OBJECTIVE: Axitinib is a potent and selective inhibitor of vascular endothelial 
growth factor receptors 1-3. This analysis compared efficacy and safety of 
axitinib plus gemcitabine in patients with advanced pancreatic cancer from 
Japan, North America and the European Union, enrolled in a randomized Phase III 
study.
METHODS: Patients (n = 632), stratified by disease extent, were randomly 
assigned (1:1) to receive axitinib/gemcitabine or placebo/gemcitabine. Axitinib 
was administered at a starting dose of 5 mg orally twice daily and gemcitabine 
at 1000 mg/m(2) once weekly for 3 weeks in 4 week cycles. Primary endpoint was 
overall survival.
RESULTS: Among Japanese patients, median overall survival was not estimable (95% 
confidence interval, 7.4 months-not estimable) with axitinib/gemcitabine (n = 
58) and 9.9 months (95% confidence interval, 7.4-10.5) with placebo/gemcitabine 
(n = 56) (hazard ratio 1.093 [95% confidence interval, 0.525-2.274]). Median 
survival follow-up (range) was 5.1 months (0.02-12.3) with axitinib/gemcitabine 
vs. 5.4 months (1.8-10.5) with placebo/gemcitabine. Similarly, no difference was 
detected in overall survival between axitinib/gemcitabine and 
placebo/gemcitabine in patients from North America or the European Union. Common 
adverse events with axitinib/gemcitabine in Japanese patients were fatigue, 
anorexia, dysphonia, nausea and decreased platelet count. Axitinib safety 
profile was generally similar in patients from the three regions, although there 
were differences in incidence of some adverse events. An exploratory analysis 
did not show any correlation between axitinib/gemcitabine-related hypertension 
and overall survival.
CONCLUSIONS: Axitinib/gemcitabine, while tolerated, did not provide survival 
benefit over gemcitabine alone in patients with advanced pancreatic cancer from 
Japan or other regions.