Clinical trials in pediatric neuro-oncology: what is missing and how we can improve

Written by Byer L, Kline C, Mueller S

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.

In the USA, the number of adult survivors of pediatric brain tumors has been steadily increasing over recent years. The average 5-year survival rate for all-comers of pediatric brain cancers has risen to approximately 73% [1]. However, there is a broad range of survivorship depending on tumor type. Pure germinomas and pilocytic astrocytomas have 5-year survival rates greater than 90%; however, for diffuse intrinsic pontine glioma and other high-grade gliomas, outcomes remain extremely poor [2–5]. The main clinical trial objective for brain tumors with poor prognoses is to improve survival; however, quality of life (QoL) remains an important aspect for these children – especially if median survival is relatively short.

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