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LGG-52. Volumetry-based response characterization of recurrent pediatric low-grade gliomas in PNOC clinical Neuro-oncology trials
BACKGROUND: Endpoints in clinical trials using novel treatments are evaluated by RANO criteria, which provide an estimate of tumor size from two-dimensional measurements along the most prominent axial slice. However, pediatric low-grade gliomas (pLGG) commonly have variegated shapes with solid and c...
Autores principales: | , , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9165161/ http://dx.doi.org/10.1093/neuonc/noac079.364 |
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author | von Reppert, Marc Lin, MingDe Bousabarah, Khaled Familiar, Ariana Velasco, Ryan Waanders, Angela Vossough, Arastoo Haddock, Amanda Nicolaides, Theodore Swanson, Kristin Kazerooni, Anahita Kline, Cassie Nabavizadeh, Ali Haas-Kogan, Daphne Prados, Michael Rubin, Joshua Mueller, Sabine Aboian, Mariam |
author_facet | von Reppert, Marc Lin, MingDe Bousabarah, Khaled Familiar, Ariana Velasco, Ryan Waanders, Angela Vossough, Arastoo Haddock, Amanda Nicolaides, Theodore Swanson, Kristin Kazerooni, Anahita Kline, Cassie Nabavizadeh, Ali Haas-Kogan, Daphne Prados, Michael Rubin, Joshua Mueller, Sabine Aboian, Mariam |
author_sort | von Reppert, Marc |
collection | PubMed |
description | BACKGROUND: Endpoints in clinical trials using novel treatments are evaluated by RANO criteria, which provide an estimate of tumor size from two-dimensional measurements along the most prominent axial slice. However, pediatric low-grade gliomas (pLGG) commonly have variegated shapes with solid and cystic components, potentially resulting in misevaluation of true tumor volume and thus, ultimately, trial outcome. OBJECTIVES: We aim to characterize treatment response through volumetric assessment of progressive/recurrent pLGGs treated with single-agent everolimus on PNOC001 clinical trial. We seek to identify clinically relevant criteria that provide added value to response assessment beyond 2D measurements. METHODS: In a cohort of 44 patients we performed 3D-segmentation of solid, cystic and whole tumor within our PACS-framework and compared results to previously carried-out central imaging review by RANO criteria which had yielded 15 PD, 27 SD, 2 PR and 0 CR. RESULTS: 8 tumors were solid only and 36 had a mixed solid-cystic appearance. When evaluating the entire tumor (i.e. solid and cystic components combined) and using the same RANO cutoff criteria, one case changed from PR to SD, one changed from SD to PR, 3 changed from SD to PD, and 7 changed from PD to SD, resulting in an overall discordance of 27% of cases. CONCLUSION: We propose that incorporation of volumetrics into response assessment provides additional and potentially more accurate information beyond RANO-based measurements. It is crucial to note that the above-reported changes represent numerical discrepancies as opposed to true-to-reality changes in clinical outcome. Determining representative thresholds for the deployment of volumetric measures in clinical trials will be critical. Future work will include data from the PNOC002 clinical trial and evaluate inter-reader agreement and reader discordance. With the availability of PACS-based 3D-tools in neuroradiology practice, well-defined volumetric criteria could be incorporated prospectively into treatment response analysis in clinical trials. |
format | Online Article Text |
id | pubmed-9165161 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | Oxford University Press |
record_format | MEDLINE/PubMed |
