A phase II study of the EGFR inhibitor gefitinib in patients with acute myeloid leukemia.

Leuk Res
Authors
Keywords
Abstract

Novel therapies for the treatment of acute myeloid leukemia are required to overcome disease resistance and to provide potentially less toxic therapies for older adults. Prior clinical trials involving patients with non-small cell lung cancer have demonstrated the safety and biologic activity of the administration of EGFR inhibitors in carefully selected patients. The potential efficacy of this approach in patients with acute myeloid leukemia is unknown. The effects of gefitinib on differentiation induction and cell viability in AML cell lines and primary patient AML cells were previously reported and cell viability was inhibited in a clinically achievable range. To determine if EGFR inhibitors would be therapeutically efficacious in advanced AML, we performed a phase II trial in which 18 patients with a median age of 72 (range, 57-84 years) were treated with gefitinib (750mg orally daily). While there were no unexpected toxicities, no patients experienced an objective response, though one had stable disease lasting 16 months. We conclude that in spite of pre-clinical activity and anecdotal cases of response to EGFR inhibitors, routine use of the EGFR inhibitor gefitinib as a single agent for advanced AML is not appropriate.

Year of Publication
2014
Journal
Leuk Res
Volume
38
Issue
4
Pages
430-4
Date Published
2014 Apr
ISSN
1873-5835
URL
DOI
10.1016/j.leukres.2013.10.026
PubMed ID
24522247
PubMed Central ID
PMC4860003
Links
Grant list
R01 CA140292 / CA / NCI NIH HHS / United States
NCIR01 CA140292 / CA / NCI NIH HHS / United States