Concurrent chemoradiotherapy with temozolomide in newly pediatric diffuse intrinsic pontine glioma: clinical outcomes and safety analysis.

BACKGROUND

Diffuse intrinsic pontine glioma (DIPG) is one of the most aggressive pediatric brain tumors, with a median overall survival of less than 12 months despite radiotherapy remaining the current standard of care. This study aimed to evaluate the clinical outcomes and toxicity of concurrent radiotherapy with temozolomide followed by adjuvant therapy in newly diagnosed pediatric DIPG.

METHODS

Retrospective analysis of 24 consecutive pediatric DIPG patients (2006-2020) treated with focal radiotherapy (45-59.4 Gy) plus concurrent temozolomide (75 mg/m2/day), followed by adjuvant temozolomide (200 mg/m2/day, days 1-5, every 28 days for ≤13 cycles) was conducted. Primary endpoints included overall survival (OS), progression-free survival (PFS), and treatment-related toxicity. Response assessment utilized standardized neuroimaging criteria at predetermined intervals.

RESULTS

Median age was 7 years (range, 3-16 years). H3K27M mutation was detected in 74% of biopsied cases (14/19). Post-radiotherapy imaging showed partial response in 12 patients (50%), stable disease in 9 (38%), and progression in 3 (12%). Median OS was 12.0 months [95% confidence interval (CI): 7.0-14.0]; median PFS was 9.0 months (95% CI: 7.0-12.0). One- and two-year OS rates were 54% and 8%, respectively. Grade 3-4 hematological toxicity occurred in 10 patients (42%): leucopenia (n=4), thrombocytopenia (n=6). No treatment-related deaths occurred.

CONCLUSIONS

Concurrent chemoradiotherapy achieved encouraging response rates with manageable toxicity profile. However, survival outcomes remain limited, underscoring the need for novel therapeutic approaches targeting specific molecular pathways in this devastating malignancy.