Challenges with defining response to antitumor agents in pediatric neuro-oncology: A report from the response assessment in pediatric neuro-oncology (RAPNO) working group

Katherine E. Warren, Tina Y. Poussaint, Gilbert Vezina, Darren Hargrave, Roger J. Packer, Stewart Goldman, Patrick Y. Wen, Ian F. Pollack, David Zurakowski, Larry E. Kun, Michael D. Prados, Stefan Rutkowski, Mark W. Kieran

Research output: Contribution to journalReview articlepeer-review

61 Scopus citations

Abstract

Criteria for new drug approval include demonstration of efficacy. In neuro-oncology, this is determined radiographically utilizing tumor measurements on MRI scans. Limitations of this method have been identified where drug activity is not reflected in decreased tumor size. The RANO (Response Assessment in Neuro-Oncology) working group was established to address limitations in defining endpoints for clinical trials in adult neuro-oncology and to develop standardized response criteria. RAPNO was subsequently established to address unique issues in pediatric neuro-oncology. The aim of this paper is to delineate response criteria issues in pediatric clinical trials as a basis for subsequent recommendations.

Original languageEnglish (US)
Pages (from-to)1397-1401
Number of pages5
JournalPediatric Blood and Cancer
Volume60
Issue number9
DOIs
StatePublished - Sep 2013

Keywords

  • Brain
  • Imaging
  • Pediatric
  • RANO
  • Response
  • Tumor

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

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