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Krepper, D.; Giesinger, J.M.; Dirven, L.; Efficace, F.; Martini, C.; Thurner, A.M.M.; ... ; Sztankay, M.J. (2023)

Information about missing patient-reported outcome data in breast cancer trials is frequently not documented: a scoping review

Article / Letter to editor
ObjectivesThis review addresses the common problem of missing patient-reported outcome (PRO) data in clinical trials by assessing the current practice of their statistical handling as reported in publications of randomized controlled trials (RCTs) in patients with breast cancer.Study Design and SettingWe searched PubMed to identify RCTs evaluating biomedical treatments in breast cancer patients with at least one PRO endpoint published between January 2019 and February 2022. Two reviewers independently assessed the eligibility of the publications for this scoping review and extracted prespecified information on missing PRO data and related statistical practices.ResultsOf 1,598 publications identified, 118 trials met the inclusion criteria. Eighty-eight (74.6%) trials reported the extent of missing data, with 11 (9.3%) not containing any missing PRO data. Twenty-one (19.6%) trials explicitly stated the statistical approach for...Show moreObjectivesThis review addresses the common problem of missing patient-reported outcome (PRO) data in clinical trials by assessing the current practice of their statistical handling as reported in publications of randomized controlled trials (RCTs) in patients with breast cancer.Study Design and SettingWe searched PubMed to identify RCTs evaluating biomedical treatments in breast cancer patients with at least one PRO endpoint published between January 2019 and February 2022. Two reviewers independently assessed the eligibility of the publications for this scoping review and extracted prespecified information on missing PRO data and related statistical practices.ResultsOf 1,598 publications identified, 118 trials met the inclusion criteria. Eighty-eight (74.6%) trials reported the extent of missing data, with 11 (9.3%) not containing any missing PRO data. Twenty-one (19.6%) trials explicitly stated the statistical approach for handling missing data, with a preference for single imputation over multiple imputation approaches (57.2%/19.0%). Only six (5.6%) trials reported a sensitivity analysis to examine the extent to the results being affected by changes in assumptions made about missing PRO data.ConclusionInternational efforts to raise awareness of the importance of accurately reporting state-of-the-art handling of missing PRO data are not yet fully reflected in the current literature of breast cancer RCTs.Show less
Patient-reported outcomes
Missing data
Imputation
Sensitivity analysis
Breast cancer
Randomized controlled trials
All authors
Krepper, D.; Giesinger, J.M.; Dirven, L.; Efficace, F.; Martini, C.; Thurner, A.M.M.; Al-Naesan, I.; Gross, F.; Sztankay, M.J.
Date
2023-08-26
Journal
Journal of Clinical Epidemiology
Volume
162
Pages
1 - 9
DOI
doi:10.1016/j.jclinepi.2023.07.012
Link
https://www.jclinepi.com/article/S0895-4356(23)00184-1/fulltext