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Neoadjuvant therapy, liver transplant effectively treated perihilar cholangiocarcinoma

Patients with perihilar cholangiocarcinoma treated with neoadjuvant chemoradiation and liver transplantation experienced high rates of recurrence-free survival in a recent study.

Researchers evaluated data on 287 patients with perihilar cholangiocarcinoma treated with neoadjuvant chemoradiation followed by liver transplant at 12 US facilities between 1993 and 2010. Among these patients, 99% completed external radiation, 98% received radiosensitizing therapy, 75% underwent brachytherapy and 65% received maintenance chemotherapy. Follow-up was conducted for a median of 2.5 years on each patient from listing for transplantation.

Of the original cohort, 71 dropped out before receiving transplantation after a median of 4.6 months, with an average increase in drop-out rate of 11.5% every 3 months. Survival rates among intent-to-treat patients were 68% within 2 years and 53% after 5 years. Among the patients who received transplants, 78% survived (95% CI, 72-84%) after 2 years, 65% (95% CI, 57-73%) after 5 years and 59% (95% CI, 49-69%) after 10 years.

After a median of 1.2 years, 122 participants died, including 60 before transplant. The most common causes of death included tumor progression in 52 pre-transplant patients and recurrence in 40 post-transplant patients. Following transplant, recurrence was seen in 43 patients (20%), and re-transplant was necessary in 22 participants after a median of 4.6 months.

Survival time was impacted by the presence of prior malignancy within 5 years and by not fitting the United Network of Organ Sharing criteria (HR=2.98; 95% CI, 1.79-4.95). Tumor size had the largest impact on survival, with a survival rate of 32% in 5 years among patients with a tumor larger than 3 cm compared with 69% among those with smaller tumors (P<.001). Neither differences in surgical staging (HR=0.73; 95% CI, 0.37-1.43) nor the use of brachytherapy (HR=1.05; 95% CI, 0.60-1.85) were found to significantly impact recurrence-free survival.

“This study confirms excellent outcomes of neoadjuvant chemoradiotherapy followed by [liver transplantation] for patients with perihilar cholangiocarcinoma,” the researchers wrote. “The central challenge for the future will be to gain a greater understanding of the tumor biology to reduce waiting list drop-out and post-transplant recurrence either by further refinements in patient selection or, ideally, by more effective chemoradiotherapy.”