What are the most effective treatments for cholangiocarcinoma?

After the sudden onset of jaundice, our uncle (72 years old and in excellent health) was diagnosed with cholangiocarcinoma, unfortunately already in an advanced stage. We have never heard of this tumor and we are in shock. What are the best cures?

He answers Lorenza RimassaAssociate Professor of Medical Oncology at Humanitas University

Cholangiocarcinoma a tumor that arises from the bile ducts, in or outside the liver, or from the gallbladder
, very often it has no initial symptoms and grows rapidly. Even when present, the first signs are quite generic, such as for example abdominal pain, weight loss, nausea, tiredness, which can easily be underestimated or confused with those of other pathologies. It is not infrequently so diagnosed when it is already in an advanced or metastatic stage. a rare tumor (there are about 5 thousand new cases diagnosed every year in Italy), but rising and aggressive. Once the diagnosis is obtained, the first thing to understand whether the patient is a candidate for surgery, which could allow him to obtain definitive healing. Unfortunately only about 25% of patients are operable and, even so, a high percentage go through disease recurrence.

For these patients we proceed with the
chemotherapy alone or, more recently, combined with immunotherapy
with durvalumab (a drug not yet reimbursed by our Italian health system, but available free of charge through the so-called early access programme, for which the oncologist applies to the pharmaceutical manufacturer for each patient), and with new targeted drugs. therefore it is essential to perform the molecular profiling test which is used to understand if there are any genetic alterations (by now we know several such as IDH1 mutations, FGFR2 translocations, HER2 amplifications, to name a few), which make the disease potentially treatable precisely with the new molecular target drugs. One of these, pemigatinib, already available through the Italian health system, for patients with tumors with translocations of FGFR2; others are still awaiting approval. However, the possibility of use depends on the possible mutation present in the tumor of the individual patient. A few days ago, for example, the European green light arrived (and the Italian one is expected) for patients with locally advanced or metastatic cholangiocarcinoma, previously treated with at least one line of medical therapy, to receive ivosideniba targeted drug against the IDH1 mutation, which works by slowing the evolution of the tumor and therefore prolonging the life of patients.

An equally important aspect is that the treatment is well tolerated and has been shown to offer a benefit to patients also in terms of quality of life. We therefore hope to be able to use it as soon as possible also for our patients, within the context of the national health system or even in research programs aimed at gaining even better knowledge of the characteristics of this drug. Since for inoperable patients the goal we set ourselves with medical therapy is to lengthen survival while maintaining a good quality of lifethe new targeted drugs are of great help as they allow for longer survival without the side effects of chemotherapy. Finally, since cholangiocarcinoma is a rare tumor and the definition of the therapeutic strategy is complex, it is essential to be treated in a referral centrewhere there is a multidisciplinary team and the doctors have specific experience in all aspects of the pathology, from diagnosis to molecular profiling, from surgery (not simple due to the area itself in which the neoplasm is located) to medical therapy.