A phase III, randomized, double-blind, placebo-controlled, study of iseganan for the reduction of stomatitis in patients receiving stomatotoxic chemotherapy.
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.
Giles
Francis J
FJ
M.D. Anderson Cancer Center, Department of Leukemia, The University of Texas, 1515 Holcombe Boulevard, Box 428, Houston, TX 77030, USA. frankgiles@aol.com
Rodriguez
Roberto
R
Weisdorf
Daniel
D
Wingard
John R
JR
Martin
Paul J
PJ
Fleming
Thomas R
TR
Goldberg
Stuart L
SL
Anaissie
Elias J
EJ
Bolwell
Brian J
BJ
Chao
Nelson J
NJ
Shea
Thomas C
TC
Brunvand
Mark M
MM
Vaughan
William
W
Petersen
Finn
F
Schubert
Mark
M
Lazarus
Hillard M
HM
Maziarz
Richard T
RT
Silverman
Margarida
M
Beveridge
Roy A
RA
Redman
Rebecca
R
Pulliam
Janis G
JG
Devitt-Risse
Patricia
P
Fuchs
Henry J
HJ
Hurd
David D
DD
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
D023181:Antimicrobial Cationic Peptides; D000970:Antineoplastic Agents; D010455:Peptides; D010919:Placebos; D011506:Proteins; C119872:antimicrobial peptide IB-367