title: Clinical applications of palifermin: amelioration of oral mucositis and other potential indications.
abstract: Mucositis is one of the most significant toxicities in cancer patients undergoing cytotoxic treatment. It can have a negative impact on both quality of life and health economics. Severe oral mucositis can contribute to hospitalization, need for narcotic analgesics, total parentral nutrition, suboptimal delivery of anti-neoplastic treatment, and morbidity and mortality. Palifermin, a recombinant derivative of human keratinocyte growth factor, is the first active agent approved by the FDA for the prevention of severe oral mucositis in patients undergoing haematopoietic stem cell transplantation (HSCT). Several studies have also shown significant reduction in the incidence, severity and/or duration of oral mucositis in other high-risk settings such as concurrent chemoradiotherapy (CT/RT) for patients with head and neck cancer, and use of mucotoxic chemotherapeutic agents such as doxorubicin in sarcoma and fluorouracil for the treatment of colorectal cancer. The reduction in mucositis has translated into amelioration of symptoms and improvement in daily functioning as measured by patient-reported outcome in multiple studies. The clinical response to palifermin appears to be related in part to epithelial proliferation and mucosal thickening. Palifermin also has other potential clinical applications including the acceleration of immune reconstitution and inhibition of graft-versus-host disease in patients undergoing HSCT, and mitigation of dysphagia in lung cancer patients treated with concurrent CT/RT. Palifermin is generally well tolerated with mild-to-moderate skin and oral adverse events. Future studies may expand the use of palifermin into other areas that would benefit from its cytoprotective and regenerative effects.
authors.affiliation: Department of Sarcoma Medical Oncology, Section of Cytokines and Supportive Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Saroj
authors.initials: S
authors.lastname: Vadhan-Raj
authors.forename: Jenna D
authors.initials: JD
authors.lastname: Goldberg
authors.forename: Miguel-Angel
authors.initials: MA
authors.lastname: Perales
authors.forename: Dietmar P
authors.initials: DP
authors.lastname: Berger
authors.forename: Marcel R M
authors.initials: MR
authors.lastname: van den Brink
mesh_terms: D000818:Animals; D000893:Anti-Inflammatory Agents / Q000494:pharmacology / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000009:adverse effects; D059248:Chemoradiotherapy; D002986:Clinical Trials as Topic; D003680:Deglutition Disorders / Q000139:chemically induced / Q000188:drug therapy; D051523:Fibroblast Growth Factor 7 / Q000494:pharmacology / Q000627:therapeutic use*; D006258:Head and Neck Neoplasms / Q000188:drug therapy* / Q000532:radiotherapy; D006801:Humans; D008175:Lung Neoplasms / Q000188:drug therapy / Q000532:radiotherapy; D013280:Stomatitis / Q000139:chemically induced / Q000188:drug therapy*; D016896:Treatment Outcome
chemical_list: D000893:Anti-Inflammatory Agents; D000970:Antineoplastic Agents; D051523:Fibroblast Growth Factor 7
keywords: GVHD; HSCT; KGF; dysphagia; immune reconstitution; mucositis; oral mucositis; palifermin; palliative care