Successful photodynamic therapy for nonresectable cholangiocarcinoma: a randomized prospective study

Gastroenterology. 2003 Nov;125(5):1355-63. doi: 10.1016/j.gastro.2003.07.015.


Background & aims: In nonrandomized trials, photodynamic therapy (PDT) had a promising effect on nonresectable cholangiocarcinoma (NCC). This prospective, open-label, randomized, multicenter study with a group sequential design compared PDT in addition to stenting (group A) with stenting alone (group B) in patients with NCC.

Methods: In patients with histologically confirmed cholangiocarcinoma, endoscopic or percutaneous double stenting was performed. Patients fulfilling inclusion criteria were randomized to group A (stenting and subsequent PDT) and group B (stenting alone). For PDT, Photofrin 2 mg/kg body wt was injected intravenously 2 days before intraluminal photoactivation (wavelength, 630 nm; light dose, 180 J/cm(2)). Further treatments were performed in cases of residual tumor in the bile duct. The primary outcome parameter was survival time. Secondary outcome parameters were cholestasis and quality of life.

Results: PDT resulted in prolongation of survival (group A: n = 20, median 493 days; group B: n = 19, median 98 days; P < 0.0001). It also improved biliary drainage and quality of life.

Conclusions: PDT, given in addition to best supportive care, improves survival in patients with NCC. The study was terminated prematurely because PDT proved to be so superior to simple stenting treatment that further randomization was deemed unethical.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Bile Duct Neoplasms / diagnosis
  • Bile Duct Neoplasms / drug therapy*
  • Bile Duct Neoplasms / physiopathology
  • Bile Duct Neoplasms / therapy
  • Cholangiocarcinoma / diagnosis
  • Cholangiocarcinoma / drug therapy*
  • Cholangiocarcinoma / physiopathology
  • Cholangiocarcinoma / therapy
  • Dihematoporphyrin Ether / administration & dosage
  • Dihematoporphyrin Ether / adverse effects
  • Dihematoporphyrin Ether / therapeutic use*
  • Endoscopy
  • Humans
  • Injections, Intravenous
  • Mass Screening
  • Photochemotherapy
  • Photosensitizing Agents / administration & dosage
  • Photosensitizing Agents / adverse effects
  • Photosensitizing Agents / therapeutic use*
  • Quality of Life
  • Stents
  • Survival Analysis


  • Photosensitizing Agents
  • Dihematoporphyrin Ether