Initial results of a phase II trial of high dose radiation therapy, 5- fluorouracil, and cisplatin for patients with anal cancer (E4292): An Eastern Cooperative Oncology Group study

James A. Martenson*, Stuart R. Lipsitz, Henry Wagner, Edward H. Kaplan, Larry A. Otteman, Lynn M. Schuchter, Edward G. Mansour, Mark S. Talamonti, Al Bowen Benson

*Corresponding author for this work

Research output: Contribution to journalArticle

110 Scopus citations


Purpose: A prospective clinical trial was performed to assess the response and toxicity associated with the use of high dose radiation therapy, 5-fluorouracil, and cisplatin in patients with anal cancer. Methods and Materials: Patients with anal cancer without distant metastasis were eligible for this study. Radiation therapy consisted of 59.4 Gy in 33 fractions; a 2 week break in treatment was taken after 36 Gy had been given. A treatment of 5-fluorouracil, 1,000 mg/m2 per day intravenously, was given for the first 4 days of radiation therapy, and cisplatin, 75 mg/m2 intravenously, was given on day 1 of radiation therapy. A second course of 5-fluorouracil and cisplatin was given after 36 Gy of radiation, when the radiation therapy was resumed. Results: Nineteen patients entered this study and received treatment. Thirteen (68%) had a complete response, 5 (26%) had a partial response, and 1 (5%) had stable disease. The patient with stable disease and one of the patients with a partial response had complete disappearance of tumor more than 8 weeks after completion of radiation therapy. Fifteen patients had toxicity of Grade 3 or higher: the worst toxicity was Grade 3 in eight patients, Grade 4 in six patients, and Grade 5 in one patient. The most common form of toxicity of Grade 3 or higher was hematologic. The one lethal toxicity was due to pseudomembranous colitis, which was a complication of antibiotic therapy for a urinary tract infection. Conclusion: Radiation therapy, cisplatin, and 5-fluorouracil resulted in an overall response rate of 95%. Significant toxicity occurred, an indication that this regimen is near the maximal tolerated dose. A Phase III clinical trial is planned in which radiation therapy, cisplatin, and 5-fluorouracil will be used as an experimental arm.

Original languageEnglish (US)
Pages (from-to)745-749
Number of pages5
JournalInternational Journal of Radiation Oncology Biology Physics
Issue number4
StatePublished - Jul 1 1996



  • 5-Fluorouracil
  • Anal cancer
  • Cisplatin
  • Radiation therapy
  • Sphincter preservation

ASJC Scopus subject areas

  • Radiation
  • Oncology
  • Radiology Nuclear Medicine and imaging
  • Cancer Research

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