Adverse Event Burden Score—A Versatile Summary Measure for Cancer Clinical Trials
This article introduces the adverse event (AE) burden score. The AE burden by treatment cycle is a weighted sum of all grades and AEs that the patient experienced in a cycle. The overall AE burden score is the total AE burden the patient experienced across all treatment cycles. AE data from two comp...
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| Format: | Article |
| Language: | English |
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MDPI AG
2020-11-01
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| Series: | Cancers |
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| Online Access: | https://www.mdpi.com/2072-6694/12/11/3251 |
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| author | Jennifer G. Le-Rademacher Shauna Hillman Elizabeth Storrick Michelle R. Mahoney Peter F. Thall Aminah Jatoi Sumithra J. Mandrekar |
| author_facet | Jennifer G. Le-Rademacher Shauna Hillman Elizabeth Storrick Michelle R. Mahoney Peter F. Thall Aminah Jatoi Sumithra J. Mandrekar |
| author_sort | Jennifer G. Le-Rademacher |
| collection | DOAJ |
| description | This article introduces the adverse event (AE) burden score. The AE burden by treatment cycle is a weighted sum of all grades and AEs that the patient experienced in a cycle. The overall AE burden score is the total AE burden the patient experienced across all treatment cycles. AE data from two completed Alliance multi-center randomized double-blind placebo-controlled trials, with different AE profiles (NCCTG 97-24-51: 176 patients, and A091105: 83 patients), were utilized for illustration. Results of the AE burden score analyses corroborated the trials’ primary results. In 97-24-51, the overall AE burden for patients on the treatment arm was 2.2 points higher than those on the placebo arm, with a higher AE burden for patients who went off treatment early due to AE. Similarly, in A091105, the overall AE burden was 1.6 points higher on the treatment arm. On the placebo arms, the AE burden in 97-24-51 remained constant over time; and increased in later cycles in A091105, likely attributable to the increase in disease morbidity. The AE burden score enables statistical comparisons analogous to other quantitative endpoints in clinical trials, and can readily accommodate different trial settings, diseases, and treatments, with diverse AE profiles. |
| first_indexed | 2024-03-10T15:06:06Z |
| format | Article |
| id | doaj.art-6cde63b1bfc649e2bc052a463d91986f |
| institution | Directory Open Access Journal |
| issn | 2072-6694 |
| language | English |
| last_indexed | 2024-03-10T15:06:06Z |
| publishDate | 2020-11-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Cancers |
| spelling | doaj.art-6cde63b1bfc649e2bc052a463d91986f2023-11-20T19:44:11ZengMDPI AGCancers2072-66942020-11-011211325110.3390/cancers12113251Adverse Event Burden Score—A Versatile Summary Measure for Cancer Clinical TrialsJennifer G. Le-Rademacher0Shauna Hillman1Elizabeth Storrick2Michelle R. Mahoney3Peter F. Thall4Aminah Jatoi5Sumithra J. Mandrekar6Department of Health Sciences Research, Mayo Clinic, Rochester, MN 55905, USADepartment of Health Sciences Research, Mayo Clinic, Rochester, MN 55905, USADepartment of Health Sciences Research, Mayo Clinic, Rochester, MN 55905, USAIQVIA, Raleigh-Durham, NC 27703, USADepartment of Biostatistics, M.D. Anderson Cancer Center, Houston, TX 77230, USADepartment of Oncology, Mayo Clinic, Rochester, MN 55905, USADepartment of Health Sciences Research, Mayo Clinic, Rochester, MN 55905, USAThis article introduces the adverse event (AE) burden score. The AE burden by treatment cycle is a weighted sum of all grades and AEs that the patient experienced in a cycle. The overall AE burden score is the total AE burden the patient experienced across all treatment cycles. AE data from two completed Alliance multi-center randomized double-blind placebo-controlled trials, with different AE profiles (NCCTG 97-24-51: 176 patients, and A091105: 83 patients), were utilized for illustration. Results of the AE burden score analyses corroborated the trials’ primary results. In 97-24-51, the overall AE burden for patients on the treatment arm was 2.2 points higher than those on the placebo arm, with a higher AE burden for patients who went off treatment early due to AE. Similarly, in A091105, the overall AE burden was 1.6 points higher on the treatment arm. On the placebo arms, the AE burden in 97-24-51 remained constant over time; and increased in later cycles in A091105, likely attributable to the increase in disease morbidity. The AE burden score enables statistical comparisons analogous to other quantitative endpoints in clinical trials, and can readily accommodate different trial settings, diseases, and treatments, with diverse AE profiles.https://www.mdpi.com/2072-6694/12/11/3251adverse eventclinical trialsadverse event burden scoresafety profile |
| spellingShingle | Jennifer G. Le-Rademacher Shauna Hillman Elizabeth Storrick Michelle R. Mahoney Peter F. Thall Aminah Jatoi Sumithra J. Mandrekar Adverse Event Burden Score—A Versatile Summary Measure for Cancer Clinical Trials Cancers adverse event clinical trials adverse event burden score safety profile |
| title | Adverse Event Burden Score—A Versatile Summary Measure for Cancer Clinical Trials |
| title_full | Adverse Event Burden Score—A Versatile Summary Measure for Cancer Clinical Trials |
| title_fullStr | Adverse Event Burden Score—A Versatile Summary Measure for Cancer Clinical Trials |
| title_full_unstemmed | Adverse Event Burden Score—A Versatile Summary Measure for Cancer Clinical Trials |
| title_short | Adverse Event Burden Score—A Versatile Summary Measure for Cancer Clinical Trials |
| title_sort | adverse event burden score a versatile summary measure for cancer clinical trials |
| topic | adverse event clinical trials adverse event burden score safety profile |
| url | https://www.mdpi.com/2072-6694/12/11/3251 |
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