spelling | pubmed-91651612022-06-05 LGG-52. Volumetry-based response characterization of recurrent pediatric low-grade gliomas in PNOC clinical Neuro-oncology trials von Reppert, Marc Lin, MingDe Bousabarah, Khaled Familiar, Ariana Velasco, Ryan Waanders, Angela Vossough, Arastoo Haddock, Amanda Nicolaides, Theodore Swanson, Kristin Kazerooni, Anahita Kline, Cassie Nabavizadeh, Ali Haas-Kogan, Daphne Prados, Michael Rubin, Joshua Mueller, Sabine Aboian, Mariam Neuro Oncol Low Grade Glioma BACKGROUND: Endpoints in clinical trials using novel treatments are evaluated by RANO criteria, which provide an estimate of tumor size from two-dimensional measurements along the most prominent axial slice. However, pediatric low-grade gliomas (pLGG) commonly have variegated shapes with solid and cystic components, potentially resulting in misevaluation of true tumor volume and thus, ultimately, trial outcome. OBJECTIVES: We aim to characterize treatment response through volumetric assessment of progressive/recurrent pLGGs treated with single-agent everolimus on PNOC001 clinical trial. We seek to identify clinically relevant criteria that provide added value to response assessment beyond 2D measurements. METHODS: In a cohort of 44 patients we performed 3D-segmentation of solid, cystic and whole tumor within our PACS-framework and compared results to previously carried-out central imaging review by RANO criteria which had yielded 15 PD, 27 SD, 2 PR and 0 CR. RESULTS: 8 tumors were solid only and 36 had a mixed solid-cystic appearance. When evaluating the entire tumor (i.e. solid and cystic components combined) and using the same RANO cutoff criteria, one case changed from PR to SD, one changed from SD to PR, 3 changed from SD to PD, and 7 changed from PD to SD, resulting in an overall discordance of 27% of cases. CONCLUSION: We propose that incorporation of volumetrics into response assessment provides additional and potentially more accurate information beyond RANO-based measurements. It is crucial to note that the above-reported changes represent numerical discrepancies as opposed to true-to-reality changes in clinical outcome. Determining representative thresholds for the deployment of volumetric measures in clinical trials will be critical. Future work will include data from the PNOC002 clinical trial and evaluate inter-reader agreement and reader discordance. With the availability of PACS-based 3D-tools in neuroradiology practice, well-defined volumetric criteria could be incorporated prospectively into treatment response analysis in clinical trials. Oxford University Press 2022-06-03 /pmc/articles/PMC9165161/ http://dx.doi.org/10.1093/neuonc/noac079.364 Text en © The Author(s) 2022. Published by Oxford University Press on behalf of the Society for Neuro-Oncology. https://creativecommons.org/licenses/by-nc/4.0/This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial License (https://creativecommons.org/licenses/by-nc/4.0/), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited. For commercial re-use, please contact journals.permissions@oup.com |
spellingShingle | Low Grade Glioma von Reppert, Marc Lin, MingDe Bousabarah, Khaled Familiar, Ariana Velasco, Ryan Waanders, Angela Vossough, Arastoo Haddock, Amanda Nicolaides, Theodore Swanson, Kristin Kazerooni, Anahita Kline, Cassie Nabavizadeh, Ali Haas-Kogan, Daphne Prados, Michael Rubin, Joshua Mueller, Sabine Aboian, Mariam LGG-52. Volumetry-based response characterization of recurrent pediatric low-grade gliomas in PNOC clinical Neuro-oncology trials |
title | LGG-52. Volumetry-based response characterization of recurrent pediatric low-grade gliomas in PNOC clinical Neuro-oncology trials |
title_full | LGG-52. Volumetry-based response characterization of recurrent pediatric low-grade gliomas in PNOC clinical Neuro-oncology trials |
title_fullStr | LGG-52. Volumetry-based response characterization of recurrent pediatric low-grade gliomas in PNOC clinical Neuro-oncology trials |
title_full_unstemmed | LGG-52. Volumetry-based response characterization of recurrent pediatric low-grade gliomas in PNOC clinical Neuro-oncology trials |
title_short | LGG-52. Volumetry-based response characterization of recurrent pediatric low-grade gliomas in PNOC clinical Neuro-oncology trials |
title_sort | lgg-52. volumetry-based response characterization of recurrent pediatric low-grade gliomas in pnoc clinical neuro-oncology trials |
topic | Low Grade Glioma |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9165161/ http://dx.doi.org/10.1093/neuonc/noac079.364 |
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