Patient questionnaires may add a new layer to chronic lymphocytic leukemia prognosis

Patients’ responses to questions routinely raised by oncologists may provide important clues about their chances of survival from chronic lymphocytic leukemia (CLL), according to a new study published in the journal Therapeutic Advances in Hematology.

The research suggests that carefully listening to patients and systematically evaluating what they say about their symptoms and daily functioning can help predict outcomes, improve prognosis and identify individuals at higher risk.

The study pooled individual patient data from 1,238 people with CLL enrolled in three major international randomized clinical trials and reanalyzed outcomes with ibrutinib-based treatment strategies.

Before treatment, participants completed a widely used cancer quality-of-life questionnaire that assessed physical functioning, fatigue, pain, shortness of breath, social functioning and other symptoms.

Rather than categorizing responses simply as “high” or “low,” the researchers applied internationally recognized thresholds to determine whether patients were experiencing problems severe enough to be clinically meaningful.

The study found that patient-reported outcomes have the potential to reveal hidden risks associated with the disease. The authors argue that patients’ own reports about their health could serve as a powerful new dimension to prognosis, treatment and risk stratification.

“This is already an interesting finding independently of survival,” said Dr. Ahmad Y. Abuhelwa, associate professor of pharmacotherapeutics at the University of Sharjah and the study’s lead author. “A large proportion of patients entering clinical trials had meaningful functional or symptom burdens that could be identified simply by asking them directly.”

Leukemia survival and patients’ voice

In 2021, more than 721,000 people were living with CLL worldwide, while the number of deaths attributed to the disease rose from 29,278 in 1990 to 45,573 in the same year.

The authors describe their work as the first comprehensive evaluation of the prevalence and clinical significance of clinically important patient-reported outcomes (PROs), defined using thresholds for clinical importance derived from the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), one of the most widely used instruments for assessing health-related quality of life among cancer patients.

The study is the result of a broad international collaboration involving researchers and clinicians from Harvard Medical School and the University of South Florida in the United States, Jordan University of Science and Technology, Flinders University in Australia and Qatar University. They worked alongside colleagues from the University of Sharjah, Burjeel Cancer Institute and Medcare Hospital Sharjah in the United Arab Emirates.

“We found that most patients (about three out of four) reported at least one area of poor functioning or symptom burden before starting therapy,” the authors write. “Those with more impaired areas, especially five or more, had a higher chance of experiencing severe side effects and a shorter overall survival.”

The researchers note that poor physical functioning emerged as the strongest predictor of adverse outcomes. According to the study, patients’ own assessment of their health may reveal important health risks that are not captured by conventional clinical measures alone.

“Using PRO scores in routine care could help doctors identify higher-risk patients, offer earlier support and personalize treatment plans to improve both quality of life and (clinical) outcomes,” they note.

Patient perspective matters for prognosis

The findings are particularly relevant to health care providers, cancer centers, clinical groups, patient advocacy organizations and developers of electronic patient-reported outcome systems.

As health care increasingly embraces digital symptom monitoring and patient-centered care, the ability to translate patient-reported information into meaningful risk indicators is likely to attract growing interest beyond academia.

“The study suggests that asking patients directly about how they are functioning and feeling may provide clinically valuable information that complements what clinicians can obtain from routine clinical assessments alone,” Abuhelwa said.

“The clinical implication is that a short patient questionnaire before treatment could potentially help clinicians identify patients who are more vulnerable and may need closer monitoring, supportive care, symptom management, functional interventions or more individualized treatment planning.”

One of the study’s key strengths is the relative ease with which patient-reported outcomes can be collected, either using questionnaires administered in clinical settings or electronically before appointments. In practice, patients could complete a short, standardized assessment before or during a consultation, enabling clinical teams to identify those experiencing a particularly high burden of symptoms or functional limitations.

Abuhelwa sees these findings as an opportunity to integrate patient-reported data with existing clinical and molecular risk factors, thereby creating a more comprehensive understanding of each patient’s condition.

“Our study does not suggest that treatment decisions should currently be made solely on questionnaire scores,” he concludes. “The results require prospective validation before a specific patient-reported outcome threshold can be considered a stand-alone clinical decision-making tool. Rather, the findings underscore the potential value of incorporating the patient’s perspective into established clinical assessments.”

Who’s behind this story?

Sadie Harley

BSc Life Sciences & Ecology. Microbiology lab background with pharmaceutical news experience in oil, gas, and renewable industries.

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Andrew Zinin

Master’s in physics with research experience. Long-time science news enthusiast. Plays key role in Science X’s editorial success.

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