Table 2.
HER2/Neu (ERBB2) expression in cholangiocarcinoma and response to therapy
| Study | N/year | Patients | Therapy used | Outcome (response/duration—months) |
|---|---|---|---|---|
| Javle et al. (MyPathway Clinical trial—NCT02091141)18 | 11 patients/ 2017 | Refractory metastatic biliary cancer with HER2 amplification/overexpression (8 patients) or putative activating mutations by gene sequencing (3 patients),18 FISH, or IHC | Pertuzumab + trastuzumab until disease progression or unacceptable toxicity | 4 patients had partial responses (PR) and 3 had stable disease (SD) for >4 months |
| Javle et al.27 | 2015 | Gallbladder cancer—9 patients CCA—5 patients | Trastuzumab, lapatinib, or pertuzumab | 1 CR, 4 PR and 4 SD in gallbladder cases; 1 mixed response on lapatinib with HER2 mutation. Duration of response 8–160+ weeks (median 40 weeks); 3 still on therapy. 1 HER2 amplification was acquired post-FGFR fusion therapy. No responses in CCA cases |
| Nam et al.32 | 2016 | 3 patients with gallbladder cancer | All 3 patients received trastuzumab alongside single- or doublet chemotherapy | 2 PR; 1 SD. Duration of response 12–34 weeks. |
| Subbaiah et al.33 | 2013 | 1 patient with gallbladder cancer | Phase-1 study: anti-HER-2/neu antibody trastuzumab and the HER-2/neu tyrosine kinase inhibitor lapatinib, and then with trastuzumab and erlotinib | SD Duration of response 10.8 months |
| Churi et al.19 | 2014 | 2 patients with HER2/neu mutations | Trastuzumab and lapatinib, respectively | No responses observed |
| Lee et al.22 | 2012 | Overexpression of HER2 and HER3 was observed in 6 % (13/224) and 39 % (90/230) of EHCCs, respectively | No treatment data provided | – |
| Mckay et al.34 | 2011 | Expression data only; only CC patients; no gallbladder cancer patients. 19 cases were positive per IHC (10 were scored as +2 and one as +3) | No treatment data provided | – |
| Sorscher et al.30 | 2013 | Gallbladder cancer—1 patient HER2/neu+++ | Trastuzumab + weekly paclitaxel (chemo later discontinued) | Marked partial response (5.4 cm → 1.3 cm). Duration of response ongoing (at least 6 months) |
| Jung et al.28 | 2017 | 3/84 patients with HER2/neu+ distal extrahepatic CC (3.6%) | No treatment data provided | – |
| Yoshikawa et al.21 | 2007 | HER2/neu expression 0.9% in intrahepatic CC and 8.5% in extrahepatic CC | No treatment data provided | – |