Role of temozolomide in the treatment of newly diagnosed diffuse brainstem glioma in children: experience at a single institution

The purpose of this study was to assess the efficacy of TMZ on diffuse brainstem glioma, either concomitant with radiotherapy or as an adjuvant treatment after radiotherapy in children.

Eighteen children (median age at diagnosis was 8.3 years) meet the following criteria: (1) newly diagnosed diffuse brainstem glioma; (2) aged less than 18 years old, which were treated with TMZ at Taipei Veterans General Hospital from January 2004 to December 2008. They were divided into two groups according to treatment modalities: a radiotherapy alone followed by adjuvant TMZ (RT+TMZ) group received conventional radiation after initial diagnosis, and a concomitant chemoradiotherapy followed by adjuvant TMZ (CCRT+TMZ) group received concurrent chemotherapy during radiation with TMZ (75 mg/M²/day). After completion of the radiotherapy, TMZ (150 mg/M²) was administered once per day for five consecutive days for all enrolled patients in each 28-day cycle. We evaluated the progression-free survival in both groups of patients.

There were 10 patients in RT+TMZ group and eight in CCRT+TMZ group. All patients experienced progression of disease. Twelve patients (75%) died, and all deaths were attributed to the disease progression. The median progression-free survival (PFS) was 7.4 months for the RT+TMZ group and 6.4 months for the CCRT+TMZ group. The 6-month and 1-year PFS in the RT+TMZ group were 70% (SD 14%) and 30% (SD 14%), respectively, and in the CCRT+TMZ group, they were 50% (SD 17%) and 0%, respectively. The log-rank test in PFS between the two groups was not statistically significant.

In this study, CCRT with TMZ followed by adjuvant TMZ did not result in a better outcome when compared with RT alone followed by adjuvant TMZ. In addition, TMZ either as adjuvant therapy or as CCRT did not improve the prognosis of the patients with newly diagnosed diffuse brainstem glioma.