'Dismal' survival demands change in bile duct cancer therapy

An international group of cancer experts has called for fundamental change to healthcare approaches for bile duct cancer, which kills three-quarters of patients within a year of diagnosis.

The group of 147 opinion leaders, which includes Prof John Bridgewater of UCL Cancer Institute in the UK, is calling for rapid improvements in diagnosis, treatment, and research for this type of cancer, also known as cholangiocarcinoma (CCA), in a consensus statement published in Nature Reviews Gastroenterology & Hepatology.

CCA is a rare but aggressive disease that usually develops and progresses silently, meaning most people are diagnosed at a late stage when treatment options are limited. Symptoms can include jaundice, abdominal pain, weight loss, pale stools and itching, but are often mistaken for less serious conditions.

Despite several advancements in treatment over the past decade, the prognosis of patients with CCA continues to be dismal, with a five-year overall survival rate of less than 20%. Up to 90% of patients are diagnosed too late for surgery, which is currently the only potential cure, and the cancer kills around 3,000 people in England alone every year.

There is a desperate need for earlier diagnosis, through greater awareness of symptoms and faster referral pathways, along with wider access to specialist surgery and cancer centres, according to the experts from the European Network for the Study of Cholangiocarcinoma (ENSCCA).

They also call for routine molecular testing to match patients with available targeted treatments, such as drugs that address genetic alterations such as IDH1/2 mutations, FGFR2 fusions, HER2, and BRAF V600E mutations, and increased funding for research and clinical trials of new therapies.

Molecular testing is critical because targeted drugs and immunotherapies such as checkpoint inhibitors can help patients live longer, but they can only be used if doctors know the cancer's specific genetic makeup.

"Bile duct cancer remains one of the hardest cancers to treat. Earlier diagnosis, better access to specialist care, and routine molecular testing could dramatically improve survival," said Prof Bridgewater, who also noted that the incidence of CCA has been steadily rising, including among younger adults, placing growing pressure on NHS cancer services.

"Too many UK patients are diagnosed when it is already too late," he added. "Faster diagnosis and fair access to advanced testing and treatments must become national priorities."

The UK's recently revealed National Cancer Plan does not include a specific target for CCA, but does include measures for rare and less common cancers and an objective for England to be in the top 25% of 28 countries in terms of survival for these diseases by 2035.

Image by Mohamed Hassan from Pixabay