Experimental Therapeutic Trial Design for Pediatric Brain Tumors. (October 2016)

• Record Type: Journal Article
• Authors: Bornhorst, Miriam; Hwang, Eugene I.
• Other Names: Khakoo Yasmin guest-editor.; Kramer Kim guest-editor.
• Is Part Of: Journal of child neurology. Volume 31:Number 12(2016:Dec.)
• Page Start: 1421
• Page End: 1432
• Publication Date: 2016-10
• Subjects: clinical trial -- pediatric -- targeted therapy -- design -- treatment; Nervous system -- Diseases -- Periodicals; 618.928
• DOI: 10.1177/0883073815604221
• Languages: English
• ISSNs: 0883-0738

Abstract

Pediatric brain tumors are the leading cause of cancer-related death during childhood. Since the first pediatric brain tumor clinical trials, the field has seen improved outcomes in some, but not all tumor types. In the past few decades, a number of promising new therapeutic agents have emerged, yet only a few of these agents have been incorporated into clinical trials for pediatric brain tumors. In this review, the authors discuss the process of and challenges in pediatric clinical trial design; this will allow for highly efficient and effective clinical trials with appropriate endpoints to ensure rapid and safe investigation of novel therapeutics for children with brain tumors.