Multicentre phase II studies evaluating imatinib plus hydroxyurea in patients with progressive glioblastoma

D. A. Reardon, G. Dresemann, S. Taillibert, M. Campone, M. Van Den Bent, P. Clement, E. Blomquist, L. Gordower, H. Schultz, J. Raizer, P. Hau, J. Easaw, M. Gil, J. Tonn, A. Gijtenbeek, U. Schlegel, P. Bergstrom, S. Green, A. Weir, Z. Nikolova

Research output: Contribution to journalArticlepeer-review

107 Scopus citations


Background: We evaluated the efficacy of imatinib mesylate in addition to hydroxyurea in patients with recurrent glioblastoma (GBM) who were either on or not on enzyme-inducing anti-epileptic drugs (EIAEDs). Methods: A total of 231 patients with GBM at first recurrence from 21 institutions in 10 countries were enrolled. All patients received 500 mg of hydroxyurea twice a day. Imatinib was administered at 600 mg per day for patients not on EIAEDs and at 500 mg twice a day if on EIAEDs. The primary end point was radiographic response rate and secondary end points were safety, progression-free survival at 6 months (PFS-6), and overall survival (OS). Results: The radiographic response rate after centralised review was 3.4%. Progression-free survival at 6 months and median OS were 10.6% and 26.0 weeks, respectively. Outcome did not appear to differ based on EIAED status. The most common grade 3 or greater adverse events were fatigue (7%), neutropaenia (7%), and thrombocytopaenia (7%). Conclusions: Imatinib in addition to hydroxyurea was well tolerated among patients with recurrent GBM but did not show clinically meaningful anti-tumour activity.

Original languageEnglish (US)
Pages (from-to)1995-2004
Number of pages10
JournalBritish Journal of Cancer
Issue number12
StatePublished - Dec 2009


  • C-KIT
  • Glioblastoma
  • Imatinib mesylate
  • Platelet-derived growth factor

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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