title: Safety and tolerability of velafermin (CG53135-05) in patients receiving high-dose chemotherapy and autologous peripheral blood stem cell transplant.
abstract: OBJECTIVE
  The objective of this study was to evaluate the safety and tolerability of velafermin in patients at risk of developing severe oral mucositis (OM) from chemotherapy.
  
  
  METHODS
  This study was a single-center, open-label, single-dose escalation, phase I trial in patients undergoing high-dose chemotherapy (HDCT) and autologous peripheral blood stem cell transplant (PBSCT). Velafermin was administered 24 h after stem cell infusion as a single intravenous dose infused over 15 min. Clinical safety variables were assessed and OM status scored daily for 30 days using the World Health Organization (WHO) grading scale.
  
  
  RESULTS
  Thirty patients were treated with velafermin at doses of 0.03 (n = 10), 0.1 (n = 10), 0.2 (n = 8), or 0.33 mg/kg (n = 2). Patients were diagnosed with multiple myeloma (n = 16), non-Hodgkin's lymphoma (n = 12), acute myelogenous leukemia (n = 1), or desmoplasmic round cell tumor (n = 1). Velafermin was well tolerated at doses up to 0.2 mg/kg. There were no drug-related serious adverse events. No patient discontinued because of adverse events; however, two patients administered 0.33 mg/kg developed adverse reactions immediately after infusion of the study drug. No other patients were treated at this dose level. The most frequent (>35% of patients) treatment-emergent adverse events were diarrhea, fatigue, pyrexia, vomiting, and nausea. Most adverse events were mild or moderate and resolved the same day without sequelae. Eight (27%) patients developed WHO grade 3 or 4 OM during the study; seven of these patients received high-dose melphalan as a conditioning regimen.
  
  
  CONCLUSIONS
  Velafermin was well tolerated by autologous PBSCT patients at doses up to 0.2 mg/kg.
authors: The Weill Medical College of Cornell University, New York, NY 10021, USA. mwschust@med.cornell.edu
authors: Michael W
authors: MW
authors: Schuster
authors: Tsiporah B
authors: TB
authors: Shore
authors: John G
authors: JG
authors: Harpel
authors: June
authors: J
authors: Greenberg
authors: Bita
authors: B
authors: Jalilizeinali
authors: Scott
authors: S
authors: Possley
authors: Robert W
authors: RW
authors: Gerwien
authors: William
authors: W
authors: Hahne
authors: Yuan-Di C
authors: YD
authors: Halvorsen
mesh_terms: D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D005346:Fibroblast Growth Factors / Q000009:adverse effects* / Q000627:therapeutic use; D019337:Hematologic Neoplasms / Q000150:complications / Q000188:drug therapy / Q000628:therapy; D006801:Humans; D008297:Male; D008875:Middle Aged; D036102:Peripheral Blood Stem Cell Transplantation; D013280:Stomatitis / Q000139:chemically induced / Q000188:drug therapy*; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; C416003:FGF20 protein, human; D005346:Fibroblast Growth Factors
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