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

Objectives: This 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 Setting: We 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. Results: Of 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. Conclusion: International 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. (c) 2023 The Author(s). Published by Elsevier Inc. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).