Contemporary survival endpoints: an International Diffuse Intrinsic Pontine Glioma Registry study

Our understanding of diffuse intrinsic pontine glioma (DIPG) biology has rapidly evolved in recent years. Promising agents such as panobinostat, with demonstrated disease-specific preclinical efficacy, are currently in clinical trials. Determination of clinical benefit by measuring improved progression-free survival (PFS) and overall survival (OS) requires well-defined historical controls. However, many previous studies evaluating these endpoints included small numbers of patients, were single institution studies, and were not limited to DIPG but rather included all brainstem tumors.^1–4 The most recent study investigating median post-progression survival (PPS) time (ie, time from initial tumor progression to death) in DIPG patients was published over 20 years ago.⁵

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