Evolving Methods of Treatment Evaluation in Pediatric Glioma Clinical Trials
While the Response Assessment in Neuro-Oncology (RANO) Working Group criteria have been pivotal in assessing adult high-grade gliomas, the more recently established Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria address the unique challenges of evaluating pediatric gliomas, emphasizing the need for tailored imaging and clinical assessment strategies. Imaging Endpoints has vast experience in the use of RAPNO in clinical trials and supported the first FDA approvals utilizing the criteria.
Central Nervous System (CNS) tumors account for 21% of all pediatric cancers. The main secondary disorders are endocrine and cognitive disorders, which can greatly inhibit patients’ quality of life as adults. Although most pediatric gliomas present as benign, slow-growing lesions (low-grade gliomas [LGG]), a significant percentage of gliomas develop over a short time and progress rapidly (high-grade gliomas [HGG]). Improvement in the diagnosis and treatment of pediatric CNS tumors is urgently needed.
The RAPNO criteria emphasizes changes in bi-dimensional contrast-enhancing tumor size in order to define response and incorporates the neurological status and corticosteroid treatment of the patient in a similar fashion to other neuro-oncology response criteria. A key difference from adult glioma assessment lies in RAPNO’s handling of cystic components frequently found in pediatric LGGs. RAPNO provides specific guidance on measuring these cystic components to accurately track the development of the remaining solid tumor portion. This is crucial because the cystic component depending on its characteristics significantly influences the overall assessment of tumor response and progression.
In addition to these newer assessment methods, quantitative radiomics offers the potential to extract detailed quantitative data from images, identifying disease characteristics (like heterogeneity) not easily assessed visually. This can improve diagnostic accuracy, prognostic assessment, and prediction of therapy response.
Imaging Endpoints has been at the forefront of glioma trials in pediatric patients, leveraging the regulatory experience with RAPNO criteria. When designing Charter for pediatric glioma clinical trials, it is important to define how cystic components should be measured, especially when they are integral to the tumor, such as with a “soap bubble” appearance or significant cyst wall enhancement. Establishing clear criteria for excluding reactive cysts at the tumor-brain interface and predominantly cystic tumors with small solid nodules, as well as resection cavities is also crucial.
With a strong track record of improving trial outcomes by implementing the right strategy, our team specializes in optimizing imaging charter and assessment criteria for Glioma clinical trials including being a pioneer for utilizing RANO & RAPNO for regulatory submission. Reach out to us to connect with our experienced medical and scientific professionals and explore how our tailored imaging solutions can support your pediatric glioma clinical trials. For more information,
