A Pilot Phase II Study of Erlotinib for the Treatment of Patients with Relapsed/Refractory Acute Myeloid Leukemia

Iman Abou Dalle, Jorge E. Cortes, Pramod Pinnamaneni, Betty Lamothe, Adolfo Diaz Duque, Jasleen Randhawa, Naveen Pemmaraju, Elias Jabbour, Alessandra Ferrajoli, William G. Wierda, Zeev Estrov, Marina Konopleva, Farhad Ravandi, Yesid Alvarado, Gautam Borthakur, Varsha Gandhi, Hagop M. Kantarjian

Research output: Contribution to journalArticlepeer-review

12 Scopus citations


Erlotinib, an epidermal growth factor receptor (EGFR) inhibitor, may have off-target activity inducing acute myeloid leukemia (AML) differentiation, possibly through SYK inhibition. We investigated erlotinib in a pilot phase II study for efficacy in relapsed/refractory AML patients at a dose of 150 mg once daily in 28-day cycles. Twenty-nine patients were treated for a median of 29 days (range 12-142 days). Seven patients (24%) received > 1 cycle of therapy and 12 (41%) discontinued treatment before day 28 due to disease progression. One patient (3%) achieved complete remission and 2 (7%) a > 50% reduction in blasts. The most common toxicities associated with erlotinib were fatigue in 10 patients (34%), diarrhea in 10 (34%), nausea in 8 (28%), and rash in 7 (24%). Only 2 patients (7%) had study drug-related adverse events requiring dose reductions and eventual discontinuation. The main reason for treatment discontinuation was disease progression in 26 patients (90%). All patients had died by the time of the last follow-up. Progression of disease was the primary cause of death in all patients. Median overall survival was 14 weeks (range 2.3-96.9 weeks) and median event-free survival was 5 weeks (range 1.7-21.0 weeks). Erlotinib as a single agent has limited clinical efficacy in patients with relapsed/refractory AML.

Original languageEnglish (US)
Pages (from-to)30-39
Number of pages10
JournalActa Haematologica
Issue number1
StatePublished - Sep 1 2018


  • Acute myeloid leukemia
  • Erlotinib
  • Relapsed/refractory AML

ASJC Scopus subject areas

  • Hematology


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