Review Article

Gallbladder Cancer in the 21st Century

Table 7

Targeted therapy alone or in combination with chemotherapy in gallbladder and biliary tract cancer.

Targeted agent Disease siteNumber
GBC/total
Line of therapyResponse rateComments

Single agent targeting VGF
 Sorafenib [145]BTC12/31First6%
 Sunitinib [146]BTCNA/56Second 9%
Single agent targeting HER2
 Lapatinib [147]HCC or BTC17 BTC/57First & second0% in BTC
 Lapatinib [148]HCC or BTCNA/9Any0% Trial was stopped early due to futility
Other single agents
 Bortezomib [149]BTC6/20First, second, third5%Trial was stopped early due to futility
 Selumetinib [150]BTC7/28Second10%
Doublet of targeted agents
 Bevacizumab + erlotinib [151]BTC10/53First 17%
 Bevacizumab + erlotinib [152]Upper GI cancer16 BTC/102Second or later 6%
Targeted agents with chemotherapy
 GEMOX ± cetuximab [153]BTCNA/50First23%Response rate in control group: 29%
  GEMOX + cetuximab [154]BTCNA/30First63%9 patients underwent resection after response
  GEMOX ± cetuximab [155]BTC50/122First27.3%Response rate in control group: 15%
  GEMOX + bevacizumab [156]BTCNA/35First & second40%
 Gem + triapine [157]BTC18/33First 9%
 5FU/LV + imatinib [158]BTC19/41First8%
 GEMOX + erlotinib [159]BTC82/268First30%16% in chemotherapy arm alone. No difference in OS

BTC, biliary tract cancer; GEMOX, gemcitabine and oxaliplatin; HCC, hepatocellular cancer; NA, not applicable; OS, overall survival. Modified from [143].