title: Azacitidine in untreated acute myeloid leukemia: a report on 149 patients.
abstract: Limited data are available on azacitidine (AZA) treatment and its prognostic factors in acute myeloid leukemia (AML). One hundred and forty-nine previously untreated AML patients considered ineligible for intensive chemotherapy received AZA in a compassionate patient-named program. AML diagnosis was de novo, post-myelodysplastic syndromes (MDS), post-MPN, and therapy-related AML in 51, 55, 13, and 30 patients, respectively. Median age was 74 years, median white blood cell count (WBC) was 3.2 × 10⁹ /L and 58% of the patients had ≥ 30% marrow blasts. Cytogenetics was adverse in 60 patients. Patients received AZA for a median of five cycles (range 1-31). Response rate (including complete remission/CR with incomplete recovery/partial remission) was 27.5% after a median of three cycles (initial response), and 33% at any time (best response). Only adverse cytogenetics predicted poorer response. Median overall survival (OS) was 9.4 months. Two-year OS was 51% in responders and 10% in non-responders (P<0.0001). Adverse cytogenetics, WBC >15 × 10⁹ /L and ECOG-PS ≥ 2 predicted poorer OS, while age and marrow blast percentage had no impact. Using MDS IWG 2006 response criteria, among patients with stable disease, those with hematological improvement had no significant survival benefit in a 7 months landmark analysis. Outcomes observed in this high-risk AML population treated with AZA deserve comparison with those of patients treated intensively in prospective studies.
authors.affiliation: Service d'Hématologie Clinique, Hôpital Avicenne, Assistance Publique-Hôpitaux de Paris (AP-HP), and Université Paris 13, Bobigny, France.
authors.forename: Sylvain
authors.initials: S
authors.lastname: Thépot
authors.forename: Raphael
authors.initials: R
authors.lastname: Itzykson
authors.forename: Valerie
authors.initials: V
authors.lastname: Seegers
authors.forename: Christian
authors.initials: C
authors.lastname: Recher
authors.forename: Emmanuel
authors.initials: E
authors.lastname: Raffoux
authors.forename: Bruno
authors.initials: B
authors.lastname: Quesnel
authors.forename: Jacques
authors.initials: J
authors.lastname: Delaunay
authors.forename: Thomas
authors.initials: T
authors.lastname: Cluzeau
authors.forename: Anne
authors.initials: A
authors.lastname: Marfaing Koka
authors.forename: Aspasia
authors.initials: A
authors.lastname: Stamatoullas
authors.forename: Marie-Pierre
authors.initials: MP
authors.lastname: Chaury
authors.forename: Caroline
authors.initials: C
authors.lastname: Dartigeas
authors.forename: Stéphane
authors.initials: S
authors.lastname: Cheze
authors.forename: Anne
authors.initials: A
authors.lastname: Banos
authors.forename: Pierre
authors.initials: P
authors.lastname: Morel
authors.forename: Isabelle
authors.initials: I
authors.lastname: Plantier
authors.forename: Anne-Laure
authors.initials: AL
authors.lastname: Taksin
authors.forename: Jean Pierre
authors.initials: JP
authors.lastname: Marolleau
authors.forename: Cecile
authors.initials: C
authors.lastname: Pautas
authors.forename: Xavier
authors.initials: X
authors.lastname: Thomas
authors.forename: Francoise
authors.initials: F
authors.lastname: Isnard
authors.forename: Blandine
authors.initials: B
authors.lastname: Beve
authors.forename: Yasmine
authors.initials: Y
authors.lastname: Chait
authors.forename: Agnes
authors.initials: A
authors.lastname: Guerci
authors.forename: Norbert
authors.initials: N
authors.lastname: Vey
authors.forename: Francois
authors.initials: F
authors.lastname: Dreyfus
authors.forename: Lionel
authors.initials: L
authors.lastname: Ades
authors.forename: Norbert
authors.initials: N
authors.lastname: Ifrah
authors.forename: Herve
authors.initials: H
authors.lastname: Dombret
authors.forename: Pierre
authors.initials: P
authors.lastname: Fenaux
authors.forename: Claude
authors.initials: C
authors.lastname: Gardin
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000964:Antimetabolites, Antineoplastic / Q000009:adverse effects / Q000627:therapeutic use*; D001374:Azacitidine / Q000009:adverse effects / Q000627:therapeutic use*; D015331:Cohort Studies; D057176:Compassionate Use Trials; D064147:Febrile Neutropenia / Q000139:chemically induced; D005260:Female; D006801:Humans; D053208:Kaplan-Meier Estimate; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D012074:Remission Induction; D018805:Sepsis / Q000209:etiology; D016896:Treatment Outcome
chemical_list: D000964:Antimetabolites, Antineoplastic; D001374:Azacitidine