Statistical methods and graphical displays of quality of life with survival outcomes in oncology clinical trials for supporting the estimand framework
Abstract Background Although there are discussions regarding standards of the analysis of patient-reported outcomes and quality of life (QOL) in oncology clinical trials, that of QOL with death events is not within their scope. For example, ignoring death can lead to bias in the QOL analysis for pat...
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| Format: | Article |
| Language: | English |
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BMC
2022-10-01
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| Series: | BMC Medical Research Methodology |
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| Online Access: | https://doi.org/10.1186/s12874-022-01735-1 |
| _version_ | 1811226662495322112 |
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| author | Kentaro Sakamaki Takuya Kawahara |
| author_facet | Kentaro Sakamaki Takuya Kawahara |
| author_sort | Kentaro Sakamaki |
| collection | DOAJ |
| description | Abstract Background Although there are discussions regarding standards of the analysis of patient-reported outcomes and quality of life (QOL) in oncology clinical trials, that of QOL with death events is not within their scope. For example, ignoring death can lead to bias in the QOL analysis for patients with moderate or high mortality rates in the palliative care setting. This is discussed in the estimand framework but is controversial. Information loss by summary measures under the estimand framework may make it challenging for clinicians to interpret the QOL analysis results. This study illustrated the use of graphical displays in the framework. They can be helpful for discussions between clinicians and statisticians and decision-making by stakeholders. Methods We reviewed the time-to-deterioration analysis, prioritized composite outcome approach, semi-competing risk analysis, survivor analysis, linear mixed model for repeated measures, and principal stratification approach. We summarized attributes of estimands and graphs in the statistical analysis and evaluated them in various hypothetical randomized controlled trials. Results Graphs for each analysis method provide different information and impressions. In the time-to-deterioration analysis, it was not easy to interpret the difference in the curves as an effect on QOL. The prioritized composite outcome approach provided new insights for QOL considering death by defining better conditions based on the distinction of OS and QOL. The semi-competing risk analysis provided different insights compared with the time-to-deterioration analysis and prioritized composite outcome approach. Due to the missing assumption, graphs by the linear mixed model for repeated measures should be carefully interpreted, even for descriptive purposes. The principal stratification approach provided pure comparison, but the interpretation was difficult because the target population was unknown. Conclusions Graphical displays can capture different aspects of treatment effects that should be described in the estimand framework. |
| first_indexed | 2024-04-12T09:28:53Z |
| format | Article |
| id | doaj.art-39cb4cdea4934ff9a45cb964a89c959b |
| institution | Directory Open Access Journal |
| issn | 1471-2288 |
| language | English |
| last_indexed | 2024-04-12T09:28:53Z |
| publishDate | 2022-10-01 |
| publisher | BMC |
| record_format | Article |
| series | BMC Medical Research Methodology |
| spelling | doaj.art-39cb4cdea4934ff9a45cb964a89c959b2022-12-22T03:38:24ZengBMCBMC Medical Research Methodology1471-22882022-10-0122111110.1186/s12874-022-01735-1Statistical methods and graphical displays of quality of life with survival outcomes in oncology clinical trials for supporting the estimand frameworkKentaro Sakamaki0Takuya Kawahara1Center for Data Science, Yokohama City UniversityClinical Research Promotion Center, The University of Tokyo HospitalAbstract Background Although there are discussions regarding standards of the analysis of patient-reported outcomes and quality of life (QOL) in oncology clinical trials, that of QOL with death events is not within their scope. For example, ignoring death can lead to bias in the QOL analysis for patients with moderate or high mortality rates in the palliative care setting. This is discussed in the estimand framework but is controversial. Information loss by summary measures under the estimand framework may make it challenging for clinicians to interpret the QOL analysis results. This study illustrated the use of graphical displays in the framework. They can be helpful for discussions between clinicians and statisticians and decision-making by stakeholders. Methods We reviewed the time-to-deterioration analysis, prioritized composite outcome approach, semi-competing risk analysis, survivor analysis, linear mixed model for repeated measures, and principal stratification approach. We summarized attributes of estimands and graphs in the statistical analysis and evaluated them in various hypothetical randomized controlled trials. Results Graphs for each analysis method provide different information and impressions. In the time-to-deterioration analysis, it was not easy to interpret the difference in the curves as an effect on QOL. The prioritized composite outcome approach provided new insights for QOL considering death by defining better conditions based on the distinction of OS and QOL. The semi-competing risk analysis provided different insights compared with the time-to-deterioration analysis and prioritized composite outcome approach. Due to the missing assumption, graphs by the linear mixed model for repeated measures should be carefully interpreted, even for descriptive purposes. The principal stratification approach provided pure comparison, but the interpretation was difficult because the target population was unknown. Conclusions Graphical displays can capture different aspects of treatment effects that should be described in the estimand framework.https://doi.org/10.1186/s12874-022-01735-1Quality of lifeTruncation by deathEstimand frameworkGraphical displaysPrioritized composite outcomeSemi-competing risk analysis |
| spellingShingle | Kentaro Sakamaki Takuya Kawahara Statistical methods and graphical displays of quality of life with survival outcomes in oncology clinical trials for supporting the estimand framework BMC Medical Research Methodology Quality of life Truncation by death Estimand framework Graphical displays Prioritized composite outcome Semi-competing risk analysis |
| title | Statistical methods and graphical displays of quality of life with survival outcomes in oncology clinical trials for supporting the estimand framework |
| title_full | Statistical methods and graphical displays of quality of life with survival outcomes in oncology clinical trials for supporting the estimand framework |
| title_fullStr | Statistical methods and graphical displays of quality of life with survival outcomes in oncology clinical trials for supporting the estimand framework |
| title_full_unstemmed | Statistical methods and graphical displays of quality of life with survival outcomes in oncology clinical trials for supporting the estimand framework |
| title_short | Statistical methods and graphical displays of quality of life with survival outcomes in oncology clinical trials for supporting the estimand framework |
| title_sort | statistical methods and graphical displays of quality of life with survival outcomes in oncology clinical trials for supporting the estimand framework |
| topic | Quality of life Truncation by death Estimand framework Graphical displays Prioritized composite outcome Semi-competing risk analysis |
| url | https://doi.org/10.1186/s12874-022-01735-1 |
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