Table 1.

Small-molecule EGFR TKIs: phase II trials in recurrent and newly diagnosed glioblastoma and comparison with historical controls

ReferenceDosenObjective response (CR + PR), %Disease control (CR + PR + SD), %Median PFS (mo)6-mo PFS (%)Median OS
Gefitinib (recurrent GBM)
    Rich et al. (24)500–1,000 g5304221310
    Lieberman et al. (25)*500–1,500 g3813 (5 PR)NA29NA
    Franceschi et al. (26)*250 mg160132126
Gefitinib (newly diagnosed GBM)
    Uhm et al. (27)*500–1,000 g98NANA7NA11
Gefitinib + everolimus (recurrent GBM)
    Nguyen et al. (50)*250 mg (gefitinib); 30–70 mg (everolimus)1910 (2 PR)47 (including 2 minor responses)3NA7
Erlotinib (recurrent GBM)
    Raizer et al. (28)*150 mg3001330NA
    Cloughesy et al. (29)*150–500 mg488 (1 CR; 3 PR)41217NA
    Vogelbaum et al. (30)*150 mg3126 (8 PR)423266
Historical controls (recurrent GBM; refs. 2, 3)5–633–422–315–216–7
  • Abbreviations: GBM, glioblastoma; CR, complete response; PR, partial response; SD, stable disease; NA, not available.

  • * Preliminary results presented in the form of abstract.