Pilot study of FMC (5-fluorouracil, mitomycin C, and cisplatin) with radiotherapy for patients with anal cancer

Objectives: Concurrent chemoradiotherapy (CRT) is the current standard of treatment for anal squamous carcinoma. However, local or metastatic recurrences remain significant after CRT with 5-fluorouracil (5-FU) and mitomycin C (MMC). Therefore, the present study evaluated the feasibility and efficacy of adding cisplatin to the classic CRT (5-FU, MMC, and radiotherapy).

Methods: Twenty patients with histologically confirmed squamous cell carcinoma of the anus without metastatic disease were enrolled at Kyungpook National University Medical Center (Daegu, Korea) between January 2005 and December 2014. The CRT comprised of two cycles of 5-FU 750 mg/m² days 1-4, MMC 10 mg/m² day 1, and cisplatin 60 mg/m² day 1 every 4 weeks and radiotherapy (59.6 Gy in 33 daily fractions). The primary endpoint was to determine the feasibility of FMC, while the secondary endpoints were the pathologic complete response (pCR) rate at 8 weeks following the completion of CRT, progression-free survival (PFS), and overall survival (OS).

Results: The treatment was generally well tolerated, and all patients received two cycles of FMC chemotherapy. Among the 20 patients, 17 were assessed for their pathologic response and 12 patients (70.6%) achieved pCR. The most common grade 3 or 4 hematologic toxicity was neutropenia (n = 3), while the most frequent severe non-hematologic toxicity was radiation dermatitis (n = 6, 30%). After a median follow-up duration of 45.5 months, the estimated 5-year PFS and overall survival rates were 88.9 and 88.1%, respectively.

Conclusion: CRT with two cycles of a FMC regimen was found to be feasible for patients with anal squamous carcinoma. Further study is warranted to evaluate the efficacy of CRT with a FMC regimen.