title: Phase II Study of Bortezomib as a Single Agent in Patients with Previously Untreated or Relapsed/Refractory Acute Myeloid Leukemia Ineligible for Intensive Therapy.
abstract: We explored the safety and efficacy of bortezomib given as single agent in patients with untreated or relapsed/refractory acute myeloid leukemia (AML), unfit for conventional chemotherapy. Fourteen patients were treated with bortezomib 1.5 mg/m(2) administered twice weekly for two weeks, every 3 weeks. Median age was 70 years (range 60-81) and the median number of cycles delivered was 2 (range 1-4). Of 13 evaluable patients, in 8 (61%), the administration of bortezomib resulted in an antileukemic effect as demonstrated by peripheral blood and/or bone marrow blast reduction. In 4 (50%) of these 8, a decrease by 37% of transfusion requirement was also observed (P = 0.009). Overall median survival was 4 months (range 0.25-10). Neurotoxicity was the most frequent adverse event with 7 of 13 (54%) patients experiencing grades 3-4 peripheral neuropathy. Neurotoxicity led to treatment discontinuation in 4 (57%) of 7. In conclusion, the observed anti-leukemic activity of bortezomib indicates that there is room for designing additional studies in which combination with other chemotherapeutic agents should be considered. Clinical registration no.: EUDRACT 2006-006923-38.
authors.affiliation: Department of Hematology, University of Rome Tor Vergata, Viale Oxford 81, 00133 Rome, Italy.
authors.forename: Chiara
authors.initials: C
authors.lastname: Sarlo
authors.forename: Francesco
authors.initials: F
authors.lastname: Buccisano
authors.forename: Luca
authors.initials: L
authors.lastname: Maurillo
authors.forename: Mariagiovanna
authors.initials: M
authors.lastname: Cefalo
authors.forename: Luigi
authors.initials: L
authors.lastname: Di Caprio
authors.forename: Laura
authors.initials: L
authors.lastname: Cicconi
authors.forename: Concetta
authors.initials: C
authors.lastname: Ditto
authors.forename: Licia
authors.initials: L
authors.lastname: Ottaviani
authors.forename: Ambra
authors.initials: A
authors.lastname: Di Veroli
authors.forename: Maria Ilaria
authors.initials: MI
authors.lastname: Del Principe
authors.forename: Maria Assunta
authors.initials: MA
authors.lastname: Grasso
authors.forename: Daniela
authors.initials: D
authors.lastname: Nasso
authors.forename: Giovanna
authors.initials: G
authors.lastname: De Santis
authors.forename: Sergio
authors.initials: S
authors.lastname: Amadori
authors.forename: Adriano
authors.initials: A
authors.lastname: Venditti