Contributions
Abstract: EP1171
Type: E-Poster Presentation
Session title: Quality of life, palliative care, ethics and health economics
Background
The PRO-CTCAE is a patient-reported outcome measure of symptomatic toxicity in oncology trials designed to complement CTCAE criteria (clinician’s measure of adverse events [AEs]). Items for evaluation are selected from 78 symptomatic toxicities in the CTCAE. PRO-CTCAE uptake and use has not been uniformly assessed; understanding how to best analyze and present PRO-CTCAE data is important.
Aims
We reviewed literature on clinical trials reporting the PRO-CTCAE to ascertain its use and current analytical methods to inform future analyses.
Methods
A review of the literature and ongoing oncology trials using PubMed, oncology and outcomes research conferences (ASCO, ASH, ISOQOL, ISPOR), and ClinicalTrials.gov was completed August 2019; updated November 2020. Selected literature included guidance on: item selection; analytical methods; PRO-CTCAE data/endpoint use in clinical trials. Analytical and visualization methods from the literature were used to inform the specification of PRO-CTCAE analyses conducted on data from the DREAMM-2 (NCT03525678) trial of single-agent belantamab mafodotin (belamaf), a B-cell maturation antigen–binding antibody–drug conjugate, in relapsed/refractory multiple myeloma.
Results
After screening (n=197), 45 articles were reviewed: 13 related to recommendations for use/analysis, 7 to item selection, and 25 to examples of PRO-CTCAE use. 118 completed/ongoing trials reported PRO-CTCAE use (17 [14%] Phase I or I/II; 73 [62%] Phase II, II/III, or III; 3 [3%] Phase IV; 25 had no phase reported). Most (92/118, 78%) trials included the PRO-CTCAE as a secondary endpoint. The number of trials using the PRO-CTCAE increased from 3 in 2015 to 30 in 2020. PRO-CTCAE data in the literature were reported descriptively using tables (maximum post-baseline ratings; mean change over time; ratings higher than a predefined cutoff) and graphs (line graphs; stacked bar charts; heatmaps). These and more sophisticated analyses were applied to DREAMM-2 data to best capture the dynamics of patient-reported AEs over time, or control for baseline PRO-CTCAE ratings (eg, Toxicity over Time approach; ordinal log-linear models).
Conclusion
The importance of tolerability and symptomatic AEs for patients is increasingly recognized, particularly for new therapies. This landscape review demonstrates recent growth in interest and adoption of PRO-CTCAE. There are few existing standards for analysis; this research increases understanding of existing methods and further evaluates ways of analyzing and presenting PRO-CTCAE data over time. Additional research exploring innovative methods to analyze PRO-CTCAE data and address its challenges is needed.
Funding Source: GlaxoSmithKline (212225). Drug linker technology licensed from Seagen Inc.; mAb produced using POTELLIGENT Technology licensed from BioWa.
Encore statement: ©2021 American Society of Clinical Oncology, Inc. Reused with permission. All rights reserved. This abstract was previously submitted to the American Society of Clinical Oncology (ASCO) Annual Meeting, June 4–8, 2021, and is submitted on behalf of the original authors with their permission.
Keyword(s): B-cell maturation antigen, Multiple myeloma, Quality of life, Safety
Abstract: EP1171
Type: E-Poster Presentation
Session title: Quality of life, palliative care, ethics and health economics
Background
The PRO-CTCAE is a patient-reported outcome measure of symptomatic toxicity in oncology trials designed to complement CTCAE criteria (clinician’s measure of adverse events [AEs]). Items for evaluation are selected from 78 symptomatic toxicities in the CTCAE. PRO-CTCAE uptake and use has not been uniformly assessed; understanding how to best analyze and present PRO-CTCAE data is important.
Aims
We reviewed literature on clinical trials reporting the PRO-CTCAE to ascertain its use and current analytical methods to inform future analyses.
Methods
A review of the literature and ongoing oncology trials using PubMed, oncology and outcomes research conferences (ASCO, ASH, ISOQOL, ISPOR), and ClinicalTrials.gov was completed August 2019; updated November 2020. Selected literature included guidance on: item selection; analytical methods; PRO-CTCAE data/endpoint use in clinical trials. Analytical and visualization methods from the literature were used to inform the specification of PRO-CTCAE analyses conducted on data from the DREAMM-2 (NCT03525678) trial of single-agent belantamab mafodotin (belamaf), a B-cell maturation antigen–binding antibody–drug conjugate, in relapsed/refractory multiple myeloma.
Results
After screening (n=197), 45 articles were reviewed: 13 related to recommendations for use/analysis, 7 to item selection, and 25 to examples of PRO-CTCAE use. 118 completed/ongoing trials reported PRO-CTCAE use (17 [14%] Phase I or I/II; 73 [62%] Phase II, II/III, or III; 3 [3%] Phase IV; 25 had no phase reported). Most (92/118, 78%) trials included the PRO-CTCAE as a secondary endpoint. The number of trials using the PRO-CTCAE increased from 3 in 2015 to 30 in 2020. PRO-CTCAE data in the literature were reported descriptively using tables (maximum post-baseline ratings; mean change over time; ratings higher than a predefined cutoff) and graphs (line graphs; stacked bar charts; heatmaps). These and more sophisticated analyses were applied to DREAMM-2 data to best capture the dynamics of patient-reported AEs over time, or control for baseline PRO-CTCAE ratings (eg, Toxicity over Time approach; ordinal log-linear models).
Conclusion
The importance of tolerability and symptomatic AEs for patients is increasingly recognized, particularly for new therapies. This landscape review demonstrates recent growth in interest and adoption of PRO-CTCAE. There are few existing standards for analysis; this research increases understanding of existing methods and further evaluates ways of analyzing and presenting PRO-CTCAE data over time. Additional research exploring innovative methods to analyze PRO-CTCAE data and address its challenges is needed.
Funding Source: GlaxoSmithKline (212225). Drug linker technology licensed from Seagen Inc.; mAb produced using POTELLIGENT Technology licensed from BioWa.
Encore statement: ©2021 American Society of Clinical Oncology, Inc. Reused with permission. All rights reserved. This abstract was previously submitted to the American Society of Clinical Oncology (ASCO) Annual Meeting, June 4–8, 2021, and is submitted on behalf of the original authors with their permission.
Keyword(s): B-cell maturation antigen, Multiple myeloma, Quality of life, Safety