title: Safety and efficacy of azacitidine in Belgian patients with high-risk myelodysplastic syndromes, acute myeloid leukaemia, or chronic myelomonocytic leukaemia: results of a real-life, non-interventional post-marketing survey.
abstract: OBJECTIVE
  We evaluated azacitidine (Vidaza(®)) safety and efficacy in patients with myelodysplastic syndrome (MDS), acute myeloid leukaemia (AML), and chronic myelomonocytic leukaemia (CMML), in a real-life setting. Treatment response, dose, and schedule were assessed.
  
  
  METHODS
  This non-interventional, post-marketing survey included 49/50 patients receiving azacitidine at 14 Belgian haematology centres from 2010-2012. Treatment-emergent adverse events (TEAEs), including treatment-related TEAEs, and serious TEAEs (TESAEs) were recorded throughout the study. Treatment response [complete response (CR), partial response (PR), haematological improvement (HI), stable disease (SD), treatment failure (TF)) and transfusion-independence (TI) were evaluated at completion of a 1-year observation period (1YOP) or at treatment discontinuation, and overall survival (OS), at study conclusion.
  
  
  RESULTS
  The median age of patients was 74·7 (range: 43·9-87·8) years; 69·4% had MDS, 26·5% had primary or secondary AML, and 4·1% had CMML. Treatment-related TEAEs, grade 3-4 TEAEs, and TESAEs were reported in 67·3%, 28·6%, and 18·4% of patients, respectively. During 1YOP, patients received a median of 7 (1-12) treatment cycles. Treatment response was assessed for 38/49 patients. Among MDS and CMML patients (n = 29), 41·4% had CR, PR, or HI, 41·4% had SD, and 17·2% had TF. Among AML patients (n = 9), 44·4% had CR or PR, 33·3% had SD, and 22·2% had TF. TI was observed in 14/32 (43·8%) patients who were transfusion-dependent at baseline. Median (95% confidence interval) OS was 490 (326-555) days; 1-year OS estimate was 0·571 (0·422-0·696).
  
  
  CONCLUSIONS
  Our data support previous findings that azacitidine has a clinically acceptable safety profile and shows efficacy.
authors.forename: Y
authors.initials: Y
authors.lastname: Beguin
authors.forename: D
authors.initials: D
authors.lastname: Selleslag
authors.forename: S
authors.initials: S
authors.lastname: Meers
authors.forename: C
authors.initials: C
authors.lastname: Graux
authors.forename: G
authors.initials: G
authors.lastname: Bries
authors.forename: D
authors.initials: D
authors.lastname: Deeren
authors.forename: I
authors.initials: I
authors.lastname: Vrelust
authors.forename: C
authors.initials: C
authors.lastname: Ravoet
authors.forename: K
authors.initials: K
authors.lastname: Theunissen
authors.forename: V
authors.initials: V
authors.lastname: Voelter
authors.forename: H
authors.initials: H
authors.lastname: Potier
authors.forename: F
authors.initials: F
authors.lastname: Trullemans
authors.forename: L
authors.initials: L
authors.lastname: Noens
authors.forename: P
authors.initials: P
authors.lastname: Mineur
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*; D001530:Belgium / Q000453:epidemiology; D005260:Female; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy* / Q000401:mortality; D015477:Leukemia, Myelomonocytic, Chronic / Q000188:drug therapy* / Q000401:mortality; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000401:mortality; D011358:Product Surveillance, Postmarketing; D016896:Treatment Outcome
chemical_list: D000964:Antimetabolites, Antineoplastic; D001374:Azacitidine
keywords: Acute myeloid leukaemia,; Azacitidine,; Chronic myelomonocytic leukaemia; Myelodysplastic syndromes,