ArticleJNCI cancer spectrum2023
Assessing the impact of open-label designs in patient-reported outcomes: investigation in oncology clinical trials.
Article in JNCI cancer spectrum, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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13 citing papers in PubMed.
- Patient-reported outcomes in patients with paroxysmal nocturnal hemoglobinuria treated with crovalimab and approved C5 inhibitors in the phase III COMMODORE 2 and 1 studies.Annals of hematology · 2025Trial
- Patient-reported outcomes from DESTINY-Breast04: trastuzumab deruxtecan versus physician's choice of chemotherapy in patients with HER2-low mBC.The oncologist · 2025Trial
- Trial
- Patient-reported improvements in paroxysmal nocturnal hemoglobinuria treated with iptacopan from 2 phase 3 studies.Blood advances · 2025Trial
- Home-based high tone therapy may alleviate chemotherapy-induced neuropathic symptoms in patients with colorectal cancer: A randomized double-blind placebo-controlled pilot evaluation.Supportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer · 2024Trial
- Pharmacist-led, digitally enabled transitional care after acute myocardial infarction: design and baseline characteristics of the AMI-HOPE nationwide stepped-wedge trial.European heart journal. Digital health · 2026Article
- Article
- Anlotinib and penpulimab in advanced hepatocellular carcinoma: a new contender emerges.Journal of gastrointestinal oncology · 2026Article
- Assessing the patient reported outcomes (PROs) of enfortumab vedotin plus pembrolizumab versus platinum-based chemotherapy in patients with advanced urothelial carcinoma in the EV-302 study.Translational andrology and urology · 2026Article
- Review
- Study design of the phase IV, randomized, placebo-controlled REMOdeling with Dupilumab in Eosinophilic esophagitis Long-term (REMODEL) trial.Therapeutic advances in gastroenterology · 2025Article
- Review
- Double-blinded is not better than "mutually enlightened": implications of Lord-Besson et al.JNCI cancer spectrum · 2023Article
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5 authors.
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No grant is acknowledged in the PubMed record.
Abstract
backgroundKnowledge of treatment assignment may affect patient-reported outcomes (PROs), which is of concern in oncology, where open-label trials are common. This study measured the magnitude of open-label bias by comparing PROs for similar patient groups in oncology trials with different degrees of concealment.
methodsIndividual patient data from ipilimumab arms of 2 melanoma and docetaxel arms of 2 non-small cell lung cancer (NSCLC) trials were adjusted for differences using propensity score weighting. Patients were aware of treatment assignment in CA184-022 and CheckMate 057 (open-label) but not in MDX010-20 and VITAL (blinded). Overall survival (OS) and mean changes from baseline to week 12 in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (melanoma) and Lung Cancer Symptom Scale (NSCLC) scores were compared between open-label and blinded groups.
resultsAfter adjustment, baseline characteristics were balanced between blinded (melanoma, n = 125; NSCLC, n = 424) and open-label (melanoma, n = 69; NSCLC, n = 205) groups. Study discontinuation and PRO completion rates at week 12 and OS were similar. There was no clear direction in differences in change scores between groups. In the melanoma trials, role functioning (mean = -5.2, 95% confidence interval [CI] = -15.4 to 5.0), global health status (mean = -1.3, 95% CI = -8.7 to 6.1), and pain (mean = 6.2 , 95% CI = -1.8 to 14.2) favored the blinded, whereas emotional functioning (mean = 2.2, 95% CI = -5.8 to 10.2) and diarrhea (mean = -8.3, 95% CI = -17.3 to 0.7) favored the open-label group. In the NSCLC trials, changes in dyspnea (mean = 5.4, 95% CI = -0.7 to 11.5) favored the blinded and changes in appetite (mean = -1.2, 95% CI = -8.1 to 5.7) favored the open-label group. None were clinically or statistically significant.
conclusionsThis study adds to the growing evidence demonstrating that concerns regarding open-label bias should not prohibit the interpretation of large and meaningful treatment effects on PROs.
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