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Evaluating Treatment Tolerability Using the Toxicity Index With Patient-Reported Outcomes Data
Blake Langlais1, Gina L Mazza1, Gita Thanarajasingam2
1Mayo Clinic, Department of Quantitative Health Sciences, Division of Biostatistics and Clinical Trials (B.L., G.L.M., B.G., A.C.D.), Phoenix, Arizona, USA.
Journal of Pain and Symptom Management
|August 9, 2021
Summary
The toxicity index effectively summarizes patient-reported adverse events in clinical trials, similar to severity proportions. Further research is needed to clarify its patient-facing interpretation for treatment tolerability.
Area of Science:
- Clinical Trials
- Patient-Reported Outcomes
- Health Outcomes Research
Background:
- Summarizing symptomatic adverse events is crucial for assessing treatment tolerability in clinical trials.
- The Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) captures patient-reported tolerability data.
- Existing summary measures like maximum score and proportions of severe events are commonly used.
Purpose of the Study:
- To apply the toxicity index to PRO-CTCAE data from the COMET-2 trial.
- To compare the toxicity index with maximum score as a tolerability summary measure.
- To discuss the utility of the toxicity index for communicating tolerability to patients and clinicians.
Main Methods:
- Calculated proportions of maximum PRO-CTCAE severity levels and median toxicity index by treatment arm.
- Adjusted for baseline symptoms in the analysis.
- Compared group-wise statistical differences between the two summary measures.
Main Results:
- Statistical differences between treatment arms were comparable using either severity level proportions or the toxicity index.
- Adjusting for baseline symptoms had a similar impact on comparisons regardless of the summary measure used.
- The toxicity index proved useful for ranking patients by symptomatic adverse event burden.
Conclusions:
- The toxicity index is a viable method for analyzing PRO-CTCAE data.
- The toxicity index can be applied to assess patient-reported adverse events.
- Further investigation is required to establish a clear clinical or patient-facing interpretation of the toxicity index for tolerability assessment.
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