research.”

The study tested the reliability and validity of a group of measures previously developed by Basch and colleagues for the National Cancer Institute to test patient-reported adverse events. Basch as well as other authors were contracted to develop the measures, which are called the Patient Reported Outcomes version of the Common Terminology Criteria for Adverse Events, or PRO-CTCAE.

The researchers tested 124 of the measures that had been developed for the NCI to question patients about 78 potential adverse events. The study involved nearly 1,000 patients who were undergoing outpatient chemotherapy, radiation therapy or both at one of nine U.S. cancer centers or oncology practices

“In order to establish the validity of the 124 questions in the survey we developed using the NCI measures, we conducted statistical analyses to compare the new measurement tools to other metrics, such as to clinician reports of patient function and to data on the underlying characteristics of the patients’ cancer and their treatments,” Basch said.

The study found that the PRO-CTCAE measures performed extremely well, demonstrating validity and reliability across items.

Basch said that the study is important as it validates measures developed to obtain reports of adverse events directly from patients. He noted a study by researchers in Europe published earlier this year in the Journal of Clinical Oncology found that doctors under-report side effects by as much as half in cancer trials.  (Di Maio, et al. 2015)

“Patient reporting improves the quality of information that we have about how patients feel and function, and it provides a fuller picture of the impact of treatments on people’s lives,” Basch said. “This is important because in risk-benefit balancing for drugs, we need to have comprehensive and valid information about risk.”

The study was supported by contracts from the National Cancer Institute.

In addition to Basch, the other authors include: first and corresponding author Amylou C. Dueck, PhD, Department of Health Sciences at the Mayo Clinic in Arizona; Tito R. Mendoza, PhD, of the Department of Symptom Research at the University of Texas MD Anderson Cancer Center; Sandra A. Mitchell, PhD, CRNP, AOCN, of the Division of Cancer Control and Population Sciences at the National Cancer Institute; Bryce B. Reeve, PhD, of UNC Lineberger; Kathleen M. Castro, RN, MS, AOCN, of the Division of Cancer Control and Population Sciences at the National Cancer Institute; Lauren J. Rogak, MA, of the Department of Epidemiology and Biostatistics at the Memorial Sloan Kettering Cancer Center; Thomas M. Atkinson, PhD, of the Department of Psychiatry and Behavioral Sciences at the Memorial Sloan Kettering Cancer Center; Antonia V. Bennett, PhD, of UNC Lineberger; Andrea M. Denicoff, MS, RN, ANP, of the Division of Cancer Treatment and Diagnosis at the National Cancer Institute; Ann M. O’Mara, PhD, RN, FAAN, of the Division of Cancer Prevention at the National Cancer Institute; Yuelin Li, PhD, of the Department of Psychiatry and Behavioral Sciences at the Memorial Sloan Kettering Cancer Center; Steven B. Clauser, PhD, MPA, of the Division of Cancer Control and Population Sciences at the National Cancer Institute; Donna M. Bryant, MSN, ANP-BC, OCN, CCRC, of the Department of Clinical Research at the Cancer Program of our Lady of the Lake and Mary Bird Perkins; James D. Bearden III, MD, FACP, of the Gibbs Cancer Center and Research Institute; Theresa A. Gillis, MD, of the Helen F. Graham Cancer Center and Research Institute at the Christiana Care Health System; Jay K. Harness, MD, of the Center for Cancer Prevention and Treatment at the St. Joseph Hospital of Orange; Robert D. Siegel, MD, FACP, of the Helen and Harry Gray Cancer Center at Hartford Hospital; Diane B. Paul, AAS, a patient advocate and cancer survivor; Charles S. Cleeland, PhD, Department of Symptom Research at the University of Texas MD Anderson Cancer Center; Deborah Schrag, MD, MPH, of the Division of Population Sciences at the Dana-Farber Cancer Institute; Jeff A. Sloan, PhD, of the Department of Health Sciences Research at the Mayo Clinic in Rochester; Amy P. Abernethy, MD, PhD, of the Duke University Medical Center Department of Medicine; Deborah W. Bruner, RN, PhD, FAAN, of the Nell Hodgson Woodruff School of Nursing at Emory University; and Lori M. Minasian, MD, FACP, of the Division of Cancer Treatment and Diagnosis at the National Cancer Institute.