Patient-reported outcomes (PROs) in oncology trials are similar whether the trials have open-label or blinded designs, according to research published in JNCI Cancer Spectrum.
Researchers found no significant differences in PRO measures between open-label and blinded trials of patients with melanoma or non-small cell lung cancer (NSCLC).
For this study, researchers compared an open-label trial and a blinded trial of melanoma patients, as well as an open-label trial and a blinded trial of NSCLC patients.
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In the melanoma cohort, there were 69 evaluable patients from the open-label trial and 125 evaluable patients from the blinded trial. In the NSCLC cohort, there were 205 evaluable patients from the open-label trial and 424 evaluable patients from the blinded trial.
The researchers obtained PRO measurements using the European Organization for Research and Treatment of Cancer (EORTC) quality of life questionnaire-core 30 (QLQ-C30) and the Lung Cancer Symptoms Scale (LCSS).
The researchers noted that, after adjustment, baseline characteristics were balanced between the blinded and open-label groups. For both the melanoma and NSCLC patients, the 12-week study discontinuation and PRO completion rates were similar between the open-label and blinded cohorts.
There were no significant differences in PRO scores between the open-label and blinded cohorts. However, there were trends favoring either cohort for individual PRO measures.
Melanoma Trial Results
In the melanoma trials, both before and after adjustment, there were no significant differences in the change in QLQ-C30 scores from baseline to week 12 between the open-label and blinded groups (P ≥ .05 for all).
However, changes in role functioning (mean difference between the groups, -5.2), physical functioning (-3.3), global health status (-1.3), and social functioning (-0.3) were slightly worse in the open-label group. Changes in emotional functioning (2.2) and cognitive functioning (1.1) were slightly better in the open-label group.
The researchers also found that changes in insomnia (-7.9), constipation (-2.5), and diarrhea (-8.3) were slightly better for the open-label group. However, changes in fatigue (0.9), nausea and vomiting (2.7), pain (6.2), dyspnea (2.0), and appetite loss (4.6) were slightly worse for the open-label group.
NSCLC Trial Results
In the NSCLC trials, both before and after adjustment, there were no significant differences in the change in LCSS scores between the open-label and blinded groups (P ≥ .05 for all).
After adjustment, changes in appetite (-1.2) and hemoptysis (-1.0) were slightly better in the open-label group.
However, changes in fatigue (1.9), cough (1.3), dyspnea (5.4), pain (5.4), symptom distress (1.2), activity level (0.8), health-related quality of life (4.8), and average symptom burden index (1.9) were slightly worse in the open-label group.
“This study adds to the growing body of evidence demonstrating that concerns regarding open-label bias should not prohibit the interpretation of large and meaningful treatment effects on PROs,” the researchers concluded.
Disclosures: This research was supported by Bristol Myers Squibb Company. Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.
Reference
Lord-Bessen J, Signorovitch J, Yang M, Georgieva M, Roydhouse J. Assessing the impact of open-label designs in patient-reported outcomes: Investigation in oncology clinical trials. JNCI Cancer Spectr. Published online January 20, 2023. doi:10.1093/jncics/pkad002
