The use of minor response, volumetric assessment, and growth rate kinetics as endpoints in grade 1-3 glioma clinical trials: a RANO perspective

The Response Assessment in NeuroOncology (RANO) criteria for response to treatment in glioma make a distinction between enhancing and unenhancing tumors, which reflects assessment of a different biological process underlying the magnetic resonance imaging findings in enhancing and nonenhancing tumors. The recent regulatory approval of tovorafenib has also considered the patients with minor responses (25%-50% decrease) based on the 2D measurement on T2/FLAIR MR imaging while incorporating other measures of patient benefit. The INDIGO placebo controlled trial led to the regulatory approval of vorasidenib based on the increase in PFS in the active treatment arm, as measured by blinded independent radiology review using the 2D RANO criteria. With that, more insight in the regulatory requirements for approval in low-grade tumors has been obtained, but other issues in assessing outcome in studies on low-grade enhancing tumors remain. These include the added value of volumetric assessment instead of 2D assessments, and assessment of tumor growth rate as an early marker of outcome. This manuscript gives an overview of the current perspective on and challenges of assessing outcome in trials on unenhancing glioma, with both regulatory and clinical perspectives.

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Publication Details

Journal
Neuro-Oncology
Published
2025-07-19
DOI
https://doi.org/10.1093/neuonc/noaf173
Citations
5
Primary Topic
Radiomics and Machine Learning in Medical Imaging
Type
article
Field-Weighted Citation Impact
6.80

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article

The use of minor response, volumetric assessment, and growth rate kinetics as endpoints in grade 1-3 glioma clinical trials: a RANO perspective

J. C. Tonn, Joshua E. Allen, Martin J. van den Bent, Robert J. Young et al.
5 citations
Neuro-Oncology
Radiomics and Machine Learning in Medical Imaging
6.80
article

The use of minor response, volumetric assessment, and growth rate kinetics as endpoints in grade 1-3 glioma clinical trials: a RANO perspective

J. C. Tonn, Joshua E. Allen, Martin J. van den Bent, Robert J. Young, Maarten M.J. Wijnenga, Ingo K. Mellinghoff, Patrick Yung Wen, Benjamin M. Ellingson, Gilbert Youssef, Philipp Karschnia, Shawn L. Hervey‐Jumper, Anouk van der Hoorn, John de Groot, Mark W. Kieran, Timothy F. Cloughesy, Raymond Y. Huang, Johan Pallud, Susan M. Chang, Michael A. Vogelbaum, Samuel C. Blackman
article en
5 citations

Abstract

The Response Assessment in NeuroOncology (RANO) criteria for response to treatment in glioma make a distinction between enhancing and unenhancing tumors, which reflects assessment of a different biological process underlying the magnetic resonance imaging findings in enhancing and nonenhancing tumors. The recent regulatory approval of tovorafenib has also considered the patients with minor responses (25%-50% decrease) based on the 2D measurement on T2/FLAIR MR imaging while incorporating other measures of patient benefit. The INDIGO placebo controlled trial led to the regulatory approval of vorasidenib based on the increase in PFS in the active treatment arm, as measured by blinded independent radiology review using the 2D RANO criteria. With that, more insight in the regulatory requirements for approval in low-grade tumors has been obtained, but other issues in assessing outcome in studies on low-grade enhancing tumors remain. These include the added value of volumetric assessment instead of 2D assessments, and assessment of tumor growth rate as an early marker of outcome. This manuscript gives an overview of the current perspective on and challenges of assessing outcome in trials on unenhancing glioma, with both regulatory and clinical perspectives.

Neuro-OncologyVol. 27(10)
Brigham and Women's Hospital (US), Google (United States) (US), University Medical Center Groningen (NL), Memorial Sloan Kettering Cancer Center (US), Harvard University (US), Inserm (FR), University of California, Los Angeles (US), University of Groningen (NL), German Cancer Research Center (DE), University of California, San Francisco (US), Friedrich-Alexander-Universität Erlangen-Nürnberg (DE), Université Paris Cité (FR), LMU Klinikum (DE), Moffitt Cancer Center (US), Massachusetts General Hospital (US), Universitätsklinikum Erlangen (DE), Dana-Farber Cancer Institute (US), Chimerix (United States) (US), NGM Biopharmaceuticals (United States) (US), German Centre for Cardiovascular Research (DE), Institut de Psychiatrie et Neurosciences de Paris (FR), FHU Neurovasc (FR), Comprehensive Cancer Center Erlangen (DE), Erasmus MC Cancer Institute (NL), Neurological Surgery (US), Mass General Brigham (US), Ludwig-Maximilians-Universität München (DE)
Amgen, Alexion Pharmaceuticals, Novocure, Agios Pharmaceuticals, Rijksuniversiteit Groningen, Servier, Sumitomo Dainippon Pharma Oncology
Openalex Percentile: Top 3%
Radiomics and Machine Learning in Medical Imaging
6.80
DOIOpen Access PDFOpenAlex
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