Identification of meaningful individual-level change thresholds for worsening on the patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE®) Journal Article
| Authors: | Lee, M. K.; Mitchell, S. A.; Basch, E.; Mazza, G. L.; Langlais, B. T.; Thanarajasingam, G.; Ginos, B. F.; Rogak, L.; Meek, E. A.; Jansen, J.; Deal, A. M.; Carr, P.; Blinder, V. S.; Jonsson, M.; Mody, G. N.; Mendoza, T. R.; Bennett, A. V.; Schrag, D.; Dueck, A. C. |
| Article Title: | Identification of meaningful individual-level change thresholds for worsening on the patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE®) |
| Abstract: | Background: We derived meaningful individual-level change thresholds for worsening in selected patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE®) items and their composite scores. Methods: We used two data sources, the PRO-TECT trial (Alliance AFT-39) that collected PRO-CTCAE data from adults with advanced cancer at 26 United States (U.S.) community oncology practices and the PRO-CTCAE validation study that collected PRO-CTCAE data from adults undergoing chemotherapy or radiation therapy at nine U.S. cancer centers or community oncology practices. Both studies administered selected PRO-CTCAE items and EORTC QLQ-C30 scales. Conceptually, relevant QLQ-C30 domains were used as anchors to estimate meaningful change thresholds for deterioration in corresponding PRO-CTCAE items and their composite scores. Items or composites with ǀρǀ ≥ 0.30 correlation with QLQ-C30 scales were included. Changes in PRO-CTCAE scores and composites were estimated for patients who met or exceeded a 10-point deterioration on the corresponding QLQ-C30 scale. Change scores were computed between baseline and the 3-month timepoint in PRO-TECT, and in the PRO-CTCAE validation study between baseline and a single follow-up visit that occurred between 1 and 7 weeks later. For each PRO-CTCAE item, change scores could range from − 4 to 4; for a composite, change scores could range from − 3 to 3. Results: Change scores in QLQ-C30 and PRO-CTCAE were available in 406 and 792 patients in PRO-TECT and the validation study, respectively. Across QLQ-C30 scales, the proportion of patients with a 10-point or greater worsening on QLQ-C30 ranged from 15 to 30% in the PRO-TECT data and 13% to 34% in the validation data. Across PRO-CTCAE items, anchor-based meaningful change estimates for deterioration ranged from 0.05 to 0.30 (mean 0.19) in the PRO-TECT data and from 0.19 to 0.53 (mean 0.36) in the validation data. For composites, they ranged from 0.06 to 0.27 (mean 0.17) in the PRO-TECT data and 0.22 to 0.51 (mean 0.37) in the validation data. Conclusion: In both datasets, the minimal meaningful individual-level change threshold for worsening was one point for all items and composite scores. ClinicalTrials.gov: NCT03249090 (AFT-39), NCT02158637 (MC1091) © The Author(s) 2024. |
| Keywords: | adult; cancer chemotherapy; treatment outcome; aged; middle aged; major clinical study; constipation; advanced cancer; diarrhea; united states; cancer patient; cancer radiotherapy; follow up; neoplasm; neoplasms; quality of life; breast cancer; nausea; vomiting; psychology; lung cancer; cancer pain; validation study; urogenital tract cancer; dyspnea; questionnaire; insomnia; cancer center; taste disorder; cancer fatigue; cognition; patient reported outcome measures; drug therapy; adverse drug reaction; gastrointestinal cancer; deterioration; decreased appetite; female genital tract cancer; patient-reported outcome; pro-ctcae; Common Terminology Criteria for Adverse Events; predictive model; humans; human; male; female; article; loss of appetite; drug-related side effects and adverse reactions; surveys and questionnaires; ecog performance status; european organization for research and treatment of cancer quality of life questionnaire core 30; data accuracy; data source; data classification; individual-level meaningful change threshold; meaningful worsening; meaningful individual-level change threshold |
| Journal Title: | Quality of Life Research |
| Volume: | 34 |
| Issue: | 2 |
| ISSN: | 0962-9343 |
| Publisher: | Springer |
| Date Published: | 2025-02-01 |
| Start Page: | 495 |
| End Page: | 507 |
| Language: | English |
| DOI: | 10.1007/s11136-024-03819-5 |
| PUBMED: | 39503942 |
| PROVIDER: | scopus |
| PMCID: | PMC11865169 |
| DOI/URL: | |
| Notes: | Article -- Source: Scopus |
Altmetric
Citation Impact
BMJ Impact Analytics
Related MSK Work