The invasion and colonization of oral cavity mucosal tissues by microflora may contribute to the pathophysiology of ulcerative
oral mucositis (UOM).
Iseganan is an analog of
protegrin-1, a naturally occurring
peptide with broad-spectrum microbicidal activity. A randomized, double-blind, placebo-controlled study was conducted to evaluate the efficacy and safety of
iseganan in preventing UOM after stomatotoxic
therapy. Patients received an oral rinse, consisting of
iseganan 9mg or placebo, to be swished/swallowed six times daily, starting with stomatotoxic
therapy and continuing up to 21 days. Patients were assessed for
stomatitis and UOM, and administered a questionnaire evaluating mouth
pain and difficulty swallowing thrice weekly. The primary study efficacy endpoint was the proportion of patients who did not have peak
stomatitis NCI-CTC grade >or=2. Between November 2001 and June 2002, 502 patients were randomized to receive
iseganan (251) or placebo (251). Equivalent numbers of patients in both cohorts received bone marrow or peripheral blood allogeneic or autologous
stem cell transplantation (SCT). Forty-three percent and 37% of
iseganan and placebo patients, respectively, did not have peak
stomatitis grade =2 (P = 0.182). There was no significant difference between the cohorts in
stomatitis severity, incidence of UOM, peak mouth
pain, peak difficulty swallowing, amount of
opiate analgesics used, or adverse event type or incidence. A major impact of
Iseganan on reducing
stomatitis, UOM, or its clinical sequelae in patients receiving stomatotoxic
therapy was not detected on this study.