Long-term survivors of diffuse intrinsic pontine glioma (DIPG): myth or reality

Diffuse intrinsic pontine gliomas (DIPG) represent 75–80% of the brainstem tumors occurring in children, with the highest incidence between the ages of 5 and 10 years (1). The diagnosis is based on clinico-radiological findings and biopsies have historically been reserved for atypical cases, or performed in the context of clinical trials (2). Standard treatment is based on palliative radiotherapy (1,2). Multiple studies over past decades evaluating a myriad of chemotherapeutic and targeted agents have not shown any significant improvement in the survival outcomes compared to standard radiotherapy alone (1,2). The estimated median progression-free survival (PFS) and overall survival (OS) for DIPG patients are 6 and 11 months, respectively (3). In this context, DIPG constitutes the most challenging pediatric brain tumor, given the dismal outcomes and the scarcity of effective therapies.