BACKGROUND: Biliary tract cancer (BTC) is a rare and lethal disease with few 
therapeutic options. Preclinical data suggest that the epidermal growth factor 
receptor (EGFR) pathway could be involved in its progression.
METHODS: This open-label, randomized phase 2 trial recruited chemotherapy-naive 
patients with advanced BTC displaying a wild-type (WT) KRAS status. Patients 
were randomized to gemcitabine (1000 mg/m(2) ) and oxaliplatin (100 mg/m(2) ) 
with (arm A) or without (arm B) panitumumab (6 mg/kg) for up to 12 cycles. The 
primary endpoint was progression-free survival (PFS) analyzed in an 
intention-to-treat fashion.
RESULTS: Eighty-nine patients (45 in arm A and 44 in arm B) were enrolled 
between June 2010 and September 2013. After a median follow-up of 10.1 months, 
the median PFS was 5.3 months (95% confidence interval, 3.3-7.2 months) in arm A 
and 4.4 months (95% confidence interval, 2.6-6.2 months) in arm B (P = .27). No 
survival differences were observed: the median overall survival was 9.9 months 
in arm A and 10.2 months in arm B (P = .42). In a subgroup analysis, no 
differences in PFS according to the site of the primary tumor were observed; 
patients with intrahepatic cholangiocarcinoma treated with panitumumab may have 
had a survival benefit in comparison with the control group (15.1 vs 11.8 
months, P = .13). As for safety, skin toxicity was the main adverse event in arm 
A (80% of the patients). A higher incidence of diarrhea (55.5% vs 31.8%), 
mucositis (22.2% vs 13.6%), and constipation (24.4% vs 15.9%) was seen in arm A.
CONCLUSIONS: These results confirm the marginal role of anti-EGFR therapy even 
for WT KRAS-selected BTC.