One hundred and ten patients with primary epidermoid cancers of the an
al canal were treated in a series of prospectively designed, nonrandom
ized protocols of split-course radiation therapy with concurrent admin
istration of 5-fluorouracil (5-FU) with or without mitomycin. The addi
tion of mitomycin was associated with improved primary tumor control r
ates (87 vs. 58% at 4 years, p = 0.005) and improved 4-year actuarial
cause-specific survival (80 vs. 64%, p = 0.02). Hematologic toxicity w
as the most frequent acute side effect of mitomycin use. No long-term
toxicity was attributed to mitomycin only. Mitomycin appeared to benef
it patients principally through improved control of cancer in the irra
diated volume; there was no evidence of reduced risk of extrapelvic me
tastases. Several investigators have reported high rates of control of
epidermoid anal cancers with preservation of anorectal function follo
wing concurrent treatment with mitomycin, 5-FU, and radiation. Mitomyc
in's role in anal cancer is being evaluated in a randomized clinical t
rial by the Radiation Therapy Oncology Group. The mechanisms of any in
teractions between mitomycin and radiation or other cytotoxic drugs in
clinical practice remain to be determined.