title: Azacytidine for acute myeloid leukemia in elderly or frail patients: a phase II trial (SAKK 30/07).
abstract: This phase II trial treated elderly or frail patients with acute myeloid leukemia (AML) with single-agent subcutaneous azacytidine at 100 mg/m(2), on 5 of 28 days for up to six cycles. Treatment was stopped for lack of response, or continued to progression in responders. The primary endpoint was response within 6 months. A response rate ≥ 34% was considered a positive trial outcome. From September 2008 to April 2010, 45 patients from 10 centers (median age 74 [55-86] years) were accrued. Patients received four (1-21) cycles. Best response was complete response/complete response with incomplete recovery of neutrophils and/or platelets (CR/CRi) in eight (18%; 95% confidence interval [CI]: 8-32%.), 0 (0%) partial response (PR), seven (16%) hematologic improvement, 17 (38%) stable disease. Three non-responding patients stopped treatment after six cycles, 31 patients stopped early and 11 patients continued treatment for 8-21 cycles. Adverse events (grade ≥ III) were infections (n = 13), febrile neutropenia (n = 8), thrombocytopenia (n = 7), dyspnea (p = 6), bleeding (n = 5) and anemia (n = 4). Median overall survival was 6 months. Peripheral blood blast counts, grouped at 30%, had a borderline significant association with response (p = 0.07). This modified azacytidine schedule is feasible for elderly or frail patients with AML in an outpatient setting with moderate, mainly hematologic, toxicity and response in a proportion of patients, although the primary objective was not reached.
authors.affiliation: Universitätsspital Basel , Basel , Switzerland.
authors.forename: Jakob R
authors.initials: JR
authors.lastname: Passweg
authors.forename: Thomas
authors.initials: T
authors.lastname: Pabst
authors.forename: Sabine
authors.initials: S
authors.lastname: Blum
authors.forename: Mario
authors.initials: M
authors.lastname: Bargetzi
authors.forename: Qiyu
authors.initials: Q
authors.lastname: Li
authors.forename: Dominik
authors.initials: D
authors.lastname: Heim
authors.forename: Georg
authors.initials: G
authors.lastname: Stussi
authors.forename: Michael
authors.initials: M
authors.lastname: Gregor
authors.forename: Leda
authors.initials: L
authors.lastname: Leoncini
authors.forename: Sandrine
authors.initials: S
authors.lastname: Meyer-Monard
authors.forename: Peter
authors.initials: P
authors.lastname: Brauchli
authors.forename: Yves
authors.initials: Y
authors.lastname: Chalandon
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D000964:Antimetabolites, Antineoplastic / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D001374:Azacitidine / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D016330:Frail Elderly; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000175:diagnosis / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D016896:Treatment Outcome
chemical_list: D000964:Antimetabolites, Antineoplastic; D001374:Azacitidine