Peer-reviewed article
IMG-09. RESPONSE ASSESSMENT IN DIFFUSE INTRINSIC PONTINE GLIOMA (DIPG): RECOMMENDATIONS FROM THE RESPONSE ASSESSMENT IN PEDIATRIC NEURO-ONCOLOGY COMMITTEE
Cooney T, Cohen K, Guimaraes C, Dhall G, Leach J et al. ·
Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001This document provides technical recommendations from an international committee of neuro-oncologists on how to assess treatment response in children and young adults with diffuse intrinsic pontine glioma (DIPG), a rare brain tumor. The recommendations cover standardized imaging protocols, neurological examination methods, and response criteria for clinical trials. The work addresses challenges specific to DIPG assessment including pseudoprogression and focal drug delivery approaches. While relevant to pediatric cancer, the document is clinical guidance rather than research on child well-being outcomes.
Key points
- RAPNO committee developed consensus recommendations for standardized response assessment in DIPG using MRI, neurologic examination, and medication use
- Recommended response duration of eight weeks and 25% decrease in tumor size for partial response
- Addresses unique DIPG challenges including pseudoprogression and focal drug delivery methods
- Distinguishes between pontine and extra-pontine response assessment for targeted drug delivery trials
- Recommendations designed to standardize clinical trial conduct and enable comparison of outcomes
Limitations
This document provides technical clinical guidance for oncology trials rather than empirical data on child health outcomes or effectiveness of treatments. It does not evaluate whether treatments improve survival, quality of life, or other measures of child well-being.
Abstract
Abstract Optimizing the conduct of clinical trials for diffuse intrinsic pontine glioma (DIPG) involves use of consistent, objective disease assessments and standardized response criteria. The Response Assessment in Pediatric Neuro-Oncology (RAPNO) committee, an international panel of pediatric and adult neuro-oncologists, clinicians, radiologists, radiation oncologists, and neurosurgeons, was established to address unique challenges in assessing response in children with CNS tumors. A subcommittee of RAPNO was formed to specifically address response assessment in children and young adults with DIPG and to develop a consensus on recommendations for response assessment. Distinct issues related to the response assessment of DIPG include its definition and recent molecular classifications, dearth of imaging response data, the phenomena of pseudoprogression, and measuring response in the era of focal drug delivery. The committee has recommended response be assessed using magnetic resonance imaging (MRI) of brain and spine, neurologic examination, and use of supportive medication, i.e. steroids and anti-angiogenic agents. Clinical imaging standards and imaging quality control are defined. Unique recommendations for DIPG response include an eight-week response duration, a twenty-five percent decrease for partial response, and the distinction of pontine and extra-pontine response for trials that use focal drug delivery. The recommendations presented here represent an initial effort to uniformly collect and evaluate response assessment criteria; these recommendations can now be incorporated into clinical trials to assess feasibility and corroboration with patient outcomes.