Safety and tolerability of velafermin (CG53135-05) in patients receiving high-dose chemotherapy and autologous peripheral blood stem cell transplant.
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.
Schuster
Michael W
MW
The Weill Medical College of Cornell University, New York, NY 10021, USA. mwschust@med.cornell.edu
Shore
Tsiporah B
TB
Harpel
John G
JG
Greenberg
June
J
Jalilizeinali
Bita
B
Possley
Scott
S
Gerwien
Robert W
RW
Hahne
William
W
Halvorsen
Yuan-Di C
YD
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
D000970:Antineoplastic Agents; C416003:FGF20 protein, human; D005346:Fibroblast Growth Factors
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