Characteristics of Long-Term Survivors of Diffuse Intrinsic Pontine Glioma


Key Points

  • Long-term survivors were more likely to present at < 3 or > 10 years of age, to have longer symptom duration, and to have received systemic therapy at diagnosis.
  • Long-term survivors were more likely to have HIST1H3B mutation. 

In a report from the diffuse intrinsic pontine glioma (DIPG) registries of the International and European Societies for Pediatric Oncology, published in the Journal of Clinical Oncology, Hoffman et al identified characteristics of long-term survivors among pediatric and young adult patients with the disease.

The study involved data from 1,008 patients with radiographically confirmed DIPG from registry databases in North America, Australia, Germany, Austria, Switzerland, the Netherlands, Italy, France, the United Kingdom, and Croatia.

Characteristics Associated With Survival Duration

Among all patients, 101 (10%) were long-term survivors (survival ≥ 2 years), and median overall survival was 11 months (interquartile range = 7.5–16 months). The 1-, 2-, 3-, 4-, and 5-year survival rates among all patients were 42.3%, 9.6%, 4.3%, 3.2%, and 2.2%, respectively.

Compared with short-term survivors (< 24 months), long-term survivors were more likely to present at age < 3 years (11% vs 3%) or > 10 years (33% vs 23%; P < .001 for both), to have longer symptom duration before diagnosis (eg, 12–24 weeks for 12% vs 8%, > 24 weeks for 16% vs 4%; overall P < .001), and to have received systemic therapy at diagnosis (88% vs 75%, P = .005). Short-term survivors were more likely to present with cranial nerve palsy (83% vs 73%, P = .008), ring enhancement (38% vs 23%, P = .007), necrosis (42% vs 26%, P = .009), and extrapontine extension (92% vs 86%, P = .04).

Molecular characterization of 181 tumors, accounting for 48% of tumors assessed by biopsy (n = 299) or autopsy (n = 77), showed that long-term survivors were more likely to have HIST1H3B mutation (odds ratio = 1.28, P = .002).

The investigators concluded, “We report clinical, radiologic, and molecular factors that correlate with survival in children and young adults with DIPG, which are important for risk stratification in future clinical trials.”

The study was supported by the Diffuse Intrinsic Pontine Glioma Collaborative, The Cure Starts Now Foundation, Hope for Caroline Foundation, Julian Boivin Courage for Cures Foundation, Abbie’s Army, and many others.

Maryam Fouladi, MD, of Cincinnati Children’s Hospital Medical Center, is the corresponding author for the Journal of Clinical Oncology article.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.