Photodynamic therapy for advanced bile duct cancer: Evidence for improved palliation and extended survival

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 fro m less than 50% to greater than 70% in a single-arm phase-II trial with a s tatistical power of 80% (Fleming's single step procedure; alpha or 0.05), T wenty-three consecutive patients (8 women, 15 men; 67 +/- 14 years) with no nresectable bile duct cancer (Bismuth type III n = 2, type IV n = 21) were treated with photodynamic therapy and biliary endoprosthesis. Photofrin (QL T Pharmaceuticals, Vancouver, Canada) (2 mg/kg body weight intravenously) w as 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 d iagnosis 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). T he median rate of local tumor response was 74%, 54%, 29%, and 67% after the first, second, third, fourth, and fifth photodynamic therapy. Time to prog ression ranged from 3 to 8 months. All patients, except 1 with diffuse live r metastases, improved in cholestasis, performance, and quality of life. Ph otodynamic therapy can prevent tumor occlusion of hilar bile ducts. The app arent benefit in survival time should be confirmed in a controlled trial ve rsus palliation by endoprosthesis only.