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Collection of patient-reported symptoms, adverse events ‘highly feasible’ during clinical trials
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Patient reports of their own symptomatic adverse events during multicenter clinical cancer trials proved possible and led to improved efficacy and safety monitoring in clinical research, according to results of a prospective study.
“It is highly feasible to collect patient-reported symptoms and adverse events in multicenter trials,” Ethan Basch, MD, MSc, director of UNC Lineberger Cancer Outcomes Research Program, told HemOnc Today. “Most patients are willing and able to do this, and there are multiple successful strategies for collecting this information.”
It is standard practice for clinical researchers to report patients’ adverse events in clinical trials via the Common Terminology Criteria for Adverse Events (CTCAE) database. However, clinicians may miss up to half of the symptomatic adverse events, leading to lower reliability.
Patient-reported outcomes (PROs) — the standard for trials of health-related quality of life, physical functioning and disease-related symptoms — may help to improve reliability of adverse event reporting. Although becoming more common, PRO reporting is not standard in clinical trials.
Therefore, Basch and colleagues conducted a study of patients from nine U.S. multicenter cancer treatment clinical trials to assess feasibility of PRO collection.
“Clinical investigators miss up to half of patients’ symptomatic adverse events — nausea, neuropathy, dysphagia and fatigue — [and] we miss many of the low-grade toxicities, tend to downplay or even miss the higher-grade events, and miss most of patients’ baseline symptoms, which we later may incorrectly attribute to [the] study drug,” Basch said. “Patient self-reporting fills out the picture of the patient experience with cancer drugs, and I believe that without patient self-reports of adverse events, we cannot truly understand a drug’s impact on patients, and any attempts to balance risk with benefits are done with incomplete information.”
Between March 2007 and August 2011, 285 patients (median age, 57 years; range, 24-88) were invited to self-report 13 common symptomatic adverse events using a PRO adaptation of the CTCAE database via tablet computers at five clinical visits. Among the patients, 202 (74.3%) were women, 241 (85.5%) were white, 73 (26.8%) had a high school education or less, and 176 (64.7%) reported regular internet use.
The researchers tracked patient adherence and reasons for missed self-reports.
Patients completed self-reports for 1,202 of 1,280 visits, for an overall adherence of 93.9%.
An electronic system — such as a handheld device, web-based app or automated telephone system — is the most ideal method for PRO collection, Basch said. However, paper questionnaires in clinic also can be used, and a study team can monitor compliance in real-time and call patients who don’t self-report on time.
“This strategy can bump questionnaire compliance rates by 10% to 15%,” Basch said.
There were no differences in age or ethnicity between patients who were willing and able to self-report compared with those who were not. However, researchers noted it was difficult to compare nonreporting patients because a majority of patients regularly provided self-reports.
Reasons for missing PRO submission included institutional errors (56.3%), patients feeling ill (16.7%), patient refusal (16.7%) and internet connectivity problems (10.4%).
“In cancer clinical trials, generally between 85% to 95% of patients will self-report their own symptoms and adverse events at any given time, even among those who are elderly, less computer savvy or physically ill,” Basch said. “When the research team works hard to engage patients and emphasize the importance of the patient-reported information, there can be up to 100% patient compliance, whereas teams that don’t emphasize importance or don’t accommodate patients will yield much lower numbers into the 40% to 50% range.”
Researchers analyzed weighted statistics to analyze the agreement with clinician-reported adverse events.
The patient–investigator CTCAE agreement was moderate or worse for most symptoms (most <0.05). Investigators reported fewer adverse events than patients across symptoms.
Questionnaires showed that most patients believed the system was easy to use (93.2%) and useful 93.1%). Among the investigators, 94.3% believed the system was useful and 83.2% believed it was accurate.
This study shows patients may be able to bridge the gap and become an important source of adverse events reporting in clinical trials, Zaina P. Qureshi, PhD, MPH, assistant professor in the department of health services policy and management at Arnold School of Public Health of University of South Carolina, and colleagues wrote in a related editorial.
“However, the study ... leaves open an important question: are patients and clinicians similar in their assessments of these toxic effects?” Qureshi and colleagues wrote. “... It is hoped that the next generation of self-reporting of adverse events will provide empirical data on settings where patients and clinicians differ in their assessments of occurrence and severity of an adverse event.” – by Melinda Stevens
Disclosure: The researchers report no relevant financial disclosures. Qureshi and colleagues report no disclosures.
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