BACKGROUND: Fibroblast growth factor receptor 1 (FGFR1) amplification is a 
potential driving oncogene in squamous cell cancer (SCC) of the lung. The 
current phase 2 study evaluated the efficacy and tolerability of dovitinib, an 
FGFR inhibitor, in patients with advanced SCC of the lung.
METHODS: Patients with pretreated advanced SCC of the lung whose tumors 
demonstrated FGFR1 amplification of > 5 copies by fluorescence in situ 
hybridization were enrolled. Dovitinib at a dose of 500 mg was administered 
orally, once daily, on days 1 to 5 of every week, followed by 2 days off. The 
primary endpoint was overall response. Secondary endpoints were progression-free 
survival, overall survival, and toxicity.
RESULTS: All 26 patients were men with a median age of 68 years (range, 52-80 
years). The majority of patients were ever-smokers. The median duration of 
dovitinib administration (28 days per cycle) was 2.5 months (range, 0.7-8.6 
months). The overall response rate was 11.5% (95% confidence interval [95% CI], 
0.8%-23.8%) and the disease control rate was 50% (95% CI, 30.8%-69.2%), with 3 
patients achieving partial responses. Response durations for the patients with 
partial responses were ≥4.5 months, ≥ 5.1 months, and 6.1 months, respectively. 
After a median follow-up of 15.7 months (range, 1.2-25.6 months), the median 
overall survival was 5.0 months (95% CI, 3.6-6.4 months) and the median 
progression-free survival was 2.9 months (95% CI, 1.5-4.3 months). The most 
common grade 3 or higher adverse events were fatigue (19.2%), anorexia (11.5%), 
and hyponatremia (11.5%) (event severity was graded based on National Cancer 
Institute Common Terminology Criteria for Adverse Events [version 4.0]).
CONCLUSIONS: Treatment with dovitinib demonstrated modest efficacy in patients 
with advanced SCC with FGFR1 amplification. Further studies to evaluate other 
biomarkers correlated with the efficacy of dovitinib in patients with SCC are 
warranted. Cancer 2016;122:3024-3031. © 2016 American Cancer Society.