Clinical trials in pediatric neuro-oncology: what is missing and how we can improve.
Brain tumors are the most common solid tumor in childhood, yet outcomes vary dramatically. High-grade gliomas have dismal outcomes with poor survival. By contrast, low-grade gliomas, have high survival rates, but children suffer from morbidity of tumor burden and therapy-associated side effects. In this article, we discuss how current trial designs often miss the opportunity to include end points beyond tumor response and thus fail to offer complete assessments of therapeutic approaches. Quality of life, neurocognitive function and neurofunctional deficits need to be considered when assessing overall success of a therapy. Herein, we identify specific end points that should be included in the interpretation of clinical trial results and accordingly, offer a more comprehensive approach to treatment decision-making.
School of Medicine, University of California, San Francisco (UCSF), 513 Parnassus Ave, San Francisco, CA 94143, USA.
Lennox
L
Byer
Department of Pediatrics, Division of Pediatric Hematology/Oncology, University of California, San Francisco, 550 16th Street, 4th Floor, San Francisco, CA 94158, USA.
Cassie
C
Kline
Department of Pediatrics, Division of Pediatric Hematology/Oncology, University of California, San Francisco, 550 16th Street, 4th Floor, San Francisco, CA 94158, USA.|Department of Neurology & Neurosurgery San Francisco, University of California, 625 Nelson Rising Lane, 4th Floor, San Francisco, CA 94158, USA.
Sabine
S
Mueller
D001932:Brain Neoplasms / Q000150:complications / Q000523:psychology / Q000628:therapy*; D002986:Clinical Trials as Topic; D003072:Cognition Disorders / Q000209:etiology; D006801:Humans; D008495:Medical Oncology / Q000379:methods*; D009422:Nervous System Diseases / Q000209:etiology; D009462:Neurology / Q000379:methods*; D010372:Pediatrics; D011788:Quality of Life
clinical trials; late effects; neurocognition; pediatric brain tumors; quality of life