title: Challenges with defining response to antitumor agents in pediatric neuro-oncology: a report from the response assessment in pediatric neuro-oncology (RAPNO) working group.
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.
authors.affiliation: Pediatric Oncology Branch, National Cancer Institute, Bethesda, Maryland 20892, USA. warrenk@mail.nih.gov
authors.forename: Katherine E
authors.initials: KE
authors.lastname: Warren
authors.forename: Tina Y
authors.initials: TY
authors.lastname: Poussaint
authors.forename: Gilbert
authors.initials: G
authors.lastname: Vezina
authors.forename: Darren
authors.initials: D
authors.lastname: Hargrave
authors.forename: Roger J
authors.initials: RJ
authors.lastname: Packer
authors.forename: Stewart
authors.initials: S
authors.lastname: Goldman
authors.forename: Patrick Y
authors.initials: PY
authors.lastname: Wen
authors.forename: Ian F
authors.initials: IF
authors.lastname: Pollack
authors.forename: David
authors.initials: D
authors.lastname: Zurakowski
authors.forename: Larry E
authors.initials: LE
authors.lastname: Kun
authors.forename: Michael D
authors.initials: MD
authors.lastname: Prados
authors.forename: Stefan
authors.initials: S
authors.lastname: Rutkowski
authors.forename: Mark W
authors.initials: MW
authors.lastname: Kieran
mesh_terms: D000293:Adolescent; D000328:Adult; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic / Q000379:methods* / Q000592:standards; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009380:Neoplasms, Nerve Tissue / Q000188:drug therapy*
chemical_list: D000970:Antineoplastic Agents
keywords: RANO; brain; imaging; pediatric; response; tumor