title: Topical Recombinant Human Epidermal Growth Factor for Oral Mucositis Induced by Intensive Chemotherapy with Hematopoietic Stem Cell Transplantation: Final Analysis of a Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial.
abstract: The aim of this study was to evaluate the efficacy and safety of recombinant human epidermal growth factor (rhEGF) oral spray for oral mucositis (OM) induced by intensive chemotherapy with hematopoietic stem cell transplantation. In this phase 2 study, patients were randomized to either rhEGF (50 microg/mL) or placebo in a 1:1 ratio. The primary endpoint was incidence of National Cancer Institute (NCI) grade ≥2 OM. A total of 138 patients were enrolled in this study. In the intention-to-treat analysis, rhEGF did not reduce the incidence of NCI grade ≥2 OM (p = 0.717) nor reduce its duration (p = 0.725). Secondary endpoints including the day of onset and duration of NCI grade ≥2 OM, the incidence of NCI grade ≥3 OM and its duration, and patient-reported quality of life were also similar between the two groups. In the per-protocol analysis, however, the duration of opioid analgesic use was shorter in the rhEGF group (p = 0.036), and recipients in the rhEGF group required a lower cumulative dose of opioid analgesics than those in the placebo group (p = 0.046), among patients with NCI grade ≥2 OM. Adverse events were mild and transient. This study found no evidence to suggest that rhEGF oral spray reduces the incidence of OM. However, further studies are needed to investigate the effect of rhEGF on OM-induced pain reduction after intensive chemotherapy.
authors.affiliation: Department of Internal Medicine, Seoul National University Bundang Hospital, Seongnam, Republic of Korea.
authors.forename: Ji-Won
authors.initials: JW
authors.lastname: Kim
authors.affiliation: College of Pharmacy, Seoul National University, Seoul, Republic of Korea.
authors.forename: Myeong Gyu
authors.initials: MG
authors.lastname: Kim
authors.affiliation: Department of Internal Medicine, Dongguk University Ilsan Medical Center, Goyang, Republic of Korea.
authors.forename: Hyun Jung
authors.initials: HJ
authors.lastname: Lee
authors.affiliation: Department of Internal Medicine, Seoul National University Hospital, Seoul, Republic of Korea.|Cancer Research Institute, Seoul National University, Seoul, Republic of Korea.|Biomedical Research Institute, Seoul National University Hospital, Seoul, Republic of Korea.
authors.forename: Youngil
authors.initials: Y
authors.lastname: Koh
authors.affiliation: Department of Internal Medicine, Chungbuk National University Hospital, Cheongju, Republic of Korea.
authors.forename: Ji-Hyun
authors.initials: JH
authors.lastname: Kwon
authors.affiliation: Department of Internal Medicine, Seoul National University Hospital, Seoul, Republic of Korea.|Cancer Research Institute, Seoul National University, Seoul, Republic of Korea.|Biomedical Research Institute, Seoul National University Hospital, Seoul, Republic of Korea.
authors.forename: Inho
authors.initials: I
authors.lastname: Kim
authors.affiliation: Department of Internal Medicine, Seoul National University Hospital, Seoul, Republic of Korea.|Cancer Research Institute, Seoul National University, Seoul, Republic of Korea.|Biomedical Research Institute, Seoul National University Hospital, Seoul, Republic of Korea.
authors.forename: Seonyang
authors.initials: S
authors.lastname: Park
authors.affiliation: Department of Internal Medicine, Seoul National University Hospital, Seoul, Republic of Korea.|Cancer Research Institute, Seoul National University, Seoul, Republic of Korea.|Biomedical Research Institute, Seoul National University Hospital, Seoul, Republic of Korea.
authors.forename: Byoung Kook
authors.initials: BK
authors.lastname: Kim
authors.affiliation: College of Pharmacy, Seoul National University, Seoul, Republic of Korea.|Research Institute of Pharmaceutical Sciences, Seoul National University, Seoul, Republic of Korea.
authors.forename: Jung Mi
authors.initials: JM
authors.lastname: Oh
authors.affiliation: College of Pharmacy, Korea University, Sejong, Republic of Korea.|Biomedical Research Center, Korea University Guro Hospital, Seoul, Republic of Korea.
authors.forename: Kyung Im
authors.initials: KI
authors.lastname: Kim
authors.affiliation: Department of Internal Medicine, Seoul National University Hospital, Seoul, Republic of Korea.|Cancer Research Institute, Seoul National University, Seoul, Republic of Korea.|Biomedical Research Institute, Seoul National University Hospital, Seoul, Republic of Korea.
authors.forename: Sung-Soo
authors.initials: SS
authors.lastname: Yoon
mesh_terms: D000287:Administration, Topical; D000293:Adolescent; D000328:Adult; D000368:Aged; D000701:Analgesics, Opioid / Q000627:therapeutic use; D000970:Antineoplastic Agents / Q000627:therapeutic use; D004311:Double-Blind Method; D004815:Epidermal Growth Factor / Q000008:administration & dosage* / Q000627:therapeutic use; D005260:Female; D019337:Hematologic Neoplasms / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015994:Incidence; D008297:Male; D008875:Middle Aged; D011788:Quality of Life; D011994:Recombinant Proteins / Q000008:administration & dosage* / Q000627:therapeutic use; D013280:Stomatitis / Q000188:drug therapy*; D055815:Young Adult
chemical_list: D000701:Analgesics, Opioid; D000970:Antineoplastic Agents; D011994:Recombinant Proteins; D004815:Epidermal Growth Factor