A pediatric brain tumor consortium phase II trial of capecitabine rapidly disintegrating tablets with concomitant radiation therapy in children with newly diagnosed diffuse intrinsic pontine gliomas

Pediatr Blood Cancer. 2018 Feb;65(2):10.1002/pbc.26832. doi: 10.1002/pbc.26832. Epub 2017 Nov 1.


Background: We conducted a phase II study of oral capecitabine rapidly disintegrating tablets given concurrently with radiation therapy (RT) to assess progression-free survival (PFS) in children with newly diagnosed diffuse intrinsic pontine gliomas (DIPG).

Patients and methods: Children 3-17 years with newly diagnosed DIPG were eligible. Capecitabine, 650 mg/m2 /dose BID (maximum tolerated dose [MTD] in children with concurrent radiation), was administered for 9 weeks starting the first day of RT. Following a 2-week break, three courses of capecitabine, 1,250 mg/m2 /dose BID for 14 days followed by a 7-day rest, were administered. As prospectively designed, 10 evaluable patients treated at the MTD on the phase I trial were included in the phase II analyses. The design was based on comparison of the PFS distribution to a contemporary historical control (n = 140) with 90% power to detect a 15% absolute improvement in the 1-year PFS with a type-1 error rate, α = 0.10.

Results: Forty-four patients were evaluable for the phase II objectives. Capecitabine and RT was well tolerated with low-grade palmar plantar erythrodyesthesia, increased alanine aminotransferase, cytopenias, and vomiting the most commonly reported toxicities. Findings were significant for earlier progression with 1-year PFS of 7.21% (SE = 3.47%) in the capecitabine-treated cohort versus 15.59% (SE = 3.05%) in the historical control (P = 0.007), but there was no difference for overall survival (OS) distributions (P = 0.30). Tumor enhancement at diagnosis was associated with shorter PFS and OS. Capecitabine was rapidly absorbed and converted to its metabolites.

Conclusion: Capecitabine did not improve the outcome for children with newly diagnosed DIPG.

Keywords: capecitabine; child; clinical trial; diffuse intrinsic pontine glioma (DIPG); malignant glioma; radiation.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study

MeSH terms

  • Administration, Oral
  • Adolescent
  • Brain Stem Neoplasms / diagnosis
  • Brain Stem Neoplasms / therapy*
  • Capecitabine / administration & dosage*
  • Chemoradiotherapy*
  • Child
  • Child, Preschool
  • Female
  • Follow-Up Studies
  • Glioma / diagnosis
  • Glioma / therapy*
  • Humans
  • Male
  • Prospective Studies
  • Tablets


  • Tablets
  • Capecitabine