Abstract
BackgroundInterpretation of differences or changes in patient-reported outcome scores should not only consider statistical significance, but also clinical relevance. Accordingly, accurate determination of the minimally important difference (MID) is crucial to assess the effectiveness of health care interventions, as well as for sample size calculation. Several methods have been proposed to determine the MID. Our aim was to review the statistical methods used to determine MID in patient-reported outcome (PRO) questionnaires in cancer patients, focusing on the distribution- and anchor-based approaches and to present the variability of criteria used as well as possible limitations.MethodsWe performed a systematic search using PubMed. We searched for all cancer studies related to MID determination on a PRO questionnaire. Two reviewers independently screened titles and abstracts to identify relevant articles. Data were extracted from eligible articles using a predefined data collection form. Discrepancies were resolved by discussion and the involvement of a third reviewer.ResultsSixty-three articles were identified, of which 46 were retained for final analysis. Both distribution- and anchor-based approaches were used to assess the MID in 37 studies (80.4%). Different time points were used to apply the distribution-based method and the most frequently reported distribution was the 0.5 standard deviation at baseline. A change in a PRO external scale (N = 13, 30.2%) and performance status (N = 15, 34.9%) were the most frequently used anchors. The stability of the MID over time was rarely investigated and only 28.2% of studies used at least 3 assessment timepoints. The robustness of anchor-based MID was questionable in 37.2% of the studies where the minimal number of patients by anchor category was less than 20.ConclusionEfforts are needed to improve the quality of the methodology used for MID determination in PRO questionnaires used in oncology. In particular, increased attention to the sample size should be paid to guarantee reliable results. This could increase the use of these specific thresholds in future studies.
Highlights
The use of patient-reported outcomes (PRO), including health-related quality of life (HRQOL), in cancer clinical trials has substantially increased over the years [1]
A statistically significant result may not be clinically relevant, as it should reflect changes or differences that are meaningful for the patient, i.e., they should take into account a minimally important difference (MID)
After reading the full text of the remaining 49 articles, three additional articles were excluded as they explored cut-off scores and not MID
Summary
The use of patient-reported outcomes (PRO), including health-related quality of life (HRQOL), in cancer clinical trials has substantially increased over the years [1]. A statistically significant result may not be clinically relevant, as it should reflect changes or differences that are meaningful for the patient, i.e., they should take into account a minimally important difference (MID). The determination of the MID is crucial in order to assess the effectiveness of health care interventions, as well as for sample size calculation when HRQOL is the primary or co-primary endpoint in clinical trials. Interpretation of differences or changes in patient-reported outcome scores should consider statistical significance, and clinical relevance. Accurate determination of the minimally important difference (MID) is crucial to assess the effectiveness of health care interventions, as well as for sample size calculation. Our aim was to review the statistical methods used to determine MID in patient-reported outcome (PRO) questionnaires in cancer patients, focusing on the distribution- and anchor-based approaches and to present the variability of criteria used as well as possible limitations
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