title: A phase III, randomized, double-blind, placebo-controlled, study of iseganan for the reduction of stomatitis in patients receiving stomatotoxic chemotherapy.
abstract: 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.
authors.affiliation: M.D. Anderson Cancer Center, Department of Leukemia, The University of Texas, 1515 Holcombe Boulevard, Box 428, Houston, TX 77030, USA. frankgiles@aol.com
authors.forename: Francis J
authors.initials: FJ
authors.lastname: Giles
authors.forename: Roberto
authors.initials: R
authors.lastname: Rodriguez
authors.forename: Daniel
authors.initials: D
authors.lastname: Weisdorf
authors.forename: John R
authors.initials: JR
authors.lastname: Wingard
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Martin
authors.forename: Thomas R
authors.initials: TR
authors.lastname: Fleming
authors.forename: Stuart L
authors.initials: SL
authors.lastname: Goldberg
authors.forename: Elias J
authors.initials: EJ
authors.lastname: Anaissie
authors.forename: Brian J
authors.initials: BJ
authors.lastname: Bolwell
authors.forename: Nelson J
authors.initials: NJ
authors.lastname: Chao
authors.forename: Thomas C
authors.initials: TC
authors.lastname: Shea
authors.forename: Mark M
authors.initials: MM
authors.lastname: Brunvand
authors.forename: William
authors.initials: W
authors.lastname: Vaughan
authors.forename: Finn
authors.initials: F
authors.lastname: Petersen
authors.forename: Mark
authors.initials: M
authors.lastname: Schubert
authors.forename: Hillard M
authors.initials: HM
authors.lastname: Lazarus
authors.forename: Richard T
authors.initials: RT
authors.lastname: Maziarz
authors.forename: Margarida
authors.initials: M
authors.lastname: Silverman
authors.forename: Roy A
authors.initials: RA
authors.lastname: Beveridge
authors.forename: Rebecca
authors.initials: R
authors.lastname: Redman
authors.forename: Janis G
authors.initials: JG
authors.lastname: Pulliam
authors.forename: Patricia
authors.initials: P
authors.lastname: Devitt-Risse
authors.forename: Henry J
authors.initials: HJ
authors.lastname: Fuchs
authors.forename: David D
authors.initials: DD
authors.lastname: Hurd
mesh_terms: D023181:Antimicrobial Cationic Peptides; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation; D015331:Cohort Studies; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009061:Mouth Mucosa / Q000187:drug effects; D009369:Neoplasms / Q000188:drug therapy*; D010455:Peptides; D010919:Placebos; D011506:Proteins / Q000627:therapeutic use*; D033581:Stem Cell Transplantation; D013280:Stomatitis / Q000139:chemically induced / Q000517:prevention & control*; D011795:Surveys and Questionnaires; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D023181:Antimicrobial Cationic Peptides; D000970:Antineoplastic Agents; D010455:Peptides; D010919:Placebos; D011506:Proteins; C119872:antimicrobial peptide IB-367