New Tool Makes It Easier for Patients in Clinical Trials to Report Side Effects

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It’s important for doctors and patients to discuss the side effects of treatment.

It’s important for doctors and patients to discuss the side effects of treatment.

The main role of clinical trials is to measure whether new cancer therapies shrink or control tumors. But increasingly, as cancer experts acknowledge the importance of patients’ quality of life, investigators are looking for ways to also scientifically evaluate the side effects of treatment.

“Over the past decade or so, we have increasingly recognized that we as clinicians under-detect some symptoms in our patients,” explains Ethan Basch, a medical oncologist and health outcomes researcher. “In clinical trials, this may mean that the burden of side effects on patients is underreported. Therefore, when we go to evaluate the risks and benefits of a new treatment, we often underestimate the risks.”

Previous studies have shown that the underreporting of side effects happens for a variety of reasons. Patients may focus on competing issues, such as test results, at appointments with their healthcare team and may also be reluctant to share their symptoms. Clinicians also may not fully document what patients tell them.

Some cancer patients may feel reluctant to discuss their side effects with their healthcare team.

Now a team funded by the National Cancer Institute (NCI) and led by investigators from Memorial Sloan Kettering and several other cancer centers has developed an effective system for measuring patients’ experiences during clinical trials. The group recently published an analysis of the tool’s effectiveness in the journal JAMA Oncology.

Validating a New System

The questionnaire — called the Patient Reported Outcomes version of the Common Terminology Criteria for Adverse Events, or PRO-CTCAE — asks patients 124 questions about 78 potential side effects, such as pain, fatigue, nausea, and skin problems. In the study, about 1,000 patients filled out the questionnaire once a week while they were in active treatment.

“The largest stakeholder in clinical research is the patient,” says Thomas Atkinson, head of the Behavioral Research Methods Core Facility at MSK and one of the study’s authors. “When we developed the questionnaire we worked with patients to determine which side effects they were able to measure and to develop simple, patient-friendly language that would allow us to capture these symptoms.”

The largest stakeholder in clinical research is the patient.
Thomas Atkinson outcomes researcher

The patients filled out the questionnaire before each of their appointments, including when they were seen for follow-up care. The researchers compared those results against already established ways to measure symptoms, including reports from clinicians, data on the characteristics of the patients’ tumors and treatments, and prescription information.

Statistical analysis comparing the information found that the PRO-CTCAE was accurate when compared with standard measurement tools. Researchers believe the tool also will greatly increase the likelihood that side effects will be conveyed to clinicians.

“A study that assesses this many symptoms is really unprecedented,” says Dr. Basch, an outcomes researcher at MSK who is also affiliated with the Lineberger Cancer Center at the University of North Carolina and is the study’s senior author. “The fact that we were able to validate 124 items is a methodological breakthrough in many ways.”

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Measuring Patients’ Experiences

“Patients who take part in clinical trials are active partners in the work and are very enthusiastic about participating in different aspects of research,” Dr. Basch adds. “The majority of patients who participated in this study and regularly filled out the questionnaire said that they found it valuable and that it helped them to have better discussions with their doctors and nurses.”

Patients who take part in clinical trials are [our] active partners.
Ethan Basch oncologist and outcomes researcher

Both researchers explain that once the new tool is adopted by the NCI, they expect it will be integrated into the clinical trials that the institute funds.

“This tool will impact clinical decision-making and may potentially influence which drugs are approved,” Dr. Atkinson says. “There may be situations where patients are expected to tolerate a treatment, yet they consistently report side effects that are severe enough to stop the treatment. On the flip side, there may be a drug that clinicians expect will be too toxic for patients to continue, and yet the patients report few or no side effects.”

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This study was funded by the National Cancer Institute under grants HHSN261200800043C, HHSN261201000063C, and HHSN261200800001E.