Evaluating treatment tolerability using the toxicity index with patient-reported outcomes data Journal Article

   


Authors: Langlais, B.; Mazza, G. L.; Thanarajasingam, G.; Rogak, L. J.; Ginos, B.; Heon, N.; Scher, H. I.; Schwab, G.; Ganz, P. A.; Basch, E.; Dueck, A. C.
Article Title: Evaluating treatment tolerability using the toxicity index with patient-reported outcomes data
Abstract: Context: Summarizing longitudinal symptomatic adverse events during clinical trials is necessary for understanding treatment tolerability. The Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) provides insight for capturing treatment tolerability within trials. Tolerability summary measures, such as the maximum score, are often used to communicate the potential negative symptoms both in the medical literature and directly to patients. Commonly, the proportions of present and severe symptomatic adverse events are used and reported between treatment arms among adverse event types. The toxicity index is also a summary measure previously applied to clinician-reported CTCAE data. Objectives: Apply the toxicity index to PRO-CTCAE data from the COMET-2 trial alongside the maximum score, then present and discuss considerations for using the toxicity index as a summary measure for communicating tolerability to patients and clinicians. Methods: Proportions of maximum PRO-CTCAE severity levels and median toxicity index were computed by arm using all trial data and adjusting for baseline symptoms. Results: Group-wise statistical differences were similar whether using severity level proportions or the toxicity index. The impact of adjusting for baseline symptoms was equivalently seen when comparing arms using severity rates or the toxicity index. Conclusion: The toxicity index is a useful method when ranking patients from those with the least to most symptomatic adverse event burden. This study showed the toxicity index can be applied to PRO-CTCAE data. Though as a tolerability summary measure, further study is needed to provide a clear clinical or patient-facing interpretation of the toxicity index. © 2021 The Authors
Keywords: adult; controlled study; major clinical study; prednisone; constipation; drug tolerability; fatigue; paresthesia; placebo; diarrhea; comparative study; outcome assessment; multiple cycle treatment; pain; nausea; randomized controlled trial; vomiting; clinical assessment; dyspnea; insomnia; symptom; disease severity; mitoxantrone; clinical evaluation; patient-reported outcomes; double blind procedure; adverse drug reaction; castration resistant prostate cancer; decreased appetite; patient-reported outcome; tolerability; pro-ctcae; drug intoxication; skin tingling; Common Terminology Criteria for Adverse Events; cabozantinib; human; male; article; cancer clinical trials; malignant neoplasm; symptomatic adverse event; toxicity index
Journal Title: Journal of Pain and Symptom Management
Volume: 63
Issue: 2
ISSN: 0885-3924
Publisher: Elsevier Inc.  
Date Published: 2022-02-01
Start Page: 311
End Page: 320
Language: English
DOI: 10.1016/j.jpainsymman.2021.07.031
PUBMED: 34371138
PROVIDER: scopus
PMCID: PMC8816875
DOI/URL:

https://www.scopus.com/inward/record.uri?eid=2-s2.0-85114319122&doi=10.1016%2fj.jpainsymman.2021.07.031&partnerID=40&md5=6f884095fb67e880278f4ca65fd58b04
Notes: Article -- Export Date: 1 March 2022 -- Source: Scopus
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MSK Authors
  1. Ethan Martin Basch
    180 Basch
  2. Howard Scher
    1130 Scher
  3. Lauren Jayne Rogak
    76 Rogak
  4. Narre Heon
    16 Heon
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