Median survival time of nonresectable hilar
bile duct cancer is only 4 to 6 months owing to
tumor spread in the biliary tree, refractory
cholestasis, and
sepsis or
liver failure. We explored whether local
photodynamic therapy of nonresectable
bile duct cancer could improve survival. A sample size of 23 patients is required to detect an increase in 6-month survival rate from less than 50% to greater than 70% in a single-arm phase-II trial with a statistical power of 80% (Fleming's single step procedure; alpha = 0.05). Twenty-three consecutive patients (8 women, 15 men; 67 +/- 14 years) with nonresectable
bile duct cancer (
Bismuth type III n = 2, type IV n = 21) were treated with
photodynamic therapy and biliary
endoprosthesis.
Photofrin (QLT
Pharmaceuticals, Vancouver, Canada) (2 mg/kg
body weight intravenously) was photoactivated after 1 to 4 days with
laser light (630 nm; 242 J/cm(2)) via endoscopic retrograde access. The 6-month survival rate was 91% after diagnosis and 74% after start of
photodynamic therapy (30-day mortality rate was 4%) at a median follow-up time of 10.3 months after diagnosis. Causes of death were
tumor progression (n = 9) and
bacterial infections (n = 4). The median rate of local
tumor response was 74%, 54%, 29%, and 67% after the first, second, third, fourth, and fifth
photodynamic therapy. Time to progression ranged from 3 to 8 months. All patients, except 1 with diffuse liver
metastases, improved in
cholestasis, performance, and quality of life.
Photodynamic therapy can prevent
tumor occlusion of hilar bile ducts. The apparent benefit in survival time should be confirmed in a controlled trial versus palliation by
endoprosthesis only.