How do you treat metastatic adenocarcinoma of the ampulla of Vater?

True adenocarcinoma of the ampulla of Vater is an extremely rare cancer of the GI tract (<5 pts per million). Consequently, data is mostly derived from retrospective studies or large databases with few clinical trials available, especially with locally advanced and metastatic disease.

I personally have used gemcitabine with cisplatin based on the phase III ABC-02 trial, the largest randomized controlled trial in biliary tract cancers, which did include 20 patients with ampulla primary. There was...

Given its rarity, ampullary adenocarcinoma has often been grouped with other tumor types, such as biliary tract and small bowel cancers in prospective clinical studies (Valle et al., NEJM 2010; Overman et al., JCO 2009). As such, the optimal systemic treatment regimen has been debated and there are no clear consensus guidelines. Fluoropyrimidine or gemcitabine based regimens have been most commonly used.

As noted, ampullary adenocarcinoma may be further divided histologically into subtypes—intest...

In the resected setting: it would be prudent to ask the surgeon and pathologist about ampullary vs peri-ampullary cancer. Prognosis and treatment are different for both entities and also for subtype. As discussed above, there is not much data. Most oncologists tend to use mFOLFIRINOX or pancreatic type regimens for true ampullary cancer. If it is peri-ampullary, people tend to use biliary regimens such as ABC-02. If the patient is metastatic as in this case, it may be difficult to know for sure ...

I would think the treatments of both are becoming progressively similar; mFOLFIRINOX and FOLFOXIRI are virtually the same drugs and have proven the most active in the 2 individual types.

I would not favor a gemcitabine approach: there is activity, but more desirable outcomes can be reached with alternatives.