Study cites importance of patient self-reports

Oncology patients' AE reports studied

Patient self-reporting has been successfully used for purposes of evaluating quality of life, satisfaction with care, and looking at symptom end points in clinical trials, but questions have remained about whether or not patient self reports are useful in collecting adverse event data.

One new study has found that the answer is an enthusiastic "yes."

The study asked for symptoms reports by lung cancer patients receiving chemotherapy and compared their reports with those made by their clinicians for six items pertaining to the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE).1

Researchers found that clinician CTCAE assessments were better at predicting unfavorable clinical events, such as emergency room visits and death, but that patient reports provided a better assessment of daily health status.1

"The method of what we call patient-reported outcomes (PROs) is very well-established and quite widespread in research," says Ethan M. Basch, MD, a medical oncologist with Memorial Sloan-Kettering Cancer Center in New York, NY. Basch is a co-author of the study on using PROs for adverse event data collection.

"This is really a new application of that approach with some methodological and technological refinement," Basch says. "I do think it's feasible to implement this strategy on a widespread basis because we can improve our understanding of the toxicities."

The recent study is part of a broader research program of looking at patient self-reporting, Basch says.

"We're looking to standardize the approach," he adds.

Both clinician reports and patient reports provide useful information, Basch notes.

"The final model remains to be fully developed," he says. "This is really a methods paper that describes the usefulness of information provided by patients and clinicians."

One possible drawback to relying on patient self-reports is that patients might have a low ceiling for their self-report ratings because they haven't had enough experience to put a particular symptom into perspective, critics suggest.

For example, a patient might rate his pain a 10 out of 10, saying this is as bad as it can be, while the clinician would rate it an eight, knowing from experience that the patient's pain will get worse.2

This argument involves response shift or recalibration, which suggests a patient might change his or her orientation about severity based on context, Basch says.

"So when first starting treatment a patient might have one perspective about what the worst thing that could happen to him could be, and that could change," he explains. "In the setting of controlled clinical trials, the ultimate context for these approaches, these sorts of shifts are accounted for and presumably balanced between arms."

Also, the severity of the response shift is an important dimension of the patient's self-report of their experience, and it's not captured by the clinician because he doesn't have the benefit of a changing perception, Basch adds.

"While it's true there's a response shift and clinicians have an overview of how bad things could be, that likely leads clinicians to downgrade the severity of patients' symptoms based on how bad they think it will be," he says. "And that's probably an unfair downgrading of patients' symptoms at any one point in time."

Just as a very complex host of phenomenon contribute to a patient's self report, there also is a very complex group of factors contributing to a clinician's report, Basch says.

"This may include contextualizing a patient along a continuum of patients that the clinician has seen," he says.

For example, a clinician's context could change his or her report of the following:

  • Is the patient particularly stoic?
  • Does the patient overstate severity?
  • Does the patient's health status make him or her ineligible for a trial?

"The final methodological piece is that it's important to have a mechanism for minimizing missing data," Basch says.

"One could imagine that patients who are particularly ill might not report as regularly, and we don't want to bias our data as a result of that," he explains. "So we have to have back-up data collection methods."

A back-up method could be using an automated telephone system to call patients regularly, screening for adverse symptoms, Basch says.

"If a patient doesn't answer there's a second call, and for the third call a nurse will call the patient," he adds. "If the patient still doesn't respond then the nurse might call a family member because the patient is in trouble."

When cost is a consideration, clinical trial sites could use inexpensive technologies, such as Web sites that harness the patient's own telephones and computers to collect the information.

"It might save in cost because the time spent by physicians in eliciting and abstracting adverse symptom information could be transitioned over to the patient's side," Basch says. "And if one uses electronic data capture this information would be directly imported into a research database."

The electronic data capture via Internet would work by having an Internet-based questionnaire available on a touch screen in which the patient would log in and enter his or her own toxicity information. It's then sent directly to the electronic database, he explains.

"We've been doing this research now for six to seven years, and we have found that patient familiarity with self-entering data by computer and telephone has changed fundamentally," Basch says. "So many of us do our banking via automated telephone systems or computers, and we get movie tickets this way, so these are ubiquitous technology."

Investigators who fear their patients might not want to use the technology for self-reported outcomes should know that their patients likely already are exchanging this kind of information with each other online through support communities, he adds.

"Particularly patients who are elderly and frail are very comfortable with using this technology to report their own health information," Basch says.

The benefits of using new technology to collect patient reported outcomes outweighs the risks pertaining to patients' discomfort with the technology or potential security issues, he says.

The logistics and security systems can be designed to ensure only the patient responds to the questions, Basch says.

"If the security of these technologies is satisfactory for the banking industry, it probably should be satisfactory for this context as well," he adds. "We have the opportunity to harness patients' enthusiasm for this information and improve the efficiency of clinical trials and quality of information."


  1. Basch E, Jia X, Heller G, et al. Adverse symptom event reporting by patients vs clinicians: relationships with clinical outcomes. JNCI. 2009;101:1-9.
  2. Gotay C. Patient symptoms and clinician toxicity ratings: both have a role in cancer care. JNCI. 2009;101(23):(Editorial)1-2.