Defining clinically meaningful outcomes in the evaluation of new treatments for oral mucositis: oral mucositis patient provider advisory board.
Oral mucositis (OM)-related outcomes constituting a meaningful clinical advance in bone marrow transplant patients were considered by an interdisciplinary panel. Meaningful outcomes are essential in product development for OM, a condition without effective prevention or treatment. The most important outcomes to measure, the feasibility of measuring these in a clinical trial, and clinically meaningful differences in these outcomes were determined by the panel. Most important are reduction in oral pain and use of opioid analgesics, improvement in oral intake and quality of life, and reduction of hospitalization duration. Reduction in the severity of OM measured by an objective evaluation of oral mucosa could provide insight regarding the biologic activity of an intervention. Further data are required to define the precise relationship between reduction in visible OM and improvement in outcome. Minimally, clinical trials for OM should assess oral pain, opioid use, oral intake, and include objective assessment of OM.
IntraBiotics Pharmaceuticals, Inc., 1245 Terra Bella Avenue, Mountain View, CA 94043, USA.
Lisa A
LA
Bellm
Gail
G
Cunningham
Laurie
L
Durnell
June
J
Eilers
Joel B
JB
Epstein
Tom
T
Fleming
Henry J
HJ
Fuchs
Martha Nash
MN
Haskins
Mary M
MM
Horowitz
Paul J
PJ
Martin
Deborah B
DB
McGuire
Kevin
K
Mullane
Gerry
G
Oster
D000701:Analgesics, Opioid / Q000627:therapeutic use; D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002986:Clinical Trials as Topic; D004435:Eating; D023381:Endpoint Determination; D006760:Hospitalization; D006801:Humans; D009061:Mouth Mucosa / Q000473:pathology; D010146:Pain; D011788:Quality of Life; D013280:Stomatitis / Q000628:therapy*; D016896:Treatment Outcome
D000701:Analgesics, Opioid