BACKGROUND: We conducted a multicenter, 2-stage, open-label, phase II trial to 
assess the efficacy and safety of dacomitinib in adult patients with recurrent 
glioblastoma (GB) and epidermal growth factor receptor gene (EGFR) amplification 
with or without variant III (EGFRvIII) deletion.
METHODS: Patients with first recurrence were enrolled in 2 cohorts. Cohort A 
included patients with EGFR gene amplification without EGFRvIII mutation. Cohort 
B included patients with EGFR gene amplification and EGFRvIII mutation. 
Dacomitinib was administered (45 mg/day) until disease progression/unacceptable 
adverse events (AEs). Primary endpoint was progression-free survival (PFS; RANO 
criteria) at 6 months (PFS6).
RESULTS: Thirty patients in Cohort A and 19 in Cohort B were enrolled. Median 
age was 59 years (range 39-81), 65.3% were male, and Eastern Cooperative 
Oncology Group Performance Status 0/1/2 were 10.2%/65.3%/24.5%, respectively. 
PFS6 was 10.6% (Cohort A: 13.3%; Cohort B: 5.9%) with a median PFS of 2.7 months 
(Cohort A: 2.7 mo; Cohort B: 2.6 mo). Four patients were progression free at 6 
months and 3 patients were so at 12 months. Median overall survival was 7.4 
months (Cohort A: 7.8 mo; Cohort B: 6.7 mo). The best overall response included 
1 complete response and 2 partial responses (4.1%). Stable disease was observed 
in 12 patients (24.5%: eight in Cohort A and four in Cohort B). Diarrhea and 
rash were the most common AEs; 20 (40.8%) patients experienced grade 3-4 
drug-related AEs.
CONCLUSIONS: Dacomitinib has a limited single-agent activity in recurrent GB 
with EGFR amplification. The detailed molecular characterization of the 4 
patients with response in this trial can be useful to select patients who could 
benefit from dacomitinib.