Diffuse intrinsic pontine gliomas (DIPG) at recurrence: is there a window to test new therapies in some patients?
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Updated: Feb 17, 2026
A Protocol for Rapid Post-mortem Cell Culture of Diffuse Intrinsic Pontine Glioma DIPG
Published on: March 7, 2017
M J Lobon-Iglesias1,2, G Giraud1,3, D Castel1,2
1Department of Pediatric and Adolescent Oncology, Gustave Roussy and University Paris-Saclay, Villejuif, France.
Abstract:
Children with diffuse intrinsic pontine glioma (DIPG) need new and more efficient treatments. They can be developed at relapse or at diagnosis, but therefore they must be combined with radiotherapy. Survival of children after recurrence and its predictors were studied to inform the possibility to design early phase clinical trials for DIPG at this stage. Among 142 DIPG patients treated between 1998 and 2014, 114 had biopsy-proven DIPG with histone H3 status available for 83. We defined as long survivors' patients who survived more than 3 months after relapse which corresponds to the minimal life expectancy requested for phase I/II trials. Factors influencing post-relapse survival were accordingly compared between short and long-term survivors after relapse. Fifty-seven percent of patients were considered long survivors and 70% of them had a Lansky Play Scale (LPS) above 50% at relapse. Patients who became steroids-independent after initial treatment for at least 2 months had better survival after relapse (3.7 versus 2.6 months, p = 0.001). LPS above 50% at relapse was correlated with better survival after relapse (3.8 versus 1.8 months, p < 0.001). Patients with H3.1 mutation survived longer after relapse (4.9 versus 2.7 months, p = 0.007). Patients who received a second radiotherapy at the time of relapse had an improved survival (7.5 versus 4 months, p = 0.001). In the two-way ANOVA analysis, steroid-independence and LPS predicted survival best and the type of histone H3 (H3.1 or H3.3) mutated did not improve prediction. Survival of many DIPG patients after relapse over 3 months would make possible to propose specific trials for this condition. Steroid-independence, H3 mutation status and LPS should be considered to predict eligibility.
Insights
New treatments for diffuse intrinsic pontine glioma (DIPG) are needed. This study identified factors like steroid independence and Lansky Play Scale performance that predict survival after relapse, informing future clinical trial eligibility for DIPG patients.
Area of Science:
- Pediatric Oncology
- Neuro-oncology
- Clinical Trial Design
Background:
- Diffuse intrinsic pontine glioma (DIPG) is a rare and aggressive childhood brain tumor with limited treatment options.
- Developing new therapies requires understanding survival predictors at diagnosis and relapse.
Purpose of the Study:
- To analyze survival rates and identify predictors of post-relapse survival in pediatric DIPG patients.
- To inform the design of early-phase clinical trials for DIPG.
Main Methods:
- Retrospective analysis of 142 DIPG patients treated between 1998 and 2014.
- Comparison of factors influencing survival between short-term (<3 months) and long-term (≥3 months) survivors post-relapse.
- Histone H3 mutation status and Lansky Play Scale (LPS) were assessed.
Main Results:
- 57% of patients survived over 3 months post-relapse. Steroid independence, LPS >50%, and H3.1 mutation were associated with better survival.
- Second radiotherapy at relapse significantly improved survival (7.5 vs. 4 months).
- Steroid independence and LPS were the strongest predictors of survival, outperforming H3 mutation type.
Conclusions:
- A significant proportion of DIPG patients can survive >3 months post-relapse, supporting the feasibility of early-phase clinical trials.
- Steroid independence, LPS, and H3 mutation status are crucial factors for predicting eligibility in DIPG clinical trials.
- Future trials should consider these predictors to effectively stratify patients and evaluate novel therapeutic strategies.
