This study evaluated the effect of capecitabine and concomitant radiation therapy in children with newly diagnosed brainstem gliomas.

The open-label phase 2 study NO21125 (NCT01118377) evaluated the progression-free survival, safety, and pharmacokinetics of capecitabine (Xeloda®) rapidly disintegrating tablets and concomitant radiation therapy in children and adolescent patients with newly diagnosed brainstem glioma. There were 2 phases to the study: A 9-week radiation phase, followed by a 2-week rest period, and a 9-week post-radiation phase. In the radiation phase, capecitabine 650 mg/m^2 was administered orally twice daily for 9 weeks. Concomitantly, patients received radiation therapy (180 cGy fractions) 5 days a week for a total target dose of 56 Gy. During the 9-week post-radiation phase of the study, capecitabine 1250 mg/m^2 was administered orally twice daily for 14 days followed by a 7-day rest period. This cycle of 14 days treatment followed by 7 days rest was repeated 2 additional times. The dose could be adjusted according to toxicity and body surface area.

The single-arm phase 1 study NO18517 (NCT00532948) assessed the maximum tolerated dose and dose-limiting toxicities of capecitabine (Xeloda®) administered concurrently with radiation therapy in children with newly diagnosed diffuse intrinsic brain stem gliomas and high grade gliomas. Patients in the phase 1 study NO18517 who were diagnosed with intrinsic brainstem glioma and who were treated at the established maximum tolerated dose of capecitabine 650 mg/m^2/dose twice a day were included in the analyses of the phase 2 study NO21125.

The efficacy and safety results of study NO21125 are reported below.

Inclusion Criteria:

• Pediatric and adolescent patients ≥ 3 to < 18 years of age.
• Patients must have a newly diagnosed non-disseminated intrinsic infiltrating brainstem glioma.
• Karnofsky Performance Scale (if > 16 years of age) or Lansky Performance Score (if ≤ 16 years of age) ≥ 50% assessed within 2 weeks prior to registration to study.
• Patients must not have received any prior chemotherapy or bone marrow transplant for the treatment of brainstem glioma. Prior dexamethasone and/or surgery are allowed.
• Adequate organ function.

Exclusion Criteria:

• Patients receiving any other anticancer or experimental drug therapy.
• Patients with uncontrolled infection.
• Known dihydropyrimidine dehydrogenase (DPD) deficiency